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News

Autism experts hedge bets on hormone as treatment target

by  /  11 May 2017
Drug spray: Vasopressin improves social skills in a subset of children with autism.

© sashahaltam/iStock

Drugs that alter the activity of vasopressin, a hormone involved in social behavior, may improve social abilities in people with autism. Researchers presented these promising results from two clinical trials today at the 2017 International Meeting for Autism Research in San Francisco, California.

“I think this research has high potential, with continued investigation, to lead to the development of the first effective and personalized medication to treat the social features of autism,” says Karen Parker, assistant professor at Stanford University and lead investigator on one of the trials.

In Parker’s study, children with autism inhaled a nasal spray containing vasopressin, which boosts levels of the hormone in the body. In the other trial, men with autism took a drug that dampens the hormone’s activity in the brain.

People in both trials improved more on a test of social functioning than did those who got a placebo.

It’s a mystery how two treatments with opposite effects on vasopressin could both improve social abilities, says Bryan King, vice chair of child and adolescent psychiatry at the University of California, San Francisco, who was not involved in either study.

“It’s conceivable that there’s a subgroup of people for whom the approach is to improve function in the system, and a group that’s going to benefit from the other direction,” he says. Alternatively, the two treatments may alter social abilities through different mechanisms.

In any case, the findings are encouraging because no drugs are now available for the social difficulties seen in people with autism, a core feature of the condition. Both studies hint that the treatments are safe and effective.

Sniff test:

In Parker’s study, 15 children with autism were randomly selected to sniff vasopressin twice a day for four weeks. Another 13 children with the condition instead received a placebo spray that lacks the hormone. All of the children ranged in age from 6 to 12 years and have intelligence quotients of 50 or above.

Parker and her colleagues measured the children’s social abilities before and after treatment using the Social Responsiveness Scale (SRS), a questionnaire that parents complete. The researchers also measured blood levels of vasopressin as well as expression levels of genes encoding receptors for vasopressin and the related hormone oxytocin. They monitored the children weekly for side effects.

Children who received vasopressin improved by nearly 13 points, on average, on the SRS. Those who had the lowest vasopressin levels, the highest levels of the vasopressin receptor, or the lowest levels of the oxytocin receptor before treatment improved the most.

This finding suggests that blood tests would help researchers identify children for whom the treatment is most likely to work.

“We know that these drugs are not going to work in everyone, so I think the biomarker work has a lot of promise,” says Adam Guastella, professor of psychology at the Mind and Brain Centre in Sydney, Australia, who was not involved in the study.

Children who sniffed the placebo did not show any statistically significant improvement. The two groups did not differ in side effects, which were minor.

Drug test:

In the other study, 141 men with autism took a drug called RG7314, which blocks V1a, a brain receptor for vasopressin. Researchers led by Federico Bolognani at the Swiss drug company Roche tested three different doses of the drug: 30 men received pills containing 1.5 milligrams, 73 got 4 milligrams, and 38 got 10 milligrams. Another 72 men took a placebo pill.

All of the men, who have intelligence quotients above 70, took their assigned pills once a day for 12 weeks. Before and after treatment, caregivers rated the men’s social abilities on the SRS, the study’s primary outcome measure. The researchers also assessed the drug’s effects using a variety of secondary outcome measures, including the Vineland Adaptive Behavior Scales.

The drug seems to be safe, but none of the doses led to significant improvements in scores on the SRS. However, the intermediate and high doses significantly improved socialization and communication abilities as measured by the Vineland.

These doses boosted total scores on this test by about 4 or 5 points compared with controls. “These numbers are above the threshold for what we consider a minimum clinically important difference,” Bolognani says.

He and his colleagues are testing the same drug in children and teenagers with autism. For that study, they are using the Vineland as the primary outcome measure, instead of the SRS.

For more reports from the 2017 International Meeting for Autism Research, please click here.


  • Elaine Dolan

    Children? Male only…or were there females in the study….and how many? Also why not
    use Oxytocin?

  • C Stargazer

    General thoughts on this and other popular forms of research. I’m going to give you a few quotes from someone that rejected relationships to pursue his special interest.

