Everolimus (RAD001), a mTOR inhibitor, was initially used as an 
immunosuppressant in organ transplant patients; however, it also has significant 
antineoplastic properties. In patients with subependymal giant cell astrocytomas 
(SEGAs) associated with tuberous sclerosis complex who are not candidates for 
surgery, single-agent everolimus has demonstrated the ability to significantly 
reduce SEGA volume with good tolerability. In the Phase III, randomized, 
placebo-controlled trial, everolimus was associated with a SEGA response rate of 
35% compared with 0% in the placebo group. The most common adverse events in 
clinical trials were stomatitis/mouth ulceration and upper respiratory tract 
infections, and most adverse events were grade 1 or 2; grade 4 events were rare.