Study Testing First Medication To Treat Autism
NEW HAVEN, Conn. — Children and teenagers with autism face challenges every day fitting in with friends, classmates and others in the world around them.
“My perspective is that people with autism are different, just like every other human being — unique, one of a kind,” said Dr. Roger Jou, a psychiatrist at the Yale Child Study Center who treats and works with those on the autism spectrum. But they often feel lonely, unable to find others they can relate to, he said.
There are medications to address behavioral problems, such as irritability and aggression, and to treat secondary symptoms that those on the autism spectrum often deal with, including depression, anxiety, attention deficit hyperactivity disorder and mood disorders.
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But a new medication, which is being tested in a nationwide clinical trial, could become the first drug to increase the ability of young people with autism to connect with others and to overcome the other symptoms of the disorder.
“The core symptoms of autism are deficits or challenges with social interaction, communication, restricted interests and repetitive behaviors,” said Jou, Yale’s principal investigator for the trial, known as the aViation study. Yale is one of more than 30 sites nationwide taking part.
“Really it is the core symptoms that make navigating the world so difficult” for those with autism, Jou said. “Everything we do kind of relies on one’s ability to interact with others. … So anything that could help with that … could potentially lead to a benefit. The ultimate goal is better quality of life.”
People with autism may “have a tendency to be intensely interested in one or a few things to the point where it crowds out other things that are going on around you,” Jou said. That could be something such as trains or even a fascination with a fan or washing machine, he said.
Among repetitive behaviors are lining things up or flapping the hands. “Some kids like to spin around in circles,” Jou said.
The aViation study is testing whether balovaptan, developed by the Swiss pharmaceutical company Roche, will relieve these symptoms by stopping a form of the molecule vasopressin, V1a, from binding to receptors in the brain. Vasopressin is thought to influence social behavior.
“There are studies that are done in animals that show that if you block this receptor, their social behaviors will improve,” Jou said. “In early human studies, it’s been shown to lead to subtle improvements in social behavior.”
The Food and Drug Administration has designated balovaptan a breakthrough therapy, “which is kind of a big deal because it’s mainly reserved for medicines that show a certain amount of promise,” Jou said. The lack of any medication to treat the symptoms of autism “creates more of a sense of urgency,” he said.
The aViation study is a phase two study, which includes 300 high-functioning children and teens (ages 5 to 17) over 39 weeks. The participants take either of two strengths of the drug or a placebo for 24 weeks, giving them a two in three chance of getting the medicine. There are 12 visits, half at the Child Study Center and half at the youth’s home, for which the participants are paid, up to $100 for a site visit.
The study is “looking more at children who don’t have a lot of medical issues,” Jou said. “They have to be ‘high functioning’ only because we need to get feedback from people participating and their parents.”
Jou said younger children are especially needed. “It’s been harder to get the smaller kids in, but that’s also very important because we want to make sure they’re represented in the study so the results can be applied to them.”
He said the guidance in giving medications to children is “start low, go slow.”
“There’s definitely a degree of caution because somebody may not respond in the same way and they may be more or less prone to side effects,” he said.
The need for an effective medicine is great. According to the Centers for Disease Control and Prevention, about one in 68 children is diagnosed with autism spectrum disorder. Boys are 4.5 times more likely to have autism than girls.
The FDA has approved only two medications for those with autism: risperidone and aripiprazole, which are also used for schizophrenia and bipolar disorder. “They’re used for the treatment of irritability and aggression,” Jou said.
© 2018 New Haven Register
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