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Gene Unknown
Variant unknown
Impact List
Protein Effect
Gene Variant Descriptions Unknown variant is used in combination with unknown gene (Unknown, Unknown) to connect evidence to therapies and tumor types when a specific gene variant has not been identified in the cited literature.
Associated Drug Resistance

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No Variant Reference Transcript is Available.
No transcript is Available.

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown head and neck squamous cell carcinoma not applicable Buparlisib Preclinical Actionable In a preclinical study, Buparlisib (BKM-120) reduced viability of head and neck squamous cell carcinoma (HNSSC) cells in culture and demonstrated anti-tumor activity in HNSSC xenograft models (PMID: 22116303). 22116303
Unknown unknown ovarian cancer not applicable Cisplatin + TRX-E-002-1 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of TRX-E-002-1 and Platinol (cisplatin) synergized to induce cell death in human chemoresistant ovarian cancer cell lines in culture and decreased tumor burden in xenograft models (PMID: 27196760). 27196760
Unknown unknown multiple myeloma not applicable Bortezomib + Dexamethasone + Venetoclax Phase III Actionable In a Phase III trial (BELLINI), addition of Venclexta (venetoclax) to Velcade (bortezomib) and dexamethasone significantly improved progression-free survival (23.2 vs 11.4 mo, HR=0.60) in patients with relapsed or refractory multiple myeloma, but also increased mortality rate (33%, 64/194 vs 25%, 24/97) and did not improve overall survival (33.5 vs not reached, HR=1.46) (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 8509-8509; NCT02755597). detail...
Unknown unknown gastroesophageal junction adenocarcinoma not applicable Zenocutuzumab Phase Ib/II Actionable In a Phase I/II trial, Zenocutuzumab (MCLA-128) resulted in stable disease for 5 months in a patient with gastroesophageal junction cancer (Cancer Res 2016;76(14 Suppl):Abstract nr CT050). detail...
Unknown unknown hepatocellular carcinoma not applicable Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) improved median progression free survival to 10.7 months in patients with unresectable hepatocellular carcinoma (PMID: 19144678). 19144678 detail...
Unknown unknown ovarian cancer not applicable ABT-737 + AZD8055 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of AZD8055 and Mekinist (trametinib) enhanced the sensitivity of ovarian cancer cells to ABT-737 in culture, resulting in greater apoptotic activity and cell cycle arrest when compared to Mekinist (trametinib) alone (PMID: 27980105). 27980105
Unknown unknown lung non-squamous non-small cell carcinoma not applicable Carboplatin + Demcizumab + Pemetrexed Disodium Phase Ib/II Actionable In a Phase Ib trial, Demcizumab (OMP-21M18) in combination with Paraplatin (carboplatin) and Alimta (pemetrexed) demonstrated safety and preliminary efficacy, resulted in objective tumor response in 50% (20/40) of treatment-naive patients with metastatic non-squamous non-small cell lung cancer (PMID: 29188408; NCT01189968). 29188408
Unknown unknown endometrial cancer not applicable Lenvatinib + Pembrolizumab Phase Ib/II Actionable Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 52% (12/23) in patients with metastatic endometrial cancer, with a median duration of response not evaluable, and a median progression-free survival of 9.7 months (PMID: 31961766; NCT02501096). 31961766
Unknown unknown breast cancer not applicable PF-00562271 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with PF-00562271 inhibited PTK2 (FAK) phosphorylation and resulted in apoptosis and tumor regression in breast cancer cell line xenograft models (PMID: 18339875). 18339875
Unknown unknown lung non-squamous non-small cell carcinoma no benefit Bavituximab + Docetaxel Phase II Actionable In a Phase II trial, Tarvacin (bavituximab) and Taxotere (docetaxel) combination treatment did not improve overall survival (10.5 vs 10.9 months, HR=1.06, p=0.533) or progression-free survival (HR=1.00, p=0.990) compared to Taxotere (docetaxel) and placebo in patients with previously treated advanced non-squamous non-small-cell lung cancer (PMID: 29767677; NCT01999673). 29767677
Unknown unknown alveolar soft part sarcoma not applicable Crizotinib Phase II Actionable In a Phase II trial, Xalkori (crizotinib) treatment resulted in partial response (PR) in 2.5% (1/40) and stable disease (SD) in 87.5% (35/40) of patients with TFE3-rearranged, MET-positive alveolar soft part sarcoma, with a 1-year progression-free survival (PFS) rate of 37.5%, and resulted in PR in 25% (1/4) and SD in 75% (3/4) of patients without TFE3 rearrangement and MET expression, with a 1-year PFS rate of 50% (PMID: 29216400; NCT01524926). 29216400
Unknown unknown Advanced Solid Tumor not applicable GSK126 Phase I Actionable In a Phase I trial, GSK126 was tolerated and demonstrated preliminary efficacy, resulted in partial response in 2% (1/41) and stable disease in 34% (14/41) of patients with advanced solid tumor (n=21) or B-cell lymphoma (n=20) (PMID: 31471312; NCT02082977). 31471312
Unknown unknown 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 colon cancer not applicable BO-112 Preclinical - Cell culture Actionable In a preclinical study, BO-112 treatment inhibited viability of mouse colon carcinoma cells, and reduced cell viability, and induced cytotoxicity and apoptosis in human colon cancer cell lines in culture, and intratumoral delivery of BO-112 reduced tumor growth in a syngeneic mouse model of colon carcinoma (PMID: 31046839). 31046839
Unknown unknown acute leukemia not applicable CNDO-109 Phase I Actionable In a Phase I trial, acute leukemia patients treated with CNDO-109 demonstrated a relapse-free survival (RFS) rate of 33% at 12 months, with three patients demonstrating a long-term RFS of at least 32.6 months in a follow-up (PMID: 29597002; NCT01520558). 29597002
Unknown unknown chronic myeloid leukemia not applicable ST7612AA1 Preclinical Actionable In a preclinical study, ST7612AA1 inhibited proliferation of chronic myeloid leukemia cells in culture (PMID: 25671299). 25671299
Unknown unknown prostate cancer not applicable PF-00562271 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with PF-00562271 inhibited PTK2 (FAK) phosphorylation and resulted in apoptosis and tumor regression in prostate cancer cell line xenograft models (PMID: 18339875). 18339875
Unknown unknown Advanced Solid Tumor not applicable PQR309 Phase I Actionable In a Phase I trial, PQR309 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2560)). detail...
Unknown unknown neuroblastoma not applicable Alisertib + Irinotecan + Temozolomide Phase I Actionable In a Phase I trial, the combination of Alisertib (MLN8237), Camptosar (irinotecan), and Temodar (temozolomide) demonstrated safety and preliminary efficacy in neuroblastoma patients, resulting in an overall response rate of 31.8% and a 2-year progression-free survival rate of 52.4% (PMID: 26884555). 26884555
Unknown unknown neuroblastoma not applicable Alisertib + Irinotecan + Temozolomide Phase II Actionable In a Phase II trial, the combination of Alisertib (MLN8237), Camptosar (irinotecan), and Temodar (temozolomide) resulted in a response rate of 21.1% (4/19) in patients with neuroblastoma, with 4 partial responses, 2 minor responses, and 5 stable diseases (PMID: 30093449; NCT01601535). 30093449
Unknown unknown 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 clear cell renal cell carcinoma not applicable PT2385 Phase I Actionable In a Phase I trial, treatment with PT2385 in patients with renal clear cell carcinoma resulted in a disease control rate of 66% (33/50), including one patient with a complete response, six patients achieving a partial response, and twenty-six patients with stable disease, and led to a progression-free survival of more than 14 months in 25% of patients (PMID: 29257710). 29257710 detail...
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 triple-receptor negative breast cancer not applicable SMI#9-GNP Preclinical - Cell line xenograft Actionable In a preclinical study, SMI#9-GNP treatment inhibited proliferation, and colony formation, and induced apoptosis in triple-negative breast cancer cell lines harboring either wild-type or mutant BRCA1 in culture, and inhibited tumor growth in cell line xenograft models (PMID: 30242094). 30242094
Unknown unknown Advanced Solid Tumor not applicable GDC-0575 + Gemcitabine Phase I Actionable In a Phase I trial, the combination of GDC-0575 and Gemzar (gemcitabine) demonstrated safety and preliminary activity in patients with advanced solid tumors, resulting in a best overall response of stable disease or partial response in 66% (59/90) of patients, with partial responses in 4% (4/90) of patients, including patients with TP53 mutations (PMID: 29788155; NCT01564251). 29788155 detail...
Unknown unknown B-cell lymphoma not applicable Tazemetostat Phase I Actionable In a Phase I trial, Tazemetostat (EPZ-6438) demonstrated safety and preliminary efficacy, resulted in an objective response in 38% (8/21, 3 complete response, 5 partial response) in patients with relapsed or refractory B-cell non-Hodgkin lymphoma (PMID: 29650362; NCT01897571). 29650362
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 colorectal cancer not applicable Fluorouracil + Quinacrine Preclinical - Cell line xenograft Actionable In a preclinical study, Acrichine (quinacrine) and Adrucil (5-fluorouracil) synergized to inhibit tumor growth in colorectal cancer cell line xenograft models (PMID: 21725213). 21725213
Unknown unknown clear cell renal cell carcinoma not applicable Pazopanib Phase II Actionable In a Phase II trial, 84% (84/100) of patients with renal clear cell cancer demonstrated a clinical benefit, which included a median tumor reduction of 14.4%, when treated with Votrient (pazopanib) prior to a planned nephrectomy (PMID: 27254750). 27254750
Unknown unknown clear cell renal cell carcinoma not applicable Pazopanib Phase III Actionable In a Phase III trial, Votrient (pazopanib) did not result in a significantly improved disease free survival compared to placebo in patients with renal clear cell carcinoma (PMID: 28902533). 28902533
Unknown unknown colon cancer not applicable GANT61 Preclinical Actionable In a preclinical study, treatment with the GLI1/GLI2 inhibitor, GANT61, resulted in cytotoxicity in colon cancer cell lines (PMID: 21135115). 21135115
Unknown unknown acute myeloid leukemia not applicable A-485 Preclinical - Cell culture Actionable In a preclinical study, A-485 treatment inhibited proliferation in acute myeloid leukemia cell lines in culture (PMID: 28953875). 28953875
Unknown unknown triple-receptor negative breast cancer not applicable Selumetinib Preclinical - Cell line xenograft Actionable In a preclinical study, the Mek inhibitor Selumetinib (AZD6244) inhibited tumorigenicity and invasiveness of triple-receptor negative breast cancer cell lines in culture and in cell line xenograft models (PMID: 26384399). 26384399
Unknown unknown leiomyosarcoma not applicable Doxorubicin + Nilotinib Phase I Actionable In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin resulted in 1 short-duration stable disease and 1 progressive disease in 2 patients with leiomyosarcoma, consistent with synergistic growth inhibition in liposarcoma cells in culture and in xenograft models (PMID: 30037815; NCT02587169). 30037815
Unknown unknown prostate cancer not applicable Bicalutamide + Everolimus Phase II Actionable In a Phase II trial, Casodex (bicalutamide) and Afinitor (everolimus) combination treatment resulted in PSA response in 75% (18/24) of patients with castration-resistant prostate cancer, with a median overall survival of 28 months (PMID: 27019001). 27019001
Unknown unknown multiple myeloma no benefit Selumetinib Phase II Actionable In a Phase II clinical trial, Selumetinib (AZD6244) demonstrated safety and tolerability but had minimal activity with a complete response achieved in 5.6% (2/36) of refractory multiple myeloma patients (PMID: 26446942). 26446942
Unknown unknown glioblastoma multiforme no benefit Bevacizumab + Trebananib Phase II Actionable In a Phase II trial (NRG/RTOG 1122), addition of Trebananib (AMG 386) to Avastin (bevacizumab) therapy was tolerable, but did not improve 6-month progression-free survival (PFS) rate (22.6%, 12/53 vs 41.1%, 23/56), median overall survival (7.5 vs 11.5 months), median PFS (4.2 vs 4.8 months, HR=1.51, p=0.04), or radiographic response rate (4.2% vs 5.9%) in patients with recurrent glioblastoma (PMID: 32154928; NCT01609790). 32154928
Unknown unknown Advanced Solid Tumor not applicable SSR128129E Preclinical - Cell line xenograft Actionable In a preclinical study, SSR128129E inhibited tumor growth in several solid tumor cell line xenograft models (PMID: 23597562). 23597562
Unknown unknown stomach cancer not applicable Olaparib + Paclitaxel Phase II Actionable In a Phase II trial, addition of Lynparza (olaparib) to Taxol (paclitaxel) did not significantly improve progression free survival (3.91 vs 3.55 months) compared to Taxol alone, but did significantly prolong overall survival (13.1 vs 8.3 months) in patients with metastatic gastric cancer (PMID: 26282658; NCT01063517). 26282658
Unknown unknown stomach cancer not applicable Olaparib + Paclitaxel Phase III Actionable In a Phase III trial (GOLD), addition of Lynparza (olaparib) to Taxol (paclitaxel) did not significantly improve overall survival (8.8 vs 6.9 months, HR=0.79, p=0.026) compared to Taxol (paclitaxel) alone in Asian patients with advanced gastric cancer (PMID: 29103871; NCT01924533). 29103871
Unknown unknown esophagus squamous cell carcinoma not applicable Cisplatin + Paclitaxel + Toripalimab Phase II Actionable In a Phase II trial, Toripalimab (JS001) in combination with Taxol (paclitaxel) and Platinol (cisplatin) resulted in an objective response rate of 66.7% (8/12) and a disease control rate of 91.7% (11/12) in patients with metastatic esophageal squamous cell carcinoma, with a median duration of response of 6.8 months (J Clin Oncol 38: 2020 (suppl; abstr e15083); NCT02915432). detail...
Unknown unknown stomach cancer not applicable Chiauranib Preclinical - Cell line xenograft Actionable In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a gastric cancer cell line xenograft model (PMID: 28004478). 28004478
Unknown unknown ovarian carcinoma not applicable Disarib Preclinical - Cell line xenograft Actionable In a preclinical study, Disarib treatment led to apoptotic induction and resulted in tumor regression in ovarian carcinoma xenograft models (PMID: 27693384). 27693384
Unknown unknown breast cancer not applicable OBI-999 Preclinical - Cell line xenograft Actionable In a preclinical study, OBI-999 treatment inhibited tumor growth in a Globo H-expressing breast cancer cell line xenograft model (Cancer Res 2019;79(13 Suppl):Abstract nr 4815). detail...
Unknown unknown prostate cancer not applicable NSC156529 Preclinical - Cell line xenograft Actionable In a preclinical study, NSC156529 inhibited growth of human prostate cancer cell lines in culture, induced expression of differentiation markers and inhibited tumor growth in cell line xenograft models (PMID: 26294745). 26294745
Unknown unknown lung small cell carcinoma not applicable Ganetespib Preclinical - Cell line xenograft Actionable In a preclinical study, small cell lung carcinoma cell line xenograft models treated with Ganetespib demonstrated partial tumor growth inhibition, but with tumor progression, weight loss ensued (PMID: 27267850). 27267850
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 lung non-small cell carcinoma not applicable Abemaciclib + Gemcitabine Phase Ib/II Actionable In a Phase Ib trial, the combination of Verzenio (abemaciclib) and Gemzar (gemcitabine) was well-tolerated and demonstrated preliminary activity in patients with previously treated metastatic non-small cell lung cancer, resulting in a response rate of 4% (1/24; partial response (PR)), a disease control rate of 25% (6/24; 1 PR and 5 stable disease), and a median progression-free survival of 1.58 months (95% CI, 1.15, 4.24) (PMID: 30082474; NCT02079636). 30082474
Unknown unknown ovarian carcinoma no benefit Denileukin diftitox + Sirolimus Preclinical Actionable In a preclinical study, an ovarian carcinoma mouse model did not respond to the combination of Ontak (denileukin diftitox) and Rapamune (sirolimus), demonstrating no decrease in tumor burden (PMID: 27737881). 27737881
Unknown unknown renal cell carcinoma not applicable Avelumab + Axitinib FDA approved Actionable In a Phase III trial that supported FDA approval (JAVELIN Renal 101), Inlyta (axitinib) plus Bavencio (avelumab) resulted in a median progression-free survival of 13.8 mo. and an objective response rate of 51.4%, vs. 8.4 mo. and 25.7% with Sutent (sunitinib), respectively in patients with advanced renal cell carcinoma, and at median follow-up 14.3% (63) of patients treated with Inlyta (axitinib) plus Bavencio (avelumab) had died vs. 16.9% (75) with Sutent (sunitinib) (PMID: 30779531; NCT02684006). detail... 30779531
Unknown unknown hepatocellular carcinoma not applicable Orlistat Preclinical - Cell culture Actionable In a preclinical study, Xenical (orlistat) inhibited lipid metabolism and induced cell death in hepatocellular carcinoma cells in culture (PMID: 30667213). 30667213
Unknown unknown melanoma not applicable Dizocilpine Preclinical - Cell culture Actionable In a preclinical study, the NMDA antagonist, Dizocilpine (MK-801), demonstrated effectiveness against melanoma cells in culture (PMID: 24240127). 24240127
Unknown unknown multiple myeloma not applicable Bevacizumab + Dexamethasone + Evofosfamide Phase Ib/II Actionable In a Phase I/II trial, treatment with combined Avastin (bevacizumab), Evofosfamide (TH-302) therapy, and Adexone (dexamethasone) was well tolerated in patients with relapsed/refractory multiple myeloma and resulted in a disease control rate of 78.6% (22/28, stable disease or better), 3.6% (1/28) complete and 7.1% (2/28) partial responses, a 25% 6-month progression-free survival rate, a median progression-free survival of 2.2 months, and a median overall survival of 9.0 months (PMID: 30279233; NCT01522872). 30279233
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 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 lung non-small cell carcinoma no benefit Cetuximab + Cisplatin + Vinorelbine Phase III Actionable In a Phase III clinical trial, the addition of Erbitux (cetuximab) to Platinol (cisplatin) and Navelbine (vinorelbine) chemotherapy resulted in improved median overall survival of 11.3 months, compared to 10.1 months with chemotherapy alone, and an overall response rate of 36% (203/557) vs. 29% (166/568) with chemotherapy alone in patients with non-small cell lung cancer (PMID: 19410716). 19410716
Unknown unknown osteosarcoma not applicable YW3-56 Preclinical Actionable In a preclinical study, YW3-56 inhibited proliferation of a human osteosarcoma cell line in culture (PMID: 25612620). 25612620
Unknown unknown lung non-small cell carcinoma not applicable Cisplatin + Gemcitabine + Metformin Phase 0 Actionable In a pilot clinical trial, treatment with Glucophage (metformin) in combination with Gemzar (gemcitabine) and Platinol (cisplatin) resulted in an overall response rate of 46.7%, compared to 13.3% with Gemzar (gemcitabine) plus Platinol (cisplatin) therapy in patients with non-small cell lung cancer, but this difference was not statistically significant (PMID: 26434885). 26434885
Unknown unknown sarcoma not applicable Dasatinib Phase II Actionable In a Phase II clinical trial, 19% (8/42) of patients with undifferentiated pleomorphic sarcoma demonstrated clinical benefit when treated with Sprycel (dasatinib), however the 6 month progression free survival rate in patients was only 12% (6/42) (PMID: 26710211). 26710211
Unknown unknown Hodgkin's lymphoma not applicable Brentuximab vedotin + Umbralisib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of TGR-1202 and Brentuximab vedotin synergized to inhibit proliferation of several human Hodgkin's lymphoma cell lines in culture and to suppress tumor growth in xenograft models (Blood 124(21): 4486). detail...
Unknown unknown 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 lung non-small cell carcinoma not applicable AZD7648 + Radiotherapy Preclinical - Cell line xenograft Actionable In a preclinical study, AZD7648 treatment increased sensitivity to radiotherapy, inhibiting proliferation and inducing cell cycle arrest in non-small cell lung carcinoma cells in culture, and inhibiting tumor growth and inducing tumor regression in cell line xenograft models (PMID: 31699977). 31699977
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 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 diffuse large B-cell lymphoma not applicable CPI-0610 Phase I Actionable In a Phase I trial, treatment with CPI-0610 resulted in a complete response in two patients with diffuse large B-cell lymphoma (Blood 2015 126:1491). detail...
Unknown unknown hepatocellular carcinoma not applicable LCL161 + Paclitaxel Preclinical Actionable In a preclinical study, the combination of LCL161 and Taxol (paclitaxel) inhibited proliferation of hepatocellular carcinoma cells in culture (PMID: 24976294). 24976294
Unknown unknown prostate cancer not applicable 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 transitional cell carcinoma not applicable Paclitaxel + Pazopanib Phase II Actionable In a Phase II trial, patients with urothelial carcinoma treated with the combination of Taxol (paclitaxel) and Votrient (pazopanib) demonstrated an overall response rate of 54% (15/28), in which 11% (3/28) experienced a complete response and 43% (12/28) experienced stable disease (PMID: 27068017). 27068017
Unknown unknown rhabdomyosarcoma not applicable Cisplatin + YM155 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of YM155 and Platinol (cisplatin) inhibited tumor growth in a human rhabdomyosarcoma cell line xenograft model (PMID: 26983495). 26983495
Unknown unknown 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 esophagus squamous cell carcinoma not applicable Valproic acid Preclinical Actionable In a preclinical study, valproic acid enhanced the effectiveness of radiation in esophagus squamous cell carcinoma cell lines (PMID: 26135807). 26135807
Unknown unknown pancreatic cancer no benefit Capecitabine + Cisplatin + Epirubicin + Gemcitabine + Metformin Phase II Actionable In a Phase II trial, pancreatic cancer patients treated with PEXG combined with Glucophage (metformin) demonstrated a 6 month PFS of 42% (13/31), which did not differ from the control group 6 month PFS of 52% (15/29) (PMID: 26459175). 26459175
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 head and neck squamous cell carcinoma not applicable Cetuximab + EGFR Antisense DNA Preclinical - Cell line xenograft Actionable In a preclinical study, combination treatment with Erbitux (cetuximab) and intratumoral injection of EGFR antisense DNA reduced EGFR expression and tumor growth rate in a head and neck squamous cell carcinoma cell line xenograft mouse model to a greater extent than either treatment alone (PMID: 30291796). 30291796
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 Advanced Solid Tumor not applicable M2698 Phase I Actionable In a Phase I trial, M2698 (MSC2363318A) demonstrated safety and preliminary efficacy in a variety of advanced solid tumor patients (Ann. Oncol. 26 (Suppl 2): ii25-ii27, 2015). detail...
Unknown unknown Advanced Solid Tumor not applicable M2698 Phase I Actionable In a Phase I trial, M2698 (MSC2363318A) demonstrated safety and preliminary efficacy, with 19% (6/32) of advanced solid tumor patients remained on treatment for more than 180 days (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 370PD; NCT01971515). detail...
Unknown unknown Advanced Solid Tumor not applicable M2698 Preclinical Actionable In a preclinical study, MSC2363318A demonstrated sustained inhibition of S6K phosphorylation, and inhibited tumor growth in several human cancer xenograft models of breast, pancreatic, glioblastoma and ovarian cancers (Mol Cancer Ther 2013;12(11 Suppl):A162). detail...
Unknown unknown neuroblastoma not applicable Ribociclib Phase I Actionable In a Phase I trial, Kisqali (ribociclib) treatment demonstrated safety and resulted in stable disease in 28% (9/32) of pediatric patients with neuroblastoma or malignant rhabdoid tumor (MRT) (7 patients with neuroblastoma and 2 with CNS primary MRT), and 5 patients demonstrated stable disease for greater than 6 months (PMID: 28432176). 28432176
Unknown unknown head and neck squamous cell carcinoma not applicable Cisplatin + Docetaxel + Fluorouracil + Toripalimab Phase II Actionable In a Phase II trial, Toripalimab (JS001) in combination with Taxotere (docetaxel), Adrucil (fluorouracil), and Platinol (cisplatin) resulted in an objective response rate of 33.3% (1/3) and a disease control rate of 100% (3/3) in patients with metastatic head and neck squamous cell carcinoma, with a median duration of response of 7.1 months (J Clin Oncol 38: 2020 (suppl; abstr e15083); NCT02915432). detail...
Unknown unknown urinary bladder cancer not applicable Celecoxib Preclinical - Cell culture Actionable In a preclinical study, Celebra (celecoxib) treatment resulted in decreased cell viability in multiple human bladder cancer cell lines in culture (PMID: 27406983). 27406983
Unknown unknown bone cancer not applicable Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in an overall response rate of 5% (2/40) and 12-week progression free rate of 28% (11/40) in patients with bone sarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). detail...
Unknown unknown anal squamous cell carcinoma not applicable Prexasertib Phase I Actionable In a Phase I trial, Prexasertib (LY2606368) treatment resulted in partial response in a patient with anal squamous cell carcinoma (PMID: 27044938). 27044938
Unknown unknown anal squamous cell carcinoma not applicable Prexasertib Phase Ib/II Actionable In a Phase Ib trial, treatment with Prexasertib (LY2606368) resulted in an overall response rate of 15% (4/26; 1 complete response (CR) and 3 partial responses (PR)), a clinical benefit rate (CR+PR+stable disease) of 58% (15/26), and a median progression-free survival of 2.8 months in patients with squamous cell carcinoma of the anus (PMID: 29643063; NCT0115790). 29643063
Unknown unknown lung cancer not applicable SKI-G-801 Preclinical Actionable In a preclinical study, SKI-G-801 treatment reduced tumor growth in a syngeneic mouse model of lung cancer (Cancer Res 2019;79(13 Suppl):Abstract nr 2010). detail...
Unknown unknown diffuse large B-cell lymphoma not applicable Loncastuximab tesirine Phase I Actionable In a Phase I trial, Loncastuximab tesirine treatment resulted in an objective response rate of 57.1% (20/35) with a complete response rate of 34.3% (12/35) in patients with diffuse large B-cell lymphoma (Blood 2017 130(Suppl 1):187, NCT02669017). detail...
Unknown unknown NUT midline carcinoma not applicable Birabresib Preclinical - Cell line xenograft Actionable In a preclinical study, Birabresib (OTX015) inhibited tumor growth in cell line xenograft models of NUT midline carcinoma (Mol Cancer Ther November 2013 12; C244). detail...
Unknown unknown NUT midline carcinoma not applicable Birabresib Phase Ib/II Actionable In a Phase Ib trial, Birabresib (OTX015) demonstrated favorable safety and resulted in partial response in 33% (3/9) and stable disease in 33% (3/9) patients with NUT midline carcinoma (PMID: 29733771; NCT02259114). 29733771
Unknown unknown non-Hodgkin lymphoma not applicable CUDC-907 Preclinical - Cell line xenograft Actionable In a preclinical study, CUDC-907 inhibited cell growth in a human non-Hodgkin lymphoma cell line in culture, and stabilized tumor growth in xenograft models (PMID: 22693356). 22693356
Unknown unknown prostate cancer not applicable ARV-771 Preclinical - Cell line xenograft Actionable In a preclinical study, ARV-771 treatment induced apoptosis and inhibited proliferation of prostate cancer cell lines in culture, and inhibited tumor growth and induced regression in a cell line xenograft model (PMID: 27274052). 27274052
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 acute myeloid leukemia not applicable CPI-0610 + Doxorubicin Preclinical - Cell line xenograft Actionable In a preclinical study, the combination treatment of CPI-0610 and Adriamycin (doxorubicin) resulted in inhibition of tumor growth in acute myeloid leukemia xenograft models (PMID: 26815195). 26815195
Unknown unknown glioblastoma multiforme no benefit MP7 Preclinical - Cell culture Actionable In a preclinical study, MP7 treatment did not result in significant inhibition of cell proliferation and showed minimal change in cell viability in a glioblastoma cell line in culture, thus, providing no benefit (PMID: 27797168). 27797168
Unknown unknown acute myeloid leukemia no benefit AZD1208 Phase I Actionable In a Phase I trial, AZD1208 treatment was tolerated and demonstrated activity in pharmacodynamic studies, but resulted in no clinical response (0/32) in patients with acute myeloid leukemia (PMID: 29765150; NCT01489722). 29765150
Unknown unknown esophageal 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 urinary bladder cancer not applicable E7766 Preclinical - Cell line xenograft Actionable In a preclinical study, E7766 treatment induced production of cytokines of the STING pathway, resulted in dose-dependent curative effect in orthotopic mouse models mimicking BCG-unresponsive non-muscle invasive bladder cancer (Cancer Res 2019;79(13 Suppl):Abstract nr 3269). detail...
