The role of new medical treatments for the management of developmental and epileptic encephalopathies: Novel concepts and results

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...

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Published inEpilepsia (Copenhagen) Vol. 62; no. 4; pp. 857 - 873
Main Authors Johannessen Landmark, Cecilie, Potschka, Heidrun, Auvin, Stéphane, Wilmshurst, Jo M., Johannessen, Svein I., Kasteleijn‐Nolst Trenité, Dorothée, Wirrell, Elaine C.
Format Journal Article
LanguageEnglish
Published United States Wiley Subscription Services, Inc 01.04.2021
Subjects
antiseizure medications
cannabidiol
Dravet syndrome
drug repurposing
Drug resistance
Epilepsy
everolimus
Fenfluramine
Lennox–Gastaut syndrome
Medical treatment
orphan drugs
Rapamycin
TOR protein
Tuberous sclerosis
cannabidiol
Lennox-Gastaut syndrome
everolimus
drug repurposing
orphan drugs
Dravet syndrome
antiseizure medications
tuberous sclerosis
fenfluramine
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Summary: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.
ISSN:0013-9580
1528-1167
DOI:10.1111/epi.16849