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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 multiple myeloma not applicable ABT-348 Preclinical - Cell line xenograft Actionable In a preclinical study, Ilorasertib (ABT-348) displayed efficacy in multiple myeloma cell line xenograft models (PMID: 22935731). 22935731
Unknown unknown clear cell renal cell carcinoma no benefit Bevacizumab + Sorafenib Phase III Actionable In a Phase II clinical trial, treatment with the combination of Nexavar (sorafenib) and Avastin (bevacizumab) did not result in a significant improvement in progression-free survival compared to treatment with Avastin (bevacizumab) as a single agent (9.2 months vs 7.4 months) in patients with renal clear cell carcinoma (PMID: 26077237). 26077237
Unknown unknown triple-receptor negative breast cancer not applicable RhFzd7 Preclinical - Cell line xenograft Actionable In a preclinical study, RhFzd7 treatment inhibited cellular proliferation, invasion, and induced apoptosis in triple-negative breast cancer cell lines in culture, and inhibited tumor growth in cell line xenograft models (PMID: 30096373). 30096373
Unknown unknown colon carcinoma not applicable DRP-104 + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, DRP-104 treatment in combination with an anti-PD-1 antibody resulted in enhanced tumor growth inhibition and survival in a syngeneic mouse model of colon carcinoma (J Immunother Cancer. 2019; 7(Suppl 1): 282, Abs nr: P497). detail...
Unknown unknown Advanced Solid Tumor 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 27% (17/63) of patients with advanced solid tumors (PMID: 30756308; NCT01335269). 30756308
Unknown unknown renal cell carcinoma not applicable Bevacizumab + Everolimus Phase II Actionable In a Phase II trial, of 34 evaluable patients with non clear cell RCC treated with a combination of Afinitor (everolimus) and Avastin (bevacizumab), 9 patients had a partial response, 1 patient experienced a complete response, and 15 had stable disease, and the median PFS was 11 months (PMID: 27601542). 27601542
Unknown unknown follicular lymphoma not applicable Buparlisib + Ibrutinib Phase Ib/II Actionable In a Phase I/II trial, Buparlisib (BKM120) and Imbruvica (ibrutinib) combination treatment resulted in a best overall response rate of 20% (1/5, 1 complete response) in patients with relapsed/refractory follicular lymphoma (J Clin Oncol 36, 2018 (suppl; abstr 7520); NCT02756247). detail...
Unknown unknown renal cell carcinoma not applicable Famitinib Phase I Actionable In a Phase I trial, renal carcinoma patients treated with Famitinib demonstrated a disease control rate of 87.5%, which included 50% (12/24) with a partial response and 37.5% (9/24) with stable disease, and a PFS of 10.7 mo and an OS of 33 mo (PMID: 24238512). 24238512
Unknown unknown pancreatic endocrine carcinoma not applicable Nintedanib Preclinical Actionable In a preclinical study, Ofev (nintedanib) induced tumor cell apoptosis, decreased microvessel density, inhibited tumor growth, and improved survival in transgenic mouse models of pancreatic neuroendocrine carcinoma (PMID: 26206868). 26206868
Unknown unknown pancreatic cancer not applicable PF-00562271 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with PF-00562271 inhibited PTK2 (FAK) phosphorylation and resulted in apoptosis and tumor regression in pancreatic cancer cell line xenograft models (PMID: 18339875). 18339875
Unknown unknown neuroblastoma not applicable iC9.GD2.CAR.IL-15 T-cells Preclinical - Cell line xenograft Actionable In a preclinical study, iC9.GD2.CAR.IL-15 T-cell treatment inhibited tumor growth and improved tumor-free survival in a cell line xenograft model of neuroblastoma expressing the disialoganglioside GD2 (PMID: 30617136). 30617136
Unknown unknown 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 diffuse large B-cell lymphoma not applicable MLN0905 Preclinical Actionable In a preclinical study, MLN0905 treatment resulted in decreased tumor volume in a diffuse large B-cell lymphoma xenograft model (PMID: 22609854). 22609854
Unknown unknown peritoneum cancer not applicable ENMD-2076 Phase II Actionable In a Phase II clinical trial, ENMD-2076 demonstrated efficacy in patients with recurrent, platinum-resistant ovarian, fallopian tube or peritoneal cancers (PMID: 22921155). 22921155
Unknown unknown Advanced Solid Tumor not applicable ON123300 Preclinical Actionable In a preclinical study ON123300 inhibited growth of a variety of human solid tumor cell lines in culture (PMID: 24417566). 24417566
Unknown unknown head and neck squamous cell carcinoma not applicable Lenvatinib + Pembrolizumab Phase Ib/II Actionable Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 46% (10/22) in patients with metastatic head and neck squamous cell carcinoma, with a median duration of response of 8.2 months, and a median progression-free survival of 4.7 months (PMID: 31961766; NCT02501096). 31961766
Unknown unknown 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 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 myelodysplastic syndrome no benefit Rigosertib Sodium Phase III Actionable In a Phase III trial (ONTIME), Rigosertib (ON 01910.Na) did not improve median overall survival compared to best supportive care (8.2 vs 5.9 months, HR=0.87, p=0.33) in patients with myelodysplastic syndrome (PMID: 26968357; NCT01241500). 26968357
Unknown unknown glioblastoma multiforme not applicable Olaparib + Temozolomide Phase II Actionable In a Phase II trial (OPARATIC), 36% (14/39) of evaluable patients with glioblastoma were progression-free at 6 months when treated with the combination therapy of Lynparza (olaparib) and Temodar (temozolomide) (PMID: 32347934; NCT0139057). 32347934
Unknown unknown chronic myeloid leukemia not applicable AG-14361 + Camptothecin Preclinical - Cell culture Actionable In a preclinical study, the addition of AG-14361 to Camptothecin increased sensitivity 2-fold in chronic myeloid leukemia cells in culture, demonstrating growth inhibition and cell death (PMID: 16322308). 16322308
Unknown unknown Sezary's disease not applicable CPI-818 Preclinical - Patient cell culture Actionable In a preclinical study, CPI-818 treatment resulted in dose-dependent growth inhibition of malignant T-cell derived from patients with Sezary's disease (Cancer Res 2019;79(13 Suppl):Abstract nr 1313). detail...
Unknown unknown colorectal cancer not applicable Adavosertib Preclinical - Cell line xenograft Actionable In a preclinical study, Adavosertib (MK-1775) inhibited cell proliferation and promoted DNA damage in a human colorectal cancer cell line in culture, and promoted tumor regression in xenograft models (PMID: 23699655). 23699655
Unknown unknown 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 colon cancer not applicable Panobinostat Preclinical Actionable In a preclinial study, Faridak (panobinostat) enhanced the cytotoxicity of TRAIL, tumor necrosis factor-related apoptosis-inducing ligand, in colon cancer cell lines (PMID: 21965751). 21965751
Unknown unknown Advanced Solid Tumor not applicable Durvalumab + LY2510924 Phase I Actionable In a Phase I trial, the combination of Imfinzi (durvalumab) and LY2510924 demonstrated safety and resulted in stable disease in 44.4% (4/9) of patients with advanced solid tumors, including one patient demonstrating an unconfirmed partial response (PMID: 32219196; NCT02737072). 32219196
Unknown unknown breast cancer not applicable Tamoxifen + Verrucarin A Preclinical Actionable In a preclinical study, Verrucarin A treatment sensitized breast cancer cells to Nolvadex (tamoxifen) in culture, resulting in dose-dependent growth inhibition (PMID: 24743578). 24743578
Unknown unknown 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 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 B-cell lymphoma not applicable KTE-C19 Phase Ib/II Actionable In a Phase I/II trial supporting FDA approval, Yescarta (KTE-C19) treatment resulted in an objective response rate of 82% (92/110), with complete response in 54% (60/110) and partial response in 28% (31/110) of patients with refractory B-cell non-Hodgkin lymphoma (Cancer Res 2017;77(13 Suppl):Abstract nr CT019). detail...
Unknown unknown B-cell lymphoma not applicable KTE-C19 Phase I Actionable In a Phase I trial, KTE-C19 treatment resulted in complete response in 67% (2/3) and partial response in 33% (1/3) of evaluable patients with refractory aggressive B-cell non-Hodgkin lymphoma after 1 month of treatment (Blood 2015 126:3991). detail...
Unknown unknown lung non-small cell carcinoma not applicable Carboplatin + Paclitaxel + Veliparib Phase II Actionable In a Phase II trial, the combination of Veliparib (ABT-888) with Paraplatin (carboplatin) and Taxol (paclitaxel) resulted in both an improved median PFS (5.8 mo vs 4.2 mo) and median OS (11.7 mo vs 9.1 mo) compared to placebo plus Paraplatin (carboplatin) and Taxol (paclitaxel) in patients with non-small cell lung carcinoma (PMID: 27803064). 27803064
Unknown unknown lung non-small cell carcinoma not applicable Aflibercept + Docetaxel Phase III Actionable In a Phase III trial, the combination of Zaltrap (aflibercept) and Taxotere (docetaxel) did not result in improved overall survival compared to Taxotere (docetaxel) with placebo, but did result in an improved overall response rate of 23.3% (94/404) vs. 8.9% (36/406) with Taxotere (docetaxel) plus placebo in non-small cell lung cancer patients that had failed platinum therapy (PMID: 22965962). 22965962
Unknown unknown 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 triple-receptor negative breast cancer not applicable Pemetrexed Disodium + Sorafenib Phase I Actionable In a Phase I clinical trial in patients with advanced solid tumors, the combination of Alimta (pemetrexed) and Nexavar (sorafenib) demonstrated safety and preliminary efficacy in patients with triple-receptor breast cancer (TNBC), with 60% (3/5) of TNBC patients demonstrating an objective response and 100% (5/5) of patients achieving stable disease or better (PMID: 27213589). 27213589
Unknown unknown triple-receptor negative breast cancer not applicable Birabresib + GSK2801 Preclinical - Cell culture Actionable In a preclinical study, Birabresib (OTX015) and GSK2801 treatment synergistically inhibited growth of a triple-negative breast cancer cell line in culture (PMID: 31000582). 31000582
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 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 acute myeloid leukemia not applicable RG7112 Phase I Actionable In a Phase I clinical trial, RG7112 demonstrated clinical activity in acute myeloid leukemia, with complete response in 7% (2/30), complete response with incomplete platelet recovery in 3% (1/30), partial response in 7% (2/30), and stable disease in 30% (9/30) of patients (PMID: 26459177). 26459177
Unknown unknown pancreatic adenocarcinoma not applicable CGP57380 Preclinical Actionable In a preclinical study, CGP57380 decreased migration of pancreatic adenocarcinoma (PDAC) cells in culture and inhibited growth of organoids generated from human PDAC samples (PMID: 26609108). 26609108
Unknown unknown pancreatic cancer not applicable E7449 Phase I Actionable In a Phase I trial, E7449 treatment inhibited Parp activity in peripheral blood mononuclear cells and resulted in stable disease for more than 24 weeks in 7 patients with pancreatic cancer (J Clin Oncol 36, 2018 (suppl; abstr 2505); abstr e19531; NCT01618136). detail...
Unknown unknown triple-receptor negative breast cancer not applicable Foretinib Phase II Actionable In a Phase II trial, Foretinib (GSK1363089) treatment resulted in a clinical benefit rate of 46% (17/37) in triple-receptor negative breast cancer patients, which comprised two patients with a partial response and fifteen patients with stable disease (PMID: 27116183; NCT01147484). 27116183
Unknown unknown acral lentiginous melanoma not applicable Toripalimab Phase I Actionable In a Phase I trial, Toripalimab (JS001) demonstrated safety and preliminary efficacy, resulted in an objective response rate of 23.1% (3/13) and a disease control rate of 46.2% (6/13, 1 complete response, 2 partial response, 3 stable disease) in patients with acral melanoma (PMID: 30642373; NCT02836795). 30642373
Unknown unknown acral lentiginous melanoma not applicable Toripalimab Phase II Actionable In a Phase II trial (POLARIS-01), Toripalimab (JS001) resulted in an objective response rate of 17.3% (22/127, 1 CR, 21 PR) and a disease control rate of 57.5% in melanoma patients, with a median progression-free survival (mPFS) of 3.6 mo and a median overall survival (mOS) of 22.2 mo, better ORR (31.0%, 14.0%, 0%), mPFS (5.5, 3.2, 1.9 mo), and mOS (not reached, 16.9, 10.3 mo) were observed in non-acral cutaneous melanoma (n=29) than in acral (n=50) and mucosal (n=22) subtypes (PMID: 32321714; NCT03013101). 32321714
Unknown unknown 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 lung non-squamous non-small cell carcinoma not applicable Carboplatin + Motesanib Diphosphate Phase III Actionable In a Phase III study, Motesanib plus Paraplatin (carboplatin) or Taxol (paclitaxel) improved overall survival, progression free survival and objective response rate for a subset of Asian patients with advanced nonsquamous non-small cell lung cancer (PMID: 24419239; NCT00460317). 24419239
Unknown unknown Advanced Solid Tumor not applicable PU-H71 Phase I Actionable In a Phase I trial, treatment with PU-H71 was generally well-tolerated, and resulted in stable disease as best response in 35% (6/14) patients with advanced solid tumors (PMID: 28808818; NCT01581541). 28808818
Unknown unknown 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 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 breast cancer not applicable MC180295 Preclinical - Cell culture Actionable In a preclinical study, MC180295 decreased proliferation of a breast cancer cell line in culture (PMID: 30454645). 30454645
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 thyroid gland cancer not applicable Lenvatinib FDA approved Actionable In a Phase III trial (SELECT) that supported FDA approval, treatment with Lenvima (lenvatinib) improved progression free survival (18.3 vs 3.6 months, HR=0.21, p<0.001) and response rates (64.8% vs 1.5%, p<0.001) compared to placebo in patients with radioiodine-refractory differentiated thyroid cancer (PMID: 25671254; NCT01321554). 25671254 detail...
Unknown unknown thyroid gland cancer not applicable Lenvatinib Phase II Actionable In a Phase II clinical trial, Lenvima (lenvatinib) demonstrated partial response in 36% (21/59) of patients, partial response or stable disease in 80% (47/59), and median progression free survival of 9 months in patients with advanced medullary thyroid cancer (PMID: 26311725). 26311725
Unknown unknown Advanced Solid Tumor no benefit MINT1526A Phase I Actionable In a Phase I trial, MINT1526A monotherapy did not result in partial response in patients with advanced solid tumors (PMID: 29905898). 29905898
Unknown unknown Advanced Solid Tumor not applicable CYT01B + Rucaparib Preclinical - Cell culture Actionable In a preclinical study, CYT01B and Rubraca (rucaparib) synergistically inhibited growth of tumor cell lines in culture (AACR Annual Meeting 2019, Abstract 363). 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 neuroblastoma not applicable Prexasertib Preclinical - Cell line xenograft Actionable In a preclinical study, Prexasertib (LY2606368) decreased proliferation and increased apoptosis of neuroblastoma cell lines in culture, and induced tumor regression in neuroblastoma cell line xenograft models (PMID: 28270495). 28270495
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 ovarian cancer not applicable SST0116CL1 Preclinical - Cell line xenograft Actionable In a preclinical study, SST0116CL1 inhibited tumor growth in p-glycoprotein over expressing ovarian cancer cell line xenograft models (PMID: 25096516). 25096516
Unknown unknown head and neck 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 of a head and neck cancer cell line in culture (PMID: 27559053). 27559053
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 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 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 endometrial cancer not applicable Sunitinib Phase II Actionable In Phase II clinical trials, Sutent (sunitinib) demonstrated efficacy in patients with metastatic or recurrent endometrial carcinoma (PMID: 24882554). 24882554
Unknown unknown 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 Advanced Solid Tumor not applicable AC480 Phase I Actionable In a Phase I trial, AC480 demonstrated safety and potential efficacy in patients with several solid tumor types (PMID: 21576284). 21576284
Unknown unknown Advanced Solid Tumor not applicable PRN1371 Preclinical - Pdx & cell culture Actionable In a preclinical study, PRN1371 inhibited proliferation of various FGFR-driven tumor cell lines in culture and inhibited tumor growth in a variety of patient-derived xenograft models with FGFR pathway alterations (AACR; 2016. Abstract nr 1249). detail...
Unknown unknown colon cancer not applicable CVX-060 + Sunitinib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of CVX-060 and Sutent (suntinib) resulted in increased tumor growth inhibition compared to either agent alone in a colon cancer cell line xenograft model (PMID: 21233403). 21233403
Unknown unknown 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 lung cancer no benefit Carboplatin + Cediranib + Paclitaxel Phase I Actionable In a Phase II clinical trial of patients with advanced NSCLC, the combination of Cediranib (AZD-2171) and carboplatin/paclitaxel (CP) chemotherapy resulted in improved response rate over placebo plus CP, but did not improve progression-free survival, and the study did not proceed to Phase III due to toxicity (PMID: 24360368). 24360368
Unknown unknown triple-receptor negative breast cancer not applicable KP372-1 Preclinical - Cell culture Actionable In a preclinical study, KP372-1 induced complete growth inhibition of triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown sarcoma not applicable Conatumumab + Ganitumab Phase Ib/II Actionable In a Phase Ib/II trial, Ganitumab and Conatumumab (AMG 655) combination treatment resulted in stable disease in 33% (5/15) of patients with sarcoma, including one with leiomyosarcoma (PMID: 24816908). 24816908
Unknown unknown prostate cancer not applicable Barasertib + Navitoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of Barasertib (AZD1152) and Navitoclax (ABT-263) resulted in synergy, inhibiting proliferation of prostate cancer cells in culture (PMID: 28179288). 28179288
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 Hodgkin's lymphoma not applicable Pembrolizumab FDA approved Actionable In a Phase II trial (KEYNOTE-087) that supported FDA approval, Keytrude (pembrolizumab) treatment resulted in an overall response rate of 69% (145/210) in patients with relapsed or refractory classical Hodgkin lymphoma (PMID: 28441111; NCT02453594). detail... 28441111
Unknown unknown prostate adenocarcinoma not applicable VT-464 Phase I Actionable In a Phase I study, VT-464 treatment led to safety and tolerability in patients with castration-resistant prostate adenocarcinoma and resulted in a best response of stable disease in 58% (10/17), a partial response in 6% (1/17), and no complete responses (PMID: 30012563; NCT02361086). 30012563
Unknown unknown central nervous system cancer not applicable CC-122 Phase I Actionable In a Phase I trial, CC-122 demonstrated safety and preliminary efficacy in patients with glioblastom and other central nervous system tumors, resulted in a meidan progressin-free survival of 58 days (Neuro Oncol (2016) 18 (suppl 6): vi24.). detail...
Unknown unknown central nervous system cancer not applicable CC-122 Phase I Actionable In a Phase I trial, Avadomide (CC-122) treatment was well tolerated in patients with central nervous system tumors, and resulted in a 6-month progression-free survival (PFS) rate of 83% (5/6) and a PFS ranging from 58 to 1,079 days (PMID: 30201761; NCT01421524). 30201761
Unknown unknown breast cancer not applicable H3B-6545 Preclinical - Cell line xenograft Actionable In a preclinical study, a breast cancer cell line xenograft model was sensitive to treatment with H3B-6545, demonstrating greater antitumor activity compared to Faslodex (fulvestrant) (Cancer Res 2017;77(13 Suppl):Abstract nr DDT01-04). detail...
Unknown unknown Advanced Solid Tumor not applicable Everolimus + Vatalanib Phase I Actionable In a Phase I trial, Vatalanib and Afinitor (everolimus) combination therapy demonstrated acceptable toxicity, resulted in a partial response in 12.9% (9/70) and stable disease in 58.6% (41/70) of patients with advanced solid tumors (PMID: 32328844; NCT00655655). 32328844
Unknown unknown 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 gastrointestinal stromal tumor not applicable Regorafenib FDA approved Actionable In a Phase III clinical trial (GRID) that supported FDA approval, Stivarga (regorafenib) demonstrated safety and improved progression free survival compared to placebo (4.8 vs 0.9 months, HR=0.27, p<0.0001) in patients with gastrointestinal stromal tumors (PMID: 23177515; NCT01271712). detail... 23177515
Unknown unknown head and neck squamous cell carcinoma not applicable Sirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Rapamune (sirolimus) decreased tumor growth and increased survival of cell line xenograft models of head and neck squamous cell carcinoma (PMID: 21520111). 21520111
Unknown unknown breast cancer not applicable Tanibirumab Preclinical - Cell culture Actionable In a preclinical study, Tanibirumab (TTAC-0001) inhibited angiogenesis in a cell culture assay with human breast cancer cells (PMID: 26325365). 26325365
Unknown unknown liposarcoma not applicable Pazopanib Clinical Study Actionable In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 8 weeks and median survival of 7.3 months in patients with liposarcoma (PMID: 26970174). 26970174
Unknown unknown hepatocellular carcinoma not applicable Domatinostat Preclinical - Cell culture Actionable In a preclinical study, Domatinostat (4SC-202) inhibited growth and induced apoptosis in hepatocellular carcinoma cell lines in culture (PMID: 26773495). 26773495
Unknown unknown prostate cancer not applicable ARV-110 Phase I Actionable In a Phase I trial, ARV-110 demonstrated safety and preliminary efficacy, resulted in an over 50% decline of PSA in 13.3% (2/15) of patients with metastatic castrate-resistant prostate cancer (J Clin Oncol 38: 2020 (suppl; abstr 3500); NCT03888612). 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 Advanced Solid Tumor not applicable Ulixertinib Phase I Actionable In a Phase I trial, BVD-523 (Ulixertinib) displayed safety and preliminary efficacy in advanced solid tumor patients (J Clin Oncol 33, 2015 (suppl; abstr 2506)). detail...
Unknown unknown Advanced Solid Tumor not applicable Elenagen Phase Ib/II Actionable In a Phase I/IIa trial, Elenagen treatment resulted in a best response of stable disease in 44% (12/27) of patients with advanced solid tumors (PMID: 28881846). 28881846
Unknown unknown peripheral T-cell lymphoma not applicable Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in partial response in 50% (2/4) of patients with peripheral T-cell lymphoma (PMID: 24852792). 24852792
Unknown unknown estrogen-receptor positive breast cancer not applicable RO6839921 Preclinical - Cell line xenograft Actionable In a preclinical study, RO6839921 demonstrated anti-tumor activity in ER-positive breast cancer cell line xenograft models (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A156). detail...
Unknown unknown Advanced Solid Tumor not applicable Erlotinib + Lumretuzumab Phase Ib/II Actionable In a Phase Ib trial, treatment with the combination of Tarceva (erlotinib) and Lumretuzumab demonstrated minimal clinical efficacy in ERBB3 (HER3)-positive advanced solid tumor patients, resulting in an objective response rate of 4.2% (3/71), including a partial response in a patient with ovarian cancer and in two patients with squamous non-small cell lung carcinoma (PMID: 28600476). 28600476
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 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 Advanced Solid Tumor not applicable Berzosertib + Carboplatin Phase I Actionable In a Phase I trial, Berzosertib (VX-970) and Paraplatin (carboplatin) combination treatment resulted in a best response of stable disease in 71% (15/21) of evaluable patients with advanced solid tumors, including 10 patients with stable disease for 4 months or more and 6 patients with stable disease for 6 months or more (PMID: 32568634; NCT02157792). 32568634
Unknown unknown lung non-small cell carcinoma not applicable Panobinostat + Radiotherapy Phase I Actionable In a Phase I trial, Farydak (panobinostat) in combination with palliative radiotherapy resulted in a disease control rate of 66% (6/9), with a progression-free survival of 3 months and a median overall survival of 9 months in patients with stage III non-small cell lung cancer (PMID: 26317683). 26317683
Unknown unknown biliary tract cancer not applicable M7824 Phase I Actionable In a Phase I trial, Bintrafusp alfa (M7824) treatment in patients with advanced biliary tract cancer resulted in an objective response rate of 20% (6/30), with an additional six patients demonstrating stable disease, and median progression-free survival of 2.5 months, and median overall survival of 12.7 months (PMID: 32461347; NCT02699515). 32461347
Unknown unknown 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 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 pancreatic adenocarcinoma not applicable Gemcitabine + Pimasertib Phase I Actionable In a Phase I trial, Pimasertib in combination with Gemzar (gemcitabine) demonstrated safety and efficacy in metastatic pancreatic adenocarcinoma patients (PMID: 23846936). 23846936
Unknown unknown lung non-small cell carcinoma not applicable TMU-35435 Preclinical - Cell line xenograft Actionable In a preclinical study, TMU-35435 induced cell-cycle arrest and apoptosis and decreased viability of non-small cell lung cancer cell lines in culture, and inhibited tumor growth in a lung cancer cell line xenograft model (PMID: 28233309). 28233309
Unknown unknown 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 lung non-small cell carcinoma not applicable Docetaxel + Ramucirumab FDA approved Actionable In a Phase III trial (REVEL) that supported FDA approval, Cyramza (ramucirumab) and Taxotere (docetaxel) combination treatment resulted in improved overall survival (10.5 vs 9.1 mo, HR=0.86, p=0.023) compared to Taxotere (docetaxel) and a placebo in patients with metastatic non-small cell lung cancer whose disease progressed on prior chemotherapy (PMID: 24933332; NCT01168973). 24933332 detail...
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 melanoma not applicable MIW815 + Spartalizumab Phase Ib/II Actionable In a Phase Ib trial, MIW815 (ADUS100), in combination with Spartalizumab (PDR001), demonstrated preliminary efficacy in PD-1 (PDCD1)-relapsed/refractory melanoma (J of Clin Oncol 37, 2019 (suppl; abstr 2507); NCT03172936). detail...
