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Relevant Treatment Approaches

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown prostate cancer not applicable PX-866 Phase II Actionable In a Phase II trial, Sonolisib (PX-866) treatment was tolerated, resulted in a partial response in 11% (2/18) and stable disease in 56% (10/18) of evaluable patients with recurrent or metastatic castration-resistant prostate cancer, PSA response was observed in 2% (1/43) of evaluable patients, and 32.6% (14/43) of all patients were progression-free at 12 weeks (PMID: 31056399). 31056399
Unknown unknown colon cancer not applicable BOS172722 Preclinical - Cell culture Actionable In a preclinical study, BOS172722 treatment resulted in increased apoptosis and decreased growth of a colon cancer cell line in culture (PMID: 31575759). 31575759
Unknown unknown uveal melanoma not applicable AU-011 Preclinical - Cell culture Actionable In a preclinical study, treatment with AU-011 resulted in cell binding and subsequent cell death in uveal melanoma cells in culture (Cancer Res 2014;74(19 Suppl):Abstract nr 1770). detail...
Unknown unknown lung non-small cell carcinoma not applicable Cabozantinib Phase Ib/II Actionable In a Phase Ib/II trial, Cometriq (cabozantinib) treatment resulted in partial response in 6.7% (1/15) of patients with non-small cell lung carcinoma that had progressed during treatment with Tarceva (erlotinib), with a median progression free survival of 1.9 months (PMID: 28352985). 28352985
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 lung non-small cell carcinoma not applicable Gemcitabine + SRA737 Preclinical - Cell line xenograft Actionable In a preclinical study, a non-small cell lung carcinoma cell line demonstrated sensitivity to the combination of SRA737 (CCT245737) and Gemzar (gemcitabine), resulting in antitumor efficacy in both culture and xenograft models (PMID: 27167172). 27167172
Unknown unknown 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 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 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 gastroesophageal junction adenocarcinoma 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 colorectal cancer not applicable Danusertib Phase II Actionable In a Phase II clinical trial, Danusertib (PHA-739358) showed limited efficacy in patients with metastatic colorectal cancer (J Clin Oncol 28, 2010 (suppl; abstr e13558)). detail...
Unknown unknown 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 squamous cell carcinoma not applicable PMX-53 Preclinical Actionable In a preclinical study, PMX-53 treatment did not inhibit tumor growth in a syngeneic mouse model of poorly differentiated squamous cell carcinoma (PMID: 30300579). 30300579
Unknown unknown stomach cancer not applicable Nivolumab Phase Ib/II Actionable In a Phase I/II trial, Opdivo (nivolumab), alone or incombination with Yervoy (ipilimumab), demonstrated safety and efficacy in patients with chemotherapy-refractory gastric cancer, resulted in a disease control rate of 38% (61/160) (J Clin Oncol 34, 2016 (suppl; abstr 4010)). detail...
Unknown unknown uterine carcinosarcoma not applicable Navicixizumab Case Reports/Case Series Actionable In a Phase I trial, Navicixizumab (OMP-305B83) treatment resulted in partial response in a patient with uterine carcinosarcoma (PMID: 30229512). 30229512
Unknown unknown colorectal cancer not applicable Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) showed anti-tumor activity in human colorectal cancer explants (PMID: 25242168). 25242168
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 lung small cell carcinoma not applicable Carboplatin + Durvalumab + Etoposide FDA approved Actionable In a Phase III (CASPIAN) trial that supported FDA approval, Imfinzi (durvalumab) in combination with Vepesid (etoposide) and Paraplatin (carboplatin) or Platinol (cisplatin) resulted in significantly improved overall survival (13.0 vs 10.3 mo, HR=0.73, p=0.0047) compared to platinum-etoposide therapy in patients with untreated extensive-stage small cell lung cancer (PMID: 31590988; NCT03043872). 31590988
Unknown unknown breast cancer not applicable VS-5584 Preclinical - Patient cell culture Actionable In a preclinical study, treatment with VS-5584 resulted in decreased cancer stem cell (CSC) number in a triple-negative breast cancer cell line in culture and in xenograft models, and decreased CSCs in patient breast cancer tumor samples in culture (PMID: 25432176). 25432176
Unknown unknown 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 colon carcinoma not applicable Oncolytic HSV-1 rRp450 Preclinical - Cell culture Actionable In a preclinical study, Oncolytic HSV-1 rRp450 treatment induced cytotoxicity in human and mouse colon carcinoma cell lines in culture, and reduced colon carcinoma liver metastases in a mouse model (PMID: 12209705). 12209705
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 prostate cancer not applicable OPB-111077 Phase I Actionable In a Phase I trial, a patient with prostate cancer 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 SH-1242 Preclinical - Pdx & cell culture Actionable In a preclinical study, SH-1242 inhibited growth of several non-small cell lung cancer (NSCLC) cell lines in culture, including cell lines with acquired drug resistance, and inhibited tumor growth in cell line and patient-derived xenograft models (PMID: 26645561). 26645561
Unknown unknown triple-receptor negative breast cancer not applicable YLL545 Preclinical - Cell line xenograft Actionable In a preclinical study, YLL545 inhibited proliferation of a human triple-negative breast cancer (TNBC) cell line in culture, and inhibited activation of STAT3 and ERK and decreased tumor growth in TNBC cell line xenograft models (PMID: 27203384). 27203384
Unknown unknown Ewing sarcoma 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 Ewing sarcoma (PMID: 30724423). 30724423
Unknown unknown pancreatic ductal adenocarcinoma not applicable Arsenic trioxide + JQ1 Preclinical - Cell line xenograft Actionable In a preclinical study, JQ1 and Trisenox (arsenic trioxide) combination treatment reduced viability of Trisenox (arsenic trioxide)-insensitive pancreatic ductal adenocarcinoma cell lines in culture, and synergistically inhibited tumor growth in a cell line xenograft model (PMID: 31420604). 31420604
Unknown unknown CLL/SLL not applicable Ibrutinib FDA approved Actionable In a Phase III trial (RESONATE) that supported FDA approval, treatment with Imbruvica (ibrutinib) in chronic lymphoid leukemia (CLL) or small lymphocytic lymphoma (SLL) patients resulted in improved progression-free survival compared to treatment with Arzerra (ofatumumab) (median duration not reached vs. 8.1 months), and an overall response rate of 43% (83/195) versus 4% (8/196) with Arzerra (ofatumumab) (PMID: 24881631; NCT01578707). detail... 24881631
Unknown unknown CLL/SLL not applicable Ibrutinib FDA approved Actionable In a Phase III trial (RESONATE-2) that supported FDA approval, Imbruvica (ibrutinib) treatment resulted in a greater median progression-free survival (not reached vs 18.9 months, HR=0.16, p<0.001) and response rate (86% vs 35%, p<0.001) when compared to chlorambucil treatment in patients with chronic lymphoid leukemia (CLL) or small lymphocytic lymphoma (SLL) aged 65 or older (PMID: 26639149; NCT01722487). detail... 26639149
Unknown unknown CLL/SLL not applicable Ibrutinib FDA approved Actionable In a Phase III trial (RESONATE) that supported FDA approval, treatment with Imbruvica (ibrutinib) in chronic lymphoid leukemia (CLL) or small lymphocytic lymphoma (SLL) patients with del 17p resulted in improved progression-free survival compared to Arzerra (ofatumumab) (40.6 vs. 8.1 months), and improved overall response rate (47.6%, 30/63 vs 4.7%, 3/64) (PMID: 31512258; NCT01578707). 31512258 detail...
Unknown unknown NUT midline carcinoma not applicable Birabresib Preclinical - Cell line xenograft Actionable In a preclinical study, Birabresib (OTX015) inhibited tumor growth in cell line xenograft models of NUT midline carcinoma (Mol Cancer Ther November 2013 12; C244). detail...
Unknown unknown NUT midline carcinoma not applicable Birabresib Phase Ib/II Actionable In a Phase Ib trial, Birabresib (OTX015) demonstrated favorable safety and resulted in partial response in 33% (3/9) and stable disease in 33% (3/9) patients with NUT midline carcinoma (PMID: 29733771; NCT02259114). 29733771
Unknown unknown neuroblastoma not applicable Verteporfin Preclinical - Cell line xenograft Actionable In a preclinical study, verteporfin inhibited growth and metastasis of neuroblastoma cell lines in culture, and pretreatment of a metastatic subpopulation of a neuroblastoma cell line with verteporfin decreased metastasis in xenograft models (PMID: 27899382). 27899382
Unknown unknown chronic 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 breast cancer not applicable PI-273 Preclinical - Cell line xenograft Actionable In a preclinical study, PI-273 induced cell-cycle arrest and apoptosis and decreased proliferation in breast cancer cell lines with wild-type RAS in culture, and inhibited tumor growth in a xenograft model (PMID: 28827373). 28827373
Unknown unknown 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 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 neuroendocrine carcinoma not applicable DS6051b Phase I Actionable In a Phase I clinical trial, treatment with DS-6051b was well-tolerated in patients with advanced solid tumors, and resulted in partial responses in two patients, including a patient with neuroendocrine carcinoma (Cancer Res July 15 2016 (76) (14 Supplement) CT024). detail...
Unknown unknown Advanced Solid Tumor not applicable Litronesib Phase I Actionable In an analysis of two Phase I trial, litronesib (LY2523355) treatmetn resulted in partial response in 2.3% (2/86) and stable disease in 20% (17/86) of patients with advanced cancer (PMID: 28097385). 28097385
Unknown unknown lung carcinoma not applicable SF2523 Preclinical Actionable In a preclinical study, SF2523 decreased tumor immunosuppression, increased T-lymphocyte infiltration, and reduced tumor growth in syngeneic mouse lung carcinoma models (PMID: 31018997). 31018997
Unknown unknown lung non-small cell carcinoma not applicable Antroquinonol Phase I Actionable In a Phase I trial, antroquinonol (Hocena) demonstrated safety and preliminary efficacy in metastatic NSCLC patients previously treated with two prior chemotherapy regimens (PMID: 26807250). 26807250
Unknown unknown pancreatic cancer not applicable Conatumumab + Ganitumab Phase Ib/II Actionable In a Phase Ib/II clinical trial, Ganitumab and Conatumumab combination treatment resulted in stable disease in one patient out of sixteen with pancreatic cancer (PMID: 24816908). 24816908
Unknown unknown neuroblastoma not applicable Ellipticine + Valproic acid Preclinical Actionable In a preclincal study, Depakene (valproic acid) enhanced the cytotoxicity of ellipticine in human neuroblastoma cell lines (PMID: 22167207). 22167207
Unknown unknown transitional cell carcinoma not applicable Docetaxel + Ramucirumab Phase II Actionable In a Phase II trial, Cyramza (ramucirumab) and Taxotere (docetaxel) combination treatment resulted in improved median progression-free survival (5.4 months) when compared to Taxotere (docetaxel) single treatment (2.8 months) in urothelial carcinoma patients (PMID: 26926681). 26926681
Unknown unknown transitional cell carcinoma not applicable Docetaxel + Ramucirumab Phase III Actionable In a Phase III trial, Cyramza (ramucirumab) and Taxotere (docetaxel) combination treatment resulted in improved median progression-free survival (4.1 vs 2.8 months) and objective response rate (24.5% vs 14.0%) when compared to Taxotere (docetaxel) single treatment in urothelial carcinoma patients (Annals of Oncology (2017) 28 (suppl_5): v605-v649; NCT02426125). detail...
Unknown unknown colon cancer not applicable Enzastaurin Preclinical - Cell line xenograft Actionable In a preclinical study, Enzastaurin (LY317615) demonstrated efficacy by suppressing proliferation of cultured cells and growth in cell line xenograft models of colon cancer (PMID: 16103100). 16103100
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 lung non-small cell carcinoma not applicable Everolimus Phase Ib/II Actionable In a Phase Ib trial, Afinitor (everolimus) demonstrated safety and preliminary efficacy in patients with non-small cell lung cancer (PMID: 25673697). 25673697
Unknown unknown multiple myeloma not applicable Bortezomib + Mivebresib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Velcade (bortezomib) and Mivebresib (ABBV-075) resulted in increased tumor growth inhibition in a multiple myeloma cell line xenograft model compared to Velcade (bortezomib) alone (PMID: 28416490). 28416490
Unknown unknown ovarian cancer not applicable AZD7648 + Olaparib Preclinical - Cell culture Actionable In a preclinical study, AZD7648 and Lynparza (olaparib) combination treatment inhibited viability of ovarian cancer cell lines harboring wild-type ATM in culture (PMID: 31699977). 31699977
Unknown unknown urinary bladder cancer not applicable CG0070 Phase II Actionable In a Phase II trial, treatment with CG0070 resulted in a complete response rate of 47% (21/45) in patients with BGC-unresponsive high grade non-muscle invasive bladder cancer (PMID: 28755959; NCT02365818). 28755959
Unknown unknown lung cancer not applicable BOS172722 Preclinical - Cell culture Actionable In a preclinical study, lung cancer cell lines demonstrated sensitivity to growth inhibition by BOS172722 in culture (PMID: 31575759). 31575759
Unknown unknown diffuse large B-cell lymphoma not applicable Bendamustine + Polatuzumab vedotin-piiq + Rituximab FDA approved Actionable In a Phase Ib/II (GO29365) trial that supported FDA approval, addition of Polivy (polatuzumab vedotin-piiq) to Treanda (bendamustine) and Rituxan (rituximab) combination (BR) therapy resulted in improved complete response rate (40% vs 18%) and best overall response rate (63% vs 25%) compared to BR therapy alone in patients with relapsed or refractory diffuse large B-cell lymphoma (Blood 2017 130:2821; NCT02257567). detail... detail...
Unknown unknown Advanced Solid Tumor not applicable MLN4924 Phase I Actionable In a Phase I trial, Pevonedistat (MLN4924) treatment resulted in stable disease in 74% (23/31) of patients with advanced solid tumors (PMID: 26423795). 26423795
Unknown unknown transitional cell carcinoma not applicable Ascrinvacumab Phase II Actionable In a Phase II trial, PF-03446962 demonstrated multiple toxicities and limited efficacy in patients with urothelial cancer (PMID: 24566706). 24566706
Unknown unknown Advanced Solid Tumor not applicable OSI-027 Phase I Actionable In a Phase I trial, OSI-027 treatment resulted in no RECIST response and stable disease in 5% (6/128) of patients with advanced solid tumors (PMID: 27002938). 27002938
Unknown unknown colon cancer not applicable Chiauranib Preclinical - Cell line xenograft Actionable In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a colon cancer cell line xenograft model (PMID: 28004478). 28004478
Unknown unknown hepatocellular carcinoma not applicable Orlistat + Paclitaxel Preclinical - Cell culture Actionable In a preclinical study, Xenical (orlistat) and Taxol (paclitaxel) synergistically inhibited growth and induced apoptosis in hepatocellular carcinoma cells in culture (PMID: 30667213). 30667213
Unknown unknown malignant fibrous histiocytoma not applicable EHop-016 Preclinical - Cell line xenograft Actionable In a preclinical study, EHop-016 inhibited AKT activation and increased apoptosis and reduced growth of a myxofibrosarcoma cell line in culture, and inhibited tumor growth in xenograft models (PMID: 27577794). 27577794
Unknown unknown triple-receptor negative breast cancer not applicable Irinotecan + SR-4835 Preclinical - Pdx Actionable In a preclinical study, SR-4835 and Camptosar (irinotecan) combination treatment induced DNA damage and cell death, inhibited tumor growth, leading to tumor regression in a patient-derived xenograft (PDX) model of triple-negative breast cancer harboring a BRCA1 mutation (PMID: 31668947). 31668947
Unknown unknown Ewing sarcoma not applicable Niraparib + SN-38 Preclinical - Cell culture Actionable In a preclinical study, treatment with SN-38 combined with Zejula (niraparib) demonstrated synergism in Ewing sarcoma cells in culture, resulting in reduced cell viability (PMID: 26438158). 26438158
Unknown unknown multiple myeloma not applicable Bortezomib + Daratumumab + Dexamethasone + Thalidomide FDA approved Actionable In a Phase III trial (CASSIOPEIA) that supported FDA approval, Darzalex (daratumumab) in combination with Velcade (bortezomib), thalidomide, and dexamethasone (VTd) resulted in improved stringent complete response at 100 days post autologous stem-cell transplantation compared to VTd (29%, 157/543 vs 20%, 110/542, OR=1.60, p=0.0010) in patients with newly diagnosed multiple myeloma (PMID: 31171419; NCT02541383). detail... 31171419
Unknown unknown diffuse large B-cell lymphoma not applicable AT-7867 Preclinical - Cell culture Actionable In a preclinical study, AT-7867 inhibited the growth of diffuse large B-cell lymphoma cell lines in culture (PMID: 26824321). 26824321
Unknown unknown Her2-receptor negative breast cancer not applicable Paclitaxel + Vantictumab Phase Ib/II Actionable In a Phase Ib trial, the combination of Vantictumab (OMP-18R5) and Taxol (paclitaxel) was well-tolerated and demonstrated preliminary efficacy in patients with metastatic HER2-negative breast cancer (J Clin Oncol 34, 2016 (suppl; abstr 2516)). detail...
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 breast cancer not applicable CYH33 Preclinical - Cell culture Actionable In a preclinical study, CYH33 inhibited proliferation of 56% (18/32) of breast cancer cell lines in culture and demonstrated increased activity compared to Piqray (Alpelisib) (PMID: 30003928). 30003928
Unknown unknown acute lymphoblastic leukemia not applicable OBI-3424 Preclinical - Pdx Actionable In a preclinical study, OBI-3424 treatment resulted in objective response in 89% (8/9, 2 complete response) of patient-derived xenograft (PDX) models of T-Cell acute lymphoblastic leukemia (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr LB-B16). detail...
Unknown unknown Advanced Solid Tumor not applicable Ganetespib Preclinical - Cell line xenograft Actionable In a preclinical study, Ganetespib demonstrated potent anti-tumor effects against a variety of advanced solid tumor cell types and in cell line xenograft models (PMID: 22144665). 22144665
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 Advanced Solid Tumor not applicable CFI-401870 Preclinical Actionable In a preclinical study, CFI-401870 inhibited tumor growth in advanced solid tumor xenograft models (PMID: 25763473). 25763473
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 resulted in 7 patients with a partial response, one patient with a complete response, and 6 patients experiencing stable disease for greater than 6 months (J Clin Oncol 35, 2017 (suppl; abstr 3013)). detail...
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 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 pancreatic cancer not applicable E7449 Phase I Actionable In a Phase I trial, E7449 treatment inhibited Parp activity in peripheral blood mononuclear cells and resulted in stable disease for more than 24 weeks in 7 patients with pancreatic cancer (J Clin Oncol 36, 2018 (suppl; abstr 2505); abstr e19531; NCT01618136). detail...
Unknown unknown renal cell carcinoma not applicable Gemcitabine + MU380 Preclinical - Cell culture Actionable In a preclinical study, the addition of MU380 resulted in increased sensitivity to Gemzar (gemcitabine) in a renal cell carcinoma cell line in culture, leading to decreased proliferation (PMID: 28619751). 28619751
Unknown unknown breast cancer not applicable AB61 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the synthetic nucleoside AB61 resulted in repressed protein translation, decreased tumor volume, and prolonged survival in breast cancer cell line xenograft models (PMID: 26819331). 26819331
Unknown unknown marginal zone 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 100% (4/4) in patients with relapsed or refractory marginal zone B-cell lymphoma (J Clin Oncol 38: 2020 (suppl; abstr 8016); NCT02914938). 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 Advanced Solid Tumor not applicable DS6051b Phase I Actionable In a Phase I clinical trial, DS-6051b was well-tolerated and demonstrated some preliminary efficacy in patients with advanced solid tumors (Cancer Res July 15 2016 (76) (14 Supplement) CT024). detail...
Unknown unknown lung carcinoma not applicable INCB001158 Preclinical Actionable In a preclinical study, INCB001158 (CB-1158) had no effect on Lewis Lung carcinoma cells growth in culture, but inhibited tumor growth in syngeneic animal models through regulation of host immune response (Cancer Res 2016;76(14 Suppl):Abstract nr 552). detail...
Unknown unknown prostate cancer not applicable Durvalumab + Olaparib Phase II Actionable In a Phase II trial, Imfinzi (durvalumab) plus Lynparza (olaparib) resulted in a decrease in PSA greater than or equal to 50% in 53% (9/17) of patients with metastatic castrate-resistant prostate cancer, with radiographic response in 4 of those 9, a median radiographic progression-free survival (PFS) of 16.1 mo., and a 12-mo. PFS probability of 83.3% in patients with mutations in DNA damage response (DDR) genes, compared to 36.4% in patients without DDR gene mutations (PMID: 30514390; NCT02484404). 30514390
Unknown unknown Advanced Solid Tumor not applicable GC1118 Phase I Actionable In a Phase I clinical trial, treatment with GC1118 was well-tolerated, and resulted in partial response in 13% (3/24) and stable disease in 50% (12/24) of patients with advanced solid tumors (PMID: 31164456; NCT02352571). 31164456
Unknown unknown melanoma not applicable Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in partial response in 5% (4/77) and sttable disease in 39% (30/77) of patients with metastatic melanoma, with a median overall progression free survival of 3.8 months (PMID: 28103611). 28103611
Unknown unknown ovarian cancer not applicable AZD7648 + Pegylated liposomal-doxorubicin Preclinical - Cell culture Actionable In a preclinical study, AZD7648 and Doxil (pegylated liposomal-doxorubicin) combination treatment induced DNA damage and synergistically inhibited viability of ovarian cancer cell lines in culture (PMID: 31699977). 31699977
Unknown unknown Advanced Solid Tumor not applicable BAY 1238097 Clinical Study Actionable In a Phase I trial, BAY 1238097 therapy resulted in zero objective responses and stable disease in 25% (2/8) of patients with refractory advanced solid tumors; due to high toxicity the trial was terminated prematurely (PMID: 30711772; NCT02369029). 30711772
Unknown unknown colon carcinoma not applicable Mps-BAY2b + Paclitaxel Preclinical Actionable In a preclinical study, Mps-BAY2b, in combination with paclitaxel, had increased efficacy in inhibiting cell proliferation of colon carcinoma cell in culture (PMID: 23933817). 23933817
Unknown unknown renal cell carcinoma not applicable Axitinib Phase II Actionable In a Phase II clinical trial, treatment with Inlyta (axtinib) as first-line therapy resulted a prolonged median overall survival of 42.7 months compared to 30.4 months with placebo in patients with metastatic renal cell carcinoma (PMID: 27236772). 27236772
Unknown unknown renal cell carcinoma not applicable Axitinib Clinical Study Actionable In a meta-analysis, Inlyta (axitinib) improved progression-free survival in patients with metastatic renal cell carcinoma (PMID: 24037486). 24037486
Unknown unknown renal cell carcinoma not applicable Axitinib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Inlyta (axitinib) as second-line therapy resulted in an improved progression-free survival of 8.3 months compared to 5.7 months with Nexavar (sorafenib) (HR 0.656, 95% CI 0.552-0.779; p<0.0001) in patients with metastatic renal cell carcinoma (PMID: 23598172). 23598172 detail...
Unknown unknown multiple myeloma not applicable Bendamustine + Veliparib Phase I Actionable In a Phase I trial, a patient with multiple myeloma demonstrated a partial response when treated with a combination of Bendamustine and Veliparib (ABT-888) (PMID: 28314788; NCT01326702). 28314788
Unknown unknown melanoma not applicable SKI-G-801 Preclinical Actionable In a preclinical study, SKI-G-801 treatment reduced tumor burden in a metastatic mouse model of melanoma (Cancer Res 2019;79(13 Suppl):Abstract nr 2010). detail...
Unknown unknown renal 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 70% (21/30) in patients with metastatic renal cell carcinoma, with a median duration of response of 20.0 months, and a median progression-free survival of 19.8 months (PMID: 31961766; NCT02501096). 31961766
Unknown unknown colorectal adenocarcinoma not applicable Berzosertib + Irinotecan Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of VX-970 and Camptosar (irinotecan) synergized to inhibit proliferation of a colorectal adenocarcinoma cell line in culture and to inhibit tumor growth in a human colorectal adenocarcinoma cell line xenograft model (PMID: 25269479). 25269479
Unknown unknown Ewing sarcoma not applicable Temozolomide + Veliparib Preclinical Actionable In a preclinical study, Ewing sarcoma cells treated with Temodar (temozolomide) combined with Veliparib (ABT-888) resulted in strong synergism, demonstrating reduced cell viability in culture (PMID: 26438158). 26438158
Unknown unknown stomach cancer not applicable Geldanamycin Preclinical Actionable In a preclinical study, Geldanamycin inhibited proliferation of a human gastric cancer cell line in culture (PMID: 26788199). 26788199
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 breast cancer no benefit Imatinib Phase II Actionable In a Phase II trial, Gleevec (imatinib mesylate) treatment demonstrated significant toxicity and no clinical benefit in patients with heavily pre-treated metastatic breast cancer, of the 13 tested patients, one was positive for Kit and 4 were positive for Pdgfr (PMID: 15803362). 15803362
Unknown unknown 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 chronic myeloid leukemia not applicable CP-870,893 Preclinical - Cell line xenograft Actionable In a preclinical study, CP-870,893 treatment inhibited tumor growth when co-injected with human T-lymphocytes and mouse dendritic cells in a cell line xenograft model of chronic myeloid leukemia (PMID: 21479995). 21479995
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 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 esophageal carcinoma not applicable BI 853520 Phase I Actionable In a Phase I trial, BI 853520 demonstrated safety and some anti-tumor efficacy, resulting in stable disease in 12.5% (2/16) of patients with esophageal carcinoma (PMID: 30756308; NCT01335269). 30756308
Unknown unknown gastric adenocarcinoma no benefit Panitumumab Phase III Actionable In a Phase III trial, addition of Vectibix (panitumumab) to chemotherapy consisted of epirubicin, oxaliplatin, and capecitabine (EOC) did not improve overall survival and increased side effects, therefore was not recommended in an unselected population of patients with advanced oesophagogastric adenocarcinoma (PMID: 23594787). 23594787
Unknown unknown colorectal cancer not applicable trifluridine/tipiracil hydrochloride FDA approved Actionable In a Phase III trial supporting FDA approval, Lonsurf (trifluridine/tipiracil hydrochloride) demonstrated improved median overall survival (7.1 vs 5.3 months), median progression free survival (2.0 vs 1.7 months), and 32% reduction of death risk compared to placebo in treatment-refractory metastatic colorectal cancer patients (PMID: 27126991). 27126991 detail...
