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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 | breast cancer | not applicable | OSI-027 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, OSI-027 inhibited mTORC1 and mTORC2 signaling and growth in breast cancer cells in culture and in cell line xenograft models (PMID: 21673091). | 21673091 | |
| Unknown unknown | prostate cancer | not applicable | AB928 + Oxaliplatin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of AB928 and Eloxatin (oxaliplatin) 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 | lung non-small cell carcinoma | not applicable | Merestinib | Preclinical | Actionable | In a preclinical study, LY2801653 inhibited tumor growth in mouse xenograft models of non-small cell lung cancer (PMID: 24305878). | 24305878 | |
| Unknown unknown | 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 | epithelioid sarcoma | not applicable | Dasatinib | Phase II | Actionable | In a Phase II trial, patients with epithelioid sarcoma demonstrated a median progression free survival of 7.9 months and two patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). | 27696380 | |
| Unknown unknown | 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 | osteosarcoma | not applicable | NKTR-214 | Preclinical | Actionable | In a preclinical study, NKTR-214 treatment inhibited growth of primary tumor, regrowth after amputation, and lung metastasis in disseminated and orthotopic mouse models of osteosarcoma (AACR Annual Meeting 2019, Abstract 3210). | detail... | |
| Unknown unknown | mantle cell lymphoma | not applicable | GS-5829 + Venetoclax | Preclinical - Cell culture | Actionable | In a preclinical study, mantle cell lymphoma cell lines demonstrated greater cell growth inhibition when treated with a combination of GS-5829 and Venclexta (venetoclax) compared to either agent alone in culture (Blood 2016 128:5104). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Paclitaxel + TAS-119 | Preclinical | Actionable | In a preclinical study, TAS-119 demonstrated synergistic effects with Taxol (paclitaxel) or Taxotere (docetaxel) in multiple tumor cell lines by promoting apoptosis (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr A268). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | Dexamethasone + Elotuzumab + Lenalidomide | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with the combination of Empliciti (elotuzumab) with Revlimid (lenalidomide) and dexamethosone resulted in an overall response rate of 79%, compared to 66% with Revlimid (lenalidomide) and dexamethosone combination therapy in patients with multiple myeloma (PMID: 27493709). | detail... 27493709 | |
| 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 | colon cancer | not applicable | Enzastaurin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Enzastaurin (LY317615) demonstrated efficacy by suppressing proliferation of cultured cells and growth in cell line xenograft models of colon cancer (PMID: 16103100). | 16103100 | |
| Unknown unknown | 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 | 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 | colorectal cancer | not applicable | MGCD516 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MGCD516 decreased tumor cell proliferation, tumor vascularization, and target phosphorylation in human colorectal carcinoma cell line xenograft models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1, Abstract 930). | detail... | |
| Unknown unknown | 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 | colorectal cancer | not applicable | Dbait + Fluorouracil + Oxaliplatin | Preclinical | Actionable | In a preclinical study, Dbait with Eloxitan (oxaliplatin) and Adrucil (5-fluorouracil) resulted in decreased proliferation of human colorectal cancer cell lines in culture (PMID: 26637369). | 26637369 | |
| Unknown unknown | lung carcinoma | not applicable | YW3-56 | Preclinical | Actionable | In a preclinical study, YW3-56 inhibited proliferation of lung carcinoma cell lines in culture, independent of TP53 status (PMID: 25612620). | 25612620 | |
| Unknown unknown | melanoma | not applicable | A-1210477 + G-TPP | Preclinical - Cell culture | Actionable | In a preclinical study, combination of the mitochondrial Hsp90 inhibitor G-TPP and the Mcl-1 inhibitor A-1210477 resulted in increased apoptosis of melanoma cell lines in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | 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 | Advanced Solid Tumor | not applicable | Crizotinib + Dasatinib | Phase I | Actionable | In a Phase I trial, Xalkori (crizotinib) therapy, in combination with Sprycel (dasatinib), in patients with advanced solid tumors resulted in limited efficacy, including one patient with a partial response and three patients with stable disease for at least six months or more (PMID: 29047029). | 29047029 | |
| Unknown unknown | acute myeloid leukemia | not applicable | GS87 | Preclinical | Actionable | In a preclinical study, treatment with GS87 induced terminal differentiation and inhibited growth of acute myeloid leukemia (AML) cell lines in culture and resulted in increased survival and decreased disease burden in mouse models of AML (PMID: 27196775). | 27196775 | |
| Unknown unknown | gastroesophageal junction adenocarcinoma | not applicable | Ramucirumab | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Cyramza (ramucirumab) increased progression-free and overall survival in patients with gastroesophageal junction adenocarcinoma (PMID: 24094768). | 24094768 detail... | |
| 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 | 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 | Advanced Solid Tumor | not applicable | BAY1125976 | Phase I | Actionable | In a Phase I trial, BAY 1125976 treatment demonstrated safety and pharmacological inhibition of Akt signaling, but only resulted in partial response in 1% (1/78) and stable disease in 38% (30/78) of patients with advanced solid tumors, clinical benefit rate at recommended Phase II dose was 27.9% (12/43) (PMID: 31835495; NCT01915576). | 31835495 | |
| 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 | dermatofibrosarcoma protuberans | not applicable | Imatinib | FDA approved | Actionable | In a Phase II clinical trial that supported FDA approval, treatment with Gleevec (imatinib) resulted in a median time-to-progression of 23.9 months, and complete response in 33% (4/12) and partial response in 50% (6/12) of patients with dermatofibrosarcoma protuberans (PMID: 18451237). | 18451237 detail... | |
| Unknown unknown | peritoneal carcinoma | no benefit | Motolimod + Pegylated liposomal-doxorubicin | Phase II | Actionable | In a Phase II trial, the inclusion of Motolimod (VTX-2337) with Doxil (pegylated liposomal-doxorubicin) did not result in improved survival in patients with either ovarian, fallopian tube, or primary peritoneal carcinoma (PMID: 28453702; NCT01666444). | 28453702 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | OBP-801 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, OBP-201 (YM753) inhibited growth of several solid tumor cell lines in culture and demonstrated anti-tumor activity in a colon cancer cell line xenograft model (PMID: 18292931). | 18292931 | |
| Unknown unknown | 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 | triple-receptor negative breast cancer | not applicable | Capivasertib + Olaparib | Phase I | Actionable | In a Phase I trial, the combination of AZD5363 and Lynparza (olaparib) was well-tolerated and demonstrated preliminary activity in patients with endometrial, ovarian, or triple-negative breast cancer (TNBC), with an overall response rate of 24% (7/30; all partial responses, 1 ovarian, 4 endometrial, and 2 TNBC) and stable disease for greater than 4 months in 6 additional patients (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #391P; NCT02208375). | detail... | |
| Unknown unknown | leukemia | not applicable | MC180295 | Preclinical - Cell culture | Actionable | In a preclinical study, MC180295 decreased proliferation and increased differentiation of leukemia cells in culture (PMID: 30454645). | 30454645 | |
| Unknown unknown | marginal zone B-cell lymphoma | not applicable | Umbralisib | Phase I | Actionable | In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in partial response in 20% (1/5) and stable disease in 80% (4/5) of patients with marginal zone B-cell lymphoma (PMID: 29475723; NCT01767766). | 29475723 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | SST0116CL1 | Preclinical - Cell culture | Actionable | In a preclinical study, SST0116CL1 inhibited proliferation of several human tumor cell lines in culture (PMID: 25096516). | 25096516 | |
| Unknown unknown | acute lymphoblastic leukemia | not applicable | Torin 2 | Preclinical - Cell culture | Actionable | In a preclinical study, Torin 2 treatment inhibited viability of T-cell acute lymphoblastic leukemia cell lines harboring NUP214-ABL1 fusion in culture (PMID: 27821800). | 27821800 | |
| 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 | thymoma | not applicable | Resminostat | Phase I | Actionable | In a Phase I trial, Resminostat (4SC-201) treatment resulted in stable disease in 58% (11/19) of heavily pretreated patients with advanced solid tumors, and a 27% reduction of tumor in a thymoma patient (PMID: 24065624). | 24065624 | |
| Unknown unknown | acute myeloid leukemia | predicted - sensitive | Daunorubicin + Venetoclax | Preclinical - Patient cell culture | Actionable | In a preclinical study, Venclexta (venetoclax) and Daunorubicin combination treatment synergistically induced cell death in patient-derived acute myeloid leukemia cells in culture (PMID: 27103402). | 27103402 | |
| Unknown unknown | non-Hodgkin lymphoma | not applicable | BCL201 | Phase I | Actionable | In a Phase I trial, BCL201 (S55746) demonstrated safety and preliminary activity in patients with relapsed or recurrent non-Hodgkin lymphoma (Hematol Oncol. 2017;35(S2):47-48; NCT02920697). | detail... | |
| Unknown unknown | neuroendocrine tumor | not applicable | Surufatinib | Phase Ib/II | Actionable | In a Phase Ib/II trial, Surufatinib (HMPL-012) treatment resulted in an objective response rate of 19% (8/42) and 15% (6/39), a disease control rate of 91% (38/42) and 92% (36/39), and a median progression-free survival of 21.2 and 13.4 months in patients with advanced, well-differentiated pancreatic and extrapancreatic neuroendocrine tumors, respectively (PMID: 30833272; NCT02267967). | 30833272 | |
| Unknown unknown | neuroendocrine tumor | not applicable | Surufatinib | Phase I | Actionable | In a Phase I trial, Surufatinib (HMPL-012) demonstrated an objective response rate in 44% (8/18) of patients with neuroendocrine tumors (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A1). | detail... | |
| Unknown unknown | chronic leukemia | not applicable | RG7112 | Phase I | Actionable | In a Phase I clinical trial, 5% (1/19) of chronic leukemia patients achieved partial response, and 79% (15/19) achieved stable disease following treatment with RG7112 (PMID: 26459177). | 26459177 | |
| Unknown unknown | colorectal cancer | not applicable | Fluorouracil + Trametinib | Preclinical | Actionable | In preclinical studies, Mekinist (trametinib) enhanced the efficacy of Adrucil (fluorouracil) in colorectal cancer cells in culture (PMID: 23438367). | 23438367 | |
| Unknown unknown | colorectal cancer | not applicable | Regorafenib | Phase III | Actionable | In a Phase III trial (CONSIGN), Stivarga (regorafenib) treatment demonstrated safety profile and efficacy consistent with previous studies, median progression-free survival (PFS) was 2.7 months overall, 2.8 months in KRAS wild-type, and 2.5 months in KRAS mutant colorectal cancer patients, and with no difference in KRAS status between long and short PFS groups (PMID: 30190299; NCT01538680). | 30190299 | |
| Unknown unknown | colorectal cancer | not applicable | Regorafenib | FDA approved | Actionable | In a Phase III clinical trial (CORRECT) that supported FDA approval, Stivarga (regorafenib) demonstrated safety and improved overall survival compared to placebo (6.4 vs 5.0 months, HR=0.77, p=0.0052) in patients with refractory metastatic colorectal cancer (PMID: 23177514; NCT01103323). | 23177514 detail... | |
| Unknown unknown | myelofibrosis | not applicable | Ruxolitinib | FDA approved | Actionable | In a Phase III clinical trial that supported FDA approval, treatment with Jakafi (ruxolitinib) resulted in a decrease in spleen volume of greater than or equal to 35% in 41.9% of patients with intermediate or high-risk myelofibrosis, compared to 0.7% of patients treated with the placebo (PMID: 22474318). | detail... 22474318 | |
| Unknown unknown | pancreatic cancer | not applicable | G-TPP + Navitoclax | Preclinical - Cell culture | Actionable | In a preclinical study, combination of the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Navitoclax (ABT-263) resulted in enhanced growth inhibition of pancreatic cancer cells in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | malignant pleural mesothelioma | not applicable | Trabectedin + Venetoclax | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Yondelis (trabectedin) and Venclexta (venetoclax) demonstrated synergistic or additive effects on decreasing viability of malignant pleural mesothelioma cell lines in culture (PMID: 27512118). | 27512118 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BAY1217389 | Preclinical | Actionable | In a preclinical study, BAY1217389 inhibited proliferation of a variety of human solid tumor cell lines in culture (PMID: 26832791). | detail... 26832791 | |
| 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 | lung carcinoma | not applicable | AGI-134 | Preclinical - Cell culture | Actionable | In a preclinical study, AGI-134 treatment induced killing of lung carcinoma cells in the presence of normal human serum in culture and induced phagocytosis when co-cultured with human macrophages (PMID: 31889898). | 31889898 | |
| Unknown unknown | tenosynovial giant cell tumor | not applicable | Pexidartinib | FDA approved | Actionable | In a Phase III trial (ENLIVEN) that supported FDA approval, Turalio (pexidartinib) treatment resulted in improved overall response rate at week 25 (24/61, 39% vs 0/59, 0%, p<0.0001) compared to placebo in patients with advanced tenosynovial giant cell tumour (PMID: 31229240; NCT02371369). | detail... 31229240 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Selumetinib + SHP099 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination therapy of SHP099 and Selumetinib (AZD6244) led to decreased cell viability and reduced colony formation in triple-receptor negative breast cancer cells in culture and tumor regression in xenograft models (PMID: 30045908). | 30045908 | |
| Unknown unknown | follicular lymphoma | not applicable | Copanlisib | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Aliqopa (copanlisib) treatment in patients with follicular lymphoma resulted in an objective tumor response rate of 58.7% (61/104) including 14.4% (15/61) patients experiencing a complete response and 44.2% (46/61) patients experiencing a partial response, stable disease in 33.7% (35/104) of patients, and a duration of response of 370 days (Journal of Clinical Oncology 35, no. 15_suppl (May 2017) 7535-7535; NCT01660451). | detail... detail... | |
| Unknown unknown | follicular lymphoma | not applicable | Copanlisib | Phase II | Actionable | In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 20% (2/10), partial response in 20% (2/10) and stable disease in 60% (6/10) of patients with follicular lymphoma (PMID: 24852792). | 24852792 | |
| Unknown unknown | 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 | 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 | acute leukemia | not applicable | CNDO-109 | Phase I | Actionable | In a Phase I trial, acute leukemia patients treated with CNDO-109 demonstrated a relapse-free survival (RFS) rate of 33% at 12 months, with three patients demonstrating a long-term RFS of at least 32.6 months in a follow-up (PMID: 29597002; NCT01520558). | 29597002 | |
| Unknown unknown | 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 | pancreatic ductal adenocarcinoma | not applicable | Gemcitabine + Masitinib | Phase I | Actionable | In a Phase I trial, the median OS did not differ (7.7 mo vs 7.1 mo) between pancreatic ductal adenocarcinoma patients treated with the combination of Gemzar (gemcitabine) plus Masitinib (AB1010) versus those treated with Gemzar (gemcitabine) plus placebo (PMID: 25858497). | 25858497 | |
| Unknown unknown | hematologic cancer | not applicable | ID09C3 | Phase I | Actionable | In a Phase I trial, treatment with ID09C3 in patients with B cell type leukemia/lymphoma resulted in decreased peripheral blood B cells and monocytes (PMID: 23090290). | 23090290 | |
| Unknown unknown | glioblastoma multiforme | not applicable | SR9009 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, SR9009 inhibited growth of glioblastoma cell lines in culture, resulted in apoptosis in tumors and prolonged survival in both cell line and patient-derived xenograft models (PMID: 29320480). | 29320480 | |
| Unknown unknown | 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 | granulosa cell tumor | no benefit | STM434 | Phase I | Actionable | In a Phase I trial, STM 434 treatment did not result in response in 30 evaluable patients with advanced solid tumors and only resulted in stable disease as best response in 80% (10/12) of patients with granulosa cell ovarian cancer (PMID: 31068369; NCT02262455). | 31068369 | |
| Unknown unknown | ovarian cancer | not applicable | DNIB0600A | Phase I | Actionable | In a Phase I trial, DNIB0600A (lifastuzumab vedotin) treatment demonstrated a safe profile and resulted in efficacy in patients with platinum-resistant ovarian cancer receiving a dose of 1.8-2.8 mg/kg, which included a partial response in 46% (11/24) of patients regardless of Slc34a2 expression and a partial response in 50% (11/22) of patients with over expression of Slc34a2 (PMID: 31540980; NCT01363947). | 31540980 | |
| Unknown unknown | fibrosarcoma | not applicable | Tivozanib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with Tivozanib (AV-951) resulted in partial inhibition of tumor growth and decreased tumor vasulature in a fibrosarcoma cell line xenograft model (PMID: 25995436). | 25995436 | |
| Unknown unknown | ovarian cancer | not applicable | ABT-737 + AZD8055 + Trametinib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of AZD8055 and Mekinist (trametinib) enhanced the sensitivity of ovarian cancer cells to ABT-737 in culture, resulting in greater apoptotic activity and cell cycle arrest when compared to Mekinist (trametinib) alone (PMID: 27980105). | 27980105 | |
| Unknown unknown | Ewing sarcoma | not applicable | Olaparib + SN-38 | Preclinical - Cell culture | Actionable | In a preclinical study, Ewing sarcoma cells treated with SN-38 combined with Lynparza (olaparib) resulted in synergism, demonstrating reduced cell viability in culture (PMID: 26438158). | 26438158 | |
| Unknown unknown | esophageal cancer | not applicable | DKN-01 + Paclitaxel | Phase I | Actionable | In a Phase I trial, DKN-01 and Taxol (paclitaxel) combination treatment resulted in partial response in 18% (6/34) and stable disease in 32% (11/34) of advanced esophagogastric cancer patients, with a median progression-free survival of 13.7 weeks and an overall survival of 28.4 weeks (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 91P; NCT02013154). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | EBI-2511 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, EBI-2511 treatment in diffuse large B-cell lymphoma cell line xenograft models resulted in tumor growth inhibition and a 97% decrease in tumor size (PMID: 29456795). | 29456795 | |
| Unknown unknown | 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 | colorectal cancer | no benefit | Atezolizumab | Phase III | Actionable | In a Phase III trial (IMblaze370), Tecentriq (atezolizumab) treatment did not improve median overall survival (7.1 vs 8.5 months, HR=1.19) compared to Stivarga (regorafenib) in patients with chemotherapy-refractory metastatic colorectal cancer, 91.7% of whom were microsatellite stable or microsatellite instability-low (Annals of Oncology, Volume 29, Issue suppl_5, 1 June 2018; NCT02788279). | detail... | |
| Unknown unknown | 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 | not applicable | Ipatasertib + Paclitaxel | Phase II | Actionable | In a Phase II trial, Ipatasertib (GDC-0068) in combination with Abraxane (paclitaxel) resulted in improved progression free survival (6.2 vs 4.9 months) compared to placebo in patients with triple-receptor negative breast cancer (PMID: 28800861; NCT02162719). | 28800861 detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Metformin + Temsirolimus | Phase I | Actionable | In a Phase I clinical trial, the combination of Torisel (temsirolimus) and Glucophage (metformin) demonstrated safety and resulted in a clinical benefit rate of 22% in patients with advanced or refractory tumors, with 4/18 patients achieving stable disease for greater than 6 cycles (PMID: 27014780). | 27014780 | |
| Unknown unknown | melanoma | not applicable | Ipilimumab + Radiotherapy | Clinical Study | Actionable | In a retrospective study, Yervoy (ipilimumab) in combination with local tumor treatment consisting of radiotherapy or electrochemotherapy resulted in improved median overall survival (93 weeks) compared to Yervoy (ipilimumab) alone (42 weeks) in advanced melanoma patients (PMID: 27466265). | 27466265 | |
| 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 | 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 | 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 | 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 | chronic lymphocytic leukemia | not applicable | ONO-4059 | Phase I | Actionable | In a Phase I clinical trial, treatment with ONO-4059 was well tolerated and resulted in objective responses in lymph nodes in 96% (24/25) of patients with chronic lymphocytic leukemia (PMID: 26542378). | 26542378 | |
| Unknown unknown | Hodgkin's lymphoma | not applicable | AZD7762 + Ixazomib | Preclinical | Actionable | In a preclinical study, Ixazomib (MLN9708) and AZD7762 combination treatment resulted in increased apoptosis in Hodgkin's lymphoma cells in culture (PMID: 26988986). | 26988986 | |
| Unknown unknown | prostate cancer | no benefit | Zibotentan | Phase III | Actionable | In a Phase III trial, Zibotentan (ZD4054) did not demonstrate a survival benefit compared to placebo in patients with prostate cancer, and the trial was discontinued at the interim analysis (PMID: 23381694). | 23381694 | |
| Unknown unknown | 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 | 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 | 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 | hematologic cancer | not applicable | Glasdegib | Phase I | Actionable | In a Phase I trial, Glasdegib (PF-04449913) treatment in patients with hematological malignancies resulted in some preliminary efficacy, including a complete response in one patient with acute myeloid leukemia (AML) and stable disease in four patients with AML (PMID: 28556364). | 28556364 | |
| Unknown unknown | stomach cancer | not applicable | PU-H71 | Preclinical - Cell culture | Actionable | In a preclinical study, sensitivity to PU-H71 was correlated to presence of the epichaperome, a network of chaperome complexes, in gastric cancer cell lines in culture (PMID: 27706135). | 27706135 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | AXL1717 | Phase Ib/II | Actionable | In a Phase 1b/II trial, AXL1717 (picropodophyllin) was well tolerated and 25% (4/16) of patients diagnosed with NSCLC demonstrated a partial tumor response (PMID: 26161618). | 26161618 | |
| Unknown unknown | triple-receptor negative breast cancer | no benefit | Cisplatin + Everolimus + Paclitaxel | Phase II | Actionable | In a Phase II trial, the addition of Afinitor (everolimus) to the combined treatment of Platinol (cisplatin) and Taxol (paclitaxel) in patients with triple-receptor negative breast cancer did not result in improved clinical response when compared to Platinol (cisplatin), Taxol (paclitaxel), and placebo, and resulted in greater adverse events (PMID: 28270498). | 28270498 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | S-49076 | Phase I | Actionable | In a Phase I trial, S-49076 demonstrated safety and limited preliminary clinical activity in patients with advanced solid tumors, resulting in stable disease as best response in 50% of patients, with 23% demonstrating stable disease for at least 3 months, and 9 patients demonstrating stable disease for at least 6 months (PMID: 28624695). | 28624695 | |
| 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 | nasopharynx carcinoma | not applicable | WNT-C59 | Preclinical | Actionable | In a preclinical study, nasopharynx carcinoma cells treated with WNT-C59 in culture and mouse models demonstrated inhibition of the Wnt signaling pathway and suppression of tumor growth (PMID: 25980501). | 25980501 | |
| 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 | Advanced Solid Tumor | not applicable | A-1331852 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, A-1331852 inhibited tumor growth in several solid tumor cell line xenograft models (PMID: 25787766). | 25787766 | |
| Unknown unknown | myelodysplastic/myeloproliferative neoplasm | not applicable | Ruxolitinib | Phase I | Actionable | In a Phase I trial, Jakafi (ruxolitinib) treatment demonstrated safety and preliminary efficacy in chronic myelomonocytic leukemia patients, resulted in a total response rate of 35% (7/20) and reduction of Stat5 signaling (PMID: 26858309). | 26858309 | |
| Unknown unknown | multiple myeloma | not applicable | KDM5-C70 | Preclinical - Cell culture | Actionable | In a preclinical study, KDM5-C70 inhibited cell proliferation of a multiple myeloma cell line in culture (PMID: 27214403). | 27214403 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | MCLA-128 | Phase Ib/II | Actionable | In a Phase I/II trial, MCLA-218 resulted in partial response for more than 10 months in a patient with non-small cell lung cancer (Cancer Res 2016;76(14 Suppl):Abstract nr CT050). | detail... | |
| Unknown unknown | prostate cancer | not applicable | UC-773587 | Preclinical | Actionable | In a preclinical study, UC-773587 inhibited ERK activation and growth of prostate cells in culture (PMID: 25825487). | 25825487 | |
| Unknown unknown | breast cancer | not applicable | Carboplatin + Veliparib | Phase I | Actionable | In a Phase I clinical trial, the combination of Veliparib (ABT-888) and Paraplatin (carboplatin) demonstrated preliminary efficacy in patients with metastatic breast cancer, with 18.6 % (9/43) achieving partial response and 48.8% (21/43) achieving stable disease (J Clin Oncol. 2014;32(suppl):abstr 1074). | detail... | |
| 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 | Docetaxel + Nintedanib | Phase III | Actionable | In a Phase III clinical trial, the combination of Ofev (nintedanib) and Taxotere (docetaxel) resulted in improved progression-free survival and overall survival compared to placebo plus Taxotere (docetaxel) in non-small cell lung cancer patients (PMID: 24411639). | 24411639 | |
| Unknown unknown | melanoma | not applicable | Alisertib | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, Alisertib (MLN8237) impaired mitosis, induced senescence and blocked growth in xenograft models of patient-derived and cell line-derived melanoma tumors (PMID: 23180582). | 23180582 | |
| Unknown unknown | hairy cell leukemia | not applicable | Ibrutinib | Preclinical - Cell culture | Actionable | In a preclinical study, Imbruvica (ibrutinib) inhibited Btk activity and B-cell receptor signaling, thereby resulting in inhibition of cell proliferation and decreased survival of human hairy cell leukemia cells in culture (PMID: 24697238). | 24697238 | |
| Unknown unknown | 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 | breast cancer | not applicable | JNJ-7706621 | Preclinical - Cell culture | Actionable | In a preclinical study, JNJ-7706621 disrupted cell cycle progression and inhibited growth of breast cancer cell lines in culture, with preferential inhibition of aromatase inhibitor-resistant cell lines (PMID: 25667100). | 25667100 | |
| Unknown unknown | colon cancer | not applicable | Irinotecan + Veliparib | Phase I | Actionable | In a Phase I trial, Veliparib (ABT-888) and Camptosar (irinotecan) combination therapy resulted in partial response in 50% (2/4) of patients with colon cancer (PMID: 26842236). | 26842236 | |
