Molecular Profile Detail

Profile Name Unknown unknown
Gene Variant Detail

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Relevant Treatment Approaches

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown triple-receptor negative breast cancer not applicable LY3200882 Preclinical - Cell line xenograft Actionable In a preclinical study, LY3200882 induced tumor regression in a triple-negative breast cancer cell line xenograft model (AACR Annual Meeting 2017, Abstract nr 955). detail...
Unknown unknown NUT midline carcinoma not applicable Birabresib Phase Ib/II Actionable In a Phase Ib trial, Birabresib (OTX015) demonstrated favorable safety and resulted in partial response in 33% (3/9) and stable disease in 33% (3/9) patients with NUT midline carcinoma (PMID: 29733771; NCT02259114). 29733771
Unknown unknown NUT midline carcinoma not applicable Birabresib Preclinical - Cell line xenograft Actionable In a preclinical study, Birabresib (OTX015) inhibited tumor growth in cell line xenograft models of NUT midline carcinoma (Mol Cancer Ther November 2013 12; C244). detail...
Unknown unknown prostate cancer sensitive Bicalutamide FDA approved Actionable In a clinical trial that supported FDA approval, combined with a luteinizing hormone-releasing hormone analogue (LHRH-A) therapy, Casodex (bicalutamide) demonstrated efficacy similar to Eulexin (flutamide), resulted in comparable median time to progression (97 vs 77 weeks, HR=0.93, p=0.41) and survival time (180 vs 148 weeks, HR=0.87, p=0.15) in patients with metastatic prostate cancer (PMID: 9301693). 9301693 detail...
Unknown unknown multiple myeloma not applicable ACY-1215 + Dexamethasone + lenalidomide 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 breast cancer not applicable DCBCI0901 Preclinical - Cell culture Actionable In a preclinical study, breast cancer cell lines treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown triple-receptor negative breast cancer not applicable Alisertib + Taxol Preclinical - Pdx Actionable In a preclinical study, the combination of Alisertib (MLN8237) and Taxol (paclitaxel) worked synergistically or additively to inhibit tumor growth in cell line and patient-derived xenograft (PDX) models of triple-negative breast cancer (PMID: 24980948). 24980948
Unknown unknown stomach cancer not applicable Avelumab Phase I Actionable In a Phase I trial, Bavencio (avelumab) treatment resulted in partial response in 27% (3/11) of patients with advanced gastric cancer (J Clin Oncol 33, 2015 (suppl; abstr 4047)). detail...
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 hepatocellular carcinoma not applicable NanoHHI Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with NanoHHI decreased growth and migration of hepatocellular carcinoma cells in culture, and inhibited tumor growth and metastasis in hepatocellular carcinoma cell line xenograft models (PMID: 21868763). 21868763
Unknown unknown Advanced Solid Tumor not applicable Selumetinib + Docetaxel 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 follicular lymphoma not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in complete response in 12% (2/17) and partial response in 41% (7/17) of patients with follicular lymphoma (PMID: 29475723; NCT01767766). 29475723
Unknown unknown Advanced Solid Tumor not applicable 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 non-small cell lung carcinoma not applicable Lenvatinib Phase I Actionable In a Phase I clinical trial, Lenvima (lenvatinib) demonstrated anti-tumor activity in patients with several advanced solid tumor types, including patients with non-small cell lung cancer (PMID: 26169970). 26169970
Unknown unknown 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 follicular lymphoma not applicable Bendamustine + Obinutuzumab FDA approved Actionable In a Phase III trial that supported FDA approval, the combination of Gazyva (obinutuzumab) plus Treanda (bendamustine) resulted in a greater progression free survival (29.2 mo vs 14.0 mo) than Treanda (bendamustine) alone in patients with follicular lymphoma (PMID: 27345636). 27345636
Unknown unknown acute myeloid leukemia not applicable AT-406 + Daunorubicin + Cytarabine 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 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 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 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 renal cell carcinoma not applicable Carotuximab + Axitinib Phase I Actionable In a Phase I trial, treatment the combination of Inlyta (axitinib) and Carotuximab (TRC-105) demonstrated preliminary activity in patients with renal cell carcinoma, resulting in partial response in 5 and stable disease in 10 of 17 evaluable patients, and a median progression-free survival of 11.3 months (PMID: 30190302; NCT01806064). 30190302
Unknown unknown 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 Ewing sarcoma not applicable Olaparib + Temozolomide + SN-38 Preclinical - Cell culture Actionable In a preclinical study, Ewing sarcoma cells treated with the combination of Temodar (temozolomide), SN-38, and Lynparza (olaparib) had a much greater effect on decreasing cell viability and inducing apoptosis when compared to each treatment administered as a single agent or as dual agents in culture (PMID: 26438158). 26438158
Unknown unknown 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 malignant glioma not applicable Mithramycin 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 melanoma not applicable Dovitinib Phase Ib/II Actionable In a Phase I/II study, Dovitinib (TKI258) was demonstrated safe, but of limited clinical benefit in patients with advanced melanoma (PMID: 21976540). 21976540
Unknown unknown 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 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 triple-receptor negative breast cancer not applicable Selumetinib + SHP099 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination therapy of SHP099 and Selumetinib (AZD6244) led to decreased cell viability and reduced colony formation in triple-receptor negative breast cancer cells in culture and tumor regression in xenograft models (PMID: 30045908). 30045908
Unknown unknown 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 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 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 acute myeloid leukemia not applicable Guadecitabine Phase I Actionable In a Phase I trial, SGI-110 treatment resulted in clinical response in 8% (6/74) of patients with acute myeloid leukemia (PMID: 26296954). 26296954
Unknown unknown 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 Advanced Solid Tumor not applicable Gemcitabine + LY2780301 Phase Ib/II Actionable In a Phase Ib trial, the combination of LY2780303 and Gemzar (gemcitabine) demonstrated some antitumor activity in patients with advanced solid tumors including 2 patients with a partial response, 28 patients with stable disease, and a four month disease control rate of 22% (PMID: 28750271). 28750271
Unknown unknown acute myeloid leukemia not applicable SNS-510 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with SNS-510 decreased PDPK1 pathway signaling and reduced proliferation of acute myeloid leukemia (AML) cell lines in culture, and inhibited tumor growth in an AML cell line xenograft model (Mol Cancer Ther, Dec 1 2015 (14) (12 Supplement 2) C198). detail...
Unknown unknown chordoma not applicable Dasatinib Phase II Actionable In a Phase II trial, patients with chordoma demonstrated a median progression free survival of 6.3 months and six patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown breast cancer not applicable Azacitidine + Entinostat Phase II Actionable In a Phase II trial, the combination of Vidaza (azacitidine) and Entinostat (MS-275) did not reach its primary endpoint for either cohort of breast cancer patients, TNBC or hormone-resistant, however, the optional continuation phase resulted in one patient achieving a partial response (PMID: 27979916). 27979916
Unknown unknown Advanced Solid Tumor not applicable Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors (PMID: 22516948). 22516948
Unknown unknown Advanced Solid Tumor not applicable Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) treatment resulted in partial response in 11.7% (9/77) and stable disease in 51.9% (40/77) of patients with advanced solid tumors (PMID: 26169970). 26169970
Unknown unknown leiomyosarcoma not applicable Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (3.7 vs 1.8 months), but no difference in overall survival (HR=0.50, p=0.056) compared to placebo in patients with leiomyosarcoma (PMID: 27751846). 27751846
Unknown unknown stomach cancer not applicable PU-H71 Preclinical - Cell culture Actionable In a preclinical study, sensitivity to PU-H71 was correlated to presence of the epichaperome, a network of chaperome complexes, in gastric cancer cell lines in culture (PMID: 27706135). 27706135
Unknown unknown 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 papillary thyroid carcinoma not applicable Lenvatinib Clinical Study Actionable In a clinical study, Lenvima (lenvatinib) treatment resulted in prolonged response in a papillary thyroid carcinoma patient whose disease progressed despite 3 prior therapies including Nexavar (sorafenib), Sutent (sunitinib), and Votrient (pazopanib) (PMID: 29167691). 29167691
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 Advanced Solid Tumor not applicable PF-06647263 Phase I Actionable In a Phase I trial, PF-06647263 treatment resulted in partial response in 10% (5/48) of patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2511)). detail...
Unknown unknown hepatocellular carcinoma not applicable PI-88 Phase II Actionable In a phase II trial, PI-88 treatment significantly improved survival for up to 3 years in hepatocellular carcinoma patients compared to control, as evidenced by increased recurrence-free rate (63% vs 50%) and postponed time to recurrence at the 36th percentile by 78% (PMID: 25170226). 25170226
Unknown unknown 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 glioblastoma multiforme not applicable Cediranib + Gefitinib Phase II Actionable In a Phase II trial, treatment with the combination of Cediranib (AZD-2171) and Iressa (gefitinib) resulted in a trend toward improved progression-free survival compared to Cediranib (AZD-2171) and placebo (3.6 months vs 2.8 months), and resulted in a response rate of 42% (8/19), compared to 26% (5/19) with Cediranib (AZD-2171) plus placebo in patients with recurrent glioblastoma (PMID: 27232884). 27232884
Unknown unknown lung cancer not applicable PU-H71 Preclinical - Cell culture Actionable In a preclinical study, sensitivity to PU-H71 was correlated to presence of the epichaperome, a network of chaperome complexes, in lung cancer cell lines in culture (PMID: 27706135). 27706135
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 mantle cell 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 colon cancer not applicable WANT3 Preclinical - Cell culture Actionable In a preclinical study, WANT3 treatment resulted in suppression of cell invasion of colon cancer cells in culture (PMID: 27432794). 27432794
Unknown unknown gastroesophageal junction adenocarcinoma no benefit Ramucirumab + Oxaliplatin + TS-1 Phase II Actionable In a Phase II trial, adding Cyramza (ramucirumab) to chemotherapy consisting of TS-1 and Eloxatin (oxaliplatin) did not improve progression-free survival (6.34 vs 6.74 months), objective response rate (58.2% vs 50.%), and disease control rate (90.9% vs 87.0%) compared to placebo in patients with advanced gastric or gastroesophageal junction adenocarcinoma (J Clin Oncol 36, 2018 (suppl; abstr 4036); NCT02539225). detail...
Unknown unknown multiple myeloma not applicable Pomalidomide + ACY-241 Preclinical - Cell line xenograft Actionable In a preclinical study, ACY-241, in combination with pomalidomide, demonstrated safety and prolonged survival in multiple myeloma cell line xenograft models (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 5380). detail...
Unknown unknown triple-receptor negative breast cancer not applicable YLL545 Preclinical - Cell line xenograft Actionable In a preclinical study, YLL545 inhibited proliferation of a human triple-negative breast cancer (TNBC) cell line in culture, and inhibited activation of STAT3 and ERK and decreased tumor growth in TNBC cell line xenograft models (PMID: 27203384). 27203384
Unknown unknown renal cell carcinoma not applicable Itraconazole Preclinical - Cell culture Actionable In a preclinical study, Itraconazole resulted in antiangiogenic activity and inhibited cell proliferation of renal carcinoma cells in culture (PMID: 22025615). 22025615
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 colorectal cancer not applicable TAK-960 Preclinical - Cell line xenograft Actionable In a preclinical study, colorectal cancer cells treated with TAK-960 demonstrated cell growth inhibition and decreased tumor size in both culture and cell line xenograft models (PMID: 22188812). 22188812
Unknown unknown 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 acute myeloid leukemia not applicable Cytarabine + Daunorubicin + Quizartinib Phase I Actionable In a Phase I trial (QuANTUM-First), the combination therapy, Quizartinib (AC220) with Cytosar-U (cytarabine) and Cerubidine (daunorubicin), resulted in a response in 84% (16/19) of patients with acute myeloid leukemia, including fourteen patients with a composite complete response and two patients achieving morphologic leukemia-free state (PMID: 29139135; NCT01390337). 29139135
Unknown unknown triple-receptor negative breast cancer not applicable G-TPP + Navitoclax Preclinical - Cell line xenograft Actionable In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Navitoclax (ABT-263) synergistically inhibited viability of triple-receptor negative breast cancer cells in culture, and inhibited tumor growth in cell line xenograft models (PMID: 28522750). 28522750
Unknown unknown 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 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 neuroendocrine tumor not applicable Octreotide acetate + Cixutumumab + Everolimus Phase I Actionable In a Phase I trial, patients with neuroendocrine tumors treated with the combination of Cixutumumab, Sandostatin Lar Depot (octreotide acetate), and Afinitor (everolimus) demonstrated some efficacy, however, the drug combination did result in multiple non-dose limiting toxicities preventing long term tolerance (PMID: 25900182). 25900182
Unknown unknown neuroblastoma not applicable Prexasertib Preclinical - Cell line xenograft Actionable In a preclinical study, Prexasertib (LY2606368) decreased proliferation and increased apoptosis of neuroblastoma cell lines in culture, and induced tumor regression in neuroblastoma cell line xenograft models (PMID: 28270495). 28270495
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 stomach cancer not applicable Apatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Apatinib (YN968D1) inhibited tumor growth in gastric cancer cell line xenograft models (PMID: 21443688). 21443688
Unknown unknown glioblastoma multiforme not applicable Bevacizumab + SL-701 Phase II Actionable In a Phase II trial, SL-701 and Avastin (bevacizumab) combination treatment resulted in complete response in 7.1% (2/28), partial response in 14.3% (4/28) and stable disease in 67.9.6% (19/28) of patients with relapsed/refractory glioblastoma, with a 12-month overall survival rate of 43% (median 11.7 months) (J Clin Oncol 36, 2018 (suppl; abstr 2058); NCT02078648). detail...
Unknown unknown glioblastoma multiforme not applicable Bevacizumab + SL-701 Phase II Actionable In a Phase II trial, SL-701 and Avastin (bevacizumab) combination treatment resulted in partial response in 25% (1/4) and stable disease in 75% (3/4) of patients with recurrant glioblastoma multiforme (Neuro Oncol (2016) 18 (suppl 6): vi20.). detail...
Unknown unknown colon cancer not applicable Valproic Acid + Mitomycin C Preclinical Actionable In a preclinical study, Depakene (valproic acid), in combination with Mitozytrex (mitomycin C), inhibited cell viability of colon cancer cells isolated from patients (PMID: 17024798). 17024798
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 breast cancer not applicable AKI603 + Epirubicin Preclinical Actionable In a preclinical study, AKI603 synergistically increased the cytotoxic effects of Ellence (epirubicin) in breast cancer cells, resulting in a greater decreased cell survival when compared to either agent alone (PMID: 24899685). 24899685
Unknown unknown Advanced Solid Tumor not applicable Merestinib Phase I Actionable In a Phase I trial, LY2801653 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Cancer Res October 1, 2014 74:CT237). detail...
Unknown unknown 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 breast cancer sensitive ICEC0942 Preclinical - Cell line xenograft Actionable In a preclinical study, ICEC0942 induced growth arrest of breast cancer cells in culture and in cell line xenograft models (PMID: 29545334). 29545334
Unknown unknown Advanced Solid Tumor not applicable CBT-101 Phase I Actionable CBT-101 is currently in clinical trial in patients with advanced solid tumors (clinicaltrials.gov, Oct 2018). 10
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 ovarian serous carcinoma not applicable Vistusertib + Paclitaxel Phase I Actionable In a Phase I trial, the combination therapy of Taxol (paclitaxel) and Vistusertib (AZD2014) resulted in a RECIST response rate of 64% (16/25), a CA125 response rate of 52% (13/25), and a median progression-free survival of 5.8 months in patients with high grade serous ovarian cancer (PMID: 30016392; NCT02193633). 30016392
Unknown unknown clear cell sarcoma not applicable Tivantinib Phase II Actionable In a Phase II trial, tivantinib resulted in modest activity, in which 11 patients with clear cell sarcoma had a median PFS of 1.9 months (PMID: 22605650). 22605650
Unknown unknown triple-receptor negative breast cancer not applicable ASTX-660 + Paclitaxel Preclinical - Cell line xenograft Actionable In a preclinical study, combination of ASTX-660 and Taxol (paclitaxel) treatment resulted in tumro regression and achieved partial response in cell line xenograft models of triple-receptor negative breast cancer (Cancer Res 2016;76(14 Suppl):Abstract nr 1287). detail...
Unknown unknown chronic myelomonocytic leukemia not applicable TG101209 Preclinical - Patient cell culture Actionable In a preclinical study, TG101209 inhibited colony formation of granulocytes and macrophages cultured from patients with chronic myelomonocytic leukemia (PMID: 27157043). 27157043
Unknown unknown chronic lymphocytic leukemia not applicable Bendamustine + MK2206 + Rituximab Phase Ib/II Actionable In a Phase I trial, MK2206 in combination with Bendamustine and Rituximab resulted in an overall response rate of 92% (12/13), with a median progression free survival of 16 months and a treatment free survival of 24 months in patients with relapsed or refractory chronic lymphocytic leukemia (PMID: 28402581). 28402581
Unknown unknown ovarian cancer not applicable Cisplatin + TRX-E-002-1 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of TRX-E-002-1 and Platinol (cisplatin) synergized to induce cell death in human chemoresistant ovarian cancer cell lines in culture and decreased tumor burden in xenograft models (PMID: 27196760). 27196760
Unknown unknown colorectal cancer not applicable Panitumumab FDA approved Actionable In an open-label clinical trial that supported FDA approval, Vectibix (panitumumab) treatment in patients with metastatic colorectal cancer resulted in increased progression-free survival (13.8 weeks; SD=0.76) compared to best supportive care alone (8.5 weeks; SD=0.54) (PMID: 18316547). 18316547 detail...
