Molecular Profile Detail

Profile Name Unknown unknown
Gene Variant Detail

Unknown unknown ()

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 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 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 Advanced Solid Tumor not applicable SR9009 Preclinical - Cell culture Actionable In a preclinicl study SR9009 demonstrated toxicity in a wide range of tumor cell lines harboring different driver mutations, but not in normal cell lines in culture (PMID: 29320480). 29320480
Unknown unknown 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 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 lymphoblastic lymphoma not applicable Ibrutinib + VLX1570 Preclinical - Cell culture Actionable In a preclinical study, the combination of Imbruvica (ibrutinib) and VLX1570 worked synergistically to reduce viability of an Imbruvica (ibrutinib)-resistant Waldenstrom macroglobulinemia cell line in culture (PMID: 27813535). 27813535
Unknown unknown neuroendocrine tumor not applicable Everolimus + Octreotide Phase III Actionable In a Phase III trial, addition of Afinitor (everolimus) to Octreotide did not significantly improve median overall survival (29.2 vs 35.2 months) compared to placebo in patients with advanced neuroendocrine tumors associated with carcinoid syndrome (PMID: 28444114). 28444114
Unknown unknown breast cancer not applicable H3B-6545 Preclinical - Cell line xenograft Actionable In a preclinical study, a breast cancer cell line xenograft model was sensitive to treatment with H3B-6545, demonstrating greater antitumor activity compared to Faslodex (fulvestrant) (Cancer Res 2017;77(13 Suppl):Abstract nr DDT01-04). detail...
Unknown unknown sarcoma not applicable BI 853520 Phase I Actionable In a Phase I trial, BI 853520 demonstrated safety and some anti-tumor efficacy, resulting in stable disease in 28.6% (4/14) of patients with soft tissue sarcoma, one of whom had stable disease lasting greater than 150 days (PMID: 30756308; NCT01335269). 30756308
Unknown unknown 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 hepatocellular carcinoma not applicable LCL161 + Paclitaxel Preclinical Actionable In a preclinical study, the combination of LCL161 and Taxol (paclitaxel) inhibited proliferation of hepatocellular carcinoma cells in culture (PMID: 24976294). 24976294
Unknown unknown breast cancer not applicable Altiratinib Preclinical Actionable In a preclinical study, Altiratinib (DCC-0701) inhibited tumor growth and decreased lung metastasis in a mouse breast cancer model (PMID: 26285778). 26285778
Unknown unknown non-Hodgkin lymphoma not applicable DS-3201b Phase I Actionable In a Phase I trial, DS-3201b demonstrated preliminary clinical activity in patients with non-Hodgkin lymphoma, with an overall response rate of 53% (8/15; 1 complete response/remission, and 7 partial responses), stable disease in 5 patients, and 8 patients on treatment with tumor shrinkage for greater than 24 weeks (Blood Dec 2017, 130 (Suppl 1) 4070; NCT02732275). detail...
Unknown unknown triple-receptor negative breast cancer not applicable Bortezomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, EDO-S101 and Velcade (bortezomib) worked synergistically to decrease viability of triple negative breast cancer cell lines in culture (PMID: 28753594). 28753594
Unknown unknown chronic myeloid leukemia not applicable Bosutinib Clinical Study Actionable In a meta-analysis, Bosulif (bosutinib) treatment was associated with increased rate of major molecular response compared with Gleevec (imatinib) (Odds Ratio (OR): 1.86 [1.29-2.70]), but not improved overall survival (OR: 2.38 [0.82-6.89]), and was associated with a trend toward increased risk of vascular occlusive events in chronic myeloid leukemia patients (PMID: 26847662). 26847662
Unknown unknown marginal zone B-cell lymphoma not applicable Idelalisib Phase II Actionable In a Phase II trial, Zydelig (idelalisib) treatment resulted in an overall response rate of 47% (7/15) in patients with marginal zone B-cell lymphoma (PMID: 24450858; NCT01282424). 24450858
Unknown unknown breast cancer not applicable Fluoxymesterone Phase II Actionable In a Phase II trial, a patient with advanced breast cancer that developed resistance to long term hormonal therapy demonstrated sensitivity to Halotestin (fluoxymesterone) (PMID: 1590276). 1590276
Unknown unknown breast cancer not applicable Fluoxymesterone Phase I Actionable In a Phase I study, rates of overall survival and relapse-free survival did not differ between early stage breast cancer patients treated with Halotestin (fluoxymesterone) and Nolvadex (tamoxifen) combined versus those treated with Nolvadex (Tamoxifen) alone (PMID: 16538529). 16538529
Unknown unknown ovary epithelial cancer not applicable Bevacizumab + Niraparib Phase I Actionable In a Phase I trial, Avastin (bevacizumab) and Zejula (niraparib) combination treatment resulted in complete response in 8% (1/12), partial response in 33% (4/12), and a disease control rate of 91% in platinum-sensitive ovarian cancer patients (J Clin Oncol 34, 2016 (suppl; abstr 5555)). detail...
Unknown unknown 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). 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). 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). 29305552
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
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 Advanced Solid Tumor not applicable A-1331852 Preclinical - Cell line xenograft Actionable In a preclinical study, A-1331852 inhibited tumor growth in several solid tumor cell line xenograft models (PMID: 25787766). 25787766
Unknown unknown acute myeloid leukemia not applicable GNE-781 Preclinical - Cell line xenograft Actionable In a preclinical study, GNE-781 treatment resulted in tumor growth inhibition and reduced MYC transcript levels in acute myeloid leukemia cell line xenograft models (PMID: 28892380). 28892380
Unknown unknown astrocytoma not applicable Niraparib Preclinical - Cell line xenograft Actionable In a preclinical study, pediatric high grade astrocytoma cell lines treated with Zejula (niraparib) demonstrated decreased cell viability and proliferation in culture, and a small survival benefit in xenograft models (PMID: 26351319). 26351319
Unknown unknown breast cancer not applicable COG112 Preclinical Actionable In a preclinical study, breast cancer cells treated with COG112 demonstrated a decrease in cell proliferation and cell migration in culture (PMID: 21297667). 21297667
Unknown unknown Advanced Solid Tumor not applicable APTO-253 Phase I Actionable In a Phase I clinical trial, APTO-253 demonstrated safety and resulted in stable disease in 24% (5/32) of evaluable solid tumor patients (PMID: 26268924). 26268924
Unknown unknown Advanced Solid Tumor 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 Hodgkin's lymphoma not applicable Idelalisib Phase II Actionable In a Phase II trial, Idelalisib treatment resulted in an overall response rate of 20% (5/25) in Hodgkin's lymphoma patients, with one patient experiencing a complete response and four patients experiencing a partial response (PMID: 28327905). 28327905
Unknown unknown 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 brain stem glioma not applicable MRK-003 Preclinical - Cell culture Actionable In a preclinical study, MRK-003 increased apoptosis and decreased growth of diffuse pontine glioma cell lines in culture (PMID: 26115193). 26115193
Unknown unknown 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 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 lung cancer not applicable AZD7762 + VX-970 Preclinical Actionable In a preclinical study, lung cancer cells treated with VX-970 resulted in a synthetic lethal effect when combined with AZD7762, demonstrating increased survival and decreased tumor volume in xenograft models (PMID: 26748709). 26748709
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 pancreatic cancer not applicable Galunisertib Phase I Actionable In a Phase I trial, two patients with pancreatic cancer demonstrated a best response of stable disease when treated with Galunisertib (LY2157299) (PMID: 26526984; NCT01722825). 26526984
Unknown unknown endometrial cancer not applicable Capivasertib + Olaparib Phase I Actionable In a Phase I trial, the combination of AZD5363 and Lynparza (olaparib) was well-tolerated and demonstrated preliminary activity in patients with endometrial, ovarian, or triple-negative breast cancer (TNBC), with an overall response rate of 24% (7/30; all partial responses, 1 ovarian, 4 endometrial, and 2 TNBC) and stable disease for greater than 4 months in 6 additional patients, and a response rate of 50% (4/8) in endometrial cancer patients (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #391P; NCT02208375). detail...
Unknown unknown breast carcinoma not applicable RXDX-106 + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, RXDX-106 in combination with an unspecified anti-PD-1 antibody resulted in near complete regression in orthotopic animal models of breast carcinoma (Eur J Cancer, Vol 69, Supplement 1, December 2016, Page S31). detail...
Unknown unknown Ewing sarcoma not applicable Talazoparib Phase I Actionable In a Phase I trial, Talazoparib (BMN-673) treatment in patients with Ewing sarcoma did not result in any objective responses, however, resulted in a clinical benefit rate of 23% (PMID: 28242752). 28242752
Unknown unknown Advanced Solid Tumor not applicable MCLA-128 Phase Ib/II Actionable In a Phase I/II trial, MCLA-128 demonstrated safety and preliminary efficacy, resulted in partial response in 3.6% (1/28) and stable disease in 7.1% (2/28) of patients with advanced solid tumors (Cancer Res 2016;76(14 Suppl):Abstract nr CT050). detail...
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). 20697094
Unknown unknown ovarian cancer not applicable SST0116CL1 Preclinical - Cell line xenograft Actionable In a preclinical study, SST0116CL1 inhibited tumor growth in p-glycoprotein over expressing ovarian cancer cell line xenograft models (PMID: 25096516). 25096516
Unknown unknown neuroblastoma not applicable JSH-150 Preclinical - Cell culture Actionable In a preclinical study, JSH-150 inhibited proliferation of a neuroblastoma cell line in culture (PMID: 30253346). 30253346
Unknown unknown angiosarcoma not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) treatment resulted in partial response in 2 angiosarcoma patients (PMID: 27502708). 27502708
Unknown unknown Advanced Solid Tumor not applicable Nintedanib Phase I Actionable In a Phase I trial, Ofev (nintedanib) demonstrated safety and some preliminary efficacy in patients with advanced solid tumors (PMID: 25795637). 25795637
Unknown unknown pancreatic ductal adenocarcinoma not applicable Selumetinib + SHP099 Preclinical - Pdx & cell culture Actionable In a preclinical study, the combination of SHP099 and Selumetinib (AZD6244) resulted in greater sensitivity compared to either agent alone in pancreatic ductal adenocarcinoma cells, demonstrating decreased cell viability and reduced colony formation in culture and decreased tumor growth in patient-derived xenograft (PDX) models (PMID: 30045908). 30045908
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 gastrointestinal system cancer not applicable Axitinib + Fluorouracil + Leucovorin + Oxaliplatin Phase I Actionable In a Phase I trial, Inlyta (axitinib), in combination with FOLFOX, demonstrated safety and some efficacy in patients with gastrointestinal tumors (PMID: 24423921). 24423921
Unknown unknown prostate cancer not applicable Docetaxel + Everolimus Phase I Actionable In a Phase I study, Afinitor (everolimus), in combination with Taxotere (docetaxel), demonstrated safety, but minimal efficacy in patients with prostate cancer (PMID: 25450031). 25450031
Unknown unknown pancreatic cancer not applicable AS-252424 Preclinical Actionable In a preclinical study, AS-252424 inhibited proliferation of pancreatic cancer cells in culture (PMID: 20876794). 20876794
Unknown unknown ovarian cancer not applicable Nintedanib Phase I Actionable In a Phase I clinical trial, Vargatef (nintedanib) demonstrated safety in patients with ovarian cancers, clinical trials to determine efficacy in these patients are ongoing (PMID: 19889612). 19889612
Unknown unknown acute myeloid leukemia not applicable (-)BI97D6 Preclinical - Patient cell culture Actionable In a preclinical study, (-)BI97D6 decreased live cell number in primary acute myeloid leukemia samples from refractory patients in culture (PMID: 26045609 ). 26045609
Unknown unknown triple-receptor negative breast cancer not applicable Carfilzomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of triple negative breast cancer cell lines in culture (PMID: 28753594). 28753594
Unknown unknown diffuse large B-cell lymphoma not applicable GS-5829 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, diffuse large B-cell lymphoma cell lines demonstrated greater cell growth inhibition when treated with a combination of GS-5829 and Venclexta (venetoclax) compared to either agent alone in culture (Blood 2016 128:5104). detail...
Unknown unknown diffuse large B-cell lymphoma not applicable Ibrutinib Phase II Actionable In a Phase II trial, Imbruvica (ibrutinib) treatment demonstrated preferential efficacy in the ABC subtype of diffuse large B-cell lymphoma (DLBCL) compared to the GCB subtype, resulted in complete response in 8% (2/25) and partial response in 32% (8/25) of ABC DLBCL patients, but only partial response in 5.3% (1/19) of GCB DLBCL patients (Blood 2012, 120 (21): 686). detail...
Unknown unknown clear cell renal cell carcinoma not applicable PF-03446962 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 mantle cell lymphoma not applicable BGB-3111 Preclinical - Cell line xenograft Actionable In a preclinical study, BGB-3111 inhibited BTK signaling and proliferation of mantle cell lymphoma cell lines in culture, and demonstrated antitumor activity in a mantle cell lymphoma cell line xenograft model (ibrutinib) (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 2597). detail...
Unknown unknown Advanced Solid Tumor not applicable Adavosertib Phase I Actionable In a Phase I trial, Adavosertib (MK-1775) combined with a chemotherapy resulted in a partial response of 10% (17/176) and stable disease in 53% (94/176) of patients with advanced solid tumors (PMID: 27601554). 27601554
Unknown unknown Advanced Solid Tumor not applicable Adavosertib Phase I Actionable In a Phase I trial, Adavosertib (MK-1775) treatment demonstrated safety and preliminary efficacy, resulted in an overall response rate of 3.4% (2/62), a disease control rate of 48.4% (30/62), and a median progression-free survival of 2.7 months in patients with advanced solid tumors (AACR Annual Meeting 2019, Abstract CT022). detail...
Unknown unknown chronic myeloid 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). detail... 23935038
Unknown unknown chronic myeloid leukemia not applicable Ponatinib Clinical Study Actionable In a meta-analysis, Iclusig (ponatinib) treatment was associated with increased rate of major molecular response compared with Gleevec (imatinib) (Odds Ratio (OR): 4.95 [0.97-25.19]), but not improved overall survival (OR: 2.00 [0.21-19.33]), and was associated with increased risk of vascular occlusive events (OR: 3.47 [1.23-9.78]) in patients with chronic myeloid leukemia (PMID: 26847662). 26847662
Unknown unknown Burkitt lymphoma not applicable Cerdulatinib Preclinical Actionable In a preclinical study, treatment with Cerdulatinib (PRT062070) resulted in decreased viability and increased apoptosis of Burkitt lymphoma cells in culture (PMID: 25253883). 25253883
Unknown unknown non-small cell lung carcinoma not applicable Carboplatin + Paclitaxel + Vorinostat Phase II Actionable In a Phase II trial, the combination of Paraplatin (carboplatin) and Taxol (paclitaxel) plus Zolinza (vorinostat) compared to the addition of placebo resulted in a greater median progression-free survival (6.0mo vs 4.1mo) and overall survival (13.0 mo vs 9.7 mo) in patients with non-small cell lung carcinoma (PMID: 19933908). 19933908
Unknown unknown Advanced Solid Tumor not applicable Prexasertib Phase I Actionable In a Phase I trial, Prexasertib (LY2606368) treatment resulted in partial response in 4.4% (2/45) and stable disease ranging from 1.2 to 6.7 months in 33.3% (15/45) of patients with advanced solid tumors (PMID: 27044938). 27044938
Unknown unknown melanoma not applicable LN-144 Phase II Actionable In a Phase II trial, LN-144 treatment resulted in an objective response rate of 33% (3/9, 1 complete response, 2 partial response) in patients with advanced metastatic melanoma (Journal of Clinical Oncology 35, no. 15_suppl (May 20 2017) 3045-3045; NCT02360579). detail...
Unknown unknown chronic myeloid leukemia not applicable Busulfan + Cyclophosphamide FDA approved Actionable In a clinical trial that supported FDA approval, combination of Busulfex (busulfan) and Cytoxan (cyclophosphamide) as a conditioning regimen prior to bone marrow transplantation was better tolerated and resulted in favorable 4-year probabilities of survival and event-free survival (0.86 vs 0.72) compared to Cytoxan (cyclophosphamide) plus total body irradiation in patients with chronic myeloid leukemia (PMID: 8081005). 8081005
Unknown unknown hematologic cancer no benefit Milatuzumab Phase I Actionable In a Phase I trial, Milatuzumab (hLL1) treatment of a mixed cohort of B-cell malignancies patients, resulted in stable disease in 35% (8/23), but there was no evidence of tumor shrinkage (PMID: 25754579). 25754579
Unknown unknown prostate cancer not applicable TGX-221 Preclinical - Cell line xenograft Actionable In a preclinical study, TGX-221 inhibited proliferation and decreased Akt phosphorylation in human cell line xenograft models of prostate cancer (PMID: 25620467). 25620467
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 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 endometrial cancer not applicable BEZ235 Preclinical Actionable In a preclinical study, BEZ235 suppressed tumor growth in endometrial xenografts (PMID: 22662154). 22662154
Unknown unknown hepatocellular carcinoma not applicable Linifanib Phase II Actionable In a Phase II clinical trial, single-agent linifanib was found safe and efficacious in patients with advanced hepatocellular carcinoma (PMID: 22833179). 22833179
Unknown unknown Advanced Solid Tumor no benefit MSC2490484A Phase I Actionable In a Phase I trial, MSC2490484A (M3814) demonstrated safety, but limited clinical activity, in patients with solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2556)). detail...
Unknown unknown gastrointestinal neuroendocrine tumor not applicable Pazopanib Clinical Study Actionable In a clinical study, Votrient (pazopanib) treatment in patients with gastroenteropancreatic neuroendocrine tumors resulted in an overall response rate of 24% (19/124), stable disease in 39.5% (49/124), a progression free survival of 36% at six months, and a median overall survival of 10.2 months (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 446P). detail...
Unknown unknown hematologic cancer not applicable 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 acute myeloid leukemia not applicable SY-1365 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, SY-1365 and Venclexta (venetoclax) synergistically induced apoptosis in acute myeloid leukemia cells in culture (Proceedings of the AACR, Vol 58, April 2017, Abstract # 1151). detail...
Unknown unknown prostate cancer not applicable SM88 Phase II Actionable In a Phase II trial, SM-88 treatment resulted in stable (4/10) or rising (5/10) testosterone levels, and none of the toxicity commonly associated with androgen deprivation therapy in patients with non-metastatic recurrent prostate cancer (J Clin Oncol 36, 2018 (suppl 6S; abstr 175); NCT02796898). detail...
Unknown unknown 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 urinary bladder cancer not applicable Celecoxib Preclinical - Cell culture Actionable In a preclinical study, Celebra (celecoxib) treatment resulted in decreased cell viability in multiple human bladder cancer cell lines in culture (PMID: 27406983). 27406983
Unknown unknown Advanced Solid Tumor not applicable Erlotinib + Lumretuzumab Phase Ib/II Actionable In a Phase Ib trial, treatment with the combination of Tarceva (erlotinib) and Lumretuzumab demonstrated minimal clinical efficacy in ERBB3 (HER3)-positive advanced solid tumor patients, resulting in an objective response rate of 4.2% (3/71), including a partial response in a patient with ovarian cancer and in two patients with squamous non-small cell lung carcinoma (PMID: 28600476). 28600476
Unknown unknown chondrosarcoma not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) treatment resulted in partial response in a dedifferentiated chondrosarcoma patient (PMID: 27502708). 27502708
Unknown unknown Advanced Solid Tumor not applicable DS-3078a Phase I Actionable In a Phase I trial, DS-3078a demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Mol Cancer Ther November 2013 12:C173). detail...
Unknown unknown 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 Preclinical - Cell culture Actionable In a preclinical study, head and neck squamous cell carcinoma cell lines, either human papilloma virus positive or negative, demonstrated decreased cell proliferation in culture when treated with Prexasertib (LY2606368) (PMID: 28138028). 28138028
Unknown unknown head and neck squamous cell carcinoma not applicable Prexasertib Phase 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 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 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 Advanced Solid Tumor not applicable XL147 Phase I Actionable In a Phase I study, 25/56 (43.9%) of patients with advanced solid tumors had stable disease as a best response to treatment with XL147, and one patient with NSCLC showed a partial response to XL147 (PMID: 24166903). 24166903
Unknown unknown chronic lymphocytic leukemia not applicable Bendamustine + Obinutuzumab + Venetoclax Phase II Actionable In a Phase II trial (CLL2-BAG), sequential treatment with Treanda (bendamustine) and Gazyva (obinutuzumab) combined with Venclexta (venetoclax) resulted in an response rate of 95% (60/63) in patients with chronic lymphocytic leukemia, without unexpected or cumulative toxicities (PMID: 30115596; NCT02401503). 30115596
Unknown unknown colorectal cancer not applicable INCB054329 Preclinical - Cell line xenograft Actionable In a preclinical study, INCB054329 promoted apoptosis and decreased MYC expression in colorectal cancer cell lines in culture and demonstrated anti-tumor activity in colorectal cancer cell line xenograft models (AACR; 2015. Abstract nr 3525). detail...
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 multiple myeloma no benefit Ricolinostat Phase Ib/II Actionable In a Phase Ib/II trial, Ricolinostat (ACY-1215) as a single therapy did not result in toxicity nor clinical benefit in multiple myeloma patients (PMID: 28053023). 28053023
Unknown unknown peripheral T-cell lymphoma not applicable Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in partial response in 50% (2/4) of patients with peripheral T-cell lymphoma (PMID: 24852792). 24852792
Unknown unknown acute myeloid leukemia not applicable Galinpepimut-S Phase II Actionable In a Phase II trial, Galinpepimut-S treatment in adults with acute myeloid leukemia in their first complete remission (CR1) was well tolerated and resulted in a median disease free survival of 16.9 months from CR1, 47% (9/19) survival beyond 3 years in evaluable patients, and immunologic responses in 64% (9/14) of patients tested (PMID: 29386195; NCT01266083). 29386195
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 pancreatic cancer not applicable Radiotherapy + VX-970 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with VX-970 enhanced radiotherapy efficacy in pancreatic cells resulting in apoptotic induction in culture and DNA damage and tumor growth delay in cell line xenograft models (PMID: 23222511). 23222511
Unknown unknown Advanced Solid Tumor not applicable NSC156529 Preclinical Actionable In a preclinical study, NSC156529 inhibited growth of transformed human cell lines in culture (PMID: 26294745). 26294745
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 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 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 malignant glioma not applicable GDC-0084 Phase I Actionable In a Phase I trial, GDC-0084 treatment resulted in metabolic partial response in 18.5% (5/27) and stable disease in 40.4% (19/47) of high-grade glioma patients (J Clin Oncol (Meeting Abstracts) May 2016 vol. 34 no. 15_suppl 2012). detail...
Unknown unknown colorectal cancer predicted - sensitive ST-162 + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, ST-162 therapy combined with an anti-PD-1 antibody resulted in greater tumor growth inhibition than treatment with either agent alone in a colorectal cancer mouse model (PMID: 28775144). 28775144
Unknown unknown ovarian cancer not applicable AZD1480 Preclinical Actionable In a preclinical study, AZD1480 inhibited cell proliferation of ovarian cancer cells with active Jak-Stat signaling in culture and blocked tumor growth in transgenic mouse models of ovarian cancer (PMID: 25646015). 25646015
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 Advanced Solid Tumor not applicable Parsaclisib Preclinical Actionable In a preclinical study, Parsaclisib (INCB050465) demonstrated immunomodulatory and anti-tumor activity in mouse tumor models with intact immune systems (Mol Cancer Ther December 1 2015 (14) (12 Supplement 2) C103). detail...
Unknown unknown pancreatic cancer not applicable Gemcitabine + MVT-5873 + Nab-paclitaxel Preclinical - Cell line xenograft Actionable In a preclinical study, MVT-5873 enhanced efficacy of the combination therapy, Gemzar (gemcitabine) and Abraxane (nab-paclitaxel), in pancreatic xenograft models, demonstrating increased inhibition of tumor growth and greater reduction of tumor volume when compared to single agents (Cancer Res 2016;76(24 Suppl):Abstract nr A73). detail...
Unknown unknown malignant fibroxanthoma not applicable Sapanisertib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with Sapanisertib (MLN0128) resulted in growth suppression and arrest in a myxofibrosarcoma cell line in culture, and inhibited tumor growth in myxofibrosarcoma cell line xenograft models (PMID: 27577794). 27577794
Unknown unknown Advanced Solid Tumor not applicable ARQ 751 Preclinical - Cell culture Actionable In a preclinical study, treatment with ARQ 751 resulted in anti-proliferative activity in a variety of cancer cell lines in culture (PMID: 26469692). 26469692
Unknown unknown islet cell tumor not applicable GDC-0326 Preclinical Actionable In a preclinical study, GDC-0326 inhibited AKT activation, decreased tumor growth, metastasis, and angiogenesis and increased lifespan in a transgenic mouse model of pancreatic neuroendocrine tumor (PMID: 27225693). 27225693
Unknown unknown multiple myeloma not applicable KDM5-C70 Preclinical - Cell culture Actionable In a preclinical study, KDM5-C70 inhibited cell proliferation of a multiple myeloma cell line in culture (PMID: 27214403). 27214403
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...
