Baclofen, STX209 and Autism
One night in 2006, Kathy Roberts rushed her autistic daughter, Jenny, to the hospital. Nothing had been able to stop the young woman, then in her mid-20s, from vomiting. Jenny had recently suffered several major seizures and her entire gastrointestinal system was going haywire.
To try to calm Jenny's GI tract, doctors at Massachusetts General Hospital prescribed baclofen, an antispasmodic drug that is also being studied as a potential treatment for alcoholism and other addictions. The drug relieved Jenny's vomiting, but it did something else too — a completely unexpected and welcome side effect.
"Within 24 hours, I saw a change," says Roberts. "Right away, I saw that it was globally calming. I've always described a state that she would get into where it seemed like she wasn't comfortable in her own skin, and was trying to crawl out. I saw that calmed down."
Roberts, founder of the Giant Step school for children with autism in Southport, Conn., called Mark Bear, professor of neuroscience at MIT and advisory board member of Giant Step. In 2005, Bear had co-founded a drug company called Seaside Therapeutics to develop treatments for autism and other developmental disorders. Roberts told Bear about baclofen's effect on her daughter, and a new line of research was born.
In September, Seaside announced positive results from a phase II clinical trial of STX209, an experimental drug that is chemically related to baclofen. In the trial, which was not blinded or placebo controlled, STX209 led to a reduction in agitation and related emotional outbursts in autistic people. Such behavior is common in people with autism — often, a result of anxiety caused by extreme sensory oversensitivity or frustration over being unable to communicate their needs. To cope, autistic people often develop behavioral mechanisms, include tantrums, social withdrawal or repetitive behaviors like rocking or hand flapping.
STX209, while not a cure, appeared to ease anxiety. "We're seeing reductions in a lot of types of outbursts and irritable behavior, along with increased communication and social behavior," says Dr. Randall Carpenter, co-founder, president and CEO of Seaside.
Read more: http://healthland.time.com/2010/12/01/how-a-new-version-of-an-old-drug-may-someday-help-treat-autism-and-addiction-too/#ixzz16tSJxkbc
