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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 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 ovarian cancer not applicable Conatumumab + Ganitumab Phase Ib/II Actionable In a Phase Ib/II clinical trial, Ganitumab and Conatumumab combination treatment resulted in stable disease in 56% (5/9) of patients with ovarian cancer (PMID: 24816908). 24816908
Unknown unknown ovarian cancer not applicable Pamiparib + Tislelizumab Case Reports/Case Series Actionable In a Phase Ib trial, the combination therapy of Tislelizumab (BGB-A317) and Pamiparib (BGB-290) in ovarian cancer patients resulted in 1 complete response and 5 partial responses (J Clin Oncol 35, 2017 (suppl; abstr 3013)). detail...
Unknown unknown 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 uterine corpus sarcoma not applicable Pazopanib Clinical Study Actionable In a retrospective clinical study, Votrient (pazopanib) treatment resulted in objective response in 29% (10/35) and stable disease in 31% (11/35) of patients with uterine sarcoma, with median progression-free survival of 5.8 months and overall survival of 20.0 months (PMID: 29185261). 29185261
Unknown unknown 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 alveolar rhabdomyosarcoma not applicable Infigratinib Preclinical - Pdx Actionable In a preclinical study, Infigratinib (BGJ398) resulted in antitumor activity in an alveolar rhabdomyosarcoma patient derived xenograft (PDX) model (PMID: 24687871). 24687871
Unknown unknown acute myeloid leukemia no benefit AR-42 + Decitabine Phase I Actionable In a Phase I trial, AR-42 and Dacogen (decitabine) combination treatment in patients with acute myeloid leukemia resulted in an overall response rate of 23.1% (3/13), including one patient with complete remission and two patients with complete remission with incomplete count recovery, but the trial did not meet its biological endpoint for safety and dosing (PMID: 32037935; NCT01798901). 32037935
Unknown unknown Advanced Solid Tumor not applicable PKI-402 Preclinical - Cell culture Actionable In a preclinical study, PKI-402 inhibited growth of several human solid tumor cell lines in culture (PMID: 20371716). 20371716
Unknown unknown lung small cell carcinoma not applicable Carboplatin + Etoposide + Roniciclib Phase II Actionable In a Phase II trial, the combination of Roniciclib (BAY1000394) plus chemotherapy regimen, Paraplatin (carboplatin) or Platinol (cisplatin) and Vepesid (etoposide) (n=71), did not meet its primary endpoint for progression-survival in patients with small cell lung cancer (4.9 mo vs 5.5 mo) when compared to placebo plus chemotherapy (n=71), and showed an unfavorable risk-benefit profile, therefore, leading to premature termination of the study (PMID: 30677506; NCT02161419). 30677506
Unknown unknown lung small cell carcinoma not applicable Carboplatin + Etoposide + Roniciclib Phase Ib/II Actionable In a Phase Ib/II trial, first-line treatment with the combination of Roniciclib (BAY1000394) and either carboplatin plus etoposide or cisplatin plus etoposide demonstrated tolerability and resulted in a response rate of 81.4% (35/43; all partial responses), median OS of 12.6 mo, and median PFS of 7.3 mo in extensive disease small cell lung cancer patients; however due to a safety signal in a related trial further development of Roniciclib (BAY1000394) was discontinued (PMID: 30089585; NCT01573338). 30089585
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 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 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 ovarian cancer not applicable Atezolizumab + Hu5F9-G4 Phase I Actionable In a Phase Ib trial, Tecentriq (atezolizumab) and Magrolimab (Hu5F9-G4) combination therapy demonstrated acceptable safety, and resulted in stable disease as best response in 56% (10/18) of patients with platinum-resistant ovarian cancer (J Clin Oncol 38, 2020 (suppl 5; abstr 18); NCT03558139). detail...
Unknown unknown 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 Thymoquinone Preclinical - Cell line xenograft Actionable In a preclinical study, Thymoquinone (TQ) inhibited growth, migration, and invasive behavior of human breast cancer cell lines in culture, and inhibited tumor growth and metastasis in mouse breast cancer cell line xenografts (PMID: 26023736). 26023736
Unknown unknown pancreatic ductal adenocarcinoma not applicable LMB-100 + Nab-paclitaxel Phase Ib/II Actionable In a Phase I/II trial, the combination of LMB-100 and Abraxane (nab-paclitaxel) led to a greater than 50% decrease in CA19-9 in 41% (7/17) of patients with pancreatic ductal adenocarcinoma, and one patient experienced an objective partial response, however, the combination therapy was not well tolerated (PMID: 31792036; NCT02810418). 31792036
Unknown unknown lung non-squamous non-small cell carcinoma not applicable Carboplatin + Linifanib + Paclitaxel Phase II Actionable In a Phase II clinical, Linifanib (ABT-869), in combination with Taxol (paclitaxel) and Paraplatin (carboplatin), increased progression free survival in patients with nonsquamous non-small cell lung cancer (PMID: 25559798). 25559798
Unknown unknown Advanced Solid Tumor not applicable Panvotinib-401 Preclinical Actionable In a preclinical study, Panvotinib-401 demonstrated cancer-selective cytotoxicity in transformed cell lines in culture (AACR, Cancer Res: April 2016; Volume 57, Abstract #3896). detail...
Unknown unknown hepatocellular carcinoma not applicable Regorafenib FDA approved Actionable In a Phase III trial (RESORCE) that supported FDA approval, treatment with Stivarga (regorafenib) following Nexavar (sorafenib) treatment resulted in improved overall survival (10.6 vs 7.8 months, HR=0.63) compared to Nexavar (sorafenib) followed by placebo in patients with hepatocellular carcinoma (PMID: 27932229; NCT01774344). detail... 27932229
Unknown unknown triple-receptor negative breast cancer not applicable CPI-637 + HY-16462 Preclinical - Cell culture Actionable In a preclinical study, CPI-637 and HY-16462 treatment synergistically inhibited growth of a triple-negative breast cancer cell line in culture (PMID: 31000582). 31000582
Unknown unknown brain glioma not applicable Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) decreased cell proliferation and induced apoptosis in mouse models of glioma (PMID: 21926191). 21926191
Unknown unknown Advanced Solid Tumor not applicable Abexinostat + Pazopanib Phase Ib/II Actionable In a Phase Ib/II trial, the combination of Abexinostat (PCI-24781) and Votrient (pazopanib) in advanced solid tumor patients resulted in a clinical benefit rate of 37% (16/43), a median response duration of 9.1 months, and 8 patients of 43 achieved stable disease or durable response for greater than 12 months (PMID: 28221861). 28221861
Unknown unknown Advanced Solid Tumor not applicable Roniciclib Phase I Actionable In a Phase I trial, treatment with Roniciclib (BAY 1000394) on a 3 days on/4 days off dosing schedule demonstrated safety and resulted in a disease control rate of 32.7% (34/104), with a response rate of 1% (1/104; 1 partial response) and stable disease in 31.7% (33/104) of patients with advanced solid tumors (PMID: 28463960; NCT01188252). detail... 28463960
Unknown unknown glioblastoma multiforme not applicable BMS-906024 Preclinical Actionable In a preclinical study, the pan-Notch inhibitor, BMS-906024, inhibited growth of glioblastoma xenografts (PMID: 26005526). 26005526
Unknown unknown transitional cell carcinoma not applicable Avelumab FDA approved Actionable In a Phase III trial (JAVELIN Bladder 100) that supported FDA approval, addition of maintenance Bavencio (avelumab) to best supportive care (BSC) significantly improved overall survival compared to BSC alone (21.4 vs 14.3 mo, HR=0.69, p=0.0005) in patients with advanced urothelial carcinoma (J Clin Oncol 38, (Jun 2020) no. 18_suppl; NCT02603432). detail... detail...
Unknown unknown transitional cell carcinoma not applicable Avelumab FDA approved Actionable In a Phase I trial (JAVELIN Solid Tumor) that supported FDA approval, Bavencio (avelumab) demonstrated safety and resulted in a response rate of 17% (27/161; 9 complete responses and 18 partial responses), a median progression-free survival of 6.3 weeks, and a median overall survival of 6.5 months in patients with platinum-refractory metastatic urothelial carcinoma (PMID: 29217288; NCT01772004). detail... 29217288
Unknown unknown prostate cancer not applicable Olaparib Phase II Actionable In a Phase II clinical trial, 86.7% (13/15) of metastatic castration-resistant prostate cancer patients with mutations in DNA repair genes demonstrated response to Lynparza (olaparib) treatment (Cancer Res August 1, 2015 75:CT322). detail...
Unknown unknown malignant mesothelioma not applicable MGD013 Case Reports/Case Series Actionable In a Phase I trial, MGD013 treatment resulted in a partial response in a patient with mesothelioma (J Clin Oncol 38: 2020 (suppl; abstr 3004); NCT03219268). detail...
Unknown unknown glioblastoma multiforme not applicable Dovitinib Phase I Actionable In a Phase I trial, Dovitinib (TKI258) treatment resulted in a progression free survival rate at 6 months of 16.7% (2/12) in patients with recurrent glioblastoma (PMID: 27100354). 27100354
Unknown unknown 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 gastroesophageal junction adenocarcinoma no benefit Oxaliplatin + Ramucirumab + 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 ovarian cancer not applicable Rucaparib FDA approved Actionable In a Phase III trial that supported FDA approval, Rubraca (rucaparib) maintenance therapy significantly improved median progression-free survival compared to placebo (10.8 vs 5.4 mo, HR=0.36, p<0.0001) in patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who responded to platinum-based therapy (PMID: 28916367; NCT01968213). 28916367
Unknown unknown chronic lymphocytic leukemia not applicable LAM-002A Phase I Actionable In a Phase I trial, LAM-002A demonstrated safety and preliminary efficacy, resulted in prolonged stable disease in 1 of 4 patients with chronic lymphocytic leukemia (Blood 2017 130 (Suppl 1):4119). detail...
Unknown unknown multiple myeloma not applicable Bortezomib + Dexamethasone + Ulocuplumab Phase I Actionable In a Phase I trial, Ulocuplumab (BMS-936564) in combination with Velcade (bortezomib) and Adexone (dexamethasone) demonstrated safety and preliminary efficacy, resulted in an overall response rate of 25.0% (4/16) and a clinical benefit rate of 50% (8/16) in patients with relapsed multiple myeloma (PMID: 31672767). 31672767
Unknown unknown 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 colon adenocarcinoma not applicable AGI-134 Preclinical - Cell culture Actionable In a preclinical study, AGI-134 treatment induced cell death of colon adenocarcinoma cells in the presence of normal human serum in culture (PMID: 31889898). 31889898
Unknown unknown 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 multiple myeloma not applicable BC2059 + Bortezomib Preclinical - Patient cell culture Actionable In a preclinical study, the combination of BC2059 and Velcade (bortezomib) worked additively or synergistically in several human multiple myeloma cell lines and primary multiple myeloma cells with activated canonical Wnt signaling in culture (PMID: 28500235). 28500235
Unknown unknown multiple myeloma not applicable INCB054329 + Pemigatinib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of INCB054329 and Pemazyre (pemigatinib) decreased MYC expression and FGFR3 signaling and inhibited tumor growth in a t(4;14)-positive myeloma cell line xenograft model, demonstrating increased efficacy over either agent alone (PMID: 30206163). 30206163
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 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 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 lymphoid leukemia not applicable Ponatinib FDA approved Actionable In a Phase II clinical trial which supported FDA approval, Iclusig (ponatinib) was effective in promoting disease regression in 52% of patients with accelerated phase chronic myeloid leukemia, 31% of patients with blast phase chronic myeloid leukemia, and 41% of patients with Philadelphia chromosome positive acute lymphoblastic leukemia (PMID: 23935038). 23935038 detail...
Unknown unknown oral squamous cell carcinoma not applicable Temsirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Torisel (temsirolimus) inhibited proliferation and migration of oral squamous cell carcinoma cells in culture and suppressed tumor growth in cell line xenograft models of human oral squamous cell carcinoma (PMID: 20858724). 20858724
Unknown unknown duodenum adenocarcinoma not applicable Vandetanib Preclinical Actionable In preclinical studies, Caprelsa (vandetinib) reduced the number and size of polyps in mouse models of intestinal cancer and may be a beneficial strategy in early intestinal cancer (PMID: 18347145). 18347145
Unknown unknown pancreatic cancer not applicable Gemcitabine + Nab-paclitaxel + Necuparanib Phase I Actionable In a Phase I trial, combination treatment consisted of Gemzar (gemcitabine), Abraxane (nab-paclitaxel), and Necuparanib resulted in partial response in 50% (8/16), stable disease in 38% (6/16), and median overall survival of 16.0 months in patients (pts) with metastatic pancreatic cancer (J Clin Oncol 34, 2016 (suppl; abstr 4117)). detail...
Unknown unknown 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 multiple myeloma not applicable Dinaciclib + Veliparib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Veliparib (ABT-888) and Dinaciclib (SCH 727965) had a synergistic effect on decreasing survival of multiple myeloma cell lines in culture, and resulted in delayed tumor growth and improved survival of multiple myeloma cell line xenograft models compared to either agent alone (PMID: 26719576). 26719576
Unknown unknown prostate cancer not applicable Birabresib Preclinical - Cell line xenograft Actionable In a preclinical study, Birabresib (OTX015) treatment inhibited tumor growth in a cell line xenograft model of prostate cancer (PMID: 27274052). 27274052
Unknown unknown acute myeloid leukemia not applicable INCB053914 Preclinical Actionable In a preclinical study, acute myeloid leukemia cells were sensitive to INCB053914, resulting in inhibition of cell proliferation (Cancer Res August 1, 2015 75; 5416). detail...
Unknown unknown colon cancer not applicable AsiDNA Preclinical - Cell culture Actionable In a preclinical study, AsiDNA inhibited survival of colon cancer cell lines in culture (PMID: 27559053). 27559053
Unknown unknown multiple myeloma not applicable bb2121 Phase II Actionable In a Phase II trial (KarMMa), Idecabtagene vicleucel (bb2121) treatment resulted in an objective response of 73% (94/128) in patients with relapsed or refractory multiple myeloma, with a median duration of response of 10.6 months and a median progression-free survival of 8.6 months (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 8503-8503; NCT03361748). detail...
Unknown unknown multiple myeloma not applicable bb2121 Phase I Actionable In a Phase I trial, bb2121 treatment in multiple myeloma patients resulted in an overall response rate of 78% (7/9), including two patients who experienced complete remission, four patients who achieved partial response, and one patient who experienced stable disease (EORTC-NCI-AACR Symposium, 2016, Abstract #14). detail...
Unknown unknown 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 glioblastoma multiforme not applicable CYNK-001 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with CYNK-001 resulted in tumor reduction in an orthotopic mouse model of glioblastoma multiforme (Neuro-Oncology, Vol. 21, Issue Suppl_6, Nov. 2019, Page vi85, EXTH-16). detail...
Unknown unknown prostate cancer not applicable AB928 + Doxorubicin Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of AB928 and Adriamycin (doxorubicin) resulted in a increase in tumor cell specific CD8+ T cells and increased tumor growth inhibition compared to chemotherapy alone in prostate cancer cell line xenograft models (European Journal of Cancer, Vol 92, S14-S15). detail...
Unknown unknown breast cancer not applicable PD-0325901 Phase I Actionable In a Phase I trial, PD-0325901 demonstrated some efficacy, however, the dose administered resulted in a significant degree of toxicity (PMID: 21516509). 21516509
Unknown unknown Advanced Solid Tumor not applicable Utomilumab Phase I Actionable In a Phase I trial, Utomilumab (PF-05082566) treatment resulted in an overall objective response rate of 3.8% (2/53), a median progression-free survival of 1.7 months, and a median overall survival of 11.2 months in patients with advanced solid tumors (PMID: 29549159; NCT01307267). 29549159
Unknown unknown 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 ovarian cancer not applicable Avelumab Phase I Actionable In a Phase I trial (JAVELIN), Bavencio (avelumab) treatment resulted in objective response in 9.6% (12/125, 1 complete response, 11 partial response) of patients with recurrent or refractory ovarian cancer, with a 1-year progression-free survival rate of 10.2% and a median overall survival of 11.2 months; response did not correlate with tumor CD274 (PD-L1) status (PMID: 30676622; NCT01772004). 30676622
Unknown unknown colon cancer not applicable CVX-060 + Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of CVX-060 and Sutent (suntinib) resulted in increased tumor growth inhibition compared to either agent alone in a colon cancer cell line xenograft model (PMID: 21233403). 21233403
Unknown unknown glioblastoma multiforme not applicable G-TPP + Navitoclax Preclinical - Pdx & cell culture Actionable In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Navitoclax (ABT-263) synergistically inhibited viability of established lines and patient-derived glioblastoma cells in culture, and inhibited tumor growth in cell line and patient-derived xenograft models (PMID: 28522750). 28522750
Unknown unknown malignant glioma not applicable PV1019 + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, glioma cells treated with PV1019 before and after radiotherapy resulted in greater cell death in culture than compared to radiation treatment alone (PMID: 19741151). 19741151
Unknown unknown breast cancer not applicable AJI-214 Preclinical - Cell culture Actionable In a preclinical study, AJI-214 inhibited invasion and induced apoptosis of breast cancer cells in culture (PMID: 24930769). 24930769
Unknown unknown pancreatic adenocarcinoma not applicable Adavosertib + Gemcitabine + Radiotherapy Phase I Actionable In a Phase I trial, Adavosertib (MK-1775) in combination with Gemzar (gemcitabine) and radiation therapy was tolerable, resulted in a median overall survival of 21.7 months and a median progression-free survival of 9.4 months in patients with advanced pancreatic adenocarcinoma (PMID: 31398082; NCT02037230). 31398082
Unknown unknown renal cell carcinoma not applicable Bevacizumab + Everolimus Phase II Actionable In a Phase II trial, of 34 evaluable patients with non clear cell RCC treated with a combination of Afinitor (everolimus) and Avastin (bevacizumab), 9 patients had a partial response, 1 patient experienced a complete response, and 15 had stable disease, and the median PFS was 11 months (PMID: 27601542). 27601542
Unknown unknown follicular lymphoma not applicable Ibrutinib + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of Imbruvica (ibrutinib) and Venclexta (venetoclax) resulted in a synergistic effect, demonstrating growth suppression in follicular lymphoma cell lines in culture (PMID: 28428442). 28428442
Unknown unknown ovarian cancer not applicable AZD7648 + Olaparib Preclinical - Cell culture Actionable In a preclinical study, AZD7648 and Lynparza (olaparib) combination treatment inhibited viability of ovarian cancer cell lines harboring wild-type ATM in culture (PMID: 31699977). 31699977
Unknown unknown 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 Advanced Solid Tumor not applicable SL-801 Preclinical - Cell line xenograft Actionable In a preclinical study, SL-801 resulted in tumor growth inhibition in xenograft models with various advanced solid tumors (Journal of Clinical Oncology 33, no. 15_suppl). detail...
Unknown unknown peritoneal carcinoma no benefit Motolimod + Pegylated liposomal-doxorubicin Phase II Actionable In a Phase II trial, the inclusion of Motolimod (VTX-2337) with Doxil (pegylated liposomal-doxorubicin) did not result in improved survival in patients with either ovarian, fallopian tube, or primary peritoneal carcinoma (PMID: 28453702; NCT01666444). 28453702
Unknown unknown glioblastoma multiforme not applicable BLZ945 + Buparlisib Preclinical Actionable In a preclinical study, the combination of Buparlisib (BKM120) and BLZ945 compared to BLZ945 alone resulted in enhanced survival and tumor regression in transgenic mouse models of glioblastoma (PMID: 27199435). 27199435
Unknown unknown lung cancer not applicable Nintedanib Preclinical - Cell line xenograft Actionable In a preclinical study, Nintedanib, alone or with chemotherapy, inhibited tumor growth in cell line xenograft models of lung and pancreatic cancer but not in cell culture (PMID: 23729403). 23729403
Unknown unknown lung non-small cell carcinoma not applicable Cisplatin + PJ34 Preclinical - Cell culture Actionable In a preclinical study, the combination of Platinol (cisplatin) and PJ34 worked synergistically to induce cell death in non-small cell lung carcinoma cells in culture (PMID: 23428903). 23428903
Unknown unknown breast cancer not applicable Fluoxymesterone Case Reports/Case Series 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 Advanced Solid Tumor not applicable XL147 Phase I Actionable In a Phase I study, 25/56 (43.9%) of patients with advanced solid tumors had stable disease as a best response to treatment with XL147, and one patient with NSCLC showed a partial response to XL147 (PMID: 24166903). 24166903
Unknown unknown 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 endometrial cancer not applicable Cisplatin + ETP-46464 Preclinical - Cell culture Actionable In a preclinical study, ETP-46464 increased the sensitivity of endometrial cancer cell lines to Platinol (cisplatin) in culture (PMID: 25560806). 25560806
Unknown unknown pancreatic endocrine carcinoma no benefit BEZ235 Phase II Actionable In a Phase II clinical trial, BEZ235 was not well-tolerated and demonstrated modest anti-tumor activity in pancreatic neuroendocrine tumor patients who had progressed on Afinitor (everolimus), with a 51.6% (16/31) progression-free survival rate at 16 weeks (PMID: 26851029). 26851029
Unknown unknown ovarian cancer not applicable Sunitinib Phase Ib/II Actionable In a Phase II trial, Sutent (sunitinib) treatment in ovarian cancer patients resulted in an increased PFS and a response rate of 16.7% (6/36) in those that received Sutent (sunitinib) continuously and a a response rate of 5.4% (2/37) in those that received the drug non-continuously (PMID: 22377563, PMID: 24070205). 22377563 24070205
Unknown unknown Advanced Solid Tumor not applicable Rigosertib Sodium Phase I Actionable In a Phase I study, Rigosertib (ON 01910.Na) demonstrated both safety and preliminary efficacy, resulted in a best overall response of stable disease in 41% (9/22) of patients with advanced solid tumors (PMID: 23841031; NCT01538537). 23841031
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 JIB-04 Preclinical Actionable In a preclinical study, JIB-04, a pan Jumonji histone demethylase inhibitor, reduced cell proliferation, and induced apoptosis in multiple cancer cell types while reducing tumor burden and increasing survival in mouse xenograft models (PMID: 23792809). 23792809
Unknown unknown colon adenocarcinoma not applicable CC-223 Phase I Actionable In a Phase I trial, CC-223 demonstrated safety and some efficacy in patients with solid tumors, including colon cancer (J Clin Oncol 31, 2013 (suppl; abstr 2606). detail...
Unknown unknown leukemia not applicable DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, DCBCI0901 inhibited tumor growth greater than 65% in a leukemia cell line xenograft model (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown acute myeloid leukemia not applicable SY-1365 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, SY-1365 and Venclexta (venetoclax) synergistically induced apoptosis in acute myeloid leukemia cells in culture (Proceedings of the AACR, Vol 58, April 2017, Abstract # 1151). detail...
Unknown unknown lung carcinoma not applicable SF2523 Preclinical Actionable In a preclinical study, SF2523 decreased tumor immunosuppression, increased T-lymphocyte infiltration, and reduced tumor growth in syngeneic mouse lung carcinoma models (PMID: 31018997). 31018997
Unknown unknown lung carcinoma not applicable YW3-56 Preclinical Actionable In a preclinical study, YW3-56 inhibited proliferation of lung carcinoma cell lines in culture, independent of TP53 status (PMID: 25612620). 25612620
Unknown unknown Advanced Solid Tumor not applicable Docetaxel + VIC-1911 Preclinical Actionable In a preclinical study, TAS-119 demonstrated synergistic effects with Taxol (paclitaxel) or Taxotere (docetaxel) in multiple tumor cell lines by promoting apoptosis (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr A268). detail...
Unknown unknown 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 melanoma not applicable KRT-232 + Trametinib Phase I Actionable In a Phase I trial, the combination therapy of KRT-232 (AMG 232) and Mekinist (trametinib) in 15 patients with metastatic cutaneous melanoma without a BRAF V600 mutation resulted in a partial response in 2 patients, stable disease in 11 patients, and progressive disease in 2 patients, and of the 15 patients, 11 experienced tumor reduction (J Clin Oncol 35, 2017 (suppl; abstr 2575)). detail...
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 prostate cancer not applicable AZD8186 Phase I Actionable In a Phase I trial, AZD8186 demonstrated preliminary activity in patients with select solid tumors, including castration-resistant prostate cancer (CRPC), with one CRPC patient achieving stable disease and symptomatic improvement (Cancer Res August 1 2015 (75) (15 Supplement) CT329). detail...
Unknown unknown lung non-small cell 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 lung non-small cell carcinoma not applicable XL147 Phase I Actionable In a Phase I study, 25/56 (43.9%) of patients with advanced solid tumors had stable disease as a best response to treatment with XL147, and one patient with NSCLC showed a partial response to XL147 (PMID: 24166903). 24166903
Unknown unknown neuroblastoma not applicable Vorinostat Preclinical - Cell line xenograft Actionable In a preclinical study, Zolinza (vorinostat) displayed efficacy in human neuroblastoma cell lines in culture and in xenografts, including those resistant to Adriamycin (doxorubicin) (PMID: 22703804). 22703804
Unknown unknown lung non-small cell carcinoma not applicable Ro 31-8220 Preclinical - Cell culture Actionable In a preclinical study, Ro 31-8220 decreased CREB signaling, and induced apoptosis and inhibited growth of non-small cell lung cancer cells in culture (PMID: 18281471). 18281471
Unknown unknown triple-receptor negative breast cancer not applicable Eribulin Preclinical - Cell line xenograft Actionable In a preclinical study, Halaven (eribulin) inhibited epithelial-to-mesenchymal transition and increased tumor perfusion in triple-negative breast cancer cell line xenograft models (PMID: 25838395). 25838395
Unknown unknown 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 detail...
Unknown unknown multiple myeloma not applicable Carfilzomib + Tinostamustine Preclinical - Patient cell culture Actionable In a preclinical study, the combination of Kyprolis (carfilzomib) and EDO-S101 worked synergistically to decrease viability of multiple myeloma cell lines and primary cells in culture, including a cell line with resistance to Velcade (bortezomib) (PMID: 28753594). 28753594
Unknown unknown renal cell carcinoma not applicable Famitinib Phase I Actionable In a Phase I trial, renal carcinoma patients treated with Famitinib demonstrated a disease control rate of 87.5%, which included 50% (12/24) with a partial response and 37.5% (9/24) with stable disease, and a PFS of 10.7 mo and an OS of 33 mo (PMID: 24238512). 24238512
Unknown unknown lung non-small cell carcinoma not applicable Gemcitabine + Milciclib Phase I Actionable In a Phase I trial, the combination therapy of Gemzar (gemcitabine) and Milciclib (PHA-848125AC) resulted in a clinical benefit in 36% (5/14) of patients with an advanced solid tumor, including long-term stable disease (6-14 months) in four patients and a partial response in a patient with non-small cell lung carcinoma (PMID: 28424962). 28424962
Unknown unknown acute myeloid leukemia not applicable CPX-351 FDA approved Actionable In a Phase III trial that supported FDA approval, Vyxeos (CPX-351) treatment resulted in improved overall survival (9.56 vs 5.95 months, HR=0.69, p=0.005), event-free survival (HR=0.74, p=0.021), and complete response rate (47.4% vs 33.3%, p=0.016) compared to Cytosar-U (cytarabine) and Cerubidine (daunorubicin) combination therapy in patients with acute myeloid leukemia (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 7000-7000; NCT01696084). detail... detail...