Unknown unknown malignant pleural solitary fibrous tumor not applicable Dasatinib Phase II Actionable In a Phase II trial, patients with solitary fibrous tumors demonstrated a median progression free survival of 2 months and five patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown ovarian cancer not applicable Napabucasin Phase Ib/II Actionable In a Phase Ib/II clinical trial, treatment with BBI608 demonstrated safety in patients with platinum-resistant ovarian cancer (including patients with epithelial ovarian, fallopian, or peritoneal cancer), and resulted in a disease control rate of 68% (27/40) and an objective response rate of 25% (10/40) in evaluable patients (J Clin Oncol, May 2016 vol. 34 no. 15_suppl 5578). detail...
Unknown unknown lung non-small cell carcinoma no benefit Bevacizumab + Carboplatin + Cixutumumab + Paclitaxel Phase II Actionable In a Phase II trial, the combination therapy of Avastin (bevacizumab), Paraplatin (carboplatin), Taxol (paclitaxel), and Cixutumumab resulted in greater toxicity and did not improve overall survival when compared to Avastin (bevacizumab), Paraplatin (carboplatin), and Taxol (paclitaxel) without Cixutumumab in non-small cell lung carcinoma patients (PMID: 28950351; NCT00955305). 28950351
Unknown unknown 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 hepatocellular carcinoma not applicable Foretinib Phase I Actionable In a Phase I trial, Foretinib (GSK1363089) demonstrated safety and activity in hepatocellular carcinoma patients, with an overall response rate by mRESCIST of 22.9% (8/35, all partial responses), a disease stabilization rate of 82.9% (29/35), and a median duration of response of 7.6 months (PMID: 27821605; NCT00920192). 27821605
Unknown unknown 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 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 breast carcinoma not applicable RXDX-106 + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, RXDX-106 in combination with an unspecified anti-PD-1 antibody resulted in near complete regression in orthotopic animal models of breast carcinoma (Eur J Cancer, Vol 69, Supplement 1, December 2016, Page S31). detail...
Unknown unknown 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 lymphoma not applicable Abexinostat Phase II Actionable In a Phase II trial, patients with T-cell lymphoma demonstrated an overall response rate of 40% (6/15) and a median duration response of 11.5 months when treated with Abexinostat (PCI-24781) (PMID: 28126962). 28126962
Unknown unknown melanoma not applicable Epacadostat Phase I Actionable In a Phase I trial, treatment with Epacadostat resulted in stable disease in 13% (7/52) of patients with advanced solid tumors for greater than or equal to 16 weeks, including two patients with melanoma who previously failed on immunotherapy (PMID: 28053021). 28053021
Unknown unknown Advanced Solid Tumor not applicable PV1162 Preclinical - Cell culture Actionable In a preclinical study, cultured cells treated with PV1162 showed increased loss of human artificial chromosomes, suggesting chromosomal instability (PMID: 26837770). 26837770
Unknown unknown Advanced Solid Tumor no benefit Tasquinimod Phase II Actionable In a Phase II trial, treatment with Tasquinimod provided no benefit in patients with advanced solid tumors, including those with hepatocellular cancer, ovarian cancer, renal cell cancer, or gastric cancer (PMID: 28798986; NCT01743469). 28798986
Unknown unknown colorectal cancer not applicable HL-085 + OKI-005 Preclinical - Cell culture Actionable In a preclinical study, HL085 and OKI-005 demonstrated synergistic activity in 3 of 6 colorectal cancer cell lines, and increased immunogenicity of tumor cells in culture (Cancer Res 2019;79(13 Suppl):Abstract nr 4753). detail...
Unknown unknown Advanced Solid Tumor not applicable Anlotinib Phase I Actionable In a Phase I trial, Anlotinib (AL-3818) treatment resulted in partial response in 15% (3/20) and stable disease in 70% (14/20) of patients with advanced solid tumors (J Clin Oncol 33, 2015 (suppl; abstr e13586)). detail...
Unknown unknown triple-receptor negative breast cancer not applicable Doxorubicin + DT2216 Preclinical - Cell culture Actionable In a preclinical study, the addition of DT2216 to Adriamycin (doxorubicin) treatment resulted in decreased viability of a triple-negative breast cancer cell line in culture (PMID: 31792461). 31792461
Unknown unknown myelodysplastic syndrome not applicable FF-10501-01 Phase I Actionable In a Phase I trial, FF-10501-01 demonstrated safety and preliminary efficacy, resulted in stable disease control with no disease progression over 3-14 cycles of treatment in 50% (3/6) of myelodysplastic syndrome patients, including 1 patient achieved complete marrow response (Blood 128 (22):1640). detail...
Unknown unknown glioblastoma multiforme not applicable MGCD516 Preclinical - Cell line xenograft Actionable In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human glioblastoma multiforme cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). detail...
Unknown unknown urinary bladder cancer not applicable ASP5878 Phase I Actionable In a Phase I trial, ASP5878 demonstrated safety and some preliminary efficacy in patients with advanced solid tumors, including a bladder cancer patient with an FGFR gene mutation (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A165). detail...
Unknown unknown renal carcinoma not applicable MRx0518 Preclinical Actionable In a preclinical study, MRx0518 stimulated immune response and reduced tumor size in syngeneic mouse models of renal carcinoma (Journal of Clinical Oncology 36, no. 15_suppl). detail...
Unknown unknown breast cancer not applicable NU6027 + Rucaparib Preclinical - Cell culture Actionable In a preclinical study, NU6027 enhanced the efficacy of Rubraca (rucaparib) in breast cancer cells in culture, resulting in a greater decreased cell survival (PMID: 21730979). 21730979
Unknown unknown 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 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 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 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 melanoma not applicable SF2523 Preclinical Actionable In a preclinical study, SF2523 reduced tumor growth and decreased lung metastasis in syngeneic mouse melanoma models (PMID: 31018997). 31018997
Unknown unknown 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 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 colorectal cancer not applicable V158411 Preclinical - Cell culture Actionable In a preclinical study, V158411 inhibited Chek1 autophosphorylation, induced DNA damage and cell-cycle arrest, and inhibited growth of a colorectal cancer cell line in culture (PMID: 27829224). 27829224
Unknown unknown Ewing sarcoma of bone not applicable Dasatinib Phase II Actionable In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in Ewing sarcoma patients (n=17) was suspended due to lack of drug activity (PMID: 26710211). 26710211
Unknown unknown hepatocellular carcinoma not applicable GNS561 Preclinical - Pdx & cell culture Actionable In a preclinical study, GNS561 inhibited growth of hepatocellular carcinoma cell lines in culture, and tumor growth in patient-derive xenograft (PDX) models (Cancer Res 2017;77(13 Suppl):Abstract nr 5124). detail...
Unknown unknown Ewing sarcoma no benefit Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in no objective response (0/13) in patients with Ewing sarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). detail...
Unknown unknown Advanced Solid Tumor not applicable ETC-159 Phase I Actionable In a Phase I trial, treatment with ETC-159 was well-tolerated, decreased Wnt signaling, and resulted in stable disease in 2 out of 16 treated patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2584)). detail...
Unknown unknown pancreatic cancer no benefit Gemcitabine + LY2603618 Phase II Actionable In a Phase II trial, treatment with the combination of LY2603618 and Gemzar (gemcitabine) did not result in improved overall survival, progression-free survival, duration of response, or clinical benefit compared to Gemzar (gemcitabine) alone in patients with unresectable pancreatic cancer (PMID: 28202004). 28202004
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 head and neck cancer not applicable AsiDNA Preclinical - Cell culture Actionable In a preclinical study, AsiDNA inhibited survival of a head and neck cancer cell line in culture (PMID: 27559053). 27559053
Unknown unknown stomach cancer no benefit Irinotecan + Nimotuzumab Phase II Actionable In a Phase II trial, addition of Nimotuzumab to Camptosar (irinotecan) did not significantly improve median progression-free survival (73 vs 85 days), median overall survival (250.5 vs 232.0 days) and response rate (18.4 vs 10.3 %) compared to Camptosar alone in gastric cancer patients (PMID: 25185971). 25185971
Unknown unknown Advanced Solid Tumor not applicable Cobimetinib Phase I Actionable In a Phase I trial, Cotellic (cobimetinib) treatment resulted in stable disease for five months or more in five patients with advanced solid tumors (PMID: 27424159). 27424159 detail...
Unknown unknown colon cancer not applicable Gemcitabine + SRA737 Preclinical - Cell line xenograft Actionable In a preclinical study, Gemzar (gemcitabine) resulted in enhanced antitumor efficacy when combined with SRA737 (CCT245737) in a colon cancer cell line xenograft model (PMID: 27167172). 27167172
Unknown unknown 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 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 lymphoma not applicable Carfilzomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of a lymphoma cell lines in culture (PMID: 28753594). 28753594
Unknown unknown hepatocellular carcinoma not applicable Camrelizumab Phase I Actionable In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity, resulted in partial response in 33.3% (1/3) of patients with hepatocellular carcinoma (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). detail...
Unknown unknown ovarian cancer not applicable MLN4924 Preclinical Actionable In a preclinical study, MLN4924 demonstrated moderate efficacy in ovarian cancer cell lines and enhanced synergistic effects in combination with cisplatin or carboplatin (PMID: 23939375). 23939375
Unknown unknown Advanced Solid Tumor not applicable Belvarafenib Phase I Actionable In a Phase I trial, Belvarafenib (HM95573) treatment demonstrated safety and preliminary efficacy, resulted in unconfirmed partial response in 3% (1/31) and stable disease in 29% (9/31) of patients with advanced solid tumors harboring BRAF (45%), KRAS (45%) or NRAS (10%) mutations (May 20 2016) 2570-2570; NCT02405065). detail...
Unknown unknown 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 malignant peripheral nerve sheath tumor not applicable Dasatinib Phase II Actionable In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in patients with malignant peripheral nerve sheath tumors (n=14) was suspended due to lack of drug activity (PMID: 26710211). 26710211
Unknown unknown 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 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 hematologic cancer not applicable Tefinostat Phase I Actionable In a Phase I trial, Tefinostat (CHR-2845) demonstrated safety and preliminary efficacy in patients with hematological cancers (PMID: 23647373). 23647373
Unknown unknown bladder carcinoma in situ not applicable Pembrolizumab FDA approved Actionable In a Phase II trial (KEYNOTE-057) that supported FDA approval, Keytruda (pembrolizumab) treatment demonstrated safety and efficacy, resulted in a 3-month complete response rate of 40.2% (41/102), with a median duration of follow-up of 15.8 months in patients with BCG-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are unable or refuse to undergo cystectomy (J Clin Onc 37, no. 15_suppl (May 20, 2019) 4530-4530; NCT02625961). detail... detail...
Unknown unknown Ewing sarcoma not applicable UAB30 Preclinical Actionable In a preclinical study, a renal Ewing sarcoma cell line was sensitive to UAB30 in culture, demonstrating cell-cycle arrest, decreased cell proliferation, and apoptosis (PMID: 26873726). 26873726
Unknown unknown colon cancer not applicable Ensituximab Phase I Actionable In a Phase I trial, Ensituximab (NEO-102) demonstrated safety and preliminary efficacy, resulting in stable disease in 42% (5/12) of patients with refractory colon or pancreatic cancer (PMID: 27449137; NCT01040000). 27449137
Unknown unknown nasopharynx carcinoma not applicable Cisplatin + Gemcitabine + Toripalimab Phase II Actionable In a Phase II trial, Toripalimab (JS001) in combination with Gemzar (gemcitabine) and Platinol (cisplatin) resulted in an objective response rate of 75.0% (9/12) and a disease control rate of 83.3% (10/12) in patients with metastatic nasopharyngeal carcinoma, with a median duration of response of 7.7 months (J Clin Oncol 38: 2020 (suppl; abstr e15083); NCT02915432). detail...
Unknown unknown breast cancer not applicable WANT3 Preclinical - Cell culture Actionable In a preclinical study, WANT3 treatment resulted in suppression of cell invasion of breast cancer cells in culture (PMID: 27432794). 27432794
Unknown unknown acute myeloid leukemia not applicable AKN-028 + Cytarabine Preclinical - Cell culture Actionable In a preclinical study, the sequential treatment of Cytosar-U (cytarabine) and AKN-028 resulted in a syntergistic effect in acute myeloid leukemia cells in culture, demonstrating antileukemic activity (PMID: 22864397). 22864397
Unknown unknown breast cancer not applicable Cyclophosphamide + Methotrexate + Vandetanib Phase I Actionable In a Phase I study, Caprelsa (vandetanib), in combination with Cytoxan (cyclophosphamide) and Abitrexate (methotrexate), demonstrated safety and some efficacy in breast cancer patients (PMID: 23001754). 23001754
Unknown unknown 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 mantle cell lymphoma not applicable CC214-1 Preclinical - Patient cell culture Actionable In preclinical study, mantle cell lymphoma patient derived cell lines demonstrated improved survival in culture when treated with CC214-1 (PMID: 25839159). 25839159
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 lung adenocarcinoma not applicable AC-93253 iodide + Gefitinib Preclinical - Cell culture Actionable In a preclinical study, AC-93253 iodide combined with Iressa (gefitinib) resulted in a synergistic effect, demonstrating growth inhibition of an Iressa (gefitinib)-resistant lung adenocarcinoma cell line in culture (PMID: 29132432). 29132432
Unknown unknown astrocytoma not applicable Dactolisib + Everolimus Phase Ib/II Actionable In a Phase Ib trial, the combination of BEZ235 and Afinitor (everolimus) resulted in stable disease in a patient with astrocytoma (PMID: 28357727). 28357727
Unknown unknown lung non-small cell carcinoma not applicable SH-1242 Preclinical - Pdx & cell culture Actionable In a preclinical study, SH-1242 inhibited growth of several non-small cell lung cancer (NSCLC) cell lines in culture, including cell lines with acquired drug resistance, and inhibited tumor growth in cell line and patient-derived xenograft models (PMID: 26645561). 26645561
Unknown unknown Advanced Solid Tumor not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) demonstrated safety and some anti-tumor activity across a range of advanced solid tumor types (PMID: 25728526). 25728526
Unknown unknown Advanced Solid Tumor not applicable Ixabepilone + Sunitinib Phase I Actionable In a Phase I trial, Ixabepilone and Sutent (sunitinib) combination therapy resulted in partial response in 15% (4/27) and stable disease in 48% (13/27) of patients with advanced solid tumors (PMID: 26864210). 26864210
Unknown unknown diffuse large B-cell lymphoma no benefit Fostamatinib Phase II Actionable In a Phase II clinical trial, fostamatinib demonstrated safety, but limited efficacy in patients with diffuse large B-cell lymphoma, with an objective response rate of 3% (2/68) and stable disease in 10% (7/68) of patients (PMID: 26707592). 26707592
Unknown unknown Advanced Solid Tumor not applicable LYC-55716 Phase I Actionable In a Phase I trial, Cintirorgon (LYC-55716) treatment resulted in a partial response in 8% (2/25) and stable disease in 44% (11/25) of patients with an advanced solid tumor, with stable disease lasting 2 to 12 months (PMID: 30819679). 30819679
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 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 diffuse large B-cell lymphoma not applicable Tisagenlecleucel FDA approved Actionable In a Phase II trial (JULIET) that supported FDA approval, Kymriah (tisagenlecleucel) treatment resulted in an overall response rate of 50% (34/68) , with complete response in 32% (22/68) and partial response in 18% (12/68) of patients with diffuse large B-cell lymphoma (ASH, 59th Annual Meeting and Exposition, Dec 2017, Abstract 577; NCT02445248). detail... detail...
Unknown unknown Advanced Solid Tumor not applicable Prexasertib Phase I Actionable In a Phase I trial, Prexasertib (LY2606368) treatment resulted in partial response in 4.4% (2/45) and stable disease ranging from 1.2 to 6.7 months in 33.3% (15/45) of patients with advanced solid tumors (PMID: 27044938). 27044938
Unknown unknown Hodgkin's lymphoma, nodular sclerosis not applicable Domatinostat Case Reports/Case Series Actionable In a Phase I trial, a patient with nodular sclerosis Hodgkin's lymphoma demonstrated a partial response lasting almost 6 months following treatment with Domatinostat (4SC-202) (PMID: 30347469; NCT01344707). 30347469
Unknown unknown breast cancer not applicable BNC105P Preclinical - Cell line xenograft Actionable In a preclinical study, BNC105P treatment disrupted tumor vasculature, induced apoptosis and necrosis, inhibited tumor growth, and induced regression in a cell line xenograft model of breast cancer (PMID: 20515948). 20515948
Unknown unknown melanoma not applicable PD-0325901 Phase I Actionable In a Phase I trial, PD-0325901 demonstrated some efficacy in previously treated melanoma patients, however, there was significant toxicity above 10 mg BID (PMID: 21516509). 21516509
Unknown unknown 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 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 prostate cancer not applicable Capivasertib + Enzalutamide Phase I Actionable In a Phase I trial, Capivasertib (AZD5363) and Xtandi (enzalutamide) combination treatment was well-tolerated and demonstrated safety, and resulted in an overall response rate of 25% (3/12) in evaluable metastatic castration-resistant prostate cancer patients (n=16) harboring either a PTEN loss or AKT1 E17K (PMID: 32205016; NCT02525068). 32205016
Unknown unknown acute myeloid leukemia not applicable Liposomal Annamycin Phase Ib/II Actionable In a Phase I/II trial, Liposomal annamycin demonstrated safety and preliminary efficacy, resulted in complete clearing of circulating peripheral blasts in 62% (5/8) of patients with relapsed or refractory acute lymphoblastic leukemia (PMID: 23763920). 23763920
Unknown unknown triple-receptor negative breast cancer not applicable SR-4835 Preclinical - Pdx Actionable In a preclinical study, SR-4835 treatment induced DNA damage and cell death, and inhibited tumor growth in a patient-derived xenograft (PDX) model of triple-negative breast cancer harboring a BRCA1 mutation (PMID: 31668947). 31668947
Unknown unknown 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 retinoblastoma not applicable Prexasertib Preclinical - Cell culture Actionable In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including a retinoblastoma cell line (PMID: 28270495). 28270495
Unknown unknown melanoma not applicable EX-527 Preclinical Actionable In a preclinical study, Ex-527 inhibited growth of human melanoma cell lines in culture (PMID: 25486469). 25486469
Unknown unknown Hodgkin's lymphoma not applicable Dezapelisib 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 pancreatic ductal adenocarcinoma not applicable Gemcitabine + MN58b Preclinical Actionable In a preclinical study, MN58b and Gemzar (gemcitabine) in combination demonstrated an additive effect on growth inhibition of pancreatic ductal adenocarcinoma cell lines in culture (PMID: 26769123). 26769123
Unknown unknown 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 thyroid gland cancer not applicable SNS-314 Preclinical Actionable In a preclinical study, SNS-314 reduced growth of anaplastic thyroid carcinoma cells in culture (PMID: 23099978). 23099978
Unknown unknown 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 acute myeloid leukemia not applicable Bortezomib + Cytarabine + Etoposide + Midostaurin + Mitoxantrone Phase I Actionable In a Phase I trial, Rydapt (midostaurin), in combination with Velcade (bortezomib) and mitoxantrone, Vepesid (etoposide), and Cytosar-U (cytarabine) (MEC), resulted in an overall response rate of 82.5% (19/23) in patients with relapsed or refractory acute myeloid leukemia receiving dose level 3 and above, with complete responses in 56.5% (13/23) of patients (PMID: 26784138). 26784138
Unknown unknown mantle cell lymphoma not applicable Ibrutinib FDA approved Actionable In a Phase II trial (Study 1104) that supported FDA approval, Imbruvica (ibrutinib) treatment resulted in a response rate of 68% (75/111, complete response 21%, partial response 47%), with an estimated median progression-free survival of 13.9 months in patients with relapsed or refractory mantle-cell lymphoma (PMID: 23782157, NCT01236391). 23782157 detail...
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 pancreatic cancer not applicable CBP501 + Cisplatin Preclinical - Cell culture Actionable In a preclinical study, the combination of CBP501 and Platinol (cisplatin) resulted in increased cell death compared to Platinol (cisplatin) alone in a human pancreatic cancer cell line in culture (PMID: 17237275). 17237275
Unknown unknown adrenal gland pheochromocytoma not applicable 131I-MIBG FDA approved Actionable In a Phase II trial that supported FDA approval, Azedra (iobenguane I 131) treatment resulted in partial response in 23% (15/68) of patients with pheochromocytoma or paraganglioma who received 1 therapeutic dose, with a 12-month overall survival rate of 91% (J Clin Oncol 36, 2018 (suppl; abstr 4005); NCT00874614). detail... detail...
Unknown unknown 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 MALT lymphoma not applicable Chlorambucil + Rituximab Phase III Actionable In a Phase III trial, Chlorambucil and Rituxan (Rituximab) combination treatment resulted in improved event-free survival (HR=0.54) but not longer overall survival in patients with mucosa-associated lymphoid tissue lymphoma (PMID: 28355112). 28355112
Unknown unknown colon carcinoma not applicable SLC-391 + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, treatment with SLC-391 combined with an anti-PD-1 antibody demonstrated synergy and increased overall survival rate in a syngeneic mouse model of colon carcinoma (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B148). 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 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 Advanced Solid Tumor not applicable ALT-803 Phase I Actionable In a Phase I trial, ALT-803 treatment demonstrated safety and preliminary efficacy in patients with advanced solid tumors, resulted in desirable NK cell expansion and strong reaction at injection sites (PMID: 30045932; NCT01727076). 30045932
Unknown unknown Advanced Solid Tumor not applicable Sapitinib Preclinical - Cell line xenograft Actionable In a preclinical study, Sapitinib (AZD8931) inhibited EGFR, ERBB2 (HER2), and ERBB3 (HER3) kinase activity, and inhibited tumor growth in several cell line xenograft models, including breast, NSCLC, colorectal, and head and neck squamous cell carcinoma xenograft models (PMID: 20145185). 20145185
Unknown unknown 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 lung non-small cell carcinoma not applicable Pimitespib Phase I Actionable In a Phase I trial, TAS-116 treatment resulted in partial response in 2 patients with non-small cell lung carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 2546)). detail...
Unknown unknown ovarian cancer not applicable ETP-46464 + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, ETP-46464 increased the sensitivity of ovarian cancer cell lines to ionizing radiation in culture (PMID: 25560806). 25560806
Unknown unknown skin squamous cell carcinoma not applicable R11.1.6 Preclinical - Cell culture Actionable In a preclinical study, R11.1.6 treatment did not inhibit colony formation in a skin squamous cell carcinoma cell line in culture (PMID: 29720559). 29720559
Unknown unknown colon cancer not applicable Lenvatinib Preclinical Actionable In a preclinical study, Lenvima (lenvatinib) induced apoptosis and inhibited proliferation of colorectal cancer cells in culture (PMID: 24255582). 24255582
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 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 cancer not applicable Olaparib FDA approved Actionable In a Phase II trial that supported FDA approval, Lynparza (olaparib) maintenance therapy resulted in significantly improved progression-free survival compared to placebo (8.4 vs 4.8 mo, HR=0.35, p<0.001) in patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneum cancer (PMID: 22452356; NCT00753545). 22452356 detail...
Unknown unknown Advanced Solid Tumor not applicable PTC596 Phase I Actionable In a Phase I trial, PTC596 demonstrated safety and preliminary efficacy, resulted in stable disease in 16% (5/31) of patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2574)). detail...
Unknown unknown Advanced Solid Tumor not applicable PRN1371 Preclinical - Pdx & cell culture Actionable In a preclinical study, PRN1371 inhibited proliferation of various FGFR-driven tumor cell lines in culture and inhibited tumor growth in a variety of patient-derived xenograft models with FGFR pathway alterations (AACR; 2016. Abstract nr 1249). detail...
Unknown unknown pancreatic cancer not applicable Acalabrutinib + Pembrolizumab Phase II Actionable In a Phase II trial, the combination therapy of Calquence (acalabrutinib) and Keytruda (pembrolizumab) was well tolerated, but resulted in minimal clinical benefit with an overall response rate of 7.9% (3/38) and a disease control rate of 21.1% (8/38) in patients with advanced or metastatic pancreatic cancer (PMID: 32114502). 32114502
Unknown unknown lung small cell carcinoma not applicable Alisertib + Paclitaxel Preclinical - Pdx & cell culture Actionable In a preclinical study, Alisertib (MLN8237), alone and in combination with Taxol (paclitaxel), inhibited growth of small cell lung cancer cell (SCLC) lines in culture and inhibited tumor growth in primary human SCLC xenograft models (Mol Cancer Ther 2013;12(11 Suppl):A282). detail...
Unknown unknown lung small cell carcinoma not applicable Alisertib + Paclitaxel Phase II Actionable In a Phase II trial, the combination treatment of Alisertib (MLN8237) and Taxol (paclitaxel) compared to Taxol (paclitaxel) plus placebo resulted in a progression-free survival of 3.32 months versus 2.17 months (P=0.038), and led to an overall response rate of 22% (20/89) versus 18% (16/289), and a disease control rate of 58% (52/89) versus 46% (41/89), respectively, in patients with relapsed or refractory small cell lung cancer (PMID: 31655296; NCT02038647). 31655296
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 breast cancer not applicable XL388 Preclinical - Cell culture Actionable In a preclinical study, XL388 treatment led to tumor growth inhibition in breast cancer cell line xenograft models (PMID: 23394126). 23394126
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 lymphoblastic lymphoma not applicable Ibrutinib + VLX1570 Preclinical - Cell culture Actionable In a preclinical study, the combination of Imbruvica (ibrutinib) and VLX1570 worked synergistically to reduce viability of an Imbruvica (ibrutinib)-resistant Waldenstrom macroglobulinemia cell line in culture (PMID: 27813535). 27813535
Unknown unknown CLL/SLL not applicable Idelalisib FDA approved Actionable In a Phase II trial that supported FDA approval, Zydelig (idelalisib) treatment resulted in an overall response rate of 58% (15/26, all partial response) in patients with relapsed small lymphocytic lymphoma, with a median duration of response of 11.9 months (PMID: 24450858; NCT01282424). 24450858 detail...
Unknown unknown head and neck cancer not applicable Olaparib Preclinical - Pdx Actionable In a preclinical study, a patient-dervived xenograft (PDX) model of head and neck cancer harboring mutant TP53 and wild-type ATM did not demonstrate sensitivity to Lynparza (olaparib) treatment (PMID: 31699977). 31699977
Unknown unknown chronic myelomonocytic leukemia not applicable H3B-8800 Preclinical - Pdx Actionable In a preclinical study, H3B-8800 treatment reduced tumor burden in patient-derived xenograft (PDX) models of chronic myelomonocytic leukemia harboring spliceosome mutations but not spliceosome wild-type models (Blood 2016 128:966). detail...
