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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 Advanced Solid Tumor not applicable Akt Inhibitor (Pan) M2698 Phase I Actionable In a Phase I trial, M2698 (MSC2363318A) demonstrated safety and preliminary efficacy in a variety of advanced solid tumor patients (Ann. Oncol. 26 (Suppl 2): ii25-ii27, 2015). detail...
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) M2698 Preclinical Actionable In a preclinical study, MSC2363318A demonstrated sustained inhibition of S6K phosphorylation, and inhibited tumor growth in several human cancer xenograft models of breast, pancreatic, glioblastoma and ovarian cancers (Mol Cancer Ther 2013;12(11 Suppl):A162). detail...
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) M2698 Phase I Actionable In a Phase I trial, M2698 (MSC2363318A) demonstrated safety and preliminary efficacy, with 19% (6/32) of advanced solid tumor patients remained on treatment for more than 180 days (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 370PD; NCT01971515). detail...
Unknown unknown glioblastoma multiforme not applicable Akt Inhibitor (Pan) LYS6KAKT1 Preclinical Actionable In a preclinical study, LYS6KAKT1 inhibited phosphorylation of p70S6 kinase in mice engrafted with glioblastoma cells (Mol Cancer Ther 2011;10(11 Suppl):Abstract nr B117). detail...
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) Gemcitabine + LY2780301 Phase Ib/II Actionable In a Phase Ib trial, the combination of LY2780303 and Gemzar (gemcitabine) demonstrated some antitumor activity in patients with advanced solid tumors including 2 patients with a partial response, 28 patients with stable disease, and a four month disease control rate of 22% (PMID: 28750271). 28750271
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) 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 triple-receptor negative breast cancer not applicable Akt Inhibitor (Pan) 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 immune system cancer not applicable Akt Inhibitor (Pan) Afuresertib Phase II Actionable In a Phase II trial, treatment with Afuresertib (GSK2110183) in patients with Langerhans cell histiocytosis resulted in an overall response rate of 33% in treatment-naive patients and 28% in patients with refractory disease (PMID: 27804235). 27804235
Unknown unknown ovary epithelial cancer not applicable Akt Inhibitor (Pan) Afuresertib + Carboplatin + Paclitaxel Phase I Actionable In a Phase Ib trial, the combination of Afuresertib (GSK2110183) , Paraplatin (carboplatin), and Taxol (paclitaxel) was well-tolerated and demonstrated preliminary efficacy in patients with platinum-resistant epithelial ovarian cancer, resulting in an overall response rate in the dose-expansion part of the trial (Part II) of 32% (9/28) and a median progression-free survival of 7.1 months (PMID: 30563934; NCT01653912). 30563934
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) Afuresertib + Trametinib Phase I Actionable In a Phase I trial, the combination of Mekinist (trametinib) and Afuresertib (GSK2110183) was not well-tolerated in patients with advanced solid tumors (PMID: 25417902). 25417902
Unknown unknown triple-receptor negative breast cancer not applicable Akt Inhibitor (Pan) Docetaxel + ONC201 Preclinical - Cell culture Actionable In a preclinical study, the combination of ONC201 and Docefrez (docetaxel) worked synergistically to inhibit viability of triple-negative breast cancer cell lines in culture (PMID: 28424227). 28424227
Unknown unknown mantle cell lymphoma not applicable Akt Inhibitor (Pan) 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 multiple myeloma not applicable Akt Inhibitor (Pan) ONC201 Preclinical - Cell culture Actionable In a preclinical study, a Velcade (bortezomib)-resistant multiple myeloma cell line demonstrated sensitivity to ONC201 in culture (PMID: 26884599). 26884599
Unknown unknown endometrial clear cell adenocarcinoma not applicable Akt Inhibitor (Pan) 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 breast cancer not applicable Akt Inhibitor (Pan) ONC201 Preclinical - Cell line xenograft Actionable In a preclinical study, ONC201 inhibited viability of several breast cancer cell lines in culture (including triple-negative breast cancer (TNBC) and non-TNBC cell lines), with some cell lines demonstrating increased apoptosis, and inhibited tumor growth in TNBC cell line xenograft models (PMID: 28424227). 28424227
Unknown unknown acute myeloid leukemia not applicable Akt Inhibitor (Pan) ONC201 Preclinical - Pdx & cell culture Actionable In a preclinical study, ONC201 treatment induced ATF4 expression and apoptosis in acute myeloid leukemia (AML) cell lines in culture, independent of Tp53 status, and was toxic to AML primary samples in culture, resulting in decreased engraftment in xenograft models (PMID: 26884599). 26884599
Unknown unknown triple-receptor negative breast cancer not applicable Akt Inhibitor (Pan) ONC201 Preclinical - Cell line xenograft Actionable In a preclinical study, ONC201 inhibited viability of several triple-negative breast cancer (TNBC) cell lines in culture, demonstrating variable pro-apototic and anti-proliferative activity, and inhibited tumor growth in TNBC cell line xenograft models (PMID: 28424227). 28424227
Unknown unknown prostate cancer not applicable Akt Inhibitor (Pan) 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 glioblastoma multiforme not applicable Akt Inhibitor (Pan) ONC201 Phase II Actionable In a Phase II trial, ONC201 treatment was well-tolerated, however, did not achieve the 6 month progression-free survival (PFS) at 5% in glioblastoma multiforme patients (n=20) with a median PFS of 1.8 months and a median overall survival of 7.5 months, and led to a complete tumor regression in a glioblastoma multiforme patient harboring H3 K28M (also referred to as K27M) with a durable response of over 1.5 years (PMID: 31702782; NCT02525692). 31702782
Unknown unknown prostate adenocarcinoma not applicable Akt Inhibitor (Pan) 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 mantle cell lymphoma not applicable Akt Inhibitor (Pan) ONC201 Preclinical - Patient cell culture Actionable In a preclinical study, ONC201 induced apoptosis and decreased viability of mantle cell lymphoma (MCL) cell lines in culture and demonstrated cytotoxicity in primary MCL samples in culture (PMID: 26884599). 26884599 detail...
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) ONC201 Phase I Actionable In a Phase I trial, ONC201 treatment resulted in stable disease in 80% (8/10) of patients with advanced solid tumors (J Clin Oncol 34, 2016 (suppl; abstr 2514)). detail...
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) ONC201 Preclinical - Cell line xenograft Actionable In a preclinical study, ONC-201 (TIC-10) induced apoptosis and tumor regression in a variety of cell line xenograft models, including colon, breast, and brain cancer (PMID: 23390247). 23390247
Unknown unknown endometrial cancer not applicable Akt Inhibitor (Pan) ONC201 Clinical Study Actionable In a clinical case study, a patient with endometrial cancer demonstrated stable disease for 42 weeks and continued regression of metastatic lesions in the lung when treated with ONC201 (TIC-10) (PMID: 28331050). 28331050
Unknown unknown triple-receptor negative breast cancer not applicable Akt Inhibitor (Pan) ONC201 + Paclitaxel Preclinical - Cell culture Actionable In a preclinical study, the combination of ONC201 and Taxol (paclitaxel) worked synergistically to inhibit viability of triple-negative breast cancer cell lines in culture (PMID: 28424227). 28424227
Unknown unknown acute myeloid leukemia not applicable Akt Inhibitor (Pan) ONC201 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of ONC201 and Venclexta (venetoclax) demonstrated synergy in acute myeloid leukemia cell lines in culture, resulting in increased induction of apoptosis (PMID: 26884599). 26884599
Unknown unknown mantle cell lymphoma not applicable Akt Inhibitor (Pan) ONC201 + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, the combination of ONC201and Venclexta (venetoclax) demonstrated synergy in a mantle cell lymphoma cell line in culture, resulting in increased induction of apoptosis (PMID: 26884599). 26884599
Unknown unknown ovarian cancer not applicable Akt Inhibitor (Pan) ABT-737 + MK2206 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of MK2206 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 chronic lymphocytic leukemia not applicable Akt Inhibitor (Pan) Bendamustine + MK2206 + Rituximab Phase Ib/II Actionable In a Phase I trial, MK2206 in combination with Bendamustine and Rituximab resulted in an overall response rate of 92% (12/13), with a median progression free survival of 16 months and a treatment free survival of 24 months in patients with relapsed or refractory chronic lymphocytic leukemia (PMID: 28402581). 28402581
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) Lapatinib + MK2206 Phase I Actionable In a Phase I trial, Tykerb (lapatinib) and MK2206 combination treatment resulted in stable disease for more than 4 months in 9% (2/23) of patients with advanced solid tumors (PMID: 27026198). 27026198
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) 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 gastric adenocarcinoma not applicable Akt Inhibitor (Pan) MK2206 Phase II Actionable In a Phase II trial, MK2206 demonstrated safety, but did not meet study endpoint of median overall survival of 6.5 months, however gastric adenocarcinoma patients were not stratified according to somatic profiles (PMID: 25827820). 25827820
Unknown unknown renal cell carcinoma no benefit Akt Inhibitor (Pan) 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 Advanced Solid Tumor not applicable Akt Inhibitor (Pan) MK2206 + Paclitaxel Phase Ib/II Actionable In a Phase Ib clinical trial, the combination of MK2206 and Taxol (paclitaxel) was well tolerated and demonstrated preliminary efficacy in patients with advanced solid tumors, with partial response in 24% (5/21) and stable disease in 43% (9/21) of patients (PMID: 25688104) 25688104
Unknown unknown pancreatic adenocarcinoma no benefit Akt Inhibitor (Pan) MK2206 + Selumetinib Phase II Actionable In a Phase II trial, the combination of Selumetinib (AZD6244) and MK2206 did not result in improved median overall survival compared to mFOLFOX therapy (3.9 mo vs. 6.7 mo) or improved median progression-free survival (1.9 mo vs 2.0 mo) in patients with pancreatic adenocarcinoma (PMID: 27978579). 27978579
Unknown unknown ovarian cancer not applicable Akt Inhibitor (Pan) Cisplatin + Uprosertib Preclinical - Cell line xenograft Actionable In a preclinical study, the addition of GSK2141795 re-sensitized platinum-resistant ovarian cancer cell lines to Platinol (cisplatin) in culture, and treatment with the combination of GSK2141795 and Platinol (cisplatin) resulted in increased tumor growth inhibition in ovarian cancer xenograft models compared to Platinol (cisplatin) alone (PMID: 26497682). 26497682
Unknown unknown endometrial cancer no benefit Akt Inhibitor (Pan) Trametinib + Uprosertib Phase I Actionable In a Phase I trial, Mekinist (trametinib) and Uprosertib (GSK2141795) combination treatment demonstrated increased toxicity and limited efficacy, resulted in no response (0/14) at RP2D dose and 1 response (8.3%, 1/12) at reduced dose in patients with recurrent endometrial cancer, with progression-free survival at 6 months in 14% and 25% of the patients, respectively (PMID: 31623857). 31623857
Unknown unknown Advanced Solid Tumor no benefit Akt Inhibitor (Pan) Trametinib + Uprosertib Phase I Actionable In a Phase I trial, the combination of Trametinib (GSK1120212) and Uprosertib (GSK2141795) was not well-tolerated and resulted in minimal efficacy in patients with advanced solid tumors (n=126), demonstrating an objective response rate of less than 5%, with one complete response and five partial responses (PMID: 32062691). 32062691
Unknown unknown breast cancer not applicable Akt Inhibitor (Pan) TAS0612 Preclinical - Cell line xenograft Actionable In a preclinical study, breast cancer cells harboring mutations in PIK3CA (H1047R, H1047L, or E545K), HRAS, and PTEN, and antiestrogen-resistant cells demonstrated sensitivity to TAS0612 treatment in culture, and orthotopic cell line xenograft models, including a triple-negative breast cancer, demonstrated inhibition of tumor growth (PMID: 31879363). 31879363
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) ARQ 751 Preclinical - Cell culture Actionable In a preclinical study, treatment with ARQ 751 resulted in anti-proliferative activity in a variety of cancer cell lines in culture (PMID: 26469692). 26469692
Unknown unknown diffuse large B-cell lymphoma not applicable Akt Inhibitor (Pan) AT-7867 Preclinical - Cell culture Actionable In a preclinical study, AT-7867 inhibited the growth of diffuse large B-cell lymphoma cell lines in culture (PMID: 26824321). 26824321
Unknown unknown acute myeloid leukemia not applicable Akt Inhibitor (Pan) AZD1897 + Capivasertib Preclinical Actionable In a preclinical study, acute myeloid leukemia cells treated with the combination of AZD1897 and AZD5363 produced a synergistic effect, resulting in decreased cell survival (PMID: 24975213). 24975213
Unknown unknown prostate cancer not applicable Akt Inhibitor (Pan) Capivasertib + Docetaxel + Prednisone Phase I Actionable In a Phase I trial, AZZD5363 in combination with Taxotere (docetaxel) and Prednisone resulted in more than 50% PSA reduction at 12 weeks in 70% (7/10) of patients with metastatic castration resistant prostate cancer (PMID: 28144789; NCT02121639). 28144789
Unknown unknown prostate cancer not applicable Akt Inhibitor (Pan) 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 PTEN loss or AKT1 E17K (PMID: 32205016; NCT02525068). 32205016
Unknown unknown triple-receptor negative breast cancer not applicable Akt Inhibitor (Pan) 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 endometrial cancer not applicable Akt Inhibitor (Pan) 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 ovarian cancer not applicable Akt Inhibitor (Pan) 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 Advanced Solid Tumor not applicable Akt Inhibitor (Pan) Capivasertib + Olaparib Phase I Actionable In a Phase I trial, Capivasertib (AZD5363) and Lynparza (olaparib) combination therapy was well-tolerated, and resulted in a clinical benefit rate of 44.6% (25/56, 14 partial responses, 11 stable disease >= 4 months) in patients with advanced solid tumors (PMID: 32532747; NCT02338622). 32532747
Unknown unknown triple-receptor negative breast cancer not applicable Akt Inhibitor (Pan) Capivasertib + Paclitaxel Phase II Actionable In a Phase II trial (PAKT), addition of Capivasertib (AZD5363) to Taxol (paclitaxel) as first-line therapy resulted in improved median progression-free survival (5.9 vs 4.2 months, HR=0.74, p=0.06) and median overall survival (19.1 vs 12.6 months, HR=0.61, p=0.04) compared to Taxol (paclitaxel) alone in patients with metastatic triple-negative breast cancer (PMID: 31841354; NCT02423603). 31841354
Unknown unknown Her2-receptor negative breast cancer not applicable Akt Inhibitor (Pan) 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 Advanced Solid Tumor not applicable Akt Inhibitor (Pan) 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 Advanced Solid Tumor not applicable Akt Inhibitor (Pan) Docetaxel + Ipatasertib Phase I Actionable In a Phase Ib trial, the combination of Ipatasertib (GDC-0068) and Taxotere (docetaxel) demonstrated safety in patients with advanced solid tumors, and resulted in an overall response rate of 7.7% (2/26), including partial responses in two patients, stable disease in 53.8% (14/26) of patients, a six-month progression-free survival rate of 11.5% (3/26), and maximum progression-free survival duration of 10 months in a lung cancer patient (PMID: 32205017; NCT01362374). 32205017
Unknown unknown prostate cancer not applicable Akt Inhibitor (Pan) Enzalutamide + Ipatasertib Phase I Actionable In a Phase Ib trial, the combination of Ipatasertib (GDC-0068) and Xtandi (enzalutamide) demonstrated safety in prostate cancer patients, and resulted in an overall response rate of 11.8% (2/17), including partial responses in two patients, stable disease in 23.5% (4/17) of patients, a six-month progression-free survival rate of 23.5% (4/17), and maximum progression-free survival duration of 19.5 months (PMID: 32205017; NCT01362374). 32205017
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) Fluorouracil + Ipatasertib + Leucovorin + Oxaliplatin Phase I Actionable In a Phase Ib trial, the combination of Ipatasertib (GDC-0068) and mFOLFOX6 demonstrated safety in patients with advanced solid tumors, and resulted in an overall response rate of 6.1% (2/33), including partial responses in two patients, stable disease in 51.5% (17/33) of patients, a six-month progression-free survival rate of 18.2% (6/33), and maximum progression-free survival duration of 50 months in a patient with appendix cancer (PMID: 32205017; NCT01362374). 32205017
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) 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 Akt Inhibitor (Pan) 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 triple-receptor negative breast cancer not applicable Akt Inhibitor (Pan) Ipatasertib + Paclitaxel Phase II Actionable In a Phase II trial, Ipatasertib (GDC-0068) in combination with Abraxane (paclitaxel) resulted in improved progression free survival (6.2 vs 4.9 months) compared to placebo in patients with triple-receptor negative breast cancer (PMID: 28800861; NCT02162719). 28800861 detail...
