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| Profile Name | Unknown unknown |
| Gene Variant Detail | |
| Relevant Treatment Approaches |
| Molecular Profile | Indication/Tumor Type | Response Type | Relevant Treatment Approaches | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|---|
| Unknown unknown | Advanced Solid Tumor | not applicable | Cabozantinib | Phase II | Actionable | In a Phase II clinical trial, Cometriq (cabozantinib) showed safety and anti-tumor activity in several advanced solid tumor types (J Clin Oncol 29: 2011 (suppl; abstr 3010)). | detail... | |
| Unknown unknown | Merkel cell carcinoma | not applicable | Pembrolizumab | FDA approved | Actionable | In a Phase II trial (KEYNOTE-017) that supported FDA approval, Keytruda (pembrolizumab) treatment resulted in a confirmed objective response rate of 50% (21/42, complete response 19%, partial response 31%) in patients with advanced Merkel cell carcinoma naive to systemic therapy (J Clin Oncol 36, no. 15_suppl (May 20 2018) 9506-9506; NCT02267603). | detail... detail... | |
| 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 | prostate adenocarcinoma | not applicable | VT-464 | Phase I | Actionable | In a Phase I study, VT-464 treatment led to safety and tolerability in patients with castration-resistant prostate adenocarcinoma and resulted in a best response of stable disease in 58% (10/17), a partial response in 6% (1/17), and no complete responses (PMID: 30012563; NCT02361086). | 30012563 | |
| Unknown unknown | myeloid neoplasm | not applicable | Bortezomib + Dexamethasone + Lenalidomide | Phase III | Actionable | In a Phase III trial, Velcade (bortezomib) in combination with Revlimid (lenalidomide) and dexamethasone resulted in an improved median progression-free survival of 43 months compared to 30 months with Revlimid (lenalidomide) plus dexamethasone, and a response rate of 81.5% (176/216) compared to 71.5% (153/214) with Revlimid (lenalidomide) plus dexamethasone in myeloma patients (PMID: 28017406). | 28017406 | |
| Unknown unknown | 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 | lung non-small cell carcinoma | not applicable | A-1331852 + Docetaxel | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of A-1331852 and Taxotere (docetaxel) inhibited tumor growth in non-small cell lung cancer cell line xenograft models, with increased efficacy over either agent alone (PMID: 25787766). | 25787766 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Sacituzumab govitecan-hziy | Phase Ib/II | Actionable | In a Phase I/II trial (IMMU-132-01), Trodelvy (sacituzumab govitecan-hziy) demonstrated safety and preliminary efficacy, resulted in a partial response in 8% (2/25) and stable disease in 64% (16/25) of patients with advanced solid tumors, with a time-to-progression of 3.6 months (PMID: 25944802; NCT01631552). | 25944802 | |
| Unknown unknown | breast cancer | not applicable | Alisertib + Fulvestrant | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with the combination of Alisertib (MLN8237) and Faslodex (fulvestrant) resulted in increased growth inhibition in aromatase inhibitor-resistant breast cancer cell lines compared to either agent alone (PMID: 25667100). | 25667100 | |
| Unknown unknown | follicular lymphoma | not applicable | Hu5F9-G4 + Rituximab | Phase Ib/II | Actionable | In a Phase Ib study, combined Hu5F9-G4 and Rituxan (rituximab) therapy demonstrated safety and efficacy, resulting in an objective response rate of 71% (5/7, 3 complete and 2 partial responses) in patients with follicular lymphoma, and a median duration of response longer than 6 months (PMID: 30380386). | 30380386 | |
| Unknown unknown | breast cancer | not applicable | Thymoquinone | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Thymoquinone (TQ) inhibited growth, migration, and invasive behavior of human breast cancer cell lines in culture, and inhibited tumor growth and metastasis in mouse breast cancer cell line xenografts (PMID: 26023736). | 26023736 | |
| Unknown unknown | B-cell lymphoma | not applicable | WM-8014 | Preclinical - Cell culture | Actionable | In a preclinical study, WM-8014 inhibited proliferation of a mouse B-cell lymphoma cell line in culture (PMID: 30069049). | 30069049 | |
| 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 | diffuse large B-cell lymphoma | not applicable | Mivavotinib | Phase I | Actionable | In a Phase I trial, Mivavotinib (TAK-659) treatment resulted in an objective response rate of 28% (12/43, 8 complete responses, 4 partial responses) in patients with relapsed or refractory diffuse large B-cell lymphoma, with a median duration of response not reached (PMID: 32327472; NCT02000934). | 32327472 | |
| Unknown unknown | thyroid gland cancer | not applicable | SL327 + Sunitinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of SL327 and Sutent (sunitinib) worked additively to decrease viability, induce apoptosis, and decrease migration of Taxotere (docetaxel)-resistant anaplastic thyroid cancer cell lines in culture, and to inhibit tumor growth in xenograft models (PMID: 28178630) | 28178630 | |
| Unknown unknown | renal cell carcinoma | no benefit | MK2206 | Phase II | Actionable | In a Phase II trial, MK2206 treatment resulted in shorter progression free survival compared to Afinitor (everolimus) (3.68 vs 5.98 months) in patients with renal cell carcinoma (PMID: 28049139). | 28049139 | |
| Unknown unknown | ovarian carcinoma | not applicable | Gemcitabine + MU380 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the addition of MU380 resulted in increased sensitivity to Gemzar (gemcitabine) in a ovarian carcinoma cell line, resulting in decreased proliferation in culture and improved survival in xenograft models (PMID: 28619751). | 28619751 | |
| 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 | chondrosarcoma | not applicable | Alisertib | Phase II | Actionable | In a Phase II trial, Alisertib (MLN8237) treatment resulted in partial response in a dedifferentiated chondrosarcoma patient (PMID: 27502708). | 27502708 | |
| Unknown unknown | endometrial cancer | not applicable | Adavosertib + Paclitaxel | Preclinical | Actionable | In a preclinicals study, Adavosertib (MK-1775) in combination with Paclitaxel, demonstrated efficacy in endometrial cancer cells (PMID: 24381593). | 24381593 | |
| Unknown unknown | chronic myeloid leukemia | not applicable | Ponatinib | FDA approved | Actionable | In a Phase II clinical trial which supported FDA approval, Iclusig (ponatinib) was effective in promoting disease regression in 52% of patients with accelerated phase chronic myeloid leukemia, 31% of patients with blast phase chronic myeloid leukemia, and 41% of patients with Philadelphia chromosome positive acute lymphoblastic leukemia (PMID: 23935038). | 23935038 detail... | |
| Unknown unknown | chronic myeloid leukemia | not applicable | Ponatinib | Clinical Study | Actionable | In a meta-analysis, Iclusig (ponatinib) treatment was associated with increased rate of major molecular response compared with Gleevec (imatinib) (Odds Ratio (OR): 4.95 [0.97-25.19]), but not improved overall survival (OR: 2.00 [0.21-19.33]), and was associated with increased risk of vascular occlusive events (OR: 3.47 [1.23-9.78]) in patients with chronic myeloid leukemia (PMID: 26847662). | 26847662 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Selumetinib | Phase I | Actionable | In a Phase I trial, treatment with Selumetinib (AZD6244) demonstrated safety and preliminary efficacy patients with advanced solid tumors, with several patients achieving prolonged stable disease (PMID: 18390968). | 18390968 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Selumetinib | Clinical Study | Actionable | In a meta-analysis, Selumetinib (AZD6244) was shown to have modest clinical efficacy as a monotherapy in a variety of solid tumors (PMID: 24716986). | 24716986 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | RO4987655 | Phase I | Actionable | In a Phase I trial, RO4987655 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22767668). | 22767668 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | RO4987655 | Phase I | Actionable | In a Phase I trial, RO4987655 demonstrated safety and preliminary clinical activity in Japanese patients with advanced solid tumors, including 1 partial response in a patient with esophageal cancer and 7 patients achieving progression-free survival for greater than 16 weeks (PMID: 25809858). | 25809858 | |
| Unknown unknown | cervical cancer | not applicable | PRGN-2009 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, PRGN-2009 treatment increased tumor infiltration of T-lymphocytes and decreased tumor growth in a cell line xenograft model of HPV16-positive cervical cancer (J Immunol 2020, 204 (1 Supplement) 91.6). | detail... | |
| Unknown unknown | lymphoma | not applicable | Cyclophosphamide + SB 11285 | Preclinical | Actionable | In a preclinical study, Cytoxan (cyclophosphamide) and SB 11285 combination treatment delayed and inhibited tumor growth in a syngeneic mouse model of lymphoma (Journal of Clinical Oncology 2017 35:15_suppl, e14616). | detail... | |
| 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 | gastric adenocarcinoma | not applicable | Rivoceranib | Phase III | Actionable | In a Phase III trial, Apatinib (YN968D1) treatment significantly improved median overall survival (6.5 vs 4.7 months) and median progression-free survival (2.6 vs 1.8 months) compared to placebo in chemotherapy-refractory patients with advanced gastric or gastroesophageal junction adenocarcinoma (PMID: 26884585). | 26884585 | |
| Unknown unknown | 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 | 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 | synovial sarcoma | not applicable | Pazopanib | Clinical Study | Actionable | In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 16.4 weeks and median survival of 10.6 months in patients with synovial sarcoma (PMID: 26970174). | 26970174 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Rituximab + Urelumab | Phase I | Actionable | In a Phase I trial, Urelumab (BMS-663513) and Rituxan (rituximab) combination therapy demonstrated manageable safety profile, resulted in an objective response rate of 10% (3/29) and a disease control rate of 24% (7/29) in patients with relapsed or refractory diffuse large B-cell lymphoma, with a median progression-free survival of 9.0 weeks and a median overall survival of 23.9 weeks (PMID: 32052473; NCT01775631). | 32052473 | |
| Unknown unknown | glioblastoma multiforme | not applicable | G-TPP + Navitoclax | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Navitoclax (ABT-263) synergistically inhibited viability of established lines and patient-derived glioblastoma cells in culture, and inhibited tumor growth in cell line and patient-derived xenograft models (PMID: 28522750). | 28522750 | |
| Unknown unknown | 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 | prostate cancer | not applicable | Docetaxel + PF-00562271 | Preclinical | Actionable | In a preclinical study, PF-00562271 re-sensitized chemoresistant prostate cancer cells to Taxotere (docetaxel) in culture, resulting in decreased phosphorylation of Ptk2 (Fak) and increased autophagy (PMID: 24194567). | 24194567 | |
| 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 | B-cell lymphoma | not applicable | Denileukin diftitox + Sirolimus | Preclinical - Cell culture | Actionable | In a preclinical study, Rapamune (sirolimus) enhanced the efficacy of Ontak (denileukin diftitox) in human B-cell lymphoma cells, resulting in decreased cell viability in culture (PMID: 27737881). | 27737881 | |
| Unknown unknown | 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 | 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 | stomach cancer | not applicable | Bevacizumab | Phase III | Actionable | In a Phase III trial, addition of Avastin (bevacizumab) to chemotherapy consisted of fluoropyrimidine and cisplatin resulted in improved median overall survival (12.1 vs 10.1 months), median progression-free survival (6.7 vs 5.3 months) and overall response rate (46.0% vs 37.4%) compared to chemotherapy alone in gastric cancer patients (PMID: 21844504). | 21844504 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CC-115 | Phase I | Actionable | In a Phase Ia/Ib trial, CC-115 treatment was tolerated and demonstrated manageable safety, and resulted in an overall response rate of 5.1% (n=39; 1 complete response, 1 partial response, 18 stable disease), and a disease control rate of 51.3% in advanced solid tumor patients (PMID: 31853198; NCT01353625). | 31853198 | |
| 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 | head and neck squamous cell carcinoma | not applicable | Temsirolimus | Phase II | Actionable | In a Phase II trial, treatment with Torisel (temsirolimus) resulted in disease stabilization in 57.6% (19/33) and tumor shrinkage in 39.4% (13/33) of patients with head and neck squamous cell carcinoma (PMID: 25527417). | 25527417 | |
| Unknown unknown | breast cancer | not applicable | AZD8186 | Preclinical - Cell culture | Actionable | In a preclinical study, AZD8186 inhibited proliferation of several breast cancer cell lines in culture (PMID: 25398829). | 25398829 | |
| Unknown unknown | myelodysplastic syndrome | not applicable | FF-10501-01 | Phase I | Actionable | In a Phase I trial, FF-10501-01 demonstrated preliminary efficacy, resulting in a partial remission in 10% (2/20) of heavily pretreated patients with myelodysplastic syndrome, however, Phase 2a of the trial was closed due to increased toxicity (PMID: 32264726; NCT02193958). | 32264726 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | CAR.k.28 cells | Preclinical - Cell culture | Actionable | In a preclinical study, patient-derived CAR.k.28 cells when co-cultured with either autologous or allogenic Kappa-positive B-cell chronic lymphocytic leukemia cells induced cell death in culture (PMID: 16926291). | 16926291 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | PF-04691502 | Phase I | Actionable | In a Phase I trial, PF-04691502 demonstrated safety and efficacy in patients with advanced solid tumors (PMID: 24395457). | 24395457 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Pamiparib + Tislelizumab | Phase Ib/II | Actionable | In a Phase Ib trial, the combination therapy of Tislelizumab (BGB-A317) and Pamiparib (BGB-290) in patients with advanced solid tumors was well-tolerated and resulted in an objective response in 20% (10/49), with two complete responses and eight partial responses, stable disease in 32% (16/49), a disease control rate of 53% (26/49), and a clinical benefit of 39% (PMID: 31378459; NCT02660034). | 31378459 | |
| 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 | leiomyosarcoma | not applicable | Regorafenib | Phase II | Actionable | In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (3.7 vs 1.8 months), but no difference in overall survival (HR=0.50, p=0.056) compared to placebo in patients with leiomyosarcoma (PMID: 27751846). | 27751846 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | PU-H71 | Phase I | Actionable | In a Phase I trial, treatment with PU-H71 was generally well-tolerated, and resulted in stable disease as best response in 35% (6/14) patients with advanced solid tumors (PMID: 28808818; NCT01581541). | 28808818 | |
| Unknown unknown | ovary epithelial cancer | not applicable | Afuresertib + Carboplatin + Paclitaxel | Phase I | Actionable | In a Phase Ib trial, the combination of Afuresertib (GSK2110183) , Paraplatin (carboplatin), and Taxol (paclitaxel) was well-tolerated and demonstrated preliminary efficacy in patients with platinum-resistant epithelial ovarian cancer, resulting in an overall response rate in the dose-expansion part of the trial (Part II) of 32% (9/28) and a median progression-free survival of 7.1 months (PMID: 30563934; NCT01653912). | 30563934 | |
| Unknown unknown | 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 | stomach cancer | not applicable | HD105 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, HD105 inhibited tumor progression in two of four human gastric cancer cell line xenograft models (PMID: 27049350). | 27049350 | |
| Unknown unknown | 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 | 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 | breast cancer | not applicable | MCLA-145 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MCLA-145 treatment inhibited tumor growth and induced regression in a cell line xenograft model of breast cancer engrafted with human CD34-positive cells (Cancer Res 2019;79(13 Suppl):Abstract nr 539). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | SGN-CD228A | Preclinical - Pdx | Actionable | In a preclinical study, SGN-CD228A treatment delayed tumor growth and resulted in complete responses in patient-derived xenograft (PDX) models of non-small cell lung carcinoma (Cancer Res 2019;79(13 Suppl):Abstract nr 2688). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BAY1161909 | Preclinical | Actionable | In a preclinical study, BAY1161909 inhibited proliferation of a variety of human solid tumor cell lines in culture (PMID: 26832791). | detail... 26832791 | |
| Unknown unknown | stomach cancer | not applicable | IGM-8444 | Preclinical - Pdx | Actionable | In a preclinical study, IGM-8444 treatment induced tumor regression in a patient-derived xenograft (PDX) model of gastric cancer (Journal of Clinical Oncology 2020 38:15_suppl, 3595). | detail... | |
| 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 | head and neck squamous cell carcinoma | no benefit | Cetuximab + Cisplatin + Patritumab | Phase Ib/II | Actionable | In a Phase Ib clinical trial, combined Erbitux (cetuximab), Patritumab (U3-1287), and Platinol (cisplatin) treatment was tolerable in patients with recurrent/metastatic head and neck squamous cell carcinoma and resulted in a 47% (7/15) overall response rate (3 complete responses and 4 partial responses), stable disease in 53% (8/15) of patients, a 7.9-month median progression-free survival, and a 13.5-month overall survival (PMID: 30327312; NCT02350712). | 30327312 | |
| Unknown unknown | head and neck squamous cell carcinoma | no benefit | Cetuximab + Cisplatin + Patritumab | Phase II | Actionable | In a Phase II trial, the combination of Patritumab (U3-1287) with Erbitux (cetuximab) and Platinol (cisplatin) or Paraplatin (carboplatin) in patients with recurrent or metastatic head and neck squamous cell carcinoma was well-tolerated, but resulted in a similar median progression-free survival (5.6 mo vs 5.5 mo) and median overall survival (10.0 mo vs 12.7 mo) when compared to chemotherapy alone (PMID: 31648099; NCT02633800). | 31648099 | |
| Unknown unknown | colorectal cancer | not applicable | Danusertib | Phase II | Actionable | In a Phase II clinical trial, Danusertib (PHA-739358) showed limited efficacy in patients with metastatic colorectal cancer (J Clin Oncol 28, 2010 (suppl; abstr e13558)). | detail... | |
| Unknown unknown | lung non-squamous non-small cell carcinoma | not applicable | Carboplatin + Motesanib Diphosphate | 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 | AsiDNA | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA inhibited survival of melanoma cell lines in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | transitional cell carcinoma | no benefit | Docetaxel + Icrucumab | Phase II | Actionable | In a Phase II trial, Icrucumab (IMC-18F1) and Taxotere (docetaxel) combination treatment did not result in improved median progression-free survival (1.6 months) when compared to Taxotere (docetaxel) single treatment (2.8 months) in urothelial carcinoma patients (PMID: 26926681). | 26926681 | |
| Unknown unknown | sarcoma | not applicable | Carotuximab + Pazopanib | Phase Ib/II | Actionable | In a Phase I/II trial, TRC105 and Votrient (pazopanib) combination therapy resulted in a median progression free survival of 3.95 months and ongoing complete response in 4% (3/81) of soft tissue sarcoma patients (J Clin Oncol 34, 2016 (suppl; abstr 11016)). | detail... | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Intuvax | Phase I | Actionable | In a Phase I trial, Intuvax (Ilixadencel) alone or in combination with Nexavar (sorafenib) demonstrated safety in hepatocellular carcinoma patients, and resulted in one partial response (Intuvax monotherapy), stable disease in 5 patients, increased circulating tumor-specific CD8-positive T-cells in 82% (9/11) of patients receiving Intuvax alone and 50% (2/4) of patients also receiving Nexavar, median time to progression of 5.5 months, and median overall survival of 7.5 months (PMID: 30719425; NCT01974661). | 30719425 | |
| Unknown unknown | glioblastoma multiforme | not applicable | JR-AB2-011 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, JR-AB2-011 resulted in tumor growth inhibition, increased apoptotic activity, and improved survival of glioblastoma cell line xenograft models (PMID: 28453552). | 28453552 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Metformin | Clinical Study | Actionable | In a meta-analysis, Glucophage (metformin) treatment decreased recurrence and metastasis and improved overall survival of head and neck squamous cell carcinoma patients (PMID: 25636350). | 25636350 | |
| Unknown unknown | gastric adenocarcinoma | not applicable | Irinotecan + Minnelide | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Minnelide and Camptosar (irinotecan) combination treatment resulted in increased inhibition of tumor growth in a cell line xenograft model of gastric adenocarcinoma compared to either Minnelide or Camptosar (irinotecan) treatment alone (PMID: 28192510). | 28192510 | |
| 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 | gastric adenocarcinoma | not applicable | Paclitaxel + Tegafur-gimeracil-oteracil Potassium | Phase III | Actionable | In a Phase III trial, combining intraperitoneal Taxol (paclitaxel) with TS-1 (tegafur-gimeracil-oteracil potassium) and Taxol did not show statistical improvement of overall survival, but did demonstrated clinical efficacy compared to TS-1 and cisplatin combination in gastric adenocarcinoma patients with peritoneal metastasis (J Clin Oncol 34, 2016 (suppl; abstr 4014)). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | SNS-510 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with SNS-510 decreased PDPK1 pathway signaling and proliferation of a diffuse large B-cell lymphoma cell line in culture (Mol Cancer Ther, Dec 1 2015 (14) (12 Supplement 2) C198). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Bosutinib | Phase I | Actionable | In a Phase I trial, Bosulif (bosutinib) demonstrated safety and limited efficacy in patients with advanced solid tumors (PMID: 22179664). | 22179664 | |
| Unknown unknown | colon cancer | not applicable | TPST-1495 + unspecified PD-1 antibody | Preclinical | Actionable | In a preclinical study, TPST-1495 in combination with an anti-PD-1 antibody synergistically inhibited tumor growth in a syngeneic mouse model of colon cancer (Journal for ImmunoTherapy of Cancer 2019;7:282, Abs nr: P311). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | LY3023414 | Phase I | Actionable | In a Phase I trial, LY3023414 demonstrated tolerability and inhibition of PI3K/mTOR signaling and resulted a disease control rate of 34.0% (16/47) in patients with advanced solid tumors, with one partial response and 15 patients achieving stable disease (PMID: 29636360; NCT01655225). | detail... 29636360 | |
| Unknown unknown | acute myeloid leukemia | not applicable | BI 836858 | Preclinical - Patient cell culture | Actionable | In a preclinical study, BI 836858 induced potent cytotoxic immune response against patient derived acute myeloid leukemia blast cells in culture (PMID: 27013443). | 27013443 | |
| Unknown unknown | pancreatic cancer | not applicable | Gemcitabine + Refametinib | Phase Ib/II | Actionable | In a Phase I/II trial, Refametinib (BAY86-9766) and Gemzar (gemcitabine) combination treatment resulted in an objective response rate of 23% and a disease control rate of 73% in patients with advanced pancreatic cancer (PMID: 27975152). | 27975152 | |
| Unknown unknown | 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 | mantle cell lymphoma | not applicable | Ibrutinib + Palbociclib | Phase I | Actionable | In a Phase I trial, Imbruvica (ibrutinib) and Ibrance (palbociclib) combination treatment resulted in an overall response rate of 67%, a complete response rate of 37%, and a two year progression-free survival in 59.4% of patients with previously treated mantle cell lymphoma, with a median follow up of 25.6 months (PMID: 30692121; NCT02159755). | detail... 30692121 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Chiauranib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Chiauranib (CS2164) inhibited tumor growth in a non-small cell lung cancer cell line xenograft model (PMID: 28004478). | 28004478 | |
| Unknown unknown | glioblastoma multiforme | not applicable | Durvalumab | Phase II | Actionable | In a Phase II trial, Imfinzi (durvalumab) treatment resulted in partial response in 16.7% (5/30), stable disease in 43.3% (13/30), and an estimated 6 month progression free survival of 20.0% in patients with Bevacizumab-naive, recurrent glioblastoma (Neuro Oncol (2016) 18 (suppl 6): vi18.). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | SNS-510 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with SNS-510 decreased PDPK1 pathway signaling and proliferation of multiple myeloma cell lines in culture (Mol Cancer Ther, Dec 1 2015 (14) (12 Supplement 2) C198). | detail... | |
| Unknown unknown | Indication other than cancer | not applicable | Sirolimus | FDA approved | Actionable | Rapamune (sirolimus) is FDA approved for use in patients with lymphangioleiomyomatosis and for prophylactic immunosuppression in renal transplant patients (FDA.gov). | detail... detail... | |
| Unknown unknown | glioblastoma multiforme | no benefit | Buparlisib | Preclinical | Actionable | In a preclinical study, treatment with BKM120 demonstrated a survival similar to vehicle in transgenic mouse models of glioblastoma and therefore, resulted in no benefit (PMID: 27199435). | 27199435 | |
| Unknown unknown | esophageal cancer | not applicable | Futibatinib | Phase I | Actionable | In a Phase I trial, TAS-120 treatment resulted in clinical response in an esophageal cancer patient (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 372PD). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | AZD5153 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AZD5153 inhibited proliferation of multiple myeloma cell lines harboring t (11;14) translocation in culture, resulted in tumor growth inhibition in cell line xenograft models (PMID: 27573426). | 27573426 | |
| Unknown unknown | 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 | melanoma | not applicable | CCG-203971 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a melanoma cell line treated with CCG-203971 resulted in inhibition of cell migration, invasion, and decreased cell growth in culture, and a reduced tumor burden in xenograft models (PMID: 27837031). | 27837031 | |
| Unknown unknown | follicular lymphoma | not applicable | Bendamustine + Bortezomib + Rituximab | Phase II | Actionable | In a Phase II trial, addition of Velcade (Bortezomib) to Bendamustine and Rituxan (rituximab) combination therapy resulted in improved complete response (74%, 63/85) compared to without Velcade (Bortezomib) (58%, 80/137) in high risk follicular lymphoma patients (J Clin Oncol 34, 2016 (suppl; abstr 7507)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Topotecan + Veliparib | Phase I | Actionable | In a Phase I trial, the combination of Veliparib (ABT-888) and topotecan demonstrated safety and tolerability, and resulted in an objective response rate of 10% (5/51; 1 complete response and 4 partial responses) and stable disease for at least 4 months in 43% (22/51) of patients with advanced solid tumors, with the majority of the patients having ovarian, fallopian tube, or primary peritoneal cancer (PMID: 29138343; NCT01012817). | 29138343 | |
| Unknown unknown | colon cancer | not applicable | DSP-0509 + unspecified PD-1 antibody | Preclinical | Actionable | In a preclinical study, an anti-PD-1 inhibitor combined with DSP-0509 inhibited tumor growth in a syngeneic mouse model of colon cancer (Cancer Res July 1 2018 (78) (13 Supplement) 4726). | detail... | |