Unknown unknown gastric adenocarcinoma no benefit Panitumumab Phase III Actionable In a Phase III trial, addition of Vectibix (panitumumab) to chemotherapy consisted of epirubicin, oxaliplatin, and capecitabine (EOC) did not improve overall survival and increased side effects, therefore was not recommended in an unselected population of patients with advanced oesophagogastric adenocarcinoma (PMID: 23594787). 23594787
Unknown unknown Hodgkin's lymphoma not applicable Brentuximab vedotin + Dacarbazine + Doxorubicin + Vinblastine FDA approved Actionable In a Phase III trial that supported FDA approval, Adcetris (brentuximab vedotin) in combination with Deticene (dacarbazine), Adriamycin (doxorubicin), and Velban (vinblastine) resulted in an improved modified progression-free survival rate of 82.1% compared to 77.2% with the combination of Adriamycin (doxorubicin), Blenoxane (Bleomycin), Velban (vinblastine) and Deticene (dacarbazine) (HR=0.77, p=0.04) in patients with untreated stage III or IV Hodgkin's lymphoma (PMID: 29224502; NCT01712490). 29224502
Unknown unknown non-small cell lung carcinoma no benefit Carboplatin + Paclitaxel + Motesanib Phase III Actionable In a Phase III trial, Motesanib (AMG 706) in combination with Taxol (paclitaxel) and Paraplatin (carboplatin) did not improve progression-free survival significantly compared to placebo (6.1 vs 5.6 months) in patient with non-squamous non-small cell lung cancer (PMID: 28902534). 28902534
Unknown unknown lymphoma not applicable Denileukin diftitox + Sirolimus Preclinical Actionable In a preclinical study, a low dose of Rapamune (sirolimus) enhanced the efficacy of Ontak (denileukin diftitox) treatment in lymphoma mouse models, resulting in greater inhibition of tumor growth and higher survival compared to either agent alone (PMID: 27737881). 27737881
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 cervical cancer not applicable BIRB-796 + Tozasertib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Tozasertib (VX-680) and BIRB-796 resulted in increased growth inhibition and induction of apoptosis in cervical cancer cell lines in culture, and increased tumor growth inhibition in cervical cancer cell line xenograft models, compared to either agent alone (PMID: 27082306). 27082306
Unknown unknown melanoma not applicable DT01 + Radiotherapy Phase I Actionable In a Phase I trial, DT01 treatment enhanced the sensitivity of radiotherapy, resulting in an objective response in 59% (45/76) of lesions from 21 melanoma patients, including 23 complete responses (PMID: 27140316; NCT01469455). 27140316
Unknown unknown diffuse large B-cell lymphoma not applicable BGB-3111 Preclinical - Cell line xenograft Actionable In a preclinical study, BGB-3111 inhibited BTK signaling and proliferation of diffuse large B-cell lymphoma (DLBCL) cell lines in culture, and demonstrated antitumor activity in a DLBCL cell line xenograft model, with improved efficacy compared to Imbruvica (ibrutinib) (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 2597). detail...
Unknown unknown 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 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 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 acute myeloid leukemia not applicable ABBV-075 Preclinical - Patient cell culture Actionable In a preclinical study, ABBV-075 induced apoptosis and inhibited growth of acute myeloid leukemia (AML) cell lines and patient-derived cells in culture, and inhibited tumor growth in an AML cell line xenograft model (PMID: 28416490). 28416490
Unknown unknown hepatocellular carcinoma not applicable Interferon alfa-2b + Fluorouracil 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 11148999
Unknown unknown acute myeloid leukemia not applicable Flotetuzumab Phase I Actionable In a Phase I trial, Flotetuzumab (MGD006) demonstrated safety and preliminary efficacy in patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome, resulted in anti-leukemic activity in 57% (8/14) of patients and an overall response rate of 43% (6/14, 3 complete response, 1 CRi, 1 MLF, 1 partial response) (Blood 2017 130(Suppl 1):637). detail...
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 Hodgkin's lymphoma not applicable Pembrolizumab FDA approved Actionable In a Phase II trial (KEYNOTE-087) that supported FDA approval, Keytrude (pembrolizumab) treatment resulted in an overall response rate of 69% (145/210) in patients with relapsed or refractory classical Hodgkin lymphoma (PMID: 28441111; NCT02453594). 28441111
Unknown unknown triple-receptor negative breast cancer not applicable CCT007093 + Paclitaxel Preclinical Actionable In a preclinical study, CCT007093 and Taxol (paclitaxel) worked synergistically to inhibit growth of triple-receptor negative breast cancer cell lines in culture (PMID: 20576088). 20576088
Unknown unknown lung cancer not applicable AZ628 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Mekinist (trametinib) and AZ628 resulted in greater inhibition of Mek and apoptosis in a non-BRAF V600 mutant lung cancer cell line in culture compared to the combination of Mekinist (trametinib) and Tafinlar (dabrafenib) (J Clin Oncol 35, 2017 (suppl; abstr e23154)). detail...
Unknown unknown Advanced Solid Tumor not applicable Crizotinib + Dasatinib Phase I Actionable In a Phase I trial, Xalkori (crizotinib) therapy, in combination with Sprycel (dasatinib), in patients with advanced solid tumors resulted in limited efficacy, including one patient with a partial response and three patients with stable disease for at least six months or more (PMID: 29047029). 29047029
Unknown unknown non-small cell lung carcinoma not applicable Vistusertib + Paclitaxel 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 colon cancer not applicable HM-3 Preclinical - Cell line xenograft Actionable In a preclinical study, a colon cancer cells treated with HM-3 demonstrated reduced cell migration in culture and inhibition of tumor growth in xenograft models (PMID: 27633584). 27633584
Unknown unknown Advanced Solid Tumor not applicable Binimetinib Phase I Actionable In a Phase I trial, Binimetinib (MEK162) treatment resulted in complete response in 1% (1/91), partial response in 2% (2/91), and stable disease with median duration of 3.94 months in 36% (33/91) of patients with advanced solid tumors (PMID: 28152546). 28152546
Unknown unknown Hodgkin's lymphoma not applicable INCB040093 Phase I Actionable In a Phase I trial, INCB040093 treatment resulted in complete response in 17% (1/6) of Hodgkin's lymphoma patients, with an objective response rate of 50% (J Clin Oncol 33, 2015 (suppl; abstr 8558)). detail...
Unknown unknown acute myeloid leukemia not applicable BI 836858 + Decitabine Preclinical - Patient cell culture Actionable In a preclinical study, treatment of patient derived acute myeloid leukemia blast cells with Dacogen (decitabine) enhanced BI 836858-mediated cytotoxic immune response in culture (PMID: 27013443). 27013443
Unknown unknown Her2-receptor positive breast cancer not applicable BMS-906024 Preclinical Actionable In a preclinical study, the pan-Notch inhibitor, BMS-906024, inhibited growth of ERBB2 (HER2)-positive breast cancer xenografts (PMID: 26005526). 26005526
Unknown unknown Advanced Solid Tumor not applicable XL999 Phase II Actionable In six Phase II clinical trials, XL999 demonstrated anti-tumor activity in patients with advanced solid tumors, but also resulted in significant cardiotoxicity, which improved after discontinuation of the drug (J Clin Oncol, 25:18s, 2007 (Suppl; abstr 3591)). detail...
Unknown unknown 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 colon carcinoma not applicable Mps-BAY1 Preclinical Actionable In a preclinical study, Mps-BAY1 inhibited cell cycle progression and induced cell death of colon carcinoma cells in culture (PMID: 23933817). 23933817
Unknown unknown 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 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 mantle cell lymphoma sensitive STRO-001 Preclinical - Cell culture Actionable In a preclinical study, STRO-001 potently inhibited growth of mantle cell lymphoma cell lines in culture (Blood 2016 128 (22):464). detail...
Unknown unknown brain stem glioma not applicable Olaparib Preclinical - Cell culture Actionable In a preclinical study, Lynparza (olaparib) resulted in decreased cell viability of some diffuse intrinsic pontine glioma cell lines in culture (PMID: 26351319). 26351319
Unknown unknown multiple myeloma not applicable DT204 + Bortezomib 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 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 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 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 Advanced Solid Tumor not applicable TVB-2640 Phase I Actionable In a Phase I trial, TVB-2640 demonstrated bioavailability and inhibited FASN-dependent signaling in the tumor tissue of one patient with advanced solid tumor (Cancer Res August 1, 2015 75; 2675). detail...
Unknown unknown Advanced Solid Tumor not applicable SAR566658 Phase I Actionable In a phase I trial, SAR566658 treatment resulted in complete response in 1% (1/114), partial response in 7% (8/114), and stable disease in 39% (44/114) of patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2511)). detail...
Unknown unknown Advanced Solid Tumor not applicable VS-5584 Preclinical - Cell line xenograft Actionable In a preclinical study, VS-5584 inhibited growth of a variety of human tumor cell lines in culture and inhibited tumor growth in cell line xenograft models (PMID: 23270925). 23270925
Unknown unknown ovarian cancer not applicable Ad5CMV-p53 gene Phase I Actionable In a Phase I trial, Ad5CMV-p53 gene therapy demonstrated safety and preliminary efficacy, resulting in a mixed response in 12% (2/17) and stable disease in 24% (4/17) of patients with platinum- and paclitaxel-resistant metastatic epithelial ovarian cancer (PMID: 15297186). 15297186
Unknown unknown head and neck squamous cell carcinoma not applicable Nivolumab FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Opdivo (nivolumab) resulted in a 1 year overall survival rate of 36%, compared to 18% with standard therapy, and an improved median overall survival of 7.5 months, compared to 5.1 months with standard therapy, in patients with recurrent head and neck squamous cell carcinoma (PMID: 27718784). 27718784
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 non-small cell lung carcinoma not applicable LY3164530 Preclinical - Cell line xenograft Actionable In a preclinical study, LY3164530 demonstrated anti-tumor activity in cell line xenograft models of non-small cell lung carcinoma (AACR; Cancer Res 2016;76(14 Suppl):Abstract nr 873). detail...
Unknown unknown Advanced Solid Tumor not applicable PF-562271 Preclinical - Cell line xenograft Actionable In a preclinical study, PF-562271 inhibited PTK2 signaling and growth of multiple tumor cell types in culture and in cell line xenograft models (PMID: 18339875). 18339875
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 stomach carcinoma not applicable Tivantinib Phase II Actionable In a Phase II trial, Tivantinib (ARQ197) demonstrated efficacy in previously treated patients with metastatic gastric cancer (PMID: 24337769). 24337769
Unknown unknown oral squamous cell carcinoma not applicable Mithramycin Preclinical - Cell line xenograft Actionable In a preclinical study, Mithracin (plicamycin) inhibited cell growth and induced apoptosis in human oral squamous cell carcinoma cell lines in culture, and inhibited tumor growth and induced apoptosis in a human oral squamous cell carcinoma cell line xenograft model (PMID: 23019217). 23019217
Unknown unknown biliary tract cancer not applicable Binimetinib Phase I Actionable In a Phase I trial, Binimetinib (MEK162) treatment resulted in complete response in 3.3% (1/30) and partial response in 6.7% (2/30) of patients with biliary tract cancer, with estimated progression free survival of 2.1 months and overall survival of 4.8 months (PMID: 28152546). 28152546
Unknown unknown Advanced Solid Tumor not applicable Selumetinib Clinical Study Actionable In a meta-analysis, Selumetinib (AZD6244) was shown to have modest clinical efficacy as a monotherapy in a variety of solid tumors (PMID: 24716986). 24716986
Unknown unknown Advanced Solid Tumor not applicable Selumetinib Phase I Actionable In a Phase I trial, treatment with Selumetinib (AZD6244) demonstrated safety and preliminary efficacy patients with advanced solid tumors, with several patients achieving prolonged stable disease (PMID: 18390968). 18390968
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 esophageal cancer not applicable Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) improved progression free survival compared to placebo in patients with refractory advanced oesophagogastric cancer (J Clin Oncol 33, 2015 (suppl; abstr 4003)). detail...
Unknown unknown primary mediastinal B-cell lymphoma not applicable Pembrolizumab FDA approved Actionable In a Phase II trial (KEYNOTE-170) that supported FDA approval, Keytruda (pembrolizumab) treatment resulted in an objective response rate of 45% (24/53) in patients with primary mediastinal large B-cell lymphoma, with a complete response rate of 13% (7/53), and a median progression-free survival of 5.5 months (ASH Annual Meeting Dec 2018, Session 626, abstract 228; NCT02576990). detail... detail...
Unknown unknown colon cancer not applicable AMG 900 Preclinical - Cell line xenograft Actionable In a preclinical study, AMG 900 inhibited growth of colon cancer cell lines in culture, and inhibited tumor growth in colon cancer cell line xenograft models (PMID: 20935223). 20935223
Unknown unknown 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 glioblastoma multiforme not applicable CC-115 Preclinical - Pdx Actionable In a preclinical study, CC-115 increased survival of patient derived xenograft models of glioblastoma (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 1755). detail...
Unknown unknown Advanced Solid Tumor not applicable GSK1120212 + GSK2141795 Phase I Actionable In a Phase I trial, the combination of Trametinib (GSK1120212) and GSK2141795 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol (Meeting Abstracts) 2011 29: 3085). detail...
Unknown unknown non-Hodgkin lymphoma not applicable A-485 Preclinical - Cell culture Actionable In a preclinical study, A-485 treatment inhibited proliferation in non-Hodgkin lymphoma cell lines in culture (PMID: 28953875). 28953875
Unknown unknown lung small cell carcinoma not applicable BAY1000394 Phase I Actionable In a Phase I trial, treatment with Roniciclib (BAY 1000394) at the RP2D reduced PCNA expression and resulted in a disease control rate of 17.4% (n=23) in patients with small cell lung cancer (PMID: 28463960; NCT01188252). 28463960
Unknown unknown prostate cancer not applicable AZD3514 Phase I Actionable In two Phase I trials, AZD3514 treatment resulted in more than 50% decrease of PSA level in 13% (9/70) of patients with castration-resistant prostate cancer, and an objective soft tissue response in 17% (4/24) of patients in one of the trials (PMID: 25920479). 25920479
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 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 Advanced Solid Tumor not applicable PTC596 Phase I Actionable In a Phase I trial, PTC596 demonstrated safety and preliminary efficacy, resulted in stable disease in 16% (5/31) of patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2574)). detail...
Unknown unknown mantle cell lymphoma not applicable GS-5829 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, mantle cell lymphoma cell lines demonstrated greater cell growth inhibition when treated with a combination of GS-5829 and Venclexta (venetoclax) compared to either agent alone in culture (Blood 2016 128:5104). detail...
Unknown unknown 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 brain glioma not applicable Dorsomorphin Preclinical Actionable In a preclinical study, dorsomorphin (Compound C) inhibited AMPK and reduced glioma cells viability in vitro by inhibiting proliferation and inducing cell death (PMID: 24419061) 24419061
Unknown unknown gastrointestinal stromal tumor not applicable Ponatinib Phase II Actionable In a Phase II trial, Iclusig (ponatinib) demonstrated preliminary activity in patients with advanced gastrointestinal stromal tumor (J Clin Oncol (Meeting Abstracts) 2014 32: 10506). detail...
Unknown unknown Hodgkin's lymphoma not applicable Itacitinib + INCB040093 Phase I Actionable In a Phase I trial, INCB040093 and INCB039110 combination treatment resulted in complete response in 22% (2/9) of Hodgkin's lymphoma patients, with an objective response rate of 67% (J Clin Oncol 33, 2015 (suppl; abstr 8558)). detail...
Unknown unknown germ cell cancer not applicable Ipafricept Phase I Actionable In a Phase I trial, a patient with germ cell cancer demonstrated stable disease for greater than 6 months when treated with Ipafricept (OMP-54F28) (PMID: 28954784). 28954784
Unknown unknown lymphoma not applicable Abexinostat Phase II Actionable In a Phase II trial, patients with T-cell lymphoma demonstrated an overall response rate of 40% (6/15) and a median duration response of 11.5 months when treated with Abexinostat (PCI-24781) (PMID: 28126962). 28126962
Unknown unknown Ewing sarcoma not applicable Niraparib + Temozolomide Preclinical - Cell culture Actionable In a preclinical study, treatment with Temodar (temozolomide) combined with Zejula (niraparib) demonstrated strong synergism in Ewing sarcoma cells in culture, resulting in decreased cell viability (PMID: 26438158). 26438158
Unknown unknown 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 Advanced Solid Tumor sensitive Gemcitabine + Pazopanib Phase I Actionable In a Phase I study, Votrient (pazopanib) administered with Gemzar (gemcitabine) was shown to be tolerated in patients with advanced solid tumors, and resulted in one partial response (metastatic melanoma) and prolonged disease stabilization in three patients (metastatic melanoma, cholangiocarcinoma, and colorectal carcinoma) (PMID: 23064954). 23064954
Unknown unknown hepatocellular carcinoma not applicable GNS561 Preclinical - Pdx & cell culture Actionable In a preclinical study, GNS561 inhibited growth of hepatocellular carcinoma cell lines in culture, and tumor growth in patient-derive xenograft (PDX) models (Cancer Res 2017;77(13 Suppl):Abstract nr 5124). 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 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 breast cancer not applicable Paclitaxel + TVB-2640 Phase I Actionable In a Phase I trial, TVB-2640 and Taxol (paclitaxel) combination treatment resulted in partial response in 8% (1/12) and stable disease for more than 20 weeks in 25% (3/12) of breast cancer patients (J Clin Oncol 34, 2016 (suppl; abstr 2512)). detail...
Unknown unknown hepatocellular carcinoma no benefit Codrituzumab Phase II Actionable In a Phase II trial, previously treated hepatocellular carcinoma patients treated with Codrituzumab did not demonstrate a signficantly greater PFS (2.6 vs 1.5 mo) or OS (8.7 vs 10 mo) when compared to placebo (PMID: 27085251). 27085251
Unknown unknown pancreatic cancer not applicable CHIR-124 + Gemcitabine Preclinical - Cell culture Actionable In a preclinical study, CHIR-124 and Gemzar (gemcitabine) demonstrated synergistic cytoxicity in pancreatic cancer cells in spheroid culture, resulting in increased DNA damage and apoptosis and decreased viability (PMID: 22244109). 22244109
Unknown unknown breast cancer not applicable Tanibirumab Preclinical - Cell culture Actionable In a preclinical study, Tanibirumab (TTAC-0001) inhibited angiogenesis in a cell culture assay with human breast cancer cells (PMID: 26325365). 26325365
Unknown unknown Advanced Solid Tumor not applicable Axitinib + Bevacizumab Phase I Actionable In a Phase I trial, Inlyta (axitinib) in combination with Avastin (bevacizumab) demonstrated safety and efficacy in patients with advanced solid tumors including metastatic colorectal cancer (PMID: 19940012). 19940012
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 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 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 transitional cell carcinoma sensitive 4SC-202 Preclinical - Cell culture Actionable In a preclinical study, 4SC-202 inhibited proliferation and induced cell-cycle alterations and cell death in urothelial cancer cell lines in culture (PMID: 27250763). 27250763
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 ovarian cancer not applicable BKM120 + Trametinib Phase Ib/II Actionable In a Phase Ib trial, ovarian cancer patients demonstrated an objective response rate of 21% (6/21), including 1 complete response and 5 partial responses, to treatment with Buparlisib (BKM120) in combination with Mekinist (trametinib) (PMID: 25500057) 25500057
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 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 head and neck squamous cell carcinoma not applicable Abemaciclib + Everolimus Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Abemaciclib (LY2835219) and Afinitor (everolimus) worked synergistically to reduce viability of head and neck squamous cell carcinoma (HNSCC) cells in culture, and to inhibit tumor growth in HNSCC cell line xenograft models, with increased efficacy over either agent alone (PMID: 26909611). 26909611
Unknown unknown hepatocellular carcinoma not applicable Fluorouracil + RU-A1 Preclinical - Cell culture Actionable In a preclinical study, RU-A1 enhanced the sensitivity of Adrucil (fluorouracil) treatment in hepatocellular carcinoma cells, demonstrating greater decreased cell survival and inhibition of colony formation in culture compared to treatment with either agent alone (PMID: 28589491). 28589491
Unknown unknown Advanced Solid Tumor not applicable Temozolomide + TRC102 Phase I Actionable In a Phase I trial, the combination of Temodar (temozolomide) and TRC102 demonstrated safety and preliminary activity in a patients with advanced solid tumors, with 4/37 patients demonstrating partial response and 11/37 patients achieving stable disease (J Clin Oncol 34, 2016 (suppl; abstr 2556)). detail...