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) Ipatasertib + Paclitaxel Phase I Actionable In a Phase Ib trial, the combination of Ipatasertib (GDC-0068) and Taxol (paclitaxel) demonstrated safety in patients with advanced solid tumors, and resulted in an overall response rate of 8.0% (2/25), including partial responses in two patients, stable disease in 56.0% (14/25) of patients, a six-month progression-free survival rate of 12.0% (3/25), and maximum progression-free survival duration of 14 months in a breast cancer patient (PMID: 32205017; NCT01362374). 32205017
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) Miransertib Phase I Actionable In a Phase I trial, Miransertib (ARQ092) demonstrated safety and is currently being tested for efficacy in clinical trials in patients with advanced solid tumors (American Association for Cancer Research. April 6-10, 2013. Abstract #LB-197). detail...
Unknown unknown lung cancer not applicable Akt Inhibitor (Pan) ABT-737 + Perifosine Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of ABT-737 and perifosine worked synergistically to induce apoptosis and inhibit growth of lung cancer cell lines and primary lung cancer cells in culture, and to inhibit tumor growth in a lung cancer cell line xenograft model (PMID: 27073162). 27073162
Unknown unknown astrocytoma not applicable Akt Inhibitor (Pan) Perifosine Phase II Actionable In a Phase II trial, Perifosine (KRX-0401) was well tolerated, but demonstrated limited activity in patients with anaplastic astrocytoma (n=14), with only 1 patient achieved a partial response (PMID: 31325145). 31325145
Unknown unknown Advanced Solid Tumor not applicable Akt Inhibitor (Pan) Perifosine Phase I Actionable In a Phase I trial, Perifosine demonstrated safety and preliminary efficacy in patients with a variety of advanced solid tumors (PMID: 25183650). 25183650
Unknown unknown glioblastoma multiforme no benefit Akt Inhibitor (Pan) 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 stomach cancer not applicable Akt1 Inhibitor RX-0201 Preclinical Actionable In a preclinical study, RX-0201 prevented cell proliferation of stomach cancer cells (JASCO Annual Meeting Proceedings Vol 24, No 18S (June 20 Supplement), 2006: 13102). detail...
Unknown unknown melanoma not applicable Akt1 Inhibitor A-674563 Preclinical - Cell line xenograft Actionable In a preclinical study, a melanoma cell line demonstrated sensitivity to A-674563, resulting in apoptotic activity and inhibition of Akt1 activity in culture, and inhibition of tumor growth in xenograft models (PMID: 26970307). 26970307
Unknown unknown prostate cancer not applicable Akt1 Inhibitor NSC156529 Preclinical - Cell line xenograft Actionable In a preclinical study, NSC156529 inhibited growth of human prostate cancer cell lines in culture, induced expression of differentiation markers and inhibited tumor growth in cell line xenograft models (PMID: 26294745). 26294745
Unknown unknown Advanced Solid Tumor not applicable Akt1 Inhibitor NSC156529 Preclinical Actionable In a preclinical study, NSC156529 inhibited growth of transformed human cell lines in culture (PMID: 26294745). 26294745
Unknown unknown hepatocellular carcinoma not applicable Akt1 Inhibitor NSC156529 Preclinical Actionable In a preclinical study, NSC156529 inhibited growth of human hepatocellular carcinoma cell lines in culture (PMID: 26294745). 26294745
Unknown unknown Advanced Solid Tumor not applicable Akt1 Inhibitor Akt2 Inhibitor BAY1125976 Phase I Actionable In a Phase I trial, BAY 1125976 treatment demonstrated safety and pharmacological inhibition of Akt signaling, but only resulted in a partial response in 1% (1/78) and stable disease in 38% (30/78) of patients with advanced solid tumors, and a clinical benefit rate of 27.9% (12/43) at the recommended Phase II dose (PMID: 31835495; NCT01915576). 31835495
Unknown unknown pancreatic cancer not applicable mTOR Inhibitor 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 Advanced Solid Tumor not applicable mTOR Inhibitor 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 lung cancer not applicable mTOR Inhibitor ABTL0812 Preclinical Actionable In a preclinical study, ABTL0812 inhibited growth and mTORC1 signaling and induced autophagy in a human lung cancer cell line in culture (PMID: 26671995). 26671995
Unknown unknown breast cancer not applicable mTOR Inhibitor RapaLink-1 Preclinical - Cell line xenograft Actionable In a preclinical study, a breast cancer cell line demonstrated sensitivity to RapaLink-1 in culture and in xenograft models, resulting in inhibition of both Mtor signaling and tumor growth (PMID: 27279227). 27279227
Unknown unknown T-cell acute lymphoblastic leukemia not applicable mTOR Inhibitor PI3K Inhibitor (Pan) BGT226 Preclinical - Cell culture Actionable In a preclinical study, BGT226 treatment inhibited viability of NUP214-ABL1 fusion-positive T-cell acute lymphoblastic leukemia cell lines in culture (PMID: 27821800). 27821800
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) BGT226 Phase I Actionable In a Phase I trial, BGT226 treatment was tolerated, and resulted in stable disease as best response in 28% (5/18) of patients with advanced solid tumors (PMID: 31192075). 31192075
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) BGT226 Phase I Actionable In a Phase I trial of BGT226 in patients with advanced solid tumors, limited preliminary antitumor activity and inconsistent target inhibition were observed (PMID: 22357447). 22357447
Unknown unknown CLL/SLL not applicable mTOR Inhibitor CC-115 Phase I Actionable In a Phase Ia/Ib trial, CC-115 treatment was tolerated and demonstrated manageable safety, and resulted in a partial response in 37.5% (3/8) and a stable disease in 25% (2/8) of chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) patients, and a median progression-free survival was not evaluable (PMID: 31853198; NCT01353625). 31853198
Unknown unknown Ewing sarcoma not applicable mTOR Inhibitor CC-115 Phase I Actionable In a Phase Ia/Ib trial, CC-115 treatment was tolerated and demonstrated manageable safety, and resulted in stable disease as the best response in 22.2% (2/9) of Ewing sarcoma patients, and a median progression-free survival of 56 days (PMID: 31853198; NCT01353625). 31853198
Unknown unknown prostate cancer not applicable mTOR Inhibitor CC-115 Phase I Actionable In a Phase Ia/Ib trial, CC-115 treatment was tolerated and demonstrated manageable safety, and resulted in stable disease as the best response in 63.6% (7/11) of castration-resistant prostate cancer patients, and a median progression-free survival of 345 days (PMID: 31853198; NCT01353625). 31853198
Unknown unknown glioblastoma multiforme not applicable mTOR Inhibitor CC-115 Preclinical - Pdx Actionable In a preclinical study, CC-115 increased survival of patient derived xenograft models of glioblastoma (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr 1755). detail...
Unknown unknown glioblastoma multiforme not applicable mTOR Inhibitor CC-115 Phase I Actionable In a Phase Ia/Ib trial, CC-115 treatment was tolerated and demonstrated manageable safety, and resulted in stable disease as the best response in 21.4% (3/14) of glioblastoma patients, and a median progression-free survival of 56.5 days (PMID: 31853198; NCT01353625). 31853198
Unknown unknown head and neck squamous cell carcinoma not applicable mTOR Inhibitor CC-115 Phase I Actionable In a Phase Ia/Ib trial, CC-115 treatment was tolerated and demonstrated manageable safety, and resulted in stable disease as the best response in 52.9% (9/17) of head and neck squamous cell carcinoma patients, and a median progression-free survival of 74 days (PMID: 31853198; NCT01353625). 31853198
Unknown unknown endometrial cancer not applicable mTOR Inhibitor CC-115 Case Reports/Case Series Actionable In a Phase Ia/Ib trial, an endometrial cancer patient achieved a complete response following treatment with CC-115 and remained tumor-free for over 4 years (PMID: 31853198; NCT01353625). 31853198
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor CC-115 Phase I Actionable In a Phase Ia/Ib trial, CC-115 treatment was tolerated and demonstrated manageable safety, and resulted in an overall response rate of 5.1% (n=39; 1 complete response, 1 partial response, 18 stable disease), and a disease control rate of 51.3% in advanced solid tumor patients (PMID: 31853198; NCT01353625). 31853198
Unknown unknown malignant choroid melanoma not applicable mTOR Inhibitor CC-115 Case Reports/Case Series Actionable In a Phase Ia/Ib trial, CC-115 treatment led to a partial response in a choroid melanoma patient that lasted for 56 days (PMID: 31853198; NCT01353625). 31853198
Unknown unknown colon cancer not applicable mTOR Inhibitor Torin 1 Preclinical - Pdx Actionable In a preclinical study, Torin 1 inhibited cell proliferation of colon cancer cell cultures and patient-derived xenograft models (PMID: 24185040). 24185040
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor 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 breast cancer not applicable mTOR Inhibitor OSI-027 Preclinical - Cell line xenograft Actionable In a preclinical study, OSI-027 inhibited mTORC1 and mTORC2 signaling and growth in breast cancer cells in culture and in cell line xenograft models (PMID: 21673091). 21673091
Unknown unknown colorectal cancer not applicable mTOR Inhibitor OSI-027 Preclinical - Cell line xenograft Actionable In a preclinical study, OSI-027 inhibited mTORC1 and mTORC2 signaling and growth in colorectal cancer cell line xenograft models (PMID: 21673091). 21673091
Unknown unknown neuroendocrine tumor not applicable mTOR Inhibitor Onatasertib Phase Ib/II Actionable In a Phase Ib/II trial, treatment with Onatasertib (CC-223) in patients with well-differentiated neuroendocrine tumors of non-pancreatic gastrointestinal or unknown origin resulted in a partial response in 7.3% (3/41) of patients, stable disease in 82.9% (34/41), a median progression-free survival of 19.5 months, and tumor shrinkage in 73.2% (30/41) (PMID: 31527867). 31527867
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor 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 colon adenocarcinoma not applicable mTOR Inhibitor Onatasertib Phase I Actionable In a Phase I trial, CC-223 demonstrated safety and some efficacy in patients with solid tumors, including colon cancer (J Clin Oncol 31, 2013 (suppl; abstr 2606). detail...