Unknown unknown colon cancer not applicable Doxorubicin + NU7441 Preclinical - Cell culture Actionable In a preclinical study, NU7441 increased sensitivity of colon cancer cell lines to Adriamycin (doxorubicin), resulting in reduced cell survival in culture (PMID: 16707462). 16707462
Unknown unknown Advanced Solid Tumor not applicable Fluorouracil + Ipatasertib + Leucovorin + Oxaliplatin Phase I Actionable In a Phase Ib trial, the combination of Ipatasertib (GDC-0068) and mFOLFOX6 demonstrated safety in patients with advanced solid tumors, and resulted in an overall response rate of 6.1% (2/33), including partial responses in two patients, stable disease in 51.5% (17/33) of patients, a six-month progression-free survival rate of 18.2% (6/33), and maximum progression-free survival duration of 50 months in a patient with appendix cancer (PMID: 32205017; NCT01362374). 32205017
Unknown unknown clear cell renal cell carcinoma no benefit Bevacizumab + Sorafenib Phase III Actionable In a Phase II clinical trial, treatment with the combination of Nexavar (sorafenib) and Avastin (bevacizumab) did not result in a significant improvement in progression-free survival compared to treatment with Avastin (bevacizumab) as a single agent (9.2 months vs 7.4 months) in patients with renal clear cell carcinoma (PMID: 26077237). 26077237
Unknown unknown acral lentiginous melanoma not applicable Toripalimab Phase I Actionable In a Phase I trial, Toripalimab (JS001) demonstrated safety and preliminary efficacy, resulted in an objective response rate of 23.1% (3/13) and a disease control rate of 46.2% (6/13, 1 complete response, 2 partial response, 3 stable disease) in patients with acral melanoma (PMID: 30642373; NCT02836795). 30642373
Unknown unknown acral lentiginous melanoma not applicable Toripalimab Phase II Actionable In a Phase II trial (POLARIS-01), Toripalimab (JS001) resulted in an objective response rate of 17.3% (22/127, 1 CR, 21 PR) and a disease control rate of 57.5% in melanoma patients, with a median progression-free survival (mPFS) of 3.6 mo and a median overall survival (mOS) of 22.2 mo, better ORR (31.0%, 14.0%, 0%), mPFS (5.5, 3.2, 1.9 mo), and mOS (not reached, 16.9, 10.3 mo) were observed in non-acral cutaneous melanoma (n=29) than in acral (n=50) and mucosal (n=22) subtypes (PMID: 32321714; NCT03013101). 32321714
Unknown unknown glioblastoma multiforme not applicable BI2536 Preclinical - Cell culture Actionable In a preclinical study, a glioblastoma cell line treated with BI2536 demonstrated decreased cell viability in culture (PMID: 26573800). 26573800
Unknown unknown neuroendocrine tumor not applicable Toripalimab Phase Ib/II Actionable In a Phase Ib trial, treatment with Toripalimab (JS001) resulted in an objective response rate of 20% (8/40), a disease control rate of 35% (14/40), a median duration of response of 15.2 months, a median progression-free survival of 2.5 months, and a median overall survival of 7.8 months in patients with neuroendocrine tumors (PMID: 32086343; NCT03167853). 32086343
Unknown unknown multiple myeloma not applicable Daratumumab and hyaluronidase-fihj + Dexamethasone + Lenalidomide FDA approved Actionable In a Phase II trial (PLEIADES) that supported FDA approval, Darzalex Faspro (Daratumumab and hyaluronidase-fihj) demonstrated safety and efficacy comparable to Darzalex (daratumumab) when combined with Adexone (dexamethasone) and Revlimid (lenalidomide) in patients with relapsed or refractory multiple myeloma, resulted in an objective response rate of 93.8% (61/65, complete response 21.5%) (17th International Myeloma Workshop Sep 2019, Boston, MA, US. OAB-022). detail... detail...
Unknown unknown breast cancer not applicable V158411 Preclinical - Cell culture Actionable In a preclinical study, V158411 induced DNA damage and cell-cycle arrest, and inhibited growth of a breast cancer cell line in culture (PMID: 27829224). 27829224
Unknown unknown glioblastoma multiforme not applicable VAL-083 Phase Ib/II Actionable In a Phase I/II trial, treatment with a clinically active dose of VAL-083 resulted in improved overall survival (7.9 vs 2.9 months, HR=0.341, p=0.003) compared to an inactive dose in Avastin (bevacizumab)-refractory glioblastoma multiforme patients (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 2061-2061; NCT01478178). detail...
Unknown unknown gastroesophageal junction adenocarcinoma not applicable Envafolimab + Fluorouracil + Leucovorin + Oxaliplatin Phase II Actionable In a Phase II trial, Envafolimab (KN035) in combination with FOLFOX regimen resulted in an objective response rate of 60% (9/15) and a median progression-free survival of 6.8 months in patients with advanced gastric (n=13) or gastroesophageal junction (n=2) cancer (J Clin Oncol 38: 2020 (suppl; abstr e16585); CTR20181124). detail...
Unknown unknown Advanced Solid Tumor not applicable Citarinostat + Paclitaxel Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of ACY-241 and Taxol (paclitaxel) resulted in increased efficacy in advanced solid tumor xenograft models, including pancreatic and ovarian (AACR; Cancer Res 2016;76(14 Suppl):Abstract nr 4822). detail...
Unknown unknown Advanced Solid Tumor not applicable Tivozanib Phase I Actionable In a Phase I clinical study, Tivozanib (AV-951) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Clin Cancer Res, November 15, 2011 17; 7156). detail...
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 Advanced Solid Tumor not applicable AZD1208 Phase I Actionable In a Phase I trial, AZD1208 treatment was tolerated and demonstrated activity in pharmacodynamic studies, resulted in stable disease for 6 weeks or longer as best objective response in 48% (13/27) of evaluable patients with advanced solid tumor (PMID: 29765150; NCT01588548). 29765150
Unknown unknown Advanced Solid Tumor not applicable OPB-31121 Phase I Actionable In a Phase I trial, OPB-31121 treatment resulted in stable diseases in 44% (8/18) of patients with advanced solid tumors, and tumor shrinkage in 2 patients (1 colon cancer, 1 rectal cancer) (PMID: 25715763). 25715763
Unknown unknown Advanced Solid Tumor not applicable 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 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 malignant glioma not applicable DNX-2401 Phase I Actionable In a Phase I trial, DNX-2401 treatment demonstrated virus-induced oncolysis in patients' tumors, resulted in more than 95% tumor reduction in 3 patients, and survival for more than 3 years in 20% (5/25) of patients with recurrent malignant glioma (PMID: 29432077; NCT02197169). 29432077
Unknown unknown malignant glioma not applicable DNX-2401 Phase I Actionable In a Phase I trial, DNX-2401 treatment resulted in tumor reduction in 72% (18/25), complete response in 12% (3/25) and prolonged stable disease in 8% (2/25) of patients with recurrent malignant glioma, with a median overall survival of 9.5 months and progression-free survival of more than 3 years in patients achieved complete responses (PMID: 29432077). 29432077
Unknown unknown gastric adenocarcinoma no benefit Oxaliplatin + Ramucirumab + Tegafur-gimeracil-oteracil Potassium 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 Advanced Solid Tumor not applicable Pazopanib + Trametinib Phase I Actionable In a Phase I trial, combination treatment with Votrient (pazopanib) and Mekinist (trametinib) was tolerable and resulted in partial response in 12% (3/25), and stable disease in 72% (18/25) of patients with advanced solid tumors, and 9 patients remained on study for more than 6 cycles, including patients with differentiated thyroid cancer (n=3), colorectal cancer (n=2), melanoma (n=1), cholangiocarcinoma (n=1), ovarian cancer (n=1), and synovial cell sarcoma (n=1) (PMID: 31186313; NCT01438554). 31186313
Unknown unknown non-Hodgkin lymphoma not applicable DS-3201b Phase I Actionable In a Phase I trial, DS-3201b demonstrated preliminary clinical activity in patients with non-Hodgkin lymphoma, with an overall response rate of 53% (8/15; 1 complete response/remission, and 7 partial responses), stable disease in 5 patients, and 8 patients on treatment with tumor shrinkage for greater than 24 weeks (Blood Dec 2017, 130 (Suppl 1) 4070; NCT02732275). detail...
Unknown unknown thyroid gland medullary carcinoma not applicable Cabozantinib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Cometriq (cabozantinib) resulted in improved progression free survival in patients with metastatic medullary thyroid cancer (PMID: 23319867). detail... 23319867
Unknown unknown acute myeloid leukemia not applicable ABBV-744 Preclinical - Cell line xenograft Actionable In a preclinical study, ABBV-744 inhibited growth of acute myeloid leukemia cells in culture and in cell line xenograft models (Cancer Res 2018;78(13 Suppl):Abstract nr DDT01-05). detail...
Unknown unknown 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 prostate cancer not applicable Docetaxel + MEDI5117 Preclinical Actionable In a preclinical study, MEDI5117 in combination with Taxotere (docetaxel) resulted in tumor regression in human prostate cancer xenograft models (PMID: 26744529). 26744529
Unknown unknown prostate cancer not applicable Atezolizumab + Cabozantinib Phase I Actionable In a Phase I trial (COSMIC-021), Tecentriq (atezolizumab) and Cometriq (Cabometyx, cabozantinib) combination therapy was tolerated, resulted in an objective response rate of 32% (14/44, 2 complete responses, 12 partial responses) and stable disease in 48% (21/44) of patients with metastatic castration-resistant prostate cancer whose disease progressed after Xtandi (enzalutamide) or Zytiga (abiraterone) treatment (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 5564-5564; NCT03170960). detail...
Unknown unknown Advanced Solid Tumor not applicable Gemcitabine + Milciclib Phase I Actionable In a Phase I trial, the combination therapy of Gemzar (gemcitabine) and Milciclib (PHA-848125AC) resulted in a clinical benefit in 36% (5/14) of patients with an advanced solid tumor, including long-term stable disease (6-14 months) in four patients and a partial response in a patient with non-small cell lung carcinoma (PMID: 28424962). 28424962
Unknown unknown squamous cell carcinoma not applicable Prexasertib Phase Ib/II Actionable In a Phase Ib trial, Prexasertib (LY2606368) demonstrated safety and resulted in a clinical benefit rate at 3 months of 29% (29/101) in squamous cell carcinoma patients, including patients with squamous cell carcinoma of the anus, head and neck squamous cell carcinoma, and squamous non-small cell lung cancer (PMID: 29643063; NCT0115790). 29643063
Unknown unknown squamous cell carcinoma not applicable Prexasertib Phase I Actionable In a Phase I trial, Prexasertib (LY2606368) treatment resulted in partial response in 2 patients with squamous cell carcinoma (SCC), and 40% (6/15) of solid tumor patients who achieved stable disease had SCC (PMID: 27044938; NCT0115790). 27044938
Unknown unknown 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 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 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 pancreatic cancer not applicable Pegilodecakin Phase I Actionable In a Phase I trial, AM0010 treatment in patients with pancreatic cancer resulted in 47% (8/17) of patients with stable disease, three patients with a progression free survival greater than 6 months, and a median overall survival of 5.1 months (J Clin Oncol 34, 2016 (suppl; abstr 3082). detail...
Unknown unknown Advanced Solid Tumor not applicable Panulisib Preclinical - Cell line xenograft Actionable In a preclinical study, Panulisib (P7170) inhibited growth of several solid tumor cell lines in culture and in cell line xenograft models (PMID: 25700704). 25700704
Unknown unknown malignant peripheral nerve sheath tumor not applicable Carfilzomib + Selinexor Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Selinexor (KPT-330) and Kyprolis (carfilzomib) resulted in downregulation of Birc5 (Survivin) and enhanced induction of apoptotic markers compared to Selinexor (KPT-330) alone, and worked additively to decrease viability of malignant peripheral nerve sheath tumor (MPNST) cells in culture, and resulted in enhanced tumor growth inhibition in MPNST xenograft models (PMID: 28314790). 28314790
Unknown unknown multiple myeloma not applicable SJB3-019A Preclinical - Cell culture Actionable In a preclinical study, treatment with SJB3-019A induced cell-cycle arrest and apoptosis and decreased viability of multiple myeloma cell lines in culture (PMID: 28270494). 28270494
Unknown unknown glioblastoma multiforme not applicable Regorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Stivarga, (regorafenib), inhibited tumor growth in cell line xenograft models of glioblastoma multiforme (PMID: 21170960). 21170960
Unknown unknown neuroblastoma not applicable Ellipticine + Valproic acid Preclinical Actionable In a preclincal study, Depakene (valproic acid) enhanced the cytotoxicity of ellipticine in human neuroblastoma cell lines (PMID: 22167207). 22167207
Unknown unknown malignant glioma not applicable Everolimus Phase II Actionable In a Phase II trial, Afinitor (everolimus) treatment resulted in a 6-month progression free survival rate of 87% and 55% in patients with WHO grade II and III/IV glioma, respectively (Neuro Oncol (2016) 18 (suppl 6): vi8-vi9.). detail...
Unknown unknown gastric adenocarcinoma not applicable MK2206 Phase II Actionable In a Phase II trial, MK2206 demonstrated safety, but did not meet study endpoint of median overall survival of 6.5 months, however gastric adenocarcinoma patients were not stratified according to somatic profiles (PMID: 25827820). 25827820
Unknown unknown ovarian cancer not applicable AB61 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the synthetic nucleoside AB61 resulted in decreased tumor volume in ovarian cancer cell line xenograft models (PMID: 26819331). 26819331
Unknown unknown acute myeloid leukemia not applicable Cytarabine + Filgrastim + GDC-0575 Preclinical - Pdx Actionable In a preclinical study, addition of Neupogen (filgrastim) to the combination of GDC-0575 and Cytosar-U (cytarabine) resulted in enhanced killing of acute myeloid leukemia cells in patient-derived xenograft (PDX) models (PMID: 29162833). 29162833
Unknown unknown 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 mantle cell lymphoma not applicable ST7612AA1 Preclinical Actionable In a preclinical study, ST7612AA1 inhibited proliferation of mantle cell lymphoma cell lines in culture (PMID: 25671299). 25671299
Unknown unknown Advanced Solid Tumor not applicable BI 836880 Phase I Actionable In a Phase I trial, BI 836880 treatment was well tolerated, and resulted in partial response in 7% (2/29) and stable disease in 31% (9/29) of patients with advanced solid tumors (J Clin Onc 2018 36:15_suppl, 12024; NCT02674152). detail...
Unknown unknown 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 lung non-small cell carcinoma not applicable Dasatinib Phase II Actionable In a Phase II trial, Sprycel (dasatinib) treatment in non-small cell lung carcinoma patients showed some clinical efficacy, resulting in one patient with a partial response, and 12 patients with stable disease, demonstrating a disease control rate of 43% (13/30)(PMID: 20855820). 20855820
Unknown unknown 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 invasive bladder transitional cell carcinoma not applicable Cisplatin + Gemcitabine + Sorafenib Phase II Actionable In a Phase II trial, Nexavar (sorafenib) in combination with Platinol (cisplatin) and Gemzar (gemcitabine) resulted in pathologic complete response in 42.2% (19/45) of patients with muscle-invasive urothelial bladder cancer (J Clin Oncol 35, 2017 (suppl 6S; abstract 345)). detail...
Unknown unknown 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 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 cutaneous T cell lymphoma not applicable Duvelisib Phase I Actionable In a Phase I trial, Copiktra (duvelisib) treatment resulted in an overall response rate of 31.6% (6/19, 6 partial response) with a median duration of treatment of 12.6 weeks in patients with cutaneous T cell lymphoma (PMID: 29233821; NCT01476657). 29233821
Unknown unknown gastric adenocarcinoma not applicable Paclitaxel + Ramucirumab FDA approved Actionable In a Phase III trial (RAINBOW) that supported FDA approval, Cyramza (ramucirumab) and Taxol (paclitaxel) combination treatment significantly improved overall survival (9.6 vs 7.4 mo, HR=0.807, p=0.017) compared to Taxol (paclitaxel) alone in patients with advanced gastric or gastroesophageal junction adenocarcinoma who progressed on prior chemotherapy (PMID: 25240821; NCT01170663). 25240821 detail...
Unknown unknown Ewing sarcoma no benefit GSK-LSD1 Preclinical - Cell culture Actionable In a preclinical study, Ewing sarcoma cell lines did not demonstrate sensitivity to GSK-LSD1 in culture (PMID: 29997151). 29997151
Unknown unknown colorectal adenocarcinoma not applicable CRLX101 + Fluorouracil + Radiotherapy Preclinical - Cell line xenograft Actionable In a preclinical study, the addition of CRLX101 to Adrucil (fluorouracil) and radiotherapy resulted in a greater decrease in tumor volume in colorectal adenocarcinoma xenograft models when compared to the combination of Adrucil (fluorouracil) and radiotherapy only (PMID: 27784746). 27784746
Unknown unknown breast cancer not applicable Gemcitabine + Trametinib Phase Ib/II Actionable In a Phase Ib clinical trial, the combination of Mekinist (trametinib) and Gemzar (gemcitabine) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors, including one complete response in a patient with breast cancer (PMID: 23583440). 23583440
Unknown unknown acute leukemia not applicable Volasertib Phase I Actionable In a Phase I trial, Volasertib (BI 6727) demonstrated safety and limited efficacy in pediatric patients with acute leukemia or advanced solid tumors, with stable disease as best objective response in 71% (5/7) of patients with acute leukemia and in 13% (2/15) of patients with advanced solid tumors (PMID: 31276318; NCT01971476). 31276318
Unknown unknown Advanced Solid Tumor not applicable Mivebresib Phase I Actionable In a Phase I trial, Mivebresib (ABBV-075) treatment demonstrated safety and modest clinical activity, resulted in stable disease as best response in 43% (26/61) of evaluable patients with advanced solid tumors, with a median progression-free survival of 1.8 months (PMID: 31420359; NCT02391480) . 31420359
Unknown unknown Advanced Solid Tumor not applicable Necitumumab Phase I Actionable In a Phase I clinical trial, Portrazza (necitumumab) was well-tolerated and demonstrated preliminary efficacy in patients with advanced solid tumors (PMID: 20197484). 20197484
Unknown unknown Indication other than cancer not applicable Nintedanib FDA approved Actionable Ofev (nintedanib) is FDA approved for use in patients with idiopathic pulmonary fibrosis (FDA.gov). detail... detail...
Unknown unknown prostate cancer not applicable UC-857993 Preclinical Actionable In a preclinical study, UC-773587 inhibited ERK activation and growth of prostate cells in culture (PMID: 25825487). 25825487
Unknown unknown Advanced Solid Tumor not applicable 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 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 colon carcinoma not applicable CTX-471 Preclinical Actionable In a preclinical study, CTX-471 treatment inhibited tumor growth and increased survival in a syngeneic mouse model of colon carcinoma (PMID: 32161196). 32161196
Unknown unknown myelodysplastic syndrome not applicable Azacitidine + Hu5F9-G4 Phase I Actionable In a Phase Ib trial, Magrolimab (Hu5F9-G4) and Vidaza (azacitidine) combination therapy was well tolerated, and resulted in complete response in 42% (14/33), marrow complete response in 24% (8/33), partial response in 3% (1/33), hematologic improvement in 21% (7/33), and stable disease in 9% (3/33) of patients with intermediate to very high risk myelodyaplastic syndrome (J Clin Oncol 38: 2020 (suppl; abstr 7507); NCT03248479). detail...
Unknown unknown transitional cell carcinoma not applicable Ascrinvacumab Phase II Actionable In a Phase II trial, PF-03446962 demonstrated multiple toxicities and limited efficacy in patients with urothelial cancer (PMID: 24566706). 24566706
Unknown unknown rhabdomyosarcoma not applicable AZD8055 + Selumetinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Koselugo (selumetinib) and AZD8055 worked synergistically to inhibit tumor growth in xenograft models of rhabdomyosarcoma (PMID: 23918606). 23918606
Unknown unknown prostate cancer not applicable JNJ-54302833 Preclinical Actionable In a preclinical study, JNJ-54302833 inhibited growth of prostate cancer cells in culture ( Cancer Res October 1, 2014 74; 4747 ). detail...
Unknown unknown hematologic cancer not applicable AZD4573 Preclinical - Cell culture Actionable In a preclinical study, AZD4573 induced apoptosis and cell death in a panel of hematologic cancer cell lines in culture (Cancer Res 2018;78(13 Suppl):Abstract nr 310). detail...
Unknown unknown endometrial cancer not applicable Cabozantinib Phase II Actionable In a Phase II (NCI9322/PHL86) trial, Cometriq (Cabometyx, cabozantinib) treatment resulted in a response rate (RR) of 14% (5/36), a 12-week progression-free survival (PFS) rate of 67%, and a mPFS of 4.8 mo in patients with recurrent/metastatic endometrial cancer of endometrioid histology, and a RR of 12% (4/34), a 12-week PFS of 56%, and a mPFS of 4.0 mo in patients of serous histology, and a RR of 6% (2/32) and a 12-week PFS of 47% in patients of uncommon histology (PMID: 31992589; NCT01935934). 31992589
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 breast cancer not applicable Aldoxorubicin + Rigosertib Preclinical - Cell line xenograft Actionable In a preclinical study, human breast cancer cells demonstrated a complete tumor response in cell line xenograft models when treated with a combination of Rigosertib (ON01910) and Aldoxorubicin (PMID: 15766665). 15766665
Unknown unknown 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 Indication other than cancer not applicable Imatinib FDA approved Actionable Gleevec (imatinib) is FDA approved for patients with aggressive systemic mastocytosis in which c-KIT mutational status is unknown (FDA.gov). detail... detail...
Unknown unknown ovary epithelial cancer not applicable MGD013 Phase I Actionable In a Phase I trial, MGD013 treatment resulted in a partial response in 13.3% and stable disease in 46.7% (7/15) of patients with epithelial ovarian cancer (J Clin Oncol 38: 2020 (suppl; abstr 3004); NCT03219268). detail...
Unknown unknown lung cancer not applicable AZ628 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Mekinist (trametinib) and AZ628 resulted in greater inhibition of Mek and apoptosis in a non-BRAF V600 mutant lung cancer cell line in culture compared to the combination of Mekinist (trametinib) and Tafinlar (dabrafenib) (J Clin Oncol 35, 2017 (suppl; abstr e23154)). detail...
Unknown unknown gastrointestinal system cancer not applicable Rivoceranib Phase III Actionable In a Phase III trial, treatment with Apatinib (YN968D1) at 850mg resulted in a greater progression free survival (2.6 mo vs 1.8 mo) and overall survival (6.5 mo vs 4.7 mo) when compared to placebo in patients with either gastric cancer or gastroesophageal junction adenocarcinoma (PMID: 26884585). 26884585
Unknown unknown gastrointestinal system cancer not applicable Rivoceranib Phase II Actionable In a Phase II trial, Apatinib (YN968D1) improved progression-free survival and overall survival in metastatic gastric cancer patients (PMID: 23918952). 23918952
Unknown unknown triple-receptor negative breast cancer not applicable Lucitanib Preclinical Actionable In a preclinical study, Lucitanib (E-3810) demonstrated antitumor activity in mouse xenograft models of triple negative breast cancer with synergistic effects noted when using Lucatinib plus chemotherapy (PMID: 23270924). 23270924
Unknown unknown pancreatic ductal adenocarcinoma not applicable Fluorouracil + Oxaliplatin + Veliparib Phase Ib/II Actionable In a Phase I/II trial, Veliparib (ABT-888) in combination with Adrucil (fluorouracil) and Eloxatin (oxaliplatin) demonstrated safety, and resulted in an objective response rate of 26% (15/58, 4 complete responses, 11 partial responses), a disease control rate of 52%, a progression-free survival of 4.0 months, and an overall survival of 7.8 months in patients with metastatic pancreatic ductal adenocarcinoma (PMID: 32669374; NCT01489865). 32669374
Unknown unknown triple-receptor negative breast cancer not applicable GSK2801 + JQ1 Preclinical - Cell culture Actionable In a preclinical study, GSK2801 and JQ1 treatment synergistically inhibited cell growth and viability, led to enhanced cell cycle arrest, and induced senescence and apoptosis in triple-negative breast cancer cell lines in culture (PMID: 31000582). 31000582
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 Advanced Solid Tumor not applicable Selinexor Phase I Actionable In a Phase I trial, Selinexor (KPT-330) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, resulting in an objective response rate of 4% (7/157; 1 complete response and 6 partial responses) and stable disease in 43% (67/157), with 17% (27/157) of patients demonstrating stable disease for at least 4 months (PMID: 26926685). 26926685
Unknown unknown melanoma not applicable AsiDNA + Olaparib Preclinical - Cell culture Actionable In a preclinical study, AsiDNA treatment led to increased sensitivity to Lynparza (olaparib), resulting in decreased survival of melanoma cell lines in culture (PMID: 27559053). 27559053
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 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 marginal zone B-cell lymphoma not applicable ME-401 Phase I Actionable In a Phase I trial, ME-401 treatment was well-tolerated, resulted in an objective response rate of 100% (4/4) in patients with relapsed or refractory marginal zone B-cell lymphoma (J Clin Oncol 38: 2020 (suppl; abstr 8016); NCT02914938). detail...
Unknown unknown pancreatic ductal adenocarcinoma not applicable Dinaciclib Preclinical - Cell culture Actionable In a preclinical study, Dinaciclib (SCH 727965) treatment inhibited proliferation and induced apoptosis in mouse pancreatic ductal adenocarcinoma cell lines in culture, and delayed tumor growth, and increased overall survival in a transgenic mouse model (PMID: 32269732). 32269732
Unknown unknown stomach cancer not applicable Rivoceranib 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 breast cancer not applicable AsiDNA + AZD2461 Preclinical - Cell culture Actionable In a preclinical study, AsiDNA and AZD2461 combination treatment resulted in increased cell death and inhibition of proliferation in breast cancer cell lines compared to AZD2461 alone in culture (PMID: 27559053). 27559053
Unknown unknown multiple myeloma not applicable GSK1904529A Preclinical - Cell culture Actionable In a preclinical study, multiple myeloma cells were sensitive to GSK1904529A in culture, resulting in decreased cell viability (PMID: 19383820). 19383820
Unknown unknown synovial sarcoma not applicable Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 10% (1/10) of patients with synovial sarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). detail...
Unknown unknown 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 mantle cell lymphoma not applicable Duvelisib Preclinical - Cell culture Actionable In a preclinical study, Copiktra (duvelisib) inhibited proliferation, and resulted in an increase in activation-induced cytidine deaminase (AID) expression and genomic instability in a mantle cell lymphoma cell line in culture (PMID: 28199309). 28199309
Unknown unknown Advanced Solid Tumor not applicable XL147 Phase I Actionable In a Phase I trial, 43.9% (25/56) of patients with advanced solid tumors had stable disease as a best response to treatment with Pilaralisib (XL147), and one patient with NSCLC showed a partial response, and in a separate therapy arm, Pilaralisib (XL147) in tablet formulation demonstrated safety and some antitumor activity in advanced solid tumor patients, including 11.1% (2/18) of patients with a partial response (PMID: 29593099, PMID: 24166903; NCT00486135). 29593099 24166903
Unknown unknown kidney 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 kidney cancer (PMID: 28911087). 28911087
Unknown unknown gastrointestinal system cancer not applicable Axitinib + Fluorouracil + Leucovorin + Oxaliplatin Phase I Actionable In a Phase I trial, Inlyta (axitinib), in combination with FOLFOX, demonstrated safety and some efficacy in patients with gastrointestinal tumors (PMID: 24423921). 24423921
Unknown unknown glioblastoma multiforme not applicable SR9009 Preclinical - Pdx & cell culture Actionable In a preclinical study, SR9009 inhibited growth of glioblastoma cell lines in culture, resulted in apoptosis in tumors and prolonged survival in both cell line and patient-derived xenograft models (PMID: 29320480). 29320480
Unknown unknown 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 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 plexiform neurofibroma not applicable Selumetinib FDA approved Actionable In a Phase II trial (SPRINT) that supported FDA approval, Koselugo (selumetinib) treatment resulted in an objective response rate of 70% (35/50) in pediatric patients 2 years or older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas, with 28 of the responses lasted over 1 year (PMID: 32187457; NCT01362803). 32187457
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 hematologic cancer not applicable TAS4464 Preclinical - Cell culture Actionable In a preclinical study, TAS4464 demonstrated cytotoxicity in a number of hematologic cancer cell lines in culture (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr C176). detail...
Unknown unknown Advanced Solid Tumor not applicable Copanlisib Phase I Actionable In a Phase I clinical trial, treatment with Aliqopa (copanlisib) was well-tolerated and demonstrated preliminary activity in patients with advanced solid tumors, with complete response in 2% (1/48), partial response in 4% (2/48), and stable disease in 31% (15/48) of patients (PMID: 27672108). 27672108
Unknown unknown Advanced Solid Tumor not applicable Copanlisib Phase I Actionable In a Phase I trial, Aliqopa (copanlisib) treatment resulted in no complete or partial response (0/10) and a disese control rate of 40% in patients with advanced solid tumors (PMID: 27915408). 27915408
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 prostate cancer not applicable AGS-1C4D4 Phase I Actionable In a Phase I trial, AGS-1C4D4 treatment in castration resistant prostate cancer patients was well-tolerated and resulted in stable disease at 24 weeks in 46% (6/13) of patients, but did not reduce PSA (PMID: 22020316). 22020316
Unknown unknown lung non-small cell carcinoma not applicable Atezolizumab + CPI-444 Phase I Actionable In a Phase I trial, treatment with the combination of CPI-444 and Tecentriq (atezolizumab) was well-tolerated and resulted in a disease control rate of 71% (5/7) in patients with non-small cell lung cancer (J Clin Oncol 35, 2017 (suppl; abstr 3004)). detail...
