Epilepsy: key experimental therapeutics in early clinical development.

作者: Claude Steriade , Jacqueline French , Orrin Devinsky

DOI: 10.1080/13543784.2020.1743678

关键词:

摘要: Introduction: Antiseizure medications are the mainstay of epilepsy treatment. Currently therapies not specific to etiology, and control seizures in two-thirds cases. Drugs clinical development aim bridge that gap by targeting novel receptors epileptogenesis. While currently approved antiseizure target focal or generalized epilepsies regardless newly investigational drugs also rare orphan syndrome indications, such as Lennox-Gastaut Dravet syndrome. We identified through Epilepsy Foundation pipeline tracker conference proceedings recent drug conferences (XV AEDD, XIV EILAT).Areas covered: review their targets (GABA, T-type calcium channels, 5-HT, potassium channels). discuss with unknown multiple mechanisms action (cannabinoids, carisbamate, cenobamate). Therapies potential disease-modifying effects preclinical then outlined, ranging from gene-targeted treatments (antisense oligonucleotide, gene therapy, antisense transcript regulators) genetic epilepsies, mTOR inhibitors, inflammation-targeted treatments.Expert opinion: treat well prevent epileptogenesis offer great promise. To assess disease modifying agents, we may need new trial designs. Precision medicine for restore brain health.

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