Last reviewed · How we verify
Sirolimus (SIR)
Sirolimus inhibits mTOR (mammalian target of rapamycin), a key signaling protein that controls cell growth and proliferation.
Sirolimus inhibits mTOR (mammalian target of rapamycin), a key signaling protein that controls cell growth and proliferation. Used for Prevention of organ rejection in transplant recipients, Various cancers with mTOR pathway activation (investigational).
At a glance
| Generic name | Sirolimus (SIR) |
|---|---|
| Also known as | Rapamune |
| Sponsor | H. Lee Moffitt Cancer Center and Research Institute |
| Drug class | mTOR inhibitor |
| Target | mTOR (mammalian target of rapamycin) |
| Modality | Small molecule |
| Therapeutic area | Oncology; Immunology |
| Phase | FDA-approved |
Mechanism of action
Sirolimus binds to FKBP12 and forms a complex that inhibits mTOR kinase activity, suppressing cell cycle progression and reducing protein synthesis. This mechanism makes it useful as an immunosuppressant and anti-cancer agent by blocking proliferation of T cells and tumor cells. It is particularly effective in preventing organ rejection and has activity in certain cancers driven by mTOR pathway activation.
Approved indications
- Prevention of organ rejection in transplant recipients
- Various cancers with mTOR pathway activation (investigational)
Common side effects
- Immunosuppression / increased infection risk
- Hyperlipidemia
- Thrombocytopenia
- Anemia
- Mouth ulcers / stomatitis
- Diarrhea
Key clinical trials
- A Phase I/II GVHD Prevention Trial Combining Pacritinib With Sirolimus-Based Immune Suppression (PHASE1, PHASE2)
- CD40-L Blockade for Prevention of Acute Graft-Versus-Host Disease (PHASE1)
- The SIR-POBA Bypass Trial (NA)
- The SIR-POBA Shunt Trial (NA)
- Sequential Targeting of Cluster of Differentiation 52 (CD52) and Tumor Necrosis Factor (TNF) Allows Early Minimization Therapy in Kidney Transplantation (NA)
- REpurposing SirolimUS in Compensated Advanced Chronic Liver Disease. the RESUS Proof of Concept Study (PHASE2)
- Hydroxychloroquine in Combination With Sirolimus and Dexamethasone for Treating COVID-19 Patients (NA)
- Calcineurin Inhibitor-Free GVHD Prevention Regimen After Related Haplo PBSCT (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |