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Gene Variant Detail

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Relevant Treatment Approaches

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown 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