| Unknown unknown | colorectal cancer | not applicable | Bevacizumab + Fluorouracil + Leucovorin + Oxaliplatin | Clinical Study | Actionable | In a clinical study, colorectal cancer patients demonstrated a greater median PFS when treated with FOLFOX and Avastin (bevacizumab), which was thought to be associated with a decrease in granulocytic myeloid-derived suppressor cells highly expressing PD-L1 (PMID: 27496709). | 27496709 | |
| Unknown unknown | prostate cancer | no benefit | Abiraterone + Buparlisib | Phase I | Actionable | In a Phase Ib trial, the combination of Zytiga (abiraterone) and Buparlisib (BKM120) did not demonstrate significant clinical activity in patients with castration-resistant prostate cancer, resulting in a best overall response of stable disease in 15% (3/20) patients treated at the 100 mg QD Buparlisib (BKM120) dose level, and further study of this combination is not planned (PMID: 28282611; NCT01634061). | 28282611 | |
| Unknown unknown | gastroesophageal junction adenocarcinoma | not applicable | Paclitaxel + Ramucirumab | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Cyramza (ramucirumab) in combination with Taxol (paclitaxel) increased progression-free and overall survival and improved quality of life compared to treatment with Taxol (paclitaxel) alone in patients with gastroesophageal junction adenocarcinoma (PMID: 24706672). | 24706672 detail... | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Entospletinib + Idelalisib | Phase II | Actionable | In a Phase II trial, Zydelig (idelalisib) and Entospletinib combination treatment resulted in objective response in 60% of patients with chronic lymphocytic leukemia, but was terminated due to severe treatment-emergent pneumonitis (PMID: 26968534). | 26968534 | |
| Unknown unknown | 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 | Advanced Solid Tumor | not applicable | Axitinib + Bevacizumab | Phase I | Actionable | In a Phase I trial, Inlyta (axitinib) in combination with Avastin (bevacizumab) demonstrated safety and efficacy in patients with advanced solid tumors including metastatic colorectal cancer (PMID: 19940012). | 19940012 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | IPI-549 | Preclinical | Actionable | In a preclinical study, IPI-549 inhibited tumor growth in multiple xenograft models of solid tumors (Mol Cancer Ther December 2015 14; A192). | detail... | |
| Unknown unknown | glioblastoma multiforme | not applicable | Bevacizumab + SL-701 | Phase II | Actionable | In a Phase II trial, SL-701 and Avastin (bevacizumab) combination treatment resulted in complete response in 7.1% (2/28), partial response in 14.3% (4/28) and stable disease in 67.9.6% (19/28) of patients with relapsed/refractory glioblastoma, with a 12-month overall survival rate of 43% (median 11.7 months) (J Clin Oncol 36, 2018 (suppl; abstr 2058); NCT02078648). | detail... | |
| Unknown unknown | glioblastoma multiforme | not applicable | Bevacizumab + SL-701 | Phase II | Actionable | In a Phase II trial, SL-701 and Avastin (bevacizumab) combination treatment resulted in partial response in 25% (1/4) and stable disease in 75% (3/4) of patients with recurrant glioblastoma multiforme (Neuro Oncol (2016) 18 (suppl 6): vi20.). | detail... | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | BAY1161909 + Paclitaxel | Preclinical | Actionable | In a preclinical study, BAY1161909, in combination with Taxol (paclitaxel), had increased efficacy in xenograft models of triple-negative breast cancer compared to Taxol (paclitaxel) alone, resulting in complete tumor regression (PMID: 26832791). | 26832791 | |
| Unknown unknown | colorectal cancer | no benefit | ABT-751 + Bevacizumab + Capecitabine + Irinotecan | Phase I | Actionable | In a Phase I clinical trial, the combination of ABT-751, Avastin (bevacizumab), Camptosar (irinotecan), and Xeloda (capecitabine) had modest efficacy, coupled with multiple dose limiting toxicities in colorectal cancer and will not be explored further (PMID: 26632033). | 26632033 | |
| Unknown unknown | adenoid cystic carcinoma | not applicable | Dovitinib | Phase II | Actionable | In a Phase II trial, Dovitinib (TKI258) treatment was tolerated and demonstrated limited clinical activity in patients with adenoid cystic carcinoma, resulting in partial response in 5.9% (2/34) of patients, suppression of overall tumor growth rate, and a median progression-free survival of 8.2 months (PMID: 28377480). | 28377480 | |
| Unknown unknown | basal cell carcinoma | not applicable | Taladegib | Phase I | Actionable | In a Phase I trial, Taladegib treatment resulted in a clinical response in 46.8% (22/47) of patients with basal cell carcinoma, including 16 patients with a confirmed partial response, 1 patient with an unconfirmed partial response, and 5 patients with a confirmed complete response (PMID: 29483143). | 29483143 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Nivolumab | Guideline | Actionable | Opdivo (nivolumab) is included in guidelines for small cell lung cancer patients who have relapsed after primary therapy (NCCN.org). | detail... | |
| Unknown unknown | lung small cell carcinoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase I/II trial (CheckMate 032) that supported FDA approval, Opdivo (nivolumab) as third- or later-line treatment resulted in an objective response rate of 11.9% (13/109, 1 complete response, 12 partial response) in patients with metastatic small cell lung cancer, with a median duration of response of 17.9 months (PMID: 29731394, PMID: 30316010; NCT01928394). | 29731394 30316010 detail... | |
| 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 | glioblastoma multiforme | not applicable | Dovitinib | Phase I | Actionable | In a Phase I trial, Dovitinib (TKI258) treatment resulted in a progression free survival rate at 6 months of 16.7% (2/12) in patients with recurrent glioblastoma (PMID: 27100354). | 27100354 | |
| Unknown unknown | melanoma | not applicable | AS1409 | Phase I | Actionable | In a Phase I trial, AS1409 treatment in patients with either melanoma or renal cell carcinoma resulted in stable disease in 46% (6/13) of patients and in two patients with melanoma, one demonstrated a partial response while another showed tumor shrinkage, which lasted beyond 12 months (PMID: 21447719). | 21447719 | |
| Unknown unknown | 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 | diffuse large B-cell lymphoma | not applicable | Cerdulatinib | Preclinical | Actionable | In a preclinical study, treatment with Cerdulatinib (PRT062070) resulted in decreased viability and increased apoptosis of diffuse large B-cell lymphoma cells in culture (PMID: 25253883). | 25253883 | |
| Unknown unknown | 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 | prostate cancer | not applicable | Birabresib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Birabresib (OTX015) treatment inhibited tumor growth in a cell line xenograft model of prostate cancer (PMID: 27274052). | 27274052 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Bortezomib + Mivebresib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Velcade (bortezomib) and Mivebresib (ABBV-075) resulted in increased tumor growth inhibition in an acute myeloid leukemia cell line xenograft model compared to Velcade (bortezomib) alone (PMID: 28416490). | 28416490 | |
| 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 | neuroendocrine carcinoma | sensitive | 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 | colon adenocarcinoma | not applicable | AGI-134 | Preclinical - Cell culture | Actionable | In a preclinical study, AGI-134 treatment induced killing of colon adenocarcinoma cells in the presence of normal human serum in culture (PMID: 31889898). | 31889898 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Orlistat | Preclinical - Cell culture | Actionable | In a preclinical study, Xenical (orlistat) inhibited lipid metabolism and induced cell death in hepatocellular carcinoma cells in culture (PMID: 30667213). | 30667213 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MEDI3617 | Phase I | Actionable | In a Phase I trial, MEDI3617 monotherapy resulted in no objective response (0/25) and stable disease in 52% (13/25) of patients with advanced solid tumors, with a median progression-free survival of 1.4 months (PMID: 29559563; NCT01248949). | 29559563 | |
| Unknown unknown | 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 | 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 | renal cell carcinoma | not applicable | Itraconazole | Preclinical - Cell culture | Actionable | In a preclinical study, Itraconazole resulted in antiangiogenic activity and inhibited cell proliferation of renal carcinoma cells in culture (PMID: 22025615). | 22025615 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Parsaclisib | Preclinical | Actionable | In a preclinical study, Parsaclisib (INCB050465) demonstrated immunomodulatory and anti-tumor activity in mouse tumor models with intact immune systems (Mol Cancer Ther December 1 2015 (14) (12 Supplement 2) C103). | detail... | |
| Unknown unknown | pancreatic endocrine carcinoma | not applicable | Sunitinib | FDA approved | Actionable | In a Phase III clinical trial that supported FDA approval, Sutent (sunitinib) demonstrated safety and improved progression free survival in patients with pancreatic neuroendocrine tumors (PMID: 21306237). | 21306237 detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | JCAR017 | Clinical Study | Actionable | In a clinical case study, treatment with JCAR017 resulted in complete remission and durable central nervous system response in a patient with diffuse large B-cell lymphoma (PMID: 28834486). | 28834486 | |
| Unknown unknown | lymphoid leukemia | not applicable | Ponatinib | FDA approved | Actionable | In a Phase II clinical trial which supported FDA approval, Iclusig (ponatinib) was effective in promoting disease regression in 52% of patients with accelerated phase chronic myeloid leukemia, 31% of patients with blast phase chronic myeloid leukemia, and 41% of patients with Philadelphia chromosome positive acute lymphoblastic leukemia (PMID: 23935038). | 23935038 detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | OPB-31121 | Phase I | Actionable | In a Phase I trial, OPB-31121 treatment resulted in stable diseases in 44% (8/18) of patients with advanced solid tumors, and tumor shrinkage in 2 patients (1 colon cancer, 1 rectal cancer) (PMID: 25715763). | 25715763 | |
| Unknown unknown | 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 | ovarian cancer | not applicable | JNJ-54302833 | Preclinical | Actionable | In a preclinical study, JNJ-54302833 inhibited growth of ovarian cancer cells in culture ( Cancer Res October 1, 2014 74; 4747 ). | detail... | |
| Unknown unknown | stomach cancer | not applicable | CUDC-101 | Phase I | Actionable | In a Phase I trial, a gastric cancer patient treated with CUDC-101 demonstrated a partial response for 57 days, in which the tumor regressed by 56% (PMID: 25107918). | 25107918 | |
| Unknown unknown | Burkitt lymphoma | not applicable | RVX2135 + VE-821 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of RVX2135 and VE-821 resulted in decreased cell viability in Burkitt lymphoma cell lines in culture (PMID: 26804177). | 26804177 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | XL999 | Phase II | Actionable | In six Phase II clinical trials, XL999 demonstrated anti-tumor activity in patients with advanced solid tumors, but also resulted in significant cardiotoxicity, which improved after discontinuation of the drug (J Clin Oncol, 25:18s, 2007 (Suppl; abstr 3591)). | detail... | |
| Unknown unknown | lung cancer | not applicable | AZD7762 + VX-970 | Preclinical | Actionable | In a preclinical study, lung cancer cells treated with VX-970 resulted in a synthetic lethal effect when combined with AZD7762, demonstrating increased survival and decreased tumor volume in xenograft models (PMID: 26748709). | 26748709 | |
| Unknown unknown | follicular lymphoma | not applicable | Abexinostat | Phase II | Actionable | In a Phase II clinical trial, Abexinostat treatment reduced tumor size in 86% (12/14) of follicular lymphoma patients with an observed response rate of 64.3% (9/14) and median progression-free survival of 20.5 months (PMID: 26482040). | 26482040 | |
| Unknown unknown | cervical cancer | not applicable | Tisotumab Vedotin | Phase II | Actionable | In a Phase II trial, Tisotumab vedotin treatment resulted in partial response in a patient with cervical cancer (Journal of Clinical Oncology 33, no. 15_suppl (May 20 2015) 2570-2570; NCT02001623). | detail... | |
| Unknown unknown | epithelioid sarcoma | not applicable | Vorinostat | Preclinical | Actionable | In a preclinical study, Zolinza (vorinostat) inhibited growth and colony formation of epithelioid sarcoma cells in culture (PMID: 26396249). | 26396249 | |
| Unknown unknown | pancreatic endocrine carcinoma | not applicable | Cabozantinib | Preclinical | Actionable | In a preclinical study, Cometriq (cabozantinib) inhibited pancreatic neuroendocrine tumor growth and invasion in transgenic mouse models (PMID: 22585997). | 22585997 | |
| Unknown unknown | 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 | estrogen-receptor positive breast cancer | not applicable | Everolimus + Tamoxifen | Preclinical - Cell culture | Actionable | In a preclinical study, the combination treatment of Afinitor (everolimus) and Nolvadex (tamoxifen) resulted in decreased colony formation by 95% in estrogen-receptor (ER) positive breast cancer cell lines while in ER positive breast cancer cell lines resistant to Nolvadex (tamoxifen), colony formation formation decreased by 76% with the addition of Afinitor (everolimus) (PMID: 27421652). | 27421652 | |
| Unknown unknown | colon adenocarcinoma | not applicable | Cetuximab + Temsirolimus | Preclinical | Actionable | In a preclinical study, Torisel (temsirolimus) decreased resistance to Erbitux (cetuximab) in colon cancer cells (PMID: 24493623). | 24493623 | |
| Unknown unknown | subacute myeloid leukemia | not applicable | Cytarabine + Venetoclax | Preclinical - Patient cell culture | Actionable | In a preclinical study, Venclexta (venetoclax) and Cytosar-U (cytarabine) combination treatment synergistically induced cell death in patient-derived acute myeloid leukemia cells in culture (PMID: 27103402). | 27103402 | |
| Unknown unknown | 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 | uveal melanoma | not applicable | AU-011 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with AU-011 resulted in cell binding and subsequent cell death in uveal melanoma cells in culture (Cancer Res 2014;74(19 Suppl):Abstract nr 1770). | detail... | |
| Unknown unknown | gastrointestinal stromal tumor | not applicable | Cediranib | Phase II | Actionable | In a Phase II trial, treatment with Cediranib (AZD-2171) resulted in stable disease as best response in 55% (11/20) gastrointestinal stromal tumor patients, with 8 patients achieving stable disease for greater than or equal to 16 weeks (PMID: 24714778). | 24714778 | |
| Unknown unknown | esophageal cancer | not applicable | PP-121 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, PP-121 inhibited proliferation and growth of esophageal cancer cells in culture and in cell line xenograft models (PMID: 26235881). | 26235881 | |
| Unknown unknown | multiple myeloma | not applicable | Pomalidomide + SJB3-019A | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of SJB3-019A and Pomalyst (pomalidomide) worked synergistically to induce cytotoxicity in multiple myeloma cell lines and primary multiple myeloma cells in culture (PMID: 28270494). | 28270494 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Vandetanib | Phase II | Actionable | In a Phase II trial, Caprelsa (vandetanib) alone, or in combination with Taxotere (docetaxel), improved progression-free survival of NSCLC patients (PMID: 17243944). | 17243944 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Vandetanib | Phase II | Actionable | In a Phase II trial, treatment with Caprelsa (vandetanib) increased progression-free survival in NSCLC patients, compared to Iressa (gefitinib) (PMID: 19332730). | 19332730 | |
| Unknown unknown | 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 | MBG453 + Spartalizumab | Case Reports/Case Series | Actionable | In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in partial response in a patient with non-small cell lung cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). | detail... | |
| Unknown unknown | 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 | urinary bladder cancer | not applicable | Celecoxib + OBP-801 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Celebra (celecoxib) and OBP-801 resulted in a synergistic effect, demonstrating increased apoptotic activity and decreased tumor volume in xenograft models of bladder cancer (PMID: 27406983). | 27406983 | |
| Unknown unknown | 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 | melanoma | not applicable | Ramucirumab | Phase II | Actionable | In a Phase II trial, Cyramza (ramucirumab), with or without Deticene (dacarbazine), demonstrated safety and efficacy in patients with metastatic melanoma. The combined therapy appeared to be associated with improved progression free survival relative to monotherapy (J Clin Oncol 28:15s, 2010 (suppl; abstr 8519)). | detail... | |
| Unknown unknown | ovarian carcinoma | not applicable | NMS-P715 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, NMS-P715 inhibited tumor growth in ovarian carcinoma cell line xenograft models (PMID: 21159646). | 21159646 | |
| Unknown unknown | B-cell lymphoma | not applicable | Loncastuximab tesirine | Phase I | Actionable | In a Phase I trial, Loncastuximab tesirine treatment demonstrated safety and preliminary efficacy, resulted in an overall response rate of 59.4% (41/69, 28 complete response, 13 partial response) in patients with relapsed/refractory B-cell non-Hodgkin lymphoma, with median duration of response, progression-free survival, and overall survival of 4.8, 5.5, and 11.6 months, respectively (PMID: 31685491; NCT02669017). | 31685491 | |
| Unknown unknown | glioblastoma multiforme | not applicable | Cediranib + Gefitinib | Phase II | Actionable | In a Phase II trial, treatment with the combination of Cediranib (AZD-2171) and Iressa (gefitinib) resulted in a trend toward improved progression-free survival compared to Cediranib (AZD-2171) and placebo (3.6 months vs 2.8 months), and resulted in a response rate of 42% (8/19), compared to 26% (5/19) with Cediranib (AZD-2171) plus placebo in patients with recurrent glioblastoma (PMID: 27232884). | 27232884 | |
| Unknown unknown | multiple myeloma | not applicable | GSK2857916 | Phase I | Actionable | In a Phase I trial, GSK2857916 demonstrated manageable safety and preliminary activity in patients with multiple myeloma, with 60% (21/35) of patients in part 2 receiving the recommended phase 2 dose achieving a response (1 stringent complete response (CR), 1 CR, 2 very good partial responses (PR), and 3 PR) (PMID: 30442502; NCT02064387). | 30442502 | |
| Unknown unknown | pancreatic cancer | not applicable | CG200745 | Preclinical - Cell culture | Actionable | In a preclinical study, CG200745 decreased viability of pancreatic cancer cell lines in culture, including Gemzar (gemcitabine)-resistant cell lines (PMID: 28134290). | 28134290 | |
| Unknown unknown | prostate cancer | not applicable | Navitoclax + TAK-901 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of TAK-901 and Navitoclax (ABT-263) resulted in a synergistic effect, demonstrating reduced cell viability of prostate cancer cells in culture (PMID: 28179288). | 28179288 | |
| Unknown unknown | thymic carcinoma | not applicable | Selinexor | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Selinexor (KPT-330) induced cell-cycle arrest and apoptosis and inhibited growth of several thymic epithelial tumor cell lines, including thymoma and thymic carcinoma cell lines, in culture, and inhibited tumor growth in thymic carcinoma cell line xenograft models (PMID: 28819023). | 28819023 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CLR457 | Preclinical - Pdx | Actionable | In a preclinical study, CLR457 treatment of a variety of solid tumor patient-derived xenograft models decreased tumor volume and produced a 54% response (PMID: 26479923). | 26479923 | |
| Unknown unknown | ovarian cancer | not applicable | Nintedanib | Phase I | Actionable | In a Phase I clinical trial, Vargatef (nintedanib) demonstrated safety in patients with ovarian cancers, clinical trials to determine efficacy in these patients are ongoing (PMID: 19889612). | 19889612 | |
| Unknown unknown | 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 | prostate cancer | not applicable | MC180295 | Preclinical - Cell culture | Actionable | In a preclinical study, MC180295 decreased proliferation of prostate cancer cell lines in culture (PMID: 30454645). | 30454645 | |
| Unknown unknown | gastrointestinal neuroendocrine tumor | not applicable | Pazopanib | Clinical Study | Actionable | In a clinical study, Votrient (pazopanib) treatment in patients with gastroenteropancreatic neuroendocrine tumors resulted in an overall response rate of 24% (19/124), stable disease in 39.5% (49/124), a progression free survival of 36% at six months, and a median overall survival of 10.2 months (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 446P). | detail... | |
| Unknown unknown | 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 | grade III astrocytoma | not applicable | Trabedersen | Phase II | Actionable | In a Phase II trial, Trabedersen (AP 12009) treatment in patients with recurrent or refractory high-grade glioma (including grade IV glioblastoma multiforme or grade III astrocytoma) resulted in responses in 19 (3 complete and 16 partial responses) and stable disease for at least 6 months in 7 of 77 patients in the efficacy population, and a median progression-free survival of 86 days and median overall survival of 432 days (PMID: 31795071; NCT00431561). | 31795071 | |
| Unknown unknown | pancreatic ductal adenocarcinoma | not applicable | MitoMet-10 | Preclinical | Actionable | In a preclinical study, MitoMet-10 inhibited mitochondrial function and proliferation of pancreatic ductal adenocarcinoma cells in culture, and demonstrated increased potency compared to metformin (PMID: 27216187). | 27216187 | |
| Unknown unknown | acute myeloid leukemia | not applicable | CX-6258 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CX-6528 inhibited tumor growth in human acute myeloid leukemia cell line xenograft models (PMID: 24900437). | 24900437 | |
| Unknown unknown | multiple myeloma | not applicable | Lenalidomide | Phase III | Actionable | In a Phase III trial, Revlimid (lenalidomide) maintenance treatment after stem cell transplant resulted in improved overall survival and progression free survival in patients with multiple myeloma (J Clin Oncol 35, 2017 (suppl; abstr 8037)). | detail... | |
| Unknown unknown | 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 | breast cancer | not applicable | AsiDNA + AZD2461 | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA and AZD2461 combination treatment resulted in increased cell death and inhibition of proliferation in breast cancer cell lines compared to AZD2461 alone in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | Oprozomib | Phase I | Actionable | In a Phase I trial, Oprozomib (ONX 0912) demonstrated clinically relevant toxicity and minimal efficacy, with stable disease as best response in patients with advanced solid tumors (PMID: 26924128). | 26924128 | |
| Unknown unknown | 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 | Ewing sarcoma | not applicable | BMS-754807 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 50% (2/4) of cell line xenograft models of Ewing sarcoma (PMID: 21298745). | 21298745 | |
| Unknown unknown | lung non-small cell carcinoma | no benefit | Bevacizumab + Carboplatin + Cixutumumab + Paclitaxel | Phase II | Actionable | In a Phase II trial, the combination therapy of Avastin (bevacizumab), Paraplatin (carboplatin), Taxol (paclitaxel), and Cixutumumab resulted in greater toxicity and did not improve overall survival when compared to Avastin (bevacizumab), Paraplatin (carboplatin), and Taxol (paclitaxel) without Cixutumumab in non-small cell lung carcinoma patients (PMID: 28950351; NCT00955305). | 28950351 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ipatasertib | Phase I | Actionable | In a Phase I trial, Ipatasertib (GDC-0068) resulted in antitumor activity in 30% (16/52) of patients with advanced solid tumors, primarily demonstrating stable disease (PMID: 27872130). | 27872130 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ipatasertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Ipatasertib (GDC-0068) demonstrated activity against tumor growth in cell line xenograft models of solid tumors (PMID: 24141624). | 24141624 | |
| Unknown unknown | colorectal cancer | not applicable | PD-0325901 | Phase I | Actionable | In a Phase I study, PD-325901 demonstrated efficacy but toxicity at current dose was unacceptable (PMID: 21516509). | 21516509 | |
| Unknown unknown | 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 | 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 | Indication other than cancer | not applicable | Metformin | FDA approved | Actionable | Glucophage (metformin) is FDA approved for use in patients with type-2 diabetes (FDA.gov). | detail... detail... | |
| Unknown unknown | angioimmunoblastic T-cell lymphoma | not applicable | Domatinostat | Case Reports/Case Series | Actionable | In a Phase I trial, a patient with relapsed angioimmunoblastic T-cell lymphoma demonstrated a complete response lasting over 2 years following treatment with Domatinostat (4SC-202) (PMID: 30347469; NCT01344707). | 30347469 | |
| Unknown unknown | lymphoma | not applicable | Abexinostat | Phase II | Actionable | In a Phase II trial, patients with T-cell lymphoma demonstrated an overall response rate of 40% (6/15) and a median duration response of 11.5 months when treated with Abexinostat (PCI-24781) (PMID: 28126962). | 28126962 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Acalabrutinib + ACP-319 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Calquence (acalabrutinib) and ACP-319 resulted in decreased tumor volume in diffuse large B-cell lymphoma xenograft models (Eur J of Cancer, Dec 2016, 69;1, S39-S40). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | 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 | NSC156529 | Preclinical | Actionable | In a preclinical study, NSC156529 inhibited growth of transformed human cell lines in culture (PMID: 26294745). | 26294745 | |
| 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 | pancreatic cancer | no benefit | Gemcitabine + Vandetanib | Phase II | Actionable | In a Phase II trial, addition of Caprelsa (vandetanib) to Gemzar (gemcitabine) did not improve median overall survival (8.83 vs 8.95 months) compared to Gemzar (gemcitabine) monotherapy in patients with advanced pancreatic cancer (PMID: 28259610). | 28259610 | |
| Unknown unknown | multiple myeloma | not applicable | Daratumumab + Dexamethasone + Lenalidomide | FDA approved | Actionable | In a Phase III trial (POLLUX) that supported FDA approval, the combination of Darzalex (daratumumab), Adexone (dexamethasone), and Revlimid (lenalidomide) resulted in a greater 12 month progression-free survival (83.2% vs 60.1%) and overall response (92.9%; 261/281 vs 76.4%; 211/276) compared to Adexone (dexamethasone) and Revlimid (lenalidomide) alone in patients with relapsed or refractory multiple myeloma (PMID: 27705267; NCT02076009). | detail... 27705267 | |
| Unknown unknown | multiple myeloma | not applicable | Daratumumab + Dexamethasone + Lenalidomide | FDA approved | Actionable | In a Phase III trial (MAIA) that supported FDA approval, the combination of Darzalex (daratumumab), Adexone (dexamethasone), and Revlimid (lenalidomide) resulted in an improved rate of progression-free survival at 30 months (70.6% vs 55.6%, HR=0.56, p<0.001) compared control in newly diagnosed multiple myeloma patients ineligible for autologous stem-cell transplantation (PMID: 31141632; NCT02252172). | detail... 31141632 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Alisertib + Paclitaxel | Preclinical - Pdx | Actionable | In a preclinical study, the combination of Alisertib (MLN8237) and Taxol (paclitaxel) worked synergistically or additively to inhibit tumor growth in cell line and patient-derived xenograft (PDX) models of triple-negative breast cancer (PMID: 24980948). | 24980948 | |
| Unknown unknown | diffuse large B-cell 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 40% (6/15, 5 complete and 1 partial response) and stable disease in 20% (3/15) of patients with diffuse large B-cell lymphoma, and a median duration of response longer than 6 months (PMID: 30380386). | 30380386 | |
