Last reviewed · How we verify
Low-dose sirolimus
Low-dose sirolimus inhibits mTOR signaling to suppress immune cell proliferation and promote regulatory T cell differentiation.
Low-dose sirolimus inhibits mTOR signaling to suppress immune cell proliferation and promote regulatory T cell differentiation. Used for Autoimmune and inflammatory conditions (Phase 3 investigational).
At a glance
| Generic name | Low-dose sirolimus |
|---|---|
| Also known as | rapamycin |
| Sponsor | Icahn School of Medicine at Mount Sinai |
| Drug class | mTOR inhibitor |
| Target | mTOR (mechanistic target of rapamycin) |
| Modality | Small molecule |
| Therapeutic area | Immunology |
| Phase | Phase 3 |
Mechanism of action
Sirolimus is an mTOR inhibitor that blocks cell cycle progression by preventing the phosphorylation of downstream effectors. At low doses, it preferentially promotes the expansion of regulatory T cells (Tregs) while suppressing effector T cell proliferation, creating an immunomodulatory effect that may reduce autoimmune and inflammatory responses. This mechanism differs from higher-dose sirolimus used in transplantation, where broad immunosuppression is the goal.
Approved indications
- Autoimmune and inflammatory conditions (Phase 3 investigational)
Common side effects
- Hyperlipidemia
- Thrombocytopenia
- Mouth ulcers
- Infections
- Anemia
Key clinical trials
- Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies (PHASE2)
- Everolimus Trial in Laryngotracheal Stenosis (EARLY_PHASE1)
- Trametinib and Everolimus for Treatment of Pediatric and Young Adult Patients With Recurrent Gliomas (PNOC021) (PHASE1)
- Combination Gerotherapeutic Interventions for Healthspan Improvement (PHASE3)
- Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease (PHASE2)
- CALM-AF-AI: Counteracting Age-related Loss of Muscle With AAV-Follistatin Combined With Angiogenesis-Inducing VEGF Plasmid Gene Therapy (PHASE1, PHASE2)
- Sirolimus in Combination With Metronomic Chemotherapy in Children With High-Risk Solid Tumors (PHASE2)
- REgulatory T Cell Therapy to Achieve Immunosuppression REduction (PHASE2)
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 |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Low-dose sirolimus CI brief — competitive landscape report
- Low-dose sirolimus updates RSS · CI watch RSS
- Icahn School of Medicine at Mount Sinai portfolio CI