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Gene Variant Detail

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Relevant Treatment Approaches

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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