| Unknown unknown | 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 | mantle cell lymphoma | sensitive | STRO-001 | Preclinical - Cell culture | Actionable | In a preclinical study, STRO-001 potently inhibited growth of mantle cell lymphoma cell lines in culture (Blood 2016 128 (22):464). | detail... | |
| Unknown unknown | pancreatic cancer | not applicable | G-TPP + Obatoclax | Preclinical - Cell culture | Actionable | In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Obatoclax (GX015-070) synergistically inhibited viability of pancreatic cancer cells in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | Her2-receptor negative breast cancer | not applicable | Paclitaxel + Reparixin | Phase Ib/II | Actionable | In a Phase Ib trial, the combination therapy of Taxol (paclitaxel) and Reparixin in patients with metastatic ERBB2 (HER2)-receptor negative breast cancer resulted in a 30% (8/27) response rate and a durable response greater than 12 months in two patients (PMID: 28539464; NCT02001974). | 28539464 | |
| Unknown unknown | central nervous system lymphoma | not applicable | Ibrutinib | Phase I | Actionable | In a Phase I trial, Imbruvica (ibrutinib) treatment resulted in antitumor efficacy in 77% (10/13) of patients with primary central nervous system lymphoma, demonstrating a complete response in five patients and a partial response in five patients (PMID: 28619981). | 28619981 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Decitabine + TMU-35435 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of TMU-35435 and Dacogen (decitabine) worked synergistically to decrease viability of non-small cell lung cancer cell lines in culture, and to inhibit tumor growth in a non-small cell lung cancer cell line xenograft model (PMID: 28233309). | 28233309 | |
| Unknown unknown | renal cell carcinoma | not applicable | Nivolumab | Clinical Study | Actionable | In a retrospective analysis, Opdivo (nivolumab) treatment demonstrated an ORR of 22% (41/187), 24% (22/90) and 26% (15/58), and DOT of 5.7 mo, 6.2 mo, and 8.3 mo in the 2nd, 3rd, and 4th-line setting respectively, and a median OS in the 2nd-line setting in favorable, intermediate, and poor-risk groups of not reached (NR), 26.7 mo, and 7.4 mo, respectively; 36.1 mo, 28.2 mo, and 11.1 mo in the 3rd-line setting; and NR, NR, and 6.7 mo in the 4th-line setting in renal cell carcinoma patients (PMID: 30307610). | 30307610 | |
| Unknown unknown | renal cell carcinoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase III clinical trial (CheckMate 025) that supported FDA approval, treatment with Opdivo (nivolumab) resulted in a median overall survival of 25 months, compared to 19.6 months with Afinitor (everolimus) and an objective response rate of 25% versus 5% with Afinitor (everolimus) in patients with advanced renal cell carcinoma (PMID: 26406148; NCT01668784). | 26406148 detail... | |
| Unknown unknown | 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 | triple-receptor negative breast cancer | not applicable | HY-16462 + JQ1 | Preclinical - Cell culture | Actionable | In a preclinical study, HY-16462 and JQ1 treatment synergistically inhibited growth of a triple-negative breast cancer cell line in culture (PMID: 31000582). | 31000582 | |
| 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 | 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 | multiple myeloma | not applicable | Bortezomib + DT204 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a Velcade (bortezomib) resistant multiple myeloma cell line overcame resistance when treatment was combined with DT204, and in xenograft models the combined treatment resulted in delayed tumor growth and improved survival (PMID: 27677741). | 27677741 | |
| Unknown unknown | 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 | malignant glioma | not applicable | DNX-2401 | Phase I | Actionable | In a Phase I trial, DNX-2401 treatment demonstrated virus-induced oncolysis in patients' tumors, resulted in more than 95% tumor reduction in 3 patients, and survival for more than 3 years in 20% (5/25) of patients with recurrent malignant glioma (PMID: 29432077; NCT02197169). | 29432077 | |
| Unknown unknown | malignant glioma | not applicable | DNX-2401 | Phase I | Actionable | In a Phase I trial, DNX-2401 treatment resulted in tumor reduction in 72% (18/25), complete response in 12% (3/25) and prolonged stable disease in 8% (2/25) of patients with recurrent malignant glioma, with a median overall survival of 9.5 months and progression-free survival of more than 3 years in patients achieved complete responses (PMID: 29432077). | 29432077 | |
| Unknown unknown | B-cell lymphoma | not applicable | Alisertib + Rituximab | Phase I | Actionable | In a Phase I trial, Alisertib (MLN8237) in combination with Rituxan (rituximab) demonstrated safety and efficacy, resulted in an objective response rate of 25% (3/12, 2 complete response, 1 partial response), and stable disease in 42% (5/12) of patients with relapsed or refractory B-cell non-Hodgkin lymphomas (PMID: 30082475; NCT01397825). | 30082475 | |
| Unknown unknown | gastroesophageal cancer | 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 6% (1/16) and a disease control rate of 50% (8/16) in patients with gastroesophageal cancer, with a median progression-free survival of 1.6 months (PMID: 31142504; NCT02082210). | 31142504 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Docetaxel + S63845 | Preclinical - Pdx | Actionable | In a preclinical study, S63845 and Taxotere (docetaxel) demonstrated synergy in triple-negative breast cancer patient-derived xenograft models, resulting in enhanced tumor growth inhibition and overall survival compared to either agent alone (PMID: 28768804). | 28768804 | |
| Unknown unknown | 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 | multiple myeloma | no benefit | Tivantinib | Phase II | Actionable | In a Phase II trial, Tivantinib (ARQ197) treatment only resulted in stable disease in 36% (4/11) of patients with relapsed or refractory multiple myeloma (PMID: 28337527). | 28337527 | |
| Unknown unknown | 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 | hepatocellular carcinoma | not applicable | PEGPH20 | Phase I | Actionable | In a Phase Ib trial, PEGPH20 treatment resulted in median progression free survival of 7.2 months and overall survival of 13.0 months in hepatocellular carcinoma patients with high pretreatment tissue hyaluronan (HA) levels (n = 6), and 3.5 and 5.7 months respectively for patients with low HA levels (n = 11) (PMID: 26813359). | 26813359 | |
| Unknown unknown | liposarcoma | not applicable | MGCD516 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MGCD516 blocked cell proliferation of a human liposarcoma cell line in culture and repressed tumor growth in xenograft models (PMID: 26675259). | 26675259 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | JIB-04 | Preclinical | Actionable | In a preclinical study, JIB-04, a pan Jumonji histone demethylase inhibitor, reduced cell proliferation, and induced apoptosis in multiple cancer cell types while reducing tumor burden and increasing survival in mouse xenograft models (PMID: 23792809). | 23792809 | |
| Unknown unknown | B-cell lymphoma | not applicable | Nutlin-3 | Preclinical - Cell culture | Actionable | In a preclinical study, Nutlin-3 induced death of a mouse B-cell lymphoma cell line isolated from a tumor over expressing MYC in culture (PMID: 30069049). | 30069049 | |
| Unknown unknown | renal cell carcinoma | not applicable | Sunitinib + Trametinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, combination of Mekinist (trametinib) and Sutent (sunitinib) effectively inhibited tumor angiogenesis and growth in cell line xenograft models of Sutent (sunitinib)-refractory renal cell carcinoma (PMID: 26487278). | 26487278 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Pexidartinib + PLX9486 | Phase I | Actionable | In a Phase I trial, PLX9486 and Pexidartinib (PLX3397) combination therapy demonstrated safety and preliminary efficacy, resulted in a partial response rate of 8.3% (1/12) and progression-free survival not yet reached in patients with advanced solid tumors, 11 of these patients had gastrointestinal stromal tumor that progressed on Gleevec (imatinib mesylate), and most harbored KIT exon 11 and exon 17 mutations (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 11509-11509; NCT02401815). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | PRN1371 | Preclinical - Pdx | Actionable | In a preclinical study, PRN1371 treatment resulted in tumor regression in PDX models of non-small cell lung cancer (Eu J Cancer 2014 Vol 50, Suppl 6:157). | detail... | |
| Unknown unknown | pancreatic cancer | not applicable | Demcizumab + Gemcitabine | Phase Ib/II | Actionable | In a Phase Ib trial, Demcizumab (OMP-21M18) and Gemzar (gemcitabine) combination treatment resulted in partial response in 25% (4/16) and stable disease in 44% (7/16) of pancreatic cancer patients (J Clin Onco, Vol 32, No 3_suppl (January 20 Supplement), 2014: 279). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | ETP-46464 + Radiotherapy | Preclinical - Cell culture | Actionable | In a preclinical study, ETP-46464 increased the sensitivity of ovarian cancer cell lines to ionizing radiation in culture (PMID: 25560806). | 25560806 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | LTX-315 + Pegylated liposomal-doxorubicin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of LTX-315 and Doxil (pegylated liposomal doxorubicin) resulted in increased tumor growth inhibition and regression, increased tumor necrosis, and increased T-cell infiltration in triple-negative breast cancer (TNBC) cell line xenograft models compared to either agent alone, and in TNBC models in the neoadjuvant setting induced tumor regression in 50% and improved survival compared to either agent alone (PMID: 30670061). | 30670061 | |
| Unknown unknown | 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 | 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 | 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 | ovarian cancer | not applicable | Paclitaxel + TRX-E-002-1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TRX-E-002-1 given as a maintenance treatment after Taxol (paclitaxel) treatment resulted in less tumor recurrence compared to placebo in cell line xenograft models of ovarian cancer (PMID: 27196760). | 27196760 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ganitumab | Phase I | Actionable | In a Phase I trial, Ganitumab (AMG 479) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol (Meeting Abstracts) 2007 25: 3002). | detail... | |
| Unknown unknown | lymphoma | not applicable | S63845 | Preclinical - Cell culture | Actionable | In a preclinical study, S63845 inhibited survival of several lymphoma cell lines in culture (PMID: 27760111). | 27760111 | |
| Unknown unknown | hematologic cancer | not applicable | Cenisertib | Phase I | Actionable | In a Phase I clinical trial, Cenisertib (AS703569) demonstrated safety and preliminary efficacy in patients with advanced hematological malignancies (Blood 2008 112:2963). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Pemetrexed + Sorafenib | Phase I | Actionable | In a Phase I clinical trial, the combination of Alimta (pemetrexed) and Nexavar (sorafenib) demonstrated safety and some preliminary efficacy in patients with advanced solid tumors, with an objective response rate of 15% (5/33) and stable disease in 45% (15/33) of patients (PMID: 27213589). | 27213589 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Talazoparib | Phase I | Actionable | In a Phase I trial, Talazoparib (BMN-673) treatment in patients with lung small cell carcinoma resulted in an objective response rate of 9% (2/23), including two patients with a partial response, and four patients with stable disease for at least 16 weeks (PMID: 28242752). | 28242752 | |
| Unknown unknown | glioblastoma multiforme | 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 | prostate cancer | not applicable | Paclitaxel + SPC3042 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of SPC3042 and Taxol (paclitaxel) worked synergistically to inhibit tumor growth in prostate cancer cell line xenograft models, with increased activity over either agent alone (PMID: 18790754). | 18790754 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | BMS-906024 | Preclinical | Actionable | In a preclinical study, the pan-Notch inhibitor, BMS-906024, inhibited growth of triple-negative breast cancer xenografts (PMID: 26005526). | 26005526 | |
| Unknown unknown | cervical cancer | not applicable | Cisplatin + ETP-46464 | Preclinical - Cell culture | Actionable | In a preclinical study, ETP-46464 increased the sensitivity of cervical cancer cell lines to Platinol (cisplatin) in culture (PMID: 25560806). | 25560806 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ARQ 751 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with ARQ 751 resulted in anti-proliferative activity in a variety of cancer cell lines in culture (PMID: 26469692). | 26469692 | |
| Unknown unknown | mantle cell lymphoma | no benefit | MOR208 | Phase II | Actionable | In a Phase II trial, MOR208 treatment was well-tolerated and resulted in stable disease as the best response in 50% (6/12) of patients with mantle cell lymphoma (PMID: 29444231; NCT01685008). | 29444231 | |
| Unknown unknown | colon adenocarcinoma | not applicable | CC-223 | Phase I | Actionable | In a Phase I trial, CC-223 demonstrated safety and some efficacy in patients with solid tumors, including colon cancer (J Clin Oncol 31, 2013 (suppl; abstr 2606). | detail... | |
| Unknown unknown | 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 | follicular 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 71% (10/14), complete response/complete metabolic response rate of 21% (3/14), and median duration of response was not reached patients with follicular lymphoma (PMID: 30803990; NCT02018861). | 30803990 | |
| Unknown unknown | malignant peripheral nerve sheath tumor | not applicable | Alisertib | Phase II | Actionable | In a Phase II trial, Alisertib (MLN8237) treatment resulted in twelve-week progression free survival in 60% (6/10) of malignant peripheral nerve sheath tumor patients (PMID: 27502708). | 27502708 | |
| Unknown unknown | lung cancer | not applicable | AZ628 + Trametinib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Mekinist (trametinib) and AZ628 resulted in greater inhibition of Mek and apoptosis in a non-BRAF V600 mutant lung cancer cell line in culture compared to the combination of Mekinist (trametinib) and Tafinlar (dabrafenib) (J Clin Oncol 35, 2017 (suppl; abstr e23154)). | detail... | |
| Unknown unknown | gastrointestinal stromal tumor | not applicable | Apatinib | Phase I | Actionable | In a Phase I trial, Apatinib (YN968D1) produced a partial response in 18.9% (7/37) and stable disease in 64.9% (24/37) of patients with advanced solid tumors, including partial response in one patient with GIST (PMID: 20923544). | 20923544 | |
| Unknown unknown | pancreatic cancer | not applicable | Gemcitabine + Vantictumab | Preclinical - Pdx | Actionable | In a preclinical study, Vantictumab (OMP-18R5) worked synergistically with Gemzar (gemcitabine) to inhibit tumor growth in patient-derived xenograft models of pancreatic cancer (PMID: 22753465). | 22753465 | |
| Unknown unknown | thyroid gland medullary carcinoma | not applicable | Lenvatinib | Phase II | Actionable | In a Phase II trial, advanced medullary thyroid cancer patients experienced an objective response rate of 36% (21/59, all partial responses) and median progression free survival was 9 months when treated with Lenvima (lenvatinib) (PMID: 26311725). | 26311725 | |
| Unknown unknown | 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 | lymphoma | not applicable | Denileukin diftitox + Sirolimus | Preclinical | Actionable | In a preclinical study, a low dose of Rapamune (sirolimus) enhanced the efficacy of Ontak (denileukin diftitox) treatment in lymphoma mouse models, resulting in greater inhibition of tumor growth and higher survival compared to either agent alone (PMID: 27737881). | 27737881 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Bevacizumab + Carboplatin + Paclitaxel | FDA approved | Actionable | In an open-label trial that supported FDA approval, treatment with the combination of Avastin (bevacizumab) with Paraplatin (carboplatin) and Taxol (paclitaxel) resulted in improved overall survival compared to Paraplatin (carboplatin) and Taxol (paclitaxel) alone in patients with advanced non-small cell lung cancer (PMID: 17602060). | 17602060 detail... | |
| Unknown unknown | liposarcoma | not applicable | Trabectedin | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Yondelis (trabectedin) treatment resulted in an improved median progression-free survival of 4.2 months versus 1.5 months with Deticene (dacarbazine) in patients with unresectable or metastatic liposarcoma or leiomyosarcoma (PMID: 28774898; NCT01343277). | 28774898 detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BGB-A317 + Pamiparib | Phase Ib/II | Actionable | In a Phase Ib trial, the combination therapy of BGB-A317 and BGB-290 in patients with advanced solid tumors resulted in 7 patients with a partial response, one patient with a complete response, and 6 patients experiencing stable disease for greater than 6 months (J Clin Oncol 35, 2017 (suppl; abstr 3013)). | detail... | |
| Unknown unknown | colorectal cancer | not applicable | Ibrutinib + unspecified CTLA4 antibody | Preclinical | Actionable | In a preclinical study, the combination of Imbruvica (ibrutinib) and an anti-CTLA4 antibody resulted in complete tumor regression in colorectal cancer mouse models (Cancer Res 2016;76(14 Suppl):Abstract nr 2321). | detail... | |
| Unknown unknown | multiple myeloma | not applicable | Cyclophosphamide + LCL161 | Phase II | Actionable | In a Phase II trial, LCL161 treatment followed by Cytoxan (cyclophosphamide) resulted in complete response in 4% (1/25), partial response in 12% (3/25), and molecular response in 4% (1/25) of multiple myeloma patients (PMID: 27841872). | 27841872 | |
| Unknown unknown | breast cancer | not applicable | Pyr1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Pyr1 inhibited proliferation and migration of breast cancer cell lines in culture, and inhibited growth of primary tumors and metastases in breast cancer cell line xenograft models (PMID: 27216191). | 27216191 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | YM155 | Preclinical - Cell culture | Actionable | In a preclinical study, YM155 induced complete growth inhibition of triple-receptor negative breast cancer cells in culture (PMID: 27872098). | 27872098 | |
| Unknown unknown | ovarian cancer | not applicable | AZD7648 + Olaparib | Preclinical - Cell culture | Actionable | In a preclinical study, AZD7648 and Lynparza (olaparib) combination treatment inhibited viability of ovarian cancer cell lines harboring wild-type ATM in culture (PMID: 31699977). | 31699977 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | LY3039478 | Phase I | Actionable | In a Phase I trial, LY3039478 treatment resulted in 80% inhibition of plasma A-beta and more than 50% inhibition of Notch1-regulated genes in patients with advanced solid tumor, leading to partial response in 0.9% (1/110) and stable disease in 32.7% (36/110) of the patients (PMID: 30060061; NCT01695005). | 30060061 detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | LY3039478 | Phase I | Actionable | In a Phase I trial, treatment with LY3039478 was well-tolerated and demonstrated some preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 33, 2015 (suppl; abstr 2533)). | detail... | |
| Unknown unknown | 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 | Advanced Solid Tumor | not applicable | MBG453 + Spartalizumab | Phase Ib/II | Actionable | In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in partial response in 5% (4/86) and stable disease in 40% (34/86) of patients with advanced solid tumor (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Erlotinib + Pictilisib | Phase I | Actionable | In a Phase I trial, the combination treatment of Tarceva (erlotinib) and Pictilisib (GDC-0941) in patients with advanced solid tumors resulted in toxicity, requiring dose adjustments, and led to minimal antitumor activity including two partial responses and stable disease in nineteen patients (PMID: 28798270). | 28798270 | |
| Unknown unknown | rhabdoid cancer | not applicable | Panobinostat | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Farydak (panobinostat) treatment of rhabdoid cancer cell lines in culture resulted in cell cycle arrest and cell differentiation and in cell line xenograft models, inhibition of tumor growth and a decrease in tumor size (PMID: 26920892). | 26920892 | |
| Unknown unknown | lung small cell carcinoma | not applicable | Bevacizumab + Cisplatin + Etoposide | Phase III | Actionable | In a Phase III trial, the addition of Avastin (bevacizumab) to treatment with Platinol (cisplatin) and Vepesid (etoposide) resulted in a median progression-free survival of 6.7 months, compared to 5.7 months with Platinol (cisplatin) and Vepesid (etoposide) in combination, but did not result in significantly improved overall survival in patients with small-cell lung cancer (PMID: 28135143). | 28135143 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | IsoA | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with IsoA inhibited cell growth in a variety of cancer cell lines in culture, and induced apoptosis via ROS generation (PMID: 28011619). | 28011619 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | DS-7423 | Phase I | Actionable | In a Phase I trial, DS-7423 demonstrated safety and preliminary clinical activity in patients with advanced solid tumors (Ann Oncol (2014) 25 (suppl 4): iv153). | detail... | |
| Unknown unknown | breast cancer | not applicable | MBQ-167 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MBQ-167 increased cell-cycle arrest and decreased viability and migration of breast cancer cell lines in culture, and reduced tumor growth and metastasis in a breast cancer cell line xenograft model (PMID: 28450422). | 28450422 | |
| Unknown unknown | stomach cancer | no benefit | Ipilimumab | Phase II | Actionable | In a Phase II trial, Yervoy (ipilimumab) did not improve immune-related progression-free survival (2.9 vs 4.9 months) compared to best supportive care in patients with unresectable, locally advanced/metastatic gastric or gastroesophageal junction cancer (J Clin Oncol 34, 2016 (suppl; abstr 4011)). | detail... | |
| Unknown unknown | multiple myeloma | sensitive | CB-5083 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CB-5083 treatment of multiple myeloma cell lines and xenografts resulted in cell death and decreased tumor growth, respectively (PMID: 28878026). | 28878026 | |
| Unknown unknown | Ewing sarcoma | not applicable | SN-38 + Veliparib | Preclinical | Actionable | In a preclinical study, Ewing sarcoma cells treated with SN-38 combined with Veliparib (ABT-888) resulted in synergism, demonstrating reduced cell viability in culture (PMID: 26438158). | 26438158 | |
| Unknown unknown | prostate cancer | not applicable | Darolutamide | FDA approved | Actionable | In a Phase III trial (ARAMIS) that supported FDA approval, treatment with Nubeqa (darolutamide) resulted in improved median metastasis-free survival (40.4 vs 18.4 months, HR=0.41, p<0.001), overall survival (HR=0.71, 95% CI, 0.5-0.99, p=0.045), and time to pain progression (40.3 vs 25.4 months, HR=0.65, p<0.001) compared to placebo in non-metastatic castration-resistant prostate cancer patients (PMID: 30763142; NCT02200614). | detail... 30763142 | |
| Unknown unknown | peripheral T-cell lymphoma | not applicable | Belinostat | FDA approved | Actionable | In clinical trials that supported FDA approval, treatment with Beleodaq (belinostat) resulted in an objective response rate of 25.8% (31/120) and a median overall survival of 7.9 months in patients with peripheral T-cell lymphoma (PMID: 26101246). | detail... 26101246 | |
| Unknown unknown | urinary bladder cancer | not applicable | GSK2126458 | Phase I | Actionable | In Phase I trial, GSK2126458 treatment was well-tolerated and resulted in a partial response and stable disease in two patients and one patient with bladder cancer, respectively (PMID: 26603258). | 26603258 | |
| Unknown unknown | breast cancer | not applicable | AZ0108 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AZ0108 induced mitotic defects, apoptosis, and inhibited growth in breast cancer cell lines in culture, and inhibited tumor growth in cell line xenograft models (PMID: 30297535). | 30297535 | |
| Unknown unknown | myelodysplastic syndrome | not applicable | Cytarabine + Glasdegib | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of Glasdegib (PF-04449913) and Cytosar-U (cytarabine) resulted in an overall survival of 4.4 months in patients with either acute myeloid leukemia or myelodysplastic syndrome (MDS), with 33% (1/3) of MDS patients experiencing a complete remission (PMID: 29463550). | 29463550 | |
| Unknown unknown | 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 | glioblastoma multiforme | no benefit | MP7 | Preclinical - Cell culture | Actionable | In a preclinical study, MP7 treatment did not result in significant inhibition of cell proliferation and showed minimal change in cell viability in a glioblastoma cell line in culture, thus, providing no benefit (PMID: 27797168). | 27797168 | |
| Unknown unknown | hematologic cancer | not applicable | AsiDNA + Olaparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA treatment sensitized hematologic cancer cell lines to Lynparza (olaparib), inhibiting survival in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ALT-803 | Phase I | Actionable | In a Phase I trial, ALT-803 treatment demonstrated safety and preliminary efficacy in patients with advanced solid tumors, resulted in desirable NK cell expansion and strong reaction at injection sites (PMID: 30045932; NCT01727076). | 30045932 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | SL-801 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, SL-801 resulted in tumor growth inhibition in xenograft models with various advanced solid tumors (Journal of Clinical Oncology 33, no. 15_suppl). | detail... | |
| Unknown unknown | 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 | follicular lymphoma | not applicable | Umbralisib | Phase I | Actionable | In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in complete response in 12% (2/17) and partial response in 41% (7/17) of patients with follicular lymphoma (PMID: 29475723; NCT01767766). | 29475723 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Tivozanib | Phase I | Actionable | In a Phase I clinical study, Tivozanib (AV-951) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Clin Cancer Res, November 15, 2011 17; 7156). | detail... | |
| Unknown unknown | lung small cell carcinoma | not applicable | Navitoclax + Pictilisib | Preclinical - Pdx | Actionable | In a preclinical study, the combination of Navitoclax (ABT-263) and GDC-0941 delayed tumor growth in a circulating tumor cell (CTC)-derived xenograft model derived using CTCs from a small cell lung cancer patient (PMID: 27197306). | 27197306 | |
| 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 | esophagus squamous cell carcinoma | not applicable | Imetelstat + Radiotherapy | Preclinical - Cell line xenograft | Actionable | In a preclinical study, imetelstat increased sensitivity of human esophageal squamous cell carcinoma cell lines to radiotherapy, resulting in increased apoptosis and decreased survival in culture, and increased apoptosis, decreased proliferation, and reduced tumor growth in mouse models (PMID: 28099140). | 28099140 | |
| Unknown unknown | multiple myeloma | not applicable | Bortezomib + Tapalumab | 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 Tapalumab and Velcade (bortezomib) (PMID: 27287072). | 27287072 | |
| 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 | colon cancer | not applicable | Lenvatinib | Preclinical | Actionable | In a preclinical study, Lenvima (lenvatinib) induced apoptosis and inhibited proliferation of colorectal cancer cells in culture (PMID: 24255582). | 24255582 | |
| Unknown unknown | follicular lymphoma | not applicable | CPI-0610 | Phase I | Actionable | In a Phase I trial, treatment with CPI-0610 resulted in a partial response in one patient with follicular lymphoma (Blood 2015 126:1491). | 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 | subependymal giant cell astrocytoma | not applicable | Everolimus | FDA approved | Actionable | In a Phase III trial (EXIST-1) that supported FDA approval, Afinitor (everolimus) treatment resulted in a 50% or more tumor reduction in 35% (27/78) of adult and pediatric patients diagnosed with tuberous sclerosis complex and had subependymal giant cell astrocytoma, compared to 0% (0/39) in the placebo group (PMID: 23158522; NCT00789828). | 23158522 detail... | |