Unknown unknown glioblastoma multiforme not applicable Tepotinib Preclinical Actionable In a preclinical study, Tepotinib (EMD-1214063) induced tumor regression in mouse cell line xenograft models of glioblastoma (PMID: 23553846). 23553846
Unknown unknown acute myeloid leukemia not applicable RG7112 Phase I Actionable In a Phase I clinical trial, RG7112 demonstrated clinical activity in acute myeloid leukemia, with complete response in 7% (2/30), complete response with incomplete platelet recovery in 3% (1/30), partial response in 7% (2/30), and stable disease in 30% (9/30) of patients (PMID: 26459177). 26459177
Unknown unknown chronic lymphocytic leukemia not applicable Samalizumab Phase I Actionable In a Phase I trial, Samalizumab (ALXN6000) treatment demonstrated safety and preliminary efficacy, resulting in first-dose response in 40% (10/25) of patients with B-cell chronic lymphocytic leukemia, and reduction of tumor burden in 2 patients (Blood 2010 116:2465). detail...
Unknown unknown Advanced Solid Tumor not applicable YYB-101 Phase I Actionable In a Phase I trial, YYB-101 demonstrated safety and preliminary efficacy in patients with refractory solid tumors, with partial response in 4.5% (1/22) and stable disease in 45.5% (10/22) of patients (J Clin Onc. 2018 36:15_suppl, e14501). detail...
Unknown unknown Advanced Solid Tumor not applicable Spartalizumab Phase I Actionable In a Phase I trial, Spartalizumab (PDR001) treatment in advanced solid tumor patients demonstrated tolerability and an overall response rate of 3.4% (2/58), with one atypical carcinoid lung tumor patient and one anal cancer patient achieving partial responses, and an additional 41.4% (24/58) of patients demonstrating stable disease (PMID: 32179633; NCT02404441). 32179633
Unknown unknown diffuse large B-cell lymphoma not applicable GS-5829 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, diffuse large B-cell lymphoma cell lines demonstrated greater cell growth inhibition when treated with a combination of GS-5829 and Venclexta (venetoclax) compared to either agent alone in culture (Blood 2016 128:5104). detail...
Unknown unknown Merkel cell carcinoma not applicable Avelumab Phase Ib/II Actionable In a Phase I/II trial, addition of Bavencio (avelumab) to adoptive transfer of Merkel cell polyomavirus (MCPyV)-specific T cells and HLA upregulation resulted in sustained complete response in 75% (3/4) of patients with MCPyV-associated Merkel cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 3044)). detail...
Unknown unknown Merkel cell carcinoma not applicable Avelumab Phase III Actionable In a Phase II trial (JAVELIN Merkel 200), Bavencio (avelumab) treatment resulted in a 62.1% (18/29) objective response rate and a median progression-free survival of 9.1 months in patients with treatment-naive metastatic Merkel cell carcinoma, with response duration estimated to be greater than 3 months and 6 months in 93% and 83% of responding patients, respectively (PMID: 29566106; NCT 02155647). 29566106
Unknown unknown Merkel cell carcinoma not applicable Avelumab FDA approved Actionable In a Phase II trial (JAVELIN Merkel 200) that supported FDA approval, Bavencio (avelumab) treatment resulted in an objective response response rate of 31.8% (28/88), with complete response in 9% (8/88) and partial response in 23% (20/88) of patients with metastatic Merkel cell carcinoma that progressed after chemotherapy (PMID: 27592805; NCT02155647). 27592805 detail...
Unknown unknown acute myeloid leukemia not applicable Venetoclax Phase II Actionable In a Phase II trial, treatment with Venclexta (venetoclax) resulted in a 19% (6/32) overall response rate, a 6% (2/32) complete response, and a 13% (4/32) complete response with incomplete blood count recovery in acute myeloid leukemia patients (PMID: 27520294). 27520294
Unknown unknown renal cell carcinoma not applicable 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 ovarian cancer not applicable Cisplatin + ETP-46464 Preclinical - Cell culture Actionable In a preclinical study, ETP-46464 increased the sensitivity of ovarian cancer cell lines to Platinol (cisplatin) in culture (PMID: 25560806). 25560806
Unknown unknown prostate cancer not applicable Enzalutamide + Ipatasertib Phase I Actionable In a Phase Ib trial, the combination of Ipatasertib (GDC-0068) and Xtandi (enzalutamide) demonstrated safety in prostate cancer patients, and resulted in an overall response rate of 11.8% (2/17), including partial responses in two patients, stable disease in 23.5% (4/17) of patients, a six-month progression-free survival rate of 23.5% (4/17), and maximum progression-free survival duration of 19.5 months (PMID: 32205017; NCT01362374). 32205017
Unknown unknown CLL/SLL not applicable Venetoclax Phase I Actionable In a Phase I trial, Venclexta (venetoclax) treatment resulted in a 79% (92/116) overall response rate and 20% (23/116) complete response rate in patients with either chronic lymphocytic leukemia or small lymphocytic lymphoma (PMID: 26639348). 26639348
Unknown unknown breast cancer not applicable CS-11 Preclinical Actionable In a preclinical study, CS-11 induced apoptosis and inhibited growth of breast cancer cell lines in culture, and inhibited tumor growth and metastasis in an orthotopic breast cancer mouse model (PMID: 28500231). 28500231
Unknown unknown 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 multiple myeloma not applicable Bortezomib + Tabalumab Phase I Actionable In a Phase I trial, 42% (20/46) of patients with multiple myeloma demonstrated a partial response, including 3 with a complete response and 2 with a very good partial response, when treated with a combination of Tabalumab and Velcade (bortezomib) (PMID: 27287072). 27287072
Unknown unknown ovarian serous carcinoma not applicable Paclitaxel + Vistusertib Phase I Actionable In a Phase I trial, the combination therapy of Taxol (paclitaxel) and Vistusertib (AZD2014) resulted in a RECIST response rate of 64% (16/25), a CA125 response rate of 52% (13/25), and a median progression-free survival of 5.8 months in patients with high grade serous ovarian cancer (PMID: 30016392; NCT02193633). 30016392
Unknown unknown lymphoma not applicable Alisertib + Vorinostat Phase I Actionable In a Phase I trial, Alisertib (MLN8237) in combination with Zolinza (vorinostat) demonstrated safety and preliminary efficacy in patients with relapsed or refractory lymphomas, resulting in an overall response rate of 16.7% (3/18, 1 complete response, 2 partial responses) at the recommended Phase 2 dose (PMID: 31617432; NCT01567709). 31617432
Unknown unknown hepatocellular carcinoma not applicable Resminostat + Sorafenib Phase Ib/II Actionable In a Phase I/II trial, the combination of Resminostat (4SC-201) and Nexavar (sorafenib) demonstrated increased efficacy compared to Resminostat (4SC-201) alone in advanced hepatocellular carcinoma patients, resulting in an improved progression-free survival rate of 62.5% vs. 12.5%, a median time to progression of 4.1 vs. 1.8 months, and an overall survival of 8.0 vs. 6.5 months (PMID: 26952006). 26952006
Unknown unknown urinary bladder cancer not applicable Celecoxib + OBP-801 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Celebra (celecoxib) and OBP-801 resulted in a synergistic effect, demonstrating increased apoptotic activity and decreased tumor volume in xenograft models of bladder cancer (PMID: 27406983). 27406983
Unknown unknown melanoma not applicable CP-870,893 + Tremelimumab Phase I Actionable In a Phase I trial, the combination of CP-870,893 and Tremelimumab was well-tolerated, and metastatic melanoma patients treated with CP-870,893 and Tremelimumab combination therapy demonstrated an objective response rate of 27.3% (6/22; 2 complete and 4 partial responses), a median progression-free survival of 3.2 months, and median overall survival of 23.6 months, with a 45-month median follow-up (PMID: 30288340; NCT01103635). 30288340
Unknown unknown ovarian cancer not applicable Paclitaxel + Volasertib Preclinical - Cell culture Actionable In a preclinical study, Volasertib (BI 6727) and Taxol (paclitaxel) synergistically inhibited growth of ovarian cancer cell lines in culture (PMID: 32183025). 32183025
Unknown unknown lung squamous cell carcinoma not applicable Cisplatin + Gemcitabine + Tislelizumab Case Reports/Case Series Actionable In a Phase II trial, treatment with Tislelizumab (BGB-A317) plus platinum doublet chemotherapy (Gemzar (gemcitabine) with Platinol (cisplatin) or Paraplatin (carboplatin)) in patients with squamous non-small cell lung cancer resulted in an objective response rate of 67% (4/6) and disease control rate of 83% (5/6), including a partial response in four patients and stable disease in one patient (PMID: 32769013; NCT03432598). 32769013
Unknown unknown 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 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 prostate cancer sensitive Bicalutamide FDA approved Actionable In a clinical trial that supported FDA approval, combined with a luteinizing hormone-releasing hormone analogue (LHRH-A) therapy, Casodex (bicalutamide) demonstrated efficacy similar to Eulexin (flutamide), resulted in comparable median time to progression (97 vs 77 weeks, HR=0.93, p=0.41) and survival time (180 vs 148 weeks, HR=0.87, p=0.15) in patients with metastatic prostate cancer (PMID: 9301693). 9301693 detail...
Unknown unknown 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 alveolar soft part sarcoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 77%, median progression-free survival of 21 months, an objective response rate of 46% (n=13), and median overall survival was not reached in patients with alveolar soft part sarcoma (PMID: 29895706; NCT01878448). 29895706
Unknown unknown transitional cell carcinoma not applicable Avelumab FDA approved Actionable In a Phase III trial (JAVELIN Bladder 100) that supported FDA approval, addition of maintenance Bavencio (avelumab) to best supportive care (BSC) significantly improved overall survival compared to BSC alone (21.4 vs 14.3 mo, HR=0.69, p=0.0005) in patients with advanced urothelial carcinoma (J Clin Oncol 38, (Jun 2020) no. 18_suppl; NCT02603432). detail... detail...
Unknown unknown transitional cell carcinoma not applicable Avelumab FDA approved Actionable In a Phase I trial (JAVELIN Solid Tumor) that supported FDA approval, Bavencio (avelumab) demonstrated safety and resulted in a response rate of 17% (27/161; 9 complete responses and 18 partial responses), a median progression-free survival of 6.3 weeks, and a median overall survival of 6.5 months in patients with platinum-refractory metastatic urothelial carcinoma (PMID: 29217288; NCT01772004). detail... 29217288
Unknown unknown Ewing sarcoma not applicable Temozolomide + Veliparib Preclinical Actionable In a preclinical study, Ewing sarcoma cells treated with Temodar (temozolomide) combined with Veliparib (ABT-888) resulted in strong synergism, demonstrating reduced cell viability in culture (PMID: 26438158). 26438158
Unknown unknown glioblastoma multiforme no benefit Valproic acid Clinical Study Actionable In a pooled analysis of several clinical trials, use of Valproic acid was not associated with improved progression-free survival or overall survival in glioblastoma patients (PMID: 26786929). 26786929
Unknown unknown hepatocellular carcinoma not applicable YW3-56 Preclinical Actionable In a preclinical study, YW3-56 inhibited proliferation of a human hepatocellular carcinoma cell line in culture (PMID: 25612620). 25612620
Unknown unknown lung squamous cell carcinoma not applicable Cisplatin + Paclitaxel + Tislelizumab Phase II Actionable In a Phase II trial, treatment with Tislelizumab (BGB-A317) plus platinum doublet chemotherapy (Taxol (paclitaxel) with Platinol (cisplatin) or Paraplatin (carboplatin)) in patients with squamous non-small cell lung cancer resulted in an objective response rate of 80% (12/15) and disease control rate of 93% (14/15), including a partial response in 12 patients and stable disease in two patients, and median progression-free survival of 7.0 months (PMID: 32769013; NCT03432598). 32769013
Unknown unknown gastric adenocarcinoma not applicable Paclitaxel + Ridaforolimus Phase Ib/II Actionable In a Phase Ib trial, Ridaforolimus (MK-8669), in combination with paclitaxel, produced stable disease greater than or equal to 4 months in 67% (2/3) of gastric cancer patients (PMID: 19901013). 19901013
Unknown unknown colorectal cancer not applicable Adavosertib Preclinical - Cell line xenograft Actionable In a preclinical study, Adavosertib (MK-1775) inhibited cell proliferation and promoted DNA damage in a human colorectal cancer cell line in culture, and promoted tumor regression in xenograft models (PMID: 23699655). 23699655
Unknown unknown hepatocellular carcinoma not applicable Refametinib + Sorafenib Phase I Actionable In a Phase I trial, 43.8% (7/16) of hepatocellular carcinoma patients treated with a combination of Refametinib (BAY86-9766) and Nexavar (sorafenib) demonstrated stable disease (PMID: 26644411). 26644411
Unknown unknown 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 extraosseous Ewing sarcoma not applicable GSK1838705A Preclinical Actionable In a preclinical study, multiple cancer cell lines including multiple myeloma and Ewing's sarcoma were sensitive to GSK1838705A (PMID: 19825801). 19825801
Unknown unknown 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 chronic lymphocytic leukemia not applicable Lenalidomide + Ofatumumab Phase I Actionable In a Phase I trial, the combination treatment of Revlimid (lenalidomide) and Arzerra (ofatumumab) in patients with chronic lymphocytic leukemia resulted in an overall response rate of 71% (24/34), in which 24% (8/34) experienced a complete remission and 47% (16/34) experienced a partial response (PMID: 26733610). 26733610
Unknown unknown cholangiocarcinoma not applicable Sabatolimab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 treatment resulted in stable disease in 2 patients with cholangiocarcinoma (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown urinary bladder cancer not applicable Futibatinib Phase I Actionable In a Phase I trial, TAS-120 treatment resulted in stable disease over 24 weeks in a bladder cancer patient (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 372PD). detail...
Unknown unknown anaplastic large cell lymphoma not applicable Brentuximab vedotin FDA approved Actionable In a Phase II trial (SGN-35) that supported FDA approval, Adcetris (brentuximab vedotin) treatment resulted in complete remission in 57% (33/58), partial remission in 29% (17/58) of patients with relapsed or refractory systemic anaplastic large cell lymphoma (PMID: 22614995; NCT00866047). 22614995 detail...
Unknown unknown diffuse large B-cell lymphoma not applicable IT-901 Preclinical Actionable In a preclinical study, IT-901 inhibited growth of diffuse large B-cell lymphoma cells in culture (PMID: 26744524). 26744524
Unknown unknown 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 lung non-small cell carcinoma not applicable BMS-906024 Preclinical Actionable In a preclinical study, the pan-Notch inhibitor, BMS-906024, inhibited growth of non-small cell lung carcinoma xenografts (PMID: 26005526). 26005526
Unknown unknown multiple myeloma not applicable TNB-383B Preclinical - Cell line xenograft Actionable In a preclinical study, TNB-383B increased survival and reduced tumor burden in a cell-line xenograft model of multiple myeloma (Journal of Clinical Oncology 2018 36:15_suppl, 8034-8034). detail...
Unknown unknown diffuse large B-cell lymphoma not applicable Ibrutinib + PQR309 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination therapy of PQR309 and Imbruvica (ibrutinib) led to a synergistic effect in 6/7 diffuse large B-cell lymphoma (DLBCL) cell lines in culture, and the effect was confirmed in a DLBCL cell line xenograft model (PMID: 29066507). 29066507
Unknown unknown synovial sarcoma not applicable Rivoceranib Case Reports/Case Series Actionable In a retrospective analysis, Rivoceranib (apatinib) treatment demonstrated manageable safety profile, resulted in a partial response in 42.9% (9/21) and stable disease in 38.1% (8/21) of patients with advanced synovial sarcoma, with a median progression-free survival of 13.1 months and a median overall survival of 15.5 months (PMID: 32669874). 32669874
Unknown unknown Advanced Solid Tumor not applicable Tepotinib Phase I Actionable In a Phase I trial, treatment with Tepotinib (EMD-1214063) was well-tolerated, demonstrated safety, and resulted in some antitumor efficacy in Japanese patients with an advanced solid tumor including stable disease for 12 weeks or more in two patients, a gastric cancer patient with MET expression and another patient with urachal cancer (PMID: 32328660). 32328660
Unknown unknown uveal melanoma not applicable Mivebresib Phase I Actionable In a Phase I trial, Mivebresib (ABBV-075) treatment demonstrated safety and modest clinical activity in patients with uveal melanoma, resulting in stable disease as best response in 44% (4/9) of evaluable patients, patients achieved stable disease had lower cfDNA mutational load of pathogenic mutations (53.1 vs 893.9 median mutant molecules/mL plasma) compared to patients developed progressive disease (J Clin Oncol 37, no. 15_suppl; NCT02391480). detail...
Unknown unknown glioblastoma multiforme not applicable Olaparib + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, treatment with Lynparza (olaparib) enhanced the sensitivity of glioblastoma cell lines to radiation therapy in culture, demonstrating greater decreased cell survival when compared to cells not treated with Lynparza (olaparib) (PMID: 32347934). 32347934
Unknown unknown Advanced Solid Tumor not applicable Seliciclib Phase I Actionable In a Phase I trial, Seliciclib (R-roscovitine) demonstrated preliminary anti-tumor activity in patients with advanced solid tumors (PMID: 20822897). 20822897
Unknown unknown pancreatic adenocarcinoma not applicable Gemcitabine + Pimasertib Phase I Actionable In a Phase I trial, Pimasertib in combination with Gemzar (gemcitabine) demonstrated safety and efficacy in metastatic pancreatic adenocarcinoma patients (PMID: 23846936). 23846936
Unknown unknown chronic lymphocytic leukemia not applicable JSH-150 Preclinical - Cell culture Actionable In a preclinical study, JSH-150 induced cell-cycle arrest and inhibited proliferation of chronic lymphocytic leukemia cell lines in culture (PMID: 30253346). 30253346
Unknown unknown 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 neuroblastoma not applicable Cambinol + Doxorubicin Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Adriamycin (doxorubicin) and Cambinol inhibited growth of a human neuroblastoma cell line in culture and in xenograft models (PMID: 22703804). 22703804
Unknown unknown follicular lymphoma not applicable Bendamustine + Obinutuzumab FDA approved Actionable In a Phase III trial that supported FDA approval, the combination of Gazyva (obinutuzumab) plus Treanda (bendamustine) resulted in a greater progression free survival (29.2 mo vs 14.0 mo) than Treanda (bendamustine) alone in patients with follicular lymphoma (PMID: 27345636). 27345636 detail...
Unknown unknown colon adenocarcinoma not applicable XL147 Preclinical - Cell line xenograft Actionable In a preclinical study, XL147 (SAR245408) demonstrated modest efficacy as a single agent in human cancer cell line xenograft models (PMID: 23002019). 23002019
Unknown unknown pancreatic cancer not applicable Foretinib Preclinical Actionable In a preclinical study, Foretinib (GSK1363089) treatment resulted in regression of the tumor vasculature, extensive hypoxia, apoptosis, and decreased tumor aggressiveness in a transgenic mouse model of pancreatic islet cancer (PMID: 21613405). 21613405
Unknown unknown 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 renal cell carcinoma not applicable Sunitinib Phase III Actionable In a Phase III clinical trial, treatment with Sutent (sunitinib) resulted in prolonged disease free survival (HR = 0.761) compared to placebo in post-nephrectomy patients with renal cell carcinoma (ESMO 2016 Congress in Copenhagen, Abstract LBA11_PR). detail...
Unknown unknown renal cell carcinoma not applicable Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Sutent (sunitinib) resulted in improved median progression free survival (47.3 weeks) and objective response rate (27.5%) in patients with renal cell carcinoma (PMID: 19707433). 19707433 detail...
Unknown unknown clear cell renal cell carcinoma not applicable Bevacizumab + Vorinostat Phase Ib/II Actionable In a Phase I/II trial, Zolinza (vorinostat) and Avastin (bevacizumab) combination treatment resulted in complete response in 3% (1/33) and partial response in 15% (5/33) of patients with renal clear cell carcinoma, with median progression free survival and overall survival of 5.7 months and 13.9 months, respectively (PMID: 28222071). 28222071
Unknown unknown neuroblastoma not applicable Dinutuximab FDA approved Actionable In a Phase III trial supporting FDA approval, Unituxin (dinutuximab) in combination with Sargramostim, Aldesleukin, and 13-cis-retinoic acid resulted in improved median event-free survival (HR = 0.57) and overall survival (HR = 0.58) compared to control group in high-risk pediatric neuroblastoma patients (PMID: 26917818). detail... 26917818
Unknown unknown esophagus squamous cell carcinoma not applicable Fluorouracil + OBP-801 Preclinical Actionable In a preclinical study, OBP-801, in combination with 5-FU and radiation, enhanced growth inhibition of esophageal squamous carcinoma cells in culture (PMID: 24854104). 24854104
Unknown unknown 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 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 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 sarcoma not applicable Aldoxorubicin + Alvocidib Phase II Actionable In a Phase I trial, Alvocidib (flavopiridol), in combination with aldoxorubicin resulted in partial responses in 6% (2/31) and stable disease in 51% (16/31) of patients with advanced sarcoma (PMID: 22374332). 22374332
Unknown unknown 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 transitional cell carcinoma not applicable Domatinostat Preclinical - Cell culture Actionable In a preclinical study, Domatinostat (4SC-202) inhibited proliferation and induced cell-cycle alterations and cell death in urothelial cancer cell lines in culture (PMID: 27250763). 27250763
Unknown unknown endometrial cancer not applicable LY3023414 Phase II Actionable In a Phase II trial, patients with advanced endometrial cancer harboring a PI3K pathway mutation demonstrated a best overall response rate of 16% (4/25), a clinical benefit rate of 28% (7/25) at 12 weeks, a progression-free survival of 2.5 months, and overall survival of 9.2 months when treated with LY3023414 (PMID: 31880826; NCT01775072). 31880826
Unknown unknown prostate cancer not applicable Dactolisib + unspecified CTLA4 antibody + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, combination of myeloid-derived suppressor cell-targeting with BEZ235 and immune checkpoint blockade with anti-CTLA4 and anti-PD-1 antibodies resulted in synergistic inhibition of tumor growth and metastasis in transgenic mouse models of metastatic castration-resistant prostate cancer (PMID: 28321130). 28321130
Unknown unknown 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 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 chronic lymphocytic leukemia not applicable Cyclophosphamide + Fludarabine + Ofatumumab FDA approved Actionable In a Phase III trial supporting FDA approval, Arzerra (ofatumumab) in combination with fludarabine and cyclophosphamide (FC) resulted in improved progression free survival (28.9 vs 18.8 months) compared to FC treatment alone in patients with relapsed chronic lymphocytic leukemia (PMID: 27731748). 27731748 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 lung non-small cell carcinoma not applicable Regorafenib Phase I Actionable In a Phase I trial, Stivarga (regorafenib) treatment in patients with non-small cell lung cancer resulted in stable disease (SD) in 76% (13/17) of patients, and one patient with SD experienced a progression free survival of 279 days and tumor reduction greater than 30% (J Clin Oncol 28:15s, 2010 (suppl; abstr 7585)). detail...
Unknown unknown Advanced Solid Tumor not applicable Perifosine Phase I Actionable In a Phase I trial, Perifosine demonstrated safety and preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 25183650). 25183650
Unknown unknown head and neck squamous cell carcinoma not applicable Combretastatin A1 Diphosphate Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with OXi4503 resulted in increased tumor cell necrosis and decreased tumor growth and extended median survival in cell line xenograft models of head and neck squamous cell carcinoma (PMID: 26751478). 26751478
Unknown unknown germ cell cancer not applicable Pazopanib Phase II Actionable In a Phase II trial, Votrient (pazopanib) treatment resulted in partial responses in 4.7% (2/43), stable disease in 44.2% (19/43), and a 3-month progression free survival probability of 12.8% in patients with refractory germ cell cancer (PMID: 28383677). 28383677
Unknown unknown prostate cancer not applicable MC180295 Preclinical - Cell culture Actionable In a preclinical study, MC180295 decreased proliferation of prostate cancer cell lines in culture (PMID: 30454645). 30454645
Unknown unknown multiple myeloma not applicable Lenalidomide Phase III Actionable In a Phase III trial, Revlimid (lenalidomide) maintenance treatment after stem cell transplant resulted in improved overall survival and progression free survival in patients with multiple myeloma (J Clin Oncol 35, 2017 (suppl; abstr 8037)). detail...
Unknown unknown glioblastoma multiforme not applicable LYS6KAKT1 Preclinical Actionable In a preclinical study, LYS6KAKT1 inhibited phosphorylation of p70S6 kinase in mice engrafted with glioblastoma cells (Mol Cancer Ther 2011;10(11 Suppl):Abstract nr B117). detail...
Unknown unknown 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 multiple myeloma not applicable DT2216 + S63845 Preclinical - Cell culture Actionable In a preclinical study, the combination of DT2216 and S63845 demonstrated synergy in a myeloma cell line dependent on MCL-1 and BCL-XL for survival, resulting in decreased cell viability in culture (PMID: 31792461). 31792461
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 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 Advanced Solid Tumor not applicable OM-174 Phase I Actionable In a Phase I clinical trial, OM-174 demonstrated safety and limited preliminary efficacy as a monotherapy in patients advanced solid tumors, with 17% (3/17) of patients achieving stable disease (PMID: 23547558). 23547558
Unknown unknown acute lymphoblastic leukemia no benefit T-3775440 Preclinical - Cell culture Actionable In a preclinical study, an acute lymphocytic leukemia cell line did not respond to treatment with T-3775440 in culture, demonstrating minimal to no cell growth inhibition (PMID: 27903753). 27903753
Unknown unknown multiple myeloma not applicable MV-NIS Phase Ib/II Actionable In a Phase I/II trial, MV-NIS treatment resulted in enhanced T-cell response to tumor antigens in 40% (4/10) of patients with multiple myeloma (Journal of Clinical Oncology 36, no. 5_suppl (February 10 2018) 218-218; NCT00450814). detail...
Unknown unknown malignant glioma not applicable Bevacizumab + MEDI3617 Phase I Actionable In a Phase I trial, MEDI3617 in combination with Avastin (bevacizumab) resulted in no objective response (0/11) and stable disease in 18% (2/11) of patients with recurrent malignant glioma, with a median progression-free survival of 1.4 months (PMID: 29559563; NCT01248949). 29559563
Unknown unknown lymphoma not applicable Mivavotinib Phase I Actionable In a Phase I trial, Mivavotinib (TAK-659) treatment resulted in an objective response rate of 40% (27/67, 12 complete responses, 15 partial responses) in patients with relapsed or refractory diffuse large B-cell lymphoma, with a median duration of response of 856 days (PMID: 32327472; NCT02000934). 32327472
Unknown unknown liposarcoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 63%, median progression-free survival of 5.6 months, an objective response rate of 7.7% (n=13), and a median overall survival of 13 months in patients with liposarcoma (PMID: 29895706; NCT01878448). detail... 29895706
Unknown unknown chronic myeloid leukemia not applicable Busulfan + Cyclophosphamide FDA approved Actionable In a clinical trial that supported FDA approval, combination of Busulfex (busulfan) and Cytoxan (cyclophosphamide) as a conditioning regimen prior to bone marrow transplantation was better tolerated and resulted in favorable 4-year probabilities of survival and event-free survival (0.86 vs 0.72) compared to Cytoxan (cyclophosphamide) plus total body irradiation in patients with chronic myeloid leukemia (PMID: 8081005). 8081005 detail...
Unknown unknown lung squamous cell carcinoma not applicable CX-2029 Case Reports/Case Series Actionable In a Phase I/II trial (PROCLAIM-CX-2029), CX-2029 treatment resulted in partial response in a patient with squamous cell carcinoma of the lung (J Clin Oncol 38: 2020 (suppl; abstr 3502); NCT03543813). detail...
