ScienceIndependently confirmed2 publishers2 min readPublished
Restoring brain blood flow reversed autism-linked behaviours in adult mice
Researchers at The Ottawa Hospital reversed autism-linked behaviours in 16p11.2 mice by restoring blood flow in brain vessels that held half the normal ATP. The drug that did it is already approved for people in Japan and South Korea, though for dry eye, not autism.
The Scientist · Science desk
What happened
- The dysfunction starts in the endothelial cells lining the brain's smallest blood vessels, the capillaries, and not in the neurons those vessels feed.
- In the 16p11.2 mice, those vessels cannot open on demand to supply active neural circuits, because their P2Y2 receptor signalling is impaired.
- Switching the P2Y2 receptor back on reversed hyperactivity, repetitive movements and motor-learning deficits.
- The results were published on September 30 in the journal Neuron.
Why it matters
- capability No approved drug targets the biology behind these autism symptoms, so a specific vascular receptor gives drug developers a defined target.
- precedent The team frames the compound's existing human approval as an accelerated route to trials, shorter than developing a molecule from scratch.
- constraint Because the behaviour was measured in one autism-linked variant in mice, the work does not show the vascular route matters for autism's other causes or in people.
For years, autism research concentrated on neurons and the synapses that wire them together [5]. Lacoste's lab had found something different in mice carrying the 16p11.2 deletion: their blood vessels fail to develop and work normally [10]. The earlier work did not show which way the effect ran. Sluggish vessels might be a byproduct of a differently built brain, or they might drive the behaviour themselves.
The explanation the team lands on runs through ATP. Inside a cell it is fuel; outside the cell, ATP is a signal that binds the P2Y2 receptor on the vessel wall and triggers dilation [11]. First author Julie Ouellette and colleagues gave the mice a compound built to bind and switch that receptor on [9]. It restored healthy calcium signalling inside the endothelial cells and brought responsive blood flow back across the brain [12].
The vessels' failure to respond first shows up during early development [13], the window when a brain is being built. The treatment worked in animals that were already fully grown [14]. That points to a deficit that can still be reversed after development is over.
How far this vascular explanation reaches is unsettled. The 16p11.2 microdeletion is one of the most common copy-number variants linked to autism [17], but it is one variant, and the behavioural work was done in mice.
"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 Dr. Lacoste [18].
What to watch
- Whether a P2Y2 agonist produces any behavioural effect in a human trial, and in which autism populations.
- Whether the same vascular defect turns up in autism-linked variants other than 16p11.2.
- How such a compound would be delivered to the brain if it were tested for autism.
Clarity's read
What the record supports and how the coverage leans. The claims behind it follow.
Reality
- Evidence55
- Adoption
- Insufficient
- Hype gap+35
- Incentives55
- Confidence60
Claim ledger
Ranked by verification strength, evidence, and original report placement.
- [1]
Researchers at The Ottawa Hospital and the University of Ottawa reversed core behavioral symptoms in a mouse model of autism by restoring healthy blood vessel function in the brain.
- [2]
Endothelial cells in the 16p11.2 mouse model possessed only half the normal physiological levels of adenosine triphosphate (ATP).
- [3]
The P2Y2-activating compound used is already approved for human clinical use in Japan and South Korea for dry eye disease.
- [4]
The root of the dysfunction lies in brain endothelial cells, the single-cell layer that lines the inner lumen of cerebral capillaries.
- [5]
Neuroscientists studying autism have traditionally concentrated almost entirely on neurons and synaptic wiring; Lacoste's work challenged that neuro-centric focus by examining the cerebral vasculature.
- [6]
Brain endothelial cells in mice carrying the 16p11.2 deletion fail to deliver rapid, responsive blood flow to active neural circuits because of impaired P2Y2 purinergic receptor signaling.
- [7]
Pharmacologically activating the endothelial P2Y2 receptor reversed established behaviors including hyperactivity, repetitive movements, and motor learning impairments.
- [8]
The study was published September 30 in the journal Neuron.
- [9]
The study's first author is Dr. Julie Ouellette; the work was led by Dr. Baptiste Lacoste, senior scientist at The Ottawa Hospital and professor at the University of Ottawa.
- [10]
In previous work, Lacoste's laboratory showed that blood vessels fail to develop and function normally in mouse models carrying the 16p11.2 microdeletion.
- [11]
ATP, best known as an intracellular energy currency, also acts as an extracellular signaling molecule that binds the P2Y2 receptor on endothelial surfaces to trigger vasodilation.
- [12]
The P2Y2 agonist reestablished healthy calcium dynamics inside the endothelial cells and repaired responsive blood flow throughout the brain.
- [13]
In the 16p11.2 mice, the vessels' hemodynamic responsiveness first falters during early developmental windows.
- [14]
The reversal of established behaviors occurred even in fully mature adult mice.
- [15]
The researchers described the drug's existing approval as opening an accelerated path toward translational development.
- [16]
To date, no targeted pharmacological therapies exist to address the underlying neurobiological mechanisms behind these autism symptoms.
- [17]
The 16p11.2 microdeletion is one of the most prevalent copy number variations associated with human autism.
- [18]
"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 Dr. Lacoste.
Sources
2 independent publishers whose own reporting we read for this story.
- genengnews.comAutism Target Discovered in Endothelial Cells in Genetic Deletion Mouse Model
1 article · September 30, 2026
- neurosciencenews.comRestoring Brain Blood Vessels Reverses Autism-Related Behaviors in Mice
1 article · September 30, 2026
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Topics
- Neurovascular couplingFollow
- Preclinical researchFollow
- Drug RepurposingFollow
- Autism spectrum disorderFollow