| Unknown unknown | 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 | 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 | 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 | lung non-small cell carcinoma | not applicable | INCB059872 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, INCB059872 inhibited growth of non-small cell lung carcinoma cell lines in culture and in cell line xenoraft models (Cancer Res 2016;76(14 Suppl):Abstract nr 4704). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Urelumab | Phase I | Actionable | In a Phase I trial, Urelumab (BMS-663513) demonstrated manageable safety profile, resulted in an objective response rate of 6% (2/31) and a disease control rate of 19% (6/31) in patients with relapsed or refractory diffuse large B-cell lymphoma, with a median progression-free survival of 8.1 weeks and a median overall survival of 45.6 weeks (PMID: 32052473; NCT01471210). | 32052473 | |
| Unknown unknown | lung cancer | not applicable | TAK-960 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, lung 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 | Neratinib + Temsirolimus | Phase I | Actionable | In a Phase I study, Nerlynx (neratinib) administered with Torisel (temsirolimus) was tolerable and demonstrated antitumor activity in multiple solid tumor types, including breast cancer and NSCLC (PMID: 24323026). | 24323026 | |
| Unknown unknown | 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 | cervical cancer | not applicable | BMS-986205 + Nivolumab | Phase Ib/II | Actionable | In a Phase I/II trial, BMS-986205 in combination with Opdivo (nivolumab) resulted in an objective response rate of 14% (3/22) and a durable response rate of 64% (14/22) in patients with cervical cancer (PMID: 29167110). | 29167110 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CC-90010 | Phase I | Actionable | In a Phase I trial, CC-90010 treatment demonstrated safety, and resulted in an overall response rate of 2.9% (2/69, 1 complete response, 1 partial response), stable disease in 33.3% (23/69), and a median progression-free survival of 1.9 months in patients with advanced solid tumors or relapsed/refractory non-Hodgkin lymphoma (PMID: 32240793; NCT03220347). | 32240793 | |
| Unknown unknown | colorectal cancer | not applicable | Fluorouracil + Tanibirumab | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Tanibirumab (TTAC-0001) and Adrucil (fluorouracil) demonstrated enhanced inhibition of angiogenesis and tumor growth in colorectal cancer cell line xenograft models compared to either agent alone (PMID: 26325365). | 26325365 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Dostarlimab + TSR-033 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with TSR-033 and Dostarlimab (TSR-042) in combination resulted in increased T-cell number and increased T-cell proliferation in a non-small cell lung cancer cell line xenograft model and had an additive effect on tumor growth inhibition (PMID: 30587557). | 30587557 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CT-0508 | Preclinical - Pdx | Actionable | In a preclinical study, CT-0508 treatment resulted in prolonged survival in multiple patient-derived xenograft models of advanced solid tumors (Cancer Immunol Res 2020;8(3 Suppl):Abstract nr B65). | detail... | |
| Unknown unknown | lung cancer | not applicable | Rivoceranib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Apatinib (YN968D1) inhibited tumor growth in lung cancer cell line xenograft models (PMID: 21443688). | 21443688 | |
| 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 | 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 | malignant glioma | not applicable | Sunitinib | Phase II | Actionable | In a Phase II clinical trial, Sutent (sunitinib) was well-tolerated in young patients with high grade glioma, but did not demonstrate sufficient anti-tumor activity as a single agent, with no patients achieving a sustained objective response (PMID: 27109549). | 27109549 | |
| Unknown unknown | malignant glioma | not applicable | Sunitinib | Preclinical | Actionable | In a preclinical study, Sutent (sunitinb) induced cell death and decreased proliferation of glioma cells in culture (PMID: 25458015). | 25458015 | |
| Unknown unknown | gastrointestinal stromal tumor | not applicable | PKF118-310 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, PKF118-310 decreased viability of gastrointestinal stromal tumor (GIST) cell lines in culture, and reduced tumor growth in xenograft models (PMID: 28611108). | 28611108 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Docetaxel + MLN4924 | Phase I | Actionable | In a Phase Ib trial, the combination therapy of Pevonedistat (MLN4924) and Taxotere (docetaxel) was well-tolerated and demonstrated safety in advanced solid tumor patients, and resulted in an overall response rate of 16% (3/19), including a partial response in a cholangiocarcinoma patient and in two patients with head and neck cancer (PMID: 29781056; NCT01862328). | 29781056 | |
| Unknown unknown | clear cell renal cell carcinoma | not applicable | Ipilimumab + Nivolumab | Phase II | Actionable | In a Phase II trial (HCRN GU16-260), salvage therapy with combination of Yervoy (ipilimumab) and Opdivo (nivolumab) resulted in a partial response in 11% (3/27) and stable disease in 30% (8/27) of patients with advanced clear cell renal cell carcinoma whose disease progressed after Opdivo (nivolumab) monotherapy (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 5006-5006; NCT03117309). | detail... | |
| Unknown unknown | glioblastoma multiforme | not applicable | Imetelstat | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with imetelstat resulted in increased cell death in glioblastoma multiforme (GBM) cells in culture, and decreased tumor growth in xenograft models (PMID: 20048334). | 20048334 | |
| Unknown unknown | 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 | synovial sarcoma | not applicable | Regorafenib | Phase II | Actionable | In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (5.6 vs 1.0 months), but no difference in overall survival (HR=0.87, p=0.79) compared to placebo in patients with synovial sarcoma (PMID: 27751846). | 27751846 | |
| Unknown unknown | 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 | 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 | bladder carcinoma in situ | not applicable | Valrubicin | FDA approved | Actionable | In a Phase III clinical trial that supported FDA approval, treatment with Valstar (valrubicin) resulted in complete response in 21% (19/90) of patients with bacillus Calmette-Guerin (BCG)-refractory bladder carcinoma in situ (PMID: 10687972). | detail... 10687972 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Evofosfamide | Preclinical | Actionable | In a preclinical study, TH-302 inhibited growth of triple-receptor negative breast cancer cell lines under hypoxic conditions, regardless of their BRCA1 status (PMID: 25193512). | 25193512 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Marizomib | Phase I | Actionable | In a Phase I trial, Marizomib (NPI-0052) treatment resulted in stable disease of short duration in 29% (7/24) of patients with advanced solid tumors (PMID: 27117181). | 27117181 | |
| Unknown unknown | 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 | colorectal cancer | not applicable | Pegilodecakin | Phase I | Actionable | In a Phase I trial, AM0010 treatment in pancreatic cancer patients resulted in stable disease in 27% (4/15) of patients, one patient with progression free survival for greater than 6 months, and a median overall survival of 15.4 months (J Clin Oncol 34, 2016 (suppl; abstr 3082)). | detail... | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | STRO-001 | Preclinical - Cell culture | Actionable | In a preclinical study, STRO-001 inhibited growth of EBV-transformed chronic lymphocytic leukemia cells in culture (Blood 2016 128 (22):464). | detail... | |
| 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 | clear cell renal cell carcinoma | no benefit | Bevacizumab + Temsirolimus | Phase II | Actionable | In a Phase II clinical trial, treatment with the combination of Torisel (temsirolimus) and Avastin (bevacizumab) did prolong progression-free survival compared to treatment with Avastin (bevacizumab) as a single agent (7.6 months vs 7.4 months) in patients with renal clear cell carcinoma (PMID: 26077237). | 26077237 | |
| Unknown unknown | colorectal cancer | not applicable | Oxaliplatin + Resminostat | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Resminostat (4SC-201) and Eloxatin (oxaliplatin) induced apoptosis in a colorectal cancer (CRC) cell line and primary colon cancer cells in culture, and inhibited tumor growth in a CRC cell line xenograft model, with increased activity compared to either agent alone (PMID: 26831668). | 26831668 | |
| Unknown unknown | melanoma | not applicable | Hu3F8-BsAb | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Hu3F8-BsAb treatment induced cell killing in melanoma cell lines expressing GD2 in culture, and treatment with Hu3F8-BsAb plus human peripheral blood mononuclear cells inhibited tumor growth and improved survival in melanoma cell line xenograft models in combination with transplanted PBMCs (PMID: 25542634). | 25542634 | |
| Unknown unknown | melanoma | not applicable | Ipilimumab + Nivolumab | Phase II | Actionable | In a Phase II trial, the combination of Opdivo (nivolumab) and Yervoy (ipilimumab) resulted in improved overall response rate (ORR) and pathologic complete response rate (pCR) compared to Opdivo (nivolumab) monotherapy in patients with stage III or IV melanoma, with a ORR of 73% (8/11) and pCR of 45% (5/11), however, demonstrated higher toxicity (PMID: 30297909; NCT02519322). | 30297909 | |
| Unknown unknown | melanoma | not applicable | Ipilimumab + Nivolumab | FDA approved | Actionable | In a Phase III trial (CheckMate 067) that supported FDA approval, combination of Opdivo (nivolumab) and Yervoy (ipilimumab) and Opdivo (nivolumab) alone demonstrated improved efficacy compared to Yervoy (ipilimumab) in patients with untreated advanced melanoma, resulted in a median overall survival unreached in the combination arm, 36.9 and 19.9 months in nivolumab and ipilimumab monotherapy arms, and a median progression-free survival of 11.5, 6.9, and 2.9 months respectively (PMID: 30361170; NCT01844505). | detail... detail... 30361170 | |
| Unknown unknown | clear cell renal cell carcinoma | not applicable | Epacadostat + Pembrolizumab | Phase Ib/II | Actionable | In a Phase I/II trial, Epacadostat (INCB024360) and Keytruda (pembrolizumab) combination treatment resulted in complete response in 5% (1/19), partial response in 42% (8/19), and stable disease in 10% (2/19) of patients with advanced clear cell renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 4515)). | detail... | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Docetaxel + ONC201 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of ONC201 and Docefrez (docetaxel) worked synergistically to inhibit viability of triple-negative breast cancer cell lines in culture (PMID: 28424227). | 28424227 | |
| Unknown unknown | pancreatic cancer | not applicable | LOAd703 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, LOAd703 treatment reduced cell viability in pancreatic cancer lines in culture, and led to moderate decreased progression and reduced tumor growth in a pancreatic cancer cell line xenograft model (PMID: 28536305). | 28536305 | |
| 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 | breast adenocarcinoma | not applicable | Disarib | Preclinical | Actionable | In a preclinical study, Disarib treatment led to apoptotic induction and resulted in tumor regression in breast adenocarcinoma cell line mouse models (PMID: 27693384). | 27693384 | |
| Unknown unknown | malignant glioma | not applicable | PQ401 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, PQ401 disrupted cell migration and inhibited growth of glioma cells in culture, and suppressed tumor growth in cell line xenograft models (PMID: 25971682). | 25971682 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Talazoparib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the Talazoparib (BMN-673) selective PARP1/2 inhibitor demonstrated antitumor activity on a variety of cell lines and xenografts with defects in DNA repair (PMID: 23881923). | 23881923 | |
| Unknown unknown | 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 | endometrial carcinoma | not applicable | Lenvatinib + Pembrolizumab | FDA approved | Actionable | In a Phase II trial (Study 111/KEYNOTE-146) that supported FDA approval, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment resulted in an objective response rate of 35.6% (16/45) in patients with endometrial carcinoma that was not MSI-H or dMMR (PMID: 30922731; NCT02501096). | detail... detail... 30922731 | |
| Unknown unknown | ovarian cancer | not applicable | Cediranib | Phase III | Actionable | In a Phase III trial, Cediranib (AZD-2171) given with chemotherapy and as maintenance therapy resulted in improved median overall survival (27.3 vs 19.9 months) in platinum-sensitive ovarian cancer patients (J Clin Oncol 35, 2017 (suppl; abstr 5506)). | detail... | |
| Unknown unknown | lung small cell carcinoma | not applicable | STA-8666 | Preclinical - Pdx | Actionable | In a preclinical study, small cell lung cancer cell line xenograft and patient derived xenograft (PDX) models demonstrated stabilization of tumor growth and eventual tumor regression when treated with STA-8666 (PMID: 27267850). | 27267850 | |
| Unknown unknown | multiple myeloma | no benefit | DFRF4539A | Phase I | Actionable | In a Phase I trial, DFRF4539A demonstrated low activity in multiple myeloma patients, with partial responses in 5% (2/39), minimal response in 3% (1/39), stable disease in 46% (18/39), and progressive disease in 41% (16/39) of patients (PMID: 30718503; NCT01432353). | 30718503 | |
| 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 | 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 | 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 | colorectal cancer | not applicable | OSI-027 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, OSI-027 inhibited mTORC1 and mTORC2 signaling and growth in colorectal cancer cell line xenograft models (PMID: 21673091). | 21673091 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Carboplatin + MEDI-575 + Paclitaxel | Phase Ib/II | Actionable | In a Phase Ib/II trial, MEDI-575, Paraplatin (carboplatin), and Taxol (paclitaxel) combination treatment resulted in increased toxicity, and did not confer benefit over Paraplatin (carboplatin) plus Taxol (paclitaxel) in non-small cell lung carcinoma (NSCLC) patients (n=99), with a progression-free survival of 4.6 months vs. 5.5 months (HR=2.20, p=0.03), an overall response rate of 31.7% vs. 22.5% (p=0.49), and a median overall survival of 10 months vs. 11.8 months (HR=1.31) (PMID: 30685114). | 30685114 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | KU-0063794 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, KU-0063794 inhibited cell survival and reduced colony formation of hepatocellular carcinoma cells in culture, and inhibited tumor growth in cell line xenograft models (PMID: 26278819). | 26278819 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | 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 | prostate cancer | not applicable | RO6839921 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, RO6839921 demonstrated anti-tumor activity in prostate cancer cell line xenograft models (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A156). | detail... | |
| Unknown unknown | glioblastoma multiforme | not applicable | ST-11 | Preclinical | Actionable | In a preclinical study, ST-11 induced cell-cycle arrest and apoptosis in glioblastoma cell lines in culture, and reduced tumor burden in mouse models of glioblastoma (PMID: 27325686). | 27325686 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Zanubrutinib | FDA approved | Actionable | In a Phase II trial (BGB-3111-206) that supported FDA approval, Brukinsa (zanubrutinib) treatment resulted in an overall response rate of 83.5% (71/85, 50 complete response, 21 partial response) of patients with relapsed or refractory mantle cell lymphoma who received 1 to 4 prior treatments (Blood (2018) 132 (Supplement 1): 148; NCT03206970). | detail... detail... | |
| Unknown unknown | mantle cell lymphoma | not applicable | Zanubrutinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Brukinsa (zanubrutinib) inhibited BTK signaling and proliferation of mantle cell lymphoma cell lines in culture, and demonstrated antitumor activity in a mantle cell lymphoma cell line xenograft model (ibrutinib) (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 2597). | detail... | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Docetaxel + RhFzd7 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of RhFzd7 and Taxotere (docetaxel) resulted in increased inhibition of proliferation and decreased tumor growth and neovascularization compared to either agent alone in cell line xenograft models of triple-negative breast cancer (PMID: 30096373). | 30096373 | |
| Unknown unknown | brain glioma | not applicable | NT-I7 | Preclinical | Actionable | In a preclinical study, NT-I7 treatment improved survival in a mouse model of glioma (Journal of Clinical Oncology 37, no. 15_suppl, Abs nr: e13516). | detail... | |
| Unknown unknown | clear cell renal cell carcinoma | not applicable | Ascrinvacumab | Phase I | Actionable | In a Phase I trial, a patient with renal clear cell carcinoma demonstrated a partial response for 325 days when treated with PF-03446962 (PMID: 26655846). | 26655846 | |
| Unknown unknown | 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 | prostate cancer | not applicable | AZD1208 | Case Reports/Case Series | Actionable | In a Phase I trial, AZD1208 treatment resulted in considerable decrease of PSA levels in a patient with prostate cancer (PMID: 29765150; NCT01588548). | 29765150 | |
| Unknown unknown | prostate cancer | not applicable | AZD1208 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AZD1208 inhibited tumor growth in castrate-resistant prostate cancer cell line xenograft models (PMID: 25505253). | 25505253 | |
| Unknown unknown | head and neck squamous cell carcinoma | no benefit | Carboplatin + Cetuximab + Patritumab | Phase II | Actionable | In a Phase II trial, the combination of Patritumab (U3-1287) with Erbitux (cetuximab) and Platinol (cisplatin) or Paraplatin (carboplatin) in patients with recurrent or metastatic head and neck squamous cell carcinoma was well-tolerated, but resulted in a similar median progression-free survival (5.6 mo vs 5.5 mo) and median overall survival (10.0 mo vs 12.7 mo) when compared to chemotherapy alone (PMID: 31648099; NCT02633800). | 31648099 | |
| Unknown unknown | melanoma | not applicable | AGI-134 | Preclinical | Actionable | In a preclinical study, AGI-134 treatment induced tumor regression, reduced formation of secondary lesions and increased survival in mouse models of melanoma (PMID: 31889898). | 31889898 | |
| Unknown unknown | colon cancer | not applicable | Navicixizumab | Preclinical | Actionable | In a preclinical study, Navicixizumab (OMP-305B83) inhibited proliferation of endothelial cells in culture and demonstrated antitumor activity in vivo in multiple solid tumor types, including colon tumors (Mol Cancer Ther December 2015 14; C164). | detail... | |
| Unknown unknown | colon cancer | not applicable | AZD2811 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with AZD2811 nanoparticles resulted in tumor regression in colon cancer xenograft models (PMID: 26865565). | 26865565 | |
| Unknown unknown | ovary epithelial cancer | not applicable | Bevacizumab + Carboplatin + Paclitaxel | Phase II | Actionable | In a Phase II trial, addition of Avastin (bevacizumab) to neoadjuvant chemotherapy (Paraplatin (carboplatin) plus Taxol (paclitaxel)) did not improve complete macroscopic response rate (2/35 vs 2/33), or median progression free survival (20.4 vs 20.1 months) compared to control in patients with advanced epithelial ovarian cancer, but resulted in favorable rate of surgical feasibility (66.7 vs 88.6%) (J Clin Oncol 35, 2017 (suppl; abstr 5508)). | detail... | |
| Unknown unknown | ovary epithelial cancer | not applicable | Bevacizumab + Carboplatin + Paclitaxel | FDA approved | Actionable | In a Phase III trial (GOG-0218) that supported FDA approval, addition of Avastin (bevacizumab) during and after Paraplatin (carboplatin) plus Taxol (paclitaxel) treatment prolonged progression-free survival (14.1 vs 10.3 months) compared to placebo in patients with previously untreated, stage III/IV epithelial ovarian, primary peritoneal, or fallopian tube cancer (PMID: 22204724; NCT00262847). | detail... 22204724 | |
| Unknown unknown | ovarian clear cell carcinoma | not applicable | GDC-0980 | Phase I | Actionable | In a Phase I trial, Apitolisib (GDC-0980) treatment resulted in tumor regression by 48.3% in a patient with ovarian clear cell carcinoma (PMID: 26787751). | 26787751 | |
| Unknown unknown | acute myeloid leukemia | not applicable | CWP232291 | Phase I | Actionable | In a Phase I trial, CWP232291 demonstrated tolerability and limited preliminary efficacy in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS), resulting in one complete response and one partial response among 64 AML patients, while none of the five MDS patients achieved a response (PMID: 32396615; NCT01398462). | 32396615 | |
| Unknown unknown | colorectal cancer | not applicable | Fruquintinib | Phase III | Actionable | In a Phase III trial (FRESCO), treatment with Fruquitinib (HMPL-013) resulted in an improved median overall survival of 9.3 mo. vs. 6.57 mo. with placebo (HR=0.63), prolonged progression-free survival of 3.71 mo. vs. 1.84 mo. (HR=0.26), and an overall response rate (ORR) of 4.7% (13/278; 1 complete response, 12 partial responses) vs. 0% with placebo, in patients with metastatic colorectal cancer who had progressed on at least 2 prior chemotherapy regimens (PMID: 29946728; NCT02314819). | 29946728 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | RXDX-105 | Phase I | Actionable | In a Phase I clinical trial, RXDX-105 (CEP-32496) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, including 2 patients with heavily pretreated non-small cell lung cancer that achieved stable disease for greater that 6 months (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr C188). | detail... | |
| Unknown unknown | melanoma | 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 | TGX-221 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TGX-221 inhibited proliferation and decreased Akt phosphorylation in human cell line xenograft models of prostate cancer (PMID: 25620467). | 25620467 | |
| Unknown unknown | clear cell renal cell carcinoma | no benefit | Sorafenib + Temsirolimus | Phase II | Actionable | In a Phase II clinical trial, treatment with the combination of Nexavar (sorafenib) and Torisel (temsirolimus) did not prolong progression-free survival compared to treatment with Avastin (bevacizumab) monotherapy (7.4 months vs 7.5 months) in patients with renal clear cell carcinoma (PMID: 26077237). | 26077237 | |
| Unknown unknown | ovarian cancer | not applicable | Cisplatin + VE-821 | Preclinical - Cell culture | Actionable | In a preclinical study, VE-821 increased the sensitivity of an ovarian cancer cell line to Platinol (cisplatin) in culture (PMID: 25560806). | 25560806 | |
| 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 | leukemia | not applicable | Carboplatin + Topotecan + Veliparib | Phase I | Actionable | In a Phase I trial, the combination of Hycamtin (topotecan), Paraplatin (carboplatin), and Veliparib (ABT-888) resulted in an overall response rate of 33% (33/99) in leukemia patients and a response rate of 64% (14/22) in patients with aggressive myeloproliferative neoplasms or chronic myelomonocytic leukemia (PMID: 27551000). | 27551000 | |
| Unknown unknown | pancreatic adenocarcinoma | no benefit | Bevacizumab + Erlotinib + Gemcitabine | Phase III | Actionable | In a Phase III trial, treatment with the combination of Avastin (bevacizumab), Tarceva (erlotinib), and Gemzar (gemcitabine) did not result in a significant increase in OS in pancreatic adenocarcinoma patients compared to the combined treatment of Gemzar (gemcitabine), Tarceva (erlotinib), and placebo (PMID: 19307500). | 19307500 | |
| Unknown unknown | colorectal cancer | not applicable | YW3-56 | Preclinical | Actionable | In a preclinical study, YW3-56 inhibited proliferation of colorectal cancer cell lines in culture, independent of TP53 status (PMID: 25612620). | 25612620 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Pazopanib | Phase I | Actionable | In a Phase I trial, Votrient (pazopanib) demonstrated safety and efficacy in a variety of pediatric solid tumors (PMID: 23857966). | 23857966 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Pazopanib | Phase I | Actionable | In a Phase I study, Votrient (pazopanib) in combination with Taxol (paclitaxel) and Paraplatin (carboplatin) demonstrated efficacy in solid tumors (PMID: 22679111). | 22679111 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Abemaciclib | Preclinical | Actionable | In a preclinical study, Abemaciclib (LY2835219) reduced RB and AKT activation, and inhibited cell proliferation and colony formation in head and neck squamous cell carcinoma (HNSCC) cell lines in culture, and inhibited tumor growth in HNSCC xenograft models (PMID: 26909611). | 26909611 | |
| Unknown unknown | 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 | Hodgkin's lymphoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase I trial (CheckMate 039) that supported FDA approval, treatment with Opdivo (nivolumab) resulted in an objective response rate of 87% (20/23), with complete response in 17% (4/23) and partial response in 70% (16/23) of patients with relapsed or refractory classical Hodgkin's lymphoma (PMID: 25482239; NCT01592370). | 25482239 detail... | |
| Unknown unknown | 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 | colon cancer | not applicable | Ch282-5 + Oxaliplatin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of ch282-5 and Eloxatin (oxaliplatin) inhibited cell growth in human and mouse colon cancer cell lines in culture, and the combination synergized to inhibit tumor growth in mouse colon cancer cell line xenograft models (PMID: 26515494). | 26515494 | |
| Unknown unknown | colorectal cancer | not applicable | Resminostat | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Resminostat (4SC-201) inhibited proliferation and induced cell-cycle arrest and apoptosis in colorectal cancer (CRC) cell lines and primary colon cancer cells in culture, and inhibited tumor growth in a CRC cell line xenograft model (PMID: 26831668). | 26831668 | |
| Unknown unknown | 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 | gastric adenocarcinoma | not applicable | Triptolide | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with Triptolide (C1572) induced apoptotic cell death and resulted in decreased cell viability in gastric adenocarcinoma cell lines in culture (PMID: 28192510). | 28192510 | |
| Unknown unknown | breast cancer | not applicable | AJI-214 | Preclinical - Cell culture | Actionable | In a preclinical study, AJI-214 inhibited invasion and induced apoptosis of breast cancer cells in culture (PMID: 24930769). | 24930769 | |
| Unknown unknown | 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 | 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 | 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 | B-cell adult acute lymphocytic leukemia | not applicable | Inotuzumab ozogamicin | FDA approved | Actionable | In a Phase III trial supporting FDA approval, Besponsa (inotuzumab ozogamicin) treatment resulted in a significantly improved complete remission rate (80.7%, 88/109), duration of remission (4.6 months), progression free survival (5.0 months) and overall survival (7.7 months) in adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia when compared to standard of care (29.4%, 24/109, 3.1, 1.8, 6.7 months, respectively) (PMID: 27292104; NCT01564784). | detail... 27292104 | |
