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Unlocking the Secrets of Autism

Lab to Life-Again

Case Western Reserve neuroscientist David M. Katz, PhD, has spent his career exploring the mysteries of the brain—specifically the way neurons develop and how their progress can go awry.

But as compelling as the intellectual questions proved, in 2005 his work took on new intensity. That year he met Chelsea Coenraads, a young girl with Rett syndrome, one of the most severe of the autism spectrum disorders. Her penetrating gaze has stuck with him ever since.

“Meeting her really deepened my commitment to doing everything we could do to figure out what’s wrong in the brains of these children,” he explains.

Since that time, Katz has made significant progress not only in understanding the roots of Rett syndrome, but also in potential approaches to help reverse it. His efforts have proved so promising that this year he and his research team won the first grant that the national group Autism Speaks has awarded in Northeast Ohio. The three-year, $443,416 grant will support studies to evaluate experimental compounds that mimic a molecule that is key to normal brain function, yet deficient in Rett syndrome. The compounds were developed by a collaborator of Katz’s at Stanford University, Frank Longo, MD, PhD.

Robert Ring, vice president of translational research at Autism Speaks, says Katz’s work “is helping to rationalize and support a path that could redirect the development of these agents as treatments for Rett. This would be a win for the autism community.”

Rett syndrome affects girls almost exclusively. Children with the disorder appear healthy at birth, but within about six to 18 months they begin to show symptoms that include problems with movement, cognition, speech, breathing, feeding and other functions.

Katz hopes to discover which molecules work best to improve cognitive function in an experimental model of Rett syndrome, to identify the best treatment regimen and to understand genetic influences on treatment response. There’s no guarantee the work will lead to human trials, but Katz finds plenty of reason for hope.

“I believe it is possible,” he says, “that through the combined efforts of different laboratories, we will have treatments for at least some of the symptoms of Rett syndrome within the next three to five years.”

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