Unknown unknown 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 glioblastoma multiforme not applicable Bortezomib + Vorinostat Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Zolinza (vorinostat) and Velcade (bortezomib) synergized to decrease cell viability, DNA fragmentation and elevate caspase 9 activity in several human glioblastoma cell lines in culture and in xenograft models of one human glioblastoma cell line (PMID: 26804704). 26804704
Unknown unknown pancreatic cancer not applicable PF-00562271 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with PF-00562271 inhibited PTK2 (FAK) phosphorylation and resulted in apoptosis and tumor regression in pancreatic cancer cell line xenograft models (PMID: 18339875). 18339875
Unknown unknown Advanced Solid Tumor not applicable BMS-986156 Phase Ib/II Actionable In a Phase I/IIa trial, patients with advanced solid tumors treated with a combination of Opdivo (nivolumab) and BMS-986156 demonstrated preliminary antitumor activity (Journal of Clinical Oncology 35, no. 15_suppl (May 2017) 104-104; NCT02598960). detail...
Unknown unknown non-Hodgkin lymphoma not applicable Cyclophosphamide + Inotuzumab ozogamicin + Prednisone + Rituximab + Vincristine Sulfate Phase I Actionable In a Phase I trial, the combination of inotuzumab ozogamicin with R-CVP (rituximab, cyclophosphamide, Oncovin (vincristine), and prednisone) resulted in an ORR of 84% (32/38) in non-Hodgkin lymphoma patients, including a complete response in 24% (9/38) of patients (PMID: 27154915). 27154915
Unknown unknown 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 AXL1717 Phase Ib/II Actionable In a Phase 1b/II trial, AXL1717 (picropodophyllin) was well tolerated and 25% (4/16) of patients diagnosed with NSCLC demonstrated a partial tumor response (PMID: 26161618). 26161618
Unknown unknown head and neck squamous cell carcinoma not applicable Lenvatinib + Pembrolizumab Phase Ib/II Actionable Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 46% (10/22) in patients with metastatic head and neck squamous cell carcinoma, with a median duration of response of 8.2 months, and a median progression-free survival of 4.7 months (PMID: 31961766; NCT02501096). 31961766
Unknown unknown hematologic cancer not applicable AZD5991 Preclinical - Cell line xenograft Actionable In a preclinical study, AZD5991 inhibited tumor growth in hematologic cancer cell line xenograft models (Cancer Res July 1 2017 (77) (13 Supplement) DDT01-02). detail...
Unknown unknown B-cell lymphoma not applicable IT-901 Preclinical Actionable In a preclinical study, IT-901 inhibited tumor growth in Epstein Barr Virus-induced B-cell lymphoma xenograft models (PMID: 26744524). 26744524
Unknown unknown gastrointestinal system cancer no benefit Cetuximab + Cisplatin + Irinotecan Phase II Actionable In a Phase II trial, Erbitux (cetuximab) in combination with IC (irinotecan, cisplatin) demonstrated less efficacy and more toxicity, resulted in an overall response rate of 45.0% (32/71), median overall survival of 8.6 months; and median progression-free survival of 4.9 months in patients with metastatic esophageal or gastroesophageal junction cancers (PMID: 27382098). 27382098
Unknown unknown osteosarcoma not applicable Gemcitabine + Sirolimus Phase II Actionable In a Phase II trial, the combination of Gemzar (gemcitabine) and Rapamune (sirolimus) resulted in a progression-free survival rate of 44% after 4 months in osteosarcoma patients, including a partial response in two patients and stable disease in fourteen patients (PMID: 29045512). 29045512
Unknown unknown prostate cancer not applicable Abiraterone + Onvansertib Preclinical - Cell line xenograft Actionable In a preclinical study, Zytiga (abiraterone) and Onvansertib (PCM-075) synergistically inhibited growth of prostate cancer cells in cell line xenograft models (J Clin Oncol 36, 2018 (suppl 6S; abstr 369)). detail...
Unknown unknown multiple myeloma not applicable Elotuzumab + GDA-201 Phase I Actionable In a Phase I trial, GDA-201 and Empliciti (elotuzumab) combination therapy demonstrated safety and preliminary efficacy, resulted in complete response in 7.7% (1/13) and stable disease in 30.8% (4/13) of patients with refractory multiple myeloma, with a median duration of response of 2.5 months (Blood (2019) 134 (Supplement_1): 777). detail...
Unknown unknown 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 melanoma not applicable AS1409 Phase I Actionable In a Phase I trial, AS1409 treatment in patients with either melanoma or renal cell carcinoma resulted in stable disease in 46% (6/13) of patients and in two patients with melanoma, one demonstrated a partial response while another showed tumor shrinkage, which lasted beyond 12 months (PMID: 21447719). 21447719
Unknown unknown 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 stomach cancer not applicable AT13148 Preclinical Actionable In a preclinical study, AT13148 inhibited proliferation and induced apoptosis in gastric cancer cell lines in culture and inhibited tumor growth in a gastric cancer cell line xenograft model (PMID: 26828267). 26828267
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 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 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 oral squamous cell carcinoma not applicable Everolimus Preclinical Actionable In a preclinical study, Afinitor (everolimus) inhibited growth and mTOR signaling in oral squamous cell carcinoma cell lines (PMID: 25682238). 25682238
Unknown unknown epithelioid sarcoma not applicable Abexinostat Preclinical - Cell line xenograft Actionable In a preclinical study, Abexinostat (PCI-24781) inhibited growth and colony formation of epithelioid sarcoma cell lines in culture, and inhibited tumor growth in epithelioid sarcoma cell line xenograft models (PMID: 26396249). 26396249
Unknown unknown cervix carcinoma not applicable Bevacizumab + Cisplatin + Paclitaxel FDA approved Actionable In a Phase III trial that supported FDA approval, the addition of Avastin (bevacizumab) to Platinol (cisplatin) and Taxol (paclitaxel) chemotherapy resulted in improved overall survival and progression-free survival compared to chemotherapy alone in patients with cervical cancer (PMID: 25281440, PMID: 24552320). detail... 25281440 24552320
Unknown unknown 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 Atezolizumab + Bevacizumab FDA approved Actionable In a Phase III trial (IMbrave150) that supported FDA approval, Tecentriq (atezolizumab) and Avastin (bevacizumab) combination therapy improved survival compared to Nexavar (sorafenib) (HR for death=0.58, p<0.001) in patients with unresectable hepatocellular carcinoma, with superior overall survival at 12 months (67.2% vs 54.6%) and prolonged median progression-free survival (6.8 vs 4.3 months, HR=0.59, p<0.001) (PMID: 32402160; NCT03434379). detail... 32402160
Unknown unknown Advanced Solid Tumor not applicable Refametinib Phase I Actionable In a Phase I trial, Refametinib (BAY 86-9766) was well-tolerated and displayed some evidence of clinical benefit across several tumor types (PMID: 23434733). 23434733
Unknown unknown urinary bladder cancer not applicable Etomoxir Preclinical - Cell line xenograft Actionable In a preclinical study, Etomoxir treatment induced cell cycle arrest, and reduced cell migration, proliferation, and viability of bladder cancer cell lines in culture, and inhibited tumor growth in a cell line xenograft model (PMID: 31358595). 31358595
Unknown unknown colorectal cancer not applicable Conatumumab + Ganitumab Phase Ib/II Actionable In a Phase Ib/II clinical trial, Ganitumab and Conatumumab combination treatment resulted in one unconfirmed partial response and stable disease in 38% (6/16) of patients with colorectal cancer (PMID: 24816908). 24816908
Unknown unknown multiple myeloma not applicable JNJ-68284528 Phase I Actionable In a Phase Ib trial (CARTITUDE-1), JNJ-68284528 treatment demonstrated manageable toxicity profile, resulted in an objective response rate of 100% (29/29, 22 stringent complete response, 6 very good partial response, 1 partial response) in patients with relapsed or refractory multiple myeloma, with a 6-month progression-free rate of 93% (27/29) (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 8505-8505; NCT03548207). detail...
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 colon cancer not applicable MPT0E028 Preclinical - Cell line xenograft Actionable In a preclinical study, MPT0E028 decreased proliferation and increased apoptosis in a colon cancer cell line in culture and inhibited tumor growth in a colon cancer cell line xenograft model (PMID: 22928010). 22928010
Unknown unknown chronic myeloid leukemia not applicable Bosutinib Clinical Study Actionable In a meta-analysis, Bosulif (bosutinib) treatment was associated with increased rate of major molecular response compared with Gleevec (imatinib) (Odds Ratio (OR): 1.86 [1.29-2.70]), but not improved overall survival (OR: 2.38 [0.82-6.89]), and was associated with a trend toward increased risk of vascular occlusive events in chronic myeloid leukemia patients (PMID: 26847662). 26847662
Unknown unknown acute lymphoblastic leukemia not applicable Natalizumab Preclinical - Pdx Actionable In a preclinical study, Tysabri (natalizumab) treatment alone or in combination with chemotherapy prolonged survival in patient-derived xenograft models of acute lymphocytic leukemia (PMID: 23319569). 23319569
Unknown unknown ovarian cancer not applicable Cediranib + Durvalumab + Olaparib Phase I Actionable In a Phase I trial, the combination of Cediranib (AZD-2171), Imfinzi (durvalumab), and Lynparza (olaparib) treatment demonstrated tolerability and activity in female patients with ovarian, endometrial, or triple-negative breast cancer, with a response rate of 33% (3/9; 2 pts with ovarian cancer, and 1 pt with endometrial cancer), and stable disease in 4/9 pts (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #390P; NCT02484404). detail...
Unknown unknown thyroid gland cancer not applicable Bevacizumab Preclinical Actionable In a preclinical study, Avastin (bevacizumab) inhibited tumor growth and angiogenesis in mouse models of anaplastic thyroid cancer (PMID: 17429874). 17429874
Unknown unknown 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 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 gastroesophageal junction adenocarcinoma not applicable Erlotinib + Glesatinib Phase I Actionable In a Phase I trial, 38% (3/8) of patients with gastroesophageal cancer remained on study for approximately 12-26 cycles with Glesatinib (MGCD265) and Tarceva (erlotinib) combination therapy (J Clin Oncol 30, 2012 (suppl; abstr e13602)). detail...
Unknown unknown Advanced Solid Tumor not applicable MGD013 Phase I Actionable In a Phase I trial, MGD013 treatment resulted in a partial response in 7.3% (3/41) and stable disease in 51.2% (21/41) of response-evaluable patients with advanced solid tumors in the dose escalation cohort (J Clin Oncol 38: 2020 (suppl; abstr 3004); NCT03219268). detail...
Unknown unknown myelodysplastic syndrome not applicable Decitabine FDA approved Actionable In a Phase III trial that supported FDA approval, Dacogen (decitabine) treatment resulted in an overall response rate of 17% (15/89), with complete responses in 9% (8/89) and partial responses in 8% (7/9) of patients with myelodysplastic syndromes (PMID: 16532500). 16532500 detail...
Unknown unknown ovary epithelial cancer not applicable Bevacizumab + Niraparib Phase I Actionable In a Phase I trial, Avastin (bevacizumab) and Zejula (niraparib) combination treatment resulted in complete response in 8% (1/12), partial response in 33% (4/12), and a disease control rate of 91% in platinum-sensitive ovarian cancer patients (J Clin Oncol 34, 2016 (suppl; abstr 5555)). detail...
Unknown unknown glioblastoma multiforme not applicable GSK1838705A Preclinical - Cell line xenograft Actionable In a preclinical study, GSK1838705A inhibited cell growth and prevented tumor growth via apoptotic induction in glioma cells in both culture and xenograft models (PMID: 26238593). 26238593
Unknown unknown acute lymphoblastic leukemia not applicable AS605240 + Dexamethasone Preclinical - Pdx Actionable In a preclinical study, the combination of AS605240 and dexamethasone improved survival of primary T-ALL cell xenograft models (PMID: 25869207). 25869207
Unknown unknown medulloblastoma not applicable M344 Preclinical - Cell culture Actionable In a preclinical study, M344 induced apoptosis and inhibited proliferation of medulloblastoma cell lines in culture (PMID: 17230517). 17230517
Unknown unknown gastroesophageal junction 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 gastrointestinal stromal tumor not applicable Rivoceranib Phase I Actionable In a Phase I trial, Apatinib (YN968D1) produced a partial response in 18.9% (7/37) and stable disease in 64.9% (24/37) of patients with advanced solid tumors, including partial response in one patient with GIST (PMID: 20923544). 20923544
Unknown unknown diffuse large B-cell lymphoma not applicable Ibrutinib + SEL24-B489 Preclinical Actionable In a preclinical study, SEL24-B489 demonstrated a synergistic effect when combined with Ibruvica (ibrutinib) in diffuse large B-cell lymphoma cells in culture, resulting in cell growth inhibition ((Blood 126 (23):706.December 2015). detail...
Unknown unknown Advanced Solid Tumor not applicable NP-004255 Preclinical - Cell culture Emerging In a preclinical study, a biochemical assay with NP-004255, a RAD52 inhibitor similar to other compounds that induced cell death in cells with BRCA2 loss, demonstrated inhibition of RAD52 binding to ssDNA, suggesting NP-004255 may be a potential therapeutic target (PMID: 27434671). 27434671
Unknown unknown Advanced Solid Tumor not applicable Seribantumab + XL147 Phase Ib/II Actionable In a Phase Ib trial, treatment with the combination of Pilaralisib (SAR245408, XL147) and Seribantumab (SAR256212) resulted in stable disease as best response in 52.2% (12/23) patients with advanced solid tumors, with no difference in response between patients harboring PIK3CA mutations and those with wild-type PIK3CA (PMID: 28031425). 28031425
Unknown unknown malignant peripheral nerve sheath tumor 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 malignant peripheral nerve sheath tumor (PMID: 30724423). 30724423
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 breast cancer not applicable Avelumab + PF-06840003 Preclinical - Cell line xenograft Actionable In a preclinical study, PF-06840003 and Bavencio (avelumab) combination treatment inhibited tumor growth in a cell line xenograft model of breast cancer engrafted with human CD34-positive cells (PMID: 30232146). 30232146
Unknown unknown diffuse large B-cell lymphoma not applicable Ibrutinib Phase II Actionable In a Phase II trial, Imbruvica (ibrutinib) treatment demonstrated preferential efficacy in the ABC subtype of diffuse large B-cell lymphoma (DLBCL) compared to the GCB subtype, resulted in complete response in 8% (2/25) and partial response in 32% (8/25) of ABC DLBCL patients, but only partial response in 5.3% (1/19) of GCB DLBCL patients (Blood 2012, 120 (21): 686). detail...
Unknown unknown multiple myeloma not applicable 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 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 osteosarcoma not applicable VCN-01 Preclinical Actionable In a preclinical study, treatment with VCN-01 resulted in decreased viability of osteosarcoma cell lines in culture (PMID: 26603261). 26603261
Unknown unknown head and neck squamous cell carcinoma not applicable Cetuximab + Dasatinib Phase II Actionable In a Phase II trial, Sprycel (dasatinib) and Erbitux (cetuximab) combination treatment resulted in stable disease in 46% (6/13) and disease progression in 54% (7/13) of patients with cetuximab-resistant head and neck squamous cell carcinoma, with low serum IL6 level correlated with stable disease (PMID: 28559019). 28559019
Unknown unknown diffuse large B-cell lymphoma not applicable ST7612AA1 Preclinical Actionable In a preclinical study, ST7612AA1 inhibited proliferation of diffuse large B-cell lymphoma cell lines in culture (PMID: 25671299). 25671299
Unknown unknown hepatoblastoma not applicable UAB30 Preclinical - Cell line xenograft Actionable In a preclinical study, a human hepatoblastoma cell line was sensitive to UAB30 in both culture and in xenograft models, demonstrating cell-cycle arrest, decreased cell proliferation, and reduced tumor growth (PMID: 26873726). 26873726
Unknown unknown breast cancer not applicable TAK-960 Preclinical - Cell line xenograft Actionable In a preclinical study, breast cancer cells treated with TAK-960 demonstrated a decrease in tumor size in cell line xenograft models (PMID: 22188812). 22188812
Unknown unknown 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 GDC-0919 Phase I Actionable In a Phase I trial, GDC-0919 (Navoximod) monotherapy was well tolerated, resulted in stable disease as best response in 50% (5/10) of patients with advanced solid tumors (PMID: 31124055). 31124055
Unknown unknown triple-receptor negative breast cancer not applicable Niraparib + SY-1365 Preclinical - Cell culture Actionable In a preclinical study, SY-1365 and Zejula (niraparib) synergistically induced apoptosis in triple-receptor negative breast cancer cells in culture (Proceedings of the AACR, Vol 58, April 2017, Abstract # 1151). detail...
Unknown unknown thyroid gland cancer not applicable ABT-737 + Cisplatin Preclinical Actionable In a preclinical study, suboptimal dosing of ABT-737 and Platinol (cisplatin) did not affect cell viability of human thyroid cancer cell lines in culture (PMID: 27042160). 27042160
Unknown unknown lung carcinoma not applicable MRx0518 Preclinical Actionable In a preclinical study, MRx0518 stimulated immune response and reduced tumor size in syngeneic mouse models of lung carcinoma (Journal of Clinical Oncology 36, no. 15_suppl). detail...
Unknown unknown breast cancer not applicable AsiDNA + Rucaparib Preclinical - Cell culture Actionable In a preclinical study, AsiDNA and Rubraca (rucaparib) combination treatment resulted in increased cell death and inhibition of proliferation in breast cancer cell lines compared to Rubraca (rucaparib) alone in culture (PMID: 27559053). 27559053
Unknown unknown 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 lung carcinoma not applicable Fostamatinib Preclinical - Cell culture Actionable In a preclinical study, Fostamatinib (R788) treatment induced cytotoxicity in Lewis lung carcinoma cells in culture and inhibited tumor growth in a syngeneic mouse model (PMID: 31974273). 31974273
Unknown unknown colorectal cancer not applicable TAK-960 Preclinical - Cell line xenograft Actionable In a preclinical study, colorectal cancer cells treated with TAK-960 demonstrated cell growth inhibition and decreased tumor size in both culture and cell line xenograft models (PMID: 22188812). 22188812
Unknown unknown 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 triple-receptor negative breast cancer not applicable BOS172722 Preclinical - Cell line xenograft Actionable In a preclinical study, triple-negative breast cancer (TNBC) cell lines demonstrated increased sensitivity to BOS172722 compared to non-triple-negative breast cancer cell lines in culture, and BOS172722 treatment resulted in moderate tumor growth inhibition in a TNBC cell line xenograft model (PMID: 31575759). 31575759
Unknown unknown gastrointestinal stromal tumor not applicable Imatinib Clinical Study Actionable In a retrospective study of 16 Phase I trials, treatment with kinase inhibitors including Gleevec (imatinib mesylate), Sutent (sunitinib), or Stivarga (regorafenib) resulted in stable disease in 47.6% (10/21) and partial response in 19% (4/21) of patients with gastrointestinal stromal tumors (PMID: 27842521). 27842521
Unknown unknown colon cancer not applicable FN-1501 Preclinical - Cell culture Actionable In a preclinical study, FN-1501 induced cell-cycle arrest decreased proliferation in a colon cancer cell line in culture (PMID: 29357250). 29357250
Unknown unknown Advanced Solid Tumor not applicable Pimasertib Phase I Actionable In a Phase I trial, Pimasertib (MSC1936369B) demonstrated safety and some preliminary anti-tumor activity in patients with advanced solid tumors (J Clin Oncol 28:15s, 2010 (suppl; abstr 2504)). detail...
Unknown unknown cholangiocarcinoma not applicable Cabozantinib Phase II Actionable In a Phase II trial, Cometriq (cabozantinib) treatment resulted in a median progression free survival of 1.8 months, and a median overall survival of 5.2 months in patients with advanced cholangiocarcinoma, but also induced grade 3/4 adverse events in 89% (17/19) of the patients (PMID: 28192597). 28192597
Unknown unknown transitional cell carcinoma not applicable Pembrolizumab FDA approved Actionable In a Phase III (KEYNOTE-045) trial that supported FDA approval, treatment with Keytruda (pembrolizumab) resulted in improved median overall survival (10.3 vs 7.4 months, p=0.002) and objective response rate (21.1% vs 11.4%) compared to chemotherapy in patients with platinum-refractory advanced urothelial carcinoma (PMID: 28212060; NCT02256436). detail... 28212060
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 gastrointestinal stromal tumor not applicable Pimitespib Phase I Actionable In a Phase I trial, TAS-116 treatment resulted in partial response in a patient with gastrointestinal stromal tumor (J Clin Oncol 35, 2017 (suppl; abstr 2546)). detail...
Unknown unknown gastrointestinal stromal tumor not applicable Pimitespib Phase II Actionable In a Phase II trial, treatment with Pimitespib (TAS-116) in patients with a gastrointestinal stromal tumor who failed previous therapy (n=40) resulted in a progression-free survival of 4.4 months, a median overall survival of 11.5 months, a 0% objective response rate, and a disease control rate of 85%, with 34 patients achieving stable disease, and one patient experienced a 37.2% decrease in tumor size after the analysis cut-off date (PMID: 31536852). 31536852
Unknown unknown Ewing sarcoma not applicable Talazoparib Phase I Actionable In a Phase I trial, Talazoparib (BMN-673) treatment in patients with Ewing sarcoma did not result in any objective responses, however, resulted in a clinical benefit rate of 23% (PMID: 28242752). 28242752
Unknown unknown endometrial clear cell adenocarcinoma not applicable ONC201 Clinical Study Actionable In a clinical case study, a patient with clear cell endometrial cancer treated with ONC201 (TIC-10) demonstrated some reduction in lesion size (>30%), but also developed new lesions (PMID: 28331050). 28331050
Unknown unknown hairy cell leukemia not applicable Fenretinide Phase I Actionable In a Phase I trial, Fenretinide treatment resulted in stable disease in a patient with hairy cell leukemia (PMID: 28420721). 28420721
Unknown unknown acute myeloid leukemia not applicable MK-8242 Phase I Actionable In a Phase I trial, MK-8242 demonstrated safety and some preliminary efficacy in patients with refractory or recurrent acute myeloid leukemia, with 3 out of 24 evaluable patients demonstrating an objective response (PMID: 27544076). 27544076
Unknown unknown 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 hepatocellular carcinoma not applicable FH535 Preclinical Actionable In a preclinical study, FH535 demonstrated efficacy by inhibiting proliferation of liver cancer stem cells and hepatocellular carcinoma cells in culture (PMID: 24940873). 24940873
Unknown unknown lung non-small cell carcinoma not applicable Cediranib + Cisplatin + Gemcitabine Phase I Actionable In a Phase I trial, the combination of Cediranib with Gemzar (gemcitabine) and Platinol (cisplatin) demonstrated preliminary efficacy in patients with advanced non-small cell lung cancer (PMID: 19091548). 19091548
Unknown unknown lung small cell carcinoma not applicable AZD2811 Case Reports/Case Series Actionable In a Phase II trial, a patient with small cell lung cancer demonstrated stable disease, with a duration of 3.7 months, when treated with AZD2811 (PMID: 32584426; NCT02688894). 32584426
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 glioblastoma multiforme not applicable PRX177561 + Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of PRX177561 and Sutent (sunitinib) decreased tumor growth and improved time-to-progression, disease-free survival, and overall survival over either agent alone in glioblastoma cell line xenograft models (PMID: 28057017). 28057017
Unknown unknown 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 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 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 Advanced Solid Tumor not applicable VS-5584 Preclinical - Cell line xenograft Actionable In a preclinical study, VS-5584 inhibited growth of a variety of human tumor cell lines in culture and inhibited tumor growth in cell line xenograft models (PMID: 23270925). 23270925
Unknown unknown Advanced Solid Tumor not applicable Derazantinib Phase I Actionable In a Phase I trial, Derazantinib (ARQ 087) treatment resulted in partial response in 4.5% (3/67) and stable disease in 38.8% (26/67) of patients with advanced solid tumors (PMID: 28972963; NCT01752920). 28972963
Unknown unknown Advanced Solid Tumor not applicable TVB-2640 Phase I Actionable In a Phase I trial, TVB-2640 demonstrated bioavailability and inhibited FASN-dependent signaling in the tumor tissue of one patient with advanced solid tumor (Cancer Res August 1, 2015 75; 2675). detail...
Unknown unknown pancreatic adenocarcinoma no benefit Everolimus + Ribociclib Phase I Actionable In a Phase I trial, treatment with the combination of Afinitor (everolimus) and Kisqali (ribociclib) did not lead to a clinical benefit in pancreatic adenocarcinoma patients who previously progressed on chemotherapy (n=11), resulting in a median progression-free survival of 1.8 months, a median overall survival of 3.7 months, stable disease in two patients for 8 weeks, and progressive disease in nine patients (PMID: 32642630; NCT02985125). 32642630
Unknown unknown Advanced Solid Tumor not applicable Zenocutuzumab Phase Ib/II Actionable In a Phase I/II trial, Zenocutuzumab (MCLA-128) demonstrated safety and preliminary efficacy, resulted in partial response in 3.6% (1/28) and stable disease in 7.1% (2/28) of patients with advanced solid tumors (Cancer Res 2016;76(14 Suppl):Abstract nr CT050). detail...
Unknown unknown ovarian cancer not applicable Buparlisib + Trametinib Phase Ib/II Actionable In a Phase Ib trial, ovarian cancer patients demonstrated an objective response rate of 21% (6/21), including 1 complete response and 5 partial responses, to treatment with Buparlisib (BKM120) in combination with Mekinist (trametinib) (PMID: 25500057) 25500057
Unknown unknown follicular lymphoma not applicable Buparlisib + Ibrutinib Phase Ib/II Actionable In a Phase I/II trial, Buparlisib (BKM120) and Imbruvica (ibrutinib) combination treatment resulted in a best overall response rate of 20% (1/5, 1 complete response) in patients with relapsed/refractory follicular lymphoma (J Clin Oncol 36, 2018 (suppl; abstr 7520); NCT02756247). detail...
Unknown unknown 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 Advanced Solid Tumor not applicable 4SC-205 Phase I Actionable In a Phase I trial, 4SC-205 treatment demonstrated safety and promising long-term disease stabilization in patients with advanced solid tumors, resulted in a median time on study of 162 days in patients receiving continuous dosing (J Clin Oncol (Meeting Abstracts) May 2015 vol. 33 no. 15_suppl 2528). detail...
Unknown unknown glioblastoma multiforme not applicable Alisertib + MP7 Preclinical - Cell culture Actionable In a preclinical study, the combination of MP7 and Alisertib (MLN8237) resulted in an additive effect in glioblastoma cells in culture, demonstrating an increase in antiproliferative activity, decreased cell viability, and inhibition of glioma stem cell colony formation (PMID: 27797168). 27797168
Unknown unknown colon cancer not applicable AR-42 Preclinical - Cell line xenograft Actionable In a preclinical study, AR-42 induced cell death in primary human colon cancer cells and cell lines in culture, and inhibited tumor growth in colon cancer cell line xenograft models (PMID: 26026677). 26026677
Unknown unknown acute myeloid leukemia not applicable Azacitidine + Hu5F9-G4 Phase I Actionable In a Phase Ib trial, Magrolimab (Hu5F9-G4) and Vidaza (azacitidine) combination therapy was well tolerated, and resulted in complete response in 40% (10/25), complete response with incomplete hematologic recovery in 16% (4/25), partial response in 4% (1/25), morphologic leukemia-free state in 4% (1/25), and stable disease in 32% (8/25) of patients with acute myeloid leukemia unfit for chemotherapy (J Clin Oncol 38: 2020 (suppl; abstr 7507); NCT03248479). detail...
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 Balsalazide + Parthenolide Preclinical - Cell culture Actionable In a preclinical study, the combination of Balsalazide and Parthenolide resulted in a synergistic effect, resulting in reduced cell viability and increased apoptotic activity in colorectal cancer cells in culture (PMID: 28108625). 28108625
Unknown unknown 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 prostate cancer not applicable ABBV-744 Preclinical - Cell line xenograft Actionable In a preclinical study, ABBV-744 inhibited growth of prostate cancer cells in culture and in cell line xenograft models (Cancer Res 2018;78(13 Suppl):Abstract nr DDT01-05). detail...
