Rapamune适用于治疗什么病症呢？

Rapamycin, formerly known as rapamycin (RPM), is a lipophilic triene nitrogen-containing macrolide antibiotic compound produced by Streptomyces hygroscopieus isolated from soil samples of Easier Island in the Pacific by Vezina and others at Ayerst Laboratory in Canada in 1975. It was initially used as a low-toxic antifungal drug. In 1977, it was found to have immunosuppressive effects. In 1989, RAPA began to be tried out as a new drug to treat rejection of organ transplants. Judging from the effects of animal experiments and clinical applications, it is a new immunosuppressant with good efficacy, low toxicity, and no nephrotoxicity. Compared with cyclosporine, Rapamune oral solution has a smaller dose (only 2 to 3 mg per dose), stronger anti-rejection effect, and fewer side effects. Therefore, since its launch, Rapamune has quickly become a commonly used oral immunosuppressant for organ transplant recipients around the world (especially kidney transplants). Rapamune is a serine/threonine kinase regulated by phosphoinositide-3-kinase and an mTOR (mammalian target of rapamycin) inhibitor. mTOR is a protein that controls many cellular processes, including angiogenesis and cell synthesis, making it a reasonable target for inhibiting severe vascular tumors. Recently, in multiple clinical research reports involving vascular malformations and refractory vascular tumors, Rapamune has been proven to have a significant effect in vascular and lymphoproliferative diseases.

Rapamune was approved by the FDA on May 30, 2007 for the treatment of advanced renal disease. It has now entered clinical phase 4 for the treatment of mantle cell lymphoma. Rapamune is an inhibitor of the rapamycin target protein mTOR. mTOR is a downstream protein of the PI3K/AKT pathway. This pathway is abnormally activated in a variety of tumor cells. It can control the growth of tumor cells by inhibiting the activity of mTOR and thereby inhibiting the activity of its downstream S6 ribosomal protein kinase (S6K1). In addition, rapamycin () also has its own activity and can be converted into rapamycin in the body to inhibit mTOR.