WASHINGTON — A pill to ease a type of cognitive disability? An experiment is underway to develop one, aimed at a genetic disorder that might unravel some of the mysteries of autism along the way.
Fragile X syndrome is the most common inherited form of intellectual impairment, estimated to affect almost 100,000 Americans. It’s also the most common cause of autism yet identified, as about a third of Fragile X- affected boys have autism.
Now a handful of drugmakers are working to develop the first treatment for Fragile X.
“We are moving into a new age of reversing intellectual disabilities,” predicts Dr. Randi Hagerman, who directs the MIND Institute at the University of California, Davis, a study site.
Fragile X, more common in males than females, ranges from learning disabilities to severe cognitive impairment, along with emotional and behavioral problems. The genetic defect disrupts a basic foundation of learning: How brain cells respond to experiences by forming connections between each other, called synapses. Those structures aren’t destroyed — they’re too immature to work properly.
The experimental drugs aim to get the brain back on track by simply blocking an overactive receptor that plays a key role in weakened synapses. The goal is to strengthen synapses, to make learning easier and behavior more normal.
