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| Profile Name | Unknown unknown |
| Gene Variant Detail | |
| Relevant Treatment Approaches |
| Molecular Profile | Indication/Tumor Type | Response Type | Relevant Treatment Approaches | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|---|
| Unknown unknown | synovial sarcoma | not applicable | SU6656 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, SU6656 treatment decreased cell proliferation of synovial sarcoma cells in culture and inhibited tumor growth and blocked tumor invasion in cell line xenograft models (PMID: 22244830). | 22244830 | |
| 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 | 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 | pancreatic adenocarcinoma | not applicable | Adavosertib + Gemcitabine + Radiotherapy | Phase I | Actionable | In a Phase I trial, Adavosertib (MK-1775) in combination with Gemzar (gemcitabine) and radiation therapy was tolerable, resulted in a median overall survival of 21.7 months and a median progression-free survival of 9.4 months in patients with advanced pancreatic adenocarcinoma (PMID: 31398082; NCT02037230). | 31398082 | |
| Unknown unknown | colorectal cancer | not applicable | Adavosertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Adavosertib (MK-1775) inhibited cell proliferation and promoted DNA damage in a human colorectal cancer cell line in culture, and promoted tumor regression in xenograft models (PMID: 23699655). | 23699655 | |
| Unknown unknown | thyroid gland anaplastic carcinoma | not applicable | Spartalizumab | Phase II | Actionable | In a Phase II trial, Spartalizumab (PDR001) demonstrated tolerability in patients with anaplastic thyroid carcinoma, and resulted in an overall response rate of 19% (8/42), including three complete responses and five partial responses, median progression-free survival of 1.7 months, and median overall survival of 5.9 months (PMID: 32364844; NCT02404441). | 32364844 | |
| 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 | LY3127804 + Ramucirumab | Phase I | Actionable | In a Phase I trial, the combination treatment of LY3127804 and Cyramza (ramucirumab) at various doses in advanced solid tumor patients demonstrated safety and was well-tolerated, and led to a best overall response of partial responses observed in four patients, including one each with clear cell endometrial carcinoma, cervix squamous cell carcinoma, carcinoma of unknown primary, and gastroesophageal junction adenocarcinoma, and resulted in stable disease in 37% (13/35) (PMID: 32741971; NCT02597036). | 32741971 | |
| 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 | breast cancer | not applicable | Metformin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Glucophage (metformin) inhibited the growth of a breast cancer cell line in culture and resulted in decreased tumor volume in a cell line xenograft model (PMID: 26351208). | 26351208 | |
| Unknown unknown | colon carcinoma | not applicable | DRP-104 | Preclinical | Actionable | In a preclinical study, DRP-104 treatment inhibited tumor growth in a syngeneic mouse model of colon carcinoma (J Immunother Cancer. 2019; 7(Suppl 1): 282, Abs nr: P497). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Onalespib | Phase I | Actionable | In a Phase I trial, AT13387 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 25336693). | 25336693 | |
| 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 | Advanced Solid Tumor | not applicable | Sirolimus | Preclinical | Actionable | In preclinical studies, Rapamune (sirolimus) induced apoptosis of cancer cells and increased sensitivity to Platinol (cisplatin) (PMID: 15136596). | 15136596 | |
| 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 | pancreatic endocrine carcinoma | not applicable | Nintedanib | Preclinical | Actionable | In a preclinical study, Ofev (nintedanib) induced tumor cell apoptosis, decreased microvessel density, inhibited tumor growth, and improved survival in transgenic mouse models of pancreatic neuroendocrine carcinoma (PMID: 26206868). | 26206868 | |
| 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 | Advanced Solid Tumor | not applicable | Paclitaxel + Taladegib | Phase I | Actionable | In a Phase I trial, combination of Taladegib and Taxol (paclitaxel) treatment demonstrated safety and preliminary efficacy, resulted in partial response in 3 patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2594)). | detail... | |
| 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 | 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 | prostate cancer | not applicable | MC180295 | Preclinical - Cell culture | Actionable | In a preclinical study, MC180295 decreased proliferation of prostate cancer cell lines in culture (PMID: 30454645). | 30454645 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Bendamustine + Idelalisib + Rituximab | Phase III | Actionable | In a Phase III trial, addition of Zydelig (idelalisib) to Bendamustine and Rituximab resulted in improved median progression-free survival (20.8 vs 11.1 months, HR=0.33, p<0.0001) compared to placebo in patients with relapsed or refractory chronic lymphocytic leukemia, although treatment associated adverse events were also increased (PMID: 28139405). | 28139405 | |
| Unknown unknown | renal carcinoma | no benefit | MG 98 | Phase II | Actionable | In a Phase II trial, MG 98 treatment in seventeen evaluable patients resulted in stable disease in six patients, progressive disease in nine patients, and no objective responses, and the trial was terminated early due to a lack of responses (PMID: 16502349). | 16502349 | |
| 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 | lung adenocarcinoma | not applicable | AC-93253 iodide + Gefitinib | Preclinical - Cell culture | Actionable | In a preclinical study, AC-93253 iodide combined with Iressa (gefitinib) resulted in a synergistic effect, demonstrating growth inhibition of an Iressa (gefitinib)-resistant lung adenocarcinoma cell line in culture (PMID: 29132432). | 29132432 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Adavosertib + Carboplatin + Paclitaxel | Phase Ib/II | Actionable | In a Phase Ib trial, treatment with Adavosertib (MK-1775) in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) at the recommended Phase II does was tolerable and resulted in partial response in 16.7% (1/3) of patients and stable disease in 33.3% (2/6) of Asian patients with advanced solid tumors (PMID: 32034630). | 32034630 | |
| Unknown unknown | acute lymphoblastic leukemia | not applicable | ORY-1001 | Preclinical - Pdx | Actionable | In a preclinical study, ORY-1001 reduced leukemic blast percentage and prolonged survival in a T-cell acute lymphoblastic leukemia patient-derived xenograft (PDX) model (PMID: 29502954). | 29502954 | |
| Unknown unknown | malignant glioma | not applicable | Sunitinib | Preclinical | Actionable | In a preclinical study, Sutent (sunitinb) induced cell death and decreased proliferation of glioma cells in culture (PMID: 25458015). | 25458015 | |
| Unknown unknown | malignant glioma | not applicable | Sunitinib | Phase II | Actionable | In a Phase II clinical trial, Sutent (sunitinib) was well-tolerated in young patients with high grade glioma, but did not demonstrate sufficient anti-tumor activity as a single agent, with no patients achieving a sustained objective response (PMID: 27109549). | 27109549 | |
| Unknown unknown | endometrial carcinoma | not applicable | XL147 | Phase II | Actionable | In a Phase II trial, Pilaralisib (XL147) treatment resulted in an objective response rate of 6% (4/67) in patients with endometrial carcinoma, although anti-tumor activity is not associated with molecular alterations in PTEN and PIK3R1 (PMID: 25528496). | 25528496 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MK-1248 | Phase I | Actionable | In a Phase I trial, treatment with MK-1248 demonstrated safety and resulted in stable disease in 15% (3/20) of patients with advanced solid tumors, with no complete or partial responses observed, but demonstrated preliminary antitumor efficacy when combined with Keytruda (pembrolizumab) (PMID: 32809217; NCT02553499). | 32809217 | |
| Unknown unknown | glioblastoma | not applicable | Dovitinib | Phase I | Actionable | In a Phase I trial, Dovitinib (TKI258) treatment resulted in a progression free survival rate at 6 months of 16.7% (2/12) in patients with recurrent glioblastoma (PMID: 27100354). | 27100354 | |
| 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 | diffuse large 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 diffuse large B-cell lymphoma, high-grade B-cell lymphoma, primary mediastinal B-cell lymphoma, and follicular lymphoma grade 3B (PMID: 32888407; NCT02631044). | 32888407 detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Lisocabtagene maraleucel | Clinical Study | Actionable | In a clinical case study, treatment with Breyanzi (lisocabtagene maraleucel) resulted in complete remission and durable central nervous system response in a patient with diffuse large B-cell lymphoma (PMID: 28834486). | 28834486 | |
| Unknown unknown | urinary bladder 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 a patient with bladder cancer (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). | detail... | |
| Unknown unknown | stomach cancer | not applicable | AZD6738 + Cisplatin | Preclinical - Cell culture | Actionable | In a preclinical study, the addition of AZD6738 to Platinol (cisplatin) treatment in a gastric cancer cell line in culture resulted in enhanced chemotherapeutic sensitivity, demonstrating a synergistic effect (PMID: 28138034). | 28138034 | |
| Unknown unknown | acute myeloid leukemia | not applicable | RN-1 | Preclinical - Cell culture | Actionable | In a preclinical study, RN-1 inhibited growth of acute myeloid leukemia cell lines in culture (PMID: 26837761). | 26837761 | |
| Unknown unknown | ovarian cancer | not applicable | Cisplatin + NU6027 | Preclinical - Cell culture | Actionable | In a preclinical study, NU6027 enhanced the efficacy of Platinol (cisplatin) in ovarian cancer cells in culture, resulting in decreased cell survival (PMID: 21730979). | 21730979 | |
| Unknown unknown | colon cancer | not applicable | Vandetanib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Caprelsa (vandetanib) reduced human colon cancer cell line-induced angiogenesis and delayed tumor growth in colon cancer cell line xenograft models, with the highest delay corresponding to tumors with the highest VEGF-expression levels (PMID: 19528456). | 19528456 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ABTL0812 | Phase I | Actionable | In a Phase I/Ib trial, ABTL0812 demonstrated safety and preliminary efficacy in patients with advanced solid tumors, resulted in long lasting stable disease for more than 1 year in two patients (PMID: 33588149; NCT02201823). | 33588149 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ONCOS-102 | Phase I | Actionable | In a Phase I trial, ONCOS-102 demonstrated safety and preliminary efficacy, resulted in disease control in 40% (4/10) of patients with advanced solid tumors, and a median overall survival of 9.3 months (PMID: 26981247). | 26981247 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Atezolizumab | FDA approved | Actionable | In a Phase III trial (OAK) that supported FDA approval, treatment with Tecentriq (atezolizumab) resulted in an improved median overall survival compared to Taxotere (docetaxel) (13.8 vs 9.6 months, HR=0.73, p=0.0003) in patients with previously treated non-small cell lung cancer (PMID: 27979383; NCT02008227). | detail... 27979383 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Atezolizumab | Clinical Study - Meta-analysis | Actionable | In a meta-analysis, compared to Taxotere (docetaxel), treatment with immune checkpoint inhibitors including Opdivo (nivolumab), Keytruda (pembrolizumab), or Tecentriq (atezolizumab), resulted in prolonged overall survival (HR=0.69, p<0.001) in non-small cell lung carcinoma patients (PMID: 29270615). | 29270615 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Atezolizumab | Phase II | Actionable | In a Phase II trial (POPLAR), treatment with Tecentriq (atezolizumab) resulted in an improved median overall survival of 12.6 months compared to 9.7 months with Taxotere (docetaxel) in patients with previously treated non-small cell lung cancer, and increased PD-L1 expression level was associated with improved survival benefit (PMID: 26970723; NCT01903993). | 26970723 | |
| 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 | Burkitt lymphoma | not applicable | Cerdulatinib | Preclinical | Actionable | In a preclinical study, treatment with Cerdulatinib (PRT062070) resulted in decreased viability and increased apoptosis of Burkitt lymphoma cells in culture (PMID: 25253883). | 25253883 | |
| 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 | Advanced Solid Tumor | not applicable | CYT01B + Niraparib | Preclinical - Cell culture | Actionable | In a preclinical study, CYT01B and Zejula (niraparib) synergistically inhibited growth of tumor cell lines in culture (AACR Annual Meeting 2019, Abstract 363). | detail... | |
| Unknown unknown | prostate cancer | not applicable | MYCi975 + unspecified PD-1 antibody | Preclinical | Actionable | In a preclinical study, MYCi975 combined with an anti-PD1 therapy synergistically inhibited tumor growth in a mouse model of prostate cancer (PMID: 31679823). | 31679823 | |
| 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 | ovarian cancer | not applicable | S2101 | Preclinical - Cell culture | Actionable | In a preclinical study, S2101 altered gene expression, resulted in apoptosis in ovarian cancer cells in culture (PMID: 27914215). | 27914215 | |
| 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 | angiosarcoma | not applicable | Bevacizumab + Paclitaxel | Phase II | Actionable | In a Phase II trial, angiosarcoma patients treated with Avastin (bevacizumab), in combination with Taxol (paclitaxel), showed no improvement in PFS and OS compared to Taxol (paclitaxel) alone and resulted in higher toxicity (PMID: 26215950). | 26215950 | |
| 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 | Advanced Solid Tumor | not applicable | KW-2450 | Phase I | Actionable | In a Phase I trial, 40% (4/10) of patients with advanced solid tumors demonstrated stable disease when treated with KW-2450 (PMID: 26850678). | 26850678 | |
| Unknown unknown | astrocytoma | not applicable | Dactolisib + Everolimus | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of BEZ235 and Afinitor (everolimus) resulted in stable disease in a patient with astrocytoma (PMID: 28357727). | 28357727 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Forskolin | Preclinical | Actionable | In a preclinical study, Forskolin inhibited cell proliferation and induced apoptosis in acute myeloid leukemia cells in culture (PMID: 21233840). | 21233840 | |
| Unknown unknown | ovarian serous carcinoma | not applicable | Berzosertib + Gemcitabine | Phase II | Actionable | In a Phase II trial, the combination of Berzosertib (VX-970) and Gemzar (gemcitabine) resulted in improved median progression-free survival (22.9 vs 14.7 weeks), median overall survival (59.4 vs 43.0 weeks), and clinical benefit rate (35% (12/34) vs 25% (9/36)), and a lower objective response rate (3% (1/34) vs 11% (4/36) compared to Gemzar (gemicitabine) alone in patients with platinum-resistant high-grade serous carcinoma of the ovary, fallopian tube, or primary peritoneum (PMID: 32553118; NCT02595892). | 32553118 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | HY-16462 + JQ1 | Preclinical - Cell culture | Actionable | In a preclinical study, HY-16462 and JQ1 treatment synergistically inhibited growth of a triple-negative breast cancer cell line in culture (PMID: 31000582). | 31000582 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | GSK2126458 | Phase I | Actionable | In a Phase I trial, GSK2126458 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol (Meeting Abstracts) 2011 29: 3018). | detail... | |
| Unknown unknown | malignant glioma | not applicable | Everolimus | Phase II | Actionable | In a Phase II trial, Afinitor (everolimus) treatment resulted in a 6-month progression free survival rate of 87% and 55% in patients with WHO grade II and III/IV glioma, respectively (Neuro Oncol (2016) 18 (suppl 6): vi8-vi9.). | detail... | |
| Unknown unknown | CLL/SLL | not applicable | CC-115 | Phase I | Actionable | In a Phase I trial, CC-115 treatment was tolerated and demonstrated manageable safety, and resulted in a partial response in 37.5% (3/8) and a stable disease in 25% (2/8) of chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) patients, and a median progression-free survival was not evaluable (PMID: 31853198; NCT01353625). | 31853198 | |
| 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 | 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 | breast cancer | not applicable | Everolimus + Metformin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Afinitor (everolimus) and Glucophage (metformin) resulted in a synergistic effect in breast cancer cells, resulting in both greater inhibition of cell growth in culture and decreased tumor growth in cell line xenograft models when compared to either agent alone (PMID: 26351208). | 26351208 | |
| 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 | gastrointestinal stromal tumor | not applicable | OSI-930 | Phase I | Actionable | In a Phase I trial, 58% (11/19) of patients with a gastrointestinal stromal tumor demonstrated stable disease based on RECIST criteria when treated with OSI-930 (PMID: 23403628). | 23403628 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BAY1217389 | Preclinical | Actionable | In a preclinical study, BAY1217389 inhibited proliferation of a variety of human solid tumor cell lines in culture (PMID: 26832791). | detail... 26832791 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | Enavatuzumab | Phase I | Actionable | In a Phase I trial, Enavatuzumab treatment resulted in no objective responses and only stable disease in 13.3% (4/30) of patients with advanced solid tumors (PMID: 29054986). | 29054986 | |
| 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 | transitional cell carcinoma | not applicable | Everolimus | Phase II | Actionable | In a Phase II trial, treatment with Afinitor (everolimus) in patients with transitional cell carcinoma resulted in 2 patients with a partial response, 8 patients with stable disease, and 27 patients with progressive disease, and resulted in a median progression free survival of 61 days and a median overall survival of 101 days (PMID: 22473592). | 22473592 | |
| Unknown unknown | lung non-small cell carcinoma | 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 non-small cell lung cancer cells in culture (PMID: 28179288). | 28179288 | |
| Unknown unknown | head and neck cancer | not applicable | Cetuximab + Ficlatuzumab | Phase I | Actionable | In a Phase I trial, the combination of Erbitux (cetuximab) and Ficlatuzumab (AV-299) was tolerated and resulted in an overall response rate of 17% (2/12), a median progression-free survival (PFS) of 5.4 months, and an overall survival (OS) of 8.9 months in patients with advanced head and neck squamous cell carcinoma, baseline plasma scMet levels negatively correlated with PFS (HR = 1.92, p = 0.048), while tumor cMet or Hgf levels did not correlate with PFS or OS (PMID: 32545260; NCT02277197). | 32545260 | |
| Unknown unknown | pancreatic cancer | not applicable | Gemcitabine + Refametinib | Phase Ib/II | Actionable | In a Phase I/II trial, Refametinib (BAY86-9766) and Gemzar (gemcitabine) combination treatment resulted in an objective response rate of 23% and a disease control rate of 73% in patients with advanced pancreatic cancer (PMID: 27975152). | 27975152 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | MIW815 + Spartalizumab | Phase Ib/II | Actionable | In a Phase Ib trial, MIW815 (ADUS100), in combination with Spartalizumab (PDR001), demonstrated preliminary efficacy in PD-1 (PDCD1)-naive TNBC patients (J of Clin Oncol 37, 2019 (suppl; abstr 2507); NCT03172936). | detail... | |
| Unknown unknown | Hodgkin's lymphoma | not applicable | Idelalisib | Phase II | Actionable | In a Phase II trial, Idelalisib treatment resulted in an overall response rate of 20% (5/25) in Hodgkin's lymphoma patients, with one patient experiencing a complete response and four patients experiencing a partial response (PMID: 28327905). | 28327905 | |
| Unknown unknown | pancreatic cancer | not applicable | GP-2250 | Preclinical - Pdx | Actionable | In a preclinical study, GP-2250 treatment reduced viability and proliferation, and induced apoptosis and necrosis in pancreatic cancer cell lines in culture, and inhibited tumor growth in cell line xenograft and patient-derived xenograft (PDX) models (PMID: 28340556). | 28340556 | |
| 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 | Advanced Solid Tumor | not applicable | Berzosertib + Carboplatin | Phase I | Actionable | In a Phase I trial, Berzosertib (VX-970) and Paraplatin (carboplatin) combination treatment resulted in a best response of stable disease in 71% (15/21) of evaluable patients with advanced solid tumors, including 10 patients with stable disease for 4 months or more and 6 patients with stable disease for 6 months or more (PMID: 32568634; NCT02157792). | 32568634 | |
| 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 | appendix carcinoma | not applicable | Melanin + Phenytoin + Sirolimus + SM88 | Case Reports/Case Series | Actionable | In a Phase I trial, a patient with appendiceal carcinoma achieved a complete response following treatment with SMK therapy (sirolimus, phenytoin, and sirolimus with SM-88) (PMID: 30929156). | 30929156 | |
| 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 | 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 | small intestine 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 small intestine cancer (PMID: 30724423). | 30724423 | |
| 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 | melanoma | not applicable | Cobimetinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Cobimetinib (GDC-0973) induced cell death in several human melanoma cell lines in culture and inhibited tumor growth in xenograft models (PMID: 22084396). | 22084396 | |
| Unknown unknown | chondrosarcoma | not applicable | Doxorubicin + Nilotinib | Phase I | Actionable | In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin resulted in 5 stable disease and 2 progressive disease in 7 patients with chondrosarcoma, with a median progression-free survival of 14 months and a median overall survival of 25 months (PMID: 30037815; NCT02587169). | 30037815 | |
| 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 | triple-receptor negative breast cancer | not applicable | AMG 900 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AMG 900 induced apoptosis in triple-negative breast cancer (TNBC) cell lines in culture and inhibited tumor growth in TNBC cell line xenograft models (PMID: 23990115). | 23990115 | |
| 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 | mycosis fungoides | not applicable | Mogamulizumab | FDA approved | Actionable | In a Phase III trial (MAVORIC) that supported FDA approval, Poteligeo (mogamulizumab-kpkc) treatment resulted in significantly improved progression-free survival (7.7 vs 3.1 months, HR=0.53, p<0.0001) and objective response rate (28.0% vs 4.8%) compared to Zolinza (vorinostat) in patients with mycosis fungoides or Sézary syndrome (Blood 2017 130(Suppl 1):817). | detail... detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | KX2-391 | Phase I | Actionable | In a Phase I trial, KX2-391 demonstrated safety and preliminary efficacy, resulted in stable disease for more than 4 months in 25% (11/44) of patients with advanced solid tumors (PMID: 23361621). | 23361621 | |
| Unknown unknown | stomach cancer | not applicable | OPB-111077 | Phase I | Actionable | In a Phase I trial, two gastric cancer patients demonstrated stable disease when treated with OPB-111077 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B118). | detail... | |
| Unknown unknown | malignant peripheral nerve sheath tumor | no benefit | Ganetespib + Sirolimus | Phase II | Actionable | In a Phase II trial, treatment with the combination of Ganetespib and Rapamune (sirolimus) did not result in clinical benefit in 10 patients with malignant peripheral nerve sheath tumor (PMID: 32089640; NCT02008877). | 32089640 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Fostamatinib + Silmitasertib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Silmitasertib (CX-4945) and Fostamatinib (R788) worked synergistically to decrease cell viability and resulted in increased apoptosis and decreased AKT phosphorylation in ABC and GCB-type diffuse large B-cell lymphoma cell lines in culture (PMID: 28460620). | 28460620 | |
| Unknown unknown | follicular lymphoma | not applicable | Betalutin | Phase Ib/II | Actionable | In a Phase I/II trial, Betalutin (177Lu Lilotomab Satetraxetan) treatment in patients with follicular lymphoma resulted in an overall response rate (ORR) of 65% (37/57, 17 complete responses (CR), 20 partial responses (PR), 10 stable disease (SD)) and a median progression-free survival of 9.0 months, ORR was 70% (26/37, 12 CR, 14 PR) in patients who received two or more prior therapies, and was 67% (14/21, 5 CR, 9 PR) in those who were also rituximab-refractory (PMID: 32877524; NCT01796171). | 32877524 | |
| Unknown unknown | multiple myeloma | not applicable | AMG 701 + Lenalidomide | Preclinical | Actionable | In a preclinical study, AMG 701 and Revlimid (lenalidomide) combination treatment enhanced tumor growth inhibition and regression in a mouse model of multiple myeloma (Blood (2019) 134 (Supplement_1): 135). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | AC480 | Phase I | Actionable | In a Phase I trial, AC480 demonstrated safety and potential efficacy in patients with several solid tumor types (PMID: 21576284). | 21576284 | |
| Unknown unknown | angiosarcoma | not applicable | Alisertib | Phase II | Actionable | In a Phase II trial, Alisertib (MLN8237) treatment resulted in partial response in 2 angiosarcoma patients (PMID: 27502708). | 27502708 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Niraparib + SY-1365 | Preclinical - Cell culture | Actionable | In a preclinical study, SY-1365 and Zejula (niraparib) synergistically induced apoptosis in triple-receptor negative breast cancer cells in culture (Proceedings of the AACR, Vol 58, April 2017, Abstract # 1151). | detail... | |
| Unknown unknown | head and neck cancer | not applicable | Cisplatin + GL-ONC1 + Radiotherapy | Phase I | Actionable | In a Phase I trial, the combination therapy of GL-ONC1, Platinol (cisplatin), and radiotherapy resulted in a progression-free survival of 74.4% at 1 year and 64.1% at 2 years and an overall survival of 84.6% at 1 year and 69.2% at 2 years in patients with nonmetastatic head and neck cancer (PMID: 28679776). | 28679776 | |
| Unknown unknown | multiple myeloma | not applicable | AMG 701 | Preclinical - Patient cell culture | Actionable | In a preclinical study, AMG 701 treatment induced lysis of cells from patients with relapsed/refractory multiple myeloma in culture, and inhibited tumor growth in a cell line xenograft model reconstituted with human effector T-cells (Blood (2019) 134 (Supplement_1): 135). | detail... | |
| Unknown unknown | colorectal cancer | not applicable | HL-085 + OKI-005 | Preclinical - Cell culture | Actionable | In a preclinical study, HL085 and OKI-005 demonstrated synergistic activity in 3 of 6 colorectal cancer cell lines, and increased immunogenicity of tumor cells in culture (Cancer Res 2019;79(13 Suppl):Abstract nr 4753). | detail... | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Entospletinib | Phase II | Actionable | In a Phase II clinical trial, treatment with entospletinib resulted in a progression-free survival (PFS) rate of 70.1% at 24 weeks, with a median PFS of 13.8 months, and a objective response rate of 61% (24/41; all partial responses) in patients with chronic lymphocytic leukemia (PMID: 25696919). | 25696919 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | DS-7423 | Phase I | Actionable | In a Phase I trial, DS-7423 demonstrated safety and preliminary clinical activity in patients with advanced solid tumors (Ann Oncol (2014) 25 (suppl 4): iv153). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | Flotetuzumab | Phase I | Actionable | In a Phase I/II trial, Flotetuzumab (MGD006) treatment demonstrated acceptable safety and tolerability, and resulted in an overall response rate of 30% (9/30; 5 complete response (CR), 3 CRh, 1 CRi) and a median overall survival of 11.2 months in heavily pretreated acute myeloid leukemia patients with either primary induction failure (n=24) or early relapse (n=6), and in patients who achieved CR/CRh, resulted in a median duration of response of 6.9 months (PMID: 32929488; NCT02152956). | 32929488 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | CT-707 + PHA-665752 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of PHA-665752 and CT-707 resulted in synergism in hepatocellular carcinoma cells in culture, demonstrating near complete cell death (PMID: 27638856). | 27638856 | |
