CAMBRIDGE, Mass., Nov 5, 2009 (UPI via COMTEX) -- 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.
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Copyright 2009 by United Press International