Tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) are associated with dysfunctional mammalian target of rapamycin (mTOR) signaling, which results in increased cell growth and ribosomal S6 protein phosphorylation. TSC studies suggest mTOR inhibitors can reduce TSC tumor growth and seizure frequency, while FCD studies indicate seizure suppression. This study assessed safety of mTOR inhibitor everolimus in treatment resistant TSC and FCD patients undergoing surgical resection, and mTOR signaling and molecular pathways.

In this study, fourteen treatment resistant epilepsy patients undergoing surgical resection were treated with everolimus. After 7 days of everolimus treatment or no intervention, peripheral blood and brain tissue were collected at the time of surgical resection. Plasma was used to quantify everolimus levels, proteomics, and cytokine analyses. The dataset includes proteomics and metabolomics data. The data indicate short-term everolimus before epilepsy surgery in TSC and FCD resulted in no adverse events and reduced mTOR signaling.