| Unknown unknown | squamous cell carcinoma | not applicable | Cemiplimab | FDA approved | Actionable | In a Phase I and a Phase II trial that supported FDA approval, Libtayo (cemiplimab) treatment resulted in an objective response rate of 46.7% (35/75), a complete response rate of 5.3% (4/75), and a partial response rate of 41.3% (31/75) in patients with metastatic cutaneous squamous cell carcinoma (PMID: 29863979; NCT02383212, NCT02760498). | detail... 29863979 | |
| Unknown unknown | multiple myeloma | not applicable | TAS4464 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TAS4464 inhibited growth and induced apoptosis in multiple myeloma cell lines in culture, resulted in tumor suppression in cell line xenograft models (Cancer Res 2015;75(15 Suppl):Abstract nr 1730). | detail... | |
| 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 | breast cancer | not applicable | Danusertib + Fulvestrant | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with the combination of Danusertib (PHA-739358) 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 | follicular lymphoma | not applicable | Polatuzumab vedotin-piiq + Rituximab | Phase II | Actionable | In a Phase II trial (ROMULUS), the combination of Polivy (polatuzumab vedotin-piiq) and Rituxan (rituximab) demonstrated safety and tolerability, and resulted in an objective response rate of 70% (14/20, 9 complete responses, 5 partial responses), a median progression-free survival of 15.3 months, and a 2-year overall survival rate of 87.8% in patients with follicular lymphoma (PMID: 30935953; NCT01691898). | 30935953 | |
| 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 | Advanced Solid Tumor | not applicable | CG200745 | Phase I | Actionable | In a Phase I trial, CG200745 treatment demonstrated safety in advanced solid tumor patients and did not result in any partial or complete responses, but led to stable disease in 57.1% (16/28) of patients for at least six weeks (PMID: 26076682). | 26076682 | |
| Unknown unknown | colorectal cancer | not applicable | LY2090314 + Simmiparib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination therapy of LY2090314 and Simmiparib resulted in a synergistic effect in colorectal cancer cells with either BRCA1/2 proficiency or BRCA2 deficiency in culture, demonstrating increased cell cycle arrest, apoptotic cell death, and decreased cell survival, and led to inhibition of tumor growth, increased double-strand DNA breaks, and elevated cleaved caspase 3 in cell line xenograft models (PMID: 33589588). | 33589588 | |
| Unknown unknown | pancreatic adenocarcinoma | 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 35% (6/17) of patients with pancreatic adenocarcinoma, two of whom had stable disease lasting greater than 150 days (PMID: 30756308; NCT01335269). | 30756308 | |
| Unknown unknown | ovarian carcinoma | not applicable | Disarib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Disarib treatment led to apoptotic induction and resulted in tumor regression in ovarian carcinoma xenograft models (PMID: 27693384). | 27693384 | |
| Unknown unknown | colon carcinoma | not applicable | SB 11285 + unspecified CTLA4 antibody | Preclinical | Actionable | In a preclinical study, treatment with SB 11285 combined with an anti-CTLA4 antibody delayed tumor growth and reduced tumor volume in a syngeneic mouse model of colon carcinoma (Journal of Clinical Oncology 2017 35:15_suppl, e14616). | detail... | |
| Unknown unknown | colorectal cancer | not applicable | Labetuzumab govitecan | Phase Ib/II | Actionable | In a Phase I/II trial, treatment with Labetuzumab govitecan demonstrated safety and resulted in stable disease in 49% (42/86), a clinical benefit rate of 29% (25/86; 1 partial response and stable disease for at least 6 months in 24), a median progression-free survival of 3.6 months, and a median overall survival of 6.9 months in patients with previously treated recurrent or refractory colorectal cancer (PMID: 28817371; NCT01605318). | detail... 28817371 | |
| 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 | 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 | melanoma | not applicable | BNT111 + Nivolumab | Case Reports/Case Series | Actionable | In a Phase I trial, BNT111 treatment in combination with Opdivo (nivolumab) resulted in a partial response in a melanoma patient with a reduction in lung metastatic lesions, and in another patient, resulted in a partial response with a decrease in tumor burden in both liver and subcutaneous lesions (PMID: 32728218; NCT02410733). | 32728218 | |
| Unknown unknown | ovarian cancer | not applicable | CPI-0610 + Dasatinib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination treatment of Sprycel (dasatinb) and CPI-0610 resulted in a synergistic effect, demonstrating greater inhibition of colony formation in ovarian cancer cells in culture compared to either therapy alone (PMID: 32927276). | 32927276 | |
| 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 | hematologic cancer | not applicable | Acalisib | Phase I | Actionable | In a Phase Ib trial, Acalisib (GS-9820) treatment resulted in lymph node reduction in 70% (7/10) and nodal partial response in 40% (4/10) of patients with non-Hodgkin's lymphoma or chronic lymphocytic leukemia (Blood: 122 (21): 2881). | detail... | |
| Unknown unknown | pancreatic cancer | not applicable | Trametinib | Phase I | Actionable | In a Phase I trial, 42% (11/26) of pancreatic cancer patients demonstrated a decrease in tumor formation when treated with Mekinist (trametinib) (PMID: 22805291). | 22805291 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Cabozantinib | FDA approved | Actionable | In a Phase III trial (CELESTIAL) that supported FDA approval, Cabometyx (cabozantinib) significantly improved overall survival (10.2 vs 8.0 months, HR=0.76, p=0.005) and progression-free survival (5.2 vs 1.9 months, HR=0.44, p<0.001) compared to placebo in patients with previously treated advanced hepatocellular carcinoma (PMID: 29972759; NCT01908426). | 29972759 detail... | |
| Unknown unknown | non-Hodgkin lymphoma | not applicable | Acalisib | Phase Ib/II | Actionable | In a Phase Ib trial, Acalisib (GS-9820) treatment resulted in an overall response rate of 28.6% (4/14), with 4 partial responses, stable disease in 3 patients, and a lymph node response in 36.4% (4/11) of patients with non-Hodgkin's lymphoma or Hodgkin's lymphoma (PMID: 29434192; NCT01705847). | 29434192 | |
| Unknown unknown | clear cell renal cell carcinoma | not applicable | GSK3368715 | Preclinical - Cell culture | Actionable | In a preclinical study, GSK3368715 inhibited tumor growth in a clear cell renal cell carcinoma xenograft model (PMID: 31257072). | 31257072 | |
| Unknown unknown | stomach cancer | not applicable | DKN-01 + Paclitaxel | Phase I | Actionable | In a Phase I trial, DKN-01 and Taxol (paclitaxel) combination treatment resulted in partial response in 18% (6/34) and stable disease in 32% (11/34) of advanced esophagogastric cancer patients, with a median progression-free survival of 13.7 weeks and an overall survival of 28.4 weeks (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #91P; NCT02013154). | detail... | |
| Unknown unknown | B-cell adult acute lymphocytic leukemia | not applicable | Disarib + Paclitaxel | Preclinical - Cell culture | Actionable | In a preclinical study, Taxol (paclitaxel) efficacy was enhanced when combined with Disarib, resulting in greater cell death compared to either agent alone in B-cell acute lymphocytic leukemia cells in culture (PMID: 27693384). | 27693384 | |
| Unknown unknown | B-cell lymphoma | not applicable | BI 836826 | Phase I | Actionable | In a Phase I trial, BI 836826 (MAb 37.1) treatment led to an overall response rate of 6.3% (3/48) in heavily pretreated relapsed/refractory B-cell non-Hodgkin lymphoma patients, with 1 complete response in a diffuse large B-cell lymphoma patient, 2 partial responses with one each in a follicular lymphoma and a mantle cell lymphoma patient, and resulted in a median progression-free survival of 47 days, however, clinical development of BI 836826 (MAb 37.1) was terminated (PMID: 32172489; NCT01403948). | 32172489 | |
| Unknown unknown | follicular lymphoma | not applicable | Rituximab + Urelumab | Phase I | Actionable | In a Phase I trial, Urelumab (BMS-663513) and Rituxan (rituximab) combination therapy demonstrated manageable safety profile, resulted in an objective response rate of 35% (6/17) and a disease control rate of 71% (12/17) in patients with relapsed or refractory follicular lymphoma, with a median progression-free survival of 40.4 weeks and a median overall survival not reached (PMID: 32052473; NCT01775631). | 32052473 | |
| Unknown unknown | colon cancer | not applicable | HM-3 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a colon cancer cells treated with HM-3 demonstrated reduced cell migration in culture and inhibition of tumor growth in xenograft models (PMID: 27633584). | 27633584 | |
| Unknown unknown | pancreatic adenocarcinoma | not applicable | Gemcitabine + PRI-724 | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of PRI-724 and Gemzar (gemcitabine) demonstrated safety and preliminary efficacy in patients with advanced pancreatic adenocarcinoma, resulted in stable disease in 40% (8/20) of the patients, with a median progression-free survival of 2 months, and a median decline of serum S100P level by 49.95% (Journal of Clinical Oncology 34, no. 15_suppl; NCT01764477). | detail... detail... | |
| Unknown unknown | glioblastoma | not applicable | Trabedersen | Phase II | Actionable | In a Phase II trial, Trabedersen (AP 12009) treatment in patients with recurrent or refractory high-grade glioma (including grade IV glioblastoma multiforme or grade III astrocytoma) resulted in responses in 19 (3 complete and 16 partial responses) and stable disease for at least 6 months in 7 of 77 patients in the efficacy population, and a median progression-free survival of 86 days and median overall survival of 432 days (PMID: 31795071; NCT00431561). | 31795071 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Sapitinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Sapitinib (AZD8931) inhibited EGFR, ERBB2 (HER2), and ERBB3 (HER3) kinase activity, and inhibited tumor growth in several cell line xenograft models, including breast, NSCLC, colorectal, and head and neck squamous cell carcinoma xenograft models (PMID: 20145185). | 20145185 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | GSK3203591 + GSK3368715 | Preclinical - Cell culture | Actionable | In a preclinical study, GSK3203591 and GSK3368715 worked synergistically to inhibit viability of diffuse large B-cell lymphoma cell lines in culture (PMID: 31257072). | 31257072 | |
| Unknown unknown | sarcoma | not applicable | Doxorubicin + Nilotinib | Phase I | Actionable | In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin demonstrated safety and preliminary efficacy, resulted in 1 partial response and 9 stable disease in 13 patients with sarcomas (PMID: 30037815; NCT02587169). | 30037815 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Lenalidomide | FDA approved | Actionable | In a Phase II trial (EMERGE) that supported FDA approval, Revlimid (lenalidomide) treatment resulted in an objective response rate of 28% (37/134) with rapid time to response (2.2 months), a median duration of response of 16.6 months, a median progression-free survival of 4.0 months, and a median overall survival of 19.0 months in patients with mantle cell lymphoma who relapsed or progressed after or were refractory to Velcade (bortezomib) (PMID: 24002500; NCT00737529). | 24002500 detail... | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | OMTX705 | Preclinical - Pdx | Actionable | In a preclinical study, OMTX705 treatment inhibited tumor growth in a patient-derived xenograft (PDX) model of triple-negative breast cancer (PMID: 32161121). | 32161121 | |
| Unknown unknown | leiomyosarcoma | not applicable | Doxorubicin + Nilotinib | Phase I | Actionable | In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin resulted in 1 short-duration stable disease and 1 progressive disease in 2 patients with leiomyosarcoma, consistent with synergistic growth inhibition in liposarcoma cells in culture and in xenograft models (PMID: 30037815; NCT02587169). | 30037815 | |
| Unknown unknown | hematologic cancer | not applicable | AsiDNA + Olaparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA treatment sensitized hematologic cancer cell lines to Lynparza (olaparib), inhibiting survival in culture (PMID: 27559053). | 27559053 | |
| 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 | lung small cell carcinoma | not applicable | Olaparib + Temozolomide | Phase Ib/II | Actionable | In a Phase I/II trial, combination of Lynparza (olaparib) and Temodar (temozolomide) resulted in an objective response rate of 41.7% (20/48) in patients with relapsed small cell lung cancer, with a median progression-free survival of 4.2 months and a median overall survival of 8.5 months, similar response was recapitulated in a coclinical trial with 32 patient-derived xenograft models (PMID: 31416802; NCT02446704). | 31416802 | |
| Unknown unknown | ovarian cancer | not applicable | Everolimus + JI-101 | Phase 0 | Actionable | In a pilot study, JI-101 in combination with Afinitor (everolimus) demonstrated safety and preliminary efficacy, resulted in stable disease in a majority of patients with ovarian cancer (PMID: 26365907). | 26365907 | |
| Unknown unknown | ovarian cancer | not applicable | JNJ-54302833 | Preclinical | Actionable | In a preclinical study, JNJ-54302833 inhibited growth of ovarian cancer cells in culture ( Cancer Res October 1, 2014 74; 4747 ). | detail... | |
| 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 | neuroblastoma | not applicable | Cyclophosphamide + Topotecan | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Topotecan and Cytoxan (cyclophosphamide) combination treatment resulted in mild tumor growth delay in a neuroblastoma cell line xenograft model with wild-type ALK and TP53 H168R, and a model with wild-type ALK, wild-type TP53, and MDM2 amplification (PMID: 26438783). | 26438783 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Necitumumab | Phase I | Actionable | In a Phase I clinical trial, Portrazza (necitumumab) was well-tolerated and demonstrated preliminary efficacy in patients with advanced solid tumors (PMID: 20197484). | 20197484 | |
| 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 | prostate cancer | not applicable | EC-70124 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, EC-70124 inhibited proliferation, colony formation, Stat3 activity, and NFkappaB activity in prostate cancer cells and prostate cancer stem cells in culture and inhibited tumor growth in cell line xenografts (PMID: 26826115). | 26826115 | |
| 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 | colon 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 | pancreatic cancer | not applicable | Plicamycin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Mithracin (plicamycin) inhibited tumor growth in several human pancreatic cancer cell line xenograft models (PMID: 17973266). | 17973266 | |
| Unknown unknown | breast cancer | not applicable | MBQ-167 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MBQ-167 increased cell-cycle arrest and decreased viability and migration of breast cancer cell lines in culture, and reduced tumor growth and metastasis in a breast cancer cell line xenograft model (PMID: 28450422). | 28450422 | |
| Unknown unknown | breast cancer | not applicable | V158411 | Preclinical - Cell culture | Actionable | In a preclinical study, V158411 induced DNA damage and cell-cycle arrest, and inhibited growth of a breast cancer cell line in culture (PMID: 27829224). | 27829224 | |
| 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 | breast adenocarcinoma | not applicable | Disarib | Preclinical | Actionable | In a preclinical study, Disarib treatment led to apoptotic induction and resulted in tumor regression in breast adenocarcinoma cell line mouse models (PMID: 27693384). | 27693384 | |
| Unknown unknown | ovarian cancer | not applicable | Rucaparib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Rubraca (rucaparib) maintenance therapy significantly improved median progression-free survival compared to placebo (10.8 vs 5.4 mo, HR=0.36, p<0.0001) in patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who responded to platinum-based therapy (PMID: 28916367; NCT01968213). | 28916367 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | KHK2455 + Mogamulizumab | Phase I | Actionable | In a Phase I trial, the combination of KHK2455 and Poteligeo (mogamulizumab-kpkc) resulted in stable disease, according to RECIST, in four patients for more than 6 months and in one patient for greater than 14 months (J Clin Oncol 36, 2018 (suppl; abstr 3040). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | SGI-1027 | Preclinical - Cell culture | Actionable | In a preclinical study, SGI-1027, inhibited DNA methylation and reactivated silenced tumor suppressor genes, and induced DNMT1 degradation in a variety of cancer cell lines in culture (PMID: 19417133). | 19417133 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Midostaurin | Phase I | Actionable | In a Phase I trial, Rydapt (midostaurin) demonstrated safety in patients with advanced solid tumors (PMID: 11230495). | 11230495 | |
| Unknown unknown | hematologic cancer | not applicable | Tefinostat | Phase I | Actionable | In a Phase I trial, Tefinostat (CHR-2845) demonstrated safety and preliminary efficacy in patients with hematological cancers (PMID: 23647373). | 23647373 | |
| Unknown unknown | lymphoma | not applicable | CPI-0610 | Phase I | Actionable | In a Phase I trial, treatment with CPI-0610 in lymphoma patients resulted in decreased tumor volume in five patients and stable disease in six patients (Blood 2015 126:1491). | detail... | |
| 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 | B-cell lymphoma | not applicable | Loncastuximab tesirine | Phase I | Actionable | In a Phase I trial, Loncastuximab tesirine treatment demonstrated safety and preliminary efficacy, resulted in an overall response rate of 59.4% (41/69, 28 complete response, 13 partial response) in patients with relapsed/refractory B-cell non-Hodgkin lymphoma, with median duration of response, progression-free survival, and overall survival of 4.8, 5.5, and 11.6 months, respectively (PMID: 31685491; NCT02669017). | 31685491 | |
| Unknown unknown | prostate cancer | not applicable | Darinaparsin | Preclinical - Patient cell culture | Actionable | In a preclinical study, Darinaparsin treatment reduced tumor initiating cells and decreased viability of prostate cancer cells isolated from patients, and inhibited viability and secondary colony formation ability of tumor initiating cells (TIC), and induced cell cycle arrest in prostate cancer cell lines in culture, and inhibited tumor growth in a TIC and a cell line xenograft model (PMID: 25381261). | 25381261 | |
| Unknown unknown | glioblastoma | not applicable | MPS1-IN-3 + Vincristine Sulfate | Preclinical | Actionable | In a preclinical study, MPS1-IN-3, in combination with Oncovvin (vincristine), inhibited cell growth of glioblastoma cells in culture and in xenograft GBM models (PMID: 23940287). | 23940287 | |
| 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 | 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 | hematologic cancer | not applicable | Abexinostat | Phase II | Actionable | In a Phase II trial, treatment with Abexinostat (PCI-24781) in patients with a variety of hematological cancers resulted in an overall response rate (ORR) of 28% (24/87), a complete response in 5%, and a median duration response of 8.8 months (PMID: 28126962). | 28126962 | |
| Unknown unknown | dermatofibrosarcoma protuberans | not applicable | Imatinib | FDA approved | Actionable | In a Phase II clinical trial that supported FDA approval, treatment with Gleevec (imatinib) resulted in a median time-to-progression of 23.9 months, and complete response in 33% (4/12) and partial response in 50% (6/12) of patients with dermatofibrosarcoma protuberans (PMID: 18451237). | 18451237 detail... | |
| Unknown unknown | acral lentiginous melanoma | 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 23.1% (3/13) and a disease control rate of 46.2% (6/13, 1 complete response, 2 partial response, 3 stable disease) in patients with acral melanoma (PMID: 30642373; NCT02836795). | 30642373 | |
| Unknown unknown | acral lentiginous 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 | 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 | malignant fibrous histiocytoma | not applicable | IPA-3 | Preclinical - Cell culture | Actionable | In a preclinical study, IPA-3 increased apoptosis and inhibited growth of a myxofibrosarcoma cell line in culture (PMID: 27577794). | 27577794 | |
| Unknown unknown | urinary bladder cancer | not applicable | Celecoxib + OBP-801 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Celebra (celecoxib) and OBP-801 resulted in a synergistic effect, demonstrating increased apoptotic activity and decreased tumor volume in xenograft models of bladder cancer (PMID: 27406983). | 27406983 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Eribulin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Halaven (eribulin) inhibited epithelial-to-mesenchymal transition and increased tumor perfusion in triple-negative breast cancer cell line xenograft models (PMID: 25838395). | 25838395 | |
| Unknown unknown | stomach cancer | not applicable | MGD013 | Case Reports/Case Series | Actionable | In a Phase I trial, MGD013 treatment resulted in a partial response in a patient with gastric cancer (J Clin Oncol 38: 2020 (suppl; abstr 3004); NCT03219268). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CYT01B + Olaparib | Preclinical - Cell culture | Actionable | In a preclinical study, CYT01B and Lynparza (olaparib) synergistically inhibited growth of tumor cell lines in culture (AACR Annual Meeting 2019, Abstract 363). | detail... | |
| 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 | breast cancer | not applicable | AsiDNA + Iniparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA and Iniparib combination treatment resulted in increased cell death and inhibition of proliferation in breast cancer cell lines compared to Iniparib alone in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | transitional cell carcinoma | not applicable | Enfortumab vedotin-ejfv | FDA approved | Actionable | In a Phase II trial (EV-201) that supported FDA approval, Padcev (enfortumab vedotin-ejfv) treatment resulted in an objective response rate of 42% (53/125) with complete response in 9% (11/125) of patients with locally advanced or metastatic urothelial cancer who have previously received a PD-1 or PD-L1 inhibitor, and a platinum-containing chemotherapy (Journal of Clinical Oncology 37, no. 18_suppl (June 20, 2019) 4505-4505; NCT03219333). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | GNE-272 | Preclinical - Cell culture | Actionable | In a preclinical study, GNE-272 treatment resulted in anti-proliferative activity in multiple myeloma cell lines in culture (PMID: 27682507). | 27682507 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Docetaxel + SGT-53 | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of Taxotere (docetaxel) and SGT-53 demonstrated safety, and out of 12 evaluable patients resulted in partial response (PR) in 2 patients, an unverified PR in 1 patient, stable disease with tumor shrinkage in 2 patients, and stable disease without tumor shrinkage in 4 patients with advanced solid tumors (PMID: 27357628). | 27357628 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Docetaxel + TAK-243 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Taxotere (docetaxel) 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 | myelodysplastic/myeloproliferative neoplasm | not applicable | Ruxolitinib | Phase I | Actionable | In a Phase I trial, Jakafi (ruxolitinib) treatment demonstrated safety and preliminary efficacy in chronic myelomonocytic leukemia patients, resulted in a total response rate of 35% (7/20) and reduction of Stat5 signaling (PMID: 26858309). | 26858309 | |
| 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 | Advanced Solid Tumor | not applicable | SAR566658 | Phase I | Actionable | In a phase I trial, SAR566658 treatment resulted in complete response in 1% (1/114), partial response in 7% (8/114), and stable disease in 39% (44/114) of patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2511)). | detail... | |
| Unknown unknown | melanoma | not applicable | BO-112 | Preclinical - Patient cell culture | Actionable | In a preclinical study, BO-112 treatment inhibited cell viability and induced apoptosis in mouse melanoma cells, and induced cytotoxicity and increased apoptosis in cells derived from metastatic lesions of melanoma patients in culture, and intratumoral delivery of BO-112 enhanced infiltration of T-lymphocytes, reduced tumor growth, and delayed progression in syngeneic mouse models (PMID: 31046839). | 31046839 | |
| 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 | lung cancer | not applicable | PF-00562271 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, PF-00562271 inhibited PTK2 (FAK) phosphorylation and resulted in apoptosis and tumor suppression in lung cancer cell line xenograft models (PMID: 18339875). | 18339875 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ALM201 | Phase I | Actionable | In a Phase I trial, ALM201 demonstrated safety and acceptable pharmacokinetics in patients with advanced solid tumors, and 2 of 18 evaluable patients demonstrated stable disease for up to 6 cycles (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #383P). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | IsoA | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with IsoA inhibited cell growth in a variety of cancer cell lines in culture, and induced apoptosis via ROS generation (PMID: 28011619). | 28011619 | |
| Unknown unknown | splenic marginal zone lymphoma | not applicable | ST7612AA1 | Preclinical | Actionable | In a preclinical study, ST7612AA1 inhibited proliferation of splenic marginal zone lymphoma cell lines in culture (PMID: 25671299). | 25671299 | |
| Unknown unknown | melanoma | not applicable | Prolgolimab | Phase II | Actionable | In a Phase II trial (MIRACULUM), Prolgolimab demonstrated safety and preliminary efficacy, resulted in an objective response rate (ORR) of 38% (24/63, 5 complete response (CR), 19 partial response (PR)) and a disease control rate (DCR) of 64% when administered at 1mg/kg Q2W, and an ORR of 29% (18/63, 2 CR, 16 PR) with a DCR of 46% when administered at 3mg/kg Q3W, in patients with advanced melanoma (Annals of Oncology, Volume 30, Issue Supplement_11, December 2019, Abstract 119P; NCT03269565). | detail... | |
| 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 | renal cell carcinoma | not applicable | CPI-444 | Phase I | Actionable | In a Phase I trial, treatment with single agent CPI-444 was well-tolerated and resulted in a disease control rate of 75% (3/4) in patients with renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 3004)). | detail... | |
| Unknown unknown | neuroendocrine tumor | not applicable | Axitinib | Phase II | Actionable | In a Phase II clinical trial, treatment with Inlyta (axitinib) resulted in a median overall progression-free survival (PFS) of 26.7 months, a 12-month PFS rate of 74.5%, and median overall survival of 45.3 months, and led to partial response in 3% (1/30) and stable disease in 70% (21/30) of patients with neuroendocrine tumors, with some tumor shrinkage in 68% (15/22) of patients (PMID: 27080472). | 27080472 | |
| Unknown unknown | renal carcinoma | not applicable | Cabozantinib | Phase II | Actionable | In a Phase II trial, renal cancer patients treated with Cabozantinib demonstrated a 28 % objective response rate, a 62 % stable disease rate, and a median progression free survival of 14.7 months (PMID: 23292795). | 23292795 | |