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). 28434648
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 Advanced Solid Tumor not applicable Bendamustine + Veliparib Phase I Actionable In a Phase I trial, the combination of Bendamustine and Veliparib (ABT-888) resulted in stable disease in 63% (12/19) of patients with advanced solid tumors (PMID: 28314788; NCT01326702). 28314788
Unknown unknown prostate adenocarcinoma not applicable ONC201 Clinical Study Actionable In a clinical case study, a prostate adenocarcinoma patient demonstrated extended stable disease for 27 weeks when treated with ONC201 (TIC-10) (PMID: 28331050). 28331050
Unknown unknown epithelioid sarcoma not applicable Entinostat Preclinical Actionable In a preclinical study, Entinostat inhibited growth and colony formation of epithelioid sarcoma cells in culture (PMID: 26396249). 26396249
Unknown unknown colon adenocarcinoma not applicable NMS-P937 Preclinical - Cell line xenograft Actionable In a preclinical study, human colon adenocarcinoma cells demonstrated tumor growth inhibition in cell line xenograft models when treated with NMS-P937 (PMID: 22319201). 22319201
Unknown unknown cholangiocarcinoma not applicable Zebularine Preclinical - Cell culture Actionable In a preclinical study, Zebularine treatment decreased DNMT1 expression,and induced apoptosis in cholangiocarcinoma cell lines in culture (PMID: 25799509). 25799509
Unknown unknown neuroblastoma not applicable GANT61 Preclinical - Cell line xenograft Actionable In a preclinical study, GANT61 induced apoptosis and decreased growth of neuroblastoma cells in culture, and inhibited tumor growth in neuroblastoma cell line xenograft models (PMID: 22949014). 22949014
Unknown unknown 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 ovarian cancer not applicable Selumetinib + SHP099 Preclinical - Cell culture Actionable In a preclinical study, the combination therapy of Selumetinib (AZD6244) and SHP099 resulted in decreased colony formation and reduced cell viability in ovarian cancer cells in culture and inhibition of tumor growth and reduced tumor angiogenesis in a patient-derived xenograft (PDX) model of ovarian cancer (PMID: 30045908). 30045908
Unknown unknown colon cancer not applicable BGP-15 Preclinical - Cell line xenograft Actionable In a preclinical study, BGP-15 induced apoptosis and inhibited growth of colon cancer cells in culture and in cell line xenograft models (PMID: 22661288). 22661288
Unknown unknown multiple myeloma not applicable NSC126405 Preclinical - Cell line xenograft Actionable In a preclinical study, NSC126405 inhibited tumor growth during 11 days of treatment in multiple myeloma cell line xenograft models and 22% (2/9) of the mice demonstrated tumor regression post treatment (PMID: 27530328). 27530328
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 melanoma not applicable Denileukin diftitox + Sirolimus Preclinical Actionable In a preclinical study, the combination of Ontak (denileukin diftitox) and Rapamune (sirolimus) resulted in a greater decrease in tumor volume compared to treatment with either agent alone in a melanoma mouse model (PMID: 27737881). 27737881
Unknown unknown adenoid cystic carcinoma not applicable Dovitinib Phase II Actionable In a Phase II trial, Dovitinib (TKI258) treatment was tolerated and demonstrated limited clinical activity in patients with adenoid cystic carcinoma, resulting in partial response in 5.9% (2/34) of patients, suppression of overall tumor growth rate, and a median progression-free survival of 8.2 months (PMID: 28377480). 28377480
Unknown unknown Advanced Solid Tumor not applicable 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 rhabdomyosarcoma not applicable TAS4464 Preclinical - Cell line xenograft Actionable In a preclinical study, TAS4464 inhibited growth and induced apoptosis in rhabdomyosarcoma cell lines in culture, resulted in tumor suppression in cell line xenograft models (Cancer Res 2016;76(14 Suppl):Abstract nr 3777). detail...
Unknown unknown non-small cell lung carcinoma not applicable Regorafenib Phase I Actionable In a Phase I trial, Stivarga (regorafenib) treatment in patients with non-small cell lung cancer resulted in stable disease (SD) in 76% (13/17) of patients, and one patient with SD experienced a progression free survival of 279 days and tumor reduction greater than 30% (J Clin Oncol 28:15s, 2010 (suppl; abstr 7585)). detail...
Unknown unknown alveolar soft part sarcoma not applicable Durvalumab + Tremelimumab Clinical Study Actionable In a clinical case study, a patient with alveolar soft part sarcoma had a 73% tumor reduction and a response lasting greater than 30 months when treated with Imfinzi (durvalumab) and Tremelimumab, and the tumor was shown retrospectively to have a mismatch repair defect signature, poor immune infiltration, and 2% tumoral PD-L1 positivity (PMID: 30018044; NCT02261220). 30018044
Unknown unknown chronic lymphocytic leukemia not applicable JSH-150 Preclinical - Cell culture Actionable In a preclinical study, JSH-150 induced cell-cycle arrest and inhibited proliferation of chronic lymphocytic leukemia cell lines in culture (PMID: 30253346). 30253346
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 chronic lymphocytic leukemia not applicable Cyclophosphamide + Fludarabine + Ofatumumab FDA approved Actionable In a Phase III trial supporting FDA approval, Arzerra (ofatumumab) in combination with fludarabine and cyclophosphamide (FC) resulted in improved progression free survival (28.9 vs 18.8 months) compared to FC treatment alone in patients with relapsed chronic lymphocytic leukemia (PMID: 27731748). 27731748
Unknown unknown lung carcinoma predicted - sensitive 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 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 Advanced Solid Tumor not applicable Cabozantinib Phase II Actionable In a Phase II clinical trial, Cometriq (cabozantinib) showed safety and anti-tumor activity in several advanced solid tumor types (J Clin Oncol 29: 2011 (suppl; abstr 3010)). detail...
Unknown unknown stomach cancer not applicable HD105 Preclinical - Cell line xenograft Actionable In a preclinical study, HD105 inhibited tumor progression in two of four human gastric cancer cell line xenograft models (PMID: 27049350). 27049350
Unknown unknown non-Hodgkin lymphoma not applicable Duvelisib Phase I Actionable In a Phase I trial, Copiktra (duvelisib) treatment resulted in complete response in 16% (5/31), partial response in 45% (14/31), minor response in 3% (1/31), and stable disease in 29% (9/31) in non-Hodgkin lymphoma patients (Blood 124(21): 802). detail...
Unknown unknown Advanced Solid Tumor not applicable BAL101553 Phase I Actionable In a Phase I trial, BAL101553 treatment resulted in stable disease in 37% (7/19) of patients with advanced solid tumors (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 2530-2530; NCT02490800). detail...
Unknown unknown Advanced Solid Tumor not applicable SU14813 Phase I Actionable In a Phase I trial, SU14813 demonstrated safety and preliminary efficacy in patients with advanced solid tumors, with an objective response rate of 20% (13/65), including 1 complete response and 12 partial responses (PMID: 20605934). 20605934
Unknown unknown gastroesophageal junction adenocarcinoma not applicable MCLA-128 Phase Ib/II Actionable In a Phase I/II trial, MCLA-128 resulted in stable disease for 5 months in a patient with gastroesophageal junction cancer (Cancer Res 2016;76(14 Suppl):Abstract nr CT050). detail...
Unknown unknown non-Hodgkin lymphoma not applicable CUDC-907 Preclinical - Cell line xenograft Actionable In a preclinical study, CUDC-907 inhibited cell growth in a human non-Hodgkin lymphoma cell line in culture, and stabilized tumor growth in xenograft models (PMID: 22693356). 22693356
Unknown unknown Advanced Solid Tumor not applicable 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 oral squamous cell carcinoma not applicable Everolimus Preclinical Actionable In a preclinical study, Afinitor (everolimus) inhibited growth and mTOR signaling in oral squamous cell carcinoma cell lines (PMID: 25682238). 25682238
Unknown unknown ovarian cancer not applicable Selinexor Phase I Actionable In a Phase I clinical trial, Selinexor (KPT-330) treatment resulted in inhibition of tumor growth in 3/5 patients with platinum-refractory ovarian cancer, with one patient achieving a partial response and two patients achieving stable disease (PMID: 27649553). 27649553
Unknown unknown synovial sarcoma not applicable SU6656 Preclinical - Cell line xenograft Actionable In a preclinical study, SU6656 treatment decreased cell proliferation of synovial sarcoma cells in culture and inhibited tumor growth and blocked tumor invasion in cell line xenograft models (PMID: 22244830). 22244830
Unknown unknown mantle cell lymphoma not applicable ONC201 Preclinical - Patient cell culture Actionable In a preclinical study, ONC201 induced apoptosis and decreased viability of mantle cell lymphoma (MCL) cell lines in culture and demonstrated cytotoxicity in primary MCL samples in culture (PMID: 26884599). 26884599 detail...
Unknown unknown endometrial clear cell adenocarcinoma not applicable ONC201 Clinical Study Actionable In a clinical case study, a patient with clear cell endometrial cancer treated with ONC201 (TIC-10) demonstrated some reduction in lesion size (>30%), but also developed new lesions (PMID: 28331050). 28331050
Unknown unknown triple-receptor negative breast cancer not applicable PR-104 Preclinical Actionable In a preclinical study, PR-104 inhibited growth of triple-receptor negative breast cancer cell lines under hypoxic culture conditions, regardless of their BRCA1 status (PMID: 25193512). 25193512
Unknown unknown Advanced Solid Tumor not applicable PD-0325901 Phase I Actionable In a Phase I clinical trial, PD-0325901 demonstrated preliminary clinical activity in patients with advanced solid tumors, however late-onset dose-limiting toxicities were encountered (PMID: 20215549). 20215549
Unknown unknown Advanced Solid Tumor not applicable CLR457 Preclinical - Pdx Actionable In a preclinical study, CLR457 treatment of a variety of solid tumor patient-derived xenograft models decreased tumor volume and produced a 54% response (PMID: 26479923). 26479923
Unknown unknown ovarian cancer not applicable Cisplatin + ETP-46464 Preclinical - Cell culture Actionable In a preclinical study, ETP-46464 increased the sensitivity of ovarian cancer cell lines to Platinol (cisplatin) in culture (PMID: 25560806). 25560806
Unknown unknown acute promyelocytic leukemia not applicable Carfilzomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of an acute promyelocytic leukemia cell line in culture (PMID: 28753594). 28753594
Unknown unknown renal carcinoma not applicable Cabozantinib Phase II Actionable In a Phase II trial, renal cancer patients treated with Cabozantinib demonstrated a 28 % objective response rate, a 62 % stable disease rate, and a median progression free survival of 14.7 months (PMID: 23292795). 23292795
Unknown unknown malignant pleural mesothelioma not applicable GDC-0980 Phase I Actionable In a Phase I trial, Apitolisib (GDC-0980) treatment resulted in a partial response in 7.4% (2/27) and stable disease in 74.1% (20/27) of malignant pleural mesothelioma patients, including one with PTEN loss and another with PIK3CA E545K (PMID: 26787751). 26787751
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 colon carcinoma not applicable UD-017 Preclinical - Cell line xenograft Actionable In a preclinical study, UD-017 inhibited tumor growth in cell line xenograft models of colon carcinoma (J Clin Oncol 35, 2017 (suppl; abstr e14085)). detail...
Unknown unknown 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 non-small cell lung 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 melanoma not applicable Cobimetinib Preclinical - Cell line xenograft Actionable In a preclinical study, Cobimetinib (GDC-0973) induced cell death in several human melanoma cell lines in culture and inhibited tumor growth in xenograft models (PMID: 22084396). 22084396
Unknown unknown 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 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 Indication other than cancer not applicable Fingolimod FDA approved Actionable Gilenya (fingolimod) is FDA approved for use in patients with relapsing forms of multiple sclerosis (FDA.gov). detail...
Unknown unknown mantle cell lymphoma not applicable ONC201 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of ONC201and Venclexta (venetoclax) demonstrated synergy in a mantle cell lymphoma cell line in culture, resulting in increased induction of apoptosis (PMID: 26884599). 26884599
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 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 multiple myeloma not applicable Dinaciclib + Doxorubicin Preclinical - Cell culture Actionable In a preclinical study, the combination of Dinaciclib (SCH 727965) and Adriamycin (doxorubicin) demonstrated synergy in multiple myeloma cell lines in culture, resulting in decreased cell viability (PMID: 26719576). 26719576
Unknown unknown melanoma not applicable MIW815 + Spartalizumab Phase Ib/II Actionable In a Phase Ib trial, MIW815 (ADUS100), in combination with Spartalizumab (PDR001), demonstrated preliminary efficacy in PD-1 (PDCD1)-relapsed/refractory melanoma (J of Clin Oncol 37, 2019 (suppl; abstr 2507); NCT03172936). detail...
Unknown unknown triple-receptor negative breast cancer not applicable BAY1217389 + Paclitaxel Preclinical Actionable In a preclinical study, BAY1217389, in combination with Taxol (paclitaxel), had increased efficacy in inhibiting tumor growth in xenograft models of triple-negative breast cancer, compared to Taxol (paclitaxel) alone (PMID: 26832791). detail... 26832791
Unknown unknown 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 oral cavity cancer not applicable Duvelisib + unspecified PD-L1 antibody Preclinical Actionable In a preclinical study, treatment with the combination of low-dose Copiktra (duvelisib) and a PD-L1 antibody resulted in increased infiltration of CD8-positive tumor-infiltrating lymphocytes, decreased tumor volume, and improved survival in mice bearing T-cell inflamed murine oral cancer cell-derived tumors, when compared to either agent alone (PMID: 28364000). 28364000
Unknown unknown colorectal cancer not applicable Pegilodecakin Phase I Actionable In a Phase I trial, AM0010 treatment in pancreatic cancer patients resulted in stable disease in 27% (4/15) of patients, one patient with progression free survival for greater than 6 months, and a median overall survival of 15.4 months (J Clin Oncol 34, 2016 (suppl; abstr 3082)). detail...
Unknown unknown acute myeloid leukemia not applicable INCB053914 Preclinical Actionable In a preclinical study, acute myeloid leukemia cells were sensitive to INCB053914, resulting in inhibition of cell proliferation (Cancer Res August 1, 2015 75; 5416). detail...
Unknown unknown colorectal cancer not applicable Bevacizumab + Fluorouracil + Irinotecan + Oxaliplatin Clinical Study Actionable In a systematic review, a pooled analysis of 11 studies assessing the combination of Avastin (bevacizumab), Adrucil (fluorouracil), Eloxatin (oxaliplatin), and Camptosar (irinotecan) in colorectal cancer patients demonstrated an objective response rate of 69%, a median overall survival of 30.2 months based on six trials, and a progression free survival of 12.4 months based on nine trials (PMID: 28542671). 28542671
Unknown unknown colorectal cancer not applicable Abemaciclib Phase I Actionable In a Phase I trial, treatment with Abemaciclib (LY2835219) in colorectal cancer patients resulted in 2 patients (13%; 2/15) with stable disease (PMID: 27217383). 27217383
Unknown unknown non-small cell lung 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 myelodysplastic syndrome 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 3-14 cycles of treatment in 50% (3/6) of myelodysplastic syndrome patients, including 1 patient achieved complete marrow response (Blood 128 (22):1640). 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 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 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 ovarian cancer not applicable MLN4924 Preclinical Actionable In a preclinical study, MLN4924 demonstrated moderate efficacy in ovarian cancer cell lines and enhanced synergistic effects in combination with cisplatin or carboplatin (PMID: 23939375). 23939375
Unknown unknown breast cancer not applicable Thymoquinone Preclinical - Cell line xenograft Actionable In a preclinical study, Thymoquinone (TQ) inhibited growth, migration, and invasive behavior of human breast cancer cell lines in culture, and inhibited tumor growth and metastasis in mouse breast cancer cell line xenografts (PMID: 26023736). 26023736
Unknown unknown fibrosarcoma not applicable 23814 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with 23814 resulted in partial inhibition of tumor growth in a fibrosarcoma cell line xenograft model (PMID: 25995436). 25995436
Unknown unknown multiple myeloma not applicable GSK2857916 Phase I Actionable In a Phase I trial, GSK2857916 demonstrated manageable safety and preliminary activity in patients with multiple myeloma, with 60% (21/35) of patients in part 2 receiving the recommended phase 2 dose achieving a response (1 stringent complete response (CR), 1 CR, 2 very good partial responses (PR), and 3 PR) (PMID: 30442502; NCT02064387). 30442502
Unknown unknown thyroid cancer not applicable ABT-737 + Doxorubicin Preclinical Actionable In a preclinical study, the combination of ABT-737 and Adriamycin (doxorubicin) was synergistic towards inhibiting cell viability of a majority of human thyroid cancer cell lines tested in culture (PMID: 27042160). 27042160
Unknown unknown lung carcinoma not applicable MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human lung carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown colorectal cancer not applicable AB61 Preclinical - Cell line xenograft Actionable In a preclinical study, the synthetic nucleoside AB61 demonstrated cytotoxicity in colorectal cancer cell lines in culture and decreased tumor volume and prolonged survival in colorectal cancer cell line xenograft models (PMID: 26819331). 26819331
Unknown unknown astrocytoma no benefit Veliparib Preclinical - Cell culture Actionable In a preclinical study, Veliparib (ABT-888) had a very limited effect on the cell viability of multiple cultured pediatric high grade astrocytoma cell lines (PMID: 26351319). 26351319
Unknown unknown neuroblastoma not applicable Cyclophosphamide + Topotecan Preclinical - Cell line xenograft Actionable In a preclinical study, Topotecan and Cytoxan (cyclophosphamide) combination treatment resulted in mild tumor growth delay in a neuroblastoma cell line xenograft model with wild-type ALK and TP53 H168R, and a model with wild-type ALK, wild-type TP53, and MDM2 amplification (PMID: 26438783). 26438783
Unknown unknown prostate cancer not applicable AGS-1C4D4 Phase I Actionable In a Phase I trial, AGS-1C4D4 treatment in castration resistant prostate cancer patients was well-tolerated and resulted in stable disease at 24 weeks in 46% (6/13) of patients, but did not reduce PSA (PMID: 22020316). 22020316
Unknown unknown ovarian cancer not applicable MBG453 + Spartalizumab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in stable disease in 3 patients with ovarian cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown 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 acute myeloid leukemia not applicable SNS-229 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with SNS-229 resulted in decreased PDPK1 pathway signaling and tumor growth inhibition in an acute myeloid leukemia cell line xenograft model (Mol Cancer Ther, Dec 1 2015 (14) (12 Supplement 2) C198). detail...
Unknown unknown ovarian clear cell adenocarcinoma not applicable DS-7423 Preclinical - Cell line xenograft Actionable In a preclinical study, DS-7423 inhibited proliferation of ovarian clear cell adenocarcinoma cell lines in culture and suppressed tumor growth in cell line xenograft animal models (PMID: 24504419). 24504419
Unknown unknown triple-receptor negative breast cancer not applicable AMG 900 Preclinical - Cell line xenograft Actionable In a preclinical study, AMG 900 induced apoptosis in triple-negative breast cancer (TNBC) cell lines in culture and inhibited tumor growth in TNBC cell line xenograft models (PMID: 23990115). 23990115
Unknown unknown glioblastoma multiforme not applicable Abemaciclib Phase I Actionable In a Phase I trial, three patients with glioblastoma demonstrated stable disease when treated with Abemaciclib (LY2835219) (PMID: 27217383). 27217383
Unknown unknown Advanced Solid Tumor not applicable Glesatinib Phase I Actionable In a Phase I trial, Glesatinib (MGCD265) displayed safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 27, 2009 (suppl; abstr e14525)). detail...
Unknown unknown Advanced Solid Tumor not applicable CDX-1140 + CDX-301 Phase I Emerging In a Phase I trial, CDX-1140 and CDX-301 combination treatment demonstrated safety and expected lymphocyte activation and cytokine responses in patients with advanced solid tumors (AACR Annual Meeting 2019, Abstract LB_194; NCT03329950). detail...
Unknown unknown leukemia not applicable MC180295 Preclinical - Cell culture Actionable In a preclinical study, MC180295 decreased proliferation and increased differentiation of leukemia cells in culture (PMID: 30454645). 30454645
Unknown unknown acute lymphocytic leukemia not applicable Prexasertib Preclinical - Cell culture Actionable In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including acute lymphoblastic leukemia cell lines (PMID: 28270495). 28270495
Unknown unknown pancreatic cancer not applicable G-TPP + Obatoclax Preclinical - Cell culture Actionable In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Obatoclax (GX015-070) synergistically inhibited viability of pancreatic cancer cells in culture (PMID: 28522750). 28522750
Unknown unknown acute myeloid leukemia not applicable INCB054329 + INCB059872 Preclinical - Cell line xenograft Actionable In a preclinical study, combination of INCB054329 and INCB059872 resulted in enhanced apoptosis in acute myeloid leukemia cells in culture and in cell-line xenograft models (Cancer Res 2016;76(14 Suppl):Abstract nr 4702). detail...
Unknown unknown ovarian cancer not applicable MBG453 Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 treatment resulted in stable disease in 2 patients with ovarian cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
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 chronic lymphocytic leukemia not applicable Carfilzomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, the combination of EDO-S101 and Kyprolis (carfilzomib) decreased viability of primary chronic lymphocytic leukemia cells in culture (PMID: 28753594). 28753594
Unknown unknown esophagus squamous cell carcinoma not applicable Camrelizumab Phase I Actionable In a Phase I trial, Camrelizumab (SHR-1210) treatment resulted in an objective response rate of 33.3% (10/30, 1 complete response, 9 partial response) and a disease control rate of 56.7%, with a median progression free survival of 3.6 months in patients with advanced esophageal squamous cell carcinoma (PMID: 29358502; NCT02742935). 29358502
Unknown unknown multiple myeloma not applicable Dexamethasone + Lenalidomide + Ricolinostat Phase I Actionable In a Phase Ib trial, Ricolinostat (ACY-1215) in combination with Revlimid (lenalidomide) and Desamethasone demonstrated safety and preliminary efficacy in relapsed or refractory multiple myeloma patients, resulted in an overall response rate of 55% (21/38) (PMID: 27646843). 27646843
Unknown unknown non-small cell lung 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 ovary epithelial cancer not applicable Ramucirumab Phase II Actionable In a Phase II trial, Cyramza (ramucirumab) resulted in antitumor activity in patients with epithelial ovarian cancer (PMID: 25016924). 25016924
Unknown unknown stomach cancer no benefit Everolimus Phase III Actionable In a Phase III trial, Afinitor (everolimus) did not significantly improve overall survival (5.4 vs 4.3 months) and progression free survival (1.7 vs 1.4 months) over placebo in previously treated advanced gastric cancer patients (PMID: 24043745). 24043745
Unknown unknown melanoma not applicable Nivolumab + NKTR-214 Phase Ib/II Actionable In a Phase I/II trial, NKTR-214 and Opdivo (nivolumab) combination treatment demonstrated safety and preliminary efficacy, resulted in radiographic response in one and complete response in another patient with melanoma (Journal of Clinical Oncology 35, no. 15_suppl; NCT02983045). detail...
Unknown unknown malignant mesothelioma not applicable Avelumab Phase I Actionable In a Phase I trial (JAVELIN), Bavencio (avelumab) resulted in an objective response rate (ORR) of 9% (5/53, 1 complete response, 4 partial response) and a disease control rate of 58% (31/53) in patients with advanced unresectable mesothelioma, with a median progression-free survival of 4.1 months and a median overall survival of 10.7 months; ORR were 19% (3/16) and 7% (2/27) in patients with CD274 (PD-L1)-positive (5% or greater) or negative tumors (p=0.34) respectively (PMID: 30605211; NCT01772004). 30605211
Unknown unknown chronic lymphocytic leukemia not applicable PRT318 Preclinical Actionable In a preclinical study, treatment with PRT318 resulted in increased apoptosis and decreased migration of primary chronic lymphocytic leukemia cells in culture (PMID: 22362000). 22362000
Unknown unknown clear cell renal cell carcinoma not applicable Epacadostat + Pembrolizumab Phase Ib/II Actionable In a Phase I/II trial, Epacadostat (INCB024360) and Keytruda (pembrolizumab) combination treatment resulted in complete response in 5% (1/19), partial response in 42% (8/19), and stable disease in 10% (2/19) of patients with advanced clear cell renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 4515)). detail...
