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

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

Volume: 23 Issue: 37

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

Affiliation:

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

Abstract: 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.

Cite this article as:

Jancic Jasna, Duric Vesna, Ivancevic Nikola, Nikolic Blazo, van den Anker N. John and Samardzic Janko, Current Use of mTOR Inhibitors for the Treatment of Subependymal Giant Cell Astrocytomas and Epilepsy in Patients with TSC, Current Medicinal Chemistry 2016; 23 (37) . https://dx.doi.org/10.2174/0929867323666161013091144