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Profile Name FBXW7 W425R
Gene Variant Detail

FBXW7 W425R (loss of function)

Relevant Treatment Approaches 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 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 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 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 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 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 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 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 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 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 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 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 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 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 papillary renal cell carcinoma not applicable mTORC1 Inhibitor Bevacizumab + Everolimus Phase II Actionable In a Phase II trial, the combination of Afinitor (everolimus) and Avastin (bevacizumab) in patients with either papillary renal cell carcinoma (RCC) (n=13), unclassified RCC with papillary features (n=23), or translocation-associated RCC with papillary features (n=1) resulted in a 6-month progression-free survival (PFS) rate of 78% (29/37), median PFS of 13.7 months, median overall survival of 33.9 months, and objective response rate of 35% (13/37, all partial responses) (PMID: 32975815; NCT01399918). 32975815
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 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 Advanced Solid Tumor no benefit 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 malignant astrocytoma not applicable 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 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 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 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 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 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 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 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 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 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 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 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 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 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 high grade 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 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 cell lines in culture, including antiestrogen-resistant cells, and inhibited tumor growth in an orthotopic breast cancer cell line xenograft model (PMID: 31879363). 31879363
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 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 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 FDA approved Actionable In a Phase II clinical trial that supported FDA approval, treatment with the combination of Lenvima (lenvatinib) and Afinitor (everolimus) resulted in a prolonged median progression-free survival of 14.6 months, compared to 5.5 months for Afinitor (everolimus) alone in patients with metastatic renal cell carcinoma who had progressed after one previous VEGF-targeted therapy (PMID: 26482279; NCT01136733). 26482279 detail...
Unknown unknown renal cell carcinoma not applicable 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 endometrial cancer not applicable 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 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 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 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 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 Hodgkin's lymphoma not applicable mTORC1 Inhibitor Everolimus + Vorinostat Phase I Actionable In a Phase I trial, the combination of Afinitor (everolimus) and Zolinza (vorinostat) demonstrated safety and resulted in an objective response rate of 33% (6/18, 2 complete, 4 partial responses) and a median progression-free survival (mPFS) of 4.8 months in patients with relapsed or refractory Hodgkin's lymphoma, with no difference in mPFS between patients who responded to treatment compared to those who did not (5.7 months vs 4.8 months; p=0.9) (PMID: 33055173; NCT01087554). 33055173
Unknown unknown glioblastoma 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 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 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 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 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 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 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 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 Advanced Solid Tumor not applicable mTORC1 Inhibitor Melanin + Phenytoin + Sirolimus + SM88 Phase I Actionable In a Phase I trial, SMK therapy (sirolimus, phenytoin, and sirolimus with SM-88) demonstrated safety in advanced metastatic cancer patients and resulted in complete response (CR) in four and partial response (PR) in six patients, including three CR and three PR among breast cancer patients (n=14), and stable disease in 17 patients for a clinical benefit rate of 90% (27/30), and a median progression-free survival of 13 months and median overall survival of 29.8 months (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 (sirolimus, phenytoin, and sirolimus with SM-88) resulted in complete response in three of 14 breast cancer patients (all CR in patients with ESR1-positive, ERBB2 (HER2)-negative breast cancer), and partial response in three patients (PMID: 30929156). 30929156
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 following treatment with SMK therapy (sirolimus, phenytoin, and sirolimus with SM-88) (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 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 Hodgkin's lymphoma not applicable mTORC1 Inhibitor Sirolimus + Vorinostat Phase I Actionable In a Phase I trial, the combination of Rapamune (sirolimus) and Zolinza (vorinostat) demonstrated safety and resulted in an objective response rate of 55% (12/22, 6 complete, 6 partial responses) and a median progression-free survival (mPFS) of 5.8 months in patients with relapsed or refractory Hodgkin's lymphoma, and responding patients demonstrated longer mPFS (18 months vs 3.2 months; p=0.019) compared to patients who did not respond to treatment (PMID: 33055173; NCT01087554). 33055173
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 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
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