Unknown unknown 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 renal carcinoma not applicable MRx0518 Preclinical Actionable In a preclinical study, MRx0518 stimulated immune response and reduced tumor size in syngeneic mouse models of renal carcinoma (Journal of Clinical Oncology 36, no. 15_suppl). detail...
Unknown unknown chronic lymphocytic leukemia not applicable Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in partial response in 67% (6/9) and stable disease in 22% (2/9) of patients with chronic lymphocytic leukemia (PMID: 24852792). 24852792
Unknown unknown natural killer cell leukemia not applicable Tagraxofusp-erzs FDA approved Actionable In a Phase I/II trial that supported FDA approval, Elzonris (tagraxofusp-erzs) treatment resulted in complete response/clinical complete response in 53.8% (7/13) of patients with untreated blastic plasmacytoid dendritic cell neoplasm (BPDCN, also known as natural killer cell leukemia/lymphoma) with a median follow-up of 11.5 months, and 1 complete response and 1 clinical complete response in 15 patients with relapsed or refractory BPDCN (FDA.gov; NCT02113982). detail...
Unknown unknown non-small cell lung carcinoma not applicable BAY1217389 + Paclitaxel Preclinical Actionable In a preclinical study, the combination of BAY1217389 and Taxol (paclitaxel) resulted in enhanced growth inhibition compared to Taxol (paclitaxel) alone in non-small cell lung cancer xenograft models (PMID: 26832791). 26832791
Unknown unknown Advanced Solid Tumor not applicable varlitinib Phase I Actionable In a Phase I clinical trial, Varlitinib (ARRY-334543) demonstrated safety and some preliminary anti-tumor activity in patients with advanced solid tumors (Mol Cancer Ther 2009;8(12 Suppl):B54). 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 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 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 Advanced Solid Tumor not applicable Bosutinib Phase I Actionable In a Phase I trial, Bosulif (bosutinib) demonstrated safety and limited efficacy in patients with advanced solid tumors (PMID: 22179664). 22179664
Unknown unknown hepatocellular carcinoma no benefit Lenalidomide + Sorafenib Phase I Actionable In a Phase I clinical trial, the combination of Revlimid (lenalidomide) and Nexavar (sorafenib) was not well-tolerated and did not demonstrate clinical activity in patients with hepatocellular carcinoma, and the study was terminated early due to toxicity (PMID: 27256874). 27256874
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 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 colorectal adenocarcinoma not applicable VX-970 + Irinotecan Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of VX-970 and Camptosar (irinotecan) synergized to inhibit proliferation of a colorectal adenocarcinoma cell line in culture and to inhibit tumor growth in a human colorectal adenocarcinoma cell line xenograft model (PMID: 25269479). 25269479
Unknown unknown clear cell renal cell carcinoma no benefit girentuximab Phase III Actionable In a Phase III trial, treatment with Girentuximab did not result in a greater disease free survival or overall survival compared to placebo in patients with high risk renal clear cell carcinoma (PMID: 27787547). 27787547
Unknown unknown renal cell carcinoma not applicable Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) resulted in an improved median progression-free survival of 167 days in patients with renal cell carcinoma (PMID: 17189398). detail... 17189398
Unknown unknown triple-receptor negative breast cancer not applicable Docetaxel + S63845 Preclinical - Pdx Actionable In a preclinical study, S63845 and Taxotere (docetaxel) demonstrated synergy in triple-negative breast cancer patient-derived xenograft models, resulting in enhanced tumor growth inhibition and overall survival compared to either agent alone (PMID: 28768804). 28768804
Unknown unknown 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 breast cancer not applicable JNJ-7706621 Preclinical - Cell culture Actionable In a preclinical study, JNJ-7706621 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 gastrointestinal system cancer not applicable Cetuximab + ECF Phase II Actionable In a Phase II trial, Erbitux (cetuximab) in combination with ECF (epirubicin, cisplatin, and fluorouracil) resulted in an overall response rate of 60.9% (38/63), median overall survival of 11.6 months; and median progression-free survival of 7.1 months in patients with metastatic esophageal or gastroesophageal junction cancers (PMID: 27382098). 27382098
Unknown unknown acute myeloid leukemia not applicable Erlotinib + Ethacridine Preclinical - Cell line xenograft Actionable In a preclinical study, Tarceva (erlotinib) and the PARG inhibitor ethacridine demonstrated synergy in decreasing viability of acute myeloid leukemia (AML) cell lines and primary samples in culture, and reduced tumor growth in AML cell line xenograft models (PMID: 27587383). 27587383
Unknown unknown glioblastoma multiforme not applicable Regorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Stivarga, (regorafenib), inhibited tumor growth in cell line xenograft models of glioblastoma multiforme (PMID: 21170960). 21170960
Unknown unknown head and neck squamous cell carcinoma not applicable Abemaciclib + Torin2 Preclinical Actionable In a preclinical study, the combination of Abemaciclib (LY2835219) and Torin2 worked synergistically to reduce viability of head and neck squamous cell carcinoma cells in culture (PMID: 26909611). 26909611
Unknown unknown ovarian cancer not applicable TRX-E-002-1 Preclinical - Cell line xenograft Actionable In a preclinical study, TRX-E-002-1 inhibited proliferation and induced apoptosis in chemoresistant human ovarian cancer cell lines in culture, and reduced tumor size in ovarian cancer cell line xenograft models (PMID: 27196760). 27196760
Unknown unknown 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 renal cell carcinoma not applicable sonepcizumab Phase II Actionable In a Phase II trial, ASONEP (sonepcizumab) treatment resulted in a median overall survival of 21.7 months and partial response in 10% (4/40) of renal cell carcinoma patients (PMID: 27727447). 27727447
Unknown unknown Advanced Solid Tumor not applicable Marizomib Phase I Actionable In a Phase I trial, Marizomib (NPI-0052) treatment resulted in stable disease of short duration in 29% (7/24) of patients with advanced solid tumors (PMID: 27117181). 27117181
Unknown unknown acute lymphocytic leukemia not applicable TAS4464 Preclinical - Cell line xenograft Actionable In a preclinical study, TAS4464 resulted in complete tumor regression in cell line xenograft models of acute lymphocytic leukemia (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr C176). detail...
Unknown unknown alveolar soft part sarcoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) resulted in an overall response rate of 46% (6/13) and a disease progression free rate at 12 weeks of 77% (10/13) in patients with alveolar soft part sarcoma (J Clin Oncol 34, 2016 (suppl; abstr 11005)). detail...
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 thyroid cancer not applicable SL327 + Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of SL327 and Sutent (sunitinib) worked additively to decrease viability, induce apoptosis, and decrease migration of Taxotere (docetaxel)-resistant anaplastic thyroid cancer cell lines in culture, and to inhibit tumor growth in xenograft models (PMID: 28178630) 28178630
Unknown unknown non-small cell lung carcinoma not applicable CCT244747 + Gemcitabine Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of CCT244747 and Gemzar (gemcitabine) slowed tumor growth in non-small cell lung cancer cell line xenograft models (PMID: 22929806). 22929806
Unknown unknown Hodgkin's lymphoma not applicable Ixazomib Preclinical Actionable In a preclinical study, Ixazomib (MLN9708) inhibited survival and induced apoptosis in Hodgkin's lymphoma cell lines in culture, and reduced tumor volume in xenograft models (PMID: 26988986). 26988986
Unknown unknown Advanced Solid Tumor not applicable Temsirolimus + Neratinib Phase I Actionable In a Phase I study, Nerlynx (neratinib) administered with Torisel (temsirolimus) was tolerable and demonstrated antitumor activity in multiple solid tumor types, including breast cancer and NSCLC (PMID: 24323026). 24323026
Unknown unknown 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 AT9283 Phase I Actionable In a Phase I clinical trial, AT9283 demonstrated safety and efficacy in patients with advanced solid tumors (PMID: 22015452). 22015452
Unknown unknown rhabdomyosarcoma not applicable YM155 + Cisplatin Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of YM155 and Platinol (cisplatin) inhibited tumor growth in a human rhabdomyosarcoma cell line xenograft model (PMID: 26983495). 26983495
Unknown unknown Advanced Solid Tumor not applicable PX-866 + Sorafenib Preclinical Actionable In a preclinical study, treatment with the combination of Stivarga (regorafenib) PK-866 resulted in increased cell death in a variety of solid tumor cell lines in culture (PMID: 23877009). 23877009
Unknown unknown liposarcoma no benefit Regorafenib Phase I Actionable In a Phase II trial, Stivarga (regorafenib) treatment did not result in significant difference in median progression-free survival (1.1 vs 1.7 months) or overall survival (HR=1.57, p=0.21) compared to placebo in patients with liposarcoma (PMID: 27751846). 27751846
Unknown unknown sarcoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) resulted in partial response in 14% (22/154) of patients with soft tissue sarcomas, with a median progression free survival of 5.63 months, and a disease progression free rate at 12 weeks of 57% (J Clin Oncol 34, 2016 (suppl; abstr 11005)). detail...
Unknown unknown stomach cancer not applicable Nivolumab Phase Ib/II Actionable In a Phase I/II trial, Opdivo (nivolumab), alone or incombination with Yervoy (ipilimumab), demonstrated safety and efficacy in patients with chemotherapy-refractory gastric cancer, resulted in a disease control rate of 38% (61/160) (J Clin Oncol 34, 2016 (suppl; abstr 4010)). detail...
Unknown unknown Advanced Solid Tumor not applicable Pazopanib + Paclitaxel Phase I Actionable In a Phase I trial, the combination of Votrient (pazopanib) and Taxol (paclitaxel) demonstrated safety and resulted in partial response in 36% (10/28) and stable disease in 36% (10/28) of patients with advanced solid tumors (PMID: 25504632). 25504632
Unknown unknown lymphoplasmacytic lymphoma not applicable VLX1570 Preclinical - Cell line xenograft Actionable In a preclinical study, VLX1570 induced apoptosis in Waldenstrom macroglobulinemia (WM) cell lines, including Velcade (bortezomib)-resistant cell lines, and primary WM cells in culture, and reduced tumor growth and increased survival in WM cell line xenograft models (PMID: 27813535). 27813535
Unknown unknown colorectal cancer no benefit ABT-751 + Bevacizumab + Capecitabine + Irinotecan Phase I Actionable In a Phase I clinical trial, the combination of ABT-751, Avastin (bevacizumab), Camptosar (irinotecan), and Xeloda (capecitabine) had modest efficacy, coupled with multiple dose limiting toxicities in colorectal cancer and will not be explored further (PMID: 26632033). 26632033
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 hematologic cancer not applicable ABT-348 Phase I Actionable In a Phase I trial, ABT-348 (Ilorasertib) demonstrated safety and preliminary efficacy in patients with various hematological malignancies (PMID: 25933833). 25933833
Unknown unknown 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 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 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 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 prostate cancer not applicable JNJ-54302833 Preclinical Actionable In a preclinical study, JNJ-54302833 inhibited growth of prostate cancer cells in culture ( Cancer Res October 1, 2014 74; 4747 ). detail...
Unknown unknown sarcoma not applicable Doxorubicin + Nilotinib Phase I Actionable In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin demonstrated safety and preliminary efficacy, resulted in 1 partial response and 9 stable disease in 13 patients with sarcomas (PMID: 30037815; NCT02587169). 30037815
Unknown unknown chronic lymphocytic leukemia sensitive Milatuzumab Phase Ib/II Actionable In a Phase Ib/II trial, Milatuzumab (hLL1) treatment of elderly and refractory chronic lymphocytic leukemia patients resulted in one patient (12.5%, 1/8) with an ongoing response at 18-months (PMID: 28466956). 28466956
Unknown unknown acute monocytic leukemia not applicable Carfilzomib + EDO-S101 Preclinical - Cell culture Actionable In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of an acute monocytic leukemia cell line in culture (PMID: 28753594). 28753594
Unknown unknown pancreatic carcinoma not applicable EMD 1214063 Preclinical - Cell line xenograft Actionable In a preclinical study, EMD 1214063 induced tumor regression in mouse cell line xenograft models of pancreatic carcinoma (PMID: 23553846). 23553846
Unknown unknown head and neck squamous cell carcinoma not applicable JPH203 + Metformin Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of JPH203 (KYT-0353) and Glucophage (metformin) decreased proliferation of head and neck squamous cell cancer cell lines in culture, and reduced tumor growth in a head and neck squamous cell cancer cell line xenograft model (PMID: 27262901). 27262901
Unknown unknown immune system cancer not applicable Afuresertib Phase II Actionable In a Phase II trial, treatment with Afuresertib (GSK2110183) in patients with Langerhans cell histiocytosis resulted in an overall response rate of 33% in treatment-naive patients and 28% in patients with refractory disease (PMID: 27804235). 27804235
Unknown unknown 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 head and neck squamous cell carcinoma not applicable Cetuximab + Carboplatin + Fluorouracil FDA approved Actionable In a Phase III clinical trial that supported FDA approval, Erbitux (cetuximab) in combination with platinum-based chemotherapy (Platinol (cisplatin) or Paraplatin (carboplatin)) and Adrucil (fluorouracil) resulted in a median overall survival of 10.1 months, versus 7.4 months with chemotherapy alone, in patients with recurrent or metastatic head and neck squamous cell carcinoma (PMID: 23576486). 23576486
Unknown unknown Advanced Solid Tumor not applicable BGB-A317 + Pamiparib Phase Ib/II Actionable In a Phase Ib trial, the combination therapy of BGB-A317 and BGB-290 in patients with advanced solid tumors resulted in 7 patients with a partial response, one patient with a complete response, and 6 patients experiencing stable disease for greater than 6 months (J Clin Oncol 35, 2017 (suppl; abstr 3013)). detail...
Unknown unknown 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 ORY-1001 Preclinical - Cell line xenograft Actionable In a preclinical study, ORY-1001 decreased colony forming ability in several acute myeloid leukemia (AML) cell lines and primary AML samples in culture, and reduced tumor growth in AML cell line xenograft model (PMID: 29502954). 29502954
Unknown unknown 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 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 triple-receptor negative breast cancer not applicable AZD5363 + 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 renal 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 a renal cell carcinoma cell line in culture, leading to decreased proliferation (PMID: 28619751). 28619751
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 non-Hodgkin lymphoma not applicable Entospletinib + Idelalisib Phase II Actionable In a Phase II trial, Zydelig (idelalisib) and Entospletinib combination treatment resulted in objective response in 36% of patients with non-Hodgkin lymphoma, but was terminated due to severe treatment-emergent pneumonitis (PMID: 26968534). 26968534
Unknown unknown gastroesophageal junction adenocarcinoma 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 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 non-small cell lung carcinoma not applicable Temsirolimus Phase I Actionable In a Phase I trial, Torisel (temsirolimus) in combination with radiation therapy demonstrated safety and efficacy in 5/8 NSCLC patients (PMID: 24373609). 24373609
Unknown unknown 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 cholangiocarcinoma not applicable Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in a median progression free survival of 1.8 months, and a median overall survival of 5.2 months in patients with advanced cholangiocarcinoma, but also induced grade 3/4 adverse events in 89% (17/19) of the patients (PMID: 28192597). 28192597
Unknown unknown B-cell lymphoma not applicable Fenretinide Phase I Actionable In a Phase I trial, Fenretinide treatment resulted in stable disease in a patient with B-cell lymphoma (PMID: 28420721). 28420721
Unknown unknown triple-receptor negative breast cancer not applicable Docetaxel + ONC201 Preclinical - Cell culture Actionable In a preclinical study, the combination of ONC201 and Docefrez (docetaxel) worked synergistically to inhibit viability of triple-negative breast cancer cell lines in culture (PMID: 28424227). 28424227
Unknown unknown Advanced Solid Tumor not applicable RO5520985 Phase I Actionable In a Phase I trial, Vanucizumab (RO5520985) treatment in advanced solid tumor patients resulted in some preliminary antitumor activity including a partial response in two patients and stable disease in nineteen patients (PMID: 29217526; NCT01688206). 29217526
Unknown unknown acute lymphocytic leukemia no benefit BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in no change in event-free survival in 100% (7/7) of cell ine xenograft models of acute lymphocytic leukemia (PMID: 21298745). 21298745
Unknown unknown 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 non-small cell lung carcinoma not applicable Vantictumab Preclinical - Pdx Actionable In a preclinical study, Vantictumab (OMP-18R5) inhibited tumor growth in 7/8 patient-derived xenograft models of non-small cell lung cancer (PMID: 22753465). 22753465
Unknown unknown multiple myeloma not applicable Bortezomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, the combination of Velcade (bortezomib) and EDO-S101 induced cell-cycle arrest and apoptosis, and worked synergistically to decrease viability of multiple myeloma cell lines in culture (PMID: 28753594). 28753594
Unknown unknown osteosarcoma not applicable AZD7762 + Hydroxyurea + VE-821 Preclinical Actionable In a preclinical study, the combination of AZD7762 and VE-821 enhanced the chemotherapeutic effects of Droxia (hydroxyurea) resulting in both increased nuclear fragmentation and apoptosis of osteosarcoma cells in culture (PMID: 26748709). 26748709
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 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 head and neck squamous cell carcinoma not applicable Pazopanib + Cetuximab Phase Ib/II Actionable In a Phase Ib clinical trial, combined therapy, Votrient (pazopanib) and Erbitux (cetuximab), was well tolerated in head and neck squamous cell carcinoma patients with metastatic or resistant disease, and resulted in a 6% (2/31) complete response rate, a 29% (9/31) partial response rate, a median time to progression of 5.5 months, and a median overall survival of 9.5 months (PMID: 30001987; NCT01716416). 30001987
Unknown unknown 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 hepatocellular carcinoma not applicable KU-0063794 Preclinical - Cell line xenograft Actionable In a preclinical study, KU-0063794 inhibited cell survival and reduced colony formation of hepatocellular carcinoma cells in culture, and inhibited tumor growth in cell line xenograft models (PMID: 26278819). 26278819
Unknown unknown Advanced Solid Tumor not applicable Navitoclax Preclinical - Cell culture Actionable In a preclinical study, Navitoclax (ABT-263) treatment alone was not effective in a number of cell lines derived from solid tumors in culture (PMID: 27974663). 27974663
Unknown unknown 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 Indication other than cancer not applicable Metformin FDA approved Actionable Glucophage (metformin) is FDA approved for use in patients with type-2 diabetes (FDA.gov). detail...
