All targets
Target workspace
Mechanistic target of rapamycin
Central kinase integrating nutrient and growth signals.
Pathway & druggability
PI3K–AKT–mTOR
Druggability: High (rapalogs approved)
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.