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 acute myeloid leukemia not applicable ONC201 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of ONC201 and Venclexta (venetoclax) demonstrated synergy in acute myeloid leukemia cell lines in culture, resulting in increased induction of apoptosis (PMID: 26884599). 26884599
Unknown unknown follicular lymphoma not applicable Bendamustine + Veliparib Phase I Actionable In a Phase I trial, a patient with follicular lymphoma treated with a combination of Veliparib (ABT-888) and Bendamustine demonstrated a complete response (PMID: 28314788; NCT01326702). 28314788
Unknown unknown kidney cancer no benefit CVX-241 Preclinical Actionable In a preclinical study, CVX-241 did not improve overall survival in a mouse model of resected renal cancer (PMID: 27651308). 27651308
Unknown unknown melanoma not applicable Pembrolizumab FDA approved Actionable In a Phase II trial (KEYNOTE-002) that supported FDA approval, treatment with Keytruda (pembrolizumab) resulted in improved progression-free survival (PFS) compared to chemotherapy in patients with Yervoy (ipilimumab)-refractory melanoma, with a HR of 0.57 (p<0.0001) in the 2mg/kg group and a HR of 0.50 (p<0.0001) in the 10mg/kg group (PMID: 26115796; NCT01704287). 26115796
Unknown unknown melanoma not applicable Pembrolizumab FDA approved Actionable In a Phase III trial (KEYNOTE-006) that supported FDA approval, treatment with Keytruda (pembrolizumab) resulted in improved progression-free survival (PFS) and overall survival (OS) in patients with metastatic melanoma, with a 6-month PFS rate of 47.3% for the every 2 week schedule and 46.4% for the every 3 week schedule versus 26.5% with Yervoy (ipilimumab) (PMID: 25891173; NCT01866319). 25891173
Unknown unknown melanoma not applicable Pembrolizumab FDA approved Actionable In a Phase III trial (KEYNOTE-054) that supported FDA approval, Keytruda (pembrolizumab) treatment resulted in significantly improved recurrence-free survival rate at 1 year (75.4% vs 61.0%, HR=0.57, p<0.001) compared to placebo in patients with resected, high-risk stage III melanoma (PMID: 29658430; NCT02362594). 29658430
Unknown unknown glioblastoma multiforme not applicable Bevacizumab FDA approved Actionable In a Phase II trial that supported FDA approval, treatment with Avastin (bevacizumab) resulted in an objective response rate of 25.9% (22/85) with a mean duration of response of 4.2 months in patients with glioblastoma (PMID: 19897538). 19897538
Unknown unknown renal cell carcinoma not applicable GW5074 + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of GW5074 and Nexavar (sorafenib) worked synergistically to induce cell death in renal cell carcinoma cells in culture and inhibited tumor growth in xenograft and spontaneous mouse models of renal cell carcinoma (PMID: 26100670). 26100670
Unknown unknown colon cancer not applicable Gemcitabine + SRA737 Preclinical - Cell line xenograft Actionable In a preclinical study, Gemzar (gemcitabine) resulted in enhanced antitumor efficacy when combined with SRA737 (CCT245737) in a colon cancer cell line xenograft model (PMID: 27167172). 27167172
Unknown unknown glioblastoma multiforme not applicable A-966492 + Radiotherapy + Topotecan Preclinical - Cell culture Actionable In a preclinical study, the combination treatment of A-966492 and Hycamtin (topotecan) resulted in enhanced radiosensitivity in glioblastoma cells in culture, demonstrating a greater reduction in cell survival (PMID: 28797568). 28797568
Unknown unknown breast cancer not applicable Docetaxel + MEDI5117 Preclinical Actionable In a preclinical study, MEDI5117 in combination with Taxotere (docetaxel) resulted in complete tumor regression of human breast cancer cells in an orthotopic model (PMID: 26744529). 26744529
Unknown unknown lymphoma not applicable S63845 Preclinical - Cell culture Actionable In a preclinical study, S63845 inhibited survival of several lymphoma cell lines in culture (PMID: 27760111). 27760111
Unknown unknown breast cancer not applicable Everolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Afinitor (everolimus) inhibited the growth of a breast cancer cell line in culture and resulted in decreased tumor volume in a cell line xenograft model (PMID: 26351208). 26351208
Unknown unknown 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 ovarian carcinoma not applicable SKLB-23bb Preclinical - Cell line xenograft Actionable In a preclinical study, SKLB-23bb treatment resulted in antitumor efficacy in ovarian carcinoma cells, demonstrating decreased colony formation in vitro and inhibition of tumor growth in cell line xenograft models (PMID: 29610282). 29610282
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 prostate cancer no benefit Bevacizumab + Docetaxel Phase III Actionable In a Phase III trial, treatment with Avastin (bevacizumab) combined with Taxotere (docetaxel) in mCRPC patients did not result in an improved OS (22.6 mo vs 21.5 mo) and demonstrated increased toxicities when compared to Taxotere (docetaxel) plus placebo (PMID: 22454414). 22454414
Unknown unknown Advanced Solid Tumor not applicable BMS-986156 Phase Ib/II Actionable In a Phase I/IIa trial, patients with advanced solid tumors treated with a combination of Opdivo (nivolumab) and BMS-986156 demonstrated preliminary antitumor activity (Journal of Clinical Oncology 35, no. 15_suppl (May 2017) 104-104; NCT02598960). detail...
Unknown unknown non-Hodgkin lymphoma not applicable KW-2478 Phase I Actionable In a Phase I clinical trial, KW-2478 demonstrated safety and preliminary efficacy in patients with non-Hodgkin lymphoma, with 100% (4/4) of patients achieving stable disease (PMID: 26695442). 26695442
Unknown unknown mycosis fungoides not applicable Mogamulizumab FDA approved Actionable In a Phase III trial (MAVORIC) that supported FDA approval, Poteligeo (mogamulizumab-kpkc) treatment resulted in significantly improved progression-free survival (7.7 vs 3.1 months, HR=0.53, p<0.0001) and objective response rate (28.0% vs 4.8%) compared to Zolinza (vorinostat) in patients with mycosis fungoides or SĂ©zary syndrome (Blood 2017 130(Suppl 1):817). detail...
Unknown unknown breast cancer not applicable Elenagen + Tamoxifen Phase Ib/II Actionable In a Phase Ib/II trial, a chemorefractory patient with breast cancer demonstrated restored chemotherapeutic sensitivity upon sequential treatment of Elenagen and Nolvadex (tamoxifen), which resulted in stable disease (PMID: 28881846). 28881846
Unknown unknown pancreatic cancer not applicable Gemcitabine + Vantictumab Preclinical - Pdx Actionable In a preclinical study, Vantictumab (OMP-18R5) worked synergistically with Gemzar (gemcitabine) to inhibit tumor growth in patient-derived xenograft models of pancreatic cancer (PMID: 22753465). 22753465
Unknown unknown Hodgkin's lymphoma not applicable JNJ-40346527 Phase II Actionable In a Phase II/III clinical trial, JNJ-40346527 demonstrated safety some efficacy, with complete response in 5.0% (1/20), partial response in 5.0% (1/20), and stable disease in 55.0% (11/20) of patients with refractory Hodgkin's lymphoma (PMID: 25628399). 25628399
Unknown unknown Advanced Solid Tumor not applicable Ipafricept Phase I Actionable In a Phase I trial, Ipafricept (OMP-54F28) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 32:5s, 2014 (suppl; abstr 2505)). detail...
Unknown unknown breast cancer not applicable Alisertib + Eribulin Preclinical - Pdx Actionable In a preclinical study, patient derived xenograft (PDX) models of breast cancer treated with a combination of Alisertib (MLN8237) and Halaven (eribulin) demonstrated a 70-80% decrease in tumor volume compared to only a 40% decrease in models treated with Halaven (eribulin) alone (PMID: 27235164). 27235164
Unknown unknown mantle cell lymphoma not applicable Ibrutinib Phase II Actionable In a Phase II trial, Imbruvica (ibrutinib) treatment resulted in a response rate of 68% (75/111, complete response 21%, partial response 47%), with an estimated median progression-free survival of 13.9 months in patients with relapsed or refractory mantle-cell lymphoma (PMID: 23782157, NCT01236391). 23782157
Unknown unknown acute lymphocytic leukemia not applicable Blinatumomab FDA approved Actionable In a Phase III trial that supported FDA approval, Blincyto (blinatumomab) treatment resulted in longer overall survival (7.7 vs 4.0 months), higher remission rate (34% vs 16%), and higher rate of event-free survival (31% vs 12%) compared to chemotherapy in patients with relapsed or refractory B-cell precursor acute lymphocytic leukemia (PMID: 28249141; NCT02013167). 28249141
Unknown unknown ovarian serous carcinoma not applicable CFI-402257 Preclinical - Pdx Actionable In a preclinical study, CFI-402257 growth in patient-derived xenograft (PDX) models of platinum-sensitive and platinum-resistant high-grade serous ovarian cancer (PMID: 28270606). 28270606
Unknown unknown Advanced Solid Tumor not applicable 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 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 gastric adenocarcinoma not applicable Paclitaxel + TS-1 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 peripheral T-cell lymphoma not applicable Fenretinide Phase I Actionable In a Phase I trial, Fenretinide treatment resulted in complete response in 17% (2/11), partial response in 17% (2/11), and stable disease in 42% (5/11) of patients with peripheral T-cell lymphoma (PMID: 28420721). 28420721
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 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 Her2-receptor negative breast cancer not applicable Capivasertib + Paclitaxel Phase II Actionable In a Phase II trial, addition of AZD5363 to Taxol (paclitaxel) did not improve progression free survival compared to placebo (10.9 vs 8.4 months) in patients with Esr1-positive, Erbb2 (Her2)-negative breast cancer (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 241PD; NCT01625286). detail...
Unknown unknown non-small cell lung carcinoma not applicable Navitoclax + TAK-901 Preclinical - Cell culture Actionable In a preclinical study, the combination of TAK-901 and Navitoclax (ABT-263) resulted in a synergistic effect, demonstrating reduced cell viability of non-small cell lung cancer cells in culture (PMID: 28179288). 28179288
Unknown unknown prostate cancer not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) treatment of patients with neuroendocrine prostate cancer (NEPC) or castration-resistant prostate adenocarcinoma (CRPAC) resulted in a 6-month radiological progression-free survival (PFS) of 13.4% (8/60, 16.7% of NEPC, 5.3% of CRPAC), a median PFS of 2.2 months (2.3 months in NEPC, 2.0 months in CRPAC), and an overall survival of 9.5 months, and 30% (18/60) of patients had stable disease at the third treatment cycle (PMID: 30232224; NCT30232224). 30232224
Unknown unknown uterine cancer not applicable BGB-A317 + Pamiparib Phase Ib/II Actionable In a Phase Ib trial, the combination therapy of BGB-A317 and BGB-290 resulted in a partial response in a patient with uterine cancer (J Clin Oncol 35, 2017 (suppl; abstr 3013)). detail...
Unknown unknown rhabdoid cancer not applicable BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 100% (3/3) of cell line xenograft models of rhabdoid tumor (PMID: 21298745). 21298745
Unknown unknown 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 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 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 liposarcoma not applicable Selinexor Preclinical - Cell line xenograft Actionable In a preclinical study, Selinexor (KPT-330) decreased Birc5 (Survivin) expression and induced PARP and caspase-3 cleavage, reduced viability of liposarcoma cell lines in culture, and inhibited tumor growth in xenograft models (PMID: 28314790). 28314790
Unknown unknown triple-receptor negative breast cancer not applicable Capivasertib + Olaparib Phase I Actionable In a Phase I trial, the combination of AZD5363 and Lynparza (olaparib) was well-tolerated and demonstrated preliminary activity in patients with endometrial, ovarian, or triple-negative breast cancer (TNBC), with an overall response rate of 24% (7/30; all partial responses, 1 ovarian, 4 endometrial, and 2 TNBC) and stable disease for greater than 4 months in 6 additional patients (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #391P; NCT02208375). detail...
Unknown unknown marginal zone B-cell lymphoma not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in partial response in 20% (1/5) and stable disease in 80% (4/5) of patients with marginal zone B-cell lymphoma (PMID: 29475723; NCT01767766). 29475723
Unknown unknown Advanced Solid Tumor not applicable PLX9486 Phase I Actionable In a Phase I trial, PLX9486 demonstrated safety and preliminary efficacy, resulted in a progression-free survival of more than 24 weeks and partial response in 8.3% (2/24) of patients with advanced solid tumors, 20 of these patients had gastrointestinal stromal tumor that progressed on Gleevec (imatinib mesylate), and most harbored KIT exon 11 and exon 17 mutations (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 11509-11509; NCT02401815). detail...
Unknown unknown non-small cell lung 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 Paclitaxel + TVB-2640 Phase I Actionable In a Phase I trial, TVB-2640 and Taxol (paclitaxel) combination treatment resulted in 58-98% reduction of CA-125 level in 42% (5/12) of ovarian cancer patients (J Clin Oncol 34, 2016 (suppl; abstr 2512)). detail...
Unknown unknown triple-receptor negative breast cancer not applicable Eribulin Preclinical - Cell line xenograft Actionable In a preclinical study, Halaven (eribulin) inhibited epithelial-to-mesenchymal transition and increased tumor perfusion in triple-negative breast cancer cell line xenograft models (PMID: 25838395). 25838395
Unknown unknown prostate cancer no benefit Zibotentan Phase III Actionable In a Phase III trial, Zibotentan (ZD4054) did not demonstrate a survival benefit compared to placebo in patients with prostate cancer, and the trial was discontinued at the interim analysis (PMID: 23381694). 23381694
Unknown unknown multiple myeloma not applicable A-485 Preclinical - Cell culture Actionable In a preclinical study, A-485 treatment inhibited proliferation in multiple myeloma cell lines in culture (PMID: 28953875). 28953875
Unknown unknown Advanced Solid Tumor not applicable Afuresertib + Trametinib Phase I Actionable In a Phase I trial, the combination of Mekinist (trametinib) and Afuresertib (GSK2110183) was not well-tolerated in patients with advanced solid tumors (PMID: 25417902). 25417902
Unknown unknown non-small cell lung carcinoma not applicable Belinostat + Carboplatin + Paclitaxel Phase I Actionable In a Phase I trial, the combination therapy of Beleodaq (belinostat), Paraplatin (carboplatin, and Taxol (paclitaxel) in patients with non-small cell lung carcinoma resulted in a 5.7 month median progression-free survival, a partial response in 35% (8/23) patients, and stable disease in 17% (4/23) of patients (Journal of Thoracic Oncology, 2017, vol 12:1S, abstract #P2.03a-003). detail...
Unknown unknown stomach cancer not applicable Camrelizumab Phase I Actionable In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity, resulted in partial response in 37.5% (3/8) of patients with gastric cancer (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). detail...
Unknown unknown Advanced Solid Tumor not applicable Selinexor Phase I Actionable In a Phase I trial, Selinexor (KPT-330) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, resulting in an objective response rate of 4% (7/157; 1 complete response and 6 partial responses) and stable disease in 43% (67/157), with 17% (27/157) of patients demonstrating stable disease for at least 4 months (PMID: 26926685). 26926685
Unknown unknown non-small cell lung carcinoma not applicable Ficlatuzumab + Gefitinib Phase I Actionable In a Phase I trial, Ficlatuzumab (Av-299) in combination with Iressa (gefitinib) demonstrated safety and efficacy in patients with NSCLC (PMID: 23493885, J Clin Oncol. 2011;29(Suppl 15) Abstract 7571). detail... 23493885
Unknown unknown non-small cell lung carcinoma not applicable Pemetrexed + 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 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 prostate cancer not applicable Sapanisertib Phase II Actionable In a Phase II trial, Sapanisertib (MLN0128) treatment demonstrated limited efficacy, with a median time on treatment of 11 weeks and stable disease as best response (PMID: 29508246). 29508246
Unknown unknown prostate cancer not applicable Sapanisertib Preclinical Actionable In a preclinical study, Sapanisertib (MLN0128) inhibited cell proliferation, induced apoptosis, and prevented metastasis in xenograft models with prostate cancer (PMID: 22367541). 22367541
Unknown unknown mantle cell lymphoma not applicable ONO-4059 Phase I Actionable In a Phase I clinical trial, treatment with ONO-4059 was well tolerated and resulted in responses in 92% (11/12) of patients with mantle cell lymphoma (PMID: 26542378). 26542378
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 acute myeloid leukemia not applicable CPI-613 + Cytarabine + Mitoxantrone Phase I Actionable In Phase I trial, the combination therapy, CPI-613 with Cytosar-U and Novantrone (Mitoxantrone), resulted in an overall response rate of 50% (31/62) in acute myeloid leukemia patients and a median survival of 6.7 months, and in patients over 60 years of age, the combined therapy led to a 47% (15/32) complete response rate and median survival of 6.9 months (PMID: 29437791). 29437791
Unknown unknown chronic lymphocytic leukemia not applicable Bortezomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, the combination of EDO-S101 and Velcade (bortezomib) decreased viability of primary chronic lymphocytic leukemia cells in culture (PMID: 28753594). 28753594
Unknown unknown acute myeloid leukemia not applicable ABBV-075 + Venetoclax Preclinical - Cell line xenograft Actionable In a preclinical study, ABV-0775 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 hepatocellular carcinoma not applicable Chiauranib Preclinical - Cell line xenograft Actionable In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a hepatocellular carcinoma cell line xenograft model (PMID: 28004478). 28004478
Unknown unknown colon cancer sensitive AZ628 + CPI-455 Preclinical - Cell culture Actionable In a preclinical study, a colon cancer cell line treated with AZ628 demonstrated increased sensitivity when co-treated with CPI-455 in culture, resulting in decreased survival of cells, in particular the cells that eventually develop drug resistance (PMID: 27214401). 27214401
Unknown unknown Advanced Solid Tumor not applicable GDC-0917 Phase I Actionable In a Phase I clinical trial, GDC-0917 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 31, 2013 (suppl; abstr 2503)). detail...
Unknown unknown hematologic cancer not applicable CUDC-907 Phase I Actionable In a Phase I trial, CUDC-907 treatment was well-tolerated, demonstrated safety, and resulted in stable disease in 57% (21/37) of patients with a hematological cancer (PMID: 27049457). 27049457
Unknown unknown renal cell carcinoma not applicable Bevacizumab + Temsirolimus Phase II Actionable In a Phase II trial, patients with metastatic renal cell carcinoma (mRCC) treated with a combination of Avastin (bevacizumab) and Torisel (temsirolimus) demonstrated an overall 4 month PFS rate of 65% while those patients with VEGFR tyrosine kinase inhibitor refractory mRCC showed a median PFS and OS of 6.5 and 9.6 months, respectively (PMID: 27036973). 27036973
Unknown unknown transitional cell carcinoma not applicable Everolimus Phase II Actionable In a Phase II trial, treatment with Afinitor (everolimus) in patients with transitional cell carcinoma resulted in 2 patients with a partial response, 8 patients with stable disease, and 27 patients with progressive disease, and resulted in a median progression free survival of 61 days and a median overall survival of 101 days (PMID: 22473592). 22473592
Unknown unknown colon carcinoma not applicable Hydroxyurea + MU380 Preclinical - Cell culture Actionable In a preclinical study, the addition of MU380 resulted in increased sensitivity to Droxia (hydroxyurea) in colon carcinoma cell lines in culture, leading to decreased proliferation (PMID: 28619751). 28619751
Unknown unknown Advanced Solid Tumor no benefit AZD7762 + Gemcitabine Phase I Actionable In a Phase I trial, the CHEK2 inhibitor, AZD7762, in combination with Gemzar (gemcitabine), demonstrated some efficacy in two solid tumors patients, however the cardiac toxicity was unfavorable (PMID: 24448638). 24448638
Unknown unknown female reproductive organ cancer 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). 28453702
Unknown unknown glioblastoma multiforme not applicable Gliovac + Sargramostim Clinical Study Actionable In a clinical study, Gliovac (ERC 1671) adminstered with Leukine (sargramostim) as an adjuvant demonstrated low toxicity and resulted in an improved 40-week overall survival rate of 77% (n=9) vs. 10% (n=39) with historical controls in patients with recurrent glioblastoma multiforme (PMID: 25865468). 25865468
Unknown unknown lung cancer not applicable Nintedanib Preclinical - Cell line xenograft Actionable In a preclinical study, Nintedanib, alone or with chemotherapy, inhibited tumor growth in cell line xenograft models of lung and pancreatic cancer but not in cell culture (PMID: 23729403). 23729403
Unknown unknown melanoma not applicable G-TPP + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, combination of the mitochondrial Hsp90 inhibitor G-TPP and the Bcl2 inhibitor Venclexta (venetoclax) resulted in increased apoptosis of melanoma cell lines in culture (PMID: 28522750). 28522750
Unknown unknown Ewing sarcoma not applicable MEDI-573 + Vistusertib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of MEDI-573 and Vistusertib (AZD2014) inhibited proliferation of a Ewing sarcoma cell line in culture, and inhibited tumor growth in a Ewing sarcoma cell line xenograft model, with increased efficacy compared to either as a single agent (PMID: 25193511). 25193511
Unknown unknown thyroid cancer not applicable CLM3 Preclinical - Cell line xenograft Actionable In a preclinical study, CLM3 induced apoptosis and inhibited EGFR phosphorylation and cell proliferation in human anaplastic thyroid cancer cell lines in culture and inhibited tumor growth in xenograft models (PMID: 24423321). 24423321
Unknown unknown splenic marginal zone lymphoma not applicable ST7612AA1 Preclinical Actionable In a preclinical study, ST7612AA1 inhibited proliferation of splenic marginal zone lymphoma cell lines in culture (PMID: 25671299). 25671299
Unknown unknown 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 alveolar rhabdomyosarcoma not applicable BGJ398 Preclinical - Pdx Actionable In a preclinical study, BGJ398 resulted in antitumor activity in an alveolar rhabdomyosarcoma patient derived xenograft model (PMID: 24687871). 24687871
Unknown unknown prostate cancer not applicable Darolutamide Phase III Actionable In a Phase III trial, treatment with Darolutamide (ODM-201) resulted in improved median metastasis-free survival (40.4 vs 18.4 months, HR=0.41, p<0.001), overall survival (HR=0.71, 95% CI, 0.5-0.99, p=0.045), and time to pain progression (40.3 vs 25.4 months, HR=0.65, p<0.001) compared to placebo in non-metastatic castration-resistant prostate cancer patients (PMID: 30763142; NCT02200614). 30763142
Unknown unknown hepatocellular carcinoma not applicable YW3-56 Preclinical Actionable In a preclinical study, YW3-56 inhibited proliferation of a human hepatocellular carcinoma cell line in culture (PMID: 25612620). 25612620
Unknown unknown breast cancer not applicable EHop-016 Preclinical Actionable In a preclinical study, EHop-016 inhibited Rac activity and migration of breast cancer cells in culture, but did not have a significant effect on cell viability (PMID: 22383527) 22383527
Unknown unknown malignant fibroxanthoma not applicable EHop-016 + IPA-3 Preclinical - Cell culture Actionable In a preclinical study, treatment with the combination of EHop-016 and IPA-3 resulted in increased apoptosis and reduced growth of a myxofibrosarcoma cell line in culture compared to either agent alone (PMID: 27577794). 27577794
Unknown unknown non-small cell lung 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 retinoblastoma not applicable Prexasertib Preclinical - Cell culture Actionable In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including a retinoblastoma cell line (PMID: 28270495). 28270495
Unknown unknown chondrosarcoma not applicable Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 20% (1/5) of patients with chondrosarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). detail...
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 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 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 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 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 lymphoma not applicable Marizomib Phase I Actionable In a Phase I trial, Marizomib (NPI-0052) treatment resulted in complete response lasting more than 10 treatment cycles in 7.1% (1/14) of lymphoma patients (PMID: 27117181). 27117181
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
Unknown unknown multiple myeloma not applicable Daratumumab + Dexamethasone + Lenalidomide FDA approved Actionable In a Phase III trial that supported FDA approval, the combination of Darzalex (daratumumab), Adexone (dexamethasone), and Revlimid (lenalidomide) resulted in a greater 12 month PFS (83.2% vs 60.1%) and overall response (92.9%; 261/281 vs 76.4%; 211/276) compared to Adexone (dexamethasone) and Revlimid (lenalidomide) alone in multiple myeloma patients (PMID: 27705267). 27705267
Unknown unknown endometrial cancer not applicable GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 treatment was well-tolerated and resulted in some efficacy in endometrial cancer patients including stable disease in 27% (4/15) and one patient with a partial response (PMID: 26603258). 26603258
Unknown unknown acute myeloid leukemia not applicable ST1926-NP Preclinical - Cell line xenograft Actionable In a preclinical study, ST1926-NP treatment resulted in reduced cell proliferation in acute myeloid leukemia (AML) cells in culture, and decreased tumor burden and improved survival in AML cell line xenograft models (PMID: 28619754). 28619754
Unknown unknown colorectal cancer not applicable ABT-348 Preclinical - Cell culture Actionable In a preclinical study, ABT-348 reduced proliferation of colorectal cancer cell lines in culture (PMID: 22935731). 22935731
Unknown unknown pancreatic cancer not applicable Gemcitabine + PG545 Preclinical - Cell line xenograft Actionable In a preclinical study, human pancreatic cancer cells demonstrated increased apoptosis and tumor growth suppression both in culture and in cell line xenograft models when treated with a combination of PG545 and Gemzar (gemcitabine) (PMID: 25669977). 25669977
Unknown unknown colorectal cancer not applicable ETBX-011 Phase Ib/II Actionable In a Phase I/II trial, ETBX-011 treatment resulted in an overall survival of 11 months, and survival at 12 months follow-up in 48% of patients with metastatic colorectal cancer (Journal of Clinical Oncology 32, no. 15_suppl (May 2014) 3093-3093; NCT01147965.). detail...