Unknown unknown multiple myeloma not applicable Dexamethasone + Filanesib Phase Ib/II Actionable In a Phase I/II trial, treatment with the combination of Filansenib (ARRY-520) and dexamethasone in a Phase II cohort resulted in an overall response rate of 15% (8/54), clinical benefit rate of 20% (11/54), and median overall survival of 10.7 months in patients with refractory or relapsed multiple myeloma (PMID: 28817190, NCT00821249). 28817190
Unknown unknown pancreatic cancer not applicable Trametinib Phase I Actionable In a Phase I trial, 42% (11/26) of pancreatic cancer patients demonstrated a decrease in tumor formation when treated with Mekinist (trametinib) (PMID: 22805291). 22805291
Unknown unknown breast cancer not applicable GA001 Preclinical - Cell culture Actionable In a preclinical study, GA001 treatment in breast cancer cells resulted in increased AMPK activity and subsequent autophagy, inhibition of cell proliferation, and induced apoptosis in culture (PMID: 28460359). 28460359
Unknown unknown follicular lymphoma not applicable Lenalidomide + Rituximab FDA approved Actionable In a Phase III trial (AUGMENT) that supported FDA approval, Revlimid (lenalidomide) in combination with Rituxan (rituximab) resulted in significantly improved progression-free survival (39.4 vs 14.1 months, HR=0.46, p<0.001) compared to placebo and Rituxan (rituximab) in patients with relapsed and/or refractory follicular or marginal zone lymphoma (PMID: 30897038; NCT01938001). 30897038 detail...
Unknown unknown Advanced Solid Tumor not applicable Adavosertib + Irinotecan Phase I Actionable In a Phase I trial (ADVL1312), the combination of Adavosertib (MK-1775) and Camptosar (irinotecan) demonstrated tolerability and some preliminary efficacy in pediatric patients with advanced solid tumors, including one Ewing sarcoma patient who achieved a partial response for three months, and one patient with ependymoma and one patient with neuroblastoma who demonstrated stable disease over 12 months and 7.5 months, respectively (PMID: 31857431). 31857431
Unknown unknown colorectal cancer not applicable CBP501 + Cisplatin Preclinical - Cell culture Actionable In a preclinical study, the combination of CBP501 and Platinol (cisplatin) resulted in increased cell death compared to Platinol (cisplatin) alone in a human colorectal cancer cell line in culture (PMID: 17237275). 17237275
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 renal cell carcinoma not applicable Cabozantinib Phase II Actionable In a Phase II trial, Cabometyx (cabozantinib) treatment demonstrated improved median progression-free survival (8.2 vs 5.6 months) and overall response rate (46% vs 18%) over Sutent (sunitinib) in untreated patients with metastatic renal cell carcinoma (ESMO 2016 Congress in Copenhagen, Abstract LBA30_PR). detail... detail...
Unknown unknown renal cell carcinoma not applicable Cabozantinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Cabometyx (cabozantinib) resulted in a median progression-free survival of 7.4 months in patients with renal cell carcinoma, compared to 3.8 months with Afinitor (everolimus), and an objective response rate of 22% (17/76) versus 3% (2/77) with Afinitor (everolimus) (PMID: 26406150). detail... 26406150
Unknown unknown renal cell carcinoma not applicable Cabozantinib FDA approved Actionable In a Phase III trial, final results extending those that supported FDA approval demonstrated Cabometyx (cabozantinib) improved median overall survival compared to Afinitor (everolimus) (21.4 m vs. 16.5 m) and progression-free survival (7.4 m vs. 3.9 m), and led to a 17% (57/330) objective response rate vs. 3% (11/328) with Afinitor (everolimus) in renal cell carcinoma patients (PMID: 27279544). 27279544 detail...
Unknown unknown breast cancer not applicable COG112 Preclinical Actionable In a preclinical study, breast cancer cells treated with COG112 demonstrated a decrease in cell proliferation and cell migration in culture (PMID: 21297667). 21297667
Unknown unknown glioblastoma multiforme not applicable LB-100 Preclinical Actionable In a preclinical study, glioblastoma cells treated with a combination of LB100 and radiotherapy resulted in inhibition of Pp2a activity, decreased tumor growth, and increased cell survival (PMID: 25939762). 25939762
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 osteosarcoma not applicable AZD7762 + VE-821 Preclinical Actionable In a preclinical study, osteosarcoma cells treated with VE-821 resulted in a synthetic lethal effect when combined with AZD7762, thereby demonstrating reduced cell survival in culture (PMID: 26748709). 26748709
Unknown unknown prostate adenocarcinoma not applicable ONC201 Clinical Study Actionable In a clinical case study, a prostate adenocarcinoma patient demonstrated extended stable disease for 27 weeks when treated with ONC201 (TIC-10) (PMID: 28331050). 28331050
Unknown unknown hematologic cancer not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in disease burden reduction in 73% (53/73), complete response in 4% (3/73), and partial response in 41% (30/73) of patients with chronic lymphocytic leukemia and lymphoma (PMID: 29475723; NCT01767766). 29475723
Unknown unknown hematologic cancer not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) demonstrated safety in patients with advanced hematological malignancies and preliminary efficacy in patients with chronic lymphocytic leukemia and non-Hodgkin's lymphoma (J Clin Oncol (Meeting Abstracts) 2015 33: 7069). detail...
Unknown unknown acute lymphoblastic leukemia not applicable Natalizumab Preclinical - Pdx Actionable In a preclinical study, Tysabri (natalizumab) treatment alone or in combination with chemotherapy prolonged survival in patient-derived xenograft models of acute lymphocytic leukemia (PMID: 23319569). 23319569
Unknown unknown Advanced Solid Tumor not applicable PTC596 Phase I Actionable In a Phase I trial, PTC596 demonstrated safety and preliminary efficacy, resulted in stable disease in 16% (5/31) of patients with advanced solid tumors (J Clin Oncol 35, 2017 (suppl; abstr 2574)). detail...
Unknown unknown B-cell adult acute lymphocytic leukemia not applicable Disarib + Paclitaxel Preclinical - Cell culture Actionable In a preclinical study, Taxol (paclitaxel) efficacy was enhanced when combined with Disarib, resulting in greater cell death compared to either agent alone in B-cell acute lymphocytic leukemia cells in culture (PMID: 27693384). 27693384
Unknown unknown ovarian cancer not applicable SCH 58500 Phase Ib/II Actionable In a Phase I/II trial, SCH 58500 demonstrated safety and preliminary efficacy, resulted in measurable transgene expression in 85% (17/20) of biopsy samples, and more than 50% reduction of CEA level in 50% (8/16) of ovarian cancer patients who received three cycles of treatment (PMID: 12082455). 12082455
Unknown unknown ovarian cancer not applicable SCH 58500 Phase I Actionable In a Phase I trial, treatment with multiple doses of SCH 58500 in combination with chemotherapy resulted in a median survival of 12-13 months in heavily pretreated ovarian cancer patients, with 10 patients surviving more than 20 months (PMID: 12082456). 12082456
Unknown unknown colon carcinoma not applicable VLX600 Preclinical - Cell line xenograft Actionable In a preclinical study, VLX600 treatment induced mitochondrial dysfunction, led to autophagy induced cell death, reduced clonogenicity and viability of quiescent cells in a spheroid culture, and inhibited proliferation of colon carcinoma cells in culture, and inhibited tumor growth in cell line xenograft models (PMID: 24548894). 24548894
Unknown unknown acute myeloid leukemia not applicable Azacitidine + Nivolumab Phase II Actionable In a Phase II trial, the combination treatment of Vidaza (azacitidine) and Opdivo (nivolumab) resulted in an overall response rate of 33% (23/70) in patients with relapsed/refractory acute myeloid leukemia, including four with complete remission, 11 with complete remission with incomplete recovery counts, one partial response, and seven with hematological improvement, and led to a median overall survival of 6.3 months (PMID: 30409776; NCT02397720). 30409776
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 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 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 lung non-small cell carcinoma not applicable Belinostat + Carboplatin + Paclitaxel Phase I Actionable In a Phase I trial, the combination therapy of Beleodaq (belinostat), Paraplatin (carboplatin, and Taxol (paclitaxel) in patients with non-small cell lung carcinoma resulted in a 5.7 month median progression-free survival, a partial response in 35% (8/23) patients, and stable disease in 17% (4/23) of patients (Journal of Thoracic Oncology, 2017, vol 12:1S, abstract #P2.03a-003). detail...
Unknown unknown 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 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 triple-receptor negative breast cancer not applicable SY-1365 Preclinical - Pdx & cell culture Actionable In a preclinical study, SY-1365 induced rapid apoptosis in triple-receptor negative breast cancer cells in culture, and resulted in tumor growth inhibition in patient-derived xenograft models (Proceedings of the AACR, Vol 58, April 2017, Abstract # 1151). detail...
Unknown unknown urinary bladder cancer not applicable PD-0325901 + PF-04691502 Preclinical - Pdx Actionable In a preclinical study, PD-0325901, in combination with PF-04691502, delayed tumor growth in patient-derived xenograft models of bladder cancer (PMID: 24442130). 24442130
Unknown unknown peritoneal carcinoma not applicable Paclitaxel + TVB-2640 Phase I Actionable In a Phase I trial, TVB-2640 and Taxol (paclitaxel) combination treatment resulted in partial response and a 58% reduction in CA-125 level in a peritoneal carcinoma patient (J Clin Oncol 34, 2016 (suppl; abstr 2512)). detail...
Unknown unknown ovarian cancer not applicable MIV-818 Preclinical - Cell culture Actionable In a preclinical study, MIV-818 inhibited proliferation of ovarian cancer cell in culture, and enhanced immune-mediated tumor cell killing in a co-culture of ovarian cancer cells and activated peripheral blood mononuclear cells (Mol Cancer Ther 2019;18(12 Suppl):Abstract nr C056). detail...
Unknown unknown lung non-small cell carcinoma not applicable ALT-803 + Nivolumab Phase Ib/II Actionable In a Phase Ib trial, the addition of ALT-803 to Opdivo (nivolumab) treatment at the time of relapse resulted in an objective response in two patients with non-small cell lung carcinoma, demonstrating antitumor activity, and a third patient who initially responded to the combination, progressed, enrolled on a trial due to a KRAS mutation, but did not respond, and was then retreated with the ALT-803 and Opdivo (nivolumab) combination, demonstrating a 100% decrease in the target lesion (PMID: 29628312). 29628312
Unknown unknown ovarian carcinoma no benefit Denileukin diftitox + Sirolimus Preclinical Actionable In a preclinical study, an ovarian carcinoma mouse model did not respond to the combination of Ontak (denileukin diftitox) and Rapamune (sirolimus), demonstrating no decrease in tumor burden (PMID: 27737881). 27737881
Unknown unknown melanoma not applicable Toripalimab Phase II Actionable In a Phase II trial (POLARIS-01), Toripalimab (JS001) resulted in an objective response rate of 17.3% (22/127, 1 CR, 21 PR) and a disease control rate of 57.5% in melanoma patients, with a median progression-free survival (mPFS) of 3.6 mo and a median overall survival (mOS) of 22.2 mo, better ORR (31.0%, 14.0%, 0%), mPFS (5.5, 3.2, 1.9 mo), and mOS (not reached, 16.9, 10.3 mo) were observed in non-acral cutaneous melanoma (n=29) than in acral (n=50) and mucosal (n=22) subtypes (PMID: 32321714; NCT03013101). 32321714
Unknown unknown melanoma not applicable Toripalimab Phase I Actionable In a Phase I trial, Toripalimab (JS001) demonstrated safety and preliminary efficacy, resulted in an objective response rate of 18.2% (4/22) and a disease control rate of 45.5% (10/22, 1 complete response, 4 partial response, 6 stable disease) in patients with advanced melanoma (PMID: 30642373; NCT02836795). 30642373
Unknown unknown melanoma not applicable Toripalimab Phase I Actionable In a Phase I trial, JS001 demonstrated safety and preliminary activity in patients with melanoma, renal cell carcinoma, or urothelial carcinoma, with an overall response rate (ORR) of 22% (7/32; 1 complete response (melanoma), 6 partial responses), and an ORR of 20% and 25% and DCR of 53% and 50% in patients with acral or mucosal melanoma, respectively (J Clin Oncol 35, 2017 (suppl; abstr 3067)). detail...
Unknown unknown Advanced Solid Tumor not applicable Talazoparib Preclinical - Cell line xenograft Actionable In a preclinical study, the Talazoparib (BMN-673) selective PARP1/2 inhibitor demonstrated antitumor activity on a variety of cell lines and xenografts with defects in DNA repair (PMID: 23881923). 23881923
Unknown unknown 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 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 Advanced Solid Tumor not applicable Milademetan Tosylate Phase I Actionable In a Phase I trial, DS-3032b demonstrated safety and some preliminary activity in patients with advanced solid tumors, with 77% (20/26) of patients achieving stable disease (J Clin Oncol 34, 2016 (suppl; abstr 2581)). detail...
Unknown unknown breast cancer not applicable GDC-0349 Preclinical - Cell line xenograft Actionable In a preclinical study, the mTOR inhibitor GDC-0349 inhibited tumor growth in cell line xenograft models of solid tumors, including breast cancer (PMID: 24900569). 24900569
Unknown unknown prostate cancer not applicable Docetaxel + Everolimus Phase I Actionable In a Phase I study, Afinitor (everolimus), in combination with Taxotere (docetaxel), demonstrated safety, but minimal efficacy in patients with prostate cancer (PMID: 25450031). 25450031
Unknown unknown colorectal cancer not applicable HL-085 + OKI-005 Preclinical - Cell culture Actionable In a preclinical study, HL085 and OKI-005 demonstrated synergistic activity in 3 of 6 colorectal cancer cell lines, and increased immunogenicity of tumor cells in culture (Cancer Res 2019;79(13 Suppl):Abstract nr 4753). detail...
Unknown unknown 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 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 renal Wilms' tumor not applicable BMS-754807 Preclinical - Cell line xenograft Actionable In a preclinical study, BMS-754807 treatment resulted in significant tumor growth delay in 100% (3/3) of cell line xenograft models of Wilms tumor (PMID: 21298745). 21298745
Unknown unknown triple-receptor negative breast cancer not applicable Carboplatin + JQ1 Preclinical - Cell culture Actionable In a preclinical study, the addition of JQ1 to Paraplatin (carboplatin) resulted in a synergistic effect in triple-receptor negative breast cancer cells in culture (PMID: 27256375). 27256375
Unknown unknown 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 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 multiple myeloma not applicable Dexamethasone + Pomalidomide FDA approved Actionable In a Phase III trial (MM-003) that supported FDA approval, combination of Pomalyst (pomalidomide) and low-dose dexamethasone resulted in improved median progression-free survival (4.0 vs 1.9 mo, HR=0.48, p<0.0001) compared to high-dose dexamethasone in patients with multiple myeloma whose disease progressed on 2 or more therapies (PMID: 24007748; NCT01311687). 24007748
Unknown unknown stomach cancer not applicable Capecitabine + Oxaliplatin + Toripalimab Phase Ib/II Actionable In a Phase I/II trial, Toripalimab (JS001) in combination with Eloxatin (oxaliplatin) and Xeloda (capecitabine) resulted in an objective response rate (ORR) of 66.7% (12/18, 1 complete response, 11 partial responses) and a disease control rate of 88.9% (16/18) in patients with chemotherapy-naive advanced gastric cancer, with a progression-free survival of 5.8 months and overall survival not reached, same ORRs (66.7%) were observed in CD274 (PD-L1)-positive and -negative groups (PMID: 31236579; NCT02915432). 31236579
Unknown unknown colorectal cancer no benefit Utomilumab Phase I Actionable In a Phase I trial, Utomilumab (PF-05082566) treatment resulted in no overall objective response (0/12) in patients with colorectal cancer (PMID: 29549159; NCT01307267). 29549159
Unknown unknown pancreatic cancer not applicable Berzosertib + Radiotherapy Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with Berzosertib (VX-970) enhanced radiotherapy efficacy in pancreatic cells resulting in apoptotic induction in culture and DNA damage and tumor growth delay in cell line xenograft models (PMID: 23222511). 23222511
Unknown unknown acute lymphoblastic leukemia not applicable Prexasertib Preclinical - Cell culture Actionable In a preclinical study, Prexasertib (LY2606368) decreased proliferation of several pediatric tumor cell lines in culture, including acute lymphoblastic leukemia cell lines (PMID: 28270495). 28270495
Unknown unknown 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 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 glioblastoma multiforme not applicable Pexidartinib Phase II Actionable In a Phase II trial, Pexidartinib (PLX3397) in combination with radiation therapy and Temodar (temozolomide) demonstrated safety and improved efficacy over standard therapy, resulting in a median progression-free survival of 9.7 months and an estimated overall survival of 25.1 months in newly diagnosed glioblastoma multiforme patients (Neuro Oncol (2016) 18 (suppl 6): vi6). 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 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 CFI-401870 Preclinical Actionable In a preclinical study, CFI-401870 inhibited tumor growth in advanced solid tumor xenograft models (PMID: 25763473). 25763473
Unknown unknown lung small cell carcinoma not applicable Cisplatin + Durvalumab + Etoposide FDA approved Actionable In a Phase III (CASPIAN) trial that supported FDA approval, Imfinzi (durvalumab) in combination with Vepesid (etoposide) and Paraplatin (carboplatin) or Platinol (cisplatin) resulted in significantly improved overall survival (13.0 vs 10.3 mo, HR=0.73, p=0.0047) compared to platinum-etoposide therapy in patients with untreated extensive-stage small cell lung cancer (PMID: 31590988; NCT03043872). 31590988
Unknown unknown triple-receptor negative breast cancer not applicable KW-2450 + Selumetinib Preclinical Actionable In a preclinical study, the combination of KW-2450 and Selumetinib worked synergistically to inhibit growth of triple-negative breast cancer cells in culture (PMID: 26443806). 26443806
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 glioblastoma multiforme not applicable Adavosertib Phase 0 Actionable In a Phase 0 trial, Adavosertib (MK-1775) demonstrated safety and preliminary efficacy, resulted in good brain tumor penetration and Wee1 pathway inhibition in intraoperatively collected tumor samples from patients with first-recurrence glioblastoma (PMID: 29798906). 29798906
Unknown unknown Advanced Solid Tumor not applicable MEDI0639 Phase I Actionable In a Phase I trial, MEDI0639 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 33, 2015 (suppl; abstr 3024)). detail...
Unknown unknown colon cancer not applicable Doxorubicin + NU7441 Preclinical - Cell culture Actionable In a preclinical study, NU7441 increased sensitivity of colon cancer cell lines to Adriamycin (doxorubicin), resulting in reduced cell survival in culture (PMID: 16707462). 16707462
Unknown unknown prostate cancer not applicable GDC-0349 Preclinical - Cell line xenograft Actionable In a preclinical study, the mTOR inhibitor GDC-0349 demonstrated inhibition of tumor growth in cell line xenograft models of solid tumors, including prostate cancer (PMID: 24900569). 24900569
Unknown unknown Advanced Solid Tumor not applicable NHS-IL12 Phase I Actionable In a Phase I trial, NHS-IL12 was well tolerated in patients with metastatic advanced solid tumors and resulted in stable disease in 50% (15/30) of patients with measurable disease, with durable stable disease in 5 patients (two prostate, one colorectal, one breast, and one chordoma) lasting more than 6 months (PMID: 30131389; NCT01417546). 30131389
Unknown unknown colorectal cancer not applicable CVX-060 Preclinical - Cell line xenograft Actionable In a preclinical study, CVX-060 did not inhibit primary tumor growth in two colorectal cancer cell line xenograft models, but reduced liver metastases in a xenograft model of colorectal cancer metastasis (PMID: 27651308). 27651308
Unknown unknown triple-receptor negative breast cancer not applicable Cisplatin + SR-4835 Preclinical - Pdx Actionable In a preclinical study, SR-4835 and Platinol (cisplatin) combination treatment induced apoptosis, inhibited proliferation and tumor growth, leading to tumor regression in an orthotopic patient-derived xenograft (PDX) model of triple-negative breast cancer (PMID: 31668947). 31668947
Unknown unknown hepatocellular carcinoma not applicable Novaferon Preclinical - Cell line xenograft Actionable In a preclinical study, Novaferon treatment induced cell cycle arrest and apoptosis, and inhibited tumor growth in a hepatocellular carcinoma cell line xenograft model (PMID: 24467885). 24467885
Unknown unknown acute myeloid leukemia not applicable Azacitidine + Hu5F9-G4 Phase I Actionable In a Phase Ib trial, Magrolimab (Hu5F9-G4) and Vidaza (azacitidine) combination therapy was well tolerated, and resulted in complete response in 40% (10/25), complete response with incomplete hematologic recovery in 16% (4/25), partial response in 4% (1/25), morphologic leukemia-free state in 4% (1/25), and stable disease in 32% (8/25) of patients with acute myeloid leukemia unfit for chemotherapy (J Clin Oncol 38: 2020 (suppl; abstr 7507); NCT03248479). detail...
Unknown unknown Advanced Solid Tumor not applicable Ganetespib Preclinical - Cell line xenograft Actionable In a preclinical study, Ganetespib demonstrated potent anti-tumor effects against a variety of advanced solid tumor cell types and in cell line xenograft models (PMID: 22144665). 22144665
Unknown unknown 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 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 pancreatic cancer not applicable 2G8 Preclinical Actionable In a preclinical study, inhibition of stromal Tgfbr2 with the murine antibody 2G8 resulted in decreased proliferation, increased apoptosis and reduced metastasis in pancreatic tumor cell line xenograft models (PMID: 25060520). 25060520
Unknown unknown Advanced Solid Tumor not applicable Emibetuzumab + Ramucirumab Phase Ib/II Actionable In a Phase Ib/II trial, Emibetuzumab (LY2875358) and Cyramza (ramucirumab) combination treatment demonstrated safety, resulted in an objective response rate of 5.2% (5/97, 5 partial response) and a disease control rate of 62% (60/97) in patients with advanced solid tumors (PMID: 31142504; NCT02082210). 31142504
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 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 pancreatic adenocarcinoma no benefit Bevacizumab + Erlotinib + Gemcitabine Phase III Actionable In a Phase III trial, treatment with the combination of Avastin (bevacizumab), Tarceva (erlotinib), and Gemzar (gemcitabine) did not result in a significant increase in OS in pancreatic adenocarcinoma patients compared to the combined treatment of Gemzar (gemcitabine), Tarceva (erlotinib), and placebo (PMID: 19307500). 19307500
Unknown unknown acute myeloid leukemia not applicable Cytarabine + Daunorubicin + Debio 1143 Phase I Actionable In a Phase I clinical trial, the combination of Debio1143 (AT-406), daunorubicin, and cytarabine was well tolerated, and resulted in complete remission in 38% (11/23) of patients with poor-risk acute myeloid leukemia, with 6 of those patients (56%) developing relapse during the study period (PMID: 25842225). 25842225
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 diffuse large B-cell lymphoma not applicable Itacitinib + Parsaclisib Preclinical - Cell line xenograft Actionable In a preclinical study, Parsaclisib (INCB050465) and INCB039110 combination treatment inhibited proliferation of diffuse large B-cell lymphoma cell lines in culture and induced tumor regression in xenograft models (Cancer Res August 1, 2015 75; 2671). detail...
Unknown unknown breast cancer not applicable Carboplatin + E7449 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of E7449 and Paraplatin (carboplatin) synergized to inhibit tumor growth in a human breast cancer cell line xenograft model (PMID: 26513298). 26513298
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 Ewing sarcoma not applicable Niraparib + SN-38 Preclinical - Cell culture Actionable In a preclinical study, treatment with SN-38 combined with Zejula (niraparib) demonstrated synergism in Ewing sarcoma cells in culture, resulting in reduced cell viability (PMID: 26438158). 26438158
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 bone giant cell tumor not applicable Denosumab FDA approved Actionable In a Phase II clinical trial that supported FDA approval, Xgeva (denosumab) treatment resulted in an overall objective response rate of 25% (47/187) in patients with giant cell bone tumor, with a median duration of response of 20 months among patients demonstrating response (PMID: 25617146). detail... 25617146
Unknown unknown meningioma not applicable Everolimus + Octreotide Phase II Actionable In a Phase II trial (CEVOREM), the combination therapy of Afinitor (everolimus) and Sandostatin Lar Depot (octreotide acetate) in patients with a recurrent meningioma resulted in a 6 month progression-free survival of 55% (11/20), 6 month and 12 month overall survivals of 90% (18/20) and 75% (15/20), respectively, and a decreased tumor growth rate in 78% of patients at 3 months (PMID: 31969329; NCT02333565). 31969329
Unknown unknown colorectal cancer not applicable Merestinib Phase I Actionable In a Phase I trial, LY2801653 demonstrated safety and preliminary efficacy in patients with advanced solid tumors, including patients with colorectal cancer (Cancer Res October 1, 2014 74:CT237). detail...