| Unknown unknown | prostate cancer | not applicable | C6-ceramide | Preclinical | Actionable | In a preclinical study, C6-ceramide treatment of prostate cancer cells prevented the association between SET and PP2A, which resulted in cell death in culture (PMID: 24025258). | 24025258 | |
| Unknown unknown | 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 breast cancer, with 58% (7/12) of breast cancer patients achieving objective response or stable disease (PMID: 27213589). | 27213589 | |
| Unknown unknown | lymphoma | not applicable | Toripalimab | Phase I | Actionable | In a Phase I trial, Toripalimab (JS001) treatment in patients with advanced lymphomas resulted in an objective response rate of 90.9% (10/11) and median progression-free survival of 8.3 months, with all nine Hodgkin's lymphoma patients demonstrating objective responses including six complete responses and four partial responses, while one diffuse large B-cell lymphoma patient achieved a partial response and the other resulted in disease progression (PMID: 32224416; NCT02836834). | 32224416 | |
| Unknown unknown | multiple myeloma | no benefit | Selumetinib | Phase II | Actionable | In a Phase II clinical trial, Selumetinib (AZD6244) demonstrated safety and tolerability but had minimal activity with a complete response achieved in 5.6% (2/36) of refractory multiple myeloma patients (PMID: 26446942). | 26446942 | |
| Unknown unknown | ovarian serous carcinoma | not applicable | CFI-402257 | Preclinical - Pdx | Actionable | In a preclinical study, CFI-402257 growth in patient-derived xenograft (PDX) models of platinum-sensitive and platinum-resistant high-grade serous ovarian cancer (PMID: 28270606). | 28270606 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BI-847325 | Phase I | Actionable | In a Phase I trial, BI-847325 treatment resulted in stable disease in 30% (21/69) of patients with advanced solid tumors, and one partial response for 67 days in a patient with esophageal cancer (PMID: 26650227). | 26650227 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BI-847325 | Phase I | Actionable | In a Phase I trial, BI-847325 demonstrated safety and some efficacy in a variety of advanced solid tumors (Mol Cancer Ther 2013;12(11 Suppl):B281). | detail... | |
| Unknown unknown | Hodgkin's lymphoma | not applicable | Edicotinib | Phase II | Actionable | In a Phase II/III clinical trial, JNJ-40346527 demonstrated safety some efficacy, with complete response in 5.0% (1/20), partial response in 5.0% (1/20), and stable disease in 55.0% (11/20) of patients with refractory Hodgkin's lymphoma (PMID: 25628399). | 25628399 | |
| 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 | endometrial cancer | not applicable | Lenvatinib + Pembrolizumab | Phase Ib/II | Actionable | Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 52% (12/23) in patients with metastatic endometrial cancer, with a median duration of response not evaluable, and a median progression-free survival of 9.7 months (PMID: 31961766; NCT02501096). | 31961766 | |
| Unknown unknown | melanoma | not applicable | CBT-502 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CBT-502 treatment inhibited tumor growth in a cell line xenograft model of melanoma (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr A200). | detail... | |
| 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 | gastrointestinal stromal tumor | not applicable | Pimitespib | Phase I | Actionable | In a Phase I trial, TAS-116 treatment resulted in partial response in a patient with gastrointestinal stromal tumor (J Clin Oncol 35, 2017 (suppl; abstr 2546)). | detail... | |
| Unknown unknown | gastrointestinal stromal tumor | not applicable | Pimitespib | Phase II | Actionable | In a Phase II trial, treatment with Pimitespib (TAS-116) in patients with a gastrointestinal stromal tumor who failed previous therapy (n=40) resulted in a progression-free survival of 4.4 months, a median overall survival of 11.5 months, a 0% objective response rate, and a disease control rate of 85%, with 34 patients achieving stable disease, and one patient experienced a 37.2% decrease in tumor size after the analysis cut-off date (PMID: 31536852). | 31536852 | |
| Unknown unknown | glioblastoma | not applicable | Cediranib + Lomustine | Clinical Study | Actionable | In a clinical case study, a patient with recurrent glioblastoma treated with Cediranib (AZD-2171), in combination with Lomustine (CCNU), demonstrated 50% tumor regression at 6 weeks, complete response at 24 weeks, and achieved clinical remission for over 6 years (PMID: 26929887). | 26929887 | |
| 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 | Camptothecin + NU6027 | Preclinical - Cell culture | Actionable | In a preclinical study, NU6027 enhanced the efficacy of Camptothecin in breast cancer cells in culture, resulting in decreased cell survival (PMID: 21730979). | 21730979 | |
| Unknown unknown | childhood B-cell acute lymphoblastic leukemia | not applicable | Tisagenlecleucel | FDA approved | Actionable | In a Phase II trial (ELIANA) that supported FDA approval, Kymriah (tisagenlecleucel) treatment led to complete remission or complete remission with incomplete blood count recovery in 83% (52/63) of pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (22nd Congress of EHA, June 2017, Abstract S476; NCT02435849). | detail... detail... detail... detail... | |
| Unknown unknown | prostate cancer | not applicable | Abiraterone + Olaparib | Phase II | Actionable | In a Phase II trial, treatment with the combination of Lynparza (olaparib) and Zytiga (abiraterone) resulted in a prolonged median radiographic progression-free survival of 13.8 months, compared to 8.2 months with placebo plus Zytiga (abiraterone), in patients with metastatic castration-resistant prostate cancer (PMID: 29880291; NCT01972217). | 29880291 | |
| Unknown unknown | melanoma | not applicable | Axitinib | Phase II | Actionable | In a Phase II study in patients with metastatic melanoma, Inlyta (axitinib) was well tolerated and demonstrated antitumor activity (PMID: 21976544). | 21976544 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Decitabine + Glasdegib | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of Glasdegib (PF-04449913) and Dacogen (decitabine) resulted in an overall survival of 11.5 months in patients with either acute myeloid leukemia (AML) or myelodysplastic syndrome, with 40% (2/5) of AML patients experiencing a complete remission (PMID: 29463550). | 29463550 | |
| 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 | chronic myeloid leukemia | not applicable | RN-1 | Preclinical - Cell culture | Actionable | In a preclinical study, RN-1 inhibited growth of chronic myeloid leukemia cell lines in culture (PMID: 26837761). | 26837761 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Lenalidomide + Tafasitamab-cxix | FDA approved | Actionable | In a Phase II trial (L-MIND) that supported FDA approval, the combination of Revlimid (lenalidomide) and Monjuvi (tafasitamab-cxix) was well tolerated in patients with previously treated diffuse large B-cell lymphoma and resulted in an objective response rate of 60% (48/80, 34 complete responses, 14 partial responses) and stable disease in 14% (11/80) of patients, with a median progression-free survival of 12.1 months (PMID: 32511983; NCT02399085). | 32511983 detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | TTI-621 | Preclinical - Cell culture | Actionable | In a preclinical study, TTI-621 (SIRPalpha-Fc) increased phagocytosis in 67% (8/12) of the human solid tumor cell lines tested in culture (PMID: 27856600). | 27856600 | |
| 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 | juvenile astrocytoma | not applicable | MRK-003 | Preclinical | Actionable | In a preclinical study, MRK-003 inhibited HES1 expression in pediatric low-grade astrocytoma cell lines in culture and decreased migration in 1 of 2 cell lines, but did not have a significant effect on cell growth (PMID: 25575134). | 25575134 | |
| Unknown unknown | glioblastoma | not applicable | LYS6KAKT1 | Preclinical | Actionable | In a preclinical study, LYS6KAKT1 inhibited phosphorylation of p70S6 kinase in mice engrafted with glioblastoma cells (Mol Cancer Ther 2011;10(11 Suppl):Abstract nr B117). | detail... | |
| Unknown unknown | prostate 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 43% (3/7) of metastatic castration-resistant prostate cancer patients with a duration of response of 50-124 days, and a median progression-free survival of 1-3 months (PMID: 32847933; NCT02265536). | 32847933 | |
| 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 | lung non-squamous non-small cell carcinoma | not applicable | Carboplatin + Linifanib + Paclitaxel | Phase II | Actionable | In a Phase II clinical, Linifanib (ABT-869), in combination with Taxol (paclitaxel) and Paraplatin (carboplatin), increased progression free survival in patients with nonsquamous non-small cell lung cancer (PMID: 25559798). | 25559798 | |
| Unknown unknown | pancreatic cancer | not applicable | G-TPP + Navitoclax | Preclinical - Cell culture | Actionable | In a preclinical study, combination of the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Navitoclax (ABT-263) resulted in enhanced growth inhibition of pancreatic cancer cells in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | IT-901 | Preclinical | Actionable | In a preclinical study, IT-901 inhibited growth of diffuse large B-cell lymphoma cells in culture (PMID: 26744524). | 26744524 | |
| Unknown unknown | lung non-squamous non-small cell carcinoma | not applicable | Itraconazole + Pemetrexed Disodium | Phase II | Actionable | In a Phase II trial, Itraconazole, in combination with Alimta (pemetrexed), increased overall survival by 24 months in patients with metastatic nonsquamous non-small cell lung cancer (PMID: 23546045). | 23546045 | |
| Unknown unknown | multiple myeloma | not applicable | Daratumumab + Dexamethasone + Pomalidomide | FDA approved | Actionable | In a Phase I trial (EQUULEUS) that supported FDA approval, Darzalex (Daratumumab) in combination with Pomalyst (pomalidomide) and Adexone (dexamethasone) resulted in an objective response rate of 60% (61/103) in patients with relapsed or refractory multiple myeloma, with a median progression-free survival of 8.8 months and a median overall survival of 17.5 months (PMID: 28637662; NCT01998971). | detail... 28637662 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ETC-159 | Phase I | Actionable | In a Phase I trial, treatment with ETC-159 was well-tolerated, decreased Wnt signaling, and resulted in stable disease in 2 out of 16 treated patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2584)). | detail... | |
| Unknown unknown | pancreatic cancer | not applicable | Napabucasin | Preclinical | Actionable | In a preclinical study, BBI608 inhibited tumor growth and metastasis in xenograft models of pancreatic cancer (PMID: 25605917). | 25605917 | |
| Unknown unknown | thyroid gland cancer | not applicable | ABT-737 + Cisplatin | Preclinical | Actionable | In a preclinical study, suboptimal dosing of ABT-737 and Platinol (cisplatin) did not affect cell viability of human thyroid cancer cell lines in culture (PMID: 27042160). | 27042160 | |
| Unknown unknown | stomach cancer | not applicable | RX-0201 | Preclinical | Actionable | In a preclinical study, RX-0201 prevented cell proliferation of stomach cancer cells (JASCO Annual Meeting Proceedings Vol 24, No 18S (June 20 Supplement), 2006: 13102). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Abexinostat | Phase II | Actionable | In a Phase II trial, patients with diffuse large B-cell lymphoma demonstrated an overall response rate of 31% (5/16) and a median duration response of 1.9 months when treated with Abexinostat (PCI-24781) (PMID: 28126962). | 28126962 | |
| Unknown unknown | melanoma | not applicable | Ramucirumab | Phase II | Actionable | In a Phase II trial, Cyramza (ramucirumab), with or without Deticene (dacarbazine), demonstrated safety and efficacy in patients with metastatic melanoma. The combined therapy appeared to be associated with improved progression free survival relative to monotherapy (J Clin Oncol 28:15s, 2010 (suppl; abstr 8519)). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | CPI-0610 + Rucaparib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination treatment of Rubraca (rucaparib) and CPI-0610 resulted in a synergistic effect, demonstrating greater inhibition of cell growth and colony formation in cultured ovarian cancer cells and a 58% decrease in tumor weight in cell line xenograft models (PMID: 32927276). | 32927276 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | A-1331852 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, A-1331852 inhibited tumor growth in several solid tumor cell line xenograft models (PMID: 25787766). | 25787766 | |
| 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 | acute myeloid leukemia | not applicable | SNS-229 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with SNS-229 resulted in decreased PDPK1 pathway signaling and tumor growth inhibition in an acute myeloid leukemia cell line xenograft model (Mol Cancer Ther, Dec 1 2015 (14) (12 Supplement 2) C198). | detail... | |
| Unknown unknown | sarcoma | not applicable | Aldoxorubicin + Alvocidib | Phase II | Actionable | In a Phase I trial, Alvocidib (flavopiridol), in combination with aldoxorubicin resulted in partial responses in 6% (2/31) and stable disease in 51% (16/31) of patients with advanced sarcoma (PMID: 22374332). | 22374332 | |
| Unknown unknown | acute lymphoblastic leukemia | not applicable | AS605240 + Dexamethasone | Preclinical - Pdx | Actionable | In a preclinical study, the combination of AS605240 and dexamethasone improved survival of primary T-ALL cell xenograft models (PMID: 25869207). | 25869207 | |
| Unknown unknown | colon cancer | not applicable | GANT61 | Preclinical | Actionable | In a preclinical study, treatment with the GLI1/GLI2 inhibitor, GANT61, resulted in cytotoxicity in colon cancer cell lines (PMID: 21135115). | 21135115 | |
| 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 | 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 | multiple myeloma | not applicable | Elotuzumab + GDA-201 | Phase I | Actionable | In a Phase I trial, GDA-201 and Empliciti (elotuzumab) combination therapy demonstrated safety and preliminary efficacy, resulted in complete response in 7.7% (1/13) and stable disease in 30.8% (4/13) of patients with refractory multiple myeloma, with a median duration of response of 2.5 months (Blood (2019) 134 (Supplement_1): 777). | detail... | |
| Unknown unknown | mantle cell lymphoma | not applicable | Buparlisib + Ibrutinib | Phase Ib/II | Actionable | In a Phase I/II trial, Buparlisib (BKM120) and Imbruvica (ibrutinib) combination treatment resulted in a best overall response rate of 88% (15/17, 11 complete response, 4 partial response) in patients with relapsed/refractory mantle cell lymphoma (J Clin Oncol 36, 2018 (suppl; abstr 7520); NCT02756247). | detail... | |
| 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 | 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 | 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 | prostate adenocarcinoma | not applicable | NSC23766 | Preclinical - Cell culture | Actionable | In a preclinical study, NSC23766 inhibited proliferation and invasion of a prostate adenocarcinoma cell line in culture (PMID: 15128949). | 15128949 | |
| Unknown unknown | neuroendocrine tumor | not applicable | Toripalimab | Phase Ib/II | Actionable | In a Phase Ib trial, treatment with Toripalimab (JS001) resulted in an objective response rate of 20% (8/40), a disease control rate of 35% (14/40), a median duration of response of 15.2 months, a median progression-free survival of 2.5 months, and a median overall survival of 7.8 months in patients with neuroendocrine tumors (PMID: 32086343; NCT03167853). | 32086343 | |
| Unknown unknown | gastric adenocarcinoma | not applicable | Regorafenib | Phase II | Actionable | In a Phase II trial, Stivarga (regorafenib) resulted in a PFS of 2.6 months compared to .9 months with placebo in gastric adenocarcinoma patients (PMID: 27325864). | 27325864 | |
| 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 | chronic lymphocytic leukemia | not applicable | AMG 176 + Venetoclax | Preclinical - Cell culture | Actionable | In a preclinical study, the combination therapy of AMG 176 and Venclexta (venetoclax) resulted in synergistic and additive effects in patient-derived cells of chronic lymphocytic leukemia, demonstrating apoptotic activity in culture (PMID: 31937611). | 31937611 | |
| Unknown unknown | colorectal cancer | not applicable | Olaparib + Temozolomide | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the addition of Lynparza (olaparib) 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 | MLN1117 + Sapanisertib | Preclinical | Actionable | In a preclinical study, the combination of Sapanisertib (MLN0128) and MLN1117 demonstrated synergistic anti-tumor activity in a variety of solid tumor xenograft models (Mol Cancer Ther 2013;12(11 Suppl):C176)). | detail... | |
| Unknown unknown | melanoma | not applicable | CCT129254 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CCT129254 inhibited motility of two human melanoma cell lines in invasion assays in culture and inhibited metastasis in a mouse melanoma cell line xenograft model (PMID: 25840982). | 25840982 | |
| Unknown unknown | colorectal cancer | not applicable | Aflibercept | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Zaltrap (aflibercept) improved median progression free survival to 6.9 months and overall survival to 13.5 months in patients with metastatic colorectal cancer (PMID: 23444216). | 23444216 detail... | |
| Unknown unknown | glioblastoma | 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 21.4% (3/14) of glioblastoma patients, and a median progression-free survival of 56.5 days (PMID: 31853198; NCT01353625). | 31853198 | |
| Unknown unknown | glioblastoma | not applicable | CC-115 | Preclinical - Pdx | Actionable | In a preclinical study, CC-115 increased survival of patient derived xenograft models of glioblastoma (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 1755). | detail... | |
| Unknown unknown | malignant glioma | not applicable | RES-529 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, RES-529 (Palomid 529) inhibited tumor growth and angiogenesis in glioma cell line xenograft models (PMID: 19010932). | 19010932 | |
| Unknown unknown | pancreatic cancer | not applicable | siG12D-LODER | Preclinical | Actionable | In a preclinical study, siG12D-LODER treatment inhibited migration of pancreatic cancer cells in culture, and induced apoptosis, and reduced lung, liver, and spleen metastases in a syngeneic mouse model (PMID: 26009994). | 26009994 | |
| 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 | lung non-small cell carcinoma | not applicable | Durvalumab | FDA approved | Actionable | In a Phase III trial (PACIFIC) that supported FDA approval, Imfinzi (durvalumab) treatment resulted significantly improved median progression-free survival (PFS) (16.8 vs 5.6 months, HR=0.52, p<0.001), and superior 12-month PFS rate (55.9% vs 35,3%), 18-month PFS rate (44.2% vs 27.0%), response rate (28.4% vs 16%), and median time to death or distant metastasis (23.2 vs 14.6 months) compared to placebo in patients with unresectable, non-small cell lung cancer (PMID: 28885881; NCT02125461). | 28885881 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 | ovarian carcinoma | not applicable | Rucaparib | Phase II | Actionable | In a Phase II trial, Rubraca (rucaparib) demonstrated activity in patients with platinum-sensitive high-grade ovarian carcinoma, with patients in the BRCA mutant and BRCA wild-type with high genomic loss-of-heterozygosity subgroups demonstrating increased progression-free survival compared to the BRCA wild-type with low genomic loss-of-heterozygosity subgroup (PMID: 27908594). | 27908594 | |
| 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 | prostate cancer | not applicable | Ipilimumab + Nivolumab | Phase II | Actionable | In a Phase II trial (CheckMate 650), Opdivo (nivolumab) plus Yervoy (ipilimumab) in metastatic castration-resistant prostate cancer patients who had not received prior chemotherapy versus those who had resulted in respective objective response rates of 25% (8/32, 2 complete responses) v 10% (3/30, 2 complete responses), median radiographic progression-free survival of 5.5 v 3.8 mo, median overall survival of 19.0 v 15.2 mo, and PSA response rate of 17.6% (6/34) v 10.0% (4/40) (PMID: 32916128; NCT02985957). | 32916128 | |
| Unknown unknown | colorectal cancer | not applicable | Zenocutuzumab | Phase Ib/II | Actionable | In a Phase I/II trial, Zenocutuzumab (MCLA-128) resulted in stable disease for 5 months in a patient with colorectal cancer (Cancer Res 2016;76(14 Suppl):Abstract nr CT050). | detail... | |
| Unknown unknown | follicular lymphoma | not applicable | Tazemetostat | Case Reports/Case Series | Actionable | In a Phase I trial, Tazemetostat (EPZ-6438) demonstrated safety and preliminary efficacy, resulted in 2 complete responses and 1 partial response in patients with relapsed or refractory follicular lymphoma (PMID: 29650362; NCT01897571). | 29650362 | |
| Unknown unknown | follicular lymphoma | not applicable | Tazemetostat | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Tazemetostat (EPZ-6438) treatment was well-tolerated and demonstrated clinical activity in patients with relapsed/refractory follicular lymphoma, resulting in an objective response rate of 77% (33/43) and 34% (18/53), stable disease in 23% (10/43) and 30% (16/53), and median progression-free survival of 11.1 and 5.7 months in EZH2 mutant and wild-type cohorts, respectively (Blood (2019) 134 (Supplement_1):123; NCT01897571). | detail... | |
| 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 | Advanced Solid Tumor | not applicable | CDX-1140 + CDX-301 | Phase I | Emerging | In a Phase I trial, CDX-1140 and CDX-301 combination treatment demonstrated safety and expected lymphocyte activation and cytokine responses in patients with advanced solid tumors (AACR Annual Meeting 2019, Abstract LB_194; NCT03329950). | detail... | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | JQ1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, triple-receptor negative breast cancer cells demonstrated sensitivity to treatment with JQ1, resulting in decrease cell proliferation and cell cycle arrest in culture and tumor growth inhibition in cell line xenograft models (PMID: 27256375). | 27256375 | |
| Unknown unknown | endometrial cancer | not applicable | Sunitinib | Phase II | Actionable | In Phase II clinical trials, Sutent (sunitinib) demonstrated efficacy in patients with metastatic or recurrent endometrial carcinoma (PMID: 24882554). | 24882554 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | GS-5829 + Venetoclax | Preclinical - Cell culture | Actionable | In a preclinical study, diffuse large B-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 | 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 an overall response rate of 33% (33/99) in leukemia patients and a response rate of 64% (14/22) in patients with aggressive myeloproliferative neoplasms or chronic myelomonocytic leukemia (PMID: 27551000). | 27551000 | |
| 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 | stomach cancer | no benefit | Fluorouracil + Ipatasertib + Leucovorin + Oxaliplatin | Phase II | Actionable | In a Phase II trial, the combination of Ipatasertib (GDC-0068) and mFOLFOX6 did not improve median progression-free survival (6.6 vs 7.5 months; HR=1.12, p=0.56), median overall survival (12.12 vs 15.67 months; HR=1.85), or objective response rate (52% (37/71) vs 56% (46/82)) compared to placebo plus mFOLFOX6 in patients with advanced gastric or gastroesophageal junction cancer (PMID: 30592991; NCT01896531). | 30592991 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Docetaxel + S63845 | Preclinical - Pdx | Actionable | In a preclinical study, S63845 and Taxotere (docetaxel) demonstrated synergy in triple-negative breast cancer patient-derived xenograft models, resulting in enhanced tumor growth inhibition and overall survival compared to either agent alone (PMID: 28768804). | 28768804 | |
| Unknown unknown | breast cancer | not applicable | Avelumab + PF-06840003 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, PF-06840003 and Bavencio (avelumab) combination treatment inhibited tumor growth in a cell line xenograft model of breast cancer engrafted with human CD34-positive cells (PMID: 30232146). | 30232146 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Pexidartinib + PLX9486 | Phase I | Actionable | In a Phase I trial, PLX9486 and Pexidartinib (PLX3397) combination therapy demonstrated safety and preliminary efficacy, resulted in a partial response rate of 8.3% (1/12) and progression-free survival not yet reached in patients with advanced solid tumors, 11 of these patients had gastrointestinal stromal tumor that progressed on Gleevec (imatinib mesylate), and most harbored KIT exon 11 and exon 17 mutations (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 11509-11509; NCT02401815). | detail... | |
| Unknown unknown | colon cancer | not applicable | TPST-1495 + unspecified PD-1 antibody | Preclinical | Actionable | In a preclinical study, TPST-1495 in combination with an anti-PD-1 antibody synergistically inhibited tumor growth in a syngeneic mouse model of colon cancer (Journal for ImmunoTherapy of Cancer 2019;7:282, Abs nr: P311). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Pimasertib | Phase I | Actionable | In a Phase I trial, Pimasertib (MSC1936369B) demonstrated safety and some preliminary anti-tumor activity in patients with advanced solid tumors (J Clin Oncol 28:15s, 2010 (suppl; abstr 2504)). | detail... | |
| Unknown unknown | breast cancer | not applicable | Aldoxorubicin + Rigosertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, human breast cancer cells demonstrated a complete tumor response in cell line xenograft models when treated with a combination of Rigosertib (ON01910) and Aldoxorubicin (PMID: 15766665). | 15766665 | |
| Unknown unknown | melanoma | not applicable | Ipilimumab + Radiotherapy | Clinical Study | Actionable | In a retrospective study, Yervoy (ipilimumab) in combination with local tumor treatment consisting of radiotherapy or electrochemotherapy resulted in improved median overall survival (93 weeks) compared to Yervoy (ipilimumab) alone (42 weeks) in advanced melanoma patients (PMID: 27466265). | 27466265 | |
| Unknown unknown | colon 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 50% (2/4) of patients with colon cancer (PMID: 26842236). | 26842236 | |
| Unknown unknown | adrenal gland pheochromocytoma | not applicable | 131I-MIBG | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Azedra (iobenguane I 131) treatment resulted in partial response in 23% (15/68) of patients with pheochromocytoma or paraganglioma who received 1 therapeutic dose, with a 12-month overall survival rate of 91% (J Clin Oncol 36, 2018 (suppl; abstr 4005); NCT00874614). | detail... detail... | |
| Unknown unknown | mantle cell lymphoma | not applicable | Parsaclisib | Phase Ib/II | Actionable | In a Phase I/II trial, Parsaclisib (INCB050465) treatment demonstrated tolerability and preliminary activity in patients with refractory B-cell malignancies, and resulted in an overall response rate of 67 (6/9), complete response/complete metabolic response rate of 44% (4/9), and median duration of response was not reached in patients with mantle cell lymphoma (PMID: 30803990; NCT02018861). | 30803990 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Cytarabine + Filgrastim + GDC-0575 | Preclinical - Pdx | Actionable | In a preclinical study, addition of Neupogen (filgrastim) to the combination of GDC-0575 and Cytosar-U (cytarabine) resulted in enhanced killing of acute myeloid leukemia cells in patient-derived xenograft (PDX) models (PMID: 29162833). | 29162833 | |
| 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 | prostate cancer | not applicable | ABBV-744 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, ABBV-744 inhibited growth of prostate cancer cells in culture and in cell line xenograft models (Cancer Res 2018;78(13 Suppl):Abstract nr DDT01-05). | detail... | |
| Unknown unknown | melanoma | not applicable | PF-06840003 | Preclinical | Actionable | In a preclinical study, PF-06840003 inhibited tumor growth in a syngeneic mouse model of melanoma (PMID: 30232146). | 30232146 | |
| Unknown unknown | breast cancer | not applicable | AZD8186 | Preclinical - Cell culture | Actionable | In a preclinical study, AZD8186 inhibited proliferation of several breast cancer cell lines in culture (PMID: 25398829). | 25398829 | |
