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New research seems to show encouraging progress in the treatment of the complex neurodevelopmental disorder, autism. Two unrelated clinical trials, involving men and children with autism, suggest that the use of drugs to interact with a hormone called vasopressin could improve the social functioning of people with the disease, although an approach could to be better than the other.
Vasopressin is used by the body to maintain kidney function and regulate the blood pressure of our arteries. It is already used medically for certain types of circulation problems. However, studies in animals, mainly in rodents called voles, have shown that vasopressin in the brain also seems essential to the healthy social behaviors of these animals, including the creation of links with their companions. In humans, research has also begun to show that administering vasopressin to healthy people and to people with cognitive problems such as dementia can improve memory and social skills such as co-operation.
There have been significant improvements in children under vasopressin. And we have seen this converging evidence about parent assessments, clinician assessments, and laboratory tests of children's performance.
Karen Parker, a scientist in behavioral science at Stanford University, was very much aware of research on the social life of voles, after studying them early in her career. Since then, she has continued her work as the director of the university's social neuroscience research program. She and her team have found evidence that children living with Autism Spectrum Disorder (ASD), as it is officially known, have less circulating vasopressin in the cerebrospinal fluid than children without ASD. They also found that children with ASD with the lowest levels of vasopressin had the most severe symptoms, which may include speech and language disorders, sensory hypersensitivity, poor cognition, and IQ.
On top of that, Parker and his team decided to conduct a double-blind, randomized, controlled trial of 30 children with ASD aged 6 to 12 years. Over a period of four weeks, they either gave their subjects a nasal treatment. spray containing a form of vasopressin or a placebo.
Compared to those who received the placebo, they found that on average, children with ASD under vasopressin had significantly improved their social skills, with little or no apparent side effects. These improvements included better social communication, more accurate recognition of people's facial emotions, and better "theory of the mind" or the concept of knowing how to guess what others are feeling or thinking. The results were based not only on surveys conducted by their parents (the primary outcome of the trial), but also on physician examinations and social cognition tests.
"It's a pilot test, so it's important to recognize it," Parker told Gizmodo. "But there have been significant improvements in children with vasopressin. And we have seen this converging evidence about parent assessments, clinician assessments, and laboratory tests of children's performance. "
Parker added that this same type of test should be done with much larger groups of people and preferably with more than one research team before one of their results is considered concrete. Another factor to consider is that the diagnosis rate of ASD diagnosed in boys is much higher than in girls. Although there is some evidence that the social effects of vasopressin on ASDs should not be gender specific, her case alone can not tell us if this is really the case, given the small number of girls (5 total) studied.
Meanwhile, another double-blind, randomized, and controlled trial conducted by scientists at Roche – the Swiss-based pharmaceutical company – has revealed some interesting, but less clear, results of their attempts to use a treatment medications to improve the social functioning of adults with ASD. They gave 223 men with ASD a placebo or different doses of an experimental drug designed to block a type of brain cell receptor that binds to vasopressin for 12 weeks.
In contrast to the Parker study, the Roche trial failed to achieve its primary goal, that is, men with ASD did not improve more socially than those with placebo, according to a survey conducted by their caregivers. However, communication and socialization showed some improvement in men who had received the second highest and highest doses of the experimental drug, based on another questionnaire that clinicians gave directly to the person.
Both essays were published in Science Translational Medicine on Wednesday.
As noted by both teams (who have not previously coordinated, according to Parker) in their papers, their approaches to interacting with vasopressin in the brains of people with ASD are fundamentally opposite. But if Roche's results should probably be treated with even more skepticism than Parker's, we know that ASD is a complex disorder with no clear cause or root cause. It is therefore always possible that blocking vasopressin may help some people with a certain type of autism, while stimulating vasopressin may help others.
In any case, these trials bring us closer to something we do not have right now, which is a drug that can directly treat and manage the major symptoms of ASD. Currently, behavioral interventions can help people with ASD to better navigate socially around the world, especially at the beginning of their treatment. But the only FDA-approved drugs for ASDs are antipsychotics, which are supposed to lessen the irritability often felt by people with ASD.
Parker and her team are already considering a larger-scale trial of vasopressin nasal spray in children with ASD, she said. Previous work by his team has also shown that low levels of oxytocin may be a potential therapeutic target for some patients with ASD. She also hopes to begin testing in monkeys the theory that giving vasopressin to high-risk children early in life may not only reduce their potential symptoms of ASD, but may even prevent it. Roche also conducts his own trial of the same experimental drug in children aged 5 to 17 years.
However, discussing potential treatments for ASD has its own set of ethical issues. Many members of the autism community have begun to denounce the fact that ASDs are considered an inherently harmful condition that must be cured or prevented. some also argue that more emphasis should be placed on the destigmatization of ASDs rather than on treatment. Parker said she was sensitive to these arguments, although she feels compelled to do what she can for people with ASD who say they need help.
"I respect that movement a lot," she said. "What I am going to say is that parents and children can come to the clinic and say they are suffering. And clinicians have a duty to help them alleviate this suffering. "
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