Drug could get into the autistic mind

New Scientist Magazine
01 June 2010 by Celeste Biever

CAN people with autism take a pill to improve their social skills? For the first time, drugs are being tested that could address the social difficulties associated with autism and other learning disorders by tackling some of the brain chemistry thought to underlie them.

The only drugs currently prescribed to people with autism seek to dampen aggression and anxiety. The new drugs, now in the very early stages of clinical testing, address some of the classic symptoms of autism.

"People may learn more, learn to speak better, learn social skills and to be more communicative," says Randall Carpenter of Seaside Therapeutics in Cambridge, Massachusetts, which is testing one of the drugs.

Geraldine Dawson, chief science officer at the charity Autism Speaks and a psychiatrist at the University of North Carolina at Chapel Hill, is equally enthusiastic about the prospect of a new class of drugs. "For the first time we are seeing drugs that could tackle core autism symptoms," she says.

For the first time we are seeing drugs that could tackle the core symptoms of autism
The Seaside trial is aimed at a learning disorder called fragile X, which is associated with autism. People with fragile X carry a mutation in a gene involved in strengthening brain connections associated with salient experiences. Stronger brain connections allow people to distinguish these events from background noise, making this a key process in learning.

Carpenter and his colleagues are testing a drug called arbaclofen, which seems to reverse the effect of the mutation. At the International Meeting for Autism Research in Philadelphia, Pennsylvania, on 23 May, they presented initial results suggesting that the drug may improve the social skills of people with fragile X and autism, including improved communication and general sociability, and fewer outbursts.

Seaside's trial is not the only attempt to alter the brain chemistry of people with autism. The hormone oxytocin, also known as the cuddle chemical, helps us connect social contact with feelings of pleasure, and some people with autism produce less of it. Several teams are looking into boosting oxytocin to relieve symptoms of autism.

At the Philadelphia meeting, a team led by Evdokia Anagnostou, a child neurologist at Bloorview Research Institute in Toronto, Canada, reported that people given the hormone twice daily for six weeks were more likely to be better at recognising emotions and at social functioning, and had a better quality of life than others given a placebo.

Trying to alter the brain chemistry thought to underlie autistic behaviour has never been done before in this way, says Uta Frith of University College London. "If they succeed it would be marvellous." But she cautions that the drugs have not yet been shown to work better than behavioural interventions and that most causes of autism are still deeply mysterious.

Carpenter points out that behavioural interventions don't work for everyone, and both approaches could be useful. "If we come up with an effective treatment, parents are going to embrace that."