| Unknown unknown | mantle cell lymphoma | not applicable | STRO-001 | Preclinical - Cell culture | Actionable | In a preclinical study, STRO-001 potently inhibited growth of mantle cell lymphoma cell lines in culture (Blood 2016 128 (22):464). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ADG116 | Preclinical | Actionable | In a preclinical study, ADG116 demonstrated anti-tumor activity and increased overall survival in syngeneic mouse models of a variety of solid tumors (PMID: 31694708). | 31694708 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MGCD516 | Phase I | Actionable | In a Phase I trial, MGCD516 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2575)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MGCD516 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in a variety of human advanced solid tumor cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). | detail... | |
| Unknown unknown | spindle cell carcinoma | not applicable | LYC-55716 | Case Reports/Case Series | Actionable | In a Phase I trial, Cintirorgon (LYC-55716) treatment resulted in a partial response in a patient with metastatic spindle cell carcinoma, demonstrating decreased tumor burden (PMID: 30819679). | 30819679 | |
| Unknown unknown | glioblastoma multiforme | not applicable | AOH1160 | Preclinical - Patient cell culture | Actionable | In a preclinical study, AOH1160 inhibited growth of glioblastoma cells from patients in culture (PMID: 29967249). | 29967249 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Bevacizumab + Sorafenib | Phase I | Actionable | In a Phase I trial, the treatment combination of Avastin (bevacizumab) and Nexavar (sorafenib) in patients with advanced solid tumors resulted in stable disease in 25% (29/115) of patients and a partial response in 5% (6/115) of patients (PMID: 25363205). | 25363205 | |
| Unknown unknown | ovarian cancer | not applicable | C3742 + Cisplatin | Preclinical - Cell culture | Actionable | In a preclinical study, C3742 treatment enhanced the efficacy of Platinol (cisplatin) in wild-type TP53 ovarian cancer cells in culture, demonstrating greater inhibition of cell growth when compared to Platinol (cisplatin) alone (PMID: 22312557). | 22312557 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | SN30000 | Preclinical | Actionable | In a preclinical study, SN30000 inhibited growth of triple-receptor negative breast cancer cell lines under hypoxic culture conditions, regardless of their BRCA1 status (PMID: 25193512). | 25193512 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ME-344 | Phase I | Actionable | In a Phase I trial, ME-344 demonstrated preliminary tolerability and efficacy in patients with advanced solid tumors (PMID: 25411085). | 25411085 | |
| Unknown unknown | papillary renal cell carcinoma | not applicable | Abexinostat + Pazopanib | Phase Ib/II | Actionable | In a Phase Ib/II trial, 17% (1/6) of patients with papillary renal cell carcinoma demonstrated a response to the combination of Abexinostat (PCI-24781) and Votrient (pazopanib) (PMID: 28221861). | 28221861 | |
| 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 | renal cell carcinoma | not applicable | Axitinib + Pembrolizumab | FDA approved | Actionable | In a Phase III trial (KEYNOTE-426) that supported FDA approval, the combination therapy of Keytruda (pembrolizumab) and Inlyta (axitinib) resulted in a greater 12-month overall survival rate (89.9% vs 78.3%), median progression-free survival (15.1mo vs 11.1mo), and objective response rate (59.3% vs 35.7%) compared to treatment with Sutent (sunitinib) in patients with untreated advanced renal cell carcinoma (PMID: 30779529; NCT02853331). | detail... 30779529 | |
| Unknown unknown | urinary bladder cancer | not applicable | BMS-986205 + Nivolumab | Phase I | Actionable | In a Phase I/II trial, BMS-986205 in combination with Opdivo (nivolumab) resulted in an objective response rate of 32% (8/25) and a durable response rate of 44% (11/25) in patients with bladder cancer (PMID: 29167110). | 29167110 | |
| Unknown unknown | marginal zone B-cell lymphoma | not applicable | Tazemetostat | Case Reports/Case Series | Actionable | In a Phase I trial, Tazemetostat (EPZ-6438) demonstrated safety and preliminary efficacy, resulted in a partial response in 1 patient with relapsed or refractory marginal zone B-cell lymphoma (PMID: 29650362; NCT01897571). | 29650362 | |
| Unknown unknown | ovarian cancer | no benefit | Vandetanib | Phase II | Actionable | In a Phase II trial, treatment with Caprelsa (vandetanib) in ovarian cancer patients resulted in no benefit and led to early termination of the trial (PMID: 20068097). | 20068097 | |
| 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 | transitional cell carcinoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase II trial (CheckMate 275) that supported FDA approval, treatment with Opdivo (nivolumab) resulted in objective response in 19.6% (52/265) of urothelial carcinoma patients with prior platinum therapy, with complete response in 2% (6/265), and partial response in 17% (46/265) of patients (PMID: 28131785; NCT02387996). | detail... 28131785 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MEDI0639 | Phase I | Actionable | In a Phase I trial, MEDI0639 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 33, 2015 (suppl; abstr 3024)). | detail... | |
| Unknown unknown | Ewing sarcoma | no benefit | Pembrolizumab | Phase II | Actionable | In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in no objective response (0/13) in patients with Ewing sarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). | detail... | |
| Unknown unknown | breast cancer | not applicable | GDC-0349 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the mTOR inhibitor GDC-0349 inhibited tumor growth in cell line xenograft models of solid tumors, including breast cancer (PMID: 24900569). | 24900569 | |
| Unknown unknown | breast carcinoma | not applicable | MGCD516 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human breast carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | BI 836858 + Decitabine | Preclinical - Patient cell culture | Actionable | In a preclinical study, treatment of patient derived acute myeloid leukemia blast cells with Dacogen (decitabine) enhanced BI 836858-mediated cytotoxic immune response in culture (PMID: 27013443). | 27013443 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ASN003 | Phase I | Actionable | In a Phase I trial, ASN003 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 371PD; NCT02961283). | detail... | |
| Unknown unknown | 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 | Advanced Solid Tumor | not applicable | Aflibercept | Phase I | Actionable | In a Phase I trial, Zaltrap (aflibercept) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors (PMID: 20028764). | 20028764 | |
| Unknown unknown | renal carcinoma | no benefit | MG 98 | Phase II | Actionable | In a Phase II trial, MG 98 treatment in seventeen evaluable patients resulted in stable disease in six patients, progressive disease in nine patients, and no objective responses, and the trial was terminated early due to a lack of responses (PMID: 16502349). | 16502349 | |
| Unknown unknown | colon cancer | not applicable | Pracinostat | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Pracinostat (SB939) inhibited proliferation of colon cancer cell lines in culture, and increased histone H3 acetylation and inhibited tumor growth in a colon cancer cell line xenograft model (PMID: 20197387). | 20197387 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Berzosertib + Carboplatin | Phase I | Actionable | In a Phase I trial, Berzosertib (VX-970) and Paraplatin (carboplatin) combination treatment resulted in a best response of stable disease in 71% (15/21) of evaluable patients with advanced solid tumors, including 10 patients with stable disease for 4 months or more and 6 patients with stable disease for 6 months or more (PMID: 32568634; NCT02157792). | 32568634 | |
| Unknown unknown | marginal zone B-cell lymphoma | not applicable | Parsaclisib | Phase Ib/II | Actionable | In a Phase I/II trial, Parsaclisib (INCB050465) treatment demonstrated tolerability and preliminary activity in patients with refractory B-cell malignancies, and resulted in an overall response rate of 78% (7/9), complete response/complete metabolic response rate of 33% (3/9), and median duration of response of 4.4 months in patients with marginal zone lymphoma (PMID: 30803990; NCT02018861). | 30803990 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Erlotinib + Glesatinib | Phase I | Actionable | In a Phase I trial, Glesatinib (MGCD265) and Tarceva (erlotinib) combination therapy demonstrated safety and preliminary clinical efficacy, resulted in partial response in 1 patient, and stable disease for 6 cycles or more in 16% (7/45) of patients with advanced solid tumors (J Clin Oncol 30, 2012 (suppl; abstr e13602)). | detail... | |
| Unknown unknown | Sezary's disease | not applicable | Mogamulizumab | FDA approved | Actionable | In a Phase III trial (MAVORIC) that supported FDA approval, Poteligeo (mogamulizumab-kpkc) treatment resulted in significantly improved progression-free survival (7.7 vs 3.1 months, HR=0.53, p<0.0001) and objective response rate (28.0% vs 4.8%) compared to Zolinza (vorinostat) in patients with mycosis fungoides or Sézary syndrome (Blood 2017 130(Suppl 1):817). | detail... detail... | |
| Unknown unknown | 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 | 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 | 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 | head and neck squamous cell carcinoma | not applicable | Alpelisib + Cisplatin + Radiotherapy | Phase I | Actionable | In a Phase I trial, Piqray (Alpelisib) treatment in combination with Platinol (cisplatin) and radiotherapy demonstrated manageable safety, and resulted in an objective response rate of 66.7% (6/9) in head and neck squamous cell carcinoma patients, the 3-year progression-free survival (PFS) and overall survival (OS) were 77.8%, and the median progression-free survival and overall survival were not reached (PMID: 32464516; NCT02537223). | 32464516 | |
| Unknown unknown | colon cancer | not applicable | MPT0E028 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MPT0E028 decreased proliferation and increased apoptosis in a colon cancer cell line in culture and inhibited tumor growth in a colon cancer cell line xenograft model (PMID: 22928010). | 22928010 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | 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 | pancreatic endocrine carcinoma | no benefit | Dactolisib | Phase II | Actionable | In a Phase II clinical trial, BEZ235 was not well-tolerated and demonstrated modest anti-tumor activity in pancreatic neuroendocrine tumor patients who had progressed on Afinitor (everolimus), with a 51.6% (16/31) progression-free survival rate at 16 weeks (PMID: 26851029). | 26851029 | |
| Unknown unknown | 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 | oral cavity cancer | not applicable | Duvelisib + unspecified PD-L1 antibody | Preclinical | Actionable | In a preclinical study, treatment with the combination of low-dose Copiktra (duvelisib) and a PD-L1 antibody resulted in increased infiltration of CD8-positive tumor-infiltrating lymphocytes, decreased tumor volume, and improved survival in mice bearing T-cell inflamed murine oral cancer cell-derived tumors, when compared to either agent alone (PMID: 28364000). | 28364000 | |
| Unknown unknown | 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 | 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 | malignant pleural mesothelioma | not applicable | Cisplatin + Trabectedin | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Yondelis (trabectedin) and Platinol (cisplatin) demonstrated synergy in inducing apoptosis and decreasing viability of malignant pleural mesothelioma cell lines in culture (PMID: 27512118). | 27512118 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Everolimus | Phase Ib/II | Actionable | In a Phase Ib trial, Afinitor (everolimus) demonstrated safety and preliminary efficacy in patients with non-small cell lung cancer (PMID: 25673697). | 25673697 | |
| Unknown unknown | squamous cell carcinoma | not applicable | Paclitaxel + PMX-53 | Preclinical | Actionable | In a preclinical study, PMX-53 treatment enhanced the sensitivity of Taxol (paclitaxel) therapy, leading to a synergistic effect and resulting in inhibition of cell proliferation and tumor growth in a syngeneic mouse model of poorly differentiated squamous cell carcinoma (PMID: 30300579). | 30300579 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Cabozantinib + CT-707 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Cometriq (Cabometyx, cabozantinib) and CT-707 resulted in synergism in hepatocellular carcinoma cells, demonstrating increased apoptosis and inhibition of colony formation in culture and decreased tumor weight in xenograft models (PMID: 27638856). | 27638856 | |
| Unknown unknown | lung small cell carcinoma | no benefit | Vandetanib | Phase II | Actionable | In a Phase II trial, addition of Caprelsa (vandetanib) to platinum (cisplatin or carboplatin) and etoposide did not improve time to progression (5.62 vs 5.68 months) or overall survival (12.24 vs 9.23 months) compared to placebo in untreated extensive-stage small cell lung cancer (PMID: 27583688). | 27583688 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Gemcitabine + Trametinib | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of Mekinist (trametinib) and Gemzar (gemcitabine) demonstrated safety and preliminary anti-tumor activity in patients with advanced solid tumors (PMID: 23583440). | 23583440 | |
| Unknown unknown | colorectal cancer | not applicable | R11.1.6 | Preclinical - Cell culture | Actionable | In a preclinical study, R11.1.6 treatment did not inhibit cell proliferation and colony formation in a colorectal cancer cell line in culture (PMID: 29720559). | 29720559 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | CC-671 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, CC-671 induced apoptosis and inhibited growth of triple-negative breast cancer (TNBC) cell lines in culture, and inhibited tumor growth in TNBC cell line and patient-derived xenograft (PDX) models (PMID: 29866747). | 29866747 | |
| Unknown unknown | pancreatic endocrine carcinoma | not applicable | Nintedanib | Preclinical | Actionable | In a preclinical study, Ofev (nintedanib) induced tumor cell apoptosis, decreased microvessel density, inhibited tumor growth, and improved survival in transgenic mouse models of pancreatic neuroendocrine carcinoma (PMID: 26206868). | 26206868 | |
| Unknown unknown | 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 | osteosarcoma | not applicable | V158411 | Preclinical - Cell culture | Actionable | In a preclinical study, V158411 inhibited Chk1 autophosphorylation and induced DNA damage and apoptosis in an osteosarcoma cell line in culture (PMID: 27829224). | 27829224 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | PRI-724 | Phase I | Actionable | In a Phase I trial, Pri-724 displayed safety and preliminary efficacy in patients with a variety of advanced solid tumors (J Clin Oncol 31, 2013 (suppl; abstr 2501)). | detail... | |
| Unknown unknown | melanoma | not applicable | V158411 | Preclinical - Cell culture | Actionable | In a preclinical study, V158411 induced DNA damage and cell-cycle arrest, and inhibited growth of a melanoma cell line in culture (PMID: 27829224). | 27829224 | |
| Unknown unknown | prostate cancer | not applicable | CC-115 | Phase I | Actionable | In a Phase Ia/Ib trial, CC-115 treatment was tolerated and demonstrated manageable safety, and resulted in stable disease as the best response in 63.6% (7/11) of castration-resistant prostate cancer patients, and a median progression-free survival of 345 days (PMID: 31853198; NCT01353625). | 31853198 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Fostamatinib + Silmitasertib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Silmitasertib (CX-4945) and Fostamatinib (R788) worked synergistically to decrease cell viability and resulted in increased apoptosis and decreased AKT phosphorylation in ABC and GCB-type diffuse large B-cell lymphoma cell lines in culture (PMID: 28460620). | 28460620 | |
| 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 | acute lymphoblastic leukemia | not applicable | Zotiraciclib | Preclinical - Cell culture | Actionable | In a preclinical study, TG02 inhibited growth of acute lymphocytic leukemia cells in culture (PMID: 21860433). | 21860433 | |
| Unknown unknown | lung carcinoma | not applicable | SF1126 | Preclinical | Actionable | In a preclinical study, SF1126 decreased tumor immunosuppression and reduced tumor growth in syngeneic mouse lung carcinoma models (PMID: 31018997). | 31018997 | |
| Unknown unknown | pancreatic carcinoma | not applicable | ABT-348 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with Ilorasertib (ABT-348) inhibited tumor growth and led to regression of advanced tumors in cell line xenograft models of pancreatic carcinoma (PMID: 22935731). | 22935731 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | 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 | pancreatic cancer | not applicable | Gemcitabine + siG12D-LODER | Phase I | Actionable | In a Phase I/IIa trial, siG12D-LODER treatment in combination with concurrent chemotherapy, including Gemzar (gemcitabine), in patients with pancreatic cancer was well-tolerated and demonstrated safety, and resulted in a median overall survival of 15.12 months, a median time to metastasis of 8.25 months, and partial response in 25% (2/12) and stable disease in 75% (10/12) of patients (PMID: 26009994; NCT01188785). | 26009994 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | MLN0905 | Preclinical | Actionable | In a preclinical study, MLN0905 treatment resulted in decreased tumor volume in a diffuse large B-cell lymphoma xenograft model (PMID: 22609854). | 22609854 | |
| Unknown unknown | multiple myeloma | not applicable | INCB054329 + Ruxolitinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, INCB054329 and Jakafi (ruxolitinib) demonstrated synergy in a myeloma cell line in culture, resulting in decreased viability, increased apoptosis, and increased inhibition of STAT3 phosphorylation, and treatment with the combination of INCB054329 and Jakafi (ruxolitinib) resulted in increased efficacy in myeloma cell line xenograft models over either agent alone, with tumor regressions in 5/8 animals (PMID: 30206163). | 30206163 | |
| Unknown unknown | 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 | Hodgkin's lymphoma | not applicable | Edicotinib | Phase II | Actionable | In a Phase II/III clinical trial, JNJ-40346527 demonstrated safety some efficacy, with complete response in 5.0% (1/20), partial response in 5.0% (1/20), and stable disease in 55.0% (11/20) of patients with refractory Hodgkin's lymphoma (PMID: 25628399). | 25628399 | |
| Unknown unknown | stomach cancer | not applicable | Capecitabine + Nivolumab + Oxaliplatin + Tegafur-gimeracil-oteracil Potassium | Phase II | Actionable | In a Phase II trial (ATTRACTION-4), the combination therapy of Xeloda (capecitabine), Opdivo (nivolumab), Eloxatin (oxaliplatin), and TS-1 (tegafur-gimeracil-oteracil potassium) was well-tolerated and resulted in an objective response rate of 76.5% (13/17), a median progression-free survival of 10.6 months, and a median overall survival that was not yet reached in patients with either gastric cancer or gastroesophageal junction cancer (PMID: 30566590; NCT02746796). | 30566590 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Carboplatin + Paclitaxel + Temsirolimus | Phase II | Actionable | In a Phase II trial, the combination of Torisel (temsirolimus), Paraplatin (carboplatin), and Taxol (paclitaxel) resulted in an objective response rate of 41.7% (15/36), which included all partial responses, and 52.3% (19/36) had stable disease (PMID: 28961834). | 28961834 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | 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 | 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 | smoldering myeloma | not applicable | Lenalidomide + PVX-410 | Phase Ib/II | Actionable | In a Phase I/IIa clinical trial, combination therapy with PVX-410 and Revlimid (lenalidomide) was well-tolerated, and resulted in a partial response in 11% (1/9), minimal response in 44% (4/9), and stable disease in 44% (4/9) of patients with smoldering multiple myeloma with moderate/high risk of progression, and the magnitude of the immune response observed was significantly larger than in patients treated with PVX-410 alone (PMID: 30128502; NCT01718899). | 30128502 | |
| Unknown unknown | multiple myeloma | not applicable | Oprozomib | Phase II | Actionable | In a Phase II trial, Oprozomib (ONX 0912) treatment resulted in an objective response rate of 41.0%, 28.1%, and 25.0% in the 2/7, 240/300-mg/day; 5/14, 150/180-mg/day; and 5/14, 240-mg/day cohorts of patients with multiple myeloma (n=95) (PMID: 31142508; NCT01416428). | 31142508 | |
| Unknown unknown | renal cell carcinoma | no benefit | Pazopanib + Pembrolizumab | Phase I | Actionable | In a Phase I trial, Votrient (pazopanib) and Keytruda (pembrolizumab) combination treatment resulted in significant hepatotoxicity in patients with advanced renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 4506)). | detail... | |
| Unknown unknown | chronic myeloid leukemia | not applicable | Busulfan + Cyclophosphamide | FDA approved | Actionable | In a clinical trial that supported FDA approval, combination of Busulfex (busulfan) and Cytoxan (cyclophosphamide) as a conditioning regimen prior to bone marrow transplantation was better tolerated and resulted in favorable 4-year probabilities of survival and event-free survival (0.86 vs 0.72) compared to Cytoxan (cyclophosphamide) plus total body irradiation in patients with chronic myeloid leukemia (PMID: 8081005). | 8081005 detail... | |
| Unknown unknown | renal cell carcinoma | not applicable | Alpha 2 Interferon + Bevacizumab | FDA approved | Actionable | In a Phase III clinical trial that supported FDA approval, treatment with the combination of Avastin (bevacizumab) and Roferon (interferon alpha 2a) resulted in improved progression-free survival in patients with metastatic renal cell carcinoma compared to Roferon (interferon alpha 2a) with placebo (PMID: 20061402). | 20061402 detail... | |
| Unknown unknown | ovarian cancer | not applicable | Axitinib + Carboplatin + Paclitaxel | Phase I | Actionable | In a Phase I clinical trial, Inlyta (axitinib) in combination with paclitaxel and carboplatin, demonstrated safety and efficacy in patients with advanced solid tumors including ovarian cancers (PMID: 22990652). | 22990652 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Roniciclib | Phase I | Actionable | In a Phase I trial, treatment with Roniciclib (BAY 1000394) at the RP2D reduced PCNA expression and resulted in a disease control rate of 17.4% (n=23) in patients with small cell lung cancer (PMID: 28463960; NCT01188252). | 28463960 | |
| Unknown unknown | rhabdoid cancer | 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 | prostate cancer | not applicable | Enzalutamide | FDA approved | Actionable | In a Phase III trial (AFFIRM) that supported FDA approval, treatment with Xtandi (enzalutamide) resulted in improved median overall survival compared to placebo (18.4 vs 13.6 months HR=0.63, p<0.001) in patients with metastatic castration-resistant prostate cancer (PMID: 22894553; NCT00974311). | detail... 22894553 | |
| Unknown unknown | prostate cancer | not applicable | Enzalutamide | FDA approved | Actionable | In a Phase III trial (PROSPER) that supported FDA approval, Xtandi (enzalutamide) significantly improved median metastasis-free survival (36.6 vs 14.7 months, HR=0.29, p<0.0001) compared to placebo in patients with non-metastatic castration-resistant prostate cancer (Journal of Clinical Oncology 36, no. 6_suppl (February 20 2018) 3-3; NCT020032924). | detail... detail... | |
| Unknown unknown | prostate cancer | not applicable | Enzalutamide | FDA approved | Actionable | Ina Phase III trial (ARCHES) that supported FDA approval, Xtandi (enzalutamide) treatment with androgen deprivation therapy (ADT) significantly reduced the risk of radiographic progression or death compared to ADT plus placebo (HR=0.39, P < .001; median not reached vs 19.0 months) in patients with metastatic hormone-sensitive prostate cancer (PMID: 31329516; NCT02677896). | detail... 31329516 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Napabucasin | Phase I | Actionable | In a Phase I trial, BBI608 treatment demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 32:5s, 2014 (suppl; abstr 2546)). | detail... | |
| Unknown unknown | 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 | hepatocellular carcinoma | not applicable | Droxinostat | Preclinical - Cell culture | Actionable | In a preclinical study, droxinostat induced apoptosis and reduced growth of hepatocellular carcinoma cell lines in culture (PMID: 26947884). | 26947884 | |
| 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 | 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 | Waldenstroem's macroglobulinemia | not applicable | Oprozomib | Phase II | Actionable | In a Phase II trial, Oprozomib (ONX 0912) treatment resulted in an objective response rate of 71.4% and 47.1% in the 2/7 and 5/14 cohorts of patients with Waldenstroem's macroglobulinemia (n=31) (PMID: 31142508; NCT01416428). | 31142508 | |
| Unknown unknown | 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 | chronic lymphocytic leukemia | not applicable | ARQ 531 | Preclinical - Patient cell culture | Actionable | In a preclinical study, ARQ 531 treatment inhibited activation of BCR signaling and cell migration, and induced enhanced cytotoxicity in patient-derived chronic lymphocytic leukemia cells in culture, and increased survival in a transgenic mouse model (PMID: 30093506). | 30093506 | |
| Unknown unknown | urinary bladder cancer | not applicable | OBP-801 | Preclinical - Cell culture | Actionable | In a preclinical study, OBP-801 treatment resulted in decreased cell viability in multiple human bladder cancer cell lines in culture (PMID: 27406983). | 27406983 | |
| Unknown unknown | esophagus squamous cell carcinoma | not applicable | Camrelizumab | Phase I | Actionable | In a Phase I trial, Camrelizumab (SHR-1210) treatment resulted in an objective response rate of 33.3% (10/30, 1 complete response, 9 partial response) and a disease control rate of 56.7%, with a median progression free survival of 3.6 months in patients with advanced esophageal squamous cell carcinoma (PMID: 29358502; NCT02742935). | 29358502 | |
| Unknown unknown | colon carcinoma | not applicable | Mps-BAY1 | Preclinical | Actionable | In a preclinical study, Mps-BAY1 inhibited cell cycle progression and induced cell death of colon carcinoma cells in culture (PMID: 23933817). | 23933817 | |
| Unknown unknown | CLL/SLL | not applicable | Venetoclax | Phase I | Actionable | In a Phase I trial, Venclexta (venetoclax) treatment resulted in a 79% (92/116) overall response rate and 20% (23/116) complete response rate in patients with either chronic lymphocytic leukemia or small lymphocytic lymphoma (PMID: 26639348). | 26639348 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | Copanlisib + Refametinib | Phase I | Actionable | In a Phase I trial, Aliqopa (copanlisib) and Refametinib (BAY86-9766) combination treatment resulted in stable disease as best response in 33% (21/64) of patients with advanced solid tumors, however, the study failed to establish a tolerable and efficacious dose and schedule of this combination (PMID: 32314268; NCT01392521). | 32314268 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Docetaxel + TAK-243 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Taxotere (docetaxel) and TAK-243 (MLN7243) resulted in synergistic and additive effects, demonstrating anti-tumor activity in xenograft tumor models (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A164). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | BAY 1238097 | Preclinical | Actionable | In a preclinical study, BAY 1238097 inhibited tumor growth and was well tolerated in acute myeloid leukemia and multiple myeloma models (American Association for Cancer Research; 2015 Apr 18-22; Abstract nr 3524). | detail... | |