Unknown unknown acute myeloid leukemia not applicable Combretastatin A1 Diphosphate Phase I Actionable In a Phase I study, OXi4503 demonstrated safety and preliminary efficacy in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS), resulting in one complete response and one partial response among 14 patients, with four patients demonstrating stable disease (PMID: 32236943). 32236943
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 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 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 lung small cell carcinoma not applicable Cisplatin + Etoposide + Tislelizumab Phase II Actionable In a Phase II trial, treatment with Tislelizumab (BGB-A317) plus platinum doublet chemotherapy (Vepesid (etoposide) with Platinol (cisplatin) or Paraplatin (carboplatin)) in patients with small cell lung cancer resulted in an objective response rate of 77% (13/17) and disease control rate of 88% (15/17), including a partial response in 13 patients and stable disease in two patients, and median progression-free survival of 6.9 months (PMID: 32769013; NCT03432598). 32769013
Unknown unknown 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 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 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 stomach cancer not applicable Lenvatinib + Pembrolizumab Phase II Actionable In a Phase II trial (EPOC1706), the combination of Lenvima (lenvatinib) and Keytruda (pembrolizumab) treatment in patients with either stomach cancer or gastroesophageal junction cancer resulted in a 69% (20/29) objective response rate, a disease control rate of 100% (29/29), a median progression-free survival of 7.1 months, and a median overall survival that had not yet been reached, and 8 patients were still experiencing an ongoing response at data cut-off (PMID: 32589866; NCT03609359). 32589866
Unknown unknown rhabdomyosarcoma not applicable Prexasertib Preclinical - Cell culture Actionable In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including rhabdomyosarcoma cell lines (PMID: 28270495). 28270495
Unknown unknown Hodgkin's lymphoma not applicable Brentuximab vedotin + Umbralisib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of TGR-1202 and Brentuximab vedotin synergized to inhibit proliferation of several human Hodgkin's lymphoma cell lines in culture and to suppress tumor growth in xenograft models (Blood 124(21): 4486). detail...
Unknown unknown glioblastoma multiforme 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 thymoma no benefit Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 28.6% (2/7) and stable disease in 71.6% (5/7) of patients with refractory or relapsed thymoma, with a median progression-free survival of 6.1 months, however, grade 3 or higher immune-related adverse events were seen in 71.4% (5/7) of the patients (PMID: 29906252). 29906252
Unknown unknown Advanced Solid Tumor not applicable Temozolomide + TRC102 Phase I Actionable In a Phase I trial, the combination of Temodar (temozolomide) and TRC102 demonstrated safety and preliminary activity in a patients with advanced solid tumors, with 4/37 patients demonstrating partial response and 11/37 patients achieving stable disease (J Clin Oncol 34, 2016 (suppl; abstr 2556)). detail...
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 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 acute promyelocytic leukemia not applicable Tamibarotene Phase III Actionable In a Phase III trial (JALSG-APL204), treatment with Tamibarotene (SY-1425) resulted in a greater relapse-free survival (RFS) when compared to treatment with Tretinoin, demonstrating a 7-year RFS of 93% (n=134) versus 84% (n=135), and a 7-year RFS of 89% (n=26) vs 62% (n=26) in high risk patients, while overall survival after randomization did not differ between the two treatment groups after 7 years, 97% versus 96%, respectively (PMID: 30093681). 30093681
Unknown unknown glioblastoma multiforme not applicable DCVax-L Phase III Actionable In a Phase III trial, addition of DCVax-L to standard therapy resulted in a median overall survival (mOS) of 23.1 months in the intended to treat group (n=331) of patients with newly diagnosed glioblastoma, with a mOS of 34.7 months in patients with methylated MGMT (n?=?131) (PMID: 29843811; NCT00045968). 29843811
Unknown unknown Advanced Solid Tumor not applicable JNJ-63723283 Phase Ib/II Actionable In a Phase I/II trial, JNJ-63723283 demonstrated safety and preliminary efficacy, resulted in partial response in 9.4% (3/32) and stable disease in 56.3% (18/32) of patients with advanced solid tumors (Journal of Clinical Oncology 36, no. 5_suppl (February 10 2018) 58-58). detail...
Unknown unknown diffuse large B-cell lymphoma not applicable OPB-111077 Phase I Actionable In a Phase I trial, a patient with diffuse large B-cell lymphoma demonstrated a partial response when treated with OPB-111077 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B118). detail...
Unknown unknown 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 esophageal cancer no benefit Cetuximab + Cisplatin + Paclitaxel + Radiotherapy Phase III Actionable In a Phase III trial, the addition of Erbitux (cetuximab) to therapy with Taxol (paclitaxel), Platinol (cisplatin), and radiotherapy did not demonstrate increased clinical benefit over Taxol (paclitaxel) and Platinol (cisplatin) plus radiotherapy in esophageal cancer patients, with the experimental and control groups demonstrating similar median overall survival (19.7 mo vs. 19 mo, respectively (PMID: 28687830; NCT00566852). 28687830
Unknown unknown Advanced Solid Tumor not applicable Gemcitabine + LY2780301 Phase Ib/II Actionable In a Phase Ib trial, the combination of LY2780303 and Gemzar (gemcitabine) demonstrated some antitumor activity in patients with advanced solid tumors including 2 patients with a partial response, 28 patients with stable disease, and a four month disease control rate of 22% (PMID: 28750271). 28750271
Unknown unknown ovarian cancer not applicable Enoticumab Phase I Actionable In a Phase I study, Enoticumab (REGN421) demonstrated safety and preliminary efficacy in ovarian cancer patients (PMID: 25724527). 25724527
Unknown unknown 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 ovarian cancer not applicable AIM-100 Preclinical Actionable In a preclinical study, AIM-100 inhibited growth of ovarian cancer cells in culture (PMID: 22322295). 22322295
Unknown unknown acute myeloid leukemia not applicable Bortezomib + Tinostamustine Preclinical - Patient cell culture Actionable In a preclinical study, the combination of EDO-S101 and Velcade (bortezomib) decreased viability of primary acute myeloid leukemia cells in culture (PMID: 28753594). 28753594
Unknown unknown Advanced Solid Tumor no benefit AMG 820 Phase I Actionable In a Phase I trial, AMG 820 treatment was well-tolerated and resulted in safety, however, demonstrated limited antitumor activity in patients with advanced solid tumors, and thus, AMG 820 therapy combined with an immunotherapy was suggested based on clinical and preclinical evidence (PMID: 28655795). 28655795
Unknown unknown aggressive NK-cell leukemia not applicable tagraxofusp-erzs FDA approved Actionable In a Phase I/II trial that supported FDA approval, Elzonris (tagraxofusp-erzs) treatment resulted in complete response/clinical complete response in 53.8% (7/13) of patients with untreated blastic plasmacytoid dendritic cell neoplasm (BPDCN, also known as natural killer cell leukemia/lymphoma) with a median follow-up of 11.5 months, and 1 complete response and 1 clinical complete response in 15 patients with relapsed or refractory BPDCN (FDA.gov; NCT02113982). detail... detail...
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 melanoma not applicable Nab-paclitaxel + Oblimersen + Temozolomide Phase I Actionable In a Phase I trial, treatment with the combination of Genasense (oblimersen), Temodar (temozolomide), and Abraxane (nab-paclitaxel) resulted in an objective response rate of 40.6% (13/32) and disease control rate of 75% (24/32) in patients with advanced melanoma (PMID: 23064957). 23064957
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 lung cancer not applicable Triolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Triolimus led to cytotoxicity and inhibited Ras/Raf/MAPK and PI3K/Akt/mTOR pathway signaling in lung cancer cells in culture, and induced apoptosis, which resulted in tumor growth inhibition, in cell line xenograft models (PMID: 22896668). 22896668
Unknown unknown lymphoma not applicable Alisertib + Vorinostat Phase I Actionable In a Phase I trial, Alisertib (MLN8237) in combination with Zolinza (vorinostat) demonstrated safety and preliminary efficacy in patients with relapsed or refractory lymphomas, resulting in an overall response rate of 16.7% (3/18, 1 complete response, 2 partial responses) at the recommended Phase 2 dose (PMID: 31617432; NCT01567709). 31617432
Unknown unknown pancreatic cancer not applicable Foretinib Preclinical Actionable In a preclinical study, Foretinib (GSK1363089) treatment resulted in regression of the tumor vasculature, extensive hypoxia, apoptosis, and decreased tumor aggressiveness in a transgenic mouse model of pancreatic islet cancer (PMID: 21613405). 21613405
Unknown unknown glioblastoma multiforme not applicable BI2536 Preclinical - Cell culture Actionable In a preclinical study, a glioblastoma cell line treated with BI2536 demonstrated decreased cell viability in culture (PMID: 26573800). 26573800
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 follicular lymphoma not applicable Rituximab Phase III Actionable In a Phase III trial, Rituxan (rituximab) treatment in patients with follicular lymphoma resulted in an overall survival of 84.9% in patients receiving the drug intravenously and 84.4% in patients receiving the drug subcutaneously (PMID: 28476440). 28476440
Unknown unknown breast cancer not applicable DCBCI0901 Preclinical - Cell culture Actionable In a preclinical study, breast cancer cell lines treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown myelodysplastic syndrome not applicable Cytarabine + Glasdegib Phase Ib/II Actionable In a Phase Ib trial, the combination of Glasdegib (PF-04449913) and Cytosar-U (cytarabine) resulted in an overall survival of 4.4 months in patients with either acute myeloid leukemia or myelodysplastic syndrome (MDS), with 33% (1/3) of MDS patients experiencing a complete remission (PMID: 29463550). 29463550
Unknown unknown pancreatic cancer not applicable SF2523 Preclinical Actionable In a preclinical study, SF2523 decreased tumor immunosuppression and inhibited tumor growth in orthotopic mouse pancreatic cancer models (PMID: 31018997). 31018997
Unknown unknown prostate cancer 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 ovarian cancer not applicable Atezolizumab + Hu5F9-G4 Phase I Actionable In a Phase Ib trial, Tecentriq (atezolizumab) and Magrolimab (Hu5F9-G4) combination therapy demonstrated acceptable safety, and resulted in stable disease as best response in 56% (10/18) of patients with platinum-resistant ovarian cancer (J Clin Oncol 38, 2020 (suppl 5; abstr 18); NCT03558139). detail...
Unknown unknown Advanced Solid Tumor not applicable AVID100 Phase I Actionable In a Phase I trial, AVID100 treatment resulted in prolonged stable disease in 12.5% (3/24) of patients with advanced solid tumors not selected for Egfr overexpression (AACR Annual Meeting 2019, Abstract CT056). detail...
Unknown unknown acute leukemia not applicable CNDO-109 Phase I Actionable In a Phase I trial, acute leukemia patients treated with CNDO-109 demonstrated a relapse-free survival (RFS) rate of 33% at 12 months, with three patients demonstrating a long-term RFS of at least 32.6 months in a follow-up (PMID: 29597002; NCT01520558). 29597002
Unknown unknown non-Hodgkin lymphoma not applicable Ibritumomab tiuxetan FDA approved Actionable In a Phase I trial that supported FDA approval, Zevalin (ibritumomab tiuxetan) treatment resulted in significantly improved objective response rate (83% vs 55%, p<0.001) compared to Rituxan (rituximab) in patients with relapsed or refractory low-grade or follicular non-Hodgkin's lymphoma (PMID: 27497027). 27497027 detail...
Unknown unknown triple-receptor negative breast cancer not applicable Alisertib + Sapanisertib Preclinical - Pdx Actionable In a preclinical study, the combination treatment of Alisertib (MLN8237) and Sapanisertib (MLN0128) resulted in decreased proliferation in triple-receptor negative breast cancer (TNBC) cell lines in culture and reduced tumor growth in patient-derived xenograft (PDX) TNBC models, with one model showing tumor growth inhibition of 94.27% compared to 54.47% with Sapanisertib (MLN0128) alone and 35.09% with Alisertib (MLN8237) alone (PMID: 32414750). 32414750
Unknown unknown lung non-small cell carcinoma not applicable Abemaciclib + Pemetrexed Disodium Phase Ib/II Actionable In a Phase Ib trial, the combination of Verzenio (abemaciclib) and Alimta (pemetrexed) was well-tolerated and demonstrated preliminary activity in patients with previously treated metastatic non-small cell lung cancer, resulting in a response rate of 4% (1/23; partial response (PR)), a disease control rate of 57% (13/23; 1 PR and 12 stable disease), and a median progression-free survival of 5.55 months (95% CI, 1.81, 10.05) (PMID: 30082474; NCT02079636). 30082474
Unknown unknown liposarcoma not applicable Doxorubicin + Selinexor Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Selinexor (KPT-330) and Kyprolis (carfilzomib) resulted in enhanced induction of apoptotic markers compared to Selinexor (KPT-330) alone, and worked additively decrease viability of a liposarcoma cell line in culture (PMID: 28314790). 28314790
Unknown unknown stomach cancer not applicable Capecitabine + Lapatinib Phase II Actionable In a Phase II trial, Tykerb (lapatinib) and Xeloda (capecitabine) combination treatment resulted in partial response in 17.9% (12/67) and stable disease in 46.3% (31/67) of gastric cancer patients regardless of their ERBB2 (HER2) status, although increased Erbb3 (Her3) expression level correlated with higher response rate (PMID: 27325685). 27325685
Unknown unknown prostate cancer not applicable Selinexor Phase II Actionable In a Phase II trial, Selinexor demonstrated preliminary efficacy and poor toxicity profile in patients with abiraterone- and/or enzalutamide-refractory metastatic castration-resistant prostate cancer, resulted in partial response in 25% (2/8) and stable disease in 50% (4/8) of evaluable patients (PMID: 29487219). 29487219
Unknown unknown plasmacytoma not applicable REGN5458 Case Reports/Case Series Actionable In a Phase Ib/II study, REGN5458 treatment resulted in a partial response in a patient with medullary and cutaneous extramedullary plasmacytoma (Blood (2019) 134 (Supplement_1): 3176; NCT03761108). detail...
Unknown unknown breast cancer not applicable Pazopanib Phase II Actionable In a Phase II trial, Votrient (pazopanib) displayed safety and some efficacy in breast cancer patients (PMID: 20682606). 20682606
Unknown unknown Advanced Solid Tumor not applicable CB-103 Preclinical - Pdx Actionable In a preclinical study, CB-103 treatment resulted in antitumor efficacy in in vivo models and patient derived xenograft (PDX) models with advanced solid tumors (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 411P). detail...
Unknown unknown germ cell cancer not applicable Pazopanib Phase II Actionable In a Phase II trial, Votrient (pazopanib) treatment resulted in partial responses in 4.7% (2/43), stable disease in 44.2% (19/43), and a 3-month progression free survival probability of 12.8% in patients with refractory germ cell cancer (PMID: 28383677). 28383677
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 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 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 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 Advanced Solid Tumor not applicable Capmatinib Phase I Actionable In a Phase I trial, Tabrecta (capmatinib) demonstrated safety and preliminary efficacy, resulted in stable disease as best overall response in 18% (8/44) of patients with advanced solid tumors (PMID: 30724423). 30724423
Unknown unknown epithelioid sarcoma not applicable Abexinostat Preclinical - Cell line xenograft Actionable In a preclinical study, Abexinostat (PCI-24781) inhibited growth and colony formation of epithelioid sarcoma cell lines in culture, and inhibited tumor growth in epithelioid sarcoma cell line xenograft models (PMID: 26396249). 26396249
Unknown unknown connective tissue benign neoplasm not applicable Ipafricept Phase I Actionable In a Phase I trial, two desmoid tumor patients demonstrated stable disease for greater than 6 months when treated with Ipafricept (OMP-54F28) (PMID: 28954784). 28954784
Unknown unknown anal squamous cell carcinoma not applicable Prexasertib Phase I Actionable In a Phase I trial, Prexasertib (LY2606368) treatment resulted in partial response in a patient with anal squamous cell carcinoma (PMID: 27044938). 27044938
Unknown unknown anal squamous cell carcinoma not applicable Prexasertib Phase Ib/II Actionable In a Phase Ib trial, treatment with Prexasertib (LY2606368) resulted in an overall response rate of 15% (4/26; 1 complete response (CR) and 3 partial responses (PR)), a clinical benefit rate (CR+PR+stable disease) of 58% (15/26), and a median progression-free survival of 2.8 months in patients with squamous cell carcinoma of the anus (PMID: 29643063; NCT0115790). 29643063
Unknown unknown sarcoma not applicable Epirubicin + Panobinostat Phase I Actionable In a Phase I trial, 55% (11/20) of sarcoma patients demonstrated stable disease while one patient (1/20) experienced a partial response when treated with a combination of Farydak (panobinostat) and Ellence (epirubicin) (PMID: 26903311). 26903311
Unknown unknown breast cancer not applicable AZ0108 Preclinical - Cell line xenograft Actionable In a preclinical study, AZ0108 induced mitotic defects, apoptosis, and inhibited growth in breast cancer cell lines in culture, and inhibited tumor growth in cell line xenograft models (PMID: 30297535). 30297535
Unknown unknown Hodgkin's lymphoma not applicable Ixazomib + JQ1 Preclinical Actionable In a preclinical study, Ixazomib (MLN9708) and JQ1 combination treatment resulted in increased apoptosis in Hodgkin's lymphoma cells in culture (PMID: 26988986). 26988986
Unknown unknown Advanced Solid Tumor not applicable Amuvatinib + Carboplatin + Paclitaxel Phase I Actionable In a Phase I clinical trial, Amuvatinib (MP470) in combination with chemotherapeutic agents, demonstrated safety and some efficacy in patients with advanced solid tumors (PMID: 24849582). 24849582
Unknown unknown neuroblastoma not applicable Alisertib + Irinotecan + Temozolomide Phase II Actionable In a Phase II trial, the combination of Alisertib (MLN8237), Camptosar (irinotecan), and Temodar (temozolomide) resulted in a response rate of 21.1% (4/19) in patients with neuroblastoma, with 4 partial responses, 2 minor responses, and 5 stable diseases (PMID: 30093449; NCT01601535). 30093449
Unknown unknown neuroblastoma not applicable Alisertib + Irinotecan + Temozolomide Phase I Actionable In a Phase I trial, the combination of Alisertib (MLN8237), Camptosar (irinotecan), and Temodar (temozolomide) demonstrated safety and preliminary efficacy in neuroblastoma patients, resulting in an overall response rate of 31.8% and a 2-year progression-free survival rate of 52.4% (PMID: 26884555). 26884555
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 lung non-small cell carcinoma not applicable Pegilodecakin + Pembrolizumab Phase I Actionable In a Phase I trial, Pegilodecakin (AM0010) and Keytruda (pembrolizumab) (n=5) or Opdivo (nivolumab) (n=29) combination therapy resulted in an objective response rate of 41% (11/26, 11 partial response) and stable disease in 46% (12/26) of evaluable patients with non-small cell lung carcinoma, median progression-free survival and overall survival was 10.9 and 32.2 months in patients received Pegilodecakin and Keytruda, 4 of 4 tested had PD-L1 < 1% (J Clin Oncol 36, 2018 (suppl; abstr 9018); NCT02009449). detail...
Unknown unknown lung cancer not applicable OBI-999 Preclinical - Pdx Actionable In a preclinical study, OBI-999 treatment inhibited tumor growth in a Globo H-expressing lung cancer cell line xenograft model and a lung cancer patient-derived xenograft (PDX) model (Cancer Res 2019;79(13 Suppl):Abstract nr 4815). detail...
Unknown unknown lung non-small cell carcinoma not applicable Vandetanib Phase II Actionable In a Phase II trial, Caprelsa (vandetanib) alone, or in combination with Taxotere (docetaxel), improved progression-free survival of NSCLC patients (PMID: 17243944). 17243944
Unknown unknown lung non-small cell carcinoma not applicable Vandetanib Phase II Actionable In a Phase II trial, treatment with Caprelsa (vandetanib) increased progression-free survival in NSCLC patients, compared to Iressa (gefitinib) (PMID: 19332730). 19332730
Unknown unknown lung non-small cell carcinoma not applicable Vandetanib Clinical Study Actionable In a meta-analysis of 2,284 NSCLC patients, Caprelsa (vandetanib) in combination with chemotherapy, increased progression-free survival (PFS) and overall response rate (ORR) but not overall survival (PMID: 23861835). 23861835
Unknown unknown pancreatic cancer not applicable Demcizumab + Gemcitabine Phase Ib/II Actionable In a Phase Ib trial, Demcizumab (OMP-21M18) and Gemzar (gemcitabine) combination treatment resulted in partial response in 25% (4/16) and stable disease in 44% (7/16) of pancreatic cancer patients (J Clin Onco, Vol 32, No 3_suppl (January 20 Supplement), 2014: 279). detail...
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 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 acute myeloid leukemia not applicable GSK3368715 Preclinical - Cell culture Actionable In a preclinical study, GSK3368715 inhibited growth of acute myeloid leukemia cell lines in culture, and demonstrated cytotoxicity in 50% of cell lines tested (PMID: 31257072). 31257072
Unknown unknown lung cancer not applicable CAB-AXL-ADC Preclinical - Cell line xenograft Actionable In a preclinical study, CAB-AXL-ADC treatment inhibited tumor growth in a lung cancer cell line xenograft model (Cancer Res 2018;78(13 Suppl):Abstract nr 827). detail...
Unknown unknown sarcoma not applicable Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (2.9 vs 1.0 months), but no difference in overall survival (HR=0.75, p=0.37) compared to placebo in patients with soft tissue sarcoma excluding liposarcoma, leiomyosarcoma, and synovial sarcoma (PMID: 27751846). 27751846
Unknown unknown triple-receptor negative breast cancer not applicable ASTX-660 Preclinical - Cell line xenograft Actionable In a preclinical study, ASTX-660 inhibited growth of triple-receptor negative breast cancer cell lines in culture and in cell line xenograft models (Cancer Res 2016;76(14 Suppl):Abstract nr 1287). detail...
Unknown unknown glioblastoma multiforme 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 ovarian cancer not applicable PF-06647263 Phase I Actionable In a Phase I trial, PF-06647263 treatment resulted in partial response in 3 patients with ovarian cancer (J Clin Oncol 35, 2017 (suppl; abstr 2511)). detail...
Unknown unknown acute myeloid leukemia not applicable Pexmetinib Preclinical - Cell culture Actionable In a preclinical study, treatment with Pexmetinib (ARRY-614) inhibited cell proliferation of acute myeloid leukemia cells in culture (PMID: 27287719). 27287719
Unknown unknown sarcoma not applicable NBTXR3 + Radiotherapy Phase II Actionable In a Phase II/III trial, no difference in objective response rate was observed between soft tissue sarcoma patients treated with NBTXR3 plus radiotherapy vs. radiotherapy alone (7% vs. 10%, p=0.86), however, NBTXR3 plus radiotherapy resulted in an increased pathological complete response rate of 16% (14/87) vs. 8% (7/89), and a higher proportion of patients receiving NBTXR3 and radiotherapy demonstrated negative margins (84% (61/73) vs. 70% (57/82), p=0.30) (PMID: 31296491; NCT02379845). 31296491
Unknown unknown cervical cancer not applicable BIND-014 Phase I Actionable In a Phase I clinical trial, BIND-014 treatment resulted in a complete response in a cervical cancer patient (PMID: 26847057). 26847057
Unknown unknown head and neck squamous cell carcinoma no benefit Cetuximab + Cisplatin + Docetaxel + Fluorouracil Phase II Actionable In a Phase II trial, addition of Erbitux (cetuximab) to induction chemotherapy consisting of Platinol (cisplatin), Taxotere (docetaxel), and Fluorouracil, resulted in early termination of the study due to unacceptable clinical outcomes (PMID: 28911062). 28911062
Unknown unknown Advanced Solid Tumor not applicable Paclitaxel + 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 BI2536 + Metformin Preclinical - Pdx Actionable In a preclinical study, the combination of BI2536 and Metformin worked synergistically to inhibit tumor growth in patient-derived prostate cancer xenograft models (PMID: 25505174). 25505174
Unknown unknown colon carcinoma not applicable Mps-BAY1 Preclinical Actionable In a preclinical study, Mps-BAY1 inhibited cell cycle progression and induced cell death of colon carcinoma cells in culture (PMID: 23933817). 23933817
Unknown unknown Advanced Solid Tumor not applicable Ficlatuzumab Phase I Actionable In a Phase I trial, Ficlatuzumab treatment resulted in stable disease in 57% (12/21) of patients with advanced solid tumors and a decrease in phosphorylated Met in one patient with multiple myeloma (PMID: 24901237). 24901237
Unknown unknown chronic lymphocytic leukemia not applicable ARQ 531 Preclinical - Patient cell culture Actionable In a preclinical study, ARQ 531 treatment inhibited activation of BCR signaling and cell migration, and induced enhanced cytotoxicity in patient-derived chronic lymphocytic leukemia cells in culture, and increased survival in a transgenic mouse model (PMID: 30093506). 30093506
Unknown unknown lymphoma not applicable Copanlisib Phase II Actionable In a Phase II trial, treatment with Aliqopa (copanlisib) in patients with indolent lymphoma resulted in an objective response rate of 59% (84/142), including a complete response in 12%, and demonstrated a median duration of response of 22.6 months and a median progression-free survival of 11.2 months (PMID: 28976790, PMID: 28633365; NCT01660451). 28633365 28976790
Unknown unknown lymphoma not applicable Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in partial response in 20% (1/5) and stable disease in 40% (2/5) of patients with transformed lymphoma (PMID: 24852792). 24852792
Unknown unknown ovarian serous carcinoma not applicable Paclitaxel + Vistusertib Phase I Actionable In a Phase I trial, the combination therapy of Taxol (paclitaxel) and Vistusertib (AZD2014) resulted in a RECIST response rate of 64% (16/25), a CA125 response rate of 52% (13/25), and a median progression-free survival of 5.8 months in patients with high grade serous ovarian cancer (PMID: 30016392; NCT02193633). 30016392
Unknown unknown multiple myeloma not applicable Ricolinostat + SJB3-019A Preclinical - Patient cell culture Actionable In a preclinical study, the combination of SJB3-019A and Ricolinostat (ACY-1215) worked synergistically to induce cytotoxicity in multiple myeloma cell lines and primary multiple myeloma cells in culture (PMID: 28270494). 28270494
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 renal cell carcinoma not applicable SRF388 Preclinical Actionable In a preclinical study, SRF388 treatment demonstrated antitumor activity in an orthotopic mouse model of renal cell carcinoma (AACR 2020, Therapeutic Antibodies 3; PO.IM02.25, Abs nr: 4550/18). detail...
