By Julie Steenhuysen
Reuters
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.
Even so, the number of studies of the drugs remains small, and the trials themselves have been relatively small. Of those that have been completed, the effect has been dramatic in some patients but negligible in others.
"This is a new day, and that's exciting," says Dr. Thomas Insel, director of the National Institute of Mental Health in Bethesda, Maryland, one of the National Institutes of Health. "But we also have to temper that with the reality that there may be many failures along the way before we come up with a treatment that is both effective and safe."
Nine-year-old Jack Balter plays tug-of-war with his sister Brianna at their home in Scottsdale, Arizona, on May 17.
Mad scientist
As a toddler, Jack Balter was hyperactive. He would be mesmerized by the wheels on toy trucks or anything else that spins, stand on his tiptoes, cover his ears - all classic signs of autism. "We didn't even know what autism really was, so we didn't know it was something out of the ordinary," says Lynn, 52.
Jack was diagnosed with autism just before his third birthday. Since then he has had about 60 hours of intensive therapy a week, including speech and physical therapy and social interaction therapy. About 10 percent of autistic children who receive intensive therapy "bloom," enjoying rapid improvement as they grow older, according to a recent study published in the journal Pediatrics.
"We've been lucky. He's responded," says Lynn. Jack is now in a mainstream classroom with a full-time aide.
In a family video posted to YouTube, Jack lays out his ambitions: "On the outside, I'm just a regular old kid. On the inside, I'm a mad scientist." He says that when he grows up, he wants to be an oceanographer, a meteorologist, a climatologist, a volcanologist and a seismologist.
But even with therapy, Jack has struggled to interact with the world around him, talking in numbing detail about his favorite subjects, never playing sports or watching cartoons, standing too close to people. To other children, he seems odd.
"He's always been in his own tunnel," Lynn says.
Last November, Jack was enrolled in a trial of Seaside's compound STX209, or arbaclofen. Arbaclofen contains one of the active ingredients in an older drug, baclofen, that has been used since the 1960s to treat cerebral palsy.
Jack was part of a randomized study of 150 children with autism. Neither his parents nor his doctors knew whether he received the drug or a placebo.
Jack's parents are convinced he got the drug. Neil, 51, who founded custom storage maker California Closets at age 17 and now runs another custom closet and home organization business, graphed Jack's progress during the trial. "You can see when he got to the 10-milligram dose, 18 days in a row" he came home with a positive report from his teacher, Neil says.
Teachers didn't know about the trial, but they knew something was suddenly different. Carrie Cunningham, Jack's third-grade teacher, says Jack was more focused and could express himself better, and his emotions were mostly in check. "His aide and I both noticed a change almost immediately," she says.
They also noticed when the study ended. "It was a nightmare," Cunningham says. "All of a sudden, everything brought tears to his eyes. Everything was a battle."
Two weeks later, Jack started on a follow-up study in which his parents know he is getting the drug, and Jack has once again improved.
Neil says that on a scale of 1 to 10, with 1 being severe disability and 10 being normal, he used to think of Jack as a 5. Since Jack has been taking arbaclofen, Neil sees him at 7.5.
"It's not a cure," he says, "but it definitely moved the needle."
Seaside is running two late-stage trials in adults and children with Fragile X, and a study in children with different types of autism, including autism with no identifiable cause, like Jack's. Results of the study in which Jack participated are expected later this year.
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