Unknown unknown breast cancer not applicable CVX-241 Preclinical Actionable In a preclinical study, CVX-241 treatment resulted in improved overall survival in a breast cancer cell line xenograft model and syngeneic mouse model of breast cancer (PMID: 27651308). 27651308
Unknown unknown acute myeloid leukemia not applicable Cytarabine + GDC-0575 Preclinical - Pdx & cell culture Actionable In a preclinical study, combination of GDC-0575 and Cytosar-U (cytarabine) resulted in enhanced killing of acute myeloid leukemia cells in both cell line and patient-derived xenograft models (PMID: 29162833). 29162833
Unknown unknown peritoneum cancer not applicable ENMD-2076 Phase II Actionable In a Phase II clinical trial, ENMD-2076 demonstrated efficacy in patients with recurrent, platinum-resistant ovarian, fallopian tube or peritoneal cancers (PMID: 22921155). 22921155
Unknown unknown Advanced Solid Tumor not applicable Amuvatinib + Carboplatin + Paclitaxel Phase I Actionable In a Phase I clinical trial, Amuvatinib (MP470) in combination with chemotherapeutic agents, demonstrated safety and some efficacy in patients with advanced solid tumors (PMID: 24849582). 24849582
Unknown unknown renal cell carcinoma not applicable Alpha 2 Interferon + Bevacizumab FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with the combination of Avastin (bevacizumab) and Roferon (interferon alpha 2a) resulted in improved progression-free survival in patients with metastatic renal cell carcinoma compared to Roferon (interferon alpha 2a) with placebo (PMID: 20061402). 20061402 detail...
Unknown unknown estrogen-receptor positive breast cancer not applicable RO6839921 Preclinical - Cell line xenograft Actionable In a preclinical study, RO6839921 demonstrated anti-tumor activity in ER-positive breast cancer cell line xenograft models (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A156). detail...
Unknown unknown pancreatic cancer not applicable Brivanib Preclinical Actionable In preclinical studies, Brivanib was effective in a mouse pancreatic neuroendocrine tumor (PNET) model, demonstrating efficacy both as a front line treatment and as a second-line therapy following failure of VEGF receptor inhibitors (PMID: 21622725). 21622725
Unknown unknown Her2-receptor positive breast cancer not applicable Abemaciclib + Trastuzumab Preclinical - Pdx & cell culture Actionable In a preclinical study, the combination of Herceptin (trastuzumab) and Abemaciclib (LY2835219) resulted in tumor growth regresssion in an ERBB2 (HER2)-receptor positive breast cancer treatment refractory patient derived xenograft model and in ERBB2 (HER2)-receptor positive cultured cells, resulted in decreased cell viability (PMID: 26977878). 26977878
Unknown unknown 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 CLL/SLL not applicable Zanubrutinib Phase I Actionable In a Phase I trial, Brukinsa (zanubrutinib) treatment resulted in an overall response rate of 96.2% (75/78, 2 complete response, 63 partial response (PR), 10 PR with lymphocytosis) in patients with CLL/SLL (PMID: 31340982; NCT02343120). 31340982
Unknown unknown marginal zone B-cell lymphoma not applicable Idelalisib Phase II Actionable In a Phase II trial, Zydelig (idelalisib) treatment resulted in an overall response rate of 47% (7/15) in patients with marginal zone B-cell lymphoma (PMID: 24450858; NCT01282424). 24450858
Unknown unknown Merkel cell carcinoma not applicable Utomilumab Phase I Actionable In a Phase I trial, Utomilumab (PF-05082566) treatment resulted in an overall objective response rate of 13.3% (2/15, 1 complete response, 1 partial response) in patients with Merkel cell carcinoma (PMID: 29549159; NCT01307267). 29549159
Unknown unknown prostate cancer not applicable CAB-AXL-ADC Preclinical - Cell line xenograft Actionable In a preclinical study, CAB-AXL-ADC treatment inhibited tumor growth in a prostate cancer cell line xenograft model (Cancer Res 2018;78(13 Suppl):Abstract nr 827). detail...
Unknown unknown follicular lymphoma not applicable APG-2575 + Ibrutinib Preclinical - Cell line xenograft Actionable In a preclinical study, APG-2575 and Imbruvica (ibrutinib) combination therapy exhibited synergistic antitumor activity in a cell-line xenograft model of follicular lymphoma (Cancer Res July 1 2019 (79) (13 Supplement) 2058). detail...
Unknown unknown hepatocellular carcinoma not applicable Cabozantinib FDA approved Actionable In a Phase III trial (CELESTIAL) that supported FDA approval, Cabometyx (cabozantinib) significantly improved overall survival (10.2 vs 8.0 months, HR=0.76, p=0.005) and progression-free survival (5.2 vs 1.9 months, HR=0.44, p<0.001) compared to placebo in patients with previously treated advanced hepatocellular carcinoma (PMID: 29972759; NCT01908426). 29972759 detail...
Unknown unknown colorectal cancer not applicable ICEC0942 Preclinical - Cell line xenograft Actionable In a preclinical study, ICEC0942 induced growth arrest of colorectal cancer cells in culture and in cell line xenograft models (PMID: 29545334). 29545334
Unknown unknown 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 colon carcinoma not applicable Radiotherapy + XL-844 Preclinical - Cell culture Actionable In a preclinical study, radiation effects were enhanced by the addition of XL-844 in colon carcinoma cells in culture, demonstrating inhibition of DNA repair, mitotic catastrophe, and decreased cell survival (PMID: 20024691). 20024691
Unknown unknown Advanced Solid Tumor not applicable Glesatinib Phase I Actionable In a Phase I trial, Glesatinib (MGCD265) displayed safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 27, 2009 (suppl; abstr e14525)). detail...
Unknown unknown gastrointestinal stromal tumor not applicable Pimitespib Phase II Actionable In a Phase II trial, treatment with Pimitespib (TAS-116) in patients with a gastrointestinal stromal tumor who failed previous therapy (n=40) resulted in a progression-free survival of 4.4 months, a median overall survival of 11.5 months, a 0% objective response rate, and a disease control rate of 85%, with 34 patients achieving stable disease, and one patient experienced a 37.2% decrease in tumor size after the analysis cut-off date (PMID: 31536852). 31536852
Unknown unknown gastrointestinal stromal tumor not applicable Pimitespib Phase I Actionable In a Phase I trial, TAS-116 treatment resulted in partial response in a patient with gastrointestinal stromal tumor (J Clin Oncol 35, 2017 (suppl; abstr 2546)). detail...
Unknown unknown acute myeloid leukemia not applicable SST0116CL1 Preclinical - Cell line xenograft Actionable In a preclinical study, SST0116CL1 demonstrated antitumor activity in cell line xenograft models of acute myeloid leukemia (PMID: 25096516). 25096516
Unknown unknown lung non-small cell carcinoma not applicable CPI-444 Phase I Actionable In a Phase I trial, treatment with single agent CPI-444 was well-tolerated and resulted in a disease control rate of 36% (4/11) in patients with non-small cell lung cancer (J Clin Oncol 35, 2017 (suppl; abstr 3004)). detail...
Unknown unknown lung non-small cell carcinoma not applicable Docetaxel + Pembrolizumab Phase II Actionable In a Phase II trial (PROLUNG), combination of Keytruda (pembrolizumab) and Taxotere (docetaxel) significantly improved objective response rate (42.5% vs 15.8%, OR=3.94, p=0.01) and progression-free survival (PFS) (9.5 vs 3.9 mo, HR=0.24, p<0.001) compared to Taxotere (docetaxel) alone in patients with advanced non-small cell lung cancer, PFS was improved in patients with (6.8 vs 3.5 mo, p=0.04) and without (9.5 vs 4.1 mo, p<0.01) EGFR alterations (PMID: 32271354; NCT02574598). 32271354
Unknown unknown triple-receptor negative breast cancer not applicable JQ1 + Paclitaxel Preclinical - Cell culture Actionable In a preclinical study, the combination of JQ1 and Taxol (paclitaxel) resulted in a synergistic effect and inhibited the growth of triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown head and neck squamous cell carcinoma not applicable Carboplatin + Paclitaxel + Temsirolimus Phase II Actionable In a Phase II trial, the combination of Torisel (temsirolimus), Paraplatin (carboplatin), and Taxol (paclitaxel) resulted in an objective response rate of 41.7% (15/36), which included all partial responses, and 52.3% (19/36) had stable disease (PMID: 28961834). 28961834
Unknown unknown Advanced Solid Tumor not applicable BI 754091 Phase I Actionable In a Phase I trial, BI 754091 treatment resulted in partial response in 6% (1/17) and stable disease in 47% (8/17) of patients with advanced solid tumor (J Clin Oncol. 36, no. 5_suppl (February 2018) 212-212). detail...
Unknown unknown thyroid gland cancer not applicable ABT-737 Preclinical Actionable In a preclinical study, ABT-737 inhibited growth and induced cell death of human thyroid cancer cell lines in culture (PMID: 27042160). 27042160
Unknown unknown clear cell renal cell carcinoma no benefit Sorafenib + Temsirolimus Phase II Actionable In a Phase II clinical trial, treatment with the combination of Nexavar (sorafenib) and Torisel (temsirolimus) did not prolong progression-free survival compared to treatment with Avastin (bevacizumab) monotherapy (7.4 months vs 7.5 months) in patients with renal clear cell carcinoma (PMID: 26077237). 26077237
Unknown unknown Advanced Solid Tumor not applicable 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 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 Nivolumab Phase III Actionable In an analysis of two Phase III trial, Opdivo (nivolumab) treatment after disease progression demonstrated safety and clinical benefit, with 76% (65/85) of patients still alive at time of analysis in melanoma patients who received the last dose of Opdivo (nivolumab) more than 6 weeks after progression (PMID: 28662232). 28662232
Unknown unknown melanoma not applicable Nivolumab Phase II Actionable In a Phase II trial, Opdivo (nivolumab) monotherapy resulted an overall response rate (ORR) of 25% (3/12) and pathologic complete response rate (pCR) of 25% (3/12) in patients with stage III or IV melanoma, compared to a ORR of 73% (8/11) and pCR of 45% (5/11) with the combination of Opdivo (nivolumab) and Yervoy (ipilimumab), but demonstrated lower toxicity than the combination therapy (PMID: 30297909; NCT02519322). 30297909
Unknown unknown melanoma not applicable Nivolumab FDA approved Actionable In a Phase III trial (CheckMate 037) that supported FDA approval, 31.7% (38/120) of patients with advanced melanoma treated with Opdivo (nivolumab) experienced an objective response whereas only 10.6% (5/47) of advanced melanoma patients treated with investigator's choice of therapy demonstrated an objective response (PMID: 25795410; NCT01721746). 25795410 detail...
Unknown unknown melanoma not applicable Nivolumab FDA approved Actionable In a Phase III trial (CheckMate 238) that supported FDA approval, adjuvant Opdivo (nivolumab) treatment resulted in improved rate of recurrence-free survival at 12-month compared to Yervoy (ipilimumab) (70.5% vs 60.8%, HR=0.65, P<0.001) in patients with resected stage III or IV melanoma (PMID: 28891423, NCT02388906). detail... 28891423
Unknown unknown 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 Salirasib Phase I Actionable In a Phase I clinical trial, Salirasib treatment was well tolerated in Japanese patients with advanced solid tumors (n=21) and resulted in a median progression-free survival of 53 days (PMID: 29992354). 29992354
Unknown unknown colorectal cancer not applicable Cetuximab + Fluorouracil + Leucovorin + Oxaliplatin Phase II Actionable In a Phase II trial, Erbitux (cetuximab) in combination with FOLFOX resulted in improved median progression-free survival (6.4 months) comparing to FOLFOX alone (4.5 months) in colorectal cancer patients (hazard ratio =0.81) (PMID: 27002107). 27002107
Unknown unknown chondrosarcoma not applicable Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 20% (1/5) of patients with chondrosarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). detail...
Unknown unknown glioblastoma multiforme not applicable Marizomib + Vorinostat Preclinical Actionable In a preclinical study, glioblastoma cells treated with a combination of Zolinza (vorinostat) and Marizomib resulted in a synergistic effect, demonstrating decreased cell viability, DNA fragmentation and elevated caspase 9 activity in both culture and xenograft models (PMID: 26804704). 26804704
Unknown unknown multiple myeloma not applicable KW-2478 Phase I Actionable In a Phase I clinical trial, KW-2478 demonstrated safety and preliminary efficacy in patients with multiple myeloma, with 95.2% (20/21) of patients achieving stable disease (PMID: 26695442). 26695442
Unknown unknown neuroendocrine tumor no benefit Everolimus + Pasireotide Phase II Actionable In a Phase II trial, addition of Pasireotide to Afinitor (everolimus) did not significantly affect progression free survival (16.8 vs 16.6 months) or overall disease control rate (77.2% vs 82.7%) in patients with well-differentiated, progressive pancreatic neuroendocrine tumors (PMID: 28327907). 28327907
Unknown unknown Advanced Solid Tumor not applicable 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 colon cancer not applicable Vorinostat Preclinical Actionable In a preclinical study, Zolinza (vorinostat) decreased the growth of colon tumors in mice, and this effect was enhanced by co-administration of Lipitor (atorvastatin) (PMID: 22161747). 22161747
Unknown unknown Advanced Solid Tumor not applicable AMG208 Phase I Actionable In a Phase I trial, AMG208 demonstrated safety and preliminary efficacy, resulted in complete response in 2% (1/43), partial response in 7% (3/43) and stable disease in 65% (28/43) of patients with advanced solid tumors (PMID: 26155941; NCT00813384). 26155941
Unknown unknown pancreatic cancer not applicable Gemcitabine + Vantictumab Preclinical - Pdx Actionable In a preclinical study, Vantictumab (OMP-18R5) worked synergistically with Gemzar (gemcitabine) to inhibit tumor growth in patient-derived xenograft models of pancreatic cancer (PMID: 22753465). 22753465
Unknown unknown melanoma not applicable PF-06840003 Preclinical Actionable In a preclinical study, PF-06840003 inhibited tumor growth in a syngeneic mouse model of melanoma (PMID: 30232146). 30232146
Unknown unknown ovarian cancer not applicable Sabatolimab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 treatment resulted in stable disease in 2 patients with ovarian cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown Advanced Solid Tumor not applicable GSK1070916 Preclinical - Cell line xenograft Actionable In a preclinical study, GSK1070916 inhibited proliferation of a variety of advanced solid tumors in cell culture and in xenografts (PMID: 19567821). 19567821
Unknown unknown malignant fibrous histiocytoma not applicable IPA-3 Preclinical - Cell culture Actionable In a preclinical study, IPA-3 increased apoptosis and inhibited growth of a myxofibrosarcoma cell line in culture (PMID: 27577794). 27577794
Unknown unknown colon adenocarcinoma not applicable VE800 Preclinical Actionable In a preclinical study, VE800 treatment inhibited tumor growth in a mouse model of colon adenocarcinoma (PMID: 30675064). 30675064
Unknown unknown B-cell lymphoma not applicable Alisertib + Rituximab Phase I Actionable In a Phase I trial, Alisertib (MLN8237) in combination with Rituxan (rituximab) demonstrated safety and efficacy, resulted in an objective response rate of 25% (3/12, 2 complete response, 1 partial response), and stable disease in 42% (5/12) of patients with relapsed or refractory B-cell non-Hodgkin lymphomas (PMID: 30082475; NCT01397825). 30082475
Unknown unknown 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 Advanced Solid Tumor not applicable Gemcitabine + Milciclib Phase I Actionable In a Phase I trial, the combination therapy of Gemzar (gemcitabine) and Milciclib (PHA-848125AC) resulted in a clinical benefit in 36% (5/14) of patients with an advanced solid tumor, including long-term stable disease (6-14 months) in four patients and a partial response in a patient with non-small cell lung carcinoma (PMID: 28424962). 28424962
Unknown unknown multiple myeloma not applicable AMG 701 + Lenalidomide Preclinical Actionable In a preclinical study, AMG 701 and Revlimid (lenalidomide) combination treatment enhanced tumor growth inhibition and regression in a mouse model of multiple myeloma (Blood (2019) 134 (Supplement_1): 135). detail...
Unknown unknown Advanced Solid Tumor not applicable LMP400 Preclinical Actionable In a preclinical study, LMP400 inhibited proliferation of a variety of human tumor cell lines in culture (PMID: 23215354). 23215354
Unknown unknown gastric adenocarcinoma not applicable Napabucasin + Paclitaxel Phase III Actionable In a Phase III (BRIGHTER) trial, combination of Napabucasin (BBI608) and Taxol (paclitaxel) did not significantly improve overall survival (6.93 vs 7.36 months, HR=1.01, p=0.8596) or progression-free survival (3.55 vs 3.65 months, HR=1.00, p=0.9679) compared to placebo in patients with pretreated, advanced gastric and gastroesophageal junction adenocarcinoma (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 4010-4010; NCT02178956). detail...
Unknown unknown gastric adenocarcinoma not applicable Napabucasin + Paclitaxel Phase Ib/II Actionable In a Phase I/II tiral, combination of BBI608 (Napabucasin) and Taxol (paclitaxel) demonstrated safety and clinical efficacy in patients with advanced gastric and gastroesophageal junction adenocarcinoma (J Clin Oncol 33, 2015 (suppl; abstr 4069)). detail...
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 multiple myeloma not applicable CB-5083 Preclinical - Cell line xenograft Actionable In a preclinical study, CB-5083 treatment of multiple myeloma cell lines and xenografts resulted in cell death and decreased tumor growth, respectively (PMID: 28878026). 28878026
Unknown unknown islet cell tumor not applicable Everolimus FDA approved Actionable In a Phase III trial (RADIANT-3) that supported FDA approval, treatment with Afinitor (everolimus) prolonged progression-free survival (11.0 vs 4.6 months, HR=0.35, p<0.001) compared to placebo in patients with progressive pancreatic neuroendocrine tumors (PMID: 21306238; NCT00510068). detail... 21306238
Unknown unknown colorectal cancer not applicable Tanibirumab Preclinical - Cell line xenograft Actionable In a preclinical study, Tanibirumab (TTAC-0001) inhibited angiogenesis and tumor growth in human cell line xenograft models of colorectal cancer (PMID: 26325365). 26325365
Unknown unknown renal cell carcinoma not applicable Atezolizumab + CPI-444 Phase I Actionable In a Phase I trial, treatment with the combination of CPI-444 and Tecentriq (atezolizumab) was well-tolerated and resulted in a disease control rate of 100% (3/3) in patients with renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 3004)). detail...
Unknown unknown multiple myeloma not applicable Daratumumab + Dexamethasone + Lenalidomide FDA approved Actionable In a Phase III trial (MAIA) that supported FDA approval, the combination of Darzalex (daratumumab), Adexone (dexamethasone), and Revlimid (lenalidomide) resulted in an improved rate of progression-free survival at 30 months (70.6% vs 55.6%, HR=0.56, p<0.001) compared control in newly diagnosed multiple myeloma patients ineligible for autologous stem-cell transplantation (PMID: 31141632; NCT02252172). detail... 31141632
Unknown unknown multiple myeloma not applicable Daratumumab + Dexamethasone + Lenalidomide FDA approved Actionable In a Phase III trial (POLLUX) that supported FDA approval, the combination of Darzalex (daratumumab), Adexone (dexamethasone), and Revlimid (lenalidomide) resulted in a greater 12 month progression-free survival (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 patients with relapsed or refractory multiple myeloma (PMID: 27705267; NCT02076009). detail... 27705267
Unknown unknown stomach cancer not applicable OBI-999 Preclinical - Cell line xenograft Actionable In a preclinical study, OBI-999 treatment inhibited tumor growth in a Globo H-expressing gastric cancer cell line xenograft model (Cancer Res 2019;79(13 Suppl):Abstract nr 4815). detail...
Unknown unknown colon carcinoma not applicable Mps-BAY2a Preclinical Actionable In a preclinical study, Mps-BAY2a inhibited cell cycle progression and induced cell death of colon carcinoma cells in culture (PMID: 23933817). 23933817
Unknown unknown head and neck squamous cell carcinoma not applicable ALX148 + Pembrolizumab Phase I Actionable In a Phase I trial, ALX148 and Keytruda (pembrolizumab) combination treatment resulted in a partial response in 18% (3/17) and stable disease in 24% (4/17) of patients with head and neck squamous cell carcinoma that progressed on platinum therapy (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 2514-2514; NCT03013218). detail...
Unknown unknown diffuse large B-cell lymphoma not applicable Ublituximab + Umbralisib Phase I Actionable In a Phase I trial, high-dose TGR-1202 in combination with Ublituximab resulted in complete response in 50% (2/4) and partial response in 25% (1/4) of patients with diffuse large B-cell lymphoma (J Clin Oncol 33, 2015 (suppl; abstr 8548)). detail...
Unknown unknown peritoneal mesothelioma not applicable BEZ235 Preclinical Actionable In a preclinical study, BEZ235 treatment resulted in suppression of PI3K and mTOR signaling and growth inhibition in patient-derived malignant peritoneal mesothelioma cell lines in culture (PMID: 20839315). 20839315
Unknown unknown Advanced Solid Tumor not applicable Afuresertib + Trametinib Phase I Actionable In a Phase I trial, the combination of Mekinist (trametinib) and Afuresertib (GSK2110183) was not well-tolerated in patients with advanced solid tumors (PMID: 25417902). 25417902
Unknown unknown hepatocellular carcinoma no benefit OPB-31121 Phase I Actionable In a Phase I trial, OPB-31121 treatment resulted in only stable disease in 26% (6/23) of patients with advanced hepatocellular carcinoma, and was considered insufficient for clinical efficacy (PMID: 25676869). 25676869
Unknown unknown olfactory neuroblastoma not applicable OPB-111077 Phase I Actionable In a Phase I trial, a patient with esthesioneuroblastoma demonstrated stable disease when treated with OPB-111077 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B118). detail...
Unknown unknown acute myeloid leukemia not applicable ENMD-2076 Phase I Actionable In a Phase I trial, treatment with ENMD-2076 resulted in a 25% (4/16) overall response rate in patients with acute myeloid leukemia as well as three patients achieving a complete response (with incomplete count recovery) and one patient with a morphological leukemia free state (PMID: 27406088). 27406088
Unknown unknown thyroid gland carcinoma not applicable Vandetanib Phase II Actionable In a Phase II trial, Caprelsa (vandetanib) demonstrated efficacy in patients with advanced differentiated thyroid carcinoma (PMID: 22898678). 22898678
Unknown unknown 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 melanoma not applicable Prolgolimab Phase II Actionable In a Phase II trial (MIRACULUM), Prolgolimab demonstrated safety and preliminary efficacy, resulted in an objective response rate (ORR) of 38% (24/63, 5 complete response (CR), 19 partial response (PR)) and a disease control rate (DCR) of 64% when administered at 1mg/kg Q2W, and an ORR of 29% (18/63, 2 CR, 16 PR) with a DCR of 46% when administered at 3mg/kg Q3W, in patients with advanced melanoma (Annals of Oncology, Volume 30, Issue Supplement_11, December 2019, Abstract 119P; NCT03269565). detail...
Unknown unknown breast cancer not applicable AsiDNA + Veliparib Preclinical - Cell culture Actionable In a preclinical study, AsiDNA and Veliparib (ABT-888) combination treatment resulted in increased cell death and inhibition of proliferation in breast cancer cell lines compared to Veliparib (ABT-888) alone in culture (PMID: 27559053). 27559053
Unknown unknown prostate cancer not applicable SPC3042 Preclinical Actionable In a preclinical study, SPC3042 decreased Survivin expression and induced apoptosis of prostate cancer cells in culture (PMID: 18790754). 18790754
Unknown unknown Advanced Solid Tumor not applicable Neratinib + Temsirolimus Phase I Actionable In a Phase I study, Nerlynx (neratinib) administered with Torisel (temsirolimus) was tolerable and demonstrated antitumor activity in multiple solid tumor types, including breast cancer and NSCLC (PMID: 24323026). 24323026
Unknown unknown lung cancer not applicable CAB-AXL-ADC Preclinical - Cell line xenograft Actionable In a preclinical study, CAB-AXL-ADC treatment inhibited tumor growth in a lung cancer cell line xenograft model (Cancer Res 2018;78(13 Suppl):Abstract nr 827). detail...
Unknown unknown Sezary's disease not applicable Lacutamab Phase I Actionable In a Phase I trial, Lacutamab (IPH4102) demonstrated safety, and treatment resulted in an overall response rate of 43% (15/35; 2 complete responses, 13 partial responses), a median progression-free survival (PFS) of 11.7 months, and a median duration of response (DOR) of 13.8 months in patients with Sezary syndrome, with a median PFS of 16.8 months, and a median DOR of 13.8 months in Sezary syndrome patients with prior Poteligeo (mogamulizumab-kpkc) treatment (PMID: 31253572; NCT02593045). 31253572
Unknown unknown lung small cell carcinoma not applicable Doxorubicin + SLCB050 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination treatment of Adriamycin (doxorubicin) plus SLCB050 at low doses in a lung small cell carcinoma cell line xenograft model resulted in decreased tumor volume (PMID: 27302160). 27302160
Unknown unknown 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 hematologic cancer not applicable TTI-621 Preclinical - Cell culture Actionable In a preclinical study, TTI-621 (SIRPalpha-Fc) increased phagocytosis in 77% (23/30) of the human hematological tumor cell lines tested in culture (PMID: 27856600). 27856600
Unknown unknown multiple myeloma not applicable Marizomib Phase I Actionable In a Phase I trial, Marizomib (NPI-0052) treatment resulted in objective response in 14.8% (4/27) and stable disease in 44.4% (12/27) of patients with relapsed and/or refractory multiple myeloma (PMID: 27117181). 27117181
Unknown unknown hepatocellular carcinoma not applicable PF-03446962 Phase I Actionable In a Phase I trial, a patient with hepatocellular carcinoma demonstrated a partial response for 44 days when treated with PF-03446962 (PMID: 26655846). 26655846
Unknown unknown stomach cancer not applicable MGD013 Case Reports/Case Series Actionable In a Phase I trial, MGD013 treatment resulted in a partial response in a patient with gastric cancer (J Clin Oncol 38: 2020 (suppl; abstr 3004); NCT03219268). detail...
Unknown unknown multiple myeloma not applicable Daratumumab + Dexamethasone + Pomalidomide FDA approved Actionable In a Phase I trial (EQUULEUS) that supported FDA approval, Darzalex (Daratumumab) in combination with Pomalyst (pomalidomide) and Adexone (dexamethasone) resulted in an objective response rate of 60% (61/103) in patients with relapsed or refractory multiple myeloma, with a median progression-free survival of 8.8 months and a median overall survival of 17.5 months (PMID: 28637662; NCT01998971). detail... 28637662
Unknown unknown 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 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 Advanced Solid Tumor not applicable Danusertib Phase I Actionable In a phase I trial, Danusertib (PHA-739358) demonstrated safety and minimal efficacy in a patients with advanced solid tumors (PMID: 19770380, PMID: 22242557). 22242557 19770380
Unknown unknown ovarian cancer not applicable Niraparib Phase I Actionable In a Phase I clinical trial, Zejula (niraparib) demonstrated safety and preliminary efficacy, resulted in a durable partial response (PR) in 67% (2/3) of patients with plantinum-sensitive high-grade serous ovarian cancer, PR in 16% (3/19) and stable disease over 120 days in 16% (3/19) of patients with plantinum-resistant high-grade serous ovarian cancer (PMID: 23810788; NCT00749502) 23810788
Unknown unknown mantle cell lymphoma not applicable ONC201 Preclinical - Patient cell culture Actionable In a preclinical study, ONC201 induced apoptosis and decreased viability of mantle cell lymphoma (MCL) cell lines in culture and demonstrated cytotoxicity in primary MCL samples in culture (PMID: 26884599). 26884599 detail...
