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UIC researchers test autism treatment

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Edwin Cook

Edwin Cook, professor of psychiatry and director of autism and genetics, leads a team testing an experimental drug to treat social withdrawal in children with autism.

Photo: Roberta Dupuis-Devlin

UIC is the only site in Illinois conducting clinical trials of an experimental drug to treat social withdrawal in children and young adults with autism.

Children with autism — or autism spectrum disorders — often have difficulty communicating and interacting with others. Although behavioral and psychological interventions are often beneficial, currently there is no medication to address social communication difficulties, a core symptom of ASD.

A drug treatment is needed that would address symptoms that are "often disabling for patients and families," said Edwin Cook, professor of psychiatry and director of autism and genetics.

UIC’s Institute for Juvenile Research is one of 25 sites nationwide for the study, sponsored by Seaside Therapeutics Inc.

The clinical trial of about 150 patients, ages 5 to 21, diagnosed with autism spectrum disorders, will evaluate the efficacy, safety and tolerability of the medication STX209 (arbaclofen).

Participants in the 22-week study will be randomized to receive either the study drug, STX209, or a placebo. The clinical trial will include screening, treatment, withdrawal of medication and a follow-up period.

Subjects who complete the study may be eligible to enroll in a subsequent open-label study in which all subjects are treated with STX209.

STX209 has been studied in children with Fragile X syndrome, a genetic disorder that is the most common identified cause of autism. Previous research has found that from one-quarter to one-half of people with Fragile X have autism spectrum disorders, said Cook, principal investigator of the UIC study.

"This trial is exciting, because it represents the culmination of 20 years work in Fragile X research," said Cook, who describes the preliminary data leading to the STX209 study in autism as a "scientific and rational approach" to medication development.

"We're not expecting this to cure Fragile X or autism, but it's a very important step in the development of new treatments," Cook said.

"Finding genes is great, but it's all about treatment."

Cook's UIC study team includes co-investigator Fedra Najjar, assistant professor of psychiatry, and study coordinators Sarah Youngkin and Clare Tessman.

For more information about the clinical trial, call Sarah Youngkin at 312-413-9061 or email syoungkin@psych.uic.edu


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