Unknown unknown chordoma not applicable Dasatinib Phase II Actionable In a Phase II trial, patients with chordoma demonstrated a median progression free survival of 6.3 months and six patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown Advanced Solid Tumor not applicable CYT01B + Talazoparib Preclinical - Cell culture Actionable In a preclinical study, CYT01B and Talzenna (talazoparib) synergistically inhibited growth of tumor cell lines in culture (AACR Annual Meeting 2019, Abstract 363). detail...
Unknown unknown renal cell carcinoma not applicable Sunitinib Phase III Actionable In a Phase III clinical trial, treatment with Sutent (sunitinib) resulted in prolonged disease free survival (HR = 0.761) compared to placebo in post-nephrectomy patients with renal cell carcinoma (ESMO 2016 Congress in Copenhagen, Abstract LBA11_PR). detail...
Unknown unknown renal cell carcinoma not applicable Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Sutent (sunitinib) resulted in improved median progression free survival (47.3 weeks) and objective response rate (27.5%) in patients with renal cell carcinoma (PMID: 19707433). 19707433 detail...
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 multiple myeloma not applicable Carfilzomib + Ricolinostat Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Kyprolis (carfilzomib) and Ricolinostat (ACY-1215) promoted apoptosis and reduced tumor volume in a human multiple myeloma cell line xenograft model (PMID: 25709080). 25709080
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 ovarian cancer not applicable Pamiparib + Tislelizumab Case Reports/Case Series Actionable In a Phase Ib trial, the combination therapy of Tislelizumab (BGB-A317) and Pamiparib (BGB-290) in ovarian cancer patients resulted in 1 complete response and 5 partial responses (J Clin Oncol 35, 2017 (suppl; abstr 3013)). detail...
Unknown unknown Advanced Solid Tumor not applicable Camrelizumab Phase I Actionable In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors, with an objective response rate of 31% (18/58, all partial responses) and disease control rate of 46.5% (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). detail...
Unknown unknown desmoid tumor not applicable Sorafenib Phase III Actionable In a Phase III trial, Nexavar (sorafenib) treatment resulted in an increased 2-year progression-free survival compared to placebo (81% vs 36%), an objective response rate of 33% (16/49; 1 complete response and 15 partial responses (PR)) compared in 20% (7/25; all PR) with placebo, and a median time to objective response of 9.6 months, compared to 13.3 months with placebo, in patients with refractory desmoid tumors (PMID: 30575484; NCT02066181). 30575484
Unknown unknown colon adenocarcinoma not applicable REGN1400 Preclinical Actionable In a preclinical study, REGN1400, alone and in combination with anti-EGFR antibodies, showed efficacy in treating mouse xenograft models of colorectal cancer (PMID: 24634416). 24634416
Unknown unknown rhabdomyosarcoma not applicable BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 33% (2/6) of cell line xenograft models of rhabdomyosarcoma (PMID: 21298745). 21298745
Unknown unknown breast cancer not applicable GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 was well-tolerated and resulted in some efficacy in patients with breast cancer, including stable disease in 31% (7/22) and one patient with a partial response (PMID: 26603258). 26603258
Unknown unknown cervical cancer not applicable BIRB-796 + Tozasertib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Tozasertib (VX-680) and BIRB-796 resulted in increased growth inhibition and induction of apoptosis in cervical cancer cell lines in culture, and increased tumor growth inhibition in cervical cancer cell line xenograft models, compared to either agent alone (PMID: 27082306). 27082306
Unknown unknown multiple myeloma not applicable BC2059 + Bortezomib Preclinical - Patient cell culture Actionable In a preclinical study, the combination of BC2059 and Velcade (bortezomib) worked additively or synergistically in several human multiple myeloma cell lines and primary multiple myeloma cells with activated canonical Wnt signaling in culture (PMID: 28500235). 28500235
Unknown unknown glioblastoma multiforme not applicable ST-11 Preclinical Actionable In a preclinical study, ST-11 induced cell-cycle arrest and apoptosis in glioblastoma cell lines in culture, and reduced tumor burden in mouse models of glioblastoma (PMID: 27325686). 27325686
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 chronic lymphocytic leukemia not applicable Chlorambucil + Ofatumumab FDA approved Actionable In a Phase III trial supporting FDA approval, Arzerra (ofatumumab) and Chlorambucil combination treatment resulted in improved median progression-free survival (22.4 vs. 13.1 months) compared to Chlorambucil alone in treatment-naive patients with chronic lymphocytic leukaemia (PMID: 25882396). 25882396 detail...
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 ovarian cancer not applicable Bevacizumab Phase III Actionable In a meta-analysis of three phase III trials, Avastin (bevacizumab) significantly increased progression free survival and overall survival comparied to standard therapy, with hazard ratios of 0.53 and 0.87, respectively (PMID: 26657509). 26657509
Unknown unknown ovarian cancer not applicable Bevacizumab FDA approved Actionable In a Phase III trial that supported FDA approval, ovarian cancer patients treated with Avastin (bevacizumab) combined with chemotherapy versus chemotherapy alone resulted in a greater PFS (6.7 mo vs 3.4 mo), overall response rate (27.3% vs 11.8%), and OS (16.6 mo vs 13.3 mo) (PMID: 24637997). detail... 24637997
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 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 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 Advanced Solid Tumor not applicable Conatumumab + Ganitumab Phase Ib/II Actionable In a Phase Ib/II trial, Ganitumab and Conatumumab (AMG 655) combination treatment resulted in stable disease in 36% (28/78) of patients with an advanced solid tumor (PMID: 24816908). 24816908
Unknown unknown acute lymphoblastic leukemia no benefit BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in no change in event-free survival in 100% (7/7) of cell ine xenograft models of acute lymphocytic leukemia (PMID: 21298745). 21298745
Unknown unknown multiple myeloma not applicable Azacitidine + Mivebresib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Vidaza (azacitidine) and Mivebresib (ABBV-075) resulted in increased tumor growth inhibition in a multiple myeloma cell line xenograft model compared to either agent alone (PMID: 28416490). 28416490
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 gastroesophageal junction adenocarcinoma not applicable trifluridine/tipiracil hydrochloride FDA approved Actionable In a Phase III trial (TAGS) that supproted FDA approval, Lonsurf (trifluridine/tipiracil hydrochloride) treatment resulted in improved overall survival (5.7 vs 3.6 months, HR=0.69, p=0.00029) compared to placebo in patients with heavily pretreated metastatic or advanced gastric cancer or gastroesophageal junction adenocarcinoma (PMID: 30355453; NCT02500043). detail... 30355453
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 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 lymphoid leukemia not applicable Ponatinib FDA approved Actionable In a Phase II clinical trial which supported FDA approval, Iclusig (ponatinib) was effective in promoting disease regression in 52% of patients with accelerated phase chronic myeloid leukemia, 31% of patients with blast phase chronic myeloid leukemia, and 41% of patients with Philadelphia chromosome positive acute lymphoblastic leukemia (PMID: 23935038). 23935038 detail...
Unknown unknown Advanced Solid Tumor not applicable Futibatinib Phase I Actionable In a Phase I trial, TAS-120 treatment resulted in clinical response in 5.5% (2/36) and stable disease over 24 weeks in 5.5% (2/36) of patients with advanced solid tumors (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 372PD). detail...
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 UD-017 Preclinical - Patient cell culture Actionable In a preclinical study, UD-017 inhibited growth of patient-derived colorectal cancer cells in culture (J Clin Oncol 35, 2017 (suppl; abstr e14085)). detail...
Unknown unknown pulmonary neuroendocrine tumor not applicable Spartalizumab Case Reports/Case Series Actionable In a Phase I trial, Spartalizumab (PDR001) treatment in a patient with an atypical carcinoid lung tumor with initial CD8-positive lymphocyte tumor infiltration of 5.6% resulted in a partial response lasting 8.5 months, with an increase in CD8-positive lymphocyte infiltration to 37.6% after two weeks and subsequent reduction of target lesion size (PMID: 32179633; NCT02404441). 32179633
Unknown unknown breast cancer not applicable ONC201 Preclinical - Cell line xenograft Actionable In a preclinical study, ONC201 inhibited viability of several breast cancer cell lines in culture (including triple-negative breast cancer (TNBC) and non-TNBC cell lines), with some cell lines demonstrating increased apoptosis, and inhibited tumor growth in TNBC cell line xenograft models (PMID: 28424227). 28424227
Unknown unknown thyroid gland carcinoma not applicable MLN8054 Preclinical - Patient cell culture Actionable In a preclinical study, MLN8054 inhibited growth of tumor cells derived from a patient with anaplastic thyroid carcinoma (PMID: 27379749). 27379749
Unknown unknown triple-receptor negative breast cancer not applicable SKLB646 Preclinical - Cell line xenograft Actionable In a preclinical study, SKLB646 inhibited proliferation and migration and induced apoptosis of triple-negative breast cancer (TNBC) cell lines in culture, and inhibited tumor growth in TNBC cell line xenograft models (PMID: 26721945). 26721945
Unknown unknown Advanced Solid Tumor not applicable AMC303 Preclinical - Cell line xenograft Actionable In a preclinical study, AMC303 resulted in decreased cell invasion and migration in solid tumor cell lines in culture and tumor regression of metastatic lesions of the liver in xenograft models (Cancer Res 2017;77(13 Suppl):Abstract nr 4911). detail...
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 Advanced Solid Tumor not applicable MEDI3617 + Paclitaxel Phase I Actionable In a Phase I trial, MEDI3617 in combination with Taxol (paclitaxel) resulted in objective response in 15% (2/13) and stable disease in 31% (4/13) of patients with advanced solid tumors, with a median progression-free survival of 3.5 months (PMID: 29559563; NCT01248949). 29559563
Unknown unknown colorectal cancer not applicable Rucaparib + Temozolomide Preclinical - Cell line xenograft Actionable In a preclinical trial, Rubraca (rucaparib) sensitized colorectal cancer cell lines to Temodar (temozolomide) treatment both in culture and in cell line xenograft models (PMID: 17363489). 17363489
Unknown unknown prostate cancer not applicable TAK-960 Preclinical - Cell line xenograft Actionable In a preclinical study, prostate cancer cells treated with TAK-960 demonstrated a decrease in tumor size in cell line xenograft models (PMID: 22188812). 22188812
Unknown unknown pancreatic cancer not applicable ABTL0812 Preclinical Actionable In a preclinical study, ABTL0812 inhibited growth and mTORC1 signaling and induced autophagy in a human pancreatic cancer cell line in culture (PMID: 26671995). 26671995
Unknown unknown acute lymphoblastic leukemia not applicable AS605240 Preclinical - Pdx & cell culture Actionable In a preclinical study, AS605240 increased apoptosis and decreased viability of T-acute lymphocytic leukemia (T-ALL) cell lines and primary T-ALL cells in culture, and decreased leukemic progression in a primary T-ALL cell xenograft model (PMID: 25869207). 25869207
Unknown unknown lung carcinoma not applicable SRX3207 Preclinical - Cell culture Actionable In a preclinical study, SRX3207 treatment induced cytotoxicity in Lewis lung carcinoma cells in culture, and inhibited tumor growth, and increased survival in a syngeneic mouse model (PMID: 31974273). 31974273
Unknown unknown CLL/SLL not applicable Idelalisib FDA approved Actionable In a Phase II trial that supported FDA approval, Zydelig (idelalisib) treatment resulted in an overall response rate of 58% (15/26, all partial response) in patients with relapsed small lymphocytic lymphoma, with a median duration of response of 11.9 months (PMID: 24450858; NCT01282424). 24450858 detail...
Unknown unknown Advanced Solid Tumor not applicable VS-5584 Preclinical - Cell line xenograft Actionable In a preclinical study, VS-5584 inhibited growth of a variety of human tumor cell lines in culture and inhibited tumor growth in cell line xenograft models (PMID: 23270925). 23270925
Unknown unknown pancreatic cancer not applicable SRT3025 Preclinical - Cell line xenograft Actionable In a preclinical study, SRT3025 decreased viability of human pancreatic cancer cell lines in culture and inhibited tumor growth in pancreatic cancer cell line xenograft models (PMID: 26655844). 26655844
Unknown unknown glioblastoma multiforme 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 breast cancer not applicable CWP232228 Preclinical - Cell line xenograft Actionable In a preclinical study, CWP232228 inhibited Wnt pathway signaling, inhibited growth of breast cancer stem cells, and reduced tumor growth in breast cancer xenograft models (PMID: 25660951). 25660951
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 hepatocellular carcinoma 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 50% (8/16), a disease control rate of 93.8% (15/16, stable disease or better), a median progression-free survival (PFS) of 5.8 months, and a 9-month PFS rate of 41.0% in evaluable patients with advanced hepatocellular carcinoma (PMID: 30348638; NCT02942329). 30348638
Unknown unknown hepatocellular carcinoma not applicable Foretinib Phase I Actionable In a Phase I trial, Foretinib (GSK1363089) demonstrated safety and activity in hepatocellular carcinoma patients, with an overall response rate by mRESCIST of 22.9% (8/35, all partial responses), a disease stabilization rate of 82.9% (29/35), and a median duration of response of 7.6 months (PMID: 27821605; NCT00920192). 27821605
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 transitional cell carcinoma not applicable Sabatolimab + Spartalizumab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in stable disease in 3 patients with transitional cell carcinoma (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown alveolar rhabdomyosarcoma not applicable Infigratinib Preclinical - Pdx Actionable In a preclinical study, Infigratinib (BGJ398) resulted in antitumor activity in an alveolar rhabdomyosarcoma patient derived xenograft (PDX) model (PMID: 24687871). 24687871
Unknown unknown prostate cancer not applicable Metformin Phase II Actionable In a Phase II trial, Glucophage (metformin) demonstrated safety and preliminary efficacy in patients with castration-resistant prostate cancer (PMID: 24412228). 24412228
Unknown unknown endometrial cancer not applicable Cisplatin + VE-821 Preclinical - Cell culture Actionable In a preclinical study, VE-821 increased the sensitivity of an endometrial cancer cell line to Platinol (cisplatin) in culture (PMID: 25560806). 25560806
Unknown unknown B-cell lymphoma not applicable ARQ 531 Preclinical Actionable In a preclinical study, ARQ 531 treatment increased survival in a transgenic mouse model of aggressive B-cell lymphoma similar to Richter transformation, with a median of 47 days vs. 38 days with Imbruvica (ibrutinib) (p=0.036) (PMID: 30093506). 30093506
Unknown unknown Advanced Solid Tumor not applicable Carboplatin + Paclitaxel + Pazopanib Phase I Actionable In a Phase I study, Votrient (pazopanib) in combination with Taxol (paclitaxel) and Paraplatin (carboplatin) demonstrated efficacy in solid tumors (PMID: 22679111). 22679111
Unknown unknown breast cancer not applicable LDC1267 Preclinical Actionable In a preclinical study, LDC1267 treatment resulted in reduced l metastatic liver lesions, but did not impact primary tumor growth, in a mouse model of metastatic breast cancer (PMID: 24553136). 24553136
Unknown unknown breast cancer not applicable Gemcitabine + Trametinib Phase Ib/II Actionable In a Phase Ib clinical trial, the combination of Mekinist (trametinib) and Gemzar (gemcitabine) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors, including one complete response in a patient with breast cancer (PMID: 23583440). 23583440
Unknown unknown breast cancer not applicable AIM-100 Preclinical Actionable In a preclinical study, AIM-100 inhibited growth of breast cancer cells in culture (PMID: 22322295). 22322295
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 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 multiple myeloma not applicable Carfilzomib + Tinostamustine Preclinical - Patient cell culture Actionable In a preclinical study, the combination of Kyprolis (carfilzomib) and EDO-S101 worked synergistically to decrease viability of multiple myeloma cell lines and primary cells in culture, including a cell line with resistance to Velcade (bortezomib) (PMID: 28753594). 28753594
Unknown unknown 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 neuroendocrine tumor not applicable Cixutumumab + Everolimus + Octreotide acetate Phase I Actionable In a Phase I trial, patients with neuroendocrine tumors treated with the combination of Cixutumumab, Sandostatin Lar Depot (octreotide acetate), and Afinitor (everolimus) demonstrated some efficacy, however, the drug combination did result in multiple non-dose limiting toxicities preventing long term tolerance (PMID: 25900182). 25900182
Unknown unknown clear cell sarcoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 54%, median progression-free survival of 11 months, an objective response rate of 14% (n=7), and a median overall survival of 16 months in patients with clear cell sarcoma (PMID: 29895706; NCT01878448). 29895706
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 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 non-small cell carcinoma not applicable Carboplatin + Ganetespib + Paclitaxel + Radiotherapy Preclinical - Cell line xenograft Actionable In a preclinical study, Ganetespib had a variable impact on chemoradiation therapy (consisting of Paraplatin (carboplatin), Taxol (paclitaxel), and radiation) in non-small cell lung cancer cell lines in culture and in xenograft models, resulting in increased sensitivity in some cell lines, and decreased sensitivity in others (PMID: 27354472). 27354472
Unknown unknown lung small cell carcinoma not applicable DT2216 + Venetoclax Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of DT2216 and Venclexta (venetoclax) demonstrated synergy in a small cell lung cancer cell line dependent on BCL-XL and BCL-2 for survival, resulting in decreased cell viability in culture, and led to increased tumor growth inhibition in xenograft models with a mean tumor growth inhibition of 98.2% (PMID: 31792461). 31792461
Unknown unknown mantle cell lymphoma no benefit Tafasitamab-cxix Phase II Actionable In a Phase II trial, Monjuvi (tafasitamab-cxix) treatment was well-tolerated and resulted in stable disease as the best response in 50% (6/12) of patients with mantle cell lymphoma (PMID: 29444231; NCT01685008). 29444231
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 Bosutinib Phase I Actionable In a Phase I trial, Bosulif (bosutinib) demonstrated safety and limited efficacy in patients with advanced solid tumors (PMID: 22179664). 22179664
Unknown unknown Burkitt lymphoma not applicable CAR.k.28 cells Preclinical - Cell culture Actionable In a preclinical study, patient-derived CAR.k.28 cells when co-cultured with a Kappa-positive Burkitt lymphoma cell line induced tumor cell lysis in culture, and inhibited tumor growth in a cell line xenograft model (PMID: 16926291). 16926291
Unknown unknown lung non-small cell carcinoma not applicable Cabozantinib + Erlotinib Phase Ib/II Actionable In a Phase Ib/II trial, Cometriq (cabozantinib) and Tarceva (erlotinib) combination treatment resulted in no response (0/13) in patients with non-small cell lung carcinoma that had progressed during treatment with Tarceva (erlotinib) in Phase II, despite an objective response rate of 8.2% (5/61) in Phase I (PMID: 28352985). 28352985
Unknown unknown Waldenstroem's macroglobulinemia not applicable Oprozomib Phase II Actionable In a Phase II trial, Oprozomib (ONX 0912) treatment resulted in an objective response rate of 71.4% and 47.1% in the 2/7 and 5/14 cohorts of patients with Waldenstroem's macroglobulinemia (n=31) (PMID: 31142508; NCT01416428). 31142508
Unknown unknown triple-receptor negative breast cancer not applicable KW-2450 Preclinical Actionable In a preclinical study, KW-2450 inhibited the growth of triple-negative breast cancer cells in culture and in xenograft models (PMID: 26443806). 26443806
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 sarcoma not applicable Pembrolizumab Phase II Actionable In a Phase II trial, treatment with Keytruda (pembrolizumab) resulted in a progression-free survival (PFS) of 18 weeks and an objective response rate of 18% (7/40) in patients with soft tissue sarcoma, including four with pleomorphic sarcoma, two with liposarcoma, and one with synovial sarcoma, and PFS of 8 weeks and an objective response rate of 5% (2/40) in patients with bone sarcoma, including one with osteosarcoma and one with chondrosarcoma (PMID: 28988646; NCT02301039). detail... 28988646
Unknown unknown stomach cancer not applicable Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) improved progression free survival compared to placebo in patients with refractory advanced oesophagogastric cancer (J Clin Oncol 33, 2015 (suppl; abstr 4003)). detail...
Unknown unknown leiomyosarcoma not applicable Dasatinib Phase II Actionable In a Phase II clinical trial, 13% (6/42) of patients with leiomyosarcoma demonstrated clinical benefit with a median progression free survival of 2.2 months when treated with Sprycel (dasatinib) but were below levels considered to result from drug activity (PMID: 26710211). 26710211
Unknown unknown glioblastoma multiforme not applicable Olaparib + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, treatment with Lynparza (olaparib) enhanced the sensitivity of glioblastoma cell lines to radiation therapy in culture, demonstrating greater decreased cell survival when compared to cells not treated with Lynparza (olaparib) (PMID: 32347934). 32347934
Unknown unknown Ewing sarcoma of bone not applicable Dasatinib Phase II Actionable In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in Ewing sarcoma patients (n=17) was suspended due to lack of drug activity (PMID: 26710211). 26710211
Unknown unknown gastric adenocarcinoma not applicable Paclitaxel + Tegafur-gimeracil-oteracil Potassium Phase III Actionable In a Phase III trial, combining intraperitoneal Taxol (paclitaxel) with TS-1 (tegafur-gimeracil-oteracil potassium) and Taxol did not show statistical improvement of overall survival, but did demonstrated clinical efficacy compared to TS-1 and cisplatin combination in gastric adenocarcinoma patients with peritoneal metastasis (J Clin Oncol 34, 2016 (suppl; abstr 4014)). detail...
Unknown unknown hepatocellular carcinoma not applicable Ramucirumab FDA approved Actionable In a Phase III trial (REACH-2) that supported FDA approval, Cyramza (ramucirumab) treatment resulted in significantly improved overall survival (8.7 vs 7.3 months, HR=0.71, p=0.0199) and progression-free survival (2.8 vs 1.6 months, HR=0.452, p<0.0001) in hepatocellular carcinoma patients with baseline AFP of 400 ng/ml or higher, and progressed on or were intolerant to Nexavar (sorafenib) (PMID: 30665869; NCT02435433). 30665869 detail...
Unknown unknown hepatocellular carcinoma not applicable Ramucirumab Phase III Actionable In a Phase III trial (REACH), treatment with Cyramza (ramucirumab) did not result in improved overall survival (OS) compared to placebo (9.2 vs. 7.6 months; HR 0.87 [95% CI 0.72-1.05]; p=0.14) in patients with hepatocellular carcinoma (PMID: 26095784). However, potential benefit on OS (HR 0.80, p=0.06) was observed in patients with a Child-Pugh score of 5 (PMID: 27657674; NCT01140347). 26095784 27657674
Unknown unknown gastrointestinal stromal tumor not applicable Imatinib Clinical Study Actionable In a retrospective study of 16 Phase I trials, treatment with kinase inhibitors including Gleevec (imatinib mesylate), Sutent (sunitinib), or Stivarga (regorafenib) resulted in stable disease in 47.6% (10/21) and partial response in 19% (4/21) of patients with gastrointestinal stromal tumors (PMID: 27842521). 27842521
Unknown unknown Hodgkin's lymphoma not applicable Camidanlumab Tesirine Phase I Actionable In a Phase I trial, Camidanlumab Tesirine treatment resulted in an objective response rate of 63.6% (14/22) and a complete response rate of 27.3% (6/22) in Hodgkin's lymphoma patients (Blood 2017 130(Suppl 1):1510; NCT02432235). detail...
Unknown unknown head and neck squamous cell carcinoma not applicable Cisplatin + Fluorouracil + Pembrolizumab FDA approved Actionable In a Phase III trial (KEYNOTE-048) that supported FDA approval, Keytruda (pembrolizumab) in combination with platinum and Adrucil (fluorouracil) significantly improved overall survival compared to Erbitux (cetuximab) plus chemotherapy (13.0 vs 10.7 months, HR=0.77, p=0.0067) in patients with metastatic or unresectable recurrent head and neck squamous cell carcinoma (J Clin Oncol 37, no. 15_suppl (May 20 2019) 6000-6000; NCT02358031). detail... detail...
Unknown unknown B-cell lymphoma not applicable SKLB-23bb Preclinical - Cell line xenograft Actionable In a preclinical study, SKLB-23bb treatment inhibited tumor growth in B-cell lymphoma xenograft models (PMID: 29610282). 29610282
Unknown unknown ovarian carcinoma not applicable BMS-906024 Preclinical Actionable In a preclinical study, the pan-Notch inhibitor, BMS-906024, inhibited growth of ovarian carcinoma xenografts (PMID: 26005526). 26005526
Unknown unknown diffuse large B-cell lymphoma not applicable Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 6% (1/17), partial response in 6% (1/17) and stable disease in 41% (7/17) of patients with diffuse large B-cell lymphoma (PMID: 24852792). 24852792
Unknown unknown Advanced Solid Tumor not applicable Bevacizumab + Telatinib Phase I Actionable In a Phase I trial, the combination of BAY 57-9352 (telatinib) with Avastin (bevacizumab) in patients with advanced solid tumors resulted in antitumor activity, but demonstrated increasing toxicity over time (PMID: 21378200). 21378200
Unknown unknown triple-receptor negative breast cancer not applicable SN30000 Preclinical Actionable In a preclinical study, SN30000 inhibited growth of triple-receptor negative breast cancer cell lines under hypoxic culture conditions, regardless of their BRCA1 status (PMID: 25193512). 25193512
Unknown unknown triple-receptor negative breast cancer not applicable Docetaxel + ONC201 Preclinical - Cell culture Actionable In a preclinical study, the combination of ONC201 and Docefrez (docetaxel) worked synergistically to inhibit viability of triple-negative breast cancer cell lines in culture (PMID: 28424227). 28424227
Unknown unknown pancreatic cancer not applicable Pegilodecakin Phase I Actionable In a Phase I trial, AM0010 treatment in patients with pancreatic cancer resulted in 47% (8/17) of patients with stable disease, three patients with a progression free survival greater than 6 months, and a median overall survival of 5.1 months (J Clin Oncol 34, 2016 (suppl; abstr 3082). detail...