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) DS-7423 Phase I Actionable In a Phase I trial, DS-7423 demonstrated safety and preliminary clinical activity in patients with advanced solid tumors (Ann Oncol (2014) 25 (suppl 4): iv153). detail...
Unknown unknown ovarian clear cell adenocarcinoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) DS-7423 Preclinical - Cell line xenograft Actionable In a preclinical study, DS-7423 inhibited proliferation of ovarian clear cell adenocarcinoma cell lines in culture and suppressed tumor growth in cell line xenograft animal models (PMID: 24504419). 24504419
Unknown unknown triple-receptor negative breast cancer not applicable mTOR Inhibitor Carboplatin + Docetaxel + Gemcitabine + Itraconazole Clinical Study Actionable In a retrospective analysis, triple-negative breast cancer patients treated with the combination of Itraconazole with Docefrez (docetaxel), Paraplatin (carboplatin), and Gemzar (gemcitabine) chemotherapy demonstrated an overall response rate of 62% (8/13), a median progression-free survival of 10.8 months, and a median overall survival of 20.4 months (PMID: 24982411). 24982411
Unknown unknown ovarian cancer not applicable mTOR Inhibitor Itraconazole Phase I Actionable In a Phase I clinical trial, Itraconazole, in combination with chemotherapy, demonstrated safety and efficacy in patients with ovarian cancer (PMID: 24778064). 24778064
Unknown unknown renal cell carcinoma not applicable mTOR Inhibitor Itraconazole Preclinical - Cell culture Actionable In a preclinical study, Itraconazole resulted in antiangiogenic activity and inhibited cell proliferation of renal carcinoma cells in culture (PMID: 22025615). 22025615
Unknown unknown lung non-squamous non-small cell carcinoma not applicable mTOR Inhibitor Itraconazole + Pemetrexed Disodium Phase II Actionable In a Phase II trial, Itraconazole, in combination with Alimta (pemetrexed), increased overall survival by 24 months in patients with metastatic nonsquamous non-small cell lung cancer (PMID: 23546045). 23546045
Unknown unknown hepatocellular carcinoma not applicable mTOR Inhibitor KU-0063794 Preclinical - Cell line xenograft Actionable In a preclinical study, KU-0063794 inhibited cell survival and reduced colony formation of hepatocellular carcinoma cells in culture, and inhibited tumor growth in cell line xenograft models (PMID: 26278819). 26278819
Unknown unknown breast cancer not applicable mTOR Inhibitor 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 Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 colorectal cancer not applicable mTOR Inhibitor WYE-354 Preclinical Actionable In a preclinical study, WYE-354 induced cell cycle arrest and inhibited cell proliferation of colorectal cancer cells in culture (PMID: 19584280). 19584280
Unknown unknown melanoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 esophageal cancer not applicable mTOR Inhibitor PP-121 Preclinical - Cell line xenograft Actionable In a preclinical study, PP-121 inhibited proliferation and growth of esophageal cancer cells in culture and in cell line xenograft models (PMID: 26235881). 26235881
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (J Clin Oncol (Meeting Abstracts) 2011 29: 3018). detail...
Unknown unknown urinary bladder cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) GSK2126458 Phase I Actionable In Phase I trial, GSK2126458 treatment was well-tolerated and resulted in a partial response and stable disease in two patients and one patient with bladder cancer, respectively (PMID: 26603258). 26603258
Unknown unknown renal cell carcinoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 treatment was well-tolerated and resulted in some efficacy in renal cell carcinoma patients including stable disease in 13% (3/24), one patient with a complete response, and one patient with a partial response (PMID: 26603258). 26603258
Unknown unknown breast cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 was well-tolerated and resulted in some efficacy in patients with breast cancer, including stable disease in 31% (7/22) and one patient with a partial response (PMID: 26603258). 26603258
Unknown unknown endometrial cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) GSK2126458 Phase I Actionable In a Phase I trial, GSK2126458 treatment was well-tolerated and resulted in some efficacy in endometrial cancer patients including stable disease in 27% (4/15) and one patient with a partial response (PMID: 26603258). 26603258
Unknown unknown multiple myeloma not applicable mTOR Inhibitor Bortezomib + Torkinib Preclinical - Cell culture Actionable In a preclinical study, Torkinib (PP242) worked synergistically with Velcade (Bortezomib) to induce apoptosis in multiple myoloma cell lines in culture (PMID: 20686120). 20686120
Unknown unknown ovarian serous carcinoma not applicable mTOR Inhibitor Carboplatin + Torkinib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of the mTORC1/2 inhibitor Torkinib (PP242) and Paraplatin (carboplatin) increased survival and inhibited tumor growth in platinum-resistant ovarian serous carcinoma cell line xenograft models, with increased efficacy over either agent alone or mTORC1 inhibitor treatment (PMID: 27196780). 27196780
Unknown unknown gastric adenocarcinoma not applicable mTOR Inhibitor Torkinib Preclinical Actionable In a preclinical study, Torkinib (PP242) inhibited signaling of the PI3K/AKT/mTOR pathway and subsequent cell proliferation of gastric cancer cells in culture (PMID: 25035961). 25035961
Unknown unknown multiple myeloma not applicable mTOR Inhibitor 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 mantle cell lymphoma not applicable mTOR Inhibitor 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 Advanced Solid Tumor not applicable mTOR Inhibitor GDC-0349 Preclinical - Cell line xenograft Actionable In a preclinical study, the mTOR inhibitor GDC-0349 demonstrated inhibition of tumor growth in cell line xenograft models of solid tumors (PMID: 24900569). 24900569
Unknown unknown renal cell carcinoma not applicable mTOR Inhibitor GDC-0349 Preclinical - Cell line xenograft Actionable In a preclinical study, the mTOR inhibitor GDC-0349 demonstrated inhibition of tumor growth in cell line xenograft models of solid tumors, including renal adenocarcinoma (PMID: 24900569). 24900569
Unknown unknown prostate cancer not applicable mTOR Inhibitor 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 breast cancer not applicable mTOR Inhibitor 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 head and neck squamous cell carcinoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) GSK1059615 Preclinical - Cell line xenograft Actionable In a preclinical study, GSK1059615 treatment in a head and neck squamous cell carcinoma cell line resulted in inhibition of both cell growth and cell proliferation and induced necrosis in culture, and inhibited tumor growth in cell line xenograft models (PMID: 28187451). 28187451
Unknown unknown lymphoma not applicable mTOR Inhibitor Aldoxorubicin + Metformin Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Glucophage (metformin) and Aldoxorubicin inhibited cell growth in human lymphoma cell lines in culture and inhibited tumor growth in xenograft models (PMID: 22378068). 22378068
Unknown unknown prostate cancer not applicable mTOR Inhibitor 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 pancreatic cancer no benefit mTOR Inhibitor Capecitabine + Cisplatin + Epirubicin + Gemcitabine + Metformin Phase II Actionable In a Phase II trial, pancreatic cancer patients treated with PEXG combined with Glucophage (metformin) demonstrated a 6 month PFS of 42% (13/31), which did not differ from the control group 6 month PFS of 52% (15/29) (PMID: 26459175). 26459175
Unknown unknown lung non-small cell carcinoma not applicable mTOR Inhibitor 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 endometrial cancer not applicable mTOR Inhibitor mTORC1 Inhibitor Everolimus + Letrozole + Metformin Phase II Actionable In a Phase II trial, combination therapy consisted of Afinitor (everolimus), Femara (letrozole), and Glucophage (metformin) resulted in partial response in 29% (14/48) and stable disease in 38% (18/48) of patients with recurrent endometrioid endometrial cancer, the clinical benefit rate was not associated with KRAS mutation status in these patients (J Clin Oncol 34, 2016 (suppl; abstr 5506)). detail...
Unknown unknown breast cancer not applicable mTOR Inhibitor mTORC1 Inhibitor Everolimus + Metformin Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Afinitor (everolimus) and Glucophage (metformin) resulted in a synergistic effect in breast cancer cells, resulting in both greater inhibition of cell growth in culture and decreased tumor growth in cell line xenograft models when compared to either agent alone (PMID: 26351208). 26351208
Unknown unknown head and neck squamous cell carcinoma not applicable mTOR Inhibitor JPH203 + Metformin Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of JPH203 (KYT-0353) and Glucophage (metformin) decreased proliferation of head and neck squamous cell cancer cell lines in culture, and reduced tumor growth in a head and neck squamous cell cancer cell line xenograft model (PMID: 27262901). 27262901
Unknown unknown breast cancer not applicable mTOR Inhibitor 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 prostate cancer not applicable mTOR Inhibitor Metformin Phase II Actionable In a Phase II trial, Glucophage (metformin) demonstrated safety and preliminary efficacy in patients with castration-resistant prostate cancer (PMID: 24412228). 24412228
Unknown unknown lung non-small cell carcinoma not applicable mTOR Inhibitor Metformin Preclinical - Cell culture Actionable In a preclinical study, Glucophage (metformin) inhibited proliferation and induced cell-cycle arrest and apoptosis in non-small cell lung cancer cell lines in culture, independent of STK11 (LKB1) status (PMID: 26847819). 26847819
Unknown unknown lung cancer not applicable mTOR Inhibitor Metformin Preclinical - Cell culture Actionable In a preclinical study, Glucophage (metformin) inhibited proliferation of several lung cancer cell lines in culture, including squamous, adenocarcinoma, large cell, and small celll lung cancer cell lines (PMID: 22576795). 22576795
Unknown unknown pancreatic cancer no benefit mTOR Inhibitor 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 head and neck squamous cell carcinoma not applicable mTOR Inhibitor Metformin Clinical Study Actionable In a meta-analysis, Glucophage (metformin) treatment decreased recurrence and metastasis and improved overall survival of head and neck squamous cell carcinoma patients (PMID: 25636350). 25636350
Unknown unknown Indication other than cancer not applicable mTOR Inhibitor Metformin FDA approved Actionable Glucophage (metformin) is FDA approved for use in patients with type-2 diabetes (FDA.gov). detail... detail...
Unknown unknown hepatocellular carcinoma not applicable mTOR Inhibitor Metformin + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, addition of Glucophage (metformin) sensitized hepatocellular carcinoma cells to Nexavar (sorafenib) induced apoptosis in culture, resulted in suppression of postoperative intrahepatic recurrence and lung metastasis in cell line xenograft models (PMID: 26957312). 26957312
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor mTORC1 Inhibitor 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 Advanced Solid Tumor not applicable mTOR Inhibitor DS-3078a Phase I Actionable In a Phase I trial, DS-3078a demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Mol Cancer Ther November 2013 12:C173). detail...
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, DCBCI0901 inhibited PI3K and mTOR activation and inhibited growth of several human tumor cell lines in culture and in xenograft models (Mol Cancer Ther November 2013 12; C270). detail...
Unknown unknown lung non-small cell carcinoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 leukemia not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 prostate cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) DCBCI0901 Preclinical - Cell line xenograft Actionable In a preclinical study, prostate cancer cells treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture and inhibition of tumor growth in cell-line xenograft models (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown breast cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) DCBCI0901 Preclinical - Cell culture Actionable In a preclinical study, breast cancer cell lines treated with DCBCI0901 demonstrated inhibition of cell proliferation in culture (Mol Cancer Ther 2013;12(11 Suppl):Abstract nr C270). detail...
