Mechanistic Target of Rapamycin (mTOR) in Tuberous Sclerosis Complex-Associated Epilepsy

Various preclinical models provide substantial evidence for the role of mTOR inhibition in the treatment of epilepsy in individuals with tuberous sclerosis complex. Preliminary clinical studies provide supportive evidence for a role of mTOR inhibition in the management of tuberous sclerosis complex-associated epilepsy and pave the way for new randomized placebo-controlled studies. This article reviews current treatment recommendations for the management of tuberous sclerosis complex-associated epilepsy as well as the rationale and evidence to support the use of mTOR inhibitors.