Current Use of mTOR Inhibitors for the Treatment of Subependymal Giant Cell Astrocytomas and Epilepsy in Patients with TSC
John N. van den Anker,
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.
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