Unknown unknown Advanced Solid Tumor not applicable SF1126 Phase I Actionable In a Phase I trial, SF1126 demonstrated safety and preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 22921184). 22921184
Unknown unknown Advanced Solid Tumor not applicable Sacituzumab govitecan-hziy Phase Ib/II Actionable In a Phase I/II trial (IMMU-132-01), Trodelvy (sacituzumab govitecan-hziy) demonstrated safety and preliminary efficacy, resulted in a partial response in 8% (2/25) and stable disease in 64% (16/25) of patients with advanced solid tumors, with a time-to-progression of 3.6 months (PMID: 25944802; NCT01631552). 25944802
Unknown unknown ovarian cancer not applicable Cisplatin + KPT-185 Preclinical - Patient cell culture Actionable In a preclinical study, the combination of KPT-185 and Platinol (cisplatin) worked synergistically to decrease viability of platinum-resistant and platinum-sensitive immortalized and patient-derived human ovarian cancer cell lines in culture (PMID: 27649553). 27649553
Unknown unknown Advanced Solid Tumor not applicable JIB-04 Preclinical Actionable In a preclinical study, JIB-04, a pan Jumonji histone demethylase inhibitor, reduced cell proliferation, and induced apoptosis in multiple cancer cell types while reducing tumor burden and increasing survival in mouse xenograft models (PMID: 23792809). 23792809
Unknown unknown colon adenocarcinoma not applicable Epoetin beta + LFM-A13 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of Epoetin beta (erythropoietin beta) and LFM-A13 resulted in decreased BTK and AKT expression, reduced proliferation, and increased apoptosis compared to either agent alone in colon adenocarcinoma cell lines in culture, and inhibited tumor growth in xenograft models (PMID: 29160911). 29160911
Unknown unknown mantle cell lymphoma not applicable Bendamustine + Rituximab Phase III Actionable In a Phase III trial, Rituximab and Bendamustine combination therapy resulted in improved 5-year progression free survival rate (66.5% vs 55.8%), but no difference in overall survival rate (81.7% vs 85%) compared to R-CHOP/R-CVP regimen in patients with indolent non-Hodgkin lymphoma or mantle cell lymphoma (J Clin Oncol 35, 2017 (suppl; abstr 7500)). detail...
Unknown unknown endometrial 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, and a response rate of 50% (4/8) in endometrial cancer patients (Ann Oncol 2017, Vol 28, Suppl 5, Abstract #391P; NCT02208375). detail...
Unknown unknown esophageal cancer not applicable Futibatinib Phase I Actionable In a Phase I trial, TAS-120 treatment resulted in clinical response in an esophageal cancer patient (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 372PD). detail...
Unknown unknown 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 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 neuroendocrine tumor not applicable Cixutumumab + Everolimus + Octreotide acetate Phase I Actionable In a Phase I trial, patients with neuroendocrine tumors treated with the combination of Cixutumumab, Sandostatin Lar Depot (octreotide acetate), and Afinitor (everolimus) demonstrated some efficacy, however, the drug combination did result in multiple non-dose limiting toxicities preventing long term tolerance (PMID: 25900182). 25900182
Unknown unknown malignant glioma not applicable RES-529 Preclinical - Cell line xenograft Actionable In a preclinical study, RES-529 (Palomid 529) inhibited tumor growth and angiogenesis in glioma cell line xenograft models (PMID: 19010932). 19010932
Unknown unknown stomach cancer not applicable AZD6738 + Cisplatin Preclinical - Cell culture Actionable In a preclinical study, the addition of AZD6738 to Platinol (cisplatin) treatment in a gastric cancer cell line in culture resulted in enhanced chemotherapeutic sensitivity, demonstrating a synergistic effect (PMID: 28138034). 28138034
Unknown unknown lung 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 33.3% (1/3) of patients with lung cancer (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). detail...
Unknown unknown B-cell lymphoma not applicable Fenretinide Phase I Actionable In a Phase I trial, Fenretinide treatment resulted in stable disease in a patient with B-cell lymphoma (PMID: 28420721). 28420721
Unknown unknown breast cancer not applicable NSC23766 Preclinical - Cell culture Actionable In a preclinical study, NSC23766 induced cell-cycle arrest and inhibited growth of breast cancer cell lines in culture (PMID: 20515940). 20515940
Unknown unknown pancreatic adenocarcinoma not applicable GSK1904529A Preclinical - Cell line xenograft Actionable In a preclinical study, GSK1904529A treatment resulted in inhibition of tumor growth by 52% in xenograft models of pancreas adenocarcinoma (PMID: 19383820). 19383820
Unknown unknown 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 thyroid gland cancer not applicable LY2874455 Phase I Actionable In a Phase I trial, a patient with thyroid cancer demonstrated stable disease when treated with LY2874455 (PMID: 28589492). 28589492
Unknown unknown breast cancer not applicable WANT3 Preclinical - Cell culture Actionable In a preclinical study, WANT3 treatment resulted in suppression of cell invasion of breast cancer cells in culture (PMID: 27432794). 27432794
Unknown unknown osteosarcoma not applicable YW3-56 Preclinical Actionable In a preclinical study, YW3-56 inhibited proliferation of a human osteosarcoma cell line in culture (PMID: 25612620). 25612620
Unknown unknown urinary bladder cancer not applicable Celecoxib Preclinical - Cell culture Actionable In a preclinical study, Celebra (celecoxib) treatment resulted in decreased cell viability in multiple human bladder cancer cell lines in culture (PMID: 27406983). 27406983
Unknown unknown 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 non-small cell carcinoma not applicable Ganetespib + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, Ganetespib increased the sensitivity of various non-small cell lung cancer cell lines to radiation in culture, resulting in increased DNA damage and cell-cycle arrest and decreased cell survival (PMID: 27354472). 27354472
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 lung adenocarcinoma not applicable AC-93253 iodide + Gefitinib Preclinical - Cell culture Actionable In a preclinical study, AC-93253 iodide combined with Iressa (gefitinib) resulted in a synergistic effect, demonstrating growth inhibition of an Iressa (gefitinib)-resistant lung adenocarcinoma cell line in culture (PMID: 29132432). 29132432
Unknown unknown pulmonary neuroendocrine tumor not applicable Spartalizumab Case Reports/Case Series Actionable In a Phase I trial, Spartalizumab (PDR001) treatment in a patient with an atypical carcinoid lung tumor with initial CD8-positive lymphocyte tumor infiltration of 5.6% resulted in a partial response lasting 8.5 months, with an increase in CD8-positive lymphocyte infiltration to 37.6% after two weeks and subsequent reduction of target lesion size (PMID: 32179633; NCT02404441). 32179633
Unknown unknown Advanced Solid Tumor not applicable MEDI3617 Phase I Actionable In a Phase I trial, MEDI3617 monotherapy resulted in no objective response (0/25) and stable disease in 52% (13/25) of patients with advanced solid tumors, with a median progression-free survival of 1.4 months (PMID: 29559563; NCT01248949). 29559563
Unknown unknown pancreatic ductal adenocarcinoma not applicable JQ1 Preclinical - Pdx & cell culture Actionable In a preclinical study, treatment with JQ1 in patient derived xenograft (PDX) models of pancreatic ductal adenocarcinoma (PDAC) demonstrated tumor growth suppression and in PDAC cells, resulted in inhibition of Hedgehog and TGF-beta signaling and a decrease in desmoplastic stroma (PMID: 27528027). 27528027
Unknown unknown acute myeloid leukemia not applicable INCB054329 + INCB059872 Preclinical - Cell line xenograft Actionable In a preclinical study, combination of INCB054329 and INCB059872 resulted in enhanced apoptosis in acute myeloid leukemia cells in culture and in cell-line xenograft models (Cancer Res 2016;76(14 Suppl):Abstract nr 4702). detail...
Unknown unknown multiple myeloma not applicable Bortezomib + Daratumumab + Melphalan + Prednisone FDA approved Actionable In a Phase III trial (ALCYONE) that supported FDA approval, the combination of Darzalex (daratumumab), Velcade (bortezomib), Alkeran (melphalan), and Adasone (prednisone) resulted in improved 18-month progression-free survival rate (71.6% vs 50.2%, HR=0.50, p<0.001) and overall response rate (90.9% vs 73.9%, p<0.001) compared to control in newly diagnosed multiple myeloma patients ineligible for autologous stem-cell transplantation (PMID: 29231133; NCT02195479). detail... 29231133
Unknown unknown 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 leukemia not applicable GNE-272 Preclinical - Cell culture Actionable In a preclinical study, GNE-272 treatment resulted in anti-proliferative activity in leukemia cell lines in culture (PMID: 27682507). 27682507
Unknown unknown 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 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 renal carcinoma not applicable Hydroxyurea + MU380 Preclinical - Cell culture Actionable In a preclinical study, the addition of MU380 resulted in increased sensitivity to Droxia (hydroxyurea) in a renal cell carcinoma cell line in culture, leading to decreased proliferation (PMID: 28619751). 28619751
Unknown unknown Advanced Solid Tumor not applicable 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 ovarian cancer not applicable BAY1161909 Preclinical Actionable In a preclinical study, BAY1161909 demonstrated moderate efficacy in ovarian cancer xenograft models (PMID: 26832791). 26832791
Unknown unknown 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 head and neck squamous cell carcinoma not applicable Prexasertib Preclinical - Cell culture Actionable In a preclinical study, head and neck squamous cell carcinoma cell lines, either human papilloma virus positive or negative, demonstrated decreased cell proliferation in culture when treated with Prexasertib (LY2606368) (PMID: 28138028). 28138028
Unknown unknown head and neck squamous cell carcinoma not applicable Prexasertib Phase I Actionable In a Phase I trial, Prexasertib (LY2606368) treatment resulted in partial response in a patient with head and neck squamous cell carcinoma (PMID: 27044938; NCT0115790). 27044938
Unknown unknown head and neck squamous cell carcinoma not applicable Prexasertib Phase Ib/II Actionable In a Phase Ib trial, treatment with Prexasertib (LY2606368) resulted in an overall response rate of 5% (3/57, all partial responses), clinical benefit rate (complete response+partial response+stable disease) of 49% (28/57), and a median progression-free survival of 1.6 months in patients with head and neck squamous cell carcinoma (PMID: 29643063; NCT0115790). 29643063
Unknown unknown 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 head and neck squamous cell carcinoma not applicable Cisplatin + Docetaxel + Fluorouracil + Toripalimab Phase II Actionable In a Phase II trial, Toripalimab (JS001) in combination with Taxotere (docetaxel), Adrucil (fluorouracil), and Platinol (cisplatin) resulted in an objective response rate of 33.3% (1/3) and a disease control rate of 100% (3/3) in patients with metastatic head and neck squamous cell carcinoma, with a median duration of response of 7.1 months (J Clin Oncol 38: 2020 (suppl; abstr e15083); NCT02915432). detail...
Unknown unknown ovary epithelial cancer not applicable Bevacizumab + Niraparib Phase I Actionable In a Phase I trial, Avastin (bevacizumab) and Zejula (niraparib) combination treatment resulted in complete response in 8% (1/12), partial response in 33% (4/12), and a disease control rate of 91% in platinum-sensitive ovarian cancer patients (J Clin Oncol 34, 2016 (suppl; abstr 5555)). detail...
Unknown unknown 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 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 ovarian cancer not applicable Axitinib + Carboplatin + Paclitaxel Phase I Actionable In a Phase I clinical trial, Inlyta (axitinib) in combination with paclitaxel and carboplatin, demonstrated safety and efficacy in patients with advanced solid tumors including ovarian cancers (PMID: 22990652). 22990652
Unknown unknown hepatocellular carcinoma not applicable Cabozantinib FDA approved Actionable In a Phase III trial (CELESTIAL) that supported FDA approval, Cabometyx (cabozantinib) significantly improved overall survival (10.2 vs 8.0 months, HR=0.76, p=0.005) and progression-free survival (5.2 vs 1.9 months, HR=0.44, p<0.001) compared to placebo in patients with previously treated advanced hepatocellular carcinoma (PMID: 29972759; NCT01908426). 29972759 detail...
Unknown unknown 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 follicular lymphoma not applicable Bendamustine + Veliparib Phase I Actionable In a Phase I trial, a patient with follicular lymphoma treated with a combination of Veliparib (ABT-888) and Bendamustine demonstrated a complete response (PMID: 28314788; NCT01326702). 28314788
Unknown unknown hepatocellular carcinoma not applicable Camrelizumab Phase I Actionable In a Phase I trial, Camrelizumab (SHR-1210) demonstrated safety and preliminary anti-tumor activity, resulted in partial response in 33.3% (1/3) of patients with hepatocellular carcinoma (J Clin Onc. 2017 35:15_suppl, e15572-e15572; NCT02742935). detail...
Unknown unknown breast cancer not applicable AJI-214 Preclinical - Cell culture Actionable In a preclinical study, AJI-214 inhibited invasion and induced apoptosis of breast cancer cells in culture (PMID: 24930769). 24930769
Unknown unknown 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 hepatocellular carcinoma not applicable FH535 Preclinical Actionable In a preclinical study, FH535 demonstrated efficacy by inhibiting proliferation of liver cancer stem cells and hepatocellular carcinoma cells in culture (PMID: 24940873). 24940873
Unknown unknown Advanced Solid Tumor not applicable Carboplatin + Paclitaxel + Panobinostat Phase I Actionable In a Phase I trial, 52% (11/21) of patients with advanced solid tumors demonstrated stable disease when treated with Farydak (panobinostat), in combination with Paraplatin (carboplatin) and Taxol (paclitaxel) (PMID: 22851205). 22851205
Unknown unknown 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 lymphoma not applicable Panobinostat + PQR309 Preclinical - Cell culture Actionable In a preclinical study, the combination therapy of Farydak (panobinostat) and PQR309 induced apoptosis and led to synergistic and additive effects in lymphoma cell lines in culture (PMID: 29066507). 29066507
Unknown unknown prostate cancer not applicable CX-6258 Preclinical - Cell line xenograft Actionable In a preclinical study, CX-6258 inhibited tumor growth in human prostate cancer cell line xenograft models (PMID: 24900437). 24900437
Unknown unknown 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 ovarian cancer not applicable Sunitinib Phase Ib/II Actionable In a Phase II trial, Sutent (sunitinib) treatment in ovarian cancer patients resulted in an increased PFS and a response rate of 16.7% (6/36) in those that received Sutent (sunitinib) continuously and a a response rate of 5.4% (2/37) in those that received the drug non-continuously (PMID: 22377563, PMID: 24070205). 22377563 24070205
Unknown unknown malignant mesothelioma not applicable Ad-NK4 Preclinical - Cell line xenograft Actionable In a preclinical study, Ad-NK4 decreased phosphorylation of Met and Akt, decreased beta-catenin signaling, and inhibited viability and invasiveness of mesothelioma cell lines in culture, and inhibited tumor growth in xenograft models (PMID: 25501304). 25501304
Unknown unknown 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 Advanced Solid Tumor not applicable Sabatolimab Phase Ib/II Actionable In a Phase Ib/II trial, MBG453 treatment resulted in stable disease in 29% (25/87) of patients with advanced solid tumors (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). 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 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 triple-receptor negative breast cancer not applicable BOS172722 Preclinical - Cell line xenograft Actionable In a preclinical study, triple-negative breast cancer (TNBC) cell lines demonstrated increased sensitivity to BOS172722 compared to non-triple-negative breast cancer cell lines in culture, and BOS172722 treatment resulted in moderate tumor growth inhibition in a TNBC cell line xenograft model (PMID: 31575759). 31575759
Unknown unknown mantle cell lymphoma not applicable Ibrutinib + ONC201 Preclinical - Patient cell culture Actionable In a preclinical study, the combination of ONC201 and Imbruvica (ibrutinib) induced apoptosis in primary mantle cell lymphoma samples in culture, including Imbruvica (ibrutinib)-resistant samples, with greater efficacy than either agent alone (PMID: 26884599). 26884599
Unknown unknown Advanced Solid Tumor not applicable YW3-56 Preclinical Actionable In a preclinical study, YW3-56 inhibited proliferation of a variety of human tumor cell lines in culture, independent of TP53 mutational status (PMID: 25612620). 25612620
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 bladder urothelial carcinoma not applicable Pembrolizumab Phase II Actionable In a Phase II clinical study (PURE-01), 42% (21/50) of patients with muscle invasive bladder cancer treated with neoadjuvant Keytruda (pembrolizumab) had complete pathological responses at disease resection (PMID: 30343614; NCT02736266). 30343614
Unknown unknown anaplastic large cell lymphoma not applicable Brentuximab vedotin FDA approved Actionable In a Phase II trial (SGN-35) that supported FDA approval, Adcetris (brentuximab vedotin) treatment resulted in complete remission in 57% (33/58), partial remission in 29% (17/58) of patients with relapsed or refractory systemic anaplastic large cell lymphoma (PMID: 22614995; NCT00866047). 22614995 detail...
Unknown unknown melanoma not applicable JQ1 Preclinical Actionable In a preclinical study, JQ1 reduced tumor growth in syngeneic mouse melanoma models (PMID: 31018997). 31018997
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 colorectal cancer not applicable VLX600 Preclinical - Patient cell culture Actionable In a preclinical study, VLX600 treatment inhibited proliferation of cells derived from colorectal cancer patients in culture (PMID: 24548894). 24548894
Unknown unknown 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 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 Advanced Solid Tumor not applicable AZD8330 Phase I Actionable In a Phase I trial, AZD8330 demonstrated safety and some preliminary clinical activity in patients with advanced solid tumors (PMID: 23433846). 23433846
Unknown unknown prostate cancer no benefit VT-464 Phase II Actionable In a Phase II trial, Seviteronel (VT-464) monotherapy demonstrated an undesirable toxicity profile and limited activity, with only 6% (1/17) of patients with metastatic castration-resistant prostate cancer achieved significant PSA response (PMID: 32327394). 32327394
Unknown unknown lung non-small cell carcinoma not applicable Vantictumab Preclinical - Pdx Actionable In a preclinical study, Vantictumab (OMP-18R5) inhibited tumor growth in 7/8 patient-derived xenograft models of non-small cell lung cancer (PMID: 22753465). 22753465
Unknown unknown pancreatic cancer not applicable Gemcitabine + MVT-5873 + Nab-paclitaxel Preclinical - Cell line xenograft Actionable In a preclinical study, MVT-5873 enhanced efficacy of the combination therapy, Gemzar (gemcitabine) and Abraxane (nab-paclitaxel), in pancreatic xenograft models, demonstrating increased inhibition of tumor growth and greater reduction of tumor volume when compared to single agents (Cancer Res 2016;76(24 Suppl):Abstract nr A73). detail...
Unknown unknown hepatocellular carcinoma not applicable Orlistat + Paclitaxel Preclinical - Cell culture Actionable In a preclinical study, Xenical (orlistat) and Taxol (paclitaxel) synergistically inhibited growth and induced apoptosis in hepatocellular carcinoma cells in culture (PMID: 30667213). 30667213
Unknown unknown triple-receptor negative breast cancer not applicable Fluzoparib Preclinical - Cell culture Actionable In a preclinical study, Fluzoparib inhibited DNA-damage-induced PARylation in breast cancer cell lines in culture, however, the cells demonstrated resistance to Fluzoparib treatment (PMID: 30949414). 30949414
Unknown unknown Advanced Solid Tumor not applicable Carboplatin + LY2090314 + Pemetrexed Disodium Phase I Actionable In a Phase I trial, LY2090314 in combination with Alimta (pemetrexed) and Paraplatin (carboplatin) resulted in partial response in 13.5% (5/37) and stable disease in 51.4% (19/37) of patients with advanced solid tumors (PMID: 26403509; NCT01287520). 26403509
Unknown unknown leiomyosarcoma no benefit Pembrolizumab Phase II Actionable In a Phase II trial, Keytruda (pembrolizumab) treatment resulted in an objective response rate of 0% (0/10) in patients with leiomyosarcoma (J Clin Oncol 35, 2017 (suppl; abstr 11008)). detail...
Unknown unknown pancreatic cancer not applicable OBI-999 Preclinical - Cell line xenograft Actionable In a preclinical study, OBI-999 treatment inhibited tumor growth in a Globo H-expressing pancreatic cancer cell line xenograft model (Cancer Res 2019;79(13 Suppl):Abstract nr 4815). detail...
Unknown unknown Advanced Solid Tumor not applicable Docetaxel + Glesatinib Phase I Actionable In a Phase I trial, Glesatinib (MGCD265) and Taxotere (docetaxel) combination treatment resulted in partial response in 12% (4/34) and stable disease for 6 cycles or more in 18% (6/34) of patients with advanced solid tumors (J Clin Oncol 30, 2012 (suppl; abstr e13604)). detail...
Unknown unknown leiomyosarcoma not applicable LY3039478 Phase I Actionable In a Phase I trial, treatment with LY3039478 resulted in tumor necrosis in a patient with leiomyosarcoma (European Journal of Cancer, Volume 69, S15). detail...
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 multiple myeloma not applicable Azacitidine + Mivebresib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Vidaza (azacitidine) and Mivebresib (ABBV-075) resulted in increased tumor growth inhibition in a multiple myeloma cell line xenograft model compared to either agent alone (PMID: 28416490). 28416490
Unknown unknown esophagus squamous cell carcinoma not applicable Nivolumab FDA approved Actionable In a Phase III trial (ATTRACTION-3) that supported FDA approval, Opdivo (nivolumab) treatment demonstrated favorable safety profile and significantly improved median overall survival (10.9 vs 8.4 mo, HR=0.77, p=0.019) compared to chemotherapy in patients with advanced esophageal squamous cell carcinoma refractory or intolerant to prior chemotherapy (PMID: 31582355; NCT02569242). 31582355 detail...
Unknown unknown 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 E7449 Phase I Actionable In a Phase I trial, E7449 treatment demonstrated safety and resulted in an overall response rate of 4.9% (2/41, partial responses) and stable disease in 31.7% (13/41) of patients with advanced solid tumors, including stable disease lasting greater than 23 weeks in 8 patients (PMID: 32523090; NCT01618136). 32523090
Unknown unknown stomach cancer not applicable ALX148 + Trastuzumab Phase I Actionable In a Phase I trial, ALX148 and Herceptin (trastuzumab) combination treatment resulted in a partial response in 19% (4/21) and stable disease in 29% (6/21) of patients with gastric or gastroesophageal junction cancer that progressed on chemotherapy (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 2514-2514; NCT03013218). detail...
Unknown unknown Hodgkin's lymphoma not applicable Camidanlumab Tesirine Phase I Actionable In a Phase I trial, Camidanlumab Tesirine treatment resulted in an objective response rate of 63.6% (14/22) and a complete response rate of 27.3% (6/22) in Hodgkin's lymphoma patients (Blood 2017 130(Suppl 1):1510; NCT02432235). detail...
Unknown unknown acute myeloid leukemia not applicable HM43239 Preclinical - Cell culture Actionable In a preclinical study, HM43239 treatment inhibited downstream signaling, reduced proliferation, and induced apoptosis in an acute myeloid leukemia cell line expressing leukemic stem cell marker in culture (Cancer Res 2019;79(13 Suppl):Abstract nr 1293). detail...
Unknown unknown hepatocellular carcinoma not applicable DRP-104 Preclinical Actionable In a preclinical study, DRP-104 treatment inhibited tumor growth and increased survival in a syngeneic mouse model of hepatocellular carcinoma (J Immunother Cancer. 2019; 7(Suppl 1): 282, Abs nr: P497). detail...
Unknown unknown lymphoma not applicable CPI-0610 Phase I Actionable In a Phase I trial, treatment with CPI-0610 in lymphoma patients resulted in decreased tumor volume in five patients and stable disease in six patients (Blood 2015 126:1491). detail...
Unknown unknown Her2-receptor negative breast cancer not applicable Bevacizumab + Cyclophosphamide + Docetaxel + Epirubicin + Fluorouracil Phase III Actionable In a Phase III trial, treatment with the combination of Avastin (bevacizumab) and Taxotere (docetaxel), Adrucil (fluorouracil), Ellence (epirubicin), and Cytoxan (cyclophosphamide) (D-FEC) resulted in improved efficacy over D-FEC alone in ERBB2 (HER2)-negative breast cancer patients, with pathological complete response in breast and lymph nodes in 22% (87/388) of patients (PMID: 25975632). 25975632
Unknown unknown chronic lymphocytic leukemia not applicable Chlorambucil + Ofatumumab FDA approved Actionable In a Phase III trial supporting FDA approval, Arzerra (ofatumumab) and Chlorambucil combination treatment resulted in improved median progression-free survival (22.4 vs. 13.1 months) compared to Chlorambucil alone in treatment-naive patients with chronic lymphocytic leukaemia (PMID: 25882396). 25882396 detail...
Unknown unknown prostate cancer not applicable ONC201 Clinical Study Actionable In a clinical case study, a prostate cancer patient demonstrated tumor regression in the primary tumor and metastatic lesions when treated with ONC201 (TIC-10) (PMID: 28331050). 28331050
Unknown unknown Advanced Solid Tumor not applicable Ascrinvacumab Phase I Actionable In a Phase I trial, PF-03446962 treatment was deemed safe and resulted in anti-tumor activity in patients with advanced solid tumors, demonstrating a partial response in 6.8% (3/44) and stable disease in 27.3% (12/44) (PMID: 26655846). detail... 26655846
Unknown unknown acute myeloid leukemia not applicable GSK3368715 Preclinical - Cell culture Actionable In a preclinical study, GSK3368715 inhibited growth of acute myeloid leukemia cell lines in culture, and demonstrated cytotoxicity in 50% of cell lines tested (PMID: 31257072). 31257072
Unknown unknown renal cell carcinoma not applicable Axitinib + X4P-001 Phase I Actionable In a Phase I trial, X4P-001 in combination with Axitinib, displayed safety and efficacy in patients with renal cell carcinoma (AACR; Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B201, NCT02667886). detail...
