An experimental drug that helps people who have Fragile X syndrome is raising hopes of a treatment for autism.
The drug, called Arbaclofen, made people with Fragile X less likely to avoid social interactions, according to a study in Science Translational Medicine. Researchers suspect it might do the same for people with autism.
Fragile X is a rare genetic syndrome that can cause intellectual and social problems that are very similar to autism.
The discovery that Arbaclofen helps people with Fragile X might never have occurred without the efforts of Katie Clapp and Michael Tranfaglia, a Massachusetts couple whose son was born with the syndrome more than 20 years ago.
When the couple learned that their son Andy had Fragile X, they started a foundation called FRAXA. It's goal was to fund research that would lead to a treatment for their son.
Clapp says that goal is now in sight.
"Back in 1994, we knew exactly what we were aiming for, and it's gotten closer and closer, little by little," she says.
Research funded in part by FRAXA led to the discovery that Fragile X disrupts a system that regulates connections in the brain. That discovery eventually led to drugs with the potential to fix the problem including Arbaclofen.
The new study of the drug involved 63 children and adults with the syndrome and was conducted by a team from Rush University Medical Center and the University of California, Davis MIND Institute.
Becky Zorovic is a physician whose son Anders was in the trial and continues to take Arbaclofen. Before the treatment, she says, Anders was terrified by things like birthday parties.
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New Experimental Drug Offers Autism Hope