Unknown unknown non-small cell lung carcinoma not applicable Sirolimus + SBI-0206965 Preclinical - Cell culture Actionable In a preclinical study, the addition of SBI-0206965 to treatment with Rapamune (sirolimus) resulted in increased apoptosis in a non-small cell lung cancer cell line in culture (PMID: 26118643). 26118643
Unknown unknown colon cancer not applicable CVX-060 Preclinical - Cell line xenograft Actionable In a preclinical study, CVX-060 inhibited tumor growth in a colon cancer cell line xenograft model (PMID: 21233403). 21233403
Unknown unknown central nervous system lymphoma not applicable Ibrutinib Phase I Actionable In a Phase I trial, Imbruvica (ibrutinib) treatment resulted in antitumor efficacy in 77% (10/13) of patients with primary central nervous system lymphoma, demonstrating a complete response in five patients and a partial response in five patients (PMID: 28619981). 28619981
Unknown unknown non-small cell lung carcinoma not applicable Axitinib Phase II Actionable In a Phase II clinical trial, Inlyta (axitinib) was well-tolerated and demonstrated activity in patients with advanced non-small cell lung cancer, with a disease control rate of 41% (13/32), median progression-free survival of 4.9 months, and median overall survival of 14.8 months (PMID: 19597027). 19597027
Unknown unknown 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 Advanced Solid Tumor not applicable SR9011 Preclinical - Cell culture Actionable In a preclinicl study SR9011 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 colon cancer not applicable AR-42 Preclinical - Cell line xenograft Actionable In a preclinical study, AR-42 induced cell death in primary human colon cancer cells and cell lines in culture, and inhibited tumor growth in colon cancer cell line xenograft models (PMID: 26026677). 26026677
Unknown unknown pancreatic cancer not applicable Foretinib Preclinical Actionable In a preclinical study, Foretinib (GSK1363089) treatment resulted in regression of the tumor vasculature, extensive hypoxia, apoptosis, and decreased tumor aggressiveness in a transgenic mouse model of pancreatic islet cancer (PMID: 21613405). 21613405
Unknown unknown colorectal cancer no benefit Utomilumab Phase I Actionable In a Phase I trial, Utomilumab (PF-05082566) treatment resulted in no overall objective response (0/12) in patients with colorectal cancer (PMID: 29549159; NCT01307267). 29549159
Unknown unknown 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 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 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 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 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 triple-receptor negative breast cancer not applicable Cyclophosphamide + Doxorubicin + Elenagen Phase Ib/II Actionable In a Phase Ib/II trial, chemorefractory patients with triple-receptor negative breast cancer demonstrated restored chemotherapeutic sensitivity upon sequential treatment of Elenagen and the combined therapy, Cytoxan (cyclophosphamide) and Adriamycin (doxorubicin), which resulted in stable disease (PMID: 28881846). 28881846
Unknown unknown diffuse large B-cell lymphoma not applicable LAM-002A Phase I Actionable In a Phase I trial, LAM-002A demonstrated safety and preliminary efficacy, resulted in systemic partial metabolic responses and decreased tumor size in 3 of 11 patients with diffuse large B-cell lymphoma (Blood 2017 130 (Suppl 1):4119). detail...
Unknown unknown Advanced Solid Tumor not applicable KX2-391 Phase I Actionable In a Phase I trial, KX2-391 demonstrated safety and preliminary efficacy, resulted in stable disease for more than 4 months in 25% (11/44) of patients with advanced solid tumors (PMID: 23361621). 23361621
Unknown unknown B-cell lymphoma not applicable Ibrutinib + Ublituximab + Umbralisib Phase I Actionable In a Phase I trial, Imbruvica (Ibrutinib), TGR01201, and Ublituximab combination treatment resulted in complete response in 14% (1/7) and partial response in 71% of patients with relapsed B-cell lymphoas within 8 weeks of treatment (J Clin Oncol 33, 2015 (suppl; abstr 8501)). detail...
Unknown unknown prostate cancer not applicable Enzalutamide FDA approved Actionable In a Phase III trial (PROSPER) that supported FDA approval, Xtandi (enzalutamide) significantly improved median metastasis-free survival (36.6 vs 14.7 months, HR=0.29, p<0.0001) compared to placebo in patients with non-metastatic castration-resistant prostate cancer (Journal of Clinical Oncology 36, no. 6_suppl (February 20 2018) 3-3; NCT020032924). detail...
Unknown unknown prostate cancer not applicable Enzalutamide FDA approved Actionable In a Phase III trial (AFFIRM) that supported FDA approval, treatment with Xtandi (enzalutamide) resulted in improved median overall survival compared to placebo (18.4 vs 13.6 months HR=0.63, p<0.001) in patients with metastatic castration-resistant prostate cancer (PMID: 22894553; NCT00974311). 22894553
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 endometrial cancer not applicable BEZ235 Preclinical Actionable In a preclinical study, BEZ235 suppressed tumor growth in endometrial xenografts (PMID: 22662154). 22662154
Unknown unknown prostate cancer not applicable Forskolin Preclinical Actionable In a preclinical study, prostate cancer cells treated with Forskolin demonstrated PP2A activation and inhibition of cell proliferation in culture (PMID: 26023836). 26023836
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 sarcoma not applicable Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (2.9 vs 1.0 months), but no difference in overall survival (HR=0.75, p=0.37) compared to placebo in patients with soft tissue sarcoma excluding liposarcoma, leiomyosarcoma, and synovial sarcoma (PMID: 27751846). 27751846
Unknown unknown prostate cancer not applicable SPC3042 + Taxol Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of SPC3042 and Taxol (paclitaxel) worked synergistically to inhibit tumor growth in prostate cancer cell line xenograft models, with increased activity over either agent alone (PMID: 18790754). 18790754
Unknown unknown peritoneal mesothelioma not applicable GDC-0980 Phase I Actionable In a Phase I trial, Apitolisib (GDC-0980) treatment resulted in tumor regression in two patients with peritoneal mesothelioma (PMID: 26787751). 26787751
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 acute myeloid leukemia not applicable Carfilzomib + EDO-S101 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 non-small cell lung carcinoma not applicable PF-03446962 Phase I Actionable In a Phase I trial, a patient with non-small cell lung carcinoma demonstrated a partial response for 308 days when treated with PF-03446962 (PMID: 26655846). 26655846
Unknown unknown 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 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 epithelioid sarcoma not applicable Vorinostat Preclinical Actionable In a preclinical study, Zolinza (vorinostat) inhibited growth and colony formation of epithelioid sarcoma cells in culture (PMID: 26396249). 26396249
Unknown unknown pancreatic ductal adenocarcinoma not applicable MN58b + Fluorouracil 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 chronic lymphocytic leukemia not applicable Ibrutinib Clinical Study Actionable In a clinical study, treatment with Imbruvica (ibrutinib) resulted in a discontinuation-free survival rate at 1 year of 73.7% (232/315) and an absolute 1 year survival rate of 83.3% (264/315) in patients with relapsed or refractory chronic lymphocytic leukemia (PMID: 27756834). 27756834
Unknown unknown chronic lymphocytic leukemia not applicable Ibrutinib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Imbruvica (ibrutinib) in chronic lymphoid leukemia (CLL) or small lymphocytic lymphoma (SLL) patients resulted in improved progression-free survival compared to treatment with Arzerra (ofatumumab) (median duration not reached vs. 8.1 months), and an overall response rate of 43% (83/195) versus 4% (8/196) with Arzerra (ofatumumab) (PMID: 24881631). 24881631
Unknown unknown chronic lymphocytic leukemia not applicable Ibrutinib Phase III Actionable In a Phase III clinical trial, Imbruvica (ibrutinib) treatment resulted in a greater progression-free survival, overall survival, and response rate when compared to chlorambucil treatment in chronic lymphocytic leukemia patients aged 65 or older (PMID: 26639149). 26639149
Unknown unknown Advanced Solid Tumor not applicable NP-004255 Preclinical - Cell culture Emerging In a preclinical study, a biochemical assay with NP-004255, a RAD52 inhibitor similar to other compounds that induced cell death in cells with BRCA2 loss, demonstrated inhibition of RAD52 binding to ssDNA, suggesting NP-004255 may be a potential therapeutic target (PMID: 27434671). 27434671
Unknown unknown multiple myeloma not applicable ABT-348 Preclinical - Cell line xenograft Actionable In a preclinical study, Ilorasertib (ABT-348) displayed efficacy in multiple myeloma cell line xenograft models (PMID: 22935731). 22935731
Unknown unknown 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 ovarian cancer not applicable Everolimus + Bevacizumab 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 oral squamous cell carcinoma not applicable Olaparib + Cisplatin Preclinical - Cell line xenograft Actionable In a preclinical study, treatment of oral squamous carcinoma cells with Platinol (cisplatin) and Lynparza (olaparib) resulted in a synergistic effect demonstrating increased apoptosis and tumor growth inhibition in culture and cell line xenograft models (PMID: 26927065). 26927065
Unknown unknown gastrointestinal system cancer not applicable Axitinib + FOLFOX 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 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 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 neuroendocrine tumor not applicable Axitinib Phase II Actionable In a Phase II clinical trial, treatment with Inlyta (axitinib) resulted in a median overall progression-free survival (PFS) of 26.7 months, a 12-month PFS rate of 74.5%, and median overall survival of 45.3 months, and led to partial response in 3% (1/30) and stable disease in 70% (21/30) of patients with neuroendocrine tumors, with some tumor shrinkage in 68% (15/22) of patients (PMID: 27080472). 27080472
Unknown unknown non-small cell lung carcinoma not applicable CEP-32496 Phase I Actionable In a Phase I clinical trial, RXDX-105 (CEP-32496) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, including 2 patients with heavily pretreated non-small cell lung cancer that achieved stable disease for greater that 6 months (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr C188). detail...
Unknown unknown Advanced Solid Tumor not applicable Refametinib + Sorafenib Phase I Actionable In a Phase I trial, the combination treatment of Refametinib (BAY86-9766) and Nexavar (sorafenib) in patients with advanced solid tumors resulted in a disease control rate of 65.8% (25/38), specifically, 2.6% (1/38) experienced a partial response and 63.2% (24/38) demonstrated stable disease (PMID: 26644411). 26644411
Unknown unknown diffuse large B-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 31% (4/13, 3 complete response, 1 partial response) in patients with relapsed/refractory diffuse large B-cell lymphoma (J Clin Oncol 36, 2018 (suppl; abstr 7520); NCT02756247). detail...
Unknown unknown Advanced Solid Tumor not applicable XL228 Phase I Actionable In a Phase I trial, XL228 treatment resulted in stable disease for 12 weeks or longer in 31% (25/80) of patients with an advanced solid tumor and a partial response in one patient (J Clin Oncol 28:15s, 2010 (suppl; abstr 3105)). detail...
Unknown unknown lymphoma not applicable Copanlisib Phase II Actionable In a Phase II trial, treatment with Aliqopa (copanlisib) in patients with indolent lymphoma resulted in an objective response rate of 59% (84/142), including a complete response in 12%, and demonstrated a median duration of response of 22.6 months and a median progression-free survival of 11.2 months (PMID: 28976790; NCT01660451). 28633365 28976790
Unknown unknown lymphoma not applicable Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in partial response in 20% (1/5) and stable disease in 40% (2/5) of patients with transformed lymphoma (PMID: 24852792). 24852792
Unknown unknown colorectal cancer not applicable Regorafenib FDA approved Actionable In a Phase III clinical trial (CORRECT) that supported FDA approval, Stivarga (regorafenib) demonstrated safety and improved overall survival compared to placebo (6.4 vs 5.0 months, HR=0.77, p=0.0052) in patients with refractory metastatic colorectal cancer (PMID: 23177514; NCT01103323). 23177514 detail...
Unknown unknown colorectal cancer not applicable Regorafenib Phase III Actionable In a Phase III trial (CONSIGN), Stivarga (regorafenib) treatment demonstrated safety profile and efficacy consistent with previous studies, median progression-free survival (PFS) was 2.7 months overall, 2.8 months in KRAS wild-type, and 2.5 months in KRAS mutant colorectal cancer patients, and with no difference in KRAS status between long and short PFS groups (PMID: 30190299; NCT01538680). 30190299
Unknown unknown colorectal cancer not applicable Olaparib + Temozolomide Preclinical - Cell line xenograft Actionable In a preclinical study, the addition of Lynparza (olaparib) to Temodar (temozolomide) resulted in greater antitumor activity than Temodar (temozolomide) alone in colorectal cancer cell line xenograft models, demonstrating decreased tumor volume (PMID: 27550455). 27550455
Unknown unknown Advanced Solid Tumor not applicable MLN1117 + Sapanisertib Preclinical Actionable In a preclinical study, the combination of Sapanisertib (MLN0128) and MLN1117 demonstrated synergistic anti-tumor activity in a variety of solid tumor xenograft models (Mol Cancer Ther 2013;12(11 Suppl):C176)). detail...
Unknown unknown lymphoma not applicable Carfilzomib + EDO-S101 Preclinical - Cell culture Actionable In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of a lymphoma cell lines in culture (PMID: 28753594). 28753594
Unknown unknown 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 non-small cell lung carcinoma not applicable Nivolumab Clinical Study Actionable In a meta-analysis, compared to Taxotere (docetaxel), treatment with check point inhibitors including Opdivo (nivolumab), Keytruda (pembrolizumab), or Tecentriq (atezolizumab), resulted in prolonged overall survival (HR=0.69, p<0.001) in non-small cell lung carcinoma patients (PMID: 29270615). 29270615
Unknown unknown non-small cell lung carcinoma not applicable Nivolumab FDA approved Actionable In a Phase III randomized trial that supported FDA approval, squamous non-small cell lung carcinoma patients treated with Opdivo (nivolumab) demonstrated a greater OS (9.2 mo vs 6.0 mo), response rate (20% vs 9%), and PFS (3.5 mo vs 2.8 mo) when compared to treatment with Taxotere (docetaxel) (PMID: 26028407). 26028407
Unknown unknown colorectal cancer not applicable Balsalazide + Parthenolide Preclinical - Cell culture Actionable In a preclinical study, the combination of Balsalazide and Parthenolide resulted in a synergistic effect, resulting in reduced cell viability and increased apoptotic activity in colorectal cancer cells in culture (PMID: 28108625). 28108625
Unknown unknown liposarcoma not applicable Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 20% (2/10) of patients with dedifferentiated liposarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). detail...
Unknown unknown papillary renal cell carcinoma not applicable Everolimus + Bevacizumab Phase II Actionable In a Phase II trial, 50% (2/4) of patients with papillary renal cell carcinoma achieved a 6 month PFS when treated with a combination of Afinitor (everolimus) and Avastin (bevacizumab) (PMID: 27601542). 27601542
Unknown unknown 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 subacute myeloid leukemia not applicable Cytarabine + Venetoclax Preclinical - Patient cell culture Actionable In a preclinical study, Venclexta (venetoclax) and Cytosar-U (cytarabine) combination treatment synergistically induced cell death in patient-derived acute myeloid leukemia cells in culture (PMID: 27103402). 27103402
Unknown unknown triple-receptor negative breast cancer not applicable Evofosfamide Preclinical Actionable In a preclinical study, TH-302 inhibited growth of triple-receptor negative breast cancer cell lines under hypoxic conditions, regardless of their BRCA1 status (PMID: 25193512). 25193512
Unknown unknown 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 triple-receptor negative breast cancer not applicable KW-2450 + Selumetinib Preclinical Actionable In a preclinical study, the combination of KW-2450 and Selumetinib worked synergistically to inhibit growth of triple-negative breast cancer cells in culture (PMID: 26443806). 26443806
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 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 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 stomach cancer not applicable Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) improved progression free survival compared to placebo in patients with refractory advanced oesophagogastric cancer (J Clin Oncol 33, 2015 (suppl; abstr 4003)). detail...
Unknown unknown 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 triple-receptor negative breast cancer not applicable Everolimus + MK-1775 Preclinical - Cell culture Actionable In a preclinical study, the combination of Afinitor (everolimus) and MK-1775 resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
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 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 melanoma not applicable Ipilimumab Phase III Actionable In a Phase III trial, adjuvant Opdivo (nivolumab) treatment resulted in improved rate of recurrence-free survival at 12-months compared to Yervoy (ipilimumab) (70.5% vs 60.8%, HR=0.65, P<0.001) in patients with resected stage III or IV melanoma (PMID: 28891423, NCT02388906). 28891423
Unknown unknown melanoma not applicable Ipilimumab FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Yervoy (ipilimumab) resulted in an improved overall survival of 10.1 months in patients with metastatic melanoma, compared to 6.4 months in those treated with gp100 peptide vaccine alone (PMID: 20525992, PMID: 21900389). 20525992 21900389
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 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 lymphocytic 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 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 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 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 Advanced Solid Tumor not applicable MK-1454 + Pembrolizumab Phase I Actionable In a Phase I trial, MK-1454 in combination with Keytruda (pembrolizumab) resulted in partial response in 24% (6/25) and a disease control rate of 48% (12/25) in patients with advanced solid tumors (Ann Oncol, Oct 2018, 29 (suppl 8), abstract LBA15; NCT03010176). detail...
Unknown unknown melanoma no benefit Cediranib Phase II Actionable In a Phase II trial, treatment with Cediranib (AZD-2171) in melanoma patients resulted in only two patients with stable disease at 6 months, no objective responses, and a short median time to progression of 3.5 months thereby demonstrating no benefit (PMID: 26841902). 26841902
Unknown unknown chronic lymphocytic leukemia not applicable Obinutuzumab + Chlorambucil FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Gazyva (obinutuzumab) plus chlorambucil resulted in an improved median progression-free survival of 23.0 months, compared to 11.1 months with chlorambucil alone, and an overall response rate of 75.9% vs. 32.1% with chlorambucil in patients with previously untreated CD20-positive chronic lymphocytic leukemia (PMID: 24824310). 24824310
Unknown unknown acute myeloid leukemia not applicable MLN4924 Phase I Actionable In a Phase I trial, treatment with MLN4924 in patients with acute myeloid leukemia resulted in two patients acheiving a complete remission, five patients with partial remission, and 62% (34/55) of patients achieving stable disease (PMID: 28157218). 28157218
Unknown unknown hematologic cancer not applicable CC-122 Phase I Actionable In a Phase I trial, Avadomide (CC-122) treatment was well tolerated in patients with hematological malignancies (n=7) and resulted in an objective response in 60% (3/5) of patients with non-Hodgkin's lymphoma, including 1 complete response in a patient with follicular lymphoma and a partial response in patients with follicular lymphoma and mantle cell lymphoma lasting more than 234 days, and stable disease in one patient with multiple myeloma lasting 932 days (PMID: 30201761; NCT01421524). 30201761
Unknown unknown lung cancer not applicable HD105 Preclinical - Cell line xenograft Actionable In a preclinical study, HD105 inhibited tumor progression in human lung cancer cell line xenograft models (PMID: 27049350). 27049350
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 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 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 epithelioid sarcoma not applicable Abexinostat Preclinical - Cell line xenograft Actionable In a preclinical study, Abexinostat (PCI-24781) inhibited growth and colony formation of epithelioid sarcoma cell lines in culture, and inhibited tumor growth in epithelioid sarcoma cell line xenograft models (PMID: 26396249). 26396249
Unknown unknown multiple myeloma not applicable BAY 1238097 Preclinical Actionable In a preclinical study, BAY 1238097 inhibited tumor growth and was well tolerated in acute myeloid leukemia and multiple myeloma models (American Association for Cancer Research; 2015 Apr 18-22; Abstract nr 3524). detail...
