University of Ottawa researchers target brain blood vessels for autism

Researchers from the University of Ottawa and The Ottawa Hospital have identified a potential new therapeutic pathway for treating behavioral symptoms of autism spectrum disorder (ASD) by targeting brain blood vessels rather than neurons. The study, published in the journal Neuron, focuses on the 16p11.2 genetic deletion, one of the most common mutations associated with autism.

The research team discovered that in mouse models with this deletion, endothelial cells—which line the interior of blood vessels—lack a critical surface receptor required for energy regulation. This deficiency causes the brain’s vascular system to react too slowly to developmental demands, contributing to symptoms such as hyperactivity, repetitive movements, and motor learning difficulties.

By administering a medication currently used in Japan and South Korea to treat dry eye syndrome, the researchers were able to activate the missing P2Y2 receptors. This intervention increased cerebral blood flow and successfully reversed established behavioral symptoms in adult mice. While the researchers noted that the path to clinical trials is long, the findings suggest that targeting brain endothelial dysfunction represents a promising strategy for future ASD treatments.