Unknown unknown alveolar soft part sarcoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 77%, median progression-free survival of 21 months, an objective response rate of 46% (n=13), and median overall survival was not reached in patients with alveolar soft part sarcoma (PMID: 29895706; NCT01878448). 29895706
Unknown unknown 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 lung non-small cell carcinoma not applicable Carboplatin + Lenvatinib + Paclitaxel Phase I Actionable In a Phase I trial of patients with advanced or metastatic NSCLC, treatment with Lenvima (lenvatinib) in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) was tolerated and demonstrated anti-tumor activity (PMID: 23860537). 23860537
Unknown unknown Waldenstroem's macroglobulinemia not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in stable disease in 2 patients with Waldenstroem's macroglobulinemia (PMID: 29475723; NCT01767766). 29475723
Unknown unknown hematologic cancer not applicable TAS4464 Preclinical - Cell culture Actionable In a preclinical study, TAS4464 demonstrated cytotoxicity in a number of hematologic cancer cell lines in culture (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr C176). detail...
Unknown unknown adrenal cortex cancer not applicable Linsitinib Phase I Actionable In a Phase I trial, Linsitinib (OSI-906) was well-tolerated and resulted in stable disease in 41% (27/66) of patients with an advanced solid tumor and a partial response in two patients with adrenocortical carcinoma (PMID: 25208878). 25208878
Unknown unknown diffuse large B-cell lymphoma not applicable GSK3368715 Preclinical - Patient cell culture Actionable In a preclinical study, GSK3368715 treatment inhibited growth of a diffuse large B-cell lymphoma (DLBCL) cell line in culture and inhibited tumor growth in xenograft models, and inhibited growth of patient-derived DLBCL cells in culture PMID: 31257072). 31257072
Unknown unknown thyroid gland papillary carcinoma not applicable Ramucirumab Phase I Actionable In a Phase I trial, Cyramza (ramucirumab) demonstrated safety and efficacy resulting in partial response or stable disease in patients with advanced solid tumors, including papillary thyroid carcinoma (PMID: 20048182). 20048182
Unknown unknown 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 multiple myeloma not applicable DT204 + Ixazomib Preclinical - Cell culture Actionable In a preclinical study, multiple myeloma cells resistant to Ninlaro (ixazomib) demonstrated re-sensitization to Ninlaro (ixazomib) in culture when treatment was combined with DT204, showing a synergistic effect and increased apoptotic activity (PMID: 27677741). 27677741
Unknown unknown 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 peritoneal carcinoma no benefit Motolimod + Pegylated liposomal-doxorubicin Phase II Actionable In a Phase II trial, the inclusion of Motolimod (VTX-2337) with Doxil (pegylated liposomal-doxorubicin) did not result in improved survival in patients with either ovarian, fallopian tube, or primary peritoneal carcinoma (PMID: 28453702; NCT01666444). 28453702
Unknown unknown ovarian cancer not applicable Itraconazole Phase I Actionable In a Phase I clinical trial, Itraconazole, in combination with chemotherapy, demonstrated safety and efficacy in patients with ovarian cancer (PMID: 24778064). 24778064
Unknown unknown urinary bladder cancer not applicable PD-0325901 + PF-04691502 Preclinical - Pdx Actionable In a preclinical study, PD-0325901, in combination with PF-04691502, delayed tumor growth in patient-derived xenograft models of bladder cancer (PMID: 24442130). 24442130
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 chronic lymphocytic leukemia not applicable Idelalisib Preclinical - Cell culture Actionable In a preclinical study, Zydelig (idelalisib) inhibited proliferation, however, also resulted in increased activation-induced cytidine deaminase (AID) expression and genomic instability in a chronic lymphocytic leukemia cell line in culture (PMID: 28199309). 28199309
Unknown unknown diffuse large B-cell lymphoma not applicable TTI-622 Case Reports/Case Series Actionable In a Phase I trial, TTI-622 treatment resulted in a partial response by week 8 and a complete response by week 36 in a patient with advanced diffuse large B-cell lymphoma (J Clin Oncol 38: 2020 (suppl; abstr 3030); NCT03530683). detail...
Unknown unknown Indication other than cancer not applicable Valproic acid FDA approved Actionable Valproic acid is FDA approved for use in patients with seizures, migraines, and bipolar disorder (FDA.gov). detail... detail...
Unknown unknown Ewing sarcoma no benefit GSK-LSD1 Preclinical - Cell culture Actionable In a preclinical study, Ewing sarcoma cell lines did not demonstrate sensitivity to GSK-LSD1 in culture (PMID: 29997151). 29997151
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 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 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 lung squamous cell carcinoma not applicable Cisplatin + 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 lung non-small cell carcinoma not applicable BMS-690514 Phase Ib/II Actionable In a Phase 1b/II trial, BMS-690514 was demonstrated to be safe and efficacious in patients with NSCLC (PMID: 23490650). 23490650
Unknown unknown ovarian cancer not applicable SNS-032 Preclinical Actionable In a preclinical study, SNS-032 decreased tumor burden and increased survival in a syngeneic mouse ovarian cancer model (PMID: 30454645). 30454645
Unknown unknown prostate cancer not applicable Abiraterone + Prednisone FDA approved Actionable In a Phase III trial (LATITUDE) that supported FDA approval, treatment with the combination of Zytiga (abiraterone) and Predisone, along with androgen-deprivation therapy, resulted in improved median overall survival (not reached vs. 34.7 months; HR=0.62) and median progression-free survival (33.0 months vs. 14.8 months; HR=0.47) compared to treatment with placebos in patients with metastatic castration-sensitive prostate cancer (PMID: 28578607; NCT01715285). detail... 28578607
Unknown unknown prostate cancer not applicable BGP-15 Preclinical - Cell culture Actionable In a preclinical study, BGP-15 induced apoptosis and inhibited growth of prostate cancer cells in culture (PMID: 22661288). 22661288
Unknown unknown Advanced Solid Tumor not applicable CHR-3996 Phase I Actionable In a Phase I trial, the HDAC inhibitor, CHR-3996, demonstrated safety and preliminary efficacy in patients with a variety of refractory solid tumors (PMID: 22553374). 22553374
Unknown unknown colorectal cancer no benefit Atezolizumab Phase III Actionable In a Phase III trial (IMblaze370), Tecentriq (atezolizumab) treatment did not improve median overall survival (7.1 vs 8.5 months, HR=1.19) compared to Stivarga (regorafenib) in patients with chemotherapy-refractory metastatic colorectal cancer, 91.7% of whom were microsatellite stable or microsatellite instability-low (Annals of Oncology, Volume 29, Issue suppl_5, 1 June 2018; NCT02788279). detail...
Unknown unknown thyroid gland cancer not applicable LY2874455 Phase I Actionable In a Phase I trial, a patient with thyroid cancer demonstrated stable disease when treated with LY2874455 (PMID: 28589492). 28589492
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 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 CLL/SLL not applicable Obinutuzumab + Venetoclax FDA approved Actionable In a Phase III trial that supported FDA approval (CLL14), treatment with the combination of Venclexta (venetoclax) and Gazyva (obinutuzumab) resulted in improved progression-free survival (PFS) compared to treatment with Gazyva (obinutuzumab) plus chlorambucil ((HR 0.35; 95% CI 0.23-0.53; P<0.0001) (J Clin Oncol 37, 2019 (suppl; abstr 7502; NCT02242942). detail... detail...
Unknown unknown breast cancer not applicable PD-0325901 Phase I Actionable In a Phase I trial, PD-0325901 demonstrated some efficacy, however, the dose administered resulted in a significant degree of toxicity (PMID: 21516509). 21516509
Unknown unknown breast cancer not applicable Triolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Triolimus led to cytotoxicity and inhbition of Ras/Raf/MAPK and PI3K/Akt/mTOR pathway signaling in breast cancer cells in culture and inhibited tumor growth in cell line xenograft models (PMID: 22896668). 22896668
Unknown unknown hepatocellular carcinoma no benefit Erlotinib + Sorafenib Phase III Actionable In a Phase III trial, the combination treatment of Nexavar (sorafenib) with Tarceva (erlotinib) compared to Nexavar (sorafenib) plus placebo did not demonstrate an improved overall survival and time to progression in patients with hepatocellular carcinoma (PMID: 25547503). 25547503
Unknown unknown pancreatic cancer not applicable 2G8 Preclinical Actionable In a preclinical study, inhibition of stromal Tgfbr2 with the murine antibody 2G8 resulted in decreased proliferation, increased apoptosis and reduced metastasis in pancreatic tumor cell line xenograft models (PMID: 25060520). 25060520
Unknown unknown multiple myeloma not applicable FT671 Preclinical - Cell line xenograft Actionable In a preclinical study, FT671 treatment blocked proliferation of multiple myeloma cells in culture and inhibited tumor growth in multiple myeloma cell line xenograft models (PMID: 29045389). 29045389
Unknown unknown stomach cancer not applicable Mogamulizumab + Nivolumab Phase I Actionable In a Phase I trial, Poteligeo (mogamulizumab-kpkc) and Opdivo (nivolumab) combination treatment demonstrated acceptable safety, resulted in an objective response rate of 0% (0/15) and a disease control rate of 27% (4/15) in immunotherapy-naive patients with advanced or metastatic gastric cancer, response occurred regardless of PD-L1, CCR4, CD8 expression levels and tumor mutational burden (PMID: 31455681; NCT02476123). 31455681
Unknown unknown 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 osteosarcoma not applicable Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 5% (1/22) of patients with osteosarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). detail...
Unknown unknown Advanced Solid Tumor not applicable LB-100 Phase I Actionable In a Phase I trial, LB-100 demonstrated safety and preliminary clinical activity in patients with advanced solid tumors, including a partial response in a pancreatic cancer patient, and stable disease for at least 4 cycles in 50% (10/20) of patients (PMID: 28039265; NCT01837667). 28039265
Unknown unknown glioblastoma multiforme not applicable Bevacizumab + SL-701 Phase II Actionable In a Phase II trial, SL-701 and Avastin (bevacizumab) combination treatment resulted in partial response in 25% (1/4) and stable disease in 75% (3/4) of patients with recurrant glioblastoma multiforme (Neuro Oncol (2016) 18 (suppl 6): vi20.). detail...
Unknown unknown glioblastoma multiforme not applicable Bevacizumab + SL-701 Phase II Actionable In a Phase II trial, SL-701 and Avastin (bevacizumab) combination treatment resulted in complete response in 7.1% (2/28), partial response in 14.3% (4/28) and stable disease in 67.9.6% (19/28) of patients with relapsed/refractory glioblastoma, with a 12-month overall survival rate of 43% (median 11.7 months) (J Clin Oncol 36, 2018 (suppl; abstr 2058); NCT02078648). detail...
Unknown unknown triple-receptor negative breast cancer not applicable Cyclophosphamide + Doxorubicin + Elenagen Phase Ib/II Actionable In a Phase Ib/II trial, chemorefractory patients with triple-receptor negative breast cancer demonstrated restored chemotherapeutic sensitivity upon sequential treatment of Elenagen and the combined therapy, Cytoxan (cyclophosphamide) and Adriamycin (doxorubicin), which resulted in stable disease (PMID: 28881846). 28881846
Unknown unknown Advanced Solid Tumor not applicable BI 836880 Phase I Actionable In a Phase I trial, BI 836880 treatment was well tolerated, and resulted in partial response in 7% (2/29) and stable disease in 31% (9/29) of patients with advanced solid tumors (J Clin Onc 2018 36:15_suppl, 12024; NCT02674152). detail...
Unknown unknown prostate cancer no benefit VT-464 Phase II Actionable In a Phase II trial, Seviteronel (VT-464) monotherapy demonstrated an undesirable toxicity profile and limited activity, with only 6% (1/17) of patients with metastatic castration-resistant prostate cancer achieved significant PSA response (PMID: 32327394). 32327394
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 breast cancer not applicable SKLB-23bb Preclinical - Cell line xenograft Actionable In a preclinical study, SKLB-23bb treatment inhibited tumor growth in breast cancer xenograft models (PMID: 29610282). 29610282
Unknown unknown 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 ovarian cancer not applicable ABT-737 + AZD8055 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of AZD8055 and Mekinist (trametinib) enhanced the sensitivity of ovarian cancer cells to ABT-737 in culture, resulting in greater apoptotic activity and cell cycle arrest when compared to Mekinist (trametinib) alone (PMID: 27980105). 27980105
Unknown unknown breast cancer not applicable GDC-0349 Preclinical - Cell line xenograft Actionable In a preclinical study, the mTOR inhibitor GDC-0349 inhibited tumor growth in cell line xenograft models of solid tumors, including breast cancer (PMID: 24900569). 24900569
Unknown unknown head and neck squamous cell carcinoma not applicable Temsirolimus Phase II Actionable In a Phase II trial, treatment with Torisel (temsirolimus) resulted in disease stabilization in 57.6% (19/33) and tumor shrinkage in 39.4% (13/33) of patients with head and neck squamous cell carcinoma (PMID: 25527417). 25527417
Unknown unknown Advanced Solid Tumor not applicable Bardoxolone methyl Phase I Actionable In a Phase I trial, Bardoxolone methyl (CDDO-Me) was well tolerated by patients with advanced solid tumors, with a complete tumor response observed in a mantle cell lymphoma patient (PMID: 25364233). 25364233
Unknown unknown cholangiocarcinoma not applicable OPB-111077 Phase I Actionable In a Phase I trial, a patient with cholangiocarcinoma demonstrated stable disease when treated with OPB-111077 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B118). detail...
Unknown unknown 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 triple-receptor negative breast cancer not applicable SMI#9-GNP Preclinical - Cell line xenograft Actionable In a preclinical study, SMI#9-GNP treatment inhibited proliferation, and colony formation, and induced apoptosis in triple-negative breast cancer cell lines harboring either wild-type or mutant BRCA1 in culture, and inhibited tumor growth in cell line xenograft models (PMID: 30242094). 30242094
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 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 Advanced Solid Tumor not applicable Metformin + Temsirolimus Phase I Actionable In a Phase I clinical trial, the combination of Torisel (temsirolimus) and Glucophage (metformin) demonstrated safety and resulted in a clinical benefit rate of 22% in patients with advanced or refractory tumors, with 4/18 patients achieving stable disease for greater than 6 cycles (PMID: 27014780). 27014780
Unknown unknown adrenocortical carcinoma not applicable Avelumab Phase Ib/II Actionable In a Phase Ib trial (JAVELIN), Bavencio (avelumab) treatment demonstrated safety and some anti-tumor activity in patients with metastatic adrenocortical carcinoma, resulting in an overall response rate of 6% (3/50, all partial responses), stable disease in 42% (21/50), a median progression-free survival (PFS) of 2.6 months, 6 mo. and 12 mo. PFS rates of 20.9% and 8.7% respectively, a median overall survival (OS) of 10.6 mo, and a 1-year OS of 43.4% (PMID: 30348224; NCT01772004). 30348224
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 ovarian cancer not applicable Camptothecin + PV1019 Preclinical - Cell culture Actionable In a preclinical study, treatment with PV1019 enhanced the anti-tumor effects of Camptothecin in ovarian cancer cells in culture, demonstrating greater growth inhibition (PMID: 19741151). 19741151
Unknown unknown endometrial cancer not applicable ONC201 Clinical Study Actionable In a clinical case study, a patient with endometrial cancer demonstrated stable disease for 42 weeks and continued regression of metastatic lesions in the lung when treated with ONC201 (TIC-10) (PMID: 28331050). 28331050
Unknown unknown primary mediastinal B-cell lymphoma not applicable Pembrolizumab FDA approved Actionable In a Phase II trial (KEYNOTE-170) that supported FDA approval, Keytruda (pembrolizumab) treatment resulted in an objective response rate of 45% (24/53) in patients with primary mediastinal large B-cell lymphoma, with a complete response rate of 13% (7/53), and a median progression-free survival of 5.5 months (ASH Annual Meeting Dec 2018, Session 626, abstract 228; NCT02576990). detail... detail... detail...
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 pancreatic ductal adenocarcinoma no benefit Atezolizumab + BKT140 Phase Ib/II Actionable In a combined analysis of 2 Phase I/II trials, Tecentriq (atezolizumab) and BL-8040 (BKT140) combination therapy demonstrated expected toxicity profile and limited efficacy, did not improve objective response rate (0/14 vs 0/15), median progression-free survival (1.6 vs 2.5 mo), or median overall survival (5.2 vs 6.8 mo) compared to control in patients with metastatic pancreatic ductal adenocarcinoma (J Clin Oncol 38, 2020 (suppl 4; abstr 712); NCT03281369, NCT03193190). detail...
Unknown unknown B-cell lymphoma not applicable Denileukin diftitox + Temsirolimus Preclinical - Cell culture Actionable In a preclinical study, Torisel (temsirolimus) enhanced the efficacy of Ontak (denileukin diftitox) in human B-cell lymphoma cells, resulting in decreased cell viability in culture (PMID: 27737881). 27737881
Unknown unknown glioblastoma multiforme not applicable JR-AB2-011 Preclinical - Cell line xenograft Actionable In a preclinical study, JR-AB2-011 resulted in tumor growth inhibition, increased apoptotic activity, and improved survival of glioblastoma cell line xenograft models (PMID: 28453552). 28453552
Unknown unknown colorectal cancer not applicable TP-1454 + unspecified CTLA4 antibody Preclinical Actionable In a preclinical study, TP-1454 treatment combined with an anti-CTLA4 antibody inhibited tumor growth and increased survival in a syngeneic mouse model of colorectal cancer (Mol Cancer Ther 2019;18(12 Suppl):Abstract nr B080). detail...
Unknown unknown malignant glioma not applicable NEO212 Preclinical Actionable In a preclinical study, treatment with NEO212 resulted in decreased cell migration and invasion, thereby slowing tumor progression in a glioma mouse model (PMID: 29440289). 29440289
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 lung small cell carcinoma not applicable T-3775440 Preclinical - Cell line xenograft Actionable In a preclinical study, T-3775440 treatment led to decreased cell proliferation in lung small cell carcinoma cell lines in culture, demonstrating reduced expression of INSM1 or GFI1B, and inhibited tumor growth in lung small cell carcinoma cell line xenograft models (PMID: 28667074). 28667074
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 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 ovarian cancer not applicable Onvansertib Preclinical - Cell culture Actionable In a preclinical study, Onvansertib (PCM-075) inhibited growth of ovarian cancer cell lines in culture (PMID: 32183025). 32183025
Unknown unknown glioblastoma multiforme not applicable BLZ945 Preclinical Actionable In a preclinical study, long term treatment with BLZ945 resulted in resistance in 56% of transgenic glioblastoma multiforme mouse models (PMID: 27199435). 27199435
Unknown unknown Advanced Solid Tumor not applicable Famitinib Phase I Actionable In a Phase I trial, patients with advanced solid tumors had antitumor activity in response to the receptor tyrosine kinase inhibitor, famitinib (PMID: 24043137). 24043137
Unknown unknown neuroblastoma not applicable Go 6983 Preclinical - Cell culture Actionable In a preclinical study, Go 6983 decreased growth of a neuroblastoma cell line in culture (PMID: 10209967). 10209967
Unknown unknown ovarian cancer not applicable Cabozantinib Phase II Actionable In a Phase II clinical trial, Cometriq (cabozantinib ) demonstrated safety and efficacy in patients with ovarian cancers (J Clin Oncol 29: 2011 (suppl; abstr 5008)). detail...
Unknown unknown endometrial cancer not applicable LY3023414 Phase II Actionable In a Phase II trial, patients with advanced endometrial cancer harboring a PI3K pathway mutation demonstrated a best overall response rate of 16% (4/25), a clinical benefit rate of 28% (7/25) at 12 weeks, a progression-free survival of 2.5 months, and overall survival of 9.2 months when treated with LY3023414 (PMID: 31880826; NCT01775072). 31880826
Unknown unknown lung non-small cell carcinoma not applicable Carboplatin + MEDI-575 + Paclitaxel Phase Ib/II Actionable In a Phase Ib/II trial, MEDI-575, Paraplatin (carboplatin), and Taxol (paclitaxel) combination treatment resulted in increased toxicity, and did not confer benefit over Paraplatin (carboplatin) plus Taxol (paclitaxel) in non-small cell lung carcinoma (NSCLC) patients (n=99), with a progression-free survival of 4.6 months vs. 5.5 months (HR=2.20, p=0.03), an overall response rate of 31.7% vs. 22.5% (p=0.49), and a median overall survival of 10 months vs. 11.8 months (HR=1.31) (PMID: 30685114). 30685114
Unknown unknown chronic lymphocytic leukemia not applicable Idelalisib + Rituximab FDA approved Actionable In a Phase III trial that supported FDA approval, Zydelig (idelalisib) and Rituxan (rituximab) combination therapy resulted in improved overall response rate (81%) and overall survival at 12 months (92%) when compared to Rituxan (rituximab) plus placebo (13% and 80%, respectively) in patients with relapsed chronic lymphocytic leukemia (PMID: 24450857; NCT01539512). detail... 24450857
Unknown unknown squamous cell carcinoma not applicable Prexasertib Phase I Actionable In a Phase I trial, Prexasertib (LY2606368) treatment resulted in partial response in 2 patients with squamous cell carcinoma (SCC), and 40% (6/15) of solid tumor patients who achieved stable disease had SCC (PMID: 27044938; NCT0115790). 27044938
Unknown unknown squamous cell carcinoma not applicable Prexasertib Phase Ib/II Actionable In a Phase Ib trial, Prexasertib (LY2606368) demonstrated safety and resulted in a clinical benefit rate at 3 months of 29% (29/101) in squamous cell carcinoma patients, including patients with squamous cell carcinoma of the anus, head and neck squamous cell carcinoma, and squamous non-small cell lung cancer (PMID: 29643063; NCT0115790). 29643063
Unknown unknown breast cancer not applicable F14512 Preclinical - Cell line xenograft Actionable In a preclinical study, F14512 induced tumor regression in breast cancer cell line xenograft models (PMID: 19047165). 19047165
Unknown unknown fallopian tube 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 Gedatolisib Phase I Actionable In a Phase I trial, Gedatolisib (PF-05212384) demonstrated safety and efficacy, which resulted in antitumor activity and stable disease greater than six months in 10.4% (8/27) of patients with solid malignant tumors (PMID: 25652454). 25652454
Unknown unknown mantle cell lymphoma not applicable Mivavotinib Case Reports/Case Series Actionable In a Phase I trial, Mivavotinib (TAK-659) treatment resulted in an objective response rate of 33% (1/3, 1 partial responses) in patients with relapsed or refractory mantle cell lymphoma, with a median duration of response of 109 days (PMID: 32327472; NCT02000934). 32327472
Unknown unknown Advanced Solid Tumor not applicable Belinostat Phase I Actionable In a Phase I trial, Beleodaq (belinostat) demonstrated safety and promoted stable disease in 39% (18/46) of patients with a variety of solid tumors (PMID: 18245542). 18245542
Unknown unknown renal cell carcinoma not applicable AGS16F Phase I Actionable In a Phase I trial, treatment with AGS16F (AGS-16CSF) at the recommended phase 2 dose demonstrated safety and resulted in a partial response in 23% (3/13) of patients with metastatic renal cell carcinoma including 2 patients with clear cell and 1 patient with papillary histology, and a disease control rate of 92% (12/13) (PMID: 29848572). 29848572
Unknown unknown liposarcoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 63%, median progression-free survival of 5.6 months, an objective response rate of 7.7% (n=13), and a median overall survival of 13 months in patients with liposarcoma (PMID: 29895706; NCT01878448). detail... 29895706
Unknown unknown follicular lymphoma not applicable Copanlisib FDA approved Actionable In a Phase II trial that supported FDA approval, Aliqopa (copanlisib) treatment in patients with follicular lymphoma resulted in an objective tumor response rate of 58.7% (61/104) including 14.4% (15/61) patients experiencing a complete response and 44.2% (46/61) patients experiencing a partial response, stable disease in 33.7% (35/104) of patients, and a duration of response of 370 days (Journal of Clinical Oncology 35, no. 15_suppl (May 2017) 7535-7535; NCT01660451). detail... detail...
Unknown unknown follicular lymphoma not applicable Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 20% (2/10), partial response in 20% (2/10) and stable disease in 60% (6/10) of patients with follicular lymphoma (PMID: 24852792). 24852792
Unknown unknown breast cancer not applicable Azacitidine + Entinostat Phase II Actionable In a Phase II trial, the combination of Vidaza (azacitidine) and Entinostat (MS-275) did not reach its primary endpoint for either cohort of breast cancer patients, TNBC or hormone-resistant, however, the optional continuation phase resulted in one patient achieving a partial response (PMID: 27979916). 27979916
Unknown unknown lymphoma not applicable ACP-319 Preclinical - Cell culture Actionable In a preclinical study, ACP-319 treatment blocked cell proliferation in multiple lymphoma cell lines in culture (Eur J of Cancer, Dec 2016, 69;1, S39-S40). detail...
Unknown unknown lung small cell carcinoma not applicable Roniciclib Phase I Actionable In a Phase I trial, treatment with Roniciclib (BAY 1000394) at the RP2D reduced PCNA expression and resulted in a disease control rate of 17.4% (n=23) in patients with small cell lung cancer (PMID: 28463960; NCT01188252). 28463960
Unknown unknown malignant peripheral nerve sheath tumor not applicable Everolimus Preclinical - Cell line xenograft Actionable In a preclinical study, malignant peripheral nerve sheath cell line xenograft models treated with Afinitor (everolimus) demonstrated a 76% decrease in tumor growth (PMID: 18483311). 18483311
Unknown unknown breast cancer not applicable INCB081776 + unspecified PD-L1 antibody Preclinical Actionable In a preclinical study, INCB081776 and anti-PD-L1 therapy synergistically inhibited tumor growth in a mouse model of breast cancer (Cancer Res 2018;78(13 Suppl):Abstract nr 3759). detail...
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 glioblastoma multiforme 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 Cediranib + Olaparib Phase I Actionable In a Phase I clinical trial, the combination therapy of Cediranib (AZD-2171) and Lynparza (olaparib) demonstrated safety and efficacy in patients with ovarian cancer (J Clin Oncol 32:5s, 2014 (suppl; abstr LBA5500)). detail...
Unknown unknown glioblastoma multiforme not applicable Tanibirumab Preclinical - Cell line xenograft Actionable In a preclinical study, Tanibirumab (TTAC-0001) inhibited angiogenesis and tumor growth in human glioblastoma cell line xenograft models (PMID: 26325365). 26325365
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 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 brain glioma not applicable NT-I7 Preclinical Actionable In a preclinical study, NT-I7 treatment improved survival in a mouse model of glioma (Journal of Clinical Oncology 37, no. 15_suppl, Abs nr: e13516). detail...
Unknown unknown head and neck squamous cell carcinoma not applicable GSK1059615 Preclinical - Cell line xenograft Actionable In a preclinical study, GSK1059615 treatment in a head and neck squamous cell carcinoma cell line resulted in inhibition of both cell growth and cell proliferation and induced necrosis in culture, and inhibited tumor growth in cell line xenograft models (PMID: 28187451). 28187451
Unknown unknown lung small cell carcinoma not applicable Alisertib Preclinical - Pdx & cell culture Actionable In a preclinical study, Alisertib (MLN8237), alone and in combination with Taxol (paclitaxel), inhibited growth of small cell lung cancer cell (SCLC) lines in culture and inhibited tumor growth in primary human SCLC xenograft models (Mol Cancer Ther 2013;12(11 Suppl):A282). detail...
Unknown unknown lung small cell carcinoma not applicable Alisertib Phase II Actionable In a Phase II trial, treatment with Alisertib (MLN8237) resulted in an objective response in 21% (10/48) of patients with small cell lung cancer (PMID: 25728526). 25728526
Unknown unknown colon cancer not applicable Vorinostat Preclinical Actionable In a preclinical study, Zolinza (vorinostat) decreased the growth of colon tumors in mice, and this effect was enhanced by co-administration of Lipitor (atorvastatin) (PMID: 22161747). 22161747
Unknown unknown glioblastoma multiforme not applicable Selinexor Phase II Actionable In a Phase II trial, Selinexor (KPT-330) treatment resulted in partial response in 11% (3/27) and stable disease in 22% (6/27) of patients with glioblastoma multiforme (J Clin Oncol 34, 2016 (suppl; abstr 2077)). detail...
Unknown unknown Advanced Solid Tumor not applicable Panitumumab Phase I Actionable In a Phase I trial, Vectibix (panitumumab) demonstrated safety and anti-tumor activity in patients with advanced solid tumors (PMID: 18223225). 18223225
Unknown unknown malignant pleural solitary fibrous tumor not applicable Dasatinib Phase II Actionable In a Phase II trial, patients with solitary fibrous tumors demonstrated a median progression free survival of 2 months and five patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown prostate cancer not applicable GDC-0349 Preclinical - Cell line xenograft Actionable In a preclinical study, the mTOR inhibitor GDC-0349 demonstrated inhibition of tumor growth in cell line xenograft models of solid tumors, including prostate cancer (PMID: 24900569). 24900569
Unknown unknown breast cancer not applicable AZD7762 + VE-821 Preclinical Actionable In a preclinical study, breast cancer cells treated with VE-821 resulted in a synthetic lethal effect when combined with AZD7762, thereby demonstrating decreased cell survival in culture (PMID: 26748709). 26748709
Unknown unknown ovarian cancer not applicable Carboplatin + Ipafricept + Paclitaxel Phase Ib/II Actionable In a Phase Ib clinical trial, the combination of Ipafricept (OMP-54F28) with Paraplatin (carboplatin) and Taxol (paclitaxel) was well-tolerated and resulted in complete response in 35% (6/17), partial response in 47% (8/17) and stable disease in 18% (3/17) evaluable patients with recurrent platinum-sensitive ovarian cancer (J Clin Oncol 34, 2016 (suppl; abstr 2515)). detail...
Unknown unknown colorectal cancer not applicable GDC-0980 Preclinical - Cell line xenograft Actionable In a preclinical study, the dual PI3K/mTOR inhibitor Apitolisib (GDC-0980) reduced vascularization in cell line xenograft models of colorectal cancer (PMID: 23814482). 23814482
Unknown unknown cervical cancer not applicable ETP-46464 + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, ETP-46464 increased the sensitivity of cervical cancer cell lines to ionizing radiation in culture (PMID: 25560806). 25560806
Unknown unknown melanoma not applicable IMC-20D7S Phase Ib/II Actionable In a Phase I/Ib trial, treatment with IMC-20D7S in patients with melanoma resulted in stable disease in 37% (10/27) and one complete response (PMID: 27797971). 27797971
Unknown unknown non-Hodgkin lymphoma not applicable Cyclophosphamide + Inotuzumab ozogamicin + Prednisone + Rituximab + Vincristine Sulfate Phase I Actionable In a Phase I trial, the combination of inotuzumab ozogamicin with R-CVP (rituximab, cyclophosphamide, Oncovin (vincristine), and prednisone) resulted in an ORR of 84% (32/38) in non-Hodgkin lymphoma patients, including a complete response in 24% (9/38) of patients (PMID: 27154915). 27154915
Unknown unknown liposarcoma not applicable MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 blocked cell proliferation of a human liposarcoma cell line in culture and repressed tumor growth in xenograft models (PMID: 26675259). 26675259
Unknown unknown 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 colorectal cancer not applicable Balsalazide + Parthenolide Preclinical - Cell culture Actionable In a preclinical study, the combination of Balsalazide and Parthenolide resulted in a synergistic effect, resulting in reduced cell viability and increased apoptotic activity in colorectal cancer cells in culture (PMID: 28108625). 28108625
Unknown unknown acute myeloid leukemia not applicable ST7612AA1 Preclinical - Cell line xenograft Actionable In a preclinical study, ST7612AA1 inhibited proliferation of acute myeloid leukemia (AML) cell lines in culture and demonstrated antitumor activity in AML cell line xenograft models (PMID: 25671299). 25671299
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 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 multiple myeloma not applicable Dexamethasone + Pomalidomide FDA approved Actionable In a Phase III trial (MM-003) that supported FDA approval, combination of Pomalyst (pomalidomide) and low-dose dexamethasone resulted in improved median progression-free survival (4.0 vs 1.9 mo, HR=0.48, p<0.0001) compared to high-dose dexamethasone in patients with multiple myeloma whose disease progressed on 2 or more therapies (PMID: 24007748; NCT01311687). 24007748
Unknown unknown follicular lymphoma not applicable Bendamustine + Rituximab + Veliparib Phase I Actionable In a Phase I trial, five patients with follicular lymphoma treated with a combination of Bendamustine, Rituxan (rituximab), and Veliparib (ABT-888) demonstrated a complete response (PMID: 28314788; NCT01326702). 28314788
Unknown unknown glioblastoma multiforme 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 Hodgkin's lymphoma not applicable Brentuximab vedotin + Dacarbazine + Doxorubicin + Vinblastine FDA approved Actionable In a Phase III trial (ECHELON-1) that supported FDA approval, Adcetris (brentuximab vedotin) in combination with Deticene (dacarbazine), Adriamycin (doxorubicin), and Velban (vinblastine) improved modified progression-free survival rate (82.1% vs 77.2%, HR=0.77, p=0.04) compared to the combination of Adriamycin (doxorubicin), Blenoxane (Bleomycin), Velban (vinblastine) and Deticene (dacarbazine) in patients with untreated stage III or IV classical Hodgkin's lymphoma (PMID: 29224502; NCT01712490). 29224502 detail...
Unknown unknown lung non-small cell carcinoma not applicable Pazopanib Phase II Actionable In a Phase II trial, Votrient (pazopanib) treatment resulted in 86% (30/35) of patients with non-small cell lung cancer experiencing a decrease in tumor size, including one patient with a greater than 50% reduction, and resulted in a partial response based on RECIST criteria in three patients (PMID: 20516450). 20516450
Unknown unknown acute myeloid leukemia not applicable MK-8242 Phase I Actionable In a Phase I trial, MK-8242 demonstrated safety and some preliminary efficacy in patients with refractory or recurrent acute myeloid leukemia, with 3 out of 24 evaluable patients demonstrating an objective response (PMID: 27544076). 27544076
Unknown unknown synovial sarcoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 75%, median progression-free survival of 7.7 months, an objective response rate of 17% (n=47), and a median overall survival of 12 months in patients with synovial sarcoma (PMID: 29895706; NCT01878448). 29895706
Unknown unknown osteosarcoma not applicable V158411 Preclinical - Cell culture Actionable In a preclinical study, V158411 inhibited Chk1 autophosphorylation and induced DNA damage and apoptosis in an osteosarcoma cell line in culture (PMID: 27829224). 27829224
Unknown unknown lung non-small cell carcinoma no benefit Bevacizumab + Carboplatin + Cixutumumab + Paclitaxel Phase II Actionable In a Phase II trial, the combination therapy of Avastin (bevacizumab), Paraplatin (carboplatin), Taxol (paclitaxel), and Cixutumumab resulted in greater toxicity and did not improve overall survival when compared to Avastin (bevacizumab), Paraplatin (carboplatin), and Taxol (paclitaxel) without Cixutumumab in non-small cell lung carcinoma patients (PMID: 28950351; NCT00955305). 28950351
Unknown unknown multiple myeloma not applicable INCB054329 + Ruxolitinib Preclinical - Cell line xenograft Actionable In a preclinical study, INCB054329 and Jakafi (ruxolitinib) demonstrated synergy in a myeloma cell line in culture, resulting in decreased viability, increased apoptosis, and increased inhibition of STAT3 phosphorylation, and treatment with the combination of INCB054329 and Jakafi (ruxolitinib) resulted in increased efficacy in myeloma cell line xenograft models over either agent alone, with tumor regressions in 5/8 animals (PMID: 30206163). 30206163
Unknown unknown bladder urothelial carcinoma not applicable VAX014 Preclinical Actionable In a preclinical study, VAX014 induced cell-killing in dissociated mouse and human urothelial carcinoma cell lines in culture, and inhibited tumor growth and improved survival in a syngeneic bladder cancer model (PMID: 27919942). 27919942
Unknown unknown Her2-receptor negative breast 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 hormone receptor-positive, ERBB2 (HER2)-negative breast cancer patients who completed standard treatment, and all patients (n=8) remained alive 2 years after initial immunization, with a median relapse-free survival not reached; however, T cell response did not correlate with tumor Folr1 expression level (PMID: 29545464; NCT01606241). 29545464
Unknown unknown colon cancer not applicable Panobinostat Preclinical Actionable In a preclinial study, Faridak (panobinostat) enhanced the cytotoxicity of TRAIL, tumor necrosis factor-related apoptosis-inducing ligand, in colon cancer cell lines (PMID: 21965751). 21965751
Unknown unknown 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 lung non-small cell carcinoma not applicable ALT-803 + Nivolumab Phase Ib/II Actionable In a Phase Ib trial, the addition of ALT-803 to Opdivo (nivolumab) treatment at the time of relapse resulted in an objective response in two patients with non-small cell lung carcinoma, demonstrating antitumor activity, and a third patient who initially responded to the combination, progressed, enrolled on a trial due to a KRAS mutation, but did not respond, and was then retreated with the ALT-803 and Opdivo (nivolumab) combination, demonstrating a 100% decrease in the target lesion (PMID: 29628312). 29628312
Unknown unknown melanoma not applicable NMS-P715 Preclinical - Cell line xenograft Actionable In a preclinical study, NMS-P715 inhibited tumor growth in melanoma cell line xenograft models (PMID: 21159646). 21159646
Unknown unknown renal cell carcinoma not applicable Axitinib + X4P-001 Phase I Actionable In a Phase I trial, X4P-001 in combination with Axitinib, displayed safety and efficacy in patients with renal cell carcinoma (AACR; Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B201, NCT02667886). detail...
Unknown unknown chronic myeloid leukemia not applicable Nilotinib + Tazemetostat Preclinical Actionable In a preclinical study, treatment with the combination of Tasigna (Nilotinib) and EPZ-6438 resulted in decreased levels of leukemic cells and progenitors in primary chronic myeloid leukemia cell xenograft models, with increased efficacy compared to Tasigna (Nilotinib) alone (PMID: 27630125). 27630125
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 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 Advanced Solid Tumor not applicable Vorolanib Phase I Actionable In a Phase I trial, Vorolanib (X-82) was well tolerated and demonstrated preliminary efficacy, resulting in a complete response in 2% (1/49), a partial response in 2% (1/49), and stable disease in 51% (25/49) of patients with advanced solid tumors, with a median progression-free survival of 2 months (PMID: 30478190; NCT01296581). 30478190
Unknown unknown lung non-small cell carcinoma not applicable Pimitespib Phase I Actionable In a Phase I trial, TAS-116 treatment resulted in partial response in 2 patients with non-small cell lung carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 2546)). detail...
Unknown unknown triple-receptor negative breast cancer not applicable ONC201 Preclinical - Cell line xenograft Actionable In a preclinical study, ONC201 inhibited viability of several triple-negative breast cancer (TNBC) cell lines in culture, demonstrating variable pro-apototic and anti-proliferative activity, and inhibited tumor growth in TNBC cell line xenograft models (PMID: 28424227). 28424227
Unknown unknown gastric adenocarcinoma not applicable Minnelide Preclinical - Cell line xenograft Actionable In a preclinical study, Minnelide treatment inhibited tumor growth and induced regression in gastric adenocarcinoma cell line xenograft models (PMID: 28192510). 28192510
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 ovarian cancer not applicable BAY1161909 Preclinical Actionable In a preclinical study, BAY1161909 demonstrated moderate efficacy in ovarian cancer xenograft models (PMID: 26832791). 26832791
Unknown unknown hematologic cancer not applicable Domatinostat Phase I Actionable In a Phase I trial, treatment with Domatinostat (4SC-202) demonstrated safety and resulted in stable disease in 75% (18/24) and objective response in 2 patients (1 complete response and 1 partial response) with advanced hematological malignancies (PMID: 30347469; NCT01344707). 30347469
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 B-cell lymphoma not applicable KTE-C19 Phase I Actionable In a Phase I trial, KTE-C19 treatment resulted in complete response in 67% (2/3) and partial response in 33% (1/3) of evaluable patients with refractory aggressive B-cell non-Hodgkin lymphoma after 1 month of treatment (Blood 2015 126:3991). detail...
Unknown unknown B-cell lymphoma not applicable KTE-C19 Phase Ib/II Actionable In a Phase I/II trial supporting FDA approval, Yescarta (KTE-C19) treatment resulted in an objective response rate of 82% (92/110), with complete response in 54% (60/110) and partial response in 28% (31/110) of patients with refractory B-cell non-Hodgkin lymphoma (Cancer Res 2017;77(13 Suppl):Abstract nr CT019). detail...
Unknown unknown synovial sarcoma not applicable Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (5.6 vs 1.0 months), but no difference in overall survival (HR=0.87, p=0.79) compared to placebo in patients with synovial sarcoma (PMID: 27751846). 27751846
Unknown unknown skin melanoma not applicable Toripalimab Phase II Actionable In a Phase II trial (POLARIS-01), Toripalimab (JS001) resulted in an objective response rate of 17.3% (22/127, 1 CR, 21 PR) and a disease control rate of 57.5% in melanoma patients, with a median progression-free survival (mPFS) of 3.6 mo and a median overall survival (mOS) of 22.2 mo, better ORR (31.0%, 14.0%, 0%), mPFS (5.5, 3.2, 1.9 mo), and mOS (not reached, 16.9, 10.3 mo) were observed in non-acral cutaneous melanoma (n=29) than in acral (n=50) and mucosal (n=22) subtypes (PMID: 32321714; NCT03013101). 32321714
Unknown unknown Advanced Solid Tumor not applicable AZD3965 Phase I Actionable In a Phase I trial, AZD3965 treatment demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Annals of Oncology, Volume 29, Issue suppl_3, 1 March 2018, abstract 500). detail...
Unknown unknown cervix carcinoma not applicable Bevacizumab + Cisplatin + Paclitaxel FDA approved Actionable In a Phase III trial that supported FDA approval, the addition of Avastin (bevacizumab) to Platinol (cisplatin) and Taxol (paclitaxel) chemotherapy resulted in improved overall survival and progression-free survival compared to chemotherapy alone in patients with cervical cancer (PMID: 25281440, PMID: 24552320). detail... 25281440 24552320
Unknown unknown myelodysplastic syndrome no benefit Rigosertib Sodium Phase III Actionable In a Phase III trial (ONTIME), Rigosertib (ON 01910.Na) did not improve median overall survival compared to best supportive care (8.2 vs 5.9 months, HR=0.87, p=0.33) in patients with myelodysplastic syndrome (PMID: 26968357; NCT01241500). 26968357
Unknown unknown esophageal cancer not applicable Mogamulizumab + Nivolumab Phase I Actionable In a Phase I trial, Poteligeo (mogamulizumab-kpkc) and Opdivo (nivolumab) combination treatment demonstrated acceptable safety, resulted in an objective response rate of 13% (3/15, 2 partial responses) and a disease control rate of 33% (5/15) in immunotherapy-naive patients with advanced or metastatic esophageal cancer, response occurred regardless of PD-L1, CCR4, CD8 expression levels and tumor mutational burden (PMID: 31455681; NCT02476123). 31455681
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 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 diffuse large B-cell lymphoma not applicable Tomivosertib Preclinical Actionable In a preclinical study, eFT508 inhibited proliferation of diffuse large B-cell lymphoma cell lines in culture (Blood Dec 2015, 126 (23) 1554). detail...
Unknown unknown smoldering myeloma not applicable Lenalidomide + PVX-410 Phase Ib/II Actionable In a Phase I/IIa clinical trial, combination therapy with PVX-410 and Revlimid (lenalidomide) was well-tolerated, and resulted in a partial response in 11% (1/9), minimal response in 44% (4/9), and stable disease in 44% (4/9) of patients with smoldering multiple myeloma with moderate/high risk of progression, and the magnitude of the immune response observed was significantly larger than in patients treated with PVX-410 alone (PMID: 30128502; NCT01718899). 30128502
Unknown unknown neuroblastoma not applicable 131I-MIBG + Vorinostat Phase I Actionable In a Phase I clinical trial, the combination of 131I-MIBG with Zolinza (vorinostat) as a radiosensitizer demonstrated preliminary efficacy at the recommended Phase II dose, with in an overall objective response rate of 17% (1/6) and MIBG response rate of 67% (4/6) in patients with neuroblastoma (PMID: 25695691). 25695691
Unknown unknown head and neck squamous cell carcinoma not applicable Prexasertib Phase I Actionable In a Phase I trial, Prexasertib (LY2606368) treatment resulted in partial response in a patient with head and neck squamous cell carcinoma (PMID: 27044938; NCT0115790). 27044938
Unknown unknown head and neck squamous cell carcinoma not applicable Prexasertib Phase Ib/II Actionable In a Phase Ib trial, treatment with Prexasertib (LY2606368) resulted in an overall response rate of 5% (3/57, all partial responses), clinical benefit rate (complete response+partial response+stable disease) of 49% (28/57), and a median progression-free survival of 1.6 months in patients with head and neck squamous cell carcinoma (PMID: 29643063; NCT0115790). 29643063
Unknown unknown head and neck squamous cell carcinoma not applicable Prexasertib Preclinical - Cell culture Actionable In a preclinical study, head and neck squamous cell carcinoma cell lines, either human papilloma virus positive or negative, demonstrated decreased cell proliferation in culture when treated with Prexasertib (LY2606368) (PMID: 28138028). 28138028
Unknown unknown glioblastoma multiforme not applicable G-TPP + WEHI-539 Preclinical - Patient cell culture Actionable In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the Bcl-xL inhibitor WEHI-539 synergistically inhibited viability of established lines and patient-derived glioblastoma cells in culture (PMID: 28522750). 28522750
Unknown unknown ovarian cancer not applicable Brivanib Phase II Actionable In a Phase II trial, treatment with Brivanib (BMS-540215) demonstrated safety and resulted in a median progression-free survival of 4.0 months in ovarian cancer patients, compared to 2.0 months with placebo, and FGF2 expression was not found to be a predictive biomarker for response (PMID: 31522033; NCT00633789). 31522033
Unknown unknown gastrointestinal neuroendocrine tumor not applicable Pazopanib Clinical Study Actionable In a clinical study, Votrient (pazopanib) treatment in patients with gastroenteropancreatic neuroendocrine tumors resulted in an overall response rate of 24% (19/124), stable disease in 39.5% (49/124), a progression free survival of 36% at six months, and a median overall survival of 10.2 months (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 446P). detail...
Unknown unknown mantle cell lymphoma not applicable Venetoclax Phase I Actionable In a Phase I trial, Venclexta (venetoclax) treatment resulted in an overall response rate of 60% (12/20, 4 complete response), with a median progression-free survival of 2.6 months, and a median overall survival of 4.3 months in patients with relapsed/refractory mantle cell lymphoma who failed BTK inhibitor therapy (EHA23, June 2018, abstract S855). detail...
Unknown unknown pancreatic cancer not applicable Gemcitabine + Nab-paclitaxel + Necuparanib Phase I Actionable In a Phase I trial, combination treatment consisted of Gemzar (gemcitabine), Abraxane (nab-paclitaxel), and Necuparanib resulted in partial response in 50% (8/16), stable disease in 38% (6/16), and median overall survival of 16.0 months in patients (pts) with metastatic pancreatic cancer (J Clin Oncol 34, 2016 (suppl; abstr 4117)). detail...
Unknown unknown lung non-small cell carcinoma not applicable Docetaxel + Nintedanib Phase III Actionable In a Phase III clinical trial, the combination of Ofev (nintedanib) and Taxotere (docetaxel) resulted in improved progression-free survival and overall survival compared to placebo plus Taxotere (docetaxel) in non-small cell lung cancer patients (PMID: 24411639). 24411639
Unknown unknown smoldering myeloma not applicable PVX-410 Phase Ib/II Actionable In a Phase I/IIa clinical trial, PVX-410 treatment was well-tolerated, elicited immune responses, and resulted in a best response of stable disease in all patients (n=12) with smoldering multiple myeloma with moderate/high risk of progression and during a 12-month follow up period 41.7% (5/12) of patients had disease progression (PMID: 30128502; NCT01718899). 30128502
Unknown unknown glioblastoma multiforme not applicable BLZ945 + Buparlisib Preclinical Actionable In a preclinical study, the combination of Buparlisib (BKM120) and BLZ945 compared to BLZ945 alone resulted in enhanced survival and tumor regression in transgenic mouse models of glioblastoma (PMID: 27199435). 27199435
Unknown unknown hematologic cancer not applicable APR-246 Phase I Actionable In a Phase I clinical trial, APR-246 demonstrated safety and preliminary clinical activity in patients with hematological cancers (PMID: 22965953). 22965953
Unknown unknown esophagus squamous cell carcinoma not applicable Fluorouracil + OBP-801 Preclinical Actionable In a preclinical study, OBP-801, in combination with 5-FU and radiation, enhanced growth inhibition of esophageal squamous carcinoma cells in culture (PMID: 24854104). 24854104
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 Birabresib Preclinical - Cell line xenograft Actionable In a preclinical study, Birabresib (OTX015) treatment inhibited tumor growth in a cell line xenograft model of prostate cancer (PMID: 27274052). 27274052
Unknown unknown prostate adenocarcinoma not applicable VT-464 Phase I Actionable In a Phase I study, VT-464 treatment led to safety and tolerability in patients with castration-resistant prostate adenocarcinoma and resulted in a best response of stable disease in 58% (10/17), a partial response in 6% (1/17), and no complete responses (PMID: 30012563; NCT02361086). 30012563
Unknown unknown chronic lymphocytic leukemia not applicable Lenalidomide + Ofatumumab Phase I Actionable In a Phase I trial, the combination treatment of Revlimid (lenalidomide) and Arzerra (ofatumumab) in patients with chronic lymphocytic leukemia resulted in an overall response rate of 71% (24/34), in which 24% (8/34) experienced a complete remission and 47% (16/34) experienced a partial response (PMID: 26733610). 26733610
Unknown unknown diffuse large B-cell lymphoma not applicable EBI-2511 Preclinical - Cell line xenograft Actionable In a preclinical study, EBI-2511 treatment in diffuse large B-cell lymphoma cell line xenograft models resulted in tumor growth inhibition and a 97% decrease in tumor size (PMID: 29456795). 29456795
Unknown unknown lung non-small cell carcinoma not applicable Chiauranib Preclinical - Cell line xenograft Actionable In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a non-small cell lung cancer cell line xenograft model (PMID: 28004478). 28004478
Unknown unknown stomach cancer no benefit Irinotecan + Nimotuzumab Phase II Actionable In a Phase II trial, addition of Nimotuzumab to Camptosar (irinotecan) did not significantly improve median progression-free survival (73 vs 85 days), median overall survival (250.5 vs 232.0 days) and response rate (18.4 vs 10.3 %) compared to Camptosar alone in gastric cancer patients (PMID: 25185971). 25185971
Unknown unknown subependymal giant cell astrocytoma not applicable Everolimus FDA approved Actionable In a Phase III trial (EXIST-1) that supported FDA approval, Afinitor (everolimus) treatment resulted in a 50% or more tumor reduction in 35% (27/78) of adult and pediatric patients diagnosed with tuberous sclerosis complex and had subependymal giant cell astrocytoma, compared to 0% (0/39) in the placebo group (PMID: 23158522; NCT00789828). 23158522 detail...
