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The role of new medical treatments for the management of developmental and epileptic encephalopathies: Novel concepts and results
Author(s) -
Johannessen Landmark Cecilie,
Potschka Heidrun,
Auvin Stéphane,
Wilmshurst Jo M.,
Johannessen Svein I.,
KasteleijnNolst Trenité Dorothée,
Wirrell Elaine C.
Publication year - 2021
Publication title -
epilepsia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.687
H-Index - 191
eISSN - 1528-1167
pISSN - 0013-9580
DOI - 10.1111/epi.16849
Subject(s) - tuberous sclerosis , dravet syndrome , lennox–gastaut syndrome , context (archaeology) , medicine , everolimus , intensive care medicine , orphan drug , epilepsy , neuropsychopharmacology , psychiatry , bioinformatics , paleontology , biology
Abstract Developmental and epileptic encephalopathies (DEEs) are among the most challenging of all epilepsies to manage, given the exceedingly frequent and often severe seizure types, pharmacoresistance to conventional antiseizure medications, and numerous comorbidities. During the past decade, efforts have focused on development of new treatment options for DEEs, with several recently approved in the United States or Europe, including cannabidiol as an orphan drug in Dravet and Lennox–Gastaut syndromes and everolimus as a possible antiepileptogenic and precision drug for tuberous sclerosis complex, with its impact on the mammalian target of rapamycin pathway. Furthermore, fenfluramine, an old drug, was repurposed as a novel therapy in the treatment of Dravet syndrome. The evolution of new insights into pathophysiological processes of various DEEs provides possibilities to investigate novel and repurposed drugs and to place them into the context of their role in future management of these patients. The purpose of this review is to provide an overview of these new medical treatment options for the DEEs and to discuss the clinical implications of these results for improved treatment.