Unknown unknown lung cancer not applicable Cediranib + Gefitinib Phase I Actionable In a Phase I trial, the combination of Cediranib and Iressa (gefitinib) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, including a partial response in a patient with lung cancer (PMID: 20061136). 20061136
Unknown unknown Advanced Solid Tumor no benefit AZD0424 Phase I Actionable In a Phase I trial, AZD0424 treatment in patients with advanced solid tumors did not result in antitumor activity and is not recommended for further testing as a monotherapy in advanced solid tumor patients (PMID: 29438361). 29438361
Unknown unknown malignant pleural mesothelioma not applicable GDC-0980 Phase I Actionable In a Phase I trial, Apitolisib (GDC-0980) treatment resulted in a partial response in 7.4% (2/27) and stable disease in 74.1% (20/27) of malignant pleural mesothelioma patients, including one with PTEN loss and another with PIK3CA E545K (PMID: 26787751). 26787751
Unknown unknown acute myeloid leukemia not applicable RN-1 Preclinical - Cell culture Actionable In a preclinical study, RN-1 inhibited growth of acute myeloid leukemia cell lines in culture (PMID: 26837761). 26837761
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 PF-00562271 Phase I Actionable In a Phase I trial, PF-00562271 was well-tolerated and resulted in stable disease in 34% (31/91) of patients with advanced solid tumors as well as a metabolic response of 50% (7/14) (PMID: 22454420). 22454420
Unknown unknown Advanced Solid Tumor not applicable PF-00562271 Preclinical - Cell line xenograft Actionable In a preclinical study, PF-00562271 inhibited PTK2 (FAK) phosphorylation and growth of multiple tumor cell types in cell line xenograft models (PMID: 18339875). 18339875
Unknown unknown lung cancer not applicable Dinaciclib + Paclitaxel Preclinical - Cell culture Actionable In a preclinical study, combination of Dinaciclib (SCH 727965) and Taxol (paclitaxel) demonstrated enhanced growth inhibition in lung cancer cell lines in culture (PMID: 27550941). 27550941
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 colorectal adenocarcinoma not applicable Berzosertib + Irinotecan Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of VX-970 and Camptosar (irinotecan) synergized to inhibit proliferation of a colorectal adenocarcinoma cell line in culture and to inhibit tumor growth in a human colorectal adenocarcinoma cell line xenograft model (PMID: 25269479). 25269479
Unknown unknown 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 glioblastoma multiforme not applicable CID613034 Preclinical - Cell culture Actionable In a preclinical study, CID613034 resulted in reduced colony formation, apoptotic activity, and inhibition of migration in glioblastoma cells in culture (PMID: 28453552). 28453552
Unknown unknown Advanced Solid Tumor not applicable NP-004255 Preclinical - Cell culture Emerging In a preclinical study, a biochemical assay with NP-004255, a RAD52 inhibitor similar to other compounds that induced cell death in cells with BRCA2 loss, demonstrated inhibition of RAD52 binding to ssDNA, suggesting NP-004255 may be a potential therapeutic target (PMID: 27434671). 27434671
Unknown unknown Advanced Solid Tumor not applicable Rucaparib Phase I Actionable In a Phase I trial, Rubraca (rucaparib) was well-tolerated and demonstrated preliminary efficacy, with a disease control rate of 86% (6/7), in patients with advanced solid tumors (J Clin Oncol 31, 2013 (suppl; abstr 2585)). detail...
Unknown unknown lung small cell carcinoma not applicable Carboplatin + Durvalumab + Etoposide FDA approved Actionable In a Phase III (CASPIAN) trial that supported FDA approval, Imfinzi (durvalumab) in combination with Vepesid (etoposide) and Paraplatin (carboplatin) or Platinol (cisplatin) resulted in significantly improved overall survival (13.0 vs 10.3 mo, HR=0.73, p=0.0047) compared to platinum-etoposide therapy in patients with untreated extensive-stage small cell lung cancer (PMID: 31590988; NCT03043872). 31590988
Unknown unknown diffuse large B-cell lymphoma not applicable LAM-002A Phase I Actionable In a Phase I trial, LAM-002A demonstrated safety and preliminary efficacy, resulted in systemic partial metabolic responses and decreased tumor size in 3 of 11 patients with diffuse large B-cell lymphoma (Blood 2017 130 (Suppl 1):4119). detail...
Unknown unknown fibrous histiocytoma not applicable Pazopanib Clinical Study Actionable In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 15.3 weeks and median survival of 9.5 months in patients with undifferentiated pleomorphic sarcoma/malignant fibrous histiocytoma (PMID: 26970174). 26970174
Unknown unknown astrocytoma no benefit Veliparib Preclinical - Cell culture Actionable In a preclinical study, Veliparib (ABT-888) had a very limited effect on the cell viability of multiple cultured pediatric high grade astrocytoma cell lines (PMID: 26351319). 26351319
Unknown unknown ovarian cancer not applicable ABT-737 + Dactolisib + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of BEZ235 and Mekinist (trametinib) enhanced the sensitivity of ovarian cancer cells to ABT-737 in culture, resulting in decreased cell viability (PMID: 27980105). 27980105
Unknown unknown melanoma not applicable 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 small cell carcinoma not applicable Alisertib + Paclitaxel Preclinical - Pdx & cell culture Actionable In a preclinical study, Alisertib (MLN8237), alone and in combination with Taxol (paclitaxel), inhibited growth of small cell lung cancer cell (SCLC) lines in culture and inhibited tumor growth in primary human SCLC xenograft models (Mol Cancer Ther 2013;12(11 Suppl):A282). detail...
Unknown unknown lung small cell carcinoma not applicable Alisertib + Paclitaxel Phase II Actionable In a Phase II trial, the combination treatment of Alisertib (MLN8237) and Taxol (paclitaxel) compared to Taxol (paclitaxel) plus placebo resulted in a progression-free survival of 3.32 months versus 2.17 months (P=0.038), and led to an overall response rate of 22% (20/89) versus 18% (16/289), and a disease control rate of 58% (52/89) versus 46% (41/89), respectively, in patients with relapsed or refractory small cell lung cancer (PMID: 31655296; NCT02038647). 31655296
Unknown unknown colon carcinoma not applicable SB 11285 Preclinical Actionable In a preclinical study, treatment with SB 11285 delayed tumor growth and reduced tumor volume in a syngeneic mouse model of colon carcinoma (Journal of Clinical Oncology 2017 35:15_suppl, e14616). detail...
Unknown unknown colon cancer not applicable Ch282-5 Preclinical - Cell line xenograft Actionable In a preclinical study, ch282-5 induced apoptosis and inhibited growth and migration of several human and mouse colon cancer cell lines in culture, and inhibited tumor growth and metastasis in xenograft models (PMID: 26515494). 26515494
Unknown unknown mantle cell lymphoma not applicable Ibrutinib + Palbociclib Phase I Actionable In a Phase I trial, Imbruvica (ibrutinib) and Ibrance (palbociclib) combination treatment resulted in an overall response rate of 67%, a complete response rate of 37%, and a two year progression-free survival in 59.4% of patients with previously treated mantle cell lymphoma, with a median follow up of 25.6 months (PMID: 30692121; NCT02159755). detail... 30692121
Unknown unknown 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 glioblastoma multiforme not applicable Tanibirumab Preclinical - Cell line xenograft Actionable In a preclinical study, Tanibirumab (TTAC-0001) inhibited angiogenesis and tumor growth in human glioblastoma cell line xenograft models (PMID: 26325365). 26325365
Unknown unknown brain glioma not applicable Dorsomorphin Preclinical Actionable In a preclinical study, dorsomorphin (Compound C) inhibited AMPK and reduced glioma cells viability in vitro by inhibiting proliferation and inducing cell death (PMID: 24419061) 24419061
Unknown unknown colorectal cancer not applicable Afatinib Preclinical Actionable In a preclinical study, Gilotrif (afatinib) inhibited proliferation and increased apoptosis in several human colorectal tumor cell lines in culture (PMID: 21617858). 21617858
Unknown unknown Advanced Solid Tumor not applicable Navitoclax Preclinical - Cell culture Actionable In a preclinical study, Navitoclax (ABT-263) treatment alone was not effective in a number of cell lines derived from solid tumors in culture (PMID: 27974663). 27974663
Unknown unknown 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 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 gastric adenocarcinoma no benefit Panitumumab Phase III Actionable In a Phase III trial, addition of Vectibix (panitumumab) to chemotherapy consisted of epirubicin, oxaliplatin, and capecitabine (EOC) did not improve overall survival and increased side effects, therefore was not recommended in an unselected population of patients with advanced oesophagogastric adenocarcinoma (PMID: 23594787). 23594787
Unknown unknown thyroid gland carcinoma not applicable Sorafenib Clinical Study Actionable In a clinical case report, Nexavar (sorafenib) therapy based on in vitro efficacy testing using patient-derived tumor cells resulted in 43 months of disease-free in a patient with anaplastic thyroid carcinoma (PMID: 27379749). 27379749
Unknown unknown colon cancer not applicable Mitomycin C + Valproic acid Preclinical Actionable In a preclinical study, Depakene (valproic acid), in combination with Mitozytrex (mitomycin C), inhibited cell viability of colon cancer cells isolated from patients (PMID: 17024798). 17024798
Unknown unknown pancreatic cancer not applicable Pamiparib + Tislelizumab Case Reports/Case Series Actionable In a Phase Ib trial, the combination therapy of of Tislelizumab (BGB-A317) and Pamiparib (BGB-290) in patients with pancreatic cancer resulted in one partial response and stable disease for greater than 6 months in two patients (J Clin Oncol 35, 2017 (suppl; abstr 3013)). detail...
Unknown unknown 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 Advanced Solid Tumor not applicable Enoticumab Phase I Actionable In a Phase I trial, Enoticumab (REGN421) resulted in partial response in 3% (2/53) and stable disease in 30% (16/53) of patients with advanced solid tumors (PMID: 25724527). 25724527
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 Advanced Solid Tumor not applicable LY2780301 Phase I Actionable In a Phase I trial, LY2780301 demonstrated safety and preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 25902900). 25902900
Unknown unknown squamous cell carcinoma not applicable PMX-53 Preclinical Actionable In a preclinical study, PMX-53 treatment did not inhibit tumor growth in a syngeneic mouse model of poorly differentiated squamous cell carcinoma (PMID: 30300579). 30300579
Unknown unknown breast carcinoma not applicable BI 836880 Case Reports/Case Series Actionable In a Phase I trial, BI 836880 treatment was well tolerated in patients with advanced solid tumors, and resulted in 2 partial responses (n=29), including a partial response in a patient with breast carcinoma (J Clin Onc 2018 36:15_suppl, 12024; NCT02674152). detail...
Unknown unknown thyroid gland carcinoma not applicable Obatoclax Preclinical Actionable In a preclinical study, Obatoclax induced cell death in human thyroid carcinoma cell lines culture (PMID: 26198850). 26198850
Unknown unknown pancreatic adenocarcinoma not applicable Mogamulizumab + Nivolumab Phase I Actionable In a Phase I trial, Poteligeo (mogamulizumab-kpkc) and Opdivo (nivolumab) combination treatment demonstrated acceptable safety, resulted in an objective response rate of 7% (1/15, 1 partial responses) and a disease control rate of 40% (6/15) in immunotherapy-naive patients with advanced or metastatic pancreatic adenocarcinoma, response occurred regardless of PD-L1, CCR4, CD8 expression levels and tumor mutational burden (PMID: 31455681; NCT02476123). 31455681
Unknown unknown Ewing sarcoma not applicable MEDI-573 + Vistusertib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of MEDI-573 and Vistusertib (AZD2014) inhibited proliferation of a Ewing sarcoma cell line in culture, and inhibited tumor growth in a Ewing sarcoma cell line xenograft model, with increased efficacy compared to either as a single agent (PMID: 25193511). 25193511
Unknown unknown diffuse large B-cell lymphoma not applicable CPI-0610 Phase I Actionable In a Phase I trial, treatment with CPI-0610 resulted in a complete response in two patients with diffuse large B-cell lymphoma (Blood 2015 126:1491). detail...
Unknown unknown breast cancer not applicable Barasertib Preclinical - Cell culture Actionable In a preclinical study, Barasertib (AZD1152) treatment disrupted cell cycle progression and inhibited growth of breast cancer cell lines in culture, with preferential inhibition of aromatase inhibitor-resistant cell lines (PMID: 25667100). 25667100
Unknown unknown Advanced Solid Tumor not applicable Panulisib Preclinical - Cell line xenograft Actionable In a preclinical study, Panulisib (P7170) inhibited growth of several solid tumor cell lines in culture and in cell line xenograft models (PMID: 25700704). 25700704
Unknown unknown Advanced Solid Tumor not applicable 4SC-205 Phase I Actionable In a Phase I trial, 4SC-205 treatment demonstrated safety and promising long-term disease stabilization in patients with advanced solid tumors, resulted in a median time on study of 162 days in patients receiving continuous dosing (J Clin Oncol (Meeting Abstracts) May 2015 vol. 33 no. 15_suppl 2528). detail...
Unknown unknown 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 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 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 breast cancer not applicable AZD7648 Preclinical - Cell line xenograft Actionable In a preclinical study, AZD7648 treatment inhibited tumor growth in a cell line xenograft model of breast cancer, however, with decreased response compared to AZD7648 and Doxil (pegylated liposomal-doxorubicin) combination treatment (PMID: 31699977). 31699977
Unknown unknown B-cell lymphoma not applicable ARQ 531 Preclinical Actionable In a preclinical study, ARQ 531 treatment increased survival in a transgenic mouse model of aggressive B-cell lymphoma similar to Richter transformation, with a median of 47 days vs. 38 days with Imbruvica (ibrutinib) (p=0.036) (PMID: 30093506). 30093506
Unknown unknown Advanced Solid Tumor not applicable JNJ-63723283 Phase Ib/II Actionable In a Phase I/II trial, JNJ-63723283 demonstrated safety and preliminary efficacy, resulted in partial response in 9.4% (3/32) and stable disease in 56.3% (18/32) of patients with advanced solid tumors (Journal of Clinical Oncology 36, no. 5_suppl (February 10 2018) 58-58). detail...
Unknown unknown 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 stomach cancer not applicable Capmatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Tabrecta (capmatinib) did not induce tumor regression, however, inhibited tumor growth in an autocrine cell line xenograft model of gastric cancer overexpressing HGF (PMID: 30674502). 30674502
Unknown unknown 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 thyroid gland cancer not applicable Bevacizumab Preclinical Actionable In a preclinical study, Avastin (bevacizumab) inhibited tumor growth and angiogenesis in mouse models of anaplastic thyroid cancer (PMID: 17429874). 17429874
Unknown unknown Advanced Solid Tumor not applicable APN401 Phase I Actionable In a Phase I trial, APN401 infusion demonstrated safety in patients with advanced solid tumors, and resulted in stable disease in two patients with pancreatic cancer and one patient with with colorectal cancer (J Clin Oncol 34, 2016 (suppl; abstr e14541)). detail...
Unknown unknown triple-receptor negative breast cancer not applicable Lucitanib Preclinical Actionable In a preclinical study, Lucitanib (E-3810) demonstrated antitumor activity in mouse xenograft models of triple negative breast cancer with synergistic effects noted when using Lucatinib plus chemotherapy (PMID: 23270924). 23270924
Unknown unknown 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 lung non-small cell carcinoma not applicable Ascrinvacumab Phase I Actionable In a Phase I trial, a patient with non-small cell lung carcinoma demonstrated a partial response for 308 days when treated with PF-03446962 (PMID: 26655846). 26655846
Unknown unknown lymphoma not applicable OKI-179 Preclinical - Cell culture Actionable In a preclinical study, OKI-005 induced cell cycle arrest and apoptosis in lymphoma cells in culture (PMID: 31235619). 31235619
Unknown unknown melanoma not applicable Ipilimumab Phase III Actionable In a Phase III trial, adjuvant Opdivo (nivolumab) treatment resulted in improved rate of recurrence-free survival at 12-months compared to Yervoy (ipilimumab) (70.5% vs 60.8%, HR=0.65, P<0.001) in patients with resected stage III or IV melanoma (PMID: 28891423, NCT02388906). 28891423
Unknown unknown melanoma not applicable Ipilimumab FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Yervoy (ipilimumab) resulted in an improved overall survival of 10.1 months in patients with metastatic melanoma, compared to 6.4 months in those treated with gp100 peptide vaccine alone (PMID: 20525992, PMID: 21900389). 20525992 detail... 21900389
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 sarcoma not applicable Ganitumab Phase II Actionable In a Phase II trial, Ganitumab treatment resulted in a 6% (2/35) partial response and stable disease in 49% (17/35) of patients with metastatic Ewing's family of tumors or desmoplastic small round cell tumor (PMID: 22508822). 22508822
Unknown unknown hepatocellular carcinoma not applicable Atezolizumab + Bevacizumab FDA approved Actionable In a Phase III trial (IMbrave150) that supported FDA approval, Tecentriq (atezolizumab) and Avastin (bevacizumab) combination therapy improved survival compared to Nexavar (sorafenib) (HR for death=0.58, p<0.001) in patients with unresectable hepatocellular carcinoma, with superior overall survival at 12 months (67.2% vs 54.6%) and prolonged median progression-free survival (6.8 vs 4.3 months, HR=0.59, p<0.001) (PMID: 32402160; NCT03434379). detail... 32402160
Unknown unknown 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 colorectal cancer not applicable Barasertib + Navitoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of Barasertib (AZD1152) and Navitoclax (ABT-263) resulted in synergy, demonstrating inhibition of cell proliferation and increased apoptotic activity in colorectal cancer cells in culture (PMID: 28179288). 28179288
Unknown unknown lung cancer not applicable SKI-G-801 Preclinical Actionable In a preclinical study, SKI-G-801 treatment reduced tumor growth in a syngeneic mouse model of lung cancer (Cancer Res 2019;79(13 Suppl):Abstract nr 2010). detail...
Unknown unknown estrogen-receptor positive breast cancer not applicable Everolimus Preclinical - Cell culture Actionable In a preclinical study, treatment with Afinitor (everolimus) resulted in decreased cell proliferation, reduced anchorage-independent cell growth and a decrease in PI3K/Akt/mTOR pathway signaling in estrogen-receptor positive breast cancer cell lines resistant to aromatase inhibitors (PMID: 27421652). 27421652
Unknown unknown 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 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 colon adenocarcinoma not applicable Staurosporine Preclinical - Cell culture Actionable In a preclinical study, Staurosporine reduced proliferation and increased apoptosis in a colon adenocarcinoma cell line in culture (PMID: 8913270). 8913270
Unknown unknown pancreatic carcinoma not applicable Gemcitabine + MU380 Preclinical - Cell line xenograft Actionable In a preclinical study, the addition of MU380 resulted in increased sensitivity to Gemzar (gemcitabine) in a pancreatic carcinoma cell line in culture and in xenograft models (PMID: 28619751). 28619751
Unknown unknown 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 hepatocellular carcinoma not applicable Ascrinvacumab Phase I Actionable In a Phase I trial, a patient with hepatocellular carcinoma demonstrated a partial response for 44 days when treated with PF-03446962 (PMID: 26655846). 26655846
Unknown unknown hematologic cancer not applicable Tefinostat Phase I Actionable In a Phase I trial, Tefinostat (CHR-2845) demonstrated safety and preliminary efficacy in patients with hematological cancers (PMID: 23647373). 23647373
Unknown unknown 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 meningioma not applicable AR-42 Preclinical Actionable In a preclinical study, AR-42 induced apoptosis and inhibited proliferation of meningioma cells in culture (PMID: 21778190). 21778190
Unknown unknown 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 ovary epithelial cancer not applicable Alpelisib + Olaparib Phase I Actionable In a Phase Ib trial, Alpelisib (BYL719) and Lynparza (olaparib) combination therapy demonstrated safety and preliminary efficacy, resulted in partial response in 36% (10/28) and stable disease in 50% (14/28) of patients with epithelial ovarian cancer, overall response rate was similar for germline BRCA mutated and wild-type patients (30%, 3/10 vs 35%, 6/17, p=0.42) (PMID: 30880072; NCT01623349). 30880072
Unknown unknown colorectal cancer not applicable Sabatolimab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 treatment resulted in stable disease in 2 patients with colorectal cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown ovarian cancer not applicable Napabucasin Phase Ib/II Actionable In a Phase Ib/II clinical trial, treatment with BBI608 demonstrated safety in patients with platinum-resistant ovarian cancer (including patients with epithelial ovarian, fallopian, or peritoneal cancer), and resulted in a disease control rate of 68% (27/40) and an objective response rate of 25% (10/40) in evaluable patients (J Clin Oncol, May 2016 vol. 34 no. 15_suppl 5578). detail...
Unknown unknown 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 chronic lymphocytic leukemia not applicable Bortezomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, the combination of EDO-S101 and Velcade (bortezomib) decreased viability of primary chronic lymphocytic leukemia cells in culture (PMID: 28753594). 28753594
Unknown unknown 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 Advanced Solid Tumor not applicable GC1118 Phase I Actionable In a Phase I clinical trial, treatment with GC1118 was well-tolerated, and resulted in partial response in 13% (3/24) and stable disease in 50% (12/24) of patients with advanced solid tumors (PMID: 31164456; NCT02352571). 31164456
Unknown unknown pancreatic cancer not applicable ABTL0812 Preclinical Actionable In a preclinical study, ABTL0812 inhibited growth and mTORC1 signaling and induced autophagy in a human pancreatic cancer cell line in culture (PMID: 26671995). 26671995
Unknown unknown bone giant cell tumor not applicable Bortezomib Preclinical Actionable In a preclinical study, Velcade (Bortezomib) treatment resulted in decreased NF-kappaB signaling, increased apoptosis, and decreased growth of bone giant cell tumor cells in culture, and decreased bone giant cell tumor cell-mediated bone destruction in mouse models (PMID: 26861247). 26861247
Unknown unknown non-Hodgkin lymphoma not applicable CC-90010 Phase I Actionable In a Phase I trial, CC-90010 treatment demonstrated safety, and resulted in an overall response rate of 2.9% (2/69, 1 complete response, 1 partial response), stable disease in 33.3% (23/69), and a median progression-free survival of 1.9 months in patients with advanced solid tumors or relapsed/refractory non-Hodgkin lymphoma (PMID: 32240793; NCT03220347). 32240793
Unknown unknown sarcoma not applicable Doxorubicin + Olaratumab FDA approved Actionable In a Phase I/IIb trial that supported FDA approval, Lartruvo (olaratumab) and Adriamycin (doxorubicin) combination treatment improved median progression free survival (6.6 vs. 4.1 months) and median overall survival (26.5 vs. 14.7 months) compared to Adriamycin (doxorubicin) treatment alone in patients with advanced soft tissue sarcoma (PMID: 27291997; NCT01185964). 27291997 detail... detail...
Unknown unknown lung cancer not applicable SNG12 Preclinical - Cell line xenograft Actionable In a preclinical study, the TTK (MPS1) inhibitor, SNG12, inhibited proliferation of lung cancer cells in culture and suppressed tumor growth in cell line xenograft models (PMID: 24900510). 24900510
Unknown unknown Advanced Solid Tumor not applicable AMG 900 Preclinical - Cell line xenograft Actionable In a preclinical study, AMG 900 inhibited the growth of a variety of human solid tumor cell lines in culture and inhibited tumor growth in cell line xenograft models of several tumor types including breast, colon, lung, pancreatic, and uterine cancer (PMID: 20935223). 20935223
Unknown unknown prostate cancer not applicable BI2536 + Metformin Preclinical - Pdx Actionable In a preclinical study, the combination of BI2536 and Metformin worked synergistically to inhibit tumor growth in patient-derived prostate cancer xenograft models (PMID: 25505174). 25505174
Unknown unknown ovarian cancer not applicable Cisplatin + TRX-E-002-1 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of TRX-E-002-1 and Platinol (cisplatin) synergized to induce cell death in human chemoresistant ovarian cancer cell lines in culture and decreased tumor burden in xenograft models (PMID: 27196760). 27196760
Unknown unknown prostate cancer no benefit Bevacizumab + Docetaxel Phase III Actionable In a Phase III trial, treatment with Avastin (bevacizumab) combined with Taxotere (docetaxel) in mCRPC patients did not result in an improved OS (22.6 mo vs 21.5 mo) and demonstrated increased toxicities when compared to Taxotere (docetaxel) plus placebo (PMID: 22454414). 22454414
Unknown unknown rhabdomyosarcoma not applicable SP-2509 Preclinical - Cell culture Actionable In a preclinical study, SP-2509 treatment inhibited viability of rhabdomyosarcoma cell lines in culture (PMID: 29997151). 29997151
Unknown unknown head and neck squamous cell carcinoma not applicable Cetuximab + Motolimod Phase Ib/II Actionable In a Phase Ib trial, Motolimod (VTX-2337) and Erbitux (cetuximab) combination treatment resulted in partial response in 15% (2/13) and stable disease in 39% (5/13) of patients with head and neck squamous cell carcinoma (PMID: 27810904). 27810904
Unknown unknown duodenum adenocarcinoma not applicable Ramucirumab Phase II Actionable In a Phase II study, Cyramza (ramuciramab), in combination with mFOLFOX-6 chemotherapy regimen (Wellcovorin (leucovorin), Adrucil (fluorouracil) and Eloxatin (oxaliplatin)), demonstrated safety and efficacy in metastatic colorectal cancer (PMID: 24674871). 24674871
Unknown unknown ovarian carcinoma not applicable SKLB-23bb Preclinical - Cell line xenograft Actionable In a preclinical study, SKLB-23bb treatment resulted in antitumor efficacy in ovarian carcinoma cells, demonstrating decreased colony formation in vitro and inhibition of tumor growth in cell line xenograft models (PMID: 29610282). 29610282
Unknown unknown pancreatic cancer no benefit Metformin Preclinical - Pdx Actionable In a preclinical study, long term treatment (28 days) of Glucophage (metformin) did not result in decreased tumor growth in patient-derived pancreatic cancer xenograft models (PMID: 26760500). 26760500
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 Litronesib Phase I Actionable In an analysis of two Phase I trial, litronesib (LY2523355) treatmetn resulted in partial response in 2.3% (2/86) and stable disease in 20% (17/86) of patients with advanced cancer (PMID: 28097385). 28097385
Unknown unknown 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 Advanced Solid Tumor not applicable LB-100 Phase I Actionable In a Phase I trial, LB-100 demonstrated safety and preliminary clinical activity in patients with advanced solid tumors, including a partial response in a pancreatic cancer patient, and stable disease for at least 4 cycles in 50% (10/20) of patients (PMID: 28039265; NCT01837667). 28039265
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 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 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 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 kidney cancer not applicable ISTH0047 Preclinical Actionable In a preclinical study, ISTH0047 reduced lung metastasis, but not primary tumor growth, in a mouse model of metastatic kidney cancer (PMID: 28911087). 28911087
Unknown unknown acute myeloid leukemia not applicable Cytarabine + Onvansertib Phase I Actionable In a Phase I trial, Onvansertib (PCM-075) and low-dose Cytosar-U (cytarabine) combination therapy resulted in complete response in 33.3% (1/3) of evaluable patients with relapsed or refractory acute myeloid leukemia (Ann Oncol, 30 (Supplement 5): v435-v448, 2019; NCT03303339). detail...
