Article from Autism Speaks about NEW MEDICINE

 

The next step toward treatment for the core symptoms of autism

June 23, 2011 Autism Speaks

by Chief Science Officer of Autism Speaks, Geraldine Dawson, Ph.D.
I often get the question: How is the research we are funding on single gene disorders, such as Fragile X, relevant to the larger population of individuals with ASD? My answer is that, although autism has many different causes – including single gene mutations, multiple genetic factors, and even environmental factors – it is likely that these causes affect common underlying biological pathways. By studying the “simpler” single gene disorders, especially by studying animal models of these disorders, we can discover these pathways and develop medications that hopefully can help restore the functioning of these pathways.

As you will see in the press release, this strategy is being implemented by Seaside Therapeutics. With the help of funding from Autism Speaks and NIH, Mark Bear and other scientists developed an animal model for Fragile X and discovered that glutamate, an excitatory neurotransmitter, is affected by the Fragile X mutation. An overabundance of glutamate is interfering with the ability of neurons to communicate with each other (synaptic functioning). SeasideTherapeutics then tested a medicine, STX209 (arbaclofen), which helps to restore normal synaptic functioning, in a clinical trial with people with Fragile X. They found encouraging results! The next step, which was launched yesterday, is to test the efficacy of STX209 in individuals with ASD. The hope is that this medicine will improve social behavior and reduce irritability (e.g. aggression, tantrums) in people with ASD.

In the press release Randall L. Carpenter, M.D., President and Chief Executive Officer of Seaside Therapeutics says, “In our open-label Phase 2a study of STX209, we observed significant improvements in social impairment—a core symptom of autism spectrum disorders—including symptoms such as preference to be alone, being withdrawn or isolated, and lack of social reactivity. We are spearheading late-stage development of a drug candidate that has the potential to change the treatment paradigm for autism spectrum disorders—addressing core symptoms—and are truly excited about the prospect of helping patients and their families achieve an improved quality of life.”
Arbaclofen acts by stimulating the release of GABA in the brain. To make an simplified analogy, if we think of glutamate as the accelerator pedal in brain, then GABA is the brake pedal. By reducing glutamate through stimulating GABA receptors, the first clinical trial with people who have Fragile X syndrome demonstrated positive effects on behavior.

In Phase 2b of the trial, 25 sites will conduct a randomized, placebo-controlled trial of arbaclofen, enrolling 150 people with ASD for a total duration of treatment of 12 weeks. For more information about the clinical trial visit http://www.clinicaltrials.gov/ .

We will be sure to keep you informed as this study and other translational research progresses!
Here is the link to the blog:
Autism Speaks Blog

Autism Therapy More Successful When Peers Involved Too

By Shaun Heasley

November 29, 2011 Text Size A A

Kids with autism fare significantly better socially when their typically developing classmates are taught how to interact with them, new research suggests.
Often students with autism are targeted for social skills training. But that alone may not be the best approach, according to a study published this week in the Journal of Child Psychology and Psychiatry.
For the study, researchers at the University of California, Los Angeles looked at 60 kids with autism ages 6 to 11, all of whom were in mainstream classes at least 80 percent of the day.
Some of the children received one-on-one social skills training from a professional. In other cases, a professional taught a group of three typically developing kids from the child’s class how to engage with students who have social difficulties but did not interact with the child who had autism.
Meanwhile, other students in the study benefited from both approaches — individual training and peer training. A control group did not experience either social skills intervention.
Ultimately, the researchers found that children whose peers received training were less likely to spend time alone on the playground as compared to those who received only individual training. In addition, kids who benefited from peer training were more likely to have classmates say that they were friends.
Moreover, the benefits of peer training appear to stick around. Even when kids advanced to new classrooms with different students, researchers found that the children with autism who experienced peer training continued to see improved socialization.
The findings suggest that focusing on typically developing peers in addition to the child with autism may offer the best results, the researchers said. What’s more, they indicated that individual social skills intervention may only be beneficial if professionals also work with the child’s peers.
“Real life doesn’t happen in a lab, but few research studies reflect that,” said Thomas Insel, director of the National Institute of Mental Health, which helped fund the study. “As this study shows, taking into account a person’s typical environment may improve treatment outcomes.”
Despite the improvements seen with peer intervention, however, the researchers said it’s not a total fix. Children with autism continued to say they had few friends and struggled with issues like taking turns even when their classmates were taught how to work with them.