Unknown unknown 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 Advanced Solid Tumor not applicable Ulixertinib Phase I Actionable In a Phase I trial, BVD-523 (Ulixertinib) displayed safety and preliminary efficacy in advanced solid tumor patients (J Clin Oncol 33, 2015 (suppl; abstr 2506)). detail...
Unknown unknown colon cancer not applicable MPT0E028 Preclinical - Cell line xenograft Actionable In a preclinical study, MPT0E028 decreased proliferation and increased apoptosis in a colon cancer cell line in culture and inhibited tumor growth in a colon cancer cell line xenograft model (PMID: 22928010). 22928010
Unknown unknown Advanced Solid Tumor not applicable Poloxin-2 Preclinical - Cell culture Actionable In a preclinical study, human cancer cells demonstrated sensitivity to treatment with Poloxin-2 in culture, resulting in cell-cycle arrest and apoptotic induction (PMID: 26279064). 26279064
Unknown unknown colon cancer not applicable MC180295 Preclinical - Cell line xenograft Actionable In a preclinical study, MC180295 decreased growth of colon cancer cell lines in culture, and reduced tumor growth rate and improved survival in colon cancer cell line xenograft models (PMID: 30454645). 30454645
Unknown unknown Advanced Solid Tumor not applicable Epacadostat Phase I Actionable In a Phase I trial, treatment with Epacadostat resulted in stable disease in 13% (7/52) of patients with advanced solid tumors for greater than or equal to 16 weeks, including two patients with melanoma who previously failed on immunotherapy (PMID: 28053021). 28053021
Unknown unknown lung small cell carcinoma not applicable STA-8666 Preclinical - Pdx Actionable In a preclinical study, small cell lung cancer cell line xenograft and patient derived xenograft (PDX) models demonstrated stabilization of tumor growth and eventual tumor regression when treated with STA-8666 (PMID: 27267850). 27267850
Unknown unknown Advanced Solid Tumor not applicable Buparlisib Phase I Actionable In a Phase I trial, Buparlisib (BKM120) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22162589). 22162589
Unknown unknown B-cell lymphoma not applicable IT-901 Preclinical Actionable In a preclinical study, IT-901 inhibited tumor growth in Epstein Barr Virus-induced B-cell lymphoma xenograft models (PMID: 26744524). 26744524
Unknown unknown 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 non-small cell lung 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 non-small cell lung carcinoma not applicable Afatinib + Nimotuzumab Phase Ib/II Actionable In a Phase I/II trial, Gilotrif (afatinib) and Nimotuzumab combination treatment resulted in a median progression-free survival of 4.0 months, an overall survival of 11.7 months, and overall response rate of 23% (10/44) in non-small cell lung cancer patients with acquired resistance to Iressa (gefitinib) or Tarceva (erlotinib) (PMID: 26667485). 26667485
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 hematologic cancer not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) demonstrated safety in patients with advanced hematological malignancies and preliminary efficacy in patients with chronic lymphocytic leukemia and non-Hodgkin's lymphoma (J Clin Oncol (Meeting Abstracts) 2015 33: 7069). detail...
Unknown unknown hematologic cancer not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in disease burden reduction in 73% (53/73), complete response in 4% (3/73), and partial response in 41% (30/73) of patients with chronic lymphocytic leukemia and lymphoma (PMID: 29475723; NCT01767766). 29475723
Unknown unknown uterine corpus sarcoma not applicable Pazopanib Clinical Study Actionable In a retrospective clinical study, Votrient (pazopanib) treatment resulted in objective response in 29% (10/35) and stable disease in 31% (11/35) of patients with uterine sarcoma, with median progression-free survival of 5.8 months and overall survival of 20.0 months (PMID: 29185261). 29185261
Unknown unknown non-small cell lung 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 leukemia not applicable DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, DCBCI0901 inhibited tumor growth greater than 65% in a leukemia cell line xenograft model (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown B-cell adult acute lymphocytic leukemia not applicable Disarib + Paclitaxel Preclinical - Cell culture Actionable In a preclinical study, Taxol (paclitaxel) efficacy was enhanced when combined with Disarib, resulting in greater cell death compared to either agent alone in B-cell acute lymphocytic leukemia cells in culture (PMID: 27693384). 27693384
Unknown unknown pancreatic cancer not applicable Chidamide Preclinical - Cell line xenograft Actionable In a preclinical study, Chidamide (CS055) inhibited growth of a human pancreatic cancer cell line in culture and inhibited tumor growth in xenograft models (PMID: 25384499). 25384499
Unknown unknown lymphoma not applicable Bortezomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, EDO-S101 and Velcade (bortezomib) worked synergistically to decrease viability of lymphoma cell lines in culture (PMID: 28753594). 28753594
Unknown unknown colon cancer not applicable Bevacizumab + CVX-060 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of CVX-060 and Avastin (bevacizumab) resulted in increased tumor growth inhibition compared to either agent alone in a colon cancer cell line xenograft model (PMID: 21233403). 21233403
Unknown unknown chronic lymphocytic leukemia not applicable Ofatumumab FDA approved Actionable In a Phase III trial supporting FDA approval, Arzerra (ofatumumab) maintenance treatment resulted in improved progression free survival (29.4 vs 15.2 months) compared to observation group in chronic lymphocytic leukemia patients in complete or partial remission after second-line or third-line treatment (PMID: 20194866, PMID: 26377300). 26377300 20194866
Unknown unknown Advanced Solid Tumor not applicable GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol (Meeting Abstracts) 2011 29: 3018). detail...
Unknown unknown colorectal cancer not applicable Bleomycin + CBP501 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of Blenoxane (bleomycin) and CBP501 resulted in enhanced tumor growth inhibition in cell line xenograft models of colorectal cancer, compared to either agent alone (PMID: 17237275). 17237275
Unknown unknown gastrointestinal stromal tumor not applicable JSH-150 Preclinical - Cell culture Actionable In a preclinical study, JSH-150 inhibited proliferation of a gastrointestinal stromal tumor cell line in culture (PMID: 30253346). 30253346
Unknown unknown 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 angiosarcoma not applicable Carotuximab + Pazopanib Phase Ib/II Actionable In a Phase I/II trial, TRC105 and Votrient (pazopanib) combination therapy resulted complete response in 40% (2/5) of angiosarcoma patients (J Clin Oncol 34, 2016 (suppl; abstr 11016)). detail...
Unknown unknown 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 non-small cell lung carcinoma not applicable Abemaciclib + Pemetrexed 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 renal cell carcinoma not applicable Famitinib Phase I Actionable In a Phase I trial, renal carcinoma patients treated with Famitinib demonstrated a disease control rate of 87.5%, which included 50% (12/24) with a partial response and 37.5% (9/24) with stable disease, and a PFS of 10.7 mo and an OS of 33 mo (PMID: 24238512). 24238512
Unknown unknown breast cancer not applicable GSK1904529A Preclinical - Cell culture Actionable In a preclinical study, GSK1904529A treatment resulted in decreased cell viability of breast cancer cells in culture (PMID: 19383820). 19383820
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 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 Advanced Solid Tumor not applicable GW5074 + Sorafenib Preclinical Actionable In a preclinical study, the combination of GW5074 and Nexavar (sorafenib) induced cell death in several tumor cell lines, and phosphorylation of DAPK at amino acid S308 correlated positively with response to therapy (PMID: 26100670). 26100670
Unknown unknown colon adenocarcinoma not applicable CVX-241 Preclinical - Cell line xenograft Actionable In a preclinical study, CVX-241 decreased Ang2 and phosphorylated and total Vegfr2 levels and inhibited tumor growth in a colon adenocarcinoma cell line xenograft model (PMID: 21149738). 21149738
Unknown unknown triple-receptor negative breast cancer not applicable MIW815 + Spartalizumab Phase Ib/II Actionable In a Phase Ib trial, MIW815 (ADUS100), in combination with Spartalizumab (PDR001), demonstrated preliminary efficacy in PD-1 (PDCD1)-naive TNBC patients (J of Clin Oncol 37, 2019 (suppl; abstr 2507); NCT03172936). detail...
Unknown unknown colorectal cancer not applicable Aflibercept FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Zaltrap (aflibercept) improved median progression free survival to 6.9 months and overall survival to 13.5 months in patients with metastatic colorectal cancer (PMID: 23444216). detail... 23444216
Unknown unknown gastrointestinal stromal tumor not applicable Dasatinib Phase 0 Actionable In a clinical trial, Sprycel (dasatinib) treatment resulted in a 6-month progression-free survival (PFS) rate of 29% (n=48), median PFS of 2.9 months, median overall survival of 19 months, and partial response in 25% (12/48) of patients with Gleevec (imatinib)-resistant gastrointestinal stromal tumor (PMID: 29710216). 29710216
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 cervical cancer not applicable Tisotumab Vedotin Phase II Actionable In a Phase II trial, Tisotumab vedotin treatment resulted in partial response in a patient with cervical cancer (Journal of Clinical Oncology 33, no. 15_suppl (May 20 2015) 2570-2570; NCT02001623). detail...
Unknown unknown thyroid cancer not applicable Bevacizumab Preclinical Actionable In a preclinical study, Avastin (bevacizumab) inhibited tumor growth and angiogenesis in mouse models of anaplastic thyroid cancer (PMID: 17429874). 17429874
Unknown unknown neuroblastoma not applicable Doxorubicin + GANT61 Preclinical Actionable In a preclinical study, GANT61 and Adriamycin (doxorubicin) worked synergistically to inhibit growth of neuroblastoma cells in culture (PMID: 22949014). 22949014
Unknown unknown multiple myeloma not applicable KW-2478 Phase I Actionable In a Phase I clinical trial, KW-2478 demonstrated safety and preliminary efficacy in patients with multiple myeloma, with 95.2% (20/21) of patients achieving stable disease (PMID: 26695442). 26695442
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 Ewing sarcoma 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 Ewing sarcoma (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 10542-10542; NCT02909777). 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 Advanced Solid Tumor not applicable ETC-159 Phase I Actionable In a Phase I trial, treatment with ETC-159 was well-tolerated, decreased Wnt signaling, and resulted in stable disease in 2 out of 16 treated patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2584)). detail...
Unknown unknown glioblastoma multiforme not applicable LYS6KAKT1 Preclinical Actionable In a preclinical study, LYS6KAKT1 inhibited phosphorylation of p70S6 kinase in mice engrafted with glioblastoma cells (Mol Cancer Ther 2011;10(11 Suppl):Abstract nr B117). detail...
Unknown unknown prostate cancer not applicable X480 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with X480 in prostate cancer cell lines resulted in reduced cell proliferation and induced apoptotic activity in culture, and decreased tumor load and metastasis in cell line xenograft models (Eur J Cancer, 2012, 48, S5:235). detail...
Unknown unknown 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 melanoma not applicable AT13148 Preclinical Actionable In a preclinical study, AT13148 inhibited human melanoma cell motility in invasion assays in culture (PMID: 25840982). 25840982
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 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 marginal zone B-cell lymphoma not applicable LAM-002A Phase I Actionable In a Phase I trial, LAM-002A demonstrated safety and preliminary efficacy, resulted in prolonged stable disease in 1 of 3 patients with marginal zone lymphoma (Blood 2017 130 (Suppl 1):4119). detail...
Unknown unknown renal cell carcinoma not applicable Axitinib + Dalantercept Phase II Actionable In a Phase II trial, the combination of Dalantercept (ACE-041) and Inlyta (axitinib) resulted in an objective response rate of 25% (7/28) and a median PFS of 8.3 months in renal cell carcinoma patients (PMID: 28031424). 28031424
Unknown unknown acute myeloid leukemia not applicable ABBV-075 + Azacitidine Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Vidaza (azacitidine) and ABBV-075 resulted in increased tumor growth inhibition in an acute myeloid leukemia cell line xenograft model compared to either agent alone (PMID: 28416490). 28416490
Unknown unknown prostate cancer not applicable AZD8186 + Vistusertib Phase I Actionable In a Phase I trial, the combination of AZD8186 and Vistusertib (AZD2014) resulted in a partial response in a patient with castration resistant prostate cancer (J Clin Oncol 35, 2017 (suppl; abstr 2570)). detail...
Unknown unknown 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 prostate cancer not applicable AGS-PSCA Phase Ib/II Actionable In a Phase Ib/II trial, AGS-PSCA treatment was deemed safe, but only resulted in limited antitumor activity in patients with castration resistant prostate cancer (PMID: 22553195). 22553195
Unknown unknown estrogen-receptor positive breast cancer not applicable Everolimus + Tamoxifen Preclinical - Cell culture Actionable In a preclinical study, the combination treatment of Afinitor (everolimus) and Nolvadex (tamoxifen) resulted in decreased colony formation by 95% in estrogen-receptor (ER) positive breast cancer cell lines while in ER positive breast cancer cell lines resistant to Nolvadex (tamoxifen), colony formation formation decreased by 76% with the addition of Afinitor (everolimus) (PMID: 27421652). 27421652
Unknown unknown triple-receptor negative breast cancer not applicable JQ1 + Vinorelbine Preclinical - Cell culture Actionable In a preclinical study, the addition of JQ1 to Taxotere (docetaxel) resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27256375). 27256375
Unknown unknown acute myeloid leukemia not applicable Carboplatin + Topotecan + Veliparib Phase I Actionable In a Phase I trial, the combination of Hycamtin (topotecan), Paraplatin (carboplatin), and Veliparib (ABT-888) resulted in a response rate of 25% (19/77) in patients with acute myeloid leukemia (PMID: 27551000). 27551000
Unknown unknown Advanced Solid Tumor not applicable TB-403 Phase I Actionable In a Phase I trial, TB-403 (RO5323441) demonstrated safety and some preliminary evidence of activity in patients with advanced solid tumors (PMID: 22333707). 22333707
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 colon cancer not applicable Torin 1 Preclinical - Pdx Actionable In a preclinical study, Torin 1 inhibited cell proliferation of colon cancer cell cultures and patient-derived xenograft models (PMID: 24185040). 24185040
Unknown unknown Advanced Solid Tumor not applicable AMG208 Phase I Actionable In a Phase I trial, AMG208 demonstrated safety and preliminary efficacy, resulted in complete response in 2% (1/43), partial response in 7% (3/43) and stable disease in 65% (28/43) of patients with advanced solid tumors (PMID: 26155941; NCT00813384). 26155941
Unknown unknown lung squamous cell carcinoma not applicable Docetaxel + Trametinib Phase I Actionable In a Phase I/Ib trial, treatment with the combination of Mekinist (trametinib) and Taxotere (docetaxel) resulted in partial response in 1 patient and stable disease in 3 patients within the subset of 5 evaluable patients with squamous non-small cell lung cancer patients (PMID: 27876675). 27876675
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). 30361170
Unknown unknown Advanced Solid Tumor not applicable Bevacizumab + Sorafenib Phase I Actionable In a Phase I trial, the treatment combination of Avastin (bevacizumab) and Nexavar (sorafenib) in patients with advanced solid tumors resulted in stable disease in 25% (29/115) of patients and a partial response in 5% (6/115) of patients (PMID: 25363205). 25363205
Unknown unknown hepatocellular carcinoma not applicable Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) improved median progression free survival to 10.7 months in patients with unresectable hepatocellular carcinoma (PMID: 19144678). 19144678 detail...
Unknown unknown diffuse large B-cell 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 germinal center B-cell diffuse large B-cell lymphoma (DLBCL) cell lines in culture, increased apoptotic activity and inhibition of colony formation in activated B-cell (ABC) DLBCL cell lines, and complete tumor growth inhibition in ABC-DLBCL cell line xenograft models (PMID: 28428442). 28428442
Unknown unknown melanoma not applicable GNE-317 Preclinical - Cell line xenograft Actionable In a preclinical study, a melanoma cell line xenograft model demonstrated cell death of metastasized tumor cells within a small region of the brain when treated with GNE-317 (PMID: 27521448). 27521448
Unknown unknown breast cancer not applicable Cisplatin + NU6027 Preclinical - Cell culture Actionable In a preclinical study, NU6027 enhanced the efficacy of Platinol (cisplatin) in breast cancer cells in culture, resulting in decreased cell survival (PMID: 21730979). 21730979
Unknown unknown Advanced Solid Tumor not applicable Refametinib Phase I Actionable In a Phase I trial, Refametinib (BAY 86-9766) was well-tolerated and displayed some evidence of clinical benefit across several tumor types (PMID: 23434733). 23434733
Unknown unknown non-small cell lung 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 Advanced Solid Tumor not applicable OBP-801 Preclinical - Cell line xenograft Actionable In a preclinical study, OBP-201 (YM753) inhibited growth of several solid tumor cell lines in culture and demonstrated anti-tumor activity in a colon cancer cell line xenograft model (PMID: 18292931). 18292931
Unknown unknown bladder carcinoma in situ not applicable BCG solution Phase III Actionable In a Phase III trial supporting FDA approval, TICE BCG solution (BCG solution) treatment resulted in an estimated 2 year disease-free survival rate of 57% (109/191) in patients with bladder carcinoma in situ (PMID: 21224104). 21224104
Unknown unknown melanoma not applicable Ad5CMV-p53 gene + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, the combination of Advexin (Ad5CMV-p53) and an unspecified anti-PD-1 antibody resulted in a synergistic effect and abscopal effect in an immune therapy resistant syngeneic melanoma mouse model, demonstrating decreased tumor growth and improved survival compared to either agent alone (J Clin Oncol 35, 2017 (suppl; abstr e14610)). detail...
Unknown unknown colorectal cancer not applicable Conatumumab + Ganitumab Phase Ib/II Actionable In a Phase Ib/II clinical trial, Ganitumab and Conatumumab combination treatment resulted in one unconfirmed partial response and stable disease in 38% (6/16) of patients with colorectal cancer (PMID: 24816908). 24816908
Unknown unknown Advanced Solid Tumor not applicable Paclitaxel + TAS-119 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 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 non-small cell lung 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 triple-receptor negative breast cancer not applicable BETd-246 + Navitoclax Preclinical - Cell culture Actionable In a preclinical study, the Bet inhibitor BETd-246 and the Bcl-xl inhibitor ABT-263 synergistically induced apoptosis in triple-receptor negative breast cancer cell lines in culture (PMID: 28209615). 28209615
Unknown unknown Advanced Solid Tumor not applicable Talazoparib Preclinical - Cell line xenograft Actionable In a preclinical study, the Talazoparib (BMN-673) selective PARP1/2 inhibitor demonstrated antitumor activity on a variety of cell lines and xenografts with defects in DNA repair (PMID: 23881923). 23881923
Unknown unknown multiple myeloma not applicable Bortezomib + DT204 Preclinical - Cell line xenograft Actionable In a preclinical study, a Velcade (bortezomib) resistant multiple myeloma cell line overcame resistance when treatment was combined with DT204, and in xenograft models the combined treatment resulted in delayed tumor growth and improved survival (PMID: 27677741). 27677741
Unknown unknown leiomyosarcoma not applicable Aldoxorubicin + BEZ235 Preclinical Actionable In a preclinical study, the combination of doxorubicin and BEZ235 produced a synergistic effect in leiomyosarcoma cells both in culture and in mouse models, resulting in apoptotic induction and a 68% reduction in tumor volume (PMID: 26952093). 26952093
Unknown unknown triple-receptor negative breast cancer not applicable JQ1 Preclinical - Cell line xenograft Actionable In a preclinical study, triple-receptor negative breast cancer cells demonstrated sensitivity to treatment with JQ1, resulting in decrease cell proliferation and cell cycle arrest in culture and tumor growth inhibition in cell line xenograft models (PMID: 27256375). 27256375
Unknown unknown triple-receptor negative breast cancer no benefit CCT007093 Preclinical Actionable In a preclinical study, CCT007093 did not inhibit growth of triple-receptor negative breast cancer cell lines in culture (PMID: 20576088). 20576088
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 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 pancreatic ductal adenocarcinoma not applicable Gemcitabine + MN58b Preclinical Actionable In a preclinical study, MN58b and Gemzar (gemcitabine) in combination demonstrated an additive effect on growth inhibition of pancreatic ductal adenocarcinoma cell lines in culture (PMID: 26769123). 26769123
Unknown unknown colorectal cancer not applicable Nivolumab FDA approved Actionable In a Phase II trial (CheckMate 142) that supported FDA approval, treatment with Opdivo (nivolumab) resulted in an objective response rate of 36% (19/53), with 1 complete response and 18 partial responses, and disease control for 12 weeks or more in 70% (37/53) of patients with mismatch repair deficient or microsatellite instability-high metastatic colorectal cancer (PMID: 28734759; NCT02060188). 28734759
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 myeloid neoplasm not applicable Bortezomib + Dexamethasone + Lenalidomide Phase III Actionable In a Phase III trial, Velcade (bortezomib) in combination with Revlimid (lenalidomide) and dexamethasone resulted in an improved median progression-free survival of 43 months compared to 30 months with Revlimid (lenalidomide) plus dexamethasone, and a response rate of 81.5% (176/216) compared to 71.5% (153/214) with Revlimid (lenalidomide) plus dexamethasone in myeloma patients (PMID: 28017406). 28017406
Unknown unknown cervical cancer not applicable BMS-986205 + Nivolumab Phase Ib/II Actionable In a Phase I/II trial, BMS-986205 in combination with Opdivo (nivolumab) resulted in an objective response rate of 14% (3/22) and a durable response rate of 64% (14/22) in patients with cervical cancer (PMID: 29167110). 29167110
Unknown unknown Her2-receptor positive breast cancer not applicable Abemaciclib + Lapatinib Preclinical - Cell culture Actionable In a preclinical study, the addition of Abemaciclib (LY2835219) to Tykerb (lapatinib) treatment enhanced growth inhibitory effects of Tykerb (lapatinib)-resistant ERBB2 (HER2)-receptor positive breast cancer cells in culture (PMID: 26977878). 26977878
Unknown unknown Advanced Solid Tumor not applicable Roniciclib Phase I Actionable In a Phase I trial, treatment with Roniciclib (BAY 1000394) on a 3 days on/4 days off dosing schedule demonstrated safety and resulted in a disease control rate of 32.7% (34/104), with a response rate of 1% (1/104; 1 partial response) and stable disease in 31.7% (33/104) of patients with advanced solid tumors (PMID: 28463960; NCT01188252). detail... 28463960
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 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 ovarian cancer not applicable Sunitinib Phase Ib/II Actionable In a Phase II trial, Sutent (sunitinib) treatment in ovarian cancer patients resulted in an increased PFS and a response rate of 16.7% (6/36) in those that received Sutent (sunitinib) continuously and a a response rate of 5.4% (2/37) in those that received the drug non-continuously (PMID: 22377563, PMID: 24070205). 22377563 24070205
Unknown unknown colorectal cancer not applicable OSI-027 Preclinical - Cell line xenograft Actionable In a preclinical study, OSI-027 inhibited mTORC1 and mTORC2 signaling and growth in colorectal cancer cell line xenograft models (PMID: 21673091). 21673091
Unknown unknown colon cancer not applicable Fluorouracil + Sorafenib Phase I Actionable In a Phase I trial, Nexavar (sorafenib) in combination with Adrucil (fluorouracil) displayed safety and efficacy in advanced solid tumors, including colon cancer (PMID: 22232731). 22232731
Unknown unknown non-small cell lung 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 non-small cell lung 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 non-small cell lung 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 non-small cell lung carcinoma not applicable Motesanib + 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 NSCLC (PMID: 24419239). 24419239
Unknown unknown colorectal cancer not applicable Cetuximab + Fluorouracil + Leucovorin + Oxaliplatin Phase II Actionable In a Phase II trial, Erbitux (cetuximab) in combination with FOLFOX resulted in improved median progression-free survival (6.4 months) comparing to FOLFOX alone (4.5 months) in colorectal cancer patients (hazard ratio =0.81) (PMID: 27002107). 27002107
Unknown unknown triple-receptor negative breast cancer not applicable CFI-402257 Preclinical - Cell line xenograft Actionable In a preclinical study, CFI-402257 inhibited growth of triple-negative breast cancer cell lines in culture, and inhibited tumor growth in xenograft models (PMID: 28270606). 28270606
Unknown unknown hairy cell leukemia not applicable Ibrutinib Preclinical - Cell culture Actionable In a preclinical study, Imbruvica (ibrutinib) inhibited Btk activity and B-cell receptor signaling, thereby resulting in inhibition of cell proliferation and decreased survival of human hairy cell leukemia cells in culture (PMID: 24697238). 24697238
Unknown unknown prostate cancer not applicable Capivasertib + Docetaxel + Prednisone Phase I Actionable In a Phase I trial, AZZD5363 in combination with Taxotere (docetaxel) and Prednisone resulted in more than 50% PSA reduction at 12 weeks in 70% (7/10) of patients with metastatic castration resistant prostate cancer (PMID: 28144789; NCT02121639). 28144789
Unknown unknown glioblastoma multiforme not applicable SL-701 Phase II Actionable In a Phase II trial, SL-701 treatment resulted in partial response in 2.2% (1/46) and stable disease in 32.6% (15/46) of patients with relapsed/refractory glioblastoma, with a 12-month overall survival rate of 37% (median 11 months) (J Clin Oncol 36, 2018 (suppl; abstr 2058); NCT02078648). detail...
