New uOBMRI research has potential for treatment of Huntington’s patients
Anyone who has seen the devastating effects of Huntington disease (HD) first-hand wouldn’t be surprised that the Huntington Society of Canada likens it to having Alzheimer’s, Parkinson’s and ALS all in one disease. One in 7,000 Canadians suffer HD, while one in 1,000 know someone who does.
What makes a diagnosis of HD particularly difficult to hear is that there is currently no cure. But new research published by Dr. Stephen Ferguson of the University of Ottawa Brain and Mind Research Institute has shown reduced Huntington disease (HD) symptoms in mice, results with great potential for treatment in humans.
A professor in the Department of Cellular and Molecular Medicine at the Faculty of Medicine, Dr. Ferguson studies how cells communicate with each other, often done via the transfer of proteins to another cell’s protein receptor.
Patients with HD experience a buildup of a particular mutated form of a protein, referred to as the ‘huntingtin’ protein. Cell death results, leading to progressive incapacitation and death within 15-20 years. Based on past research successes with Alzheimer’s, Dr. Ferguson and his team correctly hypothesized that blocking a receptor in the brain, called mGluR5, would block the buildup of the mutated huntingtin protein and prevent the progression of Huntington’s disease. Their results were published recently in Science Signaling.
The researchers gave mice with HD a compound called CTEP, which blocks mGluR5. CTEP also activates a pathway that stimulates the breakdown of the built-up mutant huntingtin proteins. The mice showed a reduction in cell death in their brain tissue, and decreased Huntington pathology in their brains.
“CTEP works to reduce HD-like symptoms in mice because it affects the levels of huntingtin in mouse brains,” says Dr. Ferguson. “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, explains the potential of the work of Dr. Ferguson’s team and others to transform HD research. “The answers we find will likely lead to better understanding of treatments for other neurological diseases such as Parkinson’s, Alzheimer’s and ALS,” she says.
Photo credit: Taylor Jacobs