New drugs, fresh hope for autism patients

CHICAGO Lynn and Neil Balter always dreaded stage productions at their son Jack's elementary school.

When Jack was up there with the other performers, the noise, the lights, the crowd almost always got to him, and he would "start spinning," wandering around the stage or turning in circles, Lynn says. "It usually turned into an embarrassing situation," she adds.

But at a dance performance at Jack's Scottsdale, Arizona, school last December, something was different. "He was half a beat behind in the dance, but he did the whole thing," Neil says. "He participated and took the bow with his class."

Afterward, Jack's teacher greeted the Balters in tears. "I don't know what is going on with this kid, but there is this miracle happening and I have a different kid at school," she told the Balters.

Jack, 9 years old, has autism. What his teacher didn't know is that Jack was taking part in a clinical trial for a drug aimed at overcoming some of the social impairment associated with autism, a spectrum of disorders that range from the social awkwardness and narrow interests seen in Asperger syndrome to severe communication and intellectual disabilities.

For years the best that doctors have been able to offer patients with autism is intensive therapy and anti-psychotic drugs such as Johnson & Johnson's Risperdal to blunt some of the extreme behaviors associated with their disorder - tantrums, aggression and self-harm. Anti-psychotics quiet the patients. But they do nothing to address the core social and communication problems that make it impossible for many autistic children to develop deep relationships with their families and peers and grow into independently functioning adults.

As Jack's experience suggests, that may be about to change. Researchers are conducting advanced trials of the first drugs expressly designed to correct the genetically induced signaling problems in the brain that result in autism. The early indications are positive enough to offer new hope for families and spark interest from drug companies.

For patients, this research "may not solve their autism, but it may solve aggression, it may solve sensory overload, which leads to a lot of behavioral issues," says Isaac Pessah, an autism researcher at the University of California at Davis, who has not been involved in any of the drug trials.

Swiss drugmakers Novartis International AG and Roche Holding AG, as well as privately held Seaside Therapeutics in Cambridge, Massachusetts, all have drugs in late-stage tests targeting Fragile X syndrome, the most common form of inherited intellectual disability and the most common known genetic cause of autism. And though Fragile X accounts for only a small percentage of autism patients, early studies suggest the drugs may work in other forms of autism, too.

If the current trials show enough benefit to satisfy U.S. regulators, the first drugs could be available in a few years. And if that happens, they will serve a growing population. The latest figures from the Centers for Disease Control and Prevention estimate that as many as 1 in 88 children in the United States has autism, a near-doubling of the rate reported in 2002.

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New drugs, fresh hope for autism patients