| Unknown unknown | 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 | lymphoma | not applicable | PQR309 + Venetoclax | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination therapy of PQR309 and Venclexta (venetoclax) led to antitumor activity in lymphoma cells in culture and cell line xenograft models, demonstrating both synergistic and additive effects (PMID: 29066507). | 29066507 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Rituximab + Venetoclax | FDA approved | Actionable | In a Phase III trial (MURANO) that supported FDA approval, Venclexta (venetoclax) treatment combined with Rituxan (rituximab) at the beginning of treatment resulted in significantly improved 2-year progression-free survival rate (84.9% vs 36.3%) compared to Treanda (bendamustine) and Rituxan (rituximab) combination in patients with relapsed or refractory chronic lymphocytic leukemia, with (81.5% vs 27.8%) or without (85.9% vs 41.0%) chromosome 17p deletion (PMID: 29562156; NCT02005471). | detail... 29562156 | |
| 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 | 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 | mantle cell lymphoma | not applicable | Umbralisib | Phase I | Actionable | In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in partial response in 17% (1/6) and stable disease in 67% (4/6) of patients with mantle cell lymphoma (PMID: 29475723; NCT01767766). | 29475723 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Selectikine | Phase I | Actionable | In a Phase I trial, Selectikine (EMD 521873) demonstrated safety and some preliminary activity in patients with advanced solid tumors (PMID: 22918078). | 22918078 | |
| 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 | colon cancer | not applicable | JSH-150 | Preclinical - Cell culture | Actionable | In a preclinical study, JSH-150 inhibited proliferation of a colon cancer cell line in culture (PMID: 30253346). | 30253346 | |
| Unknown unknown | breast cancer | not applicable | AB61 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the synthetic nucleoside AB61 resulted in repressed protein translation, decreased tumor volume, and prolonged survival in breast cancer cell line xenograft models (PMID: 26819331). | 26819331 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Erlotinib + Everolimus | Phase I | Actionable | In a Phase I trial, Afinitor (everolimus) demonstrated safety and some efficacy in combination with Tarceva (erlotinib) in patients with advanced NSCLC (PMID: 22968184). | 22968184 | |
| Unknown unknown | ovarian cancer | not applicable | Paclitaxel + VB-111 | Case Reports/Case Series | Actionable | In a clinical study, VB-111 in combination with Taxol (paclitaxel) resulted in increased tumor lymphocyte infiltration and tumor necrosis in biopsies obtained from 3 patients with ovarian cancer (AACR Annual Meeting 2019, Abstract 4979). | detail... | |
| Unknown unknown | 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 | Hodgkin's lymphoma | not applicable | INCB040093 | Phase I | Actionable | In a Phase I trial, INCB040093 treatment resulted in complete response in 17% (1/6) of Hodgkin's lymphoma patients, with an objective response rate of 50% (J Clin Oncol 33, 2015 (suppl; abstr 8558)). | detail... | |
| Unknown unknown | non-Hodgkin lymphoma | not applicable | Cyclophosphamide + inotuzumab ozogamicin + Prednisone + Rituximab + Vincristine | Phase I | Actionable | In a Phase I trial, the combination of inotuzumab ozogamicin with R-CVP (rituximab, cyclophosphamide, Oncovin (vincristine), and prednisone) resulted in an ORR of 84% (32/38) in non-Hodgkin lymphoma patients, including a complete response in 24% (9/38) of patients (PMID: 27154915). | 27154915 | |
| Unknown unknown | stomach cancer | not applicable | Camrelizumab | Phase I | Actionable | In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity, resulted in partial response in 37.5% (3/8) of patients with gastric cancer (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). | detail... | |
| Unknown unknown | melanoma | not applicable | GNE-317 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a melanoma cell line xenograft model demonstrated cell death of metastasized tumor cells within a small region of the brain when treated with GNE-317 (PMID: 27521448). | 27521448 | |
| Unknown unknown | malignant pleural mesothelioma | not applicable | SEPREHVIR | Phase Ib/II | Actionable | In a Phase I/II trial, SEPREHVIR treatment demonstrated safety and preliminary efficacy, induced anti-tumor immune response in patients with malignant pleural mesothelioma (Cancer Immunol Res 2016;4(1 Suppl):Abstract nr B033). | detail... | |
| Unknown unknown | rhabdomyosarcoma | not applicable | TAS4464 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TAS4464 inhibited growth and induced apoptosis in rhabdomyosarcoma cell lines in culture, resulted in tumor suppression in cell line xenograft models (Cancer Res 2016;76(14 Suppl):Abstract nr 3777). | detail... | |
| Unknown unknown | stomach cancer | not applicable | Ipilimumab + 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 | ovarian cancer | not applicable | Cabozantinib | Phase II | Actionable | In a Phase II clinical trial, Cometriq (cabozantinib ) demonstrated safety and efficacy in patients with ovarian cancers (J Clin Oncol 29: 2011 (suppl; abstr 5008)). | 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 | acute myeloid leukemia | not applicable | MK-8242 | Phase I | Actionable | In a Phase I trial, MK-8242 demonstrated safety and some preliminary efficacy in patients with refractory or recurrent acute myeloid leukemia, with 3 out of 24 evaluable patients demonstrating an objective response (PMID: 27544076). | 27544076 | |
| Unknown unknown | glioblastoma multiforme | not applicable | CC-115 | Preclinical - Pdx | Actionable | In a preclinical study, CC-115 increased survival of patient derived xenograft models of glioblastoma (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 1755). | detail... | |
| Unknown unknown | osteosarcoma | not applicable | Pembrolizumab | Phase II | Actionable | In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in partial response in 5% (1/22) of patients with osteosarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). | detail... | |
| Unknown unknown | cutaneous T cell lymphoma | not applicable | IPH4102 | Phase I | Actionable | In a Phase I trial, IPH4102 demonstrated safety, and cutaneous T-cell lymphoma patients treated with IPH4102 demonstrated an overall response rate of 36% (16/44), a median progression-free survival of 8.2 months, and a median duration of response of 13.8 months (PMID: 31253572; NCT02593045). | 31253572 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | TSR-033 + TSR-042 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with TSR-033 and 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 | lung carcinoma | predicted - sensitive | INCB001158 | Preclinical | Actionable | In a preclinical study, INCB001158 (CB-1158) had no effect on Lewis Lung carcinoma cells growth in culture, but inhibited tumor growth in syngeneic animal models through regulation of host immune response (Cancer Res 2016;76(14 Suppl):Abstract nr 552). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | 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 18% (5/28) of patients with non-small cell lung cancer, regardless of KRAS/BRAF status (PMID: 30918950; NCT01988896). | 30918950 | |
| Unknown unknown | melanoma | not applicable | Abemaciclib | Phase I | Actionable | In a Phase I trial, treatment with Abemaciclib (LY2835219) in melanoma patients resulted in a disease control rate of 27% (7/26), a partial response in one patient and six patients with stable disease (PMID: 27217383). | 27217383 | |
| Unknown unknown | 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 | malignant glioma | not applicable | Everolimus | Phase II | Actionable | In a Phase II trial, Afinitor (everolimus) treatment resulted in a 6-month progression free survival rate of 87% and 55% in patients with WHO grade II and III/IV glioma, respectively (Neuro Oncol (2016) 18 (suppl 6): vi8-vi9.). | detail... | |
| Unknown unknown | hematologic cancer | no benefit | OPB-51602 | Phase I | Actionable | In a Phase I trial, OPB-51602 treatment resulted in no objective response and stable disease in 15% (3/20) of patients with hematological malignancies (PMID: 25912076). | 25912076 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Doxorubicin + Safingol | Phase I | Actionable | In a Phase I trial, the combination of Kynacyte (safingol) and Adriamycin (doxorubicin) demonstrated safety and some preliminary activity in patients with advanced solid tumors (PMID: 9815717). | 9815717 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | KW-2450 | Phase I | Actionable | In a Phase I trial, 40% (4/10) of patients with advanced solid tumors demonstrated stable disease when treated with KW-2450 (PMID: 26850678). | 26850678 | |
| Unknown unknown | multiple myeloma | not applicable | Citarinostat + Pomalidomide | Preclinical - Cell line xenograft | Actionable | In a preclinical study, ACY-241, in combination with pomalidomide, demonstrated safety and prolonged survival in multiple myeloma cell line xenograft models (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 5380). | detail... | |
| Unknown unknown | breast cancer | not applicable | AsiDNA + Iniparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA and Iniparib combination treatment resulted in increased cell death and inhibition of proliferation in breast cancer cell lines compared to Iniparib alone in culture (PMID: 27559053). | 27559053 | |
| 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 | osteosarcoma | not applicable | SP-2509 | Preclinical - Cell culture | Actionable | In a preclinical study, SP-2509 treatment inhibited viability of osteosarcoma cell lines in culture (PMID: 29997151). | 29997151 | |
| Unknown unknown | ovarian cancer | not applicable | Conatumumab + Ganitumab | Phase Ib/II | Actionable | In a Phase Ib/II clinical trial, Ganitumab and Conatumumab combination treatment resulted in stable disease in 56% (5/9) of patients with ovarian cancer (PMID: 24816908). | 24816908 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | IT-901 | Preclinical | Actionable | In a preclinical study, IT-901 inhibited growth of diffuse large B-cell lymphoma cells in culture (PMID: 26744524). | 26744524 | |
| Unknown unknown | 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 | non-Hodgkin lymphoma | not applicable | Duvelisib | Phase I | Actionable | In a Phase I trial, Copiktra (duvelisib) treatment resulted in complete response in 16% (5/31), partial response in 45% (14/31), minor response in 3% (1/31), and stable disease in 29% (9/31) in non-Hodgkin lymphoma patients (Blood 124(21): 802). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | 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 | Hodgkin's lymphoma | not applicable | Ixazomib | Preclinical | Actionable | In a preclinical study, Ixazomib (MLN9708) inhibited survival and induced apoptosis in Hodgkin's lymphoma cell lines in culture, and reduced tumor volume in xenograft models (PMID: 26988986). | 26988986 | |
| Unknown unknown | gastroesophageal junction adenocarcinoma | not applicable | Napabucasin + Paclitaxel | Phase Ib/II | Actionable | In a Phase I/II tiral, combination of BBI608 (Napabucasin) and Taxol (paclitaxel) demonstrated safety and clinical efficacy in patients with advanced gastric and gastroesophageal junction adenocarcinoma (J Clin Oncol 33, 2015 (suppl; abstr 4069)). | detail... | |
| Unknown unknown | gastroesophageal junction adenocarcinoma | not applicable | Napabucasin + Paclitaxel | Phase III | Actionable | In a Phase III (BRIGHTER) trial, combination of Napabucasin (BBI608) and Taxol (paclitaxel) did not significantly improve overall survival (6.93 vs 7.36 months, HR=1.01, p=0.8596) or progression-free survival (3.55 vs 3.65 months, HR=1.00, p=0.9679) compared to placebo in patients with pretreated, advanced gastric and gastroesophageal junction adenocarcinoma (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 4010-4010; NCT02178956). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | MOR208 | Phase II | Actionable | In a Phase II trial, diffuse large B-cell lymphoma patients tolerated MOR208 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 | ovarian cancer | 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 40.9% (n=22) in patients with ovarian cancer (PMID: 28463960; NCT01188252). | 28463960 | |
| Unknown unknown | colorectal cancer | not applicable | Demcizumab | Phase I | Actionable | In a Phase I trial, treatment with Demcizumab (OMP-21M18) resulted in downregulation of Notch target genes and demonstrated preliminary efficacy in patients with advanced solid tumors, with reductions in tumor size in patients with several tumor types, including colorectal cancer (PMID: 25324140). | 25324140 | |
| Unknown unknown | pancreatic adenocarcinoma | not applicable | Gemcitabine + NSC109555 | Preclinical - Cell culture | Actionable | In a preclinical study, NSC109555 enhanced the anti-tumor effects of Gemzar (gemcitabine) in pancreatic adenocarcinoma cells in culture, resulting in greater decreased colony formation, inhibition of cell proliferation, and apoptotic activity (PMID: 23855452). | 23855452 | |
| 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 | Advanced Solid Tumor | not applicable | Golvatinib | Phase I | Actionable | In a Phase I trial, Golvatinib (E7050) demonstrated safety and preliminary efficacy, with stable disease as best overall response in patients with advanced solid tumors (PMID: 25278451). | 25278451 | |
| Unknown unknown | prostate cancer | not applicable | AS605240 | Preclinical | Actionable | In a preclinical study, AS605240 reduced invasiveness of prostate cancer cells in culture (PMID: 24416348). | 24416348 | |
| Unknown unknown | breast cancer | not applicable | 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 | 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 | prostate cancer | sensitive | Bicalutamide | FDA approved | Actionable | In a clinical trial that supported FDA approval, combined with a luteinizing hormone-releasing hormone analogue (LHRH-A) therapy, Casodex (bicalutamide) demonstrated efficacy similar to Eulexin (flutamide), resulted in comparable median time to progression (97 vs 77 weeks, HR=0.93, p=0.41) and survival time (180 vs 148 weeks, HR=0.87, p=0.15) in patients with metastatic prostate cancer (PMID: 9301693). | 9301693 detail... | |
| Unknown unknown | colon adenocarcinoma | not applicable | JQ1 | Preclinical | Actionable | In a preclinical study, JQ1 increased T-lymphocyte infiltration and reduced tumor growth in syngeneic mouse colon adenocarcinoma models (PMID: 31018997). | 31018997 | |
| Unknown unknown | myelofibrosis | not applicable | Fedratinib | FDA approved | Actionable | In a Phase III trial (JAKARTA) that supported FDA approval, Inrebic (fedratinib) demonstrated both clinical benefits and toxicity in myelofibrosis patients, resulting in symptom response rates at week 24 of 36% (33/91), 34% (31/91), and 7% (6/85) in the Fedratinib (SAR302503) 400 mg, 500 mg, and placebo groups, respectively (P<.001) (PMID: 26181658; NCT01437787). | 26181658 detail... | |
| Unknown unknown | lung squamous cell carcinoma | not applicable | Carboplatin + Nab-paclitaxel + Pembrolizumab | FDA approved | Actionable | In a Phase III trial (KEYNOTE-407) that supported FDA approval, Keytruda (pembrolizumab) in combination with chemotherapy consisted of carboplatin and paclitaxel or nab-paclitaxel significantly improved overall survival (15.9 vs 11.3 months), progression-free survival (6.4 vs 4.8 months), and overall response rate (58% vs 35%) compared to placebo plus chemotherapy in patients with untreated metastatic squamous non-small cell lung cancer (J Clin Oncol 36, no. 15_suppl, 105-105; NCT02775435). | detail... detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Antroquinonol | Phase I | Actionable | In a Phase I trial, antroquinonol (Hocena) demonstrated safety and preliminary efficacy in metastatic NSCLC patients previously treated with two prior chemotherapy regimens (PMID: 26807250). | 26807250 | |
| 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 | prostate cancer | not applicable | Durvalumab + Olaparib | Phase II | Actionable | In a Phase II trial, Imfinzi (durvalumab) plus Lynparza (olaparib) resulted in a decrease in PSA greater than or equal to 50% in 53% (9/17) of patients with metastatic castrate-resistant prostate cancer, with radiographic response in 4 of those 9, a median radiographic progression-free survival (PFS) of 16.1 mo., and a 12-mo. PFS probability of 83.3% in patients with mutations in DNA damage response (DDR) genes, compared to 36.4% in patients without DDR gene mutations (PMID: 30514390; NCT02484404). | 30514390 | |
| Unknown unknown | Ewing sarcoma | not applicable | Olaparib + Temozolomide | Preclinical - Cell culture | Actionable | In a preclinical study, Ewing sarcoma cells treated with Temodar (temozolomide) combined with Lynparza (olaparib) resulted in very strong synergism, inducing apoptosis and reducing cell viability in culture (PMID: 26438158). | 26438158 | |
| Unknown unknown | neuroblastoma | not applicable | Cambinol + Doxorubicin | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Adriamycin (doxorubicin) and Cambinol inhibited growth of a human neuroblastoma cell line in culture and in xenograft models (PMID: 22703804). | 22703804 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Adavosertib + Everolimus | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Afinitor (everolimus) and Adavosertib (MK-1775) resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27872098). | 27872098 | |
| Unknown unknown | myelofibrosis | not applicable | XL019 | Phase I | Actionable | In a Phase I trial, treatment with XL019 demonstrated clinical activity in 23% (7/30) of myelofibrosis patients, including 27% (6/22) of patients with JAK2 V617F and 13% (1/8) with wild-type JAK2, and resulted in responses in 10% (3/30) of patients, however, resulted in neurotoxicity and the trial was discontinued (PMID: 24374145; NCT00595829). | 24374145 | |
| Unknown unknown | 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 | 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 | renal cell carcinoma | not applicable | AGS-003 + Sunitinib | Phase II | Actionable | In a Phase II clinical trial, treatment with the combination of AGS-003 and Sutent (sunitinib) resulted in clinical benefit in 62% (13/21) of patients with advanced renal cell carcinoma, with 9 partial responses and 4 patients achieving stable disease, a median overall survival of 30.2 months, and median progression-free survival if 11.2 months (PMID: 25901286). | 25901286 | |
| Unknown unknown | breast cancer | not applicable | Cabozantinib | Preclinical | Actionable | In a preclinical study, Cometriq (cabozantinib) suppressed metastasis, angiogenesis, and tumor growth in mouse models of breast cancer (PMID: 21926191). | 21926191 | |
| Unknown unknown | 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 | non-Hodgkin lymphoma | not applicable | DS-3201b | Phase I | Actionable | In a Phase I trial, DS-3201b demonstrated preliminary clinical activity in patients with non-Hodgkin lymphoma, with an overall response rate of 53% (8/15; 1 complete response/remission, and 7 partial responses), stable disease in 5 patients, and 8 patients on treatment with tumor shrinkage for greater than 24 weeks (Blood Dec 2017, 130 (Suppl 1) 4070; NCT02732275). | detail... | |
| Unknown unknown | 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 | multiple myeloma | not applicable | Delanzomib + Tinostamustine | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Delanzomib (CEP-1877) and EDO-S101 worked synergistically to decrease viability of a multiple myeloma cell line in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | follicular lymphoma | 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 54% (39/72, 6 complete response, 33 partial response) in patients with relapsed follicular lymphoma (PMID: 24450858; NCT01282424). | 24450858 detail... | |
| Unknown unknown | glioblastoma multiforme | not applicable | Bortezomib + Panobinostat | Preclinical | Actionable | In a preclinical study, glioblastoma cells treated with a combination of Farydak (panobinostat) and Velcade (bortezomib) resulted in a synergistic effect, demonstrating decreased cell viability in culture (PMID: 26804704). | 26804704 | |
| Unknown unknown | Ewing sarcoma | not applicable | Carfilzomib + Selinexor | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Selinexor (KPT-330) and Kyprolis (carfilzomib) resulted in downregulation of Birc5 (Survivin) and enhanced induction of apoptotic markers compared to Selinexor (KPT-330) alone, and worked synergistically to decrease viability of Ewing sarcoma cells in culture (PMID: 28314790). | 28314790 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CHR-3996 | Phase I | Actionable | In a Phase I trial, the HDAC inhibitor, CHR-3996, demonstrated safety and preliminary efficacy in patients with a variety of refractory solid tumors (PMID: 22553374). | 22553374 | |
| Unknown unknown | ovarian cancer | not applicable | Cediranib + Durvalumab + Olaparib | Phase I | Actionable | In a Phase I trial, the combination of Cediranib (AZD-2171), Imfinzi (durvalumab), and Lynparza (olaparib) treatment demonstrated tolerability and activity in female patients with ovarian, endometrial, or triple-negative breast cancer, with a response rate of 33% (3/9; 2 pts with ovarian cancer, and 1 pt with endometrial cancer), and stable disease in 4/9 pts (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #390P; NCT02484404). | detail... | |
| Unknown unknown | colorectal cancer | not applicable | Vistusertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Vistusertib (AZD2014) induced apoptosis and decreased viability of colorectal cancer cell lines in culture, and inhibited tumor growth in colorectal cancer cell line xenograft models (PMID: 24309100). | 24309100 | |
| Unknown unknown | glioblastoma multiforme | not applicable | PVSRIPO | Phase I | Actionable | In a Phase I clinical trial, treatment with PVSRIPO by convection-enhanced delivery in patients with recurrent grade IV malignant glioma demonstrated safety and resulted in a median overall survival (OS) of 12.5 months, compared to 11.3 months in historical controls, and a 24-month and 36-month OS of 21% (95% CI, 11 to 33), compared to 14% and 4%, respectively in historical controls (PMID: 29943666; NCT02986178). | 29943666 | |
| Unknown unknown | chronic myeloid leukemia | not applicable | Nilotinib + Tazemetostat | Preclinical | Actionable | In a preclinical study, treatment with the combination of Tasigna (Nilotinib) and EPZ-6438 resulted in decreased levels of leukemic cells and progenitors in primary chronic myeloid leukemia cell xenograft models, with increased efficacy compared to Tasigna (Nilotinib) alone (PMID: 27630125). | 27630125 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Cytarabine + RN-1 | Preclinical - Cell culture | Actionable | In a preclinical study, RN-1 and Cytosar-U (cytarabine) demonstrated synergy in growth inhibition of several acute myeloid leukemia cell lines in culture (PMID: 26837761). | 26837761 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Bendamustine + MK2206 + Rituximab | Phase Ib/II | Actionable | In a Phase I trial, MK2206 in combination with Bendamustine and Rituximab resulted in an overall response rate of 92% (12/13), with a median progression free survival of 16 months and a treatment free survival of 24 months in patients with relapsed or refractory chronic lymphocytic leukemia (PMID: 28402581). | 28402581 | |
| Unknown unknown | sarcoma | not applicable | BI 853520 | Phase I | Actionable | In a Phase I trial, BI 853520 demonstrated safety and some anti-tumor efficacy, resulting in stable disease in 28.6% (4/14) of patients with soft tissue sarcoma, one of whom had stable disease lasting greater than 150 days (PMID: 30756308; NCT01335269). | 30756308 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BI 894999 | Phase I | Actionable | In a Phase I trial, treatment with BI 894999 resulted in stable disease in one patient and a partial response in three patients of 27 evaluable patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2504)). | detail... | |
| Unknown unknown | renal cell carcinoma | not applicable | Pegilodecakin | Phase I | Actionable | In a Phase I trial, AM0010 demonstrated safety and resulted in partial responses in 27% (4/15) of patients with renal cell carcinoma (PMID: 27528724; NCT02009449). | 27528724 | |
| Unknown unknown | 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 | multiple myeloma | not applicable | Daratumumab | FDA approved | Actionable | In a Phase I/IIb trial (GEN501) that supported FDA approval, Darzalex (daratumumab) treatment in patients with heavily pretreated and refractory multiple myeloma resulted in an overall response rate of 36% (15/42) in the 16 mg/kg cohort, with 1 complete response and 3 very good partial responses (PMID: 26308596; NCT00574288). | detail... 26308596 | |
| 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 | Capivasertib + Docetaxel + Prednisone | Phase I | Actionable | In a Phase I trial, AZZD5363 in combination with Taxotere (docetaxel) and Prednisone resulted in more than 50% PSA reduction at 12 weeks in 70% (7/10) of patients with metastatic castration resistant prostate cancer (PMID: 28144789; NCT02121639). | 28144789 | |
| Unknown unknown | gastrointestinal system cancer | not applicable | Axitinib + Fluorouracil + Leucovorin + Oxaliplatin | Phase I | Actionable | In a Phase I trial, Inlyta (axitinib), in combination with FOLFOX, demonstrated safety and some efficacy in patients with gastrointestinal tumors (PMID: 24423921). | 24423921 | |
| Unknown unknown | 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 | diffuse large B-cell lymphoma | not applicable | Abexinostat | Phase II | Actionable | In a Phase II trial, patients with diffuse large B-cell lymphoma demonstrated an overall response rate of 31% (5/16) and a median duration response of 1.9 months when treated with Abexinostat (PCI-24781) (PMID: 28126962). | 28126962 | |
| Unknown unknown | head and neck squamous cell carcinoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase III clinical trial (CheckMate 141) that supported FDA approval, treatment with Opdivo (nivolumab) resulted in a 1 year overall survival rate of 36%, compared to 18% with standard therapy, and an improved median overall survival of 7.5 months, compared to 5.1 months with standard therapy, in patients with recurrent head and neck squamous cell carcinoma (PMID: 27718784; NCT02105636). | detail... 27718784 | |
| Unknown unknown | breast cancer | not applicable | CVX-241 | Preclinical | Actionable | In a preclinical study, CVX-241 treatment resulted in improved overall survival in a breast cancer cell line xenograft model and syngeneic mouse model of breast cancer (PMID: 27651308). | 27651308 | |
| Unknown unknown | 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 | 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 | Advanced Solid Tumor | not applicable | CYT01B + Talazoparib | Preclinical - Cell culture | Actionable | In a preclinical study, CYT01B and Talzenna (talazoparib) synergistically inhibited growth of tumor cell lines in culture (AACR Annual Meeting 2019, Abstract 363). | detail... | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Ibrutinib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Imbruvica (ibrutinib) in chronic lymphoid leukemia (CLL) or small lymphocytic lymphoma (SLL) patients resulted in improved progression-free survival compared to treatment with Arzerra (ofatumumab) (median duration not reached vs. 8.1 months), and an overall response rate of 43% (83/195) versus 4% (8/196) with Arzerra (ofatumumab) (PMID: 24881631). | detail... 24881631 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Ibrutinib | Phase III | Actionable | In a Phase III clinical trial, Imbruvica (ibrutinib) treatment resulted in a greater progression-free survival, overall survival, and response rate when compared to chlorambucil treatment in chronic lymphocytic leukemia patients aged 65 or older (PMID: 26639149). | 26639149 | |
| Unknown unknown | 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 | anal canal squamous cell carcinoma | not applicable | Cetuximab + Cisplatin + Fluorouracil + Radiotherapy | Phase II | Actionable | In a Phase II trial, the combination of Erbitux (cetuximab) with Platinol (cisplatin), Adrucil (fluorouracil), and radiotherapy resulted in a locoregional failure rate of 23% (14/61) and a PFS of 68% and OS of 83% in patients with anal canal squamous cell carcinoma (PMID: 28068178). | 28068178 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Dasatinib | Phase II | Actionable | In a Phase II trial, Sprycel (dasatinib) treatment in non-small cell lung carcinoma patients showed some clinical efficacy, resulting in one patient with a partial response, and 12 patients with stable disease, demonstrating a disease control rate of 43% (13/30)(PMID: 20855820). | 20855820 | |