Unknown unknown acute lymphoblastic leukemia not applicable Prexasertib Preclinical - Cell culture Actionable In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including acute lymphoblastic leukemia cell lines (PMID: 28270495). 28270495
Unknown unknown multiple myeloma not applicable ABT-348 Preclinical - Cell line xenograft Actionable In a preclinical study, Ilorasertib (ABT-348) displayed efficacy in multiple myeloma cell line xenograft models (PMID: 22935731). 22935731
Unknown unknown ovary epithelial cancer no benefit Dalantercept Phase II Actionable In a Phase II trial, Dalantercept (ACE-041) demonstrated safety, but did not result in objective response in patients with persistent or recurrent epithelial ovarian cancer, with stable disease as best response in 36.7% (11/30) of patients, a median progression-free survival of 1.7 months, and a median overall survival of 19.8 months (PMID: 30041929). 30041929
Unknown unknown lung cancer not applicable Metformin Preclinical - Cell culture Actionable In a preclinical study, Glucophage (metformin) inhibited proliferation of several lung cancer cell lines in culture, including squamous, adenocarcinoma, large cell, and small celll lung cancer cell lines (PMID: 22576795). 22576795
Unknown unknown head and neck squamous cell carcinoma not applicable Cetuximab + Pazopanib Phase Ib/II Actionable In a Phase Ib clinical trial, combined therapy, Votrient (pazopanib) and Erbitux (cetuximab), was well tolerated in head and neck squamous cell carcinoma patients with metastatic or resistant disease, and resulted in a 6% (2/31) complete response rate, a 29% (9/31) partial response rate, a median time to progression of 5.5 months, and a median overall survival of 9.5 months (PMID: 30001987; NCT01716416). 30001987
Unknown unknown prostate cancer not applicable Abiraterone + Prednisone FDA approved Actionable In a Phase III trial (LATITUDE) that supported FDA approval, treatment with the combination of Zytiga (abiraterone) and Predisone, along with androgen-deprivation therapy, resulted in improved median overall survival (not reached vs. 34.7 months; HR=0.62) and median progression-free survival (33.0 months vs. 14.8 months; HR=0.47) compared to treatment with placebos in patients with metastatic castration-sensitive prostate cancer (PMID: 28578607; NCT01715285). detail... 28578607
Unknown unknown Advanced Solid Tumor not applicable ALM201 Phase I Actionable In a Phase I trial, ALM201 demonstrated safety and acceptable pharmacokinetics in patients with advanced solid tumors, and 2 of 18 evaluable patients demonstrated stable disease for up to 6 cycles (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #383P). detail...
Unknown unknown 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 lung small cell carcinoma not applicable Carboplatin + STA-8666 Preclinical - Cell line xenograft Actionable In a preclinical study, STA-8666 enhanced antitumor activity when combined with Paraplatin (carboplatin), resulting in stabilization of tumor growth and eventual tumor regression in small cell lung cancer cell line xenograft models (PMID: 27267850). 27267850
Unknown unknown Advanced Solid Tumor not applicable OSI-027 Phase I Actionable In a Phase I trial, OSI-027 treatment resulted in no RECIST response and stable disease in 5% (6/128) of patients with advanced solid tumors (PMID: 27002938). 27002938
Unknown unknown B-cell lymphoma not applicable JCAR017 Phase I Actionable In a Phase I trial, JCAR017 treatment resulted in a response rate of 80% (22/28) with a complete response rate of 60% (17/28) in patients with relapsed/refractory B-cell non-Hodgkin lymphoma (J Clin Oncol 35, 2017 (suppl; abstr 7513); NCT02631044). detail...
Unknown unknown melanoma no benefit Alvocidib Phase II Actionable In a Phase II trial, Alvocidib (flavopiridol) treatment resulted in stable disease in 44% (7/16) of patients with melanoma, but no objective response (PMID: 15122079). 15122079
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 lung cancer not applicable ABT-737 + Perifosine Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of ABT-737 and perifosine worked synergistically to induce apoptosis and inhibit growth of lung cancer cell lines and primary lung cancer cells in culture, and to inhibit tumor growth in a lung cancer cell line xenograft model (PMID: 27073162). 27073162
Unknown unknown leiomyosarcoma not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) treatment resulted in twelve-week progression free survival in 44% (4/9) of leiomyosarcoma patients (PMID: 27502708). 27502708
Unknown unknown pancreatic cancer not applicable Pamiparib + Tislelizumab Case Reports/Case Series Actionable In a Phase Ib trial, the combination therapy of of Tislelizumab (BGB-A317) and Pamiparib (BGB-290) in patients with pancreatic cancer resulted in one partial response and stable disease for greater than 6 months in two patients (J Clin Oncol 35, 2017 (suppl; abstr 3013)). detail...
Unknown unknown ovarian cancer not applicable Navicixizumab Case Reports/Case Series Actionable In a Phase I trial, Navicixizumab (OMP-305B83) treatment resulted in partial response in 3 patients with advanced ovarian cancer, and 7 of the 11 ovarian cancer patients had a reduction of target lesions (PMID: 30229512). 30229512
Unknown unknown renal cell carcinoma not applicable Axitinib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Inlyta (axitinib) as second-line therapy resulted in an improved progression-free survival of 8.3 months compared to 5.7 months with Nexavar (sorafenib) (HR 0.656, 95% CI 0.552-0.779; p<0.0001) in patients with metastatic renal cell carcinoma (PMID: 23598172). 23598172 detail...
Unknown unknown renal cell carcinoma not applicable Axitinib Phase II Actionable In a Phase II clinical trial, treatment with Inlyta (axtinib) as first-line therapy resulted a prolonged median overall survival of 42.7 months compared to 30.4 months with placebo in patients with metastatic renal cell carcinoma (PMID: 27236772). 27236772
Unknown unknown renal cell carcinoma not applicable Axitinib Clinical Study Actionable In a meta-analysis, Inlyta (axitinib) improved progression-free survival in patients with metastatic renal cell carcinoma (PMID: 24037486). 24037486
Unknown unknown prostate cancer not applicable Capivasertib + Docetaxel + Prednisone Phase I Actionable In a Phase I trial, AZZD5363 in combination with Taxotere (docetaxel) and Prednisone resulted in more than 50% PSA reduction at 12 weeks in 70% (7/10) of patients with metastatic castration resistant prostate cancer (PMID: 28144789; NCT02121639). 28144789
Unknown unknown Advanced Solid Tumor not applicable Icrucumab Phase I Actionable In a Phase I trial, Icrucumab (IMC-18F1) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 23903897). 23903897
Unknown unknown esophagus squamous cell carcinoma not applicable Nivolumab FDA approved Actionable In a Phase III trial (ATTRACTION-3) that supported FDA approval, Opdivo (nivolumab) treatment demonstrated favorable safety profile and significantly improved median overall survival (10.9 vs 8.4 mo, HR=0.77, p=0.019) compared to chemotherapy in patients with advanced esophageal squamous cell carcinoma refractory or intolerant to prior chemotherapy (PMID: 31582355; NCT02569242). 31582355 detail...
Unknown unknown multiple myeloma not applicable Bortezomib + Mivebresib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Velcade (bortezomib) and Mivebresib (ABBV-075) resulted in increased tumor growth inhibition in a multiple myeloma cell line xenograft model compared to Velcade (bortezomib) alone (PMID: 28416490). 28416490
Unknown unknown 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 endometrial cancer not applicable Cediranib Phase II Actionable In a Phase II clinical trial, treatment with Cediranib (AZD-2171) resulted in an overall response rate of 12.5% (6/48, all partial responses), stable disease in 37.5% (18/48), and a six-month event-free survival rate of 29.2% (14/48) in patients with recurrent or persistent endometrial cancer (PMID: 26186911). 26186911
Unknown unknown Advanced Solid Tumor not applicable CX-072 Phase II Actionable In a Phase II trial (PROCLAIM-CX-072), CX-072 treatment demonstrated acceptable safety, and resulted in a disease control rate of 41% (47/114, 10 partial responses) in patients with advanced solid tumors (J Clin Oncol 38: 2020 (suppl; abstr 3005); NCT03993379). detail...
Unknown unknown pancreatic carcinoma not applicable Lexatumumab + PF-573228 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Lexatumumab and PF-573228 increased Tnfrsf10b (TrailR2) expression, decreased Bcl2L1 expression, and resulted in tumor growth inhibition in cell line xenograft models of pancreatic carcinoma (PMID: 28459212). 28459212
Unknown unknown T-cell acute lymphoblastic leukemia not applicable DT2216 Preclinical - Cell line xenograft Actionable In a preclinical study, DT2216 treatment resulted in decreased BCL-XL expression and increased apoptosis and reduced viability of a T-cell acute lymphoblastic leukemia (T-ALL) cell line in culture, and decreased tumor growth in xenograft models (PMID: 31792461). 31792461
Unknown unknown thymic carcinoma not applicable Selinexor Preclinical - Cell line xenograft Actionable In a preclinical study, Selinexor (KPT-330) induced cell-cycle arrest and apoptosis and inhibited growth of several thymic epithelial tumor cell lines, including thymoma and thymic carcinoma cell lines, in culture, and inhibited tumor growth in thymic carcinoma cell line xenograft models (PMID: 28819023). 28819023
Unknown unknown stomach cancer not applicable Lenvatinib + Pembrolizumab Phase II Actionable In a Phase II trial (EPOC1706), the combination of Lenvima (lenvatinib) and Keytruda (pembrolizumab) treatment in patients with either stomach cancer or gastroesophageal junction cancer resulted in a 69% (20/29) objective response rate, a disease control rate of 100% (29/29), a median progression-free survival of 7.1 months, and a median overall survival that had not yet been reached, and 8 patients were still experiencing an ongoing response at data cut-off (PMID: 32589866; NCT03609359). 32589866
Unknown unknown diffuse large B-cell lymphoma not applicable Acalabrutinib + ACP-319 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Calquence (acalabrutinib) and ACP-319 resulted in decreased tumor volume in diffuse large B-cell lymphoma xenograft models (Eur J of Cancer, Dec 2016, 69;1, S39-S40). detail...
Unknown unknown Advanced Solid Tumor not applicable Dacarbazine + Selumetinib Phase I Actionable In a Phase I trial, the combination of Koselugo (selumetinib) and Deticene (dacarbazine) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, with partial responses in 17% (4/23) and stable disease for greater than 6 weeks in 65% (15/23) of patients (PMID: 28264648). 28264648
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 Advanced Solid Tumor not applicable SNK01 Phase I Actionable In a Phase I trial, SNK01 treatment demonstrated safety and was well-tolerated, and resulted in stable disease as the best overall response in 4 heavily pre-treated advanced solid tumor patients (n=5) (J. of Clin. Onc. 2020 38:15_suppl, e15024; NCT03941262). detail...
Unknown unknown Hodgkin's lymphoma not applicable Alisertib + Vorinostat Case Reports/Case Series Actionable In a Phase I trial, Alisertib (MLN8237) in combination with Zolinza (vorinostat) demonstrated safety and preliminary efficacy, resulting in a partial response in 2 patients with Hodgkin's lymphoma (PMID: 31617432; NCT01567709). 31617432
Unknown unknown non-Hodgkin lymphoma not applicable Bendamustine + Rituximab Phase III Actionable In a Phase III trial, Rituximab and Bendamustine combination therapy resulted in improved 5-year progression free survival rate (66.5% vs 55.8%), but no difference in overall survival rate (81.7% vs 85%) compared to R-CHOP/R-CVP regimen in patients with indolent non-Hodgkin lymphoma or mantle cell lymphoma (J Clin Oncol 35, 2017 (suppl; abstr 7500)). detail...
Unknown unknown 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 neuroblastoma not applicable Go 6983 Preclinical - Cell culture Actionable In a preclinical study, Go 6983 decreased growth of a neuroblastoma cell line in culture (PMID: 10209967). 10209967
Unknown unknown lung non-small cell carcinoma not applicable BAY1217389 + Paclitaxel Preclinical Actionable In a preclinical study, the combination of BAY1217389 and Taxol (paclitaxel) resulted in enhanced growth inhibition compared to Taxol (paclitaxel) alone in non-small cell lung cancer xenograft models (PMID: 26832791). 26832791
Unknown unknown endometrial cancer not applicable Everolimus Phase II Actionable In a Phase II trial, the mTOR inhibitor Afinitor (everolimus) demonstrated efficacy and tolerability in patients with chemotherapy-refractory advanced or metastatic endometrial cancer (PMID: 23612453). 23612453
Unknown unknown Advanced Solid Tumor not applicable 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 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 malignant pleural mesothelioma not applicable Trabectedin Preclinical - Cell line xenograft Actionable In a preclinical study, Yondelis (trabectedin) inhibited growth and increased apoptosis of malignant pleural mesothelioma (MPM) cell lines in culture, decreased viability of primary MPM cells in culture, and inhibited tumor growth in MPM cell line xenograft models (PMID: 27512118). 27512118
Unknown unknown gastroesophageal junction adenocarcinoma no benefit Oxaliplatin + Ramucirumab + Tegafur-gimeracil-oteracil Potassium 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 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 chronic lymphocytic leukemia not applicable Ibrutinib Clinical Study Actionable In a clinical study, treatment with Imbruvica (ibrutinib) resulted in a discontinuation-free survival rate at 1 year of 73.7% (232/315) and an absolute 1 year survival rate of 83.3% (264/315) in patients with relapsed or refractory chronic lymphocytic leukemia (PMID: 27756834). 27756834
Unknown unknown glioblastoma multiforme not applicable Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in stable disease in a patient with glioblastoma (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown 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 hepatocellular carcinoma not applicable Chiauranib Preclinical - Cell line xenograft Actionable In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a hepatocellular carcinoma cell line xenograft model (PMID: 28004478). 28004478
Unknown unknown 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 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 glioblastoma multiforme not applicable PF-00562271 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with PF-00562271 inhibited PTK2 (FAK) phosphorylation and resulted in apoptosis and tumor suppression in glioblastoma cancer cell line xenograft models (PMID: 18339875). 18339875
Unknown unknown thyroid gland carcinoma not applicable Sorafenib Clinical Study Actionable In a clinical case report, Nexavar (sorafenib) therapy based on in vitro efficacy testing using patient-derived tumor cells resulted in 43 months of disease-free in a patient with anaplastic thyroid carcinoma (PMID: 27379749). 27379749
Unknown unknown Advanced Solid Tumor not applicable Debio 0617B Preclinical - Patient cell culture Actionable In a preclinical study, Debio 0617B inhibited survival of a variety of patient-derived tumor cells in culture (PMID: 27439479). 27439479
Unknown unknown 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 Ascrinvacumab 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 lung non-small cell carcinoma not applicable Mogamulizumab + Nivolumab Phase I Actionable In a Phase I trial, Poteligeo (mogamulizumab-kpkc) and Opdivo (nivolumab) combination treatment demonstrated acceptable safety, resulted in an objective response rate of 20% (3/15, 3 partial responses) and a disease control rate of 40% (6/15) in immunotherapy-naive patients with advanced or metastatic non-small cell lung cancer, response occurred regardless of PD-L1, CCR4, CD8 expression levels and tumor mutational burden (PMID: 31455681; NCT02476123). 31455681
Unknown unknown lung small cell carcinoma not applicable Carboplatin + Durvalumab + Etoposide FDA approved Actionable In a Phase III (CASPIAN) trial that supported FDA approval, Imfinzi (durvalumab) in combination with Vepesid (etoposide) and Paraplatin (carboplatin) or Platinol (cisplatin) resulted in significantly improved overall survival (13.0 vs 10.3 mo, HR=0.73, p=0.0047) compared to platinum-etoposide therapy in patients with untreated extensive-stage small cell lung cancer (PMID: 31590988; NCT03043872). 31590988
Unknown unknown 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 stomach cancer not applicable Capmatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Tabrecta (capmatinib) did not induce tumor regression, however, inhibited tumor growth in an autocrine cell line xenograft model of gastric cancer overexpressing HGF (PMID: 30674502). 30674502
Unknown unknown glioblastoma multiforme not applicable Selinexor Phase II Actionable In a Phase II trial, Selinexor (KPT-330) treatment resulted in partial response in 11% (3/27) and stable disease in 22% (6/27) of patients with glioblastoma multiforme (J Clin Oncol 34, 2016 (suppl; abstr 2077)). detail...
Unknown unknown prostate cancer not applicable PX-866 Phase II Actionable In a Phase II trial, Sonolisib (PX-866) treatment was tolerated, resulted in a partial response in 11% (2/18) and stable disease in 56% (10/18) of evaluable patients with recurrent or metastatic castration-resistant prostate cancer, PSA response was observed in 2% (1/43) of evaluable patients, and 32.6% (14/43) of all patients were progression-free at 12 weeks (PMID: 31056399). 31056399
Unknown unknown 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 prostate cancer not applicable Niraparib Phase I Actionable In a Phase I trial, Zejula (niraparib) treatment resulted in stable diseases with a median duration of 254 days in 43% (9/21) of patients with castration-resistant prostate cancer (PMID: 23810788; NCT00749502). 23810788
Unknown unknown renal cell carcinoma not applicable Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in partial response in 2 patients and stable disease in 1 patient with renal cell carcinoma (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown glioblastoma multiforme not applicable 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 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 lung non-squamous non-small cell carcinoma no benefit Carboplatin + Motesanib Diphosphate + Paclitaxel Phase III Actionable In a Phase III trial, Motesanib (AMG 706) in combination with Taxol (paclitaxel) and Paraplatin (carboplatin) did not improve progression-free survival significantly compared to placebo (6.1 vs 5.6 months) in patient with non-squamous non-small cell lung cancer (PMID: 28902534). 28902534
Unknown unknown 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 hepatocellular carcinoma not applicable Metformin + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, addition of Glucophage (metformin) sensitized hepatocellular carcinoma cells to Nexavar (sorafenib) induced apoptosis in culture, resulted in suppression of postoperative intrahepatic recurrence and lung metastasis in cell line xenograft models (PMID: 26957312). 26957312
Unknown unknown Hodgkin's lymphoma not applicable Brentuximab vedotin FDA approved Actionable In a Phase III trial (AETHERA) that supported FDA approval, Adcetris (brentuximab vedotin) treatment as consolidation therapy after autologous stem-cell transplantation resulted in significantly improved progression-free survival compared to placebo (42.9 vs 24.1 months, HR=0.57, p=0.0013) in patients with classical Hodgkin's lymphoma at risk for relapse or progression after transplantation (PMID: 25796459; NCT01100502). detail... 25796459
Unknown unknown Hodgkin's lymphoma not applicable Brentuximab vedotin FDA approved Actionable In a Phase II trial that supported FDA approval, Adcetris (brentuximab vedotin) treatment resulted in an objective response rate of 73% (72/102, 33 complete response, 38 partial response) in patients with relapsed or refractory classical Hodgkin's lymphoma following autologous stem cell transplantation (PMID: 25533035; NCT00848926). detail... 25533035
Unknown unknown colorectal cancer not applicable TP-1454 + unspecified CTLA4 antibody Preclinical Actionable In a preclinical study, TP-1454 treatment combined with an anti-CTLA4 antibody inhibited tumor growth and increased survival in a syngeneic mouse model of colorectal cancer (Mol Cancer Ther 2019;18(12 Suppl):Abstract nr B080). detail...
Unknown unknown 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 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 juvenile astrocytoma not applicable MRK-003 Preclinical Actionable In a preclinical study, MRK-003 inhibited HES1 expression in pediatric low-grade astrocytoma cell lines in culture and decreased migration in 1 of 2 cell lines, but did not have a significant effect on cell growth (PMID: 25575134). 25575134
Unknown unknown pancreatic cancer not applicable Berzosertib + Gemcitabine + Radiotherapy Preclinical - Cell line xenograft Actionable In a preclinical study, Berzosertib (VX-970) enhanced the efficacy of radiotherapy combined with Gemzar (gemcitabine) in pancreatic cell line xenograft models, demonstrating a longer delay in tumor growth when compared to the models treated with only Gemzar (gemcitabine) and radiotherapy (PMID: 23222511). 23222511
Unknown unknown malignant glioma not applicable ADDA 5 Preclinical - Cell line xenograft Actionable In a preclinical study, ADDA 5 inhibited energy metabolism and proliferation in both chemo-sensitive and chemo-resistant glioma cell lines in culture, and suppressed tumor growth in cell line xenograft models (PMID: 27679486). 27679486
Unknown unknown plasmacytoma not applicable REGN5458 Case Reports/Case Series Actionable In a Phase Ib/II study, REGN5458 treatment resulted in a partial response in a patient with medullary and cutaneous extramedullary plasmacytoma (Blood (2019) 134 (Supplement_1): 3176; NCT03761108). detail...
Unknown unknown lung small cell carcinoma not applicable Alisertib Preclinical - Pdx & cell culture Actionable In a preclinical study, Alisertib (MLN8237), alone and in combination with Taxol (paclitaxel), inhibited growth of small cell lung cancer cell (SCLC) lines in culture and inhibited tumor growth in primary human SCLC xenograft models (Mol Cancer Ther 2013;12(11 Suppl):A282). detail...
Unknown unknown lung small cell carcinoma not applicable Alisertib Phase II Actionable In a Phase II trial, treatment with Alisertib (MLN8237) resulted in an objective response in 21% (10/48) of patients with small cell lung cancer (PMID: 25728526). 25728526
Unknown unknown 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 malignant glioma not applicable NEO214 Preclinical Actionable In a preclinical study, NEO214 induced apoptosis and inhibited growth of glioma cell lines in culture, including cell lines resistant to Temodar (temozolmide), and inhibited tumor growth and improved survival in a mouse glioma model (PMID: 30647121). 30647121
Unknown unknown breast cancer not applicable SKI-G-801 Preclinical Actionable In a preclinical study, SKI-G-801 treatment reduced tumor growth in a syngeneic mouse model of breast cancer (Cancer Res 2019;79(13 Suppl):Abstract nr 2010). detail...
Unknown unknown malignant ependymoma not applicable Sunitinib Phase II Actionable In a Phase II clinical trial, Sutent (sunitinib) was well-tolerated in young patients with ependymoma, but did not demonstrate sufficient anti-tumor activity as a single agent, with no patients achieving a sustained objective response (PMID: 27109549). 27109549
Unknown unknown mantle cell lymphoma not applicable Parsaclisib Phase Ib/II Actionable In a Phase I/II trial, Parsaclisib (INCB050465) treatment demonstrated tolerability and preliminary activity in patients with refractory B-cell malignancies, and resulted in an overall response rate of 67 (6/9), complete response/complete metabolic response rate of 44% (4/9), and median duration of response was not reached in patients with mantle cell lymphoma (PMID: 30803990; NCT02018861). 30803990
Unknown unknown head and neck squamous cell carcinoma not applicable Sirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Rapamune (sirolimus) decreased tumor growth and increased survival of cell line xenograft models of head and neck squamous cell carcinoma (PMID: 21520111). 21520111
Unknown unknown prostate carcinoma not applicable Dactolisib 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 Crizotinib + Dasatinib Phase I Actionable In a Phase I trial, Xalkori (crizotinib) therapy, in combination with Sprycel (dasatinib), in patients with advanced solid tumors resulted in limited efficacy, including one patient with a partial response and three patients with stable disease for at least six months or more (PMID: 29047029). 29047029
Unknown unknown hepatocellular carcinoma not applicable DRP-104 + unspecified PD-L1 antibody Preclinical Actionable In a preclinical study, DRP-104 treatment in combination with an anti-PD-L1 antibody enhanced inhibition of tumor growth, increased survival, and led to a durable response in a syngeneic mouse model of hepatocellular carcinoma (J Immunother Cancer. 2019; 7(Suppl 1): 282, Abs nr: P497). detail...
Unknown unknown Advanced Solid Tumor not applicable Litronesib Phase I Actionable In an analysis of two Phase I trial, litronesib (LY2523355) treatmetn resulted in partial response in 2.3% (2/86) and stable disease in 20% (17/86) of patients with advanced cancer (PMID: 28097385). 28097385
Unknown unknown pancreatic ductal adenocarcinoma not applicable Fluorouracil + MN58b Preclinical Actionable In a preclinical study, MN58b and Adrucil (fluorouracil) in combination demonstrated an additive effect on growth inhibition of pancreatic ductal adenocarcinoma cell lines in culture (PMID: 26769123). 26769123
Unknown unknown Advanced Solid Tumor not applicable Ipatasertib Preclinical - Cell line xenograft Actionable In a preclinical study, Ipatasertib (GDC-0068) demonstrated activity against tumor growth in cell line xenograft models of solid tumors (PMID: 24141624). 24141624
Unknown unknown Advanced Solid Tumor not applicable Ipatasertib Phase I Actionable In a Phase I trial, Ipatasertib (GDC-0068) resulted in antitumor activity in 30% (16/52) of patients with advanced solid tumors, primarily demonstrating stable disease (PMID: 27872130). 27872130
Unknown unknown 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 T-cell acute lymphoblastic leukemia not applicable Torin 2 Preclinical - Cell culture Actionable In a preclinical study, Torin 2 treatment inhibited viability of NUP214-ABL1 fusion-positive T-cell acute lymphoblastic leukemia cell lines in culture (PMID: 27821800). 27821800
Unknown unknown colon cancer not applicable SKLB-23bb Preclinical - Cell line xenograft Actionable In a preclinical study, SKLB-23bb treatment resulted in antitumor efficacy in colon cancer cells, demonstrating decreased colony formation in vitro and inhibition of tumor growth in cell line xenograft models (PMID: 29610282). 29610282
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 breast cancer not applicable GTCpFE Preclinical - Cell line xenograft Actionable In a preclinical study, GTCpFE decreased viability of breast cancer cell lines in culture and reduced the population of cancer stem cells, resulting in decreased tumorigenicity in xenograft models (PMID: 26530254). 26530254
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 Advanced Solid Tumor not applicable Camrelizumab Phase I Actionable In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors, with an objective response rate of 31% (18/58, all partial responses) and disease control rate of 46.5% (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). detail...
Unknown unknown glioblastoma multiforme not applicable Abemaciclib Phase I Actionable In a Phase I trial, three patients with glioblastoma demonstrated stable disease when treated with Abemaciclib (LY2835219) (PMID: 27217383). 27217383
Unknown unknown Advanced Solid Tumor not applicable 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 primary mediastinal B-cell lymphoma not applicable Pembrolizumab FDA approved Actionable In a Phase II trial (KEYNOTE-170) that supported FDA approval, Keytruda (pembrolizumab) treatment resulted in an objective response rate of 45% (24/53) in patients with primary mediastinal large B-cell lymphoma, with a complete response rate of 13% (7/53), and a median progression-free survival of 5.5 months (ASH Annual Meeting Dec 2018, Session 626, abstract 228; NCT02576990). detail... detail... detail...
Unknown unknown Advanced Solid Tumor not applicable CBP501 + Cisplatin + Nivolumab Phase I Actionable In a Phase Ib trial, CBP501, Platinol (cisplatin), and Opdivo (nivolumab) triple combination demonstrated safety and preliminary efficacy, resulted in partial response in 11.8% (2/17) of patients with advanced refractory solid tumors, with a disease control rate (complete response+partial response+stable disease over 3 months) of 35% (6/17) (AACR Annual Meeting 2019, Abstract CT228; NCT03113188). detail...
Unknown unknown diffuse large B-cell lymphoma not applicable ME-401 Phase I Actionable In a Phase I trial, ME-401 treatment was well-tolerated, resulted in an objective response rate of 25% (2/8) in patients with relapsed or refractory diffuse large B-cell lymphoma (J Clin Oncol 38: 2020 (suppl; abstr 8016); NCT02914938). detail...
Unknown unknown 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 colorectal cancer not applicable Bevacizumab + Fluorouracil + Irinotecan + Leucovorin FDA approved Actionable In a Phase III clinical trial that supported FDA approval, the combination of Avastin (bevacizumab) and FOLFIRI chemotherapy demonstrated increased duration of overall survival and improved progression-free survival compared to FOLFIRI alone in patients with metastatic colorectal cancer (PMID: 22477726, PMID: 15175435). 15175435 22477726 detail...