Unknown unknown renal cell carcinoma not applicable Pazopanib FDA approved Actionable In a Phase III trial that supported FDA approval, Votrient (pazopanib) improved progression free survival in patients with advanced renal cell carcinoma (PMID: 20100962). detail... 20100962
Unknown unknown renal cell carcinoma not applicable Pazopanib Phase III Actionable In a Phase III trial, adjuvant Votrient (pazopanib) therapy post nephrectomy did not improve disease-free survival (HR=0.862, p=0.165) compared to placebo in patients with renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 4507)). detail...
Unknown unknown 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 Advanced Solid Tumor not applicable MRX34 Phase I Actionable In a Phase I trial, MRX34 treatment in advanced solid tumor patients resulted in some preliminary efficacy, including a partial response lasting 48 weeks in one patient with hepatocellular carcinoma and four patients with stable disease (PMID: 27917453). 27917453 detail...
Unknown unknown peritoneum cancer not applicable Bevacizumab + Carboplatin + Paclitaxel FDA approved Actionable In a Phase III trial (GOG-0218) that supported FDA approval, addition of Avastin (bevacizumab) during and after Paraplatin (carboplatin) plus Taxol (paclitaxel) treatment prolonged progression-free survival (14.1 vs 10.3 months) compared to placebo in patients with previously untreated, stage III/IV epithelial ovarian, primary peritoneal, or fallopian-tube cancer (PMID: 22204724; NCT00262847). 22204724
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 stomach cancer not applicable Saracatinib Phase II Actionable In a Phase II trial, Saracatinib (AZD0530) treatment resulted in stable disease in 23.5% (4/17) of patients with gastric cancer (PMID: 21400081). 21400081
Unknown unknown Advanced Solid Tumor not applicable Doxorubicin + Safingol Phase I Actionable In a Phase I trial, the combination of Kynacyte (safingol) and Adriamycin (doxorubicin) demonstrated safety and some preliminary activity in patients with advanced solid tumors (PMID: 9815717). 9815717
Unknown unknown colorectal cancer not applicable Resminostat Preclinical - Cell line xenograft Actionable In a preclinical study, Resminostat (4SC-201) inhibited proliferation and induced cell-cycle arrest and apoptosis in colorectal cancer (CRC) cell lines and primary colon cancer cells in culture, and inhibited tumor growth in a CRC cell line xenograft model (PMID: 26831668). 26831668
Unknown unknown non-small cell lung carcinoma not applicable XL228 Phase I Actionable In a Phase I clinical trial, 41% (9/22) of patients with an advanced solid tumor experienced stable disease for at least 12 weeks and a partial response response was reported in one patient with NSCLC when treated with XL228 (J Clin Oncol 28:15s, 2010 (suppl; abstr 3105)). detail...
Unknown unknown chronic lymphocytic leukemia not applicable Bendamustine + Idelalisib + Rituximab Phase III Actionable In a Phase III trial, addition of Zydelig (idelalisib) to Bendamustine and Rituximab resulted in improved median progression-free survival (20.8 vs 11.1 months, HR=0.33, p<0.0001) compared to placebo in patients with relapsed or refractory chronic lymphocytic leukemia, although treatment associated adverse events were also increased (PMID: 28139405). 28139405
Unknown unknown 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 colon cancer not applicable Enzastaurin Preclinical - Cell line xenograft Actionable In a preclinical study, Enzastaurin (LY317615) demonstrated efficacy by suppressing proliferation of cultured cells and growth in cell line xenograft models of colon cancer (PMID: 16103100). 16103100
Unknown unknown acute myeloid leukemia not applicable Bortezomib + Midostaurin + MEC 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 follicular lymphoma not applicable Ocaratuzumab Phase I Actionable In a Phase I clinical trial, Ocaratuzumab (AME-133v) demonstrated safety and some preliminary activity in patients with follicular lymphoma, including those with the Fc-gamma-RIIIa genotype (PMID: 22223529). 22223529
Unknown unknown pancreatic cancer not applicable RX-3117 Phase Ib/II Actionable In a Phase I/II trial, RX-3117 demonstrated safety and preliminary efficacy, resulted in durable stable disease in 25% (2/8) of patients with pancreatic cancer (Journal of Clinical Oncology 35, no. 4_suppl (February 1 2017) 445-445; NCT02030067). detail...
Unknown unknown pancreatic cancer sensitive 2g8 Preclinical Actionable In a preclinical study, inhibition of stromal Tgfbr2 with the murine antibody 2G8 resulted in decreased proliferation, increased apoptosis and reduced metastasis in pancreatic tumor cell line xenograft models (PMID: 25060520). 25060520
Unknown unknown Advanced Solid Tumor not applicable BIND-014 Phase I Actionable In a Phase I clinical trial, BIND-014 treatment resulted in partial response in 10% (5/52) of patients with advanced solid tumors, and complete response in a cervical cancer patient (PMID: 26847057). 26847057
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 pancreatic ductal adenocarcinoma not applicable Reolysin + Gemcitabine Phase II Actionable In a Phase II trial, Reolysin (pelareorep) in combination with Gemzar (gemcitabine) resulted in partial response in 3% (1/34), stable disease in 68% (23/34) of patients with advanced pancreatic ductal adenocarcinoma, with a median overall survival of 10.2 months, and a 1- and 2-year survival rate of 45% and 24% respectively (PMID: 29799479). 29799479
Unknown unknown acute myeloid leukemia not applicable AKN-028 + Daunorubicin Preclinical - Cell culture Actionable In a preclinical study, the sequential treatment of Cerubidine (daunorubicin) and AKN-028 resulted in a syntergistic effect in acute myeloid leukemia cells in culture, demonstrating antileukemic activity (PMID: 22864397). 22864397
Unknown unknown ovarian cancer not applicable Ipafricept + Carboplatin + Paclitaxel Phase Ib/II Actionable In a Phase Ib clinical trial, the combination of Ipafricept (OMP-54F28) with Paraplatin (carboplatin) and Taxol (paclitaxel) was well-tolerated and resulted in complete response in 35% (6/17), partial response in 47% (8/17) and stable disease in 18% (3/17) evaluable patients with recurrent platinum-sensitive ovarian cancer (J Clin Oncol 34, 2016 (suppl; abstr 2515)). detail...
Unknown unknown chronic myeloid leukemia not applicable ST7612AA1 Preclinical Actionable In a preclinical study, ST7612AA1 inhibited proliferation of chronic myeloid leukemia cells in culture (PMID: 25671299). 25671299
Unknown unknown colon cancer 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 LY2090314 + Carboplatin + Pemetrexed Phase I Actionable In a Phase I trial, LY2090314 in combination with Alimta (pemetrexed) and Paraplatin (carboplatin) resulted in partial response in 13.5% (5/37) and stable disease in 51.4% (19/37) of patients with advanced solid tumors (PMID: 26403509; NCT01287520). 26403509
Unknown unknown 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 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 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 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 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 thymic carcinoma not applicable Selinexor Preclinical - Cell line xenograft Actionable In a preclinical study, Selinexor (KPT-330) induced cell-cycle arrest and apoptosis and inhibited growth of several thymic epithelial tumor cell lines, including thymoma and thymic carcinoma cell lines, in culture, and inhibited tumor growth in thymic carcinoma cell line xenograft models (PMID: 28819023). 28819023
Unknown unknown 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 Advanced Solid Tumor not applicable Docetaxel + TAK-243 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Taxotere (docetaxel) and TAK-243 (MLN7243) resulted in synergistic and additive effects, demonstrating anti-tumor activity in xenograft tumor models (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A164). detail...
Unknown unknown melanoma not applicable Sirtinol Preclinical Actionable In a preclinical study, Sirtinol inhibited growth of human melanoma cell lines in culture (PMID: 25486469). 25486469
Unknown unknown non-small cell lung carcinoma not applicable Atezolizumab FDA approved Actionable In a Phase III trial (OAK) that supported FDA approval, treatment with Tecentriq (atezolizumab) resulted in an improved median overall survival compared to Taxotere (docetaxel) (13.8 vs 9.6 months, HR=0.73, p=0.0003) in patients with previously treated non-small cell lung cancer (PMID: 27979383; NCT02008227). 27979383
Unknown unknown non-small cell lung carcinoma not applicable Atezolizumab Phase II Actionable In a Phase II trial (POPLAR), treatment with Tecentriq (atezolizumab) resulted in an improved median overall survival of 12.6 months compared to 9.7 months with Taxotere (docetaxel) in patients with previously treated non-small cell lung cancer, and increased PD-L1 expression level was associated with improved survival benefit (PMID: 26970723; NCT01903993). 26970723
Unknown unknown non-small cell lung carcinoma not applicable Atezolizumab Clinical Study Actionable In a meta-analysis, compared to Taxotere (docetaxel), treatment with check point inhibitors including Opdivo (nivolumab), Keytruda (pembrolizumab), or Tecentriq (atezolizumab), resulted in prolonged overall survival (HR=0.69, p<0.001) in non-small cell lung carcinoma patients (PMID: 29270615). 29270615
Unknown unknown colon cancer not applicable CFI-400936 Preclinical - Cell line xenograft Actionable In a preclinical study, CFI-400936 inhibited tumor growth in a human cell line xenograft model of colon cancer (PMID: 25043312). 25043312
Unknown unknown diffuse large B-cell lymphoma not applicable OPB-111077 Phase I Actionable In a Phase I trial, a patient with diffuse large B-cell lymphoma demonstrated a partial response when treated with OPB-111077 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B118). detail...
Unknown unknown hepatocellular carcinoma not applicable Foretinib Phase I Actionable In a Phase I trial, Foretinib (GSK1363089) demonstrated safety and activity in hepatocellular carcinoma patients, with an overall response rate by mRESCIST of 22.9% (8/35, all partial responses), a disease stabilization rate of 82.9% (29/35), and a median duration of response of 7.6 months (PMID: 27821605; NCT00920192). 27821605
Unknown unknown colon cancer not applicable CVX-060 + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of CVX-060 and Nexavar (sorafenib) resulted in increased tumor growth inhibition compared to either agent alone in a colon cancer cell line xenograft model (PMID: 21233403). 21233403
Unknown unknown multiple myeloma not applicable Bortezomib + Daratumumab + Dexamethasone FDA approved Actionable In a Phase III trial that supported FDA approval, the combination of Darzalex (daratumumab), Velcade (bortezomib), and Adexone (dexamethasone) resulted in a greater 12 month PFS (77.5% vs 29.4%) and overall response (82.9%; 199/240 vs 63.2%; 148/234) compared to Adexone (dexamethasone) and Velcade (bortezomib) alone in multiple myeloma patients (PMID: 27557302). 27557302
Unknown unknown urinary bladder cancer not applicable GSK2126458 Phase I Actionable In Phase I trial, GSK2126458 treatment was well-tolerated and resulted in a partial response and stable disease in two patients and one patient with bladder cancer, respectively (PMID: 26603258). 26603258
Unknown unknown 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 neuroendocrine tumor not applicable Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in stable disease ranging from 3-23 months in 10 patients with neuroendocrine tumors (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown non-small cell lung carcinoma not applicable TMU-35435 Preclinical - Cell line xenograft Actionable In a preclinical study, TMU-35435 induced cell-cycle arrest and apoptosis and decreased viability of non-small cell lung cancer cell lines in culture, and inhibited tumor growth in a lung cancer cell line xenograft model (PMID: 28233309). 28233309
Unknown unknown Advanced Solid Tumor not applicable Carboplatin + TAK-243 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Paraplatin (carboplatin) and TAK-243 (MLN7243) resulted in synergistic and additive effects, demonstrating anti-tumor activity in xenograft tumor models (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A164). detail...
Unknown unknown 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 anaplastic large cell lymphoma not applicable Brentuximab vedotin FDA approved Actionable In a Phase II trial 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 anaplastic large cell lymphoma (PMID: 22614995; NCT00866047). 22614995
Unknown unknown melanoma not applicable G-TPP + Obatoclax Preclinical - Cell culture Actionable In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Obatoclax (GX015-070) synergistically inhibited viability of established melanoma cells in culture (PMID: 28522750). 28522750
Unknown unknown lung squamous cell carcinoma not applicable Prexasertib Phase Ib/II Actionable In a Phase Ib trial, treatment with Prexasertib (LY2606368) resulted in a clinical benefit rate (complete response+partial response+stable disease) of 56% (9/16, all stable disease), and a median progression-free survival of 3.0 months in patients with squamous non-small cell lung cancer (PMID: 29643063; NCT0115790). 29643063
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 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 renal cell carcinoma not applicable Temsirolimus FDA approved Actionable In a Phase III randomized trial that supported FDA approval, treatment with Torisel (temsirolimus) resulted in an improved overall survival time of 11.1 months in patients with advanced renal cell carcinoma compared to 7.4 months in patients treated with IFN-alpha (PMID: 20332142). 20332142
Unknown unknown papillary thyroid carcinoma not applicable Ramucirumab Phase I Actionable In a Phase I trial, Cyramza (ramucirumab) demonstrated safety and efficacy resulting in partial response or stable disease in patients with advanced solid tumors, including papillary thyroid carcinoma (PMID: 20048182). 20048182
Unknown unknown follicular lymphoma not applicable Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 20% (2/10), partial response in 20% (2/10) and stable disease in 60% (6/10) of patients with follicular lymphoma (PMID: 24852792). 24852792
Unknown unknown follicular lymphoma not applicable Copanlisib FDA approved Actionable In a Phase II trial that supported FDA approval, Aliqopa (copanlisib) treatment in patients with follicular lymphoma resulted in an objective tumor response rate of 58.7% (61/104) including 14.4% (15/61) patients experiencing a complete response and 44.2% (46/61) patients experiencing a partial response, stable disease in 33.7% (35/104) of patients, and a duration of response of 370 days (Journal of Clinical Oncology 35, no. 15_suppl (May 2017) 7535-7535; NCT01660451). detail...
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 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 colon adenocarcinoma not applicable Temsirolimus + Cetuximab Preclinical Actionable In a preclinical study, Torisel (temsirolimus) decreased resistance to Erbitux (cetuximab) in colon cancer cells (PMID: 24493623). 24493623
Unknown unknown hepatocellular carcinoma not applicable 4SC-202 Preclinical Actionable In a preclinical study, 4SC-202 inhibited growth and induced apoptosis in hepatocellular carcinoma cell lines in culture (PMID: 26773495). 26773495
Unknown unknown chronic lymphocytic leukemia not applicable Ibrutinib + Venetoclax Phase II Actionable In a Phase II trial, Imbruvica (ibrutinib) and Venclexta (venetoclax) combination therapy resulted in a response rate of 100% (14/14, 9 complete response, 5 partial response) in relapsed or refractory chronic lymphocytic leukemia (CLL) patients, and a response rate of 100% (16/16, 9 complete response, 7 partial response) in untreated patients with high-risk features including del 17p, TP53 mutations, and del 11q (ASH, 59th Annual Meeting and Exposition, Dec 2017, Abstract 429; NCT02756897). detail...
Unknown unknown renal cell carcinoma not applicable GDC-0349 Preclinical - Cell line xenograft Actionable In a preclinical study, the mTOR inhibitor GDC-0349 demonstrated inhibition of tumor growth in cell line xenograft models of solid tumors, including renal adenocarcinoma (PMID: 24900569). 24900569
Unknown unknown gastroesophageal junction adenocarcinoma not applicable Ramucirumab + Paclitaxel FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Cyramza (ramucirumab) in combination with Taxol (paclitaxel) increased progression-free and overall survival and improved quality of life compared to treatment with Taxol (paclitaxel) alone in patients with gastroesophageal junction adenocarcinoma (PMID: 24706672). detail... 24706672
Unknown unknown acute myeloid leukemia not applicable Forskolin Preclinical Actionable In a preclinical study, Forskolin inhibited cell proliferation and induced apoptosis in acute myeloid leukemia cells in culture (PMID: 21233840). 21233840
Unknown unknown non-small cell lung carcinoma not applicable Chiauranib Preclinical - Cell line xenograft Actionable In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a non-small cell lung cancer cell line xenograft model (PMID: 28004478). 28004478
Unknown unknown mantle cell lymphoma not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in partial response in 17% (1/6) and stable disease in 67% (4/6) of patients with mantle cell lymphoma (PMID: 29475723; NCT01767766). 29475723
Unknown unknown non-small cell lung carcinoma no benefit Bavituximab + Docetaxel Phase II Actionable In a Phase II trial, Tarvacin (bavituximab) and Taxotere (docetaxel) combination treatment resulted in favorable outcome compared to control in non-small cell lung cancer patients, with an overall response rate of 17.1% (7/41), median progression-free survival of 4.5 months (HR=0.74), and a median overall survival of 11.7 months (HR=0.66) (PMID: 27265742). 27265742
Unknown unknown non-small cell lung carcinoma no benefit Bavituximab + Docetaxel Phase III Actionable In a Phase II trial, Tarvacin (bavituximab) and Taxotere (docetaxel) combination treatment did not improve overall survival (10.5 vs 10.9 months, HR=1.06, p=0.533) or progression-free survival (HR=1.00, p=0.990) compared to Taxotere (docetaxel) and placebo in patients with previously treated advanced non-squamous non-small-cell lung cancer (PMID: 29767677; NCT01999673). 29767677
Unknown unknown stomach cancer no benefit Irinotecan + Nimotuzumab Phase II Actionable In a Phase II trial, addition of Nimotuzumab to Camptosar (irinotecan) did not significantly improve median progression-free survival (73 vs 85 days), median overall survival (250.5 vs 232.0 days) and response rate (18.4 vs 10.3 %) compared to Camptosar alone in gastric cancer patients (PMID: 25185971). 25185971
Unknown unknown fallopian tube cancer not applicable Everolimus + Bevacizumab 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 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 prostate cancer not applicable Abiraterone + Olaparib Phase II Actionable In a Phase II trial, treatment with the combination of Lynparza (olaparib) and Zytiga (abiraterone) resulted in a prolonged median radiographic progression-free survival of 13.8 months, compared to 8.2 months with placebo plus Zytiga (abiraterone), in patients with metastatic castration-resistant prostate cancer (PMID: 29880291; NCT01972217). 29880291
Unknown unknown 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 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 gastroesophageal junction adenocarcinoma no benefit Ipilimumab Phase II Actionable In a Phase II trial, Yervoy (ipilimumab) did not improve immune-related progression-free survival (2.9 vs 4.9 months) compared to best supportive care in patients with unresectable, locally advanced/metastatic gastric or gastroesophageal junction cancer (J Clin Oncol 34, 2016 (suppl; abstr 4011)). detail...
