Therapy Detail

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Therapy Name Sirolimus
Synonyms
Therapy Description

Rapamune (sirolimus) binds to the FKBP-12 to generate an immunosuppressive complex that binds and allosterically inhibits mTOR (PMID: 25261369). Rapamune (sirolimus) is FDA approved for the prevention of renal transplant rejection and for patients with lymphangioleiomyomatosis (FDA.gov).

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Drug Name Trade Name Synonyms Drug Classes Drug Description
Sirolimus Rapamune Rapamycin mTORC1 Inhibitor 9 Rapamune (sirolimus) binds to the FKBP-12 to generate an immunosuppressive complex that binds and allosterically inhibits mTOR (PMID: 25261369). Rapamune (sirolimus) is FDA approved for the prevention of renal transplant rejection and for patients with lymphangioleiomyomatosis (FDA.gov).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
PIK3CA H1047R lung adenocarcinoma no benefit Sirolimus Preclinical Actionable In a preclinical study, Sirolimus (rapamycin) failed to inhibit tumor growth in a mouse lung adenocarcinoma model expressing the PIK3CA H1047R mutation (PMID: 19029981). 19029981
RB1 loss neuroendocrine tumor sensitive Sirolimus Preclinical Actionable In a preclinical study, Sirolimus (rapamycin) slowed pituitary tumors and decreased the occurrence of thyroid tumors in Rb1+/- mice (PMID: 23454836). 23454836
PIK3CA H1047R lung cancer no benefit Sirolimus Preclinical Actionable In a preclinical study, Sirolimus (rapamycin) did not induce tumor shrinkage in mouse lung cancer models carrying PIK3CA H1047R (PMID: 19029981). 19029981
FBXW7 E192A fibrolamellar carcinoma predicted - sensitive Sirolimus Phase I Actionable In a Phase I study, Rapamune (sirolimus) resulted in prolonged stable disease in a patient with hepatocellular fibrolamellar carcinoma harboring FBXW7 E192A (PMID: 24586741). 24586741
PIK3CA C420R Advanced Solid Tumor sensitive Sirolimus Preclinical Actionable In a preclinical study, cells expressing PIK3CA C420R were sensitive to Rapamune (sirolimus) in culture (PMID: 17376864). 17376864
PIK3CA I391M uterine cancer predicted - sensitive Sirolimus Case Reports/Case Series Actionable In a clinical case study, a uterine cancer patient harboring PIK3CA I391M demonstrated stable disease when treated with Rapamune (sirolimus) (PMID: 28514312). 28514312
RB1 loss retinoblastoma sensitive Sirolimus Preclinical Actionable In a preclinical study, Sirolimus (rapamycin) decreased tumor occurrence, tumor hypoxia and tumor vascularization in a retinoblastoma mouse model with functionally inactivated Rb protein (PMID: 21468343, PMID: 1689463). 21468343 1689463
FBXW7 loss colon cancer sensitive Sirolimus Preclinical Actionable In a preclinical study, Rapamune (sirolimus) inhibited epithelial-mesenchymal transition, motility, and invasiveness in colon cancer cells lacking FBXW7 in culture (PMID: 23558291) 23558291
Unknown unknown head and neck squamous cell carcinoma not applicable Sirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Rapamune (sirolimus) decreased tumor growth and increased survival of cell line xenograft models of head and neck squamous cell carcinoma (PMID: 21520111). 21520111
PTEN loss osteosarcoma sensitive Sirolimus Preclinical - Pdx Actionable In a preclinical study, a patient derived xenograft (PDX) model of osteosarcoma with biallelic PTEN loss was sensitive to treatment with Rapamune (sirolimus), demonstrating reduced tumor growth and increased apoptotic activity (PMID: 30266815). 30266815
PIK3CA E545K PTEN loss stomach cancer unknown Sirolimus Phase 0 Actionable In a pilot clinical trial, Rapamune (sirolimus) demonstrated modest clinical activity in patients with PIK3CA-mutant gastric cancer (n=3) or hilar cholangiocarcinoma (n=1), including 2 gastric cancer patients harboring PIK3CA E545K and PTEN loss, with a 0% response rate, median progression-free survival of 1.9 months, and median overall survival of 3.6 months (PMID: 28685070). 28685070
PIK3CA M1043V Advanced Solid Tumor sensitive Sirolimus Preclinical Actionable In a preclinical study, Rapamune (sirolimus) inhibited proliferation in cultured cells expressing PIK3CA M1043V (PMID: 17376864). 17376864
PIK3CA mutant Erdheim-Chester disease sensitive Sirolimus Guideline Actionable Rapamune (sirolimus) is included in guidelines as first-line or subsequent-line therapy for patients with Erdheim-Chester disease harboring PIK3CA mutations (NCCN Guidelines). detail...