| Unknown unknown | breast cancer | not applicable | INCB081776 + unspecified PD-L1 antibody | Preclinical | Actionable | In a preclinical study, INCB081776 and anti-PD-L1 therapy synergistically inhibited tumor growth in a mouse model of breast cancer (Cancer Res 2018;78(13 Suppl):Abstract nr 3759). | detail... | |
| Unknown unknown | chronic myeloid leukemia | not applicable | RN-1 | Preclinical - Cell culture | Actionable | In a preclinical study, RN-1 inhibited growth of chronic myeloid leukemia cell lines in culture (PMID: 26837761). | 26837761 | |
| 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 | T-cell acute lymphoblastic leukemia | not applicable | DT2216 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, DT2216 treatment resulted in decreased BCL-XL expression and increased apoptosis and reduced viability of a T-cell acute lymphoblastic leukemia (T-ALL) cell line in culture, and decreased tumor growth in xenograft models (PMID: 31792461). | 31792461 | |
| Unknown unknown | breast cancer | not applicable | SL0101 | Preclinical - Cell culture | Actionable | In a preclinical study, SL0101 inhibited proliferation of a breast cancer cell line in culture (PMID: 15705904). | 15705904 | |
| 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 | gastroesophageal junction adenocarcinoma | no benefit | Oxaliplatin + Ramucirumab + TS-1 | Phase II | Actionable | In a Phase II trial, adding Cyramza (ramucirumab) to chemotherapy consisting of TS-1 and Eloxatin (oxaliplatin) did not improve progression-free survival (6.34 vs 6.74 months), objective response rate (58.2% vs 50.%), and disease control rate (90.9% vs 87.0%) compared to placebo in patients with advanced gastric or gastroesophageal junction adenocarcinoma (J Clin Oncol 36, 2018 (suppl; abstr 4036); NCT02539225). | detail... | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Copanlisib | Phase II | Actionable | In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 6% (1/17), partial response in 6% (1/17) and stable disease in 41% (7/17) of patients with diffuse large B-cell lymphoma (PMID: 24852792). | 24852792 | |
| Unknown unknown | cervical cancer | not applicable | ETP-46464 + Radiotherapy | Preclinical - Cell culture | Actionable | In a preclinical study, ETP-46464 increased the sensitivity of cervical cancer cell lines to ionizing radiation in culture (PMID: 25560806). | 25560806 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | PRT062607 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with PRT062607 (P505-15) resulted in decreased viability of chronic lymphocytic leukemia cells in culture and in cell line xenograft models (PMID: 23220742). | 23220742 | |
| Unknown unknown | glioblastoma multiforme | not applicable | DCVax-L | Phase III | Actionable | In a Phase III trial, addition of DCVax-L to standard therapy resulted in a median overall survival (mOS) of 23.1 months in the intended to treat group (n=331) of patients with newly diagnosed glioblastoma, with a mOS of 34.7 months in patients with methylated MGMT (n?=?131) (PMID: 29843811; NCT00045968). | 29843811 | |
| Unknown unknown | melanoma | not applicable | Indoximod + Pembrolizumab | Phase II | Actionable | In a Phase II trial, combination of Indoximod and Keytruda (pembrolizumab) resulted in an objective response rate of 55.7% (39/70, 13 complete response), with a median progression-free survival of 12.4 months in patients with advanced melanoma (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 9512-9512; NCT02073123). | detail... | |
| Unknown unknown | hairy cell leukemia | not applicable | Moxetumomab pasudotox-tdfk | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Lumoxiti (moxetumomab pasudotox-tdfk) treatment resulted in durable complete response in 30% (24/80), complete response in 41% (33/80), objective response (complete response and partial response) in 75% (60/80), and hematologic remission in 80% (64/80) of patients with relapsed or refractory hairy cell leukemia who had more than 2 prior systemic therapies (PMID: 30030507; NCT01829711). | 30030507 detail... | |
| Unknown unknown | colorectal cancer | not applicable | WAY-600 | Preclinical | Actionable | In a preclinical study, WAY-600 induced cell cycle arrest and inhibited cell proliferation of colorectal cancer cells in culture (PMID: 19584280). | 19584280 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Birinapant | Phase I | Actionable | In a Phase I dose escalation study of birinapant, 50 patients with advanced solid tumors or lymphoma were enrolled and no patients achieved complete or partial remission while stable disease was observed in 3 patients (PMID: 26333381). | 26333381 | |
| Unknown unknown | 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 | lung non-small cell carcinoma | not applicable | Ro 31-8220 | Preclinical - Cell culture | Actionable | In a preclinical study, Ro 31-8220 decreased CREB signaling, and induced apoptosis and inhibited growth of non-small cell lung cancer cells in culture (PMID: 18281471). | 18281471 | |
| Unknown unknown | 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 | prostate cancer | not applicable | AZD8186 | Phase I | Actionable | In a Phase I trial, AZD8186 demonstrated preliminary activity in patients with select solid tumors, including castration-resistant prostate cancer (CRPC), with one CRPC patient achieving stable disease and symptomatic improvement (Cancer Res August 1 2015 (75) (15 Supplement) CT329). | detail... | |
| Unknown unknown | 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 | melanoma | not applicable | Cobimetinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Cobimetinib (GDC-0973) induced cell death in several human melanoma cell lines in culture and inhibited tumor growth in xenograft models (PMID: 22084396). | 22084396 | |
| Unknown unknown | melanoma | not applicable | JSH-150 | Preclinical - Cell culture | Actionable | In a preclinical study, JSH-150 inhibited proliferation of a melanoma cell line in culture (PMID: 30253346). | 30253346 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Carboplatin + Lenvatinib + Paclitaxel | Phase I | Actionable | In a Phase I trial of patients with advanced or metastatic NSCLC, treatment with Lenvima (lenvatinib) in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) was tolerated and demonstrated anti-tumor activity (PMID: 23860537). | 23860537 | |
| Unknown unknown | melanoma | not applicable | Vandetanib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with Caprelsa (vandetanib) resulted in a significant reduction in tumor outgrowth in mouse xenograft models of melanoma (PMID: 22075979). | 22075979 | |
| Unknown unknown | acute myeloid leukemia | not applicable | ONC201 + Venetoclax | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of ONC201 and Venclexta (venetoclax) demonstrated synergy in acute myeloid leukemia cell lines in culture, resulting in increased induction of apoptosis (PMID: 26884599). | 26884599 | |
| Unknown unknown | 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 | 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 | 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 | 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 | bone cancer | not applicable | Pembrolizumab | Phase II | Actionable | In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in an overall response rate of 5% (2/40) and 12-week progression free rate of 28% (11/40) in patients with bone sarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). | detail... | |
| Unknown unknown | 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 | 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 | acute myeloid leukemia | not applicable | ST1926-NP | Preclinical - Cell line xenograft | Actionable | In a preclinical study, ST1926-NP treatment resulted in reduced cell proliferation in acute myeloid leukemia (AML) cells in culture, and decreased tumor burden and improved survival in AML cell line xenograft models (PMID: 28619754). | 28619754 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | SU14813 | Phase I | Actionable | In a Phase I trial, SU14813 demonstrated safety and preliminary efficacy in patients with advanced solid tumors, with an objective response rate of 20% (13/65), including 1 complete response and 12 partial responses (PMID: 20605934). | 20605934 | |
| Unknown unknown | neuroblastoma | not applicable | Doxorubicin + Vorinostat | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Zolinza (vorinostat) and Adriamycin (doxorubicin) inhibited growth of human neuroblastoma cells in culture and in cell line xenograft models (PMID: 22703804). | 22703804 | |
| Unknown unknown | mycosis fungoides | not applicable | Mogamulizumab | FDA approved | Actionable | In a Phase III trial (MAVORIC) that supported FDA approval, Poteligeo (mogamulizumab-kpkc) treatment resulted in significantly improved progression-free survival (7.7 vs 3.1 months, HR=0.53, p<0.0001) and objective response rate (28.0% vs 4.8%) compared to Zolinza (vorinostat) in patients with mycosis fungoides or Sézary syndrome (Blood 2017 130(Suppl 1):817). | detail... detail... | |
| Unknown unknown | colorectal cancer | not applicable | Nivolumab | FDA approved | Actionable | In a Phase II trial (CheckMate 142) that supported FDA approval, treatment with Opdivo (nivolumab) resulted in an objective response rate of 36% (19/53), with 1 complete response and 18 partial responses, and disease control for 12 weeks or more in 70% (37/53) of patients with mismatch repair deficient or microsatellite instability-high metastatic colorectal cancer (PMID: 28734759; NCT02060188). | 28734759 detail... | |
| Unknown unknown | transitional cell carcinoma | not applicable | Pembrolizumab | FDA approved | Actionable | In a Phase III (KEYNOTE-045) trial that supported FDA approval, treatment with Keytruda (pembrolizumab) resulted in improved median overall survival (10.3 vs 7.4 months, p=0.002) and objective response rate (21.1% vs 11.4%) compared to chemotherapy in patients with platinum-refractory advanced urothelial carcinoma (PMID: 28212060; NCT02256436). | detail... 28212060 | |
| Unknown unknown | 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 | head and neck squamous cell carcinoma | not applicable | GSK1059615 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, GSK1059615 treatment in a head and neck squamous cell carcinoma cell line resulted in inhibition of both cell growth and cell proliferation and induced necrosis in culture, and inhibited tumor growth in cell line xenograft models (PMID: 28187451). | 28187451 | |
| Unknown unknown | B-cell lymphoma | not applicable | SKLB-23bb | Preclinical - Cell line xenograft | Actionable | In a preclinical study, SKLB-23bb treatment inhibited tumor growth in B-cell lymphoma xenograft models (PMID: 29610282). | 29610282 | |
| Unknown unknown | 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 | colorectal adenocarcinoma | not applicable | CS-11 | Preclinical - Cell culture | Actionable | In a preclinical study, CS-11 induced cytotoxicity in a colorectal adenocarcinoma cell line in culture, and inhibited tumor growth in xenograft models (PMID: 28500231). | 28500231 | |
| Unknown unknown | breast cancer | not applicable | CS-11 | Preclinical | Actionable | In a preclinical study, CS-11 induced apoptosis and inhibited growth of breast cancer cell lines in culture, and inhibited tumor growth and metastasis in an orthotopic breast cancer mouse model (PMID: 28500231). | 28500231 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Abexinostat | Phase II | Actionable | In a Phase II clinical trial, Abexinostat treatment resulted in an overall response rate of 27.3% (3/11) mantle cell lymphoma patients with a median progression-free survival of 3.9 months (PMID: 26482040). | 26482040 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | G-TPP + Obatoclax | Preclinical - Cell culture | Actionable | In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Obatoclax (GX015-070) synergistically inhibited viability of triple-receptor negative breast cancer cells in culture (PMID: 28522750). | 28522750 | |
| Unknown unknown | uveal melanoma | no benefit | Ipilimumab | Clinical Study - Meta-analysis | Actionable | In a meta-analysis, uveal melanoma patients were not responsive to Yervoy (ipilimumab) therapy (PMID: 28881222). | 28881222 | |
| Unknown unknown | breast cancer | not applicable | F14512 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, F14512 induced tumor regression in breast cancer cell line xenograft models (PMID: 19047165). | 19047165 | |
| Unknown unknown | 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 | chronic lymphocytic leukemia | not applicable | Bendamustine + Idelalisib + Rituximab | Phase III | Actionable | In a Phase III trial, addition of Zydelig (idelalisib) to Bendamustine and Rituximab resulted in improved median progression-free survival (20.8 vs 11.1 months, HR=0.33, p<0.0001) compared to placebo in patients with relapsed or refractory chronic lymphocytic leukemia, although treatment associated adverse events were also increased (PMID: 28139405). | 28139405 | |
| Unknown unknown | lung adenocarcinoma | not applicable | JNJ-64041757 | Phase I | Actionable | In a Phase I trial, JNJ-64041757 treatment demonstrated safety in patients with lung adenocarcinoma, and resulted in mesothelin-specific immune responses in several patients and stable disease as best response (J Thoracic Oncol, Vol 12, Issue 11, S2151). | detail... | |
| Unknown unknown | 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 | 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 | non-Hodgkin lymphoma | not applicable | Bendamustine + Rituximab + Veliparib | Phase I | Actionable | In a Phase I trial, seven patients with non-Hodgkin lymphoma treated with a combination of Veliparib (ABT-888), Bendamustine, and Rituxan (rituximab) demonstrated an overall response rate of 86% (6/7) and a complete response rate of 71% (5/7), and a progression free survival of 14.2 months (PMID: 28314788; NCT01326702). | 28314788 | |
| Unknown unknown | neuroendocrine tumor | no benefit | Everolimus + Pasireotide | Phase II | Actionable | In a Phase II trial, addition of Pasireotide to Afinitor (everolimus) did not significantly affect progression free survival (16.8 vs 16.6 months) or overall disease control rate (77.2% vs 82.7%) in patients with well-differentiated, progressive pancreatic neuroendocrine tumors (PMID: 28327907). | 28327907 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Paclitaxel + Pazopanib | Phase I | Actionable | In a Phase I trial, the combination of Votrient (pazopanib) and Taxol (paclitaxel) demonstrated safety and resulted in partial response in 36% (10/28) and stable disease in 36% (10/28) of patients with advanced solid tumors (PMID: 25504632). | 25504632 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Conatumumab + Ganitumab | Phase Ib/II | Actionable | In a Phase Ib/II clinical trial, Ganitumab and Conatumumab combination treatment resulted in stable disease in 50% (11/22) of patients with non-small cell lung carcinoma (PMID: 24816908). | 24816908 | |
| Unknown unknown | 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 | diffuse large B-cell lymphoma | not applicable | GSK3368715 | Preclinical - Patient cell culture | Actionable | In a preclinical study, GSK3368715 treatment inhibited growth of a diffuse large B-cell lymphoma (DLBCL) cell line in culture and inhibited tumor growth in xenograft models, and inhibited growth of patient-derived DLBCL cells in culture PMID: 31257072). | 31257072 | |
| Unknown unknown | 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 | 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 | 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 | cervical cancer | not applicable | BIRB-796 + Tozasertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Tozasertib (VX-680) and BIRB-796 resulted in increased growth inhibition and induction of apoptosis in cervical cancer cell lines in culture, and increased tumor growth inhibition in cervical cancer cell line xenograft models, compared to either agent alone (PMID: 27082306). | 27082306 | |
| Unknown unknown | osteosarcoma | not applicable | Dasatinib | Phase II | Actionable | In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in osteosarcoma patients (n=46) was suspended due to lack of drug activity (PMID: 26710211). | 26710211 | |
| Unknown unknown | thyroid gland papillary carcinoma | not applicable | Ramucirumab | Phase I | Actionable | In a Phase I trial, Cyramza (ramucirumab) demonstrated safety and efficacy resulting in partial response or stable disease in patients with advanced solid tumors, including papillary thyroid carcinoma (PMID: 20048182). | 20048182 | |
| Unknown unknown | gastrointestinal system cancer | not applicable | Cabozantinib | Preclinical - Pdx | Actionable | In a preclinical study, Cometriq (cabozantinib) demonstrated efficacy in colorectal cancer patient-derived tumor explant models (Cancer Research: April 15, 2013; Volume 73, Issue 8, Supplement 1). | detail... | |
| Unknown unknown | Advanced Solid Tumor | no benefit | GGTI-2418 | Phase I | Actionable | In a Phase I trial, GGTI-2418 demonstrated safety, but did not reach optimal target inhibition due to rapid elimination, and resulted in stable disease as best response in 31% (4/13) of patients with advanced solid tumors (PMID: 31372813). | 31372813 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | AMG208 | Phase I | Actionable | In a Phase I trial, AMG208 demonstrated safety and preliminary efficacy, resulted in complete response in 2% (1/43), partial response in 7% (3/43) and stable disease in 65% (28/43) of patients with advanced solid tumors (PMID: 26155941; NCT00813384). | 26155941 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | IHSF115 | Preclinical - Cell culture | Actionable | In a preclinical study, IHSF115 inhibited growth of a panel of cancer cell lines in culture (PMID: 28369544). | 28369544 | |
| Unknown unknown | 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 | 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 | neuroblastoma | not applicable | ABT-751 + Fenretinide | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Fenretinide and ABT-751 synergistically inhibited growth of neuroblastoma cell lines in culture and in cell line xenograft models (PMID: 27530131). | 27530131 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | BEZ235 + Everolimus | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of BEZ235 and Afinitor (everolimus) resulted in limited efficacy in patients with advanced solid tumors and was discontinued due to multiple intolerable side effects (PMID: 28357727). | 28357727 | |
| Unknown unknown | 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 | acute myeloid leukemia | not applicable | Forskolin | Preclinical | Actionable | In a preclinical study, Forskolin inhibited cell proliferation and induced apoptosis in acute myeloid leukemia cells in culture (PMID: 21233840). | 21233840 | |
| Unknown unknown | multiple myeloma | not applicable | Ricolinostat + SJB3-019A | Preclinical - Patient cell culture | Actionable | In a preclinical study, the combination of SJB3-019A and Ricolinostat (ACY-1215) worked synergistically to induce cytotoxicity in multiple myeloma cell lines and primary multiple myeloma cells in culture (PMID: 28270494). | 28270494 | |
| Unknown unknown | glioblastoma multiforme | not applicable | Marizomib + Panobinostat | Preclinical | Actionable | In a preclinical study, glioblastoma cells treated with a combination of Farydak (panobinostat) and Marizomib resulted in a synergistic effect, demonstrating decreased cell viability in culture (PMID: 26804704). | 26804704 | |
| Unknown unknown | Her2-receptor negative breast cancer | not applicable | Sorafenib | Clinical Study | Actionable | In a meta-analysis of 844 ERBB2 (HER2)-negative breast cancer patients, Nexavar (sorafenib) increased progression-free survival time, but not overall survival or objective response rate (PMID: 24940450). | 24940450 | |
| Unknown unknown | alveolar soft part sarcoma | not applicable | Durvalumab + Tremelimumab | Clinical Study | Actionable | In a clinical case study, a patient with alveolar soft part sarcoma had a 73% tumor reduction and a response lasting greater than 30 months when treated with Imfinzi (durvalumab) and Tremelimumab, and the tumor was shown retrospectively to have a mismatch repair defect signature, poor immune infiltration, and 2% tumoral PD-L1 positivity (PMID: 30018044; NCT02261220). | 30018044 | |
| Unknown unknown | leukemia | not applicable | P10 + Vorinostat | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of P10 and Zolinza (vorinostat) resulted in enhanced DNA damage leading to increased apoptotic activity and cell-cycle arrest of leukemic cells in culture (PMID: 27188390). | 27188390 | |
| Unknown unknown | angiosarcoma | not applicable | Alisertib | Phase II | Actionable | In a Phase II trial, Alisertib (MLN8237) treatment resulted in partial response in 2 angiosarcoma patients (PMID: 27502708). | 27502708 | |
| Unknown unknown | 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 | Advanced Solid Tumor | no benefit | SAR260301 | Phase I | Actionable | In a Phase I trial, SAR260301 treatment in advanced solid tumor patients failed to inhibit the PI3K pathway due to rapid clearance, and therefore, was not recommended for further clinical analysis (PMID: 28976556; NCT01673737). | 28976556 detail... | |
| Unknown unknown | multiple myeloma | not applicable | Torkinib | Preclinical - Patient cell culture | Actionable | In a preclinical study, Torkinib (PP242) treatment resulted in decreased mTORC2 signaling, growth inhibition and apoptosis in multiple myeloma cell lines and patient-derived multiple myeloma cells in culture, and reduced tumor growth in cell line xenograft animal models (PMID: 20686120). | 20686120 | |
| Unknown unknown | stomach cancer | no benefit | Everolimus | Phase III | Actionable | In a Phase III trial, Afinitor (everolimus) did not significantly improve overall survival (5.4 vs 4.3 months) and progression free survival (1.7 vs 1.4 months) over placebo in previously treated advanced gastric cancer patients (PMID: 24043745). | 24043745 | |
| Unknown unknown | 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 | brain stem glioma | not applicable | MRK-003 | Preclinical - Cell culture | Actionable | In a preclinical study, MRK-003 increased apoptosis and decreased growth of diffuse pontine glioma cell lines in culture (PMID: 26115193). | 26115193 | |
| Unknown unknown | chronic myeloid leukemia | not applicable | Nilotinib | Clinical Study | Actionable | In a meta-analysis, Tasigna (nilotinib) treatment was associated with increased rate of major molecular response compared with Gleevec (imatinib) (Odds Ratio (OR): 2.45 [1.85-3.24]), but not improved overall survival (OR: 1.51 [0.38-5.99]), and was associated with increased risk of vascular occlusive events (OR: 3.42 [2.07-5.63]) in patients with chronic myeloid leukemia (PMID: 26847662). | 26847662 | |
| Unknown unknown | chronic myeloid leukemia | not applicable | Nilotinib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Tasigna (nilotinib) resulted in improved hematologic response and overall survival rates compared to treatment with Gleevec (imatinib) in patients with Philadelphia chromosome positive chronic myeloid leukemia (PMID: 21091142). | 21091142 detail... | |
| 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 | prostate cancer | not applicable | Sapanisertib | Phase II | Actionable | In a Phase II trial, Sapanisertib (MLN0128) treatment demonstrated limited efficacy, with a median time on treatment of 11 weeks and stable disease as best response (PMID: 29508246). | 29508246 | |
| Unknown unknown | prostate cancer | not applicable | Sapanisertib | Preclinical | Actionable | In a preclinical study, Sapanisertib (MLN0128) inhibited cell proliferation, induced apoptosis, and prevented metastasis in xenograft models with prostate cancer (PMID: 22367541). | 22367541 | |
| Unknown unknown | multiple myeloma | not applicable | Bortezomib + Torkinib | Preclinical - Cell culture | Actionable | In a preclinical study, Torkinib (PP242) worked synergistically with Velcade (Bortezomib) to induce apoptosis in multiple myoloma cell lines in culture (PMID: 20686120). | 20686120 | |
| Unknown unknown | renal cell carcinoma | not applicable | Ipilimumab + Nivolumab | Phase I | Actionable | In a Phase I trial, the combination of Opdivo (nivolumab) and Yervoy (ipilimumab) demonstrated safety using 2 different dosing regimens (N3I1=nivolumab 3mg/kg+ipilumumab 1mg/kg; N1I3=nivolumab 1mg/kg+ipilumumab 3mg/kg) in patients with metastatic renal cell carcinoma, and resulted in an objective response rate of 40.4% (19/47) in both N3I1 and N1I3 arms and a 2-year overall survival of 67% in the N3I1 arm and 70% in the N1I3 arm (PMID: 28678668; NCT01472081). | 28678668 | |
| Unknown unknown | renal cell carcinoma | not applicable | Ipilimumab + Nivolumab | FDA approved | Actionable | In a Phase III trial (CheckMate 214) that supported FDA approval, the combination of Opdivo (nivolumab) and Yervoy (ipilimumab) resulted in improved median overall survival (not reached vs. 26.0 months), objective response rate (42%, 40 complete responses (CR), vs. 27%, 5 CR), and progression-free survival (11.6 vs 8.4 months) compared to Sutent (sunitinib) in patients with intermediate or poor risk renal cell carcinoma (PMID: 29562145; NCT02231749). | 29562145 detail... detail... | |
| Unknown unknown | extraosseous Ewing sarcoma | not applicable | GSK1838705A | Preclinical | Actionable | In a preclinical study, multiple cancer cell lines including multiple myeloma and Ewing's sarcoma were sensitive to GSK1838705A (PMID: 19825801). | 19825801 | |
| Unknown unknown | 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 | Hodgkin's lymphoma | not applicable | Idelalisib | Phase II | Actionable | In a Phase II trial, Idelalisib treatment resulted in an overall response rate of 20% (5/25) in Hodgkin's lymphoma patients, with one patient experiencing a complete response and four patients experiencing a partial response (PMID: 28327905). | 28327905 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | CCT007093 + Paclitaxel | Preclinical | Actionable | In a preclinical study, CCT007093 and Taxol (paclitaxel) worked synergistically to inhibit growth of triple-receptor negative breast cancer cell lines in culture (PMID: 20576088). | 20576088 | |
| Unknown unknown | stomach cancer | not applicable | Capecitabine + Nivolumab + Oxaliplatin + TS-1 | Phase II | Actionable | In a Phase II trial (ATTRACTION-4), the combination therapy of Xeloda (capecitabine), Opdivo (nivolumab), Eloxatin (oxaliplatin), and TS-1 (tegafur-gimeracil-oteracil potassium) was well-tolerated and resulted in an objective response rate of 76.5% (13/17), a median progression-free survival of 10.6 months, and a median overall survival that was not yet reached in patients with either gastric cancer or gastroesophageal junction cancer (PMID: 30566590; NCT02746796). | 30566590 | |
| Unknown unknown | cutaneous T cell lymphoma | not applicable | Mogamulizumab | Phase III | Actionable | In a Phase III trial, Mogamulizumab treatment resulted in significant improvement in progression-free survival (7.7 vs 3.1 months, HR=0.53) and overall response rate (28.0% vs 4.8%) compared to Zolinza (vorinostat) in patients with cutaneous T cell lymphoma (Blood 2017 130(Suppl 1):817; NCT01728805). | detail... | |
| Unknown unknown | paraganglioma | not applicable | 131I-MIBG | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Azedra (iobenguane I 131) treatment resulted in partial response in 23% (15/68) of patients with pheochromocytoma or paraganglioma who received 1 therapeutic dose, with a 12-month overall survival rate of 91% (J Clin Oncol 36, 2018 (suppl; abstr 4005); NCT00874614). | detail... detail... | |
| Unknown unknown | melanoma | not applicable | NMS-P715 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, NMS-P715 inhibited tumor growth in melanoma cell line xenograft models (PMID: 21159646). | 21159646 | |
| Unknown unknown | head and neck cancer | not applicable | Cisplatin + GL-ONC1 + Radiotherapy | Phase I | Actionable | In a Phase I trial, the combination therapy of GL-ONC1, Platinol (cisplatin), and radiotherapy resulted in a progression-free survival of 74.4% at 1 year and 64.1% at 2 years and an overall survival of 84.6% at 1 year and 69.2% at 2 years in patients with nonmetastatic head and neck cancer (PMID: 28679776). | 28679776 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | NP-004255 | Preclinical - Cell culture | Emerging | In a preclinical study, a biochemical assay with NP-004255, a RAD52 inhibitor similar to other compounds that induced cell death in cells with BRCA2 loss, demonstrated inhibition of RAD52 binding to ssDNA, suggesting NP-004255 may be a potential therapeutic target (PMID: 27434671). | 27434671 | |