Unknown unknown non-small cell lung carcinoma not applicable LY2874455 Phase I Actionable In a Phase I trial, 92% (11/12) of patients with non-small cell lung carcinoma demonstrated stable disease after two cycles of LY2874455, however, after four cycles, all patients had either progressed or discontinued the study (PMID: 28589492; NCT01212107). 28589492
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 pancreatic endocrine carcinoma not applicable Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) inhibited pancreatic neuroendocrine tumor growth and invasion in transgenic mouse models (PMID: 22585997). 22585997
Unknown unknown breast cancer not applicable Alisertib + Fulvestrant Preclinical - Cell culture Actionable In a preclinical study, treatment with the combination of Alisertib (MLN8237) and Faslodex (fulvestrant) resulted in increased growth inhibition in aromatase inhibitor-resistant breast cancer cell lines compared to either agent alone (PMID: 25667100). 25667100
Unknown unknown sarcoma not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) treatment resulted in twelve-week progression free survival in 36% (4/11) of undifferentiated sarcoma patients (PMID: 27502708). 27502708
Unknown unknown non-small cell lung carcinoma not applicable Metformin Preclinical - Cell culture Actionable In a preclinical study, Glucophage (metformin) inhibited proliferation and induced cell-cycle arrest and apoptosis in non-small cell lung cancer cell lines in culture, independent of STK11 (LKB1) status (PMID: 26847819). 26847819
Unknown unknown Advanced Solid Tumor not applicable CC-122 Phase I Actionable In a Phase I trial, Avadomide (CC-122) treatment was well tolerated in patients with advanced solid tumors (n=27) but did not result in any objective responses in patients with advanced solid tumors other than brain cancer (n=19), stable disease was observed in 2 patients (lasting 114 and 309 days) with hepatocellular carcinoma (PMID: 30201761; NCT01421524). 30201761
Unknown unknown diffuse large B-cell lymphoma not applicable SEL24-B489 + Ibrutinib Preclinical Actionable In a preclinical study, SEL24-B489 demonstrated a synergistic effect when combined with Ibruvica (ibrutinib) in diffuse large B-cell lymphoma cells in culture, resulting in cell growth inhibition ((Blood 126 (23):706.December 2015). 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 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 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 MEHD7945A Phase I Actionable In a Phase I trial, MEHD7945A treatment resulted in partial response in 3% (2/66) and stable disease in 21% (14/66) of patients with advanced solid tumors (PMID: 26034219). 26034219
Unknown unknown pancreatic carcinoma not applicable BMS-906024 Preclinical Actionable In a preclinical study, the pan-Notch inhibitor, BMS-906024, inhibited growth of pancreatic carcinoma xenografts (PMID: 26005526). 26005526
Unknown unknown diffuse large B-cell lymphoma not applicable MLN0905 Preclinical Actionable In a preclinical study, MLN0905 treatment resulted in decreased tumor volume in a diffuse large B-cell lymphoma xenograft model (PMID: 22609854). 22609854
Unknown unknown acute myeloid leukemia not applicable Azacitidine + 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 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 diffuse large B-cell lymphoma not applicable Abexinostat Phase II Actionable In a Phase II trial, patients with diffuse large B-cell lymphoma demonstrated an overall response rate of 31% (5/16) and a median duration response of 1.9 months when treated with Abexinostat (PCI-24781) (PMID: 28126962). 28126962
Unknown unknown gastrointestinal stromal tumor not applicable Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) treatment resulted in partial response in 13% (4/31), stable disease in 52% (16/31), a median progression-free survival of 4.9 months and an overall survivals of 9.7 months in gastrointestinal stromal tumor patients who failed prior tyrosine kinase inhibitors (PMID: 22270258). 22270258
Unknown unknown germ cell cancer not applicable Pazopanib Phase II Actionable In a Phase II trial, Votrient (pazopanib) treatment resulted in partial responses in 4.7% (2/43), stable disease in 44.2% (19/43), and a 3-month progression free survival probability of 12.8% in patients with refractory germ cell cancer (PMID: 28383677). 28383677
Unknown unknown pediatric ependymoma not applicable CUDC-907 Phase I Actionable In a Phase I trial, CUDC-907 treatment resulted in stable disease for 6 treatment cycles in a pediatric patient with ependymoma (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 10542-10542; NCT02909777). detail...
Unknown unknown 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 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 non-small cell lung carcinoma no benefit Sorafenib Phase III Actionable In a Phase III trial, Nexavar (sorafenib) treatment in non-small cell lung carcinoma patients did not reach its primary endpoint, resulting in an overall survival similar to that when treated with placebo, however, did meet its secondary endpoint, demonstrating a greater progression free survival and time to progression when compared to placebo (PMID: 26743856). 26743856
Unknown unknown medulloblastoma not applicable MS44 Preclinical - Cell culture Actionable In a preclinical study, M344 induced apoptosis and inhibited proliferation of medulloblastoma cell lines in culture (PMID: 17230517). 17230517
Unknown unknown 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 pancreatic adenocarcinoma not applicable Vandetanib + Gemcitabine Phase I Actionable In a Phase I trial, Caprelsa (vandetanib), in combination with Gemzar (gemcitabine), demonstrated safety and resulted in stable disease in metastatic pancreatic adenocarcinoma patients (PMID: 21921646). 21921646
Unknown unknown 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 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 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 glioblastoma multiforme not applicable Alisertib Preclinical Actionable In a preclinical study, Alisertib (MLN8237) decreased proliferation of primary glioblastoma cell lines in culture, and improved survival of primary glioblastoma cell line xenograft models, including models resistant to Avastin (bevacizumab) (PMID: 27816996). 27816996
Unknown unknown Advanced Solid Tumor not applicable CG200745 Phase I Actionable In a Phase I trial, CG200745 treatment demonstrated safety in advanced solid tumor patients and did not result in any partial or complete responses, but led to stable disease in 57.1% (16/28) of patients for at least six weeks (PMID: 26076682). 26076682
Unknown unknown thyroid medullary carcinoma not applicable Lenvatinib Phase II Actionable In a Phase II trial, advanced medullary thyroid cancer patients experienced an objective response rate of 36% (21/59, all partial responses) and median progression free survival was 9 months when treated with Lenvima (lenvatinib) (PMID: 26311725). 26311725
Unknown unknown triple-receptor negative breast cancer not applicable Bevacizumab + Capecitabine + Paclitaxel Phase II Actionable In a Phase II clinical trial, treatment with the combination of Avastin (bevacizumab), Xeloda (capecitabine), and Taxol (paclitaxel) resulted in an overall response rate of 77% (44/57), including complete response in 19% (11/57), and a median progression-free survival of 7.6 months and median overall survival of 19.2 months in patients with triple-negative breast cancer (PMID: 27412268). 27412268
Unknown unknown pancreatic cancer not applicable Trabedersen Phase Ib/II Actionable In a Phase I/II trial, Trabedersen (AP 12009) treatment followed by chemotherapy resulted in an overall survival of 14.5 months in advanced pancreatic cancer patients (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 119P). detail...
Unknown unknown sarcoma not applicable Abexinostat + Doxorubicin Phase I Actionable In a Phase I study, Abexinostat (PCI-24781), in combination with doxorubicin, displayed safety and preliminary efficacy in patients with metastatic sarcoma (PMID: 25536954). 25536954
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 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 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 melanoma not applicable Indoximod + Pembrolizumab Phase II Actionable In a Phase II trial, combination of Indoximod and Keytruda (pembrolizumab) resulted in an objective response rate of 55.7% (39/70, 13 complete response), with a median progression-free survival of 12.4 months in patients with advanced melanoma (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 9512-9512; NCT02073123). 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 Ramucirumab + Docetaxel FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with a combination of Cyramza (ramucirumab) and Taxotere (docetaxel) resulted in improved overall survival compared to treatment with Taxotere (docetaxel) and a placebo in patients with metastatic non-small cell lung cancer (PMID: 24933332). detail... 24933332
Unknown unknown Advanced Solid Tumor not applicable CEP-32496 Phase I Actionable In a Phase I clinical trial, RXDX-105 (CEP-32496) was tolerable and demonstrated preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2574)). detail...
Unknown unknown colon cancer not applicable Temsirolimus + Cetuximab 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 ovarian cancer not applicable ST7612AA1 Preclinical - Cell line xenograft Actionable In a preclinical study, ST7612AA1 inhibited proliferation of ovarian cancer cell lines in culture, and inhibited tumor growth in ovarian cancer cell line xenograft models (PMID: 25671299). 25671299
Unknown unknown multiple myeloma sensitive CB-5083 Preclinical - Cell line xenograft Actionable In a preclinical study, CB-5083 treatment of multiple myeloma cell lines and xenografts resulted in cell death and decreased tumor growth, respectively (PMID: 28878026). 28878026
Unknown unknown B-cell lymphoma not applicable JCAR017 Phase I Actionable In a Phase I trial, JCAR017 treatment resulted in a response rate of 80% (22/28) with a complete response rate of 60% (17/28) in patients with relapsed/refractory B-cell non-Hodgkin lymphoma (J Clin Oncol 35, 2017 (suppl; abstr 7513); NCT02631044). detail...
Unknown unknown breast carcinoma not applicable MRx0518 Preclinical Actionable In a preclinical study, MRx0518 stimulated immune response and reduced tumor size in syngeneic mouse models of breast carcinoma (Journal of Clinical Oncology 36, no. 15_suppl). detail...
Unknown unknown glioblastoma multiforme not applicable Bortezomib + Vorinostat Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Zolinza (vorinostat) and Velcade (bortezomib) synergized to decrease cell viability, DNA fragmentation and elevate caspase 9 activity in several human glioblastoma cell lines in culture and in xenograft models of one human glioblastoma cell line (PMID: 26804704). 26804704
Unknown unknown Advanced Solid Tumor not applicable Alpelisib + BGJ398 Phase I Actionable In a Phase Ib trial, BGJ398 and Alpelisib (BYL719) combination treatment resulted in partial response in 25% (8/32) of patients with advanced solid tumors, including urothelial, head and neck, melanoma, and anal cancer (J Clin Oncol 34, 2016 (suppl; abstr 2500)). detail...
Unknown unknown non-small cell lung carcinoma not applicable TAS-116 + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, TAS-116 increased sensitivity of a non-small cell lung cancer cell line to X-ray and carbon ion radiotherapy, resulting in decreased DNA double-strand break repair and increased cell-cycle arrest in culture (PMID: 28062703). 28062703
Unknown unknown glioblastoma multiforme not applicable PRX177561 + Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of PRX177561 and Sutent (sunitinib) decreased tumor growth and improved time-to-progression, disease-free survival, and overall survival over either agent alone in glioblastoma cell line xenograft models (PMID: 28057017). 28057017
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 non-small cell lung carcinoma not applicable ST7612AA1 Preclinical - Cell line xenograft Actionable In a preclinical study, ST7612AA1 inhibited proliferation of non-small cell lung cancer (NSCLC) cell lines in culture, and inhibited tumor growth in NSCLC cell line xenograft models (PMID: 25671299). 25671299
Unknown unknown biliary tract cancer not applicable Ramucirumab Phase II Actionable In a Phase II trial, Cyramza (ramucirumab) treatment resulted in a progression-free survival of 2.73 months, an overall survival of 6.31 months, 0% objective response rate, and a disease control rate of 44% (15/34) in patients with advanced biliary cancer(J Clin Oncol 36, 2018 (suppl; abstr 4081); NCT02520141). detail...
Unknown unknown Hodgkin's lymphoma not applicable Idelalisib Phase II Actionable In a Phase II trial, Idelalisib treatment resulted in an overall response rate of 20% (5/25) in Hodgkin's lymphoma patients, with one patient experiencing a complete response and four patients experiencing a partial response (PMID: 28327905). 28327905
Unknown unknown Advanced Solid Tumor not applicable Flucytosine + TG4023 Phase I Actionable In a Phase I trial, TG4023 and Flucytosine combination therapy demonstrated safety and preliminary efficacy, resulted in stable disease in 50% (8/16) of patients with advanced solid tumors (PMID: 28177438; NCT00978107). 28177438
Unknown unknown 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 peritoneum cancer not applicable Everolimus + Bevacizumab 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 triple-receptor negative breast cancer not applicable Cisplatin + JQ1 Preclinical - Cell culture Actionable In a preclinical study, the addition of JQ1 to Platinol (cisplatin) resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27256375). 27256375
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 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 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 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 gastroesophageal junction adenocarcinoma not applicable Apatinib Phase III Actionable In a Phase III trial, Apatinib (YN968D1) treatment significantly improved median overall survival (6.5 vs 4.7 months) and median progression-free survival (2.6 vs 1.8 months) compared to placebo in chemotherapy-refractory patients with advanced gastric or gastroesophageal junction adenocarcinoma (PMID: 26884585). 26884585
Unknown unknown colorectal cancer not applicable Demcizumab Phase I Actionable In a Phase I trial, treatment with Demcizumab (OMP-21M18) resulted in downregulation of Notch target genes and demonstrated preliminary efficacy in patients with advanced solid tumors, with reductions in tumor size in patients with several tumor types, including colorectal cancer (PMID: 25324140). 25324140
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 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 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-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 squamous cell carcinoma not applicable Prexasertib Phase Ib/II Actionable In a Phase Ib trial, Prexasertib (LY2606368) demonstrated safety and resulted in a clinical benefit rate at 3 months of 29% (29/101) in squamous cell carcinoma patients, including patients with squamous cell carcinoma of the anus, head and neck squamous cell carcinoma, and squamous non-small cell lung cancer (PMID: 29643063; NCT0115790). 29643063
Unknown unknown squamous cell carcinoma not applicable Prexasertib Phase I Actionable In a Phase I trial, Prexasertib (LY2606368) treatment resulted in partial response in 2 patients with squamous cell carcinoma (SCC), and 40% (6/15) of solid tumor patients who achieved stable disease had SCC (PMID: 27044938; NCT0115790). 27044938
Unknown unknown stomach 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 stomach carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
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 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 Advanced Solid Tumor not applicable Trebananib Phase I Actionable In a Phase I trial, treatment with Trebananib demonstrated tolerability in pediatric patients with advanced solid tumors, and resulted in stable disease for greater than 4 months in one patient with neuroblastoma and one patient with anaplastic astrocytoma (PMID: 28751444). 28751444
Unknown unknown Ewing sarcoma no benefit Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in no objective response (0/13) in patients with Ewing sarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). detail...
Unknown unknown breast cancer not applicable SKLB-23bb Preclinical - Cell line xenograft Actionable In a preclinical study, SKLB-23bb treatment inhibited tumor growth in breast cancer xenograft models (PMID: 29610282). 29610282
Unknown unknown breast carcinoma not applicable MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human breast carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown colon cancer not applicable Ch282-5 Preclinical - Cell line xenograft Actionable In a preclinical study, ch282-5 induced apoptosis and inhibited growth and migration of several human and mouse colon cancer cell lines in culture, and inhibited tumor growth and metastasis in xenograft models (PMID: 26515494). 26515494
Unknown unknown glioblastoma multiforme not applicable DCVax-L Phase III Actionable In a Phase III trial, addition of DCVax-L to standard therapy resulted in a median overall survival (mOS) of 23.1 months in the intended to treat group (n=331) of patients with newly diagnosed glioblastoma, with a mOS of 34.7 months in patients with methylated MGMT (n?=?131) (PMID: 29843811; NCT00045968). 29843811
Unknown unknown multiple myeloma not applicable ONC201 Preclinical - Cell culture Actionable In a preclinical study, a Velcade (bortezomib)-resistant multiple myeloma cell line demonstrated sensitivity to ONC201 in culture (PMID: 26884599). 26884599
Unknown unknown acute lymphocytic leukemia not applicable AS605240 + Dexamethasone Preclinical - Pdx Actionable In a preclinical study, the combination of AS605240 and dexamethasone improved survival of primary T-ALL cell xenograft models (PMID: 25869207). 25869207
Unknown unknown breast cancer not applicable A-1331852 + Docetaxel Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of A-1331852 and Taxotere (docetaxel) inhibited tumor growth in a metastatic breast cancer cell line xenograft model, with increased efficacy over either agent alone (PMID: 25787766). 25787766
Unknown unknown renal cell carcinoma not applicable GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 treatment was well-tolerated and resulted in some efficacy in renal cell carcinoma patients including stable disease in 13% (3/24), one patient with a complete response, and one patient with a partial response (PMID: 26603258). 26603258
Unknown unknown acute promyelocytic 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 promyelocytic leukemia cell line in culture (PMID: 28753594). 28753594
Unknown unknown head and neck cancer not applicable Cisplatin + GL-ONC1 + Radiotherapy Phase I Actionable In a Phase I trial, the combination therapy of GL-ONC1, Platinol (cisplatin), and radiotherapy resulted in a progression-free survival of 74.4% at 1 year and 64.1% at 2 years and an overall survival of 84.6% at 1 year and 69.2% at 2 years in patients with nonmetastatic head and neck cancer (PMID: 28679776). 28679776
Unknown unknown 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 Advanced Solid Tumor not applicable CCT241533 + Olaparib Preclinical - Cell culture Actionable In a preclinical study, CCT241533 enhanced the growth inhibition effects of Lynparza (olaparib) in cancer cells in culture (PMID: 21239475). 21239475
Unknown unknown acute myeloid leukemia not applicable Venetoclax Phase II Actionable In a Phase II trial, treatment with Venclexta (venetoclax) resulted in a 19% (6/32) overall response rate, a 6% (2/32) complete response, and a 13% (4/32) complete response with incomplete blood count recovery in acute myeloid leukemia patients (PMID: 27520294). 27520294
Unknown unknown renal cell carcinoma not applicable GDC-0980 Phase II Actionable In a Phase II clinical trial, Apitolisib (GDC-0980) was inferior to Afinitor (everolimus) (median PFS, 3.7 months (n=42) vs. 6.1 months (n=43), respectively) and overall survival trended lower in the Apitolisib (GDC-0980) arm in patients with metastatic renal cell carcinoma (PMID: 26951309). 26951309
Unknown unknown ovarian clear cell carcinoma not applicable GDC-0980 Phase I Actionable In a Phase I trial, Apitolisib (GDC-0980) treatment resulted in tumor regression by 48.3% in a patient with ovarian clear cell carcinoma (PMID: 26787751). 26787751
Unknown unknown non-small cell lung carcinoma not applicable Adavosertib Preclinical - Cell line xenograft Actionable In a preclinical study, MK-1775 inhibited cell proliferation and promoted DNA damage in human non-small cell lung carcinoma cell lines in culture, and promoted tumor regression in xenograft models (PMID: 23699655). 23699655
Unknown unknown 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 lung small cell carcinoma not applicable T-3775440 Preclinical - Cell line xenograft Actionable In a preclinical study, T-3775440 treatment led to decreased cell proliferation in lung small cell carcinoma cell lines in culture, demonstrating reduced expression of INSM1 or GFI1B, and inhibited tumor growth in lung small cell carcinoma cell line xenograft models (PMID: 28667074). 28667074
Unknown unknown 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 hematologic cancer not applicable Glasdegib Phase I Actionable In a Phase I trial, Glasdegib (PF-04449913) treatment in patients with hematological malignancies resulted in some preliminary efficacy, including a complete response in one patient with acute myeloid leukemia (AML) and stable disease in four patients with AML (PMID: 28556364). 28556364
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 Advanced Solid Tumor not applicable ABT-348 Preclinical - Pdx & cell culture Actionable In a preclinical study, ABT-348 (Ilorasertib) inhibited proliferation and promoted apoptosis in various solid tumor cell culture and Pdx models, including NSCLC, ovarian, and colon (AACR; Mol Cancer Ther 2011;10(11 Suppl):Abstract nr B231). detail...
