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| Profile Name | Unknown unknown |
| Gene Variant Detail | |
| Relevant Treatment Approaches |
| Molecular Profile | Indication/Tumor Type | Response Type | Relevant Treatment Approaches | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|---|
| Unknown unknown | glioblastoma | not applicable | SR9009 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, SR9009 inhibited growth of glioblastoma cell lines in culture, resulted in apoptosis in tumors and prolonged survival in both cell line and patient-derived xenograft models (PMID: 29320480). | 29320480 | |
| Unknown unknown | stomach cancer | not applicable | Rivoceranib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Apatinib (YN968D1) inhibited tumor growth in gastric cancer cell line xenograft models (PMID: 21443688). | 21443688 | |
| Unknown unknown | peritoneal serous adenocarcinoma | not applicable | Paclitaxel + TVB-2640 | Case Reports/Case Series | Actionable | In a Phase I trial, TVB-2640 and Taxol (paclitaxel) combination treatment resulted in partial response in one patient with peritoneal serous carcinoma (2015 51 S724-S724 Eur J Cancer). | detail... | |
| Unknown unknown | colon adenocarcinoma | not applicable | unspecified PD-1 antibody + VE800 | Preclinical | Actionable | In a preclinical study, PD1-antibody treatment supplemented with VE800 inhibited tumor growth in mouse models of colon adenocarcinoma (PMID: 30675064). | 30675064 | |
| Unknown unknown | neuroblastoma | not applicable | Verteporfin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, verteporfin inhibited growth and metastasis of neuroblastoma cell lines in culture, and pretreatment of a metastatic subpopulation of a neuroblastoma cell line with verteporfin decreased metastasis in xenograft models (PMID: 27899382). | 27899382 | |
| Unknown unknown | chronic myelomonocytic leukemia | not applicable | Lenzilumab | Phase I | Actionable | In a Phase I trial, Lenzilumab (KB003) demonstrated safety in patients with chronic myelomonocytic leukemia and resulted in a median response duration of 5.5 months, clinical benefit rate of 33% (5/15), including three platelet responses, one neutrophil response, and one patient who demonstrated a partial bone marrow response with a myeloblast decrease from 6% to 1%, and three of the responding patients harbored NRAS mutations (PMID: 32294158; NCT02546284). | 32294158 | |
| Unknown unknown | ovarian cancer | not applicable | Birabresib + Rucaparib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Birabresib (OTX015) and Rubraca (rucaparib) resulted in a synergistic effect, demonstrating greater growth inhibition of ovarian cancer cells in culture compared to Birabresib (OTX015) treatment alone (PMID: 32927276). | 32927276 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | BMS-690514 | Phase Ib/II | Actionable | In a Phase 1b/II trial, BMS-690514 was demonstrated to be safe and efficacious in patients with NSCLC (PMID: 23490650). | 23490650 | |
| Unknown unknown | gastroesophageal junction adenocarcinoma | not applicable | Rivoceranib | Phase III | Actionable | In a Phase III trial, Apatinib (YN968D1) treatment significantly improved median overall survival (6.5 vs 4.7 months) and median progression-free survival (2.6 vs 1.8 months) compared to placebo in chemotherapy-refractory patients with advanced gastric or gastroesophageal junction adenocarcinoma (PMID: 26884585). | 26884585 | |
| Unknown unknown | lung cancer | not applicable | Rivoceranib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Apatinib (YN968D1) inhibited tumor growth in lung cancer cell line xenograft models (PMID: 21443688). | 21443688 | |
| Unknown unknown | retinoblastoma | not applicable | Prexasertib | Preclinical - Cell culture | Actionable | In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including a retinoblastoma cell line (PMID: 28270495). | 28270495 | |
| Unknown unknown | colon cancer | not applicable | AsiDNA + Olaparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA treatment led to increased sensitivity to Lynparza (olaparib), resulting in decreased survival in colon cancer cell lines in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | leukemia | not applicable | TAK-960 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, colorectal cancer cells treated with TAK-960 demonstrated cell growth inhibition, decreased tumor size, and increased median survival in both culture and cell line xenograft models (PMID: 22188812). | 22188812 | |
| Unknown unknown | prostate cancer | not applicable | MGC018 | Case Reports/Case Series | Actionable | In a Phase I/II trial, MGC018 treatment resulted in a 29.4% decrease of target lesion in a patient with metastatic castrate-resistant prostate cancer (J Clin Oncol 38: 2020 (suppl; abstr 3071); NCT03729596). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | PRN1371 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, PRN1371 inhibited proliferation of various FGFR-driven tumor cell lines in culture and inhibited tumor growth in a variety of patient-derived xenograft models with FGFR pathway alterations (AACR; 2016. Abstract nr 1249). | detail... | |
| Unknown unknown | lung squamous cell carcinoma | not applicable | Cisplatin + Gemcitabine + Tislelizumab | Case Reports/Case Series | Actionable | In a Phase II trial, treatment with Tislelizumab (BGB-A317) plus platinum doublet chemotherapy (Gemzar (gemcitabine) with Platinol (cisplatin) or Paraplatin (carboplatin)) in patients with squamous non-small cell lung cancer resulted in an objective response rate of 67% (4/6) and disease control rate of 83% (5/6), including a partial response in four patients and stable disease in one patient (PMID: 32769013; NCT03432598). | 32769013 | |
| Unknown unknown | ovarian cancer | not applicable | Roniciclib | Phase I | Actionable | In a Phase I trial, treatment with Roniciclib (BAY 1000394) at the RP2D reduced PCNA expression and resulted in a disease control rate of 40.9% (n=22) in patients with ovarian cancer (PMID: 28463960; NCT01188252). | 28463960 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Abemaciclib + Pemetrexed Disodium | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of Verzenio (abemaciclib) and Alimta (pemetrexed) was well-tolerated and demonstrated preliminary activity in patients with previously treated metastatic non-small cell lung cancer, resulting in a response rate of 4% (1/23; partial response (PR)), a disease control rate of 57% (13/23; 1 PR and 12 stable disease), and a median progression-free survival of 5.55 months (95% CI, 1.81, 10.05) (PMID: 30082474; NCT02079636). | 30082474 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Tazemetostat | Phase I | Actionable | In a Phase I trial, Tazemetostat (EPZ-6438) demonstrated safety and preliminary efficacy, resulted in an objective response in 5% (2/43) in patients with advanced solid tumors (PMID: 29650362; NCT01897571). | 29650362 | |
| Unknown unknown | estrogen-receptor positive breast cancer | not applicable | RO6839921 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, RO6839921 demonstrated anti-tumor activity in ER-positive breast cancer cell line xenograft models (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A156). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | INCB053914 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, INCB053914 inhibited proliferation of diffuse large B-cell lymphoma cell lines in culture and suppressed tumor growth in xenograft models (PMID: 29927999). | 29927999 | |
| Unknown unknown | breast cancer | not applicable | F14512 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, F14512 induced tumor regression in breast cancer cell line xenograft models (PMID: 19047165). | 19047165 | |
| Unknown unknown | lymphoma | not applicable | DSP107 | Preclinical - Cell culture | Actionable | In a preclinical study, DSP107 treatment induced phagocytosis mediated by granulocytes and macrophages in lymphoma cells in culture (Cancer Immunol Res 2019;7(2 Suppl):Abstract nr A076). | detail... | |
| Unknown unknown | Her2-receptor negative breast cancer | not applicable | Paclitaxel + Reparixin | Phase Ib/II | Actionable | In a Phase Ib trial, the combination therapy of Taxol (paclitaxel) and Reparixin in patients with metastatic ERBB2 (HER2)-receptor negative breast cancer resulted in a 30% (8/27) response rate and a durable response greater than 12 months in two patients (PMID: 28539464; NCT02001974). | 28539464 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Zanubrutinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Brukinsa (zanubrutinib) inhibited BTK signaling and proliferation of diffuse large B-cell lymphoma (DLBCL) cell lines in culture, and demonstrated antitumor activity in a DLBCL cell line xenograft model, with improved efficacy compared to Imbruvica (ibrutinib) (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 2597). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | BI 836858 | Preclinical - Patient cell culture | Actionable | In a preclinical study, BI 836858 induced potent cytotoxic immune response against patient derived acute myeloid leukemia blast cells in culture (PMID: 27013443). | 27013443 | |
| Unknown unknown | neuroblastoma | not applicable | ABT-751 + Fenretinide | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Fenretinide and ABT-751 synergistically inhibited growth of neuroblastoma cell lines in culture and in cell line xenograft models (PMID: 27530131). | 27530131 | |
| Unknown unknown | mature T-cell and NK-cell lymphoma | not applicable | DS-3201b | Phase I | Actionable | In a Phase I trial, DS-3201b demonstrated preliminary clinical activity in patients with T-cell lymphoma, with an overall response rate of 80% (4/5; 1 complete response/remission, and 3 partial responses) (Blood Dec 2017, 130 (Suppl 1) 4070; NCT02732275). | detail... | |
| Unknown unknown | stomach cancer | no benefit | Cetuximab | Phase III | Actionable | In a Phase III trial, addition of Erbitux (cetuximab) to chemotherapy consisted of capecitabine and cisplatin did not improve progression free survival over chemotherapy alone (4.4 vs 5.6 months) in patients with advanced gastric cancer (PMID: 23594786). | 23594786 | |
| Unknown unknown | melanoma | not applicable | AsiDNA | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA inhibited survival of melanoma cell lines in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Abexinostat + Pazopanib | Phase Ib/II | Actionable | In a Phase Ib/II trial, the combination of Abexinostat (PCI-24781) and Votrient (pazopanib) in advanced solid tumor patients resulted in a clinical benefit rate of 37% (16/43), a median response duration of 9.1 months, and 8 patients of 43 achieved stable disease or durable response for greater than 12 months (PMID: 28221861). | 28221861 | |
| Unknown unknown | uterine carcinosarcoma | not applicable | Navicixizumab | Case Reports/Case Series | Actionable | In a Phase I trial, Navicixizumab (OMP-305B83) treatment resulted in partial response in a patient with uterine carcinosarcoma (PMID: 30229512). | 30229512 | |
| Unknown unknown | follicular lymphoma | not applicable | Lisocabtagene maraleucel | FDA approved | Actionable | In a Phase I trial (TRANSCEND NHL 001) that supported FDA approval, Breyanzi (lisocabtagene maraleucel) treatment resulted in an objective response in 73% (186/256, 136 complete responses) of patients with relapsed or refractory large B-cell lymphoma, including follicular lymphoma grade 3B,high-grade B-cell lymphoma, diffuse large B-cell lymphoma, and primary mediastinal B-cell lymphoma (PMID: 32888407; NCT02631044). | 32888407 detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Sabatolimab + Spartalizumab | Case Reports/Case Series | Actionable | In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in partial response in a patient with non-small cell lung cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). | detail... | |
| Unknown unknown | head and neck cancer | not applicable | AsiDNA | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA inhibited survival of a head and neck cancer cell line in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | multiple myeloma | not applicable | GSK1904529A | Preclinical - Cell culture | Actionable | In a preclinical study, multiple myeloma cells were sensitive to GSK1904529A in culture, resulting in decreased cell viability (PMID: 19383820). | 19383820 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Ibrutinib + Palbociclib | Phase I | Actionable | In a Phase I trial, Imbruvica (ibrutinib) and Ibrance (palbociclib) combination treatment resulted in an overall response rate of 67%, a complete response rate of 37%, and a two year progression-free survival in 59.4% of patients with previously treated mantle cell lymphoma, with a median follow up of 25.6 months (PMID: 30692121; NCT02159755). | detail... 30692121 | |
| Unknown unknown | multiple myeloma | not applicable | Bortezomib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, addition of Velcade (bortezomib) to melphalan and prednisone combination therapy significantly improved time to progression (24.0 vs 16.6 months, HR=0.48, p<0.001) in patients with newly diagnosed multiple myeloma, with improved partial response rate (71% vs 35%) and complete response rate (30% vs 4%), and prolonged overall survival (HR=0.61, p=0.008) (PMID: 18753647; NCT00111319). | detail... 18753647 | |
| Unknown unknown | multiple myeloma | not applicable | Bortezomib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Velcade (bortezomib) treatment resulted in superior response rate (38% vs 18%, p<0.01), improved time to progression (6.22 vs 3.49 months, HR=0.55, p<0.001), and overall survival (HR=0.57, p=0.001) compared to dexamethasone in patients with relapsed multiple myeloma (PMID: 15958804; NCT00048230). | detail... 15958804 | |
| Unknown unknown | multiple myeloma | not applicable | Bortezomib | Preclinical - Cell culture | Actionable | In a preclinical study, Velcade (bortezomib) treatment induced apoptosis in multiple myeloma cells in culture (PMID: 22538852). | 22538852 | |
| Unknown unknown | ovarian carcinoma | not applicable | VS-4718 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with VS-4718 decreased phosphorylation of Ptk2 and induced cell-cycle arrest and apoptosis in ovarian carcinoma cells in culture (PMID: 24899686). | 24899686 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MEDI3617 + Paclitaxel | Phase I | Actionable | In a Phase I trial, MEDI3617 in combination with Taxol (paclitaxel) resulted in objective response in 15% (2/13) and stable disease in 31% (4/13) of patients with advanced solid tumors, with a median progression-free survival of 3.5 months (PMID: 29559563; NCT01248949). | 29559563 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | DLYE5953A | Phase I | Actionable | In a Phase I trial, DLYE5953A treatment resulted in an overall objective response rate of 12% (8/68, all partial responses), and stable disease in 54% (37/68) of patients with advanced solid tumors, including a cohort of breast cancer patients with a partial response in 10% (3/29) and a cohort of non-small cell lung cancer patients with a partial response in 20% (5/25) (PMID: 32694157; NCT02092792). | 32694157 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Cetuximab + EGFR Antisense DNA + Radiotherapy | Phase I | Actionable | In a Phase I trial, combined Erbitux (cetuximab) and radiotherapy plus intratumoral delivery of EGFR antisense DNA was well tolerated by patients with head and neck squamous cell carcinoma and showed preliminary efficacy with complete responses in 66.7% (4/6) of patients and one partial response (PMID: 30291796; NCT00903461; NCT01592721). | 30291796 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Cetuximab | FDA approved | Actionable | In a Phase III clinical trial (BONNER) that supported FDA approval, treatment with Erbitux (cetuximab) in combination with radiotherapy resulted in a median survival of 49.0 months, compared to 29.3 months with radiotherapy alone, in patients with head and neck squamous cell carinoma (PMID: 16808060; NCT00004227). | 16808060 detail... | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Cetuximab | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Erbitux (cetuximab) inhibited tumor growth in xenograft models of a head and neck squamous cell carcinoma cell line (PMID: 25559287). | 25559287 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Cetuximab | Preclinical - Cell culture | Actionable | In a preclinical study, head and neck squamous cell carcinoma cell lines, either human papilloma virus positive or negative, demonstrated decreased cell proliferation in culture when treated with Erlotinib (cetuximab) (PMID: 28138028). | 28138028 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Cetuximab | Preclinical - Pdx | Actionable | In a preclinical study, Erbitux (cetuximab) treatment did not inhibit tumor growth in patient-derived xenograft (PDX) models of head and neck squamous cell carcinoma (PMID: 32439698). | 32439698 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ON123300 | Preclinical | Actionable | In a preclinical study ON123300 inhibited growth of a variety of human solid tumor cell lines in culture (PMID: 24417566). | 24417566 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | GGTI-2418 | Phase I | Actionable | In a Phase I trial, GGTI-2418 demonstrated safety, but did not reach optimal target inhibition due to rapid elimination, and resulted in stable disease as best response in 31% (4/13) of patients with advanced solid tumors (PMID: 31372813). | 31372813 | |
| Unknown unknown | CLL/SLL | not applicable | Ibrutinib + Rituximab | FDA approved | Actionable | In a Phase III trial that supported FDA approval, combination of Imbruvica (ibrutinib) and Rituxan (rituximab) resulted in superior progression-free survival at 3 years (89.4% vs 72.9%, HR=0.35, p<0.001) and overall survival at 3 years (98.8% vs 91.5%, HR=0.17, p<0.001) compared to chemoimmunotherapy in patients with previously untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) (PMID: 31365801; NCT02048813). | detail... 31365801 | |
| Unknown unknown | renal cell carcinoma | not applicable | Pegilodecakin | Phase I | Actionable | In a Phase I trial, AM0010 demonstrated safety and resulted in partial responses in 27% (4/15) of patients with renal cell carcinoma (PMID: 27528724; NCT02009449). | 27528724 | |
| Unknown unknown | lymphoma | not applicable | APTO-253 | Preclinical - Cell culture | Actionable | In a preclinical study, APTO-253 treatment in acute myeloid leukemia cell lines resulted in decreased proliferation in culture (PMID: 29626127). | 29626127 | |
| Unknown unknown | Sezary's disease | not applicable | CPI-818 | Preclinical - Patient cell culture | Actionable | In a preclinical study, CPI-818 treatment resulted in dose-dependent growth inhibition of malignant T-cell derived from patients with Sezary's disease (Cancer Res 2019;79(13 Suppl):Abstract nr 1313). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | JNJ-63723283 | Phase Ib/II | Actionable | In a Phase I/II trial, JNJ-63723283 demonstrated safety and preliminary efficacy, resulted in partial response in 9.4% (3/32) and stable disease in 56.3% (18/32) of patients with advanced solid tumors (Journal of Clinical Oncology 36, no. 5_suppl (February 10 2018) 58-58). | detail... | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | ACP-319 | Phase I | Actionable | In a Phase I trial, ACP-319 (AMG 319) demonstrated safety and preliminary efficacy in patients with chronic lymphocytic leukemia (Blood Nov 2013, 122 (21) 678). | detail... | |
| Unknown unknown | colorectal cancer | not applicable | AB61 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the synthetic nucleoside AB61 demonstrated cytotoxicity in colorectal cancer cell lines in culture and decreased tumor volume and prolonged survival in colorectal cancer cell line xenograft models (PMID: 26819331). | 26819331 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Cerdulatinib | Preclinical | Actionable | In a preclinical study, treatment with Cerdulatinib (PRT062070) resulted in decreased viability and increased apoptosis of diffuse large B-cell lymphoma cells in culture (PMID: 25253883). | 25253883 | |
| Unknown unknown | Ewing sarcoma | not applicable | Carfilzomib + Selinexor | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Selinexor (KPT-330) and Kyprolis (carfilzomib) resulted in downregulation of Birc5 (Survivin) and enhanced induction of apoptotic markers compared to Selinexor (KPT-330) alone, and worked synergistically to decrease viability of Ewing sarcoma cells in culture (PMID: 28314790). | 28314790 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Bendamustine + Obinutuzumab + Venetoclax | Phase II | Actionable | In a Phase II trial (CLL2-BAG), sequential treatment with Treanda (bendamustine) and Gazyva (obinutuzumab) combined with Venclexta (venetoclax) resulted in an response rate of 95% (60/63) in patients with chronic lymphocytic leukemia, without unexpected or cumulative toxicities (PMID: 30115596; NCT02401503). | 30115596 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Onatasertib | Phase I | Actionable | In a Phase I clinical trial, CC-223 demonstrated safety and some efficacy in patients with solid tumors (J Clin Oncol 31, 2013 (suppl; abstr 2606). | detail... | |
| Unknown unknown | melanoma | not applicable | AS1409 | Phase I | Actionable | In a Phase I trial, AS1409 treatment in patients with either melanoma or renal cell carcinoma resulted in stable disease in 46% (6/13) of patients and in two patients with melanoma, one demonstrated a partial response while another showed tumor shrinkage, which lasted beyond 12 months (PMID: 21447719). | 21447719 | |
| Unknown unknown | non-Hodgkin lymphoma | not applicable | Cyclophosphamide + Inotuzumab ozogamicin + Prednisone + Rituximab + Vincristine Sulfate | Phase I | Actionable | In a Phase I trial, the combination of inotuzumab ozogamicin with R-CVP (rituximab, cyclophosphamide, Oncovin (vincristine), and prednisone) resulted in an ORR of 84% (32/38) in non-Hodgkin lymphoma patients, including a complete response in 24% (9/38) of patients (PMID: 27154915). | 27154915 | |
| Unknown unknown | leiomyosarcoma | not applicable | Ganetespib + Sirolimus | Case Reports/Case Series | Actionable | In a Phase I trial, one patient with leiomyosarcoma demonstrated a partial response to treatment with the combination of Ganetespib and Rapamune (sirolimus) (PMID: 32089640; NCT02008877). | 32089640 | |
| Unknown unknown | Ewing sarcoma | not applicable | SP-2509 | Preclinical - Cell culture | Actionable | In a preclinical study, EWS fusion-positive Ewing sarcoma cell lines demonstrated reduced viability following SP-2509 treatment that correlated with induction of KDM1B expression post-treatment in culture (PMID: 29997151). | 29997151 | |
| Unknown unknown | breast cancer | not applicable | XL388 | Preclinical - Cell culture | Actionable | In a preclinical study, XL388 treatment led to tumor growth inhibition in breast cancer cell line xenograft models (PMID: 23394126). | 23394126 | |
| Unknown unknown | prostate cancer | not applicable | KX2-391 | Phase II | Actionable | In a Phase II trial, KX2-391 at tested dose did not demonstrate anti-tumor effects in patients with castration-resistant prostate cancer, resulted in 8% progression free survival (PFS) at 24 weeks and median PFS of 18.6 weeks, but had modest effects on bone turnover markers (PMID: 23314737). | 23314737 | |
| Unknown unknown | melanoma | not applicable | Pembrolizumab + SD-101 | Phase I | Actionable | In a Phase Ib trial, SD-101 in combination with Keytruda (pembrolizumab) resulted in an overall response of 78% (7/9), an estimated 12-month progression-free survival rate of 88% (8/9), and an overall survival rate of 89% (8/9) in patients with unresectable or metastatic malignant melanoma naïve to anti-PD-1 therapy, and an overall response of 15% (2/13) in patients received prior anti-PD-1 therapy (PMID: 30154193; NCT02521870). | 30154193 | |
| Unknown unknown | multiple myeloma | not applicable | MKC-3946 + Tanespimycin | Preclinical - Patient cell culture | Actionable | In a preclinical study, MKC-3946 treatment in combination with Tanespimycin (17-AAG) enhanced growth inhibition of patient-derived multiple myeloma cells in culture (PMID: 22538852). | 22538852 | |
| Unknown unknown | acute myeloid leukemia | not applicable | A-366 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, A-366 inhibited proliferation and reduced cell viability in an acute myeloid leukemia (AML) cell line in culture, and inhibited tumor growth in an AML cell line xenograft model (PMID: 26147105). | 26147105 | |
| Unknown unknown | mature T-cell and NK-cell lymphoma | not applicable | Tenalisib | Phase I | Actionable | In a Phase I trial, Tenalisib (RP6530) demonstrated safety in patients with relapsed or refractory peripheral and cutaneous T-cell lymphomas, and resulted in an overall response rate of 45.7% (16/35), including three complete responses and 13 partial responses with a median duration of response of 4.9 months, and a further 31% (11/35) patients achieved stable disease (PMID: 32824175; NCT02567656). | 32824175 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Glesatinib | Phase I | Actionable | In a Phase I trial, Glesatinib (MGCD265) displayed safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 27, 2009 (suppl; abstr e14525)). | detail... | |
| Unknown unknown | medulloblastoma | not applicable | M344 | Preclinical - Cell culture | Actionable | In a preclinical study, M344 induced apoptosis and inhibited proliferation of medulloblastoma cell lines in culture (PMID: 17230517). | 17230517 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | Dactolisib + Everolimus | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of BEZ235 and Afinitor (everolimus) resulted in limited efficacy in patients with advanced solid tumors and was discontinued due to multiple intolerable side effects (PMID: 28357727). | 28357727 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | AZD7648 + Radiotherapy | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AZD7648 treatment increased sensitivity to radiotherapy, inhibiting proliferation and inducing cell cycle arrest in non-small cell lung carcinoma cells in culture, and inhibiting tumor growth and inducing tumor regression in cell line xenograft models (PMID: 31699977). | 31699977 | |
| Unknown unknown | Her2-receptor negative breast cancer | not applicable | Sorafenib | Clinical Study | Actionable | In a meta-analysis of 844 ERBB2 (HER2)-negative breast cancer patients, Nexavar (sorafenib) increased progression-free survival time, but not overall survival or objective response rate (PMID: 24940450). | 24940450 | |
| Unknown unknown | CLL/SLL | not applicable | Cerdulatinib | Phase I | Actionable | In a Phase I trial, treatment with Cerdulatinib (PRT062070) inhibited SYK and JAK/STAT signaling, and resulted in tumor response in 50% (3/6) of patients with CLL/SLL (PMID: 30333224; NCT01994382). | 30333224 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Azacitidine + Nivolumab | Phase II | Actionable | In a Phase II trial, the combination treatment of Vidaza (azacitidine) and Opdivo (nivolumab) resulted in an overall response rate of 33% (23/70) in patients with relapsed/refractory acute myeloid leukemia, including four with complete remission, 11 with complete remission with incomplete recovery counts, one partial response, and seven with hematological improvement, and led to a median overall survival of 6.3 months (PMID: 30409776; NCT02397720). | 30409776 | |
| Unknown unknown | prostate cancer | not applicable | JNJ-54302833 | Preclinical | Actionable | In a preclinical study, JNJ-54302833 inhibited growth of prostate cancer cells in culture ( Cancer Res October 1, 2014 74; 4747 ). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Ficlatuzumab + Gefitinib | Phase I | Actionable | In a Phase I trial, Ficlatuzumab (Av-299) in combination with Iressa (gefitinib) demonstrated safety and efficacy in patients with NSCLC (PMID: 23493885, J Clin Oncol. 2011;29(Suppl 15) Abstract 7571). | detail... 23493885 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Atezolizumab + Carboplatin + Etoposide | FDA approved | Actionable | In a Phase III trial (IMpower133) that supported FDA approval, Tecentriq (atezolizumab) in combination with Paraplatin (carboplatin) and Vepesid (etoposide) resulted in significantly improved median overall survival (12.3 vs 10.3 months, HR=0.70, p=0.007) and median progression-free survival (5.2 vs 4.3 months, HR=0.77, p=0.02) compared to placebo in patients with untreated extensive-stage small-cell lung cancer (PMID: 30280641; NCT02763579). | detail... 30280641 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ABT-348 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, ABT-348 (Ilorasertib) inhibited proliferation and promoted apoptosis in various solid tumor cell culture and Pdx models, including NSCLC, ovarian, and colon (AACR; Mol Cancer Ther 2011;10(11 Suppl):Abstract nr B231). | detail... | |
| Unknown unknown | squamous cell carcinoma | not applicable | PMX-53 | Preclinical | Actionable | In a preclinical study, PMX-53 treatment did not inhibit tumor growth in a syngeneic mouse model of poorly differentiated squamous cell carcinoma (PMID: 30300579). | 30300579 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Camrelizumab | Phase I | Actionable | In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors, with an objective response rate of 31% (18/58, all partial responses) and disease control rate of 46.5% (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). | detail... | |
| Unknown unknown | renal cell carcinoma | not applicable | 23814 | Preclinical - Pdx | Actionable | In a preclinical study, treatment with 23814 resulted in tumor growth inhibition in a renal cell carcinoma patient-derived xenograft (PDX) model (PMID: 25995436). | 25995436 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Carboplatin + Topotecan + Veliparib | Phase I | Actionable | In a Phase I trial, the combination of Hycamtin (topotecan), Paraplatin (carboplatin), and Veliparib (ABT-888) resulted in a response rate of 25% (19/77) in patients with acute myeloid leukemia (PMID: 27551000). | 27551000 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | LY3295668 | Phase I | Emerging | In a Phase I trial, AK-01 demonstrated desirable pharmacokinetics in patients with locally advanced or metastatic solid tumors (AACR Annual Meeting 2019, Abstract CT019; NCT03092934). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | AMG 397 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AMG 397 treatment induced apoptosis and reduced viability of multiple myeloma cells in culture, and induced tumor regression in a cell line xenograft model (Cancer Res 2020;80(16 Suppl):Abstract nr 6218). | detail... | |
| Unknown unknown | lung cancer | not applicable | SKI-G-801 | Preclinical | Actionable | In a preclinical study, SKI-G-801 treatment reduced tumor growth in a syngeneic mouse model of lung cancer (Cancer Res 2019;79(13 Suppl):Abstract nr 2010). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | LY900009 | Phase I | Actionable | In a Phase I trial, LY900009 demonstrated manageable safety and some preliminary activity in patients with advanced solid tumors, with treatment resulting in no responses, but stable disease in 14% (5/35) of patients (PMID: 26798966). | 26798966 | |
| Unknown unknown | indolent systemic mastocytosis | not applicable | Avapritinib | Phase I | Actionable | In a Phase I trial, BLU-285 treatment reduced bone marrow mast cell infiltration in a patient with systemic mastocytosis (PMID: 29093181; NCT02561988). | 29093181 | |
| Unknown unknown | acute myeloid leukemia | not applicable | RG7112 | Phase I | Actionable | In a Phase I clinical trial, RG7112 demonstrated clinical activity in acute myeloid leukemia, with complete response in 7% (2/30), complete response with incomplete platelet recovery in 3% (1/30), partial response in 7% (2/30), and stable disease in 30% (9/30) of patients (PMID: 26459177). | 26459177 | |
| Unknown unknown | rhabdoid cancer | not applicable | Ribociclib | Phase I | Actionable | In a Phase I trial, Kisqali (ribociclib) treatment demonstrated safety and resulted in stable disease in 28% (9/32) of pediatric patients with neuroblastoma or malignant rhabdoid tumor (MRT) (7 patients with neuroblastoma and 2 with CNS primary MRT), and 5 patients demonstrated stable disease for greater than 6 months (PMID: 28432176). | 28432176 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | XL147 | Phase I | Actionable | In a Phase I study, 25/56 (43.9%) of patients with advanced solid tumors had stable disease as a best response to treatment with XL147, and one patient with NSCLC showed a partial response to XL147 (PMID: 24166903). | 24166903 | |
| Unknown unknown | ovarian cancer | no benefit | Vandetanib | Phase II | Actionable | In a Phase II trial, treatment with Caprelsa (vandetanib) in ovarian cancer patients resulted in no benefit and led to early termination of the trial (PMID: 20068097). | 20068097 | |
| Unknown unknown | melanoma | not applicable | Ad5CMV-p53 gene + unspecified PD-1 antibody | Preclinical | Actionable | In a preclinical study, the combination of Advexin (Ad5CMV-p53) and an unspecified anti-PD-1 antibody resulted in a synergistic effect and abscopal effect in an immune therapy resistant syngeneic melanoma mouse model, demonstrating decreased tumor growth and improved survival compared to either agent alone (J Clin Oncol 35, 2017 (suppl; abstr e14610)). | detail... | |
| Unknown unknown | alveolar rhabdomyosarcoma | not applicable | HSV-1 G207 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, HSV-1 G207 treatment resulted in inhibition of alveolar rhabdomyosarcoma cell proliferation in culture, and blocked tumor growth and angiogenesis in patient-derived xenograft (PDX) models of alveolar rhabdomyosarcoma (PMID: 15720798). | 15720798 | |
| Unknown unknown | fibrosarcoma | not applicable | Anlotinib | Phase II | Actionable | In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 81%, median progression-free survival of 5.6 months, an objective response rate of 11% (n=18), and a median overall survival of 12 months in patients with fibrosarcoma (PMID: 29895706; NCT01878448). | detail... 29895706 | |
| Unknown unknown | breast cancer | not applicable | Danusertib | Preclinical - Cell culture | Actionable | In a preclinical study, Danusertib (PHA-739358) disrupted cell cycle progression and inhibited growth of breast cancer cell lines, with preferential inhibition of aromatase inhibitor-resistant cell lines (PMID: 25667100). | 25667100 | |
| Unknown unknown | pancreatic cancer | not applicable | BRD4770 | Preclinical - Cell culture | Actionable | In a preclinical study, BRD4770 resulted in decreased cell viability, reduced cell proliferation, and decreased colony formation in a pancreatic cell line in culture (PMID: 22536950). | 22536950 | |
| Unknown unknown | gastrointestinal neuroendocrine tumor | not applicable | Pazopanib | Clinical Study | Actionable | In a clinical study, Votrient (pazopanib) treatment in patients with gastroenteropancreatic neuroendocrine tumors resulted in an overall response rate of 24% (19/124), stable disease in 39.5% (49/124), a progression free survival of 36% at six months, and a median overall survival of 10.2 months (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 446P). | detail... | |
| Unknown unknown | hematologic cancer | not applicable | AsiDNA | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA inhibited survival of hematologic cancer cell lines in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | colorectal cancer | not applicable | Afatinib | Preclinical | Actionable | In a preclinical study, Gilotrif (afatinib) inhibited proliferation and increased apoptosis in several human colorectal tumor cell lines in culture (PMID: 21617858). | 21617858 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Carboplatin + TAK-243 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Paraplatin (carboplatin) and TAK-243 (MLN7243) resulted in synergistic and additive effects, demonstrating anti-tumor activity in xenograft tumor models (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A164). | detail... | |