| Unknown unknown | 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 | ovarian cancer | not applicable | AZD7648 + Pegylated liposomal-doxorubicin | Preclinical - Cell culture | Actionable | In a preclinical study, AZD7648 and Doxil (pegylated liposomal-doxorubicin) combination treatment induced DNA damage and synergistically inhibited viability of ovarian cancer cell lines in culture (PMID: 31699977). | 31699977 | |
| 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 | thymic carcinoma | not applicable | Bevacizumab + MINT1526A | Phase I | Actionable | In a Phase I trial, MINT1526A in combination with Avastin (bevacizumab) resulted in partial response in a patient with thymic carcinoma (PMID: 29905898). | 29905898 | |
| Unknown unknown | gastroesophageal junction adenocarcinoma | not applicable | MCLA-128 | Phase Ib/II | Actionable | In a Phase I/II trial, MCLA-128 resulted in stable disease for 5 months in a patient with gastroesophageal junction cancer (Cancer Res 2016;76(14 Suppl):Abstract nr CT050). | detail... | |
| Unknown unknown | chondrosarcoma | not applicable | Dasatinib | Phase II | Actionable | In a Phase II trial, patients with chondrosarcoma demonstrated a median progression free survival of 5.5 months and six patients demonstrated an objective tumor response when treated with Sprycel (dasatinib) (PMID: 27696380). | 27696380 | |
| 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 | lung non-small cell carcinoma | not applicable | Metformin | Preclinical - Cell culture | Actionable | In a preclinical study, Glucophage (metformin) inhibited proliferation and induced cell-cycle arrest and apoptosis in non-small cell lung cancer cell lines in culture, independent of STK11 (LKB1) status (PMID: 26847819). | 26847819 | |
| Unknown unknown | glioblastoma multiforme | not applicable | SL-701 | Phase II | Actionable | In a Phase II trial, SL-701 treatment resulted in partial response in 3.3% (1/30) and stable disease in 50% (15/30) of patients with recurrant glioblastoma multiforme (Neuro Oncol (2016) 18 (suppl 6): vi20.). | detail... | |
| Unknown unknown | glioblastoma multiforme | not applicable | SL-701 | Phase II | Actionable | In a Phase II trial, SL-701 treatment resulted in partial response in 2.2% (1/46) and stable disease in 32.6% (15/46) of patients with relapsed/refractory glioblastoma, with a 12-month overall survival rate of 37% (median 11 months) (J Clin Oncol 36, 2018 (suppl; abstr 2058); NCT02078648). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | CPX-351 | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Vyxeos (CPX-351) treatment resulted in improved overall survival (9.56 vs 5.95 months, HR=0.69, p=0.005), event-free survival (HR=0.74, p=0.021), and complete response rate (47.4% vs 33.3%, p=0.016) compared to Cytosar-U (cytarabine) and Cerubidine (daunorubicin) combination therapy in patients with acute myeloid leukemia (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 7000-7000; NCT01696084). | detail... detail... | |
| Unknown unknown | 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 | multiple myeloma | not applicable | KW-2478 | Phase I | Actionable | In a Phase I clinical trial, KW-2478 demonstrated safety and preliminary efficacy in patients with multiple myeloma, with 95.2% (20/21) of patients achieving stable disease (PMID: 26695442). | 26695442 | |
| Unknown unknown | 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 | melanoma | not applicable | AT13148 | Preclinical | Actionable | In a preclinical study, AT13148 inhibited human melanoma cell motility in invasion assays in culture (PMID: 25840982). | 25840982 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | GS-5829 + Venetoclax | Preclinical - Cell culture | Actionable | In a preclinical study, diffuse large B-cell lymphoma cell lines demonstrated greater cell growth inhibition when treated with a combination of GS-5829 and Venclexta (venetoclax) compared to either agent alone in culture (Blood 2016 128:5104). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CI-1040 | Phase I | Actionable | In a Phase I trial, CI-1040 treatment resulted in median stable disease for 5.5 months in 28% (19/66) of patients with advanced solid tumors (including colorectal, non-small-cell lung, pancreatic, melanoma, and kidney) and partial response in 1 pancreatic cancer patient (PMID: 16009947). | 16009947 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Navicixizumab | Phase I | Actionable | In a Phase I trial, Navicixizumab (OMP-305B83) treatment resulted in partial response in 6% (4/66) and stable disease in 26% (17/66) of patients with advanced solid tumors, with 19 patients having a reduction of target lesions (PMID: 30229512). | 30229512 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Navicixizumab | Phase I | Actionable | In a Phase I trial, Navicixizumab (OMP-305B83) demonstrated safety and preliminary efficacy in a variety of advanced solid tumor patients, including ovarian, endometrial, breast, and pancreatic (Eur J Can, Vol 69, S35). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Navicixizumab | Preclinical | Actionable | In a preclinical study, Navicixizumab (OMP-305B83) inhibited cell proliferation of endothelial cells in culture and demonstrated antitumor activity of multiple tumor types in vivo (Mol Cancer Ther December 2015 14; C164). | detail... | |
| Unknown unknown | skin carcinoma | not applicable | CVX-241 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CVX-241 inhibited tumor growth in a skin carcinoma cell line xenograft model (PMID: 21149738). | 21149738 | |
| Unknown unknown | neuroendocrine tumor | not applicable | Everolimus + Octreotide | Phase III | Actionable | In a Phase III trial, addition of Afinitor (everolimus) to Octreotide did not significantly improve median overall survival (29.2 vs 35.2 months) compared to placebo in patients with advanced neuroendocrine tumors associated with carcinoid syndrome (PMID: 28444114). | 28444114 | |
| Unknown unknown | acute myeloid leukemia | not applicable | JSH-150 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, JSH-150 induced cell-cycle arrest and inhibited proliferation of acute myeloid leukemia cell lines in culture, and inhibited tumor progression in xenograft models (PMID: 30253346). | 30253346 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Sapanisertib | Phase I | Actionable | In a Phase I trial, Sapanisertib (MLN0128) demonstrated safety and some efficacy in patients with advanced solid tumors (Mol Cancer Ther 2013;12(11 Suppl):C252). | 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 | acute myeloid leukemia | not applicable | Birabresib | Phase I | Actionable | In a Phase I trial, Birabresib (OTX015) treatment resulted in complete remission lasting 2-5 months in 8% (3/36) and partial blast clearance in 6% (2/36) of acute myeloid leukaemia patients (PMID: 27063977). | 27063977 | |
| Unknown unknown | mantle cell lymphoma | not applicable | ONO-4059 | Phase I | Actionable | In a Phase I clinical trial, treatment with ONO-4059 was well tolerated and resulted in responses in 92% (11/12) of patients with mantle cell lymphoma (PMID: 26542378). | 26542378 | |
| Unknown unknown | cervix carcinoma | not applicable | Bevacizumab + Paclitaxel + Topotecan | FDA approved | Actionable | In a Phase III trial that supported FDA approval, the addition of Avastin (bevacizumab) to Hycamtin (topotecan) and Taxol (paclitaxel) chemotherapy resulted in improved overall survival and progression-free survival compared to chemotherapy alone in patients with cervical cancer (PMID: 25281440, PMID: 24552320). | 25281440 24552320 detail... | |
| Unknown unknown | pancreatic cancer | not applicable | SF2523 | Preclinical | Actionable | In a preclinical study, SF2523 decreased tumor immunosuppression and inhibited tumor growth in orthotopic mouse pancreatic cancer models (PMID: 31018997). | 31018997 | |
| Unknown unknown | 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 | lung non-small cell carcinoma | not applicable | PF-03446962 | Phase I | Actionable | In a Phase I trial, a patient with non-small cell lung carcinoma demonstrated a partial response for 308 days when treated with PF-03446962 (PMID: 26655846). | 26655846 | |
| Unknown unknown | melanoma | not applicable | JQ1 | Preclinical | Actionable | In a preclinical study, JQ1 reduced tumor growth in syngeneic mouse melanoma models (PMID: 31018997). | 31018997 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | BPM 31510 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TNBC cell lines demonstrated decreased cell viability and increased apoptotic activity in culture when treated with BPM 31510, and in cell line xenograft models, treatment resulted in reduced tumor size (Cancer Res 2016;76(14 Suppl):Abstract nr 208). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Vorolanib | Phase I | Actionable | In a Phase I clinical trial, X-82 was tolerated and demonstrated preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 30, 2012 (suppl; abstr 3041)). | detail... | |
| Unknown unknown | breast cancer | not applicable | AIM-100 | Preclinical | Actionable | In a preclinical study, AIM-100 inhibited growth of breast cancer cells in culture (PMID: 22322295). | 22322295 | |
| Unknown unknown | 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 | rhabdomyosarcoma | not applicable | AZD8055 + Selumetinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Selumetinib (AZD6244) and AZD8055 worked synergistically to inhibit tumor growth in xenograft models of rhabdomyosarcoma (PMID: 23918606). | 23918606 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Epacadostat | Phase I | Actionable | In a Phase I trial, treatment with Epacadostat resulted in stable disease in 13% (7/52) of patients with advanced solid tumors for greater than or equal to 16 weeks, including two patients with melanoma who previously failed on immunotherapy (PMID: 28053021). | 28053021 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | SY-1365 | Preclinical - Cell culture | Actionable | In a preclinical study, SY-1365 induced rapid apoptosis in a panel of solid tumor cell lines in culture, including breast, ovarian, colorectal, and lung cancer cells (Proceedings of the AACR, Vol 58, April 2017, Abstract # 1151). | detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | GNE-272 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with GNE-272 in acute myeloid leukemia xenograft models resulted in tumor growth inhibition at all doses, and at the highest dose led to a complete response in one of the eight tested mouse models (PMID: 27682507). | 27682507 | |
| Unknown unknown | glioblastoma multiforme | not applicable | Ad-RTS-IL-12 plus AL | Phase I | Actionable | In a Phase I trial, Ad-RTS-IL-12 plus AL demonstrated safety and preliminary efficacy in glioblastoma multiforme patients, with 100% (7/7) of patients remained alive and 5 patients having a follow-up of more than 90 days posttreatment (J Clin Oncol 34, 2016 (suppl; abstr 2052)). | detail... | |
| Unknown unknown | gastrointestinal stromal tumor | not applicable | Dovitinib | Phase II | Actionable | In a Phase II clinical trial, Dovitinib (TKI258) demonstrated safety and efficacy in heavily pretreated patients with advanced GISTs (PMID: 24084771). | 24084771 | |
| Unknown unknown | chronic myeloid leukemia | not applicable | ST7612AA1 | Preclinical | Actionable | In a preclinical study, ST7612AA1 inhibited proliferation of chronic myeloid leukemia cells in culture (PMID: 25671299). | 25671299 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | GSK3203591 + GSK3368715 | Preclinical - Cell culture | Actionable | In a preclinical study, GSK3203591 and GSK3368715 worked synergistically to inhibit viability of diffuse large B-cell lymphoma cell lines in culture (PMID: 31257072). | 31257072 | |
| Unknown unknown | colorectal cancer | not applicable | Abemaciclib | Phase I | Actionable | In a Phase I trial, treatment with Abemaciclib (LY2835219) in colorectal cancer patients resulted in 2 patients (13%; 2/15) with stable disease (PMID: 27217383). | 27217383 | |
| Unknown unknown | colon cancer | not applicable | Quisinostat | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Quisinostat (JNJ-26481585) induced apoptosis and inhibited proliferation of colon cancer cell lines in culture, and inhibited tumor growth in a colon cancer cell line xenograft model (PMID: 19861438). | 19861438 | |
| Unknown unknown | nasopharynx carcinoma | not applicable | Camrelizumab | Phase I | Actionable | In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity, resulted in partial response in 33.3% (1/3) of patients with nasopharynx cancer (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). | detail... | |
| Unknown unknown | nasopharynx carcinoma | not applicable | Camrelizumab | Phase I | Actionable | In a Phase I trial, Camrelizumab (SHR-1210) treatment was well tolerated in patients with refractory nasopharyngeal carcinoma, and demonstrated preliminary efficacy with an overall response rate of 34% (31/91, 2 complete responses and 29 partial responses) and a disease control rate of 59% (54/91, stable disease or better) (PMID: 30213452; NCT02721589). | 30213452 | |
| Unknown unknown | melanoma | not applicable | Axitinib | Phase II | Actionable | In a Phase II study in patients with metastatic melanoma, Inlyta (axitinib) was well tolerated and demonstrated antitumor activity (PMID: 21976544). | 21976544 | |
| 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 | melanoma | not applicable | Lenvatinib + Pembrolizumab | Phase Ib/II | Actionable | Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 48% (10/21) in patients with metastatic melanoma, with a median duration of response of 12.5 months, and a median progression-free survival of 5.5 months (PMID: 31961766; NCT02501096). | 31961766 | |
| Unknown unknown | colon cancer | not applicable | MC180295 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, MC180295 decreased growth of colon cancer cell lines in culture, and reduced tumor growth rate and improved survival in colon cancer cell line xenograft models (PMID: 30454645). | 30454645 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | LY2109761 + Paclitaxel | Preclinical | Actionable | In a preclinical study, LY2109761 and Taxol (paclitaxel) worked synergistically to inhibit the growth of triple-receptor negative breast cancer cell lines in culture (PMID: 20576088). | 20576088 | |
| Unknown unknown | 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 | 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 | lung non-small cell carcinoma | not applicable | CCT244747 + Gemcitabine | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of CCT244747 and Gemzar (gemcitabine) slowed tumor growth in non-small cell lung cancer cell line xenograft models (PMID: 22929806). | 22929806 | |
| Unknown unknown | multiple myeloma | not applicable | Daratumumab + Dexamethasone + Pomalidomide | FDA approved | Actionable | In a Phase I trial (EQUULEUS) that supported FDA approval, Darzalex (Daratumumab) in combination with Pomalyst (pomalidomide) and Adexone (dexamethasone) resulted in an objective response rate of 60% (61/103) in patients with relapsed or refractory multiple myeloma, with a median progression-free survival of 8.8 months and a median overall survival of 17.5 months (PMID: 28637662; NCT01998971). | detail... 28637662 | |
| Unknown unknown | transitional cell carcinoma | not applicable | Everolimus | Phase II | Actionable | In a Phase II trial, treatment with Afinitor (everolimus) in patients with transitional cell carcinoma resulted in 2 patients with a partial response, 8 patients with stable disease, and 27 patients with progressive disease, and resulted in a median progression free survival of 61 days and a median overall survival of 101 days (PMID: 22473592). | 22473592 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | MLN4924 | Phase I | Actionable | In a Phase I trial, Pevonedistat (MLN4924) treatment resulted in stable disease in 74% (23/31) of patients with advanced solid tumors (PMID: 26423795). | 26423795 | |
| Unknown unknown | pancreatic ductal adenocarcinoma | not applicable | LMB-100 + Nab-paclitaxel | Phase Ib/II | Actionable | In a Phase I/II trial, the combination of LMB-100 and Abraxane (nab-paclitaxel) led to a greater than 50% decrease in CA19-9 in 41% (7/17) of patients with pancreatic ductal adenocarcinoma, and one patient experienced an objective partial response, however, the combination therapy was not well tolerated (PMID: 31792036; NCT02810418). | 31792036 | |
| Unknown unknown | 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 | Her2-receptor negative breast cancer | not applicable | Capivasertib + Paclitaxel | Phase II | Actionable | In a Phase II trial, addition of AZD5363 to Taxol (paclitaxel) did not improve progression free survival compared to placebo (10.9 vs 8.4 months) in patients with Esr1-positive, Erbb2 (Her2)-negative breast cancer (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 241PD; NCT01625286). | detail... | |
| Unknown unknown | 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 | colon cancer | not applicable | Ensituximab | Phase I | Actionable | In a Phase I trial, Ensituximab (NEO-102) demonstrated safety and preliminary efficacy, resulting in stable disease in 42% (5/12) of patients with refractory colon or pancreatic cancer (PMID: 27449137; NCT01040000). | 27449137 | |
| Unknown unknown | 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 | 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 | ovarian cancer | not applicable | Itraconazole | Phase I | Actionable | In a Phase I clinical trial, Itraconazole, in combination with chemotherapy, demonstrated safety and efficacy in patients with ovarian cancer (PMID: 24778064). | 24778064 | |
| 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 | Advanced Solid Tumor | not applicable | ETC-159 | Phase I | Actionable | In a Phase I trial, treatment with ETC-159 was well-tolerated, decreased Wnt signaling, and resulted in stable disease in 2 out of 16 treated patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2584)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | F14512 | Preclinical | Actionable | In a preclinical study, F14512 inhibited growth of a variety of human solid tumor cell lines in culture (PMID: 19047165). | 19047165 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Navitoclax + TAK-901 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of TAK-901 and Navitoclax (ABT-263) resulted in a synergistic effect, demonstrating reduced cell viability of non-small cell lung cancer cells in culture (PMID: 28179288). | 28179288 | |
| Unknown unknown | melanoma | not applicable | MBG453 + Spartalizumab | Case Reports/Case Series | Actionable | In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in stable disease in 5 patients with melanoma, including cutaneous melanoma (n=2), uveal melanoma (n=2), and non-cutaneous melanoma (n=1) (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). | detail... | |
| Unknown unknown | prostate cancer | not applicable | C6-ceramide | Preclinical | Actionable | In a preclinical study, C6-ceramide treatment of prostate cancer cells prevented the association between SET and PP2A, which resulted in cell death in culture (PMID: 24025258). | 24025258 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Pegilodecakin + Pembrolizumab | Phase I | Actionable | In a Phase I trial, Pegilodecakin (AM0010) and Keytruda (pembrolizumab) (n=5) or Opdivo (nivolumab) (n=29) combination therapy resulted in an objective response rate of 41% (11/26, 11 partial response) and stable disease in 46% (12/26) of evaluable patients with non-small cell lung carcinoma, median progression-free survival and overall survival was 10.9 and 32.2 months in patients received Pegilodecakin and Keytruda, 4 of 4 tested had PD-L1 < 1% (J Clin Oncol 36, 2018 (suppl; abstr 9018); NCT02009449). | 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 | colorectal cancer | not applicable | Olaparib + Temozolomide | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the addition of Lynparza (olaparib) to Temodar (temozolomide) resulted in greater antitumor activity than Temodar (temozolomide) alone in colorectal cancer cell line xenograft models, demonstrating decreased tumor volume (PMID: 27550455). | 27550455 | |
| Unknown unknown | cervical cancer | not applicable | Mps-BAY2b + Paclitaxel | Preclinical | Actionable | In a preclinical study, Mps-BAY2b, in combination with paclitaxel, had increased efficacy in decreasing tumor volume of cervical carcinoma xenografts (PMID: 23933817). | 23933817 | |
| 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 | colon cancer | not applicable | IT-141 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, colon cancer cell line xenograft models demonstrated tumor regression when treated with IT-141 (PMID: 22187652). | 22187652 | |
| 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 | head and neck squamous cell carcinoma | not applicable | Abemaciclib + Everolimus | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Abemaciclib (LY2835219) and Afinitor (everolimus) worked synergistically to reduce viability of head and neck squamous cell carcinoma (HNSCC) cells in culture, and to inhibit tumor growth in HNSCC cell line xenograft models, with increased efficacy over either agent alone (PMID: 26909611). | 26909611 | |
| Unknown unknown | ovarian cancer | not applicable | Niraparib | FDA approved | Actionable | In a Phase III trial (NOVA) that supported FDA approval, maintenance therapy with Zejula (niraparib) improved median progression-free survival compared to placebo in patients with platinum-sensitive, recurrent ovarian cancer harboring germline BRCA mutations (21.0 vs. 5.5 mo., HR=0.27) and patients without germline BRCA mutations (9.3 vs. 3.9 mo, HR=0.45) (PMID: 27717299; NCT01847274). | 27717299 detail... | |
| Unknown unknown | ovarian cancer | not applicable | Niraparib | Phase I | Actionable | In a Phase I clinical trial, Zejula (niraparib) demonstrated safety and preliminary efficacy, resulted in a durable partial response (PR) in 67% (2/3) of patients with plantinum-sensitive high-grade serous ovarian cancer, PR in 16% (3/19) and stable disease over 120 days in 16% (3/19) of patients with plantinum-resistant high-grade serous ovarian cancer (PMID: 23810788; NCT00749502) | 23810788 | |
| Unknown unknown | 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 | 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 | Advanced Solid Tumor | not applicable | Midostaurin | Phase I | Actionable | In a Phase I trial, Rydapt (midostaurin) demonstrated safety in patients with advanced solid tumors (PMID: 11230495). | 11230495 | |
| Unknown unknown | gastroesophageal junction adenocarcinoma | no benefit | Ipilimumab | Phase II | Actionable | In a Phase II trial, Yervoy (ipilimumab) did not improve immune-related progression-free survival (2.9 vs 4.9 months) compared to best supportive care in patients with unresectable, locally advanced/metastatic gastric or gastroesophageal junction cancer (J Clin Oncol 34, 2016 (suppl; abstr 4011)). | detail... | |
| Unknown unknown | 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 | estrogen-receptor positive breast cancer | sensitive | ICEC0942 + Tamoxifen | Preclinical - Cell line xenograft | Actionable | In a preclinical study, ICEC0942, in combination with Tamoxifen, enhanced inhibition of tumor growth in ER-positive breast cancer cell-line xenograft models (PMID: 29545334). | 29545334 | |
| 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 | fibrosarcoma | not applicable | 23814 + Tivozanib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, treatment with the combination of 23814 and Tivozanib (AV-951) resulted in tumor growth inhibition in a fibrosarcoma cell line xenograft model, with increased efficacy over either agent alone (PMID: 25995436). | 25995436 | |
| Unknown unknown | Ewing sarcoma of bone | not applicable | Dasatinib | Phase II | Actionable | In a Phase II clinical trial, a trial arm assessing Sprycel (dasatinib) in Ewing sarcoma patients (n=17) was suspended due to lack of drug activity (PMID: 26710211). | 26710211 | |
| Unknown unknown | melanoma | not applicable | G-TPP + Navitoclax | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Navitoclax (ABT-263) synergistically inhibited viability of melanoma cell lines in culture, and inhibited tumor growth in cell line xenograft models (PMID: 28522750). | 28522750 | |
| Unknown unknown | hepatoblastoma | not applicable | UAB30 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a human hepatoblastoma cell line was sensitive to UAB30 in both culture and in xenograft models, demonstrating cell-cycle arrest, decreased cell proliferation, and reduced tumor growth (PMID: 26873726). | 26873726 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Crizotinib + Navitoclax | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of Navitoclax (ABT-263) and Xalkori (crizotinib) resulted in a synergistic effect and inhibited the growth of triple-receptor negative breast cancer cells in culture (PMID: 27872098). | 27872098 | |
| Unknown unknown | pediatric ependymoma | not applicable | CUDC-907 | Phase I | Actionable | In a Phase I trial, CUDC-907 treatment resulted in stable disease for 6 treatment cycles in a pediatric patient with ependymoma (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 10542-10542; NCT02909777). | detail... | |
| Unknown unknown | hairy cell leukemia | not applicable | Fenretinide | Phase I | Actionable | In a Phase I trial, Fenretinide treatment resulted in stable disease in a patient with hairy cell leukemia (PMID: 28420721). | 28420721 | |
| Unknown unknown | pancreatic adenocarcinoma | not applicable | Adavosertib + Gemcitabine + Radiotherapy | Phase I | Actionable | In a Phase I trial, Adavosertib (MK-1775) in combination with Gemzar (gemcitabine) and radiation therapy was tolerable, resulted in a median overall survival of 21.7 months and a median progression-free survival of 9.4 months in patients with advanced pancreatic adenocarcinoma (PMID: 31398082; NCT02037230). | 31398082 | |
| Unknown unknown | medulloblastoma | not applicable | M344 | Preclinical - Cell culture | Actionable | In a preclinical study, M344 induced apoptosis and inhibited proliferation of medulloblastoma cell lines in culture (PMID: 17230517). | 17230517 | |
| Unknown unknown | ovarian cancer | not applicable | S2101 | Preclinical - Cell culture | Actionable | In a preclinical study, S2101 altered gene expression, resulted in apoptosis in ovarian cancer cells in culture (PMID: 27914215). | 27914215 | |
| Unknown unknown | 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 | thyroid gland cancer | not applicable | Bevacizumab | Preclinical | Actionable | In a preclinical study, Avastin (bevacizumab) inhibited tumor growth and angiogenesis in mouse models of anaplastic thyroid cancer (PMID: 17429874). | 17429874 | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Lenalidomide + Ofatumumab | Phase I | Actionable | In a Phase I trial, the combination treatment of Revlimid (lenalidomide) and Arzerra (ofatumumab) in patients with chronic lymphocytic leukemia resulted in an overall response rate of 71% (24/34), in which 24% (8/34) experienced a complete remission and 47% (16/34) experienced a partial response (PMID: 26733610). | 26733610 | |
| Unknown unknown | Advanced Solid Tumor | no benefit | MSC2490484A | Phase I | Actionable | In a Phase I trial, MSC2490484A (M3814) demonstrated safety, but limited clinical activity, in patients with solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2556)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Bendamustine + Veliparib | Phase I | Actionable | In a Phase I trial, the combination of Bendamustine and Veliparib (ABT-888) resulted in stable disease in 63% (12/19) of patients with advanced solid tumors (PMID: 28314788; NCT01326702). | 28314788 | |
| Unknown unknown | pancreatic cancer | not applicable | Erlotinib + Gemcitabine | FDA approved | Actionable | In a Phase III clinical trial that supported FDA approval, the combination of Tarceva (erlotinib) and Gemzar (gemicitabine) resulted in an improved median overall survival of 6.24 months compared to 5.91 months with Gemzar (gemicitabine) and placebo, and prolonged progression-free survival (HR=0.77 (95% CI, 0.64 to 0.92; P = .004)) in patients with advanced pancreatic cancer (PMID: 17452677). | 17452677 detail... | |
