Pill in works to treat inherited retardation

February 02, 2010|Associated Press

WASHINGTON - An experiment is underway to develop a pill to ease a type of mental retardation.

The work aims at a genetic disorder and might unravel some of the mysteries of autism along the way.

The drug would treat Fragile X syndrome, the most common inherited form of intellectual impairment.

The syndrome is estimated to affect almost 100,000 Americans and is also the most common cause of autism yet identified.

A handful of drug makers working on the treatment were spurred by brain research that is making specialists rethink how they approach developmental disorders.

“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 are just too immature to work properly.

“The process of learning is just that much more difficult but not impossible, because there’s nothing wrong with the synapse,’’ says Dr. Stephen Warren, an Emory University geneticist who led the discovery of Fragile X’s mutated gene.