mTOR Inhibitor Therapy for Epilepsy Secondary to Tuberous Sclerosis

Authors

Heather A. Ammirata, University of LynchburgFollow

Specialty

Pediatric Neurology

Advisor

Dr. Tom Colletti, DHSc, PA-C, DFAAPA

Abstract

ABSTRACT

Purpose: The purpose of this article is to review the novel use of mTOR inhibitors (everolimus, sirolimus) for the treatment of epilepsy secondary to tuberous sclerosis complex.

Method: An advanced search was conducted using PubMed and Google Scholar, combining keywords ‘tuberous sclerosis,’ ‘everolimus’ or ‘sirolimus’ or ‘rapamycin’ and ‘seizures’ or ‘epilepsy.’

Results: All studies reviewed concluded that everolimus therapy for the treatment of epilepsy secondary to tuberous sclerosis complex significantly decreases seizure frequency. At present, there is insufficient evidence to determine if rapamycin (sirolimus) is also efficacious.

Conclusion: Epilepsy secondary to tuberous sclerosis complex has historically been managed with conventional antiepileptics that decrease neuronal hyperexcitability with poor efficacy for seizure control in tuberous sclerosis complex. Unlike conventional antiepileptics, mTOR inhibitors provide a unique mechanism of action through targeted treatment of the underlying pathology of tuberous sclerosis, mTOR overactivation. As a result of recent evidence, mTOR inhibitors are increasingly being used as a novel antiepileptic class for patients with tuberous sclerosis with resultant decreased seizure frequency.

Recommended Citation

Ammirata HA. mTOR Inhibitor Therapy for Epilepsy Secondary to Tuberous Sclerosis. University of Lynchburg DMSc Doctoral Project Assignment Repository. 2021; 3(3).