Autism is relatively common, although prevalence estimates vary by country and age group. In the United States, recent CDC surveillance estimates suggest that about 1 in 31 eight-year-old children are identified with autism spectrum disorder, while global estimates generally place prevalence at around 1% of the population. Credit: Shutterstock

A study in mice suggests that cells lining the brain’s blood vessels may play an important role in autism-related symptoms.

Researchers have reversed several autism related behavioral symptoms in adult mice by correcting a problem in the blood vessels that supply the brain. The findings point to an unexpected treatment target: endothelial cells, which line blood vessels and help control how quickly blood reaches active regions of the brain.

The study, published in Neuron, focused on mice carrying a 16p11.2 deletion, a genetic change associated with autism in humans. Activating a receptor called P2Y2 restored blood vessel function, increased blood flow in the brain, and reversed hyperactivity, repetitive movements, and impaired motor learning in the animals.

A Vascular Problem Inside the Brain

Autism is a neurodevelopmental condition with a wide range of characteristics and support needs. Some associated behavioral symptoms can make daily life more challenging, and there are currently no drug treatments that broadly address those symptoms.

The same researchers had previously discovered that brain blood vessels function abnormally in mice with the 16p11.2 deletion. Their work suggested that the problem begins early in development within endothelial cells, which form the inner lining of blood vessels.

Researchers from Dr. Baptiste Lacoste’s research team at The Ottawa Hospital and the University of Ottawa were able to reverse certain behavioural symptoms in a mouse model of autism by fixing a problem in the brain blood vessels. Their findings, published in Neuron, present a promising new treatment target for these symptoms. Credit: The Ottawa Hospital

These cells help regulate blood delivery to active brain tissue. When neurons become more active, nearby blood vessels normally respond by increasing local blood flow so the tissue receives enough oxygen and nutrients. In the mice studied by the team, that vascular response was too slow.

A Missing Signal in Blood Vessel Cells

In the new study, led by former PhD student Dr. Julie Ouellette, the researchers investigated what was preventing the endothelial cells from responding normally.

They found that the cells had about half the normal level of ATP. Although ATP is best known as the molecule cells use to store and transfer energy, it can also act as a signaling molecule outside cells. In this case, the researchers traced the defect to reduced signaling through P2Y2, a receptor found on the cell surface.

Stimulating P2Y2 restored endothelial cell function. Blood flow in the brain improved, and several behavioral abnormalities that were already established in adult mice were reversed.

An Existing Drug Points to a Possible Route Forward

The researchers achieved the effect using a drug that activates P2Y2. The compound is already approved in Japan and South Korea for the treatment of dry eye syndrome.

“It’s as if these cells are asleep, and now we can wake them up,” says Dr. Lacoste. “And we may only need to treat them once to wake them up permanently. We will test that further, but it’s an encouraging feature for a future treatment.”

Testing Whether Earlier Treatment Changes the Outcome

The team now plans to activate P2Y2 earlier in development to determine whether treatment at a younger age produces different or additional effects.

“The road from discovery to clinical trials is long, but we’re excited by the possibility that our findings might one day improve the daily lives of people with autism,” said senior author Dr. Baptiste Lacoste, senior scientist at The Ottawa Hospital and professor at the University of Ottawa.

The researchers have also filed a patent application covering the use of P2Y2 activation in blood vessels to treat autism related symptoms.

Reference: “Purinergic receptor activation rectifies autism-associated endothelial dysfunction” by Julie Ouellette, Sareen Warsi, Phinea Romero, Purva Khare, Shama Naz, Leya Aubert-Tandon, Moises Freitas-Andrade, Chantal Pileggi, Sozerko Yandiev, Joanna Raman-Nair, James Yee, Nicole Blakeley, Balaji Govindaswamy, Cesar Henrique Comin, Mary-Ellen Harper, Devika Soundara Manickam, Fabrice Dabertrand, Armen Saghatelyan and Baptiste Lacoste, 30 September 2026, Neuron.
DOI: 10.1016/j.neuron.2026.09.009

Funding: Canadian Institutes of Health Research

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