| Unknown unknown | breast cancer | not applicable | Gemcitabine + Trametinib | Phase Ib/II | Actionable | In a Phase Ib clinical trial, the combination of Mekinist (trametinib) and Gemzar (gemcitabine) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors, including one complete response in a patient with breast cancer (PMID: 23583440). | 23583440 | |
| Unknown unknown | endometrial cancer | not applicable | Capivasertib + Olaparib | Phase I | Actionable | In a Phase I trial, the combination of AZD5363 and Lynparza (olaparib) was well-tolerated and demonstrated preliminary activity in patients with endometrial, ovarian, or triple-negative breast cancer (TNBC), with an overall response rate of 24% (7/30; all partial responses, 1 ovarian, 4 endometrial, and 2 TNBC) and stable disease for greater than 4 months in 6 additional patients, and a response rate of 50% (4/8) in endometrial cancer patients (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #391P; NCT02208375). | detail... | |
| Unknown unknown | oral squamous cell carcinoma | not applicable | Palbociclib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Ibrance (palbociclib) treatment reduced phosphorylation of Rb, induced cell cycle arrest, and inhibited proliferation of oral squamous cell carcinoma cell lines in culture, and inhibited tumor growth in cell line xenograft models (PMID: 31516747). | 31516747 | |
| Unknown unknown | melanoma | not applicable | Abemaciclib | Phase I | Actionable | In a Phase I trial, treatment with Abemaciclib (LY2835219) in melanoma patients resulted in a disease control rate of 27% (7/26), a partial response in one patient and six patients with stable disease (PMID: 27217383). | 27217383 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Pembrolizumab | FDA approved | Actionable | In a Phase Ib trial (KEYNOTE-012) that supported FDA approval, treatment with Keytruda (pembrolizumab) resulted in an objective response rate of 18% (24/132; 4 complete response, 20 partial response) in patients with recurrent and/or metastatic head and neck squamous cell carcinoma (PMID: 27247226; NCT01848834). | detail... detail... 27247226 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Adavosertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Adavosertib (MK-1775) inhibited cell proliferation and promoted DNA damage in human non-small cell lung carcinoma cell lines in culture, and promoted tumor regression in xenograft models (PMID: 23699655). | 23699655 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Radiotherapy + TAK-243 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of TAK-243 (MLN7243) and radiotherapy resulted in elevated levels of DNA damage and growth inhibition in cancer cell lines in vitro (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A164). | detail... | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Carboplatin + Cetuximab + Fluorouracil | FDA approved | Actionable | In a Phase III clinical trial that supported FDA approval, Erbitux (cetuximab) in combination with platinum-based chemotherapy (Platinol (cisplatin) or Paraplatin (carboplatin)) and Adrucil (fluorouracil) resulted in a median overall survival of 10.1 months, versus 7.4 months with chemotherapy alone, in patients with recurrent or metastatic head and neck squamous cell carcinoma (PMID: 23576486). | 23576486 detail... | |
| Unknown unknown | lymphoma | not applicable | Ixazomib | Preclinical | Actionable | In a preclinical study, Ixazomib (MLN9708) inhibited survival and induced apoptosis in T-cell lymphoma cell lines in culture, and reduced tumor volume in xenograft models (PMID: 26988986). | 26988986 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MK-1248 + Pembrolizumab | Phase I | Actionable | In a Phase I trial, MK-1248 in combination with Keytruda (pembrolizumab) demonstrated safety and resulted in an objective response response rate of 18% (3/17, one complete and two partial responses) and a disease control rate of 47% (8/17) in patients with advanced solid tumors (PMID: 32809217; NCT02553499). | 32809217 | |
| Unknown unknown | neuroendocrine tumor | not applicable | Everolimus | FDA approved | Actionable | In a Phase III trial (RADIANT-4) supporting FDA approval, Afinitor (everolimus) treatment significantly improved median progression-free survival (11.0 months) comparing to placebo (3.9 months) in patients with progressive neuroendocrine tumours of the lung or gastrointestinal tract origin (PMID: 26703889; NCT01524783). | detail... 26703889 | |
| Unknown unknown | multiple myeloma | not applicable | Dexamethasone + Lenalidomide + Ricolinostat | Phase I | Actionable | In a Phase Ib trial, Ricolinostat (ACY-1215) in combination with Revlimid (lenalidomide) and Desamethasone demonstrated safety and preliminary efficacy in relapsed or refractory multiple myeloma patients, resulted in an overall response rate of 55% (21/38) (PMID: 27646843). | 27646843 | |
| Unknown unknown | colon adenocarcinoma | not applicable | AGI-134 | Preclinical - Cell culture | Actionable | In a preclinical study, AGI-134 treatment induced cell death of colon adenocarcinoma cells in the presence of normal human serum in culture (PMID: 31889898). | 31889898 | |
| Unknown unknown | mantle cell lymphoma | not applicable | GS-5829 + Venetoclax | Preclinical - Cell culture | Actionable | In a preclinical study, mantle cell lymphoma cell lines demonstrated greater cell growth inhibition when treated with a combination of GS-5829 and Venclexta (venetoclax) compared to either agent alone in culture (Blood 2016 128:5104). | detail... | |
| Unknown unknown | renal cell carcinoma | not applicable | Ipilimumab + Nivolumab | Phase I | Actionable | In a Phase I trial, the combination of Opdivo (nivolumab) and Yervoy (ipilimumab) demonstrated safety using 2 different dosing regimens (N3I1=nivolumab 3mg/kg+ipilumumab 1mg/kg; N1I3=nivolumab 1mg/kg+ipilumumab 3mg/kg) in patients with metastatic renal cell carcinoma, and resulted in an objective response rate of 40.4% (19/47) in both N3I1 and N1I3 arms and a 2-year overall survival of 67% in the N3I1 arm and 70% in the N1I3 arm (PMID: 28678668; NCT01472081). | 28678668 | |
| Unknown unknown | renal cell carcinoma | not applicable | Ipilimumab + Nivolumab | FDA approved | Actionable | In a Phase III trial (CheckMate 214) that supported FDA approval, the combination of Opdivo (nivolumab) and Yervoy (ipilimumab) resulted in improved median overall survival (not reached vs. 26.0 months), objective response rate (42%, 40 complete responses (CR), vs. 27%, 5 CR), and progression-free survival (11.6 vs 8.4 months) compared to Sutent (sunitinib) in patients with intermediate or poor risk renal cell carcinoma (PMID: 29562145; NCT02231749). | 29562145 detail... detail... | |
| Unknown unknown | B-cell lymphoma | not applicable | Denileukin diftitox + Temsirolimus | Preclinical - Cell culture | Actionable | In a preclinical study, Torisel (temsirolimus) enhanced the efficacy of Ontak (denileukin diftitox) in human B-cell lymphoma cells, resulting in decreased cell viability in culture (PMID: 27737881). | 27737881 | |
| Unknown unknown | liposarcoma | not applicable | Alisertib | Phase II | Actionable | In a Phase II trial, Alisertib (MLN8237) treatment resulted in twelve-week progression free survival in 73% (9/12) of liposarcoma patients (PMID: 27502708). | 27502708 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Bevacizumab + Sorafenib | Phase I | Actionable | In a Phase I trial, the treatment combination of Avastin (bevacizumab) and Nexavar (sorafenib) in patients with advanced solid tumors resulted in stable disease in 25% (29/115) of patients and a partial response in 5% (6/115) of patients (PMID: 25363205). | 25363205 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Refametinib + Sorafenib | Phase I | Actionable | In a Phase I trial, 43.8% (7/16) of hepatocellular carcinoma patients treated with a combination of Refametinib (BAY86-9766) and Nexavar (sorafenib) demonstrated stable disease (PMID: 26644411). | 26644411 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Ibrutinib | FDA approved | Actionable | In a Phase II trial (Study 1104) that supported FDA approval, Imbruvica (ibrutinib) treatment resulted in a response rate of 68% (75/111, complete response 21%, partial response 47%), with an estimated median progression-free survival of 13.9 months in patients with relapsed or refractory mantle-cell lymphoma (PMID: 23782157, NCT01236391). | 23782157 detail... | |
| Unknown unknown | lung small cell carcinoma | not applicable | Carboplatin + Etoposide + Tislelizumab | Phase II | Actionable | In a Phase II trial, treatment with Tislelizumab (BGB-A317) plus platinum doublet chemotherapy (Vepesid (etoposide) with Platinol (cisplatin) or Paraplatin (carboplatin)) in patients with small cell lung cancer resulted in an objective response rate of 77% (13/17) and disease control rate of 88% (15/17), including a partial response in 13 patients and stable disease in two patients, and median progression-free survival of 6.9 months (PMID: 32769013; NCT03432598). | 32769013 | |
| Unknown unknown | melanoma | not applicable | Talimogene laherparepvec | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Imlygic (talimogene laherparepvec) treatment resulted in significantly improved durable response rate (16.3% vs 2.1%, OR=8.9, p<0.001), overall response rate (26.4% vs 5.7%), and median overall survival (23.3 vs 18.9 months, HR=0.79, p=0.051) compared to GM-CSF in patients with melanoma (PMID: 26014293; NCT00769704). | 26014293 detail... | |
| Unknown unknown | pancreatic cancer | not applicable | E7449 | Phase I | Actionable | In a Phase I trial, E7449 treatment inhibited Parp activity in peripheral blood mononuclear cells and resulted in stable disease for more than 24 weeks in 7 patients with pancreatic cancer (J Clin Oncol 36, 2018 (suppl; abstr 2505); abstr e19531; NCT01618136). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Cobimetinib | Phase I | Actionable | In a Phase I trial, Cotellic (cobimetinib) treatment resulted in stable disease for five months or more in five patients with advanced solid tumors (PMID: 27424159). | 27424159 detail... | |
| Unknown unknown | lung carcinoma | not applicable | MGCD516 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human lung carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BAY 1238097 | Clinical Study | Actionable | In a Phase I trial, BAY 1238097 therapy resulted in zero objective responses and stable disease in 25% (2/8) of patients with refractory advanced solid tumors; due to high toxicity the trial was terminated prematurely (PMID: 30711772; NCT02369029). | 30711772 | |
| Unknown unknown | rhabdoid cancer | not applicable | OBP-801 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, OBP-801 treatment inhibited proliferation and induced apoptosis in rhabdoid tumor cell lines in culture, and inhibited tumor growth in cell line xenograft models (PMID: 32847975). | 32847975 | |
| Unknown unknown | renal cell carcinoma | not applicable | Dovitinib | Phase III | Actionable | In a Phase III clinical trial, Dovitinib (TKI258) demonstrated efficacy equivalent to Nexavar (sorafenib) in metastatic renal cell carcinoma patients (PMID: 24556040). | 24556040 | |
| Unknown unknown | renal cell carcinoma | not applicable | Dovitinib | Phase I | Actionable | In a Phase I trial, Dovitinib (TKI258) demonstrated safety and efficacy resulting in 10% (2/20) partial response and 60% (12/20) stable disease in renal cell carcinoma patients (PMID: 23339124). | 23339124 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Lucitanib | Preclinical | Actionable | In a preclinical study, Lucitanib (E-3810) demonstrated antitumor activity in mouse xenograft models of triple negative breast cancer with synergistic effects noted when using Lucatinib plus chemotherapy (PMID: 23270924). | 23270924 | |
| Unknown unknown | prostate cancer | not applicable | Sapanisertib | Phase II | Actionable | In a Phase II trial, Sapanisertib (MLN0128) treatment demonstrated limited efficacy, with a median time on treatment of 11 weeks and stable disease as best response (PMID: 29508246). | 29508246 | |
| Unknown unknown | prostate cancer | not applicable | Sapanisertib | Preclinical | Actionable | In a preclinical study, Sapanisertib (MLN0128) inhibited cell proliferation, induced apoptosis, and prevented metastasis in xenograft models with prostate cancer (PMID: 22367541). | 22367541 | |
| Unknown unknown | angiosarcoma | not applicable | Carotuximab + Pazopanib | Phase Ib/II | Actionable | In a Phase I/II trial, TRC105 and Votrient (pazopanib) combination therapy resulted complete response in 40% (2/5) of angiosarcoma patients (J Clin Oncol 34, 2016 (suppl; abstr 11016)). | detail... | |
| Unknown unknown | pancreatic cancer | not applicable | Berzosertib + Gemcitabine + Radiotherapy | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Berzosertib (VX-970) enhanced the efficacy of radiotherapy combined with Gemzar (gemcitabine) in pancreatic cell line xenograft models, demonstrating a longer delay in tumor growth when compared to the models treated with only Gemzar (gemcitabine) and radiotherapy (PMID: 23222511). | 23222511 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Golvatinib | Phase I | Actionable | In a Phase I trial, Golvatinib (E7050) demonstrated safety and preliminary efficacy, with stable disease as best overall response in patients with advanced solid tumors (PMID: 25278451). | 25278451 | |
| Unknown unknown | Waldenstroem's macroglobulinemia | not applicable | Umbralisib | Case Reports/Case Series | Actionable | In a Phase I trial, Ukoniq (umbralisib) treatment resulted in stable disease in 2 patients with Waldenstroem's macroglobulinemia (PMID: 29475723; NCT01767766). | 29475723 | |
| Unknown unknown | ovarian cancer | not applicable | Selumetinib + SHP099 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination therapy of Selumetinib (AZD6244) and SHP099 resulted in decreased colony formation and reduced cell viability in ovarian cancer cells in culture and inhibition of tumor growth and reduced tumor angiogenesis in a patient-derived xenograft (PDX) model of ovarian cancer (PMID: 30045908). | 30045908 | |
| Unknown unknown | acute lymphoblastic leukemia | not applicable | ABT-348 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Ilorasertib (ABT-348) displayed efficacy in acute lymphoblastic leukemia cell line xenograft models (PMID: 22935731). | 22935731 | |
| Unknown unknown | melanoma | not applicable | Epacadostat | Phase I | Actionable | In a Phase I trial, treatment with Epacadostat resulted in stable disease in 13% (7/52) of patients with advanced solid tumors for greater than or equal to 16 weeks, including two patients with melanoma who previously failed on immunotherapy (PMID: 28053021). | 28053021 | |
| Unknown unknown | pancreatic cancer | not applicable | ABC294640 + Gemcitabine | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of ABC294640 and Gemzar (gemcitabine) worked synergistically to decrease viability of pancreatic cancer cell lines in culture (PMID: 27517489). | 27517489 | |
| Unknown unknown | lung non-squamous non-small cell carcinoma | not applicable | Camrelizumab + Rivoceranib | Phase Ib/II | Actionable | In a Phase Ib/II trial, Camrelizumab (SHR-1210) plus Rivoceranib (apatinib) demonstrated safety and resulted in a 30.9% (29/94; one complete, 28 partial responses) objective response rate, 81.9% (77/94) disease control rate, 52.1% (49/94; disease control lasting 24 weeks or more) clinical benefit response rate, median progression-free survival of 5.7 months, and median overall survival of 15.5 months in evaluable patients with non-squamous NSCLC who received prior chemotherapy (PMID: 33323401; NCT03083041). | 33323401 | |
| Unknown unknown | colorectal cancer | not applicable | Bevacizumab + Fluorouracil + Irinotecan + Oxaliplatin | Clinical Study | Actionable | In a systematic review, a pooled analysis of 11 studies assessing the combination of Avastin (bevacizumab), Adrucil (fluorouracil), Eloxatin (oxaliplatin), and Camptosar (irinotecan) in colorectal cancer patients demonstrated an objective response rate of 69%, a median overall survival of 30.2 months based on six trials, and a progression free survival of 12.4 months based on nine trials (PMID: 28542671). | 28542671 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Ibrutinib + ONC201 | Preclinical - Patient cell culture | Actionable | In a preclinical study, the combination of ONC201 and Imbruvica (ibrutinib) induced apoptosis in primary mantle cell lymphoma samples in culture, including Imbruvica (ibrutinib)-resistant samples, with greater efficacy than either agent alone (PMID: 26884599). | 26884599 | |
| Unknown unknown | renal cell carcinoma | not applicable | Everolimus + Lenvatinib | FDA approved | Actionable | In a Phase II clinical trial that supported FDA approval, treatment with the combination of Lenvima (lenvatinib) and Afinitor (everolimus) resulted in a prolonged median progression-free survival of 14.6 months, compared to 5.5 months for Afinitor (everolimus) alone in patients with metastatic renal cell carcinoma who had progressed after one previous VEGF-targeted therapy (PMID: 26482279; NCT01136733). | 26482279 detail... | |
| Unknown unknown | renal cell carcinoma | not applicable | Everolimus + Lenvatinib | Phase Ib/II | Actionable | In a Phase Ib clinical trial, the combination of Lenvima (lenvatinib) and Afinitor (everolimus) demonstrated safety, and resulted in partial response in 30% (6/20) of patients with metastatic renal cell carcinoma (PMID: 24190702). | 24190702 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | TVB-2640 | Phase I | Actionable | In a Phase I trial, TVB-2640 demonstrated bioavailability and inhibited FASN-dependent signaling in the tumor tissue of one patient with advanced solid tumor (Cancer Res August 1, 2015 75; 2675). | detail... | |
| Unknown unknown | gastroesophageal junction adenocarcinoma | not applicable | Nintedanib | Phase II | Actionable | In a Phase II trial, Ofev (nintedanib) was well tolerated and the study met its primary endpoint, resulted in progression-free survival at 6-months in 19% (6/32) of patients with esophageal/GEJ (n=17) or gastric (n=15) adenocarcinoma, with a median follow-up of 14.5 months and a median overall survival of 14.2 months (PMID: 30952642; NCT02234596). | 30952642 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | ONC201 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, ONC201 inhibited viability of several triple-negative breast cancer (TNBC) cell lines in culture, demonstrating variable pro-apototic and anti-proliferative activity, and inhibited tumor growth in TNBC cell line xenograft models (PMID: 28424227). | 28424227 | |
| Unknown unknown | ovary epithelial cancer | not applicable | Bevacizumab + Niraparib | Phase I | Actionable | In a Phase I trial, Avastin (bevacizumab) and Zejula (niraparib) combination treatment resulted in complete response in 8% (1/12), partial response in 33% (4/12), and a disease control rate of 91% in platinum-sensitive ovarian cancer patients (J Clin Oncol 34, 2016 (suppl; abstr 5555)). | detail... | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | S63845 | Preclinical - Cell culture | Actionable | In a preclinical study, S63845 decreased viability of triple negative breast cancer (TNBC) cell lines and TNBC patient-derived xenograft (PDX) tumor cells in culture (PMID: 28768804). | 28768804 | |
| Unknown unknown | lung cancer | not applicable | TAK-960 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, lung cancer cells treated with TAK-960 demonstrated a decrease in tumor size in cell line xenograft models (PMID: 22188812). | 22188812 | |
| Unknown unknown | ovarian cancer | not applicable | Abemaciclib | Preclinical | Actionable | In a preclinical study, LY2835219 treatment reduced tumor growth and increased survival in ovarian cancer xenograft models (American Association for Cancer Research; 2013 Apr 6-10; Abstract LB-122). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | Abemaciclib | Preclinical | Actionable | In a preclinical study, LY2835219 alone, and in combination with Gemzar (gemcitabine), reduced tumor growth in xenograft models of solid tumors including ovarian cancers (PMID: 24919854). | 24919854 | |
| Unknown unknown | ovarian cancer | not applicable | Abemaciclib | Phase I | Actionable | In a Phase I trial, two ovarian cancer patients treated with Abemaciclib (LY2835219) demonstrated stable disease while another ovarian cancer patient achieved a partial response (PMID: 27217383). | 27217383 | |
| Unknown unknown | glioblastoma | not applicable | BI2536 + PD-0325901 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of BI 2536 and PD-0325901 resulted in greater inhibition of cell proliferation in glioblastoma cells in culture compared to treatment with either agent alone (PMID: 26573800). | 26573800 | |
| Unknown unknown | ovary epithelial cancer | not applicable | Bevacizumab + Carboplatin + Paclitaxel | FDA approved | Actionable | In a Phase III trial (GOG-0218) that supported FDA approval, addition of Avastin (bevacizumab) during and after Paraplatin (carboplatin) plus Taxol (paclitaxel) treatment prolonged progression-free survival (14.1 vs 10.3 months) compared to placebo in patients with previously untreated, stage III/IV epithelial ovarian, primary peritoneal, or fallopian tube cancer (PMID: 22204724; NCT00262847). | detail... 22204724 | |
| Unknown unknown | ovary epithelial cancer | not applicable | Bevacizumab + Carboplatin + Paclitaxel | Phase II | Actionable | In a Phase II trial, addition of Avastin (bevacizumab) to neoadjuvant chemotherapy (Paraplatin (carboplatin) plus Taxol (paclitaxel)) did not improve complete macroscopic response rate (2/35 vs 2/33), or median progression free survival (20.4 vs 20.1 months) compared to control in patients with advanced epithelial ovarian cancer, but resulted in favorable rate of surgical feasibility (66.7 vs 88.6%) (J Clin Oncol 35, 2017 (suppl; abstr 5508)). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Tafasitamab-cxix | Phase II | Actionable | In a Phase II trial, diffuse large B-cell lymphoma patients tolerated Monjuvi (tafasitamab-cxix) treatment, and achieved an overall response rate of 26% (9/35, 2 complete and 7 partial response) with 5 patients responding over 12 months, a disease control rate of 40% (14/35), a median duration of response of 20.1 months and a progression-free survival of 2.7 months at a median follow-up of 21 months (PMID: 29444231; NCT01685008). | 29444231 | |
| Unknown unknown | neuroblastoma | not applicable | Ribociclib | Phase I | Actionable | In a Phase I trial, Kisqali (ribociclib) treatment demonstrated safety and resulted in stable disease in 28% (9/32) of pediatric patients with neuroblastoma or malignant rhabdoid tumor (MRT) (7 patients with neuroblastoma and 2 with CNS primary MRT), and 5 patients demonstrated stable disease for greater than 6 months (PMID: 28432176). | 28432176 | |
| Unknown unknown | hematologic cancer | not applicable | AZD4573 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AZD4573 treatment induced caspase activation and inhibited viability of hematological cancer cell lines in culture, led to tumor regression in cell line xenograft models (PMID: 31699827). | 31699827 | |
| Unknown unknown | hematologic cancer | not applicable | AZD4573 | Preclinical - Cell culture | Actionable | In a preclinical study, AZD4573 induced apoptosis and cell death in a panel of hematologic cancer cell lines in culture (Cancer Res 2018;78(13 Suppl):Abstract nr 310). | detail... | |
| Unknown unknown | renal cell carcinoma | not applicable | Pazopanib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Votrient (pazopanib) improved progression free survival in patients with advanced renal cell carcinoma (PMID: 20100962). | 20100962 detail... | |
| Unknown unknown | renal cell carcinoma | not applicable | Pazopanib | Phase III | Actionable | In a Phase III trial, adjuvant Votrient (pazopanib) therapy post nephrectomy did not improve disease-free survival (HR=0.862, p=0.165) compared to placebo in patients with renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 4507)). | detail... | |
| Unknown unknown | glioblastoma | not applicable | Pexidartinib | Phase II | Actionable | In a Phase II trial, Pexidartinib (PLX3397) in combination with radiation therapy and Temodar (temozolomide) demonstrated safety and improved efficacy over standard therapy, resulting in a median progression-free survival of 9.7 months and an estimated overall survival of 25.1 months in newly diagnosed glioblastoma multiforme patients (Neuro Oncol (2016) 18 (suppl 6): vi6). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Panobinostat + Radiotherapy | Phase I | Actionable | In a Phase I trial, Farydak (panobinostat) in combination with palliative radiotherapy resulted in a disease control rate of 66% (6/9), with a progression-free survival of 3 months and a median overall survival of 9 months in patients with stage III non-small cell lung cancer (PMID: 26317683). | 26317683 | |
| Unknown unknown | ovarian cancer | not applicable | Brivanib | Phase II | Actionable | In a Phase II trial, treatment with Brivanib (BMS-540215) demonstrated safety and resulted in a median progression-free survival of 4.0 months in ovarian cancer patients, compared to 2.0 months with placebo, and FGF2 expression was not found to be a predictive biomarker for response (PMID: 31522033; NCT00633789). | 31522033 | |
| Unknown unknown | endometrial cancer | not applicable | GSK2126458 | Phase I | Actionable | In a Phase I trial, GSK2126458 treatment was well-tolerated and resulted in some efficacy in endometrial cancer patients including stable disease in 27% (4/15) and one patient with a partial response (PMID: 26603258). | 26603258 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Bevacizumab + Cisplatin + Etoposide | Phase III | Actionable | In a Phase III trial, the addition of Avastin (bevacizumab) to treatment with Platinol (cisplatin) and Vepesid (etoposide) resulted in a median progression-free survival of 6.7 months, compared to 5.7 months with Platinol (cisplatin) and Vepesid (etoposide) in combination, but did not result in significantly improved overall survival in patients with small-cell lung cancer (PMID: 28135143). | 28135143 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | A-1155463 + BETd-246 | Preclinical - Cell culture | Actionable | In a preclinical study, the Bet inhibitor BETd-246 and the Bcl-xl inhibitor A-1155463 synergistically induced apoptosis in triple-receptor negative breast cancer cell lines in culture (PMID: 28209615). | 28209615 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Belinostat | Phase I | Actionable | In a Phase I trial, Beleodaq (belinostat) demonstrated safety and promoted stable disease in 39% (18/46) of patients with a variety of solid tumors (PMID: 18245542). | 18245542 | |
| Unknown unknown | acute myeloid leukemia | not applicable | AKN-028 + Daunorubicin | Preclinical - Cell culture | Actionable | In a preclinical study, the sequential treatment of Cerubidine (daunorubicin) and AKN-028 resulted in a syntergistic effect in acute myeloid leukemia cells in culture, demonstrating antileukemic activity (PMID: 22864397). | 22864397 | |
| Unknown unknown | breast cancer | not applicable | Alisertib + Fulvestrant | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with the combination of Alisertib (MLN8237) and Faslodex (fulvestrant) resulted in increased growth inhibition in aromatase inhibitor-resistant breast cancer cell lines compared to either agent alone (PMID: 25667100). | 25667100 | |
| Unknown unknown | Burkitt lymphoma | not applicable | CAR.k.28 cells | Preclinical - Cell culture | Actionable | In a preclinical study, patient-derived CAR.k.28 cells when co-cultured with a Kappa-positive Burkitt lymphoma cell line induced tumor cell lysis in culture, and inhibited tumor growth in a cell line xenograft model (PMID: 16926291). | 16926291 | |
| Unknown unknown | colorectal cancer | not applicable | Fluorouracil + Quinacrine + Sorafenib | Preclinical - Cell culture | Actionable | In a preclinical study, Acrichine (quinacrine) and Adrucil (5-fluorouracil) synergistically enhanced the cytotoxicity of Nexavar (sorafenib) in human colorectal cancer cell lines in culture (PMID: 21725213). | 21725213 | |
| Unknown unknown | CLL/SLL | not applicable | Idelalisib | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Zydelig (idelalisib) treatment resulted in an overall response rate of 58% (15/26, all partial response) in patients with relapsed small lymphocytic lymphoma, with a median duration of response of 11.9 months (PMID: 24450858; NCT01282424). | 24450858 detail... | |
| Unknown unknown | Advanced Solid Tumor | no benefit | SAR260301 | Phase I | Actionable | In a Phase I trial, SAR260301 treatment in advanced solid tumor patients failed to inhibit the PI3K pathway due to rapid clearance, and therefore, was not recommended for further clinical analysis (PMID: 28976556; NCT01673737). | 28976556 detail... | |
| Unknown unknown | colon cancer | not applicable | AZD2811 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with AZD2811 nanoparticles resulted in tumor regression in colon cancer xenograft models (PMID: 26865565). | 26865565 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Dostarlimab-gxly | Phase I | Actionable | In a Phase I trial, Jemperli (Dostarlimab-gxly) demonstrated safety and preliminary efficacy, resulted in partial response in 9.5% (2/21) and stable disease in 23.8% (5.21) of patients with advanced solid tumors (Annals of Oncology (2017) 28 (suppl_5): v403-v427. Abstract # 1185P; NCT02715284). | detail... | |
| Unknown unknown | acute monocytic leukemia | not applicable | Carfilzomib + Tinostamustine | Preclinical - Cell culture | Actionable | In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of an acute monocytic leukemia cell line in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | B-cell lymphoma | not applicable | Fenretinide | Phase I | Actionable | In a Phase I trial, Fenretinide treatment resulted in stable disease in a patient with B-cell lymphoma (PMID: 28420721). | 28420721 | |
| Unknown unknown | Kaposi's sarcoma | not applicable | Pomalidomide | FDA approved | Actionable | In a Phase I/II trial (12-C-0047) that supported FDA approval, Pomalyst (pomalidomide) treatment was well-tolerated and resulted in an objective response rate (ORR) of 73% (16/22) in patients with Kaposi's sarcoma, including HIV-positive patients who failed HAART (ORR 60%, 9/15) and HIV-negative patients (ORR 100%, 7/7) (PMID: 27863194; NCT01495598). | 27863194 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CX-2029 | Phase Ib/II | Actionable | In a Phase I/II trial (PROCLAIM-CX-2029), CX-2029 treatment demonstrated acceptable safety, and resulted in a partial response in 3.1% (1/32) and stable disease in 28.1% (9/32) of patients with advanced solid tumors (J Clin Oncol 38: 2020 (suppl; abstr 3502); NCT03543813). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | Torkinib | Preclinical - Patient cell culture | Actionable | In a preclinical study, Torkinib (PP242) treatment resulted in decreased mTORC2 signaling, growth inhibition and apoptosis in multiple myeloma cell lines and patient-derived multiple myeloma cells in culture, and reduced tumor growth in cell line xenograft animal models (PMID: 20686120). | 20686120 | |
| Unknown unknown | renal cell carcinoma | not applicable | SRF388 | Preclinical | Actionable | In a preclinical study, SRF388 treatment demonstrated antitumor activity in an orthotopic mouse model of renal cell carcinoma (AACR 2020, Therapeutic Antibodies 3; PO.IM02.25, Abs nr: 4550/18). | detail... | |
| Unknown unknown | leiomyosarcoma | not applicable | Pazopanib | Clinical Study | Actionable | In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 18.6 weeks and median survival of 20.1 months in patients with leiomyosarcoma (PMID: 26970174). | 26970174 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Intuvax | Phase I | Actionable | In a Phase I trial, Intuvax (ilixadencel) alone or in combination with Nexavar (sorafenib) demonstrated safety in hepatocellular carcinoma patients, and resulted in one partial response (Intuvax monotherapy), stable disease in 5 patients, increased circulating tumor-specific CD8-positive T-cells in 82% (9/11) of patients receiving Intuvax alone and 50% (2/4) of patients also receiving Nexavar, median time to progression of 5.5 months, and median overall survival of 7.5 months (PMID: 30719425; NCT01974661). | 30719425 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Cosibelimab | Phase I | Actionable | In a Phase I trial, Cosibelimab (CK-301) was tolerated and demonstrated preliminary efficacy, resulted in a partial response in 28% (10/36) and stable disease in 47% (17/36) of patients with advanced solid tumors, with 67% (24/36) of patients achieved target lesion reduction (Ann Oncol. Oct 2019, Vol 30 (Suppl_5): v177; NCT03212404). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Atezolizumab + RO7122290 | Phase I | Actionable | In a Phase I trial, RO7122290 and Tecentriq (atezolizumab) combination therapy demonstrated acceptable safety profile, induced anti-tumor immune response in tumor samples, and resulted in an objective response rate of 18.4% (7/39) in patients with advanced solid tumors (Ann Oncol Sep 2020, 31 (Suppl 4), S707; EUDRACT 2017-003961-83). | detail... | |
| Unknown unknown | colorectal adenocarcinoma | not applicable | CS-11 | Preclinical - Cell culture | Actionable | In a preclinical study, CS-11 induced cytotoxicity in a colorectal adenocarcinoma cell line in culture, and inhibited tumor growth in xenograft models (PMID: 28500231). | 28500231 | |
| Unknown unknown | breast cancer | not applicable | AJI-100 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, AJI-100 induced apoptosis and inhibited cell growth of breast cancer cell lines and xenografts (PMID: 24930769). | 24930769 | |
| Unknown unknown | ovarian cancer | not applicable | Carboplatin + ETP-46464 | Preclinical - Cell culture | Actionable | In a preclinical study, ETP-46464 increased the sensitivity of ovarian cancer cell lines to Paraplatin (carboplatin) in culture (PMID: 25560806). | 25560806 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | PEGPH20 | Phase I | Actionable | In a Phase Ib trial, PEGPH20 treatment resulted in median progression free survival of 7.2 months and overall survival of 13.0 months in hepatocellular carcinoma patients with high pretreatment tissue hyaluronan (HA) levels (n = 6), and 3.5 and 5.7 months respectively for patients with low HA levels (n = 11) (PMID: 26813359). | 26813359 | |
| Unknown unknown | esophageal cancer | not applicable | PP-121 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, PP-121 inhibited proliferation and growth of esophageal cancer cells in culture and in cell line xenograft models (PMID: 26235881). | 26235881 | |
| Unknown unknown | malignant pleural mesothelioma | not applicable | Cisplatin + Ganetespib + Pemetrexed Disodium | Phase I | Actionable | In a Phase Ib trial (MESO-02), treatment with Ganetespib and Alimta (Pemetrexed Disodium) plus Platinol (cisplatin) or Paraplatin (carboplatin) demonstrated safety in malignant pleural mesothelioma patients and resulted in an objective response rate of 52% (14/27, all partial responses), disease control rate of 81% (22/27), median progression-free survival of 5.8 mo, and median overall survival (mOS) of 11.5 mo, with mOS of 14.4 mo for patients who received Platinol (n=16) (PMID: 32669375; NCT01590160). | 32669375 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | NKTR-214 + VB10.NEO | Preclinical | Actionable | In a preclinical study, VB10.NEO and NKTR-214 synergistically enhanced neoantigen-specific T-cell response in animal tumor models (AACR Annual Meeting 2019, Abstract 2256). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ibrutinib + unspecified PD-L1 antibody | Preclinical | Actionable | In a preclinical study, the combination treatment of Imbruvica (ibrutinib) and an anti-PD-L1 antibody in mouse models with advanced solid tumors resulted in antitumor efficacy, including decreased tumor size, minimized metastasis, and improved survival in triple-receptor negative breast cancer mouse models, and a 30% cure rate and improved survival in colon cancer mouse models (PMID: 25730880). | 25730880 | |