| Unknown unknown | colon cancer | not applicable | CVX-060 + Sunitinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of CVX-060 and Sutent (suntinib) resulted in increased tumor growth inhibition compared to either agent alone in a colon cancer cell line xenograft model (PMID: 21233403). | 21233403 | |
| Unknown unknown | melanoma | not applicable | DT01 + Radiotherapy | Phase I | Actionable | In a Phase I trial, DT01 treatment enhanced the sensitivity of radiotherapy, resulting in an objective response in 59% (45/76) of lesions from 21 melanoma patients, including 23 complete responses (PMID: 27140316; NCT01469455). | 27140316 | |
| Unknown unknown | melanoma | not applicable | CA-170 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CA-170 activated peripheral T cells and inhibited tumor growth in mouse models of melanoma (Ann Oncol. 2017 Sep 18; 28 (Suppl_5): Abstract 1141PD). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Defactinib | Phase I | Actionable | In a Phase I trial, Defactinib (VS-6063) treatment demonstrated safety in patient with advanced solid tumors (PMID: 26334219). | 26334219 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | GEN3009 | Preclinical - Patient cell culture | Actionable | In a preclinical study, GEN3009 treatment induced complement-dependent cytotoxicity in cells derived from chronic lymphocytic leukemia patients in culture (PMID: 32341336). | 32341336 | |
| Unknown unknown | peritoneal carcinoma | not applicable | Paclitaxel + TVB-2640 | Phase I | Actionable | In a Phase I trial, TVB-2640 and Taxol (paclitaxel) combination treatment resulted in partial response and a 58% reduction in CA-125 level in a peritoneal carcinoma patient (J Clin Oncol 34, 2016 (suppl; abstr 2512)). | detail... | |
| Unknown unknown | colon carcinoma | not applicable | BMS-906024 | Preclinical | Actionable | In a preclinical study, the pan-Notch inhibitor, BMS-906024, inhibited growth of colon carcinoma xenografts (PMID: 26005526). | 26005526 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Mogamulizumab + Nivolumab | Phase I | Actionable | In a Phase I trial, Poteligeo (mogamulizumab-kpkc) and Opdivo (nivolumab) combination treatment demonstrated acceptable safety, resulted in an objective response rate of 27% (4/15, 4 partial responses) and a disease control rate of 67% (10/15) in immunotherapy-naive patients with advanced or metastatic hepatocellular carcinoma, response occurred regardless of PD-L1, CCR4, CD8 expression levels and tumor mutational burden (PMID: 31455681; NCT02476123). | 31455681 | |
| Unknown unknown | meningioma | not applicable | AR-42 | Preclinical | Actionable | In a preclinical study, AR-42 induced apoptosis and inhibited proliferation of meningioma cells in culture (PMID: 21778190). | 21778190 | |
| Unknown unknown | melanoma | not applicable | Nivolumab + NKTR-214 | Phase Ib/II | Actionable | In a Phase I/II trial, NKTR-214 and Opdivo (nivolumab) combination treatment demonstrated safety and preliminary efficacy, resulted in radiographic response in one and complete response in another patient with melanoma (Journal of Clinical Oncology 35, no. 15_suppl; NCT02983045). | detail... | |
| Unknown unknown | pancreatic adenocarcinoma | 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 35% (6/17) of patients with pancreatic adenocarcinoma, two of whom had stable disease lasting greater than 150 days (PMID: 30756308; NCT01335269). | 30756308 | |
| Unknown unknown | follicular lymphoma | not applicable | Lenalidomide + Rituximab | FDA approved | Actionable | In a Phase III trial (AUGMENT) that supported FDA approval, Revlimid (lenalidomide) in combination with Rituxan (rituximab) resulted in significantly improved progression-free survival (39.4 vs 14.1 months, HR=0.46, p<0.001) compared to placebo and Rituxan (rituximab) in patients with relapsed and/or refractory follicular or marginal zone lymphoma (PMID: 30897038; NCT01938001). | 30897038 detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Vorolanib | Phase I | Actionable | In a Phase I trial, Vorolanib (X-82) was well tolerated and demonstrated preliminary efficacy, resulting in a complete response in 2% (1/49), a partial response in 2% (1/49), and stable disease in 51% (25/49) of patients with advanced solid tumors, with a median progression-free survival of 2 months (PMID: 30478190; NCT01296581). | 30478190 | |
| Unknown unknown | 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 | childhood B-cell acute lymphoblastic leukemia | not applicable | Tisagenlecleucel | FDA approved | Actionable | In a Phase II trial (ELIANA) that supported FDA approval, Kymriah (tisagenlecleucel) treatment led to complete remission or complete remission with incomplete blood count recovery in 83% (52/63) of pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (22nd Congress of EHA, June 2017, Abstract S476; NCT02435849). | detail... detail... detail... detail... | |
| Unknown unknown | ureter transitional cell carcinoma | not applicable | Mitomycin gel | FDA approved | Actionable | In a Phase III trial (OLYMPUS) that supported FDA approval, Jelmyto (mitomycin gel) treatment resulted in a complete response in 60% (41/68) of patients with low-grade upper tract urothelial cancer (J Urol. 2019 Apr; 201 (Supplement 4): abstract LBA-16; NCT02793128). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ravoxertinib | Phase I | Actionable | In a Phase I trial, Ravoxertinib (GDC-0994) demonstrated safety in patients with advanced solid tumors and resulted in stable disease in 33% (15/45) of patients, while two colorectal cancer patients harboring BRAF mutations (2/15) achieved partial responses lasting 21 and 73 weeks (PMID: 31848189; NCT01875705). | 31848189 | |
| 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 | Advanced Solid Tumor | not applicable | Navitoclax | Preclinical - Cell culture | Actionable | In a preclinical study, Navitoclax (ABT-263) treatment alone was not effective in a number of cell lines derived from solid tumors in culture (PMID: 27974663). | 27974663 | |
| Unknown unknown | 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 | prostate cancer | not applicable | AGS-PSCA | Phase Ib/II | Actionable | In a Phase Ib/II trial, AGS-PSCA treatment was deemed safe, but only resulted in limited antitumor activity in patients with castration resistant prostate cancer (PMID: 22553195). | 22553195 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | ASTX-660 + Paclitaxel | Preclinical - Cell line xenograft | Actionable | In a preclinical study, combination of ASTX-660 and Taxol (paclitaxel) treatment resulted in tumro regression and achieved partial response in cell line xenograft models of triple-receptor negative breast cancer (Cancer Res 2016;76(14 Suppl):Abstract nr 1287). | detail... | |
| Unknown unknown | 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 | 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 | colorectal cancer | not applicable | WAY-600 | Preclinical | Actionable | In a preclinical study, WAY-600 induced cell cycle arrest and inhibited cell proliferation of colorectal cancer cells in culture (PMID: 19584280). | 19584280 | |
| Unknown unknown | colorectal cancer | not applicable | Bleomycin + CBP501 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of Blenoxane (bleomycin) and CBP501 resulted in enhanced tumor growth inhibition in cell line xenograft models of colorectal cancer, compared to either agent alone (PMID: 17237275). | 17237275 | |
| Unknown unknown | breast cancer | not applicable | BO-112 | Preclinical - Cell culture | Actionable | In a preclinical study, BO-112 treatment induced cytotoxicity in human breast cancer cell lines and inhibited viability of mouse triple-negative breast cancer (TNBC) cells in culture, and intratumoral delivery of BO-112 reduced tumor growth in a syngeneic mouse model of TNBC (PMID: 31046839). | 31046839 | |
| Unknown unknown | 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 | 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 | hematologic cancer | not applicable | Domatinostat | Phase I | Actionable | In a Phase I trial, treatment with Domatinostat (4SC-202) demonstrated safety and resulted in stable disease in 75% (18/24) and objective response in 2 patients (1 complete response and 1 partial response) with advanced hematological malignancies (PMID: 30347469; NCT01344707). | 30347469 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Adavosertib + Gemcitabine | Phase I | Actionable | In a Phase I trial, the combination of Adavosertib (MK-1775) and Gemzar (gemcitabine) resulted in a partial response in 5% (4/81) of patients and stable disease in 53% (43/81) of patients, all with advanced solid tumors (PMID: 27601554). | 27601554 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Lenvatinib | Phase I | Actionable | In a Phase I clinical trial, Lenvima (lenvatinib) demonstrated anti-tumor activity in patients with several advanced solid tumor types, including patients with non-small cell lung cancer (PMID: 26169970). | 26169970 | |
| Unknown unknown | Ewing sarcoma | not applicable | CC-115 | Phase I | Actionable | In a Phase Ia/Ib trial, CC-115 treatment was tolerated and demonstrated manageable safety, and resulted in stable disease as the best response in 22.2% (2/9) of Ewing sarcoma patients, and a median progression-free survival of 56 days (PMID: 31853198; NCT01353625). | 31853198 | |
| 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 | melanoma | not applicable | A-674563 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a melanoma cell line demonstrated sensitivity to A-674563, resulting in apoptotic activity and inhibition of Akt1 activity in culture, and inhibition of tumor growth in xenograft models (PMID: 26970307). | 26970307 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | KHK2455 + Mogamulizumab | Phase I | Actionable | In a Phase I trial, the combination of KHK2455 and Poteligeo (mogamulizumab-kpkc) resulted in stable disease, according to RECIST, in four patients for more than 6 months and in one patient for greater than 14 months (J Clin Oncol 36, 2018 (suppl; abstr 3040). | detail... | |
| Unknown unknown | colorectal cancer | not applicable | Cabozantinib + SBI-0206965 | Preclinical - Cell culture | Actionable | In a preclinical study, Cometriq (Cabometyx, cabozantinib) treatment in combination with SBI-0206965 enhanced apoptosis in colorectal cancer cells in culture (PMID: 30026382). | 30026382 | |
| 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 | 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 | Ewing sarcoma | no benefit | Tranylcypromine | Preclinical - Cell culture | Actionable | In a preclinical study, Ewing sarcoma cell lines did not demonstrate sensitivity to Parnate (tranylcypromine) in culture (PMID: 29997151). | 29997151 | |
| Unknown unknown | acute promyelocytic leukemia | not applicable | Carfilzomib + Tinostamustine | Preclinical - Cell culture | Actionable | In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of an acute promyelocytic leukemia cell line in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Atezolizumab + GDC-0919 | Phase I | Actionable | In a Phase I trial, treatment with GDC-0919 and Tecentriq (atezolizumab) in combination was well-tolerated and demonstrated preliminary anti-tumor activity in patients with advanced solid tumors, with partial response in 9% (4/45) and stable disease in 24% (11/45) of patients (J Clin Oncol 35, 2017 (suppl; abstr 105)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Atezolizumab + GDC-0919 | Phase I | Actionable | In a Phase I trial, GDC-0919 (Navoximod) and Tecentriq (atezolizumab) combination therapy was well tolerated, resulted in stable disease as best response in 80% (8/10) of patients with advanced solid tumors (PMID: 31124055). | 31124055 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Decitabine + Onvansertib | Phase I | Actionable | In a Phase I trial, Onvansertib (PCM-075) and Dacogen (decitabine) combination therapy resulted in complete response in 33.3% (2/6) and complete response with incomplete hematologic remission in 16.7% (1/6) of evaluable patients with relapsed or refractory acute myeloid leukemia (Ann Oncol, 30 (Supplement 5): v435-v448, 2019; NCT03303339). | detail... | |
| Unknown unknown | stomach cancer | not applicable | RX-0201 | Preclinical | Actionable | In a preclinical study, RX-0201 prevented cell proliferation of stomach cancer cells (JASCO Annual Meeting Proceedings Vol 24, No 18S (June 20 Supplement), 2006: 13102). | detail... | |
| Unknown unknown | Advanced Solid Tumor | no benefit | AZD0424 | Phase I | Actionable | In a Phase I trial, AZD0424 treatment in patients with advanced solid tumors did not result in antitumor activity and is not recommended for further testing as a monotherapy in advanced solid tumor patients (PMID: 29438361). | 29438361 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Belinostat | Phase I | Actionable | In a Phase I trial, Beleodaq (belinostat) demonstrated safety and promoted stable disease in 39% (18/46) of patients with a variety of solid tumors (PMID: 18245542). | 18245542 | |
| Unknown unknown | prostate cancer | not applicable | Danusertib | Phase II | Actionable | In a Phase II clinical trial, Danusertib (PHA-739358) monotherapy was well tolerated, but showed minimal efficacy in patients with castration-resistant prostate cancer (PMID: 22928785). | 22928785 | |
| Unknown unknown | triple-receptor negative breast cancer | no benefit | CCT007093 | Preclinical | Actionable | In a preclinical study, CCT007093 did not inhibit growth of triple-receptor negative breast cancer cell lines in culture (PMID: 20576088). | 20576088 | |
| Unknown unknown | Burkitt lymphoma | not applicable | CAR.k.28 cells | Preclinical - Cell culture | Actionable | In a preclinical study, patient-derived CAR.k.28 cells when co-cultured with a Kappa-positive Burkitt lymphoma cell line induced tumor cell lysis in culture, and inhibited tumor growth in a cell line xenograft model (PMID: 16926291). | 16926291 | |
| Unknown unknown | lung 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 | thyroid gland cancer | not applicable | LY2874455 | Phase I | Actionable | In a Phase I trial, a patient with thyroid cancer demonstrated stable disease when treated with LY2874455 (PMID: 28589492). | 28589492 | |
| Unknown unknown | lymphoma | not applicable | ACP-319 | Preclinical - Cell culture | Actionable | In a preclinical study, ACP-319 treatment blocked cell proliferation in multiple lymphoma cell lines in culture (Eur J of Cancer, Dec 2016, 69;1, S39-S40). | detail... | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Triptolide | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with Triptolide (C1572) decreased Myc protein expression, decreased cancer stem-like cell (CSC) number, and induced senescence in a triple-negative breast cancer (TNBC) cell line in culture, and depleted CSCs and reduced tumor growth in TNBC cell line xenograft models (PMID: 28951456). | 28951456 | |
| Unknown unknown | acute myeloid leukemia | not applicable | SNS-229 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with SNS-229 resulted in decreased PDPK1 pathway signaling and tumor growth inhibition in an acute myeloid leukemia cell line xenograft model (Mol Cancer Ther, Dec 1 2015 (14) (12 Supplement 2) C198). | detail... | |
| Unknown unknown | 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 | lymphoma | not applicable | FT516 + Rituximab | Preclinical - Cell line xenograft | Actionable | In a preclinical study, FT516 in combination with Rituxan (rituximab) enhanced ADCC and cytokine production, resulted in reduced tumor burden in cell line xenograft models of disseminated lymphoma (Cancer Res 2019;79(13 Suppl):Abstract nr 3191). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BIND-014 | Phase I | Actionable | In a Phase I clinical trial, BIND-014 treatment resulted in partial response in 10% (5/52) of patients with advanced solid tumors, and complete response in a cervical cancer patient (PMID: 26847057). | 26847057 | |
| Unknown unknown | 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 | 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 | lymphoma | not applicable | Bortezomib + Tinostamustine | Preclinical - Cell culture | Actionable | In a preclinical study, EDO-S101 and Velcade (bortezomib) worked synergistically to decrease viability of lymphoma cell lines in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | pancreatic cancer | not applicable | Nintedanib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Nintedanib, alone or with chemotherapy, inhibited tumor growth in cell line xenograft models of lung and pancreatic cancer but not in cell culture (PMID: 23729403). | 23729403 | |
| Unknown unknown | Waldenstroem's macroglobulinemia | not applicable | Umbralisib | Phase I | Actionable | In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in stable disease in 2 patients with Waldenstroem's macroglobulinemia (PMID: 29475723; NCT01767766). | 29475723 | |
| Unknown unknown | colon 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 colon cancer cell line xenograft models (PMID: 18339875). | 18339875 | |
| Unknown unknown | angiosarcoma | not applicable | Bevacizumab + Paclitaxel | Phase II | Actionable | In a Phase II trial, angiosarcoma patients treated with Avastin (bevacizumab), in combination with Taxol (paclitaxel), showed no improvement in PFS and OS compared to Taxol (paclitaxel) alone and resulted in higher toxicity (PMID: 26215950). | 26215950 | |
| Unknown unknown | bladder urothelial carcinoma | not applicable | Pembrolizumab | Phase II | Actionable | In a Phase II clinical study (PURE-01), 42% (21/50) of patients with muscle invasive bladder cancer treated with neoadjuvant Keytruda (pembrolizumab) had complete pathological responses at disease resection (PMID: 30343614; NCT02736266). | 30343614 | |
| Unknown unknown | transitional cell carcinoma | not applicable | Lenvatinib + Pembrolizumab | Phase Ib/II | Actionable | Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 25% (5/20) in patients with metastatic urothelial cancer, with a median duration of response not evaluable, and a median progression-free survival of 5.4 months (PMID: 31961766; NCT02501096). | 31961766 | |
| Unknown unknown | 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 | 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 | 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 | 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 | breast cancer | not applicable | TAS0612 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, breast cancer cells harboring mutations in PIK3CA (H1047R, H1047L, or E545K), HRAS, and PTEN, and antiestrogen-resistant cells demonstrated sensitivity to TAS0612 treatment in culture, and orthotopic cell line xenograft models, including a triple-negative breast cancer, demonstrated inhibition of tumor growth (PMID: 31879363). | 31879363 | |
| Unknown unknown | gastroesophageal junction adenocarcinoma | not applicable | Capecitabine + Nivolumab + Oxaliplatin + Tegafur-gimeracil-oteracil Potassium | Phase II | Actionable | In a Phase II trial (ATTRACTION-4), the combination therapy of Xeloda (capecitabine), Opdivo (nivolumab), Eloxatin (oxaliplatin), and TS-1 (tegafur-gimeracil-oteracil potassium) was well-tolerated and resulted in an objective response rate of 76.5% (13/17), a median progression-free survival of 10.6 months, and a median overall survival that was not yet reached in patients with either gastric cancer or gastroesophageal junction cancer (PMID: 30566590; NCT02746796). | 30566590 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | NEO2734 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, NEO2734 inhibited proliferation of a variety of tumor cell lines in culture, and resulted in tumor regression in cell line xenograft models (Ann Oncol, 29(suppl_8), Oct 2018, abstract 429P). | detail... | |
| Unknown unknown | 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 | melanoma | not applicable | CBT-502 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CBT-502 treatment inhibited tumor growth in a cell line xenograft model of melanoma (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr A200). | detail... | |
| 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 | 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 | 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 | neuroblastoma | not applicable | GD2-GD3 vaccine | Phase I | Actionable | In a Phase I trial, treatment with the GD2-GD3 vaccine was well tolerated, and led to an antibody response in 80% (12/15) and loss of minimal residual disease in 60% (6/10) of neuroblastoma patients (PMID: 24520094). | 24520094 | |
| Unknown unknown | 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 | breast cancer | not applicable | Everolimus | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Afinitor (everolimus) treatment inhibited phosphorylation of Ybx1, p70S6K, and S6, and reduced proliferation of breast cancer cells harboring mutations in PIK3CA (H1047R or E545K) and PTEN, and antiestrogen-resistant cells in culture, and inhibited tumor growth in an orthotopic cell line xenograft model (PMID: 31879363). | 31879363 | |
| Unknown unknown | B-cell lymphoma | not applicable | Fenretinide | Phase I | Actionable | In a Phase I trial, Fenretinide treatment resulted in stable disease in a patient with B-cell lymphoma (PMID: 28420721). | 28420721 | |
| Unknown unknown | renal cell carcinoma | not applicable | Atezolizumab + CPI-444 | Phase I | Actionable | In a Phase I trial, treatment with the combination of CPI-444 and Tecentriq (atezolizumab) was well-tolerated and resulted in a disease control rate of 100% (3/3) in patients with renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 3004)). | detail... | |
| Unknown unknown | epithelioid sarcoma | not applicable | Entinostat | Preclinical | Actionable | In a preclinical study, Entinostat inhibited growth and colony formation of epithelioid sarcoma cells in culture (PMID: 26396249). | 26396249 | |
| Unknown unknown | endometrial cancer | not applicable | ETP-46464 + Radiotherapy | Preclinical - Cell culture | Actionable | In a preclinical study, ETP-46464 increased the sensitivity of endometrial cancer cell lines to ionizing radiation in culture (PMID: 25560806). | 25560806 | |
| Unknown unknown | 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 | 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 | non-Hodgkin lymphoma | not applicable | KW-2478 | Phase I | Actionable | In a Phase I clinical trial, KW-2478 demonstrated safety and preliminary efficacy in patients with non-Hodgkin lymphoma, with 100% (4/4) of patients achieving stable disease (PMID: 26695442). | 26695442 | |
| Unknown unknown | 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 | lung small cell carcinoma | not applicable | Olaparib + Temozolomide | Phase Ib/II | Actionable | In a Phase I/II trial, combination of Lynparza (olaparib) and Temodar (temozolomide) resulted in an objective response rate of 41.7% (20/48) in patients with relapsed small cell lung cancer, with a median progression-free survival of 4.2 months and a median overall survival of 8.5 months, similar response was recapitulated in a coclinical trial with 32 patient-derived xenograft models (PMID: 31416802; NCT02446704). | 31416802 | |
| Unknown unknown | lymphoma | not applicable | Copanlisib | Phase II | Actionable | In a Phase II trial, treatment with Aliqopa (copanlisib) in patients with indolent lymphoma resulted in an objective response rate of 59% (84/142), including a complete response in 12%, and demonstrated a median duration of response of 22.6 months and a median progression-free survival of 11.2 months (PMID: 28976790, PMID: 28633365; NCT01660451). | 28633365 28976790 | |
| Unknown unknown | lymphoma | not applicable | Copanlisib | Phase II | Actionable | In a Phase II trial, Aliqopa (copanlisib) treatment resulted in partial response in 20% (1/5) and stable disease in 40% (2/5) of patients with transformed lymphoma (PMID: 24852792). | 24852792 | |
| Unknown unknown | 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 | 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 | acute promyelocytic leukemia | not applicable | Bortezomib + Tinostamustine | Preclinical - Cell culture | Actionable | In a preclinical study, EDO-S101 and Velcade (bortezomib) worked synergistically to decrease viability of an acute promyelocytic leukemia cell line in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CEP-11981 | Phase I | Actionable | In a Phase I trial, CEP-11981 demonstrated safety and preliminary efficacy, resulted in stable disease in 51% (19/37) of patients with advanced solid tumors (PMID: 25152243). | 25152243 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | KW-2450 | Preclinical | Actionable | In a preclinical study, KW-2450 inhibited the growth of triple-negative breast cancer cells in culture and in xenograft models (PMID: 26443806). | 26443806 | |
| Unknown unknown | colon cancer | not applicable | Etoposide + NU7441 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, NU7441 increased the sensitivity of colon cancer cell lines to Vepesid (etoposide), resulting in decreased cell survival in culture and reduced tumor growth in xenograft models (PMID: 16707462). | 16707462 | |
| Unknown unknown | renal cell carcinoma | not applicable | Famitinib | Phase I | Actionable | In a Phase I trial, renal carcinoma patients treated with Famitinib demonstrated a disease control rate of 87.5%, which included 50% (12/24) with a partial response and 37.5% (9/24) with stable disease, and a PFS of 10.7 mo and an OS of 33 mo (PMID: 24238512). | 24238512 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Brexucabtagene autoleucel + Cyclophosphamide + Fludarabine | Phase II | Actionable | In a Phase II (ZUMA-2) trial, conditioning chemotherapy including Cytoxan (cyclophosphamide) and Flurdara (fludarabine) followed by a single infusion of Tecartus (brexucabtagene autoleucel) resulted in an objective response rate of 86% (24/28, 16 complete response, 8 partial response) in patients with relapsed/refractory mantle cell lymphoma (Blood (2019) 134 (Supplement_1): 754). | detail... | |
| Unknown unknown | acute promyelocytic leukemia | not applicable | Arsenic trioxide + Tretinoin | Phase III | Actionable | In a Phase III trial, a 40.6 month follow-up of acute promyelocytic leukemia (APL) patients treated with the combination of Vesanoid (tretinoin) and Trisenox (arsenic trioxide) demonstrated a better event-free survival, cumulative incidence of relapse, and overall survival when compared to APL patients treated with the combination of Vesanoid (tretinoin) and chemotherapy (PMID: 27400939). | 27400939 | |
| Unknown unknown | colon carcinoma | not applicable | UD-017 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, UD-017 inhibited tumor growth in cell line xenograft models of colon carcinoma (J Clin Oncol 35, 2017 (suppl; abstr e14085)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | DS-3078a | Phase I | Actionable | In a Phase I trial, DS-3078a demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Mol Cancer Ther November 2013 12:C173). | detail... | |
| Unknown unknown | 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 | Advanced Solid Tumor | not applicable | Poloxin-2 | Preclinical - Cell culture | Actionable | In a preclinical study, human cancer cells demonstrated sensitivity to treatment with Poloxin-2 in culture, resulting in cell-cycle arrest and apoptotic induction (PMID: 26279064). | 26279064 | |
| Unknown unknown | 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 | marginal zone B-cell lymphoma | not applicable | LAM-002A | Phase I | Actionable | In a Phase I trial, LAM-002A demonstrated safety and preliminary efficacy, resulted in prolonged stable disease in 1 of 3 patients with marginal zone lymphoma (Blood 2017 130 (Suppl 1):4119). | detail... | |
| Unknown unknown | lung carcinoma | not applicable | SF2523 | Preclinical | Actionable | In a preclinical study, SF2523 decreased tumor immunosuppression, increased T-lymphocyte infiltration, and reduced tumor growth in syngeneic mouse lung carcinoma models (PMID: 31018997). | 31018997 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | HS-110 + Nivolumab | Phase Ib/II | Actionable | In a Phase I/II trial, HS-110 (Viagenpumatucel-L) and Opdivo (nivolumab) combination treatment demonstrated safety, and resulted in an objective response rate of 21% (10/47), a clinical benefit rate of 43%, a median duration of response of 17.2 months, and a median overall survival of 28.7 months in patients with advanced non-small cell lung cancer (J Clin Oncol 38: 2020 (suppl; abstr 9546); NCT02439450). | detail... | |