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) LY3023414 Phase I Actionable In a Phase I trial, LY3023414 demonstrated tolerability and inhibition of PI3K/mTOR signaling and resulted a disease control rate of 34.0% (16/47) in patients with advanced solid tumors, with one partial response and 15 patients achieving stable disease (PMID: 29636360; NCT01655225). detail... 29636360
Unknown unknown endometrial cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) LY3023414 Phase II Actionable In a Phase II trial, patients with advanced endometrial cancer harboring a PI3K pathway mutation demonstrated a best overall response rate of 16% (4/25), a clinical benefit rate of 28% (7/25) at 12 weeks, a progression-free survival of 2.5 months, and overall survival of 9.2 months when treated with LY3023414 (PMID: 31880826; NCT01775072). 31880826
Unknown unknown ovarian cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) ABT-737 + AZD8055 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of AZD8055 and Mekinist (trametinib) enhanced the sensitivity of ovarian cancer cells to ABT-737 in culture, resulting in greater apoptotic activity and cell cycle arrest when compared to Mekinist (trametinib) alone (PMID: 27980105). 27980105
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) AZD8055 Phase I Actionable In a Phase I trial, AZD8055 treatment demonstrated safety and tolerability, and resulted in stable disease for more than 4 months in 14% (7/49) of patients with advanced solid tumors or lymphoma (PMID: 22935583). 22935583
Unknown unknown breast cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) AZD8055 Preclinical - Cell culture Actionable In a preclinical study, breast cancer cells harboring PIK3CA (H1047R or E545K) and PTEN mutations demonstrated sensitivity to AZD8055 treatment in culture (PMID: 31879363). 31879363
Unknown unknown lung non-small cell carcinoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) AZD8055 + SBI-0206965 Preclinical - Cell culture Actionable In a preclinical study, the addition of SBI-0206965 to treatment with AZD8055 resulted in increased apoptosis in a non-small cell lung cancer cell line in culture (PMID: 26118643). 26118643
Unknown unknown rhabdomyosarcoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) AZD8055 + Selumetinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Koselugo (selumetinib) and AZD8055 worked synergistically to inhibit tumor growth in xenograft models of rhabdomyosarcoma (PMID: 23918606). 23918606
Unknown unknown colorectal cancer not applicable mTOR Inhibitor WYE-687 Preclinical Actionable In a preclinical study, WYE-687 induced cell cycle arrest and inhibited cell proliferation of colorectal cancer cells in culture (PMID: 19584280). 19584280
Unknown unknown colorectal cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) GDC-0980 Preclinical - Cell line xenograft Actionable In a preclinical study, the dual PI3K/mTOR inhibitor Apitolisib (GDC-0980) reduced vascularization in cell line xenograft models of colorectal cancer (PMID: 23814482). 23814482
Unknown unknown malignant pleural mesothelioma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 ovarian clear cell carcinoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 renal cell carcinoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) GDC-0980 Phase II Actionable In a Phase II clinical trial, Apitolisib (GDC-0980) was inferior to Afinitor (everolimus) (median PFS, 3.7 months (n=42) vs. 6.1 months (n=43), respectively) and overall survival trended lower in the Apitolisib (GDC-0980) arm in patients with metastatic renal cell carcinoma (PMID: 26951309). 26951309
Unknown unknown peritoneal mesothelioma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) GDC-0980 Phase I Actionable In a Phase I trial, Apitolisib (GDC-0980) treatment resulted in tumor regression in two patients with peritoneal mesothelioma (PMID: 26787751). 26787751
Unknown unknown ovarian cancer no benefit mTOR Inhibitor PI3K Inhibitor (Pan) Pimasertib + Voxtalisib Phase II Actionable In a Phase II trial, the combination of Pimasertib (MSC1936369B) and XL765 (SAR245409) versus Pimasertib (MSC1936369B) alone resulted in an objective response rate (ORR) of 9.4% and 12.1%, and a median progression-free survival of 9.99 mo and 7.23 mo, respectively, in patients with ovarian carcinoma (n=65), and 18 pts treated with combination and 19 pts treated with single therapy discontinued the study, and thus, the study was terminated due to a low ORR and high rate of discontinuation (PMID: 31870556). 31870556
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) Voxtalisib Phase I Actionable In a Phase I trial, SAR245409 (XL765) reduced PI3K and mTORC1/mTORC2 pathway signaling and demonstrated safety and efficacy irrespective of molecular alterations in the PI3K pathway, in patients with advanced solid tumors (PMID: 24583798). 24583798
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) Voxtalisib Phase I Actionable In a Phase I trial, SAR245409 (XL765) demonstrated safety and efficacy in patients with solid tumors (PMID: 18959794). 18959794
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) PKI-402 Preclinical - Cell culture Actionable In a preclinical study, PKI-402 inhibited growth of several human solid tumor cell lines in culture (PMID: 20371716). 20371716
Unknown unknown prostate cancer no benefit mTOR Inhibitor PI3K Inhibitor (Pan) 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 ovarian cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 leiomyosarcoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) Aldoxorubicin + Dactolisib Preclinical Actionable In a preclinical study, the combination of doxorubicin and BEZ235 produced a synergistic effect in leiomyosarcoma cells both in culture and in mouse models, resulting in apoptotic induction and a 68% reduction in tumor volume (PMID: 26952093). 26952093
Unknown unknown pancreatic endocrine carcinoma no benefit mTOR Inhibitor PI3K Inhibitor (Pan) Dactolisib Phase II Actionable In a Phase II clinical trial, BEZ235 was not well-tolerated and demonstrated modest anti-tumor activity in pancreatic neuroendocrine tumor patients who had progressed on Afinitor (everolimus), with a 51.6% (16/31) progression-free survival rate at 16 weeks (PMID: 26851029). 26851029
Unknown unknown peritoneal mesothelioma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) Dactolisib Preclinical Actionable In a preclinical study, BEZ235 treatment resulted in suppression of PI3K and mTOR signaling and growth inhibition in patient-derived malignant peritoneal mesothelioma cell lines in culture (PMID: 20839315). 20839315
Unknown unknown endometrial cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) Dactolisib Preclinical Actionable In a preclinical study, BEZ235 suppressed tumor growth in endometrial xenografts (PMID: 22662154). 22662154
Unknown unknown leiomyosarcoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) Dactolisib Preclinical Actionable In a preclinical study, leiomyosarcoma cells treated with BEZ235 in culture and in mouse models demonstrated increased levels of apoptosis and a 42% reduction in tumor volume (PMID: 26952093). 26952093
Unknown unknown prostate carcinoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) Dactolisib Preclinical Actionable In a preclinical study, treatment with BEZ235 resulted in a decrease in prostate cancer progenitor cells in culture and reduced tumor growth in prostate carcinoma xenograft models (PMID: 21138868). 21138868
Unknown unknown astrocytoma not applicable mTOR Inhibitor mTORC1 Inhibitor PI3K Inhibitor (Pan) 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 Advanced Solid Tumor no benefit mTOR Inhibitor mTORC1 Inhibitor PI3K Inhibitor (Pan) Dactolisib + Everolimus Phase Ib/II Actionable In a Phase Ib trial, the combination of BEZ235 and Afinitor (everolimus) resulted in limited efficacy in patients with advanced solid tumors and was discontinued due to multiple intolerable side effects (PMID: 28357727). 28357727
Unknown unknown prostate cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) Dactolisib + unspecified CTLA4 antibody + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, combination of myeloid-derived suppressor cell-targeting with BEZ235 and immune checkpoint blockade with anti-CTLA4 and anti-PD-1 antibodies resulted in synergistic inhibition of tumor growth and metastasis in transgenic mouse models of metastatic castration-resistant prostate cancer (PMID: 28321130). 28321130
Unknown unknown breast cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 ovarian cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 urinary bladder cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 prostate cancer not applicable mTOR Inhibitor AZD8186 + Vistusertib Phase I Actionable In a Phase I trial, the combination of AZD8186 and Vistusertib (AZD2014) resulted in a partial response in a patient with castration resistant prostate cancer (J Clin Oncol 35, 2017 (suppl; abstr 2570)). detail...
Unknown unknown Ewing sarcoma not applicable mTOR Inhibitor 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 ovarian cancer not applicable mTOR Inhibitor Paclitaxel + Vistusertib Preclinical Actionable In a preclinical study, the combination of Vistusertib (AZD2014) and Taxol (paclitaxel) inhibited growth of ovarian cancer cells in culture (AACR; Cancer Res 2012;72(8 Suppl):Abstract nr 931). detail...
Unknown unknown ovarian serous carcinoma not applicable mTOR Inhibitor Paclitaxel + Vistusertib Phase I Actionable In a Phase I trial, the combination therapy of Taxol (paclitaxel) and Vistusertib (AZD2014) resulted in a RECIST response rate of 64% (16/25), a CA125 response rate of 52% (13/25), and a median progression-free survival of 5.8 months in patients with high grade serous ovarian cancer (PMID: 30016392; NCT02193633). 30016392
Unknown unknown lung non-small cell carcinoma not applicable mTOR Inhibitor Paclitaxel + Vistusertib Phase I Actionable In a Phase I trial, the combination therapy of Taxol (paclitaxel) and Vistusertib (AZD2014) resulted in a RECIST response rate of 35% (8/23) and a median progression-free survival of 5.8 months in patients with squamous non-small cell lung carcinoma (PMID: 30016392; NCT02193633). 30016392
Unknown unknown glioblastoma multiforme not applicable mTOR Inhibitor 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 colorectal cancer not applicable mTOR Inhibitor Vistusertib Preclinical - Cell line xenograft Actionable In a preclinical study, Vistusertib (AZD2014) induced apoptosis and decreased viability of colorectal cancer cell lines in culture, and inhibited tumor growth in colorectal cancer cell line xenograft models (PMID: 24309100). 24309100
Unknown unknown prostate cancer not applicable mTOR Inhibitor CC214-2 Preclinical - Cell line xenograft Actionable In a preclinical study, prostate cancer cell line xenograft models demonstrated inhibition of tumor growth when treated with CC214-2 (PMID: 23414803). 23414803
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) Gedatolisib Phase I Actionable In a Phase I trial, Gedatolisib (PF-05212384) demonstrated safety and efficacy, which resulted in antitumor activity and stable disease greater than six months in 10.4% (8/27) of patients with solid malignant tumors (PMID: 25652454). 25652454
Unknown unknown breast cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) Gedatolisib Preclinical - Cell line xenograft Actionable In a preclinical study, Gedatolisib (PF-05212384) suppressed phosphorylation of PI3K/mTOR effectors and induced apoptosis in human breast cancer cell lines with elevated PI3K/mTOR signaling in culture and in cell line xenograft models (PMID: 21325073). 21325073
Unknown unknown head and neck squamous cell carcinoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) Gedatolisib Preclinical - Cell line xenograft Actionable In a preclinical study, Gedatolisib (PF-05212384) increased the the sensitivity of head and neck squamous cancer cells to radiation as indicated by decreased downstream signaling of the PI3K/mTOR pathway and reduced growth both in culture and in cell line xenograft models (PMID: 25724523). 25724523
Unknown unknown colorectal cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) Gedatolisib + Irinotecan Case Reports/Case Series Actionable In a Phase I trial, treatment with the combination of Gedatolisib (PF-05212384) and Camptosar (irinotecan) resulted in an overall response rate of 4.7%, with 2 colorectal cancer patients achieving a partial response, and clinical benefit rate of 16.3% in an advanced solid tumor patient cohort comprising 93% colorectal cancer patients (PMID: 29067643; NCT01347866)). 29067643
Unknown unknown head and neck squamous cell carcinoma not applicable mTOR Inhibitor Abemaciclib + Torin 2 Preclinical Actionable In a preclinical study, the combination of Abemaciclib (LY2835219) and Torin2 worked synergistically to reduce viability of head and neck squamous cell carcinoma cells in culture (PMID: 26909611). 26909611
Unknown unknown T-cell acute lymphoblastic leukemia not applicable mTOR Inhibitor Torin 2 Preclinical - Cell culture Actionable In a preclinical study, Torin 2 treatment inhibited viability of NUP214-ABL1 fusion-positive T-cell acute lymphoblastic leukemia cell lines in culture (PMID: 27821800). 27821800
Unknown unknown hepatocellular carcinoma not applicable mTOR Inhibitor Torin 2 Preclinical Actionable In a preclinical study, Torin 2 inhibited proliferation of hepatocellular cells in culture (PMID: 26239364). 26239364
Unknown unknown malignant glioma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) GDC-0084 Phase I Actionable In a Phase I trial, GDC-0084 treatment demonstrated expected toxicity and blood-brain barrier penetration, resulted in stable disease as best response in 40% (19/47) of patients with recurrent high-grade glioma (J Clin Oncol (PMID: 31937616; NCT01547546). 31937616
Unknown unknown Indication other than cancer not applicable mTOR Inhibitor PP30 Preclinical Actionable In a preclinical study, PP30 inhibited proliferation of mouse embryonic fibroblasts more effectively than rapamycin (PMID: 19209957). 19209957
Unknown unknown triple-receptor negative breast cancer not applicable mTOR Inhibitor Alisertib + Sapanisertib Preclinical - Pdx Actionable In a preclinical study, the combination treatment of Alisertib (MLN8237) and Sapanisertib (MLN0128) resulted in decreased proliferation in triple-receptor negative breast cancer (TNBC) cell lines in culture and reduced tumor growth in patient-derived xenograft (PDX) TNBC models, with one model showing tumor growth inhibition of 94.27% compared to 54.47% with Sapanisertib (MLN0128) alone and 35.09% with Alisertib (MLN8237) alone (PMID: 32414750). 32414750
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor Alisertib + Sapanisertib Phase I Actionable In a Phase I trial, the combination of Alisertib (MLN8237) and Sapanisertib (MLN0128) demonstrated tolerability in patients with advanced solid tumors, with 70% (7/10) of patients demonstrating stable disease for a median duration of 4 months (PMID: 32414750). 32414750
Unknown unknown malignant fibrous histiocytoma not applicable mTOR Inhibitor EHop-016 + Sapanisertib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of EHop-016 and Sapanisertib (MLN0128) resulted in increased apoptosis and reduced growth of a myxofibrosarcoma cell line in culture, and resulted in greater tumor growth inhibition in myxofibrosarcoma cell line xenograft models compared to either agent alone (PMID: 27577794). 27577794
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor PIK3CA inhibitor MLN1117 + Sapanisertib Preclinical Actionable In a preclinical study, the combination of Sapanisertib (MLN0128) and MLN1117 demonstrated synergistic anti-tumor activity in a variety of solid tumor xenograft models (Mol Cancer Ther 2013;12(11 Suppl):C176)). detail...