Unknown unknown 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 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 neuroblastoma not applicable Doxorubicin + GANT61 Preclinical Actionable In a preclinical study, GANT61 and Adriamycin (doxorubicin) worked synergistically to inhibit growth of neuroblastoma cells in culture (PMID: 22949014). 22949014
Unknown unknown Advanced Solid Tumor not applicable Axitinib Phase I Actionable In a Phase I trial, Inlyta (axitinib), in combination with chemotherapeutics, demonstrated antitumor activity in 42% (17/42) of patients with advanced solid tumors (PMID: 22996612). 22996612
Unknown unknown hematologic cancer not applicable AsiDNA Preclinical - Cell culture Actionable In a preclinical study, AsiDNA inhibited survival of hematologic cancer cell lines in culture (PMID: 27559053). 27559053
Unknown unknown leiomyosarcoma not applicable Pazopanib Clinical Study Actionable In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 18.6 weeks and median survival of 20.1 months in patients with leiomyosarcoma (PMID: 26970174). 26970174
Unknown unknown rhabdomyosarcoma not applicable JQ1 Preclinical - Pdx & cell culture Actionable In a preclinical study, JQ1 treatment resulted in cytostatic effects in patient-derived rhabdomyosarcoma cell lines in culture, but inhibited tumor growth in patient-derived xenograft models due to inhibition of tumor angiogenesis (PMID: 26908627). 26908627
Unknown unknown triple-receptor negative breast cancer not applicable BAZ2-ICR + BI-9564 + JQ1 Preclinical - Cell culture Actionable In a preclinical study, BAZ2-ICR treatment combined with BI-9564 and JQ1 inhibited Rb1 phosphorylation, induced senescence and complete growth inhibition, and led to enhanced cell cycle arrest in triple-negative breast cancer cell lines in culture (PMID: 31000582). 31000582
Unknown unknown uterine cancer not applicable Pamiparib + Tislelizumab Case Reports/Case Series Actionable In a Phase Ib trial, the combination therapy of Tislelizumab (BGB-A317) and Pamiparib (BGB-290) resulted in a partial response in a patient with uterine cancer (J Clin Oncol 35, 2017 (suppl; abstr 3013)). detail...
Unknown unknown colon cancer not applicable LF3 Preclinical - Cell line xenograft Actionable In a preclinical study, LF3 decreased expression of Wnt target genes and reduced tumor growth in colon cancer cell line xenograft models derived from cells with high levels of Wnt signaling (PMID: 26645562). 26645562
Unknown unknown 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 prostate cancer not applicable Paclitaxel + SPC3042 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 liposarcoma not applicable Doxorubicin + Nilotinib Clinical Study Actionable In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin resulted in 1 partial response and 3 stable disease in 4 patients with liposarcoma, consistent with synergistic growth inhibition in liposarcoma cells in culture (PMID: 30037815; NCT02587169). 30037815
Unknown unknown malignant pleural mesothelioma not applicable Trabectedin + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of Yondelis (trabectedin) and Venclexta (venetoclax) demonstrated synergistic or additive effects on decreasing viability of malignant pleural mesothelioma cell lines in culture (PMID: 27512118). 27512118
Unknown unknown colorectal cancer not applicable Avelumab + PF-06840003 Preclinical Actionable In a preclinical study, PF-06840003 and Bavencio (avelumab) combination treatment inhibited IDO1-mediated immunosuppression and resulted in tumor growth inhibition of 74% while treatment with PF-06840003 alone led to a tumor growth inhibition of 41% in a syngeneic mouse model of colorectal cancer (PMID: 30232146). 30232146
Unknown unknown Advanced Solid Tumor not applicable PF-03446962 Phase I Actionable In a Phase I trial, PF-03446962 treatment was deemed safe and resulted in anti-tumor activity in patients with advanced solid tumors, demonstrating a partial response in 6.8% (3/44) and stable disease in 27.3% (12/44) (PMID: 26655846). detail... 26655846
Unknown unknown Advanced Solid Tumor not applicable Omaveloxolone Phase I Actionable In a Phase I trial, treatment with Omaveloxolone (RTA 408) in patients with an advanced solid tumor led to a median progression-free survival of 1.5 months, an overall survival of 5.8 months, and one patient with lung cancer experienced stable disease for greater than 12 months (PMID: 28919776). 28919776
Unknown unknown Advanced Solid Tumor not applicable CYC140 Preclinical - Cell culture Actionable In a preclinical study, CYC140 treatment resulted in cell cycle arrest, complete growth inhibition, and apoptosis in cancer cell lines (Cancer Res 2017;77(13 Suppl):Abstract nr 4178). detail...
Unknown unknown pancreatic cancer not applicable MitoMet-10 Preclinical Actionable In a preclinical study, MitoMet-10 decreased tumor growth in mouse models of pancreatic cancer (PMID: 27216187). 27216187
Unknown unknown paraganglioma not applicable 131I-MIBG FDA approved Actionable In a Phase II trial that supported FDA approval, Azedra (iobenguane I 131) treatment resulted in partial response in 23% (15/68) of patients with pheochromocytoma or paraganglioma who received 1 therapeutic dose, with a 12-month overall survival rate of 91% (J Clin Oncol 36, 2018 (suppl; abstr 4005); NCT00874614). detail... detail...
Unknown unknown multiple myeloma no benefit Ricolinostat Phase Ib/II Actionable In a Phase Ib/II trial, Ricolinostat (ACY-1215) as a single therapy did not result in toxicity nor clinical benefit in multiple myeloma patients (PMID: 28053023). 28053023
Unknown unknown mantle cell lymphoma not applicable Bortezomib FDA approved Actionable In a Phase III trial that supported FDA approval, substituting Velcade (bortezomib) for vincristine in R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone) resulted in improved progression-free survival (24.7 vs 14.4 months, HR=0.63, p<0.001) compared to R-CHOP in patients with newly diagnosed mantle cell lymphoma (PMID: 25738670; NCT00722137). 25738670 detail...
Unknown unknown mantle cell lymphoma not applicable Bortezomib FDA approved Actionable In a Phase II trial (PINNACLE) that supported FDA approval, Velcade (bortezomib) monotherapy resulted in an overall response rate of 31% (48/155, 12 complete response, 36 partial response) in patients with relapsed or refractory mantle cell lymphoma, with a median overall survival not reached after a median follow-up of 13.4 months (PMID: 17001068; NCT00063713). 17001068 detail...
Unknown unknown breast cancer not applicable 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 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 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 kidney rhabdoid cancer not applicable UAB30 Preclinical - Cell line xenograft Actionable In a preclinical study, a human malignant rhabdoid kidney tumor cell line was sensitive to UAB30 in both culture and in xenograft models, demonstrating cell-cycle arrest, decreased cell proliferation, apoptosis, and reduced tumor growth (PMID: 26873726). 26873726
Unknown unknown ovarian cancer not applicable Bevacizumab + Everolimus Phase II Actionable In a Phase II trial, Afinitor (everolimus) and Avastin (bevacizumab) combination treatment resulted in progression free survival at 6 months in 28% (14/50) of patients with ovarian, fallopian tube, and peritoneal cancers (J Clin Oncol 34, 2016 (suppl; abstr 5552)). detail...
Unknown unknown Advanced Solid Tumor not applicable Midostaurin Phase I Actionable In a Phase I trial, Rydapt (midostaurin) demonstrated safety in patients with advanced solid tumors (PMID: 11230495). 11230495
Unknown unknown peripheral T-cell lymphoma not applicable Duvelisib Phase I Actionable In a Phase I trial, Copiktra (duvelisib) treatment resulted in an overall response rate of 50% (8/16, 3 complete response, 5 partial response) with a median duration of treatment of 11.3 weeks in patients with peripheral T-cell lymphoma (PMID: 29233821; NCT01476657). 29233821
Unknown unknown lung non-small cell carcinoma not applicable SBI-0206965 + Sirolimus 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 multiple myeloma not applicable Dexamethasone + Evofosfamide Phase Ib/II Actionable In a Phase I/II trial, Evofosfamide (TH-302) therapy plus Adexone (dexamethasone) was well tolerated in patients with relapsed/refractory multiple myeloma and resulted in a disease control rate of 83.9% (26/31, stable disease or better), 3.3% (1/31) very good partial responses and 9.7% (3/31) partial responses, a 40% 6-month progression-free survival rate, a median progression-free survival of 4.4 months, and a median overall survival of 12.8 months (PMID: 30279233; NCT01522872). 30279233
Unknown unknown 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 Mivebresib Preclinical - Patient cell culture Actionable In a preclinical study, Mivebresib (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 triple-receptor negative breast cancer not applicable Crizotinib + Navitoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of Navitoclax (ABT-263) and Xalkori (crizotinib) resulted in a synergistic effect and inhibited the growth of triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown triple-receptor negative breast cancer not applicable Carboplatin + JQ1 Preclinical - Cell culture Actionable In a preclinical study, the addition of JQ1 to Paraplatin (carboplatin) resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27256375). 27256375
Unknown unknown Advanced Solid Tumor not applicable Bevacizumab + Sorafenib Phase I Actionable In a Phase I trial, the treatment combination of Avastin (bevacizumab) and Nexavar (sorafenib) in patients with advanced solid tumors resulted in stable disease in 25% (29/115) of patients and a partial response in 5% (6/115) of patients (PMID: 25363205). 25363205
Unknown unknown gastrointestinal stromal tumor not applicable JSH-150 Preclinical - Cell culture Actionable In a preclinical study, JSH-150 inhibited proliferation of a gastrointestinal stromal tumor cell line in culture (PMID: 30253346). 30253346
Unknown unknown 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 GDC-0349 Preclinical - Cell line xenograft Actionable In a preclinical study, the mTOR inhibitor GDC-0349 demonstrated inhibition of tumor growth in cell line xenograft models of solid tumors, including prostate cancer (PMID: 24900569). 24900569
Unknown unknown Advanced Solid Tumor not applicable CYT01B + Olaparib Preclinical - Cell culture Actionable In a preclinical study, CYT01B and Lynparza (olaparib) synergistically inhibited growth of tumor cell lines in culture (AACR Annual Meeting 2019, Abstract 363). detail...
Unknown unknown Hodgkin's lymphoma not applicable Nivolumab FDA approved Actionable In a Phase I trial (CheckMate 039) that supported FDA approval, treatment with Opdivo (nivolumab) resulted in an objective response rate of 87% (20/23), with complete response in 17% (4/23) and partial response in 70% (16/23) of patients with relapsed or refractory classical Hodgkin's lymphoma (PMID: 25482239; NCT01592370). 25482239 detail...
Unknown unknown pancreatic carcinoma not applicable UNC0642 Preclinical - Cell culture Actionable In a preclinical study, a pancreatic carcinoma cell line demonstrated sensitivity to UNC0642, resulting in inhibition of colony formation in culture (PMID: 24102134). 24102134
Unknown unknown 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 lung cancer not applicable JNJ-64619178 Preclinical - Cell line xenograft Actionable In a preclinical study, JNJ-64619178 treatment inhibited tumor growth and induced regression in cell line xenograft models of small cell and non-small cell lung carcinoma (Cancer Res 2018;78(13 Suppl):Abstract nr 4859). detail...
Unknown unknown breast cancer not applicable AZD8186 Preclinical - Cell culture Actionable In a preclinical study, AZD8186 inhibited proliferation of several breast cancer cell lines in culture (PMID: 25398829). 25398829
Unknown unknown gastrointestinal system cancer not applicable Axitinib + Fluorouracil + Irinotecan + Leucovorin Phase I Actionable In a Phase I trial, Inlyta (axitinib), in combination with FOLFIRI, demonstrated safety and some efficacy in patients with gastrointestinal tumors (PMID: 24423921). 24423921
Unknown unknown neuroendocrine tumor not applicable Cixutumumab + Everolimus + Octreotide acetate Phase I Actionable In a Phase I trial, patients with neuroendocrine tumors treated with the combination of Cixutumumab, Sandostatin Lar Depot (octreotide acetate), and Afinitor (everolimus) demonstrated some efficacy, however, the drug combination did result in multiple non-dose limiting toxicities preventing long term tolerance (PMID: 25900182). 25900182
Unknown unknown breast cancer not applicable Pyr1 Preclinical - Cell line xenograft Actionable In a preclinical study, Pyr1 inhibited proliferation and migration of breast cancer cell lines in culture, and inhibited growth of primary tumors and metastases in breast cancer cell line xenograft models (PMID: 27216191). 27216191
Unknown unknown multiple myeloma not applicable Cyclophosphamide + LCL161 Phase II Actionable In a Phase II trial, LCL161 treatment followed by Cytoxan (cyclophosphamide) resulted in complete response in 4% (1/25), partial response in 12% (3/25), and molecular response in 4% (1/25) of multiple myeloma patients (PMID: 27841872). 27841872
Unknown unknown neuroendocrine tumor not applicable Everolimus FDA approved Actionable In a Phase III trial (RADIANT-4) supporting FDA approval, Afinitor (everolimus) treatment significantly improved median progression-free survival (11.0 months) comparing to placebo (3.9 months) in patients with progressive neuroendocrine tumours of the lung or gastrointestinal tract origin (PMID: 26703889; NCT01524783). detail... 26703889
Unknown unknown 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 colorectal cancer not applicable Bevacizumab + Capecitabine Phase III Actionable In a Phase III trial, maintenance therapy with Avastin (bevacizumab) and Xeloda (capecitabine) in combination resulted in a greater benefit compared to observation in colorectal cancer patients who were re-treated with Avastin (bevacizumab), Xeloda (capecitabine), and Eloxatin (oxaliplatin) due to progression, regardless of whether patients harbored RAS or BRAF V600E mutations (PMID: 28911067). 28911067
Unknown unknown renal Wilms' tumor not applicable BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 100% (3/3) of cell line xenograft models of Wilms tumor (PMID: 21298745). 21298745
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 ovarian cancer not applicable Demcizumab + Paclitaxel Preclinical - Pdx Actionable In a preclinical study, Demcizumab (OMP-21M18) and Taxol (paclitaxel) combination treatment inhibited tumor growth and suppressed cancer stem cells in patient-derived xenograft models of ovarian cancer (Cancer Res 2013;73(8 Suppl):Abstract nr 3725). detail...
Unknown unknown thymoma no benefit Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 28.6% (2/7) and stable disease in 71.6% (5/7) of patients with refractory or relapsed thymoma, with a median progression-free survival of 6.1 months, however, grade 3 or higher immune-related adverse events were seen in 71.4% (5/7) of the patients (PMID: 29906252). 29906252
Unknown unknown acute myeloid leukemia not applicable ST7612AA1 Preclinical - Cell line xenograft Actionable In a preclinical study, ST7612AA1 inhibited proliferation of acute myeloid leukemia (AML) cell lines in culture and demonstrated antitumor activity in AML cell line xenograft models (PMID: 25671299). 25671299
Unknown unknown sarcoma not applicable Pazopanib FDA approved Actionable In a Phase III trial that supported FDA approval, Votrient (pazopanib) improved progression free survival in patients with advanced soft tissue sarcoma (PMID: 22595799). 22595799 detail...
Unknown unknown sarcoma not applicable Pazopanib Clinical Study Actionable In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 15.4 weeks and median survival of 11.2 months in patients with soft tissue sarcoma (PMID: 26970174). 26970174
Unknown unknown 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 colon adenocarcinoma not applicable Cetuximab + Temsirolimus Preclinical Actionable In a preclinical study, Torisel (temsirolimus) decreased resistance to Erbitux (cetuximab) in colon cancer cells (PMID: 24493623). 24493623
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 not applicable OPB-111077 Phase I Actionable In a Phase I trial, a patient with prostate cancer demonstrated stable disease when treated with OPB-111077 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B118). detail...
Unknown unknown breast cancer not applicable MLN1117 Phase I Actionable In a Phase I trial, treatment with MLN1117 resulted in antitumor activity, demonstrating partial responses in patients with advanced solid tumors including breast and gastric cancer (J Clin Oncol, May 2015 vol. 33 no. 15_suppl 2501). detail...
Unknown unknown neuroblastoma 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 neuroblastoma (PMID: 21298745). 21298745
Unknown unknown Advanced Solid Tumor not applicable Apatinib Phase I Actionable In a Phase I trial, Apatinib (YN968D1) demonstrated safety and efficacy in patients with a variety of solid tumor types (PMID: 20923544). 20923544
Unknown unknown Advanced Solid Tumor not applicable Citarinostat + Paclitaxel Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of ACY-241 and Taxol (paclitaxel) resulted in increased efficacy in advanced solid tumor xenograft models, including pancreatic and ovarian (AACR; Cancer Res 2016;76(14 Suppl):Abstract nr 4822). detail...
Unknown unknown Advanced Solid Tumor not applicable SGI-1027 Preclinical - Cell culture Actionable In a preclinical study, SGI-1027, inhibited DNA methylation and reactivated silenced tumor suppressor genes, and induced DNMT1 degradation in a variety of cancer cell lines in culture (PMID: 19417133). 19417133
Unknown unknown Ewing sarcoma not applicable GSK1904529A Preclinical - Cell culture Actionable In a preclinical study, Ewing's sarcoma cells were sensitive to GSK1904529A in culture, resulting in decreased cell viability (PMID: 19383820). 19383820
Unknown unknown melanoma not applicable Hu3F8-BsAb Preclinical - Cell line xenograft Actionable In a preclinical study, Hu3F8-BsAb treatment induced cell killing in melanoma cell lines expressing GD2 in culture, and treatment with Hu3F8-BsAb plus human peripheral blood mononuclear cells inhibited tumor growth and improved survival in melanoma cell line xenograft models in combination with transplanted PBMCs (PMID: 25542634). 25542634
Unknown unknown peripheral T-cell lymphoma not applicable Chidamide Phase II Actionable In a Phase II trial, Chidamide (CS055) demonstrated a 28% (22/79) overall response rate in refractory peripheral T-cell lymphoma patients (PMID: 26105599). 26105599
Unknown unknown triple-receptor negative breast cancer not applicable JQ1 + Vinorelbine Preclinical - Cell culture Actionable In a preclinical study, the addition of JQ1 to Taxotere (docetaxel) resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27256375). 27256375
Unknown unknown gastrointestinal system cancer not applicable Cabozantinib Preclinical - Pdx Actionable In a preclinical study, Cometriq (cabozantinib) demonstrated efficacy in colorectal cancer patient-derived tumor explant models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1). detail...
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 Advanced Solid Tumor not applicable Bevacizumab + Cediranib Phase I Actionable In a Phase I trial, the combination of Cediranib (AZD-2171) and Avastin (bevacizumab) demonstrated preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 24752867). 24752867
Unknown unknown pancreatic cancer not applicable Napabucasin Preclinical Actionable In a preclinical study, BBI608 inhibited tumor growth and metastasis in xenograft models of pancreatic cancer (PMID: 25605917). 25605917
Unknown unknown thyroid gland cancer not applicable CLM3 Preclinical - Cell line xenograft Actionable In a preclinical study, CLM3 induced apoptosis and inhibited EGFR phosphorylation and cell proliferation in human anaplastic thyroid cancer cell lines in culture and inhibited tumor growth in xenograft models (PMID: 24423321). 24423321
Unknown unknown ovarian cancer not applicable Bevacizumab Phase III Actionable In a meta-analysis of three phase III trials, Avastin (bevacizumab) significantly increased progression free survival and overall survival comparied to standard therapy, with hazard ratios of 0.53 and 0.87, respectively (PMID: 26657509). 26657509
Unknown unknown ovarian cancer not applicable Bevacizumab FDA approved Actionable In a Phase III trial that supported FDA approval, ovarian cancer patients treated with Avastin (bevacizumab) combined with chemotherapy versus chemotherapy alone resulted in a greater PFS (6.7 mo vs 3.4 mo), overall response rate (27.3% vs 11.8%), and OS (16.6 mo vs 13.3 mo) (PMID: 24637997). detail... 24637997
Unknown unknown head and neck squamous cell carcinoma not applicable Prexasertib Phase Ib/II Actionable In a Phase Ib trial, treatment with Prexasertib (LY2606368) resulted in an overall response rate of 5% (3/57, all partial responses), clinical benefit rate (complete response+partial response+stable disease) of 49% (28/57), and a median progression-free survival of 1.6 months in patients with head and neck squamous cell carcinoma (PMID: 29643063; NCT0115790). 29643063
Unknown unknown head and neck squamous cell carcinoma not applicable Prexasertib Preclinical - Cell culture Actionable In a preclinical study, head and neck squamous cell carcinoma cell lines, either human papilloma virus positive or negative, demonstrated decreased cell proliferation in culture when treated with Prexasertib (LY2606368) (PMID: 28138028). 28138028
Unknown unknown 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 myelodysplastic syndrome not applicable Cytarabine + Glasdegib Phase Ib/II Actionable In a Phase Ib trial, the combination of Glasdegib (PF-04449913) and Cytosar-U (cytarabine) resulted in an overall survival of 4.4 months in patients with either acute myeloid leukemia or myelodysplastic syndrome (MDS), with 33% (1/3) of MDS patients experiencing a complete remission (PMID: 29463550). 29463550
Unknown unknown pancreatic cancer not applicable Trametinib Phase I Actionable In a Phase I trial, 42% (11/26) of pancreatic cancer patients demonstrated a decrease in tumor formation when treated with Mekinist (trametinib) (PMID: 22805291). 22805291
Unknown unknown epithelioid sarcoma not applicable Tazemetostat FDA approved Actionable In a Phase II trial that supported FDA approval, Tazverik (tazemetostat) treatment resulted in an objective response rate of 15% (9/62) and a disease control rate of 26% (16/62) in patients with locally advanced or metastatic epithelioid sarcoma, with a median duration of response not reached and a median overall survival of 82.4 weeks (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 11003-11003, NCT02601950). detail...
Unknown unknown B-cell lymphoma not applicable SKLB-23bb Preclinical - Cell line xenograft Actionable In a preclinical study, SKLB-23bb treatment inhibited tumor growth in B-cell lymphoma xenograft models (PMID: 29610282). 29610282
Unknown unknown lung non-small cell carcinoma not applicable Vandetanib Clinical Study Actionable In a meta-analysis of 2,284 NSCLC patients, Caprelsa (vandetanib) in combination with chemotherapy, increased progression-free survival (PFS) and overall response rate (ORR) but not overall survival (PMID: 23861835). 23861835
Unknown unknown lung non-small cell carcinoma not applicable Vandetanib Phase II Actionable In a Phase II trial, Caprelsa (vandetanib) alone, or in combination with Taxotere (docetaxel), improved progression-free survival of NSCLC patients (PMID: 17243944). 17243944
Unknown unknown lung non-small cell carcinoma not applicable Vandetanib Phase II Actionable In a Phase II trial, treatment with Caprelsa (vandetanib) increased progression-free survival in NSCLC patients, compared to Iressa (gefitinib) (PMID: 19332730). 19332730
Unknown unknown pancreatic cancer not applicable MVT-1075 Preclinical - Cell line xenograft Actionable In a preclinical study, a pancreatic cancer xenograft model demonstrated tumor growth inhibition and tumor regression by 50% when treated with MVT-1075 (AACR 2017, Abstract #5204). detail...
Unknown unknown 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 ovarian cancer not applicable Nintedanib Phase I Actionable In a Phase I clinical trial, Vargatef (nintedanib) demonstrated safety in patients with ovarian cancers, clinical trials to determine efficacy in these patients are ongoing (PMID: 19889612). 19889612
Unknown unknown renal cell carcinoma not applicable AGS16F Phase I Actionable In a Phase I trial, treatment with AGS16F (AGS-16CSF) at the recommended phase 2 dose demonstrated safety and resulted in a partial response in 23% (3/13) of patients with metastatic renal cell carcinoma including 2 patients with clear cell and 1 patient with papillary histology, and a disease control rate of 92% (12/13) (PMID: 29848572). 29848572
Unknown unknown rhabdomyosarcoma not applicable Dasatinib Phase II Actionable In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in rhabdomyosarcoma patients (n=13) was suspended due to lack of drug activity (PMID: 26710211). 26710211
Unknown unknown renal cell carcinoma not applicable Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) was demonstrated to be well tolerated and displayed anti-tumor activity in patients with melanoma and renal cell carcinoma (PMID: 22516948). 22516948
Unknown unknown melanoma not applicable GDC-0425 + Gemcitabine Phase I Actionable In a Phase I trial, treatment with GDC-0425 followed by Gemzar (gemcitabine) resulted in a partial response in a patient with melanoma (PMID: 27815358). 27815358
Unknown unknown stomach cancer not applicable Apatinib + Camrelizumab Phase Ib/II Actionable In a Phase Ib trial, combined Camrelizumab (SHR-1210) and Apatinib (YN968D1) treatment resulted in an overall response rate of 17.4% (4/23), a disease control rate of 78.3% (18/23), a median progression-free survival (PFS) of 2.9 months, and an overall survival of 11.4 months in evaluable patients with gastric or gastroesophageal junction cancer (PMID: 30348638; NCT02942329). 30348638
Unknown unknown adult T-cell leukemia not applicable Alemtuzumab Phase II Actionable In a Phase II trial, treatment with Alemtuzumab resulted in 51.7% (15/29) of patients with adult T-cell leukemia demonstrating an overall objective response, a median progression free survival of 2.0 months, and overall survival of 5.9 months (PMID: 27486175). 27486175
Unknown unknown renal cell carcinoma not applicable Nivolumab FDA approved Actionable In a Phase III clinical trial (CheckMate 025) that supported FDA approval, treatment with Opdivo (nivolumab) resulted in a median overall survival of 25 months, compared to 19.6 months with Afinitor (everolimus) and an objective response rate of 25% versus 5% with Afinitor (everolimus) in patients with advanced renal cell carcinoma (PMID: 26406148; NCT01668784). 26406148 detail...
Unknown unknown renal cell carcinoma not applicable Nivolumab Clinical Study Actionable In a retrospective analysis, Opdivo (nivolumab) treatment demonstrated an ORR of 22% (41/187), 24% (22/90) and 26% (15/58), and DOT of 5.7 mo, 6.2 mo, and 8.3 mo in the 2nd, 3rd, and 4th-line setting respectively, and a median OS in the 2nd-line setting in favorable, intermediate, and poor-risk groups of not reached (NR), 26.7 mo, and 7.4 mo, respectively; 36.1 mo, 28.2 mo, and 11.1 mo in the 3rd-line setting; and NR, NR, and 6.7 mo in the 4th-line setting in renal cell carcinoma patients (PMID: 30307610). 30307610
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 melanoma not applicable SKI-G-801 Preclinical Actionable In a preclinical study, SKI-G-801 treatment reduced tumor burden in a metastatic mouse model of melanoma (Cancer Res 2019;79(13 Suppl):Abstract nr 2010). detail...