| Unknown unknown | stomach cancer | not applicable | Nivolumab | Phase Ib/II | Actionable | In a Phase I/II trial, Opdivo (nivolumab), alone or incombination with Yervoy (ipilimumab), demonstrated safety and efficacy in patients with chemotherapy-refractory gastric cancer, resulted in a disease control rate of 38% (61/160) (J Clin Oncol 34, 2016 (suppl; abstr 4010)). | detail... | |
| Unknown unknown | 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 | acute myeloid leukemia | not applicable | AMG 900 | Phase I | Actionable | In a Phase I clinical trial, AMG 900 treatment resulted in a complete response in 9% (3/35) of adult patients acute myeloid leukemia (PMID: 28370201). | 28370201 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | GDC-0349 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the mTOR inhibitor GDC-0349 demonstrated inhibition of tumor growth in cell line xenograft models of solid tumors (PMID: 24900569). | 24900569 | |
| Unknown unknown | glioblastoma multiforme | not applicable | G-TPP + Obatoclax | Preclinical - Patient cell culture | Actionable | In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Obatoclax (GX015-070) synergistically inhibited viability of established lines and patient-derived glioblastoma cells in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | neuroendocrine tumor | not applicable | CC-90011 | Phase I | Actionable | In a Phase I trial, CC-90011 treatment demonstrated safety, and resulted in a prolonged stable disease of at least 4 months in 7 patients (n=26) with neuroendocrine neoplasm (5 bronchial and 2 prostate) (Annals of Oncology, Volume 30, Issue Supplement_1, Feb 2019, mdz029; NCT02875223). | detail... | |
| Unknown unknown | glioblastoma multiforme | not applicable | Olaparib + Temozolomide | Phase II | Actionable | In a Phase II trial (OPARATIC), 36% (14/39) of evaluable patients with glioblastoma were progression-free at 6 months when treated with the combination therapy of Lynparza (olaparib) and Temodar (temozolomide) (PMID: 32347934; NCT0139057). | 32347934 | |
| Unknown unknown | colorectal cancer | not applicable | Gedatolisib + Irinotecan | Case Reports/Case Series | Actionable | In a Phase I trial, treatment with the combination of Gedatolisib (PF-05212384) and Camptosar (irinotecan) resulted in an overall response rate of 4.7%, with 2 colorectal cancer patients achieving a partial response, and clinical benefit rate of 16.3% in an advanced solid tumor patient cohort comprising 93% colorectal cancer patients (PMID: 29067643; NCT01347866)). | 29067643 | |
| 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 | ovarian cancer | not applicable | PF-06647263 | Phase I | Actionable | In a Phase I trial, PF-06647263 treatment in ovarian cancer patients (n=16) resulted in a partial response in 2 patients and stable disease in 9 patients (PMID: 30680712; NCT02078752). | 30680712 | |
| 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 | pancreatic carcinoma | not applicable | Gemcitabine + MU380 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the addition of MU380 resulted in increased sensitivity to Gemzar (gemcitabine) in a pancreatic carcinoma cell line in culture and in xenograft models (PMID: 28619751). | 28619751 | |
| Unknown unknown | endometrial cancer | not applicable | Dactolisib | Preclinical | Actionable | In a preclinical study, BEZ235 suppressed tumor growth in endometrial xenografts (PMID: 22662154). | 22662154 | |
| Unknown unknown | breast cancer | not applicable | 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 | colorectal cancer | not applicable | MM-151 | Clinical Study | Actionable | In a Phase I trial, MM-151 treatment resulted in partial response in 17% (5/29) and stable disease in 28% (8/29) of colorectal patients (J Clin Oncol 34, 2016 (suppl; abstr 2518)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | AMG 900 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AMG 900 inhibited the growth of a variety of human solid tumor cell lines in culture and inhibited tumor growth in cell line xenograft models of several tumor types including breast, colon, lung, pancreatic, and uterine cancer (PMID: 20935223). | 20935223 | |
| Unknown unknown | colon carcinoma | not applicable | Mps-BAY2b + Paclitaxel | Preclinical | Actionable | In a preclinical study, Mps-BAY2b, in combination with paclitaxel, had increased efficacy in inhibiting cell proliferation of colon carcinoma cell in culture (PMID: 23933817). | 23933817 | |
| Unknown unknown | chondrosarcoma | not applicable | Doxorubicin + Nilotinib | Phase I | Actionable | In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin resulted in 5 stable disease and 2 progressive disease in 7 patients with chondrosarcoma, with a median progression-free survival of 14 months and a median overall survival of 25 months (PMID: 30037815; NCT02587169). | 30037815 | |
| Unknown unknown | breast cancer | not applicable | DCBCI0901 | Preclinical - Cell culture | Actionable | In a preclinical study, breast cancer cell lines treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). | detail... | |
| Unknown unknown | lymphoma | not applicable | OKI-005 | Preclinical - Cell culture | Actionable | In a preclinical study, OKI-005 induced cell cycle arrest and apoptosis in lymphoma cells in culture (PMID: 31235619). | 31235619 | |
| Unknown unknown | peritoneum cancer | not applicable | Lurbinectedin | Phase II | Actionable | In a Phase II trial, Lurbinectedin (PM01183) treatment in patients with either ovarian, peritoneal, or fallopian tube cancer resulted in an overall response rate of 23% (12/52), which included one complete response and eleven partial responses, and a median duration of response of 4.6 months and 23% (12/52) of responses lasted for 6 months or more (PMID: 28368437). | 28368437 | |
| Unknown unknown | synovial sarcoma | not applicable | 211At-OTSA101 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, 211At-OTSA101 treatment inhibited tumor growth and increased survival of a cell line xenograft model of synovial sarcoma (PMID: 29952132). | 29952132 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CYT01B + Veliparib | Preclinical - Cell culture | Actionable | In a preclinical study, CYT01B and Veliparib (ABT-888) synergistically inhibited growth of tumor cell lines in culture (AACR Annual Meeting 2019, Abstract 363). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | Pegylated liposomal-doxorubicin + Trebananib | Phase III | Actionable | In a Phase III trial, Trebananib in combination with Doxil (pegylated liposomal doxorubicin) resulted in improved objective response rate (46% vs. 21%) and median duration of response (7.4 vs. 3.9 months) compared to placebo in patients with recurrent ovarian cancer (PMID: 27914241). | 27914241 | |
| Unknown unknown | breast cancer | not applicable | ST7612AA1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, ST7612AA1 inhibited proliferation of breast cancer cell lines in culture, and inhibited tumor growth in breast cancer cell line xenograft models (PMID: 25671299). | 25671299 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Vandetanib | Phase I | Actionable | In a Phase I trial, treatment with Caprelsa (vandetanib) resulted in stable disease in 40% (31/77) of patients with advanced solid tumors (PMID: 15905307). | 15905307 | |
| Unknown unknown | melanoma | not applicable | Nab-paclitaxel + Oblimersen + Temozolomide | Phase I | Actionable | In a Phase I trial, treatment with the combination of Genasense (oblimersen), Temodar (temozolomide), and Abraxane (nab-paclitaxel) resulted in an objective response rate of 40.6% (13/32) and disease control rate of 75% (24/32) in patients with advanced melanoma (PMID: 23064957). | 23064957 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Belinostat + Carboplatin + Paclitaxel | Phase I | Actionable | In a Phase I trial, the combination therapy of Beleodaq (belinostat), Paraplatin (carboplatin, and Taxol (paclitaxel) in patients with non-small cell lung carcinoma resulted in a 5.7 month median progression-free survival, a partial response in 35% (8/23) patients, and stable disease in 17% (4/23) of patients (Journal of Thoracic Oncology, 2017, vol 12:1S, abstract #P2.03a-003). | detail... | |
| Unknown unknown | renal cell carcinoma | not applicable | GDC-0349 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the mTOR inhibitor GDC-0349 demonstrated inhibition of tumor growth in cell line xenograft models of solid tumors, including renal adenocarcinoma (PMID: 24900569). | 24900569 | |
| Unknown unknown | ovarian carcinoma | not applicable | Onvansertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, human ovarian carcinoma cells demonstrated cell cycle arrest in cell line xenograft models when treated with Onvansertib (PCM-075) (PMID: 22319201). | 22319201 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Buparlisib + Ibrutinib | Phase Ib/II | Actionable | In a Phase I/II trial, Buparlisib (BKM120) and Imbruvica (ibrutinib) combination treatment resulted in a best overall response rate of 88% (15/17, 11 complete response, 4 partial response) in patients with relapsed/refractory mantle cell lymphoma (J Clin Oncol 36, 2018 (suppl; abstr 7520); NCT02756247). | detail... | |
| Unknown unknown | 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 | 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 | 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 | prostate cancer | not applicable | Apalutamide | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Erleada (apalutamide) resulted in a median metastasis-free survival of 40.5 months in patients with non-metastatic castration-resistant prostate cancer, compared to 16.2 months with placebo (HR=0.28) (PMID: 29420164; NCT01946204). | detail... 29420164 | |
| Unknown unknown | prostate cancer | not applicable | Apalutamide | Clinical Study | Actionable | In a clinical study, Erleada (apalutamide) demonstrated safety and efficacy, resulted in 12-week PSA response rate of 88% (22/25) and 22% (4/18), median time to PSA progression of 18.2 months and 3.7 months, in castration-resistant prostate cancer patients that were treatment-naive or those received prior abiraterone and prednisone (PMID: 28213364). | 28213364 | |
| Unknown unknown | prostate cancer | not applicable | Apalutamide | FDA approved | Actionable | In a Phase III trial (TITAN) that supported FDA approval, treatment with Erleada (apalutamide) plus androgen-deprivation therapy (ADT) resulted in a radiographic progression-free survival at 24 months in 68.2% (358/525) of patients with metastatic castration-sensitive prostate cancer, compared to 47.5% (250/527) in patients treated with ADT and placebo (HR=0.48, p<0.001), and improved overall survival at 24 months (82.4% vs 73.5%, HR=0.89, p=0.005) (PMID: 31150574; NCT02489318). | 31150574 detail... | |
| Unknown unknown | stomach cancer | not applicable | Trifluridine-tipiracil hydrochloride | Phase III | Actionable | In a Phase III trial (TAGS) that supproted FDA approval, Lonsurf (trifluridine/tipiracil hydrochloride) treatment resulted in improved overall survival (5.7 vs 3.6 months, HR=0.69, p=0.00029) compared to placebo in patients with heavily pretreated metastatic or advanced gastric cancer or gastroesophageal junction adenocarcinoma (PMID: 30355453; NCT02500043). | 30355453 detail... | |
| Unknown unknown | breast cancer | not applicable | SF2523 | Preclinical | Actionable | In a preclinical study, SF2523 decreased tumor immunosuppression, and inhibited tumor growth and progression in a mouse breast cancer model (PMID: 31018997). | 31018997 | |
| Unknown unknown | breast carcinoma | not applicable | VLX600 | Preclinical - Cell culture | Actionable | In a preclinical study, VLX600 treatment inhibited proliferation of a mouse breast carcinoma cell line in culture (PMID: 24548894). | 24548894 | |
| 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 | acute myeloid leukemia | not applicable | RO6839921 | Phase I | Actionable | In a Phase I trial, treatment with RO6839921 was well-tolerated, and resulted in a composite response rate (CR, CRc, CRp, CRi/MLFS) of 7.7% (2/26), including 1 patient with a complete remission (CR) and 1 patient with a CR with incomplete hematologic recovery/morphological leukemia-free state (CRi/MLFS), partial response in 7.7% (2/26), and hematologic improvement/stable disease (HI/SD) in 26.9% (7/26) of patients with acute myeloid leukemia (PMID: 32020437; NCT02098967). | 32020437 | |
| 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 | 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 | Advanced Solid Tumor | not applicable | Futibatinib | Phase I | Actionable | In a Phase I trial, TAS-120 treatment resulted in clinical response in 5.5% (2/36) and stable disease over 24 weeks in 5.5% (2/36) of patients with advanced solid tumors (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 372PD). | detail... | |
| Unknown unknown | 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 | lung small cell carcinoma | not applicable | Carboplatin + Etoposide + Roniciclib | Phase Ib/II | Actionable | In a Phase Ib/II trial, first-line treatment with the combination of Roniciclib (BAY1000394) and either carboplatin plus etoposide or cisplatin plus etoposide demonstrated tolerability and resulted in a response rate of 81.4% (35/43; all partial responses), median OS of 12.6 mo, and median PFS of 7.3 mo in extensive disease small cell lung cancer patients; however due to a safety signal in a related trial further development of Roniciclib (BAY1000394) was discontinued (PMID: 30089585; NCT01573338). | 30089585 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Carboplatin + Etoposide + Roniciclib | Phase II | Actionable | In a Phase II trial, the combination of Roniciclib (BAY1000394) plus chemotherapy regimen, Paraplatin (carboplatin) or Platinol (cisplatin) and Vepesid (etoposide) (n=71), did not meet its primary endpoint for progression-survival in patients with small cell lung cancer (4.9 mo vs 5.5 mo) when compared to placebo plus chemotherapy (n=71), and showed an unfavorable risk-benefit profile, therefore, leading to premature termination of the study (PMID: 30677506; NCT02161419). | 30677506 | |
| Unknown unknown | 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 | breast cancer | not applicable | Azacitidine + Entinostat | Phase II | Actionable | In a Phase II trial, the combination of Vidaza (azacitidine) and Entinostat (MS-275) did not reach its primary endpoint for either cohort of breast cancer patients, TNBC or hormone-resistant, however, the optional continuation phase resulted in one patient achieving a partial response (PMID: 27979916). | 27979916 | |
| Unknown unknown | colorectal cancer | not applicable | Refametinib + Sorafenib | Phase I | Actionable | In a Phase I trial, a patient with colorectal cancer demonstrated a durable partial response for 358 days when treated with the combination of Refametinib (BAY86-9766) and Nexavar (sorafenib) (PMID: 26644411). | 26644411 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MK-1454 + Pembrolizumab | Phase I | Actionable | In a Phase I trial, MK-1454 in combination with Keytruda (pembrolizumab) resulted in partial response in 24% (6/25) and a disease control rate of 48% (12/25) in patients with advanced solid tumors (Ann Oncol, Oct 2018, 29 (suppl 8), abstract LBA15; NCT03010176). | detail... | |
| Unknown unknown | 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 | follicular lymphoma | not applicable | Varlilumab | Case Reports/Case Series | Actionable | In a Phase I trial, Varlilumab (CDX-1127) was well tolerated in heavily pretreated patients with follicular lymphoma, and resulted in stable disease in three patients (3/5), with disease control lasting between 4.5 and 14 months, including one patient who experienced 5.6 months of stable disease and a 36% reduction of measurable disease (PMID: 32380537; NCT01460134). | 32380537 | |
| 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 | 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 | diffuse large B-cell lymphoma | not applicable | Umbralisib | Phase I | Actionable | In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in objective response in 31% (4/13) and stable disease in 15% (2/13) of patients with diffuse large B-cell lymphoma (PMID: 29475723; NCT01767766). | 29475723 | |
| Unknown unknown | 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 | 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 | 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 | 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 | lung non-small cell carcinoma | not applicable | Aflibercept + Docetaxel | Phase III | Actionable | In a Phase III trial, the combination of Zaltrap (aflibercept) and Taxotere (docetaxel) did not result in improved overall survival compared to Taxotere (docetaxel) with placebo, but did result in an improved overall response rate of 23.3% (94/404) vs. 8.9% (36/406) with Taxotere (docetaxel) plus placebo in non-small cell lung cancer patients that had failed platinum therapy (PMID: 22965962). | 22965962 | |
| Unknown unknown | colorectal cancer | no benefit | Fluorouracil + Leucovorin + Oxaliplatin + RO5520985 | Phase II | Actionable | In a Phase II trial (McCAVE), the combination of Vanucizumab (RO5520985) and mFOLFOX6 demonstrated increased toxicity, did not improve the objective response rate (52.1% (49/94) v 57.9% (55/95)), and resulted in a similar progression-free survival duration (343 v 334 days) compared to the combination of Avastin (bevacizumab) and mFOLFOX6 in metastatic colorectal cancer patients (PMID: 32162804, NCT02141295). | 32162804 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Emibetuzumab + Ramucirumab | Phase Ib/II | Actionable | In a Phase Ib/II trial, Emibetuzumab (LY2875358) and Cyramza (ramucirumab) combination treatment resulted in an objective response rate of 7% (1/15) and a disease control rate of 87% (13/15) in patients with non-small cell lung cancer, with a median progression-free survival of 6.6 months (PMID: 31142504; NCT02082210). | 31142504 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Cisplatin + Durvalumab + Etoposide | FDA approved | Actionable | In a Phase III (CASPIAN) trial that supported FDA approval, Imfinzi (durvalumab) in combination with Vepesid (etoposide) and Paraplatin (carboplatin) or Platinol (cisplatin) resulted in significantly improved overall survival (13.0 vs 10.3 mo, HR=0.73, p=0.0047) compared to platinum-etoposide therapy in patients with untreated extensive-stage small cell lung cancer (PMID: 31590988; NCT03043872). | 31590988 | |
| Unknown unknown | 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 | acute myeloid leukemia | not applicable | Bortezomib + Cytarabine + Etoposide + Midostaurin + Mitoxantrone | Phase I | Actionable | In a Phase I trial, Rydapt (midostaurin), in combination with Velcade (bortezomib) and mitoxantrone, Vepesid (etoposide), and Cytosar-U (cytarabine) (MEC), resulted in an overall response rate of 82.5% (19/23) in patients with relapsed or refractory acute myeloid leukemia receiving dose level 3 and above, with complete responses in 56.5% (13/23) of patients (PMID: 26784138). | 26784138 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | 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 | multiple myeloma | not applicable | INCB054329 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, INCB054329 treatment reduced MYC expression and proliferation of myeloma cell lines in culture, and inhibited tumor growth in myeloma cell line xenograft models (PMID: 30206163). | 30206163 | |
| Unknown unknown | melanoma | not applicable | KRT-232 + Trametinib | Phase I | Actionable | In a Phase I trial, the combination therapy of KRT-232 (AMG 232) and Mekinist (trametinib) in 15 patients with metastatic cutaneous melanoma without a BRAF V600 mutation resulted in a partial response in 2 patients, stable disease in 11 patients, and progressive disease in 2 patients, and of the 15 patients, 11 experienced tumor reduction (J Clin Oncol 35, 2017 (suppl; abstr 2575)). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | unspecified PD-1 antibody | Clinical Study | Actionable | In a retrospective analysis, treatment with an unspecified PD-1 or PD-L1 therapy in metastatic non-small cell lung cancer patients harboring a mutation in BRAF, ERBB2 (HER2), or MET, or a RET translocation led to a response rate of 29% (31/107), a 15.4 mo duration of response, a 4.7 mo median progression-free survival, and a 16.2 mo median overall survival, and resulted in clinical efficacy similar to what has been observed in studies with unselected non-small cell lung cancer patients (PMID: 31945494). | 31945494 | |
| Unknown unknown | colorectal cancer | not applicable | AZD2461 + Temozolomide | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the addition of AZD2461 to Temodar (temozolomide) resulted in greater antitumor activity than Temodar (temozolomide) alone in colorectal cancer cell line xenograft models, demonstrating decreased tumor volume (PMID: 27550455). | 27550455 | |
| Unknown unknown | leiomyosarcoma | not applicable | Aldoxorubicin + Dactolisib | Preclinical | Actionable | In a preclinical study, the combination of doxorubicin and BEZ235 produced a synergistic effect in leiomyosarcoma cells both in culture and in mouse models, resulting in apoptotic induction and a 68% reduction in tumor volume (PMID: 26952093). | 26952093 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | LY2275796 | Phase I | Actionable | In a Phase I study, treatment with LY2275796 demonstrated safety and resulted in reduced eIF-4E expression, but did not demonstrate antitumor effects in patients with advanced solid tumors (PMID: 21831956). | 21831956 | |
| Unknown unknown | ovary epithelial cancer | not applicable | Ramucirumab | Phase II | Actionable | In a Phase II trial, Cyramza (ramucirumab) resulted in antitumor activity in patients with epithelial ovarian cancer (PMID: 25016924). | 25016924 | |
| Unknown unknown | endometrial cancer | no benefit | Trametinib + Uprosertib | Phase I | Actionable | In a Phase I trial, Mekinist (trametinib) and Uprosertib (GSK2141795) combination treatment demonstrated increased toxicity and limited efficacy, resulted in no response (0/14) at RP2D dose and 1 response (8.3%, 1/12) at reduced dose in patients with recurrent endometrial cancer, with progression-free survival at 6 months in 14% and 25% of the patients, respectively (PMID: 31623857). | 31623857 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MRX34 | Phase I | Actionable | In a Phase I trial, MRX34 treatment in advanced solid tumor patients resulted in some preliminary efficacy, including a partial response lasting 48 weeks in one patient with hepatocellular carcinoma and four patients with stable disease (PMID: 27917453). | 27917453 detail... | |
| Unknown unknown | glioblastoma multiforme | no benefit | Sunitinib | Phase II | Actionable | In multiple Phase II clinical trials, Sutent (sunitinib) failed to demonstrate any benefit in patients with glioblastoma with or without concurrent bevacizumab treatment (PMID: 24424564, PMID: 23086433). | 23086433 24424564 | |
| Unknown unknown | glioblastoma multiforme | not applicable | A-1210477 + G-TPP | Preclinical - Patient cell culture | Actionable | In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the Mcl-1 inhibitor A-1210477 synergistically inhibited viability of established lines and patient-derived glioblastoma cells in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | BPI-9016M | Phase I | Actionable | In a Phase I trial, BPI-9016M treatment was well-tolerated in patients with non-small cell lung cancer who had progressed on prior therapy (n=20), and of nineteen evaluable patients, one patient had a partial response and 10 patients experienced stable disease (PMID: 31948451; NCT02478866). | 31948451 | |
| Unknown unknown | pheochromocytoma | not applicable | Sunitinib | Phase II | Actionable | In a Phase II trial (SNIPP), Sutent (sunitinib) treatment in patients with pheochromocytoma (PCC, n=14) or paraganglioma (PGL, n=11) resulted in an overall response rate of 13% (3/23), including partial responses in two PCC patients and one PGL patient, a disease control rate of 83% (19/23), and median progression-free survival of 13.4 months (PMID: 31105270). | 31105270 | |
| Unknown unknown | stomach cancer | not applicable | MGD013 | Case Reports/Case Series | Actionable | In a Phase I trial, MGD013 treatment resulted in a partial response in a patient with gastric cancer (J Clin Oncol 38: 2020 (suppl; abstr 3004); NCT03219268). | detail... | |
| Unknown unknown | Hodgkin's lymphoma | not applicable | Pembrolizumab | FDA approved | Actionable | In a Phase II trial (KEYNOTE-087) that supported FDA approval, Keytrude (pembrolizumab) treatment resulted in an overall response rate of 69% (145/210) in patients with relapsed or refractory classical Hodgkin lymphoma (PMID: 28441111; NCT02453594). | detail... 28441111 | |
| Unknown unknown | medulloblastoma | not applicable | Prexasertib | Preclinical - Cell culture | Actionable | In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including medulloblastoma cell lines (PMID: 28270495). | 28270495 | |
| Unknown unknown | breast cancer | not applicable | VS-5584 | Preclinical - Patient cell culture | Actionable | In a preclinical study, treatment with VS-5584 resulted in decreased cancer stem cell (CSC) number in a triple-negative breast cancer cell line in culture and in xenograft models, and decreased CSCs in patient breast cancer tumor samples in culture (PMID: 25432176). | 25432176 | |
| Unknown unknown | multiple myeloma | not applicable | BAY 1238097 | Preclinical | Actionable | In a preclinical study, BAY 1238097 inhibited tumor growth and was well tolerated in acute myeloid leukemia and multiple myeloma models (American Association for Cancer Research; 2015 Apr 18-22; Abstract nr 3524). | detail... | |
| Unknown unknown | non-Hodgkin lymphoma | not applicable | CC-90010 | Phase I | Actionable | In a Phase I trial, CC-90010 treatment demonstrated safety, and resulted in an overall response rate of 2.9% (2/69, 1 complete response, 1 partial response), stable disease in 33.3% (23/69), and a median progression-free survival of 1.9 months in patients with advanced solid tumors or relapsed/refractory non-Hodgkin lymphoma (PMID: 32240793; NCT03220347). | 32240793 | |
| Unknown unknown | colorectal cancer | not applicable | WYE-354 | Preclinical | Actionable | In a preclinical study, WYE-354 induced cell cycle arrest and inhibited cell proliferation of colorectal cancer cells in culture (PMID: 19584280). | 19584280 | |
| Unknown unknown | pancreatic cancer | not applicable | Gemcitabine + Pimasertib | Preclinical | Actionable | In a preclinical study, treatment with Pimasertib (MSC1936369B) followed by Gemzar (gemcitabine) resulted in enhanced inhibition of proliferation and induction of apoptosis in pancreatic cell lines in culture (PMID: 26228206). | 26228206 | |
| Unknown unknown | peripheral T-cell lymphoma | not applicable | Tenalisib | Phase I | Actionable | In a Phase I trial, Tenalisib (RP6530) demonstrated safety in patients with relapsed or refractory peripheral T-cell lymphoma, and resulted in an overall response rate of 46.7% (7/15), including three complete responses and four partial responses with a median duration of response of 6.5 months, and a further two patients achieved stable disease (PMID: 32824175; NCT02567656). | 32824175 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Zanubrutinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Brukinsa (zanubrutinib) inhibited BTK signaling and proliferation of diffuse large B-cell lymphoma (DLBCL) cell lines in culture, and demonstrated antitumor activity in a DLBCL cell line xenograft model, with improved efficacy compared to Imbruvica (ibrutinib) (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 2597). | detail... | |