Unknown unknown Advanced Solid Tumor not applicable mTOR Inhibitor Sapanisertib Phase I Actionable In a Phase I trial, Sapanisertib (MLN0128) demonstrated safety and some efficacy in patients with advanced solid tumors (Mol Cancer Ther 2013;12(11 Suppl):C252). detail...
Unknown unknown prostate cancer not applicable mTOR Inhibitor Sapanisertib Phase II Actionable In a Phase II trial, Sapanisertib (MLN0128) treatment demonstrated limited efficacy, with a median time on treatment of 11 weeks and stable disease as best response (PMID: 29508246). 29508246
Unknown unknown prostate cancer not applicable mTOR Inhibitor Sapanisertib Preclinical Actionable In a preclinical study, Sapanisertib (MLN0128) inhibited cell proliferation, induced apoptosis, and prevented metastasis in xenograft models with prostate cancer (PMID: 22367541). 22367541
Unknown unknown malignant fibrous histiocytoma not applicable mTOR Inhibitor Sapanisertib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with Sapanisertib (MLN0128) resulted in growth suppression and arrest in a myxofibrosarcoma cell line in culture, and inhibited tumor growth in myxofibrosarcoma cell line xenograft models (PMID: 27577794). 27577794
Unknown unknown lung non-small cell carcinoma not applicable mTOR Inhibitor Sapanisertib + SBI-0206965 Preclinical - Cell culture Actionable In a preclinical study, the addition of SBI-0206965 to treatment with Sapanisertib (MLN0128) resulted in increased apoptosis in a non-small cell lung cancer cell line in culture (PMID: 26118643). 26118643
Unknown unknown prostate cancer not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 colorectal cancer not applicable mTOR Inhibitor WAY-600 Preclinical Actionable In a preclinical study, WAY-600 induced cell cycle arrest and inhibited cell proliferation of colorectal cancer cells in culture (PMID: 19584280). 19584280
Unknown unknown diffuse large B-cell lymphoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 lymphoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 lymphoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 Advanced Solid Tumor not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 lymphoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) PQR309 + Rituximab Preclinical - Cell culture Actionable In a preclinical study, the combination therapy of PQR309 and Rituxan (rituximab) led to a synergistic effect in 2/5 lymphoma cell lines in culture (PMID: 29066507). 29066507
Unknown unknown lymphoma not applicable mTOR Inhibitor PI3K Inhibitor (Pan) 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 lung non-small cell carcinoma not applicable mTOR Inhibitor Antroquinonol Phase I Actionable In a Phase I trial, antroquinonol (Hocena) demonstrated safety and preliminary efficacy in metastatic NSCLC patients previously treated with two prior chemotherapy regimens (PMID: 26807250). 26807250
Unknown unknown malignant glioma not applicable mTOR Inhibitor 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 renal cell carcinoma not applicable mTORC1 Inhibitor Bevacizumab + Temsirolimus Phase II Actionable In a Phase II trial, patients with metastatic renal cell carcinoma (mRCC) treated with a combination of Avastin (bevacizumab) and Torisel (temsirolimus) demonstrated an overall 4 month PFS rate of 65% while those patients with VEGFR tyrosine kinase inhibitor refractory mRCC showed a median PFS and OS of 6.5 and 9.6 months, respectively (PMID: 27036973). 27036973
Unknown unknown clear cell renal cell carcinoma no benefit mTORC1 Inhibitor Bevacizumab + Temsirolimus Phase II Actionable In a Phase II clinical trial, treatment with the combination of Torisel (temsirolimus) and Avastin (bevacizumab) did prolong progression-free survival compared to treatment with Avastin (bevacizumab) as a single agent (7.6 months vs 7.4 months) in patients with renal clear cell carcinoma (PMID: 26077237). 26077237
Unknown unknown head and neck squamous cell carcinoma not applicable mTORC1 Inhibitor Carboplatin + Paclitaxel + Temsirolimus Phase II Actionable In a Phase II trial, the combination of Torisel (temsirolimus), Paraplatin (carboplatin), and Taxol (paclitaxel) resulted in an objective response rate of 41.7% (15/36), which included all partial responses, and 52.3% (19/36) had stable disease (PMID: 28961834). 28961834
Unknown unknown colon cancer not applicable mTORC1 Inhibitor 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 colon adenocarcinoma not applicable mTORC1 Inhibitor Cetuximab + Temsirolimus Preclinical Actionable In a preclinical study, Torisel (temsirolimus) decreased resistance to Erbitux (cetuximab) in colon cancer cells (PMID: 24493623). 24493623
Unknown unknown B-cell lymphoma not applicable mTORC1 Inhibitor Denileukin diftitox + Temsirolimus Preclinical - Cell culture Actionable In a preclinical study, Torisel (temsirolimus) 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 Advanced Solid Tumor not applicable mTORC1 Inhibitor Neratinib + Temsirolimus Phase I Actionable In a Phase I study, Nerlynx (neratinib) administered with Torisel (temsirolimus) was tolerable and demonstrated antitumor activity in multiple solid tumor types, including breast cancer and NSCLC (PMID: 24323026). 24323026
Unknown unknown endometrial cancer not applicable mTORC1 Inhibitor Pegylated liposomal-doxorubicin + Temsirolimus Phase Ib/II Actionable In a Phase Ib trial, Torisel (temsirolimus) in combination with Doxil (pegylated liposomal doxorubicin) demonstrated safety and some efficacy in patients with endometrial cancer (PMID: 24577626). 24577626
Unknown unknown glioblastoma multiforme no benefit mTORC1 Inhibitor Radiotherapy + Temsirolimus Phase II Actionable In a Phase II trial, the combination of Torisel (temsirolimus) and radiotherapy did not result in an improved overall survival or progression free survival when compared to the combination of Temodar (temozolomide) and radiotherapy in glioblastoma patients with an unmethylated MGMT promoter (PMID: 27143690). 27143690
Unknown unknown clear cell renal cell carcinoma no benefit mTORC1 Inhibitor 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 lung non-small cell carcinoma not applicable mTORC1 Inhibitor Temsirolimus Phase I Actionable In a Phase I trial, Torisel (temsirolimus) in combination with radiation therapy demonstrated safety and efficacy in 5/8 NSCLC patients (PMID: 24373609). 24373609
Unknown unknown oral squamous cell carcinoma not applicable mTORC1 Inhibitor 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 head and neck squamous cell carcinoma not applicable mTORC1 Inhibitor Temsirolimus Phase II Actionable In a Phase II trial, treatment with Torisel (temsirolimus) resulted in disease stabilization in 57.6% (19/33) and tumor shrinkage in 39.4% (13/33) of patients with head and neck squamous cell carcinoma (PMID: 25527417). 25527417
Unknown unknown renal cell carcinoma not applicable mTORC1 Inhibitor Temsirolimus FDA approved Actionable In a Phase III randomized trial that supported FDA approval, treatment with Torisel (temsirolimus) resulted in an improved overall survival time of 11.1 months in patients with advanced renal cell carcinoma compared to 7.4 months in patients treated with IFN-alpha (PMID: 20332142). 20332142 detail...
Unknown unknown head and neck squamous cell carcinoma not applicable mTORC1 Inhibitor Abemaciclib + Everolimus Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Abemaciclib (LY2835219) and Afinitor (everolimus) worked synergistically to reduce viability of head and neck squamous cell carcinoma (HNSCC) cells in culture, and to inhibit tumor growth in HNSCC cell line xenograft models, with increased efficacy over either agent alone (PMID: 26909611). 26909611
Unknown unknown triple-receptor negative breast cancer not applicable mTORC1 Inhibitor 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 fallopian tube cancer not applicable mTORC1 Inhibitor Bevacizumab + Everolimus Phase II Actionable In a Phase II trial, Afinitor (everolimus) and Avastin (bevacizumab) combination treatment resulted in progression free survival at 6 months in 28% (14/50) of patients with ovarian, fallopian tube, and peritoneal cancers (J Clin Oncol 34, 2016 (suppl; abstr 5552)). detail...
Unknown unknown papillary renal cell carcinoma not applicable mTORC1 Inhibitor Bevacizumab + Everolimus Phase II Actionable In a Phase II trial, 50% (2/4) of patients with papillary renal cell carcinoma achieved a 6 month PFS when treated with a combination of Afinitor (everolimus) and Avastin (bevacizumab) (PMID: 27601542). 27601542
Unknown unknown peritoneum cancer not applicable mTORC1 Inhibitor Bevacizumab + Everolimus Phase II Actionable In a Phase II trial, Afinitor (everolimus) and Avastin (bevacizumab) combination treatment resulted in progression free survival at 6 months in 28% (14/50) of patients with ovarian, fallopian tube, and peritoneal cancers (J Clin Oncol 34, 2016 (suppl; abstr 5552)). detail...
Unknown unknown renal cell carcinoma not applicable mTORC1 Inhibitor 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 ovarian cancer not applicable mTORC1 Inhibitor Bevacizumab + Everolimus Phase II Actionable In a Phase II trial, Afinitor (everolimus) and Avastin (bevacizumab) combination treatment resulted in progression free survival at 6 months in 28% (14/50) of patients with ovarian, fallopian tube, and peritoneal cancers (J Clin Oncol 34, 2016 (suppl; abstr 5552)). detail...
Unknown unknown chromophobe renal cell carcinoma not applicable mTORC1 Inhibitor 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 prostate cancer not applicable mTORC1 Inhibitor Bicalutamide + Everolimus Phase II Actionable In a Phase II trial, Casodex (bicalutamide) and Afinitor (everolimus) combination treatment resulted in PSA response in 75% (18/24) of patients with castration-resistant prostate cancer, with a median overall survival of 28 months (PMID: 27019001). 27019001
Unknown unknown lung carcinoma not applicable mTORC1 Inhibitor PI3K Inhibitor (Pan) Buparlisib + Everolimus Preclinical Actionable In a preclinical study, Buparlisib (BKM120) in combination with Afinitor (everolimus) inhibited tumor growth in mouse lung cancer xenograft models (PMID: 22781393). 22781393
Unknown unknown triple-receptor negative breast cancer no benefit mTORC1 Inhibitor 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 neuroendocrine tumor not applicable mTORC1 Inhibitor 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 prostate cancer not applicable mTORC1 Inhibitor 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 lung non-small cell carcinoma not applicable mTORC1 Inhibitor Erlotinib + Everolimus Phase I Actionable In a Phase I trial, Afinitor (everolimus) demonstrated safety and some efficacy in combination with Tarceva (erlotinib) in patients with advanced NSCLC (PMID: 22968184). 22968184
Unknown unknown malignant peripheral nerve sheath tumor not applicable mTORC1 Inhibitor Everolimus Preclinical - Cell line xenograft Actionable In a preclinical study, malignant peripheral nerve sheath cell line xenograft models treated with Afinitor (everolimus) demonstrated a 76% decrease in tumor growth (PMID: 18483311). 18483311
Unknown unknown colon adenocarcinoma not applicable mTORC1 Inhibitor 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 malignant glioma not applicable mTORC1 Inhibitor Everolimus Phase II Actionable In a Phase II trial, Afinitor (everolimus) treatment resulted in a 6-month progression free survival rate of 87% and 55% in patients with WHO grade II and III/IV glioma, respectively (Neuro Oncol (2016) 18 (suppl 6): vi8-vi9.). detail...
Unknown unknown kidney angiomyolipoma not applicable mTORC1 Inhibitor Everolimus FDA approved Actionable In a Phase III trial (EXIST-2) that supported FDA approval, Afinitor (everolimus) treatment resulted in significantly improved response rate (42%, 33/79) compared to placebo (0%, 0/39) in patients with renal angiomyolipoma as a feature of tuberous sclerosis (n=113) or sporadic lymphanioleiomyomatosis (n=5) (PMID: 23312829; NCT00790400). 23312829 detail...
