UR Awarded $7.5 Million to Investigate Autism Treatment

Studies to Focus on Diet, Behavioral Therapy, Genetic Links

May 21, 2003

Already a national leader in autism research due to the late-1990s discovery of a critical genetic link, the University of Rochester Medical Center is among eight institutions in the United States selected to study treatments for this early childhood brain disorder.

The $7.5 million, five-year National Institutes of Health grant will fund studies in diet and intensive behavioral therapy, with related investigations into neurobiology and genetics. The NIH initiative, announced this month, is called STAART (Studies to Advance Autism Research and Treatment.)

With awareness of autism on the rise, the funding demonstrates a belief by physicians, researchers, mental-health experts and parents of children with autism that a broad, scientific review of treatments is needed to determine which ones are most effective. There is no cure for autism, although many children do respond to therapy.

"Autism treatment is very expensive financially and emotionally - and no single approach works for all children," says Patricia M. Rodier, Ph.D., and principal investigator on the UR project. "That’s why it is so important to answer the big questions: ‘Who is going to respond? And to what treatment?’ If we could predict in advance which children would benefit from available treatments and which would not, children could be matched to the best treatments available."

Autism is a life-long brain disorder that’s usually diagnosed by age three when a child does not seem to pass the typical milestones between infancy and the toddler years. Children with autism lack language skills, have difficulty with social interactions, and often display odd, repetitive behaviors. However, people with autism do not always share the same symptoms or deficits, so experts classify it as a "spectrum disorder" with forms from mild to severe. Experts estimate that one in 200-300 children have autism spectrum disorders.

The UR’s latest research is designed to test two treatments:

  • The diet study will test a popular theory that symptoms improve when a child with autism stops eating foods containing gluten (wheat, rye, oats) and dairy products. The UR team will conduct a double blind, placebo-controlled clinical trial to study the link between diet and behavior.
  • The study on behavioral treatment will collect data on IQ, social and language skills, repetitive movements and other characteristics of children with autism. The researchers will look at how children with certain characteristics respond to treatment. The goal is to help families choose therapies that are likely to be successful.

In addition, the UR will conduct a neurobiology study of facial movement to discover why some children with autism exhibit little or no facial expression. Using sophisticated imaging techniques, researchers will plot where in the brain facial movements originate when children are shown movies, pictures and other stimuli.

Susan Hyman, M.D., associate professor of Pediatrics, co-director of the new STAART Center, and a well-known expert in developmental disabilities, credits the UR’s collaborative and multidisciplinary approach for the influx of government money into the autism research program.

"In Monroe County we have already been able to develop national models for establishing partnerships between schools, families, psychologists, physicians and scientists to support high quality clinical services that were non-existent a decade ago," Hyman says. "We believe that we have one of the best intervention systems in the country."

Led by Rodier, the UR team achieved national prominence by delving into the roots of autism. An embryologist and professor of Obstetrics and Gynecology, Rodier discovered that autism might develop in the earliest weeks of pregnancy, when the nervous system is just beginning to take shape. Her hypothesis is that a mutation in genes controlling early brain development, coupled with environmental factors, might result in autism. NIH funding for that line of research began in 1996 at the UR, and was recently renewed for another five years for approximately $8 million. Moreover, Rodier’s group is part of a national network studying more than 2,000 families struggling with autism spectrum disorders.

Besides Rodier and Hyman, the other principal investigators on the STAART project are: Rafael Klorman, professor, Clinical and Social Psychology; Tristram Smith, assistant professor, Pediatrics; Caroline Magyar, M.D., assistant professor, Pediatrics; Loisa Bennetto, assistant professor, Clinical and Social Psychology; and Christopher Stodgell, assistant professor, Obstetrics and Gynecology.

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