mTORC1 inhibitors, sirolimus (rapamycin) and everolimus (RAD001), have been approved by FDA for the treatment of TSC-associated subependymal Giant cell astrocytoma in brain (everolimus) [2, 35], renal angiomyolipoma (everolimus) [36], and pulmonary lymphangioleiomyomatosis (sirolimus) [10]. The gene discussed is TSC1; the disease is subependymal giant cell astrocytoma.