Unknown unknown lung non-small cell carcinoma not applicable mTORC1 Inhibitor Everolimus Phase Ib/II Actionable In a Phase Ib trial, Afinitor (everolimus) demonstrated safety and preliminary efficacy in patients with non-small cell lung cancer (PMID: 25673697). 25673697
Unknown unknown estrogen-receptor positive breast cancer not applicable mTORC1 Inhibitor 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 renal cell carcinoma not applicable mTORC1 Inhibitor Everolimus FDA approved Actionable In a Phase III trial (RECORD-1) that supported FDA approval, Afinitor (everolimus) improved progression-free survival (4.0 vs 1.9 months) compared to placebo in patients with metastatic renal cell carcinoma that progressed on other targeted therapies (PMID: 23659703, PMID: 18653228; NCT00410124). 23659703 detail... 18653228
Unknown unknown renal cell carcinoma not applicable mTORC1 Inhibitor Everolimus Phase II Actionable In a Phase II clinical trial, Apitolisib (GDC-0980) was inferior to Afinitor (everolimus) by median PFS, 3.7 months (n=42) vs. 6.1 months (n=43), respectively and overall survival trended lower as well in patients with metastatic renal cell carcinoma (PMID: 26951309). 26951309
Unknown unknown lymphoma not applicable mTORC1 Inhibitor Everolimus Phase II Actionable In a Phase II trial, Afinitor (everolimus) treatment resulted in an overall response rate of 44% (7/16) in T-cell lymphoma patients, with a median progression-free survival of 4.1 months and a median overall survival of 10.2 months (PMID: 25921059). 25921059
Unknown unknown islet cell tumor not applicable mTORC1 Inhibitor Everolimus FDA approved Actionable In a Phase III trial (RADIANT-3) that supported FDA approval, treatment with Afinitor (everolimus) prolonged progression-free survival (11.0 vs 4.6 months, HR=0.35, p<0.001) compared to placebo in patients with progressive pancreatic neuroendocrine tumors (PMID: 21306238; NCT00510068). detail... 21306238
Unknown unknown transitional cell carcinoma not applicable mTORC1 Inhibitor 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 breast cancer not applicable mTORC1 Inhibitor Everolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Afinitor (everolimus) treatment inhibited phosphorylation of Ybx1, p70S6K, and S6, and reduced proliferation of breast cancer cells harboring mutations in PIK3CA (H1047R or E545K) and PTEN, and antiestrogen-resistant cells in culture, and inhibited tumor growth in an orthotopic cell line xenograft model (PMID: 31879363). 31879363
Unknown unknown breast cancer not applicable mTORC1 Inhibitor Everolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Afinitor (everolimus) inhibited the growth of a breast cancer cell line in culture and resulted in decreased tumor volume in a cell line xenograft model (PMID: 26351208). 26351208
Unknown unknown endometrial cancer not applicable mTORC1 Inhibitor Everolimus Phase II Actionable In a Phase II trial, the mTOR inhibitor Afinitor (everolimus) demonstrated efficacy and tolerability in patients with chemotherapy-refractory advanced or metastatic endometrial cancer (PMID: 23612453). 23612453
Unknown unknown thyroid gland cancer not applicable mTORC1 Inhibitor Everolimus Phase II Actionable In a Phase II trial, treatment with Afinitor (everolimus) resulted in a median progression-free survival (PFS) of 12.9 months, 2-year PFS of 23.6%, and 2-year overall survival of 73.5% in patients with differentiated thyroid cancer, and a partial response in a patient with anaplastic thyroid cancer (ATC), and disease stability for 26 months in another ATC patient (PMID: 29301825). 29301825
Unknown unknown subependymal giant cell astrocytoma not applicable mTORC1 Inhibitor Everolimus FDA approved Actionable In a Phase III trial (EXIST-1) that supported FDA approval, Afinitor (everolimus) treatment resulted in a 50% or more tumor reduction in 35% (27/78) of adult and pediatric patients diagnosed with tuberous sclerosis complex and had subependymal giant cell astrocytoma, compared to 0% (0/39) in the placebo group (PMID: 23158522; NCT00789828). 23158522 detail...
Unknown unknown oral squamous cell carcinoma not applicable mTORC1 Inhibitor Everolimus Preclinical Actionable In a preclinical study, Afinitor (everolimus) inhibited growth and mTOR signaling in oral squamous cell carcinoma cell lines (PMID: 25682238). 25682238
Unknown unknown neuroendocrine tumor not applicable mTORC1 Inhibitor Everolimus FDA approved Actionable In a Phase III trial (RADIANT-4) supporting FDA approval, Afinitor (everolimus) treatment significantly improved median progression-free survival (11.0 months) comparing to placebo (3.9 months) in patients with progressive neuroendocrine tumours of the lung or gastrointestinal tract origin (PMID: 26703889; NCT01524783). detail... 26703889
Unknown unknown stomach cancer no benefit mTORC1 Inhibitor Everolimus Phase III Actionable In a Phase III trial, Afinitor (everolimus) did not significantly improve overall survival (5.4 vs 4.3 months) and progression free survival (1.7 vs 1.4 months) over placebo in previously treated advanced gastric cancer patients (PMID: 24043745). 24043745
Unknown unknown clear cell renal cell carcinoma not applicable mTORC1 Inhibitor Everolimus + Hydroxychloroquine Phase Ib/II Actionable In a Phase I/II trial, treatment with the combination of Afinitor (everolimus) and Plaquenil (hydroxychloroquine) was well-tolerated, and resulted in a median progression-free survival (PFS) of 6.3 months, PFS of 6 months or greater in 45% (15/33), and partial response (PR) or stable disease (SD) greater than 3 months in 66% (22/33; 2 PR and 20 SD) of clear cell renal cell carcinoma patients (PMID: 30635337). 30635337
Unknown unknown ovarian cancer not applicable mTORC1 Inhibitor Everolimus + JI-101 Phase 0 Actionable In a pilot study, JI-101 in combination with Afinitor (everolimus) demonstrated safety and preliminary efficacy, resulted in stable disease in a majority of patients with ovarian cancer (PMID: 26365907). 26365907
Unknown unknown renal cell carcinoma not applicable mTORC1 Inhibitor 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 renal cell carcinoma not applicable mTORC1 Inhibitor 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 meningioma not applicable mTORC1 Inhibitor 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 neuroendocrine tumor not applicable mTORC1 Inhibitor Everolimus + Octreotide Phase III Actionable In a Phase III trial, addition of Afinitor (everolimus) to Octreotide did not significantly improve median overall survival (29.2 vs 35.2 months) compared to placebo in patients with advanced neuroendocrine tumors associated with carcinoid syndrome (PMID: 28444114). 28444114
Unknown unknown neuroendocrine tumor no benefit mTORC1 Inhibitor Everolimus + Pasireotide Phase II Actionable In a Phase II trial, addition of Pasireotide to Afinitor (everolimus) did not significantly affect progression free survival (16.8 vs 16.6 months) or overall disease control rate (77.2% vs 82.7%) in patients with well-differentiated, progressive pancreatic neuroendocrine tumors (PMID: 28327907). 28327907
Unknown unknown clear cell renal cell carcinoma no benefit mTORC1 Inhibitor Everolimus + Pazopanib Phase II Actionable In a Phase II trial, alternating treatment with Votrient (pazopanib) and Afinitor (everolimus) did not improve 1-year progression free survival rate (45% vs 32%) or time to progression/death (7.4 vs 9.4 months) compared to continuous Votrient (pazopanib) treatment in patients with renal clear cell carcinoma (PMID: 27918762). 27918762
Unknown unknown pancreatic adenocarcinoma no benefit mTORC1 Inhibitor Everolimus + Ribociclib Phase I Actionable In a Phase I trial, treatment with the combination of Afinitor (everolimus) and Kisqali (ribociclib) did not lead to a clinical benefit in pancreatic adenocarcinoma patients who previously progressed on chemotherapy (n=11), resulting in a median progression-free survival of 1.8 months, a median overall survival of 3.7 months, stable disease in two patients for 8 weeks, and progressive disease in nine patients (PMID: 32642630; NCT02985125). 32642630
Unknown unknown renal cell carcinoma not applicable mTORC1 Inhibitor Everolimus + Sunitinib Phase II Actionable In a Phase II trial, Sutent (sunitinib) as first line therapy followed by second line therapy, Afinitor (everolimus), resulted in a greater overall survival (29.5 mo vs 22.4 mo) compared to the reverse treatment of the two therapies in patients with metastatic renal cell carcinoma (PMID: 28327953). 28327953
Unknown unknown estrogen-receptor positive breast cancer not applicable mTORC1 Inhibitor Everolimus + Tamoxifen Preclinical - Cell culture Actionable In a preclinical study, the combination treatment of Afinitor (everolimus) and Nolvadex (tamoxifen) resulted in decreased colony formation by 95% in estrogen-receptor (ER) positive breast cancer cell lines while in ER positive breast cancer cell lines resistant to Nolvadex (tamoxifen), colony formation formation decreased by 76% with the addition of Afinitor (everolimus) (PMID: 27421652). 27421652
Unknown unknown renal cell carcinoma not applicable mTORC1 Inhibitor 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 neuroendocrine tumor not applicable mTORC1 Inhibitor Everolimus + Vatalanib Phase I Actionable In a Phase I trial, Vatalanib and Afinitor (everolimus) combination therapy demonstrated acceptable toxicity, resulted in a disease control rate of 66.7% in patients with neuroendocrine tumors (PMID: 32328844; NCT00655655). 32328844
Unknown unknown Advanced Solid Tumor not applicable mTORC1 Inhibitor 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 neuroendocrine tumor not applicable mTORC1 Inhibitor Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in stable disease ranging from 3-23 months in 10 patients with neuroendocrine tumors (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown renal cell carcinoma not applicable mTORC1 Inhibitor Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in partial response in 2 patients and stable disease in 1 patient with renal cell carcinoma (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown glioblastoma multiforme not applicable mTORC1 Inhibitor Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in stable disease in a patient with glioblastoma (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown Advanced Solid Tumor not applicable mTORC1 Inhibitor Everolimus + Vorolanib Phase I Actionable In a Phase I trial, combination of Vorolanib (X-82) and Afinitor (everolimus) resulted in partial response in 9.5% (2/21) and stable disease in 57.1% (12/21) of patients with advanced solid tumors (Journal of Clinical Oncology 34, no. 15_suppl (May 20 2016) 2588-2588). detail...
Unknown unknown clear cell renal cell carcinoma not applicable mTORC1 Inhibitor Everolimus + Vorolanib Phase I Actionable In a Phase I trial, Vorolanib (X-82) and Afinitor (everolimus) combination therapy was well tolerated, and resulted in an objective response rate of 32% (6/19) and a disease control rate of 100% in patients with advanced clear cell renal cell carcinoma, with a median progression-free survival of 5.6 months (PMID: 32335374; NCT03095040). 32335374
Unknown unknown gastric adenocarcinoma not applicable mTORC1 Inhibitor Paclitaxel + Ridaforolimus Phase Ib/II Actionable In a Phase Ib trial, Ridaforolimus (MK-8669), in combination with paclitaxel, produced stable disease greater than or equal to 4 months in 67% (2/3) of gastric cancer patients (PMID: 19901013). 19901013
Unknown unknown diffuse large B-cell lymphoma not applicable mTORC1 Inhibitor 7-hydroxystaurosporine + Sirolimus Preclinical Actionable In a preclinical study, Rapamune (sirolimus), in combination with UCN-01 (sc-3510), resulted in apoptosis and cell cycle arrest in diffuse large B-cell lymphoma cells in culture (PMID: 19223503). 19223503
Unknown unknown gastric adenocarcinoma not applicable mTORC1 Inhibitor Celecoxib + Sirolimus Preclinical Actionable In a preclinical study, celecoxib enhanced the efficacy of Rapamune (sirolimus) by increasing the growth inhibition of gastric cancer cells in culture (PMID: 25701378). 25701378
Unknown unknown lymphoma not applicable mTORC1 Inhibitor Denileukin diftitox + Sirolimus Preclinical Actionable In a preclinical study, a low dose of Rapamune (sirolimus) enhanced the efficacy of Ontak (denileukin diftitox) treatment in lymphoma mouse models, resulting in greater inhibition of tumor growth and higher survival compared to either agent alone (PMID: 27737881). 27737881
Unknown unknown ovarian carcinoma no benefit mTORC1 Inhibitor 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 B-cell lymphoma not applicable mTORC1 Inhibitor 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 melanoma not applicable mTORC1 Inhibitor Denileukin diftitox + Sirolimus Preclinical Actionable In a preclinical study, the combination of Ontak (denileukin diftitox) and Rapamune (sirolimus) resulted in a greater decrease in tumor volume compared to treatment with either agent alone in a melanoma mouse model (PMID: 27737881). 27737881
Unknown unknown leiomyosarcoma not applicable mTORC1 Inhibitor 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: 32089640; NCT02008877). 32089640
Unknown unknown malignant peripheral nerve sheath tumor no benefit mTORC1 Inhibitor Ganetespib + Sirolimus Phase II Actionable In a Phase II trial, treatment with the combination of Ganetespib and Rapamune (sirolimus) did not result in clinical benefit in 10 patients with malignant peripheral nerve sheath tumor (PMID: 32089640; NCT02008877). 32089640
Unknown unknown osteosarcoma not applicable mTORC1 Inhibitor Gemcitabine + Sirolimus Phase II Actionable In a Phase II trial, the combination of Gemzar (gemcitabine) and Rapamune (sirolimus) resulted in a progression-free survival rate of 44% after 4 months in osteosarcoma patients, including a partial response in two patients and stable disease in fourteen patients (PMID: 29045512). 29045512
Unknown unknown brain glioblastoma multiforme not applicable mTORC1 Inhibitor Irinotecan + Sirolimus + Sunitinib + Temozolomide Clinical Study Actionable In two clinical case studies, RIST (rapamycin, irinotecan, sunitinib, temozolomide) resulted in anti-tumor activity in patients with glioblastoma (PMID: 25123598). 25123598
Unknown unknown Ewing sarcoma not applicable mTORC1 Inhibitor MEDI-573 + Sirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of MEDI-573 and Rapamune (sirolimus) resulted in decreased Rapamune (sirolimus)-induced AKT activation and inhibited growth of a Ewing sarcoma cell line in culture and inhibited tumor growth Ewing sarcoma cell line xenograft models, with increased efficacy compared to either as a single agent (PMID: 25193511). 25193511
Unknown unknown lung non-small cell carcinoma not applicable mTORC1 Inhibitor Pemetrexed Disodium + Sirolimus Phase Ib/II Actionable In a Phase Ib/II trial, Rapamune (sirolimus) in combination with Alimta (pemetrexed) demonstrated safety and some efficacy in treating patients with NSCLC (PMID: 24658085). 24658085
Unknown unknown lung non-small cell carcinoma not applicable mTORC1 Inhibitor SBI-0206965 + Sirolimus Preclinical - Cell culture Actionable In a preclinical study, the addition of SBI-0206965 to treatment with Rapamune (sirolimus) resulted in increased apoptosis in a non-small cell lung cancer cell line in culture (PMID: 26118643). 26118643
Unknown unknown Indication other than cancer not applicable mTORC1 Inhibitor Sirolimus FDA approved Actionable Rapamune (sirolimus) is FDA approved for use in patients with lymphangioleiomyomatosis and for prophylactic immunosuppression in renal transplant patients (FDA.gov). detail... detail...