Unknown unknown 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 acute myeloid leukemia not applicable CWP232291 Phase I Actionable In a Phase I trial, CWP232291 demonstrated tolerability and limited preliminary efficacy in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS), resulting in one complete response and one partial response among 64 AML patients, while none of the five MDS patients achieved a response (PMID: 32396615; NCT01398462). 32396615
Unknown unknown lung non-small cell carcinoma not applicable Docetaxel + Trametinib Phase I Actionable In a Phase I/Ib trial, treatment with the combination of Mekinist (trametinib) and Taxotere (docetaxel) resulted in an overall response rate (ORR) of 21% (10/47, all partial responses) and stable disease in 43% (20/47) of patients with non-small cell lung cancer (PMID: 27876675). 27876675
Unknown unknown ovarian cancer not applicable FR alpha peptide vaccine Phase I Actionable In a Phase I trial, FR alpha peptide vaccine demonstrated safety, induced durable immune response against Folr1 in ovarian cancer patients who completed standard treatment, and all patients (n=14) remained alive 2 years after initial immunization, with a median relapse-free survival of 528 days; however, T cell response did not correlate with tumor Folr1 expression level (PMID: 29545464; NCT01606241). 29545464
Unknown unknown sarcoma not applicable BI 853520 Phase I Actionable In a Phase I trial, BI 853520 demonstrated safety and some anti-tumor efficacy, resulting in stable disease in 28.6% (4/14) of patients with soft tissue sarcoma, one of whom had stable disease lasting greater than 150 days (PMID: 30756308; NCT01335269). 30756308
Unknown unknown breast cancer not applicable CPI-455 + Lapatinib Preclinical - Cell culture Actionable In a preclinical study, a breast cancer cell line treated with Tykerb (lapatinib) demonstrated increased sensitivity when co-treated with CPI-455 in culture, resulting in decreased survival of cells, in particular the cells that eventually develop drug resistance (PMID: 27214401). 27214401
Unknown unknown 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 endometrial cancer no benefit Trametinib + Uprosertib Phase I Actionable In a Phase I trial, Mekinist (trametinib) and Uprosertib (GSK2141795) combination treatment demonstrated increased toxicity and limited efficacy, resulted in no response (0/14) at RP2D dose and 1 response (8.3%, 1/12) at reduced dose in patients with recurrent endometrial cancer, with progression-free survival at 6 months in 14% and 25% of the patients, respectively (PMID: 31623857). 31623857
Unknown unknown myelodysplastic syndrome not applicable Guadecitabine Phase I Actionable In a Phase I trial, SGI-110 treatment resulted in clinical response in 32% (6/19) of patients with myelodysplastic syndrome (PMID: 26296954). 26296954
Unknown unknown esophagus adenocarcinoma not applicable IMR-1 Preclinical - Cell line xenograft Actionable In a preclinical study, IMR-1 treatment of esophageal adenocarcinoma cells resulted in decreased colony formation in culture and inhibition of tumor growth in xenograft models (PMID: 27197169). 27197169
Unknown unknown 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 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 prostate cancer no benefit Zibotentan Phase III Actionable In a Phase III trial, Zibotentan (ZD4054) did not demonstrate a survival benefit compared to placebo in patients with prostate cancer, and the trial was discontinued at the interim analysis (PMID: 23381694). 23381694
Unknown unknown triple-receptor negative breast cancer not applicable Birabresib Preclinical - Cell culture Actionable In a preclinical study, triple-receptor negative breast cancer cells demonstrated sensitivity to treatment with Birabresib (OTX015), resulting in decreased cell proliferation in culture (PMID: 27256375). 27256375
Unknown unknown breast cancer not applicable Everolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Afinitor (everolimus) inhibited the growth of a breast cancer cell line in culture and resulted in decreased tumor volume in a cell line xenograft model (PMID: 26351208). 26351208
Unknown unknown Advanced Solid Tumor not applicable 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 fallopian tube cancer not applicable Bevacizumab + Carboplatin + Paclitaxel FDA approved Actionable In a Phase III trial (GOG-0218) that supported FDA approval, addition of Avastin (bevacizumab) during and after Paraplatin (carboplatin) plus Taxol (paclitaxel) treatment prolonged progression-free survival (14.1 vs 10.3 months) compared to placebo in patients with previously untreated, stage III/IV epithelial ovarian, primary peritoneal, or fallopian tube cancer (PMID: 22204724; NCT00262847). detail... 22204724
Unknown unknown prostate carcinoma not applicable BEZ235 Preclinical Actionable In a preclinical study, treatment with BEZ235 resulted in a decrease in prostate cancer progenitor cells in culture and reduced tumor growth in prostate carcinoma xenograft models (PMID: 21138868). 21138868
Unknown unknown Advanced Solid Tumor not applicable Adavosertib + Carboplatin Phase Ib/II Actionable In a Phase Ib trial, Adavosertib (MK-1775) in combination with Paraplatin (carboplatin) was tolerable and resulted in partial response in 16.7% (1/3) of patients and stable disease in 33.3% (2/6) of Asian patients with advanced solid tumors (PMID: 32034630). 32034630
Unknown unknown Advanced Solid Tumor not applicable Adavosertib + Carboplatin Phase I Actionable In a Phase I trial, the combination of Adavosertib (MK-1775) and Paraplatin (carboplatin) resulted in a partial response in 6.5% (4/62) of patients and stable disease in 45% (28/62) of patients, all with advanced solid tumors (PMID: 27601554). 27601554
Unknown unknown Ewing sarcoma not applicable Talazoparib + Temozolomide Preclinical - Cell culture Actionable In a preclinical study, Ewing sarcoma cells treated with Temodar (temozolomide) combined with Talazoparib (BMN-673) resulted in strong synergism, demonstrating decreased cell viability in culture (PMID: 26438158). 26438158
Unknown unknown triple-receptor negative breast cancer not applicable LTX-315 + Pegylated liposomal-doxorubicin Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of LTX-315 and Doxil (pegylated liposomal doxorubicin) resulted in increased tumor growth inhibition and regression, increased tumor necrosis, and increased T-cell infiltration in triple-negative breast cancer (TNBC) cell line xenograft models compared to either agent alone, and in TNBC models in the neoadjuvant setting induced tumor regression in 50% and improved survival compared to either agent alone (PMID: 30670061). 30670061
Unknown unknown 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 renal pelvis transitional cell carcinoma not applicable Mitomycin gel FDA approved Actionable In a Phase III trial (OLYMPUS) that supported FDA approval, Jelmyto (mitomycin gel) treatment resulted in a complete response in 60% (41/68) of patients with low-grade upper tract urothelial cancer (J Urol. 2019 Apr; 201 (Supplement 4): abstract LBA-16; NCT02793128). detail...
Unknown unknown glioblastoma multiforme not applicable SA16 Preclinical - Cell culture Actionable In a preclinical study, SA16 treatment in glioblastoma cells resulted in decreased cell proliferation, reduced cell viability, and apoptotic induction in culture, and inhibited glioma stem cell formation (PMID: 27797168). 27797168
Unknown unknown 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 Advanced Solid Tumor not applicable BMS-690514 Phase Ib/II Actionable In a Phase Ib/II trial, BMS-690514 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 23490650). 23490650
Unknown unknown Advanced Solid Tumor not applicable 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 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 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 lung cancer not applicable BOS172722 Preclinical - Cell culture Actionable In a preclinical study, lung cancer cell lines demonstrated sensitivity to growth inhibition by BOS172722 in culture (PMID: 31575759). 31575759
Unknown unknown gastrointestinal stromal tumor not applicable Dovitinib Phase II Actionable In a Phase II clinical trial, Dovitinib (TKI258) demonstrated safety and efficacy in heavily pretreated patients with advanced GISTs (PMID: 24084771). 24084771
Unknown unknown head and neck squamous cell carcinoma not applicable Nivolumab FDA approved Actionable In a Phase III clinical trial (CheckMate 141) 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; NCT02105636). detail... 27718784
Unknown unknown Advanced Solid Tumor not applicable Pimitespib Phase I Actionable In a Phase I trial, TAS-116 demonstrated safety and resulted in a disease control rate of 27% (16/60; including stable disease for greater than or equal to 12 weeks (13) and partial responses (3)), in patients with advanced solid tumors, with partial responses in 2 patients with non-small cell lung cancer and 1 patient with gastrointestinal stromal tumor (PMID: 30679388; NCT02965885). 30679388
Unknown unknown ovarian cancer not applicable AIM-100 Preclinical Actionable In a preclinical study, AIM-100 inhibited growth of ovarian cancer cells in culture (PMID: 22322295). 22322295
Unknown unknown tenosynovial giant cell tumor not applicable Pexidartinib FDA approved Actionable In a Phase III trial (ENLIVEN) that supported FDA approval, Turalio (pexidartinib) treatment resulted in improved overall response rate at week 25 (24/61, 39% vs 0/59, 0%, p<0.0001) compared to placebo in patients with advanced tenosynovial giant cell tumour (PMID: 31229240; NCT02371369). detail... 31229240
Unknown unknown lung non-small cell carcinoma not applicable DNIB0600A Phase I Actionable In a Phase I trial, DNIB0600A (lifastuzumab vedotin) treatment demonstrated a safe profile, but resulted in limited efficacy in patients with non-small cell lung cancer receiving a dose of 1.8-2.8 mg/kg, which included a partial response in 8% (4/51) of patients regardless of Slc34a2 expression and a partial response in 12% (3/26) of patients with over expression of Slc34a2 (PMID: 31540980; NCT01363947). 31540980
Unknown unknown diffuse large B-cell lymphoma not applicable MLN0905 + Rituximab Preclinical Actionable In a preclinical study, MLN0905 combined with MabThera (rituximab) resulted in a synergistic effect when treating a diffuse large B-cell lymphoma xenograft model, demonstrating a decrease in tumor volume and increased survival (PMID: 22609854). 22609854
Unknown unknown fallopian tube cancer not applicable Lurbinectedin Phase II Actionable In a Phase II trial, Lurbinectedin (PM01183) treatment in patients with either ovarian, peritoneal, or fallopian tube cancer resulted in an overall response rate of 23% (12/52), which included one complete response and eleven partial responses, and a median duration of response of 4.6 months and 23% (12/52) of responses lasted for 6 months or more (PMID: 28368437). 28368437
Unknown unknown colorectal cancer not applicable YW3-56 Preclinical Actionable In a preclinical study, YW3-56 inhibited proliferation of colorectal cancer cell lines in culture, independent of TP53 status (PMID: 25612620). 25612620
Unknown unknown melanoma not applicable Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in partial response in 5% (4/77) and sttable disease in 39% (30/77) of patients with metastatic melanoma, with a median overall progression free survival of 3.8 months (PMID: 28103611). 28103611
Unknown unknown breast cancer not applicable ISTH0047 Preclinical Actionable In a preclinical study, ISTH0047 reduced lung metastasis, but not primary tumor growth, in a mouse model of metastatic breast cancer (PMID: 28911087). 28911087
Unknown unknown osteosarcoma not applicable Capmatinib Case Reports/Case Series Actionable In a Phase I trial, Tabrecta (capmatinib) demonstrated safety and preliminary efficacy, resulted in stable disease as best overall response in a patients with osteosarcoma (PMID: 30724423). 30724423
Unknown unknown transitional cell carcinoma not applicable Sabatolimab + Spartalizumab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in stable disease in 3 patients with transitional cell carcinoma (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown lymphoma not applicable OKI-179 + unspecified PD-1 antibody Preclinical - Cell line xenograft Actionable In a preclinical study, OKI-179 upregulated MHC expression, sensitized lymphoma cells to anti-PD-1 treatment in cell line xenograft models (PMID: 31235619). 31235619
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 non-Hodgkin lymphoma not applicable CC-90010 Phase I Actionable In a Phase I trial, CC-90010 treatment demonstrated safety, and resulted in an overall response rate of 2.9% (2/69, 1 complete response, 1 partial response), stable disease in 33.3% (23/69), and a median progression-free survival of 1.9 months in patients with advanced solid tumors or relapsed/refractory non-Hodgkin lymphoma (PMID: 32240793; NCT03220347). 32240793
Unknown unknown nasopharynx carcinoma not applicable BI 836880 Case Reports/Case Series Actionable In a Phase I trial, BI 836880 treatment was well tolerated in patients with advanced solid tumors, and resulted in 2 partial responses (n=29), including a partial response in a patient with nasopharyngeal carcinoma (J Clin Onc 2018 36:15_suppl, 12024; NCT02674152). detail...
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 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 renal cell carcinoma not applicable Pegilodecakin Phase I Actionable In a Phase I trial, AM0010 demonstrated safety and resulted in partial responses in 27% (4/15) of patients with renal cell carcinoma (PMID: 27528724; NCT02009449). 27528724
Unknown unknown 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 breast cancer not applicable UNC0642 Preclinical - Cell culture Actionable In a preclinical study, UNC0642 decreased H3K9me2 levels, but did not inhibit colony formation in a breast cancer cell line in culture (PMID: 24102134). 24102134
Unknown unknown osteosarcoma not applicable NKTR-214 Preclinical Actionable In a preclinical study, NKTR-214 treatment inhibited growth of primary tumor, regrowth after amputation, and lung metastasis in disseminated and orthotopic mouse models of osteosarcoma (AACR Annual Meeting 2019, Abstract 3210). detail...
Unknown unknown ovarian cancer not applicable Cediranib Phase III Actionable In a Phase III trial, Cediranib (AZD-2171) given with chemotherapy and as maintenance therapy resulted in improved median overall survival (27.3 vs 19.9 months) in platinum-sensitive ovarian cancer patients (J Clin Oncol 35, 2017 (suppl; abstr 5506)). detail...
Unknown unknown chronic myeloid leukemia not applicable Dasatinib + GSK343 Preclinical - Patient cell culture Actionable In a preclinical study, the combination of Sprycel (dasatinib) and GSK343 resulted in increased apoptosis and reduced viability of human primary chronic myeloid leukemia cells in culture, compared to Sprycel (dasatinib) alone (PMID: 27630125). 27630125
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 multiple myeloma not applicable Bortezomib + SJB3-019A Preclinical - Patient cell culture Actionable In a preclinical study, the combination of SJB3-019A and Velcade (bortezomib) worked synergistically to induce cytotoxicity in multiple myeloma cell lines and primary multiple myeloma cells in culture (PMID: 28270494). 28270494
Unknown unknown astrocytoma not applicable Niraparib Preclinical - Cell line xenograft Actionable In a preclinical study, pediatric high grade astrocytoma cell lines treated with Zejula (niraparib) demonstrated decreased cell viability and proliferation in culture, and a small survival benefit in xenograft models (PMID: 26351319). 26351319
Unknown unknown stomach cancer not applicable Capecitabine + Nivolumab + Oxaliplatin + TS-1 Phase II Actionable In a Phase II trial (ATTRACTION-4), the combination therapy of Xeloda (capecitabine), Opdivo (nivolumab), Eloxatin (oxaliplatin), and TS-1 (tegafur-gimeracil-oteracil potassium) was well-tolerated and resulted in an objective response rate of 76.5% (13/17), a median progression-free survival of 10.6 months, and a median overall survival that was not yet reached in patients with either gastric cancer or gastroesophageal junction cancer (PMID: 30566590; NCT02746796). 30566590
Unknown unknown colon carcinoma not applicable SLC-391 Preclinical Actionable In a preclinical study, SLC-391 treatment did not inhibit proliferation of a colon carcinoma cell line in culture, however, inhibited tumor growth by 37% in a syngeneic mouse model (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B148). detail...
Unknown unknown colorectal cancer not applicable Sabatolimab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 treatment resulted in stable disease in 2 patients with colorectal cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown pancreatic ductal adenocarcinoma not applicable Cisplatin + Gemcitabine Phase II Actionable In a Phase II trial, patients with pancreatic ductal adenocarcinoma harboring either a germline BRCA1, BRCA2, or PALB2 inactivating mutation demonstrated a response rate of 74.1% (20/27), a median progression-free survival of 10.1 months, a median overall survival of 15.5 months, and a disease control rate of 100% (27/27) when treated with a combination of Gemzar (gemcitabine) and Platinol (cisplatin) (PMID: 31976786). 31976786
Unknown unknown non-Hodgkin lymphoma not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in complete response in 11% (1/9) and stable disease in 44% (4/9) of patients with non-Hodgkin lymphoma (PMID: 29475723; NCT01767766). 29475723
Unknown unknown Advanced Solid Tumor not applicable Gemcitabine + Trametinib Phase Ib/II Actionable In a Phase Ib trial, the combination of Mekinist (trametinib) and Gemzar (gemcitabine) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors (PMID: 23583440). 23583440
Unknown unknown leukemia not applicable MYCi975 Preclinical - Cell culture Actionable In a preclinical study, MYCi975 treatment inhibited viability of a leukemia cell line in culture (PMID: 31679823). 31679823
Unknown unknown acute myeloid leukemia not applicable Combretastatin A1 Diphosphate Phase I Actionable In a Phase I study, OXi4503 demonstrated safety and preliminary efficacy in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS), resulting in one complete response and one partial response among 14 patients, with four patients demonstrating stable disease (PMID: 32236943). 32236943
Unknown unknown Advanced Solid Tumor not applicable Rucaparib Phase I Actionable In a Phase I trial, Rubraca (rucaparib) was well-tolerated and demonstrated preliminary efficacy, with a disease control rate of 86% (6/7), in patients with advanced solid tumors (J Clin Oncol 31, 2013 (suppl; abstr 2585)). detail...
Unknown unknown urinary bladder cancer not applicable OBP-801 Preclinical - Cell culture Actionable In a preclinical study, OBP-801 treatment resulted in decreased cell viability in multiple human bladder cancer cell lines in culture (PMID: 27406983). 27406983
Unknown unknown melanoma not applicable IMO-2125 Phase Ib/II Actionable In a Phase I/II trial, IMO-2125 treatment resulted in clinical benefit in 67% (6/9, 1 complete response, 2 partial response, 3 stable disease) of patients with melanoma refractory to a PD-1 or PD-L1 inhibitor (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 1187P; NCT02644967). detail...
Unknown unknown 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 lymphoma not applicable Ixazomib Preclinical Actionable In a preclinical study, Ixazomib (MLN9708) inhibited survival and induced apoptosis in T-cell lymphoma cell lines in culture, and reduced tumor volume in xenograft models (PMID: 26988986). 26988986
Unknown unknown acute myeloid leukemia not applicable Cytarabine + LGH447 Preclinical Actionable In a preclinical study, the combination of LGH447 and Cytosar-U (cytarabine) resulted in tumor regression by 50% in an acute myeloid leukemia mouse model (PMID: 24474669). 24474669
Unknown unknown papillary renal cell carcinoma not applicable Bevacizumab + Everolimus Phase II Actionable In a Phase II trial, 50% (2/4) of patients with papillary renal cell carcinoma achieved a 6 month PFS when treated with a combination of Afinitor (everolimus) and Avastin (bevacizumab) (PMID: 27601542). 27601542
Unknown unknown 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 follicular lymphoma not applicable Rituximab Phase III Actionable In a Phase III trial, Rituxan (rituximab) treatment in patients with follicular lymphoma resulted in an overall survival of 84.9% in patients receiving the drug intravenously and 84.4% in patients receiving the drug subcutaneously (PMID: 28476440). 28476440
Unknown unknown B-cell lymphoma not applicable Alisertib + Rituximab + Vincristine Sulfate Phase I Actionable In a Phase I trial, Alisertib (MLN8237) in combination with Rituxan (rituximab) and Oncovin (vincristine) demonstrated safety and efficacy, resulted in an objective response rate of 38% (14/37, 7 complete response, 7 partial response), and stable disease in 32% (12/37) of patients with relapsed or refractory B-cell non-Hodgkin lymphomas (PMID: 30082475; NCT01397825). 30082475
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 multiple myeloma not applicable BC2059 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with BC2059 decreased proliferation and induced apoptosis of several human multiple myeloma cell lines and primary multiple myeloma cells with activated canonical Wnt signaling in culture, and delayed tumor growth in a multiple myeloma cell line xenograft model (PMID: 28500235). 28500235
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 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 ovarian cancer not applicable Everolimus + JI-101 Phase 0 Actionable In a pilot study, JI-101 in combination with Afinitor (everolimus) demonstrated safety and preliminary efficacy, resulted in stable disease in a majority of patients with ovarian cancer (PMID: 26365907). 26365907
Unknown unknown Advanced Solid Tumor not applicable Tivantinib Phase I Actionable In a Phase I trial, Tivantinib (ARQ 197) demonstrated safety and some anti-tumor activity in patients with advanced solid tumors (PMID: 21976535). 21976535
Unknown unknown stomach cancer no benefit Ipilimumab Phase II Actionable In a Phase II trial, Yervoy (ipilimumab) did not improve immune-related progression-free survival (2.9 vs 4.9 months) compared to best supportive care in patients with unresectable, locally advanced/metastatic gastric or gastroesophageal junction cancer (J Clin Oncol 34, 2016 (suppl; abstr 4011)). detail...
Unknown unknown estrogen-receptor positive breast cancer not applicable Everolimus Preclinical - Cell culture Actionable In a preclinical study, treatment with Afinitor (everolimus) resulted in decreased cell proliferation, reduced anchorage-independent cell growth and a decrease in PI3K/Akt/mTOR pathway signaling in estrogen-receptor positive breast cancer cell lines resistant to aromatase inhibitors (PMID: 27421652). 27421652
Unknown unknown dermatofibrosarcoma protuberans not applicable Imatinib FDA approved Actionable In a Phase II clinical trial that supported FDA approval, treatment with Gleevec (imatinib) resulted in a median time-to-progression of 23.9 months, and complete response in 33% (4/12) and partial response in 50% (6/12) of patients with dermatofibrosarcoma protuberans (PMID: 18451237). 18451237 detail...
Unknown unknown Advanced Solid Tumor not applicable CLR457 Preclinical - Pdx Actionable In a preclinical study, CLR457 treatment of a variety of solid tumor patient-derived xenograft models decreased tumor volume and produced a 54% response (PMID: 26479923). 26479923
Unknown unknown Advanced Solid Tumor not applicable Poziotinib Phase I Actionable In a Phase I trial, Poziotinib (HM781-36B) displayed favorable pharmacokinetics in patients with advanced solid tumors (PMID: 25377158). 25377158
Unknown unknown Advanced Solid Tumor not applicable Ch282-5 Preclinical Actionable In a preclinical study, ch282-5 inhibited growth of a variety of human solid tumor cell lines in culture (PMID: 26515494). 26515494
Unknown unknown 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 neuroblastoma not applicable 131I-MIBG + Vorinostat Phase I Actionable In a Phase I clinical trial, the combination of 131I-MIBG with Zolinza (vorinostat) as a radiosensitizer demonstrated preliminary efficacy at the recommended Phase II dose, with in an overall objective response rate of 17% (1/6) and MIBG response rate of 67% (4/6) in patients with neuroblastoma (PMID: 25695691). 25695691
Unknown unknown desmoid tumor not applicable Sorafenib Phase III Actionable In a Phase III trial, Nexavar (sorafenib) treatment resulted in an increased 2-year progression-free survival compared to placebo (81% vs 36%), an objective response rate of 33% (16/49; 1 complete response and 15 partial responses (PR)) compared in 20% (7/25; all PR) with placebo, and a median time to objective response of 9.6 months, compared to 13.3 months with placebo, in patients with refractory desmoid tumors (PMID: 30575484; NCT02066181). 30575484
Unknown unknown 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 anal canal squamous cell carcinoma not applicable Cetuximab + Cisplatin + Fluorouracil + Radiotherapy Phase II Actionable In a Phase II trial, the combination of Erbitux (cetuximab) with Platinol (cisplatin), Adrucil (fluorouracil), and radiotherapy resulted in a locoregional failure rate of 23% (14/61) and a PFS of 68% and OS of 83% in patients with anal canal squamous cell carcinoma (PMID: 28068178). 28068178
Unknown unknown breast cancer not applicable GDC-0349 Preclinical - Cell line xenograft Actionable In a preclinical study, the mTOR inhibitor GDC-0349 inhibited tumor growth in cell line xenograft models of solid tumors, including breast cancer (PMID: 24900569). 24900569
Unknown unknown triple-receptor negative breast cancer not applicable Fluzoparib Preclinical - Cell culture Actionable In a preclinical study, Fluzoparib inhibited DNA-damage-induced PARylation in breast cancer cell lines in culture, however, the cells demonstrated resistance to Fluzoparib treatment (PMID: 30949414). 30949414
Unknown unknown triple-receptor negative breast cancer not applicable Docetaxel + NTRC 0066-0 Preclinical Actionable In a preclinical study, the TTK (MPS1) inhibitor, NTRC 0066-0, acted in synergy with docetaxel to induce tumor remission and increase survival of TNBC mouse models (PMID: 26153498). 26153498
Unknown unknown colon adenocarcinoma not applicable unspecified PD-1 antibody + VE800 Preclinical Actionable In a preclinical study, PD1-antibody treatment supplemented with VE800 inhibited tumor growth in mouse models of colon adenocarcinoma (PMID: 30675064). 30675064
Unknown unknown diffuse large B-cell lymphoma not applicable 7-hydroxystaurosporine + Sirolimus Preclinical Actionable In a preclinical study, Rapamune (sirolimus), in combination with UCN-01 (sc-3510), resulted in apoptosis and cell cycle arrest in diffuse large B-cell lymphoma cells in culture (PMID: 19223503). 19223503
Unknown unknown pancreatic cancer not applicable Gemcitabine + Refametinib Phase Ib/II Actionable In a Phase I/II trial, Refametinib (BAY86-9766) and Gemzar (gemcitabine) combination treatment resulted in an objective response rate of 23% and a disease control rate of 73% in patients with advanced pancreatic cancer (PMID: 27975152). 27975152
Unknown unknown breast cancer not applicable F14512 Preclinical - Cell line xenograft Actionable In a preclinical study, F14512 induced tumor regression in breast cancer cell line xenograft models (PMID: 19047165). 19047165
Unknown unknown melanoma not applicable VLX600 Preclinical - Cell culture Actionable In a preclinical study, VLX600 treatment inhibited proliferation of a mouse melanoma cell line in culture (PMID: 24548894). 24548894
Unknown unknown ovarian cancer not applicable S2101 Preclinical - Cell culture Actionable In a preclinical study, S2101 altered gene expression, resulted in apoptosis in ovarian cancer cells in culture (PMID: 27914215). 27914215
Unknown unknown glioblastoma multiforme not applicable Plerixafor Phase Ib/II Actionable In a Phase I/II trial, Plerixafor (AMD3100) treatment following standard chemoradiation in newly-diagnosed glioblastoma patients was well-tolerated, and 93% (27/29) achieved 6 months without progression, with a median progression-free survival of 14.5 months and median overall survival of 21.3 months, and Plerixafor (AMD3100) treatment was associated with decreased relative cerebral blood volume and decreased rate of in-field recurrence (PMID: 31537527; NCT01977677) 31537527
Unknown unknown melanoma not applicable JSH-150 Preclinical - Cell culture Actionable In a preclinical study, JSH-150 inhibited proliferation of a melanoma cell line in culture (PMID: 30253346). 30253346
Unknown unknown pancreatic ductal adenocarcinoma not applicable Gemcitabine + Masitinib Phase I Actionable In a Phase I trial, the median OS did not differ (7.7 mo vs 7.1 mo) between pancreatic ductal adenocarcinoma patients treated with the combination of Gemzar (gemcitabine) plus Masitinib (AB1010) versus those treated with Gemzar (gemcitabine) plus placebo (PMID: 25858497). 25858497
Unknown unknown acute myeloid leukemia not applicable Cytarabine + RN-1 Preclinical - Cell culture Actionable In a preclinical study, RN-1 and Cytosar-U (cytarabine) demonstrated synergy in growth inhibition of several acute myeloid leukemia cell lines in culture (PMID: 26837761). 26837761
Unknown unknown lung non-small cell carcinoma not applicable Navitoclax + TAK-901 Preclinical - Cell culture Actionable In a preclinical study, the combination of TAK-901 and Navitoclax (ABT-263) resulted in a synergistic effect, demonstrating reduced cell viability of non-small cell lung cancer cells in culture (PMID: 28179288). 28179288
Unknown unknown triple-receptor negative breast cancer not applicable S63845 Preclinical - Cell culture Actionable In a preclinical study, S63845 decreased viability of triple negative breast cancer (TNBC) cell lines and TNBC patient-derived xenograft (PDX) tumor cells in culture (PMID: 28768804). 28768804
Unknown unknown rectum cancer not applicable Nelfinavir + Radiotherapy Phase I Actionable In a Phase I trial, the combination of Nelfinavir and radiotherapy resulted in median tumor cell density reduction from 24.3% at baseline to 9.2%, and tumor regression in 56% (5/9) of rectal cancer patients (PMID: 26861457). 26861457
Unknown unknown 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 Advanced Solid Tumor not applicable Alpelisib + Infigratinib Phase I Actionable In a Phase Ib trial, Infigratinib (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 mycosis fungoides not applicable Mogamulizumab FDA approved Actionable In a Phase III trial (MAVORIC) that supported FDA approval, Poteligeo (mogamulizumab-kpkc) treatment resulted in significantly improved progression-free survival (7.7 vs 3.1 months, HR=0.53, p<0.0001) and objective response rate (28.0% vs 4.8%) compared to Zolinza (vorinostat) in patients with mycosis fungoides or Sézary syndrome (Blood 2017 130(Suppl 1):817). detail... detail...
