New autism drug being tested by UIC researchers 

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A drug to treat social withdrawal in autistic children is being studied in a clinical trial at the University of Illinois at Chicago's Institute for Juvenile Research. | File photo

An experimental drug to treat social withdrawal in children and young adults with autism is being studied in a clinical trial at the University of Illinois at Chicago’s Institute for Juvenile Research.

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.

Dr. Edwin Cook, professor of psychiatry and director of autism and genetics at UIC, said a drug treatment is needed that would address symptoms that are “often disabling for patients and families.”

UIC is the only study site in Illinois and one of 25 sites nationwide.

The study is sponsored by Seaside Therapeutics, Inc.

The clinical trial will enroll approximately 150 patients diagnosed with autism spectrum disorders between ages 5 and 21 to evaluate the efficacy, safety and tolerability of 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.

Cook’s UIC study team includes co-investigator Dr. 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.

For more information about UIC, visit www.uic.edu.

RESOURCES

The National Fragile X Foundation is an organization devoted to offering educational and emotional support, promoting public and professional awareness, and advancing research toward improved treatments and a cure for Fragile X Syndrome, a genetic disorder that is the most common identified cause of autism.

The Autism Support Network is a free online support community of families and individuals touched by autism spectrum disorder. Find out what’s working for others, coping strategies, and life guides from others.