Pathways to New Treatments in Autism Spectrum Disorder

Two main approaches are being pursued to identify new medication treatments that may benefit children with autism spectrum disorder. The first and most common approach is to evaluate a treatment for the total group of people diagnosed with ASD, typically targeting broad hypotheses about social function or repetitive behavior. The second approach is almost the exact opposite, to study a medication for ASD-related symptoms in a defined genetic syndrome that confers substantial risk of ASD but comprises less than 2% of individuals with ASD. With emerging knowledge of brain systems and intersections with genetic data, we can hope for a third approach that is somewhere in the middle, with a treatment being studied in a larger subgroup of individuals with ASD who share a common biomarker or biomarkers. I will highlight the possibilities of this third approach using the example of the serotonin system, long implicated by biomarker findings in ASD.

Dr. Veenstra-VanderWeele is a child and adolescent psychiatrist who uses molecular and translational neuroscience research tools in the pursuit of new treatments for autism spectrum disorder (ASD) and pediatric obsessive-compulsive disorder (OCD). His molecular neuroscience laboratory at Columbia University and the New York State Psychiatric Institute focuses on genetic mouse models with abnormal social or repetitive behavior. His clinical/translational research program at the New York-Presbyterian Hospital Center for Autism and the Developing Brain studies potential treatments for ASD and related genetic syndromes, including fragile X syndrome. Dr. Veenstra-VanderWeele serves as an associate editor of Autism Research, the Journal of the International Society for Autism Research, and of the Journal of Autism and Developmental Disorders. He also co-chairs the Autism and Intellectual Disability Committee of the American Academy of Child and Adolescent Psychiatry. His work has garnered multiple awards, most recently including the Blanche Ittelson Award for Research in Child and Adolescent Psychiatry from the American Psychiatric Association.

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Planner and Speaker’s Disclosures: 

Harold Koplewicz, MD, Ron Steingard, MD, and John Q. Young, MD, have nothing to disclose. Jeremy Veenstra-VanderWeele, MD, discloses to be the principal investigator for research contract at Roche

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