| Unknown unknown | lung cancer | not applicable | SNG12 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the TTK (MPS1) inhibitor, SNG12, inhibited proliferation of lung cancer cells in culture and suppressed tumor growth in cell line xenograft models (PMID: 24900510). | 24900510 | |
| Unknown unknown | ovarian cancer | not applicable | Carboplatin + Nintedanib + Paclitaxel | Phase III | Actionable | In a Phase III clinical trial, patients with advanced ovarian cancer treated with Ofev (nintedanib), in combination with Paraplatin (carboplatin) and Taxol (paclitaxel), experienced a progression free survival of 17.2 months versus 16.6 months in patients treated with a combination of placebo, carboplatin, and paclitaxel in first-line treatment (PMID: 26590673). | 26590673 | |
| 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 | prostate cancer | not applicable | UC-773587 | Preclinical | Actionable | In a preclinical study, UC-773587 inhibited ERK activation and growth of prostate cells in culture (PMID: 25825487). | 25825487 | |
| 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 | pancreatic cancer | not applicable | PH11 | Preclinical | Actionable | In a preclinical study, treatment with PH11 led to increased sensitivity to TRAIL in pancreatic cancer cells thereby restoring apoptosis (PMID: 25684663). | 25684663 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Tislelizumab | Phase I | Actionable | In a Phase I trial, Tislelizumab (BGB-A317) demonstrated safety and resulted in an objective response rate of 13.3% (60/451), including six complete responses and 54 partial responses, and led to stable disease in 141 patients for a disease control rate of 44.6% (201/451), and a clinical benefit rate of 25.9% (117/451) in patients with advanced solid tumors (PMID: 32540858; NCT02407990). | 32540858 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Abivertinib + Omacetaxine mepesuccinate | Preclinical - Patient cell culture | Actionable | In a preclinical study, Abivertinib (AC0010) treatment in combination with Synribo (omacetaxine mepesuccinate) enhanced reduction in viability of an acute myeloid leukemia cell line and acute leukemia blasts derived from patients compared to either agents alone in culture (PMID: 31310800). | 31310800 | |
| Unknown unknown | stomach cancer | no benefit | Ipilimumab | Phase II | Actionable | In a Phase II trial, Yervoy (ipilimumab) did not improve immune-related progression-free survival (2.9 vs 4.9 months) compared to best supportive care in patients with unresectable, locally advanced/metastatic gastric or gastroesophageal junction cancer (J Clin Oncol 34, 2016 (suppl; abstr 4011)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Citarinostat + Paclitaxel | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of ACY-241 and Taxol (paclitaxel) resulted in increased efficacy in advanced solid tumor xenograft models, including pancreatic and ovarian (AACR; Cancer Res 2016;76(14 Suppl):Abstract nr 4822). | 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 | Advanced Solid Tumor | not applicable | Emibetuzumab | Phase I | Actionable | In a Phase I trial, Emibetuzumab (LY2875358) treatment resulted in durable partial response in 4.3% (1/23) of patients with advanced solid tumors (PMID: 27803065). | 27803065 | |
| Unknown unknown | Ewing sarcoma | not applicable | MEDI-573 + Vistusertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of MEDI-573 and Vistusertib (AZD2014) inhibited proliferation of a Ewing sarcoma cell line in culture, and inhibited tumor growth in a Ewing sarcoma cell line xenograft model, with increased efficacy compared to either as a single agent (PMID: 25193511). | 25193511 | |
| Unknown unknown | Ewing sarcoma | not applicable | Talazoparib | Phase I | Actionable | In a Phase I trial, Talazoparib (BMN-673) treatment in patients with Ewing sarcoma did not result in any objective responses, however, resulted in a clinical benefit rate of 23% (PMID: 28242752). | 28242752 | |
| Unknown unknown | cutaneous T cell lymphoma | not applicable | Duvelisib | Phase I | Actionable | In a Phase I trial, Copiktra (duvelisib) treatment resulted in an overall response rate of 31.6% (6/19, 6 partial response) with a median duration of treatment of 12.6 weeks in patients with cutaneous T cell lymphoma (PMID: 29233821; NCT01476657). | 29233821 | |
| Unknown unknown | acute myeloid leukemia | 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 | Seribantumab + XL147 | Phase Ib/II | Actionable | In a Phase Ib trial, treatment with the combination of Pilaralisib (SAR245408, XL147) and Seribantumab (SAR256212) resulted in stable disease as best response in 52.2% (12/23) patients with advanced solid tumors, with no difference in response between patients harboring PIK3CA mutations and those with wild-type PIK3CA (PMID: 28031425). | 28031425 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | iNeo-Vac-P01 + Sargramostim | Phase I | Actionable | In a Phase I trial, iNeo-Vac-P01 administered with Leukine (sargramostim) as an adjuvant demonstrated safety in patients with advanced solid tumors and resulted in a disease control rate of 71.4% (15/21), including measurable tumor reduction in 38.1% (8/21) of patients, median progression-free survival of 4.6 months, and induced T-cell activation in peripheral blood samples in 19 of 21 patients (PMID: 32439700, NCT03662815). | 32439700 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | LY2780301 | Phase I | Actionable | In a Phase I trial, LY2780301 demonstrated safety and preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 25902900). | 25902900 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MM-151 | Phase I | Actionable | In a Phase I trial, MM-151 displayed safety in preliminary efficacy in a variety of advanced solid tumors (J Clin Oncol 33, 2015 (suppl 3; abstr 647)). | detail... | |
| Unknown unknown | breast cancer | not applicable | CPI-455 + Lapatinib | Preclinical - Cell culture | Actionable | In a preclinical study, a breast cancer cell line treated with Tykerb (lapatinib) demonstrated increased sensitivity when co-treated with CPI-455 in culture, resulting in decreased survival of cells, in particular the cells that eventually develop drug resistance (PMID: 27214401). | 27214401 | |
| 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 | 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 | glioblastoma | no benefit | Buparlisib | Preclinical | Actionable | In a preclinical study, treatment with BKM120 demonstrated a survival similar to vehicle in transgenic mouse models of glioblastoma and therefore, resulted in no benefit (PMID: 27199435). | 27199435 | |
| Unknown unknown | brain glioma | not applicable | Cabozantinib | Preclinical | Actionable | In a preclinical study, Cometriq (cabozantinib) decreased cell proliferation and induced apoptosis in mouse models of glioma (PMID: 21926191). | 21926191 | |
| Unknown unknown | colon adenocarcinoma | not applicable | Onatasertib | Phase I | Actionable | In a Phase I trial, CC-223 demonstrated safety and some efficacy in patients with solid tumors, including colon cancer (J Clin Oncol 31, 2013 (suppl; abstr 2606). | 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 | PU-H71 | Phase I | Actionable | In a Phase I trial, treatment with PU-H71 was generally well-tolerated, and resulted in stable disease as best response in 35% (6/14) patients with advanced solid tumors (PMID: 28808818; NCT01581541). | 28808818 | |
| 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 | 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 | chronic lymphocytic leukemia | not applicable | Acalabrutinib | FDA approved | Actionable | In a Phase III trial (ELEVATE TN) that supported FDA approval, Calquence (acalabrutinib) treatment resulted in prolonged progression-free survival compared to Gazyva (obinutuzumab) plus chlorambucil (HR=0.20, p<0.0001) in patients with treatment-naive chronic lymphocytic leukemia (PMID: 31724010; NCT02475681). | 31724010 detail... | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Acalabrutinib | Phase Ib/II | Actionable | In a Phase Ib/II clinical trial, Calquence (acalabrutinib) treatment in chronic lymphocytic leukemia patients, including patients carrying chromosome 17p13.1 deletion, resulted in a 95% (57/60) overall response rate, which consisted of 85% with partial response and 5% (3/60) with stable disease (PMID: 26641137). | 26641137 | |
| Unknown unknown | cervical cancer | not applicable | Mps-BAY2b + Paclitaxel | Preclinical | Actionable | In a preclinical study, Mps-BAY2b, in combination with paclitaxel, had increased efficacy in decreasing tumor volume of cervical carcinoma xenografts (PMID: 23933817). | 23933817 | |
| Unknown unknown | renal cell carcinoma | not applicable | Bevacizumab + Temsirolimus | Phase II | Actionable | In a Phase II trial, patients with metastatic renal cell carcinoma (mRCC) treated with a combination of Avastin (bevacizumab) and Torisel (temsirolimus) demonstrated an overall 4 month PFS rate of 65% while those patients with VEGFR tyrosine kinase inhibitor refractory mRCC showed a median PFS and OS of 6.5 and 9.6 months, respectively (PMID: 27036973). | 27036973 | |
| Unknown unknown | colon cancer | not applicable | Etoposide + NU7441 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, NU7441 increased the sensitivity of colon cancer cell lines to Vepesid (etoposide), resulting in decreased cell survival in culture and reduced tumor growth in xenograft models (PMID: 16707462). | 16707462 | |
| Unknown unknown | glioblastoma | not applicable | Everolimus + Vorolanib | Phase I | Actionable | In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in stable disease in a patient with glioblastoma (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | BAY2402234 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, BAY2402234 treatment induced differentiation, cell cycle arrest and apoptosis, and inhibited proliferation of acute myeloid leukemia cell lines in culture, and induced differentiation, reduced tumor burden and increased survival in cell line and patient-derived xenograft (PDX) models (PMID: 30940908). | 30940908 | |
| 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 | 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 | 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 | 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 | 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 | Advanced Solid Tumor | not applicable | Selinexor | Phase I | Actionable | In a Phase I trial, Selinexor (KPT-330) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, resulting in an objective response rate of 4% (7/157; 1 complete response and 6 partial responses) and stable disease in 43% (67/157), with 17% (27/157) of patients demonstrating stable disease for at least 4 months (PMID: 26926685). | 26926685 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Selinexor | FDA approved | Actionable | In a Phase II study (SADAL) that supported FDA approval, Xpovio (selinexor) treatment resulted in an objective response rate of 27.6% (35/127, 14 complete responses, 21 partial responses) in patients with relapsed or refractory diffuse large B-cell lymphoma who have received 2 or more prior therapies, with a median duration of response of 8.4 months, a median progression-free survival of 3.6 months, and a median overall survival of 9.1 months (Hematol Oncol (June 2019) 37 (S2): 62-64; NCT02227251). | 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 | chronic lymphocytic leukemia | not applicable | Duvelisib | Phase I | Actionable | In a Phase I trial, Copiktra (duvelisib) demonstrated safety and efficacy in patients with advanced chronic lymphocytic leukemia (PMID: 24501284). | 24501284 | |
| Unknown unknown | sarcoma | not applicable | Alisertib | Phase II | Actionable | In a Phase II trial, Alisertib (MLN8237) treatment resulted in twelve-week progression free survival in 36% (4/11) of undifferentiated sarcoma patients (PMID: 27502708). | 27502708 | |
| Unknown unknown | cervical cancer | not applicable | Tozasertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Tozasertib (VX-680) inhibited growth of cervical cancer cell lines in culture, and inhibited tumor growth in cervical cancer cell line xenograft models (PMID: 27082306). | 27082306 | |
| 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 | fibrous histiocytoma | not applicable | Pazopanib | Clinical Study | Actionable | In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 15.3 weeks and median survival of 9.5 months in patients with undifferentiated pleomorphic sarcoma/malignant fibrous histiocytoma (PMID: 26970174). | 26970174 | |
| Unknown unknown | breast cancer | not applicable | Gedatolisib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Gedatolisib (PF-05212384) suppressed phosphorylation of PI3K/mTOR effectors and induced apoptosis in human breast cancer cell lines with elevated PI3K/mTOR signaling in culture and in cell line xenograft models (PMID: 21325073). | 21325073 | |
| Unknown unknown | breast cancer | not applicable | ATI-450 | Preclinical | Actionable | In a preclinical study, ATI-450 did not inhibit growth of a mouse breast cancer cell line in culture, however, reduced metastatic tumor growth in bone and visceral organs and enhanced survival in a mouse breast cancer cell line metastasis model (PMID: 30093561). | 30093561 | |
| Unknown unknown | breast cancer | not applicable | AKI603 + Epirubicin | Preclinical | Actionable | In a preclinical study, AKI603 synergistically increased the cytotoxic effects of Ellence (epirubicin) in breast cancer cells, resulting in a greater decreased cell survival when compared to either agent alone (PMID: 24899685). | 24899685 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CBP501 + Cisplatin + Nivolumab | Phase I | Actionable | In a Phase Ib trial, CBP501, Platinol (cisplatin), and Opdivo (nivolumab) triple combination demonstrated safety and preliminary efficacy, resulted in partial response in 11.8% (2/17) of patients with advanced refractory solid tumors, with a disease control rate (complete response+partial response+stable disease over 3 months) of 35% (6/17) (AACR Annual Meeting 2019, Abstract CT228; NCT03113188). | detail... | |
| Unknown unknown | female reproductive organ cancer | not applicable | Durvalumab + Olaparib | Phase I | Actionable | In a Phase I trial, the combination therapy of Imfinzi (durvalumab) and Lynparza (olaparib) resulted in an overall response rate of 17% (2/12) and disease control rate of 83% (10/12) in patients with female reproductive organ cancer (PMID: 28471727). | 28471727 | |
| Unknown unknown | prostate cancer | no benefit | Abiraterone + Buparlisib | Phase I | Actionable | In a Phase Ib trial, the combination of Zytiga (abiraterone) and Buparlisib (BKM120) did not demonstrate significant clinical activity in patients with castration-resistant prostate cancer, resulting in a best overall response of stable disease in 15% (3/20) patients treated at the 100 mg QD Buparlisib (BKM120) dose level, and further study of this combination is not planned (PMID: 28282611; NCT01634061). | 28282611 | |
| 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 | 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 | lung non-squamous non-small cell carcinoma | not applicable | Carboplatin + Demcizumab + Pemetrexed Disodium | Phase Ib/II | Actionable | In a Phase Ib trial, Demcizumab (OMP-21M18) in combination with Paraplatin (carboplatin) and Alimta (pemetrexed) demonstrated safety and preliminary efficacy, resulted in objective tumor response in 50% (20/40) of treatment-naive patients with metastatic non-squamous non-small cell lung cancer (PMID: 29188408; NCT01189968). | 29188408 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | BPM 31510 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TNBC cell lines demonstrated decreased cell viability and increased apoptotic activity in culture when treated with BPM 31510, and in cell line xenograft models, treatment resulted in reduced tumor size (Cancer Res 2016;76(14 Suppl):Abstract nr 208). | 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 | peritoneum cancer | not applicable | Lurbinectedin | Phase II | Actionable | In a Phase II trial, Lurbinectedin (PM01183) treatment in patients with either ovarian, peritoneal, or fallopian tube cancer resulted in an overall response rate of 23% (12/52), which included one complete response and eleven partial responses, and a median duration of response of 4.6 months and 23% (12/52) of responses lasted for 6 months or more (PMID: 28368437). | 28368437 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ipafricept | Phase I | Actionable | In a Phase I trial, Ipafricept (OMP-54F28) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 32:5s, 2014 (suppl; abstr 2505)). | detail... | |
| Unknown unknown | ocular melanoma | not applicable | CX-2029 | Case Reports/Case Series | Actionable | In a Phase I/II trial (PROCLAIM-CX-2029), CX-2029 treatment resulted in stable in a patient with ocular melanoma who stayed on treatment for 36 weeks (J Clin Oncol 38: 2020 (suppl; abstr 3502); NCT03543813). | detail... | |
| Unknown unknown | lung small cell carcinoma | not applicable | AMG 119 | Preclinical | Actionable | In a preclinical study, AMG 119 induced ablation of DLL3-positive small cell lung carcinoma cells in culture, and led to antitumor activity in a mouse model (J of Thoracic Oncology. 2018, 13(Supplement), S971. P3.12-03). | detail... | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Tirapazamine | Preclinical | Actionable | In a preclinical study, Tirazone (tirapazamine) inhibited growth of triple-receptor negative breast cancer cell lines under hypoxic culture conditions, regardless of their BRCA1 status (PMID: 25193512). | 25193512 | |
| Unknown unknown | multiple myeloma | not applicable | Dinaciclib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Dinaciclib (SCH 727965) decreased cell viability and growth of multiple myeloma cell lines in culture, and decreased tumor growth in multiple myeloma cell line xenograft models (PMID: 26719576). | 26719576 | |
| Unknown unknown | acute myeloid leukemia | not applicable | AZD1897 + Capivasertib | Preclinical | Actionable | In a preclinical study, acute myeloid leukemia cells treated with the combination of AZD1897 and AZD5363 produced a synergistic effect, resulting in decreased cell survival (PMID: 24975213). | 24975213 | |
| Unknown unknown | ovarian cancer | no benefit | Carboplatin + Ipafricept + Paclitaxel | Phase Ib/II | Actionable | In a Phase Ib trial, Ipafricept (OMP-54F28) in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) was well tolerated but demonstrated bone toxicity in platinum-sensitive ovarian cancer patients, and efficacy was similar to historical data of standard of care with an overall response rate of 75.7% (28/37), a clinical benefit rate of 94.6% (35/37), a median progression-free survial of 10.3 months and an overall survival of 33 months (PMID: 31174889; NCT02092363). | 31174889 | |
| Unknown unknown | T-cell acute lymphoblastic leukemia | not applicable | DT2216 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, DT2216 treatment resulted in decreased BCL-XL expression and increased apoptosis and reduced viability of a T-cell acute lymphoblastic leukemia (T-ALL) cell line in culture, and decreased tumor growth in xenograft models (PMID: 31792461). | 31792461 | |
| Unknown unknown | glioblastoma | not applicable | Adavosertib | Phase 0 | Actionable | In a Phase 0 trial, Adavosertib (MK-1775) demonstrated safety and preliminary efficacy, resulted in good brain tumor penetration and Wee1 pathway inhibition in intraoperatively collected tumor samples from patients with first-recurrence glioblastoma (PMID: 29798906). | 29798906 | |
| Unknown unknown | lung squamous cell carcinoma | not applicable | CX-2029 | Case Reports/Case Series | Actionable | In a Phase I/II trial (PROCLAIM-CX-2029), CX-2029 treatment resulted in partial response in a patient with squamous cell carcinoma of the lung (J Clin Oncol 38: 2020 (suppl; abstr 3502); NCT03543813). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | JQ1 + Rucaparib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of JQ1 and Rubraca (rucaparib) resulted in a synergistic effect, demonstrating greater growth inhibition of ovarian cancer cells in culture compared to JQ1 treatment alone (PMID: 32927276). | 32927276 | |
| Unknown unknown | Sezary's disease | not applicable | Mogamulizumab | FDA approved | Actionable | In a Phase III trial (MAVORIC) that supported FDA approval, Poteligeo (mogamulizumab-kpkc) treatment resulted in significantly improved progression-free survival (7.7 vs 3.1 months, HR=0.53, p<0.0001) and objective response rate (28.0% vs 4.8%) compared to Zolinza (vorinostat) in patients with mycosis fungoides or Sézary syndrome (Blood 2017 130(Suppl 1):817). | detail... detail... | |
| Unknown unknown | Ewing sarcoma | not applicable | Niraparib + Temozolomide | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with Temodar (temozolomide) combined with Zejula (niraparib) demonstrated strong synergism in Ewing sarcoma cells in culture, resulting in decreased cell viability (PMID: 26438158). | 26438158 | |
| Unknown unknown | prostate cancer | not applicable | SPC3042 | Preclinical | Actionable | In a preclinical study, SPC3042 decreased Survivin expression and induced apoptosis of prostate cancer cells in culture (PMID: 18790754). | 18790754 | |
| Unknown unknown | breast 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 breast 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 | Binimetinib | Phase I | Actionable | In a Phase I trial, Binimetinib (MEK162) treatment resulted in complete response in 1% (1/91), partial response in 2% (2/91), and stable disease with median duration of 3.94 months in 36% (33/91) of patients with advanced solid tumors (PMID: 28152546). | 28152546 | |
| Unknown unknown | multiple myeloma | not applicable | JNJ-64007957 | Phase I | Actionable | In a Phase I trial, Teclistamab (JNJ-64007957) treatment demonstrated safety and preliminary efficacy, resulted in an overall response rate (ORR) of 38% (20/52) in patients with advanced relapsed or refractory multiple myeloma, with an ORR of 78% (7/9) in the highest dose group (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 100-100; NCT03145181). | detail... | |
| Unknown unknown | prostate cancer | not applicable | Danusertib | Phase II | Actionable | In a Phase II clinical trial, Danusertib (PHA-739358) monotherapy was well tolerated, but showed minimal efficacy in patients with castration-resistant prostate cancer (PMID: 22928785). | 22928785 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | RXDX-105 | Phase I | Actionable | In a Phase I clinical trial, RXDX-105 (CEP-32496) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, including 2 patients with heavily pretreated non-small cell lung cancer that achieved stable disease for greater that 6 months (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr C188). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Omaveloxolone | Preclinical - Cell culture | Actionable | In a preclinical study, Omaveloxolone (RTA 408) induced apoptosis and inhibited growth of several human tumor cell lines in culture (PMID: 25897966). | 25897966 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Omaveloxolone | Phase I | Actionable | In a Phase I trial, treatment with Omaveloxolone (RTA 408) in patients with an advanced solid tumor led to a median progression-free survival of 1.5 months, an overall survival of 5.8 months, and one patient with lung cancer experienced stable disease for greater than 12 months (PMID: 28919776). | 28919776 | |
| 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 | Advanced Solid Tumor | not applicable | Trebananib | Phase I | Actionable | In a Phase I trial, treatment with Trebananib demonstrated tolerability in pediatric patients with advanced solid tumors, and resulted in stable disease for greater than 4 months in one patient with neuroblastoma and one patient with anaplastic astrocytoma (PMID: 28751444). | 28751444 | |
| Unknown unknown | peripheral T-cell lymphoma | not applicable | Tenalisib | Phase I | Actionable | In a Phase I trial, Tenalisib (RP6530) demonstrated safety in patients with relapsed or refractory peripheral T-cell lymphoma, and resulted in an overall response rate of 46.7% (7/15), including three complete responses and four partial responses with a median duration of response of 6.5 months, and a further two patients achieved stable disease (PMID: 32824175; NCT02567656). | 32824175 | |
| Unknown unknown | mycosis fungoides | not applicable | Lacutamab | Phase I | Actionable | In a Phase I trial, Lacutamab (IPH4102) demonstrated safety, and treatment resulted in a median progression-free survival of 3.9 months, and a response in 1 and stable disease in 7 of 8 patients with mycosis fungoides (PMID: 31253572; NCT02593045). | 31253572 | |
| 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 | lung small cell carcinoma | not applicable | STA-8666 | Preclinical - Pdx | Actionable | In a preclinical study, small cell lung cancer cell line xenograft and patient derived xenograft (PDX) models demonstrated stabilization of tumor growth and eventual tumor regression when treated with STA-8666 (PMID: 27267850). | 27267850 | |
| Unknown unknown | brain stem glioma | no benefit | Veliparib | Preclinical - Cell culture | Actionable | In a preclinical study, Veliparib (ABT-888) had a very limited effect on the cell viability of multiple cultured pediatric diffuse intrinsic pontine glioma cell lines (PMID: 26351319). | 26351319 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CYT01B + Veliparib | Preclinical - Cell culture | Actionable | In a preclinical study, CYT01B and Veliparib (ABT-888) synergistically inhibited growth of tumor cell lines in culture (AACR Annual Meeting 2019, Abstract 363). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | Brequinar | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, Brequinar treatment induced differentiation of acute myeloid leukemia cell lines in culture, reduced colony formation of tumor cells, decreased leukemic burden and leukemia initiating cells, and increased survival in syngeneic mouse models, and led to induced differentiation of tumor cells, inhibition of tumor growth, and increased survival in cell line xenograft models and a patient-derived xenograft (PDX) model (PMID: 27641501). | 27641501 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Fluorouracil + Sorafenib | Phase I | Actionable | In a Phase I trial, Nexavar (sorafenib) in combination with Adrucil (fluorouracil) displayed safety and efficacy in advanced solid tumors, including colon cancer (PMID: 22232731). | 22232731 | |
| Unknown unknown | pancreatic 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 pancreatic cancer cell line xenograft model (Cancer Res 2019;79(13 Suppl):Abstract nr 4815). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | Durvalumab + Olaparib | Phase II | Actionable | In a Phase II trial, combination treatment with Imfinzi (durvalumab) and Lynparza (olaparib) did not meet the primary endpoint, but led to tumor immunomodulatory effects in patients with recurrent ovarian cancer and resulted in an overall response rate of 14% (5/35, all partial responses), a median progression-free survival of 3.9 months, and a disease control rate of 71% (25/35), including 12 patients that maintained a partial response or stable disease for more than 6 months (PMID: 32398324; NCT02484404). | 32398324 | |
| 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 | Advanced Solid Tumor | not applicable | MK2206 | Phase I | Actionable | In a Phase I clinical trial, MK2206 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22025163). | 22025163 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Cisplatin + SR-4835 | Preclinical - Pdx | Actionable | In a preclinical study, SR-4835 and Platinol (cisplatin) combination treatment induced apoptosis, inhibited proliferation and tumor growth, leading to tumor regression in an orthotopic patient-derived xenograft (PDX) model of triple-negative breast cancer (PMID: 31668947). | 31668947 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Axitinib | Phase II | Actionable | In a Phase II clinical trial, Inlyta (axitinib) was well-tolerated and demonstrated activity in patients with advanced non-small cell lung cancer, with a disease control rate of 41% (13/32), median progression-free survival of 4.9 months, and median overall survival of 14.8 months (PMID: 19597027). | 19597027 | |