| Unknown unknown | Hodgkin's lymphoma | not applicable | JNJ-40346527 | Phase II | Actionable | In a Phase II/III clinical trial, JNJ-40346527 demonstrated safety some efficacy, with complete response in 5.0% (1/20), partial response in 5.0% (1/20), and stable disease in 55.0% (11/20) of patients with refractory Hodgkin's lymphoma (PMID: 25628399). | 25628399 | |
| Unknown unknown | lung non-small cell carcinoma | no benefit | Bevacizumab | Phase III | Actionable | In a Phase IIIb trial, the combination of Avastin (bevacizumab) and standard of care therapy at first progression in non-small cell lung carcinoma patients previously treated with Avastin (bevacizumab) and chemotherapy and two maintenance cycles of Avastin (bevacizumab) did not result in a greater benefit compared to standard of care therapy alone, demonstrating a median overall survival of 11.9 mo vs 10.2 mo and a first to second progression-free survival of 5.5 mo vs 4.0 mo (PMID: 30177994; NCT01351415). | 30177994 | |
| Unknown unknown | 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 | breast cancer | not applicable | Danusertib + Fulvestrant | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with the combination of Danusertib (PHA-739358) 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 | Advanced Solid Tumor | not applicable | WX-554 | Phase I | Actionable | In a Phase I trial, WX-554 was well-tolerated and resulted in stable disease in 59% (20/34) and prolonged stable disease for greater than 6 months in 6% (2/34) of patients with advanced solid tumors (PMID: 27693888). | 27693888 | |
| Unknown unknown | esophageal cancer | not applicable | Capmatinib | Case Reports/Case Series | Actionable | In a Phase I trial, Capmatinib (INC280) demonstrated safety and preliminary efficacy, resulted in stable disease as best overall response in a patients with esophageal cancer (PMID: 30724423). | 30724423 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Sulindac | Preclinical - Cell culture | Actionable | In a preclinical study, Clinoril (sulindac) treatment inhibited proliferation of non-small cell lung carcinoma cells harboring either a PIK3CA mutation or amplification in culture (PMID: 30683736). | 30683736 | |
| 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 | glioblastoma multiforme | not applicable | Ponatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Iclusig (ponatinib) induced apoptosis and inhibited growth in glioblastoma cells in culture and in cell line xenograft models (PMID: 25378936). | 25378936 | |
| Unknown unknown | thyroid gland medullary carcinoma | not applicable | Cabozantinib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Cometriq (cabozantinib) resulted in improved progression free survival in patients with metastatic medullary thyroid cancer (PMID: 23319867). | detail... 23319867 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Guadecitabine | Phase I | Actionable | In a Phase I trial, SGI-110 treatment resulted in clinical response in 8% (6/74) of patients with acute myeloid leukemia (PMID: 26296954). | 26296954 | |
| Unknown unknown | brain stem glioma | not applicable | Niraparib + Radiotherapy | Preclinical - Cell culture | Actionable | In a preclinical study, a diffuse intrinsic pontine glioma cell line treated with a combination of ionizing radiation and Zejula (niraparib) in culture demonstrated a greater reduction in cell survival compared to either agent alone (PMID: 26351319). | 26351319 | |
| Unknown unknown | acute lymphoblastic leukemia | not applicable | OBI-3424 | Preclinical - Pdx | Actionable | In a preclinical study, OBI-3424 treatment resulted in objective response in 89% (8/9, 2 complete response) of patient-derived xenograft (PDX) models of T-Cell acute lymphoblastic leukemia (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr LB-B16). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | P276-00 | Phase I | Actionable | In a Phase I study, P276-00 demonstrated safety and some preliminary efficacy in patients with advanced solid tumors ( J Clin Oncol (Meeting Abstracts) June 2007 vol. 25 no. 18_suppl 14117). | detail... | |
| Unknown unknown | Merkel cell carcinoma | not applicable | Avelumab | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Bavencio (avelumab) treatment resulted in an objective response response rate of 31.8% (28/88), with complete response in 9% (8/88) and partial response in 23% (20/88) of patients with Merkel cell carcinoma (PMID: 27592805). | 27592805 detail... | |
| Unknown unknown | Merkel cell carcinoma | not applicable | Avelumab | Phase Ib/II | Actionable | In a Phase I/II trial, addition of Bavencio (avelumab) to adoptive transfer of Merkel cell polyomavirus (MCPyV)-specific T cells and HLA upregulation resulted in sustained complete response in 75% (3/4) of patients with MCPyV-associated Merkel cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 3044)). | detail... | |
| Unknown unknown | Merkel cell carcinoma | not applicable | Avelumab | FDA approved | Actionable | In a Phase II trial that supported FDA approval (JAVELIN Merkel 200), Bavencio (avelumab) treatment resulted in a 62.1% (18/29) objective response rate and a median progression-free survival of 9.1 months, and demonstrated early evidence of durable responses with response duration estimated to be greater than 3 months and 6 months in 93% and 83% of responding patients, respectively, in patients with treatment-naive metastatic Merkel cell carcinoma (PMID: 29566106; NCT 02155647). | 29566106 detail... | |
| Unknown unknown | acute myeloid leukemia | not applicable | (-)BI97D6 | Preclinical - Patient cell culture | Actionable | In a preclinical study, (-)BI97D6 decreased live cell number in primary acute myeloid leukemia samples from refractory patients in culture (PMID: 26045609 ). | 26045609 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | PKI-402 | Preclinical - Cell culture | Actionable | In a preclinical study, PKI-402 inhibited growth of several human solid tumor cell lines in culture (PMID: 20371716). | 20371716 | |
| Unknown unknown | hematologic cancer | not applicable | AZD4573 | Preclinical - Cell culture | Actionable | In a preclinical study, AZD4573 induced apoptosis and cell death in a panel of hematologic cancer cell lines in culture (Cancer Res 2018;78(13 Suppl):Abstract nr 310). | detail... | |
| Unknown unknown | kidney angiomyolipoma | not applicable | Everolimus | FDA approved | Actionable | In a Phase III trial (EXIST-2) that supported FDA approval, Afinitor (everolimus) treatment resulted in significantly improved response rate (42%, 33/79) compared to placebo (0%, 0/39) in patients with renal angiomyolipoma as a feature of tuberous sclerosis (n=113) or sporadic lymphanioleiomyomatosis (n=5) (PMID: 23312829; NCT00790400). | 23312829 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 | breast cancer | not applicable | AsiDNA + Olaparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA treatment resulted in increased sensitivity to Lynparza (olaparib), inhibiting DNA repair and cell proliferation, and inducing death in breast cancer cell lines in culture (PMID: 27559053). | 27559053 | |
| 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 | osteosarcoma | not applicable | WZ4003 | Preclinical - Cell culture | Actionable | In a preclinical study, WZ4003 inhibited MYPT1 phosphorylation and reduced invasive behavior and proliferation of an osteosarcoma cell line in culture (PMID: 24171924). | 24171924 | |
| Unknown unknown | brain glioblastoma multiforme | not applicable | Axitinib | Preclinical | Actionable | In a preclinical study, Inlyta (axitinib) demonstrated efficacy in glioblastoma cells, including those resistant to Bevacizumab (J Clin Oncol (Meeting Abstracts) May 2013 vol. 31 no. 15_suppl 2077). | detail... | |
| 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 + Daratumumab + Dexamethasone + Thalidomide | FDA approved | Actionable | In a Phase III trial (CASSIOPEIA) that supported FDA approval, Darzalex (daratumumab) in combination with Velcade (bortezomib), thalidomide, and dexamethasone (VTd) resulted in improved stringent complete response at 100 days post autologous stem-cell transplantation compared to VTd (29%, 157/543 vs 20%, 110/542, OR=1.60, p=0.0010) in patients with newly diagnosed multiple myeloma (PMID: 31171419; NCT02541383). | detail... 31171419 | |
| Unknown unknown | breast cancer | not applicable | Docetaxel + MEDI5117 | Preclinical | Actionable | In a preclinical study, MEDI5117 in combination with Taxotere (docetaxel) resulted in complete tumor regression of human breast cancer cells in an orthotopic model (PMID: 26744529). | 26744529 | |
| Unknown unknown | mucosal melanoma | not applicable | Toripalimab | Case Reports/Case Series | Actionable | In a Phase I trial, Toripalimab (JS001) demonstrated safety and preliminary efficacy, resulted in partial response in 1 and stable disease in 1 of 4 patients with mucosal melanoma (PMID: 30642373; NCT02836795). | 30642373 | |
| Unknown unknown | pancreatic cancer | not applicable | Ensituximab | Phase I | Actionable | In a Phase I trial, Ensituximab (NEO-102) demonstrated safety and preliminary efficacy, resulting in stable disease in 42% (5/12) of patients with refractory colon or pancreatic cancer (PMID: 27449137; NCT01040000). | 27449137 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Cediranib + Cisplatin + Gemcitabine | Phase I | Actionable | In a Phase I trial, the combination of Cediranib with Gemzar (gemcitabine) and Platinol (cisplatin) demonstrated preliminary efficacy in patients with advanced non-small cell lung cancer (PMID: 19091548). | 19091548 | |
| Unknown unknown | peritoneum cancer | not applicable | Bevacizumab + Everolimus | Phase II | Actionable | In a Phase II trial, Afinitor (everolimus) and Avastin (bevacizumab) combination treatment resulted in progression free survival at 6 months in 28% (14/50) of patients with ovarian, fallopian tube, and peritoneal cancers (J Clin Oncol 34, 2016 (suppl; abstr 5552)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Fluorouracil + Methylnaltrexone | Preclinical | Actionable | In a preclinical study, Relistor (Methylnaltrexone) enhanced the efficacy of Adrucil (fluorouracil) in a variety of human cancer cell lines (PMID: 19661297). | 19661297 | |
| Unknown unknown | multiple myeloma | not applicable | Bortezomib + SJB3-019A | Preclinical - Patient cell culture | Actionable | In a preclinical study, the combination of SJB3-019A and Velcade (bortezomib) worked synergistically to induce cytotoxicity in multiple myeloma cell lines and primary multiple myeloma cells in culture (PMID: 28270494). | 28270494 | |
| Unknown unknown | 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 | Advanced Solid Tumor | not applicable | Trebananib | Phase I | Actionable | In a Phase I trial, treatment with Trebananib demonstrated tolerability in pediatric patients with advanced solid tumors, and resulted in stable disease for greater than 4 months in one patient with neuroblastoma and one patient with anaplastic astrocytoma (PMID: 28751444). | 28751444 | |
| Unknown unknown | 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 | multiple myeloma | not applicable | MS417 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with MS417 altered expression of ZCCHC24, HEXIM1, SERPINI1, ZMYND8, and MYC in a multiple myeloma cell line in culture, and these changes correlated with decreased proliferation in culture (PMID: 27903752). | 27903752 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Axitinib | Phase II | Actionable | In a Phase II clinical trial, Inlyta (axitinib) was well-tolerated and demonstrated activity in patients with advanced non-small cell lung cancer, with a disease control rate of 41% (13/32), median progression-free survival of 4.9 months, and median overall survival of 14.8 months (PMID: 19597027). | 19597027 | |
| Unknown unknown | thyroid gland medullary carcinoma | not applicable | Vandetanib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Caprelsa (vandetanib) resulted in improved progression-free survival, an objective response rate of 45%, and a disease control rate of 87% in patients with medullary thyroid carcinoma (PMID: 22025146, PMID: 22723734). | 22723734 detail... 22025146 | |
| 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 | kidney rhabdoid cancer | not applicable | UAB30 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a human malignant rhabdoid kidney tumor cell line was sensitive to UAB30 in both culture and in xenograft models, demonstrating cell-cycle arrest, decreased cell proliferation, apoptosis, and reduced tumor growth (PMID: 26873726). | 26873726 | |
| Unknown unknown | prostate cancer | not applicable | Forskolin | Preclinical | Actionable | In a preclinical study, prostate cancer cells treated with Forskolin demonstrated PP2A activation and inhibition of cell proliferation in culture (PMID: 26023836). | 26023836 | |
| Unknown unknown | 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 | 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 | hepatocellular carcinoma | no benefit | Brivanib | Phase III | Actionable | In a Phase III trial, Brivanib (BMS-540215) treatment used as an adjuvant therapy to Transarterial Chemoembolization (TACE) did not result in a greater overall survival when compared to placebo with TACE in hepatocellular carcinoma patients (PMID: 24996197). | 24996197 | |
| Unknown unknown | hepatocellular carcinoma | no benefit | Brivanib | Phase III | Actionable | In a Phase III trial, treatment with Brivanib (BMS-540215) compared to Nexavar (sorafenib) resulted in similar results for overall survival, time to progression, overall response rate, and disease control rate in hepatocellular carcinoma patients (PMID: 23980084). | 23980084 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Regorafenib | FDA approved | Actionable | In a Phase III trial (RESORCE) that supported FDA approval, treatment with Stivarga (regorafenib) following Nexavar (sorafenib) treatment resulted in improved overall survival (10.6 vs 7.8 months, HR=0.63) compared to Nexavar (sorafenib) followed by placebo in patients with hepatocellular carcinoma (PMID: 27932229; NCT01774344). | detail... 27932229 | |
| Unknown unknown | cervical cancer | not applicable | Tozasertib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Tozasertib (VX-680) inhibited growth of cervical cancer cell lines in culture, and inhibited tumor growth in cervical cancer cell line xenograft models (PMID: 27082306). | 27082306 | |
| Unknown unknown | acute myeloid leukemia | not applicable | SST0116CL1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, SST0116CL1 demonstrated antitumor activity in cell line xenograft models of acute myeloid leukemia (PMID: 25096516). | 25096516 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Paclitaxel + Taladegib | Phase I | Actionable | In a Phase I trial, combination of Taladegib and Taxol (paclitaxel) treatment demonstrated safety and preliminary efficacy, resulted in partial response in 3 patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2594)). | detail... | |
| Unknown unknown | 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 | prostate cancer | not applicable | Relugolix | Phase II | Actionable | In a Phase II trial, Relugolix (TAK-385) resulted in decreased testosterone levels and greatly reduced prostate specific antigen levels after 24 weeks in prostate cancer patients (J Clin Oncol 34, 2016 (suppl 2S; abstr 200)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Gedatolisib | Phase I | Actionable | In a Phase I trial, Gedatolisib (PF-05212384) demonstrated safety and efficacy, which resulted in antitumor activity and stable disease greater than six months in 10.4% (8/27) of patients with solid malignant tumors (PMID: 25652454). | 25652454 | |
| 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 | acute myeloid leukemia | not applicable | Cytarabine + Glasdegib | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of Glasdegib (PF-04449913) and Cytosar-U (cytarabine) resulted in an overall survival of 4.4 months in patients with either acute myeloid leukemia (AML) or myelodysplastic syndrome, with 5% (1/20) of AML patients experiencing a complete remission (PMID: 29463550). | 29463550 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Cytarabine + Glasdegib | FDA approved | Actionable | In a Phase II trial that supported FDA approval, Daurismo (glasdegib) in combination with low-dose cytarabine resulted in improved median survival (8.3 vs 4.3 months, HR=0.46, p=0.0002) compared to low-dose cytarabine alone in patients with treatment-naive acute myeloid leukemia who are ineligible for intensive chemotherapy (Blood 2016 128 (22): 99; NCT01546038). | detail... detail... | |
| Unknown unknown | lung squamous cell carcinoma | not applicable | Carboplatin + Paclitaxel + Pembrolizumab | FDA approved | Actionable | In a Phase III trial (KEYNOTE-407) that supported FDA approval, Keytruda (pembrolizumab) in combination with chemotherapy consisted of carboplatin and paclitaxel or nab-paclitaxel significantly improved overall survival (15.9 vs 11.3 months), progression-free survival (6.4 vs 4.8 months), and overall response rate (58% vs 35%) compared to placebo plus chemotherapy in patients with untreated metastatic squamous non-small cell lung cancer (J Clin Oncol 36, no. 15_suppl, 105-105; NCT02775435). | detail... detail... | |
| Unknown unknown | multiple myeloma | not applicable | Elotuzumab + GDA-201 | Phase I | Actionable | In a Phase I trial, GDA-201 and Empliciti (elotuzumab) combination therapy demonstrated safety and preliminary efficacy, resulted in complete response in 7.7% (1/13) and stable disease in 30.8% (4/13) of patients with refractory multiple myeloma, with a median duration of response of 2.5 months (Blood (2019) 134 (Supplement_1): 777). | detail... | |
| Unknown unknown | B-cell lymphoma | not applicable | IT-901 | Preclinical | Actionable | In a preclinical study, IT-901 inhibited tumor growth in Epstein Barr Virus-induced B-cell lymphoma xenograft models (PMID: 26744524). | 26744524 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Cabozantinib | Phase Ib/II | Actionable | In a Phase Ib/II trial, Cometriq (cabozantinib) treatment resulted in partial response in 6.7% (1/15) of patients with non-small cell lung carcinoma that had progressed during treatment with Tarceva (erlotinib), with a median progression free survival of 1.9 months (PMID: 28352985). | 28352985 | |
| Unknown unknown | T-cell acute lymphoblastic leukemia | not applicable | Asparaginase + Dexamethasone + DT2216 + Vincristine | Preclinical - Pdx | Actionable | In a preclinical study, the addition of DT2216 to therapy with Oncovin (vincristine), dexamethasone, and Elspar (asparaginase) resulted in increased survival in a chemotherapy-resistant patient-derived xenograft (PDX) model of T-cell acute lymphoblastic leukemia that expressed high levels of BCL2, MCL1, and BCL-XL (BCL2L1) (PMID: 31792461). | 31792461 | |
| Unknown unknown | cholangiocarcinoma | not applicable | Zebularine | Preclinical - Cell culture | Actionable | In a preclinical study, Zebularine treatment decreased DNMT1 expression,and induced apoptosis in cholangiocarcinoma cell lines in culture (PMID: 25799509). | 25799509 | |
| Unknown unknown | 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 | colon carcinoma | not applicable | CA-170 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, CA-170 activated peripheral T cells inhibited tumor growth in mouse models of colon carcinoma (Ann Oncol. 2017 Sep 18; 28 (Suppl_5): Abstract 1141PD). | detail... | |
| Unknown unknown | peritoneum cancer | not applicable | ENMD-2076 | Phase II | Actionable | In a Phase II clinical trial, ENMD-2076 demonstrated efficacy in patients with recurrent, platinum-resistant ovarian, fallopian tube or peritoneal cancers (PMID: 22921155). | 22921155 | |
| Unknown unknown | pancreatic cancer | not applicable | MVT-1075 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a pancreatic cancer xenograft model demonstrated tumor growth inhibition and tumor regression by 50% when treated with MVT-1075 (AACR 2017, Abstract #5204). | detail... | |
| Unknown unknown | pancreatic ductal adenocarcinoma | not applicable | Gemcitabine + MN58b | Preclinical | Actionable | In a preclinical study, MN58b and Gemzar (gemcitabine) in combination demonstrated an additive effect on growth inhibition of pancreatic ductal adenocarcinoma cell lines in culture (PMID: 26769123). | 26769123 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Bortezomib | Preclinical - Cell culture | Actionable | In a preclinical study, Velcade (bortezomib) induced complete growth inhibition of triple-receptor negative breast cancer cells in culture (PMID: 27872098). | 27872098 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BI-847325 | Phase I | Actionable | In a Phase I trial, BI-847325 treatment resulted in stable disease in 30% (21/69) of patients with advanced solid tumors, and one partial response for 67 days in a patient with esophageal cancer (PMID: 26650227). | 26650227 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BI-847325 | Phase I | Actionable | In a Phase I trial, BI-847325 demonstrated safety and some efficacy in a variety of advanced solid tumors (Mol Cancer Ther 2013;12(11 Suppl):B281). | detail... | |
| Unknown unknown | ovarian cancer | not applicable | TRX-E-002-1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, TRX-E-002-1 inhibited proliferation and induced apoptosis in chemoresistant human ovarian cancer cell lines in culture, and reduced tumor size in ovarian cancer cell line xenograft models (PMID: 27196760). | 27196760 | |
| Unknown unknown | renal cell carcinoma | not applicable | Pazopanib + Trametinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, combination of Mekinist (trametinib) and Votrient (pazopanib) effectively inhibited tumor angiogenesis and growth in cell line xenograft models of renal cell carcinoma (PMID: 26487278). | 26487278 | |
| Unknown unknown | neuroblastoma | not applicable | Ellipticine + Valproic acid | Preclinical | Actionable | In a preclincal study, Depakene (valproic acid) enhanced the cytotoxicity of ellipticine in human neuroblastoma cell lines (PMID: 22167207). | 22167207 | |
| Unknown unknown | multiple myeloma | not applicable | Filanesib | Phase Ib/II | Actionable | In a Phase I/II trial, treatment with Filansenib (ARRY-520) demonstrated safety, and resulted in an overall response rate of 16% (5/31), clinical benefit rate of 23% (7/31), and median overall survival of 19.0 months in the Phase II portion in patients with refractory or relapsed multiple myeloma (PMID: 28817190, NCT00821249). | 28817190 | |
| Unknown unknown | CLL/SLL | not applicable | Zanubrutinib | Phase I | Actionable | In a Phase I trial, Brukinsa (zanubrutinib) treatment resulted in an overall response rate of 96.2% (75/78, 2 complete response, 63 partial response (PR), 10 PR with lymphocytosis) in patients with CLL/SLL (PMID: 31340982; NCT02343120). | 31340982 | |
| Unknown unknown | breast cancer | not applicable | AsiDNA + Rucaparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA and Rubraca (rucaparib) combination treatment resulted in increased cell death and inhibition of proliferation in breast cancer cell lines compared to Rubraca (rucaparib) alone in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | alveolar soft part sarcoma | not applicable | Anlotinib | Phase II | Actionable | In a Phase II trial, Anlotinib (AL-3818) treatment resulted a 12-week progression-free rate of 77%, median progression-free survival of 21 months, an objective response rate of 46% (n=13), and median overall survival was not reached in patients with alveolar soft part sarcoma (PMID: 29895706; NCT01878448). | 29895706 | |
| Unknown unknown | 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 | multiple myeloma | not applicable | Lenalidomide + SJB3-019A | Preclinical - Patient cell culture | Actionable | In a preclinical study, the combination of SJB3-019A and Revlimid (lenalidomide) worked synergistically to induce cytotoxicity in multiple myeloma cell lines and primary multiple myeloma cells in culture (PMID: 28270494). | 28270494 | |
| Unknown unknown | 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 | 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 | hematologic cancer | not applicable | ABT-348 | Phase I | Actionable | In a Phase I trial, ABT-348 (Ilorasertib) demonstrated safety and preliminary efficacy in patients with various hematological malignancies (PMID: 25933833). | 25933833 | |
| Unknown unknown | prostate cancer | not applicable | BGP-15 | Preclinical - Cell culture | Actionable | In a preclinical study, BGP-15 induced apoptosis and inhibited growth of prostate cancer cells in culture (PMID: 22661288). | 22661288 | |
| Unknown unknown | colorectal cancer | not applicable | AB61 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the synthetic nucleoside AB61 demonstrated cytotoxicity in colorectal cancer cell lines in culture and decreased tumor volume and prolonged survival in colorectal cancer cell line xenograft models (PMID: 26819331). | 26819331 | |
| Unknown unknown | ovarian cancer | not applicable | Paclitaxel + TVB-2640 | Phase I | Actionable | In a Phase I trial, TVB-2640 and Taxol (paclitaxel) combination treatment resulted in 58-98% reduction of CA-125 level in 42% (5/12) of ovarian cancer patients (J Clin Oncol 34, 2016 (suppl; abstr 2512)). | detail... | |
| Unknown unknown | pancreatic cancer | not applicable | ABC294640 + Gemcitabine | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of ABC294640 and Gemzar (gemcitabine) worked synergistically to decrease viability of pancreatic cancer cell lines in culture (PMID: 27517489). | 27517489 | |
| Unknown unknown | 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 | Advanced Solid Tumor | not applicable | Docetaxel + SGT-53 | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of Taxotere (docetaxel) and SGT-53 demonstrated safety, and out of 12 evaluable patients resulted in partial response (PR) in 2 patients, an unverified PR in 1 patient, stable disease with tumor shrinkage in 2 patients, and stable disease without tumor shrinkage in 4 patients with advanced solid tumors (PMID: 27357628). | 27357628 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Linifanib | Phase II | Actionable | In a Phase II clinical trial, single-agent linifanib was found safe and efficacious in patients with advanced hepatocellular carcinoma (PMID: 22833179). | 22833179 | |
| Unknown unknown | 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 | renal cell carcinoma | not applicable | Sorafenib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) resulted in an improved median progression-free survival of 167 days in patients with renal cell carcinoma (PMID: 17189398). | detail... 17189398 | |
| Unknown unknown | aggressive NK-cell leukemia | not applicable | tagraxofusp-erzs | FDA approved | Actionable | In a Phase I/II trial that supported FDA approval, Elzonris (tagraxofusp-erzs) treatment resulted in complete response/clinical complete response in 53.8% (7/13) of patients with untreated blastic plasmacytoid dendritic cell neoplasm (BPDCN, also known as natural killer cell leukemia/lymphoma) with a median follow-up of 11.5 months, and 1 complete response and 1 clinical complete response in 15 patients with relapsed or refractory BPDCN (FDA.gov; NCT02113982). | detail... detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Rosmantuzumab | Phase I | Actionable | In a Phase I clinical trial, treatment with OMP-131R10 was well-tolerated and demonstrated preliminary efficacy in patients with advanced solid tumors, with 37% (7/19) of patients achieving stable disease (EORTC-NCI-AACR 2017; Abstract nr 68). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Icrucumab | Phase I | Actionable | In a Phase I trial, Icrucumab (IMC-18F1) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 23903897). | 23903897 | |
| Unknown unknown | 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 | 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 | colon cancer | not applicable | Ch282-5 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, ch282-5 induced apoptosis and inhibited growth and migration of several human and mouse colon cancer cell lines in culture, and inhibited tumor growth and metastasis in xenograft models (PMID: 26515494). | 26515494 | |
| Unknown unknown | oral squamous cell carcinoma | not applicable | Trametinib | Phase II | Actionable | In a Phase II trial, Mekinist (trametinib) treatment in patients with oral cavity squamous cell carcinoma was associated with decreased phosphorylation of Erk1/2 and reduced CD44 expression, and resulted in a partial response in 65% (11/17), stable disease in 29% (5/17), and progressive disease in 1 patient (6%) (PMID: 28151720). | 28151720 | |