| Unknown unknown | pancreatic cancer | no benefit | Gemcitabine + Tacedinaline | Phase II | Actionable | In a Phase II trial, the combination of Gemzar (gemcitabine) and Tacedinaline (CI-944) did not demonstrate benefit over Gemzar (gemcitabine) plus placebo in pancreatic cancer patients (PMID: 16641168). | 16641168 | |
| Unknown unknown | osteosarcoma | not applicable | HTH-01-015 | Preclinical - Cell culture | Actionable | In a preclinical study, HTH-01-015 inhibited MYPT1 phosphorylation, invasive behavior, and proliferation of an osteosarcoma cell line in culture (PMID: 24171924). | 24171924 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Docetaxel + Pembrolizumab | Phase II | Actionable | In a Phase II trial (PROLUNG), combination of Keytruda (pembrolizumab) and Taxotere (docetaxel) significantly improved objective response rate (42.5% vs 15.8%, OR=3.94, p=0.01) and progression-free survival (PFS) (9.5 vs 3.9 mo, HR=0.24, p<0.001) compared to Taxotere (docetaxel) alone in patients with advanced non-small cell lung cancer, PFS was improved in patients with (6.8 vs 3.5 mo, p=0.04) and without (9.5 vs 4.1 mo, p<0.01) EGFR alterations (PMID: 32271354; NCT02574598). | 32271354 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Capivasertib + Olaparib | Phase I | Actionable | In a Phase I trial, the combination of AZD5363 and Lynparza (olaparib) was well-tolerated and demonstrated preliminary activity in patients with endometrial, ovarian, or triple-negative breast cancer (TNBC), with an overall response rate of 24% (7/30; all partial responses, 1 ovarian, 4 endometrial, and 2 TNBC) and stable disease for greater than 4 months in 6 additional patients (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #391P; NCT02208375). | detail... | |
| Unknown unknown | neuroendocrine tumor | not applicable | Surufatinib | Phase Ib/II | Actionable | In a Phase Ib/II trial, Surufatinib (HMPL-012) treatment resulted in an objective response rate of 19% (8/42) and 15% (6/39), a disease control rate of 91% (38/42) and 92% (36/39), and a median progression-free survival of 21.2 and 13.4 months in patients with advanced, well-differentiated pancreatic and extrapancreatic neuroendocrine tumors, respectively (PMID: 30833272; NCT02267967). | 30833272 | |
| Unknown unknown | neuroendocrine tumor | not applicable | Surufatinib | Phase I | Actionable | In a Phase I trial, Surufatinib (HMPL-012) demonstrated an objective response rate in 44% (8/18) of patients with neuroendocrine tumors (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A1). | detail... | |
| Unknown unknown | lung carcinoma | not applicable | BNC105P | Preclinical - Cell line xenograft | Actionable | In a preclinical study, BNC105P treatment disrupted tumor vasculature and inhibited tumor growth in a cell line xenograft model of anaplastic lung carcinoma (PMID: 20515948). | 20515948 | |
| Unknown unknown | osteosarcoma | not applicable | Prexasertib | Preclinical - Cell culture | Actionable | In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including osteosarcoma cell lines (PMID: 28270495). | 28270495 | |
| Unknown unknown | chronic myeloid leukemia | not applicable | ST7612AA1 | Preclinical | Actionable | In a preclinical study, ST7612AA1 inhibited proliferation of chronic myeloid leukemia cells in culture (PMID: 25671299). | 25671299 | |
| Unknown unknown | colon cancer | not applicable | Navicixizumab | Preclinical | Actionable | In a preclinical study, Navicixizumab (OMP-305B83) inhibited proliferation of endothelial cells in culture and demonstrated antitumor activity in vivo in multiple solid tumor types, including colon tumors (Mol Cancer Ther December 2015 14; C164). | detail... | |
| Unknown unknown | lung carcinoma | not applicable | MYCi975 | Preclinical | Actionable | In a preclinical study, MYCi975 treatment inhibited tumor growth in a mouse model of Lewis lung carcinoma (PMID: 31679823). | 31679823 | |
| Unknown unknown | melanoma | not applicable | Dovitinib | Phase Ib/II | Actionable | In a Phase I/II study, Dovitinib (TKI258) was demonstrated safe, but of limited clinical benefit in patients with advanced melanoma (PMID: 21976540). | 21976540 | |
| Unknown unknown | pancreatic ductal adenocarcinoma | not applicable | Gemcitabine + Nab-paclitaxel + Vantictumab | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of Vantictumab (OMP-18R5), Abraxane (nab-paclitaxel), and Gemzar (gemcitabine) in patients with pancreatic ductal adenocarcinoma resulted in an overall response rate of 41.9% (13/31), including 13 partial responses, stable disease in 35.5% (11/31), a clinical benefit rate of 77.4%, a median progression-free survival of 166.0 days, and a median overall survival of 305.0 days (PMID: 31338636). | 31338636 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | Prexasertib + Ralimetinib | Phase I | Actionable | In a Phase I trial, treatment with the combination of Prexasertib (LY2606368) and Ralimetinib (LY2228820) in patients with advanced solid tumors (n=9) resulted in stable disease in one patient, however, further clinical exploration of this treatment combination was discontinued due to safety concerns related to hematological toxicity (PMID: 31707688; NCT02860780). | 31707688 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | AZD8055 | Phase I | Actionable | In a Phase I trial, AZD8055 treatment demonstrated safety and tolerability, and resulted in stable disease for more than 4 months in 14% (7/49) of patients with advanced solid tumors or lymphoma (PMID: 22935583). | 22935583 | |
| Unknown unknown | high grade glioma | not applicable | DNX-2401 | Phase I | Actionable | In a Phase I trial, DNX-2401 treatment demonstrated virus-induced oncolysis in patients' tumors, resulted in more than 95% tumor reduction in 3 patients, and survival for more than 3 years in 20% (5/25) of patients with recurrent malignant glioma (PMID: 29432077; NCT02197169). | 29432077 | |
| Unknown unknown | high grade glioma | not applicable | DNX-2401 | Phase I | Actionable | In a Phase I trial, DNX-2401 treatment resulted in tumor reduction in 72% (18/25), complete response in 12% (3/25) and prolonged stable disease in 8% (2/25) of patients with recurrent malignant glioma, with a median overall survival of 9.5 months and progression-free survival of more than 3 years in patients achieved complete responses (PMID: 29432077). | 29432077 | |
| Unknown unknown | transitional cell carcinoma | not applicable | Sabatolimab + Spartalizumab | Case Reports/Case Series | Actionable | In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in stable disease in 3 patients with transitional cell carcinoma (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). | detail... | |
| Unknown unknown | melanoma | not applicable | Lenvatinib | Phase I | Actionable | In a Phase I trial, Lenvima (lenvatinib) was demonstrated to be well tolerated and displayed anti-tumor activity in patients with melanoma and renal cell carcinoma (PMID: 22516948). | 22516948 | |
| Unknown unknown | lung non-squamous non-small cell carcinoma | not applicable | Cisplatin + Pemetrexed Disodium + Tislelizumab | Phase II | Actionable | In a Phase II trial, treatment with Tislelizumab (BGB-A317) plus platinum doublet chemotherapy (Alimta (Pemetrexed Disodium) with Platinol (cisplatin) or Paraplatin (carboplatin)) in non-squamous non-small cell lung cancer patients resulted in an objective response rate of 44% (7/16) and disease control rate of 94% (15/16), including a partial response in seven patients and stable disease in eight patients, and median progression-free survival of 9.0 months (PMID: 32769013; NCT03432598). | 32769013 | |
| Unknown unknown | pancreatic cancer | not applicable | Galunisertib | Phase I | Actionable | In a Phase I trial, two patients with pancreatic cancer demonstrated a best response of stable disease when treated with Galunisertib (LY2157299) (PMID: 26526984; NCT01722825). | 26526984 | |
| Unknown unknown | transitional cell carcinoma | not applicable | Toripalimab | Phase I | Actionable | In a Phase I trial, Toripalimab (JS001) demonstrated safety and preliminary efficacy, resulted in an objective response rate of 25.0% (2/8) and a disease control rate of 67.5% (5/8, 2 partial response, 3 stable disease) in patients with advanced urothelial carcinoma (PMID: 30642373; NCT02836795). | 30642373 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | GSK2801 + JQ1 | Preclinical - Cell culture | Actionable | In a preclinical study, GSK2801 and JQ1 treatment synergistically inhibited cell growth and viability, led to enhanced cell cycle arrest, and induced senescence and apoptosis in triple-negative breast cancer cell lines in culture (PMID: 31000582). | 31000582 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Flucytosine + TG4023 | Phase I | Actionable | In a Phase I trial, TG4023 and Flucytosine combination therapy demonstrated safety and preliminary efficacy, resulted in stable disease in 50% (8/16) of patients with advanced solid tumors (PMID: 28177438; NCT00978107). | 28177438 | |
| Unknown unknown | breast cancer | not applicable | LY3022855 | Phase I | Actionable | In a Phase I trial, LY3022855 (IMC-CS4) treatment was well-tolerated and demonstrated safety, induced activation of immune cells, and resulted in stable disease as the best response in 24% (5/21) of refractory metastatic breast cancer patients with a duration of response of 82-302 days, and a median progression-free survival of 1-3 months (PMID: 32847933; NCT02265536). | 32847933 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Everolimus + Vorolanib | Phase I | Actionable | In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in partial response in 9.5% (2/21) and stable disease in 57.1% (12/21) of patients with advanced solid tumors (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). | detail... | |
| Unknown unknown | colorectal cancer | no benefit | Crizotinib + SBI-0206965 | Preclinical - Cell culture | Actionable | In a preclinical study, Xalkori (crizotinib) treatment in combination with SBI-0206965 did not enhance apoptosis over Xalkori (crizotinib) alone in colorectal cancer cells in culture (PMID: 30026382). | 30026382 | |
| Unknown unknown | colorectal cancer | not applicable | Danusertib | Phase II | Actionable | In a Phase II clinical trial, Danusertib (PHA-739358) showed limited efficacy in patients with metastatic colorectal cancer (J Clin Oncol 28, 2010 (suppl; abstr e13558)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | PQR309 | Phase I | Actionable | In a Phase I trial, PQR309 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2560)). | detail... | |
| Unknown unknown | malignant peripheral nerve sheath tumor | not applicable | Capmatinib | Case Reports/Case Series | Actionable | In a Phase I trial, Tabrecta (capmatinib) demonstrated safety and preliminary efficacy, resulted in stable disease as best overall response in a patients with malignant peripheral nerve sheath tumor (PMID: 30724423). | 30724423 | |
| Unknown unknown | triple-receptor negative breast cancer | no benefit | Buparlisib | Phase II | Actionable | In a Phase II trial, Buparlisib (BKM120) treatment in patients with triple-negative breast cancer (TNBC) resulted in median progression-free survival of 1.8 months, median overall survival of 11.2 months, stable disease lasting more than four months in 12% (6/50) of patients, and no objective responses, and due to the limited clinical efficacy, further study as a single agent in TNBC patients was not supported (PMID: 33138866; NCT01790932, NCT01629615). | 33138866 | |
| Unknown unknown | prostate cancer | not applicable | AZD8186 + Vistusertib | Phase I | Actionable | In a Phase I trial, the combination of AZD8186 and Vistusertib (AZD2014) resulted in a partial response in a patient with castration resistant prostate cancer (J Clin Oncol 35, 2017 (suppl; abstr 2570)). | detail... | |
| Unknown unknown | brain stem glioma | not applicable | MRK-003 | Preclinical - Cell culture | Actionable | In a preclinical study, MRK-003 increased apoptosis and decreased growth of diffuse pontine glioma cell lines in culture (PMID: 26115193). | 26115193 | |
| Unknown unknown | Ewing sarcoma of bone | not applicable | Dasatinib | Phase II | Actionable | In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in Ewing sarcoma patients (n=17) was suspended due to lack of drug activity (PMID: 26710211). | 26710211 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | GSK126 | Phase I | Actionable | In a Phase I trial, GSK126 was tolerated and demonstrated preliminary efficacy, resulted in partial response in 2% (1/41) and stable disease in 34% (14/41) of patients with advanced solid tumor (n=21) or B-cell lymphoma (n=20) (PMID: 31471312; NCT02082977). | 31471312 | |
| Unknown unknown | cholangiocarcinoma | not applicable | OPB-111077 | Phase I | Actionable | In a Phase I trial, a patient with cholangiocarcinoma demonstrated stable disease when treated with OPB-111077 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B118). | detail... | |
| Unknown unknown | cervix carcinoma | not applicable | Bevacizumab + Cisplatin + Paclitaxel | FDA approved | Actionable | In a Phase III trial that supported FDA approval, the addition of Avastin (bevacizumab) to Platinol (cisplatin) and Taxol (paclitaxel) chemotherapy resulted in improved overall survival and progression-free survival compared to chemotherapy alone in patients with cervical cancer (PMID: 25281440, PMID: 24552320). | detail... 25281440 24552320 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Triptolide | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with Triptolide (C1572) decreased Myc protein expression, decreased cancer stem-like cell (CSC) number, and induced senescence in a triple-negative breast cancer (TNBC) cell line in culture, and depleted CSCs and reduced tumor growth in TNBC cell line xenograft models (PMID: 28951456). | 28951456 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | XL228 | Phase I | Actionable | In a Phase I clinical trial, 41% (9/22) of patients with an advanced solid tumor experienced stable disease for at least 12 weeks and a partial response response was reported in one patient with NSCLC when treated with XL228 (J Clin Oncol 28:15s, 2010 (suppl; abstr 3105)). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Umbralisib | Phase I | Actionable | In a Phase I trial, Ukoniq (umbralisib) treatment resulted in objective response in 31% (4/13) and stable disease in 15% (2/13) of patients with diffuse large B-cell lymphoma (PMID: 29475723; NCT01767766). | 29475723 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | G-TPP + Navitoclax | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Navitoclax (ABT-263) synergistically inhibited viability of triple-receptor negative breast cancer cells in culture, and inhibited tumor growth in cell line xenograft models (PMID: 28522750). | 28522750 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Erlotinib + Lumretuzumab | Phase Ib/II | Actionable | In a Phase Ib trial, treatment with the combination of Tarceva (erlotinib) and Lumretuzumab demonstrated minimal clinical efficacy in ERBB3 (HER3)-positive advanced solid tumor patients, resulting in an objective response rate of 4.2% (3/71), including a partial response in a patient with ovarian cancer and in two patients with squamous non-small cell lung carcinoma (PMID: 28600476). | 28600476 | |
| Unknown unknown | triple-receptor negative breast cancer | no benefit | GSK2801 + HY-16462 | Preclinical - Cell culture | Actionable | In a preclinical study, GSK2801 and HY-16462 combination treatment did not synergistically inhibit growth of a triple-negative breast cancer cell line in culture (PMID: 31000582). | 31000582 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Cabozantinib + CT-707 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Cometriq (Cabometyx, cabozantinib) and CT-707 resulted in synergism in hepatocellular carcinoma cells, demonstrating increased apoptosis and inhibition of colony formation in culture and decreased tumor weight in xenograft models (PMID: 27638856). | 27638856 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Guadecitabine | Phase I | Actionable | In a Phase I trial, SGI-110 treatment resulted in clinical response in 8% (6/74) of patients with acute myeloid leukemia (PMID: 26296954). | 26296954 | |
| Unknown unknown | acute myeloid leukemia | not applicable | AMG 397 + Venetoclax | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AMG 397 treatment in combination with Venclexta (venetoclax) induced tumor regression in an orthotopic cell line xenograft model of acute myeloid leukemia (Cancer Res 2020;80(16 Suppl):Abstract nr 6218). | detail... | |
| Unknown unknown | adult T-cell leukemia | no benefit | Dinaciclib | Preclinical - Cell culture | Actionable | In a preclinical study, adult T cell leukemia cells were not sensitive to treatment with Dinaciclib (SCH 727965) in culture (PMID: 32907612). | 32907612 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | DS6051b | Phase I | Actionable | In a Phase I clinical trial, DS-6051b was well-tolerated and demonstrated some preliminary efficacy in patients with advanced solid tumors (Cancer Res July 15 2016 (76) (14 Supplement) CT024). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MVAp53 + Pembrolizumab | Phase I | Actionable | In a Phase I trial, MVAp53 and Keytruda (pembrolizumab) combination therapy demonstrated safety and preliminary efficacy, resulting in a clinical response in 27.3% (3/11) of patients with advanced solid tumors, who remained with stable disease for up to 49 weeks (PMID: 30094792). | 30094792 | |
| Unknown unknown | prostate cancer | not applicable | Atezolizumab + Cabozantinib | Phase I | Actionable | In a Phase I trial (COSMIC-021), Tecentriq (atezolizumab) and Cometriq (Cabometyx, cabozantinib) combination therapy was tolerated, resulted in an objective response rate of 32% (14/44, 2 complete responses, 12 partial responses) and stable disease in 48% (21/44) of patients with metastatic castration-resistant prostate cancer whose disease progressed after Xtandi (enzalutamide) or Zytiga (abiraterone) treatment (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 5564-5564; NCT03170960). | detail... | |
| Unknown unknown | alveolar soft part sarcoma | not applicable | Anlotinib | Phase II | Actionable | In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 77%, median progression-free survival of 21 months, an objective response rate of 46% (n=13), and median overall survival was not reached in patients with alveolar soft part sarcoma (PMID: 29895706; NCT01878448). | 29895706 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Chiauranib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a non-small cell lung cancer cell line xenograft model (PMID: 28004478). | 28004478 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Itacitinib + Parsaclisib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Parsaclisib (INCB050465) and INCB039110 combination treatment inhibited proliferation of diffuse large B-cell lymphoma cell lines in culture and induced tumor regression in xenograft models (Cancer Res August 1, 2015 75; 2671). | detail... | |
| Unknown unknown | ovarian carcinoma | not applicable | Triapine | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Triapine treatment delayed tumor growth in a cell line xenograft model of ovarian carcinoma (PMID: 10692563). | 10692563 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Bortezomib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, substituting Velcade (bortezomib) for vincristine in R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone) resulted in improved progression-free survival (24.7 vs 14.4 months, HR=0.63, p<0.001) compared to R-CHOP in patients with newly diagnosed mantle cell lymphoma (PMID: 25738670; NCT00722137). | 25738670 detail... | |
| Unknown unknown | mantle cell lymphoma | not applicable | Bortezomib | FDA approved | Actionable | In a Phase II trial (PINNACLE) that supported FDA approval, Velcade (bortezomib) monotherapy resulted in an overall response rate of 31% (48/155, 12 complete response, 36 partial response) in patients with relapsed or refractory mantle cell lymphoma, with a median overall survival not reached after a median follow-up of 13.4 months (PMID: 17001068; NCT00063713). | 17001068 detail... | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Camrelizumab | Phase I | Actionable | In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity, resulted in partial response in 33.3% (1/3) of patients with hepatocellular carcinoma (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). | detail... | |
| Unknown unknown | colon cancer | not applicable | THOR-707 | Preclinical | Actionable | In a preclinical study, treatment with THOR-707 resulted in an increase in increased CD8-positive T cell tumor infiltration and demonstrated anti-tumor activity in a syngeneic mouse model of colon cancer (Cancer Res 2019;79(13 Suppl):Abstract nr 3258). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Cabozantinib + Erlotinib | Phase Ib/II | Actionable | In a Phase Ib/II trial, Cometriq (cabozantinib) and Tarceva (erlotinib) combination treatment resulted in no response (0/13) in patients with non-small cell lung carcinoma that had progressed during treatment with Tarceva (erlotinib) in Phase II, despite an objective response rate of 8.2% (5/61) in Phase I (PMID: 28352985). | 28352985 | |
| Unknown unknown | rhabdomyosarcoma | not applicable | TAS4464 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TAS4464 inhibited growth and induced apoptosis in rhabdomyosarcoma cell lines in culture, resulted in tumor suppression in cell line xenograft models (Cancer Res 2016;76(14 Suppl):Abstract nr 3777). | detail... | |
| Unknown unknown | non-Hodgkin lymphoma | not applicable | Bendamustine + Rituximab + Veliparib | Phase I | Actionable | In a Phase I trial, seven patients with non-Hodgkin lymphoma treated with a combination of Veliparib (ABT-888), Bendamustine, and Rituxan (rituximab) demonstrated an overall response rate of 86% (6/7) and a complete response rate of 71% (5/7), and a progression free survival of 14.2 months (PMID: 28314788; NCT01326702). | 28314788 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Gemcitabine + SRA737 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a non-small cell lung carcinoma cell line demonstrated sensitivity to the combination of SRA737 (CCT245737) and Gemzar (gemcitabine), resulting in antitumor efficacy in both culture and xenograft models (PMID: 27167172). | 27167172 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Pembrolizumab | FDA approved | Actionable | In a Phase II trial (KEYNOTE-158) that supported FDA approval, Keytruda (pembrolizumab) treatment resulted in an objective response rate of 18.7% (20/107) in patients with advanced small-cell lung cancer, with a median progression-free survival of 2.0 months (J Clin Oncol 36, no. 15_suppl (May 20 2018) 8506-8506; NCT02628067). | detail... detail... | |
| Unknown unknown | endometrial carcinoma | not applicable | CC-90010 | Case Reports/Case Series | Actionable | In a Phase I trial, CC-90010 treatment demonstrated safety, and resulted in a partial response in an endometrial carcinoma patient harboring an ESR1-AKAP12 fusion, ESR1 amplification, and PIK3CA and FGFR2 mutations (PMID: 32240793; NCT03220347). | 32240793 | |
| Unknown unknown | triple-receptor negative breast cancer | no benefit | Cisplatin + Everolimus + Paclitaxel | Phase II | Actionable | In a Phase II trial, the addition of Afinitor (everolimus) to the combined treatment of Platinol (cisplatin) and Taxol (paclitaxel) in patients with triple-receptor negative breast cancer did not result in improved clinical response when compared to Platinol (cisplatin), Taxol (paclitaxel), and placebo, and resulted in greater adverse events (PMID: 28270498). | 28270498 | |
| Unknown unknown | neuroblastoma | not applicable | Ellipticine + Valproic acid | Preclinical | Actionable | In a preclincal study, Depakene (valproic acid) enhanced the cytotoxicity of ellipticine in human neuroblastoma cell lines (PMID: 22167207). | 22167207 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Pamiparib + Tislelizumab | Phase Ib/II | Actionable | In a Phase Ib trial, the combination therapy of Tislelizumab (BGB-A317) and Pamiparib (BGB-290) in patients with advanced solid tumors was well-tolerated and resulted in an objective response in 20% (10/49), with two complete responses and eight partial responses, stable disease in 32% (16/49), a disease control rate of 53% (26/49), and a clinical benefit of 39% (PMID: 31378459; NCT02660034). | 31378459 | |
| Unknown unknown | breast cancer | not applicable | PI-273 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, PI-273 induced cell-cycle arrest and apoptosis and decreased proliferation in breast cancer cell lines with wild-type RAS in culture, and inhibited tumor growth in a xenograft model (PMID: 28827373). | 28827373 | |
| Unknown unknown | lung carcinoma | not applicable | SRX3207 | Preclinical - Cell culture | Actionable | In a preclinical study, SRX3207 treatment induced cytotoxicity in Lewis lung carcinoma cells in culture, and inhibited tumor growth, and increased survival in a syngeneic mouse model (PMID: 31974273). | 31974273 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Docetaxel + Trametinib | Phase I | Actionable | In a Phase I/Ib trial, treatment with the combination of Mekinist (trametinib) and Taxotere (docetaxel) resulted in an overall response rate (ORR) of 21% (10/47, all partial responses) and stable disease in 43% (20/47) of patients with non-small cell lung cancer (PMID: 27876675). | 27876675 | |
| Unknown unknown | prostate cancer | not applicable | CC214-2 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, prostate cancer cell line xenograft models demonstrated inhibition of tumor growth when treated with CC214-2 (PMID: 23414803). | 23414803 | |
| Unknown unknown | stomach cancer | not applicable | Camrelizumab + Rivoceranib | Phase Ib/II | Actionable | In a Phase Ib trial, combined Camrelizumab (SHR-1210) and Apatinib (YN968D1) treatment resulted in an overall response rate of 17.4% (4/23), a disease control rate of 78.3% (18/23), a median progression-free survival (PFS) of 2.9 months, and an overall survival of 11.4 months in evaluable patients with gastric or gastroesophageal junction cancer (PMID: 30348638; NCT02942329). | 30348638 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase I/II trial (CheckMate 032) that supported FDA approval, Opdivo (nivolumab) as third- or later-line treatment resulted in an objective response rate of 11.9% (13/109, 1 complete response, 12 partial response) in patients with metastatic small cell lung cancer, with a median duration of response of 17.9 months (PMID: 29731394, PMID: 30316010; NCT01928394). | 29731394 30316010 detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Carboplatin + MEDI3617 + Paclitaxel | Phase I | Actionable | In a Phase I trial, MEDI3617 in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) resulted in no objective response (0/7) and stable disease in 43% (3/7) of patients with advanced solid tumors (PMID: 29559563; NCT01248949). | 29559563 | |
| Unknown unknown | head and neck cancer | not applicable | TG6002 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with TG6002 resulted in tumor growth inhibition and led to complete responses in head and neck cancer cell line xenograft models (PMID: 31011628). | 31011628 | |
| Unknown unknown | stomach cancer | not applicable | JNJ-26483327 | Preclinical | Actionable | In a preclinical study, human gastric cancer cells demonstrated sensitivity to JNJ-26483327 (PMID: 19584230). | 19584230 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Adavosertib + Irinotecan | Phase I | Actionable | In a Phase I trial (ADVL1312), the combination of Adavosertib (MK-1775) and Camptosar (irinotecan) demonstrated tolerability and some preliminary efficacy in pediatric patients with advanced solid tumors, including one Ewing sarcoma patient who achieved a partial response for three months, and one patient with ependymoma and one patient with neuroblastoma who demonstrated stable disease over 12 months and 7.5 months, respectively (PMID: 31857431). | 31857431 | |
| Unknown unknown | multiple myeloma | not applicable | Bortezomib + Daratumumab + Dexamethasone | FDA approved | Actionable | In a Phase III trial (CASTOR) that supported FDA approval, the combination of Darzalex (daratumumab), Velcade (bortezomib), and Adexone (dexamethasone) resulted in a greater 12 month progression-free survival (77.5% vs 29.4%) and overall response (82.9%; 199/240 vs 63.2%; 148/234) compared to Adexone (dexamethasone) and Velcade (bortezomib) alone in relapsed or refractory multiple myeloma patients (PMID: 27557302; NCT02136134). | detail... 27557302 | |
| Unknown unknown | stomach cancer | not applicable | Mogamulizumab + Nivolumab | Phase I | Actionable | In a Phase I trial, Poteligeo (mogamulizumab-kpkc) and Opdivo (nivolumab) combination treatment demonstrated acceptable safety, resulted in an objective response rate of 0% (0/15) and a disease control rate of 27% (4/15) in immunotherapy-naive patients with advanced or metastatic gastric cancer, response occurred regardless of PD-L1, CCR4, CD8 expression levels and tumor mutational burden (PMID: 31455681; NCT02476123). | 31455681 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Cytarabine + Daunorubicin + Glasdegib | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of Glasdegib (PF-04449913), Cytosar-U (cytarabine), and Cerubidine (daunorubicin) resulted in an overall survival of 34.7 months in patients with either acute myeloid leukemia (AML) or myelodysplastic syndrome, with 55% (11/20) of AML patients experiencing a complete remission (PMID: 29463550). | 29463550 | |
| Unknown unknown | melanoma | not applicable | Ipilimumab + Sargramostim + UV1 Telomerase peptide vaccine | Phase I | Actionable | In a Phase I/IIa trial, UV1 Telomerase peptide vaccine combined with Yervoy (ipilimumab) and Sargramostim was well-tolerated, and resulted in a complete response and three partial responses (n=12) in metastatic melanoma patients, with a three-year overall survival of 67% (Journal of Clinical Oncology 2020 38:5_suppl, 62-62; NCT02275416). | detail... | |
| Unknown unknown | thymic carcinoma | not applicable | CC-90010 | Case Reports/Case Series | Actionable | In a Phase I trial, CC-90010 treatment demonstrated safety, and resulted in a 17% tumor reduction in a patient with metastatic malignant thymoma (PMID: 32240793; NCT03220347). | 32240793 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Cetuximab + Dasatinib | Phase II | Actionable | In a Phase II trial, Sprycel (dasatinib) and Erbitux (cetuximab) combination treatment resulted in stable disease in 46% (6/13) and disease progression in 54% (7/13) of patients with cetuximab-resistant head and neck squamous cell carcinoma, with low serum IL6 level correlated with stable disease (PMID: 28559019). | 28559019 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | unspecified PD-1 antibody | Clinical Study | Actionable | In a retrospective analysis, treatment with an unspecified PD-1 or PD-L1 therapy in metastatic non-small cell lung cancer patients harboring a mutation in BRAF, ERBB2 (HER2), or MET, or a RET translocation led to a response rate of 29% (31/107), a 15.4 mo duration of response, a 4.7 mo median progression-free survival, and a 16.2 mo median overall survival, and resulted in clinical efficacy similar to what has been observed in studies with unselected non-small cell lung cancer patients (PMID: 31945494). | 31945494 | |
| Unknown unknown | clear cell renal cell carcinoma | no benefit | Everolimus + Pazopanib | Phase II | Actionable | In a Phase II trial, alternating treatment with Votrient (pazopanib) and Afinitor (everolimus) did not improve 1-year progression free survival rate (45% vs 32%) or time to progression/death (7.4 vs 9.4 months) compared to continuous Votrient (pazopanib) treatment in patients with renal clear cell carcinoma (PMID: 27918762). | 27918762 | |
| Unknown unknown | renal cell carcinoma | not applicable | Cabozantinib + Nivolumab | FDA approved | Actionable | In a Phase III trial (CHECKMATE-9ER) that supported FDA approval, Opdivo (nivolumab) in combination with Cabometyx (cabozantinib) significantly improved median progression-free survival (16.6 vs 8.3 mo, HR 0.51, P < 0.0001), overall survival (HR 0.60, p<0.0010), and objective response rate (55.7% vs 27.1%, P < 0.0001) compared to Sutent (sunitinib) in patients with advanced real cell carcinoma (Annals of Oncology (2020) 31 (suppl_4): S1142-S1215. 10.1016/annonc/annonc325; NCT03141177). | detail... detail... detail... | |
| Unknown unknown | oral cavity cancer | not applicable | Duvelisib + unspecified PD-L1 antibody | Preclinical | Actionable | In a preclinical study, treatment with the combination of low-dose Copiktra (duvelisib) and a PD-L1 antibody resulted in increased infiltration of CD8-positive tumor-infiltrating lymphocytes, decreased tumor volume, and improved survival in mice bearing T-cell inflamed murine oral cancer cell-derived tumors, when compared to either agent alone (PMID: 28364000). | 28364000 | |
| Unknown unknown | Ewing sarcoma | no benefit | Pembrolizumab | Phase II | Actionable | In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in no objective response (0/13) in patients with Ewing sarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). | detail... | |
| Unknown unknown | prostate cancer | not applicable | Abiraterone + Prednisone | FDA approved | Actionable | In a Phase III trial (LATITUDE) that supported FDA approval, treatment with the combination of Zytiga (abiraterone) and Predisone, along with androgen-deprivation therapy, resulted in improved median overall survival (not reached vs. 34.7 months; HR=0.62) and median progression-free survival (33.0 months vs. 14.8 months; HR=0.47) compared to treatment with placebos in patients with metastatic castration-sensitive prostate cancer (PMID: 28578607; NCT01715285). | detail... 28578607 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Cisplatin + Fluorouracil + Pembrolizumab | FDA approved | Actionable | In a Phase III trial (KEYNOTE-048) that supported FDA approval, Keytruda (pembrolizumab) in combination with platinum and Adrucil (fluorouracil) significantly improved overall survival compared to Erbitux (cetuximab) plus chemotherapy (13.0 vs 10.7 months, HR=0.77, p=0.0067) in patients with metastatic or unresectable recurrent head and neck squamous cell carcinoma (J Clin Oncol 37, no. 15_suppl (May 20 2019) 6000-6000; NCT02358031). | detail... detail... | |
| Unknown unknown | colorectal cancer | not applicable | LY2090314 + Talazoparib | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with LY2090314 sensitized colorectal cancer cell lines with either BRCA1/2 proficiency or BRCA2 deficiency to Talzenna (talazoparib) in culture, resulting in a synergistic effect (PMID: 33589588). | 33589588 | |
| Unknown unknown | gastrointestinal stromal tumor | not applicable | Dovitinib | Phase II | Actionable | In a Phase II clinical trial, Dovitinib (TKI258) demonstrated safety and efficacy in heavily pretreated patients with advanced GISTs (PMID: 24084771). | 24084771 | |
| Unknown unknown | skin squamous cell carcinoma | not applicable | R11.1.6 | Preclinical - Cell culture | Actionable | In a preclinical study, R11.1.6 treatment did not inhibit colony formation in a skin squamous cell carcinoma cell line in culture (PMID: 29720559). | 29720559 | |
| Unknown unknown | alveolar soft part sarcoma | not applicable | Crizotinib | Phase II | Actionable | In a Phase II trial, Xalkori (crizotinib) treatment resulted in partial response (PR) in 2.5% (1/40) and stable disease (SD) in 87.5% (35/40) of patients with TFE3-rearranged, MET-positive alveolar soft part sarcoma, with a 1-year progression-free survival (PFS) rate of 37.5%, and resulted in PR in 25% (1/4) and SD in 75% (3/4) of patients without TFE3 rearrangement and MET expression, with a 1-year PFS rate of 50% (PMID: 29216400; NCT01524926). | 29216400 | |
| Unknown unknown | thyroid gland cancer | not applicable | Sorafenib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) improved median progression free survival to 10.8 months in metastatic differentiated thyroid cancer patients (PMID: 24768112). | detail... 24768112 | |