Unknown unknown subependymal giant cell astrocytoma not applicable Everolimus FDA approved Actionable In a Phase III trial (EXIST-1) that supported FDA approval, Afinitor (everolimus) treatment resulted in a 50% or more tumor reduction in 35% (27/78) of adult and pediatric patients diagnosed with tuberous sclerosis complex and had subependymal giant cell astrocytoma, compared to 0% (0/39) in the placebo group (PMID: 23158522; NCT00789828). 23158522 detail...
Unknown unknown synovial sarcoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 75%, median progression-free survival of 7.7 months, an objective response rate of 17% (n=47), and a median overall survival of 12 months in patients with synovial sarcoma (PMID: 29895706; NCT01878448). 29895706
Unknown unknown triple-receptor negative breast cancer not applicable LY2109761 + Paclitaxel Preclinical Actionable In a preclinical study, LY2109761 and Taxol (paclitaxel) worked synergistically to inhibit the growth of triple-receptor negative breast cancer cell lines in culture (PMID: 20576088). 20576088
Unknown unknown marginal zone B-cell lymphoma not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in partial response in 20% (1/5) and stable disease in 80% (4/5) of patients with marginal zone B-cell lymphoma (PMID: 29475723; NCT01767766). 29475723
Unknown unknown transitional cell carcinoma no benefit Docetaxel + Icrucumab Phase II Actionable In a Phase II trial, Icrucumab (IMC-18F1) and Taxotere (docetaxel) combination treatment did not result in improved median progression-free survival (1.6 months) when compared to Taxotere (docetaxel) single treatment (2.8 months) in urothelial carcinoma patients (PMID: 26926681). 26926681
Unknown unknown prostate cancer not applicable Relugolix Phase III Actionable In a Phase III trial (HERO), Relugolix (TAK-385) demonstrated superior serum testosterone suppression and maintenance of castration compared to Lupron (leuprolide) (96.7%, 601/622 vs 88.8%, 273/308, p<0.0001) in patients with androgen-sensitive advanced prostate cancer (PMID: 32469183; NCT03085095). 32469183
Unknown unknown prostate cancer not applicable Relugolix Phase II Actionable In a Phase II trial, Relugolix (TAK-385) resulted in decreased testosterone levels and greatly reduced prostate specific antigen levels after 24 weeks in prostate cancer patients (J Clin Oncol 34, 2016 (suppl 2S; abstr 200)). detail...
Unknown unknown lymphoma not applicable PQR309 + Venetoclax Preclinical - Cell line xenograft Actionable In a preclinical study, the combination therapy of PQR309 and Venclexta (venetoclax) led to antitumor activity in lymphoma cells in culture and cell line xenograft models, demonstrating both synergistic and additive effects (PMID: 29066507). 29066507
Unknown unknown stomach cancer not applicable Cisplatin + Fluorouracil + Sapitinib 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 MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human colorectal carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown breast cancer not applicable Cisplatin + NU6027 Preclinical - Cell culture Actionable In a preclinical study, NU6027 enhanced the efficacy of Platinol (cisplatin) in breast cancer cells in culture, resulting in decreased cell survival (PMID: 21730979). 21730979
Unknown unknown renal cell carcinoma not applicable Emibetuzumab + Ramucirumab Phase Ib/II Actionable In a Phase Ib/II trial, Emibetuzumab (LY2875358) and Cyramza (ramucirumab) combination treatment resulted in an objective response rate of 0% and a disease control rate of 47% (7/15) in patients with renal cell carcinoma, with a median progression-free survival of 2.9 months (PMID: 31142504; NCT02082210). 31142504
Unknown unknown colorectal cancer not applicable Fluorouracil + Leucovorin + Oxaliplatin + Ramucirumab Phase II Actionable In a Phase II study, Cyramza (ramuciramab), in combination with mFOLFOX-6 chemotherapy regimen, demonstrated safety and efficacy in metastatic colorectal cancer (PMID: 24674871). 24674871
Unknown unknown acute myeloid leukemia not applicable RO3280 Preclinical Actionable In a preclinical study, acute myeloid leukemia cells treated with RO3280 in culture demonstrated inhibition of cell growth and apoptotic activity (PMID: 25574601). 25574601
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 Advanced Solid Tumor not applicable Napabucasin Phase I Actionable In a Phase I trial, BBI608 treatment demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 32:5s, 2014 (suppl; abstr 2546)). detail...
Unknown unknown chronic lymphocytic leukemia not applicable Tirabrutinib Phase I Actionable In a Phase Ib trial, Tirabrutinib (ONO-4059) treatment was well tolerated, resulted in a overall response rate of 83% (24/29) with complete response in 7% (2/29) of patients with chronic lymphocytic leukemia (PMID: 32156743; NCT02457598). 32156743
Unknown unknown chronic lymphocytic leukemia not applicable Tirabrutinib Phase I Actionable In a Phase I clinical trial, treatment with Tirabrutinib (ONO-4059) was well tolerated and resulted in objective responses in lymph nodes in 96% (24/25) of patients with chronic lymphocytic leukemia (PMID: 26542378). 26542378
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 osteosarcoma not applicable SP-2509 Preclinical - Cell culture Actionable In a preclinical study, SP-2509 treatment inhibited viability of osteosarcoma cell lines in culture (PMID: 29997151). 29997151
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 colorectal cancer not applicable UD-017 Preclinical - Patient cell culture Actionable In a preclinical study, UD-017 inhibited growth of patient-derived colorectal cancer cells in culture (J Clin Oncol 35, 2017 (suppl; abstr e14085)). detail...
Unknown unknown Advanced Solid Tumor not applicable AC480 Phase I Actionable In a Phase I trial, AC480 demonstrated safety and potential efficacy in patients with several solid tumor types (PMID: 21576284). 21576284
Unknown unknown lung non-small cell carcinoma not applicable Carboplatin + Paclitaxel + Ramucirumab Phase II Actionable In a Phase II study, preliminary results reported a 67% response rate in NSCLC patients treated with Cyramza (ramucirumab) in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) (PMID: 22481432). 22481432
Unknown unknown lymphoplasmacytic lymphoma not applicable Idelalisib Phase II Actionable In a Phase II trial, Zydelig (idelalisib) treatment resulted in an overall response rate of 80% (8/10) in patients with lymphoplasmacytic lymphoma (PMID: 24450858; NCT01282424). 24450858
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 diffuse large B-cell lymphoma not applicable Fenretinide Phase I Actionable In a Phase I trial, Fenretinide treatment resulted in partial response in a patient with diffuse large B-cell lymphoma (PMID: 28420721). 28420721
Unknown unknown multiple myeloma not applicable Bendamustine + Veliparib Phase I Actionable In a Phase I trial, a patient with multiple myeloma demonstrated a partial response when treated with a combination of Bendamustine and Veliparib (ABT-888) (PMID: 28314788; NCT01326702). 28314788
Unknown unknown pancreatic cancer not applicable Gemcitabine + MVT-5873 + Nab-paclitaxel Preclinical - Cell line xenograft Actionable In a preclinical study, MVT-5873 enhanced efficacy of the combination therapy, Gemzar (gemcitabine) and Abraxane (nab-paclitaxel), in pancreatic xenograft models, demonstrating increased inhibition of tumor growth and greater reduction of tumor volume when compared to single agents (Cancer Res 2016;76(24 Suppl):Abstract nr A73). detail...
Unknown unknown 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 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 gastric adenocarcinoma not applicable Nintedanib Phase II Actionable In a Phase II trial, Ofev (nintedanib) was well tolerated and the study met its primary endpoint, resulted in progression-free survival at 6-months in 19% (6/32) of patients with esophageal/GEJ (n=17) or gastric (n=15) adenocarcinoma, with a median follow-up of 14.5 months and a median overall survival of 14.2 months (PMID: 30952642; NCT02234596). 30952642
Unknown unknown Advanced Solid Tumor not applicable PU-H71 Phase I Actionable In a Phase I trial, treatment with PU-H71 was generally well-tolerated, and resulted in stable disease as best response in 35% (6/14) patients with advanced solid tumors (PMID: 28808818; NCT01581541). 28808818
Unknown unknown hematologic cancer no benefit Milatuzumab Phase I Actionable In a Phase I trial, Milatuzumab (hLL1) treatment of a mixed cohort of B-cell malignancies patients, resulted in stable disease in 35% (8/23), but there was no evidence of tumor shrinkage (PMID: 25754579). 25754579
Unknown unknown Advanced Solid Tumor not applicable Metformin + Temsirolimus Phase I Actionable In a Phase I clinical trial, the combination of Torisel (temsirolimus) and Glucophage (metformin) demonstrated safety and resulted in a clinical benefit rate of 22% in patients with advanced or refractory tumors, with 4/18 patients achieving stable disease for greater than 6 cycles (PMID: 27014780). 27014780
Unknown unknown pancreatic cancer not applicable Erlotinib + Gemcitabine FDA approved Actionable In a Phase III clinical trial that supported FDA approval, the combination of Tarceva (erlotinib) and Gemzar (gemicitabine) resulted in an improved median overall survival of 6.24 months compared to 5.91 months with Gemzar (gemicitabine) and placebo, and prolonged progression-free survival (HR=0.77 (95% CI, 0.64 to 0.92; P = .004)) in patients with advanced pancreatic cancer (PMID: 17452677). 17452677 detail...
Unknown unknown acute lymphoblastic leukemia not applicable ORY-1001 Preclinical - Pdx Actionable In a preclinical study, ORY-1001 reduced leukemic blast percentage and prolonged survival in a T-cell acute lymphoblastic leukemia patient-derived xenograft (PDX) model (PMID: 29502954). 29502954
Unknown unknown triple-receptor negative breast cancer not applicable Foretinib Phase II Actionable In a Phase II trial, Foretinib (GSK1363089) treatment resulted in a clinical benefit rate of 46% (17/37) in triple-receptor negative breast cancer patients, which comprised two patients with a partial response and fifteen patients with stable disease (PMID: 27116183; NCT01147484). 27116183
Unknown unknown thyroid gland cancer not applicable ABT-737 + Doxorubicin Preclinical Actionable In a preclinical study, the combination of ABT-737 and Adriamycin (doxorubicin) was synergistic towards inhibiting cell viability of a majority of human thyroid cancer cell lines tested in culture (PMID: 27042160). 27042160
Unknown unknown endometrial cancer not applicable ETP-46464 + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, ETP-46464 increased the sensitivity of endometrial cancer cell lines to ionizing radiation in culture (PMID: 25560806). 25560806
Unknown unknown multiple myeloma not applicable Dinaciclib Preclinical - Cell line xenograft Actionable In a preclinical study, Dinaciclib (SCH 727965) decreased cell viability and growth of multiple myeloma cell lines in culture, and decreased tumor growth in multiple myeloma cell line xenograft models (PMID: 26719576). 26719576
Unknown unknown malignant glioma not applicable GDC-0084 Phase I Actionable In a Phase I trial, GDC-0084 treatment demonstrated expected toxicity and blood-brain barrier penetration, resulted in stable disease as best response in 40% (19/47) of patients with recurrent high-grade glioma (J Clin Oncol (PMID: 31937616; NCT01547546). 31937616
Unknown unknown multiple myeloma not applicable Dinaciclib + Doxorubicin Preclinical - Cell culture Actionable In a preclinical study, the combination of Dinaciclib (SCH 727965) and Adriamycin (doxorubicin) demonstrated synergy in multiple myeloma cell lines in culture, resulting in decreased cell viability (PMID: 26719576). 26719576
Unknown unknown Advanced Solid Tumor not applicable RGX-104 Phase I Actionable In a Phase I trial, RGX-104 demonstrated safety and preliminary efficacy, resulted in targeted gene expression in 100% (6/6), and activation of anti-tumor immune response in 83% (5/6) of patients with solid tumors refractory to anti-PD-1 therapy, with one renal cancer and one melanoma patient achieved stable disease (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B001). detail...
Unknown unknown renal carcinoma no benefit MG 98 Phase II Actionable In a Phase II trial, MG 98 treatment in seventeen evaluable patients resulted in stable disease in six patients, progressive disease in nine patients, and no objective responses, and the trial was terminated early due to a lack of responses (PMID: 16502349). 16502349
Unknown unknown hepatocellular carcinoma not applicable Bevacizumab + MINT1526A Phase I Actionable In a Phase I trial, MINT1526A in combination with Avastin (bevacizumab) resulted in durable minor radiographic responses in 2 patients with hepatocellular carcinoma (PMID: 29905898). 29905898
Unknown unknown Advanced Solid Tumor not applicable Ganetespib Preclinical - Cell line xenograft Actionable In a preclinical study, Ganetespib demonstrated potent anti-tumor effects against a variety of advanced solid tumor cell types and in cell line xenograft models (PMID: 22144665). 22144665
Unknown unknown Advanced Solid Tumor not applicable Alisertib + Sapanisertib Phase I Actionable In a Phase I trial, the combination of Alisertib (MLN8237) and Sapanisertib (MLN0128) demonstrated tolerability in patients with advanced solid tumors, with 70% (7/10) of patients demonstrating stable disease for a median duration of 4 months (PMID: 32414750). 32414750
Unknown unknown acute myeloid leukemia not applicable BAY2402234 Preclinical - Pdx & cell culture Actionable In a preclinical study, BAY2402234 treatment induced differentiation, cell cycle arrest and apoptosis, and inhibited proliferation of acute myeloid leukemia cell lines in culture, and induced differentiation, reduced tumor burden and increased survival in cell line and patient-derived xenograft (PDX) models (PMID: 30940908). 30940908
Unknown unknown ovarian cancer not applicable PV1019 + Topotecan Preclinical - Cell culture Actionable In a preclinical study, treatment with PV1019 combined with Hycamtin (topotecan) resulted in a synergistic effect, demonstrating greater growth inhibition of ovarian cancer cells in culture than when treated with Hycamtin (topotecan) alone (PMID: 19741151). 19741151
Unknown unknown Advanced Solid Tumor not applicable Chiauranib Phase I Actionable In a Phase I trial, Chiauranib (CS2164) demonstrated safety and preliminary efficacy, resulted in stable disease as best response in 66.7% (12/18) of patients with advanced solid tumors (PMID: 30642372; NCT02122809). 30642372
Unknown unknown colorectal cancer not applicable Bevacizumab + Fluorouracil + Irinotecan + Leucovorin + Oxaliplatin Phase II Actionable In a Phase II trial, the combination of Avastin (bevacizumab) plus FOLFOXIRI chemotherapy was well-tolerated and resulted in improved progression-free survival compared to Avastin (bevacizumab) plus FOLFOX in colorectal cancer patients (J Clin Oncol 35, 2017 (suppl 4S; abstract 658)). detail...
Unknown unknown lung non-small cell carcinoma not applicable Lenvatinib Phase I Actionable In a Phase I clinical trial, Lenvima (lenvatinib) demonstrated anti-tumor activity in patients with several advanced solid tumor types, including patients with non-small cell lung cancer (PMID: 26169970). 26169970
Unknown unknown lung 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 multiple myeloma not applicable Bortezomib FDA approved Actionable In a Phase III trial that supported FDA approval, addition of Velcade (bortezomib) to melphalan and prednisone combination therapy significantly improved time to progression (24.0 vs 16.6 months, HR=0.48, p<0.001) in patients with newly diagnosed multiple myeloma, with improved partial response rate (71% vs 35%) and complete response rate (30% vs 4%), and prolonged overall survival (HR=0.61, p=0.008) (PMID: 18753647; NCT00111319). detail... 18753647
Unknown unknown multiple myeloma not applicable Bortezomib FDA approved Actionable In a Phase III trial that supported FDA approval, Velcade (bortezomib) treatment resulted in superior response rate (38% vs 18%, p<0.01), improved time to progression (6.22 vs 3.49 months, HR=0.55, p<0.001), and overall survival (HR=0.57, p=0.001) compared to dexamethasone in patients with relapsed multiple myeloma (PMID: 15958804; NCT00048230). detail... 15958804
Unknown unknown Advanced Solid Tumor not applicable SF1126 Phase I Actionable In a Phase I trial, SF1126 demonstrated safety and preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 22921184). 22921184
Unknown unknown hematologic cancer not applicable AsiDNA + Olaparib Preclinical - Cell culture Actionable In a preclinical study, AsiDNA treatment sensitized hematologic cancer cell lines to Lynparza (olaparib), inhibiting survival in culture (PMID: 27559053). 27559053
Unknown unknown cholangiocarcinoma not applicable Silmitasertib Preclinical - Cell line xenograft Actionable In a preclinical study, the protein kinase CK2 inhibitor, Silmitasertib (CX-4945), inhibited tumor growth and increased survival of cholangiocarcinoma cell line xenograft models (PMID: 30316146). 30316146
Unknown unknown lung non-small cell carcinoma not applicable Merestinib Preclinical Actionable In a preclinical study, LY2801653 inhibited tumor growth in mouse xenograft models of non-small cell lung cancer (PMID: 24305878). 24305878
Unknown unknown 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). detail... 22204724
Unknown unknown melanoma not applicable G-TPP + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, combination of the mitochondrial Hsp90 inhibitor G-TPP and the Bcl2 inhibitor Venclexta (venetoclax) resulted in increased apoptosis of melanoma cell lines in culture (PMID: 28522750). 28522750
Unknown unknown 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 Advanced Solid Tumor not applicable Panitumumab Phase I Actionable In a Phase I trial, Vectibix (panitumumab) demonstrated safety and anti-tumor activity in patients with advanced solid tumors (PMID: 18223225). 18223225
Unknown unknown chronic lymphocytic leukemia not applicable Entospletinib + Idelalisib Phase II Actionable In a Phase II trial, Zydelig (idelalisib) and Entospletinib combination treatment resulted in objective response in 60% of patients with chronic lymphocytic leukemia, but was terminated due to severe treatment-emergent pneumonitis (PMID: 26968534). 26968534
Unknown unknown pancreatic cancer not applicable Berzosertib + Radiotherapy Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with Berzosertib (VX-970) enhanced radiotherapy efficacy in pancreatic cells resulting in apoptotic induction in culture and DNA damage and tumor growth delay in cell line xenograft models (PMID: 23222511). 23222511
Unknown unknown gastrointestinal stromal tumor not applicable Cediranib Phase II Actionable In a Phase II trial, treatment with Cediranib (AZD-2171) resulted in stable disease as best response in 55% (11/20) gastrointestinal stromal tumor patients, with 8 patients achieving stable disease for greater than or equal to 16 weeks (PMID: 24714778). 24714778
Unknown unknown pancreatic cancer not applicable SF2523 Preclinical Actionable In a preclinical study, SF2523 decreased tumor immunosuppression and inhibited tumor growth in orthotopic mouse pancreatic cancer models (PMID: 31018997). 31018997
Unknown unknown acute monocytic leukemia not applicable Carfilzomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of an acute monocytic leukemia cell line in culture (PMID: 28753594). 28753594
Unknown unknown endometrial cancer not applicable GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 treatment was well-tolerated and resulted in some efficacy in endometrial cancer patients including stable disease in 27% (4/15) and one patient with a partial response (PMID: 26603258). 26603258
Unknown unknown colon carcinoma not applicable Mps-BAY2b + Paclitaxel Preclinical Actionable In a preclinical study, Mps-BAY2b, in combination with paclitaxel, had increased efficacy in inhibiting cell proliferation of colon carcinoma cell in culture (PMID: 23933817). 23933817
Unknown unknown hepatocellular carcinoma not applicable Mogamulizumab + Nivolumab Phase I Actionable In a Phase I trial, Poteligeo (mogamulizumab-kpkc) and Opdivo (nivolumab) combination treatment demonstrated acceptable safety, resulted in an objective response rate of 27% (4/15, 4 partial responses) and a disease control rate of 67% (10/15) in immunotherapy-naive patients with advanced or metastatic hepatocellular carcinoma, response occurred regardless of PD-L1, CCR4, CD8 expression levels and tumor mutational burden (PMID: 31455681; NCT02476123). 31455681
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 leukemia not applicable MC180295 Preclinical - Cell culture Actionable In a preclinical study, MC180295 decreased proliferation and increased differentiation of leukemia cells in culture (PMID: 30454645). 30454645
Unknown unknown lung non-small cell 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 lymphoma not applicable Panobinostat + PQR309 Preclinical - Cell culture Actionable In a preclinical study, the combination therapy of Farydak (panobinostat) and PQR309 induced apoptosis and led to synergistic and additive effects in lymphoma cell lines in culture (PMID: 29066507). 29066507
Unknown unknown gastrointestinal stromal tumor not applicable G007-LK Preclinical Actionable In a preclinical study, G007-LK demonstrated anti-tumor activity in a mouse model of gastrointestinal stromal tumor expressing Kit V558del (corresponding to human V559del) (PMID: 28611108). 28611108
Unknown unknown pancreatic ductal adenocarcinoma not applicable Gemcitabine + Pelareorep 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 diffuse large B-cell lymphoma not applicable GSK3368715 Preclinical - Patient cell culture Actionable In a preclinical study, GSK3368715 treatment inhibited growth of a diffuse large B-cell lymphoma (DLBCL) cell line in culture and inhibited tumor growth in xenograft models, and inhibited growth of patient-derived DLBCL cells in culture PMID: 31257072). 31257072
Unknown unknown Advanced Solid Tumor not applicable DS6051b Phase I Actionable In a Phase I clinical trial, DS-6051b was well-tolerated and demonstrated some preliminary efficacy in patients with advanced solid tumors (Cancer Res July 15 2016 (76) (14 Supplement) CT024). detail...
Unknown unknown Advanced Solid Tumor not applicable Imalumab Phase I Actionable In a Phase I trial, treatment with Imalumab (BAX069) demonstrated safety and tolerability, and resulted in a best overall response of stable disease in 26% (13/50) of patients with an advanced solid tumor, with 8 patients achieving stable disease for 4 months or longer (PMID: 32207164; NCT01765790). 32207164
Unknown unknown leukemia not applicable H8F4 CAR-T cells Preclinical - Cell culture Actionable In a preclinical study, h8F4 CAR-T cells induced lysis of leukemia cell lines expressing PR1/HLA-A2 in culture (PMID: 27265873). 27265873
Unknown unknown clear cell renal cell carcinoma not applicable GSK3368715 Preclinical - Cell culture Actionable In a preclinical study, GSK3368715 inhibited tumor growth in a clear cell renal cell carcinoma xenograft model (PMID: 31257072). 31257072
Unknown unknown Advanced Solid Tumor not applicable E7046 Phase I Actionable In a Phase I trial, E7046 treatment resulted in no objective responses, only stable disease in 13% (4/30) and metabolic responses in 13% (4/30) of patients with advanced solid tumors (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 373PD; NCT02540291). detail...
Unknown unknown Advanced Solid Tumor not applicable E7046 Preclinical Actionable In a preclinical study, multiple mouse tumor models demonstrated inhibition of tumor growth when treated with E7046 (Cancer Immunol Res 2016;4(1 Suppl):Abstract nr B034). detail...
Unknown unknown colon cancer not applicable E7046 + Radiotherapy Preclinical Actionable In a preclinical study, the combination treatment of E7046 and radiotherapy resulted in 9 out of 12 tumor free colon cancer mouse models and increased the number of T-cells infiltrating the tumor (International Journal of Rad Onc, 2016, 96;2, S128). detail...
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 ovarian cancer not applicable SCH 58500 Phase Ib/II Actionable In a Phase I/II trial, SCH 58500 demonstrated safety and preliminary efficacy, resulted in measurable transgene expression in 85% (17/20) of biopsy samples, and more than 50% reduction of CEA level in 50% (8/16) of ovarian cancer patients who received three cycles of treatment (PMID: 12082455). 12082455
Unknown unknown ovarian cancer not applicable SCH 58500 Phase I Actionable In a Phase I trial, treatment with multiple doses of SCH 58500 in combination with chemotherapy resulted in a median survival of 12-13 months in heavily pretreated ovarian cancer patients, with 10 patients surviving more than 20 months (PMID: 12082456). 12082456
Unknown unknown prostate cancer not applicable Carnosic acid Preclinical Actionable In a preclinical study, carnosic acid treatment of prostate cancer cells led to activation of PP2A, which resulted in apoptosis and inhibition of cell proliferation (PMID: 22453599). 22453599
Unknown unknown gastric 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 lung cancer not applicable Triolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Triolimus led to cytotoxicity and inhibited Ras/Raf/MAPK and PI3K/Akt/mTOR pathway signaling in lung cancer cells in culture, and induced apoptosis, which resulted in tumor growth inhibition, in cell line xenograft models (PMID: 22896668). 22896668
Unknown unknown colon cancer not applicable DSP-0509 + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, an anti-PD-1 inhibitor combined with DSP-0509 inhibited tumor growth in a syngeneic mouse model of colon cancer (Cancer Res July 1 2018 (78) (13 Supplement) 4726). detail...