| Unknown unknown | breast cancer | not applicable | OSI-027 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, OSI-027 inhibited mTORC1 and mTORC2 signaling and growth in breast cancer cells in culture and in cell line xenograft models (PMID: 21673091). | 21673091 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | SF1126 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, SF1126 inhibited proliferation of hepatocellular carcinoma cell lines in culture, and inhibited tumor growth in hepatocellular carcinoma cell line xenograft models (PMID: 27496136). | 27496136 | |
| Unknown unknown | sarcoma | 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 2.8 months in soft-tissue sarcoma patients, compared to 1.4 months with placebo, and FGF2 expression was not found to be a predictive biomarker for response (PMID: 31522033; NCT00633789). | 31522033 | |
| 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 | 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 | head and neck 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 of a head and neck cancer cell line in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | colorectal cancer | not applicable | Napabucasin | Phase I | Actionable | In a Phase I trial, colorectal cancer patients treated with BBI608 demonstrated a median PFS of 14 weeks and OS of 47 months while 67% (8/12) experienced stable disease (J Clin Oncol May 2013 vol. 31 no. 15_suppl 2542). | detail... | |
| Unknown unknown | brain stem glioma | not applicable | Olaparib | Preclinical - Cell culture | Actionable | In a preclinical study, Lynparza (olaparib) resulted in decreased cell viability of some diffuse intrinsic pontine glioma cell lines in culture (PMID: 26351319). | 26351319 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | LY3127804 | Phase I | Actionable | In a Phase I trial, treatment with LY3127804 in advanced solid tumor patients demonstrated safety and was well-tolerated, and resulted in stable disease in 55% (11/20) of patients (PMID: 32741971). | 32741971 | |
| Unknown unknown | lung adenocarcinoma | not applicable | JNJ-64041757 | Phase I | Actionable | In a Phase I trial, JNJ-64041757 treatment demonstrated safety in patients with lung adenocarcinoma, and resulted in mesothelin-specific immune responses in several patients and stable disease as best response (J Thoracic Oncol, Vol 12, Issue 11, S2151). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | RO4987655 | Phase I | Actionable | In a Phase I trial, RO4987655 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22767668). | 22767668 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | RO4987655 | Phase I | Actionable | In a Phase I trial, RO4987655 demonstrated safety and preliminary clinical activity in Japanese patients with advanced solid tumors, including 1 partial response in a patient with esophageal cancer and 7 patients achieving progression-free survival for greater than 16 weeks (PMID: 25809858). | 25809858 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CV301 | Phase I | Actionable | In a Phase I trial, CV301 treatment was well-tolerated and resulted in a median progression-free survival of 15 weeks in advanced solid tumor patients (n=12) (PMID: 31110074; NCT02840994). | 31110074 | |
| Unknown unknown | leiomyosarcoma | not applicable | Alisertib | Phase II | Actionable | In a Phase II trial, Alisertib (MLN8237) treatment resulted in twelve-week progression free survival in 44% (4/9) of leiomyosarcoma patients (PMID: 27502708). | 27502708 | |
| 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 | Neratinib + Temsirolimus | Phase I | Actionable | In a Phase I study, Nerlynx (neratinib) administered with Torisel (temsirolimus) was tolerable and demonstrated antitumor activity in multiple solid tumor types, including breast cancer and NSCLC (PMID: 24323026). | 24323026 | |
| Unknown unknown | non-Hodgkin lymphoma | not applicable | Camidanlumab Tesirine | Phase I | Actionable | In a Phase I trial, Camidanlumab Tesirine treatment resulted in partial response in 15.8% (3/19) in non-Hodgkin lymphoma patients (Blood 2017 130(Suppl 1):1510; NCT02432235). | detail... | |
| 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 | Advanced Solid Tumor | not applicable | Derazantinib | Phase I | Actionable | In a Phase I trial, Derazantinib (ARQ 087) treatment resulted in partial response in 4.5% (3/67) and stable disease in 38.8% (26/67) of patients with advanced solid tumors (PMID: 28972963; NCT01752920). | 28972963 | |
| Unknown unknown | acute myeloid leukemia | 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, cell-cycle arrest, and induced apoptosis in culture (PMID: 29626127). | 29626127 | |
| Unknown unknown | cervical cancer | not applicable | Cisplatin + ETP-46464 | Preclinical - Cell culture | Actionable | In a preclinical study, ETP-46464 increased the sensitivity of cervical cancer cell lines to Platinol (cisplatin) in culture (PMID: 25560806). | 25560806 | |
| Unknown unknown | colon adenocarcinoma | not applicable | unspecified CTLA4 antibody + VE800 | Preclinical | Actionable | In a preclinical study, CTLA4 antibody treatment supplemented with VE800 inhibited tumor growth in a mouse model of colon adenocarcinoma (PMID: 30675064). | 30675064 | |
| Unknown unknown | prostate cancer | not applicable | X480 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with X480 in prostate cancer cell lines resulted in reduced cell proliferation and induced apoptotic activity in culture, and decreased tumor load and metastasis in cell line xenograft models (Eur J Cancer, 2012, 48, S5:235). | detail... | |
| Unknown unknown | glioblastoma | not applicable | ICT-107 | Phase II | Actionable | In a Phase II trial, ICT-107 treatment was well-tolerated, and resulted in an overall survival of 17 months vs. 15 months (HR=0.87, P=0.58), and progression-free survival of 11.2 months vs. 9 months (HR=0.57, P=0.011) compared to control in glioblastoma patients (PMID: 31320597; NCT01280552). | 31320597 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | BMS-906024 | Preclinical | Actionable | In a preclinical study, the pan-Notch inhibitor, BMS-906024, inhibited growth of non-small cell lung carcinoma xenografts (PMID: 26005526). | 26005526 | |
| 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 | lung squamous cell carcinoma | not applicable | Carboplatin + Paclitaxel + Tislelizumab | Phase II | Actionable | In a Phase II trial, treatment with Tislelizumab (BGB-A317) plus platinum doublet chemotherapy (Taxol (paclitaxel) with Platinol (cisplatin) or Paraplatin (carboplatin)) in patients with squamous non-small cell lung cancer resulted in an objective response rate of 80% (12/15) and disease control rate of 93% (14/15), including a partial response in 12 patients and stable disease in two patients, and median progression-free survival of 7.0 months (PMID: 32769013; NCT03432598). | 32769013 | |
| Unknown unknown | urinary bladder cancer | not applicable | OBP-801 | Preclinical - Cell culture | Actionable | In a preclinical study, OBP-801 treatment resulted in decreased cell viability in multiple human bladder cancer cell lines in culture (PMID: 27406983). | 27406983 | |
| 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 | 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 | chronic lymphocytic leukemia | not applicable | PRT062607 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with PRT062607 (P505-15) resulted in decreased viability of chronic lymphocytic leukemia cells in culture and in cell line xenograft models (PMID: 23220742). | 23220742 | |
| 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 | Advanced Solid Tumor | not applicable | SST0116CL1 | Preclinical - Cell culture | Actionable | In a preclinical study, SST0116CL1 inhibited proliferation of several human tumor cell lines in culture (PMID: 25096516). | 25096516 | |
| Unknown unknown | neuroblastoma | not applicable | JSH-150 | Preclinical - Cell culture | Actionable | In a preclinical study, JSH-150 inhibited proliferation of a neuroblastoma cell line in culture (PMID: 30253346). | 30253346 | |
| Unknown unknown | estrogen-receptor positive breast cancer | not applicable | Everolimus + Tamoxifen | Preclinical - Cell culture | Actionable | In a preclinical study, the combination treatment of Afinitor (everolimus) and Nolvadex (tamoxifen) resulted in decreased colony formation by 95% in estrogen-receptor (ER) positive breast cancer cell lines while in ER positive breast cancer cell lines resistant to Nolvadex (tamoxifen), colony formation formation decreased by 76% with the addition of Afinitor (everolimus) (PMID: 27421652). | 27421652 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Pegilodecakin + Pembrolizumab | Phase I | Actionable | In a Phase Ib trial (IVY), Pegilodecakin (AM0010) and Keytruda (pembrolizumab) (n=5) or Opdivo (nivolumab) (n=29) combination therapy demonstrated safety and preliminary efficacy, resulting in an objective response rate of 43% (12/28, 1 complete response, 11 partial responses) and a disease control rate of 82% (23/28) in evaluable patients with non-small cell lung carcinoma, with a median duration of response of 10.3 months (PMID: 31563517; NCT02009449). | 31563517 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Fluzoparib | Preclinical - Cell culture | Actionable | In a preclinical study, Fluzoparib inhibited DNA-damage-induced PARylation in breast cancer cell lines in culture, however, the cells demonstrated resistance to Fluzoparib treatment (PMID: 30949414). | 30949414 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Evofosfamide | Preclinical | Actionable | In a preclinical study, TH-302 inhibited growth of triple-receptor negative breast cancer cell lines under hypoxic conditions, regardless of their BRCA1 status (PMID: 25193512). | 25193512 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Pembrolizumab + Utomilumab | Phase Ib/II | Actionable | In a Phase Ib trial, treatment with the combination of Keytruda (pembrolizumab) and Utomilumab (PF-05082566) resulted in an objective response rate of 26% (6/23) in patients with advanced solid tumors, which included two patients achieving a complete response and four patients demonstrating a partial response (PMID: 28634283). | 28634283 | |
| 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 | stomach cancer | not applicable | Chiauranib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a gastric cancer cell line xenograft model (PMID: 28004478). | 28004478 | |
| 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 | prostate adenocarcinoma | not applicable | ONC201 | Clinical Study | Actionable | In a clinical case study, a prostate adenocarcinoma patient demonstrated extended stable disease for 27 weeks when treated with ONC201 (TIC-10) (PMID: 28331050). | 28331050 | |
| Unknown unknown | nasopharynx carcinoma | not applicable | Axitinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Inlyta (axitinib) inhibited growth of nasopharyngeal carcinoma cell lines in culture, and inhibited tumor growth, decreased microvessel density, and increased tumor necrosis in a nasopharyngeal carcinoma xenograft model (PMID: 29301831). | 29301831 | |
| Unknown unknown | nasopharynx carcinoma | not applicable | Axitinib | Clinical Study | Actionable | In a clinical trial, treatment with Inlyta (axitinib) resulted in a 3-month clinical benefit rate (CBR) of 78.4% (29/37; 1 confirmed partial response (PR), 6 unconfirmed PR, 22 stable disease for greater than 3 months) and 6-month CBR of 43.2%, a median progression-free survival of 5.0 months, and median overall survival of 10.4 months in patients with nasopharyngeal carcinoma (PMID: 29301831). | 29301831 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CLR457 | Phase I | Actionable | In a Phase I trial, CLR457 treatment demonstrated limited efficacy, resulting in no confirmed responses, stable disease in 25.8% (8/31) , and non-complete response/non-progressive disease in 6.5% (2/31) of patients with advanced solid tumors with PI3K pathway activation (PMID: 30073466; NCT02189174). | 30073466 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CLR457 | Preclinical - Pdx | Actionable | In a preclinical study, CLR457 treatment of a variety of solid tumor patient-derived xenograft models decreased tumor volume and produced a 54% response (PMID: 26479923). | 26479923 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Temsirolimus | Phase I | Actionable | In a Phase I trial, Torisel (temsirolimus) in combination with radiation therapy demonstrated safety and efficacy in 5/8 NSCLC patients (PMID: 24373609). | 24373609 | |
| Unknown unknown | pancreatic carcinoma | not applicable | Tepotinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Tepmetko (tepotinib) induced tumor regression in mouse cell line xenograft models of pancreatic carcinoma (PMID: 23553846). | 23553846 | |
| 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 | 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 | acute myeloid leukemia | no benefit | Decitabine + Talacotuzumab | Phase II | Actionable | In a phase II trial, Talacotuzumab (JNJ-56022473) and Dacogen (decitabine) combination therapy did not improve complete response rate (15%, 12/80 vs 11%, 9/82, OR=1.4, p=0.44) or median overall survival (5.36 vs 7.26 months, HR=1.04, p=0.78) compared to Dacogen (decitabine) alone in elderly patients with acute myeloid leukemia (PMID: 32203138; NCT02472145). | 32203138 | |
| Unknown unknown | oral squamous cell carcinoma | not applicable | Cisplatin + Olaparib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment of oral squamous carcinoma cells with Platinol (cisplatin) and Lynparza (olaparib) resulted in a synergistic effect demonstrating increased apoptosis and tumor growth inhibition in culture and cell line xenograft models (PMID: 26927065). | 26927065 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Entospletinib + Idelalisib | Phase II | Actionable | In a Phase II trial, Zydelig (idelalisib) and Entospletinib combination treatment resulted in objective response in 60% of patients with chronic lymphocytic leukemia, but was terminated due to severe treatment-emergent pneumonitis (PMID: 26968534). | 26968534 | |
| Unknown unknown | prostate 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 castration-resistant prostate cancer (7/14) or advanced breast cancer (7/14) (PMID: 32878811; NCT01610050). | 32878811 | |
| Unknown unknown | chronic myeloid leukemia | not applicable | AG-14361 + Camptothecin | Preclinical - Cell culture | Actionable | In a preclinical study, the addition of AG-14361 to Camptothecin increased sensitivity 2-fold in chronic myeloid leukemia cells in culture, demonstrating growth inhibition and cell death (PMID: 16322308). | 16322308 | |
| Unknown unknown | ovarian carcinoma | no benefit | Denileukin diftitox + Sirolimus | Preclinical | Actionable | In a preclinical study, an ovarian carcinoma mouse model did not respond to the combination of Ontak (denileukin diftitox) and Rapamune (sirolimus), demonstrating no decrease in tumor burden (PMID: 27737881). | 27737881 | |
| 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 | pancreatic cancer | not applicable | Gemcitabine + MVT-5873 + Nab-paclitaxel | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MVT-5873 enhanced efficacy of the combination therapy, Gemzar (gemcitabine) and Abraxane (nab-paclitaxel), in pancreatic xenograft models, demonstrating increased inhibition of tumor growth and greater reduction of tumor volume when compared to single agents (Cancer Res 2016;76(24 Suppl):Abstract nr A73). | detail... | |
| 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 | Advanced Solid Tumor | not applicable | ADG116 | Preclinical | Actionable | In a preclinical study, ADG116 demonstrated anti-tumor activity and increased overall survival in syngeneic mouse models of a variety of solid tumors (PMID: 31694708). | 31694708 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Alisertib + Paclitaxel | Preclinical - Pdx | Actionable | In a preclinical study, the combination of Alisertib (MLN8237) and Taxol (paclitaxel) worked synergistically or additively to inhibit tumor growth in cell line and patient-derived xenograft (PDX) models of triple-negative breast cancer (PMID: 24980948). | 24980948 | |
| 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 | triple-receptor negative breast cancer | not applicable | Capivasertib + Paclitaxel | Phase II | Actionable | In a Phase II trial (PAKT), addition of Capivasertib (AZD5363) to Taxol (paclitaxel) as first-line therapy resulted in improved median progression-free survival (5.9 vs 4.2 months, HR=0.74, p=0.06) and median overall survival (19.1 vs 12.6 months, HR=0.61, p=0.04) compared to Taxol (paclitaxel) alone in patients with metastatic triple-negative breast cancer (PMID: 31841354; NCT02423603). | 31841354 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ulixertinib | Phase I | Actionable | In a Phase I trial, BVD-523 (Ulixertinib) displayed safety and preliminary efficacy in advanced solid tumor patients (J Clin Oncol 33, 2015 (suppl; abstr 2506)). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | MLN0905 | Preclinical | Actionable | In a preclinical study, MLN0905 treatment resulted in decreased tumor volume in a diffuse large B-cell lymphoma xenograft model (PMID: 22609854). | 22609854 | |
| Unknown unknown | follicular lymphoma | not applicable | Urelumab | Phase I | Actionable | In a Phase I trial, Urelumab (BMS-663513) demonstrated manageable safety profile, resulted in an objective response rate of 12% (2/17) and a disease control rate of 35% (6/17) in patients with relapsed or refractory follicular lymphoma, with a median progression-free survival of 8.9 weeks and a median overall survival not reached (PMID: 32052473; NCT01471210). | 32052473 | |
| Unknown unknown | osteosarcoma | not applicable | DSP-0509 | Preclinical | Actionable | In a preclinical study, DSP-0509 inhibited tumor growth and reduced metastatic lung nodules in a syngeneic mouse model of osteosarcoma (Cancer Res July 1 2018 (78) (13 Supplement) 4726). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Urelumab | Phase I | Actionable | In a Phase I trial, Urelumab (BMS-663513) demonstrated manageable safety profile, resulted in an objective response rate of 6% (2/31) and a disease control rate of 19% (6/31) in patients with relapsed or refractory diffuse large B-cell lymphoma, with a median progression-free survival of 8.1 weeks and a median overall survival of 45.6 weeks (PMID: 32052473; NCT01471210). | 32052473 | |
| 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 | 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 | multiple myeloma | not applicable | Daratumumab and hyaluronidase-fihj | FDA approved | Actionable | In a Phase III trial (COLUMBA) that supported FDA approval, subcutaneous administration of Darzalex Faspro (Daratumumab and hyaluronidase-fihj) demonstrated improved safety profile and resulted in an overall response rate comparable to that of intravenous Darzalex (daratumumab) (41%, 108/263, vs 37%, 96/259, relative risk 1.11) in patients with relapsed or refractory multiple myeloma who received 3 or more prior therapies (PMID: 32213342; NCT03277105). | 32213342 | |
| Unknown unknown | multiple myeloma | not applicable | Bortezomib + SJB3-019A | Preclinical - Patient cell culture | Actionable | In a preclinical study, the combination of SJB3-019A and Velcade (bortezomib) worked synergistically to induce cytotoxicity in multiple myeloma cell lines and primary multiple myeloma cells in culture (PMID: 28270494). | 28270494 | |
| Unknown unknown | breast cancer | not applicable | H3B-6545 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a breast cancer cell line xenograft model was sensitive to treatment with H3B-6545, demonstrating greater antitumor activity compared to Faslodex (fulvestrant) (Cancer Res 2017;77(13 Suppl):Abstract nr DDT01-04). | detail... | |
| 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 | alveolar soft part sarcoma | not applicable | Durvalumab | Clinical Study | Actionable | In a clinical case study, a patient with alveolar soft part sarcoma had a 58% reduction of target lesions and a response lasting 12 months when treated with Imfinzi (durvalumab), and the tumor was shown retrospectively to have a mismatch repair defect signature, poor immune infiltration, and 0% tumoral PD-L1 positivity (PMID: 30018044; NCT01693562). | 30018044 | |
| Unknown unknown | lung non-squamous non-small cell carcinoma | not applicable | Atezolizumab + Bevacizumab + Carboplatin + Paclitaxel | Phase III | Actionable | In a Phase III trial (IMpower150), addition of Tecentriq (atezolizumab) to the standard-of-care bevacizumab, paclitaxel, plus carboplatin improved overall survival (OS) in patients with metastatic non-squamous non-small cell lung cancer, improved OS was observed in the intent-to-treat population (19.8 vs 14.9 mo, HR=0.76), patients harboring EGFR sensitizing mutations (not reached vs 17.5 mo, HR=0.31), and in patients with baseline liver metastases (13.3 vs 9.4 mo, HR=0.52) (PMID: 30922878; NCT02366143). | 30922878 | |
| Unknown unknown | Ewing sarcoma | not applicable | Olaparib + SN-38 + Temozolomide | Preclinical - Cell culture | Actionable | In a preclinical study, Ewing sarcoma cells treated with the combination of Temodar (temozolomide), SN-38, and Lynparza (olaparib) had a much greater effect on decreasing cell viability and inducing apoptosis when compared to each treatment administered as a single agent or as dual agents in culture (PMID: 26438158). | 26438158 | |
| 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 | 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 | Pazopanib + Trametinib | Phase I | Actionable | In a Phase I trial, combination treatment with Votrient (pazopanib) and Mekinist (trametinib) was tolerable and resulted in partial response in 12% (3/25), and stable disease in 72% (18/25) of patients with advanced solid tumors, and 9 patients remained on study for more than 6 cycles, including patients with differentiated thyroid cancer (n=3), colorectal cancer (n=2), melanoma (n=1), cholangiocarcinoma (n=1), ovarian cancer (n=1), and synovial cell sarcoma (n=1) (PMID: 31186313; NCT01438554). | 31186313 | |
| Unknown unknown | biliary tract cancer | not applicable | Pembrolizumab | Phase II | Actionable | In a Phase II trial (KEYNOTE-158), Keytruda (pembrolizumab) demonstrated efficacy in patients with advanced biliary tract cancer regardless of CD274 (PD-L1) status, resulted in partial response in 5.8% (6/104) and stable disease in 16% (17/104) of patients; objective response rate, median progression-free survival, and median overall survival were 6.6%, 1.9, and 7.2 months in patients with CPS?1 (n?=?61), and 2.9%, 2.1, and 9.6 months in patients with CPS<1 (n?=?34) (Ann Oncol 29(suppl_8); NCT02628067). | detail... | |
| Unknown unknown | uveal melanoma | no benefit | Ipilimumab + Pembrolizumab | Case Reports/Case Series | Actionable | In a clinical study, Yervoy (ipilimumab) and Keytruda (pembrolizumab) combination treatment resulted in an objective response rate of 0% (0/9) and a disease control rate of 56% (5/9) in patients with uveal melanoma, with a median overall survival of 18.4 months (PMID: 29988983). | 29988983 | |
| 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 | multiple myeloma | not applicable | Dexamethasone + Thalidomide | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Thalomid (thalidomide) in combination with dexamethasone (n=103) resulted in significantly improved response rate (63% vs 41%, p=0.0017) compared to dexamethasone alone (n=104) in patients with newly diagnosed multiple myeloma (PMID: 16365178). | 16365178 detail... | |
| 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 | epithelioid sarcoma | not applicable | Vorinostat | Preclinical | Actionable | In a preclinical study, Zolinza (vorinostat) inhibited growth and colony formation of epithelioid sarcoma cells in culture (PMID: 26396249). | 26396249 | |
| 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 | ovarian cancer | not applicable | RO5520985 | Phase I | Actionable | In a Phase I clinical trial, Vanucizumab (RO5520985) treatment resulted in partial response in 29% (12/41) and stable disease in 54% of (22/41) platinum (Pt)- resistant/refractory epithelial ovarian cancer patients (J Clin Oncol 33, 2015 (suppl; abstr 5549)). | detail... | |
| Unknown unknown | osteosarcoma | not applicable | AZD7762 + VE-821 | Preclinical | Actionable | In a preclinical study, osteosarcoma cells treated with VE-821 resulted in a synthetic lethal effect when combined with AZD7762, thereby demonstrating reduced cell survival in culture (PMID: 26748709). | 26748709 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Atezolizumab + CPI-444 | Phase I | Actionable | In a Phase I trial, treatment with the combination of CPI-444 and Tecentriq (atezolizumab) was well-tolerated and resulted in a disease control rate of 71% (5/7) in patients with non-small cell lung cancer (J Clin Oncol 35, 2017 (suppl; abstr 3004)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ASTX-660 | Phase I | Actionable | In a Phase I trial, ASTX660 treatment was well-tolerated, and resulted in no objective responses in heavily pretreated advanced solid tumor (n=43) and lymphoma (n=2) patients with stable disease as the best response in 29% (10/35) of evaluable patients, and a median progression-free survival of 55 days and median overall survival of 265 days in the entire cohort, and clinical improvement in a patient with cutaneous T-cell lymphoma (PMID: 31900279; NCT02503423). | 31900279 | |
| 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 | 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 | ovarian cancer | not applicable | FR alpha peptide vaccine | Phase I | Actionable | In a Phase I trial, FR alpha peptide vaccine demonstrated safety, induced durable immune response against Folr1 in ovarian cancer patients who completed standard treatment, and all patients (n=14) remained alive 2 years after initial immunization, with a median relapse-free survival of 528 days; however, T cell response did not correlate with tumor Folr1 expression level (PMID: 29545464; NCT01606241). | 29545464 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | KPT-9274 | Phase I | Actionable | In a Phase I trial, KPT-9274 treatment resulted in stable disease in 29% (4/14) of patients with advanced solid tumors (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 374PD; NCT02702492). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BTP-114 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, BTP-114 inhibited growth of tumor cells in culture and in xenograft animal models (Cancer Res 2015;75(15 Suppl):Abstract nr 4484). | detail... | |
| Unknown unknown | nasopharynx carcinoma | not applicable | Camrelizumab | Phase I | Actionable | In a Phase I trial, Camrelizumab (SHR-1210) treatment was well tolerated in patients with refractory nasopharyngeal carcinoma, and demonstrated preliminary efficacy with an overall response rate of 34% (31/91, 2 complete responses and 29 partial responses) and a disease control rate of 59% (54/91, stable disease or better) (PMID: 30213452; NCT02721589). | 30213452 | |
| Unknown unknown | nasopharynx 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 nasopharynx cancer (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). | detail... | |
| Unknown unknown | stomach cancer | not applicable | Doxorubicin + Tozasertib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Adriamycin (doxorubicin) and Tozasertib (VX-680) decreased Survivin expression and worked synergistically to reduce viability of a gastric cancer cell line in culture (PMID: 28218735). | 28218735 | |
| Unknown unknown | B-cell lymphoma | not applicable | Alisertib | Phase II | Actionable | In a Phase II trial, Alisertib (MLN8237) treatment resulted in significant toxicity and an objective response rate of 15% (2/13) in patients with B-cell non Hodgkin's lymphoma (PMID: 28278718). | 28278718 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Abemaciclib | Preclinical | Actionable | In a preclinical study, Abemaciclib (LY2835219) reduced RB and AKT activation, and inhibited cell proliferation and colony formation in head and neck squamous cell carcinoma (HNSCC) cell lines in culture, and inhibited tumor growth in HNSCC xenograft models (PMID: 26909611). | 26909611 | |
| 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 | 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 | breast cancer | not applicable | CVX-241 | Preclinical | Actionable | In a preclinical study, CVX-241 treatment resulted in improved overall survival in a breast cancer cell line xenograft model and syngeneic mouse model of breast cancer (PMID: 27651308). | 27651308 | |
