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Trial Shows Low-Dose Anti-Anxiety Med Helps Curb Autism Behaviors in Young Children

Wednesday, Dec 30, 2015

Delaware Valley

The anti-anxiety medication Buspirone is effective in low doses in improving repetitive and restrictive behaviors in young children with autism, and may be useful combined with early intensive behavioral interventions that improve social communication and adaptive behavior.

The discovery is the result of a multi-year, multi-center clinical trial led by autism researcher Diane Chugani, PhD, and published today in The Journal of Pediatrics. Dr. Chugani, formerly of Wayne State University and the Children’s Hospital of Michigan at the time of the study, is now Director of Neuroscience Research at Nemours/Alfred I. duPont Hospital for Children in Wilmington, DE.

Autism spectrum disorder (ASD) is a neurodevelopmental condition characterized by impairment in social communication and the presence of repetitive and restricted behaviors, including persistent strong interest in specific objects and unusual topics, speech echolalia, and repetitive hand and body movements such as hand-flapping, spinning and odd posturing.

"I think any research that shows benefit gives families hope. They hope for more — a cure — but help with some of the symptoms can be very meaningful for everyday life," said Dr. Chugani.

The U.S. Centers for Disease Control and Prevention reports ASD is the fastest-growing developmental disability in the country, with approximately one in 68 children diagnosed nationwide. The annual growth rate has averaged 7 percent to 8 percent over the last five years.

Buspirone, also known by brand names BuSpar and Vanspar, is already an approved treatment for children with anxiety, a comorbid condition in ASD. In the study, "Efficacy of Low Dose Buspirone for Restricted and Repetitive Behavior in Young Children with Autism Spectrum Disorder: A Randomized Trial," 166 children with ASD ranging in age from 2 to 6 were randomized to receive a placebo, 2.5 mg or 5.0 mg of buspirone twice a day for two 24-week phases. Children who were given 2.5 mg of the medicine showed significant improvement in restricted and repetitive behaviors.

Using positron emission tomography (PET), Dr. Chugani discovered in the late 1990s that changes in serotonin syntheses that occur with age are not present in autistic children. Dr. Chugani designed the study based on those imaging data in children with ASD.

Before drug treatment, the children underwent PET and blood studies to evaluate whether Buspirone response can be predicted by brain tryptophan metabolism or blood serotonin levels.

"Since serotonin function is important in postnatal brain development, we hypothesized that one approach to the pharmacologic treatment of core features of ASD would be the use of serotonin agonists in children younger than six years, when their brain serotonin synthesis capacity is lower than in children without ASD," Dr. Chugani wrote.

The children in the study were more likely to improve if they had fewer foci of increased brain tryptophan metabolism on PET, or if they showed normal blood serotonin levels. Previous studies show that 30 percent to 50 percent of individuals with ASD show elevated serotonin concentrations. Dr. Chugani plans to assess drug response in conjunction with behavior therapy soon.

The trial was supported by a grant from the National Institute of Neurological Disorders and Stroke (5U01 NS61264).

About Nemours

Nemours is an internationally recognized children's health system that owns and operates the two free-standing children’s hospitals: the Nemours/Alfred I. duPont Hospital for Children in Wilmington, Del., and Nemours Children's Hospital in Orlando, Fla., along with outpatient facilities in five states, delivering pediatric primary, specialty and urgent care. Nemours also powers the world’s most-visited website for information on the health of children and teens,, and offers on-demand, online video patient visits through Nemours CareConnect. Nemours is a program dedicated to preventing reading failure in young children, grounded in Nemours’ understanding that child health and learning are inextricably linked, and that reading level is a strong predictor of adult health.

Established as The Nemours Foundation through the legacy and philanthropy of Alfred I. duPont, Nemours provides pediatric clinical care, research, education, advocacy and prevention programs to families in the communities it serves.