Unknown unknown glioblastoma multiforme not applicable SL-701 Phase II Actionable In a Phase II trial, SL-701 treatment resulted in partial response in 3.3% (1/30) and stable disease in 50% (15/30) of patients with recurrant glioblastoma multiforme (Neuro Oncol (2016) 18 (suppl 6): vi20.). detail...
Unknown unknown breast carcinoma not applicable RXDX-106 Preclinical Actionable In a preclinical study, RXDX-106 inhibited tumor growth in orthotopic animal models of breast carcinoma (Eur J Cancer, Vol 69, Supplement 1, December 2016, Page S31). 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 multiple myeloma no benefit Cabozantinib Phase I Actionable In a Phase Ib trial, Cometriq (Cabometyx, cabozantinib) treatment did not demonstrate significant efficacy, resulting in a minimal response in 9% (1/11), stable disease in 73% (8/11), and progression in 18% (2/11) of patients with relapsed and/or refractory multiple myeloma (PMID: 27020089; NCT01866293). 27020089
Unknown unknown liposarcoma not applicable Trabectedin FDA approved Actionable In a Phase III trial that supported FDA approval, Yondelis (trabectedin) treatment resulted in an improved median progression-free survival of 4.2 months versus 1.5 months with Deticene (dacarbazine) in patients with unresectable or metastatic liposarcoma or leiomyosarcoma (PMID: 28774898; NCT01343277). 28774898
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 B-cell lymphoma not applicable CPI-1205 Phase I Actionable In a Phase I trial, CPI-1205 treatment resulted in complete response in 3% (1/32), and stable disease in 16% (5/32) of patients with B-cell lymphomas (Annals of Oncology, Volume 29, Issue suppl_3, 1 March 2018, abstract 420; NCT02395601). detail...
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 colorectal cancer not applicable Capecitabine + Oxaliplatin + Vandetanib Phase I Actionable In a Phase I clinical trial, the combination of Caprelsa (vandetanib), Xeloda (capecitabine), and Eloxatin (oxaliplatin) was well tolerated and demonstrated some efficacy in patients with colorectal cancer (PMID: 21404105). 21404105
Unknown unknown colorectal cancer not applicable Bevacizumab + Fluorouracil + Irinotecan + Leucovorin FDA approved Actionable In a Phase III clinical trial that supported FDA approval, the combination of Avastin (bevacizumab) and FOLFIRI chemotherapy demonstrated increased duration of overall survival and improved progression-free survival compared to FOLFIRI alone in patients with metastatic colorectal cancer (PMID: 22477726, PMID: 15175435). 15175435 22477726
Unknown unknown lung small cell carcinoma not applicable BDA-366 Preclinical - Pdx Actionable In a preclinical study, BDA-366 inhibited tumor growth in a patient-derived xenograft (PDX) model of small cell lung cancer (PMID: 26004684). 26004684
Unknown unknown hepatocellular carcinoma not applicable Torin 2 Preclinical Actionable In a preclinical study, Torin 2 inhibited proliferation of hepatocellular cells in culture (PMID: 26239364). 26239364
Unknown unknown stomach cancer not applicable Cisplatin + Deguelin Preclinical Actionable In a preclinical study, the combination of Deguelin and Platinol (cisplatin) worked synergistically to inhibit growth of gastric cancer cells in culture (PMID: 25202376). 25202376
Unknown unknown non-small cell lung carcinoma not applicable Cediranib + Cisplatin + Gemcitabine Phase I Actionable In a Phase I trial, the combination of Cediranib with Gemzar (gemcitabine) and Platinol (cisplatin) demonstrated preliminary efficacy in patients with advanced non-small cell lung cancer (PMID: 19091548). 19091548
Unknown unknown fibrosarcoma not applicable ABT-348 Preclinical - Cell line xenograft Actionable In a preclinical study, Ilorasertib (ABT-348) inhibited tumor growth in cell line xenograft models of fibrosarcoma (PMID: 22935731). 22935731
Unknown unknown triple-receptor negative breast cancer not applicable TVB-2640 Phase I Actionable In a Phase I clinical trial, TVB-2640 treatment resulted in stable disease for 20 weeks in one patient with triple-receptor negative breast cancer (2015 51 S724-S724 Eur J Cancer). detail...
Unknown unknown cholangiocarcinoma not applicable Cisplatin + Gemcitabine + Silmitasertib Preclinical - Cell line xenograft Actionable In a preclinical study, the protein kinase CK2 inhibitor, Silmitasertib (CX-4945), in combination with Platinol (cisplatin) and Gemzar (gemcitabine), inhibited tumor growth and increased survival of cholangiocarcinoma cell line xenograft models, to a greater extent than either compound alone (PMID: 30316146). 30316146
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 lymphoma not applicable Aldoxorubicin + Metformin Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Glucophage (metformin) and Aldoxorubicin inhibited cell growth in human lymphoma cell lines in culture and inhibited tumor growth in xenograft models (PMID: 22378068). 22378068
Unknown unknown breast carcinoma not applicable CVX-241 Preclinical - Cell line xenograft Actionable In a preclinical study, CVX-241 inhibited tumor growth in a breast carcinoma cell line xenograft model (PMID: 21149738). 21149738
Unknown unknown Advanced Solid Tumor not applicable PF-06263507 Phase I Actionable In a Phase I trial, treatment with PF-06263507 resulted in no objective responses, but led to stable disease in two patients with advanced solid tumors (PMID: 28070718). 28070718
Unknown unknown 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 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 Advanced Solid Tumor not applicable PQR309 Phase I Actionable In a Phase I trial, PQR309 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2560)). detail...
Unknown unknown Advanced Solid Tumor not applicable CH5132799 Phase I Actionable In a Phase I trial, CH5132799 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 25231405). 25231405
Unknown unknown breast cancer not applicable Doxorubicin + NU6027 Preclinical - Cell culture Actionable In a preclinical study, NU6027 enhanced the efficacy of Adriamycin (doxorubicin) in breast cancer cells in culture, resulting in decreased cell survival (PMID: 21730979). 21730979
Unknown unknown 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 hepatocellular carcinoma not applicable Regorafenib FDA approved Actionable In a Phase III trial (RESORCE) that supported FDA approval, treatment with Stivarga (regorafenib) following Nexavar (sorafenib) treatment resulted in improved overall survival (10.6 vs 7.8 months, HR=0.63) compared to Nexavar (sorafenib) followed by placebo in patients with hepatocellular carcinoma (PMID: 27932229; NCT01774344). 27932229
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 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 ovarian cancer not applicable Cisplatin + NU6027 Preclinical - Cell culture Actionable In a preclinical study, NU6027 enhanced the efficacy of Platinol (cisplatin) in ovarian cancer cells in culture, resulting in decreased cell survival (PMID: 21730979). 21730979
Unknown unknown non-Hodgkin lymphoma not applicable Rituximab FDA approved Actionable In a clinical trial that supported FDA approval, treatment with Rituxan (rituximab) resulted in a response rate of 48% in patients (n=166) with relapsed low-grade or follicular non-Hodgkin lymphoma (PMID: 9704735). 9704735
Unknown unknown hepatocellular carcinoma no benefit OPB-31121 Phase I Actionable In a Phase I trial, OPB-31121 treatment resulted in only stable disease in 26% (6/23) of patients with advanced hepatocellular carcinoma, and was considered insufficient for clinical efficacy (PMID: 25676869). 25676869
Unknown unknown extraosseous Ewing's sarcoma not applicable GSK1838705A Preclinical Actionable In a preclinical study, multiple cancer cell lines including multiple myeloma and Ewing's sarcoma were sensitive to GSK1838705A (PMID: 19825801). 19825801
Unknown unknown 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 endometrial carcinoma not applicable XL147 Phase II Actionable In a Phase II trial, Pilaralisib (XL147) treatment resulted in an objective response rate of 6% (4/67) in patients with endometrial carcinoma, although anti-tumor activity is not associated with molecular alterations in PTEN and PIK3R1 (PMID: 25528496). 25528496
Unknown unknown Her2-receptor negative breast cancer not applicable Paclitaxel + Reparixin Phase Ib/II Actionable In a Phase Ib trial, the combination therapy of Taxol (paclitaxel) and Reparixin in patients with metastatic ERBB2 (HER2)-receptor negative breast cancer resulted in a 30% (8/27) response rate and a durable response greater than 12 months in two patients (PMID: 28539464; NCT02001974). 28539464
Unknown unknown ovarian cancer not applicable ABT-737 + BEZ235 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of BEZ235 and Mekinist (trametinib) enhanced the sensitivity of ovarian cancer cells to ABT-737 in culture, resulting in decreased cell viability (PMID: 27980105). 27980105
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 non-Hodgkin lymphoma not applicable Bendamustine + Rituximab Phase III Actionable In a Phase III trial, Rituximab and Bendamustine combination therapy resulted in improved 5-year progression free survival rate (66.5% vs 55.8%), but no difference in overall survival rate (81.7% vs 85%) compared to R-CHOP/R-CVP regimen in patients with indolent non-Hodgkin lymphoma or mantle cell lymphoma (J Clin Oncol 35, 2017 (suppl; abstr 7500)). detail...
Unknown unknown Advanced Solid Tumor not applicable Elenagen Phase Ib/II Actionable In a Phase I/IIa trial, Elenagen treatment resulted in a best response of stable disease in 44% (12/27) of patients with advanced solid tumors (PMID: 28881846). 28881846
Unknown unknown prostate cancer not applicable UC-773587 Preclinical Actionable In a preclinical study, UC-773587 inhibited ERK activation and growth of prostate cells in culture (PMID: 25825487). 25825487
Unknown unknown stomach cancer not applicable Ramucirumab FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Cyramza (ramucirumab) increased progression-free and overall survival in patients with advanced gastric cancer (PMID: 24094768). 24094768 detail...
Unknown unknown breast cancer not applicable Paclitaxel + Vantictumab Preclinical - Pdx Actionable In a preclinical study, Vantictumab (OMP-18R5) worked synergistically with Taxol (paclitaxel) to inhibit tumor growth in patient-derived xenograft models of breast cancer (PMID: 22753465). 22753465
Unknown unknown prostate cancer not applicable C6-ceramide Preclinical Actionable In a preclinical study, C6-ceramide treatment of prostate cancer cells prevented the association between SET and PP2A, which resulted in cell death in culture (PMID: 24025258). 24025258
Unknown unknown colorectal cancer not applicable MM-151 Clinical Study Actionable In a Phase I trial, MM-151 treatment resulted in partial response in 17% (5/29) and stable disease in 28% (8/29) of colorectal patients (J Clin Oncol 34, 2016 (suppl; abstr 2518)). detail...
Unknown unknown prostate cancer not applicable RO6839921 Preclinical - Cell line xenograft Actionable In a preclinical study, RO6839921 demonstrated anti-tumor activity in prostate cancer cell line xenograft models (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A156). detail...
Unknown unknown glioblastoma multiforme no benefit Valproic acid Clinical Study Actionable In a pooled analysis of several clinical trials, use of Valproic acid was not associated with improved progression-free survival or overall survival in glioblastoma patients (PMID: 26786929). 26786929
Unknown unknown non-small cell lung carcinoma conflicting 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 neuroblastoma not applicable ABT-751 + Fenretinide Preclinical - Cell line xenograft Actionable In a preclinical study, Fenretinide and ABT-751 synergistically inhibited growth of neuroblastoma cell lines in culture and in cell line xenograft models (PMID: 27530131). 27530131
Unknown unknown 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 Advanced Solid Tumor not applicable AT7519 Phase I Actionable In a Phase I trial, AT7519 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 25393368). 25393368
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 gastrointestinal stromal tumor not applicable Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Sutent (sunitinib) improved median progression free survival to 27.3 weeks in GIST patients (PMID: 17332278). detail... 17332278
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 lymphoma not applicable Ixazomib + JQ1 Preclinical Actionable In a preclinical study, Ixazomib (MLN9708) and JQ1 combination treatment resulted in increased apoptosis in T-cell lymphoma cells in culture (PMID: 26988986). 26988986
Unknown unknown lung small cell carcinoma not applicable Etoposide + NVP-ADW742 Preclinical Actionable In a preclinical study, NVP-ADW742, when combined with Toposaur (etoposide), demonstrated a synergistic effect in small cell lung cancer cell lines, resulting in inhibition of Akt signaling (PMID: 15746061). 15746061
Unknown unknown alveolar soft part sarcoma not applicable Durvalumab Clinical Study Actionable In a clinical case study, a patient with alveolar soft part sarcoma had a 58% reduction of target lesions and a response lasting 12 months when treated with Imfinzi (durvalumab), and the tumor was shown retrospectively to have a mismatch repair defect signature, poor immune infiltration, and 0% tumoral PD-L1 positivity (PMID: 30018044; NCT01693562). 30018044
Unknown unknown malignant glioma not applicable PQ401 Preclinical - Cell line xenograft Actionable In a preclinical study, PQ401 disrupted cell migration and inhibited growth of glioma cells in culture, and suppressed tumor growth in cell line xenograft models (PMID: 25971682). 25971682
Unknown unknown brain stem glioma not applicable Niraparib + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, a diffuse intrinsic pontine glioma cell line treated with a combination of ionizing radiation and Zejula (niraparib) in culture demonstrated a greater reduction in cell survival compared to either agent alone (PMID: 26351319). 26351319
Unknown unknown breast cancer not applicable R916562 Preclinical - Cell line xenograft Actionable In a preclinical study, R916562 inhibited tumor cell growth and resulted in tumor regression in a cell line xenograft model of breast cancer (PMID: 28711351). 28711351
Unknown unknown thyroid cancer not applicable ABT-737 + Gemcitabine Preclinical Actionable In a preclinical study, the combination of ABT-737 and Gemzar (gemcitabine) was synergistic towards inhibiting cell viability of a majority of human thyroid cancer cell lines tested in culture (PMID: 27042160). 27042160
Unknown unknown acute myeloid leukemia not applicable Decitabine + Venetoclax FDA approved Actionable In a Phase I/II trial that supported FDA approval, Venclexta (venetoclax) in combination with decitabine or azacitidine resulted in complete remission or complete remission with incomplete count recovery in 65% (40/62) of patients 75 years old or older with treatment-naive acute myeloid leukemia ineligible for intensive chemotherapy, with an overall survival of 11 months (PMID: 30361262; NCT02203773). 30361262
Unknown unknown Advanced Solid Tumor not applicable MBG453 Phase Ib/II Actionable In a Phase Ib/II trial, MBG453 treatment resulted in stable disease in 29% (25/87) of patients with advanced solid tumors (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown glioblastoma multiforme not applicable BI2536 + PD-0325901 Preclinical - Cell culture Actionable In a preclinical study, the combination of BI 2536 and PD-0325901 resulted in greater inhibition of cell proliferation in glioblastoma cells in culture compared to treatment with either agent alone (PMID: 26573800). 26573800
Unknown unknown B-cell lymphoma not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) treatment resulted in significant toxicity and an objective response rate of 15% (2/13) in patients with B-cell non Hodgkin's lymphoma (PMID: 28278718). 28278718
Unknown unknown Advanced Solid Tumor no benefit Docetaxel + MK-5108 Phase I Actionable In a Phase I clinical trial, the combination of MK-5108 and Taxotere (docetaxel) did not demonstrate improved efficacy over MK-5108 monotherapy in patients with advanced solid tumors, and toxicity of the combination prevented achievement of target therapeutic exposure levels (PMID: 26620496). 26620496
Unknown unknown diffuse large B-cell lymphoma not applicable Loncastuximab tesirine Phase I Actionable In a Phase I trial, Loncastuximab tesirine treatment resulted in an objective response rate of 57.1% (20/35) with a complete response rate of 34.3% (12/35) in patients with diffuse large B-cell lymphoma (Blood 2017 130(Suppl 1):187, NCT02669017). detail...
Unknown unknown Advanced Solid Tumor not applicable E7046 Preclinical Actionable In a preclinical study, multiple mouse tumor models demonstrated inhibition of tumor growth when treated with E7046 (Cancer Immunol Res 2016;4(1 Suppl):Abstract nr B034). detail...
Unknown unknown Advanced Solid Tumor not applicable E7046 Phase I Actionable In a Phase I trial, E7046 treatment resulted in no objective responses, only stable disease in 13% (4/30) and metabolic responses in 13% (4/30) of patients with advanced solid tumors (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 373PD; NCT02540291). detail...
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 malignant glioma not applicable PV1019 + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, glioma cells treated with PV1019 before and after radiotherapy resulted in greater cell death in culture than compared to radiation treatment alone (PMID: 19741151). 19741151
Unknown unknown multiple myeloma no benefit Tivantinib Phase II Actionable In a Phase II trial, Tivantinib (ARQ197) treatment only resulted in stable disease in 36% (4/11) of patients with relapsed or refractory multiple myeloma (PMID: 28337527). 28337527
Unknown unknown basal cell carcinoma not applicable Taladegib Phase I Actionable In a Phase I trial, Taladegib treatment resulted in a clinical response in 46.8% (22/47) of patients with basal cell carcinoma, including 16 patients with a confirmed partial response, 1 patient with an unconfirmed partial response, and 5 patients with a confirmed complete response (PMID: 29483143). 29483143
Unknown unknown diffuse large B-cell lymphoma not applicable Ublituximab + Umbralisib Phase I Actionable In a Phase I trial, high-dose TGR-1202 in combination with Ublituximab resulted in complete response in 50% (2/4) and partial response in 25% (1/4) of patients with diffuse large B-cell lymphoma (J Clin Oncol 33, 2015 (suppl; abstr 8548)). detail...
Unknown unknown Burkitt lymphoma not applicable RVX2135 + VE-821 Preclinical - Cell culture Actionable In a preclinical study, the combination of RVX2135 and VE-821 resulted in decreased cell viability in Burkitt lymphoma cell lines in culture (PMID: 26804177). 26804177
Unknown unknown acute myeloid leukemia not applicable A-485 Preclinical - Cell culture Actionable In a preclinical study, A-485 treatment inhibited proliferation in acute myeloid leukemia cell lines in culture (PMID: 28953875). 28953875
Unknown unknown thyroid cancer not applicable ABT-737 Preclinical Actionable In a preclinical study, ABT-737 inhibited growth and induced cell death of human thyroid cancer cell lines in culture (PMID: 27042160). 27042160
Unknown unknown Advanced Solid Tumor not applicable HGS1029 Phase I Actionable In a Phase I clinical trial, HGS1029 demonstrated safety and some preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 29: 2011 (suppl; abstr 3008)). detail...
Unknown unknown diffuse large B-cell lymphoma not applicable Ibrutinib + PQR309 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination therapy of PQR309 and Imbruvica (ibrutinib) led to a synergistic effect in 6/7 diffuse large B-cell lymphoma (DLBCL) cell lines in culture, and the effect was confirmed in a DLBCL cell line xenograft model (PMID: 29066507). 29066507
Unknown unknown lung small cell carcinoma not applicable Sacituzumab govitecan Clinical Study Actionable In a clinical study, treatment with Sacituzumab Govitecan resulted in an objective response rate of 14% (7/50), which included 7 patients achieving a partial response, and a median response duration of 5.7 months in patients with lung small cell carcinoma (PMID: 28679770). 28679770
Unknown unknown multiple myeloma not applicable AZD5153 Preclinical - Cell line xenograft Actionable In a preclinical study, AZD5153 inhibited proliferation of multiple myeloma cell lines harboring t (11;14) translocation in culture, resulted in tumor growth inhibition in cell line xenograft models (PMID: 27573426). 27573426
Unknown unknown Advanced Solid Tumor not applicable AMG 900 Preclinical - Cell line xenograft Actionable In a preclinical study, AMG 900 inhibited the growth of a variety of human solid tumor cell lines in culture and inhibited tumor growth in cell line xenograft models of several tumor types including breast, colon, lung, pancreatic, and uterine cancer (PMID: 20935223). 20935223
Unknown unknown hematologic cancer not applicable ID09C3 Phase I Actionable In a Phase I trial, treatment with ID09C3 in patients with B cell type leukemia/lymphoma resulted in decreased peripheral blood B cells and monocytes (PMID: 23090290). 23090290
Unknown unknown Advanced Solid Tumor not applicable PF-00562271 Phase I Actionable In a Phase I trial, PF-00562271 was well-tolerated and resulted in stable disease in 34% (31/91) of patients with advanced solid tumors as well as a metabolic response of 50% (7/14) (PMID: 22454420). 22454420
Unknown unknown malignant glioma not applicable Plicamycin Preclinical - Cell culture Actionable In a preclinical study, treatment with Mithracin (plicamycin) induced cell death and inhibited migration of glioma cell lines in culture (PMID: 20556479). 20556479
Unknown unknown pancreatic adenocarcinoma not applicable Cabozantinib Preclinical Actionable In a preclinical trial, Cometriq (cabozantinib) promoted apoptosis of pancreactic ductal adenocarcinoma cells (PMID: 23661005). 23661005
Unknown unknown Advanced Solid Tumor not applicable Napabucasin Phase I Actionable In a Phase I trial, BBI608 treatment demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 32:5s, 2014 (suppl; abstr 2546)). detail...
Unknown unknown chronic myeloid leukemia not applicable GSK343 Preclinical - Patient cell culture Actionable In a preclinical study, GSK343 decreased H3K27 trimethylation, and reduced viability and increased apoptosis of primary chronic myeloid leukemia cells in culture (PMID: 27630125). 27630125
Unknown unknown triple-receptor negative breast cancer not applicable Niraparib + SY-1365 Preclinical - Cell culture Actionable In a preclinical study, SY-1365 and Zejula (niraparib) synergistically induced apoptosis in triple-receptor negative breast cancer cells in culture (Proceedings of the AACR, Vol 58, April 2017, Abstract # 1151). detail...
Unknown unknown brain glioma not applicable Adavosertib Preclinical Actionable In a preclinical study, Adavosertib (MK-1775) in combination with radiation, reduced survival of cells derived from pediatric patients with high-grade gliomas (PMID: 24305702). 24305702
Unknown unknown Advanced Solid Tumor no benefit BEZ235 + Everolimus Phase Ib/II Actionable In a Phase Ib trial, the combination of BEZ235 and Afinitor (everolimus) resulted in limited efficacy in patients with advanced solid tumors and was discontinued due to multiple intolerable side effects (PMID: 28357727). 28357727
Unknown unknown neuroblastoma not applicable GANT61 + Vincristine Preclinical Actionable In a preclinical study, GANT61 and Oncovin (vincristine) worked synergistically to inhibit growth of neuroblastoma cells in culture (PMID: 22949014). 22949014
Unknown unknown Advanced Solid Tumor not applicable Panvotinib-401 Preclinical Actionable In a preclinical study, Panvotinib-401 demonstrated cancer-selective cytotoxicity in transformed cell lines in culture (AACR, Cancer Res: April 2016; Volume 57, Abstract #3896). detail...
Unknown unknown prostate cancer not applicable CC214-2 Preclinical - Cell line xenograft Actionable In a preclinical study, prostate cancer cell line xenograft models demonstrated inhibition of tumor growth when treated with CC214-2 (PMID: 23414803). 23414803
Unknown unknown Advanced Solid Tumor not applicable LY3039478 Phase I Actionable In a Phase I trial, treatment with LY3039478 was well-tolerated and demonstrated some preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 33, 2015 (suppl; abstr 2533)). detail...
Unknown unknown Advanced Solid Tumor not applicable LY3039478 Phase I Actionable In a Phase I trial, LY3039478 treatment resulted in 80% inhibition of plasma A-beta and more than 50% inhibition of Notch1-regulated genes in patients with advanced solid tumor, leading to partial response in 0.9% (1/110) and stable disease in 32.7% (36/110) of the patients (PMID: 30060061; NCT01695005). 30060061 detail...