| Unknown unknown | malignant peripheral nerve sheath tumor | not applicable | Carfilzomib + Selinexor | Preclinical - Cell line xenograft | 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 additively to decrease viability of malignant peripheral nerve sheath tumor (MPNST) cells in culture, and resulted in enhanced tumor growth inhibition in MPNST xenograft models (PMID: 28314790). | 28314790 | |
| Unknown unknown | ovarian clear cell adenocarcinoma | not applicable | DS-7423 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, DS-7423 inhibited proliferation of ovarian clear cell adenocarcinoma cell lines in culture and suppressed tumor growth in cell line xenograft animal models (PMID: 24504419). | 24504419 | |
| Unknown unknown | B-cell lymphoma | not applicable | Denileukin diftitox + Sirolimus | Preclinical - Cell culture | Actionable | In a preclinical study, Rapamune (sirolimus) 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 | 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 | melanoma | not applicable | G-TPP + Obatoclax | Preclinical - 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 melanoma cells in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | hepatocellular carcinoma | 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 10.7 months in patients with unresectable hepatocellular carcinoma (PMID: 19144678). | 19144678 detail... | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Sorafenib | Case Reports/Case Series | Actionable | In a Phase I trial, three hepatocellular carcinoma patients treated with Nexavar (sorafenib) following CEBPA-51 (MTL-CEBPA) treatment achieved complete radiological response, 1 with complete radiological response in the liver that was sustained at 9 months, 1 with complete radiological response in the liver and lung lesions that was sustained at 12 months, and 1 with complete radiologcial response in the liver and lungs that was sustained at 7 months (PMID: 32357963; NCT02716012). | 32357963 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Birabresib | Phase I | Actionable | In a Phase I trial, Birabresib (OTX015) treatment resulted in complete remission lasting 2-5 months in 8% (3/36) and partial blast clearance in 6% (2/36) of acute myeloid leukaemia patients (PMID: 27063977). | 27063977 | |
| Unknown unknown | lung carcinoma | not applicable | Triapine | Preclinical | Actionable | In a preclinical study, Triapine treatment delayed tumor growth in a syngeneic mouse model of lung carcinoma (PMID: 10692563). | 10692563 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | TAS-115 | Phase I | Actionable | In a Phase I trial, TAS-115 was well tolerated and demonstrated preliminary efficacy in patients with advanced solid tumors, with stable disease as best overall response in 37.8% (31/82) of the patients (PMID: 31820255). | 31820255 | |
| 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 | subacute myeloid leukemia | not applicable | Cytarabine + Venetoclax | Preclinical - Patient cell culture | Actionable | In a preclinical study, Venclexta (venetoclax) and Cytosar-U (cytarabine) combination treatment synergistically induced cell death in patient-derived acute myeloid leukemia cells in culture (PMID: 27103402). | 27103402 | |
| Unknown unknown | breast cancer | not applicable | AZD7648 + Pegylated liposomal-doxorubicin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AZD7648 and Doxil (pegylated liposomal-doxorubicin) combination treatment synergistically inhibited growth of breast cancer cell lines in culture, and inhibited tumor growth and induced regression in a cell line xenograft model (PMID: 31699977). | 31699977 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Cabozantinib | Phase II | Actionable | In a Phase II clinical trial, Cometriq (cabozantinib) showed safety and anti-tumor activity in several advanced solid tumor types (J Clin Oncol 29: 2011 (suppl; abstr 3010)). | detail... | |
| Unknown unknown | hairy cell leukemia | not applicable | Fenretinide | Phase I | Actionable | In a Phase I trial, Fenretinide treatment resulted in stable disease in a patient with hairy cell leukemia (PMID: 28420721). | 28420721 | |
| Unknown unknown | thymoma | not applicable | Resminostat | Phase I | Actionable | In a Phase I trial, Resminostat (4SC-201) treatment resulted in stable disease in 58% (11/19) of heavily pretreated patients with advanced solid tumors, and a 27% reduction of tumor in a thymoma patient (PMID: 24065624). | 24065624 | |
| Unknown unknown | melanoma | not applicable | G-TPP + Venetoclax | Preclinical - Cell culture | Actionable | In a preclinical study, combination of the mitochondrial Hsp90 inhibitor G-TPP and the Bcl2 inhibitor Venclexta (venetoclax) resulted in increased apoptosis of melanoma cell lines in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | pancreatic cancer | not applicable | PF-06840003 | Preclinical | Actionable | In a preclinical study, PF-06840003 inhibited tumor growth in a syngeneic mouse model of pancreatic cancer (PMID: 30232146). | 30232146 | |
| 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 | triple-receptor negative breast cancer | not applicable | Rivoceranib | Phase II | Actionable | In a Phase II trial, 500 mg of Apatinib (YN968D1) produced a median progression-free survival of 3.3 months in TNBC patients (PMID: 24604288). | 24604288 | |
| Unknown unknown | colorectal cancer | not applicable | Binimetinib + Fluorouracil + Leucovorin + Oxaliplatin | Phase I | Actionable | In a Phase I clinical trial, the combination of Binimetinib (MEK162) and FOLFOX chemotherapy demonstrated manageable toxicity and preliminary efficacy in metastatic colorectal cancer patients with chemotherapy resistance (J Clin Oncol 34, 2016 (suppl; abstr 2544)). | detail... | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Pembrolizumab | FDA approved | Actionable | In a Phase II trial (KEYNOTE-224) that suported FDA approval, Keytruda (pembrolizumab) treatment resulted in an overall response rate of 17% (18/104, 1 complete response, 17 partial response), and stable disease in 44% (46/104) of patients with hepatocellular carcinoma previously treated with Nexavar (sorafenib), with a median progression-free survival of 4.8 months (PMID: 29875066; NCT02702414). | detail... 29875066 | |
| 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 | 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 | 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 | 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 | Advanced Solid Tumor | not applicable | TAK-733 | Phase I | Actionable | In a Phase I clinical trial, TAK-733 demonstrated safety and some preliminary efficacy in patients with advanced solid tumors with partial response in 5% (2/41) and stable disease in 37% (15/41) of patients (PMID: 27650277). | 27650277 detail... | |
| Unknown unknown | multiple myeloma | not applicable | Bortezomib + Tabalumab | Phase I | Actionable | In a Phase I trial, 42% (20/46) of patients with multiple myeloma demonstrated a partial response, including 3 with a complete response and 2 with a very good partial response, when treated with a combination of Tabalumab and Velcade (bortezomib) (PMID: 27287072). | 27287072 | |
| Unknown unknown | adenoid cystic carcinoma | not applicable | LY3039478 | Phase I | Actionable | In a Phase I trial, a patient with adenoid cystic carcinoma demonstrated a metabolic response when treated with LY3039478 (European Journal of Cancer, Volume 69, S15). | detail... | |
| 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 | 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 | urinary bladder cancer | not applicable | MPT0L145 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with MPT0L145 resulted in decreased cell viability in bladder cancer cell lines in culture (PMID: 29222162). | 29222162 | |
| Unknown unknown | glioblastoma | not applicable | G-TPP + Venetoclax | Preclinical - Patient cell culture | Actionable | In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the Bcl2 inhibitor Venclexta (venetoclax) synergistically inhibited viability of established lines and patient-derived glioblastoma cells in culture (PMID: 28522750). | 28522750 | |
| 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 cancer | not applicable | Cediranib + Gefitinib | Phase I | Actionable | In a Phase I trial, the combination of Cediranib and Iressa (gefitinib) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, including a partial response in a patient with lung cancer (PMID: 20061136). | 20061136 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Anlotinib | Phase I | Actionable | In a Phase I trial, Anlotinib (AL-3818) treatment resulted in partial response in 15% (3/20) and stable disease in 70% (14/20) of patients with advanced solid tumors (J Clin Oncol 33, 2015 (suppl; abstr e13586)). | detail... | |
| Unknown unknown | pancreatic adenocarcinoma | not applicable | Cabozantinib | Preclinical | Actionable | In a preclinical trial, Cometriq (cabozantinib) promoted apoptosis of pancreactic ductal adenocarcinoma cells (PMID: 23661005). | 23661005 | |
| 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 | multiple myeloma | not applicable | Oprozomib | Phase II | Actionable | In a Phase II trial, Oprozomib (ONX 0912) treatment resulted in an objective response rate of 41.0%, 28.1%, and 25.0% in the 2/7, 240/300-mg/day; 5/14, 150/180-mg/day; and 5/14, 240-mg/day cohorts of patients with multiple myeloma (n=95) (PMID: 31142508; NCT01416428). | 31142508 | |
| Unknown unknown | sarcoma | not applicable | Anlotinib | Phase II | Actionable | In a Phase II trial, Anlotinib (AL-3818) treatment resulted in a 12-week progression-free rate of 68%, median progression-free survival of 5.6 months, median overall survival of 12 months, and an objective response rate of 13% (n=166), all partial responses, in patients with soft tissue sarcoma (PMID: 29895706; NCT01878448). | 29895706 | |
| 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 | glioblastoma | not applicable | Ad-RTS-IL-12 plus AL | Phase I | Actionable | In a Phase I trial, Ad-RTS-IL-12 plus AL demonstrated safety and preliminary efficacy in glioblastoma multiforme patients, with 100% (7/7) of patients remained alive and 5 patients having a follow-up of more than 90 days posttreatment (J Clin Oncol 34, 2016 (suppl; abstr 2052)). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | Molibresib + Rucaparib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Molibresib (GSK525762) and Rubraca (rucaparib) resulted in a synergistic effect, demonstrating greater growth inhibition of ovarian cancer cells in culture compared to Molibresib (GSK525762) treatment alone (PMID: 32927276). | 32927276 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | PV1162 | Preclinical - Cell culture | Actionable | In a preclinical study, cultured cells treated with PV1162 showed increased loss of human artificial chromosomes, suggesting chromosomal instability (PMID: 26837770). | 26837770 | |
| Unknown unknown | multiple myeloma | not applicable | MV-NIS | Phase Ib/II | Actionable | In a Phase I/II trial, MV-NIS treatment resulted in enhanced T-cell response to tumor antigens in 40% (4/10) of patients with multiple myeloma (Journal of Clinical Oncology 36, no. 5_suppl (February 10 2018) 218-218; NCT00450814). | detail... | |
| 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 | 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 | Advanced Solid Tumor | not applicable | S-49076 | Phase I | Actionable | In a Phase I trial, S-49076 demonstrated safety and limited preliminary clinical activity in patients with advanced solid tumors, resulting in stable disease as best response in 50% of patients, with 23% demonstrating stable disease for at least 3 months, and 9 patients demonstrating stable disease for at least 6 months (PMID: 28624695). | 28624695 | |
| 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 | lymphoma | not applicable | S63845 | Preclinical - Cell culture | Actionable | In a preclinical study, S63845 inhibited survival of several lymphoma cell lines in culture (PMID: 27760111). | 27760111 | |
| Unknown unknown | lung small cell carcinoma | 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 7% (1/15, 1 partial responses) and a disease control rate of 20% (3/15) in immunotherapy-naive patients with advanced or metastatic small cell lung cancer, response occurred regardless of PD-L1, CCR4, CD8 expression levels and tumor mutational burden (PMID: 31455681; NCT02476123). | 31455681 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Brexucabtagene autoleucel | FDA approved | Actionable | In a Phase II trial (ZUMA-2) that supported FDA approval, Tecartus (brexucabtagene autoleucel) infusion following conditioning chemotherapy resulted in an objective response (OR) in 96% (56/60, 40 complete responses (CR)) of patients with relapsed or refractory mantle cell lymphoma in the primary analysis group, with 57% (34/60) remained in remission at a median follow-up of 12.3 months, OR and CR in the intention-to-treat analysis (n=74) were 85% and 59%, respectively (PMID: 32242358; NCT02601313). | detail... 32242358 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Ascrinvacumab | Phase I | Actionable | In a Phase I trial, a patient with hepatocellular carcinoma demonstrated a partial response for 44 days when treated with PF-03446962 (PMID: 26655846). | 26655846 | |
| 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 | glioblastoma | not applicable | AOH1160 | Preclinical - Patient cell culture | Actionable | In a preclinical study, AOH1160 inhibited growth of glioblastoma cells from patients in culture (PMID: 29967249). | 29967249 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Debio 0617B | Preclinical - Patient cell culture | Actionable | In a preclinical study, Debio 0617B inhibited survival of a variety of patient-derived tumor cells in culture (PMID: 27439479). | 27439479 | |
| Unknown unknown | cervical cancer | not applicable | AsiDNA | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA inhibited survival of cervical cancer cell lines in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | lung cancer | not applicable | VB-111 | Preclinical | Actionable | In a preclinical study, VB-111 treatment resulted in increased tumor lymphocyte infiltration and reduced tumor burden in animal models of lung cancer (AACR Annual Meeting 2019, Abstract 4979). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Tisagenlecleucel | FDA approved | Actionable | In a Phase II trial (JULIET) that supported FDA approval, Kymriah (tisagenlecleucel) treatment resulted in an overall response rate of 50% (34/68) , with complete response in 32% (22/68) and partial response in 18% (12/68) of patients with diffuse large B-cell lymphoma (ASH, 59th Annual Meeting and Exposition, Dec 2017, Abstract 577; NCT02445248). | detail... detail... | |
| Unknown unknown | melanoma | not applicable | Lenvatinib + Pembrolizumab | Phase Ib/II | Actionable | Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 48% (10/21) in patients with metastatic melanoma, with a median duration of response of 12.5 months, and a median progression-free survival of 5.5 months (PMID: 31961766; NCT02501096). | 31961766 | |
| Unknown unknown | pancreatic cancer | not applicable | Gemcitabine + Pimasertib | Preclinical | Actionable | In a preclinical study, treatment with Pimasertib (MSC1936369B) followed by Gemzar (gemcitabine) resulted in enhanced inhibition of proliferation and induction of apoptosis in pancreatic cell lines in culture (PMID: 26228206). | 26228206 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | BAZ2-ICR + BI-9564 + JQ1 | Preclinical - Cell culture | Actionable | In a preclinical study, BAZ2-ICR treatment combined with BI-9564 and JQ1 inhibited Rb1 phosphorylation, induced senescence and complete growth inhibition, and led to enhanced cell cycle arrest in triple-negative breast cancer cell lines in culture (PMID: 31000582). | 31000582 | |
| Unknown unknown | colon carcinoma | not applicable | SB 11285 | Preclinical | Actionable | In a preclinical study, treatment with SB 11285 delayed tumor growth and reduced tumor volume in a syngeneic mouse model of colon carcinoma (Journal of Clinical Oncology 2017 35:15_suppl, e14616). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | KTD606 | Preclinical - Cell culture | Actionable | In a preclinical study, KTD606 treatment inhibited anchorage-dependent growth of transformed cells in culture (PMID: 12184411). | 12184411 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | SGN-CD228A | Preclinical - Pdx | Actionable | In a preclinical study, SGN-CD228A treatment delayed tumor growth and resulted in complete responses in patient-derived xenograft (PDX) models of non-small cell lung carcinoma (Cancer Res 2019;79(13 Suppl):Abstract nr 2688). | detail... | |
| Unknown unknown | prostate cancer | not applicable | Enzalutamide + Ipatasertib | Phase I | Actionable | In a Phase Ib trial, the combination of Ipatasertib (GDC-0068) and Xtandi (enzalutamide) demonstrated safety in prostate cancer patients, and resulted in an overall response rate of 11.8% (2/17), including partial responses in two patients, stable disease in 23.5% (4/17) of patients, a six-month progression-free survival rate of 23.5% (4/17), and maximum progression-free survival duration of 19.5 months (PMID: 32205017; NCT01362374). | 32205017 | |
| Unknown unknown | Merkel cell carcinoma | not applicable | Avelumab | Phase III | Actionable | In a Phase II trial (JAVELIN Merkel 200), Bavencio (avelumab) treatment resulted in a 62.1% (18/29) objective response rate and a median progression-free survival of 9.1 months in patients with treatment-naive metastatic Merkel cell carcinoma, with response duration estimated to be greater than 3 months and 6 months in 93% and 83% of responding patients, respectively (PMID: 29566106; NCT 02155647). | 29566106 | |
| Unknown unknown | Merkel cell carcinoma | not applicable | Avelumab | FDA approved | Actionable | In a Phase II trial (JAVELIN Merkel 200) that supported FDA approval, Bavencio (avelumab) treatment resulted in an objective response response rate of 31.8% (28/88), with complete response in 9% (8/88) and partial response in 23% (20/88) of patients with metastatic Merkel cell carcinoma that progressed after chemotherapy (PMID: 27592805; NCT02155647). | 27592805 detail... | |
| Unknown unknown | Merkel cell carcinoma | not applicable | Avelumab | Phase Ib/II | Actionable | In a Phase I/II trial, addition of Bavencio (avelumab) to adoptive transfer of Merkel cell polyomavirus (MCPyV)-specific T cells and HLA upregulation resulted in sustained complete response in 75% (3/4) of patients with MCPyV-associated Merkel cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 3044)). | detail... | |
| Unknown unknown | follicular 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 89% (8/9, 2 complete responses, 6 partial responses) in patients with relapsed or refractory follicular lymphoma, with a median duration of response of 137 days (PMID: 32327472; NCT02000934). | 32327472 | |
| 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 | hematologic cancer | not applicable | Umbralisib | Phase I | Actionable | In a Phase I trial, Ukoniq (umbralisib) treatment resulted in disease burden reduction in 73% (53/73), complete response in 4% (3/73), and partial response in 41% (30/73) of patients with chronic lymphocytic leukemia and lymphoma (PMID: 29475723; NCT01767766). | 29475723 | |
| Unknown unknown | hematologic cancer | not applicable | Umbralisib | Phase I | Actionable | In a Phase I trial, Ukoniq (umbralisib) demonstrated safety in patients with advanced hematological malignancies and preliminary efficacy in patients with chronic lymphocytic leukemia and non-Hodgkin's lymphoma (J Clin Oncol (Meeting Abstracts) 2015 33: 7069). | detail... | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Ofatumumab | FDA approved | Actionable | In a Phase III trial supporting FDA approval, Arzerra (ofatumumab) maintenance treatment resulted in improved progression free survival (29.4 vs 15.2 months) compared to observation group in chronic lymphocytic leukemia patients in complete or partial remission after second-line or third-line treatment (PMID: 20194866, PMID: 26377300). | 26377300 20194866 detail... | |
| Unknown unknown | transitional cell carcinoma | not applicable | Durvalumab | FDA approved | Actionable | In a Phase I/II trial that supported FDA approval, treatment with Imfinzi (durvalumab) was well tolerated and resulted in an overall response rate of 17.8% (34/191; 7 complete responses), median progression-free survival of 1.5 months, and median overall survival of 18.2 months in patients with advanced urothelial cancer (PMID: 28817753; NCT01693562). | detail... detail... 28817753 | |
| Unknown unknown | ovarian cancer | not applicable | Paclitaxel + TRX-E-002-1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TRX-E-002-1 given as a maintenance treatment after Taxol (paclitaxel) treatment resulted in less tumor recurrence compared to placebo in cell line xenograft models of ovarian cancer (PMID: 27196760). | 27196760 | |
| 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 | 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 | 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 | colorectal cancer | not applicable | CBP501 + Cisplatin | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of CBP501 and Platinol (cisplatin) resulted in increased cell death compared to Platinol (cisplatin) alone in a human colorectal cancer cell line in culture (PMID: 17237275). | 17237275 | |
| Unknown unknown | basal cell carcinoma | not applicable | Taladegib | Phase I | Actionable | In a Phase I trial, Taladegib treatment resulted in a clinical response in 46.8% (22/47) of patients with basal cell carcinoma, including 16 patients with a confirmed partial response, 1 patient with an unconfirmed partial response, and 5 patients with a confirmed complete response (PMID: 29483143). | 29483143 | |
| Unknown unknown | urinary bladder cancer | not applicable | GSK2126458 | Phase I | Actionable | In Phase I trial, GSK2126458 treatment was well-tolerated and resulted in a partial response and stable disease in two patients and one patient with bladder cancer, respectively (PMID: 26603258). | 26603258 | |
| Unknown unknown | sarcoma | not applicable | Pazopanib | Clinical Study | Actionable | In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 15.4 weeks and median survival of 11.2 months in patients with soft tissue sarcoma (PMID: 26970174). | 26970174 | |
| Unknown unknown | sarcoma | 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 soft tissue sarcoma (PMID: 22595799). | 22595799 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 | 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 | melanoma | not applicable | GNE-317 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a melanoma cell line xenograft model demonstrated cell death of metastasized tumor cells within a small region of the brain when treated with GNE-317 (PMID: 27521448). | 27521448 | |
| 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 | Advanced Solid Tumor | not applicable | Navitoclax | Preclinical - Cell culture | Actionable | In a preclinical study, Navitoclax (ABT-263) treatment alone was not effective in a number of cell lines derived from solid tumors in culture (PMID: 27974663). | 27974663 | |
| Unknown unknown | malignant glioma | not applicable | AdV-tk + Valacyclovir | Phase II | Actionable | In a Phase II trial, addition of AdV-tk and Valacyclovir to standard of care improved meidan overall survival (17.1 vs 13.5 months) compared to standard of care alone in patients with malignant glioma (PMID: 26843484). | 26843484 | |
| Unknown unknown | acute myeloid leukemia | not applicable | ORY-1001 | Phase I | Actionable | In a Phase I trial, ORY-1001 (iadademstat) treatment demonstrated safety in patients with acute myeloid leukemia and resulted in complete remission in one patient and hematologic improvement in 2 additional patients of 27 patients in the dose escalation phase, and resulted in decreased blast percentage and induction of differentiation in patients in the expansion cohort (PMID: 33052756). | 33052756 | |
| Unknown unknown | acute myeloid leukemia | not applicable | ORY-1001 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, ORY-1001 decreased colony forming ability in several acute myeloid leukemia (AML) cell lines and primary AML samples in culture, and reduced tumor growth in AML cell line xenograft model (PMID: 29502954). | 29502954 | |
| Unknown unknown | lung cancer | not applicable | TC-A2317 | Preclinical - Cell culture | Actionable | In a preclinical study, TC-A2317 inhibited growth, induced apoptosis in lung cancer cell lines in culture (PMID: 27713168). | 27713168 | |
| Unknown unknown | alveolar soft part sarcoma | not applicable | Durvalumab + Tremelimumab | Clinical Study | Actionable | In a clinical case study, a patient with alveolar soft part sarcoma had a 73% tumor reduction and a response lasting greater than 30 months when treated with Imfinzi (durvalumab) and Tremelimumab, and the tumor was shown retrospectively to have a mismatch repair defect signature, poor immune infiltration, and 2% tumoral PD-L1 positivity (PMID: 30018044; NCT02261220). | 30018044 | |
| Unknown unknown | leiomyosarcoma | no benefit | Pembrolizumab | Phase II | Actionable | In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in an objective response rate of 0% (0/10) in patients with leiomyosarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). | detail... | |
| Unknown unknown | pancreatic adenocarcinoma | not applicable | Gemcitabine + Vandetanib | Phase I | Actionable | In a Phase I trial, Caprelsa (vandetanib), in combination with Gemzar (gemcitabine), demonstrated safety and resulted in stable disease in metastatic pancreatic adenocarcinoma patients (PMID: 21921646). | 21921646 | |
| Unknown unknown | pancreatic adenocarcinoma | no benefit | MK2206 + Selumetinib | Phase II | Actionable | In a Phase II trial, the combination of Selumetinib (AZD6244) and MK2206 did not result in improved median overall survival compared to mFOLFOX therapy (3.9 mo vs. 6.7 mo) or improved median progression-free survival (1.9 mo vs 2.0 mo) in patients with pancreatic adenocarcinoma (PMID: 27978579). | 27978579 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Docetaxel + VIC-1911 | Preclinical | Actionable | In a preclinical study, TAS-119 demonstrated synergistic effects with Taxol (paclitaxel) or Taxotere (docetaxel) in multiple tumor cell lines by promoting apoptosis (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr A268). | detail... | |
| 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 | epithelioid sarcoma | not applicable | Tazemetostat | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Tazverik (tazemetostat) treatment resulted in an objective response rate of 15% (9/62) and a disease control rate of 26% (16/62) in patients with locally advanced or metastatic epithelioid sarcoma, with a median duration of response not reached and a median overall survival of 82.4 weeks (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 11003-11003, NCT02601950). | 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 | 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 | 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 | gastrointestinal stromal tumor | not applicable | Sorafenib | Phase II | Actionable | In a Phase II trial, Nexavar (sorafenib) treatment resulted in partial response in 13% (4/31), stable disease in 52% (16/31), a median progression-free survival of 4.9 months and an overall survivals of 9.7 months in gastrointestinal stromal tumor patients who failed prior tyrosine kinase inhibitors (PMID: 22270258). | 22270258 | |
| 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 | Ewing sarcoma | not applicable | GSK1904529A | Preclinical - Cell culture | Actionable | In a preclinical study, Ewing's sarcoma cells were sensitive to GSK1904529A in culture, resulting in decreased cell viability (PMID: 19383820). | 19383820 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | ALX148 + Pembrolizumab | Phase I | Actionable | In a Phase I trial, ALX148 and Keytruda (pembrolizumab) combination treatment resulted in a partial response in 18% (3/17) and stable disease in 24% (4/17) of patients with head and neck squamous cell carcinoma that progressed on platinum therapy (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 2514-2514; NCT03013218). | detail... | |
| Unknown unknown | lung cancer | not applicable | Dinaciclib + Paclitaxel | Preclinical - Cell culture | Actionable | In a preclinical study, combination of Dinaciclib (SCH 727965) and Taxol (paclitaxel) demonstrated enhanced growth inhibition in lung cancer cell lines in culture (PMID: 27550941). | 27550941 | |