PIK3CA E545K Advanced Solid Tumor sensitive Sirolimus Preclinical Actionable In a preclinical study, Rapamune (sirolimus) demonstrated efficacy in inhibiting transformation of cultured cells containing PIK3CA E545K mutations (PMID: 15647370). 15647370
APC inact mut colon cancer sensitive Sirolimus Preclinical Actionable In preclinical trials, Sirolimus (rapamycin) was shown effective in reducing tumors in mouse models of familial adenomatous polyposis and colon cancer with APC deficient tumors (PMID: 18768809, PMID: 20080688). 18768809 20080688
PIK3CA E545K Klatskin's tumor unknown Sirolimus Phase 0 Actionable In a pilot clinical trial, Rapamune (sirolimus) demonstrated modest clinical activity in patients with PIK3CA-mutant gastric cancer (n=3) or hilar cholangiocarcinoma (n=1; harboring PIK3CA E545K), with a 0% response rate, median progression-free survival of 1.9 months, and median overall survival of 3.6 months (PMID: 28685070). 28685070
Unknown unknown Advanced Solid Tumor not applicable Sirolimus Preclinical Actionable In preclinical studies, Rapamune (sirolimus) induced apoptosis of cancer cells and increased sensitivity to Platinol (cisplatin) (PMID: 15136596). 15136596
FBXW7 inact mut breast cancer sensitive Sirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, breast cancer cells harboring a FBXW7 mutation demonstrated sensitivity to Rapamune (sirolimus) in culture and in cell line xenograft models (PMID: 18787170). 18787170
PIK3CA H1047R Advanced Solid Tumor sensitive Sirolimus Preclinical Actionable In a preclinical study, transformed cells expressing PIK3CA H1047R were sensitive to Rapamune (sirolimus), resulting in inhibition of transformation in culture (PMID: 15647370). 15647370
PIK3CA E542K PTEN loss stomach cancer unknown Sirolimus Phase 0 Actionable In a pilot clinical trial, Rapamune (sirolimus) demonstrated modest clinical activity in patients with PIK3CA-mutant gastric cancer (n=3) or hilar cholangiocarcinoma (n=1), including a gastric cancer patient harboring PIK3CA E542K and PTEN loss, with a 0% response rate, median progression-free survival of 1.9 months, and median overall survival of 3.6 months (PMID: 28685070). 28685070
PIK3CA E542K Advanced Solid Tumor sensitive Sirolimus Preclinical Actionable In a preclinical study, Rapamune (sirolimus) demonstrated efficacy in inhibiting transformation of cultured cells containing PIK3CA E542K mutations (PMID: 15647370). 15647370
Unknown unknown Indication other than cancer not applicable 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...

