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Reduced Symptoms of Huntington's Disease Shown in Mice: Promising Breakthrough in Research

OTTAWA, CANADA - In a groundbreaking study, researchers at the University of Ottawa Brain and Mind Research Institute have discovered a potential treatment for Huntington's disease (HD), a devastating neurological disorder that affects thousands of Canadians. Dr. Stephen Ferguson and his team have successfully reduced HD symptoms in mice, offering hope for future treatments in humans.

Often referred to as a combination of Alzheimer's, Parkinson's, and ALS, Huntington's disease affects approximately one in 7,000 Canadians, with one in 1,000 individuals having a personal connection to someone suffering from the condition. The diagnosis of HD is particularly devastating due to the lack of a cure. However, Dr. Ferguson's recent research could change the landscape of HD treatment.


As a professor in the Department of Cellular and Molecular Medicine at the Faculty of Medicine, Dr. Ferguson specializes in studying cell communication and protein transfer between cells. HD patients experience a buildup of a mutated protein called "huntingtin," which leads to cell death and progressively worsens their condition over a span of 15 to 20 years.

Drawing inspiration from successful research in Alzheimer's disease, Dr. Ferguson hypothesized that blocking a receptor in the brain, known as mGluR5, could impede the accumulation of the mutant huntingtin protein and halt the progression of HD. The results of the study, recently published in Science Signaling, provide compelling evidence in support of this hypothesis.

The researchers administered a compound called CTEP to mice with HD, which effectively blocked mGluR5. Additionally, CTEP activated a pathway that facilitated the breakdown of the accumulated mutant huntingtin proteins. As a result, the mice demonstrated a reduction in brain tissue cell death and a decrease in Huntington pathology.

Explaining the significance of the findings, Dr. Ferguson said, "CTEP works to reduce HD-like symptoms in mice because it affects the levels of huntingtin in mouse brains. It also turns on the pathways that are currently turned off in someone who has Huntington disease."

Bev Heim-Myers, CEO of the Huntington Society of Canada, emphasized the transformative potential of Dr. Ferguson's research and its impact on HD studies. She expressed, "The answers we find will likely lead to better understanding of treatments for other neurological diseases such as Parkinson's, Alzheimer's, and ALS."

The breakthrough in HD research by Dr. Ferguson and his team offers a glimmer of hope for those affected by this debilitating disease. As scientists continue to explore the possibilities presented by this discovery, the future looks promising for finding effective treatments and, eventually, a cure for Huntington's disease.


Photo credit: Taylor Jacobs

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