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It binds to its intracellular receptor FKBP12 (FK506\u2010binding protein 12), a member of the family of FK506\u2010binding proteins, and inhibits the activity of mTOR, a serine\/threonine kinase involved in numerous cell processes linked to cell growth control. The drug is currently registered for the prophylaxis of organ rejection and for use in coronary stents. However, unique characteristics of sirolimus make it a good candidate for anti\u2010cancer therapy. Indeed, phase II and III clinical studies in humans with several types of neoplasms are already under way. The review describes molecular activity of sirolimus and its analogs, characteristic for specific applications, in view of very recent advances involving tuberous sclerosis complex (TSC)\u2010mediated signaling pathways. Current studies with sirolimus performed in tuberous sclerosis animal models are presented. 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