Oxytocin shows no benefit as autism treatment

Oxytocin, a naturally occurring hormone that acts as a chemical messenger in the brain, has shown no evidence of helping children with autism gain social skills, according to a large study published in The New England Journal of Medicine. The long-awaited finding provides clarity for a drug that has shown mixed outcomes in smaller, less robust studies.

Oxytocin is typically used to induce labour, but because of its activity in the brain, it has been investigated as a treatment for autism. Evidence has been conflicting, with several smaller studies suggesting it improved social and cognitive function among some children with autism, while other studies showed no benefit.

US researchers, led by principal investigator Dr Linmarie Sikich from Duke University, designed a multisite trial to provide the best evidence yet about whether oxytocin was a safe and effective treatment for children with autism spectrum disorder. The research team enrolled 290 children ages 3–17, stratified by age and the severity of their autism symptoms. The children were randomised in similar, equal-sized groups to receive oxytocin or a placebo via a daily nasal spray over 24 weeks.

The study aimed to see if the regimen of oxytocin would have a measurable impact on the children’s social abilities based on screenings and assessments at the start of the trial, midway through and at the end. Both researchers and the children’s parents provided assessments using standard analytic tools for autism.

While the oxytocin was well tolerated and had few side effects, it showed no significant benefit among the group of children who received it compared to those who received the placebo.

“Thousands of children with autism spectrum disorder were prescribed intranasal oxytocin before it was adequately tested,” said senior author Dr Jeremy Veenstra-VanderWeele, from New York State Psychiatric Institute and Columbia University. “Thankfully, our data show that it is safe. Unfortunately, it is no better than placebo when used daily for months. These results indicate that clinicians and families should insist that there is strong evidence for the safety and benefit of new treatments before they are provided to patients in the clinic.”

Dr Sikich said there was “a great deal of hope” among the study that oxytocin would be effective, and that they were “hugely disappointed” that it did not appear to change the social function of people with autism. Given these negative findings, she said no further study into oxytocin is likely.

“Our consensus as investigators is that there is no evidence in this large study that is strong enough to justify more investigation of oxytocin as a treatment for autism spectrum disorders,” she said.

Image credit: ©stock.adobe.com/au/dubova

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