Animal models for autism in 2017 and the consequential implications to drug discovery

Introduction: Autism spectrum disorder (ASD) is characterized by deficits in social communication and restricted interests/repetitive behaviors, for which there are currently no approved drug treatments. The core symptoms of ASD vary widely in severity and are often accompanied by other neuropsychiatric disorders. Drug discovery has been challenging because of the lack of understanding of the underlying pathophysiology of ASD as well as the heterogeneity of symptoms and symptom severity.

Areas covered: In this review, the author discusses animal models of ASD used as targets for drug discovery, focusing primarily on non-syndromic models, primarily rodents. They highlight the wide range of drug targets examined in animal models. While very little of this work has resulted in drug therapy for the behavioral symptoms of ASD yet, it has increased our knowledge of the biology of ASD that is critical for driving drug discovery and has already provided many new drug targets for investigation.

Expert opinion: The information gathered from the animal models of ASD is increasing our understanding of the underlying pathophysiology for ASD and is leading to better therapeutic targets. However, the issue of small sample size, heterogeneity within clinical samples, and a lack of replicable outcome measures must be addressed to move forward.