Unknown unknown Advanced Solid Tumor not applicable mTORC1 Inhibitor Sirolimus Preclinical Actionable In preclinical studies, Rapamune (sirolimus) induced apoptosis of cancer cells and increased sensitivity to Platinol (cisplatin) (PMID: 15136596). 15136596
Unknown unknown head and neck squamous cell carcinoma not applicable mTORC1 Inhibitor 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 mTORC1 Inhibitor Triolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Triolimus led to cytotoxicity and inhbition of Ras/Raf/MAPK and PI3K/Akt/mTOR pathway signaling in breast cancer cells in culture and inhibited tumor growth in cell line xenograft models (PMID: 22896668). 22896668
Unknown unknown lung cancer not applicable mTORC1 Inhibitor Triolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Triolimus led to cytotoxicity and inhibited Ras/Raf/MAPK and PI3K/Akt/mTOR pathway signaling in lung cancer cells in culture, and induced apoptosis, which resulted in tumor growth inhibition, in cell line xenograft models (PMID: 22896668). 22896668
Unknown unknown glioblastoma multiforme not applicable mTORC2 Inhibitor 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 glioblastoma multiforme not applicable mTORC2 Inhibitor JR-AB2-011 Preclinical - Cell line xenograft Actionable In a preclinical study, JR-AB2-011 resulted in tumor growth inhibition, increased apoptotic activity, and improved survival of glioblastoma cell line xenograft models (PMID: 28453552). 28453552
Unknown unknown colorectal cancer not applicable PI3K Inhibitor (Pan) R11.1.6 + ZSTK474 Preclinical - Cell culture Actionable In a preclinical study, R11.1.6 and ZSTK474 combination therapy did not inhibit cell proliferation in a colorectal cancer cell line in culture (PMID: 29720559). 29720559
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) ZSTK474 Preclinical - Cell line xenograft Actionable In a preclinical study, ZSTK474 inhibited cell proliferation of a variety of human advanced solid tumor cell lines in culture and in xenograft models (PMID: 25483727). 25483727
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) Seribantumab + XL147 Phase Ib/II Actionable In a Phase Ib trial, treatment with the combination of Pilaralisib (SAR245408, XL147) and Seribantumab (SAR256212) resulted in stable disease as best response in 52.2% (12/23) patients with advanced solid tumors, with no difference in response between patients harboring PIK3CA mutations and those with wild-type PIK3CA (PMID: 28031425). 28031425
Unknown unknown endometrial carcinoma not applicable PI3K Inhibitor (Pan) 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 colon adenocarcinoma not applicable PI3K Inhibitor (Pan) XL147 Preclinical - Cell line xenograft Actionable In a preclinical study, XL147 (SAR245408) demonstrated modest efficacy as a single agent in human cancer cell line xenograft models (PMID: 23002019). 23002019
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) XL147 Phase I Actionable In a Phase I trial, 43.9% (25/56) of patients with advanced solid tumors had stable disease as a best response to treatment with Pilaralisib (XL147), and one patient with NSCLC showed a partial response, and in a separate therapy arm, Pilaralisib (XL147) in tablet formulation demonstrated safety and some antitumor activity in advanced solid tumor patients, including 11.1% (2/18) of patients with a partial response (PMID: 29593099, PMID: 24166903; NCT00486135). 29593099 24166903
Unknown unknown lung non-small cell carcinoma not applicable PI3K Inhibitor (Pan) XL147 Phase I Actionable In a Phase I study, 25/56 (43.9%) of patients with advanced solid tumors had stable disease as a best response to treatment with XL147, and one patient with NSCLC showed a partial response to XL147 (PMID: 24166903). 24166903
Unknown unknown stomach cancer not applicable PI3K Inhibitor (Pan) Cisplatin + Deguelin Preclinical Actionable In a preclinical study, the combination of Deguelin and Platinol (cisplatin) worked synergistically to inhibit growth of gastric cancer cells in culture (PMID: 25202376). 25202376
Unknown unknown lung carcinoma not applicable PI3K Inhibitor (Pan) SRX3207 Preclinical - Cell culture Actionable In a preclinical study, SRX3207 treatment induced cytotoxicity in Lewis lung carcinoma cells in culture, and inhibited tumor growth, and increased survival in a syngeneic mouse model (PMID: 31974273). 31974273
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) Rigosertib Sodium Phase I Actionable In a Phase I study, Rigosertib (ON 01910.Na) demonstrated both safety and preliminary efficacy, resulted in a best overall response of stable disease in 41% (9/22) of patients with advanced solid tumors (PMID: 23841031; NCT01538537). 23841031
Unknown unknown myelodysplastic syndrome no benefit PI3K Inhibitor (Pan) 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 colorectal cancer not applicable PI3K Inhibitor (Pan) 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 Ewing sarcoma not applicable PI3K Inhibitor (Pan) CUDC-907 Phase I Actionable In a Phase I trial, CUDC-907 treatment resulted in stable disease for 6 treatment cycles in a pediatric patient with Ewing sarcoma (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 10542-10542; NCT02909777). detail...
Unknown unknown non-Hodgkin lymphoma not applicable PI3K Inhibitor (Pan) CUDC-907 Preclinical - Cell line xenograft Actionable In a preclinical study, CUDC-907 inhibited cell growth in a human non-Hodgkin lymphoma cell line in culture, and stabilized tumor growth in xenograft models (PMID: 22693356). 22693356
Unknown unknown hematologic cancer not applicable PI3K Inhibitor (Pan) CUDC-907 Phase I Actionable In a Phase I trial, CUDC-907 treatment was well-tolerated, demonstrated safety, and resulted in stable disease in 57% (21/37) of patients with a hematological cancer (PMID: 27049457). 27049457
Unknown unknown diffuse large B-cell lymphoma not applicable PI3K Inhibitor (Pan) CUDC-907 Phase I Actionable In a Phase I trial, CUDC-907 was well-tolerated, demonstrated safety, and resulted in a 56% (5/9) objective response rate, including two complete responses and three partial responses, in patients with diffuse large B-cell lymphoma (PMID: 27049457). 27049457
Unknown unknown pediatric ependymoma not applicable PI3K Inhibitor (Pan) CUDC-907 Phase I Actionable In a Phase I trial, CUDC-907 treatment resulted in stable disease for 6 treatment cycles in a pediatric patient with ependymoma (Journal of Clinical Oncology 36, no. 15_suppl (May 20 2018) 10542-10542; NCT02909777). detail...
Unknown unknown prostate cancer not applicable PI3K Inhibitor (Pan) Abiraterone + PX-866 Phase II Actionable In a Phase II trial, Sonolisib (PX-866) and Zytiga (abiraterone) combination treatment was tolerated, resulted in stable disease as best response in 54.5% (6/11) of evaluable patients with recurrent or metastatic castration-resistant prostate cancer who demonstrated early Zytiga (abiraterone) resistance, no PSA response (0/25) was observed, and 24% (6/25) of all patients were progression-free at 12 weeks (PMID: 31056399). 31056399
Unknown unknown prostate cancer not applicable PI3K Inhibitor (Pan) PX-866 Phase II Actionable In a Phase II trial, Sonolisib (PX-866) treatment was tolerated, resulted in a partial response in 11% (2/18) and stable disease in 56% (10/18) of evaluable patients with recurrent or metastatic castration-resistant prostate cancer, PSA response was observed in 2% (1/43) of evaluable patients, and 32.6% (14/43) of all patients were progression-free at 12 weeks (PMID: 31056399). 31056399
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) PX-866 + Sorafenib Preclinical Actionable In a preclinical study, treatment with the combination of Stivarga (regorafenib) PK-866 resulted in increased cell death in a variety of solid tumor cell lines in culture (PMID: 23877009). 23877009
Unknown unknown prostate cancer no benefit PI3K Inhibitor (Pan) Abiraterone + Buparlisib Phase I Actionable In a Phase Ib trial, the combination of Zytiga (abiraterone) and Buparlisib (BKM120) did not demonstrate significant clinical activity in patients with castration-resistant prostate cancer, resulting in a best overall response of stable disease in 15% (3/20) patients treated at the 100 mg QD Buparlisib (BKM120) dose level, and further study of this combination is not planned (PMID: 28282611; NCT01634061). 28282611
Unknown unknown leiomyosarcoma not applicable PI3K Inhibitor (Pan) Aldoxorubicin + Buparlisib Preclinical Actionable In a preclinical study, the combination of BKM120 and doxorubicin produced an additive effect when treating leiomyosarcoma cells (PMID: 26952093). 26952093
Unknown unknown glioblastoma multiforme not applicable PI3K Inhibitor (Pan) 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 Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) Buparlisib Phase I Actionable In a Phase I trial, Buparlisib (BKM120) demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 22162589). 22162589
Unknown unknown glioblastoma multiforme no benefit PI3K Inhibitor (Pan) Buparlisib Preclinical Actionable In a preclinical study, treatment with BKM120 demonstrated a survival similar to vehicle in transgenic mouse models of glioblastoma and therefore, resulted in no benefit (PMID: 27199435). 27199435
Unknown unknown transitional cell carcinoma not applicable PI3K Inhibitor (Pan) Buparlisib Phase II Actionable In a Phase II trial, Buparlisib (BKM120) demonstrated unfavorable toxicity profile and modest activity in patients with platinum-refractory metastatic urothelial carcinoma, resulting in a partial response in 7.7% (1/13) and stable disease in 46.2% (6/13) of the patients, which did not meet the cutoff to proceed the trial (PMID: 32767682; NCT01551030). 32767682
Unknown unknown head and neck squamous cell carcinoma not applicable PI3K Inhibitor (Pan) Buparlisib Preclinical Actionable In a preclinical study, Buparlisib (BKM-120) reduced viability of head and neck squamous cell carcinoma (HNSSC) cells in culture and demonstrated anti-tumor activity in HNSSC xenograft models (PMID: 22116303). 22116303
Unknown unknown mantle cell lymphoma not applicable PI3K Inhibitor (Pan) Buparlisib + Ibrutinib Phase Ib/II Actionable In a Phase I/II trial, Buparlisib (BKM120) and Imbruvica (ibrutinib) combination treatment resulted in a best overall response rate of 88% (15/17, 11 complete response, 4 partial response) in patients with relapsed/refractory mantle cell lymphoma (J Clin Oncol 36, 2018 (suppl; abstr 7520); NCT02756247). detail...