| Unknown unknown | 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 | 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 | prostate cancer | not applicable | AMG208 | Phase I | Actionable | In a Phase I trial, AMG208 treatment resulted in complete response in 1 and partial response in 2 patients with prostate cancer (PMID: 26155941; NCT00813384). | 26155941 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Barasertib | Phase II | Actionable | In a Phase II trial, Barasertib (AZD1152) treatment resulted in significantly improved objective complete response rate (35.4%, n=48, vs 11.5%, n=26), and median overall survival (8.2 vs 4.5 months, HR=0.88) compared to low dose cytosine arabinoside in elderly patients with acute myeloid leukemia (PMID: 23605952). | 23605952 | |
| Unknown unknown | 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 | colon carcinoma | not applicable | Mps-BAY2b | Preclinical | Actionable | In a preclinical study, Mps-BAY2b inhibited cell cycle progression and induced cell death of colon carcinoma cells in culture (PMID: 23933817). | 23933817 | |
| Unknown unknown | CLL/SLL | not applicable | Obinutuzumab + Venetoclax | FDA approved | Actionable | In a Phase III trial that supported FDA approval (CLL14), treatment with the combination of Venclexta (venetoclax) and Gazyva (obinutuzumab) resulted in improved progression-free survival (PFS) compared to treatment with Gazyva (obinutuzumab) plus chlorambucil ((HR 0.35; 95% CI 0.23-0.53; P<0.0001) (J Clin Oncol 37, 2019 (suppl; abstr 7502; NCT02242942). | detail... detail... | |
| Unknown unknown | 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 | leiomyosarcoma | not applicable | Dactolisib | Preclinical | Actionable | In a preclinical study, leiomyosarcoma cells treated with BEZ235 in culture and in mouse models demonstrated increased levels of apoptosis and a 42% reduction in tumor volume (PMID: 26952093). | 26952093 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Cerdulatinib | Preclinical - Patient cell culture | Actionable | In a preclinical study, Cerdulatinib (PRT062070) decreased BCR downstream signaling and chemokine secretion, and reduced viability of patient-derived chronic lymphocytic leukemia (CLL) cells in culture (PMID: 27697994). | 27697994 | |
| Unknown unknown | bladder carcinoma in situ | not applicable | BCG solution | Phase III | Actionable | In a Phase III trial supporting FDA approval, TICE BCG solution (BCG solution) treatment resulted in an estimated 2 year disease-free survival rate of 57% (109/191) in patients with bladder carcinoma in situ (PMID: 21224104). | 21224104 | |
| Unknown unknown | 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 | acute myeloid leukemia | not applicable | TP-1287 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TP-1287 demonstrated improved oral bioavailability, resulted in efficient tumor inhibition in cell line xenograft models of acute myeloid leukemia (Cancer Res 2017;77(13 Suppl):Abstract nr 5133). | detail... | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Pemetrexed Disodium + Sorafenib | Phase I | Actionable | In a Phase I clinical trial in patients with advanced solid tumors, the combination of Alimta (pemetrexed) and Nexavar (sorafenib) demonstrated safety and preliminary efficacy in patients with triple-receptor breast cancer (TNBC), with 60% (3/5) of TNBC patients demonstrating an objective response and 100% (5/5) of patients achieving stable disease or better (PMID: 27213589). | 27213589 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | GSK3368715 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, GSK3368715 inhibited tumor growth in a triple-negative breast cancer cell line xenograft model (PMID: 31257072). | 31257072 | |
| Unknown unknown | melanoma | not applicable | Ad5CMV-p53 gene + unspecified PD-1 antibody | Preclinical | Actionable | In a preclinical study, the combination of Advexin (Ad5CMV-p53) and an unspecified anti-PD-1 antibody resulted in a synergistic effect and abscopal effect in an immune therapy resistant syngeneic melanoma mouse model, demonstrating decreased tumor growth and improved survival compared to either agent alone (J Clin Oncol 35, 2017 (suppl; abstr e14610)). | detail... | |
| Unknown unknown | epithelioid sarcoma | not applicable | Tazemetostat | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Tazverik (tazemetostat) treatment resulted in an objective response rate of 15% (9/62) and a disease control rate of 26% (16/62) in patients with locally advanced or metastatic epithelioid sarcoma, with a median duration of response not reached and a median overall survival of 82.4 weeks (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 11003-11003, NCT02601950). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | CUDC-907 | Phase I | Actionable | In a Phase I trial, CUDC-907 was well-tolerated, demonstrated safety, and resulted in a 56% (5/9) objective response rate, including two complete responses and three partial responses, in patients with diffuse large B-cell lymphoma (PMID: 27049457). | 27049457 | |
| Unknown unknown | Her2-receptor positive breast cancer | not applicable | Abemaciclib | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, an ERBB2 (HER2)-receptor positive breast cancer cell line xenograft model treated with Abemaciclib (LY2835219) demonstrated delayed tumor growth and in culture, resulted in some decreased cell viability (PMID: 26977878). | 26977878 | |
| Unknown unknown | 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 | E7046 | Phase I | Actionable | In a Phase I trial, AN0025 (E7046) treatment was tolerated, demonstrated on target activity as indicated by upregulation of EP4 downstream genes and enhanced anti-tumor immune response, and resulted in stable disease as best response in 23% (7/30) of patients with advanced solid tumors, and 3 patients achieved metabolic responses (PMID: 32554609; NCT02540291). | 32554609 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | E7046 | Preclinical | Actionable | In a preclinical study, multiple mouse tumor models demonstrated inhibition of tumor growth when treated with E7046 (Cancer Immunol Res 2016;4(1 Suppl):Abstract nr B034). | detail... | |
| Unknown unknown | 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 | breast cancer | not applicable | AsiDNA | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA inhibited DNA repair and proliferation, and induced death in breast cancer cell lines in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | melanoma | not applicable | BNT111 + unspecified PD-1 antibody | Phase I | Actionable | In a Phase I trial, BNT111 treatment combined with an anti-PD1 antibody resulted in a partial response in 6 and stable disease in 2 patients with melanoma (n=17) (PMID: 32728218; NCT02410733). | 32728218 | |
| Unknown unknown | colon carcinoma | not applicable | Fluorouracil + VLX600 | Preclinical - Cell culture | Actionable | In a preclinical study, VLX600 and Adrucil (fluorouracil) combination treatment inhibited viability of colon carcinoma cells in culture (PMID: 24548894). | 24548894 | |
| Unknown unknown | hematologic cancer | not applicable | Abexinostat | Phase II | Actionable | In a Phase II trial, treatment with Abexinostat (PCI-24781) in patients with a variety of hematological cancers resulted in an overall response rate (ORR) of 28% (24/87), a complete response in 5%, and a median duration response of 8.8 months (PMID: 28126962). | 28126962 | |
| Unknown unknown | lung carcinoma | not applicable | MGCD516 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human lung carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). | detail... | |
| Unknown unknown | 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 | myelodysplastic syndrome | not applicable | Guadecitabine | Phase I | Actionable | In a Phase I trial, SGI-110 treatment resulted in clinical response in 32% (6/19) of patients with myelodysplastic syndrome (PMID: 26296954). | 26296954 | |
| Unknown unknown | 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 | triple-receptor negative breast cancer | not applicable | Carboplatin + JQ1 | Preclinical - Cell culture | Actionable | In a preclinical study, the addition of JQ1 to Paraplatin (carboplatin) resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27256375). | 27256375 | |
| Unknown unknown | neuroendocrine carcinoma | not applicable | DS6051b | Phase I | Actionable | In a Phase I clinical trial, treatment with DS-6051b was well-tolerated in patients with advanced solid tumors, and resulted in partial responses in two patients, including a patient with neuroendocrine carcinoma (Cancer Res July 15 2016 (76) (14 Supplement) CT024). | detail... | |
| Unknown unknown | 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 | breast cancer | not applicable | AsiDNA + Veliparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA and Veliparib (ABT-888) combination treatment resulted in increased cell death and inhibition of proliferation in breast cancer cell lines compared to Veliparib (ABT-888) alone in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | 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 | neuroendocrine tumor | not applicable | Cixutumumab + Everolimus + Octreotide acetate | Phase I | Actionable | In a Phase I trial, patients with neuroendocrine tumors treated with the combination of Cixutumumab, Sandostatin Lar Depot (octreotide acetate), and Afinitor (everolimus) demonstrated some efficacy, however, the drug combination did result in multiple non-dose limiting toxicities preventing long term tolerance (PMID: 25900182). | 25900182 | |
| Unknown unknown | stomach carcinoma | not applicable | SST0116CL1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, SST0116CL1 inhibited tumor growth in cell line xenograft models of gastric carcinoma (PMID: 25096516). | 25096516 | |
| Unknown unknown | leukemia | not applicable | TAK-960 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, colorectal cancer cells treated with TAK-960 demonstrated cell growth inhibition, decreased tumor size, and increased median survival in both culture and cell line xenograft models (PMID: 22188812). | 22188812 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | CPI-637 + HY-16462 | Preclinical - Cell culture | Actionable | In a preclinical study, CPI-637 and HY-16462 treatment synergistically inhibited growth of a triple-negative breast cancer cell line in culture (PMID: 31000582). | 31000582 | |
| Unknown unknown | 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 | mantle cell lymphoma | not applicable | Lenalidomide | FDA approved | Actionable | In a Phase II trial (EMERGE) that supported FDA approval, Revlimid (lenalidomide) treatment resulted in an objective response rate of 28% (37/134) with rapid time to response (2.2 months), a median duration of response of 16.6 months, a median progression-free survival of 4.0 months, and a median overall survival of 19.0 months in patients with mantle cell lymphoma who relapsed or progressed after or were refractory to Velcade (bortezomib) (PMID: 24002500; NCT00737529). | 24002500 detail... | |
| Unknown unknown | breast cancer | not applicable | GSK2126458 | Phase I | Actionable | In a Phase I trial, GSK2126458 was well-tolerated and resulted in some efficacy in patients with breast cancer, including stable disease in 31% (7/22) and one patient with a partial response (PMID: 26603258). | 26603258 | |
| Unknown unknown | Her2-receptor positive breast cancer | not applicable | S63845 | Preclinical - Cell culture | Actionable | In a preclinical study, S63845 decreased viability of an ERBB2 (HER2)-amplified breast cancer cell line and ERBB2 (HER2)-amplified patient-derived xenograft (PDX) tumor cells in culture (PMID: 28768804). | 28768804 | |
| Unknown unknown | 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 | 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 | 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 | renal cell carcinoma | not applicable | Everolimus + Sunitinib | Phase II | Actionable | In a Phase II trial, Sutent (sunitinib) as first line therapy followed by second line therapy, Afinitor (everolimus), resulted in a greater overall survival (29.5 mo vs 22.4 mo) compared to the reverse treatment of the two therapies in patients with metastatic renal cell carcinoma (PMID: 28327953). | 28327953 | |
| Unknown unknown | stomach cancer | not applicable | JNJ-26483327 | Preclinical | Actionable | In a preclinical study, human gastric cancer cells demonstrated sensitivity to JNJ-26483327 (PMID: 19584230). | 19584230 | |
| Unknown unknown | melanoma | not applicable | G-TPP + Venetoclax | Preclinical - Cell culture | Actionable | In a preclinical study, combination of the mitochondrial Hsp90 inhibitor G-TPP and the Bcl2 inhibitor Venclexta (venetoclax) resulted in increased apoptosis of melanoma cell lines in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | 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 | triple-receptor negative breast cancer | not applicable | Carboplatin + Gemcitabine + Trilaciclib | Phase II | Actionable | In a Phase II trial, Trilaciclib (G1T28) treatment in combination with Gemzar (gemcitabine) and Paraplatin (carboplatin) was well tolerated, but did not significantly improve myelosuppression in metastatic triple-negative breast cancer patients, and resulted in an objective response rate of 43% (n=60, 26 partial response, 24 stable disease), a median overall survival of 20.1 months, and a median progression-free survival of 8.8 months (PMID: 31575503; NCT02978716). | 31575503 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CFI-400945 | Phase I | Actionable | In a Phase I trial, treatment with CFI-400945 was tolerable and resulted in a clinical benefit rate of 6% (3/49; 1 partial response and 2 stable disease) in patients with advanced solid tumors, with 1 partial response in a patient with adenoid cystic carcinoma, and stable disease in 1 patient with KRAS-mutant microsatellite stable (MSS) colorectal cancer lasting 12 cycles with a 24% reduction in target lesions, and 1 patient with MSS ovarian cancer lasting 8 cycles (PMID: 31303643; NCT01954316) | 31303643 | |
| Unknown unknown | lung squamous cell carcinoma | not applicable | Prexasertib | Phase Ib/II | Actionable | In a Phase Ib trial, treatment with Prexasertib (LY2606368) resulted in a clinical benefit rate (complete response+partial response+stable disease) of 56% (9/16, all stable disease), and a median progression-free survival of 3.0 months in patients with squamous non-small cell lung cancer (PMID: 29643063; NCT0115790). | 29643063 | |
| Unknown unknown | prostate cancer | not applicable | Docetaxel + Everolimus | Phase I | Actionable | In a Phase I study, Afinitor (everolimus), in combination with Taxotere (docetaxel), demonstrated safety, but minimal efficacy in patients with prostate cancer (PMID: 25450031). | 25450031 | |
| Unknown unknown | B-cell lymphoma | not applicable | Alisertib | Phase II | Actionable | In a Phase II trial, Alisertib (MLN8237) treatment resulted in significant toxicity and an objective response rate of 15% (2/13) in patients with B-cell non Hodgkin's lymphoma (PMID: 28278718). | 28278718 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | AT7519 | Phase I | Actionable | In a Phase I trial, AT7519 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 25393368). | 25393368 | |
| Unknown unknown | leukemia | not applicable | H8F4 CAR-T cells | Preclinical - Cell culture | Actionable | In a preclinical study, h8F4 CAR-T cells induced lysis of leukemia cell lines expressing PR1/HLA-A2 in culture (PMID: 27265873). | 27265873 | |
| Unknown unknown | islet cell tumor | not applicable | Pictilisib | Preclinical | Actionable | In a preclinical study, Pictilisib (GDC-0941) inhibited AKT activation, reduced tumor burden, and increased lifespan in a transgenic mouse model of pancreatic neuroendocrine tumor (PMID: 27225693). | 27225693 | |
| Unknown unknown | 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 | lung non-small cell carcinoma | not applicable | Cabozantinib + Erlotinib | Phase Ib/II | Actionable | In a Phase Ib/II trial, Cometriq (cabozantinib) and Tarceva (erlotinib) combination treatment resulted in no response (0/13) in patients with non-small cell lung carcinoma that had progressed during treatment with Tarceva (erlotinib) in Phase II, despite an objective response rate of 8.2% (5/61) in Phase I (PMID: 28352985). | 28352985 | |
| Unknown unknown | 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 | Advanced Solid Tumor | not applicable | RXDX-105 | Phase I | Actionable | In a Phase I clinical trial, RXDX-105 (CEP-32496) was tolerable and demonstrated preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2574)). | detail... | |
| Unknown unknown | 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 | triple-receptor negative breast cancer | not applicable | TVB-2640 | Phase I | Actionable | In a Phase I clinical trial, TVB-2640 treatment resulted in stable disease for 20 weeks in one patient with triple-receptor negative breast cancer (2015 51 S724-S724 Eur J Cancer). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | BC2059 + Bortezomib | Preclinical - Patient cell culture | Actionable | In a preclinical study, the combination of BC2059 and Velcade (bortezomib) worked additively or synergistically in several human multiple myeloma cell lines and primary multiple myeloma cells with activated canonical Wnt signaling in culture (PMID: 28500235). | 28500235 | |
| Unknown unknown | 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 | prostate adenocarcinoma | not applicable | NSC23766 | Preclinical - Cell culture | Actionable | In a preclinical study, NSC23766 inhibited proliferation and invasion of a prostate adenocarcinoma cell line in culture (PMID: 15128949). | 15128949 | |
| 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 | non-Hodgkin lymphoma | not applicable | Entospletinib + Idelalisib | Phase II | Actionable | In a Phase II trial, Zydelig (idelalisib) and Entospletinib combination treatment resulted in objective response in 36% of patients with non-Hodgkin lymphoma, but was terminated due to severe treatment-emergent pneumonitis (PMID: 26968534). | 26968534 | |
| Unknown unknown | follicular lymphoma | not applicable | Betalutin | Phase Ib/II | Actionable | In a Phase I/II trial, Betalutin (177Lu Lilotomab Satetraxetan) treatment in patients with follicular lymphoma resulted in an overall response rate (ORR) of 65% (37/57, 17 complete, 20 partial, 10 stable disease) and median progression-free survival of 9.0 months, while patients who had received two or more prior therapies demonstrated an ORR of 70% (26/37, 12 complete, 14 partial), and those who were also rituximab-refractory had an ORR of 67% (14/21, 5 complete, 9 partial) (PMID: 32877524; NCT01796171). | 32877524 | |
| 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 | lymphoma | not applicable | GNE-272 | Preclinical - Cell culture | Actionable | In a preclinical study, GNE-272 treatment resulted in anti-proliferative activity in lymphoma cell lines in culture (PMID: 27682507). | 27682507 | |
| 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 | 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 | renal cell carcinoma | not applicable | Emibetuzumab + Ramucirumab | Phase Ib/II | Actionable | In a Phase Ib/II trial, Emibetuzumab (LY2875358) and Cyramza (ramucirumab) combination treatment resulted in an objective response rate of 0% and a disease control rate of 47% (7/15) in patients with renal cell carcinoma, with a median progression-free survival of 2.9 months (PMID: 31142504; NCT02082210). | 31142504 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | SGI-1027 | Preclinical - Cell culture | Actionable | In a preclinical study, SGI-1027, inhibited DNA methylation and reactivated silenced tumor suppressor genes, and induced DNMT1 degradation in a variety of cancer cell lines in culture (PMID: 19417133). | 19417133 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BXQ-350 | Phase I | Actionable | In a Phase I trial, BXQ-350 demonstrated safety and preliminary efficacy, resulted in partial response in 6% (1/17) and stable disease in 35% (6/17) of patients with advanced solid tumors (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 2517-2517; NCT02859857). | detail... | |
| Unknown unknown | colorectal cancer | not applicable | ICEC0942 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, ICEC0942 induced growth arrest of colorectal cancer cells in culture and in cell line xenograft models (PMID: 29545334). | 29545334 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BGT226 | Phase I | Actionable | In a Phase I trial, BGT226 treatment was tolerated, and resulted in stable disease as best response in 28% (5/18) of patients with advanced solid tumors (PMID: 31192075). | 31192075 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BGT226 | Phase I | Actionable | In a Phase I trial of BGT226 in patients with advanced solid tumors, limited preliminary antitumor activity and inconsistent target inhibition were observed (PMID: 22357447). | 22357447 | |
| Unknown unknown | 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 | 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 | renal cell carcinoma | not applicable | GDC-0980 | Phase II | Actionable | In a Phase II clinical trial, Apitolisib (GDC-0980) was inferior to Afinitor (everolimus) (median PFS, 3.7 months (n=42) vs. 6.1 months (n=43), respectively) and overall survival trended lower in the Apitolisib (GDC-0980) arm in patients with metastatic renal cell carcinoma (PMID: 26951309). | 26951309 | |
| Unknown unknown | ovarian cancer | not applicable | Eribulin + Volasertib | Preclinical - Cell culture | Actionable | In a preclinical study, Volasertib (BI 6727) and eribulin synergistically inhibited growth of ovarian cancer cell lines in culture (PMID: 32183025). | 32183025 | |
| Unknown unknown | ovarian carcinoma | no benefit | Motolimod + Pegylated liposomal-doxorubicin | Phase II | Actionable | In a Phase II trial, the inclusion of Motolimod (VTX-2337) with Doxil (pegylated liposomal-doxorubicin) did not result in improved survival in patients with either ovarian, fallopian tube, or primary peritoneal carcinoma (PMID: 28453702; NCT01666444). | 28453702 | |
| Unknown unknown | gastrointestinal system cancer | not applicable | Cetuximab + Fluorouracil + Leucovorin + Oxaliplatin | Phase II | Actionable | In a Phase II trial, Erbitux (cetuximab) in combination with FOLFOX (oxaliplatin, leucovorin, and fluorouracil) were well tolerated, and resulted in an overall response rate of 54.3% (35/66), median overall survival of 11.8 months; and median progression-free survival of 6.8 months in patients with metastatic esophageal or gastroesophageal junction cancers (PMID: 27382098). | 27382098 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Etoposide + NVP-ADW742 | Preclinical | Actionable | In a preclinical study, NVP-ADW742, when combined with Toposaur (etoposide), demonstrated a synergistic effect in small cell lung cancer cell lines, resulting in inhibition of Akt signaling (PMID: 15746061). | 15746061 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MK2206 + Paclitaxel | Phase Ib/II | Actionable | In a Phase Ib clinical trial, the combination of MK2206 and Taxol (paclitaxel) was well tolerated and demonstrated preliminary efficacy in patients with advanced solid tumors, with partial response in 24% (5/21) and stable disease in 43% (9/21) of patients (PMID: 25688104) | 25688104 | |
| Unknown unknown | pancreatic cancer | not applicable | CHIR-124 + Gemcitabine | Preclinical - Cell culture | Actionable | In a preclinical study, CHIR-124 and Gemzar (gemcitabine) demonstrated synergistic cytoxicity in pancreatic cancer cells in spheroid culture, resulting in increased DNA damage and apoptosis and decreased viability (PMID: 22244109). | 22244109 | |
| Unknown unknown | follicular lymphoma | not applicable | Bendamustine + Rituximab + Veliparib | Phase I | Actionable | In a Phase I trial, five patients with follicular lymphoma treated with a combination of Bendamustine, Rituxan (rituximab), and Veliparib (ABT-888) demonstrated a complete response (PMID: 28314788; NCT01326702). | 28314788 | |
| Unknown unknown | 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 | chordoma | not applicable | THZ1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, THZ1 treatment inhibited phosphorylation of Polr2a, reduced viability, and induced apoptosis in chordoma cells in culture, and inhibited tumor growth in a cell line xenograft model (PMID: 30664779). | 30664779 | |
| Unknown unknown | ovarian cancer | not applicable | KPT-185 | Preclinical - Patient cell culture | Actionable | In a preclinical study, KPT-185 inhibited growth of platinum-sensitive and platinum-resistant immortalized human ovarian cancer cell lines and patient-derived ovarian cancer cell lines in culture (PMID: 27649553). | 27649553 | |
| Unknown unknown | leukemia | not applicable | GNE-272 | Preclinical - Cell culture | Actionable | In a preclinical study, GNE-272 treatment resulted in anti-proliferative activity in leukemia cell lines in culture (PMID: 27682507). | 27682507 | |
| Unknown unknown | colon cancer | not applicable | Sunitinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Sutent (sunitinib) induced apoptosis in colon cancer cells in culture and in cell line xenograft models (PMID: 22912816). | 22912816 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Pembrolizumab | FDA approved | Actionable | In a Phase II trial (KEYNOTE-158) that supported FDA approval, Keytruda (pembrolizumab) treatment resulted in an objective response rate of 18.7% (20/107) in patients with advanced small-cell lung cancer, with a median progression-free survival of 2.0 months (J Clin Oncol 36, no. 15_suppl (May 20 2018) 8506-8506; NCT02628067). | detail... detail... | |
| Unknown unknown | neuroendocrine tumor | not applicable | Onatasertib | Phase Ib/II | Actionable | In a Phase Ib/II trial, treatment with Onatasertib (CC-223) in patients with well-differentiated neuroendocrine tumors of non-pancreatic gastrointestinal or unknown origin resulted in a partial response in 7.3% (3/41) of patients, stable disease in 82.9% (34/41), a median progression-free survival of 19.5 months, and tumor shrinkage in 73.2% (30/41) (PMID: 31527867). | 31527867 | |
| Unknown unknown | multiple myeloma | not applicable | Dexamethasone + Ixazomib + Lenalidomide | FDA approved | Actionable | In a Phase III trial (TOURMALINE-MM1) that supported FDA approval, addition of Ninlaro (ixazomib) to the regimen of Revlimid (lenalidomide) and dexamethasone significantly improved progression-free survival (20.6 vs 14.7 months, HR=0.74, p=0.01) in patients with relapsed or refractory multiple myeloma who received at least one prior therapy (PMID: 27119237; NCT01564537). | 27119237 detail... | |
| Unknown unknown | lung small cell carcinoma | not applicable | DT2216 + Venetoclax | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of DT2216 and Venclexta (venetoclax) demonstrated synergy in a small cell lung cancer cell line dependent on BCL-XL and BCL-2 for survival, resulting in decreased cell viability in culture, and led to increased tumor growth inhibition in xenograft models with a mean tumor growth inhibition of 98.2% (PMID: 31792461). | 31792461 | |
| Unknown unknown | 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 | colorectal cancer | no benefit | Fluorouracil + Leucovorin + Oxaliplatin + Tivozanib | Phase II | Actionable | In a Phase II trial, the combination of mFOLFOX-6 and Tivozanib (AV-951) in patients with metastatic colorectal cancer did not result in a greater progression free survival when compared to the combined therapy of mFOLFOX-6 and Avastin (bevacizumab) (9.8 vs 9.5 mo, respectively) (PMID: 27401244). | 27401244 | |
| Unknown unknown | renal cell carcinoma | not applicable | Bevacizumab + Temsirolimus | Phase II | Actionable | In a Phase II trial, patients with metastatic renal cell carcinoma (mRCC) treated with a combination of Avastin (bevacizumab) and Torisel (temsirolimus) demonstrated an overall 4 month PFS rate of 65% while those patients with VEGFR tyrosine kinase inhibitor refractory mRCC showed a median PFS and OS of 6.5 and 9.6 months, respectively (PMID: 27036973). | 27036973 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Riluzole | Preclinical - Cell culture | Actionable | In a preclinical study, Rilutek (riluzole) inhibited growth of hepatocellular cancer cell lines and primary hepatocellular cancer lines in culture (PMID: 27612558). | 27612558 | |
