Current Medicinal Chemistry

Atta-ur-Rahman, FRS
Honorary Life Fellow
Kings College
University of Cambridge


Translate in Chinese

Current Use of mTOR Inhibitors for the Treatment of Subependymal Giant Cell Astrocytomas and Epilepsy in Patients with TSC

Author(s): Jasna Jancic, Vesna Duric, Nikola Ivancevic, Blazo Nikolic, John N. van den Anker, Janko Samardzic.


The serine/threonine kinase mechanistic target of rapamycin (mTOR) is an important sensor of the cellular energy condition which, at the same time, represents a kind of master switch between anabolic and catabolic cellular processes. Tuberous sclerosis complex (TSC) is a genetic disease which is considered to be a prototype of a dysregulated mTOR signaling pathway. The dysregulated mTOR pathway in TSC leads to characteristic structural and physiologic abnormalities in multiple organs. In this review we focus on the pharmacological properties of mTOR inhibitors and clinical investigations of mTOR inhibitors for two important neurological TSC manifestations: subependymal giant cell astrocytomas (SEGAs) and epilepsy. Moreover, we present a safety profile of those agents and their current role in clinical practice.

Keywords: mTOR inhibitors, TSC, SEGA, epilepsy, safety.

Order Reprints Order Eprints Rights & PermissionsPrintExport

Article Details

Year: 2016
Page: [4260 - 4269]
Pages: 10
DOI: 10.2174/0929867323666161013091144
Price: $58