A landmark study published in the New England Journal of Medicine concludes that oxytocin, the hormone known for fostering social bonds, provides no meaningful benefits for children with autism spectrum disorder (ASD).
Research has long demonstrated oxytocin's role in boosting trust, social engagement, and stress resilience. In contrast, ASD is characterized by challenges in social interaction. Recent small-scale trials suggested intranasal oxytocin could alleviate social impairments in individuals with ASD. However, a rigorous new study challenges this optimism.
Researchers at Duke University Medical Center conducted a 24-week Phase 2 trial involving 290 children and adolescents aged 3 to 17 with ASD. Participants were randomly assigned equally to intranasal oxytocin or placebo groups, stratified by age and verbal fluency.
Social skills were assessed at baseline, midpoint, and endpoint using validated ASD tools. The primary outcome was the change from baseline on the Modified Social Withdrawal subscale of the Aberrant Behavior Checklist (13 items, scores 0-39; higher scores indicate reduced social interaction). Secondary measures included additional social function scales and a brief IQ assessment.
The findings were clear: oxytocin offered no significant benefit. While the treatment group showed a minor improvement in social interaction over placebo, it was not statistically meaningful.
“There was a lot of hope that this drug would work,” said lead author Linmarie Sikich. “All of us on the study team were extremely disappointed.”
Notably, the treatment caused no adverse effects, providing reassurance for parents. “Thousands of children with ASD were prescribed intranasal oxytocin before proper testing,” Sikich added. “Fortunately, our data confirms its safety. Unfortunately, it performs no better than placebo with daily use over months.”
Does this close the door on oxytocin for autism? Not entirely. In an accompanying editorial, UCLA neurology professor Daniel H. Geschwind notes autism's heterogeneous origins and methodological nuances, calling it premature to dismiss oxytocin as a potential treatment target for ASD.
