Pharmacological Modulation of GABA Function in Autism Spectrum Disorders: A Systematic Review of Human Studies

J Autism Dev Disord. 2016 Mar;46(3):825-39. doi: 10.1007/s10803-015-2619-y.


Autism spectrum disorders are an emerging health problem worldwide, but little is known about their pathogenesis. It has been hypothesized that autism may result from an imbalance between excitatory glutamatergic and inhibitory GABAergic pathways. Commonly used medications such as valproate, acamprosate, and arbaclofen may act on the GABAergic system and be a potential treatment for people with ASD. The present systematic review aimed at evaluating the state-of-the-art of clinical trials of GABA modulators in autism. To date there is insufficient evidence to suggest the use of these drugs in autistic subjects, even if data are promising. Of note, short-term use of all the reviewed medications appears to be safe. Future well designed trials are needed to elucidate these preliminary findings.

Keywords: Autism spectrum disorder; Clinical trials; GABA; Systematic review.

Publication types

  • Review
  • Systematic Review

MeSH terms

  • Autism Spectrum Disorder / drug therapy*
  • Autism Spectrum Disorder / metabolism
  • GABA Modulators / pharmacology*
  • GABA Modulators / therapeutic use*
  • Humans
  • gamma-Aminobutyric Acid / metabolism*


  • GABA Modulators
  • gamma-Aminobutyric Acid