Plate Nº 87 · recorded September 30, 2026
Neuroscience & MindReported finding
Fixing Brain Blood Vessels Reverses Autism Symptoms in Mice
Ottawa researchers reversed hyperactivity and repetitive movements in autistic-model mice by activating a receptor on brain blood vessel cells with an already-approved drug.
By Priya Raman3 min read652 words
In brief
- Researchers at The Ottawa Hospital reversed behavioral symptoms in a mouse model of autism by activating the P2Y2 receptor on brain blood vessel cells.
- The drug used is already approved in Japan and South Korea to treat dry eye syndrome in humans.
- The study was conducted only in adult mice; clinical trials in humans have not yet taken place.
Researchers at The Ottawa Hospital and the University of Ottawa have reversed certain behavioral symptoms in a mouse model of autism by repairing a defect in the brain's blood vessels. The findings, published in the journal Neuron, point to a promising new treatment target — though the road from mice to medicine remains long.
Autism is a neurodevelopmental condition with widely varying characteristics. Some behavioral symptoms can make daily life more challenging. While many people with autism have found ways to manage these symptoms, no drug treatment currently exists.
"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, a senior scientist at The Ottawa Hospital and a professor at the University of Ottawa.
A problem in the plumbing
The story began with an earlier discovery. Lacoste's team had previously found that blood vessels in the brain don't work properly in mouse models carrying a 16p11.2 deletion — one of the most common genetic mutations seen in autism. His group was the first to examine what was happening in the brain's blood vessels in this context.
In the new study, the researchers traced the problem to cells lining the blood vessels, called endothelial cells. Their job is to make sure blood quickly reaches the parts of the brain that are active. This responsive blood supply is essential for proper brain function.
In mice with the 16p11.2 deletion, the endothelial cells simply don't respond fast enough. This dysfunction appears early in brain development, and behavioral symptoms emerge later in life. These include hyperactivity, repetitive movements, and impaired motor learning.
A missing receptor
The new study, driven by former Ph.D. student Dr. Julie Ouellette, asked two questions: what exactly is wrong with these cells, and can it be fixed?
The team found that the endothelial cells carried only half the normal level of a molecule called ATP. ATP is best known as the cell's energy currency, but here it plays a different role. The cell was also missing its docking target for ATP — a surface protein called the P2Y2 receptor.
When the researchers activated the P2Y2 receptor with a drug, several things happened. The cells regained their function. Blood flow in the brain increased. And the behavioral symptoms reversed — in adult mice whose symptoms were already well established.
The compound the team used is already approved for human use in Japan and South Korea, where it treats dry eye syndrome.
"It's as if these cells are asleep, and now we can wake them up," Lacoste said. "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."
That possibility of a one-time, lasting effect remains unconfirmed. The researchers plan to test it in future work.
What comes next
The study has clear limits worth keeping in mind. It looked only at mice, and only at adult animals. That adults responded is encouraging, because it suggests targeting P2Y2 can reverse symptoms after they have taken hold rather than merely preventing them. Next, the team plans to treat mice earlier in life to see whether early intervention offers additional benefits.
The researchers have filed a patent application for using P2Y2 activation in blood vessels to treat autism symptoms. They are interested in pursuing drug development with the eventual aim of clinical trials in people.
For now, the results mark an early but notable step: the first evidence that repairing the brain's blood supply, rather than its neurons directly, can undo autism-related behaviors — at least in one mouse model of the condition.
Publication details: "Purinergic receptor activation rectifies autism-associated endothelial dysfunction," Neuron (2026). DOI: 10.1016/j.neuron.2026.09.009.
via Medical Xpress (Source)
More from Priya Raman
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Senior reporter covering industry trends and analytics at SciBeat.
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