Stem cells: A path towards improved epilepsy therapies

Neuropharmacology. 2020 May 15;168:107781. doi: 10.1016/j.neuropharm.2019.107781. Epub 2019 Sep 17.


Despite the immense growth of new anti-seizure drugs (ASDs), approximately one-third of epilepsy patients remain resistant to current treatment options. Advancements in whole genome sequencing technology continues to identify an increasing number of epilepsy-associated genes at a rate that is outpacing the development of in vivo animal models. Patient-derived induced pluripotent stem cells (iPSCs) show promise in providing a platform for modeling genetic epilepsies, high throughput drug screening, and personalized medicine. This is largely due to the ease of collecting donor cells for iPSC reprogramming, and their ability to be maintained in vitro, while preserving the patient's genetic background. In this review, we summarize the current state of iPSC research in epilepsy and closely related syndromes, discuss the growing need for high-throughput drug screening (HTS), and review the use of stem cell technology for the purpose of autologous transplantation for epilepsy stem cell therapy. Although the use of iPSC technology, as it applies to ASD discovery, is in its infancy, we highlight the significant progress that has been made in phenotype and assay development to facilitate systematic HTS for personalized medicine. This article is part of the special issue entitled 'New Epilepsy Therapies for the 21st Century - From Antiseizure Drugs to Prevention, Modification and Cure of Epilepsy'.

Keywords: Anti-Seizure drugs; Drug discovery; Epilepsy; Human induced pluripotent stem cells; Personalized medicine.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Review

MeSH terms

  • Animals
  • Anticonvulsants / administration & dosage
  • Drug Discovery / methods
  • Drug Discovery / trends
  • Epilepsy / diagnosis
  • Epilepsy / genetics
  • Epilepsy / therapy*
  • Humans
  • Induced Pluripotent Stem Cells / physiology
  • Induced Pluripotent Stem Cells / transplantation*
  • Precision Medicine / methods*
  • Precision Medicine / trends
  • Stem Cell Transplantation / methods*
  • Stem Cell Transplantation / trends


  • Anticonvulsants