    “I do not think there is any thrill that can go through the human heart like that felt by the inventor as he sees some creation of the brain unfolding to success… such emotions make a man forget food, sleep, friends, love, everything.”

    Sounds like a terrible social disorder among other things. Here’s another:

    “Be alone, that is the secret of invention; be alone, that is when ideas are born.”

    This vitally important for growth when a mind depends on intense focus and pattern recognition in order to learn. One needs time to sort through it all when one breaks things down into smaller parts and thinks in a more literal way, considering every possible literal meaning before considering the context of the present situation.

    The quotes come from Nikola Tesla, had anyone tried to fix his “deficits”, our modern electrical world would not exist. It does not mean everyone becomes an inventor, it simply means that in order to learn your mind functions like an inventor’s due to pattern recognition abilities. Granted one can stay focused on the same thing for a long time because one is observing something slightly different each time. Indeed it is a challenge to simplify complex context for Neurotypical communication and often backfires without writing a book.

    The fact of the matter is, many of us are willing to be highly social about specific topics but not in a generalized Neurotypical way. That’s assuming we haven’t been utterly stifled by attempts to fix us as children. These days entire cultures of forming from various interests, cult fandoms in particular are curious. “Binge Watching” used to be considered a deficit of some sort as well.

    Back to the the point I hope to make.

    In all the attempts to medicate me, and my own efforts to condition myself to be socially “acceptable” to Neurotypicals, All I had truly accomplished was to condition myself not to use my innate thinking and pattern recognition abilities. So much so that I did not become capable of self advocating or articulation until my 30s when I realized I was rejecting my own nature; as were my doctors. Prior to that I rarely communicated anything beyond my basic needs. That kind of attempt to conform oneself is also a major cause of social anxiety and suicidal ideation.

    If you were to offer this treatment to me today, I would likely flush it down the toilet. It is a mistake to seek to conform others to average standards of the majority even if the desire is to “help”. Respect for innate differences and individuality must come first. That seems to be a shared instinct among most humans, the desire for sameness in order to feel a sense of security.

    The topics at the UN time and time again are that basic human and civil rights are not being met, discrimination is the rule rather than the exception, that science is being used to undermine our basic dignity, suicide and unemployment rates are high.

    That is because we are not respected as we are, and are instead promised that we’ll be fixed so we can be included. How many researchers are even considering the psychological consequences of peer rejection on that scale when it comes to young minds? If you survive peer rejection in school, you then graduate to peer rejection from all of society. Many people will still be nice only because they believe one day you’ll be acceptable to them through cure or treatment, the extent of that kindness often ends when you ask those people to fight for your rights. Then there’s the conditioning to view oneself that way that is also extraordinarily stifling. Social ability also isn’t going to mean much if you already struggling with PTSD.

    Accommodations will continue to be scarce so long as the overwhelming belief is that you must conform others to accepted repetitive “One Size Fits All” Neurotypical patterns. The “support cliff” still exists because we choose to keep it around, usually motivated by constantly renewed fear and alarmism.

    I’d be curious about this study, if it wasn’t so focused on “social features” and instead looked at the entire complex individual. Does improved social ability come at too high a cost in other areas? The human brain is a complex system, if you only focus on one aspect and not all the complex interactions and balances involved you are metaphorically poking around with a stick.

    I find most research unethical regardless of intention because it does not even begin to consider psychological consequences which are required in order to respect human, civil and individual rights. I also find many forms of research use narrow statistics to attempt to define human averages that can never hope to realize greater complexity. These statistics are not being derived from innate understanding, but from external observation and very crude guesswork. I do believe medication has it’s place, but at the same time many of these problems can be resolved with greater respect and inclusion/accommodation of human diversity; without which, the end result is xenophobia.

    One more Tesla quote: “Today’s scientists have substituted mathematics for experiments, and they wander off through equation after equation, and eventually build a structure which has no relation to reality.” Meanwhile, in this context rights continue to be neglected or outright violated. Work with out differences, not against them.

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