| Unknown unknown | multiple myeloma | not applicable | belantamab mafodotin-blmf | Phase I | Actionable | In a Phase I trial, Blenrep (belantamab mafodotin-blmf) demonstrated manageable safety and preliminary activity in patients with multiple myeloma, with 60% (21/35) of patients in part 2 receiving the recommended phase 2 dose achieving a response (1 stringent complete response (CR), 1 CR, 2 very good partial responses (PR), and 3 PR) (PMID: 30442502; NCT02064387). | 30442502 | |
| Unknown unknown | multiple myeloma | not applicable | belantamab mafodotin-blmf | FDA approved | Actionable | In a Phase II trial (DREAMM-2) that supported FDA approval, Blenrep (belantamab mafodotin-blmf) treatment demonstrated manageable safety profile, and resulted in an overall response rate of 31% (30/97) at 2.5 mg/kg in patients with relapsed or refractory multiple myeloma who had received 4 or more prior treatments (PMID: 31859245; NCT03525678). | 31859245 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 | multiple myeloma | not applicable | JNJ-64007957 | Phase I | Actionable | In a Phase I trial, Teclistamab (JNJ-64007957) treatment demonstrated safety and preliminary efficacy, resulted in an overall response rate (ORR) of 38% (20/52) in patients with advanced relapsed or refractory multiple myeloma, with an ORR of 78% (7/9) in the highest dose group (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 100-100; NCT03145181). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Adavosertib + Carboplatin | Phase I | Actionable | In a Phase I trial, the combination of Adavosertib (MK-1775) and Paraplatin (carboplatin) resulted in a partial response in 6.5% (4/62) of patients and stable disease in 45% (28/62) of patients, all with advanced solid tumors (PMID: 27601554). | 27601554 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Adavosertib + Carboplatin | Phase Ib/II | Actionable | In a Phase Ib trial, Adavosertib (MK-1775) in combination with Paraplatin (carboplatin) was tolerable and resulted in partial response in 16.7% (1/3) of patients and stable disease in 33.3% (2/6) of Asian patients with advanced solid tumors (PMID: 32034630). | 32034630 | |
| Unknown unknown | 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 | 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 | 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 | kidney cancer | not applicable | CVX-060 + Sunitinib | Preclinical | Actionable | In a preclinical study, the combination of CVX-060 and Sutent (sunitinib) demonstrated a trend improved overall survival compared to single agent Sutent (sunitinib) in mouse models of unresected and resected renal cancer, however, also demonstrated increased toxicity (PMID: 27651308). | 27651308 | |
| Unknown unknown | breast cancer | not applicable | GA001 | Preclinical - Cell culture | Actionable | In a preclinical study, GA001 treatment in breast cancer cells resulted in increased AMPK activity and subsequent autophagy, inhibition of cell proliferation, and induced apoptosis in culture (PMID: 28460359). | 28460359 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | JPH203 + Metformin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of JPH203 (KYT-0353) and Glucophage (metformin) decreased proliferation of head and neck squamous cell cancer cell lines in culture, and reduced tumor growth in a head and neck squamous cell cancer cell line xenograft model (PMID: 27262901). | 27262901 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | AT-7867 | Preclinical - Cell culture | Actionable | In a preclinical study, AT-7867 inhibited the growth of diffuse large B-cell lymphoma cell lines in culture (PMID: 26824321). | 26824321 | |
| Unknown unknown | 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 | 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 | liposarcoma | not applicable | Alisertib | Phase II | Actionable | In a Phase II trial, Alisertib (MLN8237) treatment resulted in twelve-week progression free survival in 73% (9/12) of liposarcoma patients (PMID: 27502708). | 27502708 | |
| Unknown unknown | colorectal cancer | not applicable | WYE-687 | Preclinical | Actionable | In a preclinical study, WYE-687 induced cell cycle arrest and inhibited cell proliferation of colorectal cancer cells in culture (PMID: 19584280). | 19584280 | |
| 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 | lung non-small cell carcinoma | not applicable | Carboplatin + Paclitaxel + Vorinostat | Phase II | Actionable | In a Phase II trial, the combination of Paraplatin (carboplatin) and Taxol (paclitaxel) plus Zolinza (vorinostat) compared to the addition of placebo resulted in a greater median progression-free survival (6.0mo vs 4.1mo) and overall survival (13.0 mo vs 9.7 mo) in patients with non-small cell lung carcinoma (PMID: 19933908). | 19933908 | |
| Unknown unknown | 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 | 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 | T-cell lymphoblastic leukemia/lymphoma | no benefit | AZD1208 + Ruxolitinib | Preclinical | Actionable | In a preclinical study, Jakafi (ruxolitinib) and AZD1208 combination treatment did not inhibit proliferation of a T-cell leukemia cell line in culture (PMID: 26472029). | 26472029 | |
| Unknown unknown | malignant fibrous histiocytoma | not applicable | EHop-016 + Sapanisertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of EHop-016 and Sapanisertib (MLN0128) resulted in increased apoptosis and reduced growth of a myxofibrosarcoma cell line in culture, and resulted in greater tumor growth inhibition in myxofibrosarcoma cell line xenograft models compared to either agent alone (PMID: 27577794). | 27577794 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Rivoceranib | Phase II | Actionable | In a Phase II trial, 500 mg of Apatinib (YN968D1) produced a median progression-free survival of 3.3 months in TNBC patients (PMID: 24604288). | 24604288 | |
| Unknown unknown | astrocytoma | not applicable | Niraparib + Radiotherapy | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a pediatric high grade astrocytoma cell line treated with a combination of ionizing radiation and Zejula (niraparib) demonstrated a greater reduction in cell survival in culture and a better survival benefit in xenograft models compared to either agent alone (PMID: 26351319). | 26351319 | |
| Unknown unknown | ovarian cancer | not applicable | Atezolizumab + Hu5F9-G4 | Phase I | Actionable | In a Phase Ib trial, Tecentriq (atezolizumab) and Magrolimab (Hu5F9-G4) combination therapy demonstrated acceptable safety, and resulted in stable disease as best response in 56% (10/18) of patients with platinum-resistant ovarian cancer (J Clin Oncol 38, 2020 (suppl 5; abstr 18); NCT03558139). | detail... | |
| Unknown unknown | fibrous histiocytoma | not applicable | Pembrolizumab | Phase II | Actionable | In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in complete response in 10% (1/10) and partial response in 30% (3/10) of patients with undifferentiated pleomorphic sarcoma (malignant fibrous histiocytoma) (J Clin Oncol 35, 2017 (suppl; abstr 11008)). | detail... | |
| Unknown unknown | bladder carcinoma in situ | not applicable | Oportuzumab monatox | Phase II | Actionable | In a Phase II trial, treatment with Oportuzumab monatox (VB4-845) was well-tolerated and resulted in complete response in 44% (20/45) of patients with bladder carcinoma in situ refractory to bacillus Calmette-Guerin (BCG) therapy (PMID: 22998907; NCT00462488) | 22998907 | |
| Unknown unknown | prostate cancer | not applicable | CX-6258 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CX-6258 inhibited tumor growth in human prostate cancer cell line xenograft models (PMID: 24900437). | 24900437 | |
| Unknown unknown | 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 | 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 | esophagus squamous cell carcinoma | not applicable | YM155 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with YM155 induced non-apoptotic cell death in esophageal squamous cell carcinoma (ESCC) cell lines in culture, and inhibited tumor growth in ESCC cell line xenograft models (PMID: 26090615). | 26090615 | |
| Unknown unknown | laryngeal carcinoma | not applicable | ME22S | Preclinical | Actionable | In a preclinical study, ME22S inhibited cell migration, invasion, and proliferation of human laryngeal carcinoma cell lines in culture and inhibited tumor growth in laryngeal carcinoma xenograft models (PMID: 26812910). | 26812910 | |
| 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 | 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 | AZD3965 | Phase I | Actionable | In a Phase I trial, AZD3965 treatment demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Annals of Oncology, Volume 29, Issue suppl_3, 1 March 2018, abstract 500). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Durvalumab | FDA approved | Actionable | In a Phase III trial (PACIFIC) that supported FDA approval, Imfinzi (durvalumab) treatment resulted significantly improved median progression-free survival (PFS) (16.8 vs 5.6 months, HR=0.52, p<0.001), and superior 12-month PFS rate (55.9% vs 35,3%), 18-month PFS rate (44.2% vs 27.0%), response rate (28.4% vs 16%), and median time to death or distant metastasis (23.2 vs 14.6 months) compared to placebo in patients with unresectable, non-small cell lung cancer (PMID: 28885881; NCT02125461). | 28885881 detail... | |
| Unknown unknown | colorectal adenocarcinoma | not applicable | Berzosertib + Irinotecan | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of VX-970 and Camptosar (irinotecan) synergized to inhibit proliferation of a colorectal adenocarcinoma cell line in culture and to inhibit tumor growth in a human colorectal adenocarcinoma cell line xenograft model (PMID: 25269479). | 25269479 | |
| Unknown unknown | ovarian cancer | not applicable | VS-5584 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, VS-5584 reduced the proportion of cancer stem cells in primary ovarian tumors in culture, and reduced tumorigenicity of dissociated human ovarian tumors in xenograft models (PMID: 25432176). | 25432176 | |
| 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 | 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 | thyroid gland cancer | not applicable | CLM3 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CLM3 induced apoptosis and inhibited EGFR phosphorylation and cell proliferation in human anaplastic thyroid cancer cell lines in culture and inhibited tumor growth in xenograft models (PMID: 24423321). | 24423321 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | PF-00562271 | Phase I | Actionable | In a Phase I trial, PF-00562271 was well-tolerated and resulted in stable disease in 34% (31/91) of patients with advanced solid tumors as well as a metabolic response of 50% (7/14) (PMID: 22454420). | 22454420 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | PF-00562271 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, PF-00562271 inhibited PTK2 (FAK) phosphorylation and growth of multiple tumor cell types in cell line xenograft models (PMID: 18339875). | 18339875 | |
| Unknown unknown | glioblastoma multiforme | not applicable | WT2725 | Phase I | Actionable | In a Phase I trial, WT2725 treatment resulted in survival for more than a year in 33% (7/21) of patients with progressive or recurrent glioblastoma, with 3 patients survived over 18 months, 2 survived over 2 years (both in complete radiologic remission) (Journal of Clinical Oncology 35, no. 15_suppl (May 20 2017) 2066-2066; NCT01621542). | detail... | |
| 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 | hepatocellular carcinoma | not applicable | CEBPA-51 | Preclinical - Cell culture | Actionable | In a preclinical study, CEBPA-51 treatment resulted in induced expression of CEBPA mRNA and inhibited proliferation of hepatocellular carcinoma cells in culture (PMID: 28882451). | 28882451 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | CEBPA-51 | Phase I | Actionable | In a Phase I trial, CEBPA-51 treatment resulted in increased Cebpa expression in WBC, stable disease over 4 months in 40% (4/10) of patients with hepatocellular carcinoma, with 1 patient achieved partial response for 18 months (J Clin Oncol, 36(no. 15_suppl), May 2018, 2509-2509; NCT02716012). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | Olaparib + THZ1 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with Lynparza (olaparib) combined with THZ1 resulted in a synergistic effect in homologous recombination-proficient ovarian cancer cells, demonstrating decreased cell survival and reduced anchorage-independent growth in culture, and inhibition of tumor growth in cell line xenograft models (PMID: 32668240). | 32668240 | |
| Unknown unknown | Hodgkin's lymphoma | not applicable | Ixazomib + JQ1 | Preclinical | Actionable | In a preclinical study, Ixazomib (MLN9708) and JQ1 combination treatment resulted in increased apoptosis in Hodgkin's lymphoma cells in culture (PMID: 26988986). | 26988986 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | AMG 900 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AMG 900 induced apoptosis in triple-negative breast cancer (TNBC) cell lines in culture and inhibited tumor growth in TNBC cell line xenograft models (PMID: 23990115). | 23990115 | |
| Unknown unknown | 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 | triple-receptor negative breast cancer | not applicable | YLL545 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, YLL545 inhibited proliferation of a human triple-negative breast cancer (TNBC) cell line in culture, and inhibited activation of STAT3 and ERK and decreased tumor growth in TNBC cell line xenograft models (PMID: 27203384). | 27203384 | |
| Unknown unknown | 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 | triple-receptor negative breast cancer | not applicable | BETd-246 + BM-1197 | Preclinical - Cell culture | Actionable | In a preclinical study, the Bet inhibitor BETd-246 and the Bcl-xl inhibitor BM-1197 synergistically induced apoptosis in triple-receptor negative breast cancer cell lines in culture (PMID: 28209615). | 28209615 | |
| Unknown unknown | neuroblastoma | no benefit | Doxorubicin + Prexasertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Adriamycin (doxorubicin) and Prexasertib (LY2606368) did not demonstrate benefit over Prexasertib (LY2606368) alone in neuroblastoma cell line xenograft models (PMID: 28270495). | 28270495 | |
| Unknown unknown | breast cancer | not applicable | Tanibirumab | Preclinical - Cell culture | Actionable | In a preclinical study, Tanibirumab (TTAC-0001) inhibited angiogenesis in a cell culture assay with human breast cancer cells (PMID: 26325365). | 26325365 | |
| Unknown unknown | 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 | 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 | renal carcinoma | not applicable | Hydroxyurea + MU380 | Preclinical - Cell culture | Actionable | In a preclinical study, the addition of MU380 resulted in increased sensitivity to Droxia (hydroxyurea) in a renal cell carcinoma cell line in culture, leading to decreased proliferation (PMID: 28619751). | 28619751 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Docetaxel + Glesatinib | Phase I | Actionable | In a Phase I trial, Glesatinib (MGCD265) and Taxotere (docetaxel) combination treatment resulted in partial response in 12% (4/34) and stable disease for 6 cycles or more in 18% (6/34) of patients with advanced solid tumors (J Clin Oncol 30, 2012 (suppl; abstr e13604)). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | Cytarabine + GDC-0575 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, combination of GDC-0575 and Cytosar-U (cytarabine) resulted in enhanced killing of acute myeloid leukemia cells in both cell line and patient-derived xenograft models (PMID: 29162833). | 29162833 | |
| Unknown unknown | thyroid gland papillary carcinoma | not applicable | Lenvatinib | Clinical Study | Actionable | In a clinical study, Lenvima (lenvatinib) treatment resulted in prolonged response in a papillary thyroid carcinoma patient whose disease progressed despite 3 prior therapies including Nexavar (sorafenib), Sutent (sunitinib), and Votrient (pazopanib) (PMID: 29167691). | 29167691 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | A-1155463 + BETd-246 | Preclinical - Cell culture | Actionable | In a preclinical study, the Bet inhibitor BETd-246 and the Bcl-xl inhibitor A-1155463 synergistically induced apoptosis in triple-receptor negative breast cancer cell lines in culture (PMID: 28209615). | 28209615 | |
| Unknown unknown | B-cell lymphoma | not applicable | Urelumab | Phase I | Actionable | In a Phase I trial, Urelumab (BMS-663513) demonstrated manageable safety profile, resulted in an objective response rate of 17% (2/12) and a disease control rate of 42% (5/12) in patients with relapsed or refractory B-cell lymphomas, with a median progression-free survival of 13.4 months and a median overall survival not reached (PMID: 32052473; NCT01471210). | 32052473 | |
| 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 | AZD8186 | Phase I | Actionable | In a Phase I trial, AZD8186 demonstrated safety and preliminary efficacy in a variety of advanced solid tumor patients (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr CT329). | detail... | |
| Unknown unknown | transitional cell carcinoma | not applicable | Durvalumab | FDA approved | Actionable | In a Phase I/II trial that supported FDA approval, treatment with Imfinzi (durvalumab) was well tolerated and resulted in an overall response rate of 17.8% (34/191; 7 complete responses), median progression-free survival of 1.5 months, and median overall survival of 18.2 months in patients with advanced urothelial cancer (PMID: 28817753; NCT01693562). | detail... detail... 28817753 | |
| Unknown unknown | pancreatic cancer | not applicable | Trabedersen | Phase Ib/II | Actionable | In a Phase Ib/II trial, Trabedersen (AP 12009) treatment followed by chemotherapy resulted in improved median overall survival (9.4 vs 2.8 months, p=0.0004) compared to Trabedersen (AP 12009) treatment followed by best supportive care in patients with advanced pancreatic cancer (AACR Annual Meeting 2019, Abstract 3968). | detail... | |
| Unknown unknown | pancreatic cancer | not applicable | Trabedersen | Phase Ib/II | Actionable | In a Phase I/II trial, Trabedersen (AP 12009) treatment followed by chemotherapy resulted in an overall survival of 14.5 months in advanced pancreatic cancer patients (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 119P). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | AT9283 | Phase I | Actionable | In a Phase I clinical trial, AT9283 demonstrated safety and efficacy in patients with advanced solid tumors (PMID: 22015452). | 22015452 | |
| Unknown unknown | thyroid gland carcinoma | not applicable | Obatoclax | Preclinical | Actionable | In a preclinical study, Obatoclax induced cell death in human thyroid carcinoma cell lines culture (PMID: 26198850). | 26198850 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Carboplatin + 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 | 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 | pancreatic adenocarcinoma | not applicable | Gemcitabine + Vandetanib | Phase I | Actionable | In a Phase I trial, Caprelsa (vandetanib), in combination with Gemzar (gemcitabine), demonstrated safety and resulted in stable disease in metastatic pancreatic adenocarcinoma patients (PMID: 21921646). | 21921646 | |
| Unknown unknown | 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 | lung non-small cell carcinoma | not applicable | Vandetanib | Phase II | Actionable | In a Phase II trial, Caprelsa (vandetanib) alone, or in combination with Taxotere (docetaxel), improved progression-free survival of NSCLC patients (PMID: 17243944). | 17243944 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Vandetanib | Clinical Study | Actionable | In a meta-analysis of 2,284 NSCLC patients, Caprelsa (vandetanib) in combination with chemotherapy, increased progression-free survival (PFS) and overall response rate (ORR) but not overall survival (PMID: 23861835). | 23861835 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Vandetanib | Phase II | Actionable | In a Phase II trial, treatment with Caprelsa (vandetanib) increased progression-free survival in NSCLC patients, compared to Iressa (gefitinib) (PMID: 19332730). | 19332730 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Pimitespib | Phase I | Actionable | In a Phase I trial, TAS-116 demonstrated safety and resulted in a disease control rate of 27% (16/60; including stable disease for greater than or equal to 12 weeks (13) and partial responses (3)), in patients with advanced solid tumors, with partial responses in 2 patients with non-small cell lung cancer and 1 patient with gastrointestinal stromal tumor (PMID: 30679388; NCT02965885). | 30679388 | |
| Unknown unknown | pancreatic cancer | not applicable | RX-3117 | Phase Ib/II | Actionable | In a Phase I/II trial, RX-3117 demonstrated safety and preliminary efficacy, resulted in durable stable disease in 25% (2/8) of patients with pancreatic cancer (Journal of Clinical Oncology 35, no. 4_suppl (February 1 2017) 445-445; NCT02030067). | detail... | |
| Unknown unknown | ovarian carcinoma | not applicable | Topotecan + Veliparib | Phase I | Actionable | In a Phase I trial, the combination of Veliparib (ABT-888) and topotecan demonstrated safety and tolerability, and resulted in an objective response rate of 9% (4/45; 1 complete response and 3 partial responses) and stable disease in 21 patients with ovarian carcinoma (PMID: 29138343; NCT01012817). | 29138343 | |
| 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 | Everolimus + Vatalanib | Phase I | Actionable | In a Phase I trial, Vatalanib and Afinitor (everolimus) combination therapy demonstrated acceptable toxicity, resulted in a partial response in 12.9% (9/70) and stable disease in 58.6% (41/70) of patients with advanced solid tumors (PMID: 32328844; NCT00655655). | 32328844 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Cirmtuzumab | Phase I | Actionable | In a Phase I trial, treatment with Cirmtuzumab (UC-961) was safe and well-tolerated and resulted in stable disease in 77% (17/22) of chronic lymphocytic leukemia patients, and a median time to next treatment of 8.6 months (PMID: 29859176). | 29859176 | |
| Unknown unknown | pancreatic cancer | not applicable | MitoMet-10 | Preclinical | Actionable | In a preclinical study, MitoMet-10 decreased tumor growth in mouse models of pancreatic cancer (PMID: 27216187). | 27216187 | |
| Unknown unknown | acute monocytic leukemia | not applicable | Bortezomib + Tinostamustine | Preclinical - Cell culture | Actionable | In a preclinical study, EDO-S101 and Velcade (bortezomib) worked synergistically to decrease viability of an acute monocytic leukemia cell line in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | pancreatic cancer | not applicable | Chidamide | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Chidamide (CS055) inhibited growth of a human pancreatic cancer cell line in culture and inhibited tumor growth in xenograft models (PMID: 25384499). | 25384499 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Copanlisib | Phase II | Actionable | In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 17% (1/6) and partial response in 67% (4/6) of patients with mantle cell lymphoma (PMID: 24852792). | 24852792 | |
| Unknown unknown | breast cancer | not applicable | KW-2450 | Preclinical | Actionable | In a preclinical study, KW-2450 inhibited growth of several breast cancer cell lines in culture, including ESR1 positive, ERBB2 (HER2) positive/ESR1 positive, ERBB2 (HER2) positive, and triple-negative subtypes (PMID: 26443806). | 26443806 | |
| Unknown unknown | thyroid gland carcinoma | not applicable | MLN8054 | Preclinical - Patient cell culture | Actionable | In a preclinical study, MLN8054 inhibited growth of tumor cells derived from a patient with anaplastic thyroid carcinoma (PMID: 27379749). | 27379749 | |
| Unknown unknown | liver cancer | not applicable | Flucytosine + TG6002 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with TG6002 resulted in enhanced antitumor activity when combined with Flucytosine in a liver cancer cell line xenograft model, demonstrating inhibition of tumor growth (PMID: 31011628). | 31011628 | |
| Unknown unknown | breast cancer | not applicable | Camptothecin + NU6027 | Preclinical - Cell culture | Actionable | In a preclinical study, NU6027 enhanced the efficacy of Camptothecin in breast cancer cells in culture, resulting in decreased cell survival (PMID: 21730979). | 21730979 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Carboplatin + Paclitaxel + Veliparib | Phase II | Actionable | In a Phase II trial, the combination of Veliparib (ABT-888) with Paraplatin (carboplatin) and Taxol (paclitaxel) resulted in both an improved median PFS (5.8 mo vs 4.2 mo) and median OS (11.7 mo vs 9.1 mo) compared to placebo plus Paraplatin (carboplatin) and Taxol (paclitaxel) in patients with non-small cell lung carcinoma (PMID: 27803064). | 27803064 | |
| Unknown unknown | synovial sarcoma | not applicable | SU6656 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, SU6656 treatment decreased cell proliferation of synovial sarcoma cells in culture and inhibited tumor growth and blocked tumor invasion in cell line xenograft models (PMID: 22244830). | 22244830 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Ibrutinib + Silmitasertib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Silmitasertib (CX-4945) and Imbruvica (ibrutinib) worked synergistically to decrease cell viability and resulted in increased apoptosis and decreased AKT phosphorylation in ABC and GCB-type diffuse large B-cell lymphoma cell lines in culture (PMID: 28460620). | 28460620 | |
| Unknown unknown | Ewing sarcoma | not applicable | JQ1 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, JQ1 treatment resulted in cytostatic effects in patient-derived Ewing's sarcoma cell lines in culture, but inhibited tumor growth in patient-derived xenograft models due to inhibition of tumor angiogenesis (PMID: 26908627). | 26908627 | |
| Unknown unknown | lung cancer | not applicable | CAB-AXL-ADC | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CAB-AXL-ADC treatment inhibited tumor growth in a lung cancer cell line xenograft model (Cancer Res 2018;78(13 Suppl):Abstract nr 827). | detail... | |
| Unknown unknown | B-cell lymphoma | not applicable | CPI-1205 | Phase I | Actionable | In a Phase I trial, CPI-1205 treatment resulted in complete response in 3% (1/32), and stable disease in 16% (5/32) of patients with B-cell lymphomas (Annals of Oncology, Volume 29, Issue suppl_3, 1 March 2018, abstract 420; NCT02395601). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | Dexamethasone + Marizomib + Pomalidomide | Phase I | Actionable | In a Phase I trial, Marizomib (NPI-0052) in combination with Pomalyst (pomalidomide) and low-dose dexamethasone resulted in an overall response rate of 53% (19/36) and a clinical benefit rate of 64% (23/36) in patients with relapsed and refractory multiple myeloma (PMID: 29076150). | 29076150 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Sacituzumab govitecan-hziy | Case Reports/Case Series | Actionable | In a Phase I/II trial (IMMU-132-01), Trodelvy (sacituzumab govitecan-hziy) treatment resulted in a partial response in a patient with triple-negative breast cancer (PMID: 25944802; NCT01631552). | 25944802 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Sacituzumab govitecan-hziy | FDA approved | Actionable | In a Phase I/II trial (IMMU-132-01) that supported FDA approval, Trodelvy (sacituzumab govitecan-hziy) treatment resulted in an objective response rate of 33.3% (36/108, 3 complete response, 33 partial response) and a clinical benefit rate of 45.4% in patients with metastatic triple-negative breast cancer who received two or more prior therapies, with a median duration of response, progression-free survival, and overall survival of 7.7, 5.5, and 13.0 months respectively (PMID: 30786188; NCT01631552). | 30786188 detail... | |
| Unknown unknown | sarcoma | not applicable | Abexinostat + Aldoxorubicin | Phase I | Actionable | In a Phase I study, Abexinostat (PCI-24781), in combination with doxorubicin, displayed safety and preliminary efficacy in patients with metastatic sarcoma (PMID: 25536954). | 25536954 | |
