Can a Hormonal Nasal Spray Help People With Autism?
Two groups of scientists revealed last week that by altering the brain’s absorption of vasopressin — a hormone closely related to oxytocin — they produced promising improvements in the social skills of young people and adults with autism.
The independent studies were published in Science Translational Medicine and mark the first time that the hormone has been studied as an autism treatment in people. Still, experts say the results are preliminary, and parents should manage their expectations carefully until more research is done.
According to Karen Parker, Stanford University’s autism specialist and co-author of one of the papers, there are still currently no “objective laboratory-based tests to diagnose autism, and there are no drugs approved by the FDA to treat the core social features of autism.”
Parker is looking to fill that gap by examining the role vasopressin plays in the social abilities of people with autism. The disorder is often characterized by trouble interacting with others, like difficulty making eye contact and interpreting social cues.
In Parker’s study, researchers ran a placebo-controlled, double-blind experiment with 30 children, including 25 boys and five girls. Autism is more common among boys and men, and the hormone itself is believed to play a larger role in male social ability.
“We published a couple of papers last year showing that this one hormone vasopressin, which has been implicated in social functioning in mammals — in particular in male mammals — was lower in the spinal fluid of children with autism,” says Parker. “In a separate cohort, we replicated that finding and also found that the lower your vasopressin levels, the greater your symptoms’ severity.”
The Stanford researchers delivered the vasopressin during the study via a nasal spray. Administering the drug through the nose allows it to more efficiently target the brain. Parents and staff observed noticeable improvements in social behavior among the children who took the hormone compared to those who received a placebo. This study marks the first time interactions between the hormone and children with autism have been analyzed, says Parker.
In a larger test also published in Science Translational Medicine, Swedish drugmaker Roche studied the outcomes of a drug called balovaptan on 223 adult men with autism from across the United States. Instead of adding vasopressin to the brain, the drug blocks uptake of the hormone — the opposite approach from the researchers at Stanford.
Parker wasn’t involved in the balovaptan research, but the inverse approaches to analyzing vasopressin’s effects on autism suggests to her that there is likely some middle ground. It may be that a person’s lack or overabundance of the hormone could be the source of social-related symptoms, she says.
While there were no observed improvements on a common autism diagnostic tool called the Social Responsiveness Scale, the researchers at Roche did find that a group of 116 people who were given higher doses of the drug “displayed improvements in a second scale measuring socialization, adaptive behavior and daily living skills compared to the cohort treated with placebo,” according to a press release about the report. Balovaptan therapy was granted breakthrough status in January by the U.S. Food and Drug Administration (FDA), meaning the development of the drug could be sped up based on early evidence of its success.
While the results are promising, they’re still early and the effects were observed in a very controlled environment, says Parker. The researchers at Stanford understood the subjects’ blood chemistry and heart functions, and could detect when something was going wrong. “You want to make sure that when you push it into a larger number of people, that we continue to see the same positive safety profile,” says Parker. “You don’t want people rushing buying it on the internet.”
Both of these drug therapies were found to be safe and without the side effects experienced in other autism medications like increased appetite and sleepiness.
While the new studies are some of the first to test the relationship between vasopressin and autism, it’s not the first time scientists have turned to hormones as potential therapies. The effects of vasopressin on social interaction originally came to light during an investigation of monogamous prairie voles. Researchers at Florida State University in Tallahassee found that the voles’ monogamous behavior could be artificially prompted in non-monogamous vole species by administering a dose of both oxytocin and vasopressin.
Due to findings like these, oxytocin and vasopressin are often dubbed the “hug hormones” and are thought to underlie people’s social abilities. But the reality is much more complex, and multiple studies with small sample sizes have oversold findings and haven’t been replicated.
The challenges of trying to communicate preliminary findings are exacerbated by the misinformation surrounding autism online. Alison Cox, an assistant professor in the department of applied disability studies at Brock University in Ontario, agrees with Parker that there should be careful consideration of how to explain to families that promising new research is still in early stages. Parents should rely on the advice from their medical practitioners when trying to navigate claims about novel treatments for autism, she says.
Cox also points to the information about autism found on Facebook or “clickbait”-y articles as making the problem worse. “I would argue [the topic of autism] could be one of the most vulnerable to the spread of misinformation, because a lot of these families find themselves in very difficult situations with less than optimal resources,” she says.
Cox says she teaches her students how to critically review the sources of new research by analyzing previous efforts or the sample size of trials. “When it comes to parents, they don’t have time to do that,” she says. “They don’t have time to review, and many of them don’t have the expertise or the training to do that either.”
“The best thing a parent can do when it comes to something that’s sensationalized is to bring it up with their physician, behavior analyst, or psychologist,” Cox adds. “Someone who’s at least got some training to be able to tease apart, ‘Well, is this something to be excited about or is this very early stages?’”
Research on the impact of hormones like vasopressin on autism will continue. The researchers at Stanford plan to test vasopressin in 100 more children with autism to see if the findings can be repeated.
Ultimately, Cox says, families should follow the progress but proceed with caution. “It’s just so easy to go full-bore into something that’s not yet established because the claims can be so enticing,” she says.
Can a Hormonal Nasal Spray Help People With Autism?
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