| Unknown unknown | multiple myeloma | not applicable | CB-5083 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CB-5083 treatment of multiple myeloma cell lines and xenografts resulted in cell death and decreased tumor growth, respectively (PMID: 28878026). | 28878026 | |
| Unknown unknown | colon carcinoma | not applicable | SLC-391 | Preclinical | Actionable | In a preclinical study, SLC-391 treatment did not inhibit proliferation of a colon carcinoma cell line in culture, however, inhibited tumor growth by 37% in a syngeneic mouse model (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B148). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Aflibercept + Docetaxel | Phase III | Actionable | In a Phase III trial, the combination of Zaltrap (aflibercept) and Taxotere (docetaxel) did not result in improved overall survival compared to Taxotere (docetaxel) with placebo, but did result in an improved overall response rate of 23.3% (94/404) vs. 8.9% (36/406) with Taxotere (docetaxel) plus placebo in non-small cell lung cancer patients that had failed platinum therapy (PMID: 22965962). | 22965962 | |
| Unknown unknown | sarcoma | not applicable | Doxorubicin + Olaratumab | FDA approved | Actionable | In a Phase I/IIb trial that supported FDA approval, Lartruvo (olaratumab) and Adriamycin (doxorubicin) combination treatment improved median progression free survival (6.6 vs. 4.1 months) and median overall survival (26.5 vs. 14.7 months) compared to Adriamycin (doxorubicin) treatment alone in patients with advanced soft tissue sarcoma (PMID: 27291997; NCT01185964). | 27291997 detail... detail... | |
| Unknown unknown | prostate cancer | not applicable | CC-115 | Phase I | Actionable | In a Phase I trial, CC-115 treatment was tolerated and demonstrated manageable safety, and resulted in stable disease as the best response in 63.6% (7/11) of castration-resistant prostate cancer patients, and a median progression-free survival of 345 days (PMID: 31853198; NCT01353625). | 31853198 | |
| Unknown unknown | thyroid gland cancer | not applicable | SNS-314 | Preclinical | Actionable | In a preclinical study, SNS-314 reduced growth of anaplastic thyroid carcinoma cells in culture (PMID: 23099978). | 23099978 | |
| Unknown unknown | breast cancer | not applicable | AsiDNA + Niraparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA and Zejula (niraparib) combination treatment resulted in increased cell death and inhibition of proliferation in breast cancer cell lines compared to Zejula (niraparib) alone in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | colorectal cancer | not applicable | OSI-027 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, OSI-027 inhibited mTORC1 and mTORC2 signaling and growth in colorectal cancer cell line xenograft models (PMID: 21673091). | 21673091 | |
| Unknown unknown | hairy cell leukemia | not applicable | Moxetumomab pasudotox-tdfk | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Lumoxiti (moxetumomab pasudotox-tdfk) treatment resulted in durable complete response in 30% (24/80), complete response in 41% (33/80), objective response (complete response and partial response) in 75% (60/80), and hematologic remission in 80% (64/80) of patients with relapsed or refractory hairy cell leukemia who had more than 2 prior systemic therapies (PMID: 30030507; NCT01829711). | 30030507 detail... | |
| Unknown unknown | prostate cancer | not applicable | KB-0742 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with KB-0742 in a prostate cancer cell line xenograft model resulted in a significantly greater inhibition of tumor growth at 82% when compared to treatment with Taxotere (docetaxel) (PMID: 33086052). | 33086052 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | CUDC-907 | Phase I | Actionable | In a Phase I trial, CUDC-907 was well-tolerated, demonstrated safety, and resulted in a 56% (5/9) objective response rate, including two complete responses and three partial responses, in patients with diffuse large B-cell lymphoma (PMID: 27049457). | 27049457 | |
| Unknown unknown | kidney angiomyolipoma | not applicable | Everolimus | FDA approved | Actionable | In a Phase III trial (EXIST-2) that supported FDA approval, Afinitor (everolimus) treatment resulted in significantly improved response rate (42%, 33/79) compared to placebo (0%, 0/39) in patients with renal angiomyolipoma as a feature of tuberous sclerosis (n=113) or sporadic lymphanioleiomyomatosis (n=5) (PMID: 23312829; NCT00790400). | 23312829 detail... | |
| Unknown unknown | pancreatic adenocarcinoma | not applicable | Temozolomide + TRC102 | Case Reports/Case Series | Actionable | In a Phase I trial, combination therapy with Temodar (temozolomide) and TRC102 (methoxyamine) resulted in prolonged stable disease (4 months) in a patient with advanced pancreatic adenocarcinoma (PMID: 32556884; NCT00892385). | 32556884 | |
| Unknown unknown | urinary bladder cancer | not applicable | CG0070 | Phase II | Actionable | In a Phase II trial, treatment with CG0070 resulted in a complete response rate of 47% (21/45) in patients with BGC-unresponsive high grade non-muscle invasive bladder cancer (PMID: 28755959; NCT02365818). | 28755959 | |
| Unknown unknown | duodenum adenocarcinoma | not applicable | Ramucirumab | Phase II | Actionable | In a Phase II study, Cyramza (ramuciramab), in combination with mFOLFOX-6 chemotherapy regimen (Wellcovorin (leucovorin), Adrucil (fluorouracil) and Eloxatin (oxaliplatin)), demonstrated safety and efficacy in metastatic colorectal cancer (PMID: 24674871). | 24674871 | |
| Unknown unknown | colon cancer | not applicable | PF-06840003 | Preclinical | Actionable | In a preclinical study, PF-06840003 inhibited tumor growth in syngeneic mouse models of colon cancer (PMID: 30232146). | 30232146 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Poziotinib | Phase I | Actionable | In a Phase I trial, Poziotinib (HM781-36B) displayed favorable pharmacokinetics in patients with advanced solid tumors (PMID: 25377158). | 25377158 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | LY3164530 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, LY3164530 demonstrated anti-tumor activity in cell line xenograft models of non-small cell lung carcinoma (AACR; Cancer Res 2016;76(14 Suppl):Abstract nr 873). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | Selinexor | Phase I | Actionable | In a Phase I clinical trial, Selinexor (KPT-330) treatment resulted in inhibition of tumor growth in 3/5 patients with platinum-refractory ovarian cancer, with one patient achieving a partial response and two patients achieving stable disease (PMID: 27649553). | 27649553 | |
| Unknown unknown | esophageal cancer | not applicable | Capmatinib | Case Reports/Case Series | Actionable | In a Phase I trial, Tabrecta (capmatinib) demonstrated safety and preliminary efficacy, resulted in stable disease as best overall response in a patients with esophageal cancer (PMID: 30724423). | 30724423 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Sintilimab | Phase I | Actionable | In a Phase I trial, Sintilimab (IBI308) demonstrated safety and preliminary efficacy, resulted in partial response in 18% (2/11) and stable disease in 18% (2/11) of patients with advanced solid tumors (J Clin Oncol 36, 2018 (suppl; abstr e15125)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MEDI0680 | Phase I | Actionable | In a Phase I trial, treatment with MEDI0680 (AMP-514) was well-tolerated and demonstrated safety, and resulted in a clinical response in 14% (8/58) of patients with advanced solid tumors, including 5 patients with kidney cancer, with one complete response, and 3 patients with melanoma (PMID: 31439037). | 31439037 | |
| Unknown unknown | liver cancer | not applicable | Oxaliplatin + Rigosertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, human liver cancer cells demonstrated a complete tumor response in cell line xenograft models when treated with a combination of Rigosertib (ON01910) and Eloxatin (oxaliplatin) (PMID: 15766665). | 15766665 | |
| Unknown unknown | gastrointestinal system cancer | not applicable | Axitinib + Fluorouracil + Leucovorin + Oxaliplatin | Phase I | Actionable | In a Phase I trial, Inlyta (axitinib), in combination with FOLFOX, demonstrated safety and some efficacy in patients with gastrointestinal tumors (PMID: 24423921). | 24423921 | |
| Unknown unknown | renal cell carcinoma | not applicable | Toripalimab | Phase I | Actionable | In a Phase I trial, Toripalimab (JS001) demonstrated safety and preliminary efficacy, resulted in an objective response rate of 33.3% (2/6) and a disease control rate of 50.0% (3/6, 2 partial response, 1 stable disease) in patients with advanced renal cell carcinoma (PMID: 30642373; NCT02836795). | 30642373 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | BOS172722 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, triple-negative breast cancer (TNBC) cell lines demonstrated increased sensitivity to BOS172722 compared to non-triple-negative breast cancer cell lines in culture, and BOS172722 treatment resulted in moderate tumor growth inhibition in a TNBC cell line xenograft model (PMID: 31575759). | 31575759 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Alisertib + Paclitaxel | Phase II | Actionable | In a Phase II trial, the combination treatment of Alisertib (MLN8237) and Taxol (paclitaxel) compared to Taxol (paclitaxel) plus placebo resulted in a progression-free survival of 3.32 months versus 2.17 months (P=0.038), and led to an overall response rate of 22% (20/89) versus 18% (16/289), and a disease control rate of 58% (52/89) versus 46% (41/89), respectively, in patients with relapsed or refractory small cell lung cancer (PMID: 31655296; NCT02038647). | 31655296 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Alisertib + Paclitaxel | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, Alisertib (MLN8237), alone and in combination with Taxol (paclitaxel), inhibited growth of small cell lung cancer cell (SCLC) lines in culture and inhibited tumor growth in primary human SCLC xenograft models (Mol Cancer Ther 2013;12(11 Suppl):A282). | detail... | |
| Unknown unknown | myelodysplastic syndrome | not applicable | Guadecitabine | Phase I | Actionable | In a Phase I trial, SGI-110 treatment resulted in clinical response in 32% (6/19) of patients with myelodysplastic syndrome (PMID: 26296954). | 26296954 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | NanoHHI | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with NanoHHI decreased growth and migration of hepatocellular carcinoma cells in culture, and inhibited tumor growth and metastasis in hepatocellular carcinoma cell line xenograft models (PMID: 21868763). | 21868763 | |
| Unknown unknown | breast cancer | not applicable | Tanibirumab | Preclinical - Cell culture | Actionable | In a preclinical study, Tanibirumab (TTAC-0001) inhibited angiogenesis in a cell culture assay with human breast cancer cells (PMID: 26325365). | 26325365 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BI 894999 | Phase I | Actionable | In a Phase I trial, treatment with BI 894999 resulted in stable disease in one patient and a partial response in three patients of 27 evaluable patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2504)). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Alisertib | Phase II | Actionable | In a Phase II trial, Alisertib (MLN8237) demonstrated safety and some anti-tumor activity across a range of advanced solid tumor types, with an objective response rate of 4% (1/23) in patients with non-small cell lung cancer (PMID: 25728526). | 25728526 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MP0250 | Phase I | Actionable | In a Phase I trial, MP0250 demonstrated safety and resulted in one unconfirmed partial response in a patient with metastatic anal cancer, stable disease in 60% (24/40) of patients, median progression-free survival of 15.1 weeks, and measurable tumor shrinkage in 36% (14/39) of evaluable patients with heavily pretreated advanced solid tumors (PMID: 33301375; NCT02194426). | 33301375 | |
| Unknown unknown | lymphoma | not applicable | Aldoxorubicin + Metformin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Glucophage (metformin) and Aldoxorubicin inhibited cell growth in human lymphoma cell lines in culture and inhibited tumor growth in xenograft models (PMID: 22378068). | 22378068 | |
| Unknown unknown | melanoma | not applicable | DT01 + Radiotherapy | Phase I | Actionable | In a Phase I trial, DT01 treatment enhanced the sensitivity of radiotherapy, resulting in an objective response in 59% (45/76) of lesions from 21 melanoma patients, including 23 complete responses (PMID: 27140316; NCT01469455). | 27140316 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Cytarabine + RN-1 | Preclinical - Cell culture | Actionable | In a preclinical study, RN-1 and Cytosar-U (cytarabine) demonstrated synergy in growth inhibition of several acute myeloid leukemia cell lines in culture (PMID: 26837761). | 26837761 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Ganetespib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, small cell lung carcinoma cell line xenograft models treated with Ganetespib demonstrated partial tumor growth inhibition, but with tumor progression, weight loss ensued (PMID: 27267850). | 27267850 | |
| Unknown unknown | esophageal cancer | no benefit | Cetuximab + Cisplatin + Paclitaxel + Radiotherapy | Phase III | Actionable | In a Phase III trial, the addition of Erbitux (cetuximab) to therapy with Taxol (paclitaxel), Platinol (cisplatin), and radiotherapy did not demonstrate increased clinical benefit over Taxol (paclitaxel) and Platinol (cisplatin) plus radiotherapy in esophageal cancer patients, with the experimental and control groups demonstrating similar median overall survival (19.7 mo vs. 19 mo, respectively (PMID: 28687830; NCT00566852). | 28687830 | |
| Unknown unknown | liposarcoma | not applicable | Pazopanib | Clinical Study | Actionable | In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 8 weeks and median survival of 7.3 months in patients with liposarcoma (PMID: 26970174). | 26970174 | |
| Unknown unknown | lung carcinoma | not applicable | Fostamatinib | Preclinical - Cell culture | Actionable | In a preclinical study, Fostamatinib (R788) treatment induced cytotoxicity in Lewis lung carcinoma cells in culture and inhibited tumor growth in a syngeneic mouse model (PMID: 31974273). | 31974273 | |
| Unknown unknown | fibrillary astrocytoma | not applicable | CC-90010 | Case Reports/Case Series | Actionable | In a Phase I trial, CC-90010 treatment demonstrated safety, and resulted in a complete response in a patient with IDH-mutant diffuse astrocytoma with MGMT methylation (PMID: 32240793; NCT03220347). | 32240793 | |
| Unknown unknown | multiple myeloma | not applicable | Bortezomib + Daratumumab + Dexamethasone + Thalidomide | FDA approved | Actionable | In a Phase III trial (CASSIOPEIA) that supported FDA approval, Darzalex (daratumumab) in combination with Velcade (bortezomib), thalidomide, and dexamethasone (VTd) resulted in improved stringent complete response at 100 days post autologous stem-cell transplantation compared to VTd (29%, 157/543 vs 20%, 110/542, OR=1.60, p=0.0010) in patients with newly diagnosed multiple myeloma (PMID: 31171419; NCT02541383). | detail... 31171419 | |
| Unknown unknown | adenoid cystic carcinoma | not applicable | Dovitinib | Phase II | Actionable | In a Phase II trial, Dovitinib (TKI258) treatment was tolerated and demonstrated limited clinical activity in patients with adenoid cystic carcinoma, resulting in partial response in 5.9% (2/34) of patients, suppression of overall tumor growth rate, and a median progression-free survival of 8.2 months (PMID: 28377480). | 28377480 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | AZD1208 | Phase I | Actionable | In a Phase I trial, AZD1208 treatment was tolerated and demonstrated activity in pharmacodynamic studies, resulted in stable disease for 6 weeks or longer as best objective response in 48% (13/27) of evaluable patients with advanced solid tumor (PMID: 29765150; NCT01588548). | 29765150 | |
| Unknown unknown | glioblastoma | not applicable | Tepotinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Tepmetko (tepotinib) induced tumor regression in mouse cell line xenograft models of glioblastoma (PMID: 23553846). | 23553846 | |
| Unknown unknown | skin melanoma | not applicable | Toripalimab | Phase II | Actionable | In a Phase II trial (POLARIS-01), Toripalimab (JS001) resulted in an objective response rate of 17.3% (22/127, 1 CR, 21 PR) and a disease control rate of 57.5% in melanoma patients, with a median progression-free survival (mPFS) of 3.6 mo and a median overall survival (mOS) of 22.2 mo, better ORR (31.0%, 14.0%, 0%), mPFS (5.5, 3.2, 1.9 mo), and mOS (not reached, 16.9, 10.3 mo) were observed in non-acral cutaneous melanoma (n=29) than in acral (n=50) and mucosal (n=22) subtypes (PMID: 32321714; NCT03013101). | 32321714 | |
| Unknown unknown | invasive bladder transitional cell carcinoma | no benefit | Atezolizumab | Phase III | Actionable | In a Phase III trial (IMvigor010). adjuvant Tecentriq (atezolizumab) did not improve disease-free survival (HR=0.89, p=0.2446) or overall survival (HR=0.85, p=0.1951) compared to observation in patients with high-risk muscle-invasive urothelial carcinoma, and resulted in more treatment discontinuation due to adverse effects than previous studies (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 5000-5000; NCT02450331). | detail... | |
| Unknown unknown | prostate cancer | not applicable | NSC156529 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, NSC156529 inhibited growth of human prostate cancer cell lines in culture, induced expression of differentiation markers and inhibited tumor growth in cell line xenograft models (PMID: 26294745). | 26294745 | |
| Unknown unknown | brain stem glioma | not applicable | MRK-003 + Radiotherapy | Preclinical - Cell culture | Actionable | In a preclinical study, MRK-003 enhanced sensitivity of diffuse pontine glioma cell lines to radiotherapy in culture, resulting in increased apoptosis (PMID: 26115193). | 26115193 | |
| Unknown unknown | neuroblastoma | not applicable | AMXT1501 + Eflornithine | Preclinical - Cell culture | Actionable | In a preclinical study, AMXT1501 and Eflornithine (DFMO) combination treatment resulted in depletion of intracellular polyamine pools and decreased intracellular ATP, which led to cell cycle arrest and apoptosis in neuroblastoma cell lines in culture (PMID: 23457004). | 23457004 | |
| Unknown unknown | fallopian tube cancer | not applicable | ENMD-2076 | Phase II | Actionable | In a Phase II clinical trial, ENMD-2076 demonstrated efficacy in patients with recurrent, platinum-resistant ovarian, fallopian tube or peritoneal cancers (PMID: 22921155). | 22921155 | |
| Unknown unknown | thyroid gland carcinoma | not applicable | MLN8054 | Preclinical - Patient cell culture | Actionable | In a preclinical study, MLN8054 inhibited growth of tumor cells derived from a patient with anaplastic thyroid carcinoma (PMID: 27379749). | 27379749 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Erlotinib + Ethacridine | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Tarceva (erlotinib) and the PARG inhibitor ethacridine demonstrated synergy in decreasing viability of acute myeloid leukemia (AML) cell lines and primary samples in culture, and reduced tumor growth in AML cell line xenograft models (PMID: 27587383). | 27587383 | |
| Unknown unknown | glioblastoma | not applicable | G-TPP + Obatoclax | Preclinical - Patient cell culture | Actionable | In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Obatoclax (GX015-070) synergistically inhibited viability of established lines and patient-derived glioblastoma cells in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | prostate cancer | no benefit | Abiraterone + Dactolisib | Phase I | Actionable | In a Phase Ib trial, the combination of Zytiga (abiraterone) and Dactolisib (BEZ235) did not demonstrate positive safety and tolerability, and further study of this combination is not planned (PMID: 28282611; NCT01634061). | 28282611 | |
| Unknown unknown | lung cancer | not applicable | CAB-AXL-ADC | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CAB-AXL-ADC treatment inhibited tumor growth in a lung cancer cell line xenograft model (Cancer Res 2018;78(13 Suppl):Abstract nr 827). | detail... | |
| Unknown unknown | clear cell sarcoma | not applicable | TAS4464 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TAS4464 inhibited growth and induced apoptosis in clear cell carcinoma cell lines in culture, resulted in tumor suppression in cell line xenograft models (Cancer Res 2016;76(14 Suppl):Abstract nr 3777). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Marizomib | Phase I | Actionable | In a Phase I trial, Marizomib (NPI-0052) treatment resulted in stable disease of short duration in 29% (7/24) of patients with advanced solid tumors (PMID: 27117181). | 27117181 | |
| Unknown unknown | hematologic cancer | no benefit | OPB-51602 | Phase I | Actionable | In a Phase I trial, OPB-51602 treatment resulted in no objective response and stable disease in 15% (3/20) of patients with hematological malignancies (PMID: 25912076). | 25912076 | |
| Unknown unknown | synovial sarcoma | not applicable | Pembrolizumab | Phase II | Actionable | In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 10% (1/10) of patients with synovial sarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). | detail... | |
| Unknown unknown | mantle cell lymphoma | not applicable | Mivavotinib | Case Reports/Case Series | Actionable | In a Phase I trial, Mivavotinib (TAK-659) treatment resulted in an objective response rate of 33% (1/3, 1 partial responses) in patients with relapsed or refractory mantle cell lymphoma, with a median duration of response of 109 days (PMID: 32327472; NCT02000934). | 32327472 | |
| Unknown unknown | ovarian cancer | not applicable | SNS-032 + unspecified PD-1 antibody | Preclinical | Actionable | In a preclinical study, the addition of SNS-032 resulted in sensitization to anti-PD-1 therapy in a syngeneic mouse ovarian cancer model, resulting in increased immune response and decreased tumor burden (PMID: 30454645). | 30454645 | |
| Unknown unknown | colon cancer | not applicable | Quisinostat | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Quisinostat (JNJ-26481585) induced apoptosis and inhibited proliferation of colon cancer cell lines in culture, and inhibited tumor growth in a colon cancer cell line xenograft model (PMID: 19861438). | 19861438 | |
| Unknown unknown | glioblastoma | not applicable | G-TPP + Navitoclax | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Navitoclax (ABT-263) synergistically inhibited viability of established lines and patient-derived glioblastoma cells in culture, and inhibited tumor growth in cell line and patient-derived xenograft models (PMID: 28522750). | 28522750 | |
| Unknown unknown | lymphoma | not applicable | OKI-005 | Preclinical - Cell culture | Actionable | In a preclinical study, OKI-005 induced cell cycle arrest and apoptosis in lymphoma cells in culture (PMID: 31235619). | 31235619 | |
| Unknown unknown | pancreatic cancer | not applicable | RX-3117 | Phase Ib/II | Actionable | In a Phase I/II trial, RX-3117 demonstrated safety and preliminary efficacy, resulted in durable stable disease in 25% (2/8) of patients with pancreatic cancer (Journal of Clinical Oncology 35, no. 4_suppl (February 1 2017) 445-445; NCT02030067). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Sabatolimab | Phase Ib/II | Actionable | In a Phase Ib/II trial, MBG453 treatment resulted in stable disease in 29% (25/87) of patients with advanced solid tumors (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). | detail... | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Cetuximab + Pazopanib | Phase Ib/II | Actionable | In a Phase Ib clinical trial, combined therapy, Votrient (pazopanib) and Erbitux (cetuximab), was well tolerated in head and neck squamous cell carcinoma patients with metastatic or resistant disease, and resulted in a 6% (2/31) complete response rate, a 29% (9/31) partial response rate, a median time to progression of 5.5 months, and a median overall survival of 9.5 months (PMID: 30001987; NCT01716416). | 30001987 | |
| Unknown unknown | non-Hodgkin lymphoma | not applicable | Betalutin | Phase Ib/II | Actionable | In a Phase I/II trial, Betalutin (177Lu Lilotomab Satetraxetan) treatment in patients with non-Hodgkin lymphoma (follicular lymphoma n=57, mantle cell lymphoma n=7, marginal zone lymphoma n=9, small lymphocytic lymphoma n=1) resulted in an overall response rate of 61% (45/74, 22 complete responses, 23 partial responses, 14 stable disease), with a median duration of response of 13.6 months, and a median progression-free survival of 8.8 months (PMID: 32877524; NCT01796171). | 32877524 | |
| Unknown unknown | melanoma | not applicable | SKI-G-801 | Preclinical | Actionable | In a preclinical study, SKI-G-801 treatment reduced tumor burden in a metastatic mouse model of melanoma (Cancer Res 2019;79(13 Suppl):Abstract nr 2010). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | RO7122290 | Phase I | Actionable | In a Phase I trial, RO7122290 treatment demonstrated acceptable safety profile, induced anti-tumor immune response in tumor samples, and resulted in an objective response rate of 3.6% (2/62) in patients with advanced solid tumors (Ann Oncol Sep 2020, 31 (Suppl 4), S707; EUDRACT 2017-003961-83). | detail... | |
| Unknown unknown | renal cell carcinoma | not applicable | Everolimus + Vorolanib | Phase I | Actionable | In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in partial response in 2 patients and stable disease in 1 patient with renal cell carcinoma (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). | detail... | |
| Unknown unknown | malignant pleural mesothelioma | not applicable | Obatoclax + Trabectedin | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Yondelis (trabectedin) and Obatoclax (GX015-070) demonstrated synergy in inducing apoptosis and decreasing viability of malignant pleural mesothelioma cell lines in culture (PMID: 27512118). | 27512118 | |
| Unknown unknown | fibrosarcoma | not applicable | 23814 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with 23814 resulted in partial inhibition of tumor growth in a fibrosarcoma cell line xenograft model (PMID: 25995436). | 25995436 | |
| Unknown unknown | hematologic cancer | not applicable | Cenisertib | Phase I | Actionable | In a Phase I clinical trial, Cenisertib (AS703569) demonstrated safety and preliminary efficacy in patients with advanced hematological malignancies (Blood 2008 112:2963). | detail... | |
| Unknown unknown | prostate cancer | not applicable | SM88 | Phase II | Actionable | In a Phase II trial, SM-88 treatment resulted in stable (4/10) or rising (5/10) testosterone levels, and none of the toxicity commonly associated with androgen deprivation therapy in patients with non-metastatic recurrent prostate cancer (J Clin Oncol 36, 2018 (suppl 6S; abstr 175); NCT02796898). | detail... | |
| Unknown unknown | renal carcinoma | not applicable | MRx0518 | Preclinical | Actionable | In a preclinical study, MRx0518 stimulated immune response and reduced tumor size in syngeneic mouse models of renal carcinoma (Journal of Clinical Oncology 36, no. 15_suppl). | detail... | |
| Unknown unknown | chordoma | not applicable | Dasatinib | Phase II | Actionable | In a Phase II trial, patients with chordoma demonstrated a median progression free survival of 6.3 months and six patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). | 27696380 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | 7-hydroxystaurosporine + Sirolimus | Preclinical | Actionable | In a preclinical study, Rapamune (sirolimus), in combination with UCN-01 (sc-3510), resulted in apoptosis and cell cycle arrest in diffuse large B-cell lymphoma cells in culture (PMID: 19223503). | 19223503 | |
| Unknown unknown | colorectal cancer | not applicable | Cabozantinib + Chloroquine | Preclinical - Cell culture | Actionable | In a preclinical study, Cometriq (Cabometyx, cabozantinib) treatment in combination with Chloroquine enhanced apoptosis in a colorectal cancer cell line in culture (PMID: 30026382). | 30026382 | |
| Unknown unknown | multiple myeloma | not applicable | Carfilzomib + Dexamethasone + Isatuximab | FDA approved | Actionable | In a Phase III trial (IKEMA) that supported FDA approval, addition of Sarclisa (isatuximab-irfc) to the combination of Kyprolis (carfilzomib) and dexamethasone improved progression-free survival (not reached vs 19.15 mo, HR 0.531, p=0.0007) in patients with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy (62nd ASH Annual Meeting and Exposition Dec 2020, Abstract 2316; NCT03275285). | detail... detail... | |
| Unknown unknown | lung squamous cell carcinoma | not applicable | Carboplatin + Lumretuzumab + Paclitaxel | Phase Ib/II | Actionable | In a Phase Ib/II trial, treatment with the combination of Lumretuzumab (RO5479599), Taxol (paclitaxel), and Paraplatin (carboplatin) in squamous non-small cell lung cancer patients (N=12) resulted in an objective response rate of 25% (3/12) and a disease control rate of 91.7% (11/12), including a partial response in 3 patients and stable disease in 8 patients as a best response (PMID: 31423336; NCT02204345). | 31423336 | |
| Unknown unknown | colorectal cancer | not applicable | Refametinib + Sorafenib | Phase I | Actionable | In a Phase I trial, a patient with colorectal cancer demonstrated a durable partial response for 358 days when treated with the combination of Refametinib (BAY86-9766) and Nexavar (sorafenib) (PMID: 26644411). | 26644411 | |
| Unknown unknown | multiple myeloma | not applicable | Pomalidomide + SJB3-019A | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of SJB3-019A and Pomalyst (pomalidomide) worked synergistically to induce cytotoxicity in multiple myeloma cell lines and primary multiple myeloma cells in culture (PMID: 28270494). | 28270494 | |
| Unknown unknown | Indication other than cancer | not applicable | Sirolimus | FDA approved | Actionable | Rapamune (sirolimus) is FDA approved for use in patients with lymphangioleiomyomatosis and for prophylactic immunosuppression in renal transplant patients (FDA.gov). | detail... detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | TMU-35435 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TMU-35435 induced cell-cycle arrest and apoptosis and decreased viability of non-small cell lung cancer cell lines in culture, and inhibited tumor growth in a lung cancer cell line xenograft model (PMID: 28233309). | 28233309 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Fingolimod + Sorafenib | Preclinical | Actionable | In a preclinical study, Gilenya (fingolimod) worked synergistically with Nexavar (sorafenib) to inhibit growth and induce apoptosis in hepatocellular carcinoma cell lines in culture (PMID: 26516583). | 26516583 | |
| Unknown unknown | endometrial carcinoma | not applicable | ABTL0812 | Case Reports/Case Series | Actionable | In a Phase I/Ib trial, ABTL0812 treatment resulted in stable disease lasted for 59 weeks in a patient with endometrioid endometrial cancer (PMID: 33588149; NCT02201823). | 33588149 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | RO6839921 | Phase I | Actionable | In a Phase I trial, treatment with RO6839921 was well tolerated, and resulted in a clinical benefit rate of 41% (17/41), with no responses and stable disease in 41% (17/41) of patients with advanced solid tumors (PMID: 31734832; NCT02098967). | 31734832 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Birabresib | Preclinical - Cell culture | Actionable | In a preclinical study, triple-receptor negative breast cancer cells demonstrated sensitivity to treatment with Birabresib (OTX015), resulting in decreased cell proliferation in culture (PMID: 27256375). | 27256375 | |
| Unknown unknown | breast cancer | not applicable | AZD7648 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AZD7648 treatment inhibited tumor growth in a cell line xenograft model of breast cancer, however, with decreased response compared to AZD7648 and Doxil (pegylated liposomal-doxorubicin) combination treatment (PMID: 31699977). | 31699977 | |
| Unknown unknown | multiple myeloma | not applicable | Dexamethasone + Evofosfamide | Phase Ib/II | Actionable | In a Phase I/II trial, Evofosfamide (TH-302) therapy plus Adexone (dexamethasone) was well tolerated in patients with relapsed/refractory multiple myeloma and resulted in a disease control rate of 83.9% (26/31, stable disease or better), 3.3% (1/31) very good partial responses and 9.7% (3/31) partial responses, a 40% 6-month progression-free survival rate, a median progression-free survival of 4.4 months, and a median overall survival of 12.8 months (PMID: 30279233; NCT01522872). | 30279233 | |
| Unknown unknown | colorectal cancer | not applicable | CHIR99021 + Olaparib | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with CHIR99021 sensitized colorectal cancer cell lines with either BRCA1/2 proficiency or BRCA2 deficiency to Lynparza (olaparib) in culture, resulting in a synergistic effect (PMID: 33589588). | 33589588 | |
| Unknown unknown | multiple myeloma | not applicable | Cyclophosphamide + LCL161 | Phase II | Actionable | In a Phase II trial, LCL161 treatment followed by Cytoxan (cyclophosphamide) resulted in complete response in 4% (1/25), partial response in 12% (3/25), and molecular response in 4% (1/25) of multiple myeloma patients (PMID: 27841872). | 27841872 | |
| Unknown unknown | breast cancer | not applicable | Cyclophosphamide + Methotrexate + Vandetanib | Phase I | Actionable | In a Phase I study, Caprelsa (vandetanib), in combination with Cytoxan (cyclophosphamide) and Abitrexate (methotrexate), demonstrated safety and some efficacy in breast cancer patients (PMID: 23001754). | 23001754 | |
| Unknown unknown | anaplastic large cell lymphoma | not applicable | Brentuximab vedotin | FDA approved | Actionable | In a Phase II trial (SGN-35) that supported FDA approval, Adcetris (brentuximab vedotin) treatment resulted in complete remission in 57% (33/58), partial remission in 29% (17/58) of patients with relapsed or refractory systemic anaplastic large cell lymphoma (PMID: 22614995; NCT00866047). | 22614995 detail... | |
| Unknown unknown | esophagus adenocarcinoma | not applicable | Fluorouracil + Leucovorin + Nivolumab + Oxaliplatin | FDA approved | Actionable | In a Phase III trial (CHECKMATE-649) that supported FDA approval, Opdivo (nivolumab) in combination with chemotherapy (mFOLFOX6 or CapeOx) significantly improved overall survival (13.8 vs 11.6 months, HR 0.80, p=0.0002) compared to chemotherapy alone in patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma (Ann Oncol Sep 2020, 31 (Suppl 4), S1191; NCT02872116). | detail... detail... | |
| Unknown unknown | prostate cancer | not applicable | ARV-110 | Phase I | Actionable | In a Phase I trial, ARV-110 demonstrated safety and preliminary efficacy, resulted in an over 50% decline of PSA in 13.3% (2/15) of patients with metastatic castrate-resistant prostate cancer (J Clin Oncol 38: 2020 (suppl; abstr 3500); NCT03888612). | detail... | |
| Unknown unknown | breast cancer | not applicable | TAS0612 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, breast cancer cells, including antiestrogen-resistant cells, demonstrated sensitivity to TAS0612 treatment in culture, and TAS0612 inhibited tumor growth in orthotopic breast cancer cell line xenograft models, including a triple-negative breast cancer model (PMID: 31879363). | 31879363 | |
| Unknown unknown | nasopharynx carcinoma | not applicable | Camrelizumab + Cisplatin + Gemcitabine | Phase I | Actionable | In a Phase I trial, combination of Camrelizumab (SHR-1210), Gemzar (gemcitabine), and Platinol (cisplatin) demonstrated safety and preliminary anti-tumor activity in patients with treatment-naive nasopharyngeal carcinoma, resulted in an objective response rate of 91% (20/22, 1 complete response and 19 partial responses) and a disease control of 100% (22/22, stable disease or better (PMID: 30213452; NCT03121716). | 30213452 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | AMG 820 | Phase I | Actionable | In a Phase I trial, AMG 820 treatment was well-tolerated and resulted in safety, however, demonstrated limited antitumor activity in patients with advanced solid tumors, and thus, AMG 820 therapy combined with an immunotherapy was suggested based on clinical and preclinical evidence (PMID: 28655795). | 28655795 | |