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT02722668 Phase II Cyclophosphamide + Fludarabine + Mycophenolate mofetil Sirolimus anti-thymocyte globulin UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep Recruiting USA 0
NCT01522820 Phase I Rasdegafusp alfa Sirolimus Vaccine Therapy With or Without Sirolimus in Treating Patients With NY-ESO-1 Expressing Solid Tumors Completed USA 0
NCT01658007 Phase I Sirolimus Pilot Study Of Sirolimus Plus Multiagent Chemotherapy For Relapsed/Refractory Acute Lymphoblastic Leukemia/Lymphoma Terminated USA 0
NCT02756572 Phase I Sirolimus Mycophenolate mofetil Cyclosporine Cytarabine Melphalan Cladribine Filgrastim mitoxantrone hydrochloride Fludarabine Early Allogeneic Hematopoietic Cell Transplantation in Treating Patients With Relapsed or Refractory High-Grade Myelodysplastic Syndrome or Acute Myeloid Leukemia Completed USA 0
NCT00923845 Phase II Sirolimus Cyclophosphamide + Pentostatin Low-Intensity Stem Cell Transplantation With Multiple Lymphocyte Infusions to Treat Advanced Kidney Cancer Completed USA 0
NCT03826628 Phase II Sirolimus Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex Recruiting USA 8
NCT02110069 Phase II Vincristine Sulfate Sirolimus A Study to Compare Vincristine to Sirolimus for Treatment of High Risk Vascular Tumors Active, not recruiting USA 0
NCT02009332 Phase Ib/II Sirolimus Phase 1/2 Study of ABI-009 in Nonmuscle Invasive Bladder Cancer Completed USA 0
NCT01265030 Phase Ib/II Sirolimus A Pilot Study Evaluating the Use of mTor Inhibitor Sirolimus in Children and Young Adults With Desmoid-Type Fibromatosis Active, not recruiting USA 0
NCT01853423 Phase I Sirolimus Tuberou Sclerosis Complex: Facial Angiofibroma Skin Cream Completed USA 0
NCT02790515 Phase II Sirolimus anti-thymocyte globulin Blinatumomab Tacrolimus Thiotepa Melphalan Fludarabine Cyclophosphamide Mesna Rituximab Filgrastim Provision of TCR?? T Cells and Memory T Cells Plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies Relapsed or Refractory Despite Prior Transplantation Recruiting USA 0
NCT00092222 Phase II Sirolimus Alpha 2 Interferon Cyclophosphamide + Doxorubicin + Etoposide + Prednisone + Vincristine Sulfate Rituximab Bortezomib Pegylated liposomal-doxorubicin Virotherapy and Natural History Study of KHSV-Associated Multricentric Castleman s Disease With Correlates of Disease Activity Recruiting USA 0
NCT01087554 Phase I Temsirolimus Everolimus Sirolimus Vorinostat Sirolimus or Everolimus or Temsirolimus and Vorinostat in Advanced Cancer Active, not recruiting USA 0
NCT04393454 Phase II Sirolimus Study to Evaluate the Efficacy and Safety of Sirolimus in Subjects With Metastatic, Mismatch Repair Deficient Solid Tumors After Immunotherapy Recruiting USA 0
NCT02551718 Phase I Bosutinib Irinotecan Romidepsin Busulfan Melphalan Nilotinib Crizotinib Cytarabine Mitoxantrone Dasatinib Pazopanib Paclitaxel Clofarabine Hydroxyurea Tretinoin Carfilzomib Nelarabine Bexarotene Pentostatin Everolimus Cabozantinib Mercaptopurine Methotrexate Cladribine Thioguanine Daunorubicin Ponatinib Etoposide Afatinib Gefitinib Gemcitabine Regorafenib Arsenic trioxide Trametinib Imatinib Erlotinib Dabrafenib Decitabine Axitinib Azacitidine Ruxolitinib Fludarabine Lapatinib Ceritinib Sirolimus Sorafenib Lomustine Sunitinib Cabazitaxel Temsirolimus Topotecan Bortezomib Pralatrexate High Throughput Drug Sensitivity Assay and Genomics- Guided Treatment of Patients With Relapsed or Refractory Acute Leukemia Active, not recruiting USA 0
