Sirolimus (Rapamune)

Mechanism of action

From the NCI Drug Dictionary: A natural macrocyclic lactone produced by the bacterium Streptomyces hygroscopicus, with immunosuppressant properties. In cells, sirolimus binds to the immunophilin FK Binding Protein-12 (FKBP-12) to generate an immunosuppressive complex that binds to and inhibits the activation of the mammalian target of rapamycin (mTOR), a key regulatory kinase. This results in inhibition of T lymphocyte activation and proliferation that occurs in response to antigenic and cytokine (IL-2, IL-4, and IL-15) stimulation and inhibition of antibody production.

Diseases for which it is used

• Aplastic anemia
• Autoimmune cytopenia
• Castleman disease
• Erdheim-Chester disease

Diseases for which it was used

• Classical Hodgkin lymphoma

Also known as

• Code names: AY-22989, SILA-9268A, SLM, WY-090217
• Generic names: RAPA, rapamycin
• Brand name: Rapamune