| Unknown unknown | colon carcinoma | not applicable | Mps-BAY2b + Paclitaxel | Preclinical | Actionable | In a preclinical study, Mps-BAY2b, in combination with paclitaxel, had increased efficacy in inhibiting cell proliferation of colon carcinoma cell in culture (PMID: 23933817). | 23933817 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Fluorouracil + Ipatasertib + Leucovorin + Oxaliplatin | Phase I | Actionable | In a Phase Ib trial, the combination of Ipatasertib (GDC-0068) and mFOLFOX6 demonstrated safety in patients with advanced solid tumors, and resulted in an overall response rate of 6.1% (2/33), including partial responses in two patients, stable disease in 51.5% (17/33) of patients, a six-month progression-free survival rate of 18.2% (6/33), and maximum progression-free survival duration of 50 months in a patient with appendix cancer (PMID: 32205017; NCT01362374). | 32205017 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Ublituximab + Umbralisib | Phase I | Actionable | In a Phase I trial, high-dose Ukoniq (umbralisib) in combination with Ublituximab resulted in complete response in 50% (2/4) and partial response in 25% (1/4) of patients with diffuse large B-cell lymphoma (J Clin Oncol 33, 2015 (suppl; abstr 8548)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CEP1347 | Preclinical - Cell culture | Actionable | In a preclinical study, CEP1347 treatment inhibited anchorage-dependent growth of transformed cells in culture (PMID: 12184411). | 12184411 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Gedatolisib | Phase I | Actionable | In a Phase I trial, Gedatolisib (PF-05212384) demonstrated safety and efficacy, which resulted in antitumor activity and stable disease greater than six months in 10.4% (8/27) of patients with solid malignant tumors (PMID: 25652454). | 25652454 | |
| Unknown unknown | breast cancer | not applicable | OBI-999 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, OBI-999 treatment inhibited tumor growth in a Globo H-expressing breast cancer cell line xenograft model (Cancer Res 2019;79(13 Suppl):Abstract nr 4815). | detail... | |
| Unknown unknown | breast cancer | not applicable | ZMF-10 | Preclinical - Cell culture | Actionable | In a preclinical study, ZMF-10 inhibited Pak1-Erk signaling, induced ER stress, resulted in apoptosis, growth inhibition, and inhibition of migration of breast cancer cells in culture (PMID: 32222676). | 32222676 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | LY3023414 + Prexasertib | Phase I | Actionable | In a Phase Ib trial, the combination of Samotolisib (LY3023414) and Prexasertib (LY2606368) in triple negative breast cancer (TNBC) patients resulted in an overall response rate of 25% (4/16, all partial) and a disease control rate of 62.5% (10/16), and preclinically, showed greater inhibition of cell proliferation in TNBC cell lines, tumor regression in cell line xenograft models, and synergistic and additive effects in 30/38 patient-derived xenograft (PDX) models (PMID: 33495309; NCT02124148). | 33495309 | |
| Unknown unknown | peripheral T-cell lymphoma | not applicable | Copanlisib | Phase II | Actionable | In a Phase II trial, Aliqopa (copanlisib) treatment resulted in partial response in 50% (2/4) of patients with peripheral T-cell lymphoma (PMID: 24852792). | 24852792 | |
| Unknown unknown | gastric adenocarcinoma | not applicable | Torkinib | Preclinical | Actionable | In a preclinical study, Torkinib (PP242) inhibited signaling of the PI3K/AKT/mTOR pathway and subsequent cell proliferation of gastric cancer cells in culture (PMID: 25035961). | 25035961 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ravoxertinib | Phase I | Actionable | In a Phase I trial, Ravoxertinib (GDC-0994) demonstrated safety in patients with advanced solid tumors and resulted in stable disease in 34% (16/47) of patients, and partial response in two colorectal cancer patients harboring BRAF mutations (2/15), lasting 21 and 73 weeks (PMID: 31848189; NCT01875705). | 31848189 | |
| Unknown unknown | melanoma | not applicable | GDC-0425 + Gemcitabine | Phase I | Actionable | In a Phase I trial, treatment with GDC-0425 followed by Gemzar (gemcitabine) resulted in a partial response in a patient with melanoma (PMID: 27815358). | 27815358 | |
| Unknown unknown | acute myeloid leukemia | not applicable | JSH-150 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, JSH-150 induced cell-cycle arrest and inhibited proliferation of acute myeloid leukemia cell lines in culture, and inhibited tumor progression in xenograft models (PMID: 30253346). | 30253346 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Docetaxel + Nintedanib | Phase III | Actionable | In a Phase III clinical trial, the combination of Ofev (nintedanib) and Taxotere (docetaxel) resulted in improved progression-free survival and overall survival compared to placebo plus Taxotere (docetaxel) in non-small cell lung cancer patients (PMID: 24411639). | 24411639 | |
| Unknown unknown | melanoma | not applicable | VLX600 | Preclinical - Cell culture | Actionable | In a preclinical study, VLX600 treatment inhibited proliferation of a mouse melanoma cell line in culture (PMID: 24548894). | 24548894 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Vandetanib | Phase I | Actionable | In a Phase I trial, treatment with Caprelsa (vandetanib) resulted in stable disease in 40% (31/77) of patients with advanced solid tumors (PMID: 15905307). | 15905307 | |
| Unknown unknown | colorectal cancer | not applicable | LY2090314 + Rucaparib | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with LY2090314 sensitized colorectal cancer cell lines with either BRCA1/2 proficiency or BRCA2 deficiency to Rubraca (rucaparib) in culture, resulting in a synergistic effect (PMID: 33589588). | 33589588 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Hu8F4 | Preclinical - Pdx | Actionable | In a preclinical study, Hu8F4 treatment reduced tumor burden and prolonged survival in patient-derived xenograft models of acute myeloid leukemia (PMID: 27055866). | 27055866 | |
| Unknown unknown | multiple myeloma | not applicable | AMG 420 | Phase I | Actionable | In a Phase I trial, AMG 420 treatment resulted in an overall objective response rate (ORR) of 31% (13/42) in heavily pretreated multiple myeloma patients, including a minimum residual disease (MRD)-negative complete response (CR) in 6, a CR in an additional 3, and a partial response (PR) in 4, and at the maximum-tolerated dose, resulted in an objective response rate of 70% (7/10) with a MRD-negative CR in 5 and a PR in 2, and a median duration of response of 9 months (PMID: 31895611; NCT02514239). | 31895611 | |
| Unknown unknown | myeloid neoplasm | not applicable | Bortezomib + Dexamethasone + Lenalidomide | Phase III | Actionable | In a Phase III trial, Velcade (bortezomib) in combination with Revlimid (lenalidomide) and dexamethasone resulted in an improved median progression-free survival of 43 months compared to 30 months with Revlimid (lenalidomide) plus dexamethasone, and a response rate of 81.5% (176/216) compared to 71.5% (153/214) with Revlimid (lenalidomide) plus dexamethasone in myeloma patients (PMID: 28017406). | 28017406 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Sunitinib | Phase III | Actionable | In a Phase III trial, Sutent (sunitinib) as maintenance therapy resulted in improved progression free survival (4.3 vs 2.6 months) but not overall survival (11.7 vs 12.1 months) compared to placebo in patients with stage IIIB/IV non-small cell lung cancer (PMID: 28161554). | 28161554 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Sunitinib | Phase II | Actionable | In a Phase II trial, Sutent (sunitinib) treatment in non-small cell lung cancer patients resulted in an objective response rate of 11.1% (7/63), stable disease in 28.6% (18/63), and a PFS of 12 weeks and OS of 23.4 weeks (PMID: 18235126). | 18235126 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Sunitinib | Phase II | Actionable | In a Phase II trial, Sutent (sunitinib) treatment resulted in an objective response rate of 8% (1/13, partial response) and stable disease as the best response in 69% (9/13) of non-small cell lung carcinoma patients (PMID: 32312893; NCT01829217). | 32312893 | |
| Unknown unknown | melanoma | no benefit | Alvocidib | Phase II | Actionable | In a Phase II trial, Alvocidib (flavopiridol) treatment resulted in stable disease in 44% (7/16) of patients with melanoma, but no objective response (PMID: 15122079). | 15122079 | |
| Unknown unknown | breast cancer | not applicable | CDD111 | Preclinical | Actionable | In a preclinical study, CDD111 did not inhibit growth of a mouse breast cancer cell line in culture, however, reduced metastatic tumor growth in bone and visceral organs and increased survival in a mouse breast cancer cell line metastasis model (PMID: 30093561). | 30093561 | |
| Unknown unknown | breast cancer | no benefit | Imatinib | Phase II | Actionable | In a Phase II trial, Gleevec (imatinib mesylate) treatment demonstrated significant toxicity and no clinical benefit in patients with heavily pre-treated metastatic breast cancer, of the 13 tested patients, one was positive for Kit and 4 were positive for Pdgfr (PMID: 15803362). | 15803362 | |
| Unknown unknown | ovarian cancer | not applicable | Capivasertib + Olaparib | Phase I | Actionable | In a Phase I trial, the combination of AZD5363 and Lynparza (olaparib) was well-tolerated and demonstrated preliminary activity in patients with endometrial, ovarian, or triple-negative breast cancer (TNBC), with an overall response rate of 24% (7/30; all partial responses, 1 ovarian, 4 endometrial, and 2 TNBC) and stable disease for greater than 4 months in 6 additional patients (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #391P; NCT02208375). | detail... | |
| Unknown unknown | liposarcoma | not applicable | Trabectedin | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Yondelis (trabectedin) treatment resulted in an improved median progression-free survival of 4.2 months versus 1.5 months with Deticene (dacarbazine) in patients with unresectable or metastatic liposarcoma or leiomyosarcoma (PMID: 28774898; NCT01343277). | 28774898 detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Durvalumab + LY2510924 | Phase I | Actionable | In a Phase I trial, the combination of Imfinzi (durvalumab) and LY2510924 demonstrated safety and resulted in stable disease in 44.4% (4/9) of patients with advanced solid tumors, including one patient demonstrating an unconfirmed partial response (PMID: 32219196; NCT02737072). | 32219196 | |
| Unknown unknown | high grade glioma | not applicable | NEO214 | Preclinical | Actionable | In a preclinical study, NEO214 induced apoptosis and inhibited growth of glioma cell lines in culture, including cell lines resistant to Temodar (temozolmide), and inhibited tumor growth and improved survival in a mouse glioma model (PMID: 30647121). | 30647121 | |
| Unknown unknown | gastric adenocarcinoma | no benefit | Oxaliplatin + Ramucirumab + Tegafur-gimeracil-oteracil Potassium | Phase II | Actionable | In a Phase II trial, adding Cyramza (ramucirumab) to chemotherapy consisting of TS-1 and Eloxatin (oxaliplatin) did not improve progression-free survival (6.34 vs 6.74 months), objective response rate (58.2% vs 50.%), and disease control rate (90.9% vs 87.0%) compared to placebo in patients with advanced gastric or gastroesophageal junction adenocarcinoma (J Clin Oncol 36, 2018 (suppl; abstr 4036); NCT02539225). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | PF-04691502 | Phase I | Actionable | In a Phase I trial, PF-04691502 demonstrated safety and efficacy in patients with advanced solid tumors (PMID: 24395457). | 24395457 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Alpelisib + Cisplatin + Radiotherapy | Phase I | Actionable | In a Phase I trial, Piqray (Alpelisib) treatment in combination with Platinol (cisplatin) and radiotherapy demonstrated manageable safety, and resulted in an objective response rate of 66.7% (6/9) in head and neck squamous cell carcinoma patients, the 3-year progression-free survival (PFS) and overall survival (OS) were 77.8%, and the median progression-free survival and overall survival were not reached (PMID: 32464516; NCT02537223). | 32464516 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | ONC201 + Paclitaxel | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of ONC201 and Taxol (paclitaxel) worked synergistically to inhibit viability of triple-negative breast cancer cell lines in culture (PMID: 28424227). | 28424227 | |
| Unknown unknown | thyroid gland cancer | not applicable | ABT-737 | Preclinical | Actionable | In a preclinical study, ABT-737 inhibited growth and induced cell death of human thyroid cancer cell lines in culture (PMID: 27042160). | 27042160 | |
| Unknown unknown | acute myeloid leukemia | not applicable | ENMD-2076 | Phase I | Actionable | In a Phase I trial, treatment with ENMD-2076 resulted in a 25% (4/16) overall response rate in patients with acute myeloid leukemia as well as three patients achieving a complete response (with incomplete count recovery) and one patient with a morphological leukemia free state (PMID: 27406088). | 27406088 | |
| Unknown unknown | ovarian cancer | not applicable | Enoticumab | Phase I | Actionable | In a Phase I study, Enoticumab (REGN421) demonstrated safety and preliminary efficacy in ovarian cancer patients (PMID: 25724527). | 25724527 | |
| Unknown unknown | colon cancer | not applicable | Panobinostat | Preclinical | Actionable | In a preclinial study, Faridak (panobinostat) enhanced the cytotoxicity of TRAIL, tumor necrosis factor-related apoptosis-inducing ligand, in colon cancer cell lines (PMID: 21965751). | 21965751 | |
| Unknown unknown | cervical cancer | not applicable | TG6002 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with TG6002 led to decreased survival of cervical cancer cells in culture (PMID: 31011628). | 31011628 | |
| Unknown unknown | mast-cell leukemia | not applicable | Midostaurin | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Rydapt (midostaurin) treatment resulted in an overall response rate of 60% (53/89), a median overall survival of 28.7 months, and a median progression-free survival of 14.1 months in patients with systemic mastocytosis including aggressive systemic mastocytosis, systemic mastocytosis with an associated hematologic neoplasm, and mast-cell leukemia (PMID: 27355533; NCT00782067). | detail... 27355533 | |
| Unknown unknown | glioblastoma | not applicable | AsiDNA | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA inhibited survival of glioblastoma cell lines in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | Trametinib + Uprosertib | Phase I | Actionable | In a Phase I trial, the combination of Trametinib (GSK1120212) and Uprosertib (GSK2141795) was not well-tolerated and resulted in minimal efficacy in patients with advanced solid tumors (n=126), demonstrating an objective response rate of less than 5%, with one complete response and five partial responses (PMID: 32062691). | 32062691 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Navicixizumab | Preclinical | Actionable | In a preclinical study, Navicixizumab (OMP-305B83) inhibited cell proliferation of endothelial cells in culture and demonstrated antitumor activity of multiple tumor types in vivo (Mol Cancer Ther December 2015 14; C164). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Navicixizumab | Phase I | Actionable | In a Phase I trial, Navicixizumab (OMP-305B83) demonstrated safety and preliminary efficacy in a variety of advanced solid tumor patients, including ovarian, endometrial, breast, and pancreatic (Eur J Can, Vol 69, S35). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Navicixizumab | Phase I | Actionable | In a Phase I trial, Navicixizumab (OMP-305B83) treatment resulted in partial response in 6% (4/66) and stable disease in 26% (17/66) of patients with advanced solid tumors, with 19 patients having a reduction of target lesions (PMID: 30229512). | 30229512 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | U3-1565 | Phase I | Actionable | In a Phase I trial, treatment with U3-1565 in patients with an advanced solid tumor was well-tolerated, demonstrated safety, and resulted in a clinical benefit in 19% (7/36) of patients, with one partial response and six with stable disease, and three of the patients experienced a decrease in circulating VEGFA levels compared to baseline (PMID: 30056611). | 30056611 | |
| Unknown unknown | bladder carcinoma | not applicable | AR-42 + Cisplatin | Preclinical | Actionable | In a preclinical study, AR-42 synergized with cisplatin to induce apoptosis of bladder cancer cells in xenograft models (PMID: 25748177). | 25748177 | |
| Unknown unknown | gastric adenocarcinoma | not applicable | Triptolide | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with Triptolide (C1572) induced apoptotic cell death and resulted in decreased cell viability in gastric adenocarcinoma cell lines in culture (PMID: 28192510). | 28192510 | |
| Unknown unknown | hematologic cancer | not applicable | Fenretinide | Phase I | Actionable | In a Phase I trial, Fenretinide treatment resulted in objective response in 19% (5/26) and stable disease in 27% (7/26) of patients with hematologic cancer (PMID: 28420721). | 28420721 | |
| Unknown unknown | myelofibrosis | not applicable | Fedratinib | FDA approved | Actionable | In a Phase III trial (JAKARTA) that supported FDA approval, Inrebic (fedratinib) demonstrated both clinical benefits and toxicity in myelofibrosis patients, resulting in symptom response rates at week 24 of 36% (33/91), 34% (31/91), and 7% (6/85) in the Fedratinib (SAR302503) 400 mg, 500 mg, and placebo groups, respectively (P<.001) (PMID: 26181658; NCT01437787). | 26181658 detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ganitumab | Phase I | Actionable | In a Phase I trial, Ganitumab (AMG 479) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol (Meeting Abstracts) 2007 25: 3002). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | SY-1365 + Venetoclax | Preclinical - Cell culture | Actionable | In a preclinical study, SY-1365 and Venclexta (venetoclax) synergistically induced apoptosis in acute myeloid leukemia cells in culture (Proceedings of the AACR, Vol 58, April 2017, Abstract # 1151). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | CPX-351 | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Vyxeos (CPX-351) treatment resulted in improved overall survival (9.56 vs 5.95 months, HR=0.69, p=0.005), event-free survival (HR=0.74, p=0.021), and complete response rate (47.4% vs 33.3%, p=0.016) compared to Cytosar-U (cytarabine) and Cerubidine (daunorubicin) combination therapy in patients with acute myeloid leukemia (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 7000-7000; NCT01696084). | detail... detail... | |
| Unknown unknown | multiple myeloma | not applicable | Bevacizumab + Dexamethasone + Evofosfamide | Phase Ib/II | Actionable | In a Phase I/II trial, treatment with combined Avastin (bevacizumab), Evofosfamide (TH-302) therapy, and Adexone (dexamethasone) was well tolerated in patients with relapsed/refractory multiple myeloma and resulted in a disease control rate of 78.6% (22/28, stable disease or better), 3.6% (1/28) complete and 7.1% (2/28) partial responses, a 25% 6-month progression-free survival rate, a median progression-free survival of 2.2 months, and a median overall survival of 9.0 months (PMID: 30279233; NCT01522872). | 30279233 | |
| Unknown unknown | sarcoma | not applicable | Lenvatinib | Phase I | Actionable | In a Phase I trial, Lenvima (lenvatinib) demonstrated safety and anti-tumor activity in patients with advanced solid tumors, including partial responses in patients with soft-tissue sarcoma (PMID: 22516948). | 22516948 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Azacitidine + Hu5F9-G4 | Phase I | Actionable | In a Phase Ib trial, Magrolimab (Hu5F9-G4) and Vidaza (azacitidine) combination therapy was well tolerated, and resulted in complete response in 40% (10/25), complete response with incomplete hematologic recovery in 16% (4/25), partial response in 4% (1/25), morphologic leukemia-free state in 4% (1/25), and stable disease in 32% (8/25) of patients with acute myeloid leukemia unfit for chemotherapy (J Clin Oncol 38: 2020 (suppl; abstr 7507); NCT03248479). | detail... | |
| Unknown unknown | pancreatic cancer | no benefit | Capecitabine + Cisplatin + Epirubicin + Gemcitabine + Metformin | Phase II | Actionable | In a Phase II trial, pancreatic cancer patients treated with PEXG combined with Glucophage (metformin) demonstrated a 6 month PFS of 42% (13/31), which did not differ from the control group 6 month PFS of 52% (15/29) (PMID: 26459175). | 26459175 | |
| Unknown unknown | ovarian cancer | not applicable | ETP-46464 + Radiotherapy | Preclinical - Cell culture | Actionable | In a preclinical study, ETP-46464 increased the sensitivity of ovarian cancer cell lines to ionizing radiation in culture (PMID: 25560806). | 25560806 | |
| Unknown unknown | colorectal cancer | not applicable | Panitumumab | FDA approved | Actionable | In an open-label clinical trial that supported FDA approval, Vectibix (panitumumab) treatment in patients with metastatic colorectal cancer resulted in increased progression-free survival (13.8 weeks; SD=0.76) compared to best supportive care alone (8.5 weeks; SD=0.54) (PMID: 18316547). | 18316547 detail... | |
| Unknown unknown | transitional cell carcinoma | not applicable | Sacituzumab govitecan-hziy | FDA approved | Actionable | In a Phase II trial (TROPHY-U-01) that supported FDA approval, Trodelvy (sacituzumab govitecan-hziy) treatment resulted in an objective response rate of 27% (31/113) in patients with metastatic urothelial carcinoma whose disease progressed on platinum-based regimens or immunotherapy, with a clinical benefit rate of 37%, a median duration of response, progression-free survival, and overall survival of 5.9, 5.4, 10.5 months, respectively (Ann Oncol. 2020, 31 (Suppl_4): S1142-S1215; NCT03547973). | detail... detail... | |
| Unknown unknown | B-cell lymphoma | not applicable | Tazemetostat | Phase I | Actionable | In a Phase I trial, Tazemetostat (EPZ-6438) demonstrated safety and preliminary efficacy, resulted in an objective response in 38% (8/21, 3 complete response, 5 partial response) in patients with relapsed or refractory B-cell non-Hodgkin lymphoma (PMID: 29650362; NCT01897571). | 29650362 | |
| Unknown unknown | pancreatic ductal adenocarcinoma | no benefit | Atezolizumab + BKT140 | Phase Ib/II | Actionable | In a combined analysis of 2 Phase I/II trials, Tecentriq (atezolizumab) and BL-8040 (BKT140) combination therapy demonstrated expected toxicity profile and limited efficacy, did not improve objective response rate (0/14 vs 0/15), median progression-free survival (1.6 vs 2.5 mo), or median overall survival (5.2 vs 6.8 mo) compared to control in patients with metastatic pancreatic ductal adenocarcinoma (J Clin Oncol 38, 2020 (suppl 4; abstr 712); NCT03281369, NCT03193190). | detail... | |
| Unknown unknown | follicular lymphoma | not applicable | Cerdulatinib | Phase I | Actionable | In a Phase I trial, treatment with Cerdulatinib (PRT062070) inhibited SYK and JAK/STAT signaling, and resulted in tumor response in 4 of 13 patients with follicular lymphoma (PMID: 30333224; NCT01994382). | 30333224 | |
| Unknown unknown | multiple myeloma | not applicable | BAY 1238097 | Preclinical | Actionable | In a preclinical study, BAY 1238097 inhibited tumor growth and was well tolerated in acute myeloid leukemia and multiple myeloma models (American Association for Cancer Research; 2015 Apr 18-22; Abstract nr 3524). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Carboplatin + LY2090314 + Pemetrexed Disodium | Phase I | Actionable | In a Phase I trial, LY2090314 in combination with Alimta (pemetrexed) and Paraplatin (carboplatin) resulted in partial response in 13.5% (5/37) and stable disease in 51.4% (19/37) of patients with advanced solid tumors (PMID: 26403509; NCT01287520). | 26403509 | |
| Unknown unknown | breast cancer | not applicable | Thymoquinone | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Thymoquinone (TQ) inhibited growth, migration, and invasive behavior of human breast cancer cell lines in culture, and inhibited tumor growth and metastasis in mouse breast cancer cell line xenografts (PMID: 26023736). | 26023736 | |
| Unknown unknown | colorectal cancer | no benefit | Atezolizumab + Cobimetinib | Phase III | Actionable | In a Phase III trial (IMblaze370), Tecentriq (atezolizumab) and Cotellic (cobimetinib) combination treatment did not improve median overall survival (8.87 vs 8.51 months, HR=1.00, p=0.99) compared to Stivarga (regorafenib) in patients with chemotherapy-refractory metastatic colorectal cancer, 91.7% of whom were microsatellite stable or microsatellite instability-low (PMID: 31003911; NCT02788279). | 31003911 | |
| Unknown unknown | colorectal cancer | no benefit | Atezolizumab + Cobimetinib | Phase I | Actionable | In a Phase Ib trial, Tecentriq (atezolizumab) and Cotellic (cobimetinib) combination treatment resulted in partial response in 7% (7/84) of patients with metastatic colorectal cancer, with a median duration of response of 14.8 months, a disease control rate of 31% (26/84), a median progression-free survival of 1.9 months, and a median overall survival of 10.0 months (PMID: 30918950; NCT01988896). | 30918950 | |
| Unknown unknown | prostate cancer | not applicable | AB928 + Doxorubicin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of AB928 and Adriamycin (doxorubicin) resulted in a increase in tumor cell specific CD8+ T cells and increased tumor growth inhibition compared to chemotherapy alone in prostate cancer cell line xenograft models (European Journal of Cancer, Vol 92, S14-S15). | detail... | |
| Unknown unknown | female reproductive organ cancer | not applicable | Pamiparib | Phase I | Actionable | In a Phase I trial, Pamiparib (BGB-290) treatment resulted in objective response in 43% (10/23) of patients with gynecological cancer (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 368PD; NCT02361723). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | ONC201 + Venetoclax | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of ONC201 and Venclexta (venetoclax) demonstrated synergy in acute myeloid leukemia cell lines in culture, resulting in increased induction of apoptosis (PMID: 26884599). | 26884599 | |
| Unknown unknown | colon cancer | not applicable | CBT-502 | Preclinical | Actionable | In a preclinical study, CBT-502 treatment inhibited tumor growth in a mouse model of colon cancer (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr A200). | detail... | |
| Unknown unknown | lung small cell carcinoma | not applicable | MGC018 | Case Reports/Case Series | Actionable | In a Phase I/II trial, MGC018 treatment resulted in a 6.3% decrease of target lesion in a patient with small cell lung caner (J Clin Oncol 38: 2020 (suppl; abstr 3071); NCT03729596). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | LY3039478 | Phase I | Actionable | In a Phase I trial, LY3039478 treatment resulted in 80% inhibition of plasma A-beta and more than 50% inhibition of Notch1-regulated genes in patients with advanced solid tumor, leading to partial response in 0.9% (1/110) and stable disease in 32.7% (36/110) of the patients (PMID: 30060061; NCT01695005). | 30060061 detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | LY3039478 | Phase I | Actionable | In a Phase I trial, treatment with LY3039478 was well-tolerated and demonstrated some preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 33, 2015 (suppl; abstr 2533)). | detail... | |
| Unknown unknown | renal carcinoma | not applicable | PF-06840003 | Preclinical | Actionable | In a preclinical study, PF-06840003 inhibited tumor growth in a syngeneic mouse model of renal carcinoma (PMID: 30232146). | 30232146 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Combretastatin A1 Diphosphate | Phase I | Actionable | In a Phase I study, OXi4503 demonstrated safety and preliminary efficacy in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS), resulting in one complete remission and one partial resmission among 14 patients, with four patients demonstrating stable disease (PMID: 32236943). | 32236943 | |
| Unknown unknown | prostate cancer | not applicable | AB928 + Oxaliplatin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of AB928 and Eloxatin (oxaliplatin) resulted in a increase in tumor cell specific CD8+ T cells and increased tumor growth inhibition compared to chemotherapy alone in prostate cancer cell line xenograft models (European Journal of Cancer, Vol 92, S14-S15). | detail... | |
| Unknown unknown | Hodgkin's lymphoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase I trial (CheckMate 039) that supported FDA approval, treatment with Opdivo (nivolumab) resulted in an objective response rate of 87% (20/23), with complete response in 17% (4/23) and partial response in 70% (16/23) of patients with relapsed or refractory classical Hodgkin's lymphoma (PMID: 25482239; NCT01592370). | 25482239 detail... | |
| Unknown unknown | non-Hodgkin lymphoma | not applicable | KW-2478 | Phase I | Actionable | In a Phase I clinical trial, KW-2478 demonstrated safety and preliminary efficacy in patients with non-Hodgkin lymphoma, with 100% (4/4) of patients achieving stable disease (PMID: 26695442). | 26695442 | |
| Unknown unknown | melanoma | not applicable | IMC-20D7S | Phase Ib/II | Actionable | In a Phase I/Ib trial, treatment with IMC-20D7S in patients with melanoma resulted in stable disease in 37% (10/27) and one complete response (PMID: 27797971). | 27797971 | |
| Unknown unknown | follicular lymphoma | not applicable | Copanlisib | Phase II | Actionable | In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 20% (2/10), partial response in 20% (2/10) and stable disease in 60% (6/10) of patients with follicular lymphoma (PMID: 24852792). | 24852792 | |
| Unknown unknown | follicular lymphoma | not applicable | Copanlisib | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Aliqopa (copanlisib) treatment in patients with follicular lymphoma resulted in an objective tumor response rate of 58.7% (61/104) including 14.4% (15/61) patients experiencing a complete response and 44.2% (46/61) patients experiencing a partial response, stable disease in 33.7% (35/104) of patients, and a duration of response of 370 days (Journal of Clinical Oncology 35, no. 15_suppl (May 2017) 7535-7535; NCT01660451). | detail... detail... | |
| Unknown unknown | esophagus squamous cell carcinoma | not applicable | Camrelizumab | Phase I | Actionable | In a Phase I trial, Camrelizumab (SHR-1210) treatment resulted in an objective response rate of 33.3% (10/30, 1 complete response, 9 partial response) and a disease control rate of 56.7%, with a median progression free survival of 3.6 months in patients with advanced esophageal squamous cell carcinoma (PMID: 29358502; NCT02742935). | 29358502 | |
| Unknown unknown | head and neck cancer | not applicable | NT219 + Pembrolizumab | Preclinical - Pdx | Actionable | In a preclinical study, NT219 treatment in combination with Keytruda (pembrolizumab) inhibited tumor growth and induced regression in patient-derived xenograft (PDX) models of head and neck cancer (Cancer Res 2020;80(16 Suppl):Abstract nr 5639). | detail... | |
| Unknown unknown | Her2-receptor positive breast cancer | not applicable | Abemaciclib + Trastuzumab | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, the combination of Herceptin (trastuzumab) and Abemaciclib (LY2835219) resulted in tumor growth regresssion in an ERBB2 (HER2)-receptor positive breast cancer treatment refractory patient derived xenograft model and in ERBB2 (HER2)-receptor positive cultured cells, resulted in decreased cell viability (PMID: 26977878). | 26977878 | |
| Unknown unknown | brain stem glioma | not applicable | JQ1 + MRK-003 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of MRK-003 and JQ1 resulted in increased apoptosis of diffuse pontine glioma cell lines in culture, compared to either agent alone (PMID: 26115193). | 26115193 | |
| Unknown unknown | stomach cancer | not applicable | Camrelizumab | Phase I | Actionable | In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity, resulted in partial response in 37.5% (3/8) of patients with gastric cancer (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). | detail... | |
| Unknown unknown | gastrointestinal system cancer | not applicable | Cetuximab + Cisplatin + Epirubicin + Fluorouracil | Phase II | Actionable | In a Phase II trial, Erbitux (cetuximab) in combination with ECF (epirubicin, cisplatin, and fluorouracil) resulted in an overall response rate of 60.9% (38/63), median overall survival of 11.6 months; and median progression-free survival of 7.1 months in patients with metastatic esophageal or gastroesophageal junction cancers (PMID: 27382098). | 27382098 | |
| Unknown unknown | colorectal cancer | not applicable | Bevacizumab + Fluorouracil + Irinotecan + Leucovorin | FDA approved | Actionable | In a Phase III clinical trial that supported FDA approval, the combination of Avastin (bevacizumab) and FOLFIRI chemotherapy demonstrated increased duration of overall survival and improved progression-free survival compared to FOLFIRI alone in patients with metastatic colorectal cancer (PMID: 22477726, PMID: 15175435). | 15175435 22477726 detail... | |
| Unknown unknown | prostate cancer | not applicable | Enzalutamide + Pembrolizumab | Phase II | Actionable | In a Phase II trial, Keytruda (pembrolizumab) plus Xtandi (enzalutamide) treatment resulted in >=50% reduction in prostate-specific antigen (PSA) levels in 18% (5/28) of metastatic castrate-resistant prostate cancer patients, and led to an objective response rate of 25% (3/12, all partial response), a median PSA progression-free survival of 3.8 months, and a median overall survival of 22.2 mo. in all patients and 41.7 mo. in the three responders (PMID: 32616555; NCT02312557). | 32616555 | |
| Unknown unknown | colorectal cancer | not applicable | Pegilodecakin | Phase I | Actionable | In a Phase I trial, AM0010 treatment in pancreatic cancer patients resulted in stable disease in 27% (4/15) of patients, one patient with progression free survival for greater than 6 months, and a median overall survival of 15.4 months (J Clin Oncol 34, 2016 (suppl; abstr 3082)). | detail... | |
| Unknown unknown | ovarian carcinoma | not applicable | Topotecan + Veliparib | Phase I | Actionable | In a Phase I trial, the combination of Veliparib (ABT-888) and topotecan demonstrated safety and tolerability, and resulted in an objective response rate of 9% (4/45; 1 complete response and 3 partial responses) and stable disease in 21 patients with ovarian carcinoma (PMID: 29138343; NCT01012817). | 29138343 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Volasertib | Phase I | Actionable | In a Phase I trial, Volasertib (BI 6727) demonstrated safety and limited efficacy in pediatric patients with acute leukemia or advanced solid tumors, with stable disease as best objective response in 71% (5/7) of patients with acute leukemia and in 13% (2/15) of patients with advanced solid tumors (PMID: 31276318; NCT01971476). | 31276318 | |
| Unknown unknown | colon adenocarcinoma | not applicable | SF2523 | Preclinical | Actionable | In a preclinical study, SF2523 increased T-lymphocyte infiltration and reduced tumor growth in syngeneic mouse colon adenocarcinoma models (PMID: 31018997). | 31018997 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Senexin B | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Senexin B prevented tumor growth and enhanced the effects of Adriamycin (doxorubicin) in cell line xenograft models of triple-receptor negative breast cancer (Cancer Res January 1, 2015 75; PR08). | detail... | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | BAY1217389 + Paclitaxel | Preclinical | Actionable | In a preclinical study, BAY1217389, in combination with Taxol (paclitaxel), had increased efficacy in inhibiting tumor growth in xenograft models of triple-negative breast cancer, compared to Taxol (paclitaxel) alone (PMID: 26832791). | detail... 26832791 | |
