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Profile Name STK11 F354L
Gene Variant Detail

STK11 F354L (loss of function)

Relevant Treatment Approaches mTOR Inhibitor mTORC1 Inhibitor

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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 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 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 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 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 Advanced Solid Tumor not applicable mTOR Inhibitor 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 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 T-cell acute lymphoblastic leukemia not applicable mTOR Inhibitor 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 urinary bladder cancer not applicable mTOR Inhibitor 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 Advanced Solid Tumor not applicable mTOR Inhibitor 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 breast cancer not applicable mTOR Inhibitor 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 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 renal cell carcinoma not applicable mTOR Inhibitor 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 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 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 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 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 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 head and neck squamous cell carcinoma not applicable mTOR Inhibitor 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 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 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 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 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 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 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 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 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 lung non-small cell carcinoma not applicable mTOR Inhibitor 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 breast cancer not applicable mTOR Inhibitor 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 leukemia not applicable mTOR Inhibitor 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 Advanced Solid Tumor not applicable mTOR Inhibitor 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 prostate cancer not applicable mTOR Inhibitor 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 Advanced Solid Tumor not applicable mTOR Inhibitor 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 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 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 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 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 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 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 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 renal cell carcinoma not applicable mTOR Inhibitor GDC-0980 Phase II Actionable In a Phase II clinical trial, Apitolisib (GDC-0980) was inferior to Afinitor (everolimus) (median PFS, 3.7 months (n=42) vs. 6.1 months (n=43), respectively) and overall survival trended lower in the Apitolisib (GDC-0980) arm in patients with metastatic renal cell carcinoma (PMID: 26951309). 26951309
Unknown unknown ovarian clear cell carcinoma not applicable mTOR Inhibitor 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 malignant pleural mesothelioma not applicable mTOR Inhibitor 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 peritoneal mesothelioma not applicable mTOR Inhibitor 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 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 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 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 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 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 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 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 endometrial cancer not applicable mTOR Inhibitor Dactolisib Preclinical Actionable In a preclinical study, BEZ235 suppressed tumor growth in endometrial xenografts (PMID: 22662154). 22662154
Unknown unknown pancreatic endocrine carcinoma no benefit mTOR Inhibitor 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 prostate carcinoma not applicable mTOR Inhibitor 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 peritoneal mesothelioma not applicable mTOR Inhibitor 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 leiomyosarcoma not applicable mTOR Inhibitor 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 Advanced Solid Tumor no benefit mTOR Inhibitor mTORC1 Inhibitor 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 astrocytoma not applicable mTOR Inhibitor mTORC1 Inhibitor 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 prostate cancer not applicable mTOR Inhibitor 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 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 ovarian cancer not applicable mTOR Inhibitor 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 Advanced Solid Tumor not applicable mTOR Inhibitor 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 urinary bladder cancer not applicable mTOR Inhibitor 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 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 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 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 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 breast cancer not applicable mTOR Inhibitor 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 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 Advanced Solid Tumor not applicable mTOR Inhibitor 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 colorectal cancer not applicable mTOR Inhibitor 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 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 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 malignant glioma not applicable mTOR Inhibitor 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 + 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 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 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 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 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 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 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 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 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 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 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 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 appendix carcinoma not applicable mTORC1 Inhibitor Melanin + Phenytoin + Sirolimus + SM88 Case Reports/Case Series Actionable In a Phase I trial, a patient with appendiceal carcinoma achieved a complete response to treatment with SMK therapy (PMID: 30929156). 30929156
Unknown unknown breast cancer not applicable mTORC1 Inhibitor Melanin + Phenytoin + Sirolimus + SM88 Case Reports/Case Series Actionable In a Phase I trial, treatment with SMK therapy in breast cancer patients (n=14) resulted in a complete response in three patients (all three complete responders had ESR1-positive, ERBB2 (HER2)-negative breast cancer), and three patients who achieved a partial response (PMID: 30929156). 30929156
Unknown unknown Advanced Solid Tumor not applicable mTORC1 Inhibitor Melanin + Phenytoin + Sirolimus + SM88 Phase I Actionable In a Phase I trial, SMK therapy demonstrated safety in advanced metastatic cancer patients and resulted in a complete response in four, partial response in six, and stable disease in 17 patients for a clinical benefit rate of 90% (27/30), median progression-free survival of 13 months, median overall survival of 29.8 months, and treatment in breast cancer patients (n=14) resulted in three complete and three partial responses (PMID: 30929156). 30929156
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 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 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 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
Clinical Trial Phase Therapies Title Recruitment Status