| Unknown unknown | glioblastoma multiforme | not applicable | GSK1838705A | Preclinical - Cell line xenograft | Actionable | In a preclinical study, GSK1838705A inhibited cell growth and prevented tumor growth via apoptotic induction in glioma cells in both culture and xenograft models (PMID: 26238593). | 26238593 | |
| Unknown unknown | multiple myeloma | not applicable | Marizomib | Phase I | Actionable | In a Phase I trial, Marizomib (NPI-0052) treatment resulted in objective response in 14.8% (4/27) and stable disease in 44.4% (12/27) of patients with relapsed and/or refractory multiple myeloma (PMID: 27117181). | 27117181 | |
| Unknown unknown | 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 | breast cancer | not applicable | Elenagen + Tamoxifen | Phase Ib/II | Actionable | In a Phase Ib/II trial, a chemorefractory patient with breast cancer demonstrated restored chemotherapeutic sensitivity upon sequential treatment of Elenagen and Nolvadex (tamoxifen), which resulted in stable disease (PMID: 28881846). | 28881846 | |
| Unknown unknown | glioblastoma multiforme | not applicable | SA16 | Preclinical - Cell culture | Actionable | In a preclinical study, SA16 treatment in glioblastoma cells resulted in decreased cell proliferation, reduced cell viability, and apoptotic induction in culture, and inhibited glioma stem cell formation (PMID: 27797168). | 27797168 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Carboplatin + LY2090314 + Pemetrexed | Phase I | Actionable | In a Phase I trial, LY2090314 in combination with Alimta (pemetrexed) and Paraplatin (carboplatin) resulted in partial response in 13.5% (5/37) and stable disease in 51.4% (19/37) of patients with advanced solid tumors (PMID: 26403509; NCT01287520). | 26403509 | |
| Unknown unknown | 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 | 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 | pancreatic ductal adenocarcinoma | not applicable | Arsenic trioxide + JQ1 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, JQ1 and Trisenox (arsenic trioxide) combination treatment reduced viability of Trisenox (arsenic trioxide)-insensitive pancreatic ductal adenocarcinoma cell lines in culture, and synergistically inhibited tumor growth in a cell line xenograft model (PMID: 31420604). | 31420604 | |
| Unknown unknown | 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 | lymphoma | not applicable | PU-H71 | Preclinical - Cell culture | Actionable | In a preclinical study, sensitivity to PU-H71 was correlated to presence of the epichaperome, a network of chaperome complexes, in lymphoma cell lines in culture (PMID: 27706135). | 27706135 | |
| Unknown unknown | melanoma | no benefit | Alvocidib | Phase II | Actionable | In a Phase II trial, Alvocidib (flavopiridol) treatment resulted in stable disease in 44% (7/16) of patients with melanoma, but no objective response (PMID: 15122079). | 15122079 | |
| Unknown unknown | sarcoma | not applicable | Doxorubicin + Nilotinib | Phase I | Actionable | In a Phase I trial, Tasigna (nilotinib) in combination with doxorubicin demonstrated safety and preliminary efficacy, resulted in 1 partial response and 9 stable disease in 13 patients with sarcomas (PMID: 30037815; NCT02587169). | 30037815 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Fluorouracil + Sorafenib | Phase I | Actionable | In a Phase I trial, Nexavar (sorafenib) in combination with Adrucil (fluorouracil) displayed safety and efficacy in advanced solid tumors, including colon cancer (PMID: 22232731). | 22232731 | |
| Unknown unknown | 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 | not applicable | Pimasertib | Phase I | Actionable | In a Phase I trial, Pimasertib (MSC1936369B) demonstrated safety and some preliminary anti-tumor activity in patients with advanced solid tumors (J Clin Oncol 28:15s, 2010 (suppl; abstr 2504)). | detail... | |
| Unknown unknown | 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 | gastrointestinal system cancer | not applicable | Axitinib + Fluorouracil + Irinotecan + Leucovorin | Phase I | Actionable | In a Phase I trial, Inlyta (axitinib), in combination with FOLFIRI, demonstrated safety and some efficacy in patients with gastrointestinal tumors (PMID: 24423921). | 24423921 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ralimetinib | Phase I | Actionable | In a Phase I trial, Ralimetinib (LY2228820) resulted in stable disease in 21.3% (19/74) of patients with an advanced solid tumor, which lasted a median duration of 3.7 months (PMID: 26581242). | 26581242 | |
| Unknown unknown | renal cell carcinoma | no benefit | Bevacizumab + Carotuximab | Clinical Study | Actionable | In a clinical study, the addition of TRC105 to Avastin (bevacizumab) treatment in renal cell carcinoma patients did not result in improved progression free survival (2.8 mo vs 4.6 mo) when compared to Avastin (bevacizumab) alone (PMID: 28832978). | 28832978 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Nivolumab | Clinical Study - Meta-analysis | Actionable | In a meta-analysis, compared to Taxotere (docetaxel), treatment with immune checkpoint inhibitors including Opdivo (nivolumab), Keytruda (pembrolizumab), or Tecentriq (atezolizumab), resulted in prolonged overall survival (HR=0.69, p<0.001) in non-small cell lung carcinoma patients (PMID: 29270615). | 29270615 | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase III trial (CheckMate 017) that supported FDA approval, squamous non-small cell lung carcinoma patients treated with Opdivo (nivolumab) demonstrated a greater overall survival (9.2 mo vs 6.0 mo), response rate (20% vs 9%), and progression-free survival (3.5 mo vs 2.8 mo) when compared to treatment with Taxotere (docetaxel), regardless of CD274 (PD-L1) expression level (PMID: 26028407; NCT01642004). | 26028407 detail... | |
| Unknown unknown | lung non-small cell carcinoma | not applicable | Nivolumab | FDA approved | Actionable | In a Phase III trial (CheckMate 057) that supported FDA approval, nonsquamous non-small cell lung carcinoma patients treated with Opdivo (nivolumab) demonstrated a greater overall survival (12.2 vs 9.4 months, HR=0.73, p=0.002) when compared to treatment with Taxotere (docetaxel) (PMID: 26412456; NCT01673867). | detail... 26412456 | |
| Unknown unknown | pancreatic cancer | not applicable | Axitinib + Gemcitabine | Phase I | Actionable | In a Phase I study, Inlyta (axitinib) in combination with Gemzar (gemcitabine), exhibited safety and antitumor activity in advanced pancreatic cancer patients (PMID: 21670972). | 21670972 | |
| Unknown unknown | esophagus adenocarcinoma | not applicable | Nintedanib | Phase II | Actionable | In a Phase II trial, Ofev (nintedanib) was well tolerated and the study met its primary endpoint, resulted in progression-free survival at 6-months in 19% (6/32) of patients with esophageal/GEJ (n=17) or gastric (n=15) adenocarcinoma, with a median follow-up of 14.5 months and a median overall survival of 14.2 months (PMID: 30952642; NCT02234596). | 30952642 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | ABTL0812 | Phase I | Actionable | In a Phase I/Ib trial, ABTL0812 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol (Meeting Abstracts) May 2015 vol. 33 no. 15_suppl 2585). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Belvarafenib | Phase I | Actionable | In a Phase I trial, Belvarafenib (HM95573) treatment demonstrated safety and preliminary efficacy, resulted in unconfirmed partial response in 3% (1/31) and stable disease in 29% (9/31) of patients with advanced solid tumors harboring BRAF (45%), KRAS (45%) or NRAS (10%) mutations (May 20 2016) 2570-2570; NCT02405065). | detail... | |
| Unknown unknown | glioblastoma multiforme | not applicable | PRX177561 + Sunitinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of PRX177561 and Sutent (sunitinib) decreased tumor growth and improved time-to-progression, disease-free survival, and overall survival over either agent alone in glioblastoma cell line xenograft models (PMID: 28057017). | 28057017 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Decitabine + Glasdegib | Phase Ib/II | Actionable | In a Phase Ib trial, the combination of Glasdegib (PF-04449913) and Dacogen (decitabine) resulted in an overall survival of 11.5 months in patients with either acute myeloid leukemia (AML) or myelodysplastic syndrome, with 40% (2/5) of AML patients experiencing a complete remission (PMID: 29463550). | 29463550 | |
| Unknown unknown | ovarian cancer | not applicable | Selumetinib + SHP099 | Preclinical - Cell culture | Actionable | In a preclinical study, the combination therapy of Selumetinib (AZD6244) and SHP099 resulted in decreased colony formation and reduced cell viability in ovarian cancer cells in culture and inhibition of tumor growth and reduced tumor angiogenesis in a patient-derived xenograft (PDX) model of ovarian cancer (PMID: 30045908). | 30045908 | |
| Unknown unknown | ovarian cancer | not applicable | Navicixizumab | Case Reports/Case Series | Actionable | In a Phase I trial, Navicixizumab (OMP-305B83) treatment resulted in partial response in 3 patients with advanced ovarian cancer, and 7 of the 11 ovarian cancer patients had a reduction of target lesions (PMID: 30229512). | 30229512 | |
| Unknown unknown | acute myeloid leukemia | not applicable | GNE-781 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, GNE-781 treatment resulted in tumor growth inhibition and reduced MYC transcript levels in acute myeloid leukemia cell line xenograft models (PMID: 28892380). | 28892380 | |
| Unknown unknown | triple-receptor negative breast cancer | not applicable | Cisplatin + JQ1 | Preclinical - Cell culture | Actionable | In a preclinical study, the addition of JQ1 to Platinol (cisplatin) resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27256375). | 27256375 | |
| Unknown unknown | sarcoma | not applicable | Regorafenib | Phase II | Actionable | In a Phase II trial, Stivarga (regorafenib) treatment resulted in improved median progression-free survival (2.9 vs 1.0 months), but no difference in overall survival (HR=0.75, p=0.37) compared to placebo in patients with soft tissue sarcoma excluding liposarcoma, leiomyosarcoma, and synovial sarcoma (PMID: 27751846). | 27751846 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | BAL101553 | Phase I | Actionable | In a Phase I trial, BAL101553 treatment resulted in stable disease in 37% (7/19) of patients with advanced solid tumors (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 2530-2530; NCT02490800). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Anlotinib | Phase I | Actionable | In a Phase I trial, Anlotinib (AL-3818) treatment resulted in partial response in 15% (3/20) and stable disease in 70% (14/20) of patients with advanced solid tumors (J Clin Oncol 33, 2015 (suppl; abstr e13586)). | detail... | |
| Unknown unknown | retinoblastoma | not applicable | Prexasertib | Preclinical - Cell culture | Actionable | In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including a retinoblastoma cell line (PMID: 28270495). | 28270495 | |
| Unknown unknown | renal cell carcinoma | not applicable | Toripalimab | Phase I | Actionable | In a Phase I trial, Toripalimab (JS001) demonstrated safety and preliminary efficacy, resulted in an objective response rate of 33.3% (2/6) and a disease control rate of 50.0% (3/6, 2 partial response, 1 stable disease) in patients with advanced renal cell carcinoma (PMID: 30642373; NCT02836795). | 30642373 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Sapitinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Sapitinib (AZD8931) inhibited EGFR, ERBB2 (HER2), and ERBB3 (HER3) kinase activity, and inhibited tumor growth in several cell line xenograft models, including breast, NSCLC, colorectal, and head and neck squamous cell carcinoma xenograft models (PMID: 20145185). | 20145185 | |
| Unknown unknown | chromophobe renal cell carcinoma | not applicable | Bevacizumab + Everolimus | Phase II | Actionable | In a Phase II trial, 60% (3/5) of patients with chromophobe renal cell carcinoma achieved a 6 month PFS when treated with a combination of Afinitor (everolimus) and Avastin (bevacizumab) (PMID: 27601542). | 27601542 | |
| 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 | Advanced Solid Tumor | not applicable | Conatumumab + Ganitumab | Phase Ib/II | Actionable | In a Phase Ib/II trial, Ganitumab and Conatumumab (AMG 655) combination treatment resulted in stable disease in 36% (28/78) of patients with an advanced solid tumor (PMID: 24816908). | 24816908 | |
| Unknown unknown | ovarian cancer | not applicable | AZD1480 | Preclinical | Actionable | In a preclinical study, AZD1480 inhibited cell proliferation of ovarian cancer cells with active Jak-Stat signaling in culture and blocked tumor growth in transgenic mouse models of ovarian cancer (PMID: 25646015). | 25646015 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Ficlatuzumab | Phase I | Actionable | In a Phase I trial, Ficlatuzumab treatment resulted in stable disease in 57% (12/21) of patients with advanced solid tumors and a decrease in phosphorylated Met in one patient with multiple myeloma (PMID: 24901237). | 24901237 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | SGI-1027 | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with SGI-1027 reduced proliferation of a rat hepatoma cell line in culture in culture (PMID: 19417133). | 19417133 | |
| Unknown unknown | lymphoma | not applicable | Everolimus | Phase II | Actionable | In a Phase II trial, Afinitor (everolimus) treatment resulted in an overall response rate of 44% (7/16) in T-cell lymphoma patients, with a median progression-free survival of 4.1 months and a median overall survival of 10.2 months (PMID: 25921059). | 25921059 | |
| Unknown unknown | clear cell renal cell carcinoma | not applicable | PF-03446962 | Phase I | Actionable | In a Phase I trial, a patient with renal clear cell carcinoma demonstrated a partial response for 325 days when treated with PF-03446962 (PMID: 26655846). | 26655846 | |
| Unknown unknown | breast cancer | not applicable | R916562 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, R916562 inhibited tumor cell growth and resulted in tumor regression in a cell line xenograft model of breast cancer (PMID: 28711351). | 28711351 | |
| Unknown unknown | breast cancer | not applicable | Paclitaxel + TVB-2640 | Phase I | Actionable | In a Phase I trial, TVB-2640 and Taxol (paclitaxel) combination treatment resulted in partial response in 8% (1/12) and stable disease for more than 20 weeks in 25% (3/12) of breast cancer patients (J Clin Oncol 34, 2016 (suppl; abstr 2512)). | detail... | |
| Unknown unknown | glioblastoma multiforme | not applicable | BLZ945 | Preclinical | Actionable | In a preclinical study, long term treatment with BLZ945 resulted in resistance in 56% of transgenic glioblastoma multiforme mouse models (PMID: 27199435). | 27199435 | |
| Unknown unknown | lung cancer | not applicable | Capmatinib | Case Reports/Case Series | Actionable | In a Phase I trial, Capmatinib (INC280) demonstrated safety and preliminary efficacy, resulted in stable disease as best overall response in 2 patients with lung cancer (PMID: 30724423). | 30724423 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Azacitidine + Venetoclax | FDA approved | Actionable | In a Phase I/II trial that supported FDA approval, Venclexta (venetoclax) in combination with decitabine or azacitidine resulted in complete remission or complete remission with incomplete count recovery in 65% (40/62) of patients 75 years old or older with treatment-naive acute myeloid leukemia ineligible for intensive chemotherapy, with an overall survival of 11 months (PMID: 30361262; NCT02203773). | detail... 30361262 | |
| 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 | 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 | ovarian cancer | not applicable | Ad5CMV-p53 gene | Phase I | Actionable | In a Phase I trial, Ad5CMV-p53 gene therapy demonstrated safety and preliminary efficacy, resulting in a mixed response in 12% (2/17) and stable disease in 24% (4/17) of patients with platinum- and paclitaxel-resistant metastatic epithelial ovarian cancer (PMID: 15297186). | 15297186 | |
| Unknown unknown | Indication other than cancer | not applicable | Valproic acid | FDA approved | Actionable | Valproic acid is FDA approved for use in patients with seizures, migraines, and bipolar disorder (FDA.gov). | detail... detail... | |
| Unknown unknown | endometrial clear cell adenocarcinoma | not applicable | ONC201 | Clinical Study | Actionable | In a clinical case study, a patient with clear cell endometrial cancer treated with ONC201 (TIC-10) demonstrated some reduction in lesion size (>30%), but also developed new lesions (PMID: 28331050). | 28331050 | |
| Unknown unknown | ovarian cancer | not applicable | Bevacizumab + Everolimus | Phase II | Actionable | In a Phase II trial, Afinitor (everolimus) and Avastin (bevacizumab) combination treatment resulted in progression free survival at 6 months in 28% (14/50) of patients with ovarian, fallopian tube, and peritoneal cancers (J Clin Oncol 34, 2016 (suppl; abstr 5552)). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | 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 | prostate carcinoma | not applicable | BEZ235 | Preclinical | Actionable | In a preclinical study, treatment with BEZ235 resulted in a decrease in prostate cancer progenitor cells in culture and reduced tumor growth in prostate carcinoma xenograft models (PMID: 21138868). | 21138868 | |
| Unknown unknown | colorectal cancer | not applicable | CCT137690 + Radiotherapy | Preclinical - Cell culture | Actionable | In a preclinical study, colorectal cancer cells were more sensitive to radiotherapy when treated with CCT137690 (PMID: 24476310). | 24476310 | |
| Unknown unknown | prostate cancer | not applicable | AGS-1C4D4 | Phase I | Actionable | In a Phase I trial, AGS-1C4D4 treatment in castration resistant prostate cancer patients was well-tolerated and resulted in stable disease at 24 weeks in 46% (6/13) of patients, but did not reduce PSA (PMID: 22020316). | 22020316 | |
| Unknown unknown | liposarcoma | not applicable | Selinexor | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Selinexor (KPT-330) decreased Birc5 (Survivin) expression and induced PARP and caspase-3 cleavage, reduced viability of liposarcoma cell lines in culture, and inhibited tumor growth in xenograft models (PMID: 28314790). | 28314790 | |
| Unknown unknown | pancreatic cancer | not applicable | Galunisertib | Phase I | Actionable | In a Phase I trial, two patients with pancreatic cancer demonstrated a best response of stable disease when treated with Galunisertib (LY2157299) (PMID: 26526984; NCT01722825). | 26526984 | |
| Unknown unknown | 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 | head and neck squamous cell carcinoma | not applicable | Cetuximab + Cisplatin + Patritumab | Phase Ib/II | Actionable | In a Phase Ib clinical trial, combined Erbitux (cetuximab), Patritumab (U3-1287), and Platinol (cisplatin) treatment was tolerable in patients with recurrent/metastatic head and neck squamous cell carcinoma and resulted in a 47% (7/15) overall response rate (3 complete responses and 4 partial responses), stable disease in 53% (8/15) of patients, a 7.9-month median progression-free survival, and a 13.5-month overall survival (PMID: 30327312; NCT02350712). | 30327312 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CEBPA-51 | Phase I | Actionable | In a Phase I trial, CEBPA-51 treatment demonstrated safety and preliminary efficacy, resulted in increased Cebpa expression in WBC in patients with advanced solid tumors, including hepatocellular carcinoma, colorectal, and fibrolamellar cancer (J Clin Oncol, 36(no. 15_suppl), May 2018, 2509-2509; NCT02716012). | detail... | |
| Unknown unknown | lung non-squamous non-small cell carcinoma | not applicable | Bevacizumab-awwb + Carboplatin + Paclitaxel | FDA approved | Actionable | In a Phase III trial that supported FDA approval, Mvasi (bevacizumab-awwb) treatment resulted in an objective response rate (ORR) of 39% (128/328) similar to the ORR of Avastin (bevacizumab), 41.7% (131/314), in non-squamous NSCLC patients receiving Carboplatin and Paclitaxel as first line therapy, and was pharmacokinetically similar to Avastin (bevacizumab) further supporting extrapolation to other indications (J Clin Oncol 34, 15_sup (2016) 9095, J Clin Oncol 35, 15_sup (2017) 9050, FDA.gov; NCT01966003). | detail... detail... detail... | |
| Unknown unknown | malignant glioma | not applicable | Bevacizumab + Marizomib | Phase I | Actionable | In a Phase I trial, Marizomib (NPI-0052) and Avastin (bevacizumab) combination therapy resulted in complete target lesion response in 14% (5/36), partial response in 25% (9/36), and stable disease in 31% (11/36) of Avastin (bevacizumab)-naive patients with WHO grade IV malignant giloma (Neuro Oncol (2016) 18 (suppl 6): vi13.). | detail... | |
| Unknown unknown | prostate cancer | not applicable | Tisotumab Vedotin | Phase II | Actionable | In a Phase II trial, Tisotumab vedotin treatment resulted in prolonged stable disease in 2 patients with prostate cancer (Journal of Clinical Oncology 33, no. 15_suppl (May 20 2015) 2570-2570; NCT02001623). | detail... | |
| 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 | ovarian cancer | not applicable | Tisotumab Vedotin | Phase II | Actionable | In a Phase II trial, Tisotumab vedotin treatment resulted in prolonged stable disease in a patient with ovarian cancer (Journal of Clinical Oncology 33, no. 15_suppl (May 20 2015) 2570-2570; NCT02001623). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | Poziotinib | Phase I | Actionable | In a Phase I trial, Poziotinib (HM781-36B) displayed favorable pharmacokinetics in patients with advanced solid tumors (PMID: 25377158). | 25377158 | |
| Unknown unknown | stomach cancer | not applicable | Saracatinib | Phase II | Actionable | In a Phase II trial, Saracatinib (AZD0530) treatment resulted in stable disease in 23.5% (4/17) of patients with gastric cancer (PMID: 21400081). | 21400081 | |
| Unknown unknown | 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 | hepatocellular carcinoma | not applicable | Torin 2 | Preclinical | Actionable | In a preclinical study, Torin 2 inhibited proliferation of hepatocellular cells in culture (PMID: 26239364). | 26239364 | |
| Unknown unknown | thyroid gland cancer | not applicable | Sorafenib | FDA approved | Actionable | In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) improved median progression free survival to 10.8 months in metastatic differentiated thyroid cancer patients (PMID: 24768112). | detail... 24768112 | |
| Unknown unknown | glioblastoma multiforme | not applicable | VAL-083 | Phase Ib/II | Actionable | In a Phase I/II trial, treatment with a clinically active dose of VAL-083 resulted in improved overall survival (7.9 vs 2.9 months, HR=0.341, p=0.003) compared to an inactive dose in Avastin (bevacizumab)-refractory glioblastoma multiforme patients (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 2061-2061; NCT01478178). | detail... | |
| Unknown unknown | 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 | acute myeloid leukemia | not applicable | Bortezomib + Tinostamustine | Preclinical - Patient cell culture | Actionable | In a preclinical study, the combination of EDO-S101 and Velcade (bortezomib) decreased viability of primary acute myeloid leukemia cells in culture (PMID: 28753594). | 28753594 | |
| Unknown unknown | mantle cell lymphoma | not applicable | Parsaclisib | Phase Ib/II | Actionable | In a Phase I/II trial, Parsaclisib (INCB050465) treatment demonstrated tolerability and preliminary activity in patients with refractory B-cell malignancies, and resulted in an overall response rate of 67 (6/9), complete response/complete metabolic response rate of 44% (4/9), and median duration of response was not reached in patients with mantle cell lymphoma (PMID: 30803990; NCT02018861). | 30803990 | |
| Unknown unknown | acute myeloid leukemia | not applicable | INCB053914 | Preclinical | Actionable | In a preclinical study, acute myeloid leukemia cells were sensitive to INCB053914, resulting in inhibition of cell proliferation (Cancer Res August 1, 2015 75; 5416). | detail... | |
| Unknown unknown | chronic lymphocytic leukemia | not applicable | Safingol | Preclinical - Patient cell culture | Actionable | In a preclinical study, treatment with Kynacyte (safingol) resulted in increased apoptosis of patient-derived chronic lymphocytic leukemia cells in culture (PMID: 15929099). | 15929099 | |
| Unknown unknown | glioblastoma multiforme | not applicable | AsiDNA | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA inhibited survival of glioblastoma cell lines in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | colorectal cancer | not applicable | INCB054329 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, INCB054329 promoted apoptosis and decreased MYC expression in colorectal cancer cell lines in culture and demonstrated anti-tumor activity in colorectal cancer cell line xenograft models (AACR; 2015. Abstract nr 3525). | detail... | |
| Unknown unknown | Advanced Solid Tumor | not applicable | CFI-401870 | Preclinical | Actionable | In a preclinical study, CFI-401870 inhibited tumor growth in advanced solid tumor xenograft models (PMID: 25763473). | 25763473 | |
| Unknown unknown | colon cancer | not applicable | AsiDNA + Olaparib | Preclinical - Cell culture | Actionable | In a preclinical study, AsiDNA treatment led to increased sensitivity to Lynparza (olaparib), resulting in decreased survival in colon cancer cell lines in culture (PMID: 27559053). | 27559053 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | 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 | ovarian cancer | not applicable | ABT-737 + BEZ235 + Trametinib | Preclinical - Cell culture | Actionable | In a preclinical study, the combination of BEZ235 and Mekinist (trametinib) enhanced the sensitivity of ovarian cancer cells to ABT-737 in culture, resulting in decreased cell viability (PMID: 27980105). | 27980105 | |
| Unknown unknown | hepatocellular carcinoma | not applicable | Apatinib + Camrelizumab | Phase Ib/II | Actionable | In a Phase Ib trial, combined Camrelizumab (SHR-1210) and Apatinib (YN968D1) treatment resulted in an overall response rate of 50% (8/16), a disease control rate of 93.8% (15/16, stable disease or better), a median progression-free survival (PFS) of 5.8 months, and a 9-month PFS rate of 41.0% in evaluable patients with advanced hepatocellular carcinoma (PMID: 30348638; NCT02942329). | 30348638 | |
| Unknown unknown | breast cancer | not applicable | Pemetrexed + 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 breast cancer, with 58% (7/12) of breast cancer patients achieving objective response or stable disease (PMID: 27213589). | 27213589 | |
| Unknown unknown | gastroesophageal junction adenocarcinoma | not applicable | trifluridine/tipiracil hydrochloride | FDA approved | 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). | detail... 30355453 | |
| Unknown unknown | acute myeloid leukemia | not applicable | Venetoclax | Phase II | Actionable | In a Phase II trial, treatment with Venclexta (venetoclax) resulted in a 19% (6/32) overall response rate, a 6% (2/32) complete response, and a 13% (4/32) complete response with incomplete blood count recovery in acute myeloid leukemia patients (PMID: 27520294). | 27520294 | |
| Unknown unknown | colorectal cancer | not applicable | Cetuximab + Fluorouracil + Leucovorin + Oxaliplatin | Phase II | Actionable | In a Phase II trial, Erbitux (cetuximab) in combination with FOLFOX resulted in improved median progression-free survival (6.4 months) comparing to FOLFOX alone (4.5 months) in colorectal cancer patients (hazard ratio =0.81) (PMID: 27002107). | 27002107 | |
| Unknown unknown | diffuse large B-cell lymphoma | not applicable | Ibrutinib + SEL24-B489 | Preclinical | Actionable | In a preclinical study, SEL24-B489 demonstrated a synergistic effect when combined with Ibruvica (ibrutinib) in diffuse large B-cell lymphoma cells in culture, resulting in cell growth inhibition ((Blood 126 (23):706.December 2015). | detail... | |
| Unknown unknown | lung non-small cell carcinoma | no benefit | LY2603618 + Pemetrexed | Phase II | Actionable | In a Phase II trial, the combination treatment of LY2603618 and Alimta (pemetrexed) in patients with non-small lung carcinoma demonstrated similar results when compared to treatment with Alimta (pemetrexed) as a single agent (PMID: 27350064). | 27350064 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | OM-174 | Phase I | Actionable | In a Phase I clinical trial, OM-174 demonstrated safety and limited preliminary efficacy as a monotherapy in patients advanced solid tumors, with 17% (3/17) of patients achieving stable disease (PMID: 23547558). | 23547558 | |
| Unknown unknown | Advanced Solid Tumor | not applicable | GDC-0425 + Gemcitabine | Phase |