| Unknown unknown | colorectal cancer | not applicable | Flucytosine + TG6002 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with TG6002 resulted in enhanced antitumor activity when combined with Flucytosine in a colorectal cancer cell line xenograft model, demonstrating inhibition of tumor growth (PMID: 31011628). | 31011628 | |
| Unknown unknown | multiple myeloma | not applicable | Dexamethasone + Felzartamab + Lenalidomide | Phase I | Actionable | In a Phase I/IIa trial, the combination therapy of Felzartamab (MOR202), Adexone (dexamethasone), and Revlimid (lenalidomide) in patients with multiple myeloma resulted in an overall response rate of 65% (11/17), and a progression-free survival that had not yet been reached (PMID: 32171061; NCT01421186). | 32171061 | |
| Unknown unknown | transitional cell carcinoma | no benefit | Docetaxel + Icrucumab | Phase II | Actionable | In a Phase II trial, Icrucumab (IMC-18F1) and Taxotere (docetaxel) combination treatment did not result in improved median progression-free survival (1.6 months) when compared to Taxotere (docetaxel) single treatment (2.8 months) in urothelial carcinoma patients (PMID: 26926681). | 26926681 | |
| Unknown unknown | oral squamous cell carcinoma | not applicable | TAE226 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TAE226 treatment increased apoptosis and decreased proliferation and migration of oral squamous cell carcinoma cells in culture and inhibited tumor growth in cell line xenograft models (PMID: 22766511). | 22766511 | |
| Unknown unknown | acute myeloid leukemia | not applicable | T-3775440 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, T-3775440 resulted in cell cycle arrest and apoptotic activity in cell lines of acute erythroid leukemia and acute megakaryoblastic leukemia, and resulted in suppression of tumor growth in xenograft models of both types of cell lines (PMID: 27903753). | 27903753 | |
| Unknown unknown | stomach cancer | not applicable | PU-H71 | Preclinical - Cell culture | Actionable | In a preclinical study, sensitivity to PU-H71 was correlated to presence of the epichaperome, a network of chaperome complexes, in gastric cancer cell lines in culture (PMID: 27706135). | 27706135 | |
| Unknown unknown | head and neck cancer | not applicable | AZD7648 | Preclinical - Pdx | Actionable | In a preclinical study, AZD7648 treatment inhibited tumor growth in a patient-derived xenograft (PDX) model of head and neck cancer harboring mutant TP53 and wild-type ATM (PMID: 31699977). | 31699977 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | RhFzd7 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, RhFzd7 treatment inhibited cellular proliferation, invasion, and induced apoptosis in triple-negative breast cancer cell lines in culture, and inhibited tumor growth in cell line xenograft models (PMID: 30096373). | 30096373 | |
| Unknown unknown | breast cancer | not applicable | Vinflunine | Phase III | Actionable | In a Phase III trial, Javlor (vinflunine) treatment did not improve overall survival (9.1 vs 9.3 months, HR=1.04, p=0.67) compared to physician's choice of alkylating agent in patients with metastatic breast cancer (PMID: 29481630; NCT01091168). | 29481630 | |
| Unknown unknown | stomach cancer | not applicable | Capmatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Tabrecta (capmatinib) did not induce tumor regression, however, inhibited tumor growth in an autocrine cell line xenograft model of gastric cancer overexpressing HGF (PMID: 30674502). | 30674502 | |
| Unknown unknown | Indication other than cancer | not applicable | Nintedanib | FDA approved | Actionable | Ofev (nintedanib) is FDA approved for use in patients with idiopathic pulmonary fibrosis (FDA.gov). | detail... detail... | |
| Unknown unknown | breast cancer | not applicable | ST7612AA1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, ST7612AA1 inhibited proliferation of breast cancer cell lines in culture, and inhibited tumor growth in breast cancer cell line xenograft models (PMID: 25671299). | 25671299 | |
| Unknown unknown | ovarian cancer | not applicable | Cisplatin + LB-100 | Preclinical | Actionable | In a preclinical study, LB-100 sensitized several ovarian cancer cell lines to Platinol (cisplatin) in culture (PMID: 25376608). | 25376608 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Erlotinib + Everolimus | Phase I | Actionable | In a Phase I trial, Afinitor (everolimus) demonstrated safety and some efficacy in combination with Tarceva (erlotinib) in patients with advanced NSCLC (PMID: 22968184). | 22968184 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | SIM-89 | Preclinical - Cell culture | Actionable | In a preclinical study, non-small cell lung carcinoma cells demonstrated inhibition of cell proliferation and suppressed cell migration when treated with SIM-89 in culture (PMID: 28332364). | 28332364 | |
| Unknown unknown | lymphoma | not applicable | PQR309 | Preclinical - Cell culture | Actionable | In a preclinical study, PQR309 inhibited proliferation of lymphoma cells in culture, which was found to be due to the induction cell cycle arrest (PMID: 29066507). | 29066507 | |
| Unknown unknown | breast cancer | not applicable | AKI603 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AKI603 induced cell-cycle arrest and inhibited proliferation of breast cancer cell lines in culture and inhibited tumor growth in breast cancer cell line xenograft models (PMID: 24899685). | 24899685 | |
| Unknown unknown | sarcoma | not applicable | BI 853520 | Phase I | Actionable | In a Phase I trial, BI 853520 demonstrated safety and some anti-tumor efficacy, resulting in stable disease in 28.6% (4/14) of patients with soft tissue sarcoma, one of whom had stable disease lasting greater than 150 days (PMID: 30756308; NCT01335269). | 30756308 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | DS-8273a | Phase I | Actionable | In a Phase I trial, DS-8273a demonstrated safety and preliminary efficacy, resulted in decreased myeloid derived suppressor cells in a subset of patients, but no objective response (PMID: 28050790). | 28050790 | |
| Unknown unknown | pancreatic ductal adenocarcinoma | no benefit | Gemcitabine + Nab-paclitaxel + PEGPH20 | Phase III | Actionable | In a Phase III trial (HALO 109-301), Pegvorhyaluronidase alfa (PEGPH20) combined with Abraxane (nab-paclitaxel) and Gemzar (gemcitabine) (AG) resulted in an increased objective response rate compared to placebo plus AG (47% (154/327) vs 36% (60/165)), but did not improve the primary endpoint of median overall survival (11.2 vs 11.5 months) or median progression-free survival (7.1 vs 7.1 months) in patients with hyaluronan-high, metastatic pancreatic ductal adenocarcinoma (PMID: 32706635; NCT02715804). | 32706635 | |
| Unknown unknown | ovarian cancer | not applicable | Fluzoparib | Phase I | Actionable | In a Phase I trial, Fluzoparib treatment was well tolerated, and among evaluable ovarian cancer patients resulted in an overall response rate (ORR) of 8.1% (3/37) and stable disease at 24 weeks in 14/37, for a disease control rate of 45.9%, and a median progression-free survival (mPFS) of 7.2 months in patients dosed at >120mg/d, with an ORR of 30% (3/10) and mPFS of 9.3 months in platinum-sensitive patients (PMID: 32694901; NCT03509636). | 32694901 | |
| Unknown unknown | ovarian cancer | not applicable | Fluzoparib | Preclinical - Cell culture | Actionable | In a preclinical study, Fluzoparib inhibited DNA-damage-induced PARylation in high-grade serous ovarian cancer cell lines in culture, however, the cells demonstrated resistance to Fluzoparib treatment (PMID: 30949414). | 30949414 | |
| Unknown unknown | acute myeloid leukemia | not applicable | SNS-510 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with SNS-510 decreased PDPK1 pathway signaling and reduced proliferation of acute myeloid leukemia (AML) cell lines in culture, and inhibited tumor growth in an AML cell line xenograft model (Mol Cancer Ther, Dec 1 2015 (14) (12 Supplement 2) C198). | detail... | |
| Unknown unknown | hepatocellular carcinoma | not applicable | PI-88 | Phase II | Actionable | In a phase II trial, PI-88 treatment significantly improved survival for up to 3 years in hepatocellular carcinoma patients compared to control, as evidenced by increased recurrence-free rate (63% vs 50%) and postponed time to recurrence at the 36th percentile by 78% (PMID: 25170226). | 25170226 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | YM155 | Preclinical - Cell culture | Actionable | In a preclinical study, YM155 induced complete growth inhibition of triple-receptor negative breast cancer cells in culture (PMID: 27872098). | 27872098 | |
| Unknown unknown | renal cell carcinoma | not applicable | Sunitinib + Trametinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, combination of Mekinist (trametinib) and Sutent (sunitinib) effectively inhibited tumor angiogenesis and growth in cell line xenograft models of Sutent (sunitinib)-refractory renal cell carcinoma (PMID: 26487278). | 26487278 | |
| Unknown unknown | myelodysplastic syndrome | not applicable | Azacitidine + Hu5F9-G4 | Phase I | Actionable | In a Phase Ib trial, Magrolimab (Hu5F9-G4) and Vidaza (azacitidine) combination therapy was well tolerated, and resulted in complete response in 42% (14/33), marrow complete response in 24% (8/33), partial response in 3% (1/33), hematologic improvement in 21% (7/33), and stable disease in 9% (3/33) of patients with intermediate to very high risk myelodyaplastic syndrome (J Clin Oncol 38: 2020 (suppl; abstr 7507); NCT03248479). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | Navitoclax + Rucaparib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Rubraca (rucaparib) and Navitoclax (ABT-263) resulted in a synergistic effect in ovarian cancer cell lines and patient-derived ovarian cancer cells in culture, and led to increased PARP cleavage compared to treatment with Navitoclax (ABT-263) alone (PMID: 32927276). | 32927276 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ABC294640 | Phase I | Actionable | In a Phase I trial, treatment with ABC294640 in patients with advanced solid tumors resulted in antitumor efficacy, including stable disease in six patients and a partial response in a patient with cholangiosarcoma (PMID: 28420720). | 28420720 | |
| Unknown unknown | kidney cancer | not applicable | ISTH0047 | Preclinical | Actionable | In a preclinical study, ISTH0047 reduced lung metastasis, but not primary tumor growth, in a mouse model of metastatic kidney cancer (PMID: 28911087). | 28911087 | |
| Unknown unknown | follicular lymphoma | not applicable | Idelalisib | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Zydelig (idelalisib) treatment resulted in an overall response rate of 54% (39/72, 6 complete response, 33 partial response) in patients with relapsed follicular lymphoma (PMID: 24450858; NCT01282424). | 24450858 detail... | |
| Unknown unknown | prostate cancer | not applicable | Darolutamide | FDA approved | Actionable | In a Phase III trial (ARAMIS) that supported FDA approval, treatment with Nubeqa (darolutamide) resulted in improved median metastasis-free survival (40.4 vs 18.4 months, HR=0.41, p<0.001), overall survival (HR=0.71, 95% CI, 0.5-0.99, p=0.045), and time to pain progression (40.3 vs 25.4 months, HR=0.65, p<0.001) compared to placebo in non-metastatic castration-resistant prostate cancer patients (PMID: 30763142; NCT02200614). | detail... 30763142 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Erlotinib + Glesatinib | Phase I | Actionable | In a Phase I trial, Glesatinib (MGCD265) and Tarceva (erlotinib) combination therapy demonstrated safety and preliminary clinical efficacy, resulted in partial response in 1 patient, and stable disease for 6 cycles or more in 16% (7/45) of patients with advanced solid tumors (J Clin Oncol 30, 2012 (suppl; abstr e13602)). | detail... | |
| Unknown unknown | breast cancer | not applicable | Fulvestrant + JNJ-7706621 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with the combination of JNJ-7706621 and Faslodex (fulvestrant) resulted in increased growth inhibition in aromatase inhibitor-resistant breast cancer cell lines compared to either agent alone (PMID: 25667100). | 25667100 | |
| Unknown unknown | pancreatic cancer | not applicable | Chidamide | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Chidamide (CS055) inhibited growth of a human pancreatic cancer cell line in culture and inhibited tumor growth in xenograft models (PMID: 25384499). | 25384499 | |
| Unknown unknown | ovarian cancer | not applicable | Demcizumab + Paclitaxel | Phase I | Actionable | In a Phase I trial (SIERRA), Demcizumab (OMP-21M18) and Taxol (paclitaxel) combination treatment demonstrated safety and preliminary efficacy in patients with platinum-resistant ovarian cancer, resulting in an overall response rate of 21% (4/19) and a clinical benefit rate of 42% (8/19) (PMID: 32037195; NCT01952249). | 32037195 | |
| Unknown unknown | ovarian cancer | not applicable | Demcizumab + Paclitaxel | Preclinical - Pdx | Actionable | In a preclinical study, Demcizumab (OMP-21M18) and Taxol (paclitaxel) combination treatment inhibited tumor growth and suppressed cancer stem cells in patient-derived xenograft models of ovarian cancer (Cancer Res 2013;73(8 Suppl):Abstract nr 3725). | detail... | |
| Unknown unknown | colon cancer | not applicable | CVX-060 + Sorafenib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of CVX-060 and Nexavar (sorafenib) resulted in increased tumor growth inhibition compared to either agent alone in a colon cancer cell line xenograft model (PMID: 21233403). | 21233403 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Duvelisib | Preclinical - Cell culture | Actionable | In a preclinical study, Copiktra (duvelisib) inhibited proliferation, and resulted in an increase in activation-induced cytidine deaminase (AID) expression and genomic instability in a mantle cell lymphoma cell line in culture (PMID: 28199309). | 28199309 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MLN4924 | Phase I | Actionable | In a Phase I trial, Pevonedistat (MLN4924) treatment resulted in stable disease in 74% (23/31) of patients with advanced solid tumors (PMID: 26423795). | 26423795 | |
| Unknown unknown | multiple myeloma | not applicable | CTX120 | Preclinical | Actionable | In a preclinical study, CTX120 treatment inhibited tumor growth in a mouse model of multiple myeloma (Blood (2018) 132 (Supplement 1): 1921). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BI 836880 | Phase I | Actionable | In a Phase I trial, BI 836880 treatment was well tolerated, and resulted in partial response in 7% (2/29) and stable disease in 31% (9/29) of patients with advanced solid tumors (J Clin Onc 2018 36:15_suppl, 12024; NCT02674152). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | M2698 | Preclinical | Actionable | In a preclinical study, MSC2363318A demonstrated sustained inhibition of S6K phosphorylation, and inhibited tumor growth in several human cancer xenograft models of breast, pancreatic, glioblastoma and ovarian cancers (Mol Cancer Ther 2013;12(11 Suppl):A162). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | M2698 | Phase I | Actionable | In a Phase I trial, M2698 (MSC2363318A) demonstrated safety and preliminary efficacy, with 19% (6/32) of advanced solid tumor patients remained on treatment for more than 180 days (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 370PD; NCT01971515). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | M2698 | Phase I | Actionable | In a Phase I trial, M2698 (MSC2363318A) demonstrated safety and preliminary efficacy in a variety of advanced solid tumor patients (Ann. Oncol. 26 (Suppl 2): ii25-ii27, 2015). | detail... | |
| Unknown unknown | hematologic cancer | not applicable | SNX-7081 | Preclinical | Actionable | In a preclinical study, SNX-7081 induced cell cycle arrest and apoptosis in several hematological cell lines and chronic lymphocytic patient samples in culture (PMID: 20738310). | 20738310 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Ibrutinib + Venetoclax | Phase II | Actionable | In a Phase II trial, Imbruvica (ibrutinib) and Venclexta (venetoclax) combination therapy resulted in a response rate of 100% (14/14, 9 complete response, 5 partial response) in relapsed or refractory chronic lymphocytic leukemia (CLL) patients, and a response rate of 100% (16/16, 9 complete response, 7 partial response) in untreated patients with high-risk features including del 17p, TP53 mutations, and del 11q (ASH, 59th Annual Meeting and Exposition, Dec 2017, Abstract 429; NCT02756897). | detail... | |
| Unknown unknown | pancreatic carcinoma | not applicable | UNC0642 | Preclinical - Cell culture | Actionable | In a preclinical study, a pancreatic carcinoma cell line demonstrated sensitivity to UNC0642, resulting in inhibition of colony formation in culture (PMID: 24102134). | 24102134 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Bortezomib + Tinostamustine | Preclinical - Cell culture | Actionable | In a preclinical study, EDO-S101 and Velcade (bortezomib) worked synergistically to decrease viability of triple negative breast cancer cell lines in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | BAY1161909 + Paclitaxel | Preclinical | Actionable | In a preclinical study, BAY1161909, in combination with Taxol (paclitaxel), had increased efficacy in xenograft models of triple-negative breast cancer compared to Taxol (paclitaxel) alone, resulting in complete tumor regression (PMID: 26832791). | 26832791 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | OSI-027 | Phase I | Actionable | In a Phase I trial, OSI-027 treatment resulted in no RECIST response and stable disease in 5% (6/128) of patients with advanced solid tumors (PMID: 27002938). | 27002938 | |
| Unknown unknown | ovarian cancer | not applicable | CPI-203 + Rucaparib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of CPI-203 and Rubraca (rucaparib) resulted in a synergistic effect, demonstrating greater growth inhibition of ovarian cancer cells in culture compared to CPI-203 treatment alone (PMID: 32927276). | 32927276 | |
| Unknown unknown | Burkitt lymphoma | not applicable | TTI-621 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the mouse surrogate version of TTI-621 (SIRPalpha-Fc) decreased tumor growth in Burkitt lymphoma cell line xenograft models (PMID: 27856600). | 27856600 | |
| Unknown unknown | lung carcinoma | not applicable | MRx0518 | Preclinical | Actionable | In a preclinical study, MRx0518 stimulated immune response and reduced tumor size in syngeneic mouse models of lung carcinoma (Journal of Clinical Oncology 36, no. 15_suppl). | detail... | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | STRO-001 | Preclinical - Cell culture | Actionable | In a preclinical study, STRO-001 inhibited growth of EBV-transformed chronic lymphocytic leukemia cells in culture (Blood 2016 128 (22):464). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Lenvatinib | Phase I | Actionable | In a Phase I clinical trial, Lenvima (lenvatinib) demonstrated anti-tumor activity in patients with several advanced solid tumor types, including patients with non-small cell lung cancer (PMID: 26169970). | 26169970 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Axitinib + Radiotherapy | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Inlyta (axitinib) and radiotherapy resulted in improved efficacy compared to either agent alone, and decreased pneumonitis in non-small cell lung cancer cell line xenograft models (PMID: 24862536). | 24862536 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Copanlisib | Phase II | Actionable | In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 17% (1/6) and partial response in 67% (4/6) of patients with mantle cell lymphoma (PMID: 24852792). | 24852792 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | DS-3078a | Phase I | Actionable | In a Phase I trial, DS-3078a demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Mol Cancer Ther November 2013 12:C173). | detail... | |
| Unknown unknown | colorectal cancer | no benefit | Atezolizumab | Phase III | Actionable | In a Phase III trial (IMblaze370), Tecentriq (atezolizumab) treatment did not improve median overall survival (7.1 vs 8.5 months, HR=1.19) compared to Stivarga (regorafenib) in patients with chemotherapy-refractory metastatic colorectal cancer, 91.7% of whom were microsatellite stable or microsatellite instability-low (Annals of Oncology, Volume 29, Issue suppl_5, 1 June 2018; NCT02788279). | detail... | |
| Unknown unknown | lung carcinoma | not applicable | AGI-134 | Preclinical - Cell culture | Actionable | In a preclinical study, AGI-134 treatment induced cell death of lung carcinoma cells in the presence of normal human serum in culture and induced phagocytosis when co-cultured with human macrophages (PMID: 31889898). | 31889898 | |
| Unknown unknown | pancreatic ductal adenocarcinoma | not applicable | Dinaciclib | Preclinical - Cell culture | Actionable | In a preclinical study, Dinaciclib (SCH 727965) treatment inhibited proliferation and induced apoptosis in mouse pancreatic ductal adenocarcinoma cell lines in culture, and delayed tumor growth and increased overall survival in a transgenic mouse model (PMID: 32269732). | 32269732 | |
| Unknown unknown | ovary epithelial cancer | not applicable | MGD013 | Phase I | Actionable | In a Phase I trial, MGD013 treatment resulted in a partial response in 13.3% and stable disease in 46.7% (7/15) of patients with epithelial ovarian cancer (J Clin Oncol 38: 2020 (suppl; abstr 3004); NCT03219268). | detail... | |
| Unknown unknown | colon carcinoma | not applicable | SLC-391 + unspecified PD-1 antibody | Preclinical | Actionable | In a preclinical study, treatment with SLC-391 combined with an anti-PD-1 antibody demonstrated synergy and increased overall survival rate in a syngeneic mouse model of colon carcinoma (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B148). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Bevacizumab + Cediranib | Phase I | Actionable | In a Phase I trial, the combination of Cediranib (AZD-2171) and Avastin (bevacizumab) demonstrated preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 24752867). | 24752867 | |
| Unknown unknown | cervical cancer | not applicable | ETP-46464 + Radiotherapy | Preclinical - Cell culture | Actionable | In a preclinical study, ETP-46464 increased the sensitivity of cervical cancer cell lines to ionizing radiation in culture (PMID: 25560806). | 25560806 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | SR9009 | Preclinical - Cell culture | Actionable | In a preclinicl study SR9009 demonstrated toxicity in a wide range of tumor cell lines harboring different driver mutations, but not in normal cell lines in culture (PMID: 29320480). | 29320480 | |
| Unknown unknown | chronic leukemia | not applicable | RG7112 | Phase I | Actionable | In a Phase I clinical trial, 5% (1/19) of chronic leukemia patients achieved partial response, and 79% (15/19) achieved stable disease following treatment with RG7112 (PMID: 26459177). | 26459177 | |
| Unknown unknown | lymphoma | not applicable | Panobinostat + PQR309 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination therapy of Farydak (panobinostat) and PQR309 induced apoptosis and led to synergistic and additive effects in lymphoma cell lines in culture (PMID: 29066507). | 29066507 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Everolimus | Phase Ib/II | Actionable | In a Phase Ib trial, Afinitor (everolimus) demonstrated safety and preliminary efficacy in patients with non-small cell lung cancer (PMID: 25673697). | 25673697 | |
| Unknown unknown | breast cancer | not applicable | VS-5584 | Preclinical - Patient cell culture | Actionable | In a preclinical study, treatment with VS-5584 resulted in decreased cancer stem cell (CSC) number in a triple-negative breast cancer cell line in culture and in xenograft models, and decreased CSCs in patient breast cancer tumor samples in culture (PMID: 25432176). | 25432176 | |
| Unknown unknown | melanoma | not applicable | Atezolizumab + Cobimetinib | Phase I | Actionable | In a Phase Ib trial, Tecentriq (atezolizumab) and Cotellic (cobimetinib) combination treatment demonstrated safety and preliminary clinical activity, resulted in a confirmed response in 41% (9/22) of patients with melanoma, regardless of KRAS/BRAF status (PMID: 30918950; NCT01988896). | 30918950 | |
| Unknown unknown | colorectal cancer | not applicable | Bevacizumab + Capecitabine | Phase III | Actionable | In a Phase III trial, maintenance therapy with Avastin (bevacizumab) and Xeloda (capecitabine) in combination resulted in a greater benefit compared to observation in colorectal cancer patients who were re-treated with Avastin (bevacizumab), Xeloda (capecitabine), and Eloxatin (oxaliplatin) due to progression, regardless of whether patients harbored RAS or BRAF V600E mutations (PMID: 28911067). | 28911067 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Duligotuzumab | Phase I | Actionable | In a Phase I trial, MEHD7945A treatment resulted in partial response in 3% (2/66) and stable disease in 21% (14/66) of patients with advanced solid tumors (PMID: 26034219). | 26034219 | |
| Unknown unknown | Hodgkin's lymphoma | not applicable | Ixazomib | Preclinical | Actionable | In a preclinical study, Ixazomib (MLN9708) inhibited survival and induced apoptosis in Hodgkin's lymphoma cell lines in culture, and reduced tumor volume in xenograft models (PMID: 26988986). | 26988986 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ME-344 | Phase I | Actionable | In a Phase I trial, ME-344 demonstrated preliminary tolerability and efficacy in patients with advanced solid tumors (PMID: 25411085). | 25411085 | |
| Unknown unknown | lung carcinoma | not applicable | JQ1 | Preclinical | Actionable | In a preclinical study, JQ1 decreased tumor immunosuppression, increased T-lymphocyte infiltration and reduced tumor growth and increased survival in syngeneic mouse lung carcinoma models (PMID: 31018997). | 31018997 | |
| Unknown unknown | granulosa cell tumor | no benefit | STM434 | Phase I | Actionable | In a Phase I trial, STM 434 treatment did not result in response in 30 evaluable patients with advanced solid tumors and only resulted in stable disease as best response in 80% (10/12) of patients with granulosa cell ovarian cancer (PMID: 31068369; NCT02262455). | 31068369 | |
| Unknown unknown | colorectal cancer | no benefit | Cyclophosphamide + GVAX colorectal cancer vaccine + Pembrolizumab | Phase II | Actionable | In a Phase II trial, GVAX colorectal cancer vaccine in combination with Cytoxan (cyclophosphamide) and Keytruda (pembrolizumab) resulted in a median progression-free survival of 82 days, median overall survival of 213 days, and disease control rate of 18% (3/17), and while biochemical responses were observed, the study failed to meet its primary objective as no clinical objective responses were achieved in patients with mismatch repair proficient colorectal cancer (PMID: 31876399; NCT02981524). | 31876399 | |
| Unknown unknown | breast cancer | not applicable | DCBCI0901 | Preclinical - Cell culture | Actionable | In a preclinical study, breast cancer cell lines treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | OPB-111077 | Phase I | Actionable | In a Phase I trial, a patient with diffuse large B-cell lymphoma demonstrated a partial response when treated with OPB-111077 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B118). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Elenagen | Phase Ib/II | Actionable | In a Phase I/IIa trial, Elenagen treatment resulted in a best response of stable disease in 44% (12/27) of patients with advanced solid tumors (PMID: 28881846). | 28881846 | |
| Unknown unknown | colorectal cancer | not applicable | Bevacizumab + Fluorouracil + Leucovorin + Oxaliplatin | Clinical Study | Actionable | In a Phase IV trial, the addition of Avastin (bevacizumab) to Adrucil (fluorouracil), Wellcovorin (leucovorin), and Eloxatin (oxaliplatin) compared to without Avastin (bevacizumab) in patients with RAS mutant unresectable colorectal liver metastases resulted in a greater resection rate, 22.3% (27/121) vs 5.8% (7/120), objective response rate, 54.5% (66/121) vs 36.7% (44/12), median progression-free survival, 9.5 mo vs 5.6 mo, and median overall survival, 25.7 mo vs 20.5 mo (PMID: 32749938; NCT01972490). | 32749938 | |
| Unknown unknown | colorectal cancer | not applicable | Bevacizumab + Fluorouracil + Leucovorin + Oxaliplatin | Clinical Study | Actionable | In a clinical study, colorectal cancer patients demonstrated a greater median PFS when treated with FOLFOX and Avastin (bevacizumab), which was thought to be associated with a decrease in granulocytic myeloid-derived suppressor cells highly expressing PD-L1 (PMID: 27496709). | 27496709 | |
| Unknown unknown | fibrosarcoma | not applicable | 23814 + Tivozanib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of 23814 and Fotivda (tivozanib) resulted in tumor growth inhibition in a fibrosarcoma cell line xenograft model, with increased efficacy over either agent alone (PMID: 25995436). | 25995436 | |
| Unknown unknown | renal cell carcinoma | not applicable | Tivantinib | Phase II | Actionable | In a Phase II trial, tivantinib resulted in modest activity in which six patients with renal cell carcinoma had a median PFS of 1.9 months (PMID: 22605650). | 22605650 | |
| Unknown unknown | sarcoma | not applicable | Conatumumab + Ganitumab | Phase Ib/II | Actionable | In a Phase Ib/II trial, Ganitumab and Conatumumab (AMG 655) combination treatment resulted in stable disease in 33% (5/15) of patients with sarcoma, including one with leiomyosarcoma (PMID: 24816908). | 24816908 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Merestinib | Phase I | Actionable | In a Phase I trial, LY2801653 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Cancer Res October 1, 2014 74:CT237). | detail... | |
| Unknown unknown | follicular lymphoma | not applicable | Bendamustine + Veliparib | Phase I | Actionable | In a Phase I trial, a patient with follicular lymphoma treated with a combination of Veliparib (ABT-888) and Bendamustine demonstrated a complete response (PMID: 28314788; NCT01326702). | 28314788 | |
| Unknown unknown | colon cancer | not applicable | Gemcitabine + SRA737 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Gemzar (gemcitabine) resulted in enhanced antitumor efficacy when combined with SRA737 (CCT245737) in a colon cancer cell line xenograft model (PMID: 27167172). | 27167172 | |
| Unknown unknown | melanoma | not applicable | JSH-150 | Preclinical - Cell culture | Actionable | In a preclinical study, JSH-150 inhibited proliferation of a melanoma cell line in culture (PMID: 30253346). | 30253346 | |
| Unknown unknown | mantle cell lymphoma | not applicable | STRO-001 | Preclinical - Cell culture | Actionable | In a preclinical study, STRO-001 potently inhibited growth of mantle cell lymphoma cell lines in culture (Blood 2016 128 (22):464). | detail... | |
| Unknown unknown | glioblastoma | not applicable | Temozolomide + Vistusertib | Phase I | Actionable | In a Phase I trial, combination of Vistusertib (AZD2014) and Temodar (temozolomide) demonstrated safety in patients with previously treated glioblastoma multiforme, resulted in a partial response in 8% (1/13) and stable disease in 38% (5/13) of the patients, with a 6-month progression-free survival rate of 26% (PMID: 31707687). | 31707687 | |
| Unknown unknown | mantle cell lymphoma | not applicable | CC214-1 | Preclinical - Patient cell culture | Actionable | In preclinical study, mantle cell lymphoma patient derived cell lines demonstrated improved survival in culture when treated with CC214-1 (PMID: 25839159). | 25839159 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Melanin + Phenytoin + Sirolimus + SM88 | Phase I | Actionable | In a Phase I trial, SMK therapy (sirolimus, phenytoin, and sirolimus with SM-88) demonstrated safety in advanced metastatic cancer patients and resulted in complete response (CR) in four and partial response (PR) in six patients, including three CR and three PR among breast cancer patients (n=14), and stable disease in 17 patients for a clinical benefit rate of 90% (27/30), and a median progression-free survival of 13 months and median overall survival of 29.8 months (PMID: 30929156). | 30929156 | |
| Unknown unknown | melanoma | no benefit | Cediranib | Phase II | Actionable | In a Phase II trial, treatment with Cediranib (AZD-2171) in melanoma patients resulted in only two patients with stable disease at 6 months, no objective responses, and a short median time to progression of 3.5 months thereby demonstrating no benefit (PMID: 26841902). | 26841902 | |
| Unknown unknown | breast cancer | not applicable | RapaLink-1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a breast cancer cell line demonstrated sensitivity to RapaLink-1 in culture and in xenograft models, resulting in inhibition of both Mtor signaling and tumor growth (PMID: 27279227). | 27279227 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | HS-110 + Nivolumab | Phase Ib/II | Actionable | In a Phase I/II trial, HS-110 (Viagenpumatucel-L) and Opdivo (nivolumab) combination treatment demonstrated safety, and resulted in an objective response rate of 21% (10/47), a clinical benefit rate of 43%, a median duration of response of 17.2 months, and a median overall survival of 28.7 months in patients with advanced non-small cell lung cancer (J Clin Oncol 38: 2020 (suppl; abstr 9546); NCT02439450). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | B02 + Veliparib | Preclinical - Cell culture | Actionable | In a preclinical study, the addition of B02 resulted in increased sensitivity to Veliparib (ABT-888), in a multiple myeloma cell line in culture, leading decreased cell viability (PMID: 26719576). | 26719576 | |
| Unknown unknown | lung cancer | no benefit | Carboplatin + Cediranib + Paclitaxel | Phase I | Actionable | In a Phase II clinical trial of patients with advanced NSCLC, the combination of Cediranib (AZD-2171) and carboplatin/paclitaxel (CP) chemotherapy resulted in improved response rate over placebo plus CP, but did not improve progression-free survival, and the study did not proceed to Phase III due to toxicity (PMID: 24360368). | 24360368 | |
| Unknown unknown | breast carcinoma | not applicable | RXDX-106 + unspecified PD-1 antibody | Preclinical | Actionable | In a preclinical study, RXDX-106 in combination with an unspecified anti-PD-1 antibody resulted in near complete regression in orthotopic animal models of breast carcinoma (Eur J Cancer, Vol 69, Supplement 1, December 2016, Page S31). | detail... | |
| Unknown unknown | acute promyelocytic leukemia | not applicable | Carfilzomib + Tinostamustine | Preclinical - Cell culture | Actionable | In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of an acute promyelocytic leukemia cell line in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | esophageal carcinoma | not applicable | BI 853520 | Phase I | Actionable | In a Phase I trial, BI 853520 demonstrated safety and some anti-tumor efficacy, resulting in stable disease in 12.5% (2/16) of patients with esophageal carcinoma (PMID: 30756308; NCT01335269). | 30756308 | |
| Unknown unknown | glioblastoma | not applicable | Olaparib + Temozolomide | Phase II | Actionable | In a Phase II trial (OPARATIC), 36% (14/39) of evaluable patients with glioblastoma were progression-free at 6 months when treated with the combination therapy of Lynparza (olaparib) and Temodar (temozolomide) (PMID: 32347934; NCT0139057). | 32347934 | |
| Unknown unknown | cervical cancer | not applicable | AsiDNA + Olaparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA treatment led to increased sensitivity to Lynparza (olaparib), resulting in decreased survival in a cervical cancer cell line in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | colorectal cancer | not applicable | LY2090314 + Niraparib | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with LY2090314 sensitized colorectal cancer cell lines with either BRCA1/2 proficiency or BRCA2 deficiency to Zejula (niraparib) in culture, resulting in a synergistic effect (PMID: 33589588). | 33589588 | |