Unknown unknown follicular lymphoma not applicable PI3K Inhibitor (Pan) 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 diffuse large B-cell lymphoma not applicable PI3K Inhibitor (Pan) 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 ovarian cancer not applicable PI3K Inhibitor (Pan) Buparlisib + Trametinib Phase Ib/II Actionable In a Phase Ib trial, ovarian cancer patients demonstrated an objective response rate of 21% (6/21), including 1 complete response and 5 partial responses, to treatment with Buparlisib (BKM120) in combination with Mekinist (trametinib) (PMID: 25500057) 25500057
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) 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 lung carcinoma not applicable PI3K Inhibitor (Pan) SF1126 Preclinical Actionable In a preclinical study, SF1126 decreased tumor immunosuppression and reduced tumor growth in syngeneic mouse lung carcinoma models (PMID: 31018997). 31018997
Unknown unknown hepatocellular carcinoma not applicable PI3K Inhibitor (Pan) SF1126 Preclinical - Cell line xenograft Actionable In a preclinical study, SF1126 inhibited proliferation of hepatocellular carcinoma cell lines in culture, and inhibited tumor growth in hepatocellular carcinoma cell line xenograft models (PMID: 27496136). 27496136
Unknown unknown hepatocellular carcinoma not applicable PI3K Inhibitor (Pan) 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 Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) Erlotinib + Pictilisib Phase I Actionable In a Phase I trial, the combination treatment of Tarceva (erlotinib) and Pictilisib (GDC-0941) in patients with advanced solid tumors resulted in toxicity, requiring dose adjustments, and led to minimal antitumor activity including two partial responses and stable disease in nineteen patients (PMID: 28798270). 28798270
Unknown unknown lung small cell carcinoma not applicable PI3K Inhibitor (Pan) Navitoclax + Pictilisib Preclinical - Pdx Actionable In a preclinical study, the combination of Navitoclax (ABT-263) and GDC-0941 delayed tumor growth in a circulating tumor cell (CTC)-derived xenograft model derived using CTCs from a small cell lung cancer patient (PMID: 27197306). 27197306
Unknown unknown islet cell tumor not applicable PI3K Inhibitor (Pan) Pictilisib Preclinical Actionable In a preclinical study, Pictilisib (GDC-0941) inhibited AKT activation, reduced tumor burden, and increased lifespan in a transgenic mouse model of pancreatic neuroendocrine tumor (PMID: 27225693). 27225693
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) CLR457 Phase I Actionable In a Phase I trial, CLR457 treatment demonstrated limited efficacy, resulting in no confirmed responses, stable disease in 25.8% (8/31) , and non-complete response/non-progressive disease in 6.5% (2/31) of patients with advanced solid tumors with PI3K pathway activation (PMID: 30073466; NCT02189174). 30073466
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) CLR457 Preclinical - Pdx Actionable In a preclinical study, CLR457 treatment of a variety of solid tumor patient-derived xenograft models decreased tumor volume and produced a 54% response (PMID: 26479923). 26479923
Unknown unknown pancreatic cancer not applicable PI3K Inhibitor (Pan) SF2523 Preclinical Actionable In a preclinical study, SF2523 decreased tumor immunosuppression and inhibited tumor growth in orthotopic mouse pancreatic cancer models (PMID: 31018997). 31018997
Unknown unknown breast cancer not applicable PI3K Inhibitor (Pan) SF2523 Preclinical Actionable In a preclinical study, SF2523 decreased tumor immunosuppression, and inhibited tumor growth and progression in a mouse breast cancer model (PMID: 31018997). 31018997
Unknown unknown melanoma not applicable PI3K Inhibitor (Pan) SF2523 Preclinical Actionable In a preclinical study, SF2523 reduced tumor growth and decreased lung metastasis in syngeneic mouse melanoma models (PMID: 31018997). 31018997
Unknown unknown colon adenocarcinoma not applicable PI3K Inhibitor (Pan) SF2523 Preclinical Actionable In a preclinical study, SF2523 increased T-lymphocyte infiltration and reduced tumor growth in syngeneic mouse colon adenocarcinoma models (PMID: 31018997). 31018997
Unknown unknown lung carcinoma not applicable PI3K Inhibitor (Pan) SF2523 Preclinical Actionable In a preclinical study, SF2523 decreased tumor immunosuppression, increased T-lymphocyte infiltration, and reduced tumor growth in syngeneic mouse lung carcinoma models (PMID: 31018997). 31018997
Unknown unknown Advanced Solid Tumor not applicable PI3K Inhibitor (Pan) CH5132799 Phase I Actionable In a Phase I trial, CH5132799 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (PMID: 25231405). 25231405
Unknown unknown lymphoma not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, treatment with Aliqopa (copanlisib) in patients with indolent lymphoma resulted in an objective response rate of 59% (84/142), including a complete response in 12%, and demonstrated a median duration of response of 22.6 months and a median progression-free survival of 11.2 months (PMID: 28976790, PMID: 28633365; NCT01660451). 28633365 28976790
Unknown unknown lymphoma not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in partial response in 20% (1/5) and stable disease in 40% (2/5) of patients with transformed lymphoma (PMID: 24852792). 24852792
Unknown unknown peripheral T-cell lymphoma not applicable PIK3CA inhibitor 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 follicular lymphoma not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 20% (2/10), partial response in 20% (2/10) and stable disease in 60% (6/10) of patients with follicular lymphoma (PMID: 24852792). 24852792
Unknown unknown follicular lymphoma not applicable PIK3CA inhibitor Copanlisib FDA approved Actionable In a Phase II trial that supported FDA approval, Aliqopa (copanlisib) treatment in patients with follicular lymphoma resulted in an objective tumor response rate of 58.7% (61/104) including 14.4% (15/61) patients experiencing a complete response and 44.2% (46/61) patients experiencing a partial response, stable disease in 33.7% (35/104) of patients, and a duration of response of 370 days (Journal of Clinical Oncology 35, no. 15_suppl (May 2017) 7535-7535; NCT01660451). detail... detail...
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor Copanlisib Phase I Actionable In a Phase I clinical trial, treatment with Aliqopa (copanlisib) was well-tolerated and demonstrated preliminary activity in patients with advanced solid tumors, with complete response in 2% (1/48), partial response in 4% (2/48), and stable disease in 31% (15/48) of patients (PMID: 27672108). 27672108
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor Copanlisib Phase I Actionable In a Phase I trial, Aliqopa (copanlisib) treatment resulted in no complete or partial response (0/10) and a disese control rate of 40% in patients with advanced solid tumors (PMID: 27915408). 27915408
Unknown unknown chronic lymphocytic leukemia not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in partial response in 67% (6/9) and stable disease in 22% (2/9) of patients with chronic lymphocytic leukemia (PMID: 24852792). 24852792
Unknown unknown diffuse large B-cell lymphoma not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 6% (1/17), partial response in 6% (1/17) and stable disease in 41% (7/17) of patients with diffuse large B-cell lymphoma (PMID: 24852792). 24852792
Unknown unknown mantle cell lymphoma not applicable PIK3CA inhibitor Copanlisib Phase II Actionable In a Phase II trial, Aliqopa (copanlisib) treatment resulted in complete response in 17% (1/6) and partial response in 67% (4/6) of patients with mantle cell lymphoma (PMID: 24852792). 24852792
Unknown unknown Advanced Solid Tumor no benefit PIK3CA inhibitor Copanlisib + Refametinib Phase I Actionable In a Phase I trial, Aliqopa (copanlisib) and Refametinib (BAY86-9766) combination treatment resulted in stable disease as best response in 33% (21/64) of patients with advanced solid tumors, however, the study failed to establish a tolerable and efficacious dose and schedule of this combination (PMID: 32314268; NCT01392521). 32314268
Unknown unknown breast cancer not applicable PIK3CA inhibitor CYH33 Preclinical - Cell culture Actionable In a preclinical study, CYH33 inhibited proliferation of 56% (18/32) of breast cancer cell lines in culture and demonstrated increased activity compared to Piqray (Alpelisib) (PMID: 30003928). 30003928
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor Alpelisib Phase I Actionable In a Phase I clinical trial, Alpelisib (BYL719) demonstrated safety and some efficacy in patients with advanced solid tumors (PMID: 24617631). 24617631
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor Alpelisib Phase I Actionable In a Phase I clinical trial, Alpelisib (BYL719) treatment was well tolerated in Japanese patients with advanced solid tumors and resulted in a 3% (1/33) objective response rate and a disease control rate of 57.6% (19/33, 1 partial response, 18 stable disease); in patients with PIK3CA mutations or amplification disease control rates of 75.0% (6/8) were observed vs. 78.6% (11/14) in all patients (PMID: 30588709; NCT01387321). 30588709
Unknown unknown head and neck squamous cell carcinoma not applicable PIK3CA inhibitor Alpelisib + Cetuximab + Radiotherapy Phase II Actionable In a Phase Ib trial, 11 of 11 patients with metastatic head and neck squamous cell carcinoma demonstrated no evidence of disease at 3 to 4 months following treatment with Piqray (alpelisb) in combination with Erbitux (cetuximab) and intensity modulated radiation therapy (IMRT), and 10 of 11 remained disease-free at a median follow up of 23.5 months( (range: 8.4-37.7 months) from completion of radiation (PMID: 31678634). 31678634
Unknown unknown head and neck squamous cell carcinoma not applicable PIK3CA inhibitor Alpelisib + Cisplatin + Radiotherapy Phase I Actionable In a Phase I trial, Piqray (Alpelisib) treatment in combination with Platinol (cisplatin) and radiotherapy demonstrated manageable safety, and resulted in an objective response rate of 66.7% (6/9) in head and neck squamous cell carcinoma patients, the 3-year progression-free survival (PFS) and overall survival (OS) were 77.8%, and the median progression-free survival and overall survival were not reached (PMID: 32464516; NCT02537223). 32464516
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor Alpelisib + Infigratinib Phase I Actionable In a Phase Ib trial, Infigratinib (BGJ398) and Alpelisib (BYL719) combination treatment resulted in partial response in 25% (8/32) of patients with advanced solid tumors, including urothelial, head and neck, melanoma, and anal cancer (J Clin Oncol 34, 2016 (suppl; abstr 2500)). detail...
Unknown unknown ovary epithelial cancer not applicable PIK3CA inhibitor 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 breast cancer not applicable PIK3CA inhibitor MLN1117 Phase I Actionable In a Phase I trial, treatment with MLN1117 resulted in antitumor activity, demonstrating partial responses in patients with advanced solid tumors including breast and gastric cancer (J Clin Oncol, May 2015 vol. 33 no. 15_suppl 2501). detail...
Unknown unknown islet cell tumor not applicable PIK3CA inhibitor GDC-0326 Preclinical Actionable In a preclinical study, GDC-0326 inhibited AKT activation, decreased tumor growth, metastasis, and angiogenesis and increased lifespan in a transgenic mouse model of pancreatic neuroendocrine tumor (PMID: 27225693). 27225693
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor ASN003 Phase I Actionable In a Phase I trial, ASN003 demonstrated safety and preliminary efficacy in patients with advanced solid tumors (Ann Oncol 2017, Vol 28, Suppl 5, Abstract # 371PD; NCT02961283). detail...
Unknown unknown triple-receptor negative breast cancer not applicable PIK3CA inhibitor Enzalutamide + Taselisib Phase Ib/II Actionable In a Phase Ib/II trial, the combination of Taselisib (GDC-0032) and Xtandi (enzalutamide) resulted in a clinical benefit rate of 35.7% (5/14) in patients with triple-negative breast cancer, with partial response in one patient and stable disease in 4 patients, compared to no clinical benefit with Xtandi (enzalutamide) alone, and PIK3CA mutations and AR expression did not correlate with response (PMID: 31822498; NCT02457910). 31822498
Unknown unknown Advanced Solid Tumor not applicable PIK3CA inhibitor 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...
Clinical Trial Phase Therapies Title Recruitment Status
NCT04108858 Phase Ib/II Copanlisib + Pertuzumab + Trastuzumab Pertuzumab + Trastuzumab Testing the Addition of an Anti-cancer Drug, Copanlisib, to the Usual Maintenance Treatment (Trastuzumab and Pertuzumab) After Initial Chemotherapy in a Phase Ib/II Trial for Advanced HER2 Positive Breast Cancer Recruiting
NCT03317119 Phase I Trametinib + Trifluridine-tipiracil hydrochloride Trametinib and Trifluridine and Tipiracil Hydrochloride in Treating Patients With Colon or Rectal Cancer That is Advanced, Metastatic, or Cannot Be Removed by Surgery Active, not recruiting
NCT04216472 Phase II Alpelisib + Nab-paclitaxel Nab-paclitaxel and Alpelisib for the Treatment of Anthracycline Refractory Triple Negative Breast Cancer With PIK3CA or PTEN Alterations Recruiting
NCT02761694 Phase I ARQ 751 + Paclitaxel ARQ 751 ARQ 751 + Fulvestrant ARQ 751 as a Single Agent or in Combination With Other Anti-Cancer Agents, in Solid Tumors With PIK3CA / AKT / PTEN Mutations Active, not recruiting
NCT01833169 Phase II Buparlisib BKM120 for Patients With PI3K-activated Tumors Completed
NCT02920450 Phase Ib/II Carboplatin + Gedatolisib + Paclitaxel Study of Paclitaxel, Carboplatin, and PF-05212384 in Advanced or Metastatic NSCLC (UF-STO-LUNG-002) Terminated
NCT03213678 Phase II LY3023414 PI3K/mTOR Inhibitor LY3023414 in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With TSC or PI3K/MTOR Mutations (A Pediatric MATCH Treatment Trial) Recruiting
NCT03941782 Phase 0 Alpelisib Compassionate Use of BYL 719 Alpelisib Available
NCT04317105 Phase Ib/II Copanlisib + Ipilimumab + Nivolumab Copanlisib + Nivolumab Testing the Addition of an Anti-cancer Drug, Copanlisib, to the Usual Immunotherapy (Nivolumab With or Without Ipilimumab) in Patients With Advanced Solid Cancers That Have Changes in the Following Genes: PIK3CA and PTEN Recruiting
NCT02549989 Phase II LY3023414 Study of LY3023414 for the Treatment of Recurrent or Persistent Endometrial Cancer Active, not recruiting
NCT03155620 Phase II Tazemetostat Larotrectinib LY3023414 Vemurafenib Palbociclib Olaparib Ulixertinib Erdafitinib Selumetinib Ensartinib Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial) Recruiting