Unknown unknown 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 diffuse large B-cell lymphoma not applicable Sirolimus + UCN-01 Preclinical Actionable In a preclinical study, Rapamune (sirolimus), in combination with UCN-01 (sc-3510), resulted in apoptosis and cell cycle arrest in diffuse large B-cell lymphoma cells in culture (PMID: 19223503). 19223503
Unknown unknown 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). 24450857
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 triple-receptor negative breast cancer not applicable JQ1 + Paclitaxel Preclinical - Cell culture Actionable In a preclinical study, the combination of JQ1 and Taxol (paclitaxel) resulted in a synergistic effect and inhibited the growth of triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown fibrosarcoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 81%, median progression-free survival of 5.6 months, an objective response rate of 11% (n=18), and a median overall survival of 12 months in patients with fibrosarcoma (PMID: 29895706; NCT01878448). detail... 29895706
Unknown unknown 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 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 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 hepatocellular carcinoma not applicable Metformin + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, addition of Glucophage (metformin) sensitized hepatocellular carcinoma cells to Nexavar (sorafenib) induced apoptosis in culture, resulted in suppression of postoperative intrahepatic recurrence and lung metastasis in cell line xenograft models (PMID: 26957312). 26957312
Unknown unknown hematologic cancer not applicable GS-9820 Phase I Actionable In a Phase Ib trial, Acalisib (GS-9820) treatment resulted in lymph node reduction in 70% (7/10) and nodal partial response in 40% (4/10) of patients with non-Hodgkin's lymphoma or chronic lymphocytic leukemia (Blood: 122 (21): 2881). detail...
Unknown unknown Hodgkin's lymphoma, nodular sclerosis not applicable Domatinostat Case Reports/Case Series Actionable In a Phase I trial, a patient with nodular sclerosis Hodgkin's lymphoma demonstrated a partial response lasting almost 6 months following treatment with Domatinostat (4SC-202) (PMID: 30347469; NCT01344707). 30347469
Unknown unknown non-small cell lung 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 non-small cell lung carcinoma not applicable Cisplatin + PJ34 Preclinical - Cell culture Actionable In a preclinical study, the combination of Platinol (cisplatin) and PJ34 worked synergistically to induce cell death in non-small cell lung carcinoma cells in culture (PMID: 23428903). 23428903
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 endometrial carcinoma not applicable Sorafenib Preclinical Actionable In preclinical studies, Nexavar (sorafenib) promoted apoptosis of endometrial carcinoma cells (PMID: 23463670). 23463670
Unknown unknown Hodgkin's lymphoma not applicable Brentuximab vedotin FDA approved Actionable In a Phase III trial (AETHERA) that supported FDA approval, Adcetris (brentuximab vedotin) treatment as consolidation therapy after autologous stem-cell transplantation resulted in significantly improved progression-free survival compared to placebo (42.9 vs 24.1 months, HR=0.57, p=0.0013) in patients with classical Hodgkin's lymphoma at risk for relapse or progression after transplantation (PMID: 25796459; NCT01100502). 25796459
Unknown unknown Hodgkin's lymphoma not applicable Brentuximab vedotin FDA approved Actionable In a Phase II trial that supported FDA approval, Adcetris (brentuximab vedotin) treatment resulted in an objective response rate of 73% (72/102, 33 complete response, 38 partial response) in patients with relapsed or refractory classical Hodgkin's lymphoma following autologous stem cell transplantation (PMID: 25533035; NCT00848926). 25533035
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 Advanced Solid Tumor not applicable TTI-621 Preclinical - Cell culture Actionable In a preclinical study, TTI-621 (SIRPalpha-Fc) increased phagocytosis in 67% (8/12) of the human solid tumor cell lines tested in culture (PMID: 27856600). 27856600
Unknown unknown acute myeloid leukemia not applicable Bortezomib + Cytarabine + Etoposide + Midostaurin + Mitoxantrone Phase I Actionable In a Phase I trial, Rydapt (midostaurin), in combination with Velcade (bortezomib) and mitoxantrone, Vepesid (etoposide), and Cytosar-U (cytarabine) (MEC), resulted in an overall response rate of 82.5% (19/23) in patients with relapsed or refractory acute myeloid leukemia receiving dose level 3 and above, with complete responses in 56.5% (13/23) of patients (PMID: 26784138). 26784138
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 acute myeloid leukemia not applicable Cytarabine + LGH447 Preclinical Actionable In a preclinical study, the combination of LGH447 and Cytosar-U (cytarabine) resulted in tumor regression by 50% in an acute myeloid leukemia mouse model (PMID: 24474669). 24474669
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...
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 malignant glioma not applicable Bevacizumab + Marizomib Phase I Actionable In a Phase I trial, Marizomib (NPI-0052) and Avastin (bevacizumab) combination therapy resulted in complete target lesion response in 14% (5/36), partial response in 25% (9/36), and stable disease in 31% (11/36) of Avastin (bevacizumab)-naive patients with WHO grade IV malignant giloma (Neuro Oncol (2016) 18 (suppl 6): vi13.). detail...
Unknown unknown lung cancer not applicable ABTL0812 Preclinical Actionable In a preclinical study, ABTL0812 inhibited growth and mTORC1 signaling and induced autophagy in a human lung cancer cell line in culture (PMID: 26671995). 26671995
Unknown unknown clear cell renal cell carcinoma not applicable Bevacizumab + Vorinostat Phase Ib/II Actionable In a Phase I/II trial, Zolinza (vorinostat) and Avastin (bevacizumab) combination treatment resulted in complete response in 3% (1/33) and partial response in 15% (5/33) of patients with renal clear cell carcinoma, with median progression free survival and overall survival of 5.7 months and 13.9 months, respectively (PMID: 28222071). 28222071
Unknown unknown triple-receptor negative breast cancer not applicable Bortezomib Preclinical - Cell culture Actionable In a preclinical study, Velcade (bortezomib) induced complete growth inhibition of triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown B-cell lymphoma not applicable JSH-150 Preclinical - Cell culture Actionable In a preclinical study, JSH-150 inhibited proliferation of a B-cell lymphoma cell line in culture (PMID: 30253346). 30253346
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 pancreatic cancer not applicable MitoMet-10 Preclinical Actionable In a preclinical study, MitoMet-10 decreased tumor growth in mouse models of pancreatic cancer (PMID: 27216187). 27216187
Unknown unknown triple-receptor negative breast cancer not applicable A-1155463 + BETd-246 Preclinical - Cell culture Actionable In a preclinical study, the Bet inhibitor BETd-246 and the Bcl-xl inhibitor A-1155463 synergistically induced apoptosis in triple-receptor negative breast cancer cell lines in culture (PMID: 28209615). 28209615
Unknown unknown 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 chronic lymphocytic leukemia not applicable Entospletinib Phase II Actionable In a Phase II clinical trial, treatment with entospletinib resulted in a progression-free survival (PFS) rate of 70.1% at 24 weeks, with a median PFS of 13.8 months, and a objective response rate of 61% (24/41; all partial responses) in patients with chronic lymphocytic leukemia (PMID: 25696919). 25696919
Unknown unknown ovarian cancer not applicable Pegylated liposomal-doxorubicin + Trebananib Phase III Actionable In a Phase III trial, Trebananib in combination with Doxil (pegylated liposomal doxorubicin) resulted in improved objective response rate (46% vs. 21%) and median duration of response (7.4 vs. 3.9 months) compared to placebo in patients with recurrent ovarian cancer (PMID: 27914241). 27914241
Unknown unknown Advanced Solid Tumor not applicable Trametinib + Uprosertib Phase I Actionable In a Phase I trial, the combination of Trametinib (GSK1120212) and Uprosertib (GSK2141795) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol (Meeting Abstracts) 2011 29: 3085). detail...
Unknown unknown malignant glioma not applicable Toca 511 + Toca FC Phase Ib/II Actionable In a Phase I trial, Toca 511 and Toca FC combination treatment resulted in complete response in 3 and partial response in 2 patients with recurrent high-grade glioma, with a median duration of response of 25.2 months (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr A085; NCT01470794). detail...
Unknown unknown malignant glioma not applicable Toca 511 + Toca FC Phase I Actionable In a Phase I trial, Toca 511 and Toca FC demonstrated safety and preliminary efficacy, with median overall survival ranging from 12.1 to 13.6 months in patients with recurrent high grade glioma (Neuro Oncol (2016) 18 (suppl 6): vi18.; NCT02414165). detail...
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 chronic lymphocytic leukemia not applicable Cerdulatinib + Venetoclax Preclinical - Patient cell culture Actionable In a preclinical study, the combination of Cerdulatinib (PRT062070) and Venclexta (venetoclax) worked synergistically to induce apoptosis of patient-derived chronic lymphocytic leukemia cells in culture, and resulted in decreased cell viability compared to either drug as a single agent (PMID: 27697994). 27697994
Unknown unknown non-small cell lung 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 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 multiple myeloma not applicable Filanesib Phase Ib/II Actionable In a Phase I/II trial, treatment with Filansenib (ARRY-520) demonstrated safety, and resulted in an overall response rate of 16% (5/31), clinical benefit rate of 23% (7/31), and median overall survival of 19.0 months in the Phase II portion in patients with refractory or relapsed multiple myeloma (PMID: 28817190, NCT00821249). 28817190
Unknown unknown Advanced Solid Tumor not applicable YW3-56 Preclinical Actionable In a preclinical study, YW3-56 inhibited proliferation of a variety of human tumor cell lines in culture, independent of TP53 mutational status (PMID: 25612620). 25612620
Unknown unknown hepatocellular carcinoma not applicable Refametinib + Sorafenib Phase I Actionable In a Phase I trial, 43.8% (7/16) of hepatocellular carcinoma patients treated with a combination of Refametinib (BAY86-9766) and Nexavar (sorafenib) demonstrated stable disease (PMID: 26644411). 26644411
Unknown unknown Burkitt lymphoma not applicable TTI-621 Preclinical - Cell line xenograft Actionable In a preclinical study, the mouse surrogate version of TTI-621 (SIRPalpha-Fc) decreased tumor growth in Burkitt lymphoma cell line xenograft models (PMID: 27856600). 27856600
Unknown unknown acute myeloid leukemia not applicable Azacitidine + Nivolumab Phase II Actionable In a Phase II trial, the combination treatment of Vidaza (azacitidine) and Opdivo (nivolumab) resulted in an overall response rate of 33% (23/70) in patients with relapsed/refractory acute myeloid leukemia, including four with complete remission, 11 with complete remission with incomplete recovery counts, one partial response, and seven with hematological improvement, and led to a median overall survival of 6.3 months (PMID: 30409776; NCT02397720). 30409776
Unknown unknown esophagus squamous cell carcinoma not applicable YM155 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with YM155 induced non-apoptotic cell death in esophageal squamous cell carcinoma (ESCC) cell lines in culture, and inhibited tumor growth in ESCC cell line xenograft models (PMID: 26090615). 26090615
Unknown unknown hepatocellular carcinoma not applicable PF-03446962 Phase I Actionable In a Phase I trial, a patient with hepatocellular carcinoma demonstrated a partial response for 44 days when treated with PF-03446962 (PMID: 26655846). 26655846
Unknown unknown colon cancer not applicable Ensituximab Phase I Actionable In a Phase I trial, Ensituximab (NEO-102) demonstrated safety and preliminary efficacy, resulting in stable disease in 42% (5/12) of patients with refractory colon or pancreatic cancer (PMID: 27449137; NCT01040000). 27449137
Unknown unknown diffuse large B-cell lymphoma not applicable Cerdulatinib Preclinical Actionable In a preclinical study, treatment with Cerdulatinib (PRT062070) resulted in decreased viability and increased apoptosis of diffuse large B-cell lymphoma cells in culture (PMID: 25253883). 25253883
Unknown unknown gastrointestinal system cancer not applicable Axitinib + Fluorouracil + Irinotecan + Leucovorin Phase I Actionable In a Phase I trial, Inlyta (axitinib), in combination with FOLFIRI, demonstrated safety and some efficacy in patients with gastrointestinal tumors (PMID: 24423921). 24423921
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 glioblastoma multiforme no benefit Buparlisib Preclinical Actionable In a preclinical study, treatment with BKM120 demonstrated a survival similar to vehicle in transgenic mouse models of glioblastoma and therefore, resulted in no benefit (PMID: 27199435). 27199435
Unknown unknown 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 16.3% (17/104, 1 complete response, 16 partial response), and stable disease in 45.2% (47/104) of hepatocellular carcinoma patients previously treated with sorafenib, with a median progression-free survival of 4.8 months (J Clin Oncol, 36 (4_suppl), February 2018, 209-209; NCT02702414). detail...
Unknown unknown prostate cancer not applicable UC-857993 Preclinical Actionable In a preclinical study, UC-773587 inhibited ERK activation and growth of prostate cells in culture (PMID: 25825487). 25825487
Unknown unknown Advanced Solid Tumor not applicable 4SC-205 Phase I Actionable In a Phase I trial, 4SC-205 treatment demonstrated safety and promising long-term disease stabilization in patients with advanced solid tumors, resulted in a median time on study of 162 days in patients receiving continuous dosing (J Clin Oncol (Meeting Abstracts) May 2015 vol. 33 no. 15_suppl 2528). detail...
Unknown unknown Advanced Solid Tumor not applicable BMS-690514 Phase Ib/II Actionable In a Phase Ib/II trial, BMS-690514 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 23490650). 23490650
Unknown unknown Advanced Solid Tumor not applicable PKI-402 Preclinical - Cell culture Actionable In a preclinical study, PKI-402 inhibited growth of several human solid tumor cell lines in culture (PMID: 20371716). 20371716
Unknown unknown colorectal cancer not applicable Binimetinib + Fluorouracil + Leucovorin + Oxaliplatin Phase I Actionable In a Phase I clinical trial, the combination of Binimetinib (MEK162) and FOLFOX chemotherapy demonstrated manageable toxicity and preliminary efficacy in metastatic colorectal cancer patients with chemotherapy resistance (J Clin Oncol 34, 2016 (suppl; abstr 2544)). detail...
Unknown unknown Advanced Solid Tumor not applicable SY-1365 Preclinical - Cell culture Actionable In a preclinical study, SY-1365 induced rapid apoptosis in a panel of solid tumor cell lines in culture, including breast, ovarian, colorectal, and lung cancer cells (Proceedings of the AACR, Vol 58, April 2017, Abstract # 1151). detail...
Unknown unknown acute myeloid leukemia not applicable Cytarabine + Daunorubicin + Glasdegib Phase Ib/II Actionable In a Phase Ib trial, the combination of Glasdegib (PF-04449913), Cytosar-U (cytarabine), and Cerubidine (daunorubicin) resulted in an overall survival of 34.7 months in patients with either acute myeloid leukemia (AML) or myelodysplastic syndrome, with 55% (11/20) of AML patients experiencing a complete remission (PMID: 29463550). 29463550
Unknown unknown non-small cell lung carcinoma not applicable MBG453 Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 treatment resulted in stable disease in 2 patients with non-small cell lung cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown Merkel cell carcinoma not applicable Avelumab Phase Ib/II Actionable In a Phase I/II trial, addition of Bavencio (avelumab) to adoptive transfer of Merkel cell polyomavirus (MCPyV)-specific T cells and HLA upregulation resulted in sustained complete response in 75% (3/4) of patients with MCPyV-associated Merkel cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 3044)). detail...
Unknown unknown Merkel cell carcinoma not applicable Avelumab FDA approved Actionable In a Phase II trial that supported FDA approval (JAVELIN Merkel 200), Bavencio (avelumab) treatment resulted in a 62.1% (18/29) objective response rate and a median progression-free survival of 9.1 months, and demonstrated early evidence of durable responses with response duration estimated to be greater than 3 months and 6 months in 93% and 83% of responding patients, respectively, in patients with treatment-naive metastatic Merkel cell carcinoma (PMID: 29566106; NCT 02155647). 29566106
Unknown unknown Merkel cell carcinoma not applicable Avelumab FDA approved Actionable In a Phase II trial that supported FDA approval, Bavencio (avelumab) treatment resulted in an objective response response rate of 31.8% (28/88), with complete response in 9% (8/88) and partial response in 23% (20/88) of patients with Merkel cell carcinoma (PMID: 27592805). 27592805
Unknown unknown Advanced Solid Tumor not applicable BAY1161909 Preclinical Actionable In a preclinical study, BAY1161909 inhibited proliferation of a variety of human solid tumor cell lines in culture (PMID: 26832791). detail... 26832791
Unknown unknown Advanced Solid Tumor not applicable Ibrutinib + unspecified PD-L1 antibody Preclinical Actionable In a preclinical study, the combination treatment of Imbruvica (ibrutinib) and an anti-PD-L1 antibody in mouse models with advanced solid tumors resulted in antitumor efficacy, including decreased tumor size, minimized metastasis, and improved survival in triple-receptor negative breast cancer mouse models, and a 30% cure rate and improved survival in colon cancer mouse models (PMID: 25730880). 25730880
Unknown unknown mature T-cell and NK-cell lymphoma not applicable DS-3201b Phase I Actionable In a Phase I trial, DS-3201b demonstrated preliminary clinical activity in patients with T-cell lymphoma, with an overall response rate of 80% (4/5; 1 complete response/remission, and 3 partial responses) (Blood Dec 2017, 130 (Suppl 1) 4070; NCT02732275). detail...
Unknown unknown 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 stomach cancer not applicable Apatinib + Camrelizumab 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 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 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 renal cell carcinoma not applicable Everolimus + Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) as first line therapy followed by second line therapy, Afinitor (everolimus), resulted in a greater overall survival (29.5 mo vs 22.4 mo) compared to the reverse treatment of the two therapies in patients with metastatic renal cell carcinoma (PMID: 28327953). 28327953
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 glioblastoma multiforme not applicable Iniparib + Radiotherapy + Temozolomide Phase II Actionable In a Phase II trial, Iniparib in combination with radiotherapy and Temodar (temozolomide) resulted in improved median overall survival (22 months, HR=0.44) compared to historical control in patients with newly diagnosed glioblastoma multiforme (PMID: 30131387). 30131387
Unknown unknown colon carcinoma not applicable Bevacizumab + S-49076 Preclinical - Cell line xenograft Actionable In a preclinical study, S-49076, in combination with Avastin (bevacizumab) arrested tumor growth in cell line xenograft models of colon carcinoma with previous resistance to Avastin (bevacizumab) (PMID: 23804704). 23804704
Unknown unknown urinary bladder cancer not applicable OBP-801 Preclinical - Cell culture Actionable In a preclinical study, OBP-801 treatment resulted in decreased cell viability in multiple human bladder cancer cell lines in culture (PMID: 27406983). 27406983
Unknown unknown colorectal cancer sensitive ICEC0942 Preclinical - Cell line xenograft Actionable In a preclinical study, ICEC0942 induced growth arrest of colorectal cancer cells in culture and in cell line xenograft models (PMID: 29545334). 29545334
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 thyroid carcinoma not applicable Vandetanib Phase II Actionable In a Phase II trial, Caprelsa (vandetanib) demonstrated efficacy in patients with advanced differentiated thyroid carcinoma (PMID: 22898678). 22898678
Unknown unknown head and neck squamous cell carcinoma not applicable Sirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Rapamune (sirolimus) decreased tumor growth and increased survival of cell line xenograft models of head and neck squamous cell carcinoma (PMID: 21520111). 21520111
Unknown unknown head and neck squamous cell carcinoma not applicable Cetuximab + IPH2201 Phase II Actionable In a Phase II trial, combined Erbitux (cetuximab) and Monalizumab (IPH2201) treatment was well tolerated in patients with recurrent or metastatic head and neck squamous cell carcinoma, and resulted in a 31% (8/26) objective response rate (partial responses) and stable disease in 54% (14/26) of evaluable patients (PMID: 30503213; NCT02643550). 30503213
Unknown unknown melanoma not applicable JS001 Phase I Actionable In a Phase I trial, JS001 demonstrated safety and preliminary activity in patients with melanoma, renal cell carcinoma, or urothelial carcinoma, with an overall response rate (ORR) of 22% (7/32; 1 complete response (melanoma), 6 partial responses), and an ORR of 20% and 25% and DCR of 53% and 50% in patients with acral or mucosal melanoma, respectively (J Clin Oncol 35, 2017 (suppl; abstr 3067)). detail...
Unknown unknown Advanced Solid Tumor not applicable Gemcitabine + Milciclib Phase I Actionable In a Phase I trial, the combination therapy of Gemzar (gemcitabine) and Milciclib (PHA-848125AC) resulted in a clinical benefit in 36% (5/14) of patients with an advanced solid tumor, including long-term stable disease (6-14 months) in four patients and a partial response in a patient with non-small cell lung carcinoma (PMID: 28424962). 28424962
Unknown unknown breast cancer not applicable Axitinib Preclinical - Cell line xenograft Actionable In a preclinical study, Inlyta (axitinib) disrupted tumor microvasculature and inhibited tumor growth in breast cancer cell line xenograft models (PMID: 17371720). 17371720
Unknown unknown colorectal cancer not applicable Tanibirumab Preclinical - Cell line xenograft Actionable In a preclinical study, Tanibirumab (TTAC-0001) inhibited angiogenesis and tumor growth in human cell line xenograft models of colorectal cancer (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 transitional cell carcinoma not applicable Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in complete response in 2% (1/41), partial response in 17% (7/41), stable disease in 44% (18/41) of urothelial carcinoma patients, with a median progression-free survival of 3.7 months and median overall survival of 8.2 months (J Clin Oncol 34, 2016 (suppl; abstr 4534)). detail...
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 esophageal cancer not applicable BI-847325 Phase I Actionable In a Phase I trial, BI-847325 treatment resulted in stable disease in 30% (21/69) of patients with advanced solid tumors, and one partial response for 67 days in a patient with esophageal cancer (PMID: 26650227). 26650227
Unknown unknown prostate cancer not applicable AS605240 Preclinical Actionable In a preclinical study, AS605240 reduced invasiveness of prostate cancer cells in culture (PMID: 24416348). 24416348
Unknown unknown 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 pancreatic cancer not applicable CG200745 Preclinical - Cell culture Actionable In a preclinical study, CG200745 decreased viability of pancreatic cancer cell lines in culture, including Gemzar (gemcitabine)-resistant cell lines (PMID: 28134290). 28134290
Unknown unknown melanoma not applicable AS1409 Phase I Actionable In a Phase I trial, AS1409 treatment in patients with either melanoma or renal cell carcinoma resulted in stable disease in 46% (6/13) of patients and in two patients with melanoma, one demonstrated a partial response while another showed tumor shrinkage, which lasted beyond 12 months (PMID: 21447719). 21447719
Unknown unknown non-small cell lung carcinoma not applicable Docetaxel + Trametinib Phase I Actionable In a Phase I/Ib trial, treatment with the combination of Mekinist (trametinib) and Taxotere (docetaxel) resulted in an overall response rate (ORR) of 21% (10/47, all partial responses) and stable disease in 43% (20/47) of patients with non-small cell lung cancer (PMID: 27876675). 27876675
Unknown unknown ovarian cancer not applicable Navicixizumab Case Reports/Case Series Actionable In a Phase I trial, Navicixizumab (OMP-305B83) treatment resulted in partial response in 3 patients with advanced ovarian cancer, and 7 of the 11 ovarian cancer patients had a reduction of target lesions (PMID: 30229512). 30229512
Unknown unknown triple-receptor negative breast cancer not applicable tirapazamine Preclinical Actionable In a preclinical study, Tirazone (tirapazamine) inhibited growth of triple-receptor negative breast cancer cell lines under hypoxic culture conditions, regardless of their BRCA1 status (PMID: 25193512). 25193512
Unknown unknown prostate cancer not applicable CX-6258 Preclinical - Cell line xenograft Actionable In a preclinical study, CX-6258 inhibited tumor growth in human prostate cancer cell line xenograft models (PMID: 24900437). 24900437
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 neuroendocrine tumor no benefit Everolimus + Pasireotide Phase II Actionable In a Phase II trial, addition of Pasireotide to Afinitor (everolimus) did not significantly affect progression free survival (16.8 vs 16.6 months) or overall disease control rate (77.2% vs 82.7%) in patients with well-differentiated, progressive pancreatic neuroendocrine tumors (PMID: 28327907). 28327907
Unknown unknown Advanced Solid Tumor not applicable F14512 Preclinical Actionable In a preclinical study, F14512 inhibited growth of a variety of human solid tumor cell lines in culture (PMID: 19047165). 19047165
Unknown unknown Advanced Solid Tumor not applicable Necitumumab Phase I Actionable In a Phase I clinical trial, Portrazza (necitumumab) was well-tolerated and demonstrated preliminary efficacy in patients with advanced solid tumors (PMID: 20197484). 20197484
Unknown unknown prostate cancer not applicable Cabozantinib + unspecified CTLA4 antibody + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, combination of myeloid-derived suppressor cell-targeting with Cometriq (cabozantinib) 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 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 stomach cancer no benefit Cetuximab Phase III Actionable In a Phase III trial, addition of Erbitux (cetuximab) to chemotherapy consisted of capecitabine and cisplatin did not improve progression free survival over chemotherapy alone (4.4 vs 5.6 months) in patients with advanced gastric cancer (PMID: 23594786). 23594786
Unknown unknown NUT midline carcinoma not applicable GSK525762 Phase Ib/II Actionable In a Phase I/II trial, GSK525762 treatment resulted in partial response in 20% (2/10) and stable disease in 40% (4/10) of NUT midline carcinoma patients (Cancer Res 2016;76(14 Suppl): Abstract nr CT014). detail...
