Novel Therapeutics:

Precision Metabolic Therapies for Autism Spectrum Disorders

Maria Chahrour, Ph.D.

• Associate Professor
• Director, Neuroscience Graduate Program, Department of Neuroscience
• Eugene McDermott Center for Human Growth and Development
• Center for the Genetics of Host Defense
• Peter O’Donnell Jr. Brain Institute, UT Southwestern Medical Center

The Chahrour Lab

Autism spectrum disorder affects millions of children, yet there are no approved drugs that treat its underlying biology. Dr. Chahrour has discovered a clear and druggable metabolic pathway in autism in which abnormal protein regulation disrupts brain development and cognition. In animal models, blocking this pathway restores memory and improves behavior, proving that cognitive function can be rescued even after early brain development. Her team is now developing next-generation, brain-penetrant small-molecule drugs with strong biomarker readouts and orphan-disease entry points. This program lays the foundation for the first precision, mechanism-based therapeutic platform in autism, with expansion potential into broader neurodevelopmental and neuropsychiatric indications.