The new findings say that oxytocin, a naturally occurring hormone that functions as a chemical messenger in the brain, did not appear to improve the condition of children with autism in developing social skills.

While the discovery is disheartening for those hoping that oxytocin could treat children with autism, it provides clarity for a medicine that has demonstrated conflicting results in smaller, less rigorous studies.

“There was a great deal of hope this drug would be effective,” says the study’s principal investigator and lead author, Linmarie Sikich, associate consulting professor in the Department of Psychiatry & Behavioral Sciences at Duke University School of Medicine.

“All of us on the study team were hugely disappointed, but oxytocin does not appear to change social function of people with autism.”

While oxytocin is normally used to induce labor, its activity in the brain has led to its investigation as a possible treatment for autism. The evidence is mixed, with several small trials indicating that it enhanced social and cognitive function in certain children with autism, while others found no benefit.

The multi-site trial was designed by Sikich and colleagues, including senior author Jeremy Veenstra-VanderWeele, M.D., of the New York State Psychiatric Institute and Columbia University, to provide the best evidence yet on whether oxytocin is a safe and effective treatment for children with Autism Spectrum Disorder.

The researchers included 290 kids ages ranged from three to seventeen years, stratifying them according to their age and the severity of their autistic symptoms. Over a 24-week period, the kids were randomly assigned to receive oxytocin or a placebo via a daily nasal spray.

The study’s objective was to determine whether the oxytocin regimen had a measurable effect on the children’s social abilities, as determined by screens and evaluations conducted at the start, midway through, and at the end of the test. Both the researchers and the children’s parents administered tests using standard autism diagnostic techniques.

While oxytocin was well digested and had few negative impacts, it demonstrated no substantial advantage as compared to a placebo in the group of children who got it.

“Thousands of children with autism spectrum disorder were prescribed intranasal oxytocin before it was adequately tested,” says Veenstra-VanderWeele.

“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.”

According to Sikich:

“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.”

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