Lymphangioleiomyomatosis: From molecular research to target therapy / 国际药学研究杂志

Lymphangioleiomyomatosis (LAM), which primarily affects women, is a rare lung disease characterized by diffuse cystic lesion and abdominal tumor(angiomyolipoma or lymphangioleiomyomas). It has two forms, sporadic LAM (sLAM) and tuberous sclerosis complex associated LAM (TSC-LAM). The mutations in the TSC genes on LAM cells lead to inappropriate activation of mam-malian target of sirolimns (Rapamycin) kinase (mTOR), which causes the development of LAM. The important role of mTOR pathway in the mechanism of LAM promotes the clinical use of mTOR inhibitors (such as sirolimns) in LAM patients. This article summarizes the mechanisms of LAM and reviews the clinical trials with mTOR inhibitors in LAM patients.