Oxytocin is a neuropeptide produced by the hypothalamus which plays an important role in facilitating social bonding. Over the last decade, there has been great interest in the use of oxytocin to improve social functioning in individuals with autism spectrum disorder (ASD). While research supporting its use in this setting has been mixed, off-label use of oxytocin occurs frequently in the treatment of autism spectrum disorders in children and adolescents. Christopher McDougle MD, Director of the Lurie Center for Autism at MGH, has been part of a multicenter team looking at the use of intranasal oxytocin for ASD in children and adolescents: the Autism Centers of Excellence network Study of Oxytocin in Autism to improve Reciprocal Social Behaviors (SOARS-B). The results of this study were published in the New England Journal of Medicine.
In this 24-week, placebo-controlled trial, 290 children and adolescents 3 to 17 years of age with autism spectrum disorder were randomly assigned in a 1:1 ratio to receive intranasal oxytocin or placebo, with a total target dose of 48 IU daily. While 24 IU twice daily was the target, flexible dosing was used to select the most efficacious and well-tolerated dose. Social functioning was measured using the Aberrant Behavior Checklist modified Social Withdrawal subscale (ABC-mSW). In addition, cognitive functioning was assessed.
After 24 weeks of daily intranasal oxytocin or placebo, the research team observed no differences between the two groups in terms of social or cognitive functioning. The incidence and severity of adverse events were similar in the two groups.
Is This the End of the Road for Oxytocin?
There has been great interest in the use of oxytocin to improve social functioning in individuals with autism spectrum disorder. While previous studies have suggested that intranasal oxytocin may improve social functioning, these studies have been small and thus underpowered. In contrast, the SOARS-B study is the best powered study conducted to date. However, this placebo-controlled trial of intranasal oxytocin in children and adolescents with ASD demonstrated no significant improvements in measures of social or cognitive functioning over a period of 24 weeks of treatment.
There is a clear unmet need for safe and effective pharmacological treatments targeting the core symptoms of ASD, including deficits in social functioning. Although the findings of this study are disappointing, in a companion piece, Daniel H. Geschwind, MD, PhD, Director of the Institute for Precision Health at the University of California, Los Angeles, argues that it may be premature to abandon oxytocin as a means of either treating or understanding the social deficits in patients with ASD.
Specifically, Dr. Geschwind notes that the effects of oxytocin differ across developmental stages and that there may be critical developmental windows during which oxytocin must be administered in order to observe its prosocial effects. In addition, oxytocin in this study was administered without standardized behavioral interventions for social skills. Without concurrent social skills training, we might not be able to fully take advantage of the prosocial effects of oxytocin. Stay tuned…
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Sikich L, Kolevzon A, King BH, McDougle CJ, et al. Intranasal Oxytocin in Children and Adolescents with Autism Spectrum Disorder. N Engl J Med. 2021;385(16):1462-1473. doi:10.1056/NEJMoa2103583
Geschwind DH. Oxytocin for Autism Spectrum Disorder – Down, but Not Out. N Engl J Med. 2021;385(16):1524-1525. doi:10.1056/NEJMe2110158
Christopher McDougle, MD
Christopher McDougle, MD is the Director of the Lurie Center for Autism and the Nancy Lurie Marks Professor in the Field of Autism at Harvard Medical School. He is an internationally recognized expert in the research and treatment of neurodevelopmental disorders that extend into adulthood. He has 25 years of experience diagnosing and caring for children, adolescents, and adults with autism spectrum disorders (ASDs).
