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NIH Launches Neurological Drug Development Projects

New research targets Fragile X syndrome, nicotine addiction, and macular degeneration (July 31)

The National Institutes of Health (NIH) has launched three projects that will focus on the development of therapeutics for Fragile X syndrome, nicotine addiction, and age-related macular degeneration (AMD).

Fragile X syndrome is a genetic disorder linked to a range of neurodevelopmental disorders, including learning disabilities and cognitive impairment. Many patients experience general and social anxiety, and yet benzodiazepines, which are typically used to treat anxiety disorders, provide little relief. Their anxiety has been linked to reduced activity in the brain by a protein called the GABA A receptor. Sage Therapeutics in Cambridge, Mass., is developing positive allosteric modulators, designed to enhance the receptor’s activity and possibly relieve the anxiety.

Nicotine addiction has been attributed to the stimulatory effects of nicotine binding to brain proteins called orexin 1 receptors. Researchers at the Scripps Institute in Jupiter, Fla., will develop selective receptor antagonists as potential smoking-cessation aids to treat people who have attempted to quit smoking but have experienced high relapse rates and significant side effects.

AMD is a leading cause of blindness in the U.S. One form, called wet AMD, is associated with inflammation and blood-vessel leakage in the retina. Scientists at the University of Utah are developing small molecules that inhibit the activity of Arf6, a molecule involved in inflammation and blood-vessel leakage. This novel approach may lead to effective therapies for treating patients who do not respond to current wet AMD therapies, according to the NIH.

Source: NIH; July 31, 2013.

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