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mTOR Inhibitors in Children: Current Indications and Future Directions in Neurology

  • Pediatric Neurology (WE Kaufmann, Section Editor)
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Abstract

The mammalian/mechanistic target of rapamycin (mTOR) pathway is a key signaling pathway that has been implicated in genetic epilepsy syndromes, neurodegenerative diseases, and conditions associated with autism spectrum disorder and cognitive impairment. The mTOR pathway has become an exciting treatment target for these various disorders, with mTOR inhibitors such as rapamycin being studied for their potential therapeutic applications. In particular, tuberous sclerosis complex (TSC) is a genetic disorder resulting from overactivation of the mTOR pathway, and pharmacologic therapy with mTOR inhibitors has emerged as a viable treatment option for the systemic manifestations of the disease. In this review, we discuss the approved indications for mTOR inhibitors in TSC, the potential future applications of mTOR inhibitors in TSC and other neurological conditions, and the safety considerations applicable to mTOR therapy in the pediatric population.

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  1. Department of Neurology and the Hope Center for Neurological Disorders, Washington University School of Medicine, 660 South Euclid Avenue, Box 8111, St. Louis, MO, 63110, USA

    Anna Jeong & Michael Wong

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Correspondence to Michael Wong.

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Anna Jeong declares no conflict of interest.

Michael Wong is a site PI for a Novartis-sponsored clinical trial on the effects of everolimus on refractory seizures in tuberous sclerosis patients.

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This article does not contain any studies with human or animal subjects performed by any of the authors.

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This article is part of the Topical Collection on Pediatric Neurology

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Jeong, A., Wong, M. mTOR Inhibitors in Children: Current Indications and Future Directions in Neurology. Curr Neurol Neurosci Rep 16, 102 (2016). https://doi.org/10.1007/s11910-016-0708-8

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