| Unknown unknown | melanoma | not applicable | G-TPP + WEHI-539 | Preclinical - Cell culture | Actionable | In a preclinical study, combination of the mitochondrial Hsp90 inhibitor G-TPP and the Bcl-xL inhibitor WEHI-539 resulted in increased apoptosis of melanoma cell lines in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | prostate cancer | not applicable | AB928 + Doxorubicin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of AB928 and Adriamycin (doxorubicin) resulted in a increase in tumor cell specific CD8+ T cells and increased tumor growth inhibition compared to chemotherapy alone in prostate cancer cell line xenograft models (European Journal of Cancer, Vol 92, S14-S15). | detail... | |
| Unknown unknown | stomach cancer | not applicable | AZD6738 + Paclitaxel | Preclinical - Cell culture | Actionable | In a preclinical study, the addition of AZD6738 to Taxol (paclitaxel) treatment in a gastric cancer cell line in culture resulted in enhanced chemotherapeutic sensitivity, demonstrating a synergistic effect (PMID: 28138034). | 28138034 | |
| Unknown unknown | cervical cancer | not applicable | AsiDNA | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA inhibited survival of cervical cancer cell lines in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Gemcitabine + LY2780301 | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of LY2780303 and Gemzar (gemcitabine) demonstrated some antitumor activity in patients with advanced solid tumors including 2 patients with a partial response, 28 patients with stable disease, and a four month disease control rate of 22% (PMID: 28750271). | 28750271 | |
| Unknown unknown | 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 | 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 | 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 | alveolar soft part sarcoma | not applicable | Durvalumab + Mogamulizumab | Case Reports/Case Series | Actionable | In a Phase I trial, a patient with alveolar soft part sarcoma achieved a partial response after 3.68 months with a response duration of 10.6 months following treatment with the combination of Poteligeo (mogamulizumab-kpkc) and Imfinzi (durvalumab) (PMID: 32586937; NCT02301130). | 32586937 | |
| Unknown unknown | multiple myeloma | not applicable | Dexamethasone + Pegylated liposomal-doxorubicin + Selinexor | Phase I | Actionable | In a Phase I trial, treatment with the combination of Selinexor (KPT-330), Doxil (pegylated liposomal doxorubicin), and Dexamethasone resulted in very good partial response in 20% (2/10), partial response in 20% (2/10), minimal response in 20% (2/10), and stable disease in 30% (3/10) of patients with relapsed or refractory multiple myeloma (J Clin Oncol 34, 2016 (suppl; abstr 8013)). | detail... | |
| Unknown unknown | thyroid gland medullary carcinoma | not applicable | Sorafenib | Phase II | Actionable | In a Phase II trial, Nexavar (sorafenib) treatment resulted in partial response in 6.3% (1/16) and stable disease in 87.5% (14/16) of patients with sporadic medullary thyroid carcinoma, with a median progression free survival of 17.9 months (PMID: 20368568). | 20368568 | |
| Unknown unknown | esophageal cancer | not applicable | BI-847325 | Phase I | Actionable | In a Phase I trial, BI-847325 treatment resulted in stable disease in 30% (21/69) of patients with advanced solid tumors, and one partial response for 67 days in a patient with esophageal cancer (PMID: 26650227). | 26650227 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Bemcentinib + Cetuximab | Preclinical - Pdx | Actionable | In a preclinical study, Bemcentinib (BGB-324) treatment in combination with Erbitux (cetuximab) synergistically inhibited proliferation, induced apoptosis, and delayed tumor growth in patient-derived xenograft (PDX) models of head and neck squamous cell carcinoma (PMID: 32439698). | 32439698 | |
| Unknown unknown | liposarcoma | not applicable | Pembrolizumab | Phase II | Actionable | In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 20% (2/10) of patients with dedifferentiated liposarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). | detail... | |
| Unknown unknown | urinary bladder cancer | not applicable | BC-819 + BCG solution | Phase II | Actionable | In a Phase II trial, combination of BV-819 and BCG treatment resulted in a recurrence-free survival rate of 54.1% and a progression-free survival rate of 75.7% at 24 months in patients with non-muscle invasive bladder cancer (Journal of Clinical Oncology 36, no. 6_suppl (February 20 2018) 499-499; NCT01878188). | detail... | |
| Unknown unknown | gastrointestinal stromal tumor | not applicable | Ponatinib | Phase II | Actionable | In a Phase II trial, Iclusig (ponatinib) demonstrated preliminary activity in patients with advanced gastrointestinal stromal tumor (J Clin Oncol (Meeting Abstracts) 2014 32: 10506). | detail... | |
| Unknown unknown | gastric adenocarcinoma | not applicable | Celecoxib + Sirolimus | Preclinical | Actionable | In a preclinical study, celecoxib enhanced the efficacy of Rapamune (sirolimus) by increasing the growth inhibition of gastric cancer cells in culture (PMID: 25701378). | 25701378 | |
| Unknown unknown | lung carcinoma | not applicable | TP-1454 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TP-1454 treatment inhibited viability of an epithelial lung carcinoma cell line in culture, and reduced tumor growth in a cell line xenograft model (Mol Cancer Ther 2019;18(12 Suppl):Abstract nr B080). | detail... | |
| Unknown unknown | lymphoma | not applicable | Bendamustine + Veliparib | Phase I | Actionable | In a Phase I trial, seven patients with lymphoma treated with a combination of Veliparib (ABT-888) and Bendamustine demonstrated an overall response rate of 71% (5/7) and complete response rate of 57% (4/7) and progression free survival of 6.9 months (PMID: 28314788; NCT01326702). | 28314788 | |
| Unknown unknown | renal cell carcinoma | not applicable | 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 | Advanced Solid Tumor | not applicable | Romidepsin | Phase I | Actionable | In a Phase I trial, Istodax (romidepsin), in combination with Gemzar (gemcitabine), demonstrated safety and some efficacy in patients with a variety of advanced solid tumors (J Clin Oncol, May 2008 vol. 26 no. 15_suppl 2567). | detail... | |
| Unknown unknown | non-Hodgkin lymphoma | not applicable | Betalutin | Phase Ib/II | Actionable | In a Phase I/II trial, Betalutin (177Lu Lilotomab Satetraxetan) treatment in patients with non-Hodgkin lymphoma (follicular, mantle cell, or marginal zone subtypes) resulted in an overall response rate of 61% (45/74, 22 complete, 23 partial, 14 stable disease), median duration of response of 13.6 months, and median progression-free survival of 8.8 months, with an overall response rate of 65% (37/57, 17 complete, 20 partial, 10 stable disease) in follicular lymphoma patients (PMID: 32877524; NCT01796171). | 32877524 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | E7107 | Phase I | Actionable | In a Phase I trial, E7107 treatment resulted in stable disease in 31% (8/26) of patients with advanced solid tumors, however, the study was discontinued due to vision loss in two patients (PMID: 24258465; NCT00499499). | 24258465 | |
| Unknown unknown | 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 | pancreatic cancer | no benefit | Capecitabine + Gemcitabine + Tertomotide | Phase III | Actionable | In a Phase III trial (TeloVac), addition of Tertomotide (GV1001) sequentially or concurrently to chemotherapy consisting of Xeloda (capecitabine) and Gemzar (gemcitabine) did not improve median overall survival compared to chemotherapy alone (6.9, 8.4, and 7.9 months, respectively) in patients with locally advanced or metastatic pancreatic cancer (PMID: 24954781). | 24954781 | |
| 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 | melanoma | not applicable | Prolgolimab | Phase II | Actionable | In a Phase II trial (MIRACULUM), Prolgolimab demonstrated safety and preliminary efficacy, resulted in an objective response rate (ORR) of 38% (24/63, 5 complete response (CR), 19 partial response (PR)) and a disease control rate (DCR) of 64% when administered at 1mg/kg Q2W, and an ORR of 29% (18/63, 2 CR, 16 PR) with a DCR of 46% when administered at 3mg/kg Q3W, in patients with advanced melanoma (Annals of Oncology, Volume 30, Issue Supplement_11, December 2019, Abstract 119P; NCT03269565). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Mirvetuximab Soravtansine | Phase I | Actionable | In a Phase I trial, Mirvetuximab Soravtansine (IMGN853) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, with a clinical benefit rate of 23% (12/43), including partial response in 2 patients, both with epithelial ovarian cancer, CA125 response in 5 patients, and stable disease for greater than 4 months in 5 patients (PMID: 28440955). | 28440955 | |
| 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 | melanoma | not applicable | Atezolizumab + Cobimetinib | Phase I | Actionable | In a Phase Ib trial, Tecentriq (atezolizumab) and Cotellic (cobimetinib) combination treatment demonstrated safety and preliminary clinical activity, resulted in a confirmed response in 41% (9/22) of patients with melanoma, regardless of KRAS/BRAF status (PMID: 30918950; NCT01988896). | 30918950 | |
| 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 | gastric adenocarcinoma | not applicable | Regorafenib | Phase II | Actionable | In a Phase II trial, Stivarga (regorafenib) resulted in a PFS of 2.6 months compared to .9 months with placebo in gastric adenocarcinoma patients (PMID: 27325864). | 27325864 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | BAZ2-ICR + BI-9564 + JQ1 | Preclinical - Cell culture | Actionable | In a preclinical study, BAZ2-ICR treatment combined with BI-9564 and JQ1 inhibited Rb1 phosphorylation, induced senescence and complete growth inhibition, and led to enhanced cell cycle arrest in triple-negative breast cancer cell lines in culture (PMID: 31000582). | 31000582 | |
| Unknown unknown | malignant mesothelioma | not applicable | Ad-NK4 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Ad-NK4 decreased phosphorylation of Met and Akt, decreased beta-catenin signaling, and inhibited viability and invasiveness of mesothelioma cell lines in culture, and inhibited tumor growth in xenograft models (PMID: 25501304). | 25501304 | |
| Unknown unknown | B-cell lymphoma | not applicable | JSH-150 | Preclinical - Cell culture | Actionable | In a preclinical study, JSH-150 inhibited proliferation of a B-cell lymphoma cell line in culture (PMID: 30253346). | 30253346 | |
| Unknown unknown | Her2-receptor positive breast cancer | not applicable | BMS-906024 | Preclinical | Actionable | In a preclinical study, the pan-Notch inhibitor, BMS-906024, inhibited growth of ERBB2 (HER2)-positive breast cancer xenografts (PMID: 26005526). | 26005526 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Rigosertib Sodium | Phase I | Actionable | In a Phase I study, Rigosertib (ON 01910.Na) demonstrated both safety and preliminary efficacy, resulted in a best overall response of stable disease in 41% (9/22) of patients with advanced solid tumors (PMID: 23841031; NCT01538537). | 23841031 | |
| Unknown unknown | adrenal cortex cancer | not applicable | Linsitinib | Phase I | Actionable | In a Phase I trial, Linsitinib (OSI-906) was well-tolerated and resulted in stable disease in 41% (27/66) of patients with an advanced solid tumor and a partial response in two patients with adrenocortical carcinoma (PMID: 25208878). | 25208878 | |
| Unknown unknown | 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 | 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 | stomach cancer | not applicable | Cisplatin + Deguelin | Preclinical | Actionable | In a preclinical study, the combination of Deguelin and Platinol (cisplatin) worked synergistically to inhibit growth of gastric cancer cells in culture (PMID: 25202376). | 25202376 | |
| Unknown unknown | angiosarcoma | not applicable | Carotuximab + Pazopanib | Phase Ib/II | Actionable | In a Phase I/II trial, TRC105 and Votrient (pazopanib) combination therapy resulted complete response in 40% (2/5) of angiosarcoma patients (J Clin Oncol 34, 2016 (suppl; abstr 11016)). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Tafasitamab-cxix | Phase II | Actionable | In a Phase II trial, diffuse large B-cell lymphoma patients tolerated Monjuvi (tafasitamab-cxix) treatment, and achieved an overall response rate of 26% (9/35, 2 complete and 7 partial response) with 5 patients responding over 12 months, a disease control rate of 40% (14/35), a median duration of response of 20.1 months and a progression-free survival of 2.7 months at a median follow-up of 21 months (PMID: 29444231; NCT01685008). | 29444231 | |
| 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 | glioblastoma multiforme | not applicable | PRN1371 | Preclinical - Pdx | Actionable | In a preclinical study, PRN1371 treatment resulted in tumor regression in PDX models of glioblastoma (Eu J Cancer 2014 Vol 50, Suppl 6:157). | detail... | |
| Unknown unknown | pancreatic cancer | not applicable | Erlotinib + Gemcitabine + Oxaliplatin + siG12D-LODER | Case Reports/Case Series | Actionable | In a Phase I/IIa trial, siG12D-LODER treatment in combination with concurrent chemotherapy, including Gemzar (gemcitabine) and Eloxatin (oxaliplatin) plus Tarceva (erlotinib) (n=1), in patients with pancreatic cancer was well-tolerated and demonstrated safety, and resulted in a median overall survival of 15.12 months, a median time to metastasis of 8.25 months, and partial response in 25% (2/12) and stable disease in 75% (10/12) of patients (PMID: 26009994; NCT01188785). | 26009994 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Carfilzomib + Tinostamustine | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of EDO-S101 and Kyprolis (carfilzomib) decreased viability of primary chronic lymphocytic leukemia cells in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | ovarian cancer | not applicable | ABT-737 + Dactolisib + Trametinib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of BEZ235 and Mekinist (trametinib) enhanced the sensitivity of ovarian cancer cells to ABT-737 in culture, resulting in decreased cell viability (PMID: 27980105). | 27980105 | |
| Unknown unknown | melanoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase III trial (CheckMate 238) that supported FDA approval, adjuvant Opdivo (nivolumab) treatment resulted in improved rate of recurrence-free survival at 12-month compared to Yervoy (ipilimumab) (70.5% vs 60.8%, HR=0.65, P<0.001) in patients with resected stage III or IV melanoma (PMID: 28891423, NCT02388906). | detail... 28891423 | |
| Unknown unknown | melanoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase III trial (CheckMate 037) that supported FDA approval, 31.7% (38/120) of patients with advanced melanoma treated with Opdivo (nivolumab) experienced an objective response whereas only 10.6% (5/47) of advanced melanoma patients treated with investigator's choice of therapy demonstrated an objective response (PMID: 25795410; NCT01721746). | 25795410 detail... | |
| Unknown unknown | melanoma | not applicable | Nivolumab | Phase II | Actionable | In a Phase II trial, Opdivo (nivolumab) monotherapy resulted an overall response rate (ORR) of 25% (3/12) and pathologic complete response rate (pCR) of 25% (3/12) in patients with stage III or IV melanoma, compared to a ORR of 73% (8/11) and pCR of 45% (5/11) with the combination of Opdivo (nivolumab) and Yervoy (ipilimumab), but demonstrated lower toxicity than the combination therapy (PMID: 30297909; NCT02519322). | 30297909 | |
| Unknown unknown | melanoma | not applicable | Nivolumab | Phase III | Actionable | In an analysis of two Phase III trial, Opdivo (nivolumab) treatment after disease progression demonstrated safety and clinical benefit, with 76% (65/85) of patients still alive at time of analysis in melanoma patients who received the last dose of Opdivo (nivolumab) more than 6 weeks after progression (PMID: 28662232). | 28662232 | |
| Unknown unknown | 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 | transitional cell carcinoma | not applicable | Cabozantinib | Phase II | Actionable | In a Phase II trial, Cometriq (cabozantinib) treatment resulted in complete response in 2% (1/41), partial response in 17% (7/41), stable disease in 44% (18/41) of urothelial carcinoma patients, with a median progression-free survival of 3.7 months and median overall survival of 8.2 months (J Clin Oncol 34, 2016 (suppl; abstr 4534)). | detail... | |
| Unknown unknown | pancreatic ductal adenocarcinoma | not applicable | Gemcitabine + JQ1 | Preclinical - Pdx | Actionable | In a preclinical study, combination treatment with JQ1 and Gemzar (gemcitabine) resulted in tumor microenvironment alterations and greater reductions of tumor growth and weight when compared to Gemzar (gemcitabine) alone in patient derived xenograft (PDX) models of pancreatic ductal adenocarcinoma (PMID: 27528027). | 27528027 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Panitumumab | Phase I | Actionable | In a Phase I trial, Vectibix (panitumumab) demonstrated safety and anti-tumor activity in patients with advanced solid tumors (PMID: 18223225). | 18223225 | |
| Unknown unknown | 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 | 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 | head and neck squamous cell carcinoma | not applicable | Cetuximab + Prexasertib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Erlotinib (cetuximab) and Prexasertib (LY2606368 resulted in greater decreased cell proliferation in head and neck squamous cell carcinoma cells in culture compared to either agent alone (PMID: 28138028). | 28138028 | |
| Unknown unknown | cutaneous T cell lymphoma | not applicable | Tenalisib | Phase I | Actionable | In a Phase I trial, Tenalisib (RP6530) demonstrated safety in patients with relapsed or refractory cutaneous T-cell lymphoma, and resulted in an overall response rate of 45% (9/20), including nine partial responses with a median duration of response of 3.8 months, and a further nine patients achieved stable disease (PMID: 32824175; NCT02567656). | 32824175 | |
| 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 | breast cancer | not applicable | Barasertib | Preclinical - Cell culture | Actionable | In a preclinical study, Barasertib (AZD1152) treatment disrupted cell cycle progression and inhibited growth of breast cancer cell lines in culture, with preferential inhibition of aromatase inhibitor-resistant cell lines (PMID: 25667100). | 25667100 | |
| Unknown unknown | follicular lymphoma | not applicable | Tazemetostat | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Tazemetostat (EPZ-6438) treatment was well-tolerated and demonstrated clinical activity in patients with relapsed/refractory follicular lymphoma, resulting in an objective response rate of 77% (33/43) and 34% (18/53), stable disease in 23% (10/43) and 30% (16/53), and median progression-free survival of 11.1 and 5.7 months in EZH2 mutant and wild-type cohorts, respectively (Blood (2019) 134 (Supplement_1):123; NCT01897571). | detail... | |
| Unknown unknown | follicular lymphoma | not applicable | Tazemetostat | Case Reports/Case Series | Actionable | In a Phase I trial, Tazemetostat (EPZ-6438) demonstrated safety and preliminary efficacy, resulted in 2 complete responses and 1 partial response in patients with relapsed or refractory follicular lymphoma (PMID: 29650362; NCT01897571). | 29650362 | |
| Unknown unknown | breast 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 breast cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | Decitabine + Venetoclax | FDA approved | Actionable | In a Phase I/II trial that supported FDA approval, Venclexta (venetoclax) in combination with decitabine or azacitidine resulted in complete remission or complete remission with incomplete count recovery in 65% (40/62) of patients 75 years old or older with treatment-naive acute myeloid leukemia ineligible for intensive chemotherapy, with an overall survival of 11 months (PMID: 30361262; NCT02203773). | detail... 30361262 | |
| Unknown unknown | colon 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 | 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 | lymphoma | not applicable | Ixazomib | Preclinical | Actionable | In a preclinical study, Ixazomib (MLN9708) inhibited survival and induced apoptosis in T-cell lymphoma cell lines in culture, and reduced tumor volume in xenograft models (PMID: 26988986). | 26988986 | |
| Unknown unknown | biliary tract cancer | not applicable | Pembrolizumab | Phase II | Actionable | In a Phase II trial (KEYNOTE-158), Keytruda (pembrolizumab) demonstrated efficacy in patients with advanced biliary tract cancer regardless of CD274 (PD-L1) status, resulted in partial response in 5.8% (6/104) and stable disease in 16% (17/104) of patients; objective response rate, median progression-free survival, and median overall survival were 6.6%, 1.9, and 7.2 months in patients with CPS?1 (n?=?61), and 2.9%, 2.1, and 9.6 months in patients with CPS<1 (n?=?34) (Ann Oncol 29(suppl_8); NCT02628067). | detail... | |
| 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 | follicular lymphoma | not applicable | Mivavotinib | Case Reports/Case Series | Actionable | In a Phase I trial, Mivavotinib (TAK-659) treatment resulted in an objective response rate of 89% (8/9, 2 complete responses, 6 partial responses) in patients with relapsed or refractory follicular lymphoma, with a median duration of response of 137 days (PMID: 32327472; NCT02000934). | 32327472 | |
| Unknown unknown | transitional cell carcinoma | not applicable | Atezolizumab | FDA approved | Actionable | In a Phase II trial that supported FDA approval, treatment with Tecentriq (atezolizumab) in advanced urothelial carcinoma patients resulted in an objective response rate of 15% (45/310) in all patients, and 26% (26/100) in patients with immune cell PD-L1 expression greater than or equal to 5%, and resulted in an overall survival of 8.6 mo in patients receiving treatment post-progression compared to 1.2 mo in patients receiving no treatment post-progression (PMID: 26952546, PMID: 28950298; NCT02108652). | 26952546 28950298 detail... | |
| 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 | breast cancer | not applicable | Lucitanib | Phase II | Actionable | In a Phase II trial, Lucitanib (E-3810) demonstrated acceptable toxicity and activity, resulted in a median progression-free survival of 3.5 and 2.6 months for 10mg and 15mg treatment groups respectively, with no differences in response correlated with FGFR1 and 11q amplification status in metastatic breast cancer patients (Cancer Res 2017;77(4 Suppl):Abstract nr P6-11-03). | detail... | |
| Unknown unknown | 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 | sarcoma | no benefit | Pazopanib + Trametinib | Phase Ib/II | Actionable | In a Phase Ib/II trial, the combination of Votrient (pazopanib) and Mekinist (trametinib) demonstrated safety and resulted in partial response in 8% (2/25) and stable disease in 48% (12/25) of patients with advanced soft tissue sarcomas, but did not improve the 4 month progression-free survival rate over Votrient (pazopanib) alone (PMID: 28377484). | 28377484 | |
| 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 | prostate cancer | not applicable | Enzalutamide + Pembrolizumab | Phase II | Actionable | In a Phase II trial, Keytruda (pembrolizumab) plus Xtandi (enzalutamide) treatment resulted in >=50% reduction in prostate-specific antigen (PSA) levels in 18% (5/28) of metastatic castrate-resistant prostate cancer patients, and led to an objective response rate of 25% (3/12, all partial response), a median PSA progression-free survival of 3.8 months, and a median overall survival of 22.2 mo. in all patients and 41.7 mo. in the three responders (PMID: 32616555; NCT02312557). | 32616555 | |
| 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 | lymphoplasmacytic lymphoma | not applicable | VLX1570 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, VLX1570 induced apoptosis in Waldenstrom macroglobulinemia (WM) cell lines, including Velcade (bortezomib)-resistant cell lines, and primary WM cells in culture, and reduced tumor growth and increased survival in WM cell line xenograft models (PMID: 27813535). | 27813535 | |
| Unknown unknown | neuroblastoma | not applicable | Cisplatin + PHA-680632 | Preclinical - Cell culture | Actionable | In a preclincial study, PHA-680632, in combination with Cisplatin, decreased survival of neuroblastoma cells in culture, to a greater extent than Cisplatin alone (PMID: 27256407). | 27256407 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Cyclophosphamide + Doxorubicin + Elenagen | Phase Ib/II | Actionable | In a Phase Ib/II trial, chemorefractory patients with triple-receptor negative breast cancer demonstrated restored chemotherapeutic sensitivity upon sequential treatment of Elenagen and the combined therapy, Cytoxan (cyclophosphamide) and Adriamycin (doxorubicin), which resulted in stable disease (PMID: 28881846). | 28881846 | |
| Unknown unknown | pancreatic cancer | not applicable | Gemcitabine + Uproleselan | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Uproleselan (GMI-1271) and Gemzar (gemcitabine) combination treatment resulted in reduced tumor metastasis in cell line xenograft models of pancreatic cancer (Cancer Res 2014;74(19 Suppl):Abstract nr 4503). | detail... | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Carboplatin + Fluorouracil + Pembrolizumab | FDA approved | Actionable | In a Phase III trial (KEYNOTE-048) that supported FDA approval, Keytruda (pembrolizumab) in combination with platinum and Adrucil (fluorouracil) significantly improved overall survival compared to Erbitux (cetuximab) plus chemotherapy (13.0 vs 10.7 months, HR=0.77, p=0.0067) in patients with metastatic or unresectable recurrent head and neck squamous cell carcinoma (J Clin Oncol 37, no. 15_suppl (May 20 2019) 6000-6000; NCT02358031). | detail... detail... | |
| Unknown unknown | lymphoma | not applicable | PQR309 + Rituximab | Preclinical - Cell culture | Actionable | In a preclinical study, the combination therapy of PQR309 and Rituxan (rituximab) led to a synergistic effect in 2/5 lymphoma cell lines in culture (PMID: 29066507). | 29066507 | |
| 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 | rhabdomyosarcoma | not applicable | SP-2509 | Preclinical - Cell culture | Actionable | In a preclinical study, SP-2509 treatment inhibited viability of rhabdomyosarcoma cell lines in culture (PMID: 29997151). | 29997151 | |
| 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 | 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 | 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 | lung non-small cell carcinoma | not applicable | BJ-1301 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with BJ-1301 resulted in decreased cell proliferation and inhibited phosphorylation of molecules involved in PI3K/Akt and Ras/Erk signaling in non-small cell lung carcinoma (NSCLC) cells in culture, and repressed tumor growth in NSCLC cell line xenograft models (PMID: 28536313). | 28536313 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Sirolimus | Preclinical | Actionable | In preclinical studies, Rapamune (sirolimus) induced apoptosis of cancer cells and increased sensitivity to Platinol (cisplatin) (PMID: 15136596). | 15136596 | |
| Unknown unknown | multiple myeloma | not applicable | BC2059 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with BC2059 decreased proliferation and induced apoptosis of several human multiple myeloma cell lines and primary multiple myeloma cells with activated canonical Wnt signaling in culture, and delayed tumor growth in a multiple myeloma cell line xenograft model (PMID: 28500235). | 28500235 | |
| Unknown unknown | 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 | chronic myeloid leukemia | not applicable | Bemcentinib | Preclinical - Patient cell culture | Actionable | In a preclinical study, treatment with Bemcentinib (BGB-324) inhibited Axl signaling, and resulted in growth inhibition and apoptosis in BCR-ABL1 positive chronic myeloid leukemia cell lines and patient-derived primary cells in culture (PMID: 27856601). | 27856601 | |
| 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 | 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 | triple-receptor negative breast cancer |