| Unknown unknown | clear cell renal cell carcinoma | not applicable | Nivolumab | Phase II | Actionable | In a Phase II trial (HCRN GU16-260), Opdivo (nivolumab) treatment resulted in an objective response rate of 29.3% (34/117, 5 complete responses, 29 partial responses) and stable disease in 40.2% (47/117) of patients with treatment naive clear cell renal cell carcinoma, with a median duration of response of 13.8 months and a median progression-free survival of 7.4 months (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 5006-5006; NCT03117309). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | CEP1347 | Preclinical - Cell culture | Actionable | In a preclinical study, CEP1347 induced differentiation, inhibited self-renewal, and decreased phosphorylation of Jun and Jnk in ovarian cancer stem cells in culture (PMID: 29212273). | 29212273 | |
| 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 | transitional cell carcinoma | not applicable | Lenvatinib + Pembrolizumab | Phase Ib/II | Actionable | Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 25% (5/20) in patients with metastatic urothelial cancer, with a median duration of response not evaluable, and a median progression-free survival of 5.4 months (PMID: 31961766; NCT02501096). | 31961766 | |
| Unknown unknown | cutaneous T cell lymphoma | not applicable | Mogamulizumab | Phase III | Actionable | In a Phase III trial, Mogamulizumab treatment resulted in significant improvement in progression-free survival (7.7 vs 3.1 months, HR=0.53) and overall response rate (28.0% vs 4.8%) compared to Zolinza (vorinostat) in patients with cutaneous T cell lymphoma (Blood 2017 130(Suppl 1):817; NCT01728805). | detail... | |
| 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 | CLL/SLL | not applicable | Umbralisib | Phase II | Actionable | In a Phase II trial, Ukoniq (umbralisib) treatment resulted in an objective response rate of 50.0% (11/22, 1 complete response) and a disease control rate of 86.4% in patients with relapsed or refractory small lymphocytic lymphoma who have two or more prior lines of therapy, with a median time to response of 2.7 months and a median progression-free survival of 20.9 months (62nd ASH Annual Meeting and Expo, Dec 2020, Abstract 2934; NCT02793583). | detail... | |
| Unknown unknown | CLL/SLL | not applicable | ME-401 | Phase I | Actionable | In a Phase I trial, ME-401 treatment was well-tolerated, resulted in an objective response rate of 89% (9/10) in patients with relapsed or refractory CLL/SLL (J Clin Oncol 38: 2020 (suppl; abstr 8016); NCT02914938). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | Daunorubicin + Venetoclax | Preclinical - Patient cell culture | Actionable | In a preclinical study, Venclexta (venetoclax) and Daunorubicin combination treatment synergistically induced cell death in patient-derived acute myeloid leukemia cells in culture (PMID: 27103402). | 27103402 | |
| 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 | malignant peripheral nerve sheath tumor | not applicable | Dasatinib | Phase II | Actionable | In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in patients with malignant peripheral nerve sheath tumors (n=14) was suspended due to lack of drug activity (PMID: 26710211). | 26710211 | |
| Unknown unknown | lung non-small cell carcinoma | no benefit | LY2603618 + Pemetrexed Disodium | Phase II | Actionable | In a Phase II trial, the combination treatment of LY2603618 and Alimta (pemetrexed) in patients with non-small lung carcinoma demonstrated similar results when compared to treatment with Alimta (pemetrexed) as a single agent (PMID: 27350064). | 27350064 | |
| 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 | multiple myeloma | not applicable | Dexamethasone + Ixazomib + Lenalidomide | FDA approved | Actionable | In a Phase III trial (TOURMALINE-MM1) that supported FDA approval, addition of Ninlaro (ixazomib) to the regimen of Revlimid (lenalidomide) and dexamethasone significantly improved progression-free survival (20.6 vs 14.7 months, HR=0.74, p=0.01) in patients with relapsed or refractory multiple myeloma who received at least one prior therapy (PMID: 27119237; NCT01564537). | 27119237 detail... | |
| Unknown unknown | lung non-small cell carcinoma | 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 50% (11/22) of patients with non-small cell lung carcinoma (PMID: 24816908). | 24816908 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Chiauranib | Phase I | Actionable | In a Phase I trial, Chiauranib (CS2164) demonstrated safety and preliminary efficacy, resulted in stable disease as best response in 66.7% (12/18) of patients with advanced solid tumors (PMID: 30642372; NCT02122809). | 30642372 | |
| Unknown unknown | renal cell carcinoma | not applicable | Avelumab + Axitinib | FDA approved | Actionable | In a Phase III trial that supported FDA approval (JAVELIN Renal 101), Inlyta (axitinib) plus Bavencio (avelumab) resulted in a median progression-free survival of 13.8 mo. and an objective response rate of 51.4%, vs. 8.4 mo. and 25.7% with Sutent (sunitinib), respectively in patients with advanced renal cell carcinoma, and at median follow-up 14.3% (63) of patients treated with Inlyta (axitinib) plus Bavencio (avelumab) had died vs. 16.9% (75) with Sutent (sunitinib) (PMID: 30779531; NCT02684006). | detail... 30779531 | |
| 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 | Everolimus + Vatalanib | Phase I | Actionable | In a Phase I trial, Vatalanib and Afinitor (everolimus) combination therapy demonstrated acceptable toxicity, resulted in a partial response in 12.9% (9/70) and stable disease in 58.6% (41/70) of patients with advanced solid tumors (PMID: 32328844; NCT00655655). | 32328844 | |
| Unknown unknown | head and neck cancer | not applicable | Cetuximab + NT219 | Preclinical - Pdx | Actionable | In a preclinical study, NT219 treatment in combination with Erbitux (cetuximab) inhibited tumor growth and induced regression in patient-derived xenograft (PDX) models of head and neck cancer, and synergistically inhibited tumor growth in a PDX model of head and neck squamous cell carcinoma (Cancer Res 2020;80(16 Suppl):Abstract nr 5639). | detail... | |
| Unknown unknown | clear cell renal cell carcinoma | no benefit | Sorafenib + Temsirolimus | Phase II | Actionable | In a Phase II clinical trial, treatment with the combination of Nexavar (sorafenib) and Torisel (temsirolimus) did not prolong progression-free survival compared to treatment with Avastin (bevacizumab) monotherapy (7.4 months vs 7.5 months) in patients with renal clear cell carcinoma (PMID: 26077237). | 26077237 | |
| 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 | 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 | 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 | chromophobe renal cell carcinoma | not applicable | Bevacizumab + Everolimus | Phase II | Actionable | In a Phase II trial, 60% (3/5) of patients with chromophobe renal cell carcinoma achieved a 6 month PFS when treated with a combination of Afinitor (everolimus) and Avastin (bevacizumab) (PMID: 27601542). | 27601542 | |
| Unknown unknown | colorectal cancer | not applicable | Avelumab + PF-06840003 | Preclinical | Actionable | In a preclinical study, PF-06840003 and Bavencio (avelumab) combination treatment inhibited IDO1-mediated immunosuppression and resulted in tumor growth inhibition of 74% while treatment with PF-06840003 alone led to a tumor growth inhibition of 41% in a syngeneic mouse model of colorectal cancer (PMID: 30232146). | 30232146 | |
| 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 | sarcoma | not applicable | Carotuximab + Pazopanib | Phase Ib/II | Actionable | In a Phase I/II trial, TRC105 and Votrient (pazopanib) combination therapy resulted in a median progression free survival of 3.95 months and ongoing complete response in 4% (3/81) of soft tissue sarcoma patients (J Clin Oncol 34, 2016 (suppl; abstr 11016)). | detail... | |
| Unknown unknown | colorectal cancer | not applicable | Ibrutinib + unspecified CTLA4 antibody | Preclinical | Actionable | In a preclinical study, the combination of Imbruvica (ibrutinib) and an anti-CTLA4 antibody resulted in complete tumor regression in colorectal cancer mouse models (Cancer Res 2016;76(14 Suppl):Abstract nr 2321). | detail... | |
| Unknown unknown | colon carcinoma | not applicable | Mps-BAY2a + Paclitaxel | Preclinical | Actionable | In a preclinical study, Mps-BAY2a, in combination with paclitaxel, had increased efficacy in inhibiting cell proliferation of colon carcinoma cell in culture (PMID: 23933817). | 23933817 | |
| Unknown unknown | neuroblastoma | not applicable | GANT61 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, GANT61 induced apoptosis and decreased growth of neuroblastoma cells in culture, and inhibited tumor growth in neuroblastoma cell line xenograft models (PMID: 22949014). | 22949014 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Cetuximab + Imatinib | Preclinical - Pdx | Actionable | In a preclinical study, Gleevec (imatinib) treatment in combination with Erbitux (cetuximab) synergistically inhibited tumor growth and induced regression in an Erbitux (cetuximab)-resistant patient-derived xenograft (PDX) model of head and neck squamous cell carcinoma (PMID: 32439698). | 32439698 | |
| Unknown unknown | colorectal cancer | no benefit | Olaparib | Phase II | Actionable | In a Phase II clinical trial, treatment with Lynparza (olaparib) did not result in clinical activity in colorectal cancer patients that had progressed on prior standard therapy, including both microsatellite-stable patients and those that demonstrated high microsatellite instability (PMID: 26786262). | 26786262 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Crizotinib + Navitoclax | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Navitoclax (ABT-263) and Xalkori (crizotinib) resulted in a synergistic effect and inhibited the growth of triple-receptor negative breast cancer cells in culture (PMID: 27872098). | 27872098 | |
| Unknown unknown | gastrointestinal stromal tumor | not applicable | JSH-150 | Preclinical - Cell culture | Actionable | In a preclinical study, JSH-150 inhibited proliferation of a gastrointestinal stromal tumor cell line in culture (PMID: 30253346). | 30253346 | |
| Unknown unknown | lung squamous cell carcinoma | not applicable | Cisplatin + Necitumumab + Pemetrexed Disodium | Phase III | Actionable | In a Phase III trial, the addition of Necitumumab (IMC-11F8) to pemetrexed and cisplatin did not increase survival of stage IV non-squamous NSCLC patients (PMID: 25701171). | 25701171 | |
| Unknown unknown | lymphoma | not applicable | GSK3368715 | Preclinical - Cell culture | Actionable | In a preclinical study, GSK3368715 inhibited growth of lymphoma cell lines in culture, and demonstrated cytotoxicity in 56% of cell lines tested (PMID: 31257072). | 31257072 | |
| Unknown unknown | ovarian cancer | not applicable | Carboplatin + DNIB0600A | Phase I | Actionable | In a Phase Ib trial, the combination of DNIB0600A (Lifastuzumab vedotin) and Paraplatin (carboplatin) with or without Avastin (bevacizumab) demonstrated safety in patients with recurrent ovarian cancer and resulted in an objective response rate (ORR) of 59% (24/41), with a median progression-free survival (PFS) of 10.7 months, DNIB0600A (Lifastuzumab vedotin) at RP2D (2.4 mg/kg) with Paraplatin (carboplatin) resulted in an ORR of 50% (10/20) and a median PFS of 8.5 months (PMID: 32534811; NCT01995188). | 32534811 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | SR-4835 | Preclinical - Pdx | Actionable | In a preclinical study, SR-4835 treatment induced DNA damage and cell death, and inhibited tumor growth in a patient-derived xenograft (PDX) model of triple-negative breast cancer harboring a BRCA1 mutation (PMID: 31668947). | 31668947 | |
| Unknown unknown | non-Hodgkin lymphoma | not applicable | Entospletinib + Idelalisib | Phase II | Actionable | In a Phase II trial, Zydelig (idelalisib) and Entospletinib combination treatment resulted in objective response in 36% of patients with non-Hodgkin lymphoma, but was terminated due to severe treatment-emergent pneumonitis (PMID: 26968534). | 26968534 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | AT13148 | Phase I | Actionable | In a Phase I trial, AT13148 treatment resulted in no complete or partial response in 51 patients with advanced solid tumors, and was not recommended for further development due to the unfavorable pharmacokinetic profile (PMID: 32616501). | 32616501 | |
| Unknown unknown | malignant mesothelioma | not applicable | Pembrolizumab | Clinical Study | Actionable | In a clinical study, Keytruda (pembrolizumab) treatment in patients with MSLN positive malignant mesothelioma who had progressed on LMB-100 treatment resulted in an overall response rate of 40% (4/10) and median progression-free survival of 3.3 months, including one complete response, three partial responses, and one patient with stable disease among seven evaluable patients (PMID: 32611684). | 32611684 | |
| 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 | rhabdomyosarcoma | not applicable | JQ1 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, JQ1 treatment resulted in cytostatic effects in patient-derived rhabdomyosarcoma cell lines in culture, but inhibited tumor growth in patient-derived xenograft models due to inhibition of tumor angiogenesis (PMID: 26908627). | 26908627 | |
| Unknown unknown | ovarian 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 | lung non-small cell carcinoma | not applicable | Zenocutuzumab | Phase Ib/II | Actionable | In a Phase I/II trial, Zenocutuzumab (MCLA-218) resulted in partial response for more than 10 months in a patient with non-small cell lung cancer (Cancer Res 2016;76(14 Suppl):Abstract nr CT050). | detail... | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | PR-104 | Preclinical | Actionable | In a preclinical study, PR-104 inhibited growth of triple-receptor negative breast cancer cell lines under hypoxic culture conditions, regardless of their BRCA1 status (PMID: 25193512). | 25193512 | |
| Unknown unknown | multiple myeloma | not applicable | Daratumumab and hyaluronidase-fihj + Dexamethasone + Lenalidomide | FDA approved | Actionable | In a Phase II trial (PLEIADES) that supported FDA approval, Darzalex Faspro (Daratumumab and hyaluronidase-fihj) demonstrated safety and efficacy comparable to Darzalex (daratumumab) when combined with Adexone (dexamethasone) and Revlimid (lenalidomide) in patients with relapsed or refractory multiple myeloma, resulted in an objective response rate of 93.8% (61/65, complete response 21.5%) (17th International Myeloma Workshop Sep 2019, Boston, MA, US. OAB-022). | detail... detail... | |
| 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 | melanoma | not applicable | Pembrolizumab + Propranolol | Phase I | Actionable | In a Phase I trial, treatment with the combination of Keytruda (pembrolizumab) and Angilol (propranolol) demonstrated safety in patients with metastatic melanoma who had not received prior treatment, and resulted in an objective response rate of 78% (7/9, all partial responses) and stable disease in one patient (PMID: 33127652; NCT03384836). | 33127652 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Cetuximab + Prexasertib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Erlotinib (cetuximab) and Prexasertib (LY2606368 resulted in greater decreased cell proliferation in head and neck squamous cell carcinoma cells in culture compared to either agent alone (PMID: 28138028). | 28138028 | |
| Unknown unknown | multiple myeloma | not applicable | Dexamethasone + Elotuzumab + Lenalidomide | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with the combination of Empliciti (elotuzumab) with Revlimid (lenalidomide) and dexamethosone resulted in an overall response rate of 79%, compared to 66% with Revlimid (lenalidomide) and dexamethosone combination therapy in patients with multiple myeloma (PMID: 27493709). | detail... 27493709 | |
| Unknown unknown | renal cell carcinoma | not applicable | Pazopanib + Trametinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, combination of Mekinist (trametinib) and Votrient (pazopanib) effectively inhibited tumor angiogenesis and growth in cell line xenograft models of renal cell carcinoma (PMID: 26487278). | 26487278 | |
| Unknown unknown | melanoma | not applicable | MIW815 + Spartalizumab | Phase Ib/II | Actionable | In a Phase Ib trial, MIW815 (ADUS100), in combination with Spartalizumab (PDR001), demonstrated preliminary efficacy in PD-1 (PDCD1)-relapsed/refractory melanoma (J of Clin Oncol 37, 2019 (suppl; abstr 2507); NCT03172936). | detail... | |
| Unknown unknown | head and neck cancer | not applicable | Olaparib | Preclinical - Pdx | Actionable | In a preclinical study, a patient-dervived xenograft (PDX) model of head and neck cancer harboring mutant TP53 and wild-type ATM did not demonstrate sensitivity to Lynparza (olaparib) treatment (PMID: 31699977). | 31699977 | |
| Unknown unknown | breast cancer | not applicable | AsiDNA + Rucaparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA and Rubraca (rucaparib) combination treatment resulted in increased cell death and inhibition of proliferation in breast cancer cell lines compared to Rubraca (rucaparib) alone in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | LMP400 | Preclinical | Actionable | In a preclinical study, LMP400 inhibited proliferation of a variety of human tumor cell lines in culture (PMID: 23215354). | 23215354 | |
| Unknown unknown | melanoma | not applicable | IMO-2125 | Phase Ib/II | Actionable | In a Phase I/II trial, IMO-2125 treatment resulted in clinical benefit in 67% (6/9, 1 complete response, 2 partial response, 3 stable disease) of patients with melanoma refractory to a PD-1 or PD-L1 inhibitor (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 1187P; NCT02644967). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | TTI-10001 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TTI-10001 treatment activated STING pathway signaling in transformed cells in culture, resulted in elevated expression of pro-inflammatory cytokines and anti-tumor activity in xenograft models (AACR Annual Meeting 2019, Abstract 3854). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CC-90011 | Phase I | Actionable | In a Phase I trial, CC-90011 treatment was well-tolerated, and resulted in stable disease over 6 months in 24% (8/34) of patients with neuroendocrine tumors and carcinomas (PMID: 33046517; NCT02875223). | 33046517 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Mivavotinib | Case Reports/Case Series | Actionable | In a Phase I trial, Mivavotinib (TAK-659) treatment resulted in an objective response rate of 60% (3/5, 3 partial responses) in patients with relapsed or refractory chronic lymphocytic leukemia, with a median duration of response not reached (PMID: 32327472; NCT02000934). | 32327472 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Mivebresib + Venetoclax | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Mivebresib (ABBV-075) and Venclexta (venetoclax) worked synergistically to decrease viability of a acute myeloid leukemia (AML) cell lines in culture, and the combination resulted in increased tumor growth inhibition in an AML cell line xenograft model compared to either agent alone (PMID: 28416490). | 28416490 | |
| Unknown unknown | pancreatic ductal adenocarcinoma | no benefit | Gemcitabine + Nab-paclitaxel + PF-04136309 | Phase I | Actionable | In a Phase Ib trial, PF-04136309 in combination with Abraxane (nab-paclitaxel) and Gemzar (gemcitabine) resulted in a high incidence of pulmonary toxicity (24%, 5/21) in patients with pancreatic ductal adenocarcinoma, and resulted in an objective response rate of 23.8% (5/21, all partial responses), which did not demonstrate improvement compared to the efficacy of Abraxane (nab-paclitaxel) plus Gemzar (gemcitabine) established in prior studies (PMID: 31297636; NCT02732938). | 31297636 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | Oprozomib | Phase I | Actionable | In a Phase I trial, Oprozomib (ONX 0912) demonstrated clinically relevant toxicity and minimal efficacy, with stable disease as best response in patients with advanced solid tumors (PMID: 26924128). | 26924128 | |
| 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 | Motesanib Diphosphate + Paclitaxel | Phase III | Actionable | In a Phase III study, Motesanib plus Paraplatin (carboplatin) or Taxol (paclitaxel) improved overall survival, progression free survival and objective response rate for a subset of Asian patients with advanced nonsquamous non-small cell lung cancer (PMID: 24419239; NCT00460317). | 24419239 | |
| Unknown unknown | acute lymphoblastic leukemia | not applicable | Natalizumab | Preclinical - Pdx | Actionable | In a preclinical study, Tysabri (natalizumab) treatment alone or in combination with chemotherapy prolonged survival in patient-derived xenograft models of acute lymphocytic leukemia (PMID: 23319569). | 23319569 | |
| 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 | kidney cancer | not applicable | CVX-060 + Sunitinib | Preclinical | Actionable | In a preclinical study, the combination of CVX-060 and Sutent (sunitinib) demonstrated a trend improved overall survival compared to single agent Sutent (sunitinib) in mouse models of unresected and resected renal cancer, however, also demonstrated increased toxicity (PMID: 27651308). | 27651308 | |
| Unknown unknown | lung cancer | not applicable | TG6002 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with TG6002 led to decreased survival of lung cancer cells in culture (PMID: 31011628). | 31011628 | |
| 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 | multiple myeloma | not applicable | Carfilzomib + Zotiraciclib | Phase I | Actionable | In a Phase I trial, Zotiraciclib (TG02) in combination with Kyprolis (carfilzomib) resulted in an overall response rate of 27% (3/11) and durable stable disease in 27% (3/11) of multiple myeloma patients (Blood 2015 126:3052). | detail... | |
| Unknown unknown | breast cancer | not applicable | CS-11 | Preclinical | Actionable | In a preclinical study, CS-11 induced apoptosis and inhibited growth of breast cancer cell lines in culture, and inhibited tumor growth and metastasis in an orthotopic breast cancer mouse model (PMID: 28500231). | 28500231 | |
| Unknown unknown | prostate cancer | not applicable | ONC201 | Clinical Study | Actionable | In a clinical case study, a prostate cancer patient demonstrated tumor regression in the primary tumor and metastatic lesions when treated with ONC201 (TIC-10) (PMID: 28331050). | 28331050 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | GDC-0575 + Gemcitabine | Phase I | Actionable | In a Phase I trial, the combination of GDC-0575 and Gemzar (gemcitabine) demonstrated safety and preliminary activity in patients with advanced solid tumors, resulting in a best overall response of stable disease or partial response in 66% (59/90) of patients, with partial responses in 4% (4/90) of patients, including patients with TP53 mutations (PMID: 29788155; NCT01564251). | 29788155 detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | ADXS-503 | Phase I | Actionable | In a Phase I trial, ADXS-503 monotherapy demonstrated manageable toxicity, resulted in stable disease as best response in 3 of 7 patients with metastatic non-small cell lung cancer (J Clin Oncol 38: 2020 (suppl; abstr e21682); NCT03847519). | detail... | |
| 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 | peritoneum cancer | not applicable | CRLX101 + Paclitaxel | Phase Ib/II | Actionable | In a Phase Ib/II trial, CRLX101 in combination with weekly Taxol (paclitaxel) resulted in an objective response rate (ORR) of 31.6% (6/19, 1 complete response, 5 partial responses) and a median progression-free survival of 5.4 months in patients with recurrent or persistent epithelial ovarian, fallopian tube, or primary peritoneal cancer, although the ORR was not superior to that of historical control (PMID: 33390325; NCT02389985). | 33390325 | |
| Unknown unknown | ovarian cancer | not applicable | Lurbinectedin | Phase II | Actionable | In a Phase II trial, Lurbinectedin (PM01183) treatment resulted in an overall response rate of 23% (12/52) of patients with platinum-resistant or refractory ovarian cancer, with a median duration of response of 4.6 months (PMID: 28368437). | 28368437 | |
| 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 | colon carcinoma | not applicable | Irinotecan + VLX600 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, VLX600 combined with Camptosar (irinotecan) synergistically inhibited viability of colon carcinoma cells in culture, and inhibited tumor growth in an orthotopic cell line xenograft model (PMID: 24548894). | 24548894 | |
| Unknown unknown | B-cell lymphoma | not applicable | WM-1119 | Preclinical | Actionable | In a preclinical study, WM-1119 inhibited proliferation of a mouse B-cell lymphoma cell line in culture, and induced tumor growth arrest and decreased tumor burden in mouse models (PMID: 30069049). | 30069049 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Bortezomib + Tinostamustine | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of EDO-S101 and Velcade (bortezomib) decreased viability of primary chronic lymphocytic leukemia cells in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | prostate cancer | not applicable | WANT3 | Preclinical - Cell culture | Actionable | In a preclinical study, WANT3 treatment resulted in suppression of cell invasion of prostate cancer cells in culture (PMID: 27432794). | 27432794 | |
| Unknown unknown | CLL/SLL | not applicable | Venetoclax | Phase I | Actionable | In a Phase I trial, Venclexta (venetoclax) treatment resulted in a 79% (92/116) overall response rate and 20% (23/116) complete response rate in patients with either chronic lymphocytic leukemia or small lymphocytic lymphoma (PMID: 26639348). | 26639348 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Loncastuximab tesirine | Phase I | Actionable | In a Phase I trial, Loncastuximab tesirine treatment resulted in an objective response rate of 57.1% (20/35) with a complete response rate of 34.3% (12/35) in patients with diffuse large B-cell lymphoma (Blood 2017 130(Suppl 1):187, NCT02669017). | detail... | |
| Unknown unknown | colon cancer | not applicable | AMG 900 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AMG 900 inhibited growth of colon cancer cell lines in culture, and inhibited tumor growth in colon cancer cell line xenograft models (PMID: 20935223). | 20935223 | |
| Unknown unknown | colorectal cancer | not applicable | Dbait + Fluorouracil + Oxaliplatin | Preclinical | Actionable | In a preclinical study, Dbait with Eloxitan (oxaliplatin) and Adrucil (5-fluorouracil) resulted in decreased proliferation of human colorectal cancer cell lines in culture (PMID: 26637369). | 26637369 | |
| Unknown unknown | glioblastoma | no benefit | Radiotherapy + Temsirolimus | Phase II | Actionable | In a Phase II trial, the combination of Torisel (temsirolimus) and radiotherapy did not result in an improved overall survival or progression free survival when compared to the combination of Temodar (temozolomide) and radiotherapy in glioblastoma patients with an unmethylated MGMT promoter (PMID: 27143690). | 27143690 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Adavosertib | Preclinical | Actionable | In a preclinical study, Adavosertib (MK-1775) inhibited proliferation in various tumor cell lines, including head and neck squamous cell carcinoma cell lines, in culture (PMID: 23699655). | 23699655 | |
| Unknown unknown | cholangiocarcinoma | not applicable | Silmitasertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the protein kinase CK2 inhibitor, Silmitasertib (CX-4945), inhibited tumor growth and increased survival of cholangiocarcinoma cell line xenograft models (PMID: 30316146). | 30316146 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Acalisib | Phase Ib/II | Actionable | In a Phase Ib trial, Acalisib (GS-9820) treatment resulted in an overall response rate of 53.3% (8/15), with 8 partial responses, stable disease in 6 patients, and a lymph node response in 85.7% (12/14) of patients with chronic lymphocytic leukemia (PMID: 29434192; NCT01705847). | 29434192 | |
| Unknown unknown | ovarian cancer | 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 ovarian cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). | detail... | |
