Ottawa researchers reverse autism symptoms in mice by fixing blood vessel defects
A six-year study at The Ottawa Hospital and University of Ottawa found that restoring blood flow to the brain reversed behavioural symptoms in adult mice with autism-linked genetic mutations.
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Researchers at The Ottawa Hospital and University of Ottawa have reversed some autism symptoms in mice by fixing blood vessel defects in the brain — a discovery that could eventually lead to a drug treatment for humans.
The six-year study, led by Dr. Baptiste Lacoste, found that the problem lay in endothelial cells, which line blood vessels and normally ensure quick blood flow to active parts of the brain. In mice with a 16p11.2 deletion — one of the most common genetic mutations seen in autism — these cells weren't functioning properly. "The endothelial cells were not functioning properly," Lacoste said. "They have what it takes to stay alive. They have the machinery, all the elements the cell should have, but they looked a little bit tired, a bit lazy. They do not really respond to stimulations or challenges."
When the team activated certain receptors in the blood vessels of these mice, blood flow increased and the cells' function was restored. The result: the mice's behavioural symptoms — including hyperactivity, repetitive movements, and motor learning problems — reversed. "In the whole animal, what it means is the brain starts to function better, and it has an impact on the behaviour of the animal," Lacoste said.
The drug used in the study is already approved for human use in Japan and South Korea to treat dry-eye syndrome, making it a potential candidate for clinical trials. However, researchers must first test how the drug interacts with other parts of the body — a process that will take several more years. Safety and efficacy testing with larger patient groups would follow.
No current drug treatment exists for the core symptoms of autism. Lacoste estimated it will likely take about 10 years before a drug based on this research becomes available to patients. "It's a long road ahead, but it's opening up new avenues for treatment and giving a bit of hope to the community," he said. The study was driven largely by former PhD student Dr. Julie Ouellette.
How long will drug interaction testing actually take, and what budget has been allocated?
Will the research move to testing in younger mice, and if so, when?
We'll update this story as answers emerge.
The facts
How long did the Ottawa autism study take?
The study at The Ottawa Hospital and University of Ottawa took six years.
What genetic mutation did the researchers study in mice?
The researchers studied mice with a 16p11.2 deletion, one of the most common genetic mutations seen in autism.
What symptoms reversed in the mice when blood flow was restored?
The mice's hyperactivity, repetitive movements, and motor learning problems reversed when blood flow to the brain was restored.
How soon might a drug based on this research be available to patients?
Dr. Baptiste Lacoste estimated it will likely take about 10 years before a drug based on this research becomes available to patients.
