Study shows everolimus reduces seizures in TSC patients

The Phase III results of treating treatment-resistant epilepsy and tuberous sclerosis compex (TSC) were published in the online edition of The Lancet this week.

The international study enrolled 366 patients with TSC and epilepsy. They ranged from age two to 65. The patients were divided randomly into three groups and assigned a placebo or one of two doses of everolimus.

"The median percentage reduction in seizure frequency was more than 29 percent for patients on a low-dose regimen of everolimus and nearly 40 percent for patients with high-dose regimen to the drug," Cincinnati Children's Hospital Medical Center, TSC Clinic founding director and senior author Dr. David Franz said. "At baseline, nearly half of patients had failed treatment with six or more previous antiepileptic drugs."

TSC is a genetic disease that is characterized by malformations and an overgrowth of cells in the brain and other organs. Everolimus is an mTOR inhibitor that blocks the mTOR pathway that is associated with the cell overgrowth. Other mTOR-associated disorders include cancers and neurological conditions, such as Alzheimer's disease, Type 2 diabetes and autism.

Approximately 1 million people are affected by TSC worldwide. The Cincinnati Children's Hospital's TSC Clinic follows more than 1,000 children and adults with TSC.