Unknown unknown endometrial carcinoma not applicable XL147 Phase II Actionable In a Phase II trial, Pilaralisib (XL147) treatment resulted in an objective response rate of 6% (4/67) in patients with endometrial carcinoma, although anti-tumor activity is not associated with molecular alterations in PTEN and PIK3R1 (PMID: 25528496). 25528496
Unknown unknown clear cell renal cell carcinoma not applicable Cediranib + Saracatinib Phase II Actionable In a Phase II clinical trial, Saracatinib (AZD0530) did not increase the efficacy of Recentin (cediranib) in patients with metastatic clear-cell renal cell carcinoma (n=69 for both trial arms) (PMID: 26802156). 26802156
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 multiple myeloma not applicable Dinaciclib + Veliparib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Veliparib (ABT-888) and Dinaciclib (SCH 727965) had a synergistic effect on decreasing survival of multiple myeloma cell lines in culture, and resulted in delayed tumor growth and improved survival of multiple myeloma cell line xenograft models compared to either agent alone (PMID: 26719576). 26719576
Unknown unknown ovarian cancer not applicable Nivolumab + Varlilumab Phase Ib/II Actionable In a Phase I/II trial, Varlilumab and Opdivo (nivolumab) combination treatment resulted in partial response in 10% (5/49) and stable disease in 39% (19/49) of ovarian cancer patients, with treatment-induced increase of PD-L1 expression and CD8+ T cells more common in patients with better responses (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 3001-3001; NCT02335918). detail...
Unknown unknown sarcoma no benefit Doxorubicin + Trabectedin Phase II Actionable In a Phase II trial, the combination of Yondelis (trabectedin) and Adriamycin (doxorubicin) did not improve progression free survival (5.7 months) compared to Adriamycin (doxorubicin) alone (5.5 months) in patients with advanced soft tissue sarcoma (PMID: 27185843). 27185843
Unknown unknown lung non-small cell carcinoma not applicable LYC-55716 Case Reports/Case Series Actionable In a Phase I trial, Cintirorgon (LYC-55716) treatment resulted in a partial response in a patient with non-small cell lung cancer who was previously treated with Keytruda (pembrolizumab) and had progressed (PMID: 30819679). 30819679
Unknown unknown melanoma not applicable G-TPP + Navitoclax Preclinical - Cell line xenograft Actionable In a preclinical study, the mitochondrial Hsp90 inhibitor G-TPP and the broad BH3 mimetic Navitoclax (ABT-263) synergistically inhibited viability of melanoma cell lines in culture, and inhibited tumor growth in cell line xenograft models (PMID: 28522750). 28522750
Unknown unknown Advanced Solid Tumor not applicable JNJ-26483327 Phase I Actionable In a Phase I clinical trial, JNJ-26483327 was well-tolerated in patients with a range of advanced solid tumors, and demonstrated preliminary anti-tumor activity with 32% (6/19) of patients achieving stable disease for greater than 2 cycles (PMID: 20823884). 20823884
Unknown unknown Advanced Solid Tumor not applicable Cobimetinib + Ipatasertib Phase I Actionable In a Phase I clinical trial Ipatasertib (GDC-0068), in combination with the Mek inhibitor Cobimetinib (GDC-0973) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Cancer Res, October 1, 2014 74; CT328). detail...
Unknown unknown renal cell carcinoma not applicable Pazopanib Phase III Actionable In a Phase III trial, adjuvant Votrient (pazopanib) therapy post nephrectomy did not improve disease-free survival (HR=0.862, p=0.165) compared to placebo in patients with renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 4507)). detail...
Unknown unknown renal cell carcinoma not applicable Pazopanib FDA approved Actionable In a Phase III trial that supported FDA approval, Votrient (pazopanib) improved progression free survival in patients with advanced renal cell carcinoma (PMID: 20100962). 20100962 detail...
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 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 diffuse large B-cell lymphoma not applicable OPB-111077 Phase I Actionable In a Phase I trial, a patient with diffuse large B-cell lymphoma demonstrated a partial response when treated with OPB-111077 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B118). detail...
Unknown unknown renal cell carcinoma no benefit MK2206 Phase II Actionable In a Phase II trial, MK2206 treatment resulted in shorter progression free survival compared to Afinitor (everolimus) (3.68 vs 5.98 months) in patients with renal cell carcinoma (PMID: 28049139). 28049139
Unknown unknown 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 chordoma not applicable Dinaciclib Preclinical - Cell culture Actionable In a preclinical study, Dinaciclib (SCH 727965) treatment reduced viability of chordoma cells in culture (PMID: 30664779). 30664779
Unknown unknown multiple myeloma no benefit Cabozantinib Phase I Actionable In a Phase Ib trial, Cometriq (Cabometyx, cabozantinib) treatment did not demonstrate significant efficacy, resulting in a minimal response in 9% (1/11), stable disease in 73% (8/11), and progression in 18% (2/11) of patients with relapsed and/or refractory multiple myeloma (PMID: 27020089; NCT01866293). 27020089
Unknown unknown multiple myeloma not applicable FT671 Preclinical - Cell line xenograft Actionable In a preclinical study, FT671 treatment blocked proliferation of multiple myeloma cells in culture and inhibited tumor growth in multiple myeloma cell line xenograft models (PMID: 29045389). 29045389
Unknown unknown Advanced Solid Tumor not applicable Onatasertib Phase I Actionable In a Phase I clinical trial, CC-223 demonstrated safety and some efficacy in patients with solid tumors (J Clin Oncol 31, 2013 (suppl; abstr 2606). detail...
Unknown unknown glioblastoma multiforme not applicable Regorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Stivarga, (regorafenib), inhibited tumor growth in cell line xenograft models of glioblastoma multiforme (PMID: 21170960). 21170960
Unknown unknown renal cell carcinoma not applicable Everolimus + Lenvatinib FDA approved Actionable In a Phase II clinical trial that supported FDA approval, treatment with the combination of Lenvima (lenvatinib) and Afinitor (everolimus) resulted in a prolonged median progression-free survival of 14.6 months, compared to 5.5 months for Afinitor (everolimus) alone in patients with metastatic renal cell carcinoma who had progressed after one previous VEGF-targeted therapy (PMID: 26482279; NCT01136733). 26482279 detail...
Unknown unknown renal cell carcinoma not applicable Everolimus + Lenvatinib Phase Ib/II Actionable In a Phase Ib clinical trial, the combination of Lenvima (lenvatinib) and Afinitor (everolimus) demonstrated safety, and resulted in partial response in 30% (6/20) of patients with metastatic renal cell carcinoma (PMID: 24190702). 24190702
Unknown unknown mantle cell lymphoma not applicable Mivavotinib Case Reports/Case Series Actionable In a Phase I trial, Mivavotinib (TAK-659) treatment resulted in an objective response rate of 33% (1/3, 1 partial responses) in patients with relapsed or refractory mantle cell lymphoma, with a median duration of response of 109 days (PMID: 32327472; NCT02000934). 32327472
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 hematologic cancer no benefit Milatuzumab Phase I Actionable In a Phase I trial, Milatuzumab (hLL1) treatment of a mixed cohort of B-cell malignancies patients, resulted in stable disease in 35% (8/23), but there was no evidence of tumor shrinkage (PMID: 25754579). 25754579
Unknown unknown renal cell carcinoma not applicable Tivantinib Phase II Actionable In a Phase II trial, tivantinib resulted in modest activity in which six patients with renal cell carcinoma had a median PFS of 1.9 months (PMID: 22605650). 22605650
Unknown unknown 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 melanoma not applicable IMO-2125 Phase Ib/II Actionable In a Phase I/II trial, IMO-2125 treatment resulted in clinical benefit in 67% (6/9, 1 complete response, 2 partial response, 3 stable disease) of patients with melanoma refractory to a PD-1 or PD-L1 inhibitor (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 1187P; NCT02644967). detail...
Unknown unknown colon adenocarcinoma not applicable Everolimus Preclinical - Cell line xenograft Actionable In preclinical studies, the mTOR inhibitor Afinitor (everolimus) inhibited tumor growth in colon cancer cell line xenograft models (PMID: 20890178). 20890178
Unknown unknown osteosarcoma no benefit Saracatinib Phase II Actionable In a Phase II trial, Saracatinib (AZD0530) treatment was well tolerated, but did not result in statistically significant benefit in median progression-free survival (19.4 vs 8.6 months, p=0.47) or overall survival (not reached in both arms) compared to placebo in patients with osteosarcoma with lung metastases (PMID: 32398945; NCT00752206). 32398945
Unknown unknown glioblastoma multiforme not applicable ST-11 Preclinical Actionable In a preclinical study, ST-11 induced cell-cycle arrest and apoptosis in glioblastoma cell lines in culture, and reduced tumor burden in mouse models of glioblastoma (PMID: 27325686). 27325686
Unknown unknown melanoma not applicable Dovitinib Phase Ib/II Actionable In a Phase I/II study, Dovitinib (TKI258) was demonstrated safe, but of limited clinical benefit in patients with advanced melanoma (PMID: 21976540). 21976540
Unknown unknown colorectal cancer not applicable Cabozantinib Preclinical Actionable In a preclinical study, Cometriq (cabozantinib) showed anti-tumor activity in human colorectal cancer explants (PMID: 25242168). 25242168
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 Advanced Solid Tumor not applicable Abexinostat + Pazopanib Phase Ib/II Actionable In a Phase Ib/II trial, the combination of Abexinostat (PCI-24781) and Votrient (pazopanib) in advanced solid tumor patients resulted in a clinical benefit rate of 37% (16/43), a median response duration of 9.1 months, and 8 patients of 43 achieved stable disease or durable response for greater than 12 months (PMID: 28221861). 28221861
Unknown unknown prostate cancer not applicable RO6839921 Preclinical - Cell line xenograft Actionable In a preclinical study, RO6839921 demonstrated anti-tumor activity in prostate cancer cell line xenograft models (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A156). detail...
Unknown unknown mantle cell lymphoma not applicable Zanubrutinib Preclinical - Cell line xenograft Actionable In a preclinical study, Brukinsa (zanubrutinib) inhibited BTK signaling and proliferation of mantle cell lymphoma cell lines in culture, and demonstrated antitumor activity in a mantle cell lymphoma cell line xenograft model (ibrutinib) (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 2597). detail...
Unknown unknown mantle cell lymphoma not applicable Zanubrutinib FDA approved Actionable In a Phase II trial (BGB-3111-206) that supported FDA approval, Brukinsa (zanubrutinib) treatment resulted in an overall response rate of 83.5% (71/85, 50 complete response, 21 partial response) of patients with relapsed or refractory mantle cell lymphoma who received 1 to 4 prior treatments (Blood (2018) 132 (Supplement 1): 148; NCT03206970). detail... detail...
Unknown unknown colon carcinoma not applicable Oxaliplatin + VLX600 Preclinical - Cell culture Actionable In a preclinical study, VLX600 combined with Eloxatin (oxaliplatin) synergistically inhibited viability of colon carcinoma cells in culture (PMID: 24548894). 24548894
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 stomach cancer not applicable Cisplatin + Fluorouracil + Sapitinib Preclinical - Pdx Actionable In a preclinical study, Sapitinib (AZD8931), in combination with Platinol (cisplatin) and Adrucil (fluorouracil), demonstrated safety and an additive effect in a primary gastric cancer xenograft model (Cancer Res April 15, 2013 73; 2090). detail...
Unknown unknown prostate cancer no benefit Abiraterone + Dactolisib Phase I Actionable In a Phase Ib trial, the combination of Zytiga (abiraterone) and Dactolisib (BEZ235) did not demonstrate positive safety and tolerability, and further study of this combination is not planned (PMID: 28282611; NCT01634061). 28282611
Unknown unknown Ewing sarcoma not applicable Temozolomide + Veliparib Preclinical Actionable In a preclinical study, Ewing sarcoma cells treated with Temodar (temozolomide) combined with Veliparib (ABT-888) resulted in strong synergism, demonstrating reduced cell viability in culture (PMID: 26438158). 26438158
Unknown unknown 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 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 Advanced Solid Tumor no benefit Aprutumab ixadotin Phase I Actionable In a Phase I trial, Aprutumab ixadotin (BAY1187982) was poorly tolerated, resulted in no objective response (0/20) and 1 stable disease in patients with advanced solid tumors known to be FGFR2-positive, and the trial was terminated early as a result (PMID: 31502117; NCT02368951). 31502117
Unknown unknown mature T-cell and NK-cell lymphoma not applicable DS-3201b Phase I Actionable In a Phase I trial, DS-3201b demonstrated preliminary clinical activity in patients with T-cell lymphoma, with an overall response rate of 80% (4/5; 1 complete response/remission, and 3 partial responses) (Blood Dec 2017, 130 (Suppl 1) 4070; NCT02732275). detail...
Unknown unknown breast cancer not applicable VS-5584 Preclinical - Patient cell culture Actionable In a preclinical study, treatment with VS-5584 resulted in decreased cancer stem cell (CSC) number in a triple-negative breast cancer cell line in culture and in xenograft models, and decreased CSCs in patient breast cancer tumor samples in culture (PMID: 25432176). 25432176
Unknown unknown breast cancer not applicable GSK1904529A Preclinical - Cell culture Actionable In a preclinical study, GSK1904529A treatment resulted in decreased cell viability of breast cancer cells in culture (PMID: 19383820). 19383820
Unknown unknown leiomyosarcoma 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). detail... 28774898
Unknown unknown ovarian cancer not applicable E7449 Phase I Actionable In a Phase I trial, E7449 treatment inhibited Parp activity in peripheral blood mononuclear cells and resulted in partial response in 40% (2/5) of the patients with ovarian cancer (J Clin Oncol 36, 2018 (suppl; abstr 2505); abstr e19531; NCT01618136). detail...
Unknown unknown glioblastoma multiforme no benefit Perifosine Phase II Actionable In a Phase II trial, Perifosine (KRX-0401) was well tolerated, but did not demonstrate activity in patients with recurrent glioblastoma multiforme, with a 6-month progression-free rate of 0% (0/16), a median progression-free survival of 1.58 months, and a median overall survival of 3.68 months (PMID: 31325145). 31325145
Unknown unknown leukemia not applicable TAK-960 Preclinical - Cell line xenograft Actionable In a preclinical study, colorectal cancer cells treated with TAK-960 demonstrated cell growth inhibition, decreased tumor size, and increased median survival in both culture and cell line xenograft models (PMID: 22188812). 22188812
Unknown unknown glioblastoma multiforme not applicable Relatlimab + Urelumab Phase I Actionable In a Phase I trial, Relatlimab (BMS-986016), in combination with Urelumab (BMS-986016), resulted in a medium overall survival of 7 months, as compared to 8 months with Relatlimab (BMS-986016) alone, or 14 months with Urelumab (BMS-986016) alone, in patients with glioblastoma (J of Clin Oncol 37, 2019 (suppl; abstr 2017); NCT02658981). detail...
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 colon cancer not applicable BO-112 Preclinical - Cell culture Actionable In a preclinical study, BO-112 treatment inhibited viability of mouse colon carcinoma cells, and reduced cell viability, and induced cytotoxicity and apoptosis in human colon cancer cell lines in culture, and intratumoral delivery of BO-112 reduced tumor growth in a syngeneic mouse model of colon carcinoma (PMID: 31046839). 31046839
Unknown unknown Advanced Solid Tumor not applicable ABT-348 Preclinical - Pdx & cell culture Actionable In a preclinical study, ABT-348 (Ilorasertib) inhibited proliferation and promoted apoptosis in various solid tumor cell culture and Pdx models, including NSCLC, ovarian, and colon (AACR; Mol Cancer Ther 2011;10(11 Suppl):Abstract nr B231). detail...
Unknown unknown 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 colon cancer not applicable Cetuximab + Temsirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Torisel (temsirolimus) increased sensitivity to Erbitux (cetuximab) in cell line xenograft models of colon cancer (PMID: 24493623). 24493623
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 lung carcinoma not applicable BNC105P Preclinical - Cell line xenograft Actionable In a preclinical study, BNC105P treatment disrupted tumor vasculature and inhibited tumor growth in a cell line xenograft model of anaplastic lung carcinoma (PMID: 20515948). 20515948
Unknown unknown thyroid gland cancer not applicable Bevacizumab + Cetuximab Preclinical Actionable In a preclinical study, Erbitux (cetuximab), in combination with Avastin (bevacizumab), inhibited tumor growth and angiogenesis in mouse models of anaplastic thyroid cancer (PMID: 17429874). 17429874
Unknown unknown lymphoma not applicable SB 11285 Preclinical Actionable In a preclinical study, treatment with SB 11285 delayed and inhibited tumor growth in a syngeneic mouse model of lymphoma ournal of Clinical Oncology 2017 35:15_suppl, e14616). detail...
Unknown unknown pancreatic cancer not applicable Gemcitabine + PG545 Preclinical - Cell line xenograft Actionable In a preclinical study, human pancreatic cancer cells demonstrated increased apoptosis and tumor growth suppression both in culture and in cell line xenograft models when treated with a combination of PG545 and Gemzar (gemcitabine) (PMID: 25669977). 25669977
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 basal cell carcinoma not applicable Sonidegib FDA approved Actionable In a Phase II trial that supported FDA approval, treatment with Odomzo (sonidegib) resulted in an objective response in 36% (20/55) of patients with locally advanced basal cell carcinoma (PMID: 25981810). 25981810 detail...
Unknown unknown multiple myeloma not applicable BAY 1238097 Preclinical Actionable In a preclinical study, BAY 1238097 inhibited tumor growth and was well tolerated in acute myeloid leukemia and multiple myeloma models (American Association for Cancer Research; 2015 Apr 18-22; Abstract nr 3524). detail...
Unknown unknown 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 multiple myeloma not applicable GNE-272 Preclinical - Cell culture Actionable In a preclinical study, GNE-272 treatment resulted in anti-proliferative activity in multiple myeloma cell lines in culture (PMID: 27682507). 27682507
Unknown unknown breast cancer not applicable XL388 Preclinical - Cell culture Actionable In a preclinical study, XL388 treatment led to tumor growth inhibition in breast cancer cell line xenograft models (PMID: 23394126). 23394126
Unknown unknown uveal melanoma not applicable Fotemustine + S44563 Preclinical - Pdx Actionable In a preclinical study, treatment with S44563 in combination with or after Fotemustine resulted in improved efficacy compared to either agent alone in patient-derived xenograft models of uveal melanoma (PMID: 24454684). 24454684
Unknown unknown 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 lung non-small cell carcinoma not applicable Cisplatin + Gemcitabine + Metformin Phase 0 Actionable In a pilot clinical trial, treatment with Glucophage (metformin) in combination with Gemzar (gemcitabine) and Platinol (cisplatin) resulted in an overall response rate of 46.7%, compared to 13.3% with Gemzar (gemcitabine) plus Platinol (cisplatin) therapy in patients with non-small cell lung cancer, but this difference was not statistically significant (PMID: 26434885). 26434885
Unknown unknown gastrointestinal stromal tumor not applicable Dasatinib Phase 0 Actionable In a clinical trial, Sprycel (dasatinib) treatment resulted in a 6-month progression-free survival (PFS) rate of 29% (n=48), median PFS of 2.9 months, median overall survival of 19 months, and partial response in 25% (12/48) of patients with Gleevec (imatinib)-resistant gastrointestinal stromal tumor (PMID: 29710216). 29710216
Unknown unknown adrenocortical carcinoma not applicable Avelumab Phase Ib/II Actionable In a Phase Ib trial (JAVELIN), Bavencio (avelumab) treatment demonstrated safety and some anti-tumor activity in patients with metastatic adrenocortical carcinoma, resulting in an overall response rate of 6% (3/50, all partial responses), stable disease in 42% (21/50), a median progression-free survival (PFS) of 2.6 months, 6 mo. and 12 mo. PFS rates of 20.9% and 8.7% respectively, a median overall survival (OS) of 10.6 mo, and a 1-year OS of 43.4% (PMID: 30348224; NCT01772004). 30348224
Unknown unknown T-cell acute lymphoblastic leukemia not applicable Asparaginase + Dexamethasone + DT2216 + Vincristine Sulfate 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 acute myeloid leukemia not applicable BAY 1238097 Preclinical Actionable In a preclinical study, BAY 1238097 inhibited tumor growth and was well tolerated in acute myeloid leukemia and multiple myeloma models (American Association for Cancer Research; 2015 Apr 18-22; Abstract nr 3524). detail...
Unknown unknown 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 ovarian cancer no benefit Vandetanib Phase II Actionable In a Phase II trial, treatment with Caprelsa (vandetanib) in ovarian cancer patients resulted in no benefit and led to early termination of the trial (PMID: 20068097). 20068097
Unknown unknown glioblastoma multiforme not applicable Selinexor Phase II Actionable In a Phase II trial, Selinexor (KPT-330) treatment resulted in partial response in 11% (3/27) and stable disease in 22% (6/27) of patients with glioblastoma multiforme (J Clin Oncol 34, 2016 (suppl; abstr 2077)). detail...
Unknown unknown stomach cancer 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 0% (0/15) and a disease control rate of 27% (4/15) in immunotherapy-naive patients with advanced or metastatic gastric cancer, response occurred regardless of PD-L1, CCR4, CD8 expression levels and tumor mutational burden (PMID: 31455681; NCT02476123). 31455681
Unknown unknown colorectal cancer not applicable Fluorouracil + Quinacrine + Sorafenib Preclinical - Cell culture Actionable In a preclinical study, Acrichine (quinacrine) and Adrucil (5-fluorouracil) synergistically enhanced the cytotoxicity of Nexavar (sorafenib) in human colorectal cancer cell lines in culture (PMID: 21725213). 21725213
Unknown unknown 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 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 colon cancer not applicable CCT244747 + Irinotecan Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of CCT244747 and Camptosar (irinotecan) slowed tumor growth to a greater degree than treatment with either agent alone in colon cancer xenograft models (PMID: 22929806). 22929806
Unknown unknown ovarian cancer not applicable VS-5584 Preclinical - Cell line xenograft Actionable In a preclinical study, VS-5584 reduced the proportion of cancer stem cells in primary ovarian tumors in culture, and reduced tumorigenicity of dissociated human ovarian tumors in xenograft models (PMID: 25432176). 25432176
Unknown unknown 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 colon carcinoma not applicable Bevacizumab + S-49076 Preclinical - Cell line xenograft Actionable In a preclinical study, S-49076, in combination with Avastin (bevacizumab) arrested tumor growth in cell line xenograft models of colon carcinoma with previous resistance to Avastin (bevacizumab) (PMID: 23804704). 23804704
Unknown unknown prostate adenocarcinoma not applicable NSC23766 Preclinical - Cell culture Actionable In a preclinical study, NSC23766 inhibited proliferation and invasion of a prostate adenocarcinoma cell line in culture (PMID: 15128949). 15128949
Unknown unknown mantle cell lymphoma not applicable ST7612AA1 Preclinical Actionable In a preclinical study, ST7612AA1 inhibited proliferation of mantle cell lymphoma cell lines in culture (PMID: 25671299). 25671299
Unknown unknown 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 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 breast cancer not applicable Pazopanib Phase II Actionable In a Phase II trial, Votrient (pazopanib) displayed safety and some efficacy in breast cancer patients (PMID: 20682606). 20682606
Unknown unknown lymphoma not applicable PQR309 Preclinical - Cell culture Actionable In a preclinical study, PQR309 inhibited proliferation of lymphoma cells in culture, which was found to be due to the induction cell cycle arrest (PMID: 29066507). 29066507
Unknown unknown acute myeloid leukemia not applicable Flotetuzumab Phase I Actionable In a Phase I trial, Flotetuzumab (MGD006) demonstrated safety and preliminary efficacy in patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome, resulted in anti-leukemic activity in 57% (8/14) of patients and an overall response rate of 43% (6/14, 3 complete response, 1 CRi, 1 MLF, 1 partial response) (Blood 2017 130(Suppl 1):637). detail...
