Development of new treatment approaches for epilepsy: unmet needs and opportunities

Epilepsia. 2013 Aug:54 Suppl 4:3-12. doi: 10.1111/epi.12294.


A working group was created to address clinical "gaps to care" as well as opportunities for development of new treatment approaches for epilepsy. The working group primarily comprised clinicians, trialists, and pharmacologists. The group identified a need for better animal models for both efficacy and tolerability, and noted that animal models for potential disease-modifying or antiepileptogenic effect should mirror conditions in human trials. For antiseizure drugs (ASDs), current animal models have not been validated with respect to their relationship to efficacy in common epilepsy syndromes. The group performed an "expert opinion" survey of perceived efficacy of the available ASDs, and identified a specific unmet need for ASDs to treat tonic-atonic and myoclonic seizures. No correlation has as yet been demonstrated between animal models of tolerability and adverse effects (AEs), versus tolerability in humans. There is a clear opportunity for improved therapies in relation to dose-related AEs. The group identified common and rare epilepsy syndromes that could represent opportunities for clinical trials. They identified opportunities for antiepileptogenic (AEG) therapies in both adults and children, acknowledging that the presence of a biomarker would substantially improve the chances of a successful trial. However, the group acknowledged that disease-modifying therapies (given after the first seizure or after the development of epilepsy) would be easier to study than AEG therapies.

Keywords: Animal models; Antiepileptogenic therapy; Antiseizure therapy; Epilepsy syndromes.

Publication types

  • Review

MeSH terms

  • Animals
  • Anticonvulsants / therapeutic use*
  • Clinical Trials as Topic
  • Disease Models, Animal
  • Dose-Response Relationship, Drug
  • Drug Discovery*
  • Drug Evaluation, Preclinical*
  • Drugs, Investigational / therapeutic use*
  • Epilepsies, Myoclonic / drug therapy
  • Epilepsy / drug therapy*
  • Epilepsy, Generalized / drug therapy
  • Epilepsy, Tonic-Clonic / drug therapy
  • Health Services Needs and Demand*
  • Humans


  • Anticonvulsants
  • Drugs, Investigational