Sirolimus (Rapamune)
Other Medications · Approved since 1999
Description
Sirolimus is an mTOR (mechanistic target of rapamycin) inhibitor that has been evaluated in colorectal cancer as both monotherapy and in combination with other agents. This immunosuppressive drug, originally developed for organ transplant rejection prevention, has shown potential anticancer activity due to its ability to inhibit cell growth and proliferation pathways. In CRC trials, sirolimus has been particularly studied in patients with specific mutations including KRAS alterations and in hereditary conditions like familial adenomatous polyposis (FAP). The drug has been combined with conventional chemotherapy regimens and other targeted agents in treatment-resistant metastatic colorectal cancer.
Mechanism of Action
Sirolimus binds to the intracellular protein FKBP12, forming a complex that inhibits the mTOR kinase, specifically mTORC1. This inhibition blocks key cellular processes including protein synthesis, cell cycle progression, and angiogenesis by preventing phosphorylation of downstream targets like S6K1 and 4E-BP1. The mTOR pathway is frequently dysregulated in colorectal cancer, making it an attractive therapeutic target for tumors with activated PI3K/AKT/mTOR signaling.
Molecular Targets
Side Effects
Not all side effects are listed. Side effects vary by individual. Always consult your oncologist.
Clinical Trials
Quick Info
| Brand Name | Rapamune |
| Category | Other Medications |
 Status | Off-label |
 EMA Status | EMA Approved |
 Spain Access | Hospital use off-label in CRC |
| Administration | Oral |
| Year Approved | 1999 |
| Clinical Trials | 10 |
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Medical Disclaimer
This information is for educational purposes only and should not replace professional medical advice. Always consult your healthcare team before making treatment decisions.