Unknown unknown multiple myeloma not applicable bb2121 Phase II Actionable In a Phase II trial (KarMMa), Idecabtagene vicleucel (bb2121) treatment resulted in an objective response of 73% (94/128) in patients with relapsed or refractory multiple myeloma, with a median duration of response of 10.6 months and a median progression-free survival of 8.6 months (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 8503-8503; NCT03361748). detail...
Unknown unknown multiple myeloma not applicable bb2121 Phase I Actionable In a Phase I trial, bb2121 treatment in multiple myeloma patients resulted in an overall response rate of 78% (7/9), including two patients who experienced complete remission, four patients who achieved partial response, and one patient who experienced stable disease (EORTC-NCI-AACR Symposium, 2016, Abstract #14). detail...
Unknown unknown diffuse large B-cell lymphoma not applicable Ibrutinib + PQR309 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination therapy of PQR309 and Imbruvica (ibrutinib) led to a synergistic effect in 6/7 diffuse large B-cell lymphoma (DLBCL) cell lines in culture, and the effect was confirmed in a DLBCL cell line xenograft model (PMID: 29066507). 29066507
Unknown unknown lung small cell carcinoma not applicable AZD2811 Case Reports/Case Series Actionable In a Phase II trial, a patient with small cell lung cancer demonstrated stable disease, with a duration of 3.7 months, when treated with AZD2811 (PMID: 32584426; NCT02688894). 32584426
Unknown unknown mantle cell lymphoma not applicable Lenalidomide FDA approved Actionable In a Phase II trial (EMERGE) that supported FDA approval, Revlimid (lenalidomide) treatment resulted in an objective response rate of 28% (37/134) with rapid time to response (2.2 months), a median duration of response of 16.6 months, a median progression-free survival of 4.0 months, and a median overall survival of 19.0 months in patients with mantle cell lymphoma who relapsed or progressed after or were refractory to Velcade (bortezomib) (PMID: 24002500; NCT00737529). 24002500 detail...
Unknown unknown lung non-small cell carcinoma not applicable Adavosertib Preclinical - Cell line xenograft Actionable In a preclinical study, Adavosertib (MK-1775) inhibited cell proliferation and promoted DNA damage in human non-small cell lung carcinoma cell lines in culture, and promoted tumor regression in xenograft models (PMID: 23699655). 23699655
Unknown unknown colorectal cancer not applicable R11.1.6 + Wortmannin Preclinical - Cell culture Actionable In a preclinical study, R11.1.6 and Wortmannin combination therapy did not inhibit cell proliferation in a colorectal cancer cell line in culture (PMID: 29720559). 29720559
Unknown unknown colon cancer not applicable E7046 + Radiotherapy Preclinical Actionable In a preclinical study, the combination treatment of E7046 and radiotherapy resulted in 9 out of 12 tumor free colon cancer mouse models and increased the number of T-cells infiltrating the tumor (International Journal of Rad Onc, 2016, 96;2, S128). detail...
Unknown unknown lymphoma not applicable Ixazomib + JQ1 Preclinical Actionable In a preclinical study, Ixazomib (MLN9708) and JQ1 combination treatment resulted in increased apoptosis in T-cell lymphoma cells in culture (PMID: 26988986). 26988986
Unknown unknown neuroblastoma not applicable GANT61 Preclinical - Cell line xenograft Actionable In a preclinical study, GANT61 induced apoptosis and decreased growth of neuroblastoma cells in culture, and inhibited tumor growth in neuroblastoma cell line xenograft models (PMID: 22949014). 22949014
Unknown unknown prostate cancer not applicable Capivasertib + Enzalutamide Phase I Actionable In a Phase I trial, Capivasertib (AZD5363) and Xtandi (enzalutamide) combination treatment was well-tolerated and demonstrated safety, and resulted in an overall response rate of 25% (3/12) in evaluable metastatic castration-resistant prostate cancer patients (n=16) harboring either a PTEN loss or AKT1 E17K (PMID: 32205016; NCT02525068). 32205016
Unknown unknown colon cancer not applicable FN-1501 Preclinical - Cell culture Actionable In a preclinical study, FN-1501 induced cell-cycle arrest decreased proliferation in a colon cancer cell line in culture (PMID: 29357250). 29357250
Unknown unknown colorectal cancer not applicable Panitumumab FDA approved Actionable In an open-label clinical trial that supported FDA approval, Vectibix (panitumumab) treatment in patients with metastatic colorectal cancer resulted in increased progression-free survival (13.8 weeks; SD=0.76) compared to best supportive care alone (8.5 weeks; SD=0.54) (PMID: 18316547). 18316547 detail...
Unknown unknown oral squamous cell carcinoma not applicable Temsirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Torisel (temsirolimus) inhibited proliferation and migration of oral squamous cell carcinoma cells in culture and suppressed tumor growth in cell line xenograft models of human oral squamous cell carcinoma (PMID: 20858724). 20858724
Unknown unknown marginal zone B-cell lymphoma not applicable LAM-002A Phase I Actionable In a Phase I trial, LAM-002A demonstrated safety and preliminary efficacy, resulted in prolonged stable disease in 1 of 3 patients with marginal zone lymphoma (Blood 2017 130 (Suppl 1):4119). detail...
Unknown unknown bladder urothelial carcinoma not applicable VAX014 Preclinical Actionable In a preclinical study, VAX014 induced cell-killing in dissociated mouse and human urothelial carcinoma cell lines in culture, and inhibited tumor growth and improved survival in a syngeneic bladder cancer model (PMID: 27919942). 27919942
Unknown unknown ovarian cancer no benefit Docetaxel + Vandetanib Phase II Actionable In a Phase II trial, the combination of Caprelsa (vandetanib) plus Taxotere (docetaxel) did not result in a greater PFS when compared to Taxotere (docetaxel) alone (PMID: 24709487). 24709487
Unknown unknown Advanced Solid Tumor not applicable Toripalimab Phase I Actionable In a Phase I trial, Toripalimab (JS001) demonstrated tolerability and preliminary efficacy in patients with advanced malignancies, resulting in an objective response rate of 45.5% (15/33), including complete response in 7/33, partial response in 8/33, and stable disease in 13/33 patients, and median progression-free survival of 7.1 months, with solid tumor patients demonstrating an objective response rate of 22.7% (5/22) and median progression-free survival of 5.7 months (PMID: 32224416; NCT02836834). 32224416
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 lung non-small cell carcinoma not applicable Dostarlimab + TSR-033 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with TSR-033 and Dostarlimab (TSR-042) in combination resulted in increased T-cell number and increased T-cell proliferation in a non-small cell lung cancer cell line xenograft model and had an additive effect on tumor growth inhibition (PMID: 30587557). 30587557
Unknown unknown ovarian carcinoma not applicable VS-4718 Preclinical - Cell culture Actionable In a preclinical study, treatment with VS-4718 decreased phosphorylation of Ptk2 and induced cell-cycle arrest and apoptosis in ovarian carcinoma cells in culture (PMID: 24899686). 24899686
Unknown unknown diffuse large B-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 31% (4/13, 3 complete response, 1 partial response) in patients with relapsed/refractory diffuse large B-cell lymphoma (J Clin Oncol 36, 2018 (suppl; abstr 7520); NCT02756247). 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 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 leiomyosarcoma not applicable Ganetespib + Sirolimus Case Reports/Case Series Actionable In a Phase I trial, one patient with leiomyosarcoma demonstrated a partial response to treatment with the combination of Ganetespib and Rapamune (sirolimus) (PMID: 26244021; NCT02008877). 26244021
Unknown unknown hepatocellular carcinoma not applicable Droxinostat Preclinical - Cell culture Actionable In a preclinical study, droxinostat induced apoptosis and reduced growth of hepatocellular carcinoma cell lines in culture (PMID: 26947884). 26947884
Unknown unknown colon carcinoma not applicable Fluorouracil + VLX600 Preclinical - Cell culture Actionable In a preclinical study, VLX600 and Adrucil (fluorouracil) combination treatment inhibited viability of colon carcinoma cells in culture (PMID: 24548894). 24548894
Unknown unknown colorectal adenocarcinoma not applicable CRLX101 + Fluorouracil + Radiotherapy Preclinical - Cell line xenograft Actionable In a preclinical study, the addition of CRLX101 to Adrucil (fluorouracil) and radiotherapy resulted in a greater decrease in tumor volume in colorectal adenocarcinoma xenograft models when compared to the combination of Adrucil (fluorouracil) and radiotherapy only (PMID: 27784746). 27784746
Unknown unknown Advanced Solid Tumor not applicable Surufatinib Phase I Actionable In a Phase I clinical trial, Surufatinib (HMPL-012) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 30, 2012 (suppl; abstr 3040)). detail...
Unknown unknown Advanced Solid Tumor not applicable Surufatinib Phase I Actionable In a Phase I trial, Surufatinib (HMPL-012) demonstrated safety and preliminary efficacy in patients with advanced solid tumors, particularly in patients with neuroendocrine tumors (AACR; Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr A1). detail...
Unknown unknown colon cancer not applicable Navicixizumab Preclinical Actionable In a preclinical study, Navicixizumab (OMP-305B83) inhibited proliferation of endothelial cells in culture and demonstrated antitumor activity in vivo in multiple solid tumor types, including colon tumors (Mol Cancer Ther December 2015 14; C164). detail...
Unknown unknown Advanced Solid Tumor not applicable Romidepsin Phase I Actionable In a Phase I trial, Istodax (romidepsin), in combination with Gemzar (gemcitabine), demonstrated safety and some efficacy in patients with a variety of advanced solid tumors (J Clin Oncol, May 2008 vol. 26 no. 15_suppl 2567). detail...
Unknown unknown 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 Advanced Solid Tumor not applicable SR9009 Preclinical - Cell culture Actionable In a preclinicl study SR9009 demonstrated toxicity in a wide range of tumor cell lines harboring different driver mutations, but not in normal cell lines in culture (PMID: 29320480). 29320480
Unknown unknown multiple myeloma not applicable AMG 701 + Lenalidomide Preclinical Actionable In a preclinical study, AMG 701 and Revlimid (lenalidomide) combination treatment enhanced tumor growth inhibition and regression in a mouse model of multiple myeloma (Blood (2019) 134 (Supplement_1): 135). detail...
Unknown unknown pancreatic ductal adenocarcinoma not applicable Cisplatin + Gemcitabine Phase II Actionable In a Phase II trial, patients with pancreatic ductal adenocarcinoma harboring either a germline BRCA1, BRCA2, or PALB2 inactivating mutation demonstrated a response rate of 74.1% (20/27), a median progression-free survival of 10.1 months, a median overall survival of 15.5 months, and a disease control rate of 100% (27/27) when treated with a combination of Gemzar (gemcitabine) and Platinol (cisplatin) (PMID: 31976786). 31976786
Unknown unknown acute myeloid leukemia not applicable AMG 900 Phase I Actionable In a Phase I clinical trial, AMG 900 treatment resulted in a complete response in 9% (3/35) of adult patients acute myeloid leukemia (PMID: 28370201). 28370201
Unknown unknown glioblastoma multiforme not applicable Alisertib + MP7 Preclinical - Cell culture Actionable In a preclinical study, the combination of MP7 and Alisertib (MLN8237) resulted in an additive effect in glioblastoma cells in culture, demonstrating an increase in antiproliferative activity, decreased cell viability, and inhibition of glioma stem cell colony formation (PMID: 27797168). 27797168
Unknown unknown lymphoma not applicable Marizomib Phase I Actionable In a Phase I trial, Marizomib (NPI-0052) treatment resulted in complete response lasting more than 10 treatment cycles in 7.1% (1/14) of lymphoma patients (PMID: 27117181). 27117181
Unknown unknown hematologic cancer not applicable BP1001 + Cytarabine Phase I Actionable In a Phase I/Ib trial, BP1001 in combination with Cytosar-U (cytarabine) resulted in complete remission in 28.6% (2/7), complete remission with incomplete haematological recovery in 14.3% (1/7), and stable disease in 28.6% (2/7) of patients with refractory or relapsed hematological malignancies (PMID: 29550383; NCT01159028). 29550383
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 lung non-squamous non-small cell carcinoma not applicable Carboplatin + Demcizumab + Pemetrexed Disodium Phase Ib/II Actionable In a Phase Ib trial, Demcizumab (OMP-21M18) in combination with Paraplatin (carboplatin) and Alimta (pemetrexed) demonstrated safety and preliminary efficacy, resulted in objective tumor response in 50% (20/40) of treatment-naive patients with metastatic non-squamous non-small cell lung cancer (PMID: 29188408; NCT01189968). 29188408
Unknown unknown brain stem glioma not applicable Olaparib Preclinical - Cell culture Actionable In a preclinical study, Lynparza (olaparib) resulted in decreased cell viability of some diffuse intrinsic pontine glioma cell lines in culture (PMID: 26351319). 26351319
Unknown unknown chronic lymphocytic leukemia not applicable Umbralisib Phase I Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment resulted in objective response in 85% (17/20) and stable disease in 15% (3/20) of patients with chronic lymphocytic leukemia (PMID: 29475723; NCT01767766). 29475723
Unknown unknown chronic lymphocytic leukemia not applicable Umbralisib Phase II Actionable In a Phase I trial, Umbralisib (TGR-1202) treatment demonstrated safety and preliminary efficacy, with 90% of chronic lymphocytic leukemia patients achieved progression-free survival at 6.5 months (J Clin Oncol 36, 2018 (suppl; abstr 7530); NCT02742090). detail...
Unknown unknown acute leukemia not applicable Volasertib Phase I Actionable In a Phase I trial, Volasertib (BI 6727) demonstrated safety and limited efficacy in pediatric patients with acute leukemia or advanced solid tumors, with stable disease as best objective response in 71% (5/7) of patients with acute leukemia and in 13% (2/15) of patients with advanced solid tumors (PMID: 31276318; NCT01971476). 31276318
Unknown unknown Advanced Solid Tumor not applicable BMS-986156 Phase Ib/II Actionable In a Phase I/IIa trial, patients with advanced solid tumors treated with a combination of Opdivo (nivolumab) and BMS-986156 demonstrated preliminary antitumor activity (Journal of Clinical Oncology 35, no. 15_suppl (May 2017) 104-104; NCT02598960). detail...
Unknown unknown Advanced Solid Tumor not applicable PQR309 Phase I Actionable In a Phase I trial, PQR309 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2560)). detail...
Unknown unknown diffuse large B-cell lymphoma not applicable SEL24-B489 Preclinical - Cell line xenograft Actionable In a preclinical study, SEL24-B489 treatment induced apoptosis in diffuse large B-cell lymphoma cells in both culture and xenograft models (Blood 126 (23):706.December 2015). detail...
Unknown unknown breast cancer not applicable Paclitaxel + Vantictumab Preclinical - Pdx Actionable In a preclinical study, Vantictumab (OMP-18R5) worked synergistically with Taxol (paclitaxel) to inhibit tumor growth in patient-derived xenograft models of breast cancer (PMID: 22753465). 22753465
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 Advanced Solid Tumor not applicable Tazemetostat Phase I Actionable In a Phase I trial, Tazemetostat (EPZ-6438) demonstrated safety and preliminary efficacy, resulted in an objective response in 5% (2/43) in patients with advanced solid tumors (PMID: 29650362; NCT01897571). 29650362
Unknown unknown sarcoma not applicable Epirubicin + Panobinostat Phase I Actionable In a Phase I trial, 55% (11/20) of sarcoma patients demonstrated stable disease while one patient (1/20) experienced a partial response when treated with a combination of Farydak (panobinostat) and Ellence (epirubicin) (PMID: 26903311). 26903311
Unknown unknown Advanced Solid Tumor not applicable AZD8186 Phase I Actionable In a Phase I trial, AZD8186 demonstrated safety and preliminary efficacy in a variety of advanced solid tumor patients (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr CT329). detail...
Unknown unknown 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 B-cell lymphoma not applicable JCAR017 Phase I Actionable In a Phase I trial, JCAR017 treatment resulted in a response rate of 80% (22/28) with a complete response rate of 60% (17/28) in patients with relapsed/refractory B-cell non-Hodgkin lymphoma (J Clin Oncol 35, 2017 (suppl; abstr 7513); NCT02631044). detail...
Unknown unknown 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 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 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 uveal melanoma not applicable Pegilodecakin Phase I Actionable In a Phase I trial, treatment with AM0010 at the active dose resulted in partial response in 21% (5/24) of evaluable advanced solid tumor patients, including 1 uveal melanoma patient who also demonstrated reduction of gastric metastases (PMID: 27528724; NCT02009449). 27528724
Unknown unknown Advanced Solid Tumor not applicable BAY 1238097 Clinical Study Actionable In a Phase I trial, BAY 1238097 therapy resulted in zero objective responses and stable disease in 25% (2/8) of patients with refractory advanced solid tumors; due to high toxicity the trial was terminated prematurely (PMID: 30711772; NCT02369029). 30711772
Unknown unknown colon cancer not applicable AsiDNA Preclinical - Cell culture Actionable In a preclinical study, AsiDNA inhibited survival of colon cancer cell lines in culture (PMID: 27559053). 27559053
Unknown unknown lung small cell carcinoma not applicable Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) treatment in small cell lung cancer patients resulted in a partial response of 11% (1/9) and stable disease in 30% (3/9) (PMID: 26716400). 26716400
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 acute myeloid leukemia not applicable SNS-229 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with SNS-229 resulted in decreased PDPK1 pathway signaling and tumor growth inhibition in an acute myeloid leukemia cell line xenograft model (Mol Cancer Ther, Dec 1 2015 (14) (12 Supplement 2) C198). detail...
Unknown unknown esophageal carcinoma not applicable BI 853520 Phase I Actionable In a Phase I trial, BI 853520 demonstrated safety and some anti-tumor efficacy, resulting in stable disease in 12.5% (2/16) of patients with esophageal carcinoma (PMID: 30756308; NCT01335269). 30756308
Unknown unknown Advanced Solid Tumor not applicable Capmatinib Phase I Actionable In a Phase I trial, Tabrecta (capmatinib) demonstrated safety and preliminary efficacy, resulted in stable disease as best overall response in 18% (8/44) of patients with advanced solid tumors (PMID: 30724423). 30724423
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 Advanced Solid Tumor not applicable BTP-114 Preclinical - Cell line xenograft Actionable In a preclinical study, BTP-114 inhibited growth of tumor cells in culture and in xenograft animal models (Cancer Res 2015;75(15 Suppl):Abstract nr 4484). detail...
Unknown unknown glioblastoma multiforme not applicable Olaparib + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, treatment with Lynparza (olaparib) enhanced the sensitivity of glioblastoma cell lines to radiation therapy in culture, demonstrating greater decreased cell survival when compared to cells not treated with Lynparza (olaparib) (PMID: 32347934). 32347934
Unknown unknown thyroid gland carcinoma not applicable Vandetanib Phase II Actionable In a Phase II trial, Caprelsa (vandetanib) demonstrated efficacy in patients with advanced differentiated thyroid carcinoma (PMID: 22898678). 22898678
Unknown unknown multiple myeloma not applicable Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, treatment with EDO-S101 induced apoptosis and decreased viability of a multiple myeloma cell line in culture (PMID: 28753594). 28753594
Unknown unknown lung small cell carcinoma not applicable Sabatolimab + Spartalizumab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in partial response in a patient with small cell lung cancer (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown oral cavity cancer not applicable Capmatinib Case Reports/Case Series Actionable In a Phase I trial, Tabrecta (capmatinib) demonstrated safety and preliminary efficacy, resulted in stable disease as best overall response in a patients with oral cavity cancer (PMID: 30724423). 30724423
Unknown unknown glioblastoma multiforme not applicable BLZ945 + Buparlisib Preclinical Actionable In a preclinical study, the combination of Buparlisib (BKM120) and BLZ945 compared to BLZ945 alone resulted in enhanced survival and tumor regression in transgenic mouse models of glioblastoma (PMID: 27199435). 27199435
Unknown unknown 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 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 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 chronic lymphocytic leukemia not applicable Cerdulatinib + Venetoclax Preclinical - Patient cell culture Actionable In a preclinical study, the combination of Cerdulatinib (PRT062070) and Venclexta (venetoclax) worked synergistically to induce apoptosis of patient-derived chronic lymphocytic leukemia cells in culture, and resulted in decreased cell viability compared to either drug as a single agent (PMID: 27697994). 27697994
Unknown unknown malignant glioma not applicable PQ401 Preclinical - Cell line xenograft Actionable In a preclinical study, PQ401 disrupted cell migration and inhibited growth of glioma cells in culture, and suppressed tumor growth in cell line xenograft models (PMID: 25971682). 25971682
Unknown unknown lung non-squamous non-small cell carcinoma no benefit Cisplatin + LY2603618 + Pemetrexed Disodium Phase II Actionable In a Phase II trial, combination of LY2603618 with Alimta (pemetrexed) and Platinol (cisplatin) improved progression-free survival (4.7 vs 1.5 months) compared to placebo in nonsquamous non-small cell lung cancer patients, but also increased risk of serious thromboembolic events, thus the combination would no be developed further (PMID: 28625637; NCT01139775). 28625637
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 transitional cell carcinoma not applicable Sabatolimab + Spartalizumab Case Reports/Case Series Actionable In a Phase Ib/II trial, MBG453 and Spartalizumab (PDR001) combination treatment resulted in stable disease in 3 patients with transitional cell carcinoma (AACR Annual Meeting 2019, Abstract CT183; NCT02608268). detail...
Unknown unknown 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 colorectal cancer no benefit Atezolizumab + Cobimetinib Phase III Actionable In a Phase III trial (IMblaze370), Tecentriq (atezolizumab) and Cotellic (cobimetinib) combination treatment did not improve median overall survival (8.87 vs 8.51 months, HR=1.00, p=0.99) compared to Stivarga (regorafenib) in patients with chemotherapy-refractory metastatic colorectal cancer, 91.7% of whom were microsatellite stable or microsatellite instability-low (PMID: 31003911; NCT02788279). 31003911
Unknown unknown colorectal cancer no benefit Atezolizumab + Cobimetinib Phase I Actionable In a Phase Ib trial, Tecentriq (atezolizumab) and Cotellic (cobimetinib) combination treatment resulted in partial response in 7% (7/84) of patients with metastatic colorectal cancer, with a median duration of response of 14.8 months, a disease control rate of 31% (26/84), a median progression-free survival of 1.9 months, and a median overall survival of 10.0 months (PMID: 30918950; NCT01988896). 30918950
Unknown unknown colon cancer not applicable AZ628 + CPI-455 Preclinical - Cell culture Actionable In a preclinical study, a colon cancer cell line treated with AZ628 demonstrated increased sensitivity when co-treated with CPI-455 in culture, resulting in decreased survival of cells, in particular the cells that eventually develop drug resistance (PMID: 27214401). 27214401
Unknown unknown Advanced Solid Tumor not applicable AZD3965 Phase I Actionable In a Phase I trial, AZD3965 treatment demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Annals of Oncology, Volume 29, Issue suppl_3, 1 March 2018, abstract 500). detail...
Unknown unknown lung non-small cell carcinoma not applicable Pimitespib Phase I Actionable In a Phase I trial, TAS-116 treatment resulted in partial response in 2 patients with non-small cell lung carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 2546)). detail...
Unknown unknown ovarian cancer not applicable 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 triple-receptor negative breast cancer not applicable Selumetinib Preclinical - Cell line xenograft Actionable In a preclinical study, the Mek inhibitor Selumetinib (AZD6244) inhibited tumorigenicity and invasiveness of triple-receptor negative breast cancer cell lines in culture and in cell line xenograft models (PMID: 26384399). 26384399
Unknown unknown head and neck squamous cell carcinoma not applicable Adavosertib + Cisplatin + Docetaxel Phase I Actionable In a Phase I trial, neoadjuvant treatment with Adavosertib (MK-1775) in combination with Platinol (cisplatin) and Taxotere (docetaxel) was well-tolerated and demonstrated preliminary anti-tumor activity in patients with head and neck squamous cell carcinoma, resulting in partial response in 5 patients and stable disease in 4 patients, out of 10 evaluable patients (PMID: 29535125). 29535125
Unknown unknown colorectal cancer not applicable Bevacizumab + Fluorouracil + Irinotecan + Leucovorin + Oxaliplatin Phase II Actionable In a Phase II trial, the combination of Avastin (bevacizumab) plus FOLFOXIRI chemotherapy was well-tolerated and resulted in improved progression-free survival compared to Avastin (bevacizumab) plus FOLFOX in colorectal cancer patients (J Clin Oncol 35, 2017 (suppl 4S; abstract 658)). detail...
Unknown unknown hepatocellular carcinoma not applicable Foretinib Phase I Actionable In a Phase I trial, Foretinib (GSK1363089) demonstrated safety and activity in hepatocellular carcinoma patients, with an overall response rate by mRESCIST of 22.9% (8/35, all partial responses), a disease stabilization rate of 82.9% (29/35), and a median duration of response of 7.6 months (PMID: 27821605; NCT00920192). 27821605
Unknown unknown triple-receptor negative breast cancer not applicable PF-06647263 Phase I Actionable In a Phase I trial, PF-06647263 treatment resulted in partial response in 2 patients with triple-receptor negative breast cancer (TNBC) in the solid tumor dose escalation cohort, but no objective response (0/10) in the TNBC dose expansion cohort (J Clin Oncol 35, 2017 (suppl; abstr 2511)). detail...
