Duke University researchers concluded that children with autism showed no benefit from an experimental therapy.
“This is really a major setback,” said Dr. Linmarie Sikich, a Duke University researcher who led the multi-site U.S. study published in the New England Journal of Medicine. “We were really hoping to find a benefit and just couldn’t see it anywhere.”
The research study, funded by the U.S. government, used a synthetic form of oxytocin, the hormone produced in the brain that prompts uterus contractions and helps mothers bond with their newborn babies.
Experimental trials in mice have suggested the hormone may improve sociability, and smaller studies have signalled that it could have comparable effects in children with autism, for who social interaction is a challenge.
The six month study involved 250 children with autism, ranging from 3 to 17 years of age. The participants received daily squirts of nasal spray containing oxytocin or an inactive ingredient for seven weeks, with the dosage gradually increasing after that. The dose could be maintained or reduced if requested.
Very minute behavior improvements were observed in both groups, but they had no significant impact, Sikich said. Research analysis showed no appreciable difference in outcome in children with mild or severe autism, she said.
Approximately 1 in 54 U.S. children have autism, according to the Centers for Disease Control and Prevention. Behavior therapy has been found to be the most successful course of treatment.
Oxytocin is a naturally occurring hormone and chemical messenger. It is most commonly best known for setting off labor and the release of breast milk in women. Synthetic oxytocin is often given to pregnant women by IV to induce childbirth.
Based on early research findings, some doctors began prescribing it for children with autism. Some advocates call it “the love hormone” and it is sold online in potions and pills as a mood and relationship enhancer.
Larry Young, an Emory University scientist who does animal research with oxytocin, said it is too early to reject it as an autism treatment. He said the hormone is better understood today than when the study began nearly eight years ago.
“It makes the social world around us more vibrant in our brain, so we pay attention to it,” Young said.
He hypothesized an instance in which a child with autism gets a squirt of oxytocin each morning and starts paying more attention to other kids on the school bus. What if the enhanced attention assists the child in realizing that the kids are participating in bullying?
“This is a very important study because it does say that just willy-nilly giving daily administrations of oxytocin is not going to lead to improvement,” Young said.
“Hopefully physicians and parents will learn from this and say this is not something we give as a vitamin” without other treatment like behavior therapy.
Joyce Galaverna’s son was 13 when he enrolled in the study in 2015. He tolerated the treatments but his behavior showed no improvement.
“The irritability and anxiety levels pretty much stayed the same throughout the study,” Galaverna said.
The North Carolina family were never told if their son had received oxytocin or the placebo.
University of Virginia autism researcher Kevin Pelphrey said other studies have indicated that giving oxytocin by nose may lead to changes in brain regions involved in social behavior. He said the study’s use of a behavior checklist to evaluate the hormone’s benefits may have restricted its conclusions.
Pelphrey believes that brain-based measures could be useful in determining which children might respond best to the hormone.
“There is still much work to be done in the area of understanding how oxytocin might be utilized to improve social function in kids with autism,” Pelphrey said.