All evidence
Evidence record
Sirolimus (Rapamycin) × Tuberous Sclerosis Complex
mTOR inhibition reduces hamartoma growth in TSC1/TSC2-deficient cells.
Drug: Sirolimus (Rapamycin)Disease: Tuberous Sclerosis ComplexTarget: Mechanistic target of rapamycin
Why this drug?
Rapamycin inhibits mTOR signaling implicated in tuberous sclerosis complex (TSC) hamartoma growth, leading to approved use for TSC-associated SEGA and renal angiomyolipoma.
Known risks
- Immunosuppression
- Hyperlipidemia
- Mouth ulcers
- Impaired wound healing
Evidence behind this
Confidence score93%
Signal strength: HighLevel 1 (RCT + regulatory approval)
Evidence grade
Approved
Reduced SEGA volume and stabilized renal angiomyolipoma.
Mechanism
mTOR inhibition reduces hamartoma growth in TSC1/TSC2-deficient cells.
clinical trialfda labelPhase III
Authoritative sources
AI-generated summary
Generated March 15, 2026Last verified June 18, 2026
Everolimus and sirolimus have FDA-approved TSC indications based on Phase III trials demonstrating tumor volume reduction. Mechanistically grounded in TSC–mTOR pathway biology.
Source papers
- Bissler JJ et al., 2010
This summary is AI-assisted and human-reviewed. It does not constitute medical advice. Always consult authoritative sources and qualified professionals.