Unknown unknown prostate cancer not applicable X480 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with X480 in prostate cancer cell lines resulted in reduced cell proliferation and induced apoptotic activity in culture, and decreased tumor load and metastasis in cell line xenograft models (Eur J Cancer, 2012, 48, S5:235). detail...
Unknown unknown triple-receptor negative breast cancer not applicable Birabresib Preclinical - Cell culture Actionable In a preclinical study, triple-receptor negative breast cancer cells demonstrated sensitivity to treatment with Birabresib (OTX015), resulting in decreased cell proliferation in culture (PMID: 27256375). 27256375
Unknown unknown Advanced Solid Tumor not applicable APTO-253 Phase I Actionable In a Phase I clinical trial, APTO-253 demonstrated safety and resulted in stable disease in 24% (5/32) of evaluable solid tumor patients (PMID: 26268924). 26268924
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 Advanced Solid Tumor not applicable Chiauranib Phase I Actionable In a Phase I trial, Chiauranib (CS2164) demonstrated safety and preliminary efficacy, resulted in stable disease as best response in 66.7% (12/18) of patients with advanced solid tumors (PMID: 30642372; NCT02122809). 30642372
Unknown unknown 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 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 neuroblastoma not applicable GF109203X Preclinical - Cell culture Actionable In a preclinical study, GF109203X decreased growth of neuroblastoma cell lines in culture (PMID: 10209967). 10209967
Unknown unknown prostate cancer not applicable AGS-PSCA Phase Ib/II Actionable In a Phase Ib/II trial, AGS-PSCA treatment was deemed safe, but only resulted in limited antitumor activity in patients with castration resistant prostate cancer (PMID: 22553195). 22553195
Unknown unknown synovial sarcoma not applicable 211At-OTSA101 Preclinical - Cell line xenograft Actionable In a preclinical study, 211At-OTSA101 treatment inhibited tumor growth and increased survival of a cell line xenograft model of synovial sarcoma (PMID: 29952132). 29952132
Unknown unknown 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 stomach carcinoma not applicable Tivantinib Phase II Actionable In a Phase II trial, Tivantinib (ARQ197) demonstrated efficacy in previously treated patients with metastatic gastric cancer (PMID: 24337769). 24337769
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 acute monocytic leukemia not applicable Carfilzomib + Tinostamustine Preclinical - Cell culture Actionable In a preclinical study, EDO-S101 and Kyprolis (carfilzomib) worked synergistically to decrease viability of an acute monocytic leukemia cell line in culture (PMID: 28753594). 28753594
Unknown unknown multiple myeloma not applicable Daratumumab and hyaluronidase-fihj + Dexamethasone + Lenalidomide FDA approved Actionable In a Phase II trial (PLEIADES) that supported FDA approval, Darzalex Faspro (Daratumumab and hyaluronidase-fihj) demonstrated safety and efficacy comparable to Darzalex (daratumumab) when combined with Adexone (dexamethasone) and Revlimid (lenalidomide) in patients with relapsed or refractory multiple myeloma, resulted in an objective response rate of 93.8% (61/65, complete response 21.5%) (17th International Myeloma Workshop Sep 2019, Boston, MA, US. OAB-022). detail... detail...
Unknown unknown 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 lymphoplasmacytic lymphoma not applicable Idelalisib Phase II Actionable In a Phase II trial, Zydelig (idelalisib) treatment resulted in an overall response rate of 80% (8/10) in patients with lymphoplasmacytic lymphoma (PMID: 24450858; NCT01282424). 24450858
Unknown unknown melanoma not applicable SGN-CD228A Preclinical - Cell line xenograft Actionable In a preclinical study, SGN-CD228A treatment delayed tumor growth and resulted in complete responses in cell line xenograft models of melanoma (Cancer Res 2019;79(13 Suppl):Abstract nr 2688). detail...
Unknown unknown pancreatic ductal adenocarcinoma not applicable MitoMet-10 + Radiotherapy Preclinical Actionable In a preclinical study, MitoMet-10 increased the radiosensitivity of pancreatic ductal adenocarcinoma cells in culture (PMID: 27216187). 27216187
Unknown unknown hepatocellular carcinoma not applicable Fluorouracil + RU-A1 Preclinical - Cell culture Actionable In a preclinical study, RU-A1 enhanced the sensitivity of Adrucil (fluorouracil) treatment in hepatocellular carcinoma cells, demonstrating greater decreased cell survival and inhibition of colony formation in culture compared to treatment with either agent alone (PMID: 28589491). 28589491
Unknown unknown Advanced Solid Tumor not applicable Taselisib Phase I Actionable In a Phase I trial, Taselisib (GDC-0032) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Cancer Res Feb 2016 (76) (4 Supplement) P3-14-10). detail...
Unknown unknown Advanced Solid Tumor not applicable OSU03012 Phase I Actionable In a Phase I trial, OSU03012 (AR-12) treatment demonstrated safety and resulted in stable disease in 6% (2/30) of patients with advanced solid tumors, however, a new formulation was recommended due to limited absorption of the drug (J Clin Oncol 31, 2013 (suppl; abstr 2608)). detail...
Unknown unknown thymic carcinoma not applicable CC-90010 Case Reports/Case Series Actionable In a Phase I trial, CC-90010 treatment demonstrated safety, and resulted in a 17% tumor reduction in a patient with metastatic malignant thymoma (PMID: 32240793; NCT03220347). 32240793
Unknown unknown melanoma not applicable Lenvatinib Phase I Actionable In a Phase I trial, Lenvima (lenvatinib) was demonstrated to be well tolerated and displayed anti-tumor activity in patients with melanoma and renal cell carcinoma (PMID: 22516948). 22516948
Unknown unknown 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 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 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 colorectal cancer not applicable Oxaliplatin + Resminostat Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Resminostat (4SC-201) and Eloxatin (oxaliplatin) induced apoptosis in a colorectal cancer (CRC) cell line and primary colon cancer cells in culture, and inhibited tumor growth in a CRC cell line xenograft model, with increased activity compared to either agent alone (PMID: 26831668). 26831668
Unknown unknown Advanced Solid Tumor not applicable Pazopanib + Trametinib Phase I Actionable In a Phase I trial, combination treatment with Votrient (pazopanib) and Mekinist (trametinib) was tolerable and resulted in partial response in 12% (3/25), and stable disease in 72% (18/25) of patients with advanced solid tumors, and 9 patients remained on study for more than 6 cycles, including patients with differentiated thyroid cancer (n=3), colorectal cancer (n=2), melanoma (n=1), cholangiocarcinoma (n=1), ovarian cancer (n=1), and synovial cell sarcoma (n=1) (PMID: 31186313; NCT01438554). 31186313
Unknown unknown breast cancer not applicable BNC105P Preclinical - Cell line xenograft Actionable In a preclinical study, BNC105P treatment disrupted tumor vasculature, induced apoptosis and necrosis, inhibited tumor growth, and induced regression in a cell line xenograft model of breast cancer (PMID: 20515948). 20515948
Unknown unknown gastroesophageal junction adenocarcinoma not applicable Paclitaxel + Ramucirumab FDA approved Actionable In a Phase III trial (RAINBOW) that supported FDA approval, Cyramza (ramucirumab) and Taxol (paclitaxel) combination treatment significantly improved overall survival (9.6 vs 7.4 mo, HR=0.807, p=0.017) compared to Taxol (paclitaxel) alone in patients with advanced gastric or gastroesophageal junction adenocarcinoma who progressed on prior chemotherapy (PMID: 25240821; NCT01170663). 25240821 detail...
Unknown unknown neuroendocrine tumor not applicable Pembrolizumab Phase II Actionable In a Phase II trial (KEYNOTE-158), Keytruda (pembrolizumab) treatment demonstrated safety but limited efficacy in patients with heavily pretreated neuroendocrine tumors, resulting in a partial response in 3.7% (4/107, 3 pancreatic, 1 GI) and stable disease in 57.0% (61/107) of patients, all 4 responders were CD274 (PD-L1)-negative (PMID: 31980466; NCT02628067). 31980466
Unknown unknown breast cancer not applicable NU6027 + Rucaparib Preclinical - Cell culture Actionable In a preclinical study, NU6027 enhanced the efficacy of Rubraca (rucaparib) in breast cancer cells in culture, resulting in a greater decreased cell survival (PMID: 21730979). 21730979
Unknown unknown acute myeloid leukemia not applicable BI 836858 + Decitabine Preclinical - Patient cell culture Actionable In a preclinical study, treatment of patient derived acute myeloid leukemia blast cells with Dacogen (decitabine) enhanced BI 836858-mediated cytotoxic immune response in culture (PMID: 27013443). 27013443
Unknown unknown pancreatic ductal adenocarcinoma not applicable Fluorouracil + Oxaliplatin + Veliparib Phase Ib/II Actionable In a Phase I/II trial, Veliparib (ABT-888) in combination with Adrucil (fluorouracil) and Eloxatin (oxaliplatin) demonstrated safety, and resulted in an objective response rate of 26% (15/58, 4 complete responses, 11 partial responses), a disease control rate of 52%, a progression-free survival of 4.0 months, and an overall survival of 7.8 months in patients with metastatic pancreatic ductal adenocarcinoma (PMID: 32669374; NCT01489865). 32669374
Unknown unknown colon cancer not applicable BOS172722 Preclinical - Cell culture Actionable In a preclinical study, BOS172722 treatment resulted in increased apoptosis and decreased growth of a colon cancer cell line in culture (PMID: 31575759). 31575759
Unknown unknown multiple myeloma not applicable INCB054329 + Pemigatinib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of INCB054329 and Pemazyre (pemigatinib) decreased MYC expression and FGFR3 signaling and inhibited tumor growth in a t(4;14)-positive myeloma cell line xenograft model, demonstrating increased efficacy over either agent alone (PMID: 30206163). 30206163
Unknown unknown head and neck squamous cell carcinoma not applicable ALX148 + Pembrolizumab Phase I Actionable In a Phase I trial, ALX148 and Keytruda (pembrolizumab) combination treatment resulted in a partial response in 18% (3/17) and stable disease in 24% (4/17) of patients with head and neck squamous cell carcinoma that progressed on platinum therapy (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 2514-2514; NCT03013218). detail...
Unknown unknown glioblastoma multiforme not applicable Bevacizumab FDA approved Actionable In a Phase II trial that supported FDA approval, treatment with Avastin (bevacizumab) resulted in an objective response rate of 25.9% (22/85) with a mean duration of response of 4.2 months in patients with glioblastoma (PMID: 19897538). detail... 19897538
Unknown unknown acute myeloid leukemia no benefit AR-42 + Decitabine Phase I Actionable In a Phase I trial, AR-42 and Dacogen (decitabine) combination treatment in patients with acute myeloid leukemia resulted in an overall response rate of 23.1% (3/13), including one patient with complete remission and two patients with complete remission with incomplete count recovery, but the trial did not meet its biological endpoint for safety and dosing (PMID: 32037935; NCT01798901). 32037935
Unknown unknown Advanced Solid Tumor not applicable AMG 337 Phase I Actionable In a Phase I trial, AMG 337 demonstrated safety and preliminary efficacy in patients with a variety of advanced solid tumors (J Clin Oncol 33, 2015 (suppl; abstr e13538)). detail...
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 renal cell carcinoma not applicable Everolimus + Telaglenastat Phase I Actionable In a Phase I trial, the combination of CB-839 and Afinitor (everolimus) was well-tolerated and resulted in a disease control rate of 100% (8/8) in patients with papillary or clear cell renal cell carincoma, with 1 partial response, and 7 patients achieving stable disease (EORTC-NCI-AACR 2016, Abstract 26). detail...
Unknown unknown ovary adenocarcinoma not applicable Acalisib Preclinical - Cell line xenograft Actionable In a preclinical study, Acalisib (GS-9820) resulted in tumor growth inhibition in xenograft models of ovarian adenocarcinoma (Mol Cancer Ther 2009;8(12 Suppl):B136). detail...
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 breast cancer not applicable PI-273 Preclinical - Cell line xenograft Actionable In a preclinical study, PI-273 induced cell-cycle arrest and apoptosis and decreased proliferation in breast cancer cell lines with wild-type RAS in culture, and inhibited tumor growth in a xenograft model (PMID: 28827373). 28827373
Unknown unknown CLL/SLL not applicable ME-401 Phase I Actionable In a Phase I trial, ME-401 treatment was well-tolerated, resulted in an objective response rate of 89% (9/10) in patients with relapsed or refractory CLL/SLL (J Clin Oncol 38: 2020 (suppl; abstr 8016); NCT02914938). detail...
Unknown unknown mantle cell lymphoma not applicable Brexucabtagene autoleucel + Cyclophosphamide + Fludarabine Phase II Actionable In a Phase II (ZUMA-2) trial, conditioning chemotherapy including Cytoxan (cyclophosphamide) and Flurdara (fludarabine) followed by a single infusion of Tecartus (brexucabtagene autoleucel) resulted in an objective response rate of 86% (24/28, 16 complete response, 8 partial response) in patients with relapsed/refractory mantle cell lymphoma (Blood (2019) 134 (Supplement_1): 754). detail...
Unknown unknown 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 B-cell lymphoma not applicable Denileukin diftitox + Sirolimus Preclinical - Cell culture Actionable In a preclinical study, Rapamune (sirolimus) enhanced the efficacy of Ontak (denileukin diftitox) in human B-cell lymphoma cells, resulting in decreased cell viability in culture (PMID: 27737881). 27737881
Unknown unknown lymphoma not applicable Cyclophosphamide + SB 11285 Preclinical Actionable In a preclinical study, Cytoxan (cyclophosphamide) and SB 11285 combination treatment delayed and inhibited tumor growth in a syngeneic mouse model of lymphoma (Journal of Clinical Oncology 2017 35:15_suppl, e14616). detail...
Unknown unknown spindle cell carcinoma not applicable LYC-55716 Case Reports/Case Series Actionable In a Phase I trial, Cintirorgon (LYC-55716) treatment resulted in a partial response in a patient with metastatic spindle cell carcinoma, demonstrating decreased tumor burden (PMID: 30819679). 30819679
Unknown unknown 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 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 renal cell carcinoma not applicable Avelumab + Axitinib FDA approved Actionable In a Phase III trial that supported FDA approval (JAVELIN Renal 101), Inlyta (axitinib) plus Bavencio (avelumab) resulted in a median progression-free survival of 13.8 mo. and an objective response rate of 51.4%, vs. 8.4 mo. and 25.7% with Sutent (sunitinib), respectively in patients with advanced renal cell carcinoma, and at median follow-up 14.3% (63) of patients treated with Inlyta (axitinib) plus Bavencio (avelumab) had died vs. 16.9% (75) with Sutent (sunitinib) (PMID: 30779531; NCT02684006). detail... 30779531
Unknown unknown anal squamous cell carcinoma not applicable Prexasertib Phase Ib/II Actionable In a Phase Ib trial, treatment with Prexasertib (LY2606368) resulted in an overall response rate of 15% (4/26; 1 complete response (CR) and 3 partial responses (PR)), a clinical benefit rate (CR+PR+stable disease) of 58% (15/26), and a median progression-free survival of 2.8 months in patients with squamous cell carcinoma of the anus (PMID: 29643063; NCT0115790). 29643063
Unknown unknown anal squamous cell carcinoma not applicable Prexasertib Phase I Actionable In a Phase I trial, Prexasertib (LY2606368) treatment resulted in partial response in a patient with anal squamous cell carcinoma (PMID: 27044938). 27044938
Unknown unknown endometrial cancer not applicable ETP-46464 + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, ETP-46464 increased the sensitivity of endometrial cancer cell lines to ionizing radiation in culture (PMID: 25560806). 25560806
Unknown unknown prostate cancer not applicable Docetaxel + MEDI5117 Preclinical Actionable In a preclinical study, MEDI5117 in combination with Taxotere (docetaxel) resulted in tumor regression in human prostate cancer xenograft models (PMID: 26744529). 26744529
Unknown unknown 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 osteosarcoma not applicable HTH-01-015 Preclinical - Cell culture Actionable In a preclinical study, HTH-01-015 inhibited MYPT1 phosphorylation, invasive behavior, and proliferation of an osteosarcoma cell line in culture (PMID: 24171924). 24171924
Unknown unknown Advanced Solid Tumor not applicable Bevacizumab + Cediranib Phase I Actionable In a Phase I trial, the combination of Cediranib (AZD-2171) and Avastin (bevacizumab) demonstrated preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 24752867). 24752867
Unknown unknown gastroesophageal junction adenocarcinoma not applicable Envafolimab + Fluorouracil + Leucovorin + Oxaliplatin Phase II Actionable In a Phase II trial, Envafolimab (KN035) in combination with FOLFOX regimen resulted in an objective response rate of 60% (9/15) and a median progression-free survival of 6.8 months in patients with advanced gastric (n=13) or gastroesophageal junction (n=2) cancer (J Clin Oncol 38: 2020 (suppl; abstr e16585); CTR20181124). detail...
Unknown unknown 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 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 neuroblastoma not applicable Doxorubicin + GANT61 Preclinical Actionable In a preclinical study, GANT61 and Adriamycin (doxorubicin) worked synergistically to inhibit growth of neuroblastoma cells in culture (PMID: 22949014). 22949014
Unknown unknown colorectal cancer not applicable MSC2490484A + unspecified PD-L1 antibody Preclinical Actionable In a preclinical study, the combination of MSC2490484A and an unspecified PD-L1 antibody resulted in greater inhibition of tumor growth compared to the unspecified PD-L1 antibody alone in a colorectal cancer cell line mouse model (PMID: 32238472). 32238472
Unknown unknown fallopian tube cancer not applicable Bevacizumab + Carboplatin + Paclitaxel FDA approved Actionable In a Phase III trial (GOG-0218) that supported FDA approval, addition of Avastin (bevacizumab) during and after Paraplatin (carboplatin) plus Taxol (paclitaxel) treatment prolonged progression-free survival (14.1 vs 10.3 months) compared to placebo in patients with previously untreated, stage III/IV epithelial ovarian, primary peritoneal, or fallopian tube cancer (PMID: 22204724; NCT00262847). detail... 22204724
Unknown unknown triple-receptor negative breast cancer no benefit CCT007093 Preclinical Actionable In a preclinical study, CCT007093 did not inhibit growth of triple-receptor negative breast cancer cell lines in culture (PMID: 20576088). 20576088
Unknown unknown adrenal gland pheochromocytoma not applicable 131I-MIBG FDA approved Actionable In a Phase II trial that supported FDA approval, Azedra (iobenguane I 131) treatment resulted in partial response in 23% (15/68) of patients with pheochromocytoma or paraganglioma who received 1 therapeutic dose, with a 12-month overall survival rate of 91% (J Clin Oncol 36, 2018 (suppl; abstr 4005); NCT00874614). detail... detail...
Unknown unknown female reproductive organ cancer not applicable Pamiparib Phase I Actionable In a Phase I trial, Pamiparib (BGB-290) treatment resulted in objective response in 43% (10/23) of patients with gynecological cancer (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 368PD; NCT02361723). detail...
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 multiple myeloma not applicable Dexamethasone + Isatuximab + Pomalidomide FDA approved Actionable In a Phase III (ICARIA-MM) trial that supported FDA approval, addition of Sarclisa (isatuximab-irfc) to Pomalyst (pomalidomide) and dexamethasone significantly improved progression-free survival (11.5 vs 6.5 months, HR=0.596, p=0.001) in patients with relapsed and refractory multiple myeloma (PMID: 31735560; NCT02990338). detail... 31735560
Unknown unknown lymphoma not applicable OKI-005 Preclinical - Cell culture Actionable In a preclinical study, OKI-005 induced cell cycle arrest and apoptosis in lymphoma cells in culture (PMID: 31235619). 31235619
Unknown unknown ovarian carcinoma not applicable BI 853520 Phase I Actionable In a Phase I trial, BI 853520 demonstrated safety and some anti-tumor efficacy, resulting in stable disease in 31% (5/16) of patients with platinum-resistant ovarian carcinoma, one of whom had stable disease lasting greater than 150 days (PMID: 30756308; NCT01335269). 30756308
Unknown unknown 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 triple-receptor negative breast cancer not applicable Irinotecan + SR-4835 Preclinical - Pdx Actionable In a preclinical study, SR-4835 and Camptosar (irinotecan) combination treatment induced DNA damage and cell death, inhibited tumor growth, leading to tumor regression in a patient-derived xenograft (PDX) model of triple-negative breast cancer harboring a BRCA1 mutation (PMID: 31668947). 31668947
Unknown unknown Advanced Solid Tumor not applicable MK2206 Phase I Actionable In a Phase I clinical trial, MK2206 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22025163). 22025163
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 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 transitional cell carcinoma not applicable Domatinostat Preclinical - Cell culture Actionable In a preclinical study, Domatinostat (4SC-202) inhibited proliferation and induced cell-cycle alterations and cell death in urothelial cancer cell lines in culture (PMID: 27250763). 27250763
Unknown unknown Advanced Solid Tumor not applicable DTS-108 Phase I Actionable In a Phase I trial, DTS-108 demonstrated safety and preliminary efficacy, resulting in stable disease in 21% (9/42) of patients with advanced solid tumors (PMID: 27920527). 27920527
Unknown unknown Advanced Solid Tumor not applicable GDC-0917 Phase I Actionable In a Phase I clinical trial, GDC-0917 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol 31, 2013 (suppl; abstr 2503)). detail...
Unknown unknown 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 Advanced Solid Tumor not applicable Utomilumab Phase I Actionable In a Phase I trial, Utomilumab (PF-05082566) treatment resulted in an overall objective response rate of 3.8% (2/53), a median progression-free survival of 1.7 months, and a median overall survival of 11.2 months in patients with advanced solid tumors (PMID: 29549159; NCT01307267). 29549159
Unknown unknown transitional cell carcinoma not applicable Paclitaxel + Pazopanib Phase II Actionable In a Phase II trial, patients with urothelial carcinoma treated with the combination of Taxol (paclitaxel) and Votrient (pazopanib) demonstrated an overall response rate of 54% (15/28), in which 11% (3/28) experienced a complete response and 43% (12/28) experienced stable disease (PMID: 27068017). 27068017
Unknown unknown 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 lung small cell carcinoma not applicable Cisplatin + Etoposide + Roniciclib Phase II Actionable In a Phase II trial, the combination of Roniciclib (BAY1000394) plus chemotherapy regimen, Paraplatin (carboplatin) or Platinol (cisplatin) and Vepesid (etoposide) (n=71), did not meet its primary endpoint for progression-survival in patients with small cell lung cancer (4.9 mo vs 5.5 mo) when compared to placebo plus chemotherapy (n=71), and showed an unfavorable risk-benefit profile, therefore, leading to premature termination of the study (PMID: 30677506; NCT02161419). 30677506
Unknown unknown breast cancer not applicable Metformin Preclinical - Cell line xenograft Actionable In a preclinical study, Glucophage (metformin) inhibited the growth of a breast cancer cell line in culture and resulted in decreased tumor volume in a cell line xenograft model (PMID: 26351208). 26351208
Unknown unknown Advanced Solid Tumor not applicable Birabresib Preclinical - Cell culture Actionable In a preclinical study, Birabresib (OTX015) inhibited growth of a variety of tumor cell lines in cell culture (Mol Cancer Ther November 2013 12; C244). detail...
Unknown unknown Advanced Solid Tumor not applicable Birabresib Phase Ib/II Actionable In a Phase Ib trial, Birabresib (OTX015) demonstrated favorable safety and resulted in partial response in 7% (3/42) and stable disease in 60% (25/42) of patients with non-small cell lung cancer, prostate cancer, or NUT midline carcinoma, with all of the responses occurring in NUT midline carcinoma patients (PMID: 29733771; NCT02259114). 29733771
Unknown unknown multiple myeloma not applicable Carfilzomib + DT204 Preclinical - Cell culture Actionable In a preclinical study, multiple myeloma cells resistant to Kyprolis (carfilzomib) demonstrated re-sensitization to Kyprolis (carfilzomib) in culture when treatment was combined with DT204, showing a synergistic effect and increased apoptotic activity (PMID: 27677741). 27677741
Unknown unknown ovarian cancer not applicable Bevacizumab FDA approved Actionable In a Phase III trial that supported FDA approval, ovarian cancer patients treated with Avastin (bevacizumab) combined with chemotherapy versus chemotherapy alone resulted in a greater PFS (6.7 mo vs 3.4 mo), overall response rate (27.3% vs 11.8%), and OS (16.6 mo vs 13.3 mo) (PMID: 24637997). detail... 24637997
Unknown unknown ovarian cancer not applicable Bevacizumab Phase III Actionable In a meta-analysis of three phase III trials, Avastin (bevacizumab) significantly increased progression free survival and overall survival comparied to standard therapy, with hazard ratios of 0.53 and 0.87, respectively (PMID: 26657509). 26657509
Unknown unknown colorectal cancer not applicable trifluridine/tipiracil hydrochloride FDA approved Actionable In a Phase III trial supporting FDA approval, Lonsurf (trifluridine/tipiracil hydrochloride) demonstrated improved median overall survival (7.1 vs 5.3 months), median progression free survival (2.0 vs 1.7 months), and 32% reduction of death risk compared to placebo in treatment-refractory metastatic colorectal cancer patients (PMID: 27126991). 27126991 detail...
