Currently, the mTORC1 inhibitors, rapamycin as well as its analogues have been approved for the treatment of several types of human cancer, such as renal cell carcinoma, mantle cell lymphoma, and some TSC-related tumors, including lymphangioleiomyomatosis, angiomyolipomas, and subependymal giant cell astrocytomas 6, 16-18. The gene discussed is TSC1; the disease is lymphangioleiomyomatosis.