Autism spectrum disorder (ASD) is a neurodevelopmental disorder whose core symptoms include deficits in social interaction and communication besides restricted and repetitive behaviors. Although ASD is highly prevalent, affecting 1/100 in the general population, no medication for the core symptoms has been established. Therefore, the disorder is considered a huge unmet medical need and a heavy burden on individuals with ASD, their families, and entire society. Oxytocin is expected to be a potential therapeutic resource for the social core symptoms of ASD, since this neuropeptide can modulate human social behavior and cognition. This review article provides an overview of both experimental and clinical studies on effects of oxytocin administration on behavior, neural underpinnings, and symptomatology of ASD. Although the number of studies is increasing, several issues remain for further development of clinical application of the neuropeptide. The issues include optimization of administration route, doses, treatment duration, interval of administrations, and timing of starting treatment. Additional issues involve investigating neurobiological mechanisms of treatment and developing a reliable tool to accurately and objectively assess longitudinal changes in the core symptoms of ASD. Some of these issues are discussed in this review.
Keywords: Autism; Developmental disorders; Neuroimaging; Neuropeptide; Oxytocin; Social behavior.