The path from scientific discovery to cures for epilepsy

Neuropharmacology. 2020 May 1:167:107702. doi: 10.1016/j.neuropharm.2019.107702. Epub 2019 Jul 10.


The epilepsies are a complex group of disorders that can be caused by a myriad of genetic and acquired factors. As such, identifying interventions that will prevent development of epilepsy, as well as cure the disorder once established, will require a multifaceted approach. Here we discuss the progress in scientific discovery propelling us towards this goal, including identification of genetic risk factors and big data approaches that integrate clinical and molecular 'omics' datasets to identify common pathophysiological signatures and biomarkers. We discuss the many animal and cellular models of epilepsy, what they have taught us about pathophysiology, and the cutting edge cellular, optogenetic, chemogenetic and anti-seizure drug screening approaches that are being used to find new cures in these models. Finally, we reflect on the work that still needs to be done towards identify at-risk individuals early, targeting and stopping epileptogenesis, and optimizing promising treatment approaches. Ultimately, developing and implementing cures for epilepsy will require a coordinated and immense effort from clinicians and basic scientists, as well as industry, and should always be guided by the needs of individuals affected by epilepsy and their families. 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: Animal model; Big data; Biomarker; Cellular model; Cure; Drug-screening; Epigenetics; Epilepsy; Epileptogenesis; Genetics; Optogenetics.

Publication types

  • Review

MeSH terms

  • Animals
  • Anticonvulsants / pharmacology
  • Anticonvulsants / therapeutic use*
  • Cell- and Tissue-Based Therapy / methods*
  • Cell- and Tissue-Based Therapy / trends
  • Drug Discovery / methods*
  • Drug Discovery / trends
  • Drug Evaluation, Preclinical / methods
  • Epigenesis, Genetic / drug effects
  • Epigenesis, Genetic / physiology
  • Epilepsy / diagnosis
  • Epilepsy / genetics
  • Epilepsy / therapy*
  • Genetic Therapy / methods*
  • Genetic Therapy / trends
  • Humans


  • Anticonvulsants