Recently, mTOR inhibitors (mTORi) such as sirolimus and everolimus have been shown to be effective in the treatment of the TSC-associated tumors in the brain (subependymal giant cell astrocytoma [SEGA]), kidney (angiomyolipoma [AML] and lymphangioleiomyomatosis), and cardiac rhabdomyoma [7–10]. This evidence concerns the gene MTOR and subependymal giant cell astrocytoma.