Unknown unknown lung small cell carcinoma no benefit Vandetanib Phase II Actionable In a Phase II trial, addition of Caprelsa (vandetanib) to platinum (cisplatin or carboplatin) and etoposide did not improve time to progression (5.62 vs 5.68 months) or overall survival (12.24 vs 9.23 months) compared to placebo in untreated extensive-stage small cell lung cancer (PMID: 27583688). 27583688
Unknown unknown Advanced Solid Tumor not applicable Defactinib Phase I Actionable In a Phase I trial, Defactinib (VS-6063) treatment demonstrated safety in patient with advanced solid tumors (PMID: 26334219). 26334219
Unknown unknown non-small cell lung carcinoma not applicable INCB059872 Preclinical - Cell line xenograft Actionable In a preclinical study, INCB059872 inhibited growth of non-small cell lung carcinoma cell lines in culture and in cell line xenoraft models (Cancer Res 2016;76(14 Suppl):Abstract nr 4704). detail...
Unknown unknown osteosarcoma not applicable Capmatinib Case Reports/Case Series Actionable In a Phase I trial, Capmatinib (INC280) demonstrated safety and preliminary efficacy, resulted in stable disease as best overall response in a patients with osteosarcoma (PMID: 30724423). 30724423
Unknown unknown colon adenocarcinoma not applicable Capecitabine + Lapatinib Phase II Actionable In a Phase II trial, Tykerb (lapatinib) combined with Xeloda (capecitabine) demonstrated safety, but failed to show efficacy in patients with advanced refractory colorectal cancer (PMID: 22811876). 22811876
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 alveolar soft part sarcoma not applicable Dasatinib Phase II Actionable In a Phase II trial, patients with alveolar soft part sarcoma demonstrated a median progression free survival of 11 months and one patient demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown breast cancer not applicable Metformin Preclinical - Cell line xenograft Actionable In a preclinical study, Glucophage (metformin) inhibited the growth of a breast cancer cell line in culture and resulted in decreased tumor volume in a cell line xenograft model (PMID: 26351208). 26351208
Unknown unknown acute myeloid leukemia not applicable Alisertib Phase I Actionable In a Phase I study, Alisertib (MLN8237) treatment after Cytosar-U (cytarabine) and Idarubicin induction resulted in a composite remission rate (complete remission and complete remission with incomplete neutrophil recovery) of 86% (19/22) in patients with acute myeloid leukemia (PMID: 28034990). 28034990
Unknown unknown 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 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 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 Ewing sarcoma not applicable UAB30 Preclinical Actionable In a preclinical study, a renal Ewing sarcoma cell line was sensitive to UAB30 in culture, demonstrating cell-cycle arrest, decreased cell proliferation, and apoptosis (PMID: 26873726). 26873726
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 pancreatic cancer no benefit Gemcitabine + Tacedinaline Phase II Actionable In a Phase II trial, the combination of Gemzar (gemcitabine) and Tacedinaline (CI-944) did not demonstrate benefit over Gemzar (gemcitabine) plus placebo in pancreatic cancer patients (PMID: 16641168). 16641168
Unknown unknown endometrial cancer not applicable Cisplatin + ETP-46464 Preclinical - Cell culture Actionable In a preclinical study, ETP-46464 increased the sensitivity of endometrial cancer cell lines to Platinol (cisplatin) in culture (PMID: 25560806). 25560806
Unknown unknown acute myeloid leukemia not applicable ABBV-744 Preclinical - Cell line xenograft Actionable In a preclinical study, ABBV-744 inhibited growth of acute myeloid leukemia cells in culture and in cell line xenograft models (Cancer Res 2018;78(13 Suppl):Abstract nr DDT01-05). detail...
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 uterus leiomyosarcoma not applicable Pazopanib Clinical Study Actionable In a retrospective clinical study, 33% (9/27) of patients with uterine leiomyosarcoma responded to Votrient (pazopanib) treatment (PMID: 29185261). 29185261
Unknown unknown prostate cancer not applicable 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). 28578607
Unknown unknown pancreatic cancer not applicable Gemcitabine + Pimasertib Preclinical Actionable In a preclinical study, treatment with Pimasertib (MSC1936369B) followed by Gemzar (gemcitabine) resulted in enhanced inhibition of proliferation and induction of apoptosis in pancreatic cell lines in culture (PMID: 26228206). 26228206
Unknown unknown acute lymphocytic leukemia not applicable JQ1 + Silmitasertib Preclinical - Cell culture Actionable In a preclinical study, JQ-1 and Silmitasertib (CX-4945) demonstrated synergy in T-cell acute lymphocytic leukemia cell lines in culture, resulting in increased apoptosis, with stronger synergism in cell lines with higher CK2 expression levels (PMID: 27758824). 27758824
Unknown unknown epithelioid sarcoma not applicable Dasatinib Phase II Actionable In a Phase II trial, patients with epithelioid sarcoma demonstrated a median progression free survival of 7.9 months and two patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown triple-receptor negative breast cancer not applicable LTX-315 + Pegylated liposomal-doxorubicin Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of LTX-315 and Doxil (pegylated liposomal doxorubicin) resulted in increased tumor growth inhibition and regression, increased tumor necrosis, and increased T-cell infiltration in triple-negative breast cancer (TNBC) cell line xenograft models compared to either agent alone, and in TNBC models in the neoadjuvant setting induced tumor regression in 50% and improved survival compared to either agent alone (PMID: 30670061). 30670061
Unknown unknown pancreatic cancer not applicable Erlotinib + Gemcitabine FDA approved Actionable In a Phase III clinical trial that supported FDA approval, the combination of Tarceva (erlotinib) and Gemzar (gemicitabine) resulted in an improved median overall survival of 6.24 months compared to 5.91 months with Gemzar (gemicitabine) and placebo, and prolonged progression-free survival (HR=0.77 (95% CI, 0.64 to 0.92; P = .004)) in patients with advanced pancreatic cancer (PMID: 17452677). 17452677
Unknown unknown B-cell adult acute lymphocytic leukemia not applicable inotuzumab ozogamicin FDA approved Actionable In a Phase III trial supporting FDA approval, Besponsa (inotuzumab ozogamicin) treatment resulted in a significantly improved complete remission rate (80.7%, 88/109), duration of remission (4.6 months), progression free survival (5.0 months) and overall survival (7.7 months) in adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia when compared to standard of care (29.4%, 24/109, 3.1, 1.8, 6.7 months, respectively) (PMID: 27292104; NCT01564784). 27292104
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 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 chronic lymphocytic leukemia not applicable Samalizumab Phase I Actionable In a Phase I trial, Samalizumab (ALXN6000) treatment demonstrated safety and preliminary efficacy, resulting in first-dose response in 40% (10/25) of patients with B-cell chronic lymphocytic leukemia, and reduction of tumor burden in 2 patients (Blood 2010 116:2465). detail...
Unknown unknown non-small cell lung carcinoma not applicable Pemetrexed + Trametinib Phase I Actionable In a Phase I/Ib trial, treatment with the combination of Mekinist (trametinib) and Alimta (pemetrexed) resulted in an overall response rate of 14% (6/42, all partial responses) and stable disease in 55% (23/42) of patients with non-small cell lung cancer (PMID: 27876675). 27876675
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 hepatocellular carcinoma not applicable FH535 Preclinical Actionable In a preclinical study, FH535 demonstrated efficacy by inhibiting proliferation of liver cancer stem cells and hepatocellular carcinoma cells in culture (PMID: 24940873). 24940873
Unknown unknown juvenile astrocytoma not applicable MRK-003 Preclinical Actionable In a preclinical study, MRK-003 inhibited HES1 expression in pediatric low-grade astrocytoma cell lines in culture and decreased migration in 1 of 2 cell lines, but did not have a significant effect on cell growth (PMID: 25575134). 25575134
Unknown unknown diffuse large B-cell lymphoma not applicable MLN0905 + Rituximab Preclinical Actionable In a preclinical study, MLN0905 combined with MabThera (rituximab) resulted in a synergistic effect when treating a diffuse large B-cell lymphoma xenograft model, demonstrating a decrease in tumor volume and increased survival (PMID: 22609854). 22609854
Unknown unknown lung squamous cell carcinoma not applicable Cisplatin + Gemcitabine + Necitumumab FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Portrazza (necitumumab), in combination with gemcitabine and cisplatin, resulted in an increased median overall survival of 11.5 months in squamous NSCLC patients, compared to 9.9 months with gemcitabine and cisplatin alone (PMID: 26045340). 26045340
Unknown unknown pancreatic cancer not applicable Nintedanib Preclinical - Cell line xenograft Actionable In a preclinical study, Nintedanib, alone or with chemotherapy, inhibited tumor growth in cell line xenograft models of lung and pancreatic cancer but not in cell culture (PMID: 23729403). 23729403
Unknown unknown cervical cancer not applicable Radiotherapy + TAS-116 Preclinical - Cell line xenograft Actionable In a preclinical study, TAS-116 increased sensitivity of a cervical cancer cell line to X-ray and carbon ion radiotherapy, and the combination resulted in decreased DNA double-strand break repair and increased cell-cycle arrest in culture, and increased tumor growth delay in xenograft models (PMID: 28062703). 28062703
Unknown unknown acute myeloid leukemia not applicable Barasertib Phase II Actionable In a Phase II trial, Barasertib (AZD1152) treatment resulted in significantly improved objective complete response rate (35.4%, n=48, vs 11.5%, n=26), and median overall survival (8.2 vs 4.5 months, HR=0.88) compared to low dose cytosine arabinoside in elderly patients with acute myeloid leukemia (PMID: 23605952). 23605952
Unknown unknown Advanced Solid Tumor not applicable Demcizumab Phase I Actionable In a Phase I trial, treatment with Demcizumab (OMP-21M18) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 25324140). 25324140
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 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 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 triple-receptor negative breast cancer not applicable BAY 11-7082 + Erlotinib Preclinical - Cell culture Actionable In a preclinical study, the combination of BAY 11-7082 and Tarceva (erlotinib) resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown Advanced Solid Tumor not applicable Danusertib Phase I Actionable In a phase I trial, Danusertib (PHA-739358) demonstrated safety and minimal efficacy in a patients with advanced solid tumors (PMID: 19770380, PMID: 22242557). 22242557 19770380
Unknown unknown head and neck squamous cell carcinoma not applicable Carboplatin + Paclitaxel + Temsirolimus Phase II Actionable In a Phase II trial, the combination of Torisel (temsirolimus), Paraplatin (carboplatin), and Taxol (paclitaxel) resulted in an objective response rate of 41.7% (15/36), which included all partial responses, and 52.3% (19/36) had stable disease (PMID: 28961834). 28961834
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). 29863979
Unknown unknown ovarian cancer not applicable Nivolumab + Varlilumab Phase Ib/II Actionable In a Phase I/II trial, Varlilumab and Opdivo (nivolumab) combination treatment resulted in partial response in 10% (5/49) and stable disease in 39% (19/49) of ovarian cancer patients, with treatment-induced increase of PD-L1 expression and CD8+ T cells more common in patients with better responses (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 3001-3001; NCT02335918). detail...
Unknown unknown multiple myeloma not applicable Bortezomib + SJB3-019A Preclinical - Patient cell culture Actionable In a preclinical study, the combination of SJB3-019A and Velcade (bortezomib) worked synergistically to induce cytotoxicity in multiple myeloma cell lines and primary multiple myeloma cells in culture (PMID: 28270494). 28270494
Unknown unknown sarcoma not applicable Pazopanib Clinical Study Actionable In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 15.4 weeks and median survival of 11.2 months in patients with soft tissue sarcoma (PMID: 26970174). 26970174
Unknown unknown sarcoma not applicable Pazopanib FDA approved Actionable In a Phase III trial that supported FDA approval, Votrient (pazopanib) improved progression free survival in patients with advanced soft tissue sarcoma (PMID: 22595799). detail... 22595799
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 stomach cancer not applicable AZD6738 + Cisplatin Preclinical - Cell culture Actionable In a preclinical study, the addition of AZD6738 to Platinol (cisplatin) treatment in a gastric cancer cell line in culture resulted in enhanced chemotherapeutic sensitivity, demonstrating a synergistic effect (PMID: 28138034). 28138034
Unknown unknown head and neck squamous cell carcinoma not applicable OXi4503 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with OXi4503 resulted in increased tumor cell necrosis and decreased tumor growth and extended median survival in cell line xenograft models of head and neck squamous cell carcinoma (PMID: 26751478). 26751478
Unknown unknown ovarian cancer no benefit Docetaxel + Vandetanib Phase II Actionable In a Phase II trial, the combination of Caprelsa (vandetanib) plus Taxotere (docetaxel) did not result in a greater PFS when compared to Taxotere (docetaxel) alone (PMID: 24709487). 24709487
Unknown unknown renal cell carcinoma not applicable Pegilodecakin Phase I Actionable In a Phase I trial, AM0010 demonstrated safety and resulted in partial responses in 27% (4/15) of patients with renal cell carcinoma (PMID: 27528724; NCT02009449). 27528724
Unknown unknown breast cancer not applicable Alisertib Preclinical - Cell culture Actionable In a preclinical study, Alisertib (MLN8237) disrupted cell cycle progression and inhibited growth of breast cancer cell lines, with preferential inhibition of aromatase inhibitor-resistant cell lines (PMID: 25667100). 25667100
Unknown unknown breast cancer not applicable Alisertib Preclinical - Pdx & cell culture Actionable In a preclinical study, treatment with Alisertib (MLN8237) resulted in reduced cell migration of breast cancer cells in cell motility assays and in patient derived xenograft (PDX) models of breast cancer, improved survival and reduced metastasis was observed (PMID: 27235164). 27235164
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 colorectal cancer not applicable Refametinib + Sorafenib Phase I Actionable In a Phase I trial, a patient with colorectal cancer demonstrated a durable partial response for 358 days when treated with the combination of Refametinib (BAY86-9766) and Nexavar (sorafenib) (PMID: 26644411). 26644411
Unknown unknown 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 Advanced Solid Tumor not applicable DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, DCBCI0901 inhibited PI3K and mTOR activation and inhibited growth of several human tumor cell lines in culture and in xenograft models (Mol Cancer Ther November 2013 12; C270). detail...
Unknown unknown Advanced Solid Tumor not applicable Abexinostat + Pazopanib Phase Ib/II Actionable In a Phase Ib/II trial, the combination of Abexinostat (PCI-24781) and Votrient (pazopanib) in advanced solid tumor patients resulted in a clinical benefit rate of 37% (16/43), a median response duration of 9.1 months, and 8 patients of 43 achieved stable disease or durable response for greater than 12 months (PMID: 28221861). 28221861
Unknown unknown 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
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 mantle cell lymphoma not applicable Buparlisib + Ibrutinib Phase Ib/II Actionable In a Phase I/II trial, Buparlisib (BKM120) and Imbruvica (ibrutinib) combination treatment resulted in a best overall response rate of 88% (15/17, 11 complete response, 4 partial response) in patients with relapsed/refractory mantle cell lymphoma (J Clin Oncol 36, 2018 (suppl; abstr 7520); NCT02756247). detail...
Unknown unknown pancreatic ductal adenocarcinoma not applicable Fluorouracil + MN58b Preclinical Actionable In a preclinical study, MN58b and Adrucil (fluorouracil) in combination demonstrated an additive effect on growth inhibition of pancreatic ductal adenocarcinoma cell lines in culture (PMID: 26769123). 26769123
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 Advanced Solid Tumor not applicable BI 754091 Phase I Actionable In a Phase I trial, BI 754091 treatment resulted in partial response in 6% (1/17) and stable disease in 47% (8/17) of patients with advanced solid tumor (J Clin Oncol. 36, no. 5_suppl (February 2018) 212-212). detail...
Unknown unknown pancreatic cancer not applicable AIM-100 Preclinical Actionable In a preclinical study, treatment with AIM-100 resulted in increased apoptosis and decreased growth of pancreatic cancer cells in culture (PMID: 22322295). 22322295
Unknown unknown Advanced Solid Tumor not applicable Entinostat Phase I Actionable In a Phase I trial, Entinostat treatment in patients with advanced solid tumors resulted in a partial response in 7% (2/27) of patients and stable disease in 26% (7/27) of patients, which ranged from 45 days to 10 months (PMID: 18579665). 18579665
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 ovarian cancer not applicable Cediranib Phase III Actionable In a Phase III trial, Cediranib (AZD-2171) given with chemotherapy and as maintenance therapy resulted in improved median overall survival (27.3 vs 19.9 months) in platinum-sensitive ovarian cancer patients (J Clin Oncol 35, 2017 (suppl; abstr 5506)). detail...
Unknown unknown Advanced Solid Tumor not applicable GDC-0425 + Gemcitabine Phase I Actionable In a Phase I trial, treatment with GDC-0425 followed by Gemzar (gemcitabine) resulted in a best overall response rate (includes stable disease and partial response) of 60% (24/40) in patients with advanced solid tumors (PMID: 27815358). 27815358
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 glioblastoma multiforme 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 glioblastoma (PMID: 16103100). 16103100
Unknown unknown colon cancer not applicable NOX-A12 + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, the combination of NOX-A12 and an anti-PD-1 antibody demonstrated increased efficacy over either agent alone in a mouse colon cancer model, resulting in responses in 5/8 mice, compared to 2/8 with the anti-PD-1 antibody alone (PMID: 28963140). 28963140
Unknown unknown Advanced Solid Tumor not applicable Anlotinib Phase I Actionable In a Phase I trial, Anlotinib (AL-3818) treatment resulted in partial response in 15% (3/20) and stable disease in 70% (14/20) of patients with advanced solid tumors (J Clin Oncol 33, 2015 (suppl; abstr e13586)). detail...
Unknown unknown head and neck squamous cell carcinoma not applicable Buparlisib Preclinical Actionable In a preclinical study, Buparlisib (BKM-120) reduced viability of head and neck squamous cell carcinoma (HNSSC) cells in culture and demonstrated anti-tumor activity in HNSSC xenograft models (PMID: 22116303). 22116303
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 B-cell lymphoma not applicable HMPL-523 Preclinical - Cell line xenograft Actionable In a preclinical study, HMPL-523 decreased viability of SYK-dysregulated B-cell lymphoma cell lines in culture, and inhibited tumor growth in xenograft models (Blood Dec 2016, 128 (22) 3970). detail...
Unknown unknown hepatocellular carcinoma not applicable Fingolimod + Sorafenib Preclinical Actionable In a preclinical study, Gilenya (fingolimod) worked synergistically with Nexavar (sorafenib) to inhibit growth and induce apoptosis in hepatocellular carcinoma cell lines in culture (PMID: 26516583). 26516583
Unknown unknown acute myeloid leukemia not applicable GNE-272 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with GNE-272 in acute myeloid leukemia xenograft models resulted in tumor growth inhibition at all doses, and at the highest dose led to a complete response in one of the eight tested mouse models (PMID: 27682507). 27682507
Unknown unknown Advanced Solid Tumor not applicable NEO2734 Preclinical - Cell line xenograft Actionable In a preclinical study, NEO2734 inhibited proliferation of a variety of tumor cell lines in culture, and resulted in tumor regression in cell line xenograft models (Ann Oncol, 29(suppl_8), Oct 2018, abstract 429P). detail...
Unknown unknown breast cancer not applicable MC180295 Preclinical - Cell culture Actionable In a preclinical study, MC180295 decreased proliferation of a breast cancer cell line in culture (PMID: 30454645). 30454645
Unknown unknown ovarian cancer not applicable BGB-A317 + Pamiparib Phase Ib/II Actionable In a Phase Ib trial, the combination therapy of BGB-A317 and 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 pancreatic cancer not applicable MVT-1075 Preclinical - Cell line xenograft Actionable In a preclinical study, a pancreatic cancer xenograft model demonstrated tumor growth inhibition and tumor regression by 50% when treated with MVT-1075 (AACR 2017, Abstract #5204). detail...
Unknown unknown ovarian cancer not applicable Paclitaxel + TRX-E-002-1 Preclinical - Cell line xenograft Actionable In a preclinical study, TRX-E-002-1 given as a maintenance treatment after Taxol (paclitaxel) treatment resulted in less tumor recurrence compared to placebo in cell line xenograft models of ovarian cancer (PMID: 27196760). 27196760
Unknown unknown renal cell carcinoma not applicable GDC-0349 Preclinical - Cell line xenograft Actionable In a preclinical study, the mTOR inhibitor GDC-0349 demonstrated inhibition of tumor growth in cell line xenograft models of solid tumors, including renal adenocarcinoma (PMID: 24900569). 24900569
Unknown unknown ovarian carcinoma not applicable Disarib Preclinical - Cell line xenograft Actionable In a preclinical study, Disarib treatment led to apoptotic induction and resulted in tumor regression in ovarian carcinoma xenograft models (PMID: 27693384). 27693384
Unknown unknown Advanced Solid Tumor not applicable Onalespib Phase I Actionable In a Phase I trial, AT13387 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 25336693). 25336693
Unknown unknown pancreatic cancer not applicable CBP501 + Cisplatin Preclinical - Cell culture Actionable In a preclinical study, the combination of CBP501 and Platinol (cisplatin) resulted in increased cell death compared to Platinol (cisplatin) alone in a human pancreatic cancer cell line in culture (PMID: 17237275). 17237275
Unknown unknown colorectal adenocarcinoma not applicable Irinotecan + VX-970 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 anaplastic large cell lymphoma not applicable Brentuximab vedotin FDA approved Actionable In a Phase II trial (SGN-35) that supported FDA approval, Adcetris (brentuximab vedotin) treatment resulted in complete remission in 57% (33/58), partial remission in 29% (17/58) of patients with relapsed or refractory systemic anaplastic large cell lymphoma (PMID: 22614995; NCT00866047). 22614995
Unknown unknown Advanced Solid Tumor not applicable Rucaparib Phase I Actionable In a Phase I trial, Rubraca (rucaparib) was well-tolerated and demonstrated preliminary efficacy, with a disease control rate of 86% (6/7), in patients with advanced solid tumors (J Clin Oncol 31, 2013 (suppl; abstr 2585)). detail...
Unknown unknown lung small cell carcinoma not applicable Atezolizumab + Carboplatin + Etoposide Guideline Actionable Tecentriq (atezolizumab), Paraplatin (carboplatin), and Vepesid (etoposide) combination treatment is included in guidelines for patients with small cell lung cancer (NCCN.org). detail...
Unknown unknown lung small cell carcinoma not applicable Atezolizumab + Carboplatin + Etoposide FDA approved Actionable In a Phase III trial (IMpower133) that supported FDA approval, Tecentriq (atezolizumab) in combination with Paraplatin (carboplatin) and Vepesid (etoposide) resulted in significantly improved median overall survival (12.3 vs 10.3 months, HR=0.70, p=0.007) and median progression-free survival (5.2 vs 4.3 months, HR=0.77, p=0.02) compared to placebo in patients with untreated extensive-stage small-cell lung cancer (PMID: 30280641; NCT02763579). 30280641
Unknown unknown non-small cell lung 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 myelodysplastic syndrome not applicable Cytarabine + Daunorubicin + Glasdegib Phase Ib/II Actionable In a Phase Ib trial, the combination of Glasdegib (PF-04449913), Cytosar-U (cytarabine), and Cerubidine (daunorubicin) resulted in an overall survival of 34.7 months in patients with either acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS), with 50% (1/2) of MDS patients experiencing a complete remission (PMID: 29463550). 29463550
Unknown unknown Advanced Solid Tumor not applicable Epacadostat + Pembrolizumab Phase I Actionable In a Phase I trial (ECHO-202/KEYNOTE-037), combined treatment with Epacadostat (INCB024360) and Keytruda (pembrolizumab) was well tolerated and resulted in objective responses (OR) in 40% (25/62, 8 complete responses, 17 partial responses) of patients with advanced solid tumors, including melanoma (12/22 OR), non-small cell lung cancer (5/12 OR), and renal cell carcinoma (2/11 OR) (PMID: 30265610; NCT02178722). 30265610
Unknown unknown breast cancer not applicable NU6027 + Rucaparib Preclinical - Cell culture Actionable In a preclinical study, NU6027 enhanced the efficacy of Rubraca (rucaparib) in breast cancer cells in culture, resulting in a greater decreased cell survival (PMID: 21730979). 21730979
Unknown unknown pancreatic ductal adenocarcinoma not applicable Lestaurtinib Preclinical Actionable In preclinical studies, lestaurtinib has been shown to have an antitumor effect in xenograft models of pancreatic ductal adenocarcinoma (PMID: 10473107). 10473107
Unknown unknown lymphoma 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 lymphoma cell lines in culture (PMID: 27706135). 27706135
Unknown unknown colorectal cancer not applicable MBG453 + Spartalizumab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in partial response in 2 patients with colorectal cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown mast-cell leukemia not applicable Midostaurin FDA approved Actionable In a Phase II trial that supported FDA approval, Rydapt (midostaurin) treatment resulted in an overall response rate of 60% (53/89), a median overall survival of 28.7 months, and a median progression-free survival of 14.1 months in patients with systemic mastocytosis including aggressive systemic mastocytosis, systemic mastocytosis with an associated hematologic neoplasm, and mast-cell leukemia (PMID: 27355533; NCT00782067). 27355533
Unknown unknown Advanced Solid Tumor not applicable Docetaxel + Selumetinib Phase I Actionable In a Phase I trial, the combination of Selumetinib (AZD6244) and Taxotere (docetaxel) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, with partial responses in 22% (6/27) and stable disease for greater than 6 weeks in 52% (14/27) of patients (PMID: 28264648). 28264648
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 acute myeloid leukemia 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 (AML) or myelodysplastic syndrome, with 5% (1/20) of AML patients experiencing a complete remission (PMID: 29463550). 29463550
Unknown unknown acute myeloid leukemia not applicable Cytarabine + Glasdegib FDA approved Actionable In a Phase II trial that supported FDA approval, Daurismo (glasdegib) in combination with low-dose cytarabine resulted in improved median survival (8.3 vs 4.3 months, HR=0.46, p=0.0002) compared to low-dose cytarabine alone in patients with treatment-naive acute myeloid leukemia who are ineligible for intensive chemotherapy (Blood 2016 128 (22): 99; NCT01546038). detail...