Unknown unknown endometrial cancer no benefit Trametinib + Uprosertib Phase I Actionable In a Phase I trial, Mekinist (trametinib) and Uprosertib (GSK2141795) combination treatment demonstrated increased toxicity and limited efficacy, resulted in no response (0/14) at RP2D dose and 1 response (8.3%, 1/12) at reduced dose in patients with recurrent endometrial cancer, with progression-free survival at 6 months in 14% and 25% of the patients, respectively (PMID: 31623857). 31623857
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 chronic lymphocytic leukemia not applicable Safingol Preclinical - Patient cell culture Actionable In a preclinical study, treatment with Kynacyte (safingol) resulted in increased apoptosis of patient-derived chronic lymphocytic leukemia cells in culture (PMID: 15929099). 15929099
Unknown unknown breast cancer not applicable CDD111 Preclinical Actionable In a preclinical study, CDD111 did not inhibit growth of a mouse breast cancer cell line in culture, however, reduced metastatic tumor growth in bone and visceral organs and increased survival in a mouse breast cancer cell line metastasis model (PMID: 30093561). 30093561
Unknown unknown breast cancer not applicable BO-112 Preclinical - Cell culture Actionable In a preclinical study, BO-112 treatment induced cytotoxicity in human breast cancer cell lines and inhibited viability of mouse triple-negative breast cancer (TNBC) cells in culture, and intratumoral delivery of BO-112 reduced tumor growth in a syngeneic mouse model of TNBC (PMID: 31046839). 31046839
Unknown unknown clear cell renal cell carcinoma not applicable Ascrinvacumab Phase I Actionable In a Phase I trial, a patient with renal clear cell carcinoma demonstrated a partial response for 325 days when treated with PF-03446962 (PMID: 26655846). 26655846
Unknown unknown lung small cell carcinoma not applicable Cisplatin + Etoposide + Roniciclib Phase II Actionable In a Phase II trial, the combination of Roniciclib (BAY1000394) plus chemotherapy regimen, Paraplatin (carboplatin) or Platinol (cisplatin) and Vepesid (etoposide) (n=71), did not meet its primary endpoint for progression-survival in patients with small cell lung cancer (4.9 mo vs 5.5 mo) when compared to placebo plus chemotherapy (n=71), and showed an unfavorable risk-benefit profile, therefore, leading to premature termination of the study (PMID: 30677506; NCT02161419). 30677506
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 pancreatic ductal adenocarcinoma not applicable PI-3065 Preclinical Actionable In a preclinical study, treatment with the PIK3-delta inhibitor PI-3065 resulted in increased host anti-tumor immune response and led to prolonged survival and decreased metastasis in an mouse model of KRAS G12D and TP53 R175H-expressing pancreatic ductal adenocarcinoma (PMID: 24919154). 24919154
Unknown unknown melanoma not applicable Ipilimumab + Nivolumab Phase II Actionable In a Phase II trial, the combination of Opdivo (nivolumab) and Yervoy (ipilimumab) resulted in improved overall response rate (ORR) and pathologic complete response rate (pCR) compared to Opdivo (nivolumab) monotherapy in patients with stage III or IV melanoma, with a ORR of 73% (8/11) and pCR of 45% (5/11), however, demonstrated higher toxicity (PMID: 30297909; NCT02519322). 30297909
Unknown unknown melanoma not applicable Ipilimumab + Nivolumab FDA approved Actionable In a Phase III trial (CheckMate 067) that supported FDA approval, combination of Opdivo (nivolumab) and Yervoy (ipilimumab) and Opdivo (nivolumab) alone demonstrated improved efficacy compared to Yervoy (ipilimumab) in patients with untreated advanced melanoma, resulted in a median overall survival unreached in the combination arm, 36.9 and 19.9 months in nivolumab and ipilimumab monotherapy arms, and a median progression-free survival of 11.5, 6.9, and 2.9 months respectively (PMID: 30361170; NCT01844505). detail... detail... 30361170
Unknown unknown lung non-small cell carcinoma not applicable Erlotinib + Everolimus Phase I Actionable In a Phase I trial, Afinitor (everolimus) demonstrated safety and some efficacy in combination with Tarceva (erlotinib) in patients with advanced NSCLC (PMID: 22968184). 22968184
Unknown unknown 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 acute monocytic leukemia not applicable Bortezomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, EDO-S101 and Velcade (bortezomib) worked synergistically to decrease viability of an acute monocytic leukemia cell line in culture (PMID: 28753594). 28753594
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 rhabdoid cancer not applicable BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 100% (3/3) of cell line xenograft models of rhabdoid tumor (PMID: 21298745). 21298745
Unknown unknown colorectal cancer not applicable YW3-56 Preclinical Actionable In a preclinical study, YW3-56 inhibited proliferation of colorectal cancer cell lines in culture, independent of TP53 status (PMID: 25612620). 25612620
Unknown unknown osteosarcoma not applicable VCN-01 Preclinical Actionable In a preclinical study, treatment with VCN-01 resulted in decreased viability of osteosarcoma cell lines in culture (PMID: 26603261). 26603261
Unknown unknown malignant pleural mesothelioma not applicable Cisplatin + Trabectedin Preclinical - Cell culture Actionable In a preclinical study, the combination of Yondelis (trabectedin) and Platinol (cisplatin) demonstrated synergy in inducing apoptosis and decreasing viability of malignant pleural mesothelioma cell lines in culture (PMID: 27512118). 27512118
Unknown unknown breast cancer not applicable Vinflunine Phase III Actionable In a Phase III trial, Javlor (vinflunine) treatment did not improve overall survival (9.1 vs 9.3 months, HR=1.04, p=0.67) compared to physician's choice of alkylating agent in patients with metastatic breast cancer (PMID: 29481630; NCT01091168). 29481630
Unknown unknown colon cancer not applicable CFI-400936 Preclinical - Cell line xenograft Actionable In a preclinical study, CFI-400936 inhibited tumor growth in a human cell line xenograft model of colon cancer (PMID: 25043312). 25043312
Unknown unknown sarcoma not applicable Ganitumab Phase II Actionable In a Phase II trial, Ganitumab treatment resulted in a 6% (2/35) partial response and stable disease in 49% (17/35) of patients with metastatic Ewing's family of tumors or desmoplastic small round cell tumor (PMID: 22508822). 22508822
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 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 lung non-small cell carcinoma not applicable Paclitaxel + Vistusertib Phase I Actionable In a Phase I trial, the combination therapy of Taxol (paclitaxel) and Vistusertib (AZD2014) resulted in a RECIST response rate of 35% (8/23) and a median progression-free survival of 5.8 months in patients with squamous non-small cell lung carcinoma (PMID: 30016392; NCT02193633). 30016392
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 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 non-Hodgkin lymphoma not applicable BCL201 Phase I Actionable In a Phase I trial, BCL201 (S55746) demonstrated safety and preliminary activity in patients with relapsed or recurrent non-Hodgkin lymphoma (Hematol Oncol. 2017;35(S2):47-48; NCT02920697). detail...
Unknown unknown gastric adenocarcinoma not applicable Paclitaxel + Ridaforolimus Phase Ib/II Actionable In a Phase Ib trial, Ridaforolimus (MK-8669), in combination with paclitaxel, produced stable disease greater than or equal to 4 months in 67% (2/3) of gastric cancer patients (PMID: 19901013). 19901013
Unknown unknown melanoma not applicable A-674563 Preclinical - Cell line xenograft Actionable In a preclinical study, a melanoma cell line demonstrated sensitivity to A-674563, resulting in apoptotic activity and inhibition of Akt1 activity in culture, and inhibition of tumor growth in xenograft models (PMID: 26970307). 26970307
Unknown unknown hematologic cancer not applicable BP1001 + Cytarabine Phase I Actionable In a Phase I/Ib trial, BP1001 in combination with Cytosar-U (cytarabine) resulted in complete remission in 28.6% (2/7), complete remission with incomplete haematological recovery in 14.3% (1/7), and stable disease in 28.6% (2/7) of patients with refractory or relapsed hematological malignancies (PMID: 29550383; NCT01159028). 29550383
Unknown unknown prostate cancer not applicable Docetaxel + MEDI5117 Preclinical Actionable In a preclinical study, MEDI5117 in combination with Taxotere (docetaxel) resulted in tumor regression in human prostate cancer xenograft models (PMID: 26744529). 26744529
Unknown unknown endometrial cancer not applicable Everolimus Phase II Actionable In a Phase II trial, the mTOR inhibitor Afinitor (everolimus) demonstrated efficacy and tolerability in patients with chemotherapy-refractory advanced or metastatic endometrial cancer (PMID: 23612453). 23612453
Unknown unknown Advanced Solid Tumor not applicable E7107 Phase I Actionable In a Phase I trial, E7107 treatment resulted in stable disease in 31% (8/26) of patients with advanced solid tumors, however, the study was discontinued due to vision loss in two patients (PMID: 24258465; NCT00499499). 24258465
Unknown unknown hematologic cancer not applicable TTI-621 Preclinical - Cell culture Actionable In a preclinical study, TTI-621 (SIRPalpha-Fc) increased phagocytosis in 77% (23/30) of the human hematological tumor cell lines tested in culture (PMID: 27856600). 27856600
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 brain glioma not applicable Dasatinib + Vandetanib Phase I Actionable In a Phase I trial, Caprelsa (vandetanib), in combination with dasatinib, demonstrated safety in pediatric patients with intrinsic pontine glioma (PMID: 23536435). 23536435
Unknown unknown Advanced Solid Tumor no benefit GGTI-2418 Phase I Actionable In a Phase I trial, GGTI-2418 demonstrated safety, but did not reach optimal target inhibition due to rapid elimination, and resulted in stable disease as best response in 31% (4/13) of patients with advanced solid tumors (PMID: 31372813). 31372813
Unknown unknown colorectal cancer not applicable Fruquintinib Phase III Actionable In a Phase III trial (FRESCO), treatment with Fruquitinib (HMPL-013) resulted in an improved median overall survival of 9.3 mo. vs. 6.57 mo. with placebo (HR=0.63), prolonged progression-free survival of 3.71 mo. vs. 1.84 mo. (HR=0.26), and an overall response rate (ORR) of 4.7% (13/278; 1 complete response, 12 partial responses) vs. 0% with placebo, in patients with metastatic colorectal cancer who had progressed on at least 2 prior chemotherapy regimens (PMID: 29946728; NCT02314819). 29946728
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 triple-receptor negative breast cancer not applicable Docetaxel + NTRC 0066-0 Preclinical Actionable In a preclinical study, the TTK (MPS1) inhibitor, NTRC 0066-0, acted in synergy with docetaxel to induce tumor remission and increase survival of TNBC mouse models (PMID: 26153498). 26153498
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 Advanced Solid Tumor not applicable PRN1371 Preclinical - Pdx & cell culture Actionable In a preclinical study, PRN1371 inhibited proliferation of various FGFR-driven tumor cell lines in culture and inhibited tumor growth in a variety of patient-derived xenograft models with FGFR pathway alterations (AACR; 2016. Abstract nr 1249). detail...
Unknown unknown 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 ovarian cancer not applicable Tisotumab Vedotin Case Reports/Case Series Actionable In a Phase II trial, Tisotumab vedotin treatment resulted in prolonged stable disease in a patient with ovarian cancer (Journal of Clinical Oncology 33, no. 15_suppl (May 20 2015) 2570-2570; NCT02001623). detail...
Unknown unknown non-Hodgkin 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 73% (11/15, 4 complete responses, 7 partial responses) in patients with relapsed or refractory indolent non-Hodgkin lymphoma, with a median duration of response of 176 days (PMID: 32327472; NCT02000934). 32327472
Unknown unknown ovarian cancer not applicable Eribulin + Onvansertib Preclinical - Cell culture Actionable In a preclinical study, Onvansertib (PCM-075) and eribulin synergistically inhibited growth of ovarian cancer cell lines in culture, resulted in sustained tumor regression in cell line xenograft models (PMID: 32183025). 32183025
Unknown unknown acute lymphoblastic leukemia not applicable AS605240 + Prednisolone Preclinical - Cell culture Actionable In a preclinical study, AS605240 and prednisolone synergistically inhibited survival of T-acute lymphocytic leukemia cell lines in culture (PMID: 25869207). 25869207
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 Bevacizumab + Cediranib Phase I Actionable In a Phase I trial, the combination of Cediranib (AZD-2171) and Avastin (bevacizumab) demonstrated preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 24752867). 24752867
Unknown unknown 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 colorectal cancer not applicable Bevacizumab + Capecitabine Phase III Actionable In a Phase III trial, maintenance therapy with Avastin (bevacizumab) and Xeloda (capecitabine) in combination resulted in a greater benefit compared to observation in colorectal cancer patients who were re-treated with Avastin (bevacizumab), Xeloda (capecitabine), and Eloxatin (oxaliplatin) due to progression, regardless of whether patients harbored RAS or BRAF V600E mutations (PMID: 28911067). 28911067
Unknown unknown Advanced Solid Tumor not applicable GSK3052230 Phase I Actionable In a Phase I trial, GSK3052230 (FP-1039) resulted in some preliminary efficacy, including a 20% reduction in tumor volume in one patient with castration resistant prostate cancer and a best response of stable disease in 41.7% (15/36) of patients with advanced solid tumors (PMID: 26646757; NCT00687505). 26646757
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 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 malignant pleural mesothelioma no benefit AZD4547 Phase II Actionable In a Phase II trial, AZD4547 treatment resulted in progression-free survival at 6 months (PFS6) in 12% (3/24) of patients with malignant pleural mesothelioma, which did not achieve the required PFS6 for continuation to stage 2 and the trial was discontinued (PMID: 31901768). 31901768
Unknown unknown esophagus squamous cell carcinoma not applicable Valproic acid Preclinical Actionable In a preclinical study, valproic acid enhanced the effectiveness of radiation in esophagus squamous cell carcinoma cell lines (PMID: 26135807). 26135807
Unknown unknown follicular lymphoma not applicable Ibrutinib + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of Imbruvica (ibrutinib) and Venclexta (venetoclax) resulted in a synergistic effect, demonstrating growth suppression in follicular lymphoma cell lines in culture (PMID: 28428442). 28428442
Unknown unknown pancreatic cancer not applicable Golvatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Golvatinib (E7050) inhibited tumor angiogenesis and tumor growth in xenografts of a VEGF-overexpressing human pancreatic cancer cell line (PMID: 19832844). 19832844
Unknown unknown head and neck squamous cell carcinoma not applicable Abemaciclib + Torin 2 Preclinical Actionable In a preclinical study, the combination of Abemaciclib (LY2835219) and Torin2 worked synergistically to reduce viability of head and neck squamous cell carcinoma cells in culture (PMID: 26909611). 26909611
Unknown unknown malignant glioma not applicable GDC-0084 Phase I Actionable In a Phase I trial, GDC-0084 treatment demonstrated expected toxicity and blood-brain barrier penetration, resulted in stable disease as best response in 40% (19/47) of patients with recurrent high-grade glioma (J Clin Oncol (PMID: 31937616; NCT01547546). 31937616
Unknown unknown 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 ovarian cancer not applicable ETP-46464 + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, ETP-46464 increased the sensitivity of ovarian cancer cell lines to ionizing radiation in culture (PMID: 25560806). 25560806
Unknown unknown multiple myeloma not applicable SJB3-019A Preclinical - Cell culture Actionable In a preclinical study, treatment with SJB3-019A induced cell-cycle arrest and apoptosis and decreased viability of multiple myeloma cell lines in culture (PMID: 28270494). 28270494
Unknown unknown multiple myeloma not applicable ABT-348 Preclinical - Cell line xenograft Actionable In a preclinical study, Ilorasertib (ABT-348) displayed efficacy in multiple myeloma cell line xenograft models (PMID: 22935731). 22935731
Unknown unknown duodenum adenocarcinoma not applicable Vandetanib Preclinical Actionable In preclinical studies, Caprelsa (vandetinib) reduced the number and size of polyps in mouse models of intestinal cancer and may be a beneficial strategy in early intestinal cancer (PMID: 18347145). 18347145
Unknown unknown glioblastoma multiforme not applicable PRN1371 Preclinical - Pdx Actionable In a preclinical study, PRN1371 treatment resulted in tumor regression in PDX models of glioblastoma (Eu J Cancer 2014 Vol 50, Suppl 6:157). detail...
Unknown unknown pancreatic cancer not applicable MVT-5873 Preclinical - Cell line xenograft Actionable In a preclinical study, a pancreatic xenograft model demonstrated reduced tumor volume and tumor growth inhibition when treated with MVT-5873 (Cancer Res 2016;76(24 Suppl):Abstract nr A73). detail...
Unknown unknown lung non-small cell carcinoma not applicable Dasatinib Phase II Actionable In a Phase II trial, Sprycel (dasatinib) treatment in non-small cell lung carcinoma patients showed some clinical efficacy, resulting in one patient with a partial response, and 12 patients with stable disease, demonstrating a disease control rate of 43% (13/30)(PMID: 20855820). 20855820
Unknown unknown colon carcinoma not applicable Mps-BAY1 + Paclitaxel Preclinical Actionable In a preclinical study, Mps-BAY1, in combination with paclitaxel, had increased efficacy in inhibiting cell proliferation of colon carcinoma cell in culture (PMID: 23933817). 23933817
Unknown unknown hairy cell leukemia not applicable Moxetumomab pasudotox-tdfk FDA approved Actionable In a Phase III trial that supported FDA approval, Lumoxiti (moxetumomab pasudotox-tdfk) treatment resulted in durable complete response in 30% (24/80), complete response in 41% (33/80), objective response (complete response and partial response) in 75% (60/80), and hematologic remission in 80% (64/80) of patients with relapsed or refractory hairy cell leukemia who had more than 2 prior systemic therapies (PMID: 30030507; NCT01829711). 30030507 detail...
Unknown unknown uveal melanoma no benefit Ipilimumab Clinical Study - Meta-analysis Actionable In a meta-analysis, uveal melanoma patients were not responsive to Yervoy (ipilimumab) therapy (PMID: 28881222). 28881222
Unknown unknown melanoma not applicable LDC1267 Preclinical Actionable In a preclinical study, LDC1267 treatment resulted in decreased metastasis in a mouse model of metastatic melanoma (PMID: 24553136). 24553136
Unknown unknown diffuse large B-cell lymphoma not applicable LAM-002A Phase I Actionable In a Phase I trial, LAM-002A demonstrated safety and preliminary efficacy, resulted in systemic partial metabolic responses and decreased tumor size in 3 of 11 patients with diffuse large B-cell lymphoma (Blood 2017 130 (Suppl 1):4119). detail...
Unknown unknown urinary bladder cancer not applicable BMS-986205 + Nivolumab Phase I Actionable In a Phase I/II trial, BMS-986205 in combination with Opdivo (nivolumab) resulted in an objective response rate of 32% (8/25) and a durable response rate of 44% (11/25) in patients with bladder cancer (PMID: 29167110). 29167110
Unknown unknown 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 follicular lymphoma not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in complete response in 12% (2/17) and partial response in 41% (7/17) of patients with follicular lymphoma (PMID: 29475723; NCT01767766). 29475723
Unknown unknown Advanced Solid Tumor not applicable ABC294640 Phase I Actionable In a Phase I trial, treatment with ABC294640 in patients with advanced solid tumors resulted in antitumor efficacy, including stable disease in six patients and a partial response in a patient with cholangiosarcoma (PMID: 28420720). 28420720
Unknown unknown 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 melanoma not applicable CP-870,893 + Tremelimumab Phase I Actionable In a Phase I trial, the combination of CP-870,893 and Tremelimumab was well-tolerated, and metastatic melanoma patients treated with CP-870,893 and Tremelimumab combination therapy demonstrated an objective response rate of 27.3% (6/22; 2 complete and 4 partial responses), a median progression-free survival of 3.2 months, and median overall survival of 23.6 months, with a 45-month median follow-up (PMID: 30288340; NCT01103635). 30288340
Unknown unknown Advanced Solid Tumor not applicable Pimitespib Phase I Actionable In a Phase I trial, TAS-116 demonstrated safety and resulted in a disease control rate of 27% (16/60; including stable disease for greater than or equal to 12 weeks (13) and partial responses (3)), in patients with advanced solid tumors, with partial responses in 2 patients with non-small cell lung cancer and 1 patient with gastrointestinal stromal tumor (PMID: 30679388; NCT02965885). 30679388
Unknown unknown colon carcinoma not applicable Mps-BAY2b Preclinical Actionable In a preclinical study, Mps-BAY2b inhibited cell cycle progression and induced cell death of colon carcinoma cells in culture (PMID: 23933817). 23933817
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 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 unspecified PD-1 antibody Clinical Study - Meta-analysis Actionable In a meta-analysis, anti-PD-1 and anti-PD-L1 therapies demonstrated similar safety profiles in patients with cancer, while anti-PD1 therapies resulted in superior overall survival (HR=0.75, p<0.001) and progression-free survival (HR=073, p=0.02) compared to anti-PD-L1 therapies (PMID: 31876895). 31876895
Unknown unknown breast cancer not applicable PF-06840003 Preclinical Actionable In a preclinical study, PF-06840003 inhibited tumor growth in a syngeneic mouse model of breast cancer, however, did not inhibit tumor growth in another mouse model (PMID: 30232146). 30232146
Unknown unknown Ewing sarcoma not applicable SP-2509 Preclinical - Cell culture Actionable In a preclinical study, EWS fusion-positive Ewing sarcoma cell lines demonstrated reduced viability following SP-2509 treatment that correlated with induction of KDM1B expression post-treatment in culture (PMID: 29997151). 29997151
Unknown unknown 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 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 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 hepatocellular carcinoma not applicable Camrelizumab Phase I Actionable In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity, resulted in partial response in 33.3% (1/3) of patients with hepatocellular carcinoma (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). detail...
Unknown unknown breast cancer not applicable Eribulin Phase III Actionable In a randomized Phase III clinical trial, Halaven (eribulin) monotherapy demonstrated significantly improved survival (median 13.1 months) relative to treatment of physician’s choice (median 10.6 months) across 762 patients with heavily pretreated metastatic breast cancer (PMID: 21376385). 21376385
Unknown unknown Advanced Solid Tumor not applicable Belvarafenib Phase I Actionable In a Phase I trial, Belvarafenib (HM95573) treatment demonstrated safety and preliminary efficacy, resulted in unconfirmed partial response in 3% (1/31) and stable disease in 29% (9/31) of patients with advanced solid tumors harboring BRAF (45%), KRAS (45%) or NRAS (10%) mutations (May 20 2016) 2570-2570; NCT02405065). detail...
Unknown unknown endometrial cancer not applicable Adavosertib + Paclitaxel Preclinical Actionable In a preclinicals study, Adavosertib (MK-1775) in combination with Paclitaxel, demonstrated efficacy in endometrial cancer cells (PMID: 24381593). 24381593
Unknown unknown B-cell lymphoma not applicable GSK126 Phase I Actionable In a Phase I trial, GSK126 was tolerated and demonstrated preliminary efficacy, resulted in partial response in 2% (1/41) and stable disease in 34% (14/41) of patients with advanced solid tumor (n=21) or B-cell lymphoma (n=20) (PMID: 31471312; NCT02082977). 31471312
Unknown unknown 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 colon cancer not applicable CVX-060 + Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of CVX-060 and Sutent (suntinib) resulted in increased tumor growth inhibition compared to either agent alone in a colon cancer cell line xenograft model (PMID: 21233403). 21233403
Unknown unknown Her2-receptor positive breast cancer not applicable Abemaciclib Preclinical - Pdx & cell culture Actionable In a preclinical study, an ERBB2 (HER2)-receptor positive breast cancer cell line xenograft model treated with Abemaciclib (LY2835219) demonstrated delayed tumor growth and in culture, resulted in some decreased cell viability (PMID: 26977878). 26977878
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 breast cancer not applicable Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) suppressed metastasis, angiogenesis, and tumor growth in mouse models of breast cancer (PMID: 21926191). 21926191
Unknown unknown lung non-small cell carcinoma not applicable Docetaxel + Plinabulin Phase I Actionable In a Phase I clinical trial, Plinabulin and Taxotere (docetaxel) combination treatment resulted in partial response in 25% (2/8) and minor improvement in 50% (4/8) of patients with non-small cell lung cancer (PMID: 21327495). 21327495
Unknown unknown ovarian cancer not applicable MIV-818 Preclinical - Cell culture Actionable In a preclinical study, MIV-818 inhibited proliferation of ovarian cancer cell in culture, and enhanced immune-mediated tumor cell killing in a co-culture of ovarian cancer cells and activated peripheral blood mononuclear cells (Mol Cancer Ther 2019;18(12 Suppl):Abstract nr C056). detail...
Unknown unknown pancreatic adenocarcinoma not applicable Capecitabine + Ruxolitinib Phase II Actionable In a Phase II trial, the combination of Jakafi (ruxolitinib) and Xeloda (capecitabine) did not prolong OS (median OS 4.5 vs. 4.3 months) in patients with pancreatic adenocarcinoma when compared to placebo plus Xeloda (capecitabine), however, prolonged OS (median OS 2.7 vs. 1.8 months) was observed in pancreatic adenocarcinoma patients with elevated levels of serum CRP (PMID: 26351344). 26351344
Unknown unknown meningioma not applicable AR-42 Preclinical Actionable In a preclinical study, AR-42 induced apoptosis and inhibited proliferation of meningioma cells in culture (PMID: 21778190). 21778190
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 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 adult T-cell leukemia not applicable Alemtuzumab Phase II Actionable In a Phase II trial, treatment with Alemtuzumab resulted in 51.7% (15/29) of patients with adult T-cell leukemia demonstrating an overall objective response, a median progression free survival of 2.0 months, and overall survival of 5.9 months (PMID: 27486175). 27486175
Unknown unknown endometrial cancer not applicable Dactolisib Preclinical Actionable In a preclinical study, BEZ235 suppressed tumor growth in endometrial xenografts (PMID: 22662154). 22662154
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 Advanced Solid Tumor not applicable Cobimetinib + Ipatasertib Phase I Actionable In a Phase I clinical trial Ipatasertib (GDC-0068), in combination with the Mek inhibitor Cobimetinib (GDC-0973) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Cancer Res, October 1, 2014 74; CT328). detail...
Unknown unknown Advanced Solid Tumor not applicable Erdafitinib Phase I Actionable In a Phase I study, Balversa (erdafitinib) displayed safety and efficacy in advanced solid tumor patients (J Clin Oncol 32:5s, 2014 (suppl; abstr 2501)). detail...