Unknown unknown colorectal cancer not applicable Nivolumab FDA approved Actionable In a Phase II trial 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 colorectal cancer not applicable IMC-3C5 Phase I Actionable In a Phase I trial, 50% (4/8) of colorectal cancer patients experienced a progression free survival when treated with IMC-3C5, however, no responses were reported per RECIST (Journal of Clinical Oncology, Vol 33, No 15_suppl (May 20 Supplement), 2015: 3530). detail...
Unknown unknown glioblastoma multiforme no benefit Sunitinib Phase II Actionable In multiple Phase II clinical trials, Sutent (sunitinib) failed to demonstrate any benefit in patients with glioblastoma with or without concurrent bevacizumab treatment (PMID: 24424564, PMID: 23086433). 23086433 24424564
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 colon adenocarcinoma not applicable CVX-241 + Irinotecan Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of CVX-241 and Camptosaur (irinotecan) resulted in increased tumor growth inhibition compared to either agent alone in a colon adenoarcinoma cell line xenograft model (PMID: 21149738). 21149738
Unknown unknown brain stem glioma no benefit Veliparib Preclinical - Cell culture Actionable In a preclinical study, Veliparib (ABT-888) had a very limited effect on the cell viability of multiple cultured pediatric diffuse intrinsic pontine glioma cell lines (PMID: 26351319). 26351319
Unknown unknown Advanced Solid Tumor not applicable MK-1775 + Gemcitabine Phase I Actionable In a Phase I trial, the combination of MK-1775 and Gemzar (gemcitabine) resulted in a partial response in 5% (4/81) of patients and stable disease in 53% (43/81) of patients, all with advanced solid tumors (PMID: 27601554). 27601554
Unknown unknown osteosarcoma not applicable BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 67% (4/6) of cell line xenograft models of osteosarcoma (PMID: 21298745). 21298745
Unknown unknown melanoma not applicable Abemaciclib Phase I Actionable In a Phase I trial, treatment with Abemaciclib (LY2835219) in melanoma patients resulted in a disease control rate of 27% (7/26), a partial response in one patient and six patients with stable disease (PMID: 27217383). 27217383
Unknown unknown 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 invasive bladder transitional cell carcinoma not applicable Cisplatin + Gemcitabine + Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) in combination with Platinol (cisplatin) and Gemzar (gemcitabine) resulted in pathologic complete response in 42.2% (19/45) of patients with muscle-invasive urothelial bladder cancer (J Clin Oncol 35, 2017 (suppl 6S; abstract 345)). detail...
Unknown unknown Advanced Solid Tumor not applicable ONCOS-102 Phase I Actionable In a Phase I trial, ONCOS-102 demonstrated safety and preliminary efficacy, resulted in disease control in 40% (4/10) of patients with advanced solid tumors, and a median overall survival of 9.3 months (PMID: 26981247). 26981247
Unknown unknown malignant pleural solitary fibrous tumor not applicable Dasatinib Phase II Actionable In a Phase II trial, patients with solitary fibrous tumors demonstrated a median progression free survival of 2 months and five patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown 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 stomach cancer not applicable AZD6738 + Paclitaxel Preclinical - Cell culture Actionable In a preclinical study, the addition of AZD6738 to Taxol (paclitaxel) treatment in a gastric cancer cell line in culture resulted in enhanced chemotherapeutic sensitivity, demonstrating a synergistic effect (PMID: 28138034). 28138034
Unknown unknown liposarcoma not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) treatment resulted in twelve-week progression free survival in 73% (9/12) of liposarcoma patients (PMID: 27502708). 27502708
Unknown unknown Advanced Solid Tumor not applicable Pazopanib Phase I Actionable In a Phase I study, Votrient (pazopanib) in combination with Taxol (paclitaxel) and Paraplatin (carboplatin) demonstrated efficacy in solid tumors (PMID: 22679111). 22679111
Unknown unknown Advanced Solid Tumor not applicable Pazopanib Phase I Actionable In a Phase I trial, Votrient (pazopanib) demonstrated safety and efficacy in a variety of pediatric solid tumors (PMID: 23857966). 23857966
Unknown unknown basal cell carcinoma not applicable Sonidegib FDA approved Actionable In a Phase II trial that supported FDA approval, treatment with Odomzo (sonidegib) resulted in an objective response in 36% (20/55) of patients with locally advanced basal cell carcinoma (PMID: 25981810). 25981810
Unknown unknown hepatocellular carcinoma not applicable Cabozantinib + CT-707 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Cometriq (Cabometyx, cabozantinib) and CT-707 resulted in synergism in hepatocellular carcinoma cells, demonstrating increased apoptosis and inhibition of colony formation in culture and decreased tumor weight in xenograft models (PMID: 27638856). 27638856
Unknown unknown melanoma not applicable V158411 Preclinical - Cell culture Actionable In a preclinical study, V158411 induced DNA damage and cell-cycle arrest, and inhibited growth of a melanoma cell line in culture (PMID: 27829224). 27829224
Unknown unknown 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 lung cancer not applicable Cediranib + Gefitinib Phase I Actionable In a Phase I trial, the combination of Cediranib and Iressa (gefitinib) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, including a partial response in a patient with lung cancer (PMID: 20061136). 20061136
Unknown unknown urinary bladder cancer not applicable PD-0325901 + PF-04691502 Preclinical - Pdx Actionable In a preclinical study, PD-0325901, in combination with PF-04691502, delayed tumor growth in patient-derived xenograft models of bladder cancer (PMID: 24442130). 24442130
Unknown unknown ovarian cancer not applicable JNJ-54302833 Preclinical Actionable In a preclinical study, JNJ-54302833 inhibited growth of ovarian cancer cells in culture ( Cancer Res October 1, 2014 74; 4747 ). detail...
Unknown unknown 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 melanoma not applicable fresolimumab Phase I Actionable In a phase I clinical trial, Fresolimumab (GC1008) demonstrated safety and preliminary evidence of antitumor activity in patients with malignant melanoma (PMID: 24618589). 24618589
Unknown unknown 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 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 Advanced Solid Tumor not applicable KW-2450 Phase I Actionable In a Phase I trial, 40% (4/10) of patients with advanced solid tumors demonstrated stable disease when treated with KW-2450 (PMID: 26850678). 26850678
Unknown unknown 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 multiple myeloma not applicable FT671 Preclinical - Cell line xenograft Actionable In a preclinical study, FT671 treatment blocked proliferation of multiple myeloma cells in culture and inhibited tumor growth in multiple myeloma cell line xenograft models (PMID: 29045389). 29045389
Unknown unknown 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 urinary bladder cancer not applicable MPT0L145 Preclinical - Cell culture Actionable In a preclinical study, treatment with MPT0L145 resulted in decreased cell viability in bladder cancer cell lines in culture (PMID: 29222162). 29222162
Unknown unknown 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 peritoneal carcinoma not applicable Paclitaxel + TVB-2640 Phase I Actionable In a Phase I trial, TVB-2640 and Taxol (paclitaxel) combination treatment resulted in partial response and a 58% reduction in CA-125 level in a peritoneal carcinoma patient (J Clin Oncol 34, 2016 (suppl; abstr 2512)). detail...
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 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 S├ęzary syndrome (Blood 2017 130(Suppl 1):817). detail...
Unknown unknown prostate cancer not applicable Niraparib Phase I Actionable In a Phase I clinical trial, Zejula (niraparib) demonstrated antitumor activity in patients advanced solid tumors, including patients with prostate cancer (PMID: 23810788) 23810788
Unknown unknown hematologic cancer not applicable GSK1070916 Preclinical - Cell line xenograft Actionable In a preclinical study, GSK1070916 inhibited proliferation of a variety of hematological cancer types in cell culture and in xenografts (PMID: 19567821). 19567821
Unknown unknown renal cell carcinoma not applicable Tivantinib Phase II Actionable In a Phase II trial, tivantinib resulted in modest activity in which six patients with renal cell carcinoma had a median PFS of 1.9 months (PMID: 22605650). 22605650
Unknown unknown sarcoma not applicable Alvocidib + Aldoxorubicin Phase II Actionable In a Phase I trial, Alvocidib (flavopiridol), in combination with aldoxorubicin resulted in partial responses in 6% (2/31) and stable disease in 51% (16/31) of patients with advanced sarcoma (PMID: 22374332). 22374332
Unknown unknown 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 synovial sarcoma not applicable Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 10% (1/10) of patients with synovial sarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). detail...
Unknown unknown myelofibrosis not applicable XL019 Phase I Actionable In a Phase I trial, treatment with XL019 demonstrated clinical activity in 23% (7/30) of myelofibrosis patients, including 27% (6/22) of patients with JAK2 V617F and 13% (1/8) with wild-type JAK2, and resulted in responses in 10% (3/30) of patients, however, resulted in neurotoxicity and the trial was discontinued (PMID: 24374145; NCT00595829). 24374145
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 fibrosarcoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) resulted in an overall response rate of 11% (2/18) and a disease progression free rate at 12 weeks of 61% (11/18) in patients with fibrosarcoma (J Clin Oncol 34, 2016 (suppl; abstr 11005)). detail...
Unknown unknown endometrial cancer not applicable Everolimus Phase II Actionable In a Phase II trial, the mTOR inhibitor Afinitor (everolimus) demonstrated efficacy and tolerability in patients with chemotherapy-refractory advanced or metastatic endometrial cancer (PMID: 23612453). 23612453
Unknown unknown glioblastoma multiforme not applicable G-TPP + Venetoclax Preclinical - Patient cell culture Actionable In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the Bcl2 inhibitor Venclexta (venetoclax) synergistically inhibited viability of established lines and patient-derived glioblastoma cells in culture (PMID: 28522750). 28522750
Unknown unknown non-small cell lung carcinoma not applicable Lenvatinib + Carboplatin + 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 acute myeloid leukemia not applicable ABBV-075 + Bortezomib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Velcade (bortezomib) and ABBV-075 resulted in increased tumor growth inhibition in an acute myeloid leukemia cell line xenograft model compared to Velcade (bortezomib) alone (PMID: 28416490). 28416490
Unknown unknown Advanced Solid Tumor not applicable SOR-C13 Phase I Actionable In a Phase I trial, SOR-C13 demonstrated safety and preliminary activity in patients with advanced solid tumors, with treatment resulting in stable disease in 54.5 % (12/22) of patients (PMID: 28150073). 28150073
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 lung adenocarcinoma not applicable Nintedanib + Docetaxel Phase III Actionable In a Phase III trial, the combination of Ofev (nintedanib) and Taxotere (docetaxel) resulted in an improved overall survival in lung adenocarcinoma patients, particularly those patients with shorter time to progression after first line chemotherapy, which included measures that were time from start or time from end of first line chemotherapy (PMID: 28702806). 28702806
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 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 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 RTA-408 Preclinical Actionable In a preclinical study, RTA-408 induced apoptosis and inhibited growth of several human tumor cell lines in culture (PMID: 25897966). 25897966
Unknown unknown multiple myeloma 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 a multiple myeloma cell line xenograft model compared to either agent alone (PMID: 28416490). 28416490
Unknown unknown Advanced Solid Tumor not applicable Pimasertib Phase I Actionable In a Phase I trial, Pimasertib (MSC1936369B) demonstrated safety and some preliminary anti-tumor activity in patients with advanced solid tumors (J Clin Oncol 28:15s, 2010 (suppl; abstr 2504)). detail...
Unknown unknown ovarian cancer not applicable Napabucasin Phase Ib/II Actionable In a Phase Ib/II clinical trial, treatment with BBI608 demonstrated safety in patients with platinum-resistant ovarian cancer (including patients with epithelial ovarian, fallopian, or peritoneal cancer), and resulted in a disease control rate of 68% (27/40) and an objective response rate of 25% (10/40) in evaluable patients (J Clin Oncol, May 2016 vol. 34 no. 15_suppl 5578). detail...
Unknown unknown Indication other than cancer not applicable PP30 Preclinical Actionable In a preclinical study, PP30 inhibited proliferation of mouse embryonic fibroblasts more effectively than rapamycin (PMID: 19209957). 19209957
Unknown unknown gastrointestinal neuroendocrine tumor not applicable Pazopanib Clinical Study Actionable In a clinical study, Votrient (pazopanib) treatment in patients with gastroenteropancreatic neuroendocrine tumors resulted in an overall response rate of 24% (19/124), stable disease in 39.5% (49/124), a progression free survival of 36% at six months, and a median overall survival of 10.2 months (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 446P). detail...
Unknown unknown mantle cell lymphoma not applicable 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 triple-receptor negative breast 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 triple-receptor negative breast cancer cells in culture (PMID: 28522750). 28522750
Unknown unknown colorectal cancer not applicable PD-0325901 Phase I Actionable In a Phase I study, PD-325901 demonstrated efficacy but toxicity at current dose was unacceptable (PMID: 21516509). 21516509
Unknown unknown esophagus squamous cell carcinoma not applicable OBP-801 + 5-FU 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 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 colorectal cancer no benefit Olaparib Phase II Actionable In a Phase II clinical trial, treatment with Lynparza (olaparib) did not result in clinical activity in colorectal cancer patients that had progressed on prior standard therapy, including both microsatellite-stable patients and those that demonstrated high microsatellite instability (PMID: 26786262). 26786262
Unknown unknown lung cancer no benefit Cediranib + Carboplatin + Paclitaxel Phase I Actionable In a Phase II clinical trial of patients with advanced NSCLC, the combination of Cediranib (AZD-2171) and carboplatin/paclitaxel (CP) chemotherapy resulted in improved response rate over placebo plus CP, but did not improve progression-free survival, and the study did not proceed to Phase III due to toxicity (PMID: 24360368). 24360368
Unknown unknown 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 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 chronic lymphocytic leukemia not applicable Venetoclax FDA approved Actionable In a Phase II clinical trial that supported FDA approval, 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 Phase II Actionable In a Phase II trial, 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 pancreatic cancer not applicable Pimasertib + Gemcitabine 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 glioblastoma multiforme not applicable Imetelstat Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with imetelstat resulted in increased cell death in glioblastoma multiforme (GBM) cells in culture, and decreased tumor growth in xenograft models (PMID: 20048334). 20048334
Unknown unknown Ewing sarcoma not applicable SN-38 + Talazoparib Preclinical - Cell culture Actionable In a preclinical study, Ewing sarcoma cells treated with SN-38 combined with Talazoparib (BMN-673) resulted in synergism, demonstrating reduced cell viability in culture (PMID: 26438158). 26438158
Unknown unknown Advanced Solid Tumor not applicable ASN003 Phase I Actionable In a Phase I trial, ASN003 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 371PD; NCT02961283). detail...
Unknown unknown melanoma not applicable CA-170 Preclinical - Cell line xenograft Actionable In a preclinical study, CA-170 activated peripheral T cells and inhibited tumor growth in mouse models of melanoma (Ann Oncol. 2017 Sep 18; 28 (Suppl_5): Abstract 1141PD). detail...
Unknown unknown diffuse large B-cell lymphoma not applicable SEL24-B489 Preclinical - Cell line xenograft Actionable In a preclinical study, SEL24-B489 treatment induced apoptosis in diffuse large B-cell lymphoma cells in both culture and xenograft models (Blood 126 (23):706.December 2015). detail...
Unknown unknown acute promyelocytic leukemia not applicable Arsenic trioxide + Tretinoin Phase III Actionable In a Phase III trial, a 40.6 month follow-up of acute promyelocytic leukemia (APL) patients treated with the combination of Vesanoid (tretinoin) and Trisenox (arsenic trioxide) demonstrated a better event-free survival, cumulative incidence of relapse, and overall survival when compared to APL patients treated with the combination of Vesanoid (tretinoin) and chemotherapy (PMID: 27400939). 27400939
Unknown unknown stomach cancer not applicable Sapitinib + Fluorouracil + Cisplatin Preclinical - Pdx Actionable In a preclinical study, Sapitinib (AZD8931), in combination with Platinol (cisplatin) and Adrucil (fluorouracil), demonstrated safety and an additive effect in a primary gastric cancer xenograft model (Cancer Res April 15, 2013 73; 2090). detail...
Unknown unknown colorectal cancer not applicable Enadenotucirev Phase I Actionable In a Phase I trial, Enadenotucirev demonstrated safety and uptake by tumor cells in metastatic colorectal cancer patients (Ann Oncol (2014) 25 (suppl 4): iv367). detail...
Unknown unknown thyroid cancer not applicable SNS-314 Preclinical Actionable In a preclinical study, SNS-314 reduced growth of anaplastic thyroid carcinoma cells in culture (PMID: 23099978). 23099978
Unknown unknown hepatocellular carcinoma not applicable PEGPH20 Phase I Actionable In a Phase Ib trial, PEGPH20 treatment resulted in median progression free survival of 7.2 months and overall survival of 13.0 months in hepatocellular carcinoma patients with high pretreatment tissue hyaluronan (HA) levels (n = 6), and 3.5 and 5.7 months respectively for patients with low HA levels (n = 11) (PMID: 26813359). 26813359
Unknown unknown diffuse large B-cell lymphoma not applicable Hu5F9-G4 + Rituximab Phase Ib/II Actionable In a Phase Ib study, combined Hu5F9-G4 and Rituximab therapy demonstrated safety and efficacy, resulting in an objective response rate of 40% (6/15, 5 complete and 1 partial response) and stable disease in 14% (3/15) of patients with diffuse large B-cell lymphoma, and a median duration of response longer than 6 months (PMID: 30380386). 30380386
Unknown unknown lung cancer not applicable ABT-737 + Perifosine Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of ABT-737 and perifosine worked synergistically to induce apoptosis and inhibit growth of lung cancer cell lines and primary lung cancer cells in culture, and to inhibit tumor growth in a lung cancer cell line xenograft model (PMID: 27073162). 27073162
Unknown unknown diffuse large B-cell lymphoma not applicable STRO-001 Preclinical - Cell line xenograft Actionable In a preclinical study, STRO-001 potently inhibited growth of diffuse large B-cell lymphoma cell lines in culture, and suppressed tumor growth in cell line xenograft models (Blood 2016 128 (22):464). detail...
Unknown unknown Advanced Solid Tumor not applicable ABTL0812 Phase I Actionable In a Phase I/Ib trial, ABTL0812 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol (Meeting Abstracts) May 2015 vol. 33 no. 15_suppl 2585). detail...