| Unknown unknown | astrocytoma | not applicable | Perifosine | Phase II | Actionable | In a Phase II trial, Perifosine (KRX-0401) was well tolerated, but demonstrated limited activity in patients with anaplastic astrocytoma (n=14), with only 1 patient achieved a partial response (PMID: 31325145). | 31325145 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ch282-5 | Preclinical | Actionable | In a preclinical study, ch282-5 inhibited growth of a variety of human solid tumor cell lines in culture (PMID: 26515494). | 26515494 | |
| 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 | 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 | diffuse large B-cell lymphoma | not applicable | Hu5F9-G4 + Rituximab | Phase Ib/II | Actionable | In a Phase Ib study, combined Hu5F9-G4 and Rituxan (rituximab) therapy demonstrated safety and efficacy, resulting in an objective response rate of 40% (6/15, 5 complete and 1 partial response) and stable disease in 20% (3/15) of patients with diffuse large B-cell lymphoma, and a median duration of response longer than 6 months (PMID: 30380386). | 30380386 | |
| 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 | triple-receptor negative breast cancer | not applicable | Carfilzomib + Tinostamustine | Preclinical - Cell culture | Actionable | In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of triple negative breast cancer cell lines in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | malignant peripheral nerve sheath tumor | not applicable | Alisertib | Phase II | Actionable | In a Phase II trial, Alisertib (MLN8237) treatment resulted in twelve-week progression free survival in 60% (6/10) of malignant peripheral nerve sheath tumor patients (PMID: 27502708). | 27502708 | |
| Unknown unknown | bladder urothelial carcinoma | not applicable | Pembrolizumab | Phase II | Actionable | In a Phase II clinical study (PURE-01), 42% (21/50) of patients with muscle invasive bladder cancer treated with neoadjuvant Keytruda (pembrolizumab) had complete pathological responses at disease resection (PMID: 30343614; NCT02736266). | 30343614 | |
| Unknown unknown | melanoma | not applicable | G-TPP + WEHI-539 | Preclinical - Cell culture | Actionable | In a preclinical study, combination of the mitochondrial Hsp90 inhibitor G-TPP and the Bcl-xL inhibitor WEHI-539 resulted in increased apoptosis of melanoma cell lines in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | malignant mesothelioma | not applicable | Ad-NK4 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Ad-NK4 decreased phosphorylation of Met and Akt, decreased beta-catenin signaling, and inhibited viability and invasiveness of mesothelioma cell lines in culture, and inhibited tumor growth in xenograft models (PMID: 25501304). | 25501304 | |
| 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 | Advanced Solid Tumor | not applicable | AMG208 | Phase I | Actionable | In a Phase I trial, AMG208 demonstrated safety and preliminary efficacy, resulted in complete response in 2% (1/43), partial response in 7% (3/43) and stable disease in 65% (28/43) of patients with advanced solid tumors (PMID: 26155941; NCT00813384). | 26155941 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Carboplatin + Rebastinib | Phase Ib/II | Actionable | In a Phase I/II trial, Rebastinib (DCC-2036) and Paraplatin (carboplatin) combination therapy demonstrated acceptable safety, resulted in a partial response in 4.8% (1/21) and stable disease in 48% (10/21) of patients with advanced solid tumors (Annals of Oncology (2020) 31 (suppl_4): S481-S482; NCT03717415). | detail... | |
| Unknown unknown | prostate cancer | not applicable | Darinaparsin + Docetaxel | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Darinaparsin treatment in combination with Taxotere (docetaxel) inhibited tumor growth in a cell line xenograft model of prostate cancer, however, did not result in an additive or synergistic effect (PMID: 25381261). | 25381261 | |
| Unknown unknown | ovarian cancer | not applicable | Paclitaxel + Vistusertib | Preclinical | Actionable | In a preclinical study, the combination of Vistusertib (AZD2014) and Taxol (paclitaxel) inhibited growth of ovarian cancer cells in culture (AACR; Cancer Res 2012;72(8 Suppl):Abstract nr 931). | detail... | |
| Unknown unknown | colon carcinoma | not applicable | Mps-BAY2a | Preclinical | Actionable | In a preclinical study, Mps-BAY2a inhibited cell cycle progression and induced cell death of colon carcinoma cells in culture (PMID: 23933817). | 23933817 | |
| Unknown unknown | ovarian clear cell carcinoma | no benefit | ENMD-2076 | Phase II | Actionable | In a Phase II trial, ENMD-2076 did not meet efficacy standard, resulted in partial response in 7.9% (3/38) and stable disease in 68.4% (26/38) of patients with recurrent ovarian clear cell carcinoma, with an overall 6-month progression-free survival rate of 22% (PMID: 30108107). | 30108107 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | ME-401 | Phase I | Actionable | In a Phase I trial, ME-401 treatment was well-tolerated, resulted in an objective response rate of 25% (2/8) in patients with relapsed or refractory diffuse large B-cell lymphoma (J Clin Oncol 38: 2020 (suppl; abstr 8016); NCT02914938). | detail... | |
| Unknown unknown | pancreatic adenocarcinoma | not applicable | GSK3368715 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, GSK3368715 inhibited growth of a pancreatic adenocarcinoma cell line in culture, and inhibited tumor growth in xenograft models (PMID: 31257072). | 31257072 | |
| Unknown unknown | small intestine neuroendocrine neoplasm | 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 (5.5 months) in a patient with advanced small bowel neuroendocrine tumor (PMID: 32556884; NCT00892385). | 32556884 | |
| Unknown unknown | esophagus squamous cell carcinoma | not applicable | Imetelstat + Radiotherapy | Preclinical - Cell line xenograft | Actionable | In a preclinical study, imetelstat increased sensitivity of human esophageal squamous cell carcinoma cell lines to radiotherapy, resulting in increased apoptosis and decreased survival in culture, and increased apoptosis, decreased proliferation, and reduced tumor growth in mouse models (PMID: 28099140). | 28099140 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | RU-A1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with RU-A1 in hepatocellular carcinoma cells resulted in decreased cell survival and migration in culture and reduced tumor growth and improved survival in cell line xenograft zebrafish models (PMID: 28589491). | 28589491 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Enzastaurin | Phase III | Actionable | In a Phase III trial (PRELUDE), presence of a germline polymorphism on chromosome 8 (DGM1; Denovo Genomic Marker 1) correlated with improved overall survival following maintenance Enzastaurin (LY317615) therapy (Blood (2018) 132 (Supplement 1): 4207). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | JNJ-26483327 | Phase I | Actionable | In a Phase I clinical trial, JNJ-26483327 was well-tolerated in patients with a range of advanced solid tumors, and demonstrated preliminary anti-tumor activity with 32% (6/19) of patients achieving stable disease for greater than 2 cycles (PMID: 20823884). | 20823884 | |
| 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 | prostate cancer | not applicable | AS605240 | Preclinical | Actionable | In a preclinical study, AS605240 reduced invasiveness of prostate cancer cells in culture (PMID: 24416348). | 24416348 | |
| Unknown unknown | follicular lymphoma | not applicable | Abexinostat | Phase II | Actionable | In a Phase II clinical trial, Abexinostat treatment reduced tumor size in 86% (12/14) of follicular lymphoma patients with an observed response rate of 64.3% (9/14) and median progression-free survival of 20.5 months (PMID: 26482040). | 26482040 | |
| 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 | 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 | lung small cell carcinoma | not applicable | Carboplatin + STA-8666 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, STA-8666 enhanced antitumor activity when combined with Paraplatin (carboplatin), resulting in stabilization of tumor growth and eventual tumor regression in small cell lung cancer cell line xenograft models (PMID: 27267850). | 27267850 | |
| Unknown unknown | myelodysplastic syndrome | not applicable | FF-10501-01 | Phase I | Actionable | In a Phase I trial, FF-10501-01 demonstrated preliminary efficacy, resulting in a partial remission in 10% (2/20) of heavily pretreated patients with myelodysplastic syndrome, however, Phase 2a of the trial was closed due to increased toxicity (PMID: 32264726; NCT02193958). | 32264726 | |
| 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 | diffuse large B-cell 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 a diffuse large B-cell lymphoma cell line xenograft model (PMID: 27856600). | 27856600 | |
| Unknown unknown | colorectal cancer | not applicable | Navitoclax + TAK-901 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of TAK-901 and ABT-263 demonstrated synergy in inhibiting proliferation of colorectal cancer cell lines in culture (PMID: 25667100). | 25667100 | |
| 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 | gastroesophageal junction adenocarcinoma | not applicable | Capecitabine + Nivolumab + Oxaliplatin + Tegafur-gimeracil-oteracil Potassium | Phase II | Actionable | In a Phase II trial (ATTRACTION-4), the combination therapy of Xeloda (capecitabine), Opdivo (nivolumab), Eloxatin (oxaliplatin), and TS-1 (tegafur-gimeracil-oteracil potassium) was well-tolerated and resulted in an objective response rate of 76.5% (13/17), a median progression-free survival of 10.6 months, and a median overall survival that was not yet reached in patients with either gastric cancer or gastroesophageal junction cancer (PMID: 30566590; NCT02746796). | 30566590 | |
| Unknown unknown | ovarian cancer | not applicable | Cediranib + Durvalumab + Olaparib | Phase I | Actionable | In a Phase I trial, the combination of Cediranib (AZD-2171), Imfinzi (durvalumab), and Lynparza (olaparib) treatment demonstrated tolerability and activity in female patients with ovarian, endometrial, or triple-negative breast cancer, with a response rate of 33% (3/9; 2 pts with ovarian cancer, and 1 pt with endometrial cancer), and stable disease in 4/9 pts (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #390P; NCT02484404). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | Bortezomib + MKC-3946 | Preclinical - Patient cell culture | Actionable | In a preclinical study, MKC-3946 treatment in combination with Velcade (bortezomib) enhanced growth inhibition of patient-derived multiple myeloma cells in culture (PMID: 22538852). | 22538852 | |
| 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 | non-Hodgkin lymphoma | not applicable | CUDC-907 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CUDC-907 inhibited cell growth in a human non-Hodgkin lymphoma cell line in culture, and stabilized tumor growth in xenograft models (PMID: 22693356). | 22693356 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | SRA141 | Preclinical - Cell culture | Actionable | In a preclinical study, SRA141 inhibited growth of a variety of tumor cell lines in culture (AACR Annual Meeting 2019, Abstract LB-288). | detail... | |
| Unknown unknown | liver cancer | not applicable | FH535 | Preclinical | Actionable | In a preclinical study, FH535 demonstrated efficacy by inhibiting proliferation of liver cancer stem cells and hepatocellular carcinoma cells in culture (PMID: 24940873). | 24940873 | |
| 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 | 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 | colorectal adenocarcinoma | not applicable | CRLX101 + Fluorouracil + Radiotherapy | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the addition of CRLX101 to Adrucil (fluorouracil) and radiotherapy resulted in a greater decrease in tumor volume in colorectal adenocarcinoma xenograft models when compared to the combination of Adrucil (fluorouracil) and radiotherapy only (PMID: 27784746). | 27784746 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Acalabrutinib + Obinutuzumab | Phase III | Actionable | In a Phase III trial (ELEVATE TN), Calquence (acalabrutinib) and Gazyva (obinutuzumab) combination treatment resulted in prolonged progression-free survival compared to Gazyva (obinutuzumab) plus chlorambucil (not reached vs 22.6 months, HR=0.10, p<0.0001) in patients with treatment-naive chronic lymphocytic leukemia (PMID: 31724010; NCT02475681). | 31724010 | |
| Unknown unknown | multiple myeloma | not applicable | bb2121 | Phase II | Actionable | In a Phase II trial (KarMMa), Idecabtagene vicleucel (bb2121) treatment resulted in an objective response of 73% (94/128) in patients with relapsed or refractory multiple myeloma, with a median duration of response of 10.6 months and a median progression-free survival of 8.6 months (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 8503-8503; NCT03361748). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | bb2121 | Phase I | Actionable | In a Phase I trial, bb2121 treatment in multiple myeloma patients resulted in an overall response rate of 78% (7/9), including two patients who experienced complete remission, four patients who achieved partial response, and one patient who experienced stable disease (EORTC-NCI-AACR Symposium, 2016, Abstract #14). | detail... | |
| Unknown unknown | CLL/SLL | not applicable | Orelabrutinib | Phase II | Actionable | In a Phase II trial, treatment with Orelabrutinib (ICP-022) demonstrated safety and resulted in an objective response rate (ORR) of 88.5% (69/78), including 1 complete response, 39 partial responses, and 29 partial responses with lymphocytosis, and a 6 month duration of response of 89.8% in Chinese patients with relapsed or refractory chronic lymphocytic leukemia/small cell leukemia (Blood (2019) 134 (Supplement_1): 4319). | detail... | |
| 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 | gastrointestinal stromal tumor | not applicable | Cediranib | Phase II | Actionable | In a Phase II trial, treatment with Cediranib (AZD-2171) resulted in stable disease as best response in 55% (11/20) gastrointestinal stromal tumor patients, with 8 patients achieving stable disease for greater than or equal to 16 weeks (PMID: 24714778). | 24714778 | |
| Unknown unknown | colorectal cancer | not applicable | TAK-960 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, colorectal 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 | olfactory neuroblastoma | not applicable | OPB-111077 | Phase I | Actionable | In a Phase I trial, a patient with esthesioneuroblastoma demonstrated stable disease when treated with OPB-111077 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B118). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | LYC-55716 | Case Reports/Case Series | Actionable | In a Phase I trial, Cintirorgon (LYC-55716) treatment resulted in a partial response in a patient with non-small cell lung cancer who was previously treated with Keytruda (pembrolizumab) and had progressed (PMID: 30819679). | 30819679 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BXQ-350 | Phase I | Actionable | In a Phase I trial, BXQ-350 demonstrated safety and preliminary efficacy, resulted in partial response in 6% (1/17) and stable disease in 35% (6/17) of patients with advanced solid tumors (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 2517-2517; NCT02859857). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | Eribulin + Volasertib | Preclinical - Cell culture | Actionable | In a preclinical study, Volasertib (BI 6727) and eribulin synergistically inhibited growth of ovarian cancer cell lines in culture (PMID: 32183025). | 32183025 | |
| 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 | glioblastoma | not applicable | SA16 | Preclinical - Cell culture | Actionable | In a preclinical study, SA16 treatment in glioblastoma cells resulted in decreased cell proliferation, reduced cell viability, and apoptotic induction in culture, and inhibited glioma stem cell formation (PMID: 27797168). | 27797168 | |
| Unknown unknown | glioblastoma | not applicable | Alisertib | Preclinical | Actionable | In a preclinical study, Alisertib (MLN8237) decreased proliferation of primary glioblastoma cell lines in culture, and improved survival of primary glioblastoma cell line xenograft models, including models resistant to Avastin (bevacizumab) (PMID: 27816996). | 27816996 | |
| Unknown unknown | islet cell tumor | not applicable | Temozolomide + TRC102 | Case Reports/Case Series | Actionable | In a Phase I trial, combination therapy with Temodar (temozolomide) and TRC102 (methoxyamine) resulted in a partial response in one patient and prolonged stable disease (9 months) in another patient with advanced pancreatic neuroendocrine tumors (PMID: 32556884; NCT00892385). | 32556884 | |
| Unknown unknown | gastrointestinal stromal tumor | not applicable | Pazopanib | Phase II | Actionable | In a Phase II trial, treatment with Votrient (pazopanib) plus best supportive care (BSC) resulted in improved progression-free survival (45% at 4 months) compared to BSC alone (15% at 4 months) in patients with Gleevec (imatinib) and Sutent (sunitinib)-resistant gastrointestinal stromal tumors (J Clin Oncol 33, 2015 (suppl; abstr 10506)). | 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 | gastrointestinal stromal tumor | not applicable | Regorafenib | FDA approved | Actionable | In a Phase III clinical trial (GRID) that supported FDA approval, Stivarga (regorafenib) demonstrated safety and improved progression free survival compared to placebo (4.8 vs 0.9 months, HR=0.27, p<0.0001) in patients with gastrointestinal stromal tumors (PMID: 23177515; NCT01271712). | detail... 23177515 | |
| Unknown unknown | melanoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase III trial (CheckMate 037) that supported FDA approval, 31.7% (38/120) of patients with advanced melanoma treated with Opdivo (nivolumab) experienced an objective response whereas only 10.6% (5/47) of advanced melanoma patients treated with investigator's choice of therapy demonstrated an objective response (PMID: 25795410; NCT01721746). | 25795410 detail... | |
| Unknown unknown | melanoma | not applicable | Nivolumab | Phase III | Actionable | In an analysis of two Phase III trial, Opdivo (nivolumab) treatment after disease progression demonstrated safety and clinical benefit, with 76% (65/85) of patients still alive at time of analysis in melanoma patients who received the last dose of Opdivo (nivolumab) more than 6 weeks after progression (PMID: 28662232). | 28662232 | |
| Unknown unknown | melanoma | not applicable | Nivolumab | Phase II | Actionable | In a Phase II trial, Opdivo (nivolumab) monotherapy resulted an overall response rate (ORR) of 25% (3/12) and pathologic complete response rate (pCR) of 25% (3/12) in patients with stage III or IV melanoma, compared to a ORR of 73% (8/11) and pCR of 45% (5/11) with the combination of Opdivo (nivolumab) and Yervoy (ipilimumab), but demonstrated lower toxicity than the combination therapy (PMID: 30297909; NCT02519322). | 30297909 | |
| Unknown unknown | melanoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase III trial (CheckMate 238) that supported FDA approval, adjuvant Opdivo (nivolumab) treatment resulted in improved rate of recurrence-free survival at 12-month compared to Yervoy (ipilimumab) (70.5% vs 60.8%, HR=0.65, P<0.001) in patients with resected stage III or IV melanoma (PMID: 28891423, NCT02388906). | detail... 28891423 | |
| Unknown unknown | ovarian cancer | not applicable | Niraparib | Phase I | Actionable | In a Phase I clinical trial, Zejula (niraparib) demonstrated safety and preliminary efficacy, resulted in a durable partial response (PR) in 67% (2/3) of patients with plantinum-sensitive high-grade serous ovarian cancer, PR in 16% (3/19) and stable disease over 120 days in 16% (3/19) of patients with plantinum-resistant high-grade serous ovarian cancer (PMID: 23810788; NCT00749502) | 23810788 | |
| Unknown unknown | melanoma | not applicable | Ipilimumab | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Yervoy (ipilimumab) resulted in an improved overall survival of 10.1 months in patients with metastatic melanoma, compared to 6.4 months in those treated with gp100 peptide vaccine alone (PMID: 20525992, PMID: 21900389). | 20525992 detail... 21900389 | |
| Unknown unknown | melanoma | not applicable | Ipilimumab | Phase III | Actionable | In a Phase III trial, adjuvant Opdivo (nivolumab) treatment resulted in improved rate of recurrence-free survival at 12-months compared to Yervoy (ipilimumab) (70.5% vs 60.8%, HR=0.65, P<0.001) in patients with resected stage III or IV melanoma (PMID: 28891423, NCT02388906). | 28891423 | |
| Unknown unknown | ovarian cancer | not applicable | TRX-E-002-1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TRX-E-002-1 inhibited proliferation and induced apoptosis in chemoresistant human ovarian cancer cell lines in culture, and reduced tumor size in ovarian cancer cell line xenograft models (PMID: 27196760). | 27196760 | |
| 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 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 | lung cancer | not applicable | CBP501 + Cisplatin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of Platinol (cisplatin) and CBP501 resulted in enhanced tumor growth inhibition in cell line xenograft models of lung cancer, compared to either agent alone (PMID: 17237275). | 17237275 | |
| Unknown unknown | B-cell lymphoma | not applicable | Urelumab | Phase I | Actionable | In a Phase I trial, Urelumab (BMS-663513) demonstrated manageable safety profile, resulted in an objective response rate of 17% (2/12) and a disease control rate of 42% (5/12) in patients with relapsed or refractory B-cell lymphomas, with a median progression-free survival of 13.4 months and a median overall survival not reached (PMID: 32052473; NCT01471210). | 32052473 | |
| Unknown unknown | NUT midline carcinoma | not applicable | Molibresib | Phase Ib/II | Actionable | In a Phase I/II trial, Molibresib (GSK525762) treatment resulted in partial response in 20% (2/10) and stable disease in 40% (4/10) of NUT midline carcinoma patients (Cancer Res 2016;76(14 Suppl): Abstract nr CT014). | detail... | |
| Unknown unknown | Hodgkin's lymphoma | not applicable | Everolimus + Vorinostat | Phase I | Actionable | In a Phase I trial, the combination of Afinitor (everolimus) and Zolinza (vorinostat) demonstrated safety and resulted in an objective response rate of 33% (6/18, 2 complete, 4 partial responses) and a median progression-free survival (mPFS) of 4.8 months in patients with relapsed or refractory Hodgkin's lymphoma, with no difference in mPFS between patients who responded to treatment compared to those who did not (5.7 months vs 4.8 months; p=0.9) (PMID: 33055173; NCT01087554). | 33055173 | |
| Unknown unknown | renal cell carcinoma | not applicable | Famitinib | Phase I | Actionable | In a Phase I trial, renal carcinoma patients treated with Famitinib demonstrated a disease control rate of 87.5%, which included 50% (12/24) with a partial response and 37.5% (9/24) with stable disease, and a PFS of 10.7 mo and an OS of 33 mo (PMID: 24238512). | 24238512 | |
| Unknown unknown | stomach cancer | not applicable | Cisplatin + Deguelin | Preclinical | Actionable | In a preclinical study, the combination of Deguelin and Platinol (cisplatin) worked synergistically to inhibit growth of gastric cancer cells in culture (PMID: 25202376). | 25202376 | |
| 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 | not applicable | AZD1208 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AZD1208 inhibited tumor growth in castrate-resistant prostate cancer cell line xenograft models (PMID: 25505253). | 25505253 | |
| Unknown unknown | prostate cancer | not applicable | AZD1208 | Case Reports/Case Series | Actionable | In a Phase I trial, AZD1208 treatment resulted in considerable decrease of PSA levels in a patient with prostate cancer (PMID: 29765150; NCT01588548). | 29765150 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Riluzole | Preclinical - Cell culture | Actionable | In a preclinical study, Rilutek (riluzole) inhibited growth of hepatocellular cancer cell lines and primary hepatocellular cancer lines in culture (PMID: 27612558). | 27612558 | |
| Unknown unknown | acute lymphoblastic leukemia | not applicable | Blinatumomab | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Blincyto (blinatumomab) treatment resulted in longer overall survival (7.7 vs 4.0 months), higher remission rate (34% vs 16%), and higher rate of event-free survival (31% vs 12%) compared to chemotherapy in patients with relapsed or refractory B-cell precursor acute lymphocytic leukemia (PMID: 28249141; NCT02013167). | detail... 28249141 | |
| Unknown unknown | pancreatic endocrine carcinoma | not applicable | Cabozantinib | Preclinical | Actionable | In a preclinical study, Cometriq (cabozantinib) inhibited pancreatic neuroendocrine tumor growth and invasion in transgenic mouse models (PMID: 22585997). | 22585997 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | Emactuzumab + Paclitaxel | Phase I | Actionable | In a Phase I trial, Emactuzumab (RG7155) plus Taxol (paclitaxel) demonstrated safety and resulted in reduced tumor-associated M2-like macrophage levels in advanced solid tumor patients, but demonstrated marginal clinical antitumor activity, resulting in an objective response rate of 7.4% (4/54), with partial responses in three ESR1-positive, ERBB2 (HER2)-negative breast cancer patients and one ovarian cancer patient, and stable disease in a further 42.5% (23/54) of patients (PMID: 31114846; NCT01494688). | 31114846 | |
| Unknown unknown | chronic myeloid leukemia | not applicable | Bosutinib | Clinical Study | Actionable | In a meta-analysis, Bosulif (bosutinib) treatment was associated with increased rate of major molecular response compared with Gleevec (imatinib) (Odds Ratio (OR): 1.86 [1.29-2.70]), but not improved overall survival (OR: 2.38 [0.82-6.89]), and was associated with a trend toward increased risk of vascular occlusive events in chronic myeloid leukemia patients (PMID: 26847662). | 26847662 | |
| Unknown unknown | cholangiocarcinoma | not applicable | Cabozantinib | Phase II | Actionable | In a Phase II trial, Cometriq (cabozantinib) treatment resulted in a median progression free survival of 1.8 months, and a median overall survival of 5.2 months in patients with advanced cholangiocarcinoma, but also induced grade 3/4 adverse events in 89% (17/19) of the patients (PMID: 28192597). | 28192597 | |
| Unknown unknown | glioblastoma | not applicable | Bortezomib + Panobinostat | Preclinical | Actionable | In a preclinical study, glioblastoma cells treated with a combination of Farydak (panobinostat) and Velcade (bortezomib) resulted in a synergistic effect, demonstrating decreased cell viability in culture (PMID: 26804704). | 26804704 | |
| Unknown unknown | breast cancer | not applicable | Elenagen + Tamoxifen | Phase Ib/II | Actionable | In a Phase Ib/II trial, a chemorefractory patient with breast cancer demonstrated restored chemotherapeutic sensitivity upon sequential treatment of Elenagen and Nolvadex (tamoxifen), which resulted in stable disease (PMID: 28881846). | 28881846 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | PRI-724 | Phase I | Actionable | In a Phase I trial, Pri-724 displayed safety and preliminary efficacy in patients with a variety of advanced solid tumors (J Clin Oncol 31, 2013 (suppl; abstr 2501)). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | no benefit | Cyclophosphamide + Doxorubicin + Obinutuzumab + Prednisone + Vincristine Sulfate | Phase III | Actionable | In a Phase III trial, the combination treatment of Gazyva (obinutuzumab) with CHOP did not result in an improved 3-year progression free survival rate (70% vs 67%) compared to Rituxan (rituximab) combined with CHOP in patients with diffuse large B-cell lymphoma (PMID: 28796588). | 28796588 | |
| Unknown unknown | gastroesophageal cancer | not applicable | Capecitabine + Oxaliplatin + Sapitinib | Phase I | Actionable | In a Phase I trial, the addition of Sapitinib (AZD-8931) with chemotherapy, Eloxatin (oxaliplatin), and Xeloda (capecitabine), versus chemotherapy alone in patients with esophagogastric cancer resulted in a six month progression-free survival of 85% (N=20) and 100% (N=10), and an overall survival of 80% and 100% at 12 months and 64% and 90% at 24 months, respectively (PMID: 31765988). | 31765988 | |
| Unknown unknown | acute myeloid leukemia | not applicable | A-485 | Preclinical - Cell culture | Actionable | In a preclinical study, A-485 treatment inhibited proliferation in acute myeloid leukemia cell lines in culture (PMID: 28953875). | 28953875 | |
| 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). |