Unknown unknown glioblastoma multiforme not applicable DCVax-L Phase III Actionable In a Phase III trial, addition of DCVax-L to standard therapy resulted in a median overall survival (mOS) of 23.1 months in the intended to treat group (n=331) of patients with newly diagnosed glioblastoma, with a mOS of 34.7 months in patients with methylated MGMT (n?=?131) (PMID: 29843811; NCT00045968). 29843811
Unknown unknown 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 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 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 diffuse large B-cell lymphoma not applicable Lenalidomide + Tafasitamab Phase II Actionable In a Phase II trial (L-MIND), the combination of Revlimid (lenalidomide) and Tafasitamab (MOR208) was well tolerated in patients with previously treated diffuse large B-cell lymphoma and resulted in an objective response rate of 60% (48/80), including 34 complete responses and 14 partial responses, stable disease in 14% (11/80) of patients, and median progression-free survival of 12.1 months (PMID: 32511983; NCT02399085). 32511983
Unknown unknown ovarian cancer not applicable Enoticumab Phase I Actionable In a Phase I study, Enoticumab (REGN421) demonstrated safety and preliminary efficacy in ovarian cancer patients (PMID: 25724527). 25724527
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 ovary epithelial cancer not applicable MGD013 Phase I Actionable In a Phase I trial, MGD013 treatment resulted in a partial response in 13.3% and stable disease in 46.7% (7/15) of patients with epithelial ovarian cancer (J Clin Oncol 38: 2020 (suppl; abstr 3004); NCT03219268). detail...
Unknown unknown clear cell renal cell carcinoma no benefit Sorafenib + Temsirolimus Phase II Actionable In a Phase II clinical trial, treatment with the combination of Nexavar (sorafenib) and Torisel (temsirolimus) did not prolong progression-free survival compared to treatment with Avastin (bevacizumab) monotherapy (7.4 months vs 7.5 months) in patients with renal clear cell carcinoma (PMID: 26077237). 26077237
Unknown unknown osteosarcoma not applicable V158411 Preclinical - Cell culture Actionable In a preclinical study, V158411 inhibited Chk1 autophosphorylation and induced DNA damage and apoptosis in an osteosarcoma cell line in culture (PMID: 27829224). 27829224
Unknown unknown prostate cancer not applicable Enzalutamide FDA approved Actionable Ina Phase III trial (ARCHES) that supported FDA approval, Xtandi (enzalutamide) treatment with androgen deprivation therapy (ADT) significantly reduced the risk of radiographic progression or death compared to ADT plus placebo (HR=0.39, P < .001; median not reached vs 19.0 months) in patients with metastatic hormone-sensitive prostate cancer (PMID: 31329516; NCT02677896). detail... 31329516
Unknown unknown prostate cancer not applicable Enzalutamide FDA approved Actionable In a Phase III trial (AFFIRM) that supported FDA approval, treatment with Xtandi (enzalutamide) resulted in improved median overall survival compared to placebo (18.4 vs 13.6 months HR=0.63, p<0.001) in patients with metastatic castration-resistant prostate cancer (PMID: 22894553; NCT00974311). detail... 22894553
Unknown unknown prostate cancer not applicable Enzalutamide FDA approved Actionable In a Phase III trial (PROSPER) that supported FDA approval, Xtandi (enzalutamide) significantly improved median metastasis-free survival (36.6 vs 14.7 months, HR=0.29, p<0.0001) compared to placebo in patients with non-metastatic castration-resistant prostate cancer (Journal of Clinical Oncology 36, no. 6_suppl (February 20 2018) 3-3; NCT020032924). detail... detail...
Unknown unknown breast cancer not applicable LDC1267 Preclinical Actionable In a preclinical study, LDC1267 treatment resulted in reduced l metastatic liver lesions, but did not impact primary tumor growth, in a mouse model of metastatic breast cancer (PMID: 24553136). 24553136
Unknown unknown 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 small cell carcinoma not applicable MGC018 Case Reports/Case Series Actionable In a Phase I/II trial, MGC018 treatment resulted in a 6.3% decrease of target lesion in a patient with small cell lung caner (J Clin Oncol 38: 2020 (suppl; abstr 3071); NCT03729596). detail...
Unknown unknown lung cancer not applicable BOS172722 Preclinical - Cell culture Actionable In a preclinical study, lung cancer cell lines demonstrated sensitivity to growth inhibition by BOS172722 in culture (PMID: 31575759). 31575759
Unknown unknown renal cell carcinoma not applicable Axitinib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Inlyta (axitinib) as second-line therapy resulted in an improved progression-free survival of 8.3 months compared to 5.7 months with Nexavar (sorafenib) (HR 0.656, 95% CI 0.552-0.779; p<0.0001) in patients with metastatic renal cell carcinoma (PMID: 23598172). 23598172 detail...
Unknown unknown renal cell carcinoma not applicable Axitinib Phase II Actionable In a Phase II clinical trial, treatment with Inlyta (axtinib) as first-line therapy resulted a prolonged median overall survival of 42.7 months compared to 30.4 months with placebo in patients with metastatic renal cell carcinoma (PMID: 27236772). 27236772
Unknown unknown renal cell carcinoma not applicable Axitinib Clinical Study Actionable In a meta-analysis, Inlyta (axitinib) improved progression-free survival in patients with metastatic renal cell carcinoma (PMID: 24037486). 24037486
Unknown unknown peripheral T-cell lymphoma not applicable Fenretinide Phase I Actionable In a Phase I trial, Fenretinide treatment resulted in complete response in 17% (2/11), partial response in 17% (2/11), and stable disease in 42% (5/11) of patients with peripheral T-cell lymphoma (PMID: 28420721). 28420721
Unknown unknown acute myeloid leukemia not applicable CPI-0610 + Doxorubicin Preclinical - Cell line xenograft Actionable In a preclinical study, the combination treatment of CPI-0610 and Adriamycin (doxorubicin) resulted in inhibition of tumor growth in acute myeloid leukemia xenograft models (PMID: 26815195). 26815195
Unknown unknown colorectal cancer not applicable Bevacizumab + trifluridine/tipiracil hydrochloride Phase II Actionable In a Phase II trial, Avastin (bevacizumab) and Lonsurf (trifluridine/tipiracil hydrochloride) combination treatment resulted in improved progression-free survival (4.6 vs 2.6 months, HR=0.45, p=0.0015) compared to Lonsurf (trifluridine/tipiracil hydrochloride) monotherapy in patients with metastatic colorectal cancer refractory to standard therapies (PMID: 31999946). 31999946
Unknown unknown colorectal cancer not applicable Bevacizumab + trifluridine/tipiracil hydrochloride Phase Ib/II Actionable In a Phase I/II trial, Avastin (bevacizumab) and Lonsurf (trifluridine/tipiracil hydrochloride) combination treatment resulted in a 16-week progression-free survival rate of 42.9% (9/21) in patients with metastatic colorectal cancer refractory to standard therapies (PMID: 28760399). 28760399
Unknown unknown non-Hodgkin lymphoma not applicable Camidanlumab Tesirine Phase I Actionable In a Phase I trial, Camidanlumab Tesirine treatment resulted in partial response in 15.8% (3/19) in non-Hodgkin lymphoma patients (Blood 2017 130(Suppl 1):1510; NCT02432235). detail...
Unknown unknown Indication other than cancer not applicable PP30 Preclinical Actionable In a preclinical study, PP30 inhibited proliferation of mouse embryonic fibroblasts more effectively than rapamycin (PMID: 19209957). 19209957
Unknown unknown germ cell cancer not applicable Pazopanib Phase II Actionable In a Phase II trial, Votrient (pazopanib) treatment resulted in partial responses in 4.7% (2/43), stable disease in 44.2% (19/43), and a 3-month progression free survival probability of 12.8% in patients with refractory germ cell cancer (PMID: 28383677). 28383677
Unknown unknown 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 gastrointestinal stromal tumor not applicable JSH-150 Preclinical - Cell culture Actionable In a preclinical study, JSH-150 inhibited proliferation of a gastrointestinal stromal tumor cell line in culture (PMID: 30253346). 30253346
Unknown unknown neuroblastoma not applicable Alisertib + Irinotecan + Temozolomide Phase I Actionable In a Phase I trial, the combination of Alisertib (MLN8237), Camptosar (irinotecan), and Temodar (temozolomide) demonstrated safety and preliminary efficacy in neuroblastoma patients, resulting in an overall response rate of 31.8% and a 2-year progression-free survival rate of 52.4% (PMID: 26884555). 26884555
Unknown unknown neuroblastoma not applicable Alisertib + Irinotecan + Temozolomide Phase II Actionable In a Phase II trial, the combination of Alisertib (MLN8237), Camptosar (irinotecan), and Temodar (temozolomide) resulted in a response rate of 21.1% (4/19) in patients with neuroblastoma, with 4 partial responses, 2 minor responses, and 5 stable diseases (PMID: 30093449; NCT01601535). 30093449
Unknown unknown 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 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 Mivavotinib Phase I Actionable In a Phase I trial, Mivavotinib (TAK-659) treatment resulted in an objective response rate of 40% (27/67, 12 complete responses, 15 partial responses) in patients with relapsed or refractory diffuse large B-cell lymphoma, with a median duration of response of 856 days (PMID: 32327472; NCT02000934). 32327472
Unknown unknown gastroesophageal junction adenocarcinoma not applicable Nintedanib Phase II Actionable In a Phase II trial, Ofev (nintedanib) was well tolerated and the study met its primary endpoint, resulted in progression-free survival at 6-months in 19% (6/32) of patients with esophageal/GEJ (n=17) or gastric (n=15) adenocarcinoma, with a median follow-up of 14.5 months and a median overall survival of 14.2 months (PMID: 30952642; NCT02234596). 30952642
Unknown unknown breast cancer not applicable Thymoquinone Preclinical - Cell line xenograft Actionable In a preclinical study, Thymoquinone (TQ) inhibited growth, migration, and invasive behavior of human breast cancer cell lines in culture, and inhibited tumor growth and metastasis in mouse breast cancer cell line xenografts (PMID: 26023736). 26023736
Unknown unknown pancreatic carcinoma not applicable ABT-348 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with Ilorasertib (ABT-348) inhibited tumor growth and led to regression of advanced tumors in cell line xenograft models of pancreatic carcinoma (PMID: 22935731). 22935731
Unknown unknown leukemia not applicable DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, DCBCI0901 inhibited tumor growth greater than 65% in a leukemia cell line xenograft model (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown kidney cancer no benefit CVX-241 Preclinical Actionable In a preclinical study, CVX-241 did not improve overall survival in a mouse model of resected renal cancer (PMID: 27651308). 27651308
Unknown unknown prostate cancer not applicable Enzalutamide + ZEN-3694 Preclinical - Cell culture Actionable In a preclinical study, ZEN-3694 in combination with Xtandi (enzalutamide) synergistically inhibited proliferation of an AR-positive prostate cancer cell line in culture (Cancer Res 2016;76(14 Suppl):Abstract nr LB-207). detail...
Unknown unknown prostate cancer not applicable Enzalutamide + ZEN-3694 Phase Ib/II Actionable In a Phase Ib/II trial, combination of ZEN-3694 and Xtandi (enzalutamide) demonstrated safety in metastatic castration-resistant prostate cancer patients resistant to prior androgen signaling inhibitors, and resulted in a median radiographic progression-free survival (rPFS) of 9.0 months and a composite median radiographic or clinical PFS of 5.5 months, patients with lower baseline AR activity demonstrated prolonged median rPFS (10.4 v 4.3 months) compared to patients with high AR activity (PMID: 32694156). 32694156
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 endometrial cancer not applicable Adavosertib + Paclitaxel Preclinical Actionable In a preclinicals study, Adavosertib (MK-1775) in combination with Paclitaxel, demonstrated efficacy in endometrial cancer cells (PMID: 24381593). 24381593
Unknown unknown renal cell carcinoma not applicable ODM-203 Preclinical Actionable In a preclinical study, ODM-203 inhibited tumor growth and lung metastasis, and increased tumor microenvironment immune response in a VEGFR-dependent mouse renal carcinoma model (PMID: 30301864). 30301864
Unknown unknown pancreatic adenocarcinoma not applicable Gemcitabine + PRI-724 Phase Ib/II Actionable In a Phase Ib trial, the combination of PRI-724 and Gemzar (gemcitabine) demonstrated safety and preliminary efficacy in patients with advanced pancreatic adenocarcinoma, resulted in stable disease in 40% (8/20) of the patients, with a median progression-free survival of 2 months, and a median decline of serum S100P level by 49.95% (Journal of Clinical Oncology 34, no. 15_suppl; NCT01764477). detail... 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 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 lung small cell carcinoma not applicable Mogamulizumab + Nivolumab Phase I Actionable In a Phase I trial, Poteligeo (mogamulizumab-kpkc) and Opdivo (nivolumab) combination treatment demonstrated acceptable safety, resulted in an objective response rate of 7% (1/15, 1 partial responses) and a disease control rate of 20% (3/15) in immunotherapy-naive patients with advanced or metastatic small cell lung cancer, response occurred regardless of PD-L1, CCR4, CD8 expression levels and tumor mutational burden (PMID: 31455681; NCT02476123). 31455681
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 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 lung squamous cell carcinoma not applicable Cisplatin + Necitumumab + Pemetrexed Disodium Phase III Actionable In a Phase III trial, the addition of Necitumumab (IMC-11F8) to pemetrexed and cisplatin did not increase survival of stage IV non-squamous NSCLC patients (PMID: 25701171). 25701171
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 Hodgkin's lymphoma not applicable Nivolumab FDA approved Actionable In a Phase I trial (CheckMate 039) that supported FDA approval, treatment with Opdivo (nivolumab) resulted in an objective response rate of 87% (20/23), with complete response in 17% (4/23) and partial response in 70% (16/23) of patients with relapsed or refractory classical Hodgkin's lymphoma (PMID: 25482239; NCT01592370). 25482239 detail...
Unknown unknown acute myeloid leukemia not applicable FF-10501-01 Phase I Actionable In a Phase I trial, FF-10501-01 demonstrated safety and preliminary efficacy, resulted in stable disease control with no disease progression over 3-24 cycles of treatment in 34.8% (8/23) of acute myeloid leukemia patients, including 2 patients achieved partial response (Blood 128 (22):1640). detail...
Unknown unknown triple-receptor negative breast cancer not applicable BAY1217389 + Paclitaxel Preclinical Actionable In a preclinical study, BAY1217389, in combination with Taxol (paclitaxel), had increased efficacy in inhibiting tumor growth in xenograft models of triple-negative breast cancer, compared to Taxol (paclitaxel) alone (PMID: 26832791). detail... 26832791
Unknown unknown lung non-small cell carcinoma not applicable DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, non-small cell lung carcinoma cells treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture and inhibition of tumor growth in cell-line xenograft models (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown Advanced Solid Tumor not applicable AVID100 Phase I Actionable In a Phase I trial, AVID100 treatment resulted in prolonged stable disease in 12.5% (3/24) of patients with advanced solid tumors not selected for Egfr overexpression (AACR Annual Meeting 2019, Abstract CT056). detail...
Unknown unknown lung small cell carcinoma not applicable Ganetespib Preclinical - Cell line xenograft Actionable In a preclinical study, small cell lung carcinoma cell line xenograft models treated with Ganetespib demonstrated partial tumor growth inhibition, but with tumor progression, weight loss ensued (PMID: 27267850). 27267850
Unknown unknown breast cancer not applicable Lucitanib Phase II Actionable In a Phase II trial, Lucitanib (E-3810) demonstrated acceptable toxicity and activity, resulted in a median progression-free survival of 3.5 and 2.6 months for 10mg and 15mg treatment groups respectively, with no differences in response correlated with FGFR1 and 11q amplification status in metastatic breast cancer patients (Cancer Res 2017;77(4 Suppl):Abstract nr P6-11-03). detail...
Unknown unknown 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 brain stem glioma not applicable MRK-003 + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, MRK-003 enhanced sensitivity of diffuse pontine glioma cell lines to radiotherapy in culture, resulting in increased apoptosis (PMID: 26115193). 26115193
Unknown unknown acute myeloid leukemia not applicable Erlotinib + Ethacridine Preclinical - Cell line xenograft Actionable In a preclinical study, Tarceva (erlotinib) and the PARG inhibitor ethacridine demonstrated synergy in decreasing viability of acute myeloid leukemia (AML) cell lines and primary samples in culture, and reduced tumor growth in AML cell line xenograft models (PMID: 27587383). 27587383
Unknown unknown ovarian cancer not applicable Cediranib Phase III Actionable In a Phase III trial, Cediranib (AZD-2171) given with chemotherapy and as maintenance therapy resulted in improved median overall survival (27.3 vs 19.9 months) in platinum-sensitive ovarian cancer patients (J Clin Oncol 35, 2017 (suppl; abstr 5506)). detail...
Unknown unknown glioblastoma multiforme not applicable Temozolomide + Vistusertib Phase I Actionable In a Phase I trial, combination of Vistusertib (AZD2014) and Temodar (temozolomide) demonstrated safety in patients with previously treated glioblastoma multiforme, resulted in a partial response in 8% (1/13) and stable disease in 38% (5/13) of the patients, with a 6-month progression-free survival rate of 26% (PMID: 31707687). 31707687
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 hepatocellular carcinoma not applicable SF1126 + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of SF1126 and Nexavar (sorafenib) was synergistic or additive in inhibiting proliferation of hepatocellular carcinoma cell lines in culture, and demonstrated increased efficacy in hepatocellular carcinoma cell line xenograft models compared to SF1126 alone (PMID: 23355037). 23355037
Unknown unknown kidney cancer not applicable OPB-111077 Phase I Actionable In a Phase I trial, a patient with kidney cancer demonstrated stable disease when treated with OPB-111077 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B118). detail...
Unknown unknown cutaneous T cell lymphoma not applicable Lacutamab Phase I Actionable In a Phase I trial, Lacutamab (IPH4102) demonstrated safety, and cutaneous T-cell lymphoma patients treated with IPH4102 demonstrated an overall response rate of 36% (16/44), a median progression-free survival of 8.2 months, and a median duration of response of 13.8 months (PMID: 31253572; NCT02593045). 31253572
Unknown unknown lymphoma not applicable Toripalimab Phase I Actionable In a Phase I trial, Toripalimab (JS001) treatment in patients with advanced lymphomas resulted in an objective response rate of 90.9% (10/11) and median progression-free survival of 8.3 months, with all nine Hodgkin's lymphoma patients demonstrating objective responses including six complete responses and four partial responses, while one diffuse large B-cell lymphoma patient achieved a partial response and the other resulted in disease progression (PMID: 32224416; NCT02836834). 32224416
Unknown unknown pancreatic ductal adenocarcinoma not applicable Dinaciclib Preclinical - Cell culture Actionable In a preclinical study, Dinaciclib (SCH 727965) treatment inhibited proliferation and induced apoptosis in mouse pancreatic ductal adenocarcinoma cell lines in culture, and delayed tumor growth, and increased overall survival in a transgenic mouse model (PMID: 32269732). 32269732
Unknown unknown 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 non-Hodgkin lymphoma not applicable Mivavotinib Case Reports/Case Series Actionable In a Phase I trial, Mivavotinib (TAK-659) treatment resulted in an objective response rate of 73% (11/15, 4 complete responses, 7 partial responses) in patients with relapsed or refractory indolent non-Hodgkin lymphoma, with a median duration of response of 176 days (PMID: 32327472; NCT02000934). 32327472
Unknown unknown renal carcinoma not applicable Cabozantinib Phase II Actionable In a Phase II trial, renal cancer patients treated with Cabozantinib demonstrated a 28 % objective response rate, a 62 % stable disease rate, and a median progression free survival of 14.7 months (PMID: 23292795). 23292795
Unknown unknown Advanced Solid Tumor not applicable BIND-014 Phase I Actionable In a Phase I clinical trial, BIND-014 treatment resulted in partial response in 10% (5/52) of patients with advanced solid tumors, and complete response in a cervical cancer patient (PMID: 26847057). 26847057
Unknown unknown colon cancer not applicable Pracinostat Preclinical - Cell line xenograft Actionable In a preclinical study, Pracinostat (SB939) inhibited proliferation of colon cancer cell lines in culture, and increased histone H3 acetylation and inhibited tumor growth in a colon cancer cell line xenograft model (PMID: 20197387). 20197387
Unknown unknown transitional cell carcinoma not applicable Lenvatinib + Pembrolizumab Phase Ib/II Actionable Ina Phase Ib/II trial, Lenvima (lenvatinib) and Keytruda (pembrolizumab) combination treatment demonstrated safety and efficacy, resulted in an objective response rate of 25% (5/20) in patients with metastatic urothelial cancer, with a median duration of response not evaluable, and a median progression-free survival of 5.4 months (PMID: 31961766; NCT02501096). 31961766
Unknown unknown ovarian carcinoma not applicable BMS-906024 Preclinical Actionable In a preclinical study, the pan-Notch inhibitor, BMS-906024, inhibited growth of ovarian carcinoma xenografts (PMID: 26005526). 26005526
Unknown unknown basal cell carcinoma not applicable Vismodegib FDA approved Actionable In a Phase II trial that supported FDA approval, treatment with Erivedge (vismodegib) resulted in an objective response rate of 30% (10/33) in patients with metastatic basal cell carcinoma and 43% (27/63) in patients with locally advanced basal cell carcinoma, with 21% (13/63) patients with locally advanced basal cell carcinoma demonstrating complete response (PMID: 22679179, PMID: 22670903). 22679179 22670903 detail...
Unknown unknown Advanced Solid Tumor not applicable OSI-027 Phase I Actionable In a Phase I trial, OSI-027 treatment resulted in no RECIST response and stable disease in 5% (6/128) of patients with advanced solid tumors (PMID: 27002938). 27002938
Unknown unknown gastroesophageal cancer not applicable Ganetespib Phase II Actionable In a Phase II trial, Ganetespib demonstrated manageable toxicity but did not demonstrate single-agent activity, resulted in an objective response rate of 4% (1/26, 1 complete response), a median progression-free survival of 61 days, and a median overall survival of 94 days in patients with advanced esophagogastric cancer (PMID: 31898183). 31898183
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 ovarian 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 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 marginal zone B-cell lymphoma not applicable Ibrutinib FDA approved Actionable In a Phase II trial that supported FDA approval, Imbruvica (ibrutinib) treatment resulted in an objective response rate of 48% (29/60) in patients with previously treated marginal zone lymphoma, with a median progression-free survival of 14.2 months (PMID: 28167659; NCT01980628). 28167659 detail...
Unknown unknown pancreatic cancer not applicable PH11 Preclinical Actionable In a preclinical study, treatment with PH11 led to increased sensitivity to TRAIL in pancreatic cancer cells thereby restoring apoptosis (PMID: 25684663). 25684663
Unknown unknown mantle cell lymphoma not applicable CC214-1 Preclinical - Patient cell culture Actionable In preclinical study, mantle cell lymphoma patient derived cell lines demonstrated improved survival in culture when treated with CC214-1 (PMID: 25839159). 25839159
Unknown unknown triple-receptor negative breast cancer not applicable Navitoclax + Paclitaxel Preclinical - Cell culture Actionable In a preclinical study, the combination of Navitoclax (ABT-263) and Taxol (paclitaxel) resulted in a synergistic effect and inhibited the growth of triple-receptor negative breast cancer cells in culture (PMID: 27872098). 27872098
Unknown unknown B-cell lymphoma not applicable Alisertib Phase II Actionable In a Phase II trial, Alisertib (MLN8237) treatment resulted in significant toxicity and an objective response rate of 15% (2/13) in patients with B-cell non Hodgkin's lymphoma (PMID: 28278718). 28278718
Unknown unknown ovarian cancer not applicable Cisplatin + VE-821 Preclinical - Cell culture Actionable In a preclinical study, VE-821 increased the sensitivity of an ovarian cancer cell line to Platinol (cisplatin) in culture (PMID: 25560806). 25560806
Unknown unknown 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 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 triple-receptor negative breast cancer not applicable ASTX-660 + Paclitaxel Preclinical - Cell line xenograft Actionable In a preclinical study, combination of ASTX-660 and Taxol (paclitaxel) treatment resulted in tumro regression and achieved partial response in cell line xenograft models of triple-receptor negative breast cancer (Cancer Res 2016;76(14 Suppl):Abstract nr 1287). detail...
Unknown unknown astrocytoma not applicable Dactolisib + Everolimus Phase Ib/II Actionable In a Phase Ib trial, the combination of BEZ235 and Afinitor (everolimus) resulted in stable disease in a patient with astrocytoma (PMID: 28357727). 28357727
Unknown unknown colon carcinoma not applicable CFI-402257 + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, treatment with the combination of CFI-402257 and an anti-PD-1 antibody induced tumor regression in 2/8 tumors in syngeneic mouse models of colon carcinoma, and no regressions were demonstrated in mice treated with single agent CFI-402257 or anti-PD-1 antibody (PMID: 28270606). 28270606
Unknown unknown lymphoma not applicable Bendamustine + Veliparib Phase I Actionable In a Phase I trial, seven patients with lymphoma treated with a combination of Veliparib (ABT-888) and Bendamustine demonstrated an overall response rate of 71% (5/7) and complete response rate of 57% (4/7) and progression free survival of 6.9 months (PMID: 28314788; NCT01326702). 28314788
Unknown unknown lung non-small cell carcinoma not applicable Cisplatin + PJ34 Preclinical - Cell culture Actionable In a preclinical study, the combination of Platinol (cisplatin) and PJ34 worked synergistically to induce cell death in non-small cell lung carcinoma cells in culture (PMID: 23428903). 23428903
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