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 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 Advanced Solid Tumor not applicable SF1126 Phase I Actionable In a Phase I trial, SF1126 demonstrated safety and preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 22921184). 22921184
Unknown unknown acute myeloid leukemia not applicable KPT-8602 Preclinical - Cell culture Actionable In a preclinical study, KPT-8602 decreased viability of acute myeloid leukemia cell lines in culture (PMID: 27780859). 27780859
Unknown unknown glioblastoma multiforme 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 gastrointestinal stromal tumor not applicable PKF118-310 Preclinical - Cell line xenograft Actionable In a preclinical study, PKF118-310 decreased viability of gastrointestinal stromal tumor (GIST) cell lines in culture, and reduced tumor growth in xenograft models (PMID: 28611108). 28611108
Unknown unknown acute lymphoblastic leukemia not applicable TAS4464 Preclinical - Cell line xenograft Actionable In a preclinical study, TAS4464 resulted in complete tumor regression in cell line xenograft models of acute lymphocytic leukemia (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr C176). detail...
Unknown unknown glioblastoma multiforme not applicable Temozolomide + Vandetanib Phase II Actionable In a Phase II trial, Caprelsa (vandetinib), in combination with radiation therapy and Temodar (temozolomide), demonstrated no difference in PFS and OS when compared to radiation therapy and Temodar (temozolomide) alone in glioblastoma patients (PMID: 25910950). 25910950
Unknown unknown melanoma not applicable PD-0325901 Phase I Actionable In a Phase I trial, PD-0325901 demonstrated some efficacy in previously treated melanoma patients, however, there was significant toxicity above 10 mg BID (PMID: 21516509). 21516509
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 multiple myeloma not applicable Daratumumab FDA approved Actionable In a Phase I/IIb trial (GEN501) that supported FDA approval, Darzalex (daratumumab) treatment in patients with heavily pretreated and refractory multiple myeloma resulted in an overall response rate of 36% (15/42) in the 16 mg/kg cohort, with 1 complete response and 3 very good partial responses (PMID: 26308596; NCT00574288). detail... 26308596
Unknown unknown hepatocellular carcinoma not applicable Fluorouracil + Interferon alpha-2b Phase II Actionable In a Phase II clinical trial, Adrucil (fluorouracil) combined with interferon alfa-2b was well-tolerated and demonstrated efficacy in patients with hepatocellular carcinoma, with 25% (9/36) patients achieving complete or partial response, and a median overall survival of 19.5 months (PMID: 12560429). 12560429
Unknown unknown immune system cancer not applicable Afuresertib Phase II Actionable In a Phase II trial, treatment with Afuresertib (GSK2110183) in patients with Langerhans cell histiocytosis resulted in an overall response rate of 33% in treatment-naive patients and 28% in patients with refractory disease (PMID: 27804235). 27804235
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 triple-receptor negative breast cancer not applicable GSK3368715 Preclinical - Cell line xenograft Actionable In a preclinical study, GSK3368715 inhibited tumor growth in a triple-negative breast cancer cell line xenograft model (PMID: 31257072). 31257072
Unknown unknown colon carcinoma not applicable Cyclophosphamide + Oncolytic HSV-1 rRp450 Preclinical - Cell culture Actionable In a preclinical study, addition of Cytoxan (cyclophosphamide) to Oncolytic HSV-1 rRp450 treatment enhanced cytotoxicity in a colon carcinoma cell line in culture, and reduced colon carcinoma liver metastases in a mouse model (PMID: 12209705). 12209705
Unknown unknown papillary renal cell carcinoma not applicable Bevacizumab + Everolimus Phase II Actionable In a Phase II trial, 50% (2/4) of patients with papillary renal cell carcinoma achieved a 6 month PFS when treated with a combination of Afinitor (everolimus) and Avastin (bevacizumab) (PMID: 27601542). 27601542
Unknown unknown ovarian cancer not applicable Carboplatin + ETP-46464 Preclinical - Cell culture Actionable In a preclinical study, ETP-46464 increased the sensitivity of ovarian cancer cell lines to Paraplatin (carboplatin) in culture (PMID: 25560806). 25560806
Unknown unknown lung small cell carcinoma not applicable Cisplatin + Durvalumab + Etoposide FDA approved Actionable In a Phase III (CASPIAN) trial that supported FDA approval, Imfinzi (durvalumab) in combination with Vepesid (etoposide) and Paraplatin (carboplatin) or Platinol (cisplatin) resulted in significantly improved overall survival (13.0 vs 10.3 mo, HR=0.73, p=0.0047) compared to platinum-etoposide therapy in patients with untreated extensive-stage small cell lung cancer (PMID: 31590988; NCT03043872). 31590988
Unknown unknown lung non-small cell carcinoma not applicable Ganetespib + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, Ganetespib increased the sensitivity of various non-small cell lung cancer cell lines to radiation in culture, resulting in increased DNA damage and cell-cycle arrest and decreased cell survival (PMID: 27354472). 27354472
Unknown unknown malignant ependymoma not applicable Sunitinib Phase II Actionable In a Phase II clinical trial, Sutent (sunitinib) was well-tolerated in young patients with ependymoma, 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 sarcoma not applicable Conatumumab + Ganitumab Phase Ib/II Actionable In a Phase Ib/II trial, Ganitumab and Conatumumab (AMG 655) combination treatment resulted in stable disease in 33% (5/15) of patients with sarcoma, including one with leiomyosarcoma (PMID: 24816908). 24816908
Unknown unknown neuroendocrine tumor not applicable Surufatinib Phase I Actionable In a Phase I trial, Surufatinib (HMPL-012) demonstrated an objective response rate in 44% (8/18) of patients with neuroendocrine tumors (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A1). detail...
Unknown unknown neuroendocrine tumor not applicable Surufatinib Phase Ib/II Actionable In a Phase Ib/II trial, Surufatinib (HMPL-012) treatment resulted in an objective response rate of 19% (8/42) and 15% (6/39), a disease control rate of 91% (38/42) and 92% (36/39), and a median progression-free survival of 21.2 and 13.4 months in patients with advanced, well-differentiated pancreatic and extrapancreatic neuroendocrine tumors, respectively (PMID: 30833272; NCT02267967). 30833272
Unknown unknown lung non-small cell carcinoma not applicable Barasertib + Navitoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of Barasertib (AZD1152) and Navitoclax (ABT-263) resulted in synergy, inhibiting proliferation of non-small cell lung carcinoma cells in culture (PMID: 28179288). 28179288
Unknown unknown renal cell carcinoma not applicable Everolimus + Lenvatinib Phase Ib/II Actionable In a Phase Ib clinical trial, the combination of Lenvima (lenvatinib) and Afinitor (everolimus) demonstrated safety, and resulted in partial response in 30% (6/20) of patients with metastatic renal cell carcinoma (PMID: 24190702). 24190702
Unknown unknown renal cell carcinoma not applicable Everolimus + Lenvatinib FDA approved Actionable In a Phase II clinical trial that supported FDA approval, treatment with the combination of Lenvima (lenvatinib) and Afinitor (everolimus) resulted in a prolonged median progression-free survival of 14.6 months, compared to 5.5 months for Afinitor (everolimus) alone in patients with metastatic renal cell carcinoma who had progressed after one previous VEGF-targeted therapy (PMID: 26482279; NCT01136733). 26482279 detail...
Unknown unknown 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 multiple myeloma not applicable Cyclophosphamide + LCL161 Phase II Actionable In a Phase II trial, LCL161 treatment followed by Cytoxan (cyclophosphamide) resulted in complete response in 4% (1/25), partial response in 12% (3/25), and molecular response in 4% (1/25) of multiple myeloma patients (PMID: 27841872). 27841872
Unknown unknown ovarian cancer not applicable BAY1217389 Preclinical Actionable In a preclinical study, BAY1217389 demonstrated moderate efficacy in ovarian cancer xenograft models (PMID: 26832791). 26832791
Unknown unknown breast cancer not applicable TAK-960 Preclinical - Cell line xenograft Actionable In a preclinical study, breast cancer cells treated with TAK-960 demonstrated a decrease in tumor size in cell line xenograft models (PMID: 22188812). 22188812
Unknown unknown follicular lymphoma not applicable APG-2575 + Ibrutinib Preclinical - Cell line xenograft Actionable In a preclinical study, APG-2575 and Imbruvica (ibrutinib) combination therapy exhibited synergistic antitumor activity in a cell-line xenograft model of follicular lymphoma (Cancer Res July 1 2019 (79) (13 Supplement) 2058). detail...
Unknown unknown glioblastoma multiforme 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 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 chronic lymphocytic leukemia not applicable Venetoclax FDA approved Actionable In a Phase II clinical trial, treatment with Venclexta (venetoclax) resulted in an overall response rate of 79.4% in chronic lymphocytic leukemia patients with 17p deletion (PMID: 27014415). detail... 27014415
Unknown unknown chronic lymphocytic leukemia not applicable Venetoclax FDA approved Actionable In a Phase II trial that supported FDA approval, Venclexta (venetoclax) treatment resulted in an overall response rate of 65% (59/91) and a median follow-up of 14 months in chronic lymphocytic leukemia patients who were refractory to or relapsed on Imbruvica (ibrutinib) therapy (PMID: 29246803; NCT02141282). detail... 29246803
Unknown unknown chronic lymphocytic leukemia not applicable Venetoclax FDA approved Actionable In a Phase II trial that supported FDA approval, treatment with Venclexta (venetoclax) resulted in an overall response rate of 65% (59/91), median progression free-survival of 24.7 months, and median duration of response was not reached in chronic lymphocytic leukemia patients who had progressed on prior Zydelig (idelalisib) therapy (PMID: 29305552; NCT02141282). detail... 29305552
Unknown unknown thyroid gland cancer not applicable Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) improved median progression free survival to 10.8 months in metastatic differentiated thyroid cancer patients (PMID: 24768112). detail... 24768112
Unknown unknown 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 breast cancer not applicable Carboplatin + E7449 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of E7449 and Paraplatin (carboplatin) synergized to inhibit tumor growth in a human breast cancer cell line xenograft model (PMID: 26513298). 26513298
Unknown unknown clear cell renal cell carcinoma no benefit Bevacizumab + Temsirolimus Phase II Actionable In a Phase II clinical trial, treatment with the combination of Torisel (temsirolimus) and Avastin (bevacizumab) did prolong progression-free survival compared to treatment with Avastin (bevacizumab) as a single agent (7.6 months vs 7.4 months) in patients with renal clear cell carcinoma (PMID: 26077237). 26077237
Unknown unknown gastroesophageal junction adenocarcinoma not applicable Ramucirumab FDA approved Actionable In a Phase III trial (REGARD) that supported FDA approval, treatment with Cyramza (ramucirumab) improved median overall survival (5.2 vs 3.8 mo, HR=0.776, p=0.047) in aptients with compared to placebo in patients with advanced gastric or gastro-oesophageal junction adenocarcinoma whose disease progressed after chemotherapy (PMID: 24094768; NCT00917384). 24094768 detail...
Unknown unknown clear cell renal cell carcinoma not applicable Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in improved progression free survival (8.2 v 5.6 months) and objective response rate (46% vs 18%) compared to Sutent (sunitinib) in patients with untreated clear cell metastatic renal cell carcinoma, with a 34% reduction in rate of progression or death (HR=0.66, p=0.012) (PMID: 28199818). 28199818
Unknown unknown Ewing sarcoma no benefit Tranylcypromine Preclinical - Cell culture Actionable In a preclinical study, Ewing sarcoma cell lines did not demonstrate sensitivity to Parnate (tranylcypromine) in culture (PMID: 29997151). 29997151
Unknown unknown triple-receptor negative breast cancer not applicable Cabozantinib + Navitoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of Navitoclax (ABT-263) and Cometriq (cabozantinib) resulted in a synergistic effect and inhibited the growth of triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown hematologic cancer not applicable AZD5991 Preclinical - Cell line xenograft Actionable In a preclinical study, AZD5991 inhibited tumor growth in hematologic cancer cell line xenograft models (Cancer Res July 1 2017 (77) (13 Supplement) DDT01-02). detail...
Unknown unknown glioblastoma multiforme not applicable GDC-0152 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with GDC-0152 increased apoptosis of glioblastoma multiforme (GBM) cell lines in culture, and resulted in increased tumor cell differentiation and apoptosis, increased survival, and decreased tumor growth in GBM cell line xenograft models (PMID: 27490930). 27490930
Unknown unknown Advanced Solid Tumor not applicable Navicixizumab Phase I Actionable In a Phase I trial, Navicixizumab (OMP-305B83) treatment resulted in partial response in 6% (4/66) and stable disease in 26% (17/66) of patients with advanced solid tumors, with 19 patients having a reduction of target lesions (PMID: 30229512). 30229512
Unknown unknown Advanced Solid Tumor not applicable Navicixizumab Preclinical Actionable In a preclinical study, Navicixizumab (OMP-305B83) inhibited cell proliferation of endothelial cells in culture and demonstrated antitumor activity of multiple tumor types in vivo (Mol Cancer Ther December 2015 14; C164). detail...
Unknown unknown Advanced Solid Tumor not applicable Navicixizumab Phase I Actionable In a Phase I trial, Navicixizumab (OMP-305B83) demonstrated safety and preliminary efficacy in a variety of advanced solid tumor patients, including ovarian, endometrial, breast, and pancreatic (Eur J Can, Vol 69, S35). detail...
Unknown unknown thymic carcinoma not applicable Selinexor Preclinical - Cell line xenograft Actionable In a preclinical study, Selinexor (KPT-330) induced cell-cycle arrest and apoptosis and inhibited growth of several thymic epithelial tumor cell lines, including thymoma and thymic carcinoma cell lines, in culture, and inhibited tumor growth in thymic carcinoma cell line xenograft models (PMID: 28819023). 28819023
Unknown unknown acute myeloid leukemia not applicable Carfilzomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, the combination of EDO-S101 and Kyprolis (carfilzomib) decreased viability of primary acute myeloid leukemia cells in culture (PMID: 28753594). 28753594
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 renal cell carcinoma no benefit Bevacizumab + Carotuximab Clinical Study Actionable In a clinical study, the addition of TRC105 to Avastin (bevacizumab) treatment in renal cell carcinoma patients did not result in improved progression free survival (2.8 mo vs 4.6 mo) when compared to Avastin (bevacizumab) alone (PMID: 28832978). 28832978
Unknown unknown breast cancer not applicable BGP-15 Preclinical - Cell culture Actionable In a preclinical study, BGP-15 induced apoptosis and inhibited growth of breast cancer cells in culture (PMID: 22661288). 22661288
Unknown unknown 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 prostate cancer not applicable NSC156529 Preclinical - Cell line xenograft Actionable In a preclinical study, NSC156529 inhibited growth of human prostate cancer cell lines in culture, induced expression of differentiation markers and inhibited tumor growth in cell line xenograft models (PMID: 26294745). 26294745
Unknown unknown 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 multiple myeloma not applicable Carfilzomib + DT204 Preclinical - Cell culture Actionable In a preclinical study, multiple myeloma cells resistant to Kyprolis (carfilzomib) demonstrated re-sensitization to Kyprolis (carfilzomib) in culture when treatment was combined with DT204, showing a synergistic effect and increased apoptotic activity (PMID: 27677741). 27677741
Unknown unknown hepatocellular carcinoma not applicable Novaferon Preclinical - Cell line xenograft Actionable In a preclinical study, Novaferon treatment induced cell cycle arrest and apoptosis, and inhibited tumor growth in a hepatocellular carcinoma cell line xenograft model (PMID: 24467885). 24467885
Unknown unknown prostate cancer not applicable Dactolisib + unspecified CTLA4 antibody + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, combination of myeloid-derived suppressor cell-targeting with BEZ235 and immune checkpoint blockade with anti-CTLA4 and anti-PD-1 antibodies resulted in synergistic inhibition of tumor growth and metastasis in transgenic mouse models of metastatic castration-resistant prostate cancer (PMID: 28321130). 28321130
Unknown unknown cutaneous T cell lymphoma not applicable Romidepsin FDA approved Actionable In clinical trials that supported FDA approval, treatment with Istodax (romidepsin) produced a 35% (34/96) response rate in patients with cutaneous T cell lymphoma (PMID: 20697094). detail... 20697094
Unknown unknown B-cell lymphoma not applicable Alisertib + Rituximab Phase I Actionable In a Phase I trial, Alisertib (MLN8237) in combination with Rituxan (rituximab) demonstrated safety and efficacy, resulted in an objective response rate of 25% (3/12, 2 complete response, 1 partial response), and stable disease in 42% (5/12) of patients with relapsed or refractory B-cell non-Hodgkin lymphomas (PMID: 30082475; NCT01397825). 30082475
Unknown unknown acute myeloid leukemia not applicable FF-10501-01 Phase I Actionable In a Phase I trial, FF-10501-01 demonstrated safety and preliminary efficacy, resulted in stable disease control with no disease progression over 3-24 cycles of treatment in 34.8% (8/23) of acute myeloid leukemia patients, including 2 patients achieved partial response (Blood 128 (22):1640). detail...
Unknown unknown multiple myeloma not applicable Isatuximab Phase I Actionable In a Phase I trial, Isatuximab (SAR650984) treatment resulted in complete response in 6% (2/34), partial response in 18% (6/34), and stable disease in 41% (14/34) of patients with relapsed or refractory multiple myeloma (J Clin Oncol 32:5s, 2014 (suppl; abstr 8532)). detail...
Unknown unknown marginal zone B-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 78% (7/9), complete response/complete metabolic response rate of 33% (3/9), and median duration of response of 4.4 months in patients with marginal zone lymphoma (PMID: 30803990; NCT02018861). 30803990
Unknown unknown triple-receptor negative breast cancer not applicable BAY1161909 + Paclitaxel Preclinical Actionable In a preclinical study, BAY1161909, in combination with Taxol (paclitaxel), had increased efficacy in xenograft models of triple-negative breast cancer compared to Taxol (paclitaxel) alone, resulting in complete tumor regression (PMID: 26832791). 26832791
Unknown unknown 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 chronic lymphocytic leukemia not applicable Umbralisib Phase II Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment demonstrated safety and preliminary efficacy, with 90% of chronic lymphocytic leukemia patients achieved progression-free survival at 6.5 months (J Clin Oncol 36, 2018 (suppl; abstr 7530); NCT02742090). detail...
Unknown unknown chronic lymphocytic leukemia not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in objective response in 85% (17/20) and stable disease in 15% (3/20) of patients with chronic lymphocytic leukemia (PMID: 29475723; NCT01767766). 29475723
Unknown unknown gastric adenocarcinoma not applicable Nintedanib Phase II Actionable In a Phase II trial, Ofev (nintedanib) was well tolerated and the study met its primary endpoint, resulted in progression-free survival at 6-months in 19% (6/32) of patients with esophageal/GEJ (n=17) or gastric (n=15) adenocarcinoma, with a median follow-up of 14.5 months and a median overall survival of 14.2 months (PMID: 30952642; NCT02234596). 30952642
Unknown unknown neuroblastoma not applicable GD2-GD3 vaccine Phase I Actionable In a Phase I trial, treatment with the GD2-GD3 vaccine was well tolerated, and led to an antibody response in 80% (12/15) and loss of minimal residual disease in 60% (6/10) of neuroblastoma patients (PMID: 24520094). 24520094
Unknown unknown islet cell tumor not applicable Pictilisib Preclinical Actionable In a preclinical study, Pictilisib (GDC-0941) inhibited AKT activation, reduced tumor burden, and increased lifespan in a transgenic mouse model of pancreatic neuroendocrine tumor (PMID: 27225693). 27225693
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 stomach cancer not applicable LY2874455 Phase I Actionable In a Phase I trial, gastric cancer patients treated with LY2874455 demonstrated some efficacy, including one patient with a partial response, 12 patients with stable disease after two cycles, and 4 patients with stable disease after four cycles of treatment, and had a median progression free survival of 62 days (PMID: 28589492). 28589492
Unknown unknown ovary epithelial cancer not applicable Binimetinib + Paclitaxel Phase Ib/II Actionable In a Phase Ib trial, combination therapy with Taxol (paclitaxel) and Mektovi (binimetinib) was tolerable and resulted in an objective response rate of 18% (5/28, 1 complete response, 4 partial responses) and a clinical benefit rate of 57% (16/28, best overall response of stable disease or better) in patients with platinum resistant/refractory epithelial ovarian cancer, and clinical benefit was observed in all patients with MAPK pathway alterations (n=4) (PMID: 29844129; NCT01649336). 29844129
Unknown unknown pancreatic cancer not applicable Bleomycin + CBP501 Preclinical - Cell culture Actionable In a preclinical study, the combination of Blenoxane (bleomycin) and CBP501 resulted in increased cell death compared to Blenoxane (bleomycin) alone in a human pancreatic cancer cell line in culture (PMID: 17237275). 17237275
Unknown unknown lung non-small cell carcinoma not applicable TVB-2640 Phase I Actionable In a Phase I study, TVB-2640 as a monotherapy or in combination with Taxol (paclitaxel) resulted in stable diseases for more than 16 weeks in 43% (3/7) of patients with non-small cell lung cancer (2015 51 S724-S724 Eur J Cancer). detail...
Unknown unknown glioblastoma multiforme 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 multiple myeloma not applicable Bortezomib + Dexamethasone + Ulocuplumab Phase I Actionable In a Phase I trial, Ulocuplumab (BMS-936564) in combination with Velcade (bortezomib) and Adexone (dexamethasone) demonstrated safety and preliminary efficacy, resulted in an overall response rate of 25.0% (4/16) and a clinical benefit rate of 50% (8/16) in patients with relapsed multiple myeloma (PMID: 31672767). 31672767
Unknown unknown colorectal cancer no benefit Utomilumab Phase I Actionable In a Phase I trial, Utomilumab (PF-05082566) treatment resulted in no overall objective response (0/12) in patients with colorectal cancer (PMID: 29549159; NCT01307267). 29549159
Unknown unknown Ewing sarcoma not applicable SN-38 + Veliparib Preclinical Actionable In a preclinical study, Ewing sarcoma cells treated with SN-38 combined with Veliparib (ABT-888) resulted in synergism, demonstrating reduced cell viability in culture (PMID: 26438158). 26438158
Unknown unknown Advanced Solid Tumor not applicable MEDI0639 Phase I Actionable In a Phase I trial, MEDI0639 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 33, 2015 (suppl; abstr 3024)). detail...
Unknown unknown breast cancer not applicable PF-00562271 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with PF-00562271 inhibited PTK2 (FAK) phosphorylation and resulted in apoptosis and tumor regression in breast cancer cell line xenograft models (PMID: 18339875). 18339875
Unknown unknown clear cell renal cell carcinoma not applicable Everolimus + Hydroxychloroquine Phase Ib/II Actionable In a Phase I/II trial, treatment with the combination of Afinitor (everolimus) and Plaquenil (hydroxychloroquine) was well-tolerated, and resulted in a median progression-free survival (PFS) of 6.3 months, PFS of 6 months or greater in 45% (15/33), and partial response (PR) or stable disease (SD) greater than 3 months in 66% (22/33; 2 PR and 20 SD) of clear cell renal cell carcinoma patients (PMID: 30635337). 30635337
Unknown unknown Advanced Solid Tumor not applicable GDC-0349 Preclinical - Cell line xenograft Actionable In a preclinical study, the mTOR inhibitor GDC-0349 demonstrated inhibition of tumor growth in cell line xenograft models of solid tumors (PMID: 24900569). 24900569
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 Advanced Solid Tumor no benefit MINT1526A Phase I Actionable In a Phase I trial, MINT1526A monotherapy did not result in partial response in patients with advanced solid tumors (PMID: 29905898). 29905898
Unknown unknown neuroblastoma not applicable Hu3F8-BsAb Preclinical - Cell line xenograft Actionable In a preclinical study, Hu3F8-BsAb treatment induced cell killing in neuroblastoma cell lines expressing GD2 in culture, and treatment with Hu3F8-BsAb plus human peripheral blood mononuclear cells inhibited tumor growth in neuroblastoma cell line xenograft models (PMID: 25542634). 25542634
Unknown unknown lung non-small cell carcinoma not applicable Abemaciclib Phase I Actionable In a Phase I trial, treatment with Abemaciclib (LY2835219) in patients with non-small cell lung carcinoma resulted in a disease control rate of 49% (33/68), including 46% (31/68) with stable disease and 3% (2/68) with a partial response, and a 6 month PFS of 26% (PMID: 27217383, PMID: 24795392). 24795392 27217383
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 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 Carboplatin + Docetaxel + Gemcitabine + Itraconazole Clinical Study Actionable In a retrospective analysis, triple-negative breast cancer patients treated with the combination of Itraconazole with Docefrez (docetaxel), Paraplatin (carboplatin), and Gemzar (gemcitabine) chemotherapy demonstrated an overall response rate of 62% (8/13), a median progression-free survival of 10.8 months, and a median overall survival of 20.4 months (PMID: 24982411). 24982411
Unknown unknown lung non-small cell carcinoma no benefit Bavituximab + Docetaxel Phase II Actionable In a Phase II trial, Tarvacin (bavituximab) and Taxotere (docetaxel) combination treatment resulted in favorable outcome compared to control in non-small cell lung cancer patients, with an overall response rate of 17.1% (7/41), median progression-free survival of 4.5 months (HR=0.74), and a median overall survival of 11.7 months (HR=0.66) (PMID: 27265742). 27265742
Unknown unknown clear cell renal cell carcinoma not applicable Abexinostat + Pazopanib Phase Ib/II Actionable In a Phase Ib/II trial, 31% (5/16) of renal clear cell carcinoma patients demonstrated a response to the combination of Abexinostat (PCI-24781) and Votrient (pazopanib) (PMID: 28221861). 28221861
Unknown unknown colon cancer not applicable Doxorubicin + NU7441 Preclinical - Cell culture Actionable In a preclinical study, NU7441 increased sensitivity of colon cancer cell lines to Adriamycin (doxorubicin), resulting in reduced cell survival in culture (PMID: 16707462). 16707462
Unknown unknown renal cell carcinoma not applicable ODM-203 Preclinical Actionable In a preclinical study, ODM-203 inhibited tumor growth and lung metastasis, and increased tumor microenvironment immune response in a VEGFR-dependent mouse renal carcinoma model (PMID: 30301864). 30301864
Unknown unknown hepatocellular carcinoma not applicable Nivolumab FDA approved Actionable In a Phase I/II trial that supported FDA approval, Opdivo (nivolumab) treatment resulted in complete response in 1.9% (3/154) and partial response in 12.3% (19/154) of hepatocellular carcinoma patients who progressed on or were intolerant to Nexavar (sorafenib) (J Clin Oncol 35, 2017 (suppl; abstr 4013); NCT01658878). detail... detail...