Unknown unknown prostate cancer not applicable ONC201 Clinical Study Actionable In a clinical case study, a prostate cancer patient demonstrated tumor regression in the primary tumor and metastatic lesions when treated with ONC201 (TIC-10) (PMID: 28331050). 28331050
Unknown unknown 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 ovary epithelial cancer not applicable Alpelisib + Olaparib Phase I Actionable In a Phase Ib trial, Alpelisib (BYL719) and Lynparza (olaparib) combination therapy demonstrated safety and preliminary efficacy, resulted in partial response in 36% (10/28) and stable disease in 50% (14/28) of patients with epithelial ovarian cancer, overall response rate was similar for germline BRCA mutated and wild-type patients (30%, 3/10 vs 35%, 6/17, p=0.42) (PMID: 30880072; NCT01623349). 30880072
Unknown unknown Advanced Solid Tumor not applicable Carboplatin + LY2090314 + Pemetrexed Disodium Phase I Actionable In a Phase I trial, LY2090314 in combination with Alimta (pemetrexed) and Paraplatin (carboplatin) resulted in partial response in 13.5% (5/37) and stable disease in 51.4% (19/37) of patients with advanced solid tumors (PMID: 26403509; NCT01287520). 26403509
Unknown unknown malignant fibrous histiocytoma not applicable Sapanisertib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with Sapanisertib (MLN0128) resulted in growth suppression and arrest in a myxofibrosarcoma cell line in culture, and inhibited tumor growth in myxofibrosarcoma cell line xenograft models (PMID: 27577794). 27577794
Unknown unknown diffuse large B-cell lymphoma not applicable Alisertib + Vorinostat Case Reports/Case Series Actionable In a Phase I trial, Alisertib (MLN8237) in combination with Zolinza (vorinostat) demonstrated safety and preliminary efficacy, resulting in a durable complete response in 2 patients with diffuse large B-cell lymphoma (PMID: 31617432; NCT01567709). 31617432
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 ovarian cancer not applicable BAY1161909 Preclinical Actionable In a preclinical study, BAY1161909 demonstrated moderate efficacy in ovarian cancer xenograft models (PMID: 26832791). 26832791
Unknown unknown 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 Advanced Solid Tumor not applicable MEDI3617 Phase I Actionable In a Phase I trial, MEDI3617 monotherapy resulted in no objective response (0/25) and stable disease in 52% (13/25) of patients with advanced solid tumors, with a median progression-free survival of 1.4 months (PMID: 29559563; NCT01248949). 29559563
Unknown unknown pancreatic ductal adenocarcinoma not applicable JQ1 Preclinical - Pdx & cell culture Actionable In a preclinical study, treatment with JQ1 in patient derived xenograft (PDX) models of pancreatic ductal adenocarcinoma (PDAC) demonstrated tumor growth suppression and in PDAC cells, resulted in inhibition of Hedgehog and TGF-beta signaling and a decrease in desmoplastic stroma (PMID: 27528027). 27528027
Unknown unknown multiple myeloma not applicable Mivebresib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with Mivebresib (ABBV-075) altered expression of ZCCHC24, HEXIM1, SERPINI1, and MYC in a multiple myeloma cell line in culture and in xenograft models, and these changes correlated with inhibition of proliferation in culture and inhibition of tumor growth in xenograft models (PMID: 27903752). 27903752
Unknown unknown multiple myeloma not applicable Bortezomib + Daratumumab + Dexamethasone FDA approved Actionable In a Phase III trial (CASTOR) that supported FDA approval, the combination of Darzalex (daratumumab), Velcade (bortezomib), and Adexone (dexamethasone) resulted in a greater 12 month progression-free survival (77.5% vs 29.4%) and overall response (82.9%; 199/240 vs 63.2%; 148/234) compared to Adexone (dexamethasone) and Velcade (bortezomib) alone in relapsed or refractory multiple myeloma patients (PMID: 27557302; NCT02136134). detail... 27557302
Unknown unknown 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 chronic lymphocytic leukemia not applicable PRT062607 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with PRT062607 (P505-15) resulted in decreased viability of chronic lymphocytic leukemia cells in culture and in cell line xenograft models (PMID: 23220742). 23220742
Unknown unknown chronic lymphocytic leukemia not applicable Acalabrutinib Phase Ib/II Actionable In a Phase Ib/II clinical trial, Calquence (acalabrutinib) treatment in chronic lymphocytic leukemia patients, including patients carrying chromosome 17p13.1 deletion, resulted in a 95% (57/60) overall response rate, which consisted of 85% with partial response and 5% (3/60) with stable disease (PMID: 26641137). 26641137
Unknown unknown chronic lymphocytic leukemia not applicable Acalabrutinib FDA approved Actionable In a Phase III trial (ELEVATE TN) that supported FDA approval, Calquence (acalabrutinib) treatment resulted in prolonged progression-free survival compared to Gazyva (obinutuzumab) plus chlorambucil (HR=0.20, p<0.0001) in patients with treatment-naive chronic lymphocytic leukemia (PMID: 31724010; NCT02475681). 31724010 detail...
Unknown unknown Advanced Solid Tumor not applicable Pemetrexed Disodium + Sorafenib Phase I Actionable In a Phase I clinical trial, the combination of Alimta (pemetrexed) and Nexavar (sorafenib) demonstrated safety and some preliminary efficacy in patients with advanced solid tumors, with an objective response rate of 15% (5/33) and stable disease in 45% (15/33) of patients (PMID: 27213589). 27213589
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 renal cell carcinoma not applicable GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 treatment was well-tolerated and resulted in some efficacy in renal cell carcinoma patients including stable disease in 13% (3/24), one patient with a complete response, and one patient with a partial response (PMID: 26603258). 26603258
Unknown unknown ovarian cancer not applicable Cediranib + Olaparib Phase I Actionable In a Phase I clinical trial, the combination therapy of Cediranib (AZD-2171) and Lynparza (olaparib) demonstrated safety and efficacy in patients with ovarian cancer (J Clin Oncol 32:5s, 2014 (suppl; abstr LBA5500)). detail...
Unknown unknown acute myeloid leukemia not applicable CX-6258 Preclinical - Cell line xenograft Actionable In a preclinical study, CX-6528 inhibited tumor growth in human acute myeloid leukemia cell line xenograft models (PMID: 24900437). 24900437
Unknown unknown triple-receptor negative breast cancer not applicable G-TPP + Obatoclax Preclinical - Cell culture Actionable In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Obatoclax (GX015-070) synergistically inhibited viability of triple-receptor negative breast cancer cells in culture (PMID: 28522750). 28522750
Unknown unknown lung non-squamous non-small cell carcinoma not applicable Motesanib Diphosphate + Paclitaxel Phase III Actionable In a Phase III study, Motesanib plus Paraplatin (carboplatin) or Taxol (paclitaxel) improved overall survival, progression free survival and objective response rate for a subset of Asian patients with advanced nonsquamous non-small cell lung cancer (PMID: 24419239; NCT00460317). 24419239
Unknown unknown melanoma not applicable Lenvatinib + Pembrolizumab Phase Ib/II Actionable Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 48% (10/21) in patients with metastatic melanoma, with a median duration of response of 12.5 months, and a median progression-free survival of 5.5 months (PMID: 31961766; NCT02501096). 31961766
Unknown unknown gastrointestinal stromal tumor not applicable JSH-150 Preclinical - Cell culture Actionable In a preclinical study, JSH-150 inhibited proliferation of a gastrointestinal stromal tumor cell line in culture (PMID: 30253346). 30253346
Unknown unknown lung non-small cell carcinoma not applicable Sunitinib Phase III Actionable In a Phase III trial, Sutent (sunitinib) as maintenance therapy resulted in improved progression free survival (4.3 vs 2.6 months) but not overall survival (11.7 vs 12.1 months) compared to placebo in patients with stage IIIB/IV non-small cell lung cancer (PMID: 28161554). 28161554
Unknown unknown lung non-small cell carcinoma not applicable Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) treatment resulted in an objective response rate of 8% (1/13, partial response) and stable disease as the best response in 69% (9/13) of non-small cell lung carcinoma patients (PMID: 32312893; NCT01829217). 32312893
Unknown unknown lung non-small cell carcinoma not applicable Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) treatment in non-small cell lung cancer patients resulted in an objective response rate of 11.1% (7/63), stable disease in 28.6% (18/63), and a PFS of 12 weeks and OS of 23.4 weeks (PMID: 18235126). 18235126
Unknown unknown 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 head and neck squamous cell carcinoma not applicable Pembrolizumab FDA approved Actionable In a Phase Ib trial (KEYNOTE-012) that supported FDA approval, treatment with Keytruda (pembrolizumab) resulted in an objective response rate of 18% (24/132; 4 complete response, 20 partial response) in patients with recurrent and/or metastatic head and neck squamous cell carcinoma (PMID: 27247226; NCT01848834). detail... detail... 27247226
Unknown unknown acute myeloid leukemia not applicable Birabresib Phase I Actionable In a Phase I trial, Birabresib (OTX015) treatment resulted in complete remission lasting 2-5 months in 8% (3/36) and partial blast clearance in 6% (2/36) of acute myeloid leukaemia patients (PMID: 27063977). 27063977
Unknown unknown Advanced Solid Tumor not applicable Carboplatin + Paclitaxel + Panobinostat Phase I Actionable In a Phase I trial, 52% (11/21) of patients with advanced solid tumors demonstrated stable disease when treated with Farydak (panobinostat), in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) (PMID: 22851205). 22851205
Unknown unknown Indication other than cancer not applicable Fingolimod FDA approved Actionable Gilenya (fingolimod) is FDA approved for use in patients with relapsing forms of multiple sclerosis (FDA.gov). detail... detail...
Unknown unknown pancreatic ductal adenocarcinoma not applicable Arsenic trioxide + JQ1 Preclinical - Cell line xenograft Actionable In a preclinical study, JQ1 and Trisenox (arsenic trioxide) combination treatment reduced viability of Trisenox (arsenic trioxide)-insensitive pancreatic ductal adenocarcinoma cell lines in culture, and synergistically inhibited tumor growth in a cell line xenograft model (PMID: 31420604). 31420604
Unknown unknown pancreatic adenocarcinoma not applicable Capecitabine + Ruxolitinib Phase II Actionable In a Phase II trial, the combination of Jakafi (ruxolitinib) and Xeloda (capecitabine) did not prolong OS (median OS 4.5 vs. 4.3 months) in patients with pancreatic adenocarcinoma when compared to placebo plus Xeloda (capecitabine), however, prolonged OS (median OS 2.7 vs. 1.8 months) was observed in pancreatic adenocarcinoma patients with elevated levels of serum CRP (PMID: 26351344). 26351344
Unknown unknown ovarian cancer not applicable Alisertib Phase II Actionable In a Phase II clinical trial, Alisertib (MLN8237) demonstrated some preliminary efficacy in patients with platinum-resistant or refractory ovarian cancer, with 10% (3/31) of patients demonstrating objective response and 52% (16/31) of patients achieving stable disease (PMID: 22772063). 22772063
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 hematologic cancer not applicable M7583 Phase I Actionable In a Phase I trial, M7583 demonstrated preliminary efficacy in patients with B-cell malignancies (including Waldenstrom's macroglobulinemia, mantle cell lymphoma, and B-cell chronic lymphocytic leukemia), resulting in objective response or stable disease in 3/3 patients (J Clin Oncol 35, 2017 (suppl; abstr e14101)). detail...
Unknown unknown lung small cell carcinoma not applicable Doxorubicin + Lurbinectedin Phase I Actionable In a Phase I trial, Lurbinectedin (PM01183) and Adriamycin (doxorubicin) combination treatment resulted in complete response in 8% (2/27) and partial response in 50% (13/27) of patients with relapsed small-cell lung cancer (PMID: 28961837). 28961837
Unknown unknown head and neck squamous cell carcinoma not applicable Cetuximab + Motolimod Phase Ib/II Actionable In a Phase Ib trial, Motolimod (VTX-2337) and Erbitux (cetuximab) combination treatment resulted in partial response in 15% (2/13) and stable disease in 39% (5/13) of patients with head and neck squamous cell carcinoma (PMID: 27810904). 27810904
Unknown unknown Advanced Solid Tumor not applicable Nintedanib Phase I Actionable In a Phase I trial, Ofev (nintedanib) demonstrated safety and some preliminary efficacy in patients with advanced solid tumors (PMID: 25795637). 25795637
Unknown unknown Advanced Solid Tumor not applicable SR9011 Preclinical - Cell culture Actionable In a preclinicl study SR9011 demonstrated toxicity in a wide range of tumor cell lines harboring different driver mutations, but not in normal cell lines in culture (PMID: 29320480). 29320480
Unknown unknown acute lymphoblastic leukemia not applicable OBI-3424 Preclinical - Pdx Actionable In a preclinical study, OBI-3424 treatment resulted in objective response in 89% (8/9, 2 complete response) of patient-derived xenograft (PDX) models of T-Cell acute lymphoblastic leukemia (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr LB-B16). detail...
Unknown unknown Advanced Solid Tumor not applicable TAK-733 Phase I Actionable In a Phase I clinical trial, TAK-733 demonstrated safety and some preliminary efficacy in patients with advanced solid tumors with partial response in 5% (2/41) and stable disease in 37% (15/41) of patients (PMID: 27650277). 27650277 detail...
Unknown unknown ovarian cancer not applicable Selinexor Phase I Actionable In a Phase I clinical trial, Selinexor (KPT-330) treatment resulted in inhibition of tumor growth in 3/5 patients with platinum-refractory ovarian cancer, with one patient achieving a partial response and two patients achieving stable disease (PMID: 27649553). 27649553
Unknown unknown 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 triple-receptor negative breast cancer not applicable BOS172722 + Paclitaxel Preclinical - Pdx & cell culture Actionable In a preclinical study, BOS172722 and Taxol (paclitaxel) demonstrated synergy in triple-negative breast cancer (TNBC) cell lines in culture, resulting in increased cell death, and the combination of BOS172722 and Taxol (paclitaxel) induced tumor regression in TNBC cell line and patient-derived xenograft (PDX) models, and decreased tumor growth and increased survival in a TNBC cell line xenograft metastasis model, demonstrating increased benefit over Taxol (paclitaxel) or BOS172722 alone (PMID: 31575759). 31575759
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 Advanced Solid Tumor not applicable BAY1125976 Phase I Actionable In a Phase I trial, BAY 1125976 treatment demonstrated safety and pharmacological inhibition of Akt signaling, but only resulted in a partial response in 1% (1/78) and stable disease in 38% (30/78) of patients with advanced solid tumors, and a clinical benefit rate of 27.9% (12/43) at the recommended Phase II dose (PMID: 31835495; NCT01915576). 31835495
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 uterine carcinosarcoma not applicable Cabozantinib Phase II Actionable In a Phase II (NCI9322/PHL86) trial, Cometriq (Cabometyx, cabozantinib) treatment resulted in a partial response in 5% (1/19) and progression-free survival (PFS) at 12 weeks in 42% (8/19) of patients with endometrial carcinosarcoma, with a median PFS of 3.0 months (PMID: 31992589; NCT01935934). 31992589
Unknown unknown acute lymphoblastic leukemia not applicable ABT-348 Preclinical - Cell line xenograft Actionable In a preclinical study, Ilorasertib (ABT-348) displayed efficacy in acute lymphoblastic leukemia cell line xenograft models (PMID: 22935731). 22935731
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 multiple myeloma not applicable Dexamethasone + Isatuximab + Pomalidomide FDA approved Actionable In a Phase III (ICARIA-MM) trial that supported FDA approval, addition of Sarclisa (isatuximab-irfc) to Pomalyst (pomalidomide) and dexamethasone significantly improved progression-free survival (11.5 vs 6.5 months, HR=0.596, p=0.001) in patients with relapsed and refractory multiple myeloma (PMID: 31735560; NCT02990338). detail... 31735560
Unknown unknown Burkitt lymphoma not applicable Cerdulatinib Preclinical Actionable In a preclinical study, treatment with Cerdulatinib (PRT062070) resulted in decreased viability and increased apoptosis of Burkitt lymphoma cells in culture (PMID: 25253883). 25253883
Unknown unknown ovary epithelial cancer not applicable Binimetinib + Paclitaxel Phase Ib/II Actionable In a Phase Ib trial, combination therapy with Taxol (paclitaxel) and Mektovi (binimetinib) was tolerable and resulted in an objective response rate of 18% (5/28, 1 complete response, 4 partial responses) and a clinical benefit rate of 57% (16/28, best overall response of stable disease or better) in patients with platinum resistant/refractory epithelial ovarian cancer, and clinical benefit was observed in all patients with MAPK pathway alterations (n=4) (PMID: 29844129; NCT01649336). 29844129
Unknown unknown 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 breast cancer not applicable Tamoxifen + Verrucarin A Preclinical Actionable In a preclinical study, Verrucarin A treatment sensitized breast cancer cells to Nolvadex (tamoxifen) in culture, resulting in dose-dependent growth inhibition (PMID: 24743578). 24743578
Unknown unknown colon cancer not applicable AsiDNA + Olaparib Preclinical - Cell culture Actionable In a preclinical study, AsiDNA treatment led to increased sensitivity to Lynparza (olaparib), resulting in decreased survival in colon cancer cell lines in culture (PMID: 27559053). 27559053
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 prostate cancer not applicable KX2-391 Phase II Actionable In a Phase II trial, KX2-391 at tested dose did not demonstrate anti-tumor effects in patients with castration-resistant prostate cancer, resulted in 8% progression free survival (PFS) at 24 weeks and median PFS of 18.6 weeks, but had modest effects on bone turnover markers (PMID: 23314737). 23314737
Unknown unknown 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 multiple myeloma not applicable Carfilzomib + DT204 Preclinical - Cell culture Actionable In a preclinical study, multiple myeloma cells resistant to Kyprolis (carfilzomib) demonstrated re-sensitization to Kyprolis (carfilzomib) in culture when treatment was combined with DT204, showing a synergistic effect and increased apoptotic activity (PMID: 27677741). 27677741
Unknown unknown multiple myeloma not applicable Bortezomib + Torkinib Preclinical - Cell culture Actionable In a preclinical study, Torkinib (PP242) worked synergistically with Velcade (Bortezomib) to induce apoptosis in multiple myoloma cell lines in culture (PMID: 20686120). 20686120
Unknown unknown renal cell carcinoma not applicable Axitinib + Carotuximab Phase I Actionable In a Phase I trial, treatment the combination of Inlyta (axitinib) and Carotuximab (TRC-105) demonstrated preliminary activity in patients with renal cell carcinoma, resulting in partial response in 5 and stable disease in 10 of 17 evaluable patients, and a median progression-free survival of 11.3 months (PMID: 30190302; NCT01806064). 30190302
Unknown unknown B-cell lymphoma not applicable Nutlin-3a Preclinical - Cell culture Actionable In a preclinical study, Nutlin-3 induced death of a mouse B-cell lymphoma cell line isolated from a tumor over expressing MYC in culture (PMID: 30069049). 30069049
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 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 colon cancer not applicable Irinotecan + Veliparib Phase I Actionable In a Phase I trial, Veliparib (ABT-888) and Camptosar (irinotecan) combination therapy resulted in partial response in 50% (2/4) of patients with colon cancer (PMID: 26842236). 26842236
Unknown unknown lung non-small cell carcinoma not applicable TMU-35435 Preclinical - Cell line xenograft Actionable In a preclinical study, TMU-35435 induced cell-cycle arrest and apoptosis and decreased viability of non-small cell lung cancer cell lines in culture, and inhibited tumor growth in a lung cancer cell line xenograft model (PMID: 28233309). 28233309
Unknown unknown Advanced Solid Tumor not applicable Docetaxel + Selumetinib Phase I Actionable In a Phase I trial, the combination of Koselugo (selumetinib) and Taxotere (docetaxel) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, with partial responses in 22% (6/27) and stable disease for greater than 6 weeks in 52% (14/27) of patients (PMID: 28264648). 28264648
Unknown unknown 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 Advanced Solid Tumor not applicable Lapatinib + MK2206 Phase I Actionable In a Phase I trial, Tykerb (lapatinib) and MK2206 combination treatment resulted in stable disease for more than 4 months in 9% (2/23) of patients with advanced solid tumors (PMID: 27026198). 27026198
Unknown unknown triple-receptor negative breast cancer not applicable BAY1161909 + Paclitaxel Preclinical Actionable In a preclinical study, BAY1161909, in combination with Taxol (paclitaxel), had increased efficacy in xenograft models of triple-negative breast cancer compared to Taxol (paclitaxel) alone, resulting in complete tumor regression (PMID: 26832791). 26832791
Unknown unknown 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 colorectal cancer no benefit Olaparib Phase II Actionable In a Phase II clinical trial, treatment with Lynparza (olaparib) did not result in clinical activity in colorectal cancer patients that had progressed on prior standard therapy, including both microsatellite-stable patients and those that demonstrated high microsatellite instability (PMID: 26786262). 26786262
Unknown unknown lung non-small cell carcinoma not applicable TVB-2640 Phase I Actionable In a Phase I study, TVB-2640 as a monotherapy or in combination with Taxol (paclitaxel) resulted in stable diseases for more than 16 weeks in 43% (3/7) of patients with non-small cell lung cancer (2015 51 S724-S724 Eur J Cancer). detail...
Unknown unknown Advanced Solid Tumor not applicable IPI-549 Preclinical Actionable In a preclinical study, IPI-549 inhibited tumor growth in multiple xenograft models of solid tumors (Mol Cancer Ther December 2015 14; A192). detail...
Unknown unknown prostate cancer not applicable Abiraterone + Prednisone + Veliparib Phase II Actionable In a Phase II (NCI 9012) trial, addition of Veliparib (ABT-888) to Zytiga (abiraterone) and Prednisone did not improve PSA response rate (72.4% vs 63.9%, p=0.27), measurable disease response rate (52.2% vs 45.0%, p=0.51), or median progression-free survival (10.1 vs 11.0 months, p=0.99) in patients with metastatic castration-resistant prostate cancer (PMID: 29261439; NCT01576172). 29261439
Unknown unknown glioblastoma multiforme not applicable Gliovac + Sargramostim Clinical Study Actionable In a clinical study, Gliovac (ERC 1671) adminstered with Leukine (sargramostim) as an adjuvant demonstrated low toxicity and resulted in an improved 40-week overall survival rate of 77% (n=9) vs. 10% (n=39) with historical controls in patients with recurrent glioblastoma multiforme (PMID: 25865468). 25865468
Unknown unknown brain stem glioma not applicable MRK-003 Preclinical - Cell culture Actionable In a preclinical study, MRK-003 increased apoptosis and decreased growth of diffuse pontine glioma cell lines in culture (PMID: 26115193). 26115193
Unknown unknown Advanced Solid Tumor not applicable Ganitumab Phase I Actionable In a Phase I trial, Ganitumab (AMG 479) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol (Meeting Abstracts) 2007 25: 3002). detail...
Unknown unknown breast cancer not applicable AIM-100 Preclinical Actionable In a preclinical study, AIM-100 inhibited growth of breast cancer cells in culture (PMID: 22322295). 22322295
Unknown unknown clear cell sarcoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 54%, median progression-free survival of 11 months, an objective response rate of 14% (n=7), and a median overall survival of 16 months in patients with clear cell sarcoma (PMID: 29895706; NCT01878448). 29895706
Unknown unknown Advanced Solid Tumor not applicable GDC-0917 Phase I Actionable In a Phase I clinical trial, GDC-0917 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 31, 2013 (suppl; abstr 2503)). detail...
Unknown unknown head and neck squamous cell carcinoma no benefit Cetuximab + Cisplatin + Docetaxel + Fluorouracil Phase II Actionable In a Phase II trial, addition of Erbitux (cetuximab) to induction chemotherapy consisting of Platinol (cisplatin), Taxotere (docetaxel), and Fluorouracil, resulted in early termination of the study due to unacceptable clinical outcomes (PMID: 28911062). 28911062
Unknown unknown gastroesophageal junction adenocarcinoma not applicable ALX148 + Trastuzumab Phase I Actionable In a Phase I trial, ALX148 and Herceptin (trastuzumab) combination treatment resulted in a partial response in 19% (4/21) and stable disease in 29% (6/21) of patients with gastric or gastroesophageal junction cancer that progressed on chemotherapy (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 2514-2514; NCT03013218). detail...
Unknown unknown lymphoma not applicable APTO-253 Preclinical - Cell culture Actionable In a preclinical study, APTO-253 treatment in acute myeloid leukemia cell lines resulted in decreased proliferation in culture (PMID: 29626127). 29626127
Unknown unknown hepatocellular carcinoma not applicable CT-707 + PHA-665752 Preclinical - Cell culture Actionable In a preclinical study, the combination of PHA-665752 and CT-707 resulted in synergism in hepatocellular carcinoma cells in culture, demonstrating near complete cell death (PMID: 27638856). 27638856
Unknown unknown 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 glioblastoma multiforme not applicable Tanibirumab Preclinical - Cell line xenograft Actionable In a preclinical study, Tanibirumab (TTAC-0001) inhibited angiogenesis and tumor growth in human glioblastoma cell line xenograft models (PMID: 26325365). 26325365
Unknown unknown pancreatic ductal adenocarcinoma no benefit Atezolizumab + BKT140 Phase Ib/II Actionable In a combined analysis of 2 Phase I/II trials, Tecentriq (atezolizumab) and BL-8040 (BKT140) combination therapy demonstrated expected toxicity profile and limited efficacy, did not improve objective response rate (0/14 vs 0/15), median progression-free survival (1.6 vs 2.5 mo), or median overall survival (5.2 vs 6.8 mo) compared to control in patients with metastatic pancreatic ductal adenocarcinoma (J Clin Oncol 38, 2020 (suppl 4; abstr 712); NCT03281369, NCT03193190). detail...
Unknown unknown sarcoma not applicable Doxorubicin + Nilotinib Phase I Actionable In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin demonstrated safety and preliminary efficacy, resulted in 1 partial response and 9 stable disease in 13 patients with sarcomas (PMID: 30037815; NCT02587169). 30037815
Unknown unknown prostate cancer not applicable EC-70124 Preclinical - Cell line xenograft Actionable In a preclinical study, EC-70124 inhibited proliferation, colony formation, Stat3 activity, and NFkappaB activity in prostate cancer cells and prostate cancer stem cells in culture and inhibited tumor growth in cell line xenografts (PMID: 26826115). 26826115
Unknown unknown 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 lung small cell carcinoma not applicable Cisplatin + Etoposide + Tislelizumab Phase II Actionable In a Phase II trial, treatment with Tislelizumab (BGB-A317) plus platinum doublet chemotherapy (Vepesid (etoposide) with Platinol (cisplatin) or Paraplatin (carboplatin)) in patients with small cell lung cancer resulted in an objective response rate of 77% (13/17) and disease control rate of 88% (15/17), including a partial response in 13 patients and stable disease in two patients, and median progression-free survival of 6.9 months (PMID: 32769013; NCT03432598). 32769013
Unknown unknown glioblastoma multiforme not applicable AsiDNA Preclinical - Cell culture Actionable In a preclinical study, AsiDNA inhibited survival of glioblastoma cell lines in culture (PMID: 27559053). 27559053
Unknown unknown multiple myeloma not applicable GNE-272 Preclinical - Cell culture Actionable In a preclinical study, GNE-272 treatment resulted in anti-proliferative activity in multiple myeloma cell lines in culture (PMID: 27682507). 27682507
Unknown unknown neuroblastoma not applicable JSH-150 Preclinical - Cell culture Actionable In a preclinical study, JSH-150 inhibited proliferation of a neuroblastoma cell line in culture (PMID: 30253346). 30253346
Unknown unknown lung cancer not applicable VB-111 Preclinical Actionable In a preclinical study, VB-111 treatment resulted in increased tumor lymphocyte infiltration and reduced tumor burden in animal models of lung cancer (AACR Annual Meeting 2019, Abstract 4979). detail...
Unknown unknown Advanced Solid Tumor not applicable Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in partial response in 9.5% (2/21) and stable disease in 57.1% (12/21) of patients with advanced solid tumors (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown chronic lymphocytic leukemia not applicable Ublituximab + Umbralisib Phase I Actionable In a Phase I trial, TGR-1202 and Ublituximab combination treatment resulted in a median progression-free survival of 8 months in all 10 chronic lymphocytic leukemia patients, with partial response in 100% (3/3) of patients in the high-dose TGR-1201 group and 57% (4/7) in the low-dose group (J Clin Oncol 33, 2015 (suppl; abstr 8548)). detail...
