title: Current Use of mTOR Inhibitors for the Treatment of Subependymal Giant Cell Astrocytomas and Epilepsy in Patients with TSC.
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.
authors.forename: Jasna
authors.initials: J
authors.lastname: Jancic
authors.forename: Vesna
authors.initials: V
authors.lastname: Duric
authors.forename: Nikola
authors.initials: N
authors.lastname: Ivancevic
authors.forename: Blazo
authors.initials: B
authors.lastname: Nikolic
authors.forename: John N
authors.initials: JN
authors.lastname: van den Anker
authors.affiliation: Institute of Pharmacology, Clinical Pharmacology and Toxicology, Medical Faculty, University of Belgrade, Dr Subotica 1, 11129 Belgrade, Serbia.
authors.forename: Janko
authors.initials: J
authors.lastname: Samardzic
mesh_terms: D001254:Astrocytoma / Q000150:complications / Q000188:drug therapy*; D002986:Clinical Trials as Topic; D004827:Epilepsy / Q000150:complications / Q000188:drug therapy*; D000068338:Everolimus / Q000627:therapeutic use; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D058570:TOR Serine-Threonine Kinases / Q000037:antagonists & inhibitors / Q000378:metabolism*; D014402:Tuberous Sclerosis / Q000150:complications / Q000235:genetics / Q000473:pathology*
chemical_list: D007166:Immunosuppressive Agents; D000068338:Everolimus; C546842:MTOR protein, human; D058570:TOR Serine-Threonine Kinases