Unknown unknown pancreatic adenocarcinoma not applicable Cabozantinib Preclinical Actionable In a preclinical trial, Cometriq (cabozantinib) promoted apoptosis of pancreactic ductal adenocarcinoma cells (PMID: 23661005). 23661005
Unknown unknown acute lymphoblastic leukemia not applicable AS605240 + Prednisolone Preclinical - Cell culture Actionable In a preclinical study, AS605240 and prednisolone synergistically inhibited survival of T-acute lymphocytic leukemia cell lines in culture (PMID: 25869207). 25869207
Unknown unknown malignant glioma not applicable RES-529 Preclinical - Cell line xenograft Actionable In a preclinical study, RES-529 (Palomid 529) inhibited tumor growth and angiogenesis in glioma cell line xenograft models (PMID: 19010932). 19010932
Unknown unknown neuroblastoma not applicable GANT61 + Vincristine Sulfate 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 chronic lymphocytic leukemia not applicable PRT318 Preclinical Actionable In a preclinical study, treatment with PRT318 resulted in increased apoptosis and decreased migration of primary chronic lymphocytic leukemia cells in culture (PMID: 22362000). 22362000
Unknown unknown colon cancer not applicable Doxorubicin + NU7441 Preclinical - Cell culture Actionable In a preclinical study, NU7441 increased sensitivity of colon cancer cell lines to Adriamycin (doxorubicin), resulting in reduced cell survival in culture (PMID: 16707462). 16707462
Unknown unknown hepatocellular carcinoma not applicable Riluzole Preclinical - Cell culture Actionable In a preclinical study, Rilutek (riluzole) inhibited growth of hepatocellular cancer cell lines and primary hepatocellular cancer lines in culture (PMID: 27612558). 27612558
Unknown unknown Advanced Solid Tumor not applicable E7107 Phase I Actionable In a Phase I trial, E7107 treatment resulted in stable disease in 31% (8/26) of patients with advanced solid tumors, however, the study was discontinued due to vision loss in two patients (PMID: 24258465; NCT00499499). 24258465
Unknown unknown uveal melanoma no benefit Trametinib Phase I Actionable In a Phase I trial, Mekinist (trametinib) treatment resulted in stable disease as best response in 50% (8/16) of patients with uveal melanoma (PMID: 22805292; NCT00687622). 22805292
Unknown unknown stomach cancer not applicable DKN-01 + Paclitaxel Phase I Actionable In a Phase I trial, DKN-01 and Taxol (paclitaxel) combination treatment resulted in partial response in 18% (6/34) and stable disease in 32% (11/34) of advanced esophagogastric cancer patients, with a median progression-free survival of 13.7 weeks and an overall survival of 28.4 weeks (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #91P; NCT02013154). detail...
Unknown unknown neuroblastoma not applicable Hu3F8-BsAb Preclinical - Cell line xenograft Actionable In a preclinical study, Hu3F8-BsAb treatment induced cell killing in neuroblastoma cell lines expressing GD2 in culture, and treatment with Hu3F8-BsAb plus human peripheral blood mononuclear cells inhibited tumor growth in neuroblastoma cell line xenograft models (PMID: 25542634). 25542634
Unknown unknown gastrointestinal stromal tumor not applicable Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Sutent (sunitinib) improved median progression free survival to 27.3 weeks in GIST patients (PMID: 17332278). 17332278 detail...
Unknown unknown marginal zone B-cell lymphoma not applicable ME-401 Phase I Actionable In a Phase I trial, ME-401 treatment was well-tolerated, resulted in an objective response rate of 100% (4/4) in patients with relapsed or refractory marginal zone B-cell lymphoma (J Clin Oncol 38: 2020 (suppl; abstr 8016); NCT02914938). detail...
Unknown unknown bladder urothelial carcinoma not applicable VAX014 Preclinical Actionable In a preclinical study, VAX014 induced cell-killing in dissociated mouse and human urothelial carcinoma cell lines in culture, and inhibited tumor growth and improved survival in a syngeneic bladder cancer model (PMID: 27919942). 27919942
Unknown unknown sarcoma not applicable Brivanib Phase II Actionable In a Phase II trial, treatment with Brivanib (BMS-540215) demonstrated safety and resulted in a median progression-free survival of 2.8 months in soft-tissue sarcoma patients, compared to 1.4 months with placebo, and FGF2 expression was not found to be a predictive biomarker for response (PMID: 31522033; NCT00633789). 31522033
Unknown unknown ovarian cancer not applicable Fluzoparib Preclinical - Cell culture Actionable In a preclinical study, Fluzoparib inhibited DNA-damage-induced PARylation in high-grade serous ovarian cancer cell lines in culture, however, the cells demonstrated resistance to Fluzoparib treatment (PMID: 30949414). 30949414
Unknown unknown lung small cell carcinoma not applicable Ipilimumab + Nivolumab Guideline Actionable Opdivo (nivolumab) and Yervoy (ipilimumab) combination treatment is included in guidelines for small cell lung cancer patients who have relapsed after primary therapy (NCCN.org). detail...
Unknown unknown multiple myeloma not applicable Carfilzomib + Ricolinostat Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Kyprolis (carfilzomib) and Ricolinostat (ACY-1215) promoted apoptosis and reduced tumor volume in a human multiple myeloma cell line xenograft model (PMID: 25709080). 25709080
Unknown unknown 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 astrocytoma not applicable Niraparib + Radiotherapy Preclinical - Cell line xenograft Actionable In a preclinical study, a pediatric high grade astrocytoma cell line treated with a combination of ionizing radiation and Zejula (niraparib) demonstrated a greater reduction in cell survival in culture and a better survival benefit in xenograft models compared to either agent alone (PMID: 26351319). 26351319
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 Ewing sarcoma not applicable MEDI-573 + Sirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of MEDI-573 and Rapamune (sirolimus) resulted in decreased Rapamune (sirolimus)-induced AKT activation and inhibited growth of a Ewing sarcoma cell line in culture and inhibited tumor growth Ewing sarcoma cell line xenograft models, with increased efficacy compared to either as a single agent (PMID: 25193511). 25193511
Unknown unknown Ewing sarcoma not applicable Prexasertib Preclinical - Cell culture Actionable In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including Ewing sarcoma cell lines (PMID: 28270495). 28270495
Unknown unknown stomach cancer not applicable Geldanamycin Preclinical Actionable In a preclinical study, Geldanamycin inhibited proliferation of a human gastric cancer cell line in culture (PMID: 26788199). 26788199
Unknown unknown ovary epithelial cancer not applicable Afuresertib + Carboplatin + Paclitaxel Phase I Actionable In a Phase Ib trial, the combination of Afuresertib (GSK2110183) , Paraplatin (carboplatin), and Taxol (paclitaxel) was well-tolerated and demonstrated preliminary efficacy in patients with platinum-resistant epithelial ovarian cancer, resulting in an overall response rate in the dose-expansion part of the trial (Part II) of 32% (9/28) and a median progression-free survival of 7.1 months (PMID: 30563934; NCT01653912). 30563934
Unknown unknown mantle cell lymphoma not applicable Acalabrutinib Phase II Actionable In a Phase II trial (ACE-LY-004) that supported FDA approval, Calquence (acalabrutinib) treatment resulted in an overall response rate of 81% (100/124, 49 complete response) in patients with relapsed or refractory mantle cell lymphoma (PMID: 29241979; NCT02213926). 29241979 detail...
Unknown unknown Advanced Solid Tumor not applicable Bevacizumab + MINT1526A Phase I Actionable In a Phase I trial, MINT1526A in combination with Avastin (bevacizumab) resulted in 1 partial response and 2 durable minor radiographic responses in a total of 30 patients with advanced solid tumors (PMID: 29905898). 29905898
Unknown unknown granulosa cell tumor no benefit STM434 Phase I Actionable In a Phase I trial, STM 434 treatment did not result in response in 30 evaluable patients with advanced solid tumors and only resulted in stable disease as best response in 80% (10/12) of patients with granulosa cell ovarian cancer (PMID: 31068369; NCT02262455). 31068369
Unknown unknown 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 Her2-receptor negative breast cancer not applicable Sorafenib Clinical Study Actionable In a meta-analysis of 844 ERBB2 (HER2)-negative breast cancer patients, Nexavar (sorafenib) increased progression-free survival time, but not overall survival or objective response rate (PMID: 24940450). 24940450
Unknown unknown Advanced Solid Tumor not applicable 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 leiomyosarcoma not applicable Aldoxorubicin + Buparlisib Preclinical Actionable In a preclinical study, the combination of BKM120 and doxorubicin produced an additive effect when treating leiomyosarcoma cells (PMID: 26952093). 26952093
Unknown unknown colorectal cancer not applicable Sabatolimab + Spartalizumab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in partial response in 2 patients with colorectal cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown lung cancer not applicable CT16 Preclinical - Pdx Actionable In a preclinical study, CT16 treatment decreased NOTCH and EGFR signaling and inhibited tumor growth in lung cancer patient-derived xenograft (PDX) models sensitive to EGFR blockade (PMID: 28275151). 28275151
Unknown unknown ovarian cancer not applicable Brivanib Phase II Actionable In a Phase II trial, treatment with Brivanib (BMS-540215) demonstrated safety and resulted in a median progression-free survival of 4.0 months in ovarian cancer patients, compared to 2.0 months with placebo, and FGF2 expression was not found to be a predictive biomarker for response (PMID: 31522033; NCT00633789). 31522033
Unknown unknown breast cancer not applicable Lucitanib Phase II Actionable In a Phase II trial, Lucitanib (E-3810) demonstrated acceptable toxicity and activity, resulted in a median progression-free survival of 3.5 and 2.6 months for 10mg and 15mg treatment groups respectively, with no differences in response correlated with FGFR1 and 11q amplification status in metastatic breast cancer patients (Cancer Res 2017;77(4 Suppl):Abstract nr P6-11-03). detail...
Unknown unknown breast cancer not applicable SF2523 Preclinical Actionable In a preclinical study, SF2523 decreased tumor immunosuppression, and inhibited tumor growth and progression in a mouse breast cancer model (PMID: 31018997). 31018997
Unknown unknown hepatocellular carcinoma not applicable Ipilimumab + Nivolumab FDA approved Actionable In a Phase I (CheckMate 040) trial that supported FDA approval, combination of Opdivo (nivolumab) and Yervoy (ipilimumab) was well tolerated, resulted in an objective response rate of 32% (16/50, 4 complete response, 12 partial response) with a median duration of response of 17 months in patients with hepatocellular carcinoma previously treated with Nexavar (sorafenib) (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 4012-4012; NCT01658878). detail... detail... detail...
Unknown unknown ovarian cancer not applicable Paclitaxel + VB-111 Case Reports/Case Series Actionable In a clinical study, VB-111 in combination with Taxol (paclitaxel) resulted in increased tumor lymphocyte infiltration and tumor necrosis in biopsies obtained from 3 patients with ovarian cancer (AACR Annual Meeting 2019, Abstract 4979). detail...
Unknown unknown oral squamous cell carcinoma not applicable Trametinib Phase II Actionable In a Phase II trial, Mekinist (trametinib) treatment in patients with oral cavity squamous cell carcinoma was associated with decreased phosphorylation of Erk1/2 and reduced CD44 expression, and resulted in a partial response in 65% (11/17), stable disease in 29% (5/17), and progressive disease in 1 patient (6%) (PMID: 28151720). 28151720
Unknown unknown Advanced Solid Tumor not applicable ETC-159 Phase I Actionable In a Phase I trial, treatment with ETC-159 was well-tolerated, decreased Wnt signaling, and resulted in stable disease in 2 out of 16 treated patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2584)). detail...
Unknown unknown melanoma not applicable BO-112 Preclinical - Patient cell culture Actionable In a preclinical study, BO-112 treatment inhibited cell viability and induced apoptosis in mouse melanoma cells, and induced cytotoxicity and increased apoptosis in cells derived from metastatic lesions of melanoma patients in culture, and intratumoral delivery of BO-112 enhanced infiltration of T-lymphocytes, reduced tumor growth, and delayed progression in syngeneic mouse models (PMID: 31046839). 31046839
Unknown unknown 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 uveal melanoma not applicable Fotemustine + S44563 Preclinical - Pdx Actionable In a preclinical study, treatment with S44563 in combination with or after Fotemustine resulted in improved efficacy compared to either agent alone in patient-derived xenograft models of uveal melanoma (PMID: 24454684). 24454684
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 Advanced Solid Tumor not applicable Erlotinib + Ficlatuzumab Phase I Actionable In a Phase I trial, Ficlatuzumab (AV-299) in combination with Tarceva (erlotinib) was shown to be safe in treating patients with advanced solid tumors (PMID: 23493885, J Clin Oncol. 2010;28(Suppl) Abstract 2525). 23493885 detail...
Unknown unknown breast cancer not applicable AJI-100 Preclinical - Pdx & cell culture Actionable In a preclinical study, AJI-100 induced apoptosis and inhibited cell growth of breast cancer cell lines and xenografts (PMID: 24930769). 24930769
Unknown unknown 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 colorectal 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 5% (2/41) and stable disease in 17% (7/41) of colorectal cancer patients, with infrequent treatment-induced increase of PD-L1 expression and CD8+ T cells (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 3001-3001; NCT02335918). detail...
Unknown unknown chronic myeloid leukemia not applicable Nilotinib + Tazemetostat Preclinical Actionable In a preclinical study, treatment with the combination of Tasigna (Nilotinib) and EPZ-6438 resulted in decreased levels of leukemic cells and progenitors in primary chronic myeloid leukemia cell xenograft models, with increased efficacy compared to Tasigna (Nilotinib) alone (PMID: 27630125). 27630125
Unknown unknown melanoma 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 melanoma cell lines in culture, and inhibited tumor growth in cell line xenograft models (PMID: 28522750). 28522750
Unknown unknown Advanced Solid Tumor not applicable Sabatolimab + Spartalizumab Phase Ib/II Actionable In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in partial response in 5% (4/86) and stable disease in 40% (34/86) of patients with advanced solid tumor (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown 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 head and neck squamous cell carcinoma not applicable Abemaciclib Preclinical Actionable In a preclinical study, Abemaciclib (LY2835219) reduced RB and AKT activation, and inhibited cell proliferation and colony formation in head and neck squamous cell carcinoma (HNSCC) cell lines in culture, and inhibited tumor growth in HNSCC xenograft models (PMID: 26909611). 26909611
Unknown unknown diffuse large B-cell lymphoma not applicable TTI-621 Preclinical - Cell line xenograft Actionable In a preclinical study, the mouse surrogate version of TTI-621 (SIRPalpha-Fc) decreased tumor growth in a diffuse large B-cell lymphoma cell line xenograft model (PMID: 27856600). 27856600
Unknown unknown breast cancer not applicable Eribulin Phase III Actionable In a randomized Phase III clinical trial, Halaven (eribulin) monotherapy demonstrated significantly improved survival (median 13.1 months) relative to treatment of physician’s choice (median 10.6 months) across 762 patients with heavily pretreated metastatic breast cancer (PMID: 21376385). 21376385
Unknown unknown gastric adenocarcinoma not applicable Ramucirumab FDA approved Actionable In a Phase III trial (REGARD) that supported FDA approval, treatment with Cyramza (ramucirumab) improved median overall survival (5.2 vs 3.8 mo, HR=0.776, p=0.047) in aptients with compared to placebo in patients with advanced gastric or gastro-oesophageal junction adenocarcinoma whose disease progressed after chemotherapy (PMID: 24094768; NCT00917384). 24094768 detail...
Unknown unknown Advanced Solid Tumor not applicable IHSF115 Preclinical - Cell culture Actionable In a preclinical study, IHSF115 inhibited growth of a panel of cancer cell lines in culture (PMID: 28369544). 28369544
Unknown unknown pancreatic adenocarcinoma not applicable CGP57380 Preclinical Actionable In a preclinical study, CGP57380 decreased migration of pancreatic adenocarcinoma (PDAC) cells in culture and inhibited growth of organoids generated from human PDAC samples (PMID: 26609108). 26609108
Unknown unknown head and neck cancer not applicable Cetuximab + Ficlatuzumab Phase I Actionable In a Phase I trial, the combination of Erbitux (cetuximab) and Ficlatuzumab (AV-299) demonstrated tolerability and resulted in an overall response rate of 17% (2/12), median progression-free survival of 5.4 months, and overall survival of 8.9 months in patients with advanced head and neck squamous cell carcinoma (PMID: 32545260; NCT02277197). 32545260
Unknown unknown ovarian cancer no benefit Pimasertib + SAR245409 Phase II Actionable In a Phase II trial, the combination of Pimasertib (MSC1936369B) and XL765 (SAR245409) versus Pimasertib (MSC1936369B) alone resulted in an objective response rate (ORR) of 9.4% and 12.1%, and a median progression-free survival of 9.99 mo and 7.23 mo, respectively, in patients with ovarian carcinoma (n=65), and 18 pts treated with combination and 19 pts treated with single therapy discontinued the study, and thus, the study was terminated due to a low ORR and high rate of discontinuation (PMID: 31870556). 31870556
Unknown unknown colorectal cancer no benefit Atezolizumab Phase III Actionable In a Phase III trial (IMblaze370), Tecentriq (atezolizumab) treatment did not improve median overall survival (7.1 vs 8.5 months, HR=1.19) compared to Stivarga (regorafenib) in patients with chemotherapy-refractory metastatic colorectal cancer, 91.7% of whom were microsatellite stable or microsatellite instability-low (Annals of Oncology, Volume 29, Issue suppl_5, 1 June 2018; NCT02788279). detail...
Unknown unknown malignant glioma not applicable Plicamycin Preclinical - Cell culture Actionable In a preclinical study, treatment with Mithracin (plicamycin) induced cell death and inhibited migration of glioma cell lines in culture (PMID: 20556479). 20556479
Unknown unknown epithelioid sarcoma not applicable Dasatinib Phase II Actionable In a Phase II trial, patients with epithelioid sarcoma demonstrated a median progression free survival of 7.9 months and two patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown female reproductive organ cancer not applicable Durvalumab + Olaparib Phase I Actionable In a Phase I trial, the combination therapy of Imfinzi (durvalumab) and Lynparza (olaparib) resulted in an overall response rate of 17% (2/12) and disease control rate of 83% (10/12) in patients with female reproductive organ cancer (PMID: 28471727). 28471727
Unknown unknown neuroblastoma not applicable AMXT1501 + Eflornithine Preclinical - Cell culture Actionable In a preclinical study, AMXT1501 and Eflornithine (DFMO) combination treatment resulted in depletion of intracellular polyamine pools and decreased intracellular ATP, which led to cell cycle arrest and apoptosis in neuroblastoma cell lines in culture (PMID: 23457004). 23457004
Unknown unknown rhabdoid cancer not applicable BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 100% (3/3) of cell line xenograft models of rhabdoid tumor (PMID: 21298745). 21298745
Unknown unknown breast cancer no benefit Imatinib Phase II Actionable In a Phase II trial, Gleevec (imatinib mesylate) treatment demonstrated significant toxicity and no clinical benefit in patients with heavily pre-treated metastatic breast cancer, of the 13 tested patients, one was positive for Kit and 4 were positive for Pdgfr (PMID: 15803362). 15803362
Unknown unknown clear cell renal cell carcinoma not applicable Everolimus + Hydroxychloroquine Phase Ib/II Actionable In a Phase I/II trial, treatment with the combination of Afinitor (everolimus) and Plaquenil (hydroxychloroquine) was well-tolerated, and resulted in a median progression-free survival (PFS) of 6.3 months, PFS of 6 months or greater in 45% (15/33), and partial response (PR) or stable disease (SD) greater than 3 months in 66% (22/33; 2 PR and 20 SD) of clear cell renal cell carcinoma patients (PMID: 30635337). 30635337
Unknown unknown renal cell carcinoma not applicable Bevacizumab + Temsirolimus Phase II Actionable In a Phase II trial, patients with metastatic renal cell carcinoma (mRCC) treated with a combination of Avastin (bevacizumab) and Torisel (temsirolimus) demonstrated an overall 4 month PFS rate of 65% while those patients with VEGFR tyrosine kinase inhibitor refractory mRCC showed a median PFS and OS of 6.5 and 9.6 months, respectively (PMID: 27036973). 27036973
Unknown unknown Advanced Solid Tumor not applicable Ralimetinib Phase I Actionable In a Phase I trial, Ralimetinib (LY2228820) resulted in stable disease in 21.3% (19/74) of patients with an advanced solid tumor, which lasted a median duration of 3.7 months (PMID: 26581242). 26581242
Unknown unknown Advanced Solid Tumor not applicable AMC303 Preclinical - Cell line xenograft Actionable In a preclinical study, AMC303 resulted in decreased cell invasion and migration in solid tumor cell lines in culture and tumor regression of metastatic lesions of the liver in xenograft models (Cancer Res 2017;77(13 Suppl):Abstract nr 4911). detail...
Unknown unknown Advanced Solid Tumor not applicable Regorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Stivarga (regorafenib) showed antitumor activity in multiple murine xenograft models derived from melanoma, renal cell carcinoma, colorectal, breast, lung, pancreatic and ovarian tumor cell lines (PMID: 21170960). 21170960
Unknown unknown Advanced Solid Tumor not applicable Regorafenib Phase I Actionable In a Phase I trial, Stivarga (regorafenib) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22421192). 22421192
Unknown unknown lung non-small cell carcinoma not applicable unspecified PD-1 antibody Clinical Study Actionable In a retrospective analysis, treatment with an unspecified PD-1 or PD-L1 therapy in metastatic non-small cell lung cancer patients harboring a mutation in BRAF, ERBB2 (HER2), or MET, or a RET translocation led to a response rate of 29% (31/107), a 15.4 mo duration of response, a 4.7 mo median progression-free survival, and a 16.2 mo median overall survival, and resulted in clinical efficacy similar to what has been observed in studies with unselected non-small cell lung cancer patients (PMID: 31945494). 31945494
Unknown unknown renal cell carcinoma not applicable Sunitinib + Trametinib Preclinical - Cell line xenograft Actionable In a preclinical study, combination of Mekinist (trametinib) and Sutent (sunitinib) effectively inhibited tumor angiogenesis and growth in cell line xenograft models of Sutent (sunitinib)-refractory renal cell carcinoma (PMID: 26487278). 26487278
Unknown unknown 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 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 breast cancer not applicable ST7612AA1 Preclinical - Cell line xenograft Actionable In a preclinical study, ST7612AA1 inhibited proliferation of breast cancer cell lines in culture, and inhibited tumor growth in breast cancer cell line xenograft models (PMID: 25671299). 25671299
Unknown unknown melanoma not applicable Denileukin diftitox + Sirolimus Preclinical Actionable In a preclinical study, the combination of Ontak (denileukin diftitox) and Rapamune (sirolimus) resulted in a greater decrease in tumor volume compared to treatment with either agent alone in a melanoma mouse model (PMID: 27737881). 27737881
Unknown unknown endometrial cancer not applicable Sunitinib Phase II Actionable In Phase II clinical trials, Sutent (sunitinib) demonstrated efficacy in patients with metastatic or recurrent endometrial carcinoma (PMID: 24882554). 24882554
Unknown unknown 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 Advanced Solid Tumor not applicable Adavosertib + Gemcitabine Phase I Actionable In a Phase I trial, the combination of Adavosertib (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 diffuse large B-cell lymphoma not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in objective response in 31% (4/13) and stable disease in 15% (2/13) of patients with diffuse large B-cell lymphoma (PMID: 29475723; NCT01767766). 29475723
Unknown unknown 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 ovarian cancer not applicable RO5520985 Phase I Actionable In a Phase I clinical trial, Vanucizumab (RO5520985) treatment resulted in partial response in 29% (12/41) and stable disease in 54% of (22/41) platinum (Pt)- resistant/refractory epithelial ovarian cancer patients (J Clin Oncol 33, 2015 (suppl; abstr 5549)). detail...
Unknown unknown pancreatic cancer not applicable Nintedanib Preclinical - Cell line xenograft Actionable In a preclinical study, Nintedanib, alone or with chemotherapy, inhibited tumor growth in cell line xenograft models of lung and pancreatic cancer but not in cell culture (PMID: 23729403). 23729403
Unknown unknown ovarian cancer not applicable Lurbinectedin Phase II Actionable In a Phase II trial, Lurbinectedin (PM01183) treatment resulted in an overall response rate of 23% (12/52) of patients with platinum-resistant or refractory ovarian cancer, with a median duration of response of 4.6 months (PMID: 28368437). 28368437
Unknown unknown lung adenocarcinoma not applicable Docetaxel + Nintedanib 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 PF-03814735 Phase I Actionable In a Phase I trial, PF-03814735 demonstrated safety and preliminary efficacy in advanced solid tumor patients (PMID: 21852114). 21852114
Unknown unknown lung non-small cell carcinoma not applicable Barasertib + Navitoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of Barasertib (AZD1152) and Navitoclax (ABT-263) resulted in synergy, inhibiting proliferation of non-small cell lung carcinoma cells in culture (PMID: 28179288). 28179288
Unknown unknown B-cell lymphoma not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) treatment resulted in significant toxicity and an objective response rate of 15% (2/13) in patients with B-cell non Hodgkin's lymphoma (PMID: 28278718). 28278718
Unknown unknown malignant pleural mesothelioma not applicable Cisplatin + Trabectedin Preclinical - Cell culture Actionable In a preclinical study, the combination of Yondelis (trabectedin) and Platinol (cisplatin) demonstrated synergy in inducing apoptosis and decreasing viability of malignant pleural mesothelioma cell lines in culture (PMID: 27512118). 27512118
Unknown unknown Advanced Solid Tumor not applicable Afatinib + Vinorelbine Phase I Actionable In a Phase I trial, the combination of Gilotrif (afatinib) and Navelbine (vinorelbine) resulted in clinical efficacy, including two breast cancer patients with a partial response and stable disease in eight patients with advanced solid tumors (PMID: 26254023). 26254023
Unknown unknown aggressive digital papillary adenocarcinoma not applicable Atezolizumab + Hu5F9-G4 Case Reports/Case Series Actionable In a Phase Ib trial, Tecentriq (atezolizumab) and Magrolimab (Hu5F9-G4) combination therapy resulted in a partial response lasted 4 months in a patient with papillary adenocarcinoma of the finger (J Clin Oncol 38, 2020 (suppl 5; abstr 18); NCT03558139). detail...
Unknown unknown acute myeloid leukemia not applicable ONC201 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of ONC201 and Venclexta (venetoclax) demonstrated synergy in acute myeloid leukemia cell lines in culture, resulting in increased induction of apoptosis (PMID: 26884599). 26884599
Unknown unknown Advanced Solid Tumor not applicable Paclitaxel + Taladegib Phase I Actionable In a Phase I trial, combination of Taladegib and Taxol (paclitaxel) treatment demonstrated safety and preliminary efficacy, resulted in partial response in 3 patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2594)). detail...
Unknown unknown 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 ovarian carcinoma no benefit Motolimod + Pegylated liposomal-doxorubicin Phase II Actionable In a Phase II trial, the inclusion of Motolimod (VTX-2337) with Doxil (pegylated liposomal-doxorubicin) did not result in improved survival in patients with either ovarian, fallopian tube, or primary peritoneal carcinoma (PMID: 28453702; NCT01666444). 28453702
Unknown unknown lung non-small cell carcinoma not applicable Sabatolimab + Spartalizumab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in partial response in a patient with non-small cell lung cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown colon cancer not applicable Torin 1 Preclinical - Pdx Actionable In a preclinical study, Torin 1 inhibited cell proliferation of colon cancer cell cultures and patient-derived xenograft models (PMID: 24185040). 24185040
Unknown unknown 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 lung small cell carcinoma not applicable Etoposide + NVP-ADW742 Preclinical Actionable In a preclinical study, NVP-ADW742, when combined with Toposaur (etoposide), demonstrated a synergistic effect in small cell lung cancer cell lines, resulting in inhibition of Akt signaling (PMID: 15746061). 15746061
Unknown unknown Advanced Solid Tumor not applicable Carboxyamidotriazole Orotate + Paclitaxel Phase I Actionable In Phase I trial, carboxyamidotriazole in combination with Taxol (paclitaxel) displayed safety and efficacy in patients with solid tumors (PMID: 19738417). 19738417
Unknown unknown gastric adenocarcinoma not applicable Regorafenib Phase II Actionable In a Phase II trial, Stivarga (regorafenib) resulted in a PFS of 2.6 months compared to .9 months with placebo in gastric adenocarcinoma patients (PMID: 27325864). 27325864
Unknown unknown colorectal cancer not applicable Fluorouracil + Quinacrine + Sorafenib Preclinical - Cell culture Actionable In a preclinical study, Acrichine (quinacrine) and Adrucil (5-fluorouracil) synergistically enhanced the cytotoxicity of Nexavar (sorafenib) in human colorectal cancer cell lines in culture (PMID: 21725213). 21725213
Unknown unknown breast carcinoma not applicable BI 836880 Case Reports/Case Series Actionable In a Phase I trial, BI 836880 treatment was well tolerated in patients with advanced solid tumors, and resulted in 2 partial responses (n=29), including a partial response in a patient with breast carcinoma (J Clin Onc 2018 36:15_suppl, 12024; NCT02674152). detail...