Unknown unknown acute myeloid leukemia not applicable Azacitidine + Mivebresib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Vidaza (azacitidine) and Mivebresib (ABBV-075) resulted in increased tumor growth inhibition in an acute myeloid leukemia cell line xenograft model compared to either agent alone (PMID: 28416490). 28416490
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 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 basal cell carcinoma not applicable Taladegib Phase I Actionable In a Phase I trial, Taladegib treatment resulted in a clinical response in 46.8% (22/47) of patients with basal cell carcinoma, including 16 patients with a confirmed partial response, 1 patient with an unconfirmed partial response, and 5 patients with a confirmed complete response (PMID: 29483143). 29483143
Unknown unknown lung cancer not applicable HD105 Preclinical - Cell line xenograft Actionable In a preclinical study, HD105 inhibited tumor progression in human lung cancer cell line xenograft models (PMID: 27049350). 27049350
Unknown unknown diffuse large B-cell 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 and induced tumor regression (80% complete and 20% partial) in cell-line xenograft models of diffuse large B-cell lymphoma (Cancer Res July 1 2019 (79) (13 Supplement) 2058). detail...
Unknown unknown chronic lymphocytic leukemia not applicable Duvelisib Phase I Actionable In a Phase I trial, Copiktra (duvelisib) demonstrated safety and efficacy in patients with advanced chronic lymphocytic leukemia (PMID: 24501284). 24501284
Unknown unknown cervical cancer not applicable ETP-46464 + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, ETP-46464 increased the sensitivity of cervical cancer cell lines to ionizing radiation in culture (PMID: 25560806). 25560806
Unknown unknown Advanced Solid Tumor not applicable Rivoceranib 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 diffuse large B-cell lymphoma not applicable STRO-001 Preclinical - Cell line xenograft Actionable In a preclinical study, STRO-001 potently inhibited growth of diffuse large B-cell lymphoma cell lines in culture, and suppressed tumor growth in cell line xenograft models (Blood 2016 128 (22):464). detail...
Unknown unknown Advanced Solid Tumor not applicable NHS-IL12 Phase I Actionable In a Phase I trial, NHS-IL12 was well tolerated in patients with metastatic advanced solid tumors and resulted in stable disease in 50% (15/30) of patients with measurable disease, with durable stable disease in 5 patients (two prostate, one colorectal, one breast, and one chordoma) lasting more than 6 months (PMID: 30131389; NCT01417546). 30131389
Unknown unknown marginal zone B-cell 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 Advanced Solid Tumor not applicable Doxorubicin + Safingol Phase I Actionable In a Phase I trial, the combination of Kynacyte (safingol) and Adriamycin (doxorubicin) demonstrated safety and some preliminary activity in patients with advanced solid tumors (PMID: 9815717). 9815717
Unknown unknown 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 osteosarcoma not applicable WZ4003 Preclinical - Cell culture Actionable In a preclinical study, WZ4003 inhibited MYPT1 phosphorylation and reduced invasive behavior and proliferation of an osteosarcoma cell line in culture (PMID: 24171924). 24171924
Unknown unknown alveolar soft part sarcoma not applicable Dasatinib Phase II Actionable In a Phase II trial, patients with alveolar soft part sarcoma demonstrated a median progression free survival of 11 months and one patient demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). 27696380
Unknown unknown Advanced Solid Tumor not applicable Ibrutinib + unspecified PD-L1 antibody Preclinical Actionable In a preclinical study, the combination treatment of Imbruvica (ibrutinib) and an anti-PD-L1 antibody in mouse models with advanced solid tumors resulted in antitumor efficacy, including decreased tumor size, minimized metastasis, and improved survival in triple-receptor negative breast cancer mouse models, and a 30% cure rate and improved survival in colon cancer mouse models (PMID: 25730880). 25730880
Unknown unknown 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 gastric adenocarcinoma not applicable Capecitabine + Oxaliplatin + Toripalimab Phase II Actionable In a Phase II trial, Toripalimab (JS001) in combination with Xeloda (capecitabine) and Eloxatin (oxaliplatin) resulted in an objective response rate of 54.5% (18/33) and a disease control rate of 84.8% (28/33) in patients with metastatic gastric adenocarcinoma, with a median duration of response of 8.3 months (J Clin Oncol 38: 2020 (suppl; abstr e15083); NCT02915432). detail...
Unknown unknown peritoneum cancer not applicable Bevacizumab + Carboplatin + Paclitaxel FDA approved Actionable In a Phase III trial (GOG-0218) that supported FDA approval, addition of Avastin (bevacizumab) during and after Paraplatin (carboplatin) plus Taxol (paclitaxel) treatment prolonged progression-free survival (14.1 vs 10.3 months) compared to placebo in patients with previously untreated, stage III/IV epithelial ovarian, primary peritoneal, or fallopian-tube cancer (PMID: 22204724; NCT00262847). detail... 22204724
Unknown unknown ovarian cancer not applicable Carboplatin + Cediranib Phase III Actionable In a Phase III clinical trial, the addition of Cediranib (AZD-2171) to platinum-based chemotherapy, including Paraplatin (carboplatin)-based therapy, followed by Cediranib (AZD-2171) maintenance therapy, resulted in an improved median progression-free survival of 11 months versus 8.7 months with platinum-based therapy plus placebo in platinum-sensitive ovarian cancer patients (PMID: 27025186). 27025186
Unknown unknown Ewing sarcoma not applicable Carfilzomib + Selinexor Preclinical - Cell culture Actionable In a preclinical study, the combination of Selinexor (KPT-330) and Kyprolis (carfilzomib) resulted in downregulation of Birc5 (Survivin) and enhanced induction of apoptotic markers compared to Selinexor (KPT-330) alone, and worked synergistically to decrease viability of Ewing sarcoma cells in culture (PMID: 28314790). 28314790
Unknown unknown breast cancer not applicable Gedatolisib Preclinical - Cell line xenograft Actionable In a preclinical study, Gedatolisib (PF-05212384) suppressed phosphorylation of PI3K/mTOR effectors and induced apoptosis in human breast cancer cell lines with elevated PI3K/mTOR signaling in culture and in cell line xenograft models (PMID: 21325073). 21325073
Unknown unknown Advanced Solid Tumor not applicable CA-170 Phase I Actionable In a Phase I trial, CA-170 demonstrated safety and preliminary efficacy in patients with advanced solid tumors, resulting in peripheral T cell activation (Ann Oncol. 2017 Sep 18; 28 (Suppl_5): Abstract 1141PD; NCT02812875). detail...
Unknown unknown melanoma not applicable Cobimetinib Preclinical - Cell line xenograft Actionable In a preclinical study, Cobimetinib (GDC-0973) induced cell death in several human melanoma cell lines in culture and inhibited tumor growth in xenograft models (PMID: 22084396). 22084396
Unknown unknown hepatocellular carcinoma not applicable Torin 2 Preclinical Actionable In a preclinical study, Torin 2 inhibited proliferation of hepatocellular cells in culture (PMID: 26239364). 26239364
Unknown unknown 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 renal cell carcinoma not applicable Gemcitabine + MU380 Preclinical - Cell culture Actionable In a preclinical study, the addition of MU380 resulted in increased sensitivity to Gemzar (gemcitabine) in a renal cell carcinoma cell line in culture, leading to decreased proliferation (PMID: 28619751). 28619751
Unknown unknown thyroid gland cancer not applicable Bevacizumab + Cetuximab Preclinical Actionable In a preclinical study, Erbitux (cetuximab), in combination with Avastin (bevacizumab), inhibited tumor growth and angiogenesis in mouse models of anaplastic thyroid cancer (PMID: 17429874). 17429874
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 lung non-small cell carcinoma no benefit Sorafenib Phase III Actionable In a Phase III trial (MISSION), Nexavar (sorafenib) treatment in non-small cell lung carcinoma patients did not reach its primary endpoint, resulting in an overall survival similar to that when treated with placebo (8.2 vs 8.3 mo, HR=0.99, p=0.47), however, did meet its secondary endpoint, demonstrating a greater progression free survival (2.8 vs 1.4 mo, HR=0.61, p<0.0001) and time to progression (2.9 vs 1.4 mo, HR=0.54, p<0.0001) when compared to placebo (PMID: 26743856; NCT00863746). 26743856
Unknown unknown triple-receptor negative breast cancer not applicable Senexin B Preclinical - Cell line xenograft Actionable In a preclinical study, Senexin B prevented tumor growth and enhanced the effects of Adriamycin (doxorubicin) in cell line xenograft models of triple-receptor negative breast cancer (Cancer Res January 1, 2015 75; PR08). detail...
Unknown unknown uveal melanoma not applicable Pegilodecakin Phase I Actionable In a Phase I trial, treatment with AM0010 at the active dose resulted in partial response in 21% (5/24) of evaluable advanced solid tumor patients, including 1 uveal melanoma patient who also demonstrated reduction of gastric metastases (PMID: 27528724; NCT02009449). 27528724
Unknown unknown multiple myeloma not applicable Lenalidomide + SJB3-019A Preclinical - Patient cell culture Actionable In a preclinical study, the combination of SJB3-019A and Revlimid (lenalidomide) worked synergistically to induce cytotoxicity in multiple myeloma cell lines and primary multiple myeloma cells in culture (PMID: 28270494). 28270494
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 CH5132799 Phase I Actionable In a Phase I trial, CH5132799 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 25231405). 25231405
Unknown unknown 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 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 mantle cell lymphoma not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in partial response in 17% (1/6) and stable disease in 67% (4/6) of patients with mantle cell lymphoma (PMID: 29475723; NCT01767766). 29475723
Unknown unknown cervical cancer not applicable BIRB-796 + Tozasertib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Tozasertib (VX-680) and BIRB-796 resulted in increased growth inhibition and induction of apoptosis in cervical cancer cell lines in culture, and increased tumor growth inhibition in cervical cancer cell line xenograft models, compared to either agent alone (PMID: 27082306). 27082306
Unknown unknown fibrous histiocytoma not applicable Anlotinib Phase II Actionable In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 58%, median progression-free survival of 4.1 months, an objective response rate of 5.3% (n=18), and a median overall survival of 11 months in patients with undifferentiated pleomorphic sarcoma (PMID: 29895706; NCT01878448). detail... 29895706
Unknown unknown Advanced Solid Tumor not applicable Ipatasertib + Paclitaxel Phase I Actionable In a Phase Ib trial, the combination of Ipatasertib (GDC-0068) and Taxol (paclitaxel) demonstrated safety in patients with advanced solid tumors, and resulted in an overall response rate of 8.0% (2/25), including partial responses in two patients, stable disease in 56.0% (14/25) of patients, a six-month progression-free survival rate of 12.0% (3/25), and maximum progression-free survival duration of 14 months in a breast cancer patient (PMID: 32205017; NCT01362374). 32205017
Unknown unknown prostate cancer not applicable DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, prostate cancer cells treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture and inhibition of tumor growth in cell-line xenograft models (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown Her2-receptor positive breast cancer not applicable Abemaciclib + Lapatinib Preclinical - Cell culture Actionable In a preclinical study, the addition of Abemaciclib (LY2835219) to Tykerb (lapatinib) treatment enhanced growth inhibitory effects of Tykerb (lapatinib)-resistant ERBB2 (HER2)-receptor positive breast cancer cells in culture (PMID: 26977878). 26977878
Unknown unknown neuroendocrine tumor not applicable Axitinib Phase II Actionable In a Phase II clinical trial, treatment with Inlyta (axitinib) resulted in a median overall progression-free survival (PFS) of 26.7 months, a 12-month PFS rate of 74.5%, and median overall survival of 45.3 months, and led to partial response in 3% (1/30) and stable disease in 70% (21/30) of patients with neuroendocrine tumors, with some tumor shrinkage in 68% (15/22) of patients (PMID: 27080472). 27080472
Unknown unknown Advanced Solid Tumor not applicable Adavosertib + Olaparib Phase Ib/II Actionable In a Phase Ib trial, Adavosertib (MK-1775) and Lynparza (olaparib) combination therapy resulted in partial response in 17% (1/6) and stable disease in 67% (4/6) of patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 5562)). detail...
Unknown unknown Advanced Solid Tumor not applicable PF-06647263 Phase I Actionable In a Phase I trial, PF-06647263 treatment resulted in partial response in 10% (5/48) of patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2511)). detail...
Unknown unknown hepatocellular carcinoma not applicable RU-A1 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with RU-A1 in hepatocellular carcinoma cells resulted in decreased cell survival and migration in culture and reduced tumor growth and improved survival in cell line xenograft zebrafish models (PMID: 28589491). 28589491
Unknown unknown triple-receptor negative breast cancer not applicable BAY1217389 + Paclitaxel Preclinical Actionable In a preclinical study, BAY1217389, in combination with Taxol (paclitaxel), had increased efficacy in inhibiting tumor growth in xenograft models of triple-negative breast cancer, compared to Taxol (paclitaxel) alone (PMID: 26832791). detail... 26832791
Unknown unknown 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 prostate cancer no benefit ASG-5ME Phase I Actionable In a Phase I trial, ASG-5ME demonstrated preliminary efficacy in patients with metastatic castration-resistant prostate cancer, but was not developed further due to unfavorable pharmacokinetic and toxicity profile (PMID: 30725389). 30725389
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 gastrointestinal stromal tumor not applicable Dasatinib Phase 0 Actionable In a clinical trial, Sprycel (dasatinib) treatment resulted in a 6-month progression-free survival (PFS) rate of 29% (n=48), median PFS of 2.9 months, median overall survival of 19 months, and partial response in 25% (12/48) of patients with Gleevec (imatinib)-resistant gastrointestinal stromal tumor (PMID: 29710216). 29710216
Unknown unknown colorectal carcinoma not applicable Camrelizumab Phase I Actionable In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity, resulted in partial response in 33.3% (1/3) of patients with colorectal carcinoma (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). detail...
Unknown unknown stomach cancer not applicable Capecitabine + Lapatinib Phase II Actionable In a Phase II trial, Tykerb (lapatinib) and Xeloda (capecitabine) combination treatment resulted in partial response in 17.9% (12/67) and stable disease in 46.3% (31/67) of gastric cancer patients regardless of their ERBB2 (HER2) status, although increased Erbb3 (Her3) expression level correlated with higher response rate (PMID: 27325685). 27325685
Unknown unknown glioblastoma multiforme not applicable Adavosertib Phase 0 Actionable In a Phase 0 trial, Adavosertib (MK-1775) demonstrated safety and preliminary efficacy, resulted in good brain tumor penetration and Wee1 pathway inhibition in intraoperatively collected tumor samples from patients with first-recurrence glioblastoma (PMID: 29798906). 29798906
Unknown unknown acute myeloid leukemia not applicable Daunorubicin + Venetoclax Preclinical - Patient cell culture Actionable In a preclinical study, Venclexta (venetoclax) and Daunorubicin combination treatment synergistically induced cell death in patient-derived acute myeloid leukemia cells in culture (PMID: 27103402). 27103402
Unknown unknown 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 acute myeloid leukemia not applicable A-366 Preclinical - Cell line xenograft Actionable In a preclinical study, A-366 inhibited proliferation and reduced cell viability in an acute myeloid leukemia (AML) cell line in culture, and inhibited tumor growth in an AML cell line xenograft model (PMID: 26147105). 26147105
Unknown unknown acute myeloid leukemia not applicable AZD1897 + Capivasertib Preclinical Actionable In a preclinical study, acute myeloid leukemia cells treated with the combination of AZD1897 and AZD5363 produced a synergistic effect, resulting in decreased cell survival (PMID: 24975213). 24975213
Unknown unknown pancreatic cancer not applicable SRT3025 Preclinical - Cell line xenograft Actionable In a preclinical study, SRT3025 decreased viability of human pancreatic cancer cell lines in culture and inhibited tumor growth in pancreatic cancer cell line xenograft models (PMID: 26655844). 26655844
Unknown unknown colon cancer not applicable WANT3 Preclinical - Cell culture Actionable In a preclinical study, WANT3 treatment resulted in suppression of cell invasion of colon cancer cells in culture (PMID: 27432794). 27432794
Unknown unknown follicular lymphoma not applicable Tafasitamab-cxix Phase II Actionable In a Phase II trial, follicular lymphoma patients tolerated Monjuvi (tafasitamab-cxix) treatment, and achieved an overall response rate of 29% (10/34, 3 complete and 7 partial response) with 4 patients responding over 12 months, a disease control rate of 76% (26/34), progression-free survival of 8.8 months at a median follow-up of 21 months, and a median duration of response was not reached (PMID: 29444231; NCT01685008). 29444231
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 lung squamous cell carcinoma not applicable Cisplatin + Gemcitabine + Necitumumab FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Portrazza (necitumumab), in combination with gemcitabine and cisplatin, resulted in an increased median overall survival of 11.5 months in squamous NSCLC patients, compared to 9.9 months with gemcitabine and cisplatin alone (PMID: 26045340). detail... 26045340
Unknown unknown chronic lymphocytic leukemia not applicable Cerdulatinib + Venetoclax Preclinical - Patient cell culture Actionable In a preclinical study, the combination of Cerdulatinib (PRT062070) and Venclexta (venetoclax) worked synergistically to induce apoptosis of patient-derived chronic lymphocytic leukemia cells in culture, and resulted in decreased cell viability compared to either drug as a single agent (PMID: 27697994). 27697994
Unknown unknown Advanced Solid Tumor not applicable NSC156529 Preclinical Actionable In a preclinical study, NSC156529 inhibited growth of transformed human cell lines in culture (PMID: 26294745). 26294745
Unknown unknown uveal melanoma not applicable IDE196 Phase I Actionable In a Phase I trial, LXS196 demonstrated safety and preliminary efficacy, resulted in partial response in 9% (6/66) and stable disease in 68% (45/66) of patients with metastatic uveal melanoma (AACR Annual Meeting 2019, Abstract CT068). detail...
Unknown unknown lung non-small cell carcinoma not applicable Erlotinib + Everolimus Phase I Actionable In a Phase I trial, Afinitor (everolimus) demonstrated safety and some efficacy in combination with Tarceva (erlotinib) in patients with advanced NSCLC (PMID: 22968184). 22968184
Unknown unknown 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 chronic leukemia not applicable RG7112 Phase I Actionable In a Phase I clinical trial, 5% (1/19) of chronic leukemia patients achieved partial response, and 79% (15/19) achieved stable disease following treatment with RG7112 (PMID: 26459177). 26459177
Unknown unknown 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 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 pancreatic cancer not applicable Gemcitabine + Nimotuzumab Phase II Actionable In a Phase IIb trial, the combination of Nimotuzumab and Gemzar (gemcitabine) resulted in improved median overall survival (8.6 mo vs. 6.0 mo), overall survival rate at 12 months (34% vs. 19%) and median progression-free survival (5.1 mo vs. 3.4 mo) compared to Gemzar (gemcitabine) plus placebo in pancreatic cancer patients, with patients with wild-type KRAS demonstrating an overall survival rate at 12 months of 53.8% vs. 27.8% in patients harboring KRAS mutations (PMID: 28961832). 28961832
Unknown unknown Burkitt lymphoma not applicable CAR.k.28 cells Preclinical - Cell culture Actionable In a preclinical study, patient-derived CAR.k.28 cells when co-cultured with a Kappa-positive Burkitt lymphoma cell line induced tumor cell lysis in culture, and inhibited tumor growth in a cell line xenograft model (PMID: 16926291). 16926291
Unknown unknown colorectal cancer not applicable 2141 V-11 Preclinical - Cell line xenograft Actionable In a preclinical study, intratumoral injection of 2141 V-11 stimulated anti-tumor immune response, resulted in reduced tumor burden and prolonged survival in a cell line xenograft model of colorectal cancer (PMID: 30297432). 30297432
Unknown unknown sarcoma not applicable Conatumumab + Ganitumab Phase Ib/II Actionable In a Phase Ib/II trial, Ganitumab and Conatumumab (AMG 655) combination treatment resulted in stable disease in 33% (5/15) of patients with sarcoma, including one with leiomyosarcoma (PMID: 24816908). 24816908
Unknown unknown Advanced Solid Tumor not applicable MVAp53 + Pembrolizumab Phase I Actionable In a Phase I trial, MVAp53 and Keytruda (pembrolizumab) combination therapy demonstrated safety and preliminary efficacy, resulting in a clinical response in 27.3% (3/11) of patients with advanced solid tumors, who remained with stable disease for up to 49 weeks (PMID: 30094792). 30094792
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 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 lymphoma not applicable SB 11285 Preclinical Actionable In a preclinical study, treatment with SB 11285 delayed and inhibited tumor growth in a syngeneic mouse model of lymphoma ournal of Clinical Oncology 2017 35:15_suppl, e14616). 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 lung non-small cell carcinoma not applicable CCT244747 + Gemcitabine Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of CCT244747 and Gemzar (gemcitabine) slowed tumor growth in non-small cell lung cancer cell line xenograft models (PMID: 22929806). 22929806
Unknown unknown malignant peripheral nerve sheath tumor no benefit Ganetespib + Sirolimus Phase II Actionable In a Phase II trial, treatment with the combination of Ganetespib and Rapamune (sirolimus) did not result in clinical benefit in 10 patients with malignant peripheral nerve sheath tumor (PMID: 32089640; NCT02008877). 32089640
Unknown unknown Advanced Solid Tumor not applicable Onalespib Phase I Actionable In a Phase I trial, AT13387 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 25336693). 25336693
Unknown unknown Advanced Solid Tumor not applicable Linsitinib Phase I Actionable In a Phase I trial, Linsitinib (OSI-906) was well-tolerated and resulted in stable disease in 41% (27/66) of patients with an advanced solid tumor and a partial response in two patients with adrenocortical carcinoma (PMID: 25208878). 25208878
Unknown unknown colon adenocarcinoma not applicable Capecitabine + Lapatinib Phase II Actionable In a Phase II trial, Tykerb (lapatinib) combined with Xeloda (capecitabine) demonstrated safety, but failed to show efficacy in patients with advanced refractory colorectal cancer (PMID: 22811876). 22811876
Unknown unknown chronic lymphocytic leukemia not applicable Ibrutinib + Venetoclax Phase II Actionable In a Phase II trial, Imbruvica (ibrutinib) and Venclexta (venetoclax) combination therapy resulted in a response rate of 100% (14/14, 9 complete response, 5 partial response) in relapsed or refractory chronic lymphocytic leukemia (CLL) patients, and a response rate of 100% (16/16, 9 complete response, 7 partial response) in untreated patients with high-risk features including del 17p, TP53 mutations, and del 11q (ASH, 59th Annual Meeting and Exposition, Dec 2017, Abstract 429; NCT02756897). detail...
Unknown unknown 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 breast cancer not applicable AsiDNA + Talazoparib Preclinical - Cell culture Actionable In a preclinical study, AsiDNA and Talzenna (talazoparib) combination treatment resulted in increased cell death and inhibition of proliferation in breast cancer cell lines compared to Talzenna (talazoparib) alone in culture (PMID: 27559053). 27559053
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 CDX-1140 + CDX-301 Phase I Emerging In a Phase I trial, CDX-1140 and CDX-301 combination treatment demonstrated safety and expected lymphocyte activation and cytokine responses in patients with advanced solid tumors (AACR Annual Meeting 2019, Abstract LB_194; NCT03329950). detail...
Unknown unknown Advanced Solid Tumor not applicable Riviciclib Phase I Actionable In a Phase I study, P276-00 demonstrated safety and some preliminary efficacy in patients with advanced solid tumors ( J Clin Oncol (Meeting Abstracts) June 2007 vol. 25 no. 18_suppl 14117). detail...
Unknown unknown Advanced Solid Tumor not applicable Ganetespib Preclinical - Cell line xenograft Actionable In a preclinical study, Ganetespib demonstrated potent anti-tumor effects against a variety of advanced solid tumor cell types and in cell line xenograft models (PMID: 22144665). 22144665
Unknown unknown 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 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 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 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 Advanced Solid Tumor not applicable Gemcitabine + Itacitinib + Nab-paclitaxel Phase Ib/II Actionable In a Phase Ib/II trial, Itacitinib (INCB039110) in combination with nab-paclitaxel and gemcitabine demonstrated safety and preliminary efficacy, resulted in an overall response rate of 24% (13/55, all partial responses) in patients with advanced solid tumors (PMID: 30115734; NCT01858883). 30115734
Unknown unknown triple-receptor negative breast cancer not applicable Navitoclax + Paclitaxel Preclinical - Cell culture Actionable In a preclinical study, the combination of Navitoclax (ABT-263) 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 cutaneous T cell lymphoma not applicable Mogamulizumab Phase III Actionable In a Phase III trial, Mogamulizumab treatment resulted in significant improvement in progression-free survival (7.7 vs 3.1 months, HR=0.53) and overall response rate (28.0% vs 4.8%) compared to Zolinza (vorinostat) in patients with cutaneous T cell lymphoma (Blood 2017 130(Suppl 1):817; NCT01728805). detail...
Unknown unknown colon cancer not applicable Ch282-5 Preclinical - Cell line xenograft Actionable In a preclinical study, ch282-5 induced apoptosis and inhibited growth and migration of several human and mouse colon cancer cell lines in culture, and inhibited tumor growth and metastasis in xenograft models (PMID: 26515494). 26515494
Unknown unknown biliary tract cancer not applicable M7824 Phase I Actionable In a Phase I trial, Bintrafusp alfa (M7824) treatment in patients with advanced biliary tract cancer resulted in an objective response rate of 20% (6/30), with an additional six patients demonstrating stable disease, and median progression-free survival of 2.5 months, and median overall survival of 12.7 months (PMID: 32461347; NCT02699515). 32461347
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 lung cancer not applicable Cediranib + Gefitinib Phase I Actionable In a Phase I trial, the combination of Cediranib and Iressa (gefitinib) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, including a partial response in a patient with lung cancer (PMID: 20061136). 20061136
Unknown unknown multiple myeloma not applicable Bortezomib + Daratumumab + Melphalan + Prednisone FDA approved Actionable In a Phase III trial (ALCYONE) that supported FDA approval, the combination of Darzalex (daratumumab), Velcade (bortezomib), Alkeran (melphalan), and Adasone (prednisone) resulted in improved 18-month progression-free survival rate (71.6% vs 50.2%, HR=0.50, p<0.001) and overall response rate (90.9% vs 73.9%, p<0.001) compared to control in newly diagnosed multiple myeloma patients ineligible for autologous stem-cell transplantation (PMID: 29231133; NCT02195479). detail... 29231133
Unknown unknown sarcoma not applicable NBTXR3 + Radiotherapy Phase II Actionable In a Phase II/III trial, no difference in objective response rate was observed between soft tissue sarcoma patients treated with NBTXR3 plus radiotherapy vs. radiotherapy alone (7% vs. 10%, p=0.86), however, NBTXR3 plus radiotherapy resulted in an increased pathological complete response rate of 16% (14/87) vs. 8% (7/89), and a higher proportion of patients receiving NBTXR3 and radiotherapy demonstrated negative margins (84% (61/73) vs. 70% (57/82), p=0.30) (PMID: 31296491; NCT02379845). 31296491
Unknown unknown 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 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 Advanced Solid Tumor no benefit Trametinib + Uprosertib Phase I Actionable In a Phase I trial, the combination of Trametinib (GSK1120212) and Uprosertib (GSK2141795) was not well-tolerated and resulted in minimal efficacy in patients with advanced solid tumors (n=126), demonstrating an objective response rate of less than 5%, with one complete response and five partial responses (PMID: 32062691). 32062691
Unknown unknown pancreatic ductal adenocarcinoma not applicable Selumetinib + SHP099 Preclinical - Pdx & cell culture Actionable In a preclinical study, the combination of SHP099 and Selumetinib (AZD6244) resulted in greater sensitivity compared to either agent alone in pancreatic ductal adenocarcinoma cells, demonstrating decreased cell viability and reduced colony formation in culture and decreased tumor growth in patient-derived xenograft (PDX) models (PMID: 30045908). 30045908
Unknown unknown 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 (PMI