Fragile X drug now in clinical trials

CAMBRIDGE, Mass., Nov. 5 (UPI) -- U.S. scientists say they are starting a trial of a medication designed to treat the neurochemical defect underlying Fragile X syndrome.

Researchers at Seaside Therapeutics in Cambridge, Mass., said Fragile X syndrome -- the most common inherited cause of intellectual disability -- causes a range of developmental problems, including learning disabilities, cognitive impairment, attention deficit hyperactivity disorder and autism or autistic-like behavior.

People with Fragile X have DNA mutations in the FMR1 gene that, in effect, turn off the gene and prevent normal brain synaptic synthesis.

The new trial tests a Seaside Therapeutics' compound, STX107, which selectively and potently targets the synaptic defect.

"This project is the culmination of years of fundamental research, first identifying the genetic mutation and later deciphering the biochemical consequences of this mutation," said Dr. Thomas Insel, director of the National Institute of Mental Health "Now, with the initiation of this first clinical study, we move one step closer to understanding how this novel candidate may play a critical role in improving the lives of individuals with Fragile X Syndrome."

Dr. Randall Carpenter, president and chief executive officer of Seaside Therapeutics, and Mark Bear, a Massachusetts Institute of Technology professor of neuroscience and Seaside's scientific founder, are leading the research.