Medication Studied for Autism

Researchers at Ohio State University Medical Center are conducting a number of studies of potential therapies for children with autism, including one study seeking to determine whether a specific medication could reduce the core (main) symptoms of the disorder.

“There is currently no FDA-approved medication for the core symptoms of autism. New directions for therapy need to be based on understanding the central neurobiology of autism,” said Dr. L. Eugene Arnold, a child psychiatrist at Ohio State University Medical Center and interim director of Ohio State’s Nisonger Center. Arnold is the principal or co-investigator of all of these studies.

One consistently identified characteristic of brains of deceased individuals with autism is an abnormality in one of the receptors – nicotinic – for the neurotransmitter acetylcholine. This pilot trial aims to test the response of individuals with autism to agents that target this receptor type, which changes acetylcholine signaling. Acetylcholine is particularly important in mental function. The agent to be tested in children with autism, mecamylamine, has been shown to be safe in other childhood-onset disorders, including Tourette’s syndrome and attention-deficit/hyperactivity disorder (ADHD).

For the current study, 20 children age 4-12 with autism spectrum disorder will receive the drug or placebo over 13 weeks in a controlled trial. The outcome will be assessed by measures of a range of behavioral and communicative functions. Those who first receive placebo will have an opportunity for an open trial of the real medicine. The core symptoms of autism are social impairment, communication impairment and repetitive behavior. For more information about this study, contact coordinator Kristy Humphries at 614-292-3698 or humphries.33@osu.edu.