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 ovary epithelial cancer not applicable Bevacizumab + Carboplatin + Paclitaxel Phase II Actionable In a Phase II trial, addition of Avastin (bevacizumab) to neoadjuvant chemotherapy (Paraplatin (carboplatin) plus Taxol (paclitaxel)) did not improve complete macroscopic response rate (2/35 vs 2/33), or median progression free survival (20.4 vs 20.1 months) compared to control in patients with advanced epithelial ovarian cancer, but resulted in favorable rate of surgical feasibility (66.7 vs 88.6%) (J Clin Oncol 35, 2017 (suppl; abstr 5508)). detail...
Unknown unknown ovary epithelial cancer not applicable Bevacizumab + Carboplatin + Paclitaxel FDA approved Actionable In a Phase III trial (GOG-0218) that supported FDA approval, addition of Avastin (bevacizumab) during and after Paraplatin (carboplatin) plus Taxol (paclitaxel) treatment prolonged progression-free survival (14.1 vs 10.3 months) compared to placebo in patients with previously untreated, stage III/IV epithelial ovarian, primary peritoneal, or fallopian tube cancer (PMID: 22204724; NCT00262847). detail... 22204724
Unknown unknown triple-receptor negative breast cancer not applicable Docetaxel + JQ1 Preclinical - Cell culture Actionable In a preclinical study, the addition of JQ1 to Taxotere (docetaxel) resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27256375). 27256375
Unknown unknown bone giant cell tumor not applicable Denosumab FDA approved Actionable In a Phase II clinical trial that supported FDA approval, Xgeva (denosumab) treatment resulted in an overall objective response rate of 25% (47/187) in patients with giant cell bone tumor, with a median duration of response of 20 months among patients demonstrating response (PMID: 25617146). detail... 25617146
Unknown unknown Hodgkin's lymphoma not applicable Brentuximab vedotin + Dacarbazine + Doxorubicin + Vinblastine FDA approved Actionable In a Phase III trial (ECHELON-1) that supported FDA approval, Adcetris (brentuximab vedotin) in combination with Deticene (dacarbazine), Adriamycin (doxorubicin), and Velban (vinblastine) improved modified progression-free survival rate (82.1% vs 77.2%, HR=0.77, p=0.04) compared to the combination of Adriamycin (doxorubicin), Blenoxane (Bleomycin), Velban (vinblastine) and Deticene (dacarbazine) in patients with untreated stage III or IV classical Hodgkin's lymphoma (PMID: 29224502; NCT01712490). 29224502 detail...
Unknown unknown Advanced Solid Tumor not applicable CV301 Phase I Actionable In a Phase I trial, CV301 treatment was well-tolerated and resulted in a median progression-free survival of 15 weeks in advanced solid tumor patients (n=12) (PMID: 31110074; NCT02840994). 31110074
Unknown unknown follicular lymphoma not applicable Lenalidomide + Rituximab FDA approved Actionable In a Phase III trial (AUGMENT) that supported FDA approval, Revlimid (lenalidomide) in combination with Rituxan (rituximab) resulted in significantly improved progression-free survival (39.4 vs 14.1 months, HR=0.46, p<0.001) compared to placebo and Rituxan (rituximab) in patients with relapsed and/or refractory follicular or marginal zone lymphoma (PMID: 30897038; NCT01938001). 30897038 detail...
Unknown unknown colon carcinoma not applicable Hydroxyurea + MU380 Preclinical - Cell culture Actionable In a preclinical study, the addition of MU380 resulted in increased sensitivity to Droxia (hydroxyurea) in colon carcinoma cell lines in culture, leading to decreased proliferation (PMID: 28619751). 28619751
Unknown unknown liposarcoma not applicable Palbociclib Phase II Actionable In a Phase II clinical trial, treatment with Ibrance (palbociclib) resulted in an overall progression-free survival (PFS) of 57.2% at 12 weeks and median PFS of 17.9 weeks in patients with well-differentiated or dedifferentiated liposarcoma (PMID: 27124835). 27124835
Unknown unknown cutaneous T cell lymphoma not applicable Lacutamab Phase I Actionable In a Phase I trial, Lacutamab (IPH4102) demonstrated safety, and cutaneous T-cell lymphoma patients treated with IPH4102 demonstrated an overall response rate of 36% (16/44), a median progression-free survival of 8.2 months, and a median duration of response of 13.8 months (PMID: 31253572; NCT02593045). 31253572
Unknown unknown fibrosarcoma not applicable 23814 + Tivozanib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of 23814 and Tivozanib (AV-951) resulted in tumor growth inhibition in a fibrosarcoma cell line xenograft model, with increased efficacy over either agent alone (PMID: 25995436). 25995436
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 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 Advanced Solid Tumor not applicable Belinostat Phase I Actionable In a Phase I trial, Beleodaq (belinostat) demonstrated safety and promoted stable disease in 39% (18/46) of patients with a variety of solid tumors (PMID: 18245542). 18245542
Unknown unknown ovarian carcinoma not applicable BMS-906024 Preclinical Actionable In a preclinical study, the pan-Notch inhibitor, BMS-906024, inhibited growth of ovarian carcinoma xenografts (PMID: 26005526). 26005526
Unknown unknown Advanced Solid Tumor not applicable AZD8055 Phase I Actionable In a Phase I trial, AZD8055 treatment demonstrated safety and tolerability, and resulted in stable disease for more than 4 months in 14% (7/49) of patients with advanced solid tumors or lymphoma (PMID: 22935583). 22935583
Unknown unknown oral squamous cell carcinoma not applicable TAE226 Preclinical - Cell line xenograft Actionable In a preclinical study, TAE226 treatment increased apoptosis and decreased proliferation and migration of oral squamous cell carcinoma cells in culture and inhibited tumor growth in cell line xenograft models (PMID: 22766511). 22766511
Unknown unknown marginal zone B-cell lymphoma not applicable Tazemetostat Case Reports/Case Series Actionable In a Phase I trial, Tazemetostat (EPZ-6438) demonstrated safety and preliminary efficacy, resulted in a partial response in 1 patient with relapsed or refractory marginal zone B-cell lymphoma (PMID: 29650362; NCT01897571). 29650362
Unknown unknown colon cancer not applicable JSH-150 Preclinical - Cell culture Actionable In a preclinical study, JSH-150 inhibited proliferation of a colon cancer cell line in culture (PMID: 30253346). 30253346
Unknown unknown colorectal cancer not applicable Labetuzumab Govitecan Phase Ib/II Actionable In a Phase I/II trial, treatment with Labetuzumab govitecan demonstrated safety and resulted in stable disease in 49% (42/86), a clinical benefit rate of 29% (25/86; 1 partial response and stable disease for at least 6 months in 24), a median progression-free survival of 3.6 months, and a median overall survival of 6.9 months in patients with previously treated recurrent or refractory colorectal cancer (PMID: 28817371; NCT01605318). detail... 28817371
Unknown unknown 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 chordoma not applicable Dinaciclib Preclinical - Cell culture Actionable In a preclinical study, Dinaciclib (SCH 727965) treatment reduced viability of chordoma cells in culture (PMID: 30664779). 30664779
Unknown unknown melanoma not applicable A-1210477 + G-TPP Preclinical - Cell culture Actionable In a preclinical study, combination of the mitochondrial Hsp90 inhibitor G-TPP and the Mcl-1 inhibitor A-1210477 resulted in increased apoptosis of melanoma cell lines in culture (PMID: 28522750). 28522750
Unknown unknown gastric adenocarcinoma not applicable Triptolide Preclinical - Cell culture Actionable In a preclinical study, treatment with Triptolide (C1572) induced apoptotic cell death and resulted in decreased cell viability in gastric adenocarcinoma cell lines in culture (PMID: 28192510). 28192510
Unknown unknown lung non-small cell carcinoma not applicable Chiauranib Preclinical - Cell line xenograft Actionable In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a non-small cell lung cancer cell line xenograft model (PMID: 28004478). 28004478
Unknown unknown 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 breast cancer not applicable Axitinib Preclinical - Cell line xenograft Actionable In a preclinical study, Inlyta (axitinib) disrupted tumor microvasculature and inhibited tumor growth in breast cancer cell line xenograft models (PMID: 17371720). 17371720
Unknown unknown female reproductive organ cancer not applicable Cediranib + Durvalumab Phase I Actionable In a Phase I trial, the combination of Imfinzi (durvalumab) and Cediranib (AZD-2171) resulted in a partial response in 50% (6/12) of patients with female reproductive organ cancer (PMID: 28471727). 28471727
Unknown unknown prostate cancer not applicable Metformin Phase II Actionable In a Phase II trial, Glucophage (metformin) demonstrated safety and preliminary efficacy in patients with castration-resistant prostate cancer (PMID: 24412228). 24412228
Unknown unknown breast cancer not applicable Altiratinib Preclinical Actionable In a preclinical study, Altiratinib (DCC-0701) inhibited tumor growth and decreased lung metastasis in a mouse breast cancer model (PMID: 26285778). 26285778
Unknown unknown breast cancer not applicable MBQ-167 Preclinical - Cell line xenograft Actionable In a preclinical study, MBQ-167 increased cell-cycle arrest and decreased viability and migration of breast cancer cell lines in culture, and reduced tumor growth and metastasis in a breast cancer cell line xenograft model (PMID: 28450422). 28450422
Unknown unknown anal squamous cell carcinoma not applicable Prexasertib Phase Ib/II Actionable In a Phase Ib trial, treatment with Prexasertib (LY2606368) resulted in an overall response rate of 15% (4/26; 1 complete response (CR) and 3 partial responses (PR)), a clinical benefit rate (CR+PR+stable disease) of 58% (15/26), and a median progression-free survival of 2.8 months in patients with squamous cell carcinoma of the anus (PMID: 29643063; NCT0115790). 29643063
Unknown unknown anal squamous cell carcinoma not applicable Prexasertib Phase I Actionable In a Phase I trial, Prexasertib (LY2606368) treatment resulted in partial response in a patient with anal squamous cell carcinoma (PMID: 27044938). 27044938
Unknown unknown glioblastoma multiforme not applicable Cediranib + Lomustine Clinical Study Actionable In a clinical case study, a patient with recurrent glioblastoma treated with Cediranib (AZD-2171), in combination with Lomustine (CCNU), demonstrated 50% tumor regression at 6 weeks, complete response at 24 weeks, and achieved clinical remission for over 6 years (PMID: 26929887). 26929887
Unknown unknown 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 thyroid gland papillary 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 ovarian cancer not applicable 9-ING-41 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with 9-ING-41 induced apoptosis in ovarian cancer cells in culture and led to reduced tumor volume in cell line xenograft models (PMID: 21878813). 21878813
Unknown unknown Advanced Solid Tumor not applicable GDC-0425 + Gemcitabine Phase I Actionable In a Phase I trial, treatment with GDC-0425 followed by Gemzar (gemcitabine) resulted in a best overall response rate (includes stable disease and partial response) of 60% (24/40) in patients with advanced solid tumors (PMID: 27815358). 27815358
Unknown unknown triple-receptor negative breast cancer not applicable BETd-246 + BM-1197 Preclinical - Cell culture Actionable In a preclinical study, the Bet inhibitor BETd-246 and the Bcl-xl inhibitor BM-1197 synergistically induced apoptosis in triple-receptor negative breast cancer cell lines in culture (PMID: 28209615). 28209615
Unknown unknown lung non-small cell carcinoma not applicable Defactinib Phase I Actionable In a Phase I trial, 31% (17/55) of KRAS-mutant patients with non-small cell lung cancer demonstrated progression-free survival at 12 weeks and one patient experienced a partial response and eight patients had stable disease as a best overall response when treated with Defactinib (VS-6063) (PMID: 31739184; NCT01778803). 31739184
Unknown unknown medulloblastoma not applicable Prexasertib Preclinical - Cell culture Actionable In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including medulloblastoma cell lines (PMID: 28270495). 28270495
Unknown unknown Advanced Solid Tumor no benefit Oprozomib Phase I Actionable In a Phase I trial, Oprozomib (ONX 0912) demonstrated clinically relevant toxicity and minimal efficacy, with stable disease as best response in patients with advanced solid tumors (PMID: 26924128). 26924128
Unknown unknown pancreatic adenocarcinoma not applicable Mogamulizumab + Nivolumab Phase I Actionable In a Phase I trial, Poteligeo (mogamulizumab-kpkc) and Opdivo (nivolumab) combination treatment demonstrated acceptable safety, resulted in an objective response rate of 7% (1/15, 1 partial responses) and a disease control rate of 40% (6/15) in immunotherapy-naive patients with advanced or metastatic pancreatic adenocarcinoma, response occurred regardless of PD-L1, CCR4, CD8 expression levels and tumor mutational burden (PMID: 31455681; NCT02476123). 31455681
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 lung non-small cell carcinoma no benefit Bavituximab + Docetaxel Phase II Actionable In a Phase II trial, Tarvacin (bavituximab) and Taxotere (docetaxel) combination treatment resulted in favorable outcome compared to control in non-small cell lung cancer patients, with an overall response rate of 17.1% (7/41), median progression-free survival of 4.5 months (HR=0.74), and a median overall survival of 11.7 months (HR=0.66) (PMID: 27265742). 27265742
Unknown unknown triple-receptor negative breast cancer not applicable Carfilzomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of triple negative breast cancer cell lines in culture (PMID: 28753594). 28753594
Unknown unknown acute myeloid leukemia not applicable Decitabine + Glasdegib Phase Ib/II Actionable In a Phase Ib trial, the combination of Glasdegib (PF-04449913) and Dacogen (decitabine) resulted in an overall survival of 11.5 months in patients with either acute myeloid leukemia (AML) or myelodysplastic syndrome, with 40% (2/5) of AML patients experiencing a complete remission (PMID: 29463550). 29463550
Unknown unknown multiple myeloma not applicable INCB054329 + Itacitinib Preclinical - Cell line xenograft Actionable In a preclinical study, INCB054329 and Itacitinib (INCB039110) demonstrated synergy in a myeloma cell line in culture, resulting in decreased viability, increased apoptosis, and increased inhibition of STAT3 phosphorylation, and treatment with the combination of INCB054329 and Itacitinib (INCB039110) resulted in increased efficacy in myeloma cell line xenograft models over either agent alone (PMID: 30206163). 30206163
Unknown unknown acute myeloid leukemia not applicable Cytarabine + Onvansertib Phase I Actionable In a Phase I trial, Onvansertib (PCM-075) and low-dose Cytosar-U (cytarabine) combination therapy resulted in complete response in 33.3% (1/3) of evaluable patients with relapsed or refractory acute myeloid leukemia (Ann Oncol, 30 (Supplement 5): v435-v448, 2019; NCT03303339). detail...
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 lung cancer not applicable Capmatinib Case Reports/Case Series Actionable In a Phase I trial, Tabrecta (capmatinib) demonstrated safety and preliminary efficacy, resulted in stable disease as best overall response in 2 patients with lung cancer (PMID: 30724423). 30724423
Unknown unknown Advanced Solid Tumor not applicable A-1331852 + Docetaxel Preclinical - Cell line xenograft Actionable In a preclinical study, the addition of A-1331852 enhanced tumor growth inhibition by Taxotere (docetaxel) in several solid tumor cell line xenograft models, and the combination therapy demonstrated increased efficacy over either agent alone in 5/7 of the tested models (PMID: 25787766). 25787766
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 breast cancer not applicable Danusertib Preclinical - Cell culture Actionable In a preclinical study, Danusertib (PHA-739358) disrupted cell cycle progression and inhibited growth of breast cancer cell lines, with preferential inhibition of aromatase inhibitor-resistant cell lines (PMID: 25667100). 25667100
Unknown unknown colorectal adenocarcinoma not applicable Berzosertib + Irinotecan Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of VX-970 and Camptosar (irinotecan) synergized to inhibit proliferation of a colorectal adenocarcinoma cell line in culture and to inhibit tumor growth in a human colorectal adenocarcinoma cell line xenograft model (PMID: 25269479). 25269479
Unknown unknown Advanced Solid Tumor not applicable PF-06263507 Phase I Actionable In a Phase I trial, treatment with PF-06263507 resulted in no objective responses, but led to stable disease in two patients with advanced solid tumors (PMID: 28070718). 28070718
Unknown unknown 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 mantle cell lymphoma no benefit Tafasitamab Phase II Actionable In a Phase II trial, Tafasitamab (MOR208) treatment was well-tolerated and resulted in stable disease as the best response in 50% (6/12) of patients with mantle cell lymphoma (PMID: 29444231; NCT01685008). 29444231
Unknown unknown head and neck squamous cell carcinoma not applicable Carboplatin + Cetuximab + Fluorouracil FDA approved Actionable In a Phase III clinical trial that supported FDA approval, Erbitux (cetuximab) in combination with platinum-based chemotherapy (Platinol (cisplatin) or Paraplatin (carboplatin)) and Adrucil (fluorouracil) resulted in a median overall survival of 10.1 months, versus 7.4 months with chemotherapy alone, in patients with recurrent or metastatic head and neck squamous cell carcinoma (PMID: 23576486). 23576486 detail...
Unknown unknown 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 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 breast cancer not applicable Alisertib + Eribulin Preclinical - Pdx Actionable In a preclinical study, patient derived xenograft (PDX) models of breast cancer treated with a combination of Alisertib (MLN8237) and Halaven (eribulin) demonstrated a 70-80% decrease in tumor volume compared to only a 40% decrease in models treated with Halaven (eribulin) alone (PMID: 27235164). 27235164
Unknown unknown leiomyosarcoma not applicable BEZ235 Preclinical Actionable In a preclinical study, leiomyosarcoma cells treated with BEZ235 in culture and in mouse models demonstrated increased levels of apoptosis and a 42% reduction in tumor volume (PMID: 26952093). 26952093
Unknown unknown hematologic cancer no benefit OPB-51602 Phase I Actionable In a Phase I trial, OPB-51602 treatment resulted in no objective response and stable disease in 15% (3/20) of patients with hematological malignancies (PMID: 25912076). 25912076
Unknown unknown multiple myeloma not applicable Daratumumab and hyaluronidase-fihj FDA approved Actionable In a Phase III trial (COLUMBA) that supported FDA approval, subcutaneous administration of Darzalex Faspro (Daratumumab and hyaluronidase-fihj) demonstrated improved safety profile and resulted in an overall response rate comparable to that of intravenous Darzalex (daratumumab) (41%, 108/263, vs 37%, 96/259, relative risk 1.11) in patients with relapsed or refractory multiple myeloma who received 3 or more prior therapies (PMID: 32213342; NCT03277105). 32213342
Unknown unknown marginal zone B-cell lymphoma not applicable Ibrutinib FDA approved Actionable In a Phase II trial that supported FDA approval, Imbruvica (ibrutinib) treatment resulted in an objective response rate of 48% (29/60) in patients with previously treated marginal zone lymphoma, with a median progression-free survival of 14.2 months (PMID: 28167659; NCT01980628). 28167659 detail...
Unknown unknown colon cancer not applicable Navicixizumab Preclinical Actionable In a preclinical study, Navicixizumab (OMP-305B83) inhibited proliferation of endothelial cells in culture and demonstrated antitumor activity in vivo in multiple solid tumor types, including colon tumors (Mol Cancer Ther December 2015 14; C164). detail...
Unknown unknown neuroblastoma not applicable iC9.GD2.CAR.IL-15 T-cells Preclinical - Cell line xenograft Actionable In a preclinical study, iC9.GD2.CAR.IL-15 T-cell treatment inhibited tumor growth and improved tumor-free survival in a cell line xenograft model of neuroblastoma expressing the disialoganglioside GD2 (PMID: 30617136). 30617136
Unknown unknown colon cancer not applicable Bevacizumab + CVX-060 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of CVX-060 and Avastin (bevacizumab) resulted in increased tumor growth inhibition compared to either agent alone in a colon cancer cell line xenograft model (PMID: 21233403). 21233403
Unknown unknown 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 Advanced Solid Tumor not applicable SY-1365 Preclinical - Cell culture Actionable In a preclinical study, SY-1365 induced rapid apoptosis in a panel of solid tumor cell lines in culture, including breast, ovarian, colorectal, and lung cancer cells (Proceedings of the AACR, Vol 58, April 2017, Abstract # 1151). detail...
Unknown unknown 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 Advanced Solid Tumor not applicable A-1331852 Preclinical - Cell line xenograft Actionable In a preclinical study, A-1331852 inhibited tumor growth in several solid tumor cell line xenograft models (PMID: 25787766). 25787766
Unknown unknown Advanced Solid Tumor not applicable SR9009 Preclinical - Cell culture Actionable In a preclinicl study SR9009 demonstrated toxicity in a wide range of tumor cell lines harboring different driver mutations, but not in normal cell lines in culture (PMID: 29320480). 29320480
Unknown unknown Advanced Solid Tumor not applicable Buparlisib Phase I Actionable In a Phase I trial, Buparlisib (BKM120) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22162589). 22162589
Unknown unknown multiple myeloma not applicable Dexamethasone + Lenalidomide + Ricolinostat Phase I Actionable In a Phase Ib trial, Ricolinostat (ACY-1215) in combination with Revlimid (lenalidomide) and Desamethasone demonstrated safety and preliminary efficacy in relapsed or refractory multiple myeloma patients, resulted in an overall response rate of 55% (21/38) (PMID: 27646843). 27646843
Unknown unknown multiple myeloma not applicable Citarinostat + Pomalidomide 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 lymphoma not applicable Bendamustine + Veliparib Phase I Actionable In a Phase I trial, seven patients with lymphoma treated with a combination of Veliparib (ABT-888) and Bendamustine demonstrated an overall response rate of 71% (5/7) and complete response rate of 57% (4/7) and progression free survival of 6.9 months (PMID: 28314788; NCT01326702). 28314788
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 Waldenstroem's macroglobulinemia not applicable Ibrutinib FDA approved Actionable In a Phase II trial that supported FDA approval, Ibruvica (ibrutinib) treatment resulted in an overall response rate of 90.5% and a major response rate of 73.0% in patients with previously treated Waldenstroem's macroglobulinemia (PMID: 25853747; NCT01614821). 25853747 detail...
Unknown unknown uterus leiomyosarcoma not applicable Pazopanib Clinical Study Actionable In a retrospective clinical study, 33% (9/27) of patients with uterine leiomyosarcoma responded to Votrient (pazopanib) treatment (PMID: 29185261). 29185261
Unknown unknown Advanced Solid Tumor not applicable Elenagen Phase Ib/II Actionable In a Phase I/IIa trial, Elenagen treatment resulted in a best response of stable disease in 44% (12/27) of patients with advanced solid tumors (PMID: 28881846). 28881846
Unknown unknown gastrointestinal system cancer not applicable Axitinib + Fluorouracil + Leucovorin + Oxaliplatin Phase I Actionable In a Phase I trial, Inlyta (axitinib), in combination with FOLFOX, demonstrated safety and some efficacy in patients with gastrointestinal tumors (PMID: 24423921). 24423921
Unknown unknown hematologic cancer not applicable BP1001 Phase I Actionable In a Phase I/Ib trial, BP1001 treatment demonstrated safety and preliminary efficacy, resulting in clinical benefit and extended treatment cycle in 22% (7/32) of patients with refractory or relapsed hematological malignancies (PMID: 29550383; NCT01159028). 29550383
Unknown unknown 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 liposarcoma not applicable MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 blocked cell proliferation of a human liposarcoma cell line in culture and repressed tumor growth in xenograft models (PMID: 26675259). 26675259
Unknown unknown triple-receptor negative breast cancer not applicable Carboplatin + Docetaxel + Gemcitabine + Itraconazole Clinical Study Actionable In a retrospective analysis, triple-negative breast cancer patients treated with the combination of Itraconazole with Docefrez (docetaxel), Paraplatin (carboplatin), and Gemzar (gemcitabine) chemotherapy demonstrated an overall response rate of 62% (8/13), a median progression-free survival of 10.8 months, and a median overall survival of 20.4 months (PMID: 24982411). 24982411
Unknown unknown triple-receptor negative breast cancer not applicable CC-671 Preclinical - Pdx & cell culture Actionable In a preclinical study, CC-671 induced apoptosis and inhibited growth of triple-negative breast cancer (TNBC) cell lines in culture, and inhibited tumor growth in TNBC cell line and patient-derived xenograft (PDX) models (PMID: 29866747). 29866747
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 colorectal cancer not applicable Bevacizumab + trifluridine/tipiracil hydrochloride Phase Ib/II Actionable In a Phase I/II trial, Avastin (bevacizumab) and Lonsurf (trifluridine/tipiracil hydrochloride) combination treatment resulted in a 16-week progression-free survival rate of 42.9% (9/21) in patients with metastatic colorectal cancer refractory to standard therapies (PMID: 28760399). 28760399
Unknown unknown colorectal cancer not applicable Bevacizumab + trifluridine/tipiracil hydrochloride Phase II Actionable In a Phase II trial, Avastin (bevacizumab) and Lonsurf (trifluridine/tipiracil hydrochloride) combination treatment resulted in improved progression-free survival (4.6 vs 2.6 months, HR=0.45, p=0.0015) compared to Lonsurf (trifluridine/tipiracil hydrochloride) monotherapy in patients with metastatic colorectal cancer refractory to standard therapies (PMID: 31999946). 31999946
Unknown unknown osteosarcoma not applicable Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 5% (1/22) of patients with osteosarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). detail...
Unknown unknown breast cancer not applicable Doxorubicin + NU6027 Preclinical - Cell culture Actionable In a preclinical study, NU6027 enhanced the efficacy of Adriamycin (doxorubicin) in breast cancer cells in culture, resulting in decreased cell survival (PMID: 21730979). 21730979
Unknown unknown 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 renal cell carcinoma not applicable Axitinib + Pembrolizumab FDA approved Actionable In a Phase III trial (KEYNOTE-426) that supported FDA approval, the combination therapy of Keytruda (pembrolizumab) and Inlyta (axitinib) resulted in a greater 12-month overall survival rate (89.9% vs 78.3%), median progression-free survival (15.1mo vs 11.1mo), and objective response rate (59.3% vs 35.7%) compared to treatment with Sutent (sunitinib) in patients with untreated advanced renal cell carcinoma (PMID: 30779529; NCT02853331). detail... 30779529
Unknown unknown head and neck squamous cell carcinoma not applicable Cetuximab + Cisplatin + Patritumab Phase Ib/II Actionable In a Phase Ib clinical trial, combined Erbitux (cetuximab), Patritumab (U3-1287), and Platinol (cisplatin) treatment was tolerable in patients with recurrent/metastatic head and neck squamous cell carcinoma and resulted in a 47% (7/15) overall response rate (3 complete responses and 4 partial responses), stable disease in 53% (8/15) of patients, a 7.9-month median progression-free survival, and a 13.5-month overall survival (PMID: 30327312; NCT02350712). 30327312
Unknown unknown acute myeloid leukemia not applicable HM43239 Preclinical - Cell culture Actionable In a preclinical study, HM43239 treatment inhibited downstream signaling, reduced proliferation, and induced apoptosis in an acute myeloid leukemia cell line expressing leukemic stem cell marker in culture (Cancer Res 2019;79(13 Suppl):Abstract nr 1293). detail...