Unknown unknown hepatocellular carcinoma not applicable Nivolumab FDA approved Actionable In a Phase I/II trial (CheckMate 040) that supproted FDA approval, Opdivo (nivolumab) treatment resulted in complete response in 1% (3/214), partial response in 18% (39/214), and stable disease in 45% (96/214) of hepatocellular carcinoma patients (PMID: 28434648; NCT01658878). detail... 28434648
Unknown unknown Advanced Solid Tumor not applicable PF-06647263 Phase I Actionable In a Phase I trial, PF-06647263 treatment resulted in partial response in 10% (5/48) of patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2511)). detail...
Unknown unknown multiple myeloma not applicable Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, treatment with EDO-S101 induced apoptosis and decreased viability of a multiple myeloma cell line in culture (PMID: 28753594). 28753594
Unknown unknown prostate cancer not applicable ORCA-010 Preclinical - Cell line xenograft Actionable In a preclinical study, ORCA-010 treatment induced cytotoxicity in a prostate cancer cell line in culture, and inhibited tumor growth and increased survival in a cell line xenograft model (PMID: 25093639). 25093639
Unknown unknown lung non-small cell carcinoma not applicable Pemetrexed Disodium + Sirolimus Phase Ib/II Actionable In a Phase Ib/II trial, Rapamune (sirolimus) in combination with Alimta (pemetrexed) demonstrated safety and some efficacy in treating patients with NSCLC (PMID: 24658085). 24658085
Unknown unknown hepatocellular carcinoma not applicable PI-88 Phase II Actionable In a phase II trial, PI-88 treatment significantly improved survival for up to 3 years in hepatocellular carcinoma patients compared to control, as evidenced by increased recurrence-free rate (63% vs 50%) and postponed time to recurrence at the 36th percentile by 78% (PMID: 25170226). 25170226
Unknown unknown breast cancer not applicable Cyclophosphamide + Methotrexate + Vandetanib Phase I Actionable In a Phase I study, Caprelsa (vandetanib), in combination with Cytoxan (cyclophosphamide) and Abitrexate (methotrexate), demonstrated safety and some efficacy in breast cancer patients (PMID: 23001754). 23001754
Unknown unknown lung squamous cell carcinoma not applicable Carboplatin + 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 Indication other than cancer not applicable Nintedanib FDA approved Actionable Ofev (nintedanib) is FDA approved for use in patients with idiopathic pulmonary fibrosis (FDA.gov). detail... detail...
Unknown unknown pancreatic carcinoma not applicable ABT-348 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with Ilorasertib (ABT-348) inhibited tumor growth and led to regression of advanced tumors in cell line xenograft models of pancreatic carcinoma (PMID: 22935731). 22935731
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 TMU-35435 Preclinical - Cell line xenograft Actionable In a preclinical study, TMU-35435 induced cell-cycle arrest and apoptosis and decreased viability of non-small cell lung cancer cell lines in culture, and inhibited tumor growth in a lung cancer cell line xenograft model (PMID: 28233309). 28233309
Unknown unknown lymphoma not applicable Everolimus Phase II Actionable In a Phase II trial, Afinitor (everolimus) treatment resulted in an overall response rate of 44% (7/16) in T-cell lymphoma patients, with a median progression-free survival of 4.1 months and a median overall survival of 10.2 months (PMID: 25921059). 25921059
Unknown unknown Ewing sarcoma not applicable JQ1 Preclinical - Pdx & cell culture Actionable In a preclinical study, JQ1 treatment resulted in cytostatic effects in patient-derived Ewing's sarcoma cell lines in culture, but inhibited tumor growth in patient-derived xenograft models due to inhibition of tumor angiogenesis (PMID: 26908627). 26908627
Unknown unknown follicular lymphoma not applicable Ocaratuzumab Phase I Actionable In a Phase I clinical trial, Ocaratuzumab (AME-133v) demonstrated safety and some preliminary activity in patients with follicular lymphoma, including those with the Fc-gamma-RIIIa genotype (PMID: 22223529). 22223529
Unknown unknown acute myeloid leukemia not applicable Bortezomib + Mivebresib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Velcade (bortezomib) and Mivebresib (ABBV-075) resulted in increased tumor growth inhibition in an acute myeloid leukemia cell line xenograft model compared to Velcade (bortezomib) alone (PMID: 28416490). 28416490
Unknown unknown lung carcinoma not applicable Buparlisib + Everolimus Preclinical Actionable In a preclinical study, Buparlisib (BKM120) in combination with Afinitor (everolimus) inhibited tumor growth in mouse lung cancer xenograft models (PMID: 22781393). 22781393
Unknown unknown MALT lymphoma not applicable Chlorambucil + Rituximab Phase III Actionable In a Phase III trial, Chlorambucil and Rituxan (Rituximab) combination treatment resulted in improved event-free survival (HR=0.54) but not longer overall survival in patients with mucosa-associated lymphoid tissue lymphoma (PMID: 28355112). 28355112
Unknown unknown acute myeloid leukemia not applicable TP-1287 Preclinical - Cell line xenograft Actionable In a preclinical study, TP-1287 demonstrated improved oral bioavailability, resulted in efficient tumor inhibition in cell line xenograft models of acute myeloid leukemia (Cancer Res 2017;77(13 Suppl):Abstract nr 5133). 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 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 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 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 acute myeloid leukemia not applicable IACS-010759 Preclinical - Pdx & cell culture Actionable In a preclinical study, IACS-010759 inhibited growth of acute myeloid leukemia cells in culture, resulting in prolonged survival in patient-derived xenograft models (Mol Cancer Ther 2015; 14(12 Suppl 2): Abstract nr LB-A15). detail...
Unknown unknown 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 triple-receptor negative breast cancer no benefit GSK2801 + HY-16462 Preclinical - Cell culture Actionable In a preclinical study, GSK2801 and HY-16462 combination treatment did not synergistically inhibit growth of a triple-negative breast cancer cell line in culture (PMID: 31000582). 31000582
Unknown unknown renal cell carcinoma not applicable Tivozanib Phase III Actionable In a Phase III trial, Tivozanib (AV-951) demonstrated improved progression free survival compared with Nexavar (sorafenib) as initial targeted therapy in patients with metastatic renal cell carcinoma (PMID: 24019545; NCT01030783). 24019545
Unknown unknown renal cell carcinoma not applicable Tivozanib Phase III Actionable In a Phase III trial, Tivozanib (AV-951) was better tolerated than Nexavar (sorafenib), and improved progression-free survival (5.6 vs 3.9 months, HR=0.73, p=0.016) in patients with metastatic renal cell carcinoma that had at least two previous systemic treatments (PMID: 31810797; NCT02627963). 31810797
Unknown unknown neuroblastoma not applicable Cambinol + Doxorubicin Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Adriamycin (doxorubicin) and Cambinol inhibited growth of a human neuroblastoma cell line in culture and in xenograft models (PMID: 22703804). 22703804
Unknown unknown acute promyelocytic leukemia not applicable A-366 Preclinical - Cell culture Actionable In a preclinical study, A-366 inhibited cell proliferation and increased differentiation in an acute promyelocytic leukemia cell line in culture (PMID: 26147105). 26147105
Unknown unknown colorectal cancer not applicable CA102N Preclinical - Cell line xenograft Actionable In a preclinical study, CA102N inhibited growth of colorectal cancer cell lines in culture, and resulted in rapid tumor shrinkage in cell line xenograft models (Cancer Res 2015;75(15 Suppl):Abstract nr 4471). detail...
Unknown unknown acute myeloid leukemia not applicable HM43239 Preclinical - Cell culture Actionable In a preclinical study, HM43239 treatment inhibited downstream signaling, reduced proliferation, and induced apoptosis in an acute myeloid leukemia cell line expressing leukemic stem cell marker in culture (Cancer Res 2019;79(13 Suppl):Abstract nr 1293). detail...
Unknown unknown breast carcinoma not applicable MRx0518 Preclinical Actionable In a preclinical study, MRx0518 stimulated immune response and reduced tumor size in syngeneic mouse models of breast carcinoma (Journal of Clinical Oncology 36, no. 15_suppl). detail...
Unknown unknown pediatric ependymoma not applicable CUDC-907 Phase I Actionable In a Phase I trial, CUDC-907 treatment resulted in stable disease for 6 treatment cycles in a pediatric patient with ependymoma (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 10542-10542; NCT02909777). detail...
Unknown unknown chronic lymphocytic leukemia not applicable Ibrutinib Clinical Study Actionable In a clinical study, treatment with Imbruvica (ibrutinib) resulted in a discontinuation-free survival rate at 1 year of 73.7% (232/315) and an absolute 1 year survival rate of 83.3% (264/315) in patients with relapsed or refractory chronic lymphocytic leukemia (PMID: 27756834). 27756834
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 lung non-small cell carcinoma no benefit Bevacizumab Phase III Actionable In a Phase IIIb trial, the combination of Avastin (bevacizumab) and standard of care therapy at first progression in non-small cell lung carcinoma patients previously treated with Avastin (bevacizumab) and chemotherapy and two maintenance cycles of Avastin (bevacizumab) did not result in a greater benefit compared to standard of care therapy alone, demonstrating a median overall survival of 11.9 mo vs 10.2 mo and a first to second progression-free survival of 5.5 mo vs 4.0 mo (PMID: 30177994; NCT01351415). 30177994
Unknown unknown Ewing sarcoma not applicable Talazoparib + Temozolomide Preclinical - Cell culture Actionable In a preclinical study, Ewing sarcoma cells treated with Temodar (temozolomide) combined with Talazoparib (BMN-673) resulted in strong synergism, demonstrating decreased cell viability in culture (PMID: 26438158). 26438158
Unknown unknown esophageal cancer not applicable Capmatinib Case Reports/Case Series Actionable In a Phase I trial, Tabrecta (capmatinib) demonstrated safety and preliminary efficacy, resulted in stable disease as best overall response in a patients with esophageal cancer (PMID: 30724423). 30724423
Unknown unknown Advanced Solid Tumor not applicable Erlotinib + Glesatinib Phase I Actionable In a Phase I trial, Glesatinib (MGCD265) and Tarceva (erlotinib) combination therapy demonstrated safety and preliminary clinical efficacy, resulted in partial response in 1 patient, and stable disease for 6 cycles or more in 16% (7/45) of patients with advanced solid tumors (J Clin Oncol 30, 2012 (suppl; abstr e13602)). detail...
Unknown unknown breast cancer not applicable AsiDNA Preclinical - Cell culture Actionable In a preclinical study, AsiDNA inhibited DNA repair and proliferation, and induced death in breast cancer cell lines in culture (PMID: 27559053). 27559053
Unknown unknown gastrointestinal system cancer no benefit Cetuximab + Cisplatin + Irinotecan Phase II Actionable In a Phase II trial, Erbitux (cetuximab) in combination with IC (irinotecan, cisplatin) demonstrated less efficacy and more toxicity, resulted in an overall response rate of 45.0% (32/71), median overall survival of 8.6 months; and median progression-free survival of 4.9 months in patients with metastatic esophageal or gastroesophageal junction cancers (PMID: 27382098). 27382098
Unknown unknown mantle cell lymphoma not applicable Ibrutinib + ONC201 Preclinical - Patient cell culture Actionable In a preclinical study, the combination of ONC201 and Imbruvica (ibrutinib) induced apoptosis in primary mantle cell lymphoma samples in culture, including Imbruvica (ibrutinib)-resistant samples, with greater efficacy than either agent alone (PMID: 26884599). 26884599
Unknown unknown acute myeloid leukemia not applicable Decitabine + Onvansertib Phase I Actionable In a Phase I trial, Onvansertib (PCM-075) and Dacogen (decitabine) combination therapy resulted in complete response in 33.3% (2/6) and complete response with incomplete hematologic remission in 16.7% (1/6) of evaluable patients with relapsed or refractory acute myeloid leukemia (Ann Oncol, 30 (Supplement 5): v435-v448, 2019; NCT03303339). detail...
Unknown unknown breast cancer not applicable NSC23766 Preclinical - Cell culture Actionable In a preclinical study, NSC23766 induced cell-cycle arrest and inhibited growth of breast cancer cell lines in culture (PMID: 20515940). 20515940
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 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 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 renal cell carcinoma not applicable Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in partial response in 2 patients and stable disease in 1 patient with renal cell carcinoma (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown glioblastoma multiforme not applicable Tepotinib Preclinical Actionable In a preclinical study, Tepotinib (EMD-1214063) induced tumor regression in mouse cell line xenograft models of glioblastoma (PMID: 23553846). 23553846
Unknown unknown lung non-small cell carcinoma not applicable Ro 31-8220 Preclinical - Cell culture Actionable In a preclinical study, Ro 31-8220 decreased CREB signaling, and induced apoptosis and inhibited growth of non-small cell lung cancer cells in culture (PMID: 18281471). 18281471
Unknown unknown Advanced Solid Tumor not applicable MK-1248 + Pembrolizumab Phase I Actionable In a Phase I trial, MK-1248 in combination with Keytruda (pembrolizumab) resulted in complete response in 6% (1/17) and partial response in 12% (2/17) of patients with advanced solid tumor (J Clin Oncol 36, 2018 (suppl; abstr 3029); NCT02553499). detail...
Unknown unknown lung cancer not applicable CT16 Preclinical - Pdx Actionable In a preclinical study, CT16 treatment decreased NOTCH and EGFR signaling and inhibited tumor growth in lung cancer patient-derived xenograft (PDX) models sensitive to EGFR blockade (PMID: 28275151). 28275151
Unknown unknown lung cancer not applicable SKI-G-801 Preclinical Actionable In a preclinical study, SKI-G-801 treatment reduced tumor growth in a syngeneic mouse model of lung cancer (Cancer Res 2019;79(13 Suppl):Abstract nr 2010). detail...
Unknown unknown pancreatic ductal adenocarcinoma not applicable JQ1 Preclinical - Pdx & cell culture Actionable In a preclinical study, treatment with JQ1 in patient derived xenograft (PDX) models of pancreatic ductal adenocarcinoma (PDAC) demonstrated tumor growth suppression and in PDAC cells, resulted in inhibition of Hedgehog and TGF-beta signaling and a decrease in desmoplastic stroma (PMID: 27528027). 27528027
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 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 urinary bladder cancer not applicable ASP5878 Phase I Actionable In a Phase I trial, ASP5878 demonstrated safety and some preliminary efficacy in patients with advanced solid tumors, including a bladder cancer patient with an FGFR gene mutation (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A165). detail...
Unknown unknown Advanced Solid Tumor not applicable MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in a variety of human advanced solid tumor 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 MGCD516 Phase I Actionable In a Phase I trial, MGCD516 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2575)). detail...
Unknown unknown breast cancer not applicable ICEC0942 Preclinical - Cell line xenograft Actionable In a preclinical study, ICEC0942 induced growth arrest of breast cancer cells in culture and in cell line xenograft models (PMID: 29545334). 29545334
Unknown unknown Advanced Solid Tumor not applicable Erlotinib + Ficlatuzumab Phase I Actionable In a Phase I trial, Ficlatuzumab (AV-299) in combination with Tarceva (erlotinib) was shown to be safe in treating patients with advanced solid tumors (PMID: 23493885, J Clin Oncol. 2010;28(Suppl) Abstract 2525). 23493885 detail...
Unknown unknown hepatocellular carcinoma not applicable Cabozantinib + CT-707 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Cometriq (Cabometyx, cabozantinib) and CT-707 resulted in synergism in hepatocellular carcinoma cells, demonstrating increased apoptosis and inhibition of colony formation in culture and decreased tumor weight in xenograft models (PMID: 27638856). 27638856
Unknown unknown lung non-small cell carcinoma not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) demonstrated safety and some anti-tumor activity across a range of advanced solid tumor types, with an objective response rate of 4% (1/23) in patients with non-small cell lung cancer (PMID: 25728526). 25728526
Unknown unknown 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 lymphoma not applicable Carfilzomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of a lymphoma cell lines in culture (PMID: 28753594). 28753594
Unknown unknown glioblastoma multiforme not applicable BMS-906024 Preclinical Actionable In a preclinical study, the pan-Notch inhibitor, BMS-906024, inhibited growth of glioblastoma xenografts (PMID: 26005526). 26005526
Unknown unknown oral squamous cell carcinoma not applicable Temsirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Torisel (temsirolimus) inhibited proliferation and migration of oral squamous cell carcinoma cells in culture and suppressed tumor growth in cell line xenograft models of human oral squamous cell carcinoma (PMID: 20858724). 20858724
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 liposarcoma not applicable Pazopanib Clinical Study Actionable In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 8 weeks and median survival of 7.3 months in patients with liposarcoma (PMID: 26970174). 26970174
Unknown unknown glioblastoma multiforme 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 Advanced Solid Tumor not applicable Carboplatin + Paclitaxel + Panobinostat Phase I Actionable In a Phase I trial, 52% (11/21) of patients with advanced solid tumors demonstrated stable disease when treated with Farydak (panobinostat), in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) (PMID: 22851205). 22851205
Unknown unknown colon cancer not applicable CVX-060 Preclinical - Cell line xenograft Actionable In a preclinical study, CVX-060 inhibited tumor growth in a colon cancer cell line xenograft model (PMID: 21233403). 21233403
Unknown unknown diffuse large B-cell lymphoma not applicable CUDC-907 Phase I Actionable In a Phase I trial, CUDC-907 was well-tolerated, demonstrated safety, and resulted in a 56% (5/9) objective response rate, including two complete responses and three partial responses, in patients with diffuse large B-cell lymphoma (PMID: 27049457). 27049457
Unknown unknown medulloblastoma not applicable Prexasertib Preclinical - Cell culture Actionable In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including medulloblastoma cell lines (PMID: 28270495). 28270495
Unknown unknown lung non-small cell carcinoma not applicable Cisplatin + Etoposide + Panobinostat + Radiotherapy Phase I Actionable In a Phase I trial, Farydak (panobinostat) in combination with chemotherapy (cisplatin and etoposide) and radiotherapy resulted in durable partial response in all (3) treated patients with stage III non-small cell lung cancer (PMID: 26317683). 26317683
Unknown unknown uveal melanoma no benefit Trametinib Phase I Actionable In a Phase I trial, Mekinist (trametinib) treatment resulted in stable disease as best response in 50% (8/16) of patients with uveal melanoma (PMID: 22805292; NCT00687622). 22805292
Unknown unknown lung adenocarcinoma not applicable Nintedanib Phase III Actionable In a Phase III clinical trial, the combination of Ofev (nintedanib) and Taxotere (docetaxel) improved progression-free survival and overall survival in patients with lung adenocarcinoma compared to Taxotere (docetaxel) alone (PMID: 24411639). 24411639
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 head and neck squamous cell carcinoma not applicable Metformin Clinical Study Actionable In a meta-analysis, Glucophage (metformin) treatment decreased recurrence and metastasis and improved overall survival of head and neck squamous cell carcinoma patients (PMID: 25636350). 25636350
Unknown unknown lung non-small cell carcinoma not applicable Ascrinvacumab Phase I Actionable In a Phase I trial, a patient with non-small cell lung carcinoma demonstrated a partial response for 308 days when treated with PF-03446962 (PMID: 26655846). 26655846
Unknown unknown colorectal cancer not applicable WYE-354 Preclinical Actionable In a preclinical study, WYE-354 induced cell cycle arrest and inhibited cell proliferation of colorectal cancer cells in culture (PMID: 19584280). 19584280
Unknown unknown lung non-small cell carcinoma not applicable BPI-9016M Phase I Actionable In a Phase I trial, BPI-9016M treatment was well-tolerated in patients with non-small cell lung cancer who had progressed on prior therapy (n=20), and of nineteen evaluable patients, one patient had a partial response and 10 patients experienced stable disease (PMID: 31948451; NCT02478866). 31948451
Unknown unknown peripheral T-cell lymphoma not applicable Belinostat FDA approved Actionable In clinical trials that supported FDA approval, treatment with Beleodaq (belinostat) resulted in an objective response rate of 25.8% (31/120) and a median overall survival of 7.9 months in patients with peripheral T-cell lymphoma (PMID: 26101246). detail... 26101246
Unknown unknown acute myeloid leukemia not applicable ONC201 Preclinical - Pdx & cell culture Actionable In a preclinical study, ONC201 treatment induced ATF4 expression and apoptosis in acute myeloid leukemia (AML) cell lines in culture, independent of Tp53 status, and was toxic to AML primary samples in culture, resulting in decreased engraftment in xenograft models (PMID: 26884599). 26884599
Unknown unknown Advanced Solid Tumor not applicable BGT226 Phase I Actionable In a Phase I trial of BGT226 in patients with advanced solid tumors, limited preliminary antitumor activity and inconsistent target inhibition were observed (PMID: 22357447). 22357447
Unknown unknown Advanced Solid Tumor not applicable BGT226 Phase I Actionable In a Phase I trial, BGT226 treatment was tolerated, and resulted in stable disease as best response in 28% (5/18) of patients with advanced solid tumors (PMID: 31192075). 31192075
Unknown unknown Advanced Solid Tumor not applicable Alisertib + Sapanisertib Phase I Actionable In a Phase I trial, the combination of Alisertib (MLN8237) and Sapanisertib (MLN0128) demonstrated tolerability in patients with advanced solid tumors, with 70% (7/10) of patients demonstrating stable disease for a median duration of 4 months (PMID: 32414750). 32414750
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 colon cancer not applicable LF3 Preclinical - Cell line xenograft Actionable In a preclinical study, LF3 decreased expression of Wnt target genes and reduced tumor growth in colon cancer cell line xenograft models derived from cells with high levels of Wnt signaling (PMID: 26645562). 26645562
Unknown unknown synovial sarcoma not applicable Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 10% (1/10) of patients with synovial sarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). detail...
Unknown unknown Advanced Solid Tumor not applicable ON123300 Preclinical Actionable In a preclinical study ON123300 inhibited growth of a variety of human solid tumor cell lines in culture (PMID: 24417566). 24417566
Unknown unknown pancreatic adenocarcinoma not applicable Gemcitabine + NSC109555 Preclinical - Cell culture Actionable In a preclinical study, NSC109555 enhanced the anti-tumor effects of Gemzar (gemcitabine) in pancreatic adenocarcinoma cells in culture, resulting in greater decreased colony formation, inhibition of cell proliferation, and apoptotic activity (PMID: 23855452). 23855452
Unknown unknown prostate cancer not applicable DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, prostate cancer cells treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture and inhibition of tumor growth in cell-line xenograft models (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown leiomyosarcoma not applicable Trabectedin FDA approved Actionable In a Phase III trial that supported FDA approval, Yondelis (trabectedin) treatment resulted in an improved median progression-free survival of 4.2 months versus 1.5 months with Deticene (dacarbazine) in patients with unresectable or metastatic liposarcoma or leiomyosarcoma (PMID: 28774898; NCT01343277). detail... 28774898
Unknown unknown melanoma not applicable Hu3F8-BsAb Preclinical - Cell line xenograft Actionable In a preclinical study, Hu3F8-BsAb treatment induced cell killing in melanoma cell lines expressing GD2 in culture, and treatment with Hu3F8-BsAb plus human peripheral blood mononuclear cells inhibited tumor growth and improved survival in melanoma cell line xenograft models in combination with transplanted PBMCs (PMID: 25542634). 25542634
Unknown unknown melanoma not applicable Pembrolizumab + SD-101 Phase I Actionable In a Phase Ib trial, SD-101 in combination with Keytruda (pembrolizumab) resulted in an overall response of 78% (7/9), an estimated 12-month progression-free survival rate of 88% (8/9), and an overall survival rate of 89% (8/9) in patients with unresectable or metastatic malignant melanoma naïve to anti-PD-1 therapy, and an overall response of 15% (2/13) in patients received prior anti-PD-1 therapy (PMID: 30154193; NCT02521870). 30154193
Unknown unknown glioblastoma multiforme not applicable Temozolomide + Vistusertib Phase I Actionable In a Phase I trial, combination of Vistusertib (AZD2014) and Temodar (temozolomide) demonstrated safety in patients with previously treated glioblastoma multiforme, resulted in a partial response in 8% (1/13) and stable disease in 38% (5/13) of the patients, with a 6-month progression-free survival rate of 26% (PMID: 31707687). 31707687
Unknown unknown prostate cancer not applicable CAB-AXL-ADC Preclinical - Cell line xenograft Actionable In a preclinical study, CAB-AXL-ADC treatment inhibited tumor growth in a prostate cancer cell line xenograft model (Cancer Res 2018;78(13 Suppl):Abstract nr 827). detail...
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 breast cancer not applicable AZD7648 Preclinical - Cell line xenograft Actionable In a preclinical study, AZD7648 treatment inhibited tumor growth in a cell line xenograft model of breast cancer, however, with decreased response compared to AZD7648 and Doxil (pegylated liposomal-doxorubicin) combination treatment (PMID: 31699977). 31699977
Unknown unknown prostate cancer no benefit AT-101 + Bicalutamide Phase I Actionable In a Phase I trial, the combination of AT-101 and Casodex (bicalutamide) resulted in an undetectable PSA in 31% (17/55) of patients with castration resistant prostate cancer, however, the percentage did not reach the prespecified level and thus, the theraputic combination will not be pursued further (PMID: 26476589). 26476589
Unknown unknown hematologic cancer not applicable Idelalisib Phase I Actionable In a Phase I trial, Zydelig (idelalisib) treatment of patients with hematological malignancies produced an overall response rate of 72% (39/54) (PMID: 24615777). 24615777
Unknown unknown multiple myeloma not applicable Dexamethasone + Isatuximab + Pomalidomide FDA approved Actionable In a Phase III (ICARIA-MM) trial that supported FDA approval, addition of Sarclisa (isatuximab-irfc) to Pomalyst (pomalidomide) and dexamethasone significantly improved progression-free survival (11.5 vs 6.5 months, HR=0.596, p=0.001) in patients with relapsed and refractory multiple myeloma (PMID: 31735560; NCT02990338). detail... 31735560
Unknown unknown gastric adenocarcinoma not applicable Torkinib Preclinical Actionable In a preclinical study, Torkinib (PP242) inhibited signaling of the PI3K/AKT/mTOR pathway and subsequent cell proliferation of gastric cancer cells in culture (PMID: 25035961). 25035961
Unknown unknown 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 Advanced Solid Tumor not applicable GDC-0919