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 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 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 pancreatic ductal adenocarcinoma not applicable MitoMet-10 Preclinical Actionable In a preclinical study, MitoMet-10 inhibited mitochondrial function and proliferation of pancreatic ductal adenocarcinoma cells in culture, and demonstrated increased potency compared to metformin (PMID: 27216187). 27216187
Unknown unknown colon carcinoma not applicable S-49076 + Bevacizumab 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 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 prostate cancer not applicable EC-70124 Preclinical - Cell line xenograft Actionable In a preclinical study, EC-70124 inhibited proliferation, colony formation, Stat3 activity, and NFkappaB activity in prostate cancer cells and prostate cancer stem cells in culture and inhibited tumor growth in cell line xenografts (PMID: 26826115). 26826115
Unknown unknown 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 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 liposarcoma not applicable Alvocidib + Doxorubicin Phase I Actionable In a Phase I clinical trial, the combination of Alvocidib (flavopiridol) and Adriamycin (doxorubicin) resulted in a disease control rate of 67% (8/12) in patients with liposarcoma (PMID: 22374332). 22374332
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 non-small cell lung carcinoma not applicable DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, non-small cell lung carcinoma cells treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture and inhibition of tumor growth in cell-line xenograft models (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown 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 acute myeloid leukemia not applicable TG02 Preclinical - Patient cell culture Actionable In a preclinical study, TG02 inhibited growth of acute myeloid leukemia cell lines and patient-derived acute myeloid leukemia cells in culture (PMID: 21860433). 21860433
Unknown unknown Advanced Solid Tumor not applicable Lapatinib + MK2206 Phase I Actionable In a Phase I trial, Tykerb (lapatinib) and MK2206 combination treatment resulted in stable disease for more than 4 months in 9% (2/23) of patients with advanced solid tumors (PMID: 27026198). 27026198
Unknown unknown brain glioma not applicable Nintedanib Phase II Actionable In a Phase II clinical trial, Ofev (nintedanib) failed to show any efficacy in patients with recurrent high-grade glioma, regardless of prior bevacizumab therapy (PMID: 25338318). 25338318
Unknown unknown ovarian cancer not applicable Nintedanib + Carboplatin + Paclitaxel Phase III Actionable In a Phase III clinical trial, patients with advanced ovarian cancer treated with Ofev (nintedanib), in combination with Paraplatin (carboplatin) and Taxol (paclitaxel), experienced a progression free survival of 17.2 months versus 16.6 months in patients treated with a combination of placebo, carboplatin, and paclitaxel in first-line treatment (PMID: 26590673). 26590673
Unknown unknown pancreatic cancer not applicable MVT-5873 + Gemcitabine + 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 colorectal cancer not applicable Trametinib + Fluorouracil Preclinical Actionable In preclinical studies, Mekinist (trametinib) enhanced the efficacy of Adrucil (fluorouracil) in colorectal cancer cells in culture (PMID: 23438367). 23438367
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 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 pancreatic carcinoma not applicable Gemcitabine + MU380 Preclinical - Cell line xenograft Actionable In a preclinical study, the addition of MU380 resulted in increased sensitivity to Gemzar (gemcitabine) in a pancreatic carcinoma cell line in culture and in xenograft models (PMID: 28619751). 28619751
Unknown unknown prostate cancer not applicable Everolimus + Docetaxel 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 hepatocellular carcinoma 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 50% (8/16), a disease control rate of 93.8% (15/16, stable disease or better), a median progression-free survival (PFS) of 5.8 months, and a 9-month PFS rate of 41.0% in evaluable patients with advanced hepatocellular carcinoma (PMID: 30348638; NCT02942329). 30348638
Unknown unknown Advanced Solid Tumor not applicable Birinapant Phase I Actionable In a Phase I dose escalation study of birinapant, 50 patients with advanced solid tumors or lymphoma were enrolled and no patients achieved complete or partial remission while stable disease was observed in 3 patients (PMID: 26333381). 26333381
Unknown unknown lung small cell carcinoma not applicable Alisertib + Taxol 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 hepatocellular carcinoma not applicable Selumetinib + Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) and Selumetinib (AZD6244) combination treatment resulted in partial response in 15% (4/27) and stable disease in 48% (13/27) of hepatocellular carcinoma patients (PMID: 27681866). 27681866
Unknown unknown uveal melanoma not applicable AU-011 Preclinical - Cell culture Actionable In a preclinical study, treatment with AU-011 resulted in cell binding and subsequent cell death in uveal melanoma cells in culture (Cancer Res 2014;74(19 Suppl):Abstract nr 1770). detail...
Unknown unknown pancreatic cancer not applicable NEO-102 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 pancreatic ductal adenocarcinoma not applicable Gemcitabine + JQ1 Preclinical - Pdx Actionable In a preclinical study, combination treatment with JQ1 and Gemzar (gemcitabine) resulted in tumor microenvironment alterations and greater reductions of tumor growth and weight when compared to Gemzar (gemcitabine) alone in patient derived xenograft (PDX) models of pancreatic ductal adenocarcinoma (PMID: 27528027). 27528027
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 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 neuroendocrine tumor not applicable Sulfatinib Phase I Actionable In a Phase I trial, HMPL-012 (Sulfatinib) demonstrated an objective response rate in 44% (8/18) of patients with neuroendocrine tumors (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A1). detail...
Unknown unknown 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 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 angiosarcoma not applicable Bevacizumab + Paclitaxel Phase II Actionable In a Phase II trial, angiosarcoma patients treated with Avastin (bevacizumab), in combination with Taxol (paclitaxel), showed no improvement in PFS and OS compared to Taxol (paclitaxel) alone and resulted in higher toxicity (PMID: 26215950). 26215950
Unknown unknown Advanced Solid Tumor not applicable Cobimetinib Phase I Actionable In a Phase I trial, Cotellic (cobimetinib) treatment resulted in stable disease for five months or more in five patients with advanced solid tumors (PMID: 27424159). 27424159 detail...
Unknown unknown renal cell carcinoma no benefit MK2206 Phase II Actionable In a Phase II trial, MK2206 treatment resulted in shorter progression free survival compared to Afinitor (everolimus) (3.68 vs 5.98 months) in patients with renal cell carcinoma (PMID: 28049139). 28049139
Unknown unknown colorectal cancer not applicable WAY-600 Preclinical Actionable In a preclinical study, WAY-600 induced cell cycle arrest and inhibited cell proliferation of colorectal cancer cells in culture (PMID: 19584280). 19584280
Unknown unknown Advanced Solid Tumor not applicable Carboplatin + VX-970 Phase I Actionable In a Phase I trial, VX-970 and Paraplatin (carboplatin) combination treatment resulted in partial response in 7% (1/15) and stable disease in 53% (8/15) of patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2504)). detail...
Unknown unknown triple-receptor negative breast cancer not applicable SY-1365 Preclinical - Pdx & cell culture Actionable In a preclinical study, SY-1365 induced rapid apoptosis in triple-receptor negative breast cancer cells in culture, and resulted in tumor growth inhibition in patient-derived xenograft models (Proceedings of the AACR, Vol 58, April 2017, Abstract # 1151). 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 gastrointestinal stromal tumor not applicable DCC-2618 Phase I Actionable In a Phase I study, DCC-2618 treatment resulted in an overall response rate of 16% (16/99) in patients with drug resistant gastrointestinal stromal tumors, 71% (55/77) of analyzed patients harbored baseline KIT or PDGFRA activating mutations (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 11511-11511; NCT02571036). detail...
Unknown unknown chondrosarcoma not applicable Doxorubicin + Nilotinib Phase I Actionable In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin resulted in 5 stable disease and 2 progressive disease in 7 patients with chondrosarcoma, with a median progression-free survival of 14 months and a median overall survival of 25 months (PMID: 30037815; NCT02587169). 30037815
Unknown unknown B-cell childhood acute lymphoblastic leukemia not applicable Tisagenlecleucel FDA approved Actionable In a Phase II trial (ELIANA) that supported FDA approval, Kymriah (tisagenlecleucel) treatment led to complete remission or complete remission with incomplete blood count recovery in 83% (52/63) of pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (22nd Congress of EHA, June 2017, Abstract S476; NCT02435849). detail... detail... detail...
Unknown unknown head and neck squamous cell carcinoma not applicable Temsirolimus Phase II Actionable In a Phase II trial, treatment with Torisel (temsirolimus) resulted in disease stabilization in 57.6% (19/33) and tumor shrinkage in 39.4% (13/33) of patients with head and neck squamous cell carcinoma (PMID: 25527417). 25527417
Unknown unknown breast cancer not applicable MEDI5117 + Docetaxel 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 multiple myeloma not applicable Dexamethasone + pegylated liposomal-doxorubicin + Selinexor Phase I Actionable In a Phase I trial, treatment with the combination of Selinexor (KPT-330), Doxil (pegylated liposomal doxorubicin), and Dexamethasone resulted in very good partial response in 20% (2/10), partial response in 20% (2/10), minimal response in 20% (2/10), and stable disease in 30% (3/10) of patients with relapsed or refractory multiple myeloma (J Clin Oncol 34, 2016 (suppl; abstr 8013)). detail...
Unknown unknown Advanced Solid Tumor not applicable RO5126766 Phase I Actionable In a Phase I trial, RO5126766 demonstrated safety and preliminary efficacy in patients with a variety of solid tumors (PMID: 22761467). 22761467
Unknown unknown Advanced Solid Tumor not applicable RO5126766 Phase I Actionable In a Phase I trial, RO5126766 demonstrated safety and some preliminary activity in Japanese patients with advanced solid tumors, with 42% (5/12) of patients achieving stable disease (PMID: 23860959). 23860959
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 AIM-100 Preclinical Actionable In a preclinical study, AIM-100 inhibited growth of breast cancer cells in culture (PMID: 22322295). 22322295
Unknown unknown multiple myeloma not applicable GNE-272 Preclinical - Cell culture Actionable In a preclinical study, GNE-272 treatment resulted in anti-proliferative activity in multiple myeloma cell lines in culture (PMID: 27682507). 27682507
Unknown unknown renal cell carcinoma 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 (PMID: 21447719). 21447719
Unknown unknown cervical cancer not applicable Tozasertib Preclinical - Cell line xenograft Actionable In a preclinical study, Tozasertib (VX-680) inhibited growth of cervical cancer cell lines in culture, and inhibited tumor growth in cervical cancer cell line xenograft models (PMID: 27082306). 27082306
Unknown unknown bladder carcinoma in situ not applicable Oportuzumab monatox Phase II Actionable In a Phase II trial, treatment with Oportuzumab monatox (VB4-845) was well-tolerated and resulted in complete response in 44% (20/45) of patients with bladder carcinoma in situ refractory to bacillus Calmette-Guerin (BCG) therapy (PMID: 22998907; NCT00462488) 22998907
Unknown unknown Hodgkin's lymphoma not applicable Ixazomib + JQ1 Preclinical Actionable In a preclinical study, Ixazomib (MLN9708) and JQ1 combination treatment resulted in increased apoptosis in Hodgkin's lymphoma cells in culture (PMID: 26988986). 26988986
Unknown unknown 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 colon cancer not applicable Decitabine Preclinical Actionable In a preclinical study, Decitabine, an DNA methyltransferase inhibitor, inhibited cell growth in colon cancer cell lines (PMID: 24874286). 24874286
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 melanoma not applicable Epacadostat Phase I Actionable In a Phase I trial, treatment with Epacadostat resulted in stable disease in 13% (7/52) of patients with advanced solid tumors for greater than or equal to 16 weeks, including two patients with melanoma who previously failed on immunotherapy (PMID: 28053021). 28053021
Unknown unknown lung small cell carcinoma not applicable Talazoparib Phase I Actionable In a Phase I trial, Talazoparib (BMN-673) treatment in patients with lung small cell carcinoma resulted in an objective response rate of 9% (2/23), including two patients with a partial response, and four patients with stable disease for at least 16 weeks (PMID: 28242752). 28242752
Unknown unknown glioblastoma multiforme not applicable Pexidartinib Phase II Actionable In a Phase II trial, PLX3397 in combination with radiation therapy and Temodar (temozolomide) demonstrated safety and improved efficacy over standard therapy, resulted in a median progression free survival of 9.7 months and an estimated overall survival of 25.1 months in newly diagnosed glioblastoma multiforme patients (Neuro Oncol (2016) 18 (suppl 6): vi6.). detail...
Unknown unknown glioblastoma multiforme not applicable MPS1-IN-3 + Vincristine Preclinical Actionable In a preclinical study, MPS1-IN-3, in combination with Oncovvin (vincristine), inhibited cell growth of glioblastoma cells in culture and in xenograft GBM models (PMID: 23940287). 23940287
Unknown unknown leiomyosarcoma not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) treatment resulted in twelve-week progression free survival in 44% (4/9) of leiomyosarcoma patients (PMID: 27502708). 27502708
Unknown unknown 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 ovarian cancer not applicable Cediranib + Olaparib Phase I Actionable In a Phase I clinical trial, the combination therapy of Cediranib (AZD-2171) and Lynparza (olaparib) demonstrated safety and efficacy in patients with ovarian cancer (J Clin Oncol 32:5s, 2014 (suppl; abstr LBA5500)). detail...
Unknown unknown osteosarcoma not applicable VCN-01 Preclinical Actionable In a preclinical study, treatment with VCN-01 resulted in decreased viability of osteosarcoma cell lines in culture (PMID: 26603261). 26603261
Unknown unknown Advanced Solid Tumor not applicable OPB-31121 Phase I Actionable In a Phase I trial, OPB-31121 treatment resulted in stable diseases in 44% (8/18) of patients with advanced solid tumors, and tumor shrinkage in 2 patients (1 colon cancer, 1 rectal cancer) (PMID: 25715763). 25715763
Unknown unknown Advanced Solid Tumor not applicable SSR128129E Preclinical - Cell line xenograft Actionable In a preclinical study, SSR128129E inhibited tumor growth in several solid tumor cell line xenograft models (PMID: 23597562). 23597562
Unknown unknown non-small cell lung carcinoma not applicable Sunitinib Phase III Actionable In a Phase III trial, Sutent (sunitinib) as maintenance therapy resulted in improved progression free survival (4.3 vs 2.6 months) but not overall survival (11.7 vs 12.1 months) compared to placebo in patients with stage IIIB/IV non-small cell lung cancer (PMID: 28161554). 28161554
Unknown unknown non-small cell lung carcinoma not applicable Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) treatment in non-small cell lung cancer patients resulted in an objective response rate of 11.1% (7/63), stable disease in 28.6% (18/63), and a PFS of 12 weeks and OS of 23.4 weeks (PMID: 18235126). 18235126
Unknown unknown 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 multiple myeloma not applicable Panobinostat + Bortezomib + Dexamethasone FDA approved Actionable In clinical trials that supported FDA approval, treatment with Farydak (panobinostat), in combination with Velcade (Bortezomib) and dexamethasone, extended progression-free survival to 12 months in patients with multiple myeloma (PMID: 26362997). 26362997
Unknown unknown gastrointestinal system cancer no benefit PX-12 Phase Ib/II Actionable In a Phase Ib trial, PX-12 did not demonstrate clinical activity in patients with advanced gastrointestinal tumors (PMID: 22711542). 22711542
Unknown unknown Advanced Solid Tumor not applicable BAY 1238097 Clinical Study Actionable In a Phase I trial, BAY 1238097 therapy resulted in zero objective responses and stable disease in 25% (2/8) of patients with refractory advanced solid tumors; due to high toxicity the trial was terminated prematurely (PMID: 30711772; NCT02369029). 30711772
Unknown unknown triple-receptor negative breast cancer not applicable BAY1161909 + Paclitaxel Preclinical Actionable In a preclinical study, BAY1161909, in combination with Taxol (paclitaxel), had increased efficacy in xenograft models of triple-negative breast cancer compared to Taxol (paclitaxel) alone, resulting in complete tumor regression (PMID: 26832791). 26832791
Unknown unknown glioblastoma multiforme no benefit MP7 Preclinical - Cell culture Actionable In a preclinical study, MP7 treatment did not result in significant inhibition of cell proliferation and showed minimal change in cell viability in a glioblastoma cell line in culture, thus, providing no benefit (PMID: 27797168). 27797168
Unknown unknown diffuse large B-cell lymphoma no benefit Fostamatinib Phase II Actionable In a Phase II clinical trial, fostamatinib demonstrated safety, but limited efficacy in patients with diffuse large B-cell lymphoma, with an objective response rate of 3% (2/68) and stable disease in 10% (7/68) of patients (PMID: 26707592). 26707592
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 non-Hodgkin lymphoma not applicable Bendamustine + Rituximab + Veliparib Phase I Actionable In a Phase I trial, seven patients with non-Hodgkin lymphoma treated with a combination of Veliparib (ABT-888), Bendamustine, and Rituxan (rituximab) demonstrated an overall response rate of 86% (6/7) and a complete response rate of 71% (5/7), and a progression free survival of 14.2 months (PMID: 28314788; NCT01326702). 28314788
Unknown unknown 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 non-small cell lung carcinoma not applicable Motesanib + Carboplatin 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 hematologic cancer not applicable AS703569 Phase I Actionable In a Phase I clinical trial, Cenisertib (AS703569) demonstrated safety and preliminary efficacy in patients with advanced hematological malignancies (Blood 2008 112:2963). detail...
Unknown unknown renal cell carcinoma not applicable Dalantercept + Axitinib 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 nasopharynx carcinoma not applicable Camrelizumab Phase I Actionable In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity, resulted in partial response in 33.3% (1/3) of patients with nasopharynx cancer (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). detail...
Unknown unknown multiple myeloma not applicable Dexamethasone + Filanesib Phase Ib/II Actionable In a Phase I/II trial, treatment with the combination of Filansenib (ARRY-520) and dexamethasone in a Phase II cohort resulted in an overall response rate of 15% (8/54), clinical benefit rate of 20% (11/54), and median overall survival of 10.7 months in patients with refractory or relapsed multiple myeloma (PMID: 28817190, NCT00821249). 28817190
Unknown unknown head and neck squamous cell carcinoma not applicable Cetuximab + Prexasertib Preclinical - Cell culture Actionable In a preclinical study, the combination of Erlotinib (cetuximab) and Prexasertib (LY2606368 resulted in greater decreased cell proliferation in head and neck squamous cell carcinoma cells in culture compared to either agent alone (PMID: 28138028). 28138028
Unknown unknown Her2-receptor positive breast cancer not applicable S63845 Preclinical - Cell culture Actionable In a preclinical study, S63845 decreased viability of an ERBB2 (HER2)-amplified breast cancer cell line and ERBB2 (HER2)-amplified patient-derived xenograft (PDX) tumor cells in culture (PMID: 28768804). 28768804
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 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 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 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 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 lung carcinoma not applicable BKM120 + Everolimus Preclinical Actionable In a preclinical study, Buparlisib (BKM120) in combination with Afinitor (everolimus) inhibited tumor growth in mouse lung cancer xenograft models (PMID: 22781393). 22781393
Unknown unknown 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 diffuse large B-cell lymphoma not applicable