NCT01843998 Phase II Sirolimus Therapeutic Efficacy of Topical Sirolimus in Early Stage Cutaneous T-cell Lymphoma (CTCL) Withdrawn USA 0
NCT01331135 Phase I Sirolimus Aflac ST0901 CHOANOME - Sirolimus in Solid Tumors Completed USA 0
NCT02145559 Phase I Metformin + Sirolimus Sirolimus A Pharmacodynamic Study of Sirolimus and Metformin in Patients With Advanced Solid Tumors Completed USA 0
NCT02423915 Phase Ib/II Filgrastim Rituximab Cyclophosphamide + Fludarabine + Mycophenolate mofetil Sirolimus Fucosylated T Cells for Graft Versus Host Disease (GVHD) Prevention Completed USA 0
NCT01195922 Phase Ib/II Sirolimus Rapamycin Therapy in Head and Neck Squamous Cell Carcinoma Completed USA 0
NCT02793544 Phase II Cyclophosphamide Busulfan Sirolimus Mycophenolate mofetil Mesna Fludarabine HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide Completed USA 0
NCT01163357 Phase I Sirolimus Bortezomib + Fludarabine + Melphalan Bortezomib, Total Marrow Irradiation, Fludarabine Phosphate, and Melphalan in Treating Patients Undergoing Donor Peripheral Blood Stem Cell Transplant For High-Risk Stage I or II Multiple Myeloma Active, not recruiting USA 0
NCT01791088 Phase I Sirolimus Sirolimus in Treating Patients With Solid Tumors That Are Metastatic or Cannot Be Removed By Surgery Completed USA 0
NCT03878524 Phase I Oxaliplatin Palbociclib Vemurafenib Sirolimus Tretinoin Celecoxib Ipilimumab Ruxolitinib Dasatinib Abiraterone Idelalisib Trametinib Imatinib Erlotinib Carboplatin Olaparib Panobinostat Bortezomib Afatinib Fluorouracil Vorinostat Pembrolizumab Leucovorin Enzalutamide Ponatinib Nivolumab Everolimus Sunitinib Cabazitaxel Cabozantinib Lenvatinib Pertuzumab Sorafenib Venetoclax Bevacizumab A Personalized Medicine Study for Patients With Advanced Cancer of the Breast, Prostate, Pancreas or Those With Refractory Acute Myelogenous Leukemia (SMMART) Recruiting USA 0
NCT01203722 Phase Ib/II Tacrolimus Sirolimus Cyclophosphamide + Fludarabine + Mycophenolate mofetil Reduced Intensity, Partially HLA Mismatched BMT to Treat Hematologic Malignancies Recruiting USA 0
NCT00602693 Phase I Sirolimus Allopurinol + Cyclophosphamide + Fludarabine T-Regulatory Cell Infusion Post Umbilical Cord Blood Transplant in Patients With Advanced Hematologic Cancer Completed USA 0
NCT03298958 Phase III Sirolimus Secondary Prevention Trial of Rapamycin in Patients With Resected Non-muscle Invasive Bladder Cancer Withdrawn USA 0
NCT02753309 Phase 0 Sirolimus A Study of Rapamycin Combined With Intravesical BCG in Patients With Non-muscle Invasive Bladder Cancer Completed USA 0
NCT00305682 Phase II Sirolimus Mycophenolate mofetil anti-thymocyte globulin Cyclophosphamide Fludarabine Non-Myeloablative Conditioning for Unrelated Donor Umbilical Cord Blood Transplant Completed USA 0
NCT00513474 Phase I Rasburicase Methotrexate Sirolimus Cyclosporine Tacrolimus Filgrastim Busulfan + Cyclophosphamide + Etoposide Rasburicase in Preventing Graft-Versus-Host Disease in Patients With Hematologic Cancer or Other Disease Undergoing Donor Stem Cell Transplant Completed USA 0
NCT03363763 Phase II Sirolimus Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex Recruiting USA 1
NCT01231412 Phase III Sirolimus Fludarabine Cyclosporine + Mycophenolate mofetil Graft-Versus-Host Disease Prophylaxis in Treating Patients With Hematologic Malignancies Undergoing Unrelated Donor Peripheral Blood Stem Cell Transplant Completed USA 2


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