Unknown unknown Advanced Solid Tumor not applicable DS-7423 Phase I Actionable In a Phase I trial, DS-7423 demonstrated safety and preliminary clinical activity in patients with advanced solid tumors (Ann Oncol (2014) 25 (suppl 4): iv153). detail...
Unknown unknown prostate cancer not applicable BEZ235 + 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 Advanced Solid Tumor predicted - sensitive NKTR-214 + VB10.NEO Preclinical Actionable In a preclinical study, VB10.NEO and NKTR-214 synergistically enhanced neoantigen-specific T-cell response in animal tumor models (AACR Annual Meeting 2019, Abstract 2256). detail...
Unknown unknown bone giant cell tumor not applicable Bortezomib Preclinical Actionable In a preclinical study, Velcade (Bortezomib) treatment resulted in decreased NF-kappaB signaling, increased apoptosis, and decreased growth of bone giant cell tumor cells in culture, and decreased bone giant cell tumor cell-mediated bone destruction in mouse models (PMID: 26861247). 26861247
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 brain stem glioma not applicable MRK-003 + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, MRK-003 enhanced sensitivity of diffuse pontine glioma cell lines to radiotherapy in culture, resulting in increased apoptosis (PMID: 26115193). 26115193
Unknown unknown Advanced Solid Tumor not applicable Miransertib Phase I Actionable In a Phase I trial, Miransertib (ARQ092) demonstrated safety and is currently being tested for efficacy in clinical trials in patients with advanced solid tumors (American Association for Cancer Research. April 6-10, 2013. Abstract #LB-197). detail...
Unknown unknown Her2-receptor negative breast cancer not applicable Sorafenib Clinical Study Actionable In a meta-analysis of 844 ERBB2 (HER2)-negative breast cancer patients, Nexavar (sorafenib) increased progression-free survival time, but not overall survival or objective response rate (PMID: 24940450). 24940450
Unknown unknown acute myeloid leukemia not applicable AMG 900 Phase I Actionable In a Phase I clinical trial, AMG 900 treatment resulted in a complete response in 9% (3/35) of adult patients acute myeloid leukemia (PMID: 28370201). 28370201
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 Advanced Solid Tumor not applicable Aflibercept Phase I Actionable In a Phase I trial, Zaltrap (aflibercept) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors (PMID: 20028764). 20028764
Unknown unknown Advanced Solid Tumor not applicable Afatinib + Vinorelbine Phase I Actionable In a Phase I trial, the combination of Gilotrif (afatinib) and Navelbine (vinorelbine) resulted in clinical efficacy, including two breast cancer patients with a partial response and stable disease in eight patients with advanced solid tumors (PMID: 26254023). 26254023
Unknown unknown colorectal cancer not applicable Ibrutinib + unspecified CTLA4 antibody Preclinical Actionable In a preclinical study, the combination of Imbruvica (ibrutinib) and an anti-CTLA4 antibody resulted in complete tumor regression in colorectal cancer mouse models (Cancer Res 2016;76(14 Suppl):Abstract nr 2321). detail...
Unknown unknown breast cancer not applicable Barasertib Preclinical - Cell culture Actionable In a preclinical study, Barasertib (AZD1152) treatment disrupted cell cycle progression and inhibited growth of breast cancer cell lines in culture, with preferential inhibition of aromatase inhibitor-resistant cell lines (PMID: 25667100). 25667100
Unknown unknown malignant glioma not applicable Sunitinib Phase II Actionable In a Phase II clinical trial, Sutent (sunitinib) was well-tolerated in young patients with high grade glioma, but did not demonstrate sufficient anti-tumor activity as a single agent, with no patients achieving a sustained objective response (PMID: 27109549). 27109549
Unknown unknown malignant glioma not applicable Sunitinib Preclinical Actionable In a preclinical study, Sutent (sunitinb) induced cell death and decreased proliferation of glioma cells in culture (PMID: 25458015). 25458015
Unknown unknown 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...
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 fallopian tube cancer not applicable Bevacizumab + Everolimus Phase II Actionable In a Phase II trial, Afinitor (everolimus) and Avastin (bevacizumab) combination treatment resulted in progression free survival at 6 months in 28% (14/50) of patients with ovarian, fallopian tube, and peritoneal cancers (J Clin Oncol 34, 2016 (suppl; abstr 5552)). detail...
Unknown unknown malignant glioma not applicable DNX-2401 Phase I Actionable In a Phase I trial, DNX-2401 treatment demonstrated virus-induced oncolysis in patients' tumors, resulted in more than 95% tumor reduction in 3 patients, and survival for more than 3 years in 20% (5/25) of patients with recurrent malignant glioma (PMID: 29432077; NCT02197169). 29432077
Unknown unknown malignant glioma not applicable DNX-2401 Phase I Actionable In a Phase I trial, DNX-2401 treatment resulted in tumor reduction in 72% (18/25), complete response in 12% (3/25) and prolonged stable disease in 8% (2/25) of patients with recurrent malignant glioma, with a median overall survival of 9.5 months and progression-free survival of more than 3 years in patients achieved complete responses (PMID: 29432077). 29432077
Unknown unknown colorectal 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 colorectal carcinoma (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). detail...
Unknown unknown mantle cell lymphoma not applicable Lenalidomide FDA approved Actionable In a Phase II trial (EMERGE) that supported FDA approval, Revlimid (lenalidomide) treatment resulted in an objective response rate of 28% (37/134) with rapid time to response (2.2 months), a median duration of response of 16.6 months, a median progression-free survival of 4.0 months, and a median overall survival of 19.0 months in patients with mantle cell lymphoma who relapsed or progressed after or were refractory to Velcade (bortezomib) (PMID: 24002500; NCT00737529). 24002500
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 acute myeloid leukemia not applicable Cytarabine + Daunorubicin + Debio 1143 Phase I Actionable In a Phase I clinical trial, the combination of Debio1143 (AT-406), daunorubicin, and cytarabine was well tolerated, and resulted in complete remission in 38% (11/23) of patients with poor-risk acute myeloid leukemia, with 6 of those patients (56%) developing relapse during the study period (PMID: 25842225). 25842225
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 Advanced Solid Tumor not applicable BXQ-350 Phase I Actionable In a Phase I trial, BXQ-350 demonstrated safety and preliminary efficacy, resulted in partial response in 6% (1/17) and stable disease in 35% (6/17) of patients with advanced solid tumors (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 2517-2517; NCT02859857). detail...
Unknown unknown acute myeloid leukemia not applicable AKN-028 Preclinical - Cell culture Actionable In a preclinical study, AKN-028 treatment induced apoptosis in acute myeloid leukemia cells in culture and inhibited growth and resulted in decreased tumor mass in acute myeloid leukemia cell line xenograft models (PMID: 22864397). 22864397
Unknown unknown non-small cell lung carcinoma not applicable Carboplatin + Paclitaxel + Ramucirumab Phase II Actionable In a Phase II study, preliminary results reported a 67% response rate in NSCLC patients treated with Cyramza (ramucirumab) in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) (PMID: 22481432). 22481432
Unknown unknown pancreatic adenocarcinoma not applicable Gemcitabine + Pimasertib Phase I Actionable In a Phase I trial, Pimasertib in combination with Gemzar (gemcitabine) demonstrated safety and efficacy in metastatic pancreatic adenocarcinoma patients (PMID: 23846936). 23846936
Unknown unknown breast adenocarcinoma not applicable Disarib Preclinical Actionable In a preclinical study, Disarib treatment led to apoptotic induction and resulted in tumor regression in breast adenocarcinoma cell line mouse models (PMID: 27693384). 27693384
Unknown unknown 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 clear cell renal cell carcinoma no benefit Sorafenib + Temsirolimus Phase II Actionable In a Phase II clinical trial, treatment with the combination of Nexavar (sorafenib) and Torisel (temsirolimus) did not prolong progression-free survival compared to treatment with Avastin (bevacizumab) monotherapy (7.4 months vs 7.5 months) in patients with renal clear cell carcinoma (PMID: 26077237). 26077237
Unknown unknown 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 diffuse large B-cell lymphoma not applicable IT-901 Preclinical Actionable In a preclinical study, IT-901 inhibited growth of diffuse large B-cell lymphoma cells in culture (PMID: 26744524). 26744524
Unknown unknown esophagus adenocarcinoma not applicable IMR-1 Preclinical - Cell line xenograft Actionable In a preclinical study, IMR-1 treatment of esophageal adenocarcinoma cells resulted in decreased colony formation in culture and inhibition of tumor growth in xenograft models (PMID: 27197169). 27197169
Unknown unknown glioblastoma multiforme not applicable Marizomib + Panobinostat Preclinical Actionable In a preclinical study, glioblastoma cells treated with a combination of Farydak (panobinostat) and Marizomib resulted in a synergistic effect, demonstrating decreased cell viability in culture (PMID: 26804704). 26804704
Unknown unknown breast cancer not applicable MLN1117 Phase I Actionable In a Phase I trial, treatment with MLN1117 resulted in antitumor activity, demonstrating partial responses in patients with advanced solid tumors including breast and gastric cancer (J Clin Oncol, May 2015 vol. 33 no. 15_suppl 2501). detail...
Unknown unknown breast cancer not applicable AJI-100 Preclinical - Pdx & cell culture Actionable In a preclinical study, AJI-100 induced apoptosis and inhibited cell growth of breast cancer cell lines and xenografts (PMID: 24930769). 24930769
Unknown unknown kidney cancer not applicable CVX-060 + Sunitinib Preclinical Actionable In a preclinical study, the combination of CVX-060 and Sutent (sunitinib) demonstrated a trend improved overall survival compared to single agent Sutent (sunitinib) in mouse models of unresected and resected renal cancer, however, also demonstrated increased toxicity (PMID: 27651308). 27651308
Unknown unknown non-small cell lung carcinoma not applicable BIO-11006 Preclinical - Cell line xenograft Actionable In a preclinical study, BIO-11006 inhibited primary tumor growth and tumor metastasis in cell line xenograft models of non-small cell lung cancer (Journal of Clinical Oncology 35, no. 15_suppl). detail...
Unknown unknown head and neck squamous cell carcinoma not applicable Cetuximab + EGFR Antisense DNA + Radiotherapy Phase I Actionable In a Phase I trial, combined Erbitux (cetuximab) and radiotherapy plus intratumoral delivery of EGFR antisense DNA was well tolerated by patients with head and neck squamous cell carcinoma and showed preliminary efficacy with complete responses in 66.7% (4/6) of patients and one partial response (PMID: 30291796; NCT00903461; NCT01592721). 30291796
Unknown unknown glioblastoma multiforme not applicable A-1210477 + G-TPP Preclinical - Patient cell culture Actionable In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the Mcl-1 inhibitor A-1210477 synergistically inhibited viability of established lines and patient-derived glioblastoma cells in culture (PMID: 28522750). 28522750
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 gastrointestinal stromal tumor not applicable Regorafenib FDA approved Actionable In a Phase III clinical trial (GRID) that supported FDA approval, Stivarga (regorafenib) demonstrated safety and improved progression free survival compared to placebo (4.8 vs 0.9 months, HR=0.27, p<0.0001) in patients with gastrointestinal stromal tumors (PMID: 23177515; NCT01271712). detail... 23177515
Unknown unknown adenocarcinoma not applicable LY3039478 Phase I Actionable In a Phase I trial, a patient with adenoid cystic carcinoma demonstrated a metabolic response when treated with LY3039478 (European Journal of Cancer, Volume 69, S15). detail...
Unknown unknown ovarian cancer not applicable KPT-185 Preclinical - Patient cell culture Actionable In a preclinical study, KPT-185 inhibited growth of platinum-sensitive and platinum-resistant immortalized human ovarian cancer cell lines and patient-derived ovarian cancer cell lines in culture (PMID: 27649553). 27649553
Unknown unknown colon cancer not applicable Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, Sutent (sunitinib) induced apoptosis in colon cancer cells in culture and in cell line xenograft models (PMID: 22912816). 22912816
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 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 non-small cell lung 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 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 diffuse large B-cell lymphoma not applicable Birabresib Preclinical - Cell line xenograft Actionable In a preclinical study, Birabresib (OTX015) resulted in apoptotic activity and inhibition of Myc and downstream signaling pathways in diffuse large B-cell lymphoma cell lines in culture, and reduced tumor growth in xenograft models (PMID: 25623213). 25623213
Unknown unknown Advanced Solid Tumor not applicable Epirubicin + Valproic acid Phase I Actionable In a Phase I trial, Valproic acid combined with Ellence (epirubicin) demonstrated safety and efficacy in patients with advanced solid tumors (PMID: 17513804). 17513804
Unknown unknown Advanced Solid Tumor not applicable Vandetanib Phase I Actionable In a Phase I trial, treatment with Caprelsa (vandetanib) resulted in stable disease in 40% (31/77) of patients with advanced solid tumors (PMID: 15905307). 15905307
Unknown unknown Her2-receptor positive breast cancer not applicable Abemaciclib + Trastuzumab Preclinical - Pdx & cell culture Actionable In a preclinical study, the combination of Herceptin (trastuzumab) and Abemaciclib (LY2835219) resulted in tumor growth regresssion in an ERBB2 (HER2)-receptor positive breast cancer treatment refractory patient derived xenograft model and in ERBB2 (HER2)-receptor positive cultured cells, resulted in decreased cell viability (PMID: 26977878). 26977878
Unknown unknown non-small cell lung 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 peritoneal serous adenocarcinoma not applicable Paclitaxel + TVB-2640 Phase I Actionable In a Phase I trial, TVB-2640 and Taxol (paclitaxel) combination treatment resulted in partial response in one patient with peritoneal serous carcinoma (2015 51 S724-S724 Eur J Cancer ). detail...
Unknown unknown colon cancer not applicable Cetuximab + Temsirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Torisel (temsirolimus) increased sensitivity to Erbitux (cetuximab) in cell line xenograft models of colon cancer (PMID: 24493623). 24493623
Unknown unknown malignant pleural mesothelioma not applicable Trabectedin Preclinical - Cell line xenograft Actionable In a preclinical study, Yondelis (trabectedin) inhibited growth and increased apoptosis of malignant pleural mesothelioma (MPM) cell lines in culture, decreased viability of primary MPM cells in culture, and inhibited tumor growth in MPM cell line xenograft models (PMID: 27512118). 27512118
Unknown unknown follicular lymphoma not applicable Hu5F9-G4 + Rituximab Phase Ib/II Actionable In a Phase Ib study, combined Hu5F9-G4 and Rituxan (rituximab) therapy demonstrated safety and efficacy, resulting in an objective response rate of 71% (5/7, 3 complete and 2 partial responses) in patients with follicular lymphoma, and a median duration of response longer than 6 months (PMID: 30380386). 30380386
Unknown unknown acute myeloid leukemia not applicable Cytarabine + Venetoclax FDA approved Actionable In a Phase I/II trial that supported FDA approval, Venclexta (venetoclax) in combination with low-dose cytarabine resulted in complete remission or complete remission with incomplete count recovery in 54% (44/82) of patients with acute myeloid leukemia who were ineligible for intensive chemotherapy, with a median overall survival of 10 months (ASH Annual Meeting, Dec 2018, Abstract 284; NCT02287233). 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 follicular lymphoma not applicable Buparlisib + Ibrutinib Phase Ib/II Actionable In a Phase I/II trial, Buparlisib (BKM120) and Imbruvica (ibrutinib) combination treatment resulted in a best overall response rate of 20% (1/5, 1 complete response) in patients with relapsed/refractory follicular lymphoma (J Clin Oncol 36, 2018 (suppl; abstr 7520); NCT02756247). detail...
Unknown unknown acute myeloid leukemia not applicable MT1 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with MT1 resulted in decreased leukemic burden and improved survival in a acute myeloid leukemia cell line xenograft model (PMID: 27775715). 27775715
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 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 prostate cancer not applicable Abiraterone + Prednisone + Veliparib Phase II Actionable In a Phase II trial, addition of Veliparib (ABT-888) to Zytiga (abiraterone) and Prednisone moderately improved PSA response rate (72.4% vs 63.9%, p=0.27) and objective response rate (52.2% vs 45%, p=0.51) in patients with metastatic castration-resistant prostate cancer (J Clin Oncol 35, 2017 (suppl; abstr 5001)). 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 acute myeloid leukemia predicted - sensitive 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 malignant peripheral nerve sheath tumor not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) treatment resulted in twelve-week progression free survival in 60% (6/10) of malignant peripheral nerve sheath tumor patients (PMID: 27502708). 27502708
Unknown unknown multiple myeloma not applicable Daratumumab FDA approved Actionable In a Phase I/IIb trial that supported FDA approval, Darzalex (daratumumab) treatment in multiple myeloma patients resulted in an overall response rate of 36% (15/42) with 2 patients having a complete response, 2 patients having a good partial response and 11 patients having a partial response (PMID: 26308596). 26308596
Unknown unknown triple-receptor negative breast cancer not applicable Adavosertib + Everolimus Preclinical - Cell culture Actionable In a preclinical study, the combination of Afinitor (everolimus) and Adavosertib (MK-1775) resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown Advanced Solid Tumor not applicable AMG 337 Phase I Actionable In a Phase I trial, AMG 337 demonstrated safety and preliminary efficacy in patients with a variety of advanced solid tumors (J Clin Oncol 33, 2015 (suppl; abstr e13538)). detail...
Unknown unknown rhabdoid cancer not applicable Panobinostat Preclinical - Cell line xenograft Actionable In a preclinical study, Farydak (panobinostat) treatment of rhabdoid cancer cell lines in culture resulted in cell cycle arrest and cell differentiation and in cell line xenograft models, inhibition of tumor growth and a decrease in tumor size (PMID: 26920892). 26920892
Unknown unknown non-small cell lung 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 follicular lymphoma not applicable Bendamustine + Bortezomib + Rituximab Phase II Actionable In a Phase II trial, addition of Velcade (Bortezomib) to Bendamustine and Rituxan (rituximab) combination therapy resulted in improved complete response (74%, 63/85) compared to without Velcade (Bortezomib) (58%, 80/137) in high risk follicular lymphoma patients (J Clin Oncol 34, 2016 (suppl; abstr 7507)). detail...
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 ovarian cancer not applicable ENMD-2076 Phase II Actionable In a Phase II clinical trial, ENMD-2076 demonstrated efficacy in patients with recurrent, platinum-resistant ovarian, fallopian tube or peritoneal cancers (PMID: 22921155). 22921155
Unknown unknown 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 renal cell carcinoma not applicable CB-839 + Everolimus Phase I Actionable In a Phase I trial, the combination of CB-839 and Afinitor (everolimus) was well-tolerated and resulted in a disease control rate of 100% (8/8) in patients with papillary or clear cell renal cell carincoma, with 1 partial response, and 7 patients achieving stable disease (EORTC-NCI-AACR 2016, Abstract 26). detail...
Unknown unknown central nervous system lymphoma not applicable Rituximab Clinical Study Actionable In a meta-analysis of 580 patients, intravenous Rituximab treatment correlated with overall survival (HR=0.498, p<0.001) in patients with primary central nervous system lymphoma (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 333PD). 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). 25281440 24552320
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 ovarian cancer not applicable Carboplatin + Cediranib Phase III Actionable In a Phase III clinical trial, the addition of Cediranib (AZD-2171) to platinum-based chemotherapy, including Paraplatin (carboplatin)-based therapy, followed by Cediranib (AZD-2171) maintenance therapy, resulted in an improved median progression-free survival of 11 months versus 8.7 months with platinum-based therapy plus placebo in platinum-sensitive ovarian cancer patients (PMID: 27025186). 27025186
Unknown unknown Advanced Solid Tumor not applicable OSU03012 Phase I Actionable In a Phase I trial, OSU03012 (AR-12) treatment demonstrated safety and resulted in stable disease in 6% (2/30) of patients with advanced solid tumors, however, a new formulation was recommended due to limited absorption of the drug (J Clin Oncol 31, 2013 (suppl; abstr 2608)). detail...
Unknown unknown colon cancer not applicable Etoposide + NU7441 Preclinical - Cell line xenograft Actionable In a preclinical study, NU7441 increased the sensitivity of colon cancer cell lines to Vepesid (etoposide), resulting in decreased cell survival in culture and reduced tumor growth in xenograft models (PMID: 16707462). 16707462
Unknown unknown Advanced Solid Tumor not applicable Sapitinib Preclinical - Cell line xenograft Actionable In a preclinical study, Sapitinib (AZD8931) inhibited EGFR, ERBB2 (HER2), and ERBB3 (HER3) kinase activity, and inhibited tumor growth in several cell line xenograft models, including breast, NSCLC, colorectal, and head and neck squamous cell carcinoma xenograft models (PMID: 20145185). 20145185
Unknown unknown triple-receptor negative breast cancer not applicable Paclitaxel + Plicamycin Preclinical Actionable In a preclinical study, Mithracin (plicamycin) and Taxol (paclitaxel) worked synergistically to inhibit the growth of triple-receptor negative breast cancer cell lines in culture (PMID: 20576088). 20576088
Unknown unknown head and neck squamous cell carcinoma not applicable Cetuximab + Cisplatin + Patritumab Phase Ib/II Actionable In a Phase Ib clinical trial, combined Erbitux (cetuximab), Patritumab (U3-1287), and Platinol (cisplatin) treatment was tolerable in patients with recurrent/metastatic head and neck squamous cell carcinoma and resulted in a 47% (7/15) overall response rate (3 complete responses and 4 partial responses), stable disease in 53% (8/15) of patients, a 7.9-month median progression-free survival, and a 13.5-month overall survival (PMID: 30327312; NCT02350712). 30327312
Unknown unknown colorectal cancer not applicable Bevacizumab + Fluorouracil + Irinotecan + Leucovorin + Oxaliplatin Phase II Actionable In a Phase II trial, the combination of Avastin (bevacizumab) plus FOLFOXIRI chemotherapy was well-tolerated and resulted in improved progression-free survival compared to Avastin (bevacizumab) plus FOLFOX in colorectal cancer patients (J Clin Oncol 35, 2017 (suppl 4S; abstract 658)). detail...
Unknown unknown lung carcinoma not applicable MRx0518 Preclinical Actionable In a preclinical study, MRx0518 stimulated immune response and reduced tumor size in syngeneic mouse models of lung carcinoma (Journal of Clinical Oncology 36, no. 15_suppl). detail...
Unknown unknown stomach cancer not applicable Bevacizumab Phase III Actionable In a Phase III trial, addition of Avastin (bevacizumab) to chemotherapy consisted of fluoropyrimidine and cisplatin resulted in improved median overall survival (12.1 vs 10.1 months), median progression-free survival (6.7 vs 5.3 months) and overall response rate (46.0% vs 37.4%) compared to chemotherapy alone in gastric cancer patients (PMID: 21844504). 21844504
Unknown unknown Ewing sarcoma not applicable Olaparib + Temozolomide Preclinical - Cell culture Actionable In a preclinical study, Ewing sarcoma cells treated with Temodar (temozolomide) combined with Lynparza (olaparib) resulted in very strong synergism, inducing apoptosis and reducing cell viability in culture (PMID: 26438158). 26438158
Unknown unknown pancreatic cancer not applicable Gemcitabine + Uproleselan Preclinical - Cell line xenograft Actionable In a preclinical study, Uproleselan (GMI-1271) and Gemzar (gemcitabine) combination treatment resulted in reduced tumor metastasis in cell line xenograft models of pancreatic cancer (Cancer Res 2014;74(19 Suppl):Abstract nr 4503). detail...
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 stomach cancer not applicable OPB-111077 Phase I Actionable In a Phase I trial, two gastric cancer patients demonstrated stable disease when treated with OPB-111077 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B118). detail...
Unknown unknown non-small cell lung 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 triple-receptor negative breast cancer not applicable Alisertib + Docetaxel Preclinical - Pdx & cell culture Actionable In a preclinical study, Alisertib (MLN8237) showed additive and synergistic antitumor activity with Taxotere (docetaxel) in primary tumor and cell line xenograft models of triple-negative breast cancer, resulting in tumor regression (PMID: 24980948). 24980948
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).