Unknown unknown lung non-small cell carcinoma not applicable Zenocutuzumab Phase Ib/II Actionable In a Phase I/II trial, Zenocutuzumab (MCLA-218) resulted in partial response for more than 10 months in a patient with non-small cell lung cancer (Cancer Res 2016;76(14 Suppl):Abstract nr CT050). detail...
Unknown unknown breast cancer not applicable AZ0108 Preclinical - Cell line xenograft Actionable In a preclinical study, AZ0108 induced mitotic defects, apoptosis, and inhibited growth in breast cancer cell lines in culture, and inhibited tumor growth in cell line xenograft models (PMID: 30297535). 30297535
Unknown unknown colorectal cancer not applicable Bevacizumab + Fluorouracil + Irinotecan + Oxaliplatin Clinical Study Actionable In a systematic review, a pooled analysis of 11 studies assessing the combination of Avastin (bevacizumab), Adrucil (fluorouracil), Eloxatin (oxaliplatin), and Camptosar (irinotecan) in colorectal cancer patients demonstrated an objective response rate of 69%, a median overall survival of 30.2 months based on six trials, and a progression free survival of 12.4 months based on nine trials (PMID: 28542671). 28542671
Unknown unknown acute lymphoblastic leukemia not applicable AS605240 Preclinical - Pdx & cell culture Actionable In a preclinical study, AS605240 increased apoptosis and decreased viability of T-acute lymphocytic leukemia (T-ALL) cell lines and primary T-ALL cells in culture, and decreased leukemic progression in a primary T-ALL cell xenograft model (PMID: 25869207). 25869207
Unknown unknown 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 colorectal cancer not applicable MSC2490484A + unspecified PD-L1 antibody Preclinical Actionable In a preclinical study, the combination of MSC2490484A and an unspecified PD-L1 antibody resulted in greater inhibition of tumor growth compared to the unspecified PD-L1 antibody alone in a colorectal cancer cell line mouse model (PMID: 32238472). 32238472
Unknown unknown pancreatic cancer no benefit Gemcitabine + Vandetanib Phase II Actionable In a Phase II trial, addition of Caprelsa (vandetanib) to Gemzar (gemcitabine) did not improve median overall survival (8.83 vs 8.95 months) compared to Gemzar (gemcitabine) monotherapy in patients with advanced pancreatic cancer (PMID: 28259610). 28259610
Unknown unknown lung non-small cell carcinoma not applicable BAY1161909 + Paclitaxel Preclinical Actionable In a preclinical study, the combination of BAY1161909 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 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 renal cell carcinoma not applicable CPI-444 Phase I Actionable In a Phase I trial, treatment with single agent CPI-444 was well-tolerated and resulted in a disease control rate of 75% (3/4) in patients with renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 3004)). detail...
Unknown unknown pancreatic adenocarcinoma not applicable GSK1904529A Preclinical - Cell line xenograft Actionable In a preclinical study, GSK1904529A treatment resulted in inhibition of tumor growth by 52% in xenograft models of pancreas adenocarcinoma (PMID: 19383820). 19383820
Unknown unknown triple-receptor negative breast cancer not applicable Carfilzomib Preclinical - Cell culture Actionable In a preclinical study, Kyprolis (carfilzomib) induced complete growth inhibition of triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown esophageal carcinoma not applicable BI 853520 Phase I Actionable In a Phase I trial, BI 853520 demonstrated safety and some anti-tumor efficacy, resulting in stable disease in 12.5% (2/16) of patients with esophageal carcinoma (PMID: 30756308; NCT01335269). 30756308
Unknown unknown Advanced Solid Tumor not applicable CI-1040 Phase I Actionable In a Phase I trial, CI-1040 treatment resulted in median stable disease for 5.5 months in 28% (19/66) of patients with advanced solid tumors (including colorectal, non-small-cell lung, pancreatic, melanoma, and kidney) and partial response in 1 pancreatic cancer patient (PMID: 16009947). 16009947
Unknown unknown lung non-small cell carcinoma not applicable Sabatolimab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 treatment resulted in stable disease in 2 patients with non-small cell lung cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown 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 prostate cancer not applicable TAK-960 Preclinical - Cell line xenograft Actionable In a preclinical study, prostate cancer cells treated with TAK-960 demonstrated a decrease in tumor size in cell line xenograft models (PMID: 22188812). 22188812
Unknown unknown thyroid gland cancer not applicable Cetuximab Preclinical Actionable In a preclinical study, Erbitux (cetuximab) inhibited tumor growth and angiogenesis in mouse models of anaplastic thyroid cancer (PMID: 17429874). 17429874
Unknown unknown malignant peripheral nerve sheath tumor not applicable Everolimus Preclinical - Cell line xenograft Actionable In a preclinical study, malignant peripheral nerve sheath cell line xenograft models treated with Afinitor (everolimus) demonstrated a 76% decrease in tumor growth (PMID: 18483311). 18483311
Unknown unknown Advanced Solid Tumor not applicable BI-847325 Phase I Actionable In a Phase I trial, BI-847325 treatment resulted in stable disease in 30% (21/69) of patients with advanced solid tumors, and one partial response for 67 days in a patient with esophageal cancer (PMID: 26650227). 26650227
Unknown unknown Advanced Solid Tumor not applicable BI-847325 Phase I Actionable In a Phase I trial, BI-847325 demonstrated safety and some efficacy in a variety of advanced solid tumors (Mol Cancer Ther 2013;12(11 Suppl):B281). detail...
Unknown unknown pancreatic cancer not applicable Golvatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Golvatinib (E7050) inhibited tumor angiogenesis and tumor growth in xenografts of a VEGF-overexpressing human pancreatic cancer cell line (PMID: 19832844). 19832844
Unknown unknown urinary bladder cancer not applicable AZD4547 + Cisplatin + Gemcitabine Phase I Actionable In a Phase I trial, AZD4547 in combination with Platinol (cisplatin) and Gemzar (gemcitabine) demonstrated safety and preliminary efficacy in bladder cancer patients (J Clin Oncol 34, 2016 (suppl; abstr 4521)). detail...
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 renal cell carcinoma not applicable Axitinib + X4P-001 Phase I Actionable In a Phase I trial, X4P-001 in combination with Axitinib, displayed safety and efficacy in patients with renal cell carcinoma (AACR; Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B201, NCT02667886). detail...
Unknown unknown lung carcinoma not applicable AGI-134 Preclinical - Cell culture Actionable In a preclinical study, AGI-134 treatment induced cell death of lung carcinoma cells in the presence of normal human serum in culture and induced phagocytosis when co-cultured with human macrophages (PMID: 31889898). 31889898
Unknown unknown glioblastoma multiforme not applicable Durvalumab Phase II Actionable In a Phase II trial, Imfinzi (durvalumab) treatment resulted in partial response in 16.7% (5/30), stable disease in 43.3% (13/30), and an estimated 6 month progression free survival of 20.0% in patients with Bevacizumab-naive, recurrent glioblastoma (Neuro Oncol (2016) 18 (suppl 6): vi18.). detail...
Unknown unknown ovarian clear cell carcinoma no benefit ENMD-2076 Phase II Actionable In a Phase II trial, ENMD-2076 did not meet efficacy standard, resulted in partial response in 7.9% (3/38) and stable disease in 68.4% (26/38) of patients with recurrent ovarian clear cell carcinoma, with an overall 6-month progression-free survival rate of 22% (PMID: 30108107). 30108107
Unknown unknown CLL/SLL not applicable Ibrutinib + Rituximab FDA approved Actionable In a Phase III trial that supported FDA approval, combination of Imbruvica (ibrutinib) and Rituxan (rituximab) resulted in superior progression-free survival at 3 years (89.4% vs 72.9%, HR=0.35, p<0.001) and overall survival at 3 years (98.8% vs 91.5%, HR=0.17, p<0.001) compared to chemoimmunotherapy in patients with previously untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) (PMID: 31365801; NCT02048813). detail... 31365801
Unknown unknown Advanced Solid Tumor not applicable RRx-001 Phase I Actionable In a Phase I trial, RRx-001 demonstrated safety and preliminary efficacy, resulted in partial response in 5% (1/21) and stable disease in 67% (14/21) of patients with advanced solid tumors (PMID: 26296952; NCT01359982). 26296952
Unknown unknown diffuse large B-cell lymphoma not applicable Tomivosertib Preclinical Actionable In a preclinical study, eFT508 inhibited proliferation of diffuse large B-cell lymphoma cell lines in culture (Blood Dec 2015, 126 (23) 1554). detail...
Unknown unknown colorectal cancer not applicable VLX600 Preclinical - Patient cell culture Actionable In a preclinical study, VLX600 treatment inhibited proliferation of cells derived from colorectal cancer patients in culture (PMID: 24548894). 24548894
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 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 prostate cancer no benefit Abiraterone + Buparlisib Phase I Actionable In a Phase Ib trial, the combination of Zytiga (abiraterone) and Buparlisib (BKM120) did not demonstrate significant clinical activity in patients with castration-resistant prostate cancer, resulting in a best overall response of stable disease in 15% (3/20) patients treated at the 100 mg QD Buparlisib (BKM120) dose level, and further study of this combination is not planned (PMID: 28282611; NCT01634061). 28282611
Unknown unknown prostate cancer not applicable AS605240 Preclinical Actionable In a preclinical study, AS605240 reduced invasiveness of prostate cancer cells in culture (PMID: 24416348). 24416348
Unknown unknown acute myeloid leukemia not applicable Bortezomib + Tinostamustine Preclinical - Patient cell culture Actionable In a preclinical study, the combination of EDO-S101 and Velcade (bortezomib) decreased viability of primary acute myeloid leukemia cells in culture (PMID: 28753594). 28753594
Unknown unknown ovarian cancer not applicable Cisplatin + LB-100 Preclinical Actionable In a preclinical study, LB-100 sensitized several ovarian cancer cell lines to Platinol (cisplatin) in culture (PMID: 25376608). 25376608
Unknown unknown chronic lymphocytic leukemia not applicable Idelalisib + Tirabrutinib Phase I Actionable In a Phase Ib trial, Tirabrutinib (ONO-4059) and Zydelig (idelalisib) combination treatment was well tolerated, resulted in a overall response rate of 93% (13/14) with complete response in 7% (1/14) of patients with chronic lymphocytic leukemia (PMID: 32156743; NCT02457598). 32156743
Unknown unknown multiple myeloma not applicable NSC126405 Preclinical - Cell line xenograft Actionable In a preclinical study, NSC126405 inhibited tumor growth during 11 days of treatment in multiple myeloma cell line xenograft models and 22% (2/9) of the mice demonstrated tumor regression post treatment (PMID: 27530328). 27530328
Unknown unknown endometrial carcinoma not applicable XL147 Phase II Actionable In a Phase II trial, Pilaralisib (XL147) treatment resulted in an objective response rate of 6% (4/67) in patients with endometrial carcinoma, although anti-tumor activity is not associated with molecular alterations in PTEN and PIK3R1 (PMID: 25528496). 25528496
Unknown unknown Advanced Solid Tumor not applicable 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 cervical cancer not applicable Cisplatin + ETP-46464 Preclinical - Cell culture Actionable In a preclinical study, ETP-46464 increased the sensitivity of cervical cancer cell lines to Platinol (cisplatin) in culture (PMID: 25560806). 25560806
Unknown unknown 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 Advanced Solid Tumor not applicable PD-0325901 Phase I Actionable In a Phase I clinical trial, PD-0325901 demonstrated preliminary clinical activity in patients with advanced solid tumors, however late-onset dose-limiting toxicities were encountered (PMID: 20215549). 20215549
Unknown unknown spindle cell carcinoma not applicable LYC-55716 Case Reports/Case Series Actionable In a Phase I trial, Cintirorgon (LYC-55716) treatment resulted in a partial response in a patient with metastatic spindle cell carcinoma, demonstrating decreased tumor burden (PMID: 30819679). 30819679
Unknown unknown mantle cell lymphoma not applicable Tirabrutinib Phase I Actionable In a Phase I clinical trial, treatment with Tirabrutinib (ONO-4059) was well tolerated and resulted in responses in 92% (11/12) of patients with mantle cell lymphoma (PMID: 26542378). 26542378
Unknown unknown head and neck squamous cell carcinoma not applicable Cetuximab + Prexasertib + Radiotherapy Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Prexasertib (LY2606368), Erlotinib (cetuximab), and radiotherapy resulted in greater decreased cell proliferation, inhibition of cell growth, and apoptotic activity in culture compared to either agent alone, and suppressed tumor growth in cell line xenograft models (PMID: 28138028). 28138028
Unknown unknown Ewing sarcoma not applicable SP-2509 Preclinical - Cell culture Actionable In a preclinical study, EWS fusion-positive Ewing sarcoma cell lines demonstrated reduced viability following SP-2509 treatment that correlated with induction of KDM1B expression post-treatment in culture (PMID: 29997151). 29997151
Unknown unknown colon cancer not applicable THOR-707 Preclinical Actionable In a preclinical study, treatment with THOR-707 resulted in an increase in increased CD8-positive T cell tumor infiltration and demonstrated anti-tumor activity in a syngeneic mouse model of colon cancer (Cancer Res 2019;79(13 Suppl):Abstract nr 3258). detail...
Unknown unknown B-cell lymphoma not applicable Loncastuximab tesirine Phase I Actionable In a Phase I trial, Loncastuximab tesirine treatment demonstrated safety and preliminary efficacy, resulted in an overall response rate of 59.4% (41/69, 28 complete response, 13 partial response) in patients with relapsed/refractory B-cell non-Hodgkin lymphoma, with median duration of response, progression-free survival, and overall survival of 4.8, 5.5, and 11.6 months, respectively (PMID: 31685491; NCT02669017). 31685491
Unknown unknown lung carcinoma not applicable INCB001158 Preclinical Actionable In a preclinical study, INCB001158 (CB-1158) had no effect on Lewis Lung carcinoma cells growth in culture, but inhibited tumor growth in syngeneic animal models through regulation of host immune response (Cancer Res 2016;76(14 Suppl):Abstract nr 552). detail...
Unknown unknown acute myeloid leukemia not applicable ORY-1001 Preclinical - Cell line xenograft Actionable In a preclinical study, ORY-1001 decreased colony forming ability in several acute myeloid leukemia (AML) cell lines and primary AML samples in culture, and reduced tumor growth in AML cell line xenograft model (PMID: 29502954). 29502954
Unknown unknown lung non-small cell carcinoma not applicable Axitinib + Radiotherapy Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Inlyta (axitinib) and radiotherapy resulted in improved efficacy compared to either agent alone, and decreased pneumonitis in non-small cell lung cancer cell line xenograft models (PMID: 24862536). 24862536
Unknown unknown 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 Advanced Solid Tumor not applicable JNJ-63723283 Phase Ib/II Actionable In a Phase I/II trial, JNJ-63723283 demonstrated safety and preliminary efficacy, resulted in partial response in 9.4% (3/32) and stable disease in 56.3% (18/32) of patients with advanced solid tumors (Journal of Clinical Oncology 36, no. 5_suppl (February 10 2018) 58-58). detail...
Unknown unknown Advanced Solid Tumor not applicable GC1118 Phase I Actionable In a Phase I clinical trial, treatment with GC1118 was well-tolerated, and resulted in partial response in 13% (3/24) and stable disease in 50% (12/24) of patients with advanced solid tumors (PMID: 31164456; NCT02352571). 31164456
Unknown unknown Advanced Solid Tumor not applicable Dostarlimab Phase I Actionable In a Phase I trial, Dostarlimab (TSR-042) demonstrated safety and preliminary efficacy, resulted in partial response in 9.5% (2/21) and stable disease in 23.8% (5.21) of patients with advanced solid tumors (Annals of Oncology (2017) 28 (suppl_5): v403-v427. Abstract # 1185P; NCT02715284). detail...
Unknown unknown chronic lymphocytic leukemia not applicable Bendamustine + Obinutuzumab + Venetoclax Phase II Actionable In a Phase II trial (CLL2-BAG), sequential treatment with Treanda (bendamustine) and Gazyva (obinutuzumab) combined with Venclexta (venetoclax) resulted in an response rate of 95% (60/63) in patients with chronic lymphocytic leukemia, without unexpected or cumulative toxicities (PMID: 30115596; NCT02401503). 30115596
Unknown unknown ovarian cancer not applicable Sabatolimab + Spartalizumab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in stable disease in 3 patients with ovarian cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown breast cancer not applicable Metformin Preclinical - Cell line xenograft Actionable In a preclinical study, Glucophage (metformin) inhibited the growth of a breast cancer cell line in culture and resulted in decreased tumor volume in a cell line xenograft model (PMID: 26351208). 26351208
Unknown unknown 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 multiple myeloma not applicable Bortezomib + Dexamethasone + Ricolinostat Phase Ib/II Actionable In a Phase Ib/II trial, the Phase II recommended dose of Ricolinostat (ACY-1215) combined with Velcade (bortezomib) and Adexone (dexamethasone) resulted in safety and tolerance, and an overall response rate of 37% in patients with multiple myeloma (PMID: 28053023). 28053023
Unknown unknown triple-receptor negative breast cancer not applicable Irinotecan + SR-4835 Preclinical - Pdx Actionable In a preclinical study, SR-4835 and Camptosar (irinotecan) combination treatment induced DNA damage and cell death, inhibited tumor growth, leading to tumor regression in a patient-derived xenograft (PDX) model of triple-negative breast cancer harboring a BRCA1 mutation (PMID: 31668947). 31668947
Unknown unknown ovarian carcinoma not applicable Rucaparib Phase II Actionable In a Phase II trial, Rubraca (rucaparib) demonstrated activity in patients with platinum-sensitive high-grade ovarian carcinoma, with patients in the BRCA mutant and BRCA wild-type with high genomic loss-of-heterozygosity subgroups demonstrating increased progression-free survival compared to the BRCA wild-type with low genomic loss-of-heterozygosity subgroup (PMID: 27908594). 27908594
Unknown unknown lung adenocarcinoma not applicable ORCA-010 Preclinical - Cell line xenograft Actionable In a preclinical study, ORCA-010 treatment induced cytotoxicity in a lung adenocarcinoma cell line in culture, and inhibited tumor growth and increased survival in a cell line xenograft model (PMID: 25093639). 25093639
Unknown unknown astrocytoma not applicable Olaparib Preclinical - Cell culture Actionable In a preclinical study, Lynparza (olaparib) resulted in decreased cell viability of some pediatric high grade astrocytoma cell lines in culture (PMID: 26351319). 26351319
Unknown unknown pancreatic cancer not applicable Conatumumab + Ganitumab Phase Ib/II Actionable In a Phase Ib/II clinical trial, Ganitumab and Conatumumab combination treatment resulted in stable disease in one patient out of sixteen with pancreatic cancer (PMID: 24816908). 24816908
Unknown unknown chronic lymphocytic leukemia not applicable Cerdulatinib Preclinical - Patient cell culture Actionable In a preclinical study, Cerdulatinib (PRT062070) decreased BCR downstream signaling and chemokine secretion, and reduced viability of patient-derived chronic lymphocytic leukemia (CLL) cells in culture (PMID: 27697994). 27697994
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 colorectal cancer not applicable TP-1454 + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, TP-1454 combined with an anti-PD1 antibody reduced tumor growth in a syngeneic mouse model of colorectal cancer, and synergistically inhibited tumor growth in another syngeneic mouse model (Mol Cancer Ther 2019;18(12 Suppl):Abstract nr B080). detail...
Unknown unknown multiple myeloma not applicable Dexamethasone + Thalidomide FDA approved Actionable In a Phase III trial that supported FDA approval, Thalomid (thalidomide) in combination with dexamethasone (n=103) resulted in significantly improved response rate (63% vs 41%, p=0.0017) compared to dexamethasone alone (n=104) in patients with newly diagnosed multiple myeloma (PMID: 16365178). 16365178 detail...
Unknown unknown Advanced Solid Tumor not applicable Talazoparib Preclinical - Cell line xenograft Actionable In a preclinical study, the Talazoparib (BMN-673) selective PARP1/2 inhibitor demonstrated antitumor activity on a variety of cell lines and xenografts with defects in DNA repair (PMID: 23881923). 23881923
Unknown unknown sarcoma not applicable Ganitumab Phase II Actionable In a Phase II trial, Ganitumab treatment resulted in a 6% (2/35) partial response and stable disease in 49% (17/35) of patients with metastatic Ewing's family of tumors or desmoplastic small round cell tumor (PMID: 22508822). 22508822
Unknown unknown Advanced Solid Tumor not applicable Carboplatin + MEDI3617 + Paclitaxel Phase I Actionable In a Phase I trial, MEDI3617 in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) resulted in no objective response (0/7) and stable disease in 43% (3/7) of patients with advanced solid tumors (PMID: 29559563; NCT01248949). 29559563
Unknown unknown Advanced Solid Tumor not applicable Fluorouracil + Sorafenib Phase I Actionable In a Phase I trial, Nexavar (sorafenib) in combination with Adrucil (fluorouracil) displayed safety and efficacy in advanced solid tumors, including colon cancer (PMID: 22232731). 22232731
Unknown unknown colorectal cancer not applicable CCT241533 + Rucaparib Preclinical - Cell culture Actionable In a preclinical study, CCT241533 enhanced the growth inhibition effect of Rubraca (rucaparib) in colorectal cancer cells in culture (PMID: 21239475). 21239475
Unknown unknown lung non-small cell 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 mantle cell lymphoma not applicable Lenalidomide FDA approved Actionable In a Phase II trial (EMERGE) that supported FDA approval, Revlimid (lenalidomide) treatment resulted in an objective response rate of 28% (37/134) with rapid time to response (2.2 months), a median duration of response of 16.6 months, a median progression-free survival of 4.0 months, and a median overall survival of 19.0 months in patients with mantle cell lymphoma who relapsed or progressed after or were refractory to Velcade (bortezomib) (PMID: 24002500; NCT00737529). 24002500 detail...
Unknown unknown lung non-small cell carcinoma not applicable Pembrolizumab Clinical Study - Meta-analysis Actionable In a meta-analysis, compared to Taxotere (docetaxel), treatment with immune checkpoint inhibitors including Opdivo (nivolumab), Keytruda (pembrolizumab), or Tecentriq (atezolizumab), resulted in prolonged overall survival (HR=0.69, p<0.001) in non-small cell lung carcinoma patients (PMID: 29270615). 29270615
Unknown unknown 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 hepatocellular carcinoma no benefit Erlotinib + Sorafenib Phase III Actionable In a Phase III trial, the combination treatment of Nexavar (sorafenib) with Tarceva (erlotinib) compared to Nexavar (sorafenib) plus placebo did not demonstrate an improved overall survival and time to progression in patients with hepatocellular carcinoma (PMID: 25547503). 25547503
Unknown unknown 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 thyroid gland cancer not applicable Everolimus Phase II Actionable In a Phase II trial, treatment with Afinitor (everolimus) resulted in a median progression-free survival (PFS) of 12.9 months, 2-year PFS of 23.6%, and 2-year overall survival of 73.5% in patients with differentiated thyroid cancer, and a partial response in a patient with anaplastic thyroid cancer (ATC), and disease stability for 26 months in another ATC patient (PMID: 29301825). 29301825
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 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 Advanced Solid Tumor not applicable SU14813 Phase I Actionable In a Phase I trial, SU14813 demonstrated safety and preliminary efficacy in patients with advanced solid tumors, with an objective response rate of 20% (13/65), including 1 complete response and 12 partial responses (PMID: 20605934). 20605934
Unknown unknown ovarian carcinoma no benefit Denileukin diftitox + Sirolimus Preclinical Actionable In a preclinical study, an ovarian carcinoma mouse model did not respond to the combination of Ontak (denileukin diftitox) and Rapamune (sirolimus), demonstrating no decrease in tumor burden (PMID: 27737881). 27737881
Unknown unknown islet cell tumor not applicable GDC-0326 Preclinical Actionable In a preclinical study, GDC-0326 inhibited AKT activation, decreased tumor growth, metastasis, and angiogenesis and increased lifespan in a transgenic mouse model of pancreatic neuroendocrine tumor (PMID: 27225693). 27225693
Unknown unknown multiple myeloma not applicable Bortezomib + DT204 Preclinical - Cell line xenograft Actionable In a preclinical study, a Velcade (bortezomib) resistant multiple myeloma cell line overcame resistance when treatment was combined with DT204, and in xenograft models the combined treatment resulted in delayed tumor growth and improved survival (PMID: 27677741). 27677741
Unknown unknown head and neck cancer not applicable AZD7648 + Olaparib Preclinical - Pdx Actionable In a preclinical study, AZD7648 and Lynparza (olaparib) combination treatment inhibited tumor growth in a patient-derived xenograft (PDX) model of head and neck cancer harboring mutant TP53 and wild-type ATM (PMID: 31699977). 31699977
Unknown unknown acute lymphoblastic leukemia not applicable TAS4464 Preclinical - Cell line xenograft Actionable In a preclinical study, TAS4464 resulted in complete tumor regression in cell line xenograft models of acute lymphocytic leukemia (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr C176). detail...
Unknown unknown lung non-small cell carcinoma not applicable Pemetrexed Disodium + Trametinib Phase I Actionable In a Phase I/Ib trial, treatment with the combination of Mekinist (trametinib) and Alimta (pemetrexed) resulted in an overall response rate of 14% (6/42, all partial responses) and stable disease in 55% (23/42) of patients with non-small cell lung cancer (PMID: 27876675). 27876675
Unknown unknown ovarian carcinoma not applicable BI 853520 Phase I Actionable In a Phase I trial, BI 853520 demonstrated safety and some anti-tumor efficacy, resulting in stable disease in 31% (5/16) of patients with platinum-resistant ovarian carcinoma, one of whom had stable disease lasting greater than 150 days (PMID: 30756308; NCT01335269). 30756308
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 lung carcinoma not applicable MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human lung carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown thyroid gland medullary carcinoma not applicable Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) treatment resulted in partial response in 6.3% (1/16) and stable disease in 87.5% (14/16) of patients with sporadic medullary thyroid carcinoma, with a median progression free survival of 17.9 months (PMID: 20368568). 20368568
Unknown unknown esophagus squamous cell carcinoma not applicable Imetelstat + Radiotherapy Preclinical - Cell line xenograft Actionable In a preclinical study, imetelstat increased sensitivity of human esophageal squamous cell carcinoma cell lines to radiotherapy, resulting in increased apoptosis and decreased survival in culture, and increased apoptosis, decreased proliferation, and reduced tumor growth in mouse models (PMID: 28099140). 28099140
Unknown unknown Advanced Solid Tumor not applicable Paclitaxel + Pazopanib 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 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, abs