Oxytocin Improves Social Abilities in Some Kids With Autism

July 14, 2017

STANFORD, Calif -- July 14, 2017 -- Children with autism showed improved social behaviour when treated with oxytocin, according to a study published online in the Proceedings of the National Academy of Sciences (PNAS).

Children with low oxytocin benefited most from the medication. The study is the first to consider how baseline oxytocin levels influence autistic children's responses to the substance.

“Our results suggest that some children with autism will benefit from oxytocin treatment more than others, and that blood oxytocin levels might be a biological sign that will allow us to predict if a child will respond maximally or not,” said lead author Karen Parker, PhD, Stanford University Medical Center, Stanford, California.

“We are finally narrowing down whom oxytocin could be beneficial for,” added senior author Antonio Hardan, MD, Stanford University Medical Center. “This is what precision health looks like for autism.”

Although the effect of oxytocin was modest, the results are exciting because no other medications now exist to treat any of the core features of autism, he said.

In 2014, Dr. Parker and colleagues discovered that oxytocin levels vary greatly in children both with and without autism, and that those with low oxytocin have more social impairment regardless of whether they have autism.

That discovery made the researchers wonder if oxytocin's benefits as an autism therapy might be confined to kids whose levels were low to begin with. Other trials of oxytocin in autism have produced mixed results but did not take subjects' baseline levels into account.

The current study included 32 children with autism who were randomised to receive an intranasal oxytocin spray or a placebo spray twice daily for 4 weeks. The children's blood oxytocin levels were measured before and after the 4-week period. The children’s behaviour was assessed at the beginning and end of the trial using a standardised questionnaire completed by their parents.

As in many trials, the researchers saw some improvement even in children given the placebo, though the effect was less pronounced than it was in the oxytocin group.

Children who had low oxytocin at baseline received more benefit from placebo than those who began with high oxytocin -- and their bodies’ own production of the hormone rose modestly. This unexpected finding suggests a possible biological explanation for the placebo effect, which is common in studies of psychological and psychiatric treatments, noted Dr. Parker.

The idea that increases in natural oxytocin production might explain how patients benefit from a placebo merits future research, she added.

Among the children who got oxytocin, those with the lowest oxytocin levels at the beginning of the trial experienced the greatest improvements in social behaviour. Oxytocin’s effects were specific: the hormone did not change the frequency of repetitive behaviours, nor did it affect children’s anxiety levels.

A large trial of oxytocin for children with autism is now underway at several institutions across the United States.

“If our findings are replicated in the large NIH [National Institutes of Health]-funded trial, then I might consider doing baseline oxytocin measurements as part of my clinical practice to try to determine if specific patients will respond,” said Dr. Hardan.

He noted that blood oxytocin levels are not measured routinely in clinical labs. He also cautioned that oral or sublingual administration of oxytocin would not necessarily produce the same results as the intranasal oxytocin tested.

“Hopefully, this is a first step to identifying the characteristics of people with autism who respond to specific treatments,” said Dr. Hardan. “Because of the heterogeneity of the disorder, we need to start doing clinical trials not to see if there will be a response, but more to see who will respond to possible treatments.”

The hormone was found to be safe, with no adverse events reported.

SOURCE: Stanford University Medical Center

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