| Unknown unknown | ovarian cancer | not applicable | SST0116CL1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, SST0116CL1 inhibited tumor growth in p-glycoprotein over expressing ovarian cancer cell line xenograft models (PMID: 25096516). | 25096516 | |
| Unknown unknown | angioimmunoblastic T-cell lymphoma | not applicable | Domatinostat | Case Reports/Case Series | Actionable | In a Phase I trial, a patient with relapsed angioimmunoblastic T-cell lymphoma demonstrated a complete response lasting over 2 years following treatment with Domatinostat (4SC-202) (PMID: 30347469; NCT01344707). | 30347469 | |
| Unknown unknown | lung non-small cell carcinoma | no benefit | Bevacizumab | Phase III | Actionable | In a Phase IIIb trial, the combination of Avastin (bevacizumab) and standard of care therapy at first progression in non-small cell lung carcinoma patients previously treated with Avastin (bevacizumab) and chemotherapy and two maintenance cycles of Avastin (bevacizumab) did not result in a greater benefit compared to standard of care therapy alone, demonstrating a median overall survival of 11.9 mo vs 10.2 mo and a first to second progression-free survival of 5.5 mo vs 4.0 mo (PMID: 30177994; NCT01351415). | 30177994 | |
| Unknown unknown | acute myeloid leukemia | not applicable | CWP232291 | Phase I | Actionable | In a Phase I trial, CWP232291 demonstrated tolerability and limited preliminary efficacy in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS), resulting in one complete response and one partial response among 64 AML patients, while none of the five MDS patients achieved a response (PMID: 32396615; NCT01398462). | 32396615 | |
| Unknown unknown | acute myeloid leukemia | not applicable | GS87 | Preclinical | Actionable | In a preclinical study, treatment with GS87 induced terminal differentiation and inhibited growth of acute myeloid leukemia (AML) cell lines in culture and resulted in increased survival and decreased disease burden in mouse models of AML (PMID: 27196775). | 27196775 | |
| Unknown unknown | smoldering myeloma | not applicable | Lenalidomide + PVX-410 | Phase Ib/II | Actionable | In a Phase I/IIa clinical trial, combination therapy with PVX-410 and Revlimid (lenalidomide) was well-tolerated, and resulted in a partial response in 11% (1/9), minimal response in 44% (4/9), and stable disease in 44% (4/9) of patients with smoldering multiple myeloma with moderate/high risk of progression, and the magnitude of the immune response observed was significantly larger than in patients treated with PVX-410 alone (PMID: 30128502; NCT01718899). | 30128502 | |
| Unknown unknown | melanoma | not applicable | SGN-CD228A | Preclinical - Cell line xenograft | Actionable | In a preclinical study, SGN-CD228A treatment delayed tumor growth and resulted in complete responses in cell line xenograft models of melanoma (Cancer Res 2019;79(13 Suppl):Abstract nr 2688). | detail... | |
| Unknown unknown | pancreatic cancer | not applicable | Trabedersen | Phase Ib/II | Actionable | In a Phase I/II trial, Trabedersen (AP 12009) treatment followed by chemotherapy resulted in an overall survival of 14.5 months in advanced pancreatic cancer patients (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 119P). | detail... | |
| Unknown unknown | pancreatic cancer | not applicable | Trabedersen | Phase Ib/II | Actionable | In a Phase Ib/II trial, Trabedersen (AP 12009) treatment followed by chemotherapy resulted in improved median overall survival (9.4 vs 2.8 months, p=0.0004) compared to Trabedersen (AP 12009) treatment followed by best supportive care in patients with advanced pancreatic cancer (AACR Annual Meeting 2019, Abstract 3968). | detail... | |
| Unknown unknown | non-Hodgkin lymphoma | not applicable | Duvelisib | Phase I | Actionable | In a Phase I trial, Copiktra (duvelisib) treatment resulted in complete response in 16% (5/31), partial response in 45% (14/31), minor response in 3% (1/31), and stable disease in 29% (9/31) in non-Hodgkin lymphoma patients (Blood 124(21): 802). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | PF-06263507 | Phase I | Actionable | In a Phase I trial, treatment with PF-06263507 resulted in no objective responses, but led to stable disease in two patients with advanced solid tumors (PMID: 28070718). | 28070718 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Adavosertib + Everolimus | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Afinitor (everolimus) and Adavosertib (MK-1775) resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27872098). | 27872098 | |
| Unknown unknown | liposarcoma | not applicable | MGCD516 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MGCD516 blocked cell proliferation of a human liposarcoma cell line in culture and repressed tumor growth in xenograft models (PMID: 26675259). | 26675259 | |
| Unknown unknown | lymphoma | not applicable | OKI-179 | Preclinical - Cell culture | Actionable | In a preclinical study, OKI-005 induced cell cycle arrest and apoptosis in lymphoma cells in culture (PMID: 31235619). | 31235619 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | IPI-549 | Preclinical | Actionable | In a preclinical study, IPI-549 inhibited tumor growth in multiple xenograft models of solid tumors (Mol Cancer Ther December 2015 14; A192). | detail... | |
| Unknown unknown | lung small cell carcinoma | not applicable | Doxorubicin + SLCB050 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination treatment of Adriamycin (doxorubicin) plus SLCB050 at low doses in a lung small cell carcinoma cell line xenograft model resulted in decreased tumor volume (PMID: 27302160). | 27302160 | |
| Unknown unknown | colon adenocarcinoma | not applicable | JQ1 | Preclinical | Actionable | In a preclinical study, JQ1 increased T-lymphocyte infiltration and reduced tumor growth in syngeneic mouse colon adenocarcinoma models (PMID: 31018997). | 31018997 | |
| Unknown unknown | breast cancer | not applicable | PF-00562271 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with PF-00562271 inhibited PTK2 (FAK) phosphorylation and resulted in apoptosis and tumor regression in breast cancer cell line xenograft models (PMID: 18339875). | 18339875 | |
| Unknown unknown | colon carcinoma | not applicable | UD-017 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, UD-017 inhibited tumor growth in cell line xenograft models of colon carcinoma (J Clin Oncol 35, 2017 (suppl; abstr e14085)). | detail... | |
| Unknown unknown | thyroid gland carcinoma | not applicable | Obatoclax | Preclinical | Actionable | In a preclinical study, Obatoclax induced cell death in human thyroid carcinoma cell lines culture (PMID: 26198850). | 26198850 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ASP5878 | Phase I | Actionable | In a Phase I trial, ASP5878 demonstrated safety and some preliminary efficacy in patients with advanced solid tumors, including a bladder cancer patient with an FGFR gene mutation (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A165). | detail... | |
| Unknown unknown | stomach cancer | not applicable | Capecitabine + Nivolumab + Oxaliplatin | FDA approved | Actionable | In a Phase III trial (CHECKMATE-649) that supported FDA approval, Opdivo (nivolumab) in combination with chemotherapy (mFOLFOX6 or CapeOx) significantly improved overall survival (13.8 vs 11.6 months, HR 0.80, p=0.0002) compared to chemotherapy alone in patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma (Ann Oncol Sep 2020, 31 (Suppl 4), S1191; NCT02872116). | detail... detail... | |
| Unknown unknown | ovarian cancer | not applicable | TAK-960 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, ovarian cancer cells treated with TAK-960 demonstrated cell growth inhibition and decreased tumor size in both culture and cell line xenograft models (PMID: 22188812). | 22188812 | |
| Unknown unknown | medulloblastoma | not applicable | HSV-1 G207 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, HSV-1 G207 treatment resulted in prolonged survival, with a 44% increase, in medulloblastoma cell line xenograft models (PMID: 30918335). | 30918335 | |
| Unknown unknown | esophagus squamous cell carcinoma | not applicable | Cisplatin + Fluorouracil + Tislelizumab | Phase II | Actionable | In a Phase II trial, Tislelizumab (BGB-A317) in combination with Platinol (cisplatin) and Adrucil (fluorouracil) demonstrated tolerability in patients with advanced esophageal squamous cell carcinoma and resulted in an objective response rate of 46.7% (7/15, all partial responses), disease control rate of 80% (12/15), median time to response of 10.0 weeks, and median progression-free survival of 10.4 months (PMID: 32561664; NCT03469557). | 32561664 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Pemetrexed Disodium + Sorafenib | Phase I | Actionable | In a Phase I clinical trial in patients with advanced solid tumors, the combination of Alimta (pemetrexed) and Nexavar (sorafenib) demonstrated safety and preliminary efficacy in patients with triple-receptor breast cancer (TNBC), with 60% (3/5) of TNBC patients demonstrating an objective response and 100% (5/5) of patients achieving stable disease or better (PMID: 27213589). | 27213589 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Nidanilimab | Phase Ib/II | Actionable | In a Phase Ib/II trial, Nidanilimab treatment demonstrated manageable safety, and resulted in a stable disease (SD) in 45% (9/20) of advanced solid tumor patients with a non-small cell lung carcinoma patient having SD for 6 months and a pancreatic ductal adenocarcinoma patient having SD for 4 months (Journal of Clinical Oncology 2019 37:15_suppl, 2504). | detail... | |
| Unknown unknown | lung cancer | not applicable | Triolimus | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Triolimus led to cytotoxicity and inhibited Ras/Raf/MAPK and PI3K/Akt/mTOR pathway signaling in lung cancer cells in culture, and induced apoptosis, which resulted in tumor growth inhibition, in cell line xenograft models (PMID: 22896668). | 22896668 | |
| Unknown unknown | colorectal cancer | not applicable | UD-017 | Preclinical - Patient cell culture | Actionable | In a preclinical study, UD-017 inhibited growth of patient-derived colorectal cancer cells in culture (J Clin Oncol 35, 2017 (suppl; abstr e14085)). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Cisplatin + Gemcitabine + Metformin | Phase 0 | Actionable | In a pilot clinical trial, treatment with Glucophage (metformin) in combination with Gemzar (gemcitabine) and Platinol (cisplatin) resulted in an overall response rate of 46.7%, compared to 13.3% with Gemzar (gemcitabine) plus Platinol (cisplatin) therapy in patients with non-small cell lung cancer, but this difference was not statistically significant (PMID: 26434885). | 26434885 | |
| Unknown unknown | melanoma | not applicable | Fresolimumab | Phase I | Actionable | In a phase I clinical trial, Fresolimumab (GC1008) demonstrated safety and preliminary evidence of antitumor activity in patients with malignant melanoma (PMID: 24618589). | 24618589 | |
| Unknown unknown | colorectal cancer | not applicable | Abemaciclib | Phase I | Actionable | In a Phase I trial, treatment with Abemaciclib (LY2835219) in colorectal cancer patients resulted in 2 patients (13%; 2/15) with stable disease (PMID: 27217383). | 27217383 | |
| Unknown unknown | B-cell acute lymphoblastic leukemia | not applicable | Loncastuximab tesirine-lpyl | Phase I | Actionable | In a Phase I trial, Zynlonta (loncastuximab tesirine-lpyl) treatment demonstrated manageable safety profile, resulted in a complete response in 8.5% (3/35) of patients with relapsed or refractory B-cell acute lymphoblastic leukemia (PMID: 32012214; NCT02669264). | 32012214 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Merestinib | Preclinical | Actionable | In a preclinical study, LY2801653 inhibited tumor growth in mouse xenograft models of non-small cell lung cancer (PMID: 24305878). | 24305878 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Tivozanib | Phase I | Actionable | In a Phase I clinical study, Fotivda (tivozanib) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Clin Cancer Res, November 15, 2011 17; 7156). | detail... | |
| Unknown unknown | breast cancer | not applicable | BGP-15 | Preclinical - Cell culture | Actionable | In a preclinical study, BGP-15 induced apoptosis and inhibited growth of breast cancer cells in culture (PMID: 22661288). | 22661288 | |
| Unknown unknown | Her2-receptor positive breast cancer | not applicable | CYC065 | Preclinical - Cell culture | Actionable | In a preclinical study, a breast cancer cell line harboring an ERBB2 (HER2) amplification demonstrated decreased cell viability, induction of apoptosis, and decreased levels of Myc and Mcl1 when treated with CYC065 (Fadraciclib) in culture (PMID: 32645016). | 32645016 | |
| Unknown unknown | ovary epithelial cancer | not applicable | Alpelisib + Olaparib | Phase I | Actionable | In a Phase Ib trial, Alpelisib (BYL719) and Lynparza (olaparib) combination therapy demonstrated safety and preliminary efficacy, resulted in partial response in 36% (10/28) and stable disease in 50% (14/28) of patients with epithelial ovarian cancer, overall response rate was similar for germline BRCA mutated and wild-type patients (30%, 3/10 vs 35%, 6/17, p=0.42) (PMID: 30880072; NCT01623349). | 30880072 | |
| Unknown unknown | neuroblastoma | not applicable | Hu3F8-BsAb | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Hu3F8-BsAb treatment induced cell killing in neuroblastoma cell lines expressing GD2 in culture, and treatment with Hu3F8-BsAb plus human peripheral blood mononuclear cells inhibited tumor growth in neuroblastoma cell line xenograft models (PMID: 25542634). | 25542634 | |
| Unknown unknown | colon adenocarcinoma | not applicable | REGN1400 | Preclinical | Actionable | In a preclinical study, REGN1400, alone and in combination with anti-EGFR antibodies, showed efficacy in treating mouse xenograft models of colorectal cancer (PMID: 24634416). | 24634416 | |
| Unknown unknown | ovarian cancer | not applicable | Paclitaxel + Volasertib | Preclinical - Cell culture | Actionable | In a preclinical study, Volasertib (BI 6727) and Taxol (paclitaxel) synergistically inhibited growth of ovarian cancer cell lines in culture (PMID: 32183025). | 32183025 | |
| Unknown unknown | stomach cancer | not applicable | AT13148 | Preclinical | Actionable | In a preclinical study, AT13148 inhibited proliferation and induced apoptosis in gastric cancer cell lines in culture and inhibited tumor growth in a gastric cancer cell line xenograft model (PMID: 26828267). | 26828267 | |
| Unknown unknown | thyroid gland papillary carcinoma | not applicable | Ramucirumab | Phase I | Actionable | In a Phase I trial, Cyramza (ramucirumab) demonstrated safety and efficacy resulting in partial response or stable disease in patients with advanced solid tumors, including papillary thyroid carcinoma (PMID: 20048182). | 20048182 | |
| Unknown unknown | breast cancer | not applicable | Pyr1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Pyr1 inhibited proliferation and migration of breast cancer cell lines in culture, and inhibited growth of primary tumors and metastases in breast cancer cell line xenograft models (PMID: 27216191). | 27216191 | |
| Unknown unknown | islet cell tumor | not applicable | Ipilimumab + Nivolumab | Case Reports/Case Series | Actionable | In a Phase II trial (CA209-538), the combination of Yervoy (ipilimumab) and Opdivo (nivolumab) resulted in a 43% (3/7) objective response rate in patients with high grade pancreatic neuroendocrine neoplasms, including one patient with pancreatic neuroendocrine small cell carcinoma (PMID: 32532787; NCT02923934). | 32532787 | |
| Unknown unknown | breast cancer | not applicable | Docetaxel + MEDI5117 | Preclinical | Actionable | In a preclinical study, MEDI5117 in combination with Taxotere (docetaxel) resulted in complete tumor regression of human breast cancer cells in an orthotopic model (PMID: 26744529). | 26744529 | |
| Unknown unknown | acute myeloid leukemia | not applicable | IACS-010759 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, IACS-010759 inhibited growth of acute myeloid leukemia cells in culture, resulting in prolonged survival in patient-derived xenograft models (Mol Cancer Ther 2015; 14(12 Suppl 2): Abstract nr LB-A15). | detail... | |
| Unknown unknown | glioblastoma | not applicable | Gliovac + Sargramostim | Clinical Study | Actionable | In a clinical study, Gliovac (ERC 1671) administered with Leukine (sargramostim) as an adjuvant demonstrated low toxicity and resulted in an improved 40-week overall survival rate of 77% (n=9) vs. 10% (n=39) with historical controls in patients with recurrent glioblastoma multiforme (PMID: 25865468). | 25865468 | |
| Unknown unknown | ovarian cancer | not applicable | Olaparib + THZ1 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with Lynparza (olaparib) combined with THZ1 resulted in a synergistic effect in homologous recombination-proficient ovarian cancer cells, demonstrating decreased cell survival and reduced anchorage-independent growth in culture, and inhibition of tumor growth in cell line xenograft models (PMID: 32668240). | 32668240 | |
| Unknown unknown | breast cancer | not applicable | SL0101 | Preclinical - Cell culture | Actionable | In a preclinical study, SL0101 inhibited proliferation of a breast cancer cell line in culture (PMID: 15705904). | 15705904 | |
| Unknown unknown | glioblastoma | not applicable | WT2725 | Phase I | Actionable | In a Phase I trial, WT2725 treatment resulted in survival for more than a year in 33% (7/21) of patients with progressive or recurrent glioblastoma, with 3 patients survived over 18 months, 2 survived over 2 years (both in complete radiologic remission) (Journal of Clinical Oncology 35, no. 15_suppl (May 20 2017) 2066-2066; NCT01621542). | detail... | |
| Unknown unknown | glioblastoma | not applicable | LB-100 | Preclinical | Actionable | In a preclinical study, glioblastoma cells treated with a combination of LB100 and radiotherapy resulted in inhibition of Pp2a activity, decreased tumor growth, and increased cell survival (PMID: 25939762). | 25939762 | |
| Unknown unknown | glioblastoma | no benefit | Bevacizumab + Trebananib | Phase II | Actionable | In a Phase II trial (NRG/RTOG 1122), addition of Trebananib (AMG 386) to Avastin (bevacizumab) therapy was tolerable, but did not improve 6-month progression-free survival (PFS) rate (22.6%, 12/53 vs 41.1%, 23/56), median overall survival (7.5 vs 11.5 months), median PFS (4.2 vs 4.8 months, HR=1.51, p=0.04), or radiographic response rate (4.2% vs 5.9%) in patients with recurrent glioblastoma (PMID: 32154928; NCT01609790). | 32154928 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Carfilzomib + Tinostamustine | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of EDO-S101 and Kyprolis (carfilzomib) decreased viability of primary chronic lymphocytic leukemia cells in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | hematologic cancer | not applicable | AZD5991 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AZD5991 inhibited tumor growth in hematologic cancer cell line xenograft models (Cancer Res July 1 2017 (77) (13 Supplement) DDT01-02). | detail... | |
| Unknown unknown | hematologic cancer | not applicable | AZD5991 | Preclinical - Cell culture | Actionable | In a preclinical study, AZD5991 treatment induced caspase activation and apoptosis in hematologic cancer cell lines in culture (PMID: 31699827). | 31699827 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Mirvetuximab Soravtansine | Phase I | Actionable | In a Phase I trial, Mirvetuximab Soravtansine (IMGN853) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, with a clinical benefit rate of 23% (12/43), including partial response in 2 patients, both with epithelial ovarian cancer, CA125 response in 5 patients, and stable disease for greater than 4 months in 5 patients (PMID: 28440955). | 28440955 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Regorafenib | Phase I | Actionable | In a Phase I trial, Stivarga (regorafenib) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22421192). | 22421192 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Regorafenib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Stivarga (regorafenib) showed antitumor activity in multiple murine xenograft models derived from melanoma, renal cell carcinoma, colorectal, breast, lung, pancreatic and ovarian tumor cell lines (PMID: 21170960). | 21170960 | |
| Unknown unknown | prostate cancer | not applicable | Navitoclax + TAK-901 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of TAK-901 and Navitoclax (ABT-263) resulted in a synergistic effect, demonstrating reduced cell viability of prostate cancer cells in culture (PMID: 28179288). | 28179288 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Birinapant | Phase I | Actionable | In a Phase I dose escalation study of birinapant, 50 patients with advanced solid tumors or lymphoma were enrolled and no patients achieved complete or partial remission while stable disease was observed in 3 patients (PMID: 26333381). | 26333381 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | BAY1217389 + Paclitaxel | Preclinical | Actionable | In a preclinical study, the combination of BAY1217389 and Taxol (paclitaxel) resulted in enhanced growth inhibition compared to Taxol (paclitaxel) alone in non-small cell lung cancer xenograft models (PMID: 26832791). | 26832791 | |
| Unknown unknown | breast cancer | no benefit | LFA102 | Phase I | Actionable | In a Phase I trial, LFA102 treatment was well tolerated but did not demonstrate antitumor activity in Japanese patients with advanced breast cancer (7/14) or castration-resistant prostate cancer (7/14) (PMID: 32878811; NCT01610050). | 32878811 | |
| Unknown unknown | follicular lymphoma | not applicable | Bendamustine + Obinutuzumab | FDA approved | Actionable | In a Phase III trial that supported FDA approval, the combination of Gazyva (obinutuzumab) plus Treanda (bendamustine) resulted in a greater progression free survival (29.2 mo vs 14.0 mo) than Treanda (bendamustine) alone in patients with follicular lymphoma (PMID: 27345636). | 27345636 detail... | |
| Unknown unknown | colorectal cancer | not applicable | ETBX-011 | Phase Ib/II | Actionable | In a Phase I/II trial, ETBX-011 treatment resulted in an overall survival of 11 months, and survival at 12 months follow-up in 48% of patients with metastatic colorectal cancer (Journal of Clinical Oncology 32, no. 15_suppl (May 2014) 3093-3093; NCT01147965.). | detail... | |
| Unknown unknown | meningioma | not applicable | Everolimus + Octreotide | Phase II | Actionable | In a Phase II trial (CEVOREM), the combination therapy of Afinitor (everolimus) and Sandostatin Lar Depot (octreotide acetate) in patients with a recurrent meningioma resulted in a 6 month progression-free survival of 55% (11/20), 6 month and 12 month overall survivals of 90% (18/20) and 75% (15/20), respectively, and a decreased tumor growth rate in 78% of patients at 3 months (PMID: 31969329; NCT02333565). | 31969329 | |
| Unknown unknown | neuroblastoma | not applicable | GF109203X | Preclinical - Cell culture | Actionable | In a preclinical study, GF109203X decreased growth of neuroblastoma cell lines in culture (PMID: 10209967). | 10209967 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | YYB-101 | Phase I | Actionable | In a Phase I trial, YYB-101 demonstrated safety and preliminary efficacy in patients with refractory solid tumors, with partial response in 4.5% (1/22) and stable disease in 45.5% (10/22) of patients (J Clin Onc. 2018 36:15_suppl, e14501). | detail... | |
| Unknown unknown | B-cell lymphoma | not applicable | IT-901 | Preclinical | Actionable | In a preclinical study, IT-901 inhibited tumor growth in Epstein Barr Virus-induced B-cell lymphoma xenograft models (PMID: 26744524). | 26744524 | |
| Unknown unknown | renal cell carcinoma | no benefit | Bevacizumab + Carotuximab | Clinical Study | Actionable | In a clinical study, the addition of TRC105 to Avastin (bevacizumab) treatment in renal cell carcinoma patients did not result in improved progression free survival (2.8 mo vs 4.6 mo) when compared to Avastin (bevacizumab) alone (PMID: 28832978). | 28832978 | |
| Unknown unknown | acute leukemia | not applicable | Volasertib | Phase I | Actionable | In a Phase I trial, Volasertib (BI 6727) demonstrated safety and limited efficacy in pediatric patients with acute leukemia or advanced solid tumors, with stable disease as best objective response in 71% (5/7) of patients with acute leukemia and in 13% (2/15) of patients with advanced solid tumors (PMID: 31276318; NCT01971476). | 31276318 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Erlotinib + Pictilisib | Phase I | Actionable | In a Phase I trial, the combination treatment of Tarceva (erlotinib) and Pictilisib (GDC-0941) in patients with advanced solid tumors resulted in toxicity, requiring dose adjustments, and led to minimal antitumor activity including two partial responses and stable disease in nineteen patients (PMID: 28798270). | 28798270 | |
| Unknown unknown | peripheral T-cell lymphoma | not applicable | Duvelisib | Phase I | Actionable | In a Phase I trial, Copiktra (duvelisib) treatment resulted in an overall response rate of 50% (8/16, 3 complete response, 5 partial response) with a median duration of treatment of 11.3 weeks in patients with peripheral T-cell lymphoma (PMID: 29233821; NCT01476657). | 29233821 | |
| Unknown unknown | pancreatic ductal adenocarcinoma | not applicable | MN58b + Oxaliplatin | Preclinical | Actionable | In a preclinical study, the combination of MN58b and Eloxatin (oxaliplatin) worked synergistically to inhibit growth of pancreatic ductal adenocarcinoma cell lines in culture (PMID: 26769123). | 26769123 | |
| Unknown unknown | colon cancer | not applicable | Ch282-5 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, ch282-5 induced apoptosis and inhibited growth and migration of several human and mouse colon cancer cell lines in culture, and inhibited tumor growth and metastasis in xenograft models (PMID: 26515494). | 26515494 | |
| Unknown unknown | colorectal cancer | not applicable | AZD2461 + Temozolomide | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the addition of AZD2461 to Temodar (temozolomide) resulted in greater antitumor activity than Temodar (temozolomide) alone in colorectal cancer cell line xenograft models, demonstrating decreased tumor volume (PMID: 27550455). | 27550455 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BAY1161909 | Preclinical | Actionable | In a preclinical study, BAY1161909 inhibited proliferation of a variety of human solid tumor cell lines in culture (PMID: 26832791). | detail... 26832791 | |
| Unknown unknown | melanoma | not applicable | Pegilodecakin + Pembrolizumab | Phase I | Actionable | In a Phase Ib trial (IVY), Pegilodecakin (AM0010) and Keytruda (pembrolizumab) combination therapy demonstrated safety and preliminary efficacy, resulting in an objective response rate of 10% (3/31, 3 partial responses) and a disease control rate of 52% (16/31) in evaluable patients with melanoma, with a median duration of response not reached (PMID: 31563517; NCT02009449). | 31563517 | |
| Unknown unknown | renal cell carcinoma | not applicable | GDC-0349 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the mTOR inhibitor GDC-0349 demonstrated inhibition of tumor growth in cell line xenograft models of solid tumors, including renal adenocarcinoma (PMID: 24900569). | 24900569 | |
| Unknown unknown | gastroesophageal junction adenocarcinoma | not applicable | Lenvatinib + Pembrolizumab | Phase II | Actionable | In a Phase II trial (EPOC1706), the combination of Lenvima (lenvatinib) and Keytruda (pembrolizumab) treatment in patients with either stomach cancer or gastroesophageal junction cancer resulted in a 69% (20/29) objective response rate, a disease control rate of 100% (29/29), a median progression-free survival of 7.1 months, and a median overall survival that had not yet been reached, and 8 patients were still experiencing an ongoing response at data cut-off (PMID: 32589866; NCT03609359). | 32589866 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Irinotecan + Veliparib | Phase I | Actionable | In a Phase I trial, Veliparib (ABT-888) and Camptosar (irinotecan) combination therapy resulted in partial response in 19% (6/31) and stable disease in 42% (13/31) of patients with advanced solid tumors (PMID: 26842236). | 26842236 | |
| Unknown unknown | stomach cancer | not applicable | HD105 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, HD105 inhibited tumor progression in two of four human gastric cancer cell line xenograft models (PMID: 27049350). | 27049350 | |
| Unknown unknown | colorectal cancer | not applicable | Rucaparib + Temozolomide | Preclinical - Cell line xenograft | Actionable | In a preclinical trial, Rubraca (rucaparib) sensitized colorectal cancer cell lines to Temodar (temozolomide) treatment both in culture and in cell line xenograft models (PMID: 17363489). | 17363489 | |
| Unknown unknown | sarcoma | not applicable | NBTXR3 + Radiotherapy | Phase II | Actionable | In a Phase II/III trial, no difference in objective response rate was observed between soft tissue sarcoma patients treated with NBTXR3 plus radiotherapy vs. radiotherapy alone (7% vs. 10%, p=0.86), however, NBTXR3 plus radiotherapy resulted in an increased pathological complete response rate of 16% (14/87) vs. 8% (7/89), and a higher proportion of patients receiving NBTXR3 and radiotherapy demonstrated negative margins (84% (61/73) vs. 70% (57/82), p=0.30) (PMID: 31296491; NCT02379845). | 31296491 | |
| Unknown unknown | ovarian cancer | not applicable | Paclitaxel + TVB-2640 | Phase I | Actionable | In a Phase I trial, TVB-2640 and Taxol (paclitaxel) combination treatment resulted in 58-98% reduction of CA-125 level in 42% (5/12) of ovarian cancer patients (J Clin Oncol 34, 2016 (suppl; abstr 2512)). | detail... | |
| Unknown unknown | gastrointestinal system cancer | not applicable | Rivoceranib | Phase III | Actionable | In a Phase III trial, treatment with Apatinib (YN968D1) at 850mg resulted in a greater progression free survival (2.6 mo vs 1.8 mo) and overall survival (6.5 mo vs 4.7 mo) when compared to placebo in patients with either gastric cancer or gastroesophageal junction adenocarcinoma (PMID: 26884585). | 26884585 | |
| Unknown unknown | gastrointestinal system cancer | not applicable | Rivoceranib | Phase II | Actionable | In a Phase II trial, Apatinib (YN968D1) improved progression-free survival and overall survival in metastatic gastric cancer patients (PMID: 23918952). | 23918952 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Voxtalisib | Phase I | Actionable | In a Phase I trial, SAR245409 (XL765) reduced PI3K and mTORC1/mTORC2 pathway signaling and demonstrated safety and efficacy irrespective of molecular alterations in the PI3K pathway, in patients with advanced solid tumors (PMID: 24583798). | 24583798 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Voxtalisib | Phase I | Actionable | In a Phase I trial, SAR245409 (XL765) demonstrated safety and efficacy in patients with solid tumors (PMID: 18959794). | 18959794 | |
| Unknown unknown | osteosarcoma | not applicable | Pembrolizumab | Phase II | Actionable | In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 5% (1/22) of patients with osteosarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). | detail... | |
| Unknown unknown | prostate cancer | not applicable | Mogamulizumab + Tremelimumab | Case Reports/Case Series | Actionable | In a Phase I trial, a prostate cancer patient achieved a partial response after 1.84 months with a response duration of 3.7 months following treatment with the combination of Poteligeo (mogamulizumab-kpkc) and Tremelimumab (CP-675206) (PMID: 32586937; NCT02301130). | 32586937 | |
| Unknown unknown | extraosseous Ewing sarcoma | not applicable | GSK1838705A | Preclinical | Actionable | In a preclinical study, multiple cancer cell lines including multiple myeloma and Ewing's sarcoma were sensitive to GSK1838705A (PMID: 19825801). | 19825801 | |
| Unknown unknown | lung carcinoma | not applicable | YW3-56 | Preclinical | Actionable | In a preclinical study, YW3-56 inhibited proliferation of lung carcinoma cell lines in culture, independent of TP53 status (PMID: 25612620). | 25612620 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Irinotecan + SR-4835 | Preclinical - Pdx | Actionable | In a preclinical study, SR-4835 and Camptosar (irinotecan) combination treatment induced DNA damage and cell death, inhibited tumor growth, leading to tumor regression in a patient-derived xenograft (PDX) model of triple-negative breast cancer harboring a BRCA1 mutation (PMID: 31668947). | 31668947 | |
| Unknown unknown | Ewing sarcoma | not applicable | SN-38 + Veliparib | Preclinical | Actionable | In a preclinical study, Ewing sarcoma cells treated with SN-38 combined with Veliparib (ABT-888) resulted in synergism, demonstrating reduced cell viability in culture (PMID: 26438158). | 26438158 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Metformin + Temsirolimus | Phase I | Actionable | In a Phase I clinical trial, the combination of Torisel (temsirolimus) and Glucophage (metformin) demonstrated safety and resulted in a clinical benefit rate of 22% in patients with advanced or refractory tumors, with 4/18 patients achieving stable disease for greater than 6 cycles (PMID: 27014780). | 27014780 | |
| Unknown unknown | Hodgkin's lymphoma | not applicable | Brentuximab vedotin + Umbralisib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Ukoniq (umbralisib) and Brentuximab vedotin synergized to inhibit proliferation of several human Hodgkin's lymphoma cell lines in culture and to suppress tumor growth in xenograft models (Blood 124(21): 4486). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Utomilumab | Phase I | Actionable | In a Phase I trial, Utomilumab (PF-05082566) treatment resulted in an overall objective response rate of 3.8% (2/53), a median progression-free survival of 1.7 months, and a median overall survival of 11.2 months in patients with advanced solid tumors (PMID: 29549159; NCT01307267). | 29549159 | |
| Unknown unknown | head and neck cancer | not applicable | AZD7648 + Olaparib | Preclinical - Pdx | Actionable | In a preclinical study, AZD7648 and Lynparza (olaparib) combination treatment inhibited tumor growth in a patient-derived xenograft (PDX) model of head and neck cancer harboring mutant TP53 and wild-type ATM (PMID: 31699977). | 31699977 | |
| Unknown unknown | melanoma | not applicable | V158411 | Preclinical - Cell culture | Actionable | In a preclinical study, V158411 induced DNA damage and cell-cycle arrest, and inhibited growth of a melanoma cell line in culture (PMID: 27829224). | 27829224 | |
| Unknown unknown | glioblastoma | not applicable | Olaparib + Radiotherapy | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with Lynparza (olaparib) enhanced the sensitivity of glioblastoma cell lines to radiation therapy in culture, demonstrating greater decreased cell survival when compared to cells not treated with Lynparza (olaparib) (PMID: 32347934). | 32347934 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Milademetan Tosylate | Phase I | Actionable | In a Phase I trial, DS-3032b demonstrated safety and some preliminary activity in patients with advanced solid tumors, with 77% (20/26) of patients achieving stable disease (J Clin Oncol 34, 2016 (suppl; abstr 2581)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | IHSF115 | Preclinical - Cell culture | Actionable | In a preclinical study, IHSF115 inhibited growth of a panel of cancer cell lines in culture (PMID: 28369544). | 28369544 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | GSK1070916 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, GSK1070916 inhibited proliferation of a variety of advanced solid tumors in cell culture and in xenografts (PMID: 19567821). | 19567821 | |
| Unknown unknown | primary mediastinal B-cell lymphoma | not applicable | Lisocabtagene maraleucel | FDA approved | Actionable | In a Phase I trial (TRANSCEND NHL 001) that supported FDA approval, Breyanzi (lisocabtagene maraleucel) treatment resulted in an objective response in 73% (186/256, 136 complete responses) of patients with relapsed or refractory large B-cell lymphoma, including primary mediastinal B-cell lymphoma, high-grade B-cell lymphoma, diffuse large B-cell lymphoma, and follicular lymphoma grade 3B (PMID: 32888407; NCT02631044). | 32888407 detail... | |
