Bringing novel genetic therapies to treat MS, ALS and other disorders

Dr. Mark Tarnopolsky
Photo courtesy of McMaster University

CIHR Foundation Grant Recipient

Dr. Mark Tarnopolsky
Pediatrics & Medicine
McMaster University

Dr. Tarnopolsky's Research

Lack of exercise is a major threat to health and can increase the effects of aging and chronic diseases. While regular physical activity reduces the risk of chronic diseases and physical disability, the reasons behind these benefits are not clearly understood.

Many parts of the body work and "talk" together to produce the positive effects of exercise. Dr. Mark Tarnopolsky and his research team have coined the term "exerkines," to describe this communication system between cells.

The central aim of this research is to understand the nature of exerkines: how they work and how they might be used to fight disease.  By learning more about how exercise works, Dr. Tarnopolsky's research could also lead to the development of new therapies for people who are unable to exercise.

Supporting advances in muscular diseases

The research team plans to evaluate the potential of exerkines as a therapy to reduce muscular problems associated with aging, obesity and Type-2 diabetes. The team also plans to explore the potential of modified versions of exosomes, also known as small lipid vesicles that are secreted by cells, as genetic and protein therapy for people with genetic disorders such as mitochondrial disease, muscular dystrophy, ALS, and others.

The team hopes to be able to use the results to develop and test therapies (artificial exercise) that would improve the lives of people with genetic diseases. The work is expected to lead to the development of patents, biotech companies and partnerships. Dr. Tarnopolsky's work has led to the formation of EXERKINETM, a biotechnology company created in conjunction with McMaster University. There are plans underway to establish a manufacturing facility in Ontario to produce treatments for mitochondrial disease, muscular dystrophy and a wide range of genetic disorders.

About Dr. Tarnopolsky

Dr. Tarnopolsky is a graduate of McMaster University, first earning a degree in physical education in 1985, a medical degree in 1988, and a PhD in cell biology and metabolism in 1991. He is a Professor and Division Head of Neuromuscular and Neurometabolic Disorders in the Department of Pediatrics at McMaster University.  He has been the recipient of many honours and awards, including the Premier's Research Excellence Award in 2000 and the Muscular Dystrophy Association's Dr. David Green Award in 2004-2005, and honour awards from the Canadian Society of Exercise Physiology (2012) and the International Research Group on Biochemistry of Exercise (2015). Since 2010, he has held a Chair in Neuromuscular Disorders from McMaster Children's Hospital/Hamilton Health Sciences Foundation.

"This grant money will allow us to truly bring novel genetic therapies from the bench to the bedside. We expect that novel therapies will be in the clinic during the seven-year tenure of the grant."

Dr. Mark Tarnopolsky

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