| Unknown unknown | breast cancer | not applicable | SKLB-23bb | Preclinical - Cell line xenograft | Actionable | In a preclinical study, SKLB-23bb treatment inhibited tumor growth in breast cancer xenograft models (PMID: 29610282). | 29610282 | |
| Unknown unknown | transitional cell carcinoma | not applicable | Avelumab | FDA approved | Actionable | In a Phase III trial (JAVELIN Bladder 100) that supported FDA approval, addition of maintenance Bavencio (avelumab) to best supportive care (BSC) significantly improved overall survival compared to BSC alone (21.4 vs 14.3 mo, HR=0.69, p=0.0005) in patients with advanced urothelial carcinoma (J Clin Oncol 38, (Jun 2020) no. 18_suppl; NCT02603432). | detail... detail... | |
| Unknown unknown | transitional cell carcinoma | not applicable | Avelumab | FDA approved | Actionable | In a Phase I trial (JAVELIN Solid Tumor) that supported FDA approval, Bavencio (avelumab) demonstrated safety and resulted in a response rate of 17% (27/161; 9 complete responses and 18 partial responses), a median progression-free survival of 6.3 weeks, and a median overall survival of 6.5 months in patients with platinum-refractory metastatic urothelial carcinoma (PMID: 29217288; NCT01772004). | detail... 29217288 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Paclitaxel + Plicamycin | Preclinical | Actionable | In a preclinical study, Mithracin (plicamycin) and Taxol (paclitaxel) worked synergistically to inhibit the growth of triple-receptor negative breast cancer cell lines in culture (PMID: 20576088). | 20576088 | |
| Unknown unknown | colorectal cancer | not applicable | Resminostat | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Resminostat (4SC-201) inhibited proliferation and induced cell-cycle arrest and apoptosis in colorectal cancer (CRC) cell lines and primary colon cancer cells in culture, and inhibited tumor growth in a CRC cell line xenograft model (PMID: 26831668). | 26831668 | |
| Unknown unknown | malignant mesothelioma | not applicable | MGD013 | Case Reports/Case Series | Actionable | In a Phase I trial, MGD013 treatment resulted in a partial response in a patient with mesothelioma (J Clin Oncol 38: 2020 (suppl; abstr 3004); NCT03219268). | detail... | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Droxinostat | Preclinical - Cell culture | Actionable | In a preclinical study, droxinostat induced apoptosis and reduced growth of hepatocellular carcinoma cell lines in culture (PMID: 26947884). | 26947884 | |
| Unknown unknown | breast cancer | not applicable | Irinotecan + Veliparib | Phase I | Actionable | In a Phase I trial, Veliparib (ABT-888) and Camptosar (irinotecan) combination therapy resulted in partial response in 33% (3/9) of patients with advanced breast cancer (PMID: 26842236). | 26842236 | |
| Unknown unknown | breast cancer | not applicable | SKI-G-801 | Preclinical | Actionable | In a preclinical study, SKI-G-801 treatment reduced tumor growth in a syngeneic mouse model of breast cancer (Cancer Res 2019;79(13 Suppl):Abstract nr 2010). | detail... | |
| Unknown unknown | breast cancer | not applicable | AsiDNA + Veliparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA and Veliparib (ABT-888) combination treatment resulted in increased cell death and inhibition of proliferation in breast cancer cell lines compared to Veliparib (ABT-888) alone in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | colorectal cancer | not applicable | ST-162 + unspecified PD-1 antibody | Preclinical | Actionable | In a preclinical study, ST-162 therapy combined with an anti-PD-1 antibody resulted in greater tumor growth inhibition than treatment with either agent alone in a colorectal cancer mouse model (PMID: 28775144). | 28775144 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CX-072 + Ipilimumab | Phase II | Actionable | In a Phase II trial (PROCLAIM-CX-072), CX-072 and Yervoy (ipilimumab) combination treatment demonstrated acceptable safety, and resulted in a disease control rate of 37% (10/27, 1 complete response, 4 partial responses) in patients with advanced solid tumors (J Clin Oncol 38: 2020 (suppl; abstr 3005); NCT03993379). | detail... | |
| Unknown unknown | pancreatic cancer | not applicable | SF2523 | Preclinical | Actionable | In a preclinical study, SF2523 decreased tumor immunosuppression and inhibited tumor growth in orthotopic mouse pancreatic cancer models (PMID: 31018997). | 31018997 | |
| Unknown unknown | prostate cancer | no benefit | ASG-5ME | Phase I | Actionable | In a Phase I trial, ASG-5ME demonstrated preliminary efficacy in patients with metastatic castration-resistant prostate cancer, but was not developed further due to unfavorable pharmacokinetic and toxicity profile (PMID: 30725389). | 30725389 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Docetaxel + MLN4924 | Phase I | Actionable | In a Phase Ib trial, the combination therapy of Pevonedistat (MLN4924) and Taxotere (docetaxel) was well-tolerated and demonstrated safety in advanced solid tumor patients, and resulted in an overall response rate of 16% (3/19), including a partial response in a cholangiocarcinoma patient and in two patients with head and neck cancer (PMID: 29781056; NCT01862328). | 29781056 | |
| Unknown unknown | renal cell carcinoma | not applicable | AGS16F | Phase I | Actionable | In a Phase I trial, treatment with AGS16F (AGS-16CSF) at the recommended phase 2 dose demonstrated safety and resulted in a partial response in 23% (3/13) of patients with metastatic renal cell carcinoma including 2 patients with clear cell and 1 patient with papillary histology, and a disease control rate of 92% (12/13) (PMID: 29848572). | 29848572 | |
| Unknown unknown | stomach cancer | not applicable | AK963 | Preclinical - Cell culture | Actionable | In a preclinical study, AK963 inhibited Pak1 kinase activity and downstream signaling, led to cell cycle arrest and inhibition of proliferation, colony formation, and invasion of human gastric cancer cells in culture (PMID: 30773850). | 30773850 | |
| Unknown unknown | gastroesophageal junction adenocarcinoma | no benefit | Oxaliplatin + Ramucirumab + Tegafur-gimeracil-oteracil Potassium | Phase II | Actionable | In a Phase II trial, adding Cyramza (ramucirumab) to chemotherapy consisting of TS-1 and Eloxatin (oxaliplatin) did not improve progression-free survival (6.34 vs 6.74 months), objective response rate (58.2% vs 50.%), and disease control rate (90.9% vs 87.0%) compared to placebo in patients with advanced gastric or gastroesophageal junction adenocarcinoma (J Clin Oncol 36, 2018 (suppl; abstr 4036); NCT02539225). | detail... | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | GEN3009 | Preclinical - Patient cell culture | Actionable | In a preclinical study, GEN3009 treatment induced complement-dependent cytotoxicity in cells derived from chronic lymphocytic leukemia patients in culture (PMID: 32341336). | 32341336 | |
| Unknown unknown | melanoma | not applicable | SF2523 | Preclinical | Actionable | In a preclinical study, SF2523 reduced tumor growth and decreased lung metastasis in syngeneic mouse melanoma models (PMID: 31018997). | 31018997 | |
| Unknown unknown | ovarian cancer | not applicable | Conatumumab + Ganitumab | Phase Ib/II | Actionable | In a Phase Ib/II clinical trial, Ganitumab and Conatumumab combination treatment resulted in stable disease in 56% (5/9) of patients with ovarian cancer (PMID: 24816908). | 24816908 | |
| Unknown unknown | acute myeloid leukemia | not applicable | CPI-0610 + Doxorubicin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination treatment of CPI-0610 and Adriamycin (doxorubicin) resulted in inhibition of tumor growth in acute myeloid leukemia xenograft models (PMID: 26815195). | 26815195 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | BMS-986178 + Ipilimumab | Phase Ib/II | Actionable | In Phase I/II trial, BMS-986178 and Yervoy (ipilimumab) combination therapy demonstrated manageable safety profile, resulted in an objective response rate of 0% (0/34; 0/6) in different cohorts of patients with advanced solid tumors (PMID: 33148673; NCT02737475). | 33148673 | |
| Unknown unknown | pancreatic cancer | not applicable | CG200745 | Preclinical - Cell culture | Actionable | In a preclinical study, CG200745 decreased viability of pancreatic cancer cell lines in culture, including Gemzar (gemcitabine)-resistant cell lines (PMID: 28134290). | 28134290 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | unspecified PD-L1 antibody | Clinical Study | Actionable | In a retrospective analysis, treatment with an unspecified PD-1 or PD-L1 therapy in metastatic non-small cell lung cancer patients harboring a mutation in BRAF, ERBB2 (HER2), or MET, or a RET translocation led to a response rate of 29% (31/107), a 15.4 mo duration of response, a 4.7 mo median progression-free survival, and a 16.2 mo median overall survival, and resulted in clinical efficacy similar to what has been observed in studies with unselected non-small cell lung cancer patients (PMID: 31945494). | 31945494 | |
| Unknown unknown | acute myeloid leukemia | not applicable | AKN-028 | Preclinical - Cell culture | Actionable | In a preclinical study, AKN-028 treatment induced apoptosis in acute myeloid leukemia cells in culture and inhibited growth and resulted in decreased tumor mass in acute myeloid leukemia cell line xenograft models (PMID: 22864397). | 22864397 | |
| Unknown unknown | pancreatic ductal adenocarcinoma | not applicable | Arsenic trioxide + Chloroquine + JQ1 | Preclinical - Cell culture | Actionable | In a preclinical study, JQ1, Trisenox (arsenic trioxide) and Chloroquine combination treatment reduced viability of Trisenox (arsenic trioxide)-insensitive pancreatic ductal adenocarcinoma cell lines in culture (PMID: 31420604). | 31420604 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Sabatolimab + Spartalizumab | Phase Ib/II | Actionable | In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in partial response in 5% (4/86) and stable disease in 40% (34/86) of patients with advanced solid tumor (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). | detail... | |
| Unknown unknown | melanoma | not applicable | Prolgolimab | Phase II | Actionable | In a Phase II trial (MIRACULUM), Prolgolimab (BCD-100) treatment in advanced melanoma patients led to an objective response rate (ORR) of 38.1% (24/63, 5 complete response (CR), 19 partial response (PR)), disease control rate (DCR) of 63.5%, median progression-free survival (mPFS) of 6.6 mo., and 2-year overall survival (OS) of 57.1% when administered at 1mg/kg Q2W, and 28.6% ORR (18/63, 2 CR, 16 PR), 46% DCR, mPFS of 3.7 mo., and 46% 2-year OS when administered at 3mg/kg Q3W (PMID: 33872982; NCT03269565). | 33872982 | |
| Unknown unknown | rectum cancer | not applicable | Nelfinavir + Radiotherapy | Phase I | Actionable | In a Phase I trial, the combination of Nelfinavir and radiotherapy resulted in median tumor cell density reduction from 24.3% at baseline to 9.2%, and tumor regression in 56% (5/9) of rectal cancer patients (PMID: 26861457). | 26861457 | |
| Unknown unknown | renal cell carcinoma | not applicable | Abexinostat + Pazopanib | Phase Ib/II | Actionable | In a Phase Ib/II trial, 27% (6/22) of renal cell carcinoma patients demonstrated a response when treated with a combination of Abexinostat (PCI-24781) and Votrient (pazopanib) (PMID: 28221861). | 28221861 | |
| Unknown unknown | hematologic cancer | not applicable | BP1001 | Phase I | Actionable | In a Phase I/Ib trial, BP1001 treatment demonstrated safety and preliminary efficacy, resulting in clinical benefit and extended treatment cycle in 22% (7/32) of patients with refractory or relapsed hematological malignancies (PMID: 29550383; NCT01159028). | 29550383 | |
| Unknown unknown | chronic myelomonocytic leukemia | not applicable | H3B-8800 | Preclinical - Pdx | Actionable | In a preclinical study, H3B-8800 treatment reduced tumor burden in patient-derived xenograft (PDX) models of chronic myelomonocytic leukemia harboring spliceosome mutations but not spliceosome wild-type models (Blood 2016 128:966). | detail... | |
| Unknown unknown | thyroid gland cancer | not applicable | Bevacizumab | Preclinical | Actionable | In a preclinical study, Avastin (bevacizumab) inhibited tumor growth and angiogenesis in mouse models of anaplastic thyroid cancer (PMID: 17429874). | 17429874 | |
| Unknown unknown | colorectal cancer | not applicable | Bevacizumab + Fluorouracil + Irinotecan + Leucovorin + Oxaliplatin | Phase II | Actionable | In a Phase II trial, the combination of Avastin (bevacizumab) plus FOLFOXIRI chemotherapy was well-tolerated and resulted in improved progression-free survival compared to Avastin (bevacizumab) plus FOLFOX in colorectal cancer patients (J Clin Oncol 35, 2017 (suppl 4S; abstract 658)). | detail... | |
| Unknown unknown | lung carcinoma | not applicable | TP-1454 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TP-1454 treatment inhibited viability of an epithelial lung carcinoma cell line in culture, and reduced tumor growth in a cell line xenograft model (Mol Cancer Ther 2019;18(12 Suppl):Abstract nr B080). | detail... | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Prexasertib | Preclinical - Cell culture | Actionable | In a preclinical study, head and neck squamous cell carcinoma cell lines, either human papilloma virus positive or negative, demonstrated decreased cell proliferation in culture when treated with Prexasertib (LY2606368) (PMID: 28138028). | 28138028 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Prexasertib | Phase I | Actionable | In a Phase I trial, Prexasertib (LY2606368) treatment resulted in partial response in a patient with head and neck squamous cell carcinoma (PMID: 27044938; NCT0115790). | 27044938 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Prexasertib | Phase Ib/II | Actionable | In a Phase Ib trial, treatment with Prexasertib (LY2606368) resulted in an overall response rate of 5% (3/57, all partial responses), clinical benefit rate (complete response+partial response+stable disease) of 49% (28/57), and a median progression-free survival of 1.6 months in patients with head and neck squamous cell carcinoma (PMID: 29643063; NCT0115790). | 29643063 | |
| Unknown unknown | subependymal giant cell astrocytoma | not applicable | Everolimus | FDA approved | Actionable | In a Phase III trial (EXIST-1) that supported FDA approval, Afinitor (everolimus) treatment resulted in a 50% or more tumor reduction in 35% (27/78) of adult and pediatric patients diagnosed with tuberous sclerosis complex and had subependymal giant cell astrocytoma, compared to 0% (0/39) in the placebo group (PMID: 23158522; NCT00789828). | 23158522 detail... | |
| Unknown unknown | urinary bladder cancer | not applicable | BMS-986205 + Nivolumab | Phase I | Actionable | In a Phase I/II trial, BMS-986205 in combination with Opdivo (nivolumab) resulted in an objective response rate of 32% (8/25) and a durable response rate of 44% (11/25) in patients with bladder cancer (PMID: 29167110). | 29167110 | |
| Unknown unknown | invasive bladder transitional cell carcinoma | not applicable | Cisplatin + Gemcitabine + Sorafenib | Phase II | Actionable | In a Phase II trial, Nexavar (sorafenib) in combination with Platinol (cisplatin) and Gemzar (gemcitabine) resulted in pathologic complete response in 42.2% (19/45) of patients with muscle-invasive urothelial bladder cancer (J Clin Oncol 35, 2017 (suppl 6S; abstract 345)). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | Daunorubicin + VS-II-173 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination therapy of Cerubidine (daunorubicin) and VS-II-173 resulted in a synergistic effect in acute myeloid leukemia cells, demonstrating an increase in cell death from 40% to 80% in culture (PMID: 30679386). | 30679386 | |
| Unknown unknown | uveal melanoma | not applicable | AU-011 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with AU-011 resulted in cell binding and subsequent cell death in uveal melanoma cells in culture (Cancer Res 2014;74(19 Suppl):Abstract nr 1770). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | NSC-CRAd-Survivin-pk7 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, NSC-CRAd-Survivin-pk7 therapy resulted in decreased growth of ovarian cancer cells in culture and reduced tumor burden in cell line xenograft models (PMID: 30719498). | 30719498 | |
| Unknown unknown | colorectal cancer | not applicable | Trifluridine-tipiracil hydrochloride | FDA approved | Actionable | In a Phase III trial supporting FDA approval, Lonsurf (trifluridine/tipiracil hydrochloride) demonstrated improved median overall survival (7.1 vs 5.3 months), median progression free survival (2.0 vs 1.7 months), and 32% reduction of death risk compared to placebo in treatment-refractory metastatic colorectal cancer patients (PMID: 27126991). | 27126991 detail... | |
| Unknown unknown | Advanced Solid Tumor | no benefit | MEDI1873 | Phase I | Actionable | In a Phase I trial, MEDI1873 (Efgivanermin) demonstrated safety but a lack of tumor response, resulting in stable disease (SD) as best overall response of in 42.5% (17/40) of patients with advanced solid tumors, with SD for 24 weeks or more in 20% (8/40), SD for 52.5 weeks or more in 7.5% (3/40) of the patients, and SD in 35% (7/20) of colorectal cancer patients including 15% (3/20) with SD lasting 24 weeks or more, further development was not planned (PMID: 32887725; NCT02583165). | 32887725 | |
| Unknown unknown | pancreatic cancer | not applicable | CEP1347 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CEP1347 induced differentiation and inhibited self-renewal in pancreatic cancer stem cells in culture, and pretreatment of pancreatic cancer stem cell lines with CEP1347 in culture inhibited tumor growth in xenograft models (PMID: 29212273). | 29212273 | |
| Unknown unknown | pancreatic ductal adenocarcinoma | not applicable | Hydroxychloroquine + Trametinib | Case Reports/Case Series | Actionable | In a clinical case study, combination treatment with Mekinist (trametinib) and Hydroxychloroquine in a patient with refractory metastatic pancreatic ductal adenocarcinoma resulted in a partial response, with a 95% decrease in the cancer antigen 19-9 after 2 months, and a 50% reduction in tumor burden at 4 months (PMID: 30833748). | 30833748 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Docetaxel + DT2216 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the addition of DT2216 to Taxotere (docetaxel) treatment resulted in decreased viability of a triple-negative breast cancer cell line in culture, and resulted in increased tumor growth inhibition in xenograft models compared to either agent alone (PMID: 31792461). | 31792461 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | DLYE5953A | Case Reports/Case Series | Actionable | In a Phase I trial, a patient with non-small cell lung carcinoma expressing Ly6e (IHC 3+) who initially responded to prior treatments including chemotherapy regimens and Opdivo (nivolumab), but then progressed, achieved a partial response with a 46% decrease in tumor size after 2 cycles of DLYE5953A treatment, but treatment was discontinued after cycle 6 due to disease progression (PMID: 32694157; NCT02092792). | 32694157 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | LAM-002A | Phase I | Actionable | In a Phase I trial, LAM-002A demonstrated safety and preliminary efficacy, resulted in prolonged stable disease in 1 of 4 patients with chronic lymphocytic leukemia (Blood 2017 130 (Suppl 1):4119). | detail... | |
| Unknown unknown | thyroid gland cancer | not applicable | ABT-737 + Doxorubicin | Preclinical | Actionable | In a preclinical study, the combination of ABT-737 and Adriamycin (doxorubicin) was synergistic towards inhibiting cell viability of a majority of human thyroid cancer cell lines tested in culture (PMID: 27042160). | 27042160 | |
| Unknown unknown | kidney rhabdoid cancer | not applicable | UAB30 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a human malignant rhabdoid kidney tumor cell line was sensitive to UAB30 in both culture and in xenograft models, demonstrating cell-cycle arrest, decreased cell proliferation, apoptosis, and reduced tumor growth (PMID: 26873726). | 26873726 | |
| Unknown unknown | chondrosarcoma | not applicable | Dasatinib | Phase II | Actionable | In a Phase II trial, patients with chondrosarcoma demonstrated a median progression free survival of 5.5 months and six patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). | 27696380 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CYC140 | Preclinical - Cell culture | Actionable | In a preclinical study, CYC140 treatment resulted in cell cycle arrest, complete growth inhibition, and apoptosis in cancer cell lines (Cancer Res 2017;77(13 Suppl):Abstract nr 4178). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | Dexamethasone + Lenalidomide + Ulocuplumab | Phase I | Actionable | In a Phase I trial, Ulocuplumab (BMS-936564) in combination with Revlimid (lenalidomide) and Adexone (dexamethasone) demonstrated safety and preliminary efficacy, resulted in an overall response rate of 55.2% (16/29) and a clinical benefit rate of 72.4% (21/29) in patients with relapsed multiple myeloma (PMID: 31672767). | 31672767 | |
| Unknown unknown | multiple myeloma | not applicable | Bortezomib + Mivebresib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Velcade (bortezomib) and Mivebresib (ABBV-075) resulted in increased tumor growth inhibition in a multiple myeloma cell line xenograft model compared to Velcade (bortezomib) alone (PMID: 28416490). | 28416490 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | Cobimetinib + Ravoxertinib | Phase I | Actionable | In a Phase Ib trial, treatment with the combination of Ravoxertinib (GDC-0994) and Cotellic (cobimetinib) in patients with advanced solid tumors resulted in stable disease in 29% (7/24) of patients and one unconfirmed partial response in a patient with pancreatic adenocarcinoma; however, the study was terminated due to intolerable toxicity of the treatment combination observed in patients (PMID: 32311798; NCT02457793). | 32311798 | |
| Unknown unknown | uterus leiomyosarcoma | not applicable | Pazopanib | Clinical Study | Actionable | In a retrospective clinical study, 33% (9/27) of patients with uterine leiomyosarcoma responded to Votrient (pazopanib) treatment (PMID: 29185261). | 29185261 | |
| Unknown unknown | renal cell carcinoma | not applicable | Sonepcizumab | Phase II | Actionable | In a Phase II trial, ASONEP (sonepcizumab) treatment resulted in a median overall survival of 21.7 months and partial response in 10% (4/40) of renal cell carcinoma patients (PMID: 27727447). | 27727447 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Alisertib | Phase I | Actionable | In a Phase I study, Alisertib (MLN8237) treatment after Cytosar-U (cytarabine) and Idarubicin induction resulted in a composite remission rate (complete remission and complete remission with incomplete neutrophil recovery) of 86% (19/22) in patients with acute myeloid leukemia (PMID: 28034990). | 28034990 | |
| Unknown unknown | colon carcinoma | not applicable | DRP-104 + unspecified PD-1 antibody | Preclinical | Actionable | In a preclinical study, DRP-104 treatment in combination with an anti-PD-1 antibody resulted in enhanced tumor growth inhibition and survival in a syngeneic mouse model of colon carcinoma (J Immunother Cancer. 2019; 7(Suppl 1): 282, Abs nr: P497). | detail... | |
| Unknown unknown | breast cancer | not applicable | Paclitaxel + TVB-2640 | Phase I | Actionable | In a Phase I trial, TVB-2640 and Taxol (paclitaxel) combination treatment resulted in partial response in 8% (1/12) and stable disease for more than 20 weeks in 25% (3/12) of breast cancer patients (J Clin Oncol 34, 2016 (suppl; abstr 2512)). | detail... | |
| Unknown unknown | renal cell carcinoma | not applicable | Axitinib + Carotuximab | Phase I | Actionable | In a Phase I trial, treatment the combination of Inlyta (axitinib) and Carotuximab (TRC-105) demonstrated preliminary activity in patients with renal cell carcinoma, resulting in partial response in 5 and stable disease in 10 of 17 evaluable patients, and a median progression-free survival of 11.3 months (PMID: 30190302; NCT01806064). | 30190302 | |
| Unknown unknown | marginal zone B-cell lymphoma | not applicable | axicabtagene ciloleucel | Phase II | Actionable | In a Phase II trial (ZUMA-5), Yescarta (axicabtagene ciloleucel) treatment resulted in an objective response rate of 86% (6/7), with complete response in 71% (5/7) of patients with relapsed or refractory marginal zone lymphoma who have received two or more lines of therapies (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 8008-8008; NCT03105336). | detail... | |
| Unknown unknown | breast cancer | not applicable | Doxorubicin + NU6027 | Preclinical - Cell culture | Actionable | In a preclinical study, NU6027 enhanced the efficacy of Adriamycin (doxorubicin) in breast cancer cells in culture, resulting in decreased cell survival (PMID: 21730979). | 21730979 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Pimitespib | Phase I | Actionable | In a Phase I trial, TAS-116 demonstrated safety and resulted in a disease control rate of 27% (16/60; including stable disease for greater than or equal to 12 weeks (13) and partial responses (3)), in patients with advanced solid tumors, with partial responses in 2 patients with non-small cell lung cancer and 1 patient with gastrointestinal stromal tumor (PMID: 30679388; NCT02965885). | 30679388 | |
| Unknown unknown | pancreatic cancer | not applicable | Foretinib | Preclinical | Actionable | In a preclinical study, Foretinib (GSK1363089) treatment resulted in regression of the tumor vasculature, extensive hypoxia, apoptosis, and decreased tumor aggressiveness in a transgenic mouse model of pancreatic islet cancer (PMID: 21613405). | 21613405 | |
| Unknown unknown | breast cancer | not applicable | Sabatolimab | Case Reports/Case Series | Actionable | In a Phase Ib/II trial, MBG453 treatment resulted in stable disease in 2 patients with breast cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). | detail... | |
| Unknown unknown | peritoneal carcinoma | no benefit | Motolimod + Pegylated liposomal-doxorubicin | Phase II | Actionable | In a Phase II trial, the inclusion of Motolimod (VTX-2337) with Doxil (pegylated liposomal-doxorubicin) did not result in improved survival in patients with either ovarian, fallopian tube, or primary peritoneal carcinoma (PMID: 28453702; NCT01666444). | 28453702 | |
| Unknown unknown | multiple myeloma | not applicable | DT204 + Ixazomib | Preclinical - Cell culture | Actionable | In a preclinical study, multiple myeloma cells resistant to Ninlaro (ixazomib) demonstrated re-sensitization to Ninlaro (ixazomib) in culture when treatment was combined with DT204, showing a synergistic effect and increased apoptotic activity (PMID: 27677741). | 27677741 | |
| Unknown unknown | pancreatic adenocarcinoma | not applicable | GSK3203591 + GSK3368715 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of GSK3203591 and GSK3368715 inhibited tumor growth in a pancreatic adenocarcinoma cell line xenograft model, demonstrating increased efficacy over either agent alone (PMID: 31257072). | 31257072 | |
| Unknown unknown | ovarian cancer | not applicable | ENMD-2076 | Phase II | Actionable | In a Phase II clinical trial, ENMD-2076 demonstrated efficacy in patients with recurrent, platinum-resistant ovarian, fallopian tube or peritoneal cancers (PMID: 22921155). | 22921155 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | XL999 | Phase II | Actionable | In six Phase II clinical trials, XL999 demonstrated anti-tumor activity in patients with advanced solid tumors, but also resulted in significant cardiotoxicity, which improved after discontinuation of the drug (J Clin Oncol, 25:18s, 2007 (Suppl; abstr 3591)). | detail... | |
| Unknown unknown | lung small cell carcinoma | not applicable | Doxorubicin + Lurbinectedin | Phase I | Actionable | In a Phase I trial, Lurbinectedin (PM01183) and Adriamycin (doxorubicin) combination treatment resulted in complete response in 8% (2/27) and partial response in 50% (13/27) of patients with relapsed small-cell lung cancer (PMID: 28961837). | 28961837 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | INCB053914 + Parsaclisib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Parsaclisib (INCB050465) and INCB053914 synergized to inhibit proliferation of diffuse large B-cell lymphoma cells in culture and suppress tumor growth in xenograft models, and demonstrated improved efficacy over either agent alone (PMID: 29927999). | 29927999 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Alpelisib | Phase I | Actionable | In a Phase I clinical trial, Alpelisib (BYL719) demonstrated safety and some efficacy in patients with advanced solid tumors (PMID: 24617631). | 24617631 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Alpelisib | Phase I | Actionable | In a Phase I clinical trial, Alpelisib (BYL719) treatment was well tolerated in Japanese patients with advanced solid tumors and resulted in a 3% (1/33) objective response rate and a disease control rate of 57.6% (19/33, 1 partial response, 18 stable disease); in patients with PIK3CA mutations or amplification disease control rates of 75.0% (6/8) were observed vs. 78.6% (11/14) in all patients (PMID: 30588709; NCT01387321). | 30588709 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | AZD8330 | Phase I | Actionable | In a Phase I trial, AZD8330 demonstrated safety and some preliminary clinical activity in patients with advanced solid tumors (PMID: 23433846). | 23433846 | |
| Unknown unknown | renal cell carcinoma | not applicable | Axitinib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Inlyta (axitinib) as second-line therapy resulted in an improved progression-free survival of 8.3 months compared to 5.7 months with Nexavar (sorafenib) (HR 0.656, 95% CI 0.552-0.779; p<0.0001) in patients with metastatic renal cell carcinoma (PMID: 23598172). | 23598172 detail... | |
| Unknown unknown | renal cell carcinoma | not applicable | Axitinib | Clinical Study | Actionable | In a meta-analysis, Inlyta (axitinib) improved progression-free survival in patients with metastatic renal cell carcinoma (PMID: 24037486). | 24037486 | |
| Unknown unknown | renal cell carcinoma | not applicable | Axitinib | Phase II | Actionable | In a Phase II clinical trial, treatment with Inlyta (axtinib) as first-line therapy resulted a prolonged median overall survival of 42.7 months compared to 30.4 months with placebo in patients with metastatic renal cell carcinoma (PMID: 27236772). | 27236772 | |
| Unknown unknown | lung carcinoma | not applicable | INCB001158 | Preclinical | Actionable | In a preclinical study, INCB001158 (CB-1158) had no effect on Lewis Lung carcinoma cells growth in culture, but inhibited tumor growth in syngeneic animal models through regulation of host immune response (Cancer Res 2016;76(14 Suppl):Abstract nr 552). | detail... | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Selumetinib + SHP099 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination therapy of SHP099 and Selumetinib (AZD6244) led to decreased cell viability and reduced colony formation in triple-receptor negative breast cancer cells in culture and tumor regression in xenograft models (PMID: 30045908). | 30045908 | |
| Unknown unknown | breast cancer | not applicable | Altiratinib | Preclinical | Actionable | In a preclinical study, Altiratinib (DCC-0701) inhibited tumor growth and decreased lung metastasis in a mouse breast cancer model (PMID: 26285778). | 26285778 | |
| Unknown unknown | gastric adenocarcinoma | not applicable | Paclitaxel + Ramucirumab | FDA approved | Actionable | In a Phase III trial (RAINBOW) that supported FDA approval, Cyramza (ramucirumab) and Taxol (paclitaxel) combination treatment significantly improved overall survival (9.6 vs 7.4 mo, HR=0.807, p=0.017) compared to Taxol (paclitaxel) alone in patients with advanced gastric or gastroesophageal junction adenocarcinoma who progressed on prior chemotherapy (PMID: 25240821; NCT01170663). | 25240821 detail... | |
| Unknown unknown | endometrial cancer | not applicable | Adavosertib + Paclitaxel | Preclinical | Actionable | In a preclinicals study, Adavosertib (MK-1775) in combination with Paclitaxel, demonstrated efficacy in endometrial cancer cells (PMID: 24381593). | 24381593 | |
| Unknown unknown | cervical cancer | not applicable | BIRB-796 + Tozasertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Tozasertib (VX-680) and BIRB-796 resulted in increased growth inhibition and induction of apoptosis in cervical cancer cell lines in culture, and increased tumor growth inhibition in cervical cancer cell line xenograft models, compared to either agent alone (PMID: 27082306). | 27082306 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CYT01B + Talazoparib | Preclinical - Cell culture | Actionable | In a preclinical study, CYT01B and Talzenna (talazoparib) synergistically inhibited growth of tumor cell lines in culture (AACR Annual Meeting 2019, Abstract 363). | detail... | |
| Unknown unknown | cervical cancer | not applicable | Pimitespib + Radiotherapy | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TAS-116 increased sensitivity of a cervical cancer cell line to X-ray and carbon ion radiotherapy, and the combination resulted in decreased DNA double-strand break repair and increased cell-cycle arrest in culture, and increased tumor growth delay in xenograft models (PMID: 28062703). | 28062703 | |
| Unknown unknown | melanoma | not applicable | Sabatolimab + Spartalizumab | Case Reports/Case Series | Actionable | In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in stable disease in 5 patients with melanoma, including cutaneous melanoma (n=2), uveal melanoma (n=2), and non-cutaneous melanoma (n=1) (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ipatasertib | Phase I | Actionable | In a Phase I trial, Ipatasertib (GDC-0068) resulted in antitumor activity in 30% (16/52) of patients with advanced solid tumors, primarily demonstrating stable disease (PMID: 27872130). | 27872130 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ipatasertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Ipatasertib (GDC-0068) demonstrated activity against tumor growth in cell line xenograft models of solid tumors (PMID: 24141624). | 24141624 | |
| Unknown unknown | multiple myeloma | not applicable | Carfilzomib + Tinostamustine | Preclinical - Patient cell culture | Actionable | In a preclinical study, the combination of Kyprolis (carfilzomib) and EDO-S101 worked synergistically to decrease viability of multiple myeloma cell lines and primary cells in culture, including a cell line with resistance to Velcade (bortezomib) (PMID: 28753594). | 28753594 | |
| Unknown unknown | hematologic cancer | not applicable | ABT-348 | Phase I | Actionable | In a Phase I trial, ABT-348 (Ilorasertib) demonstrated safety and preliminary efficacy in patients with various hematological malignancies (PMID: 25933833). | 25933833 | |
| Unknown unknown | breast cancer | not applicable | Lucitanib | Phase II | Actionable | In a Phase II trial, Lucitanib (E-3810) demonstrated acceptable toxicity and activity, resulted in a median progression-free survival of 3.5 and 2.6 months for 10mg and 15mg treatment groups respectively, with no differences in response correlated with FGFR1 and 11q amplification status in metastatic breast cancer patients (Cancer Res 2017;77(4 Suppl):Abstract nr P6-11-03). | detail... | |
| Unknown unknown | rhabdoid cancer | not applicable | BMS-754807 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 100% (3/3) of cell line xenograft models of rhabdoid tumor (PMID: 21298745). | 21298745 | |
| Unknown unknown | high grade glioma | not applicable | GDC-0084 | Phase I | Actionable | In a Phase I trial, GDC-0084 treatment demonstrated expected toxicity and blood-brain barrier penetration, resulted in stable disease as best response in 40% (19/47) of patients with recurrent high-grade glioma (J Clin Oncol (PMID: 31937616; NCT01547546). | 31937616 | |
| Unknown unknown |