Jérôme Frenette: OPG for DMD
Professor Jérôme Frenette, himself lost two of his children due to a genetic disease. It’s, therefore, a decision from the heart that leads him to conduct today his research program on inherited diseases, like Duchenne Muscular Dystrophy (DMD). Professor Frenette understands very well the suffering of the people afflicted with the disease and their families. This understanding provides him with an additional motivation to conduct his work. This is an excellent conclusion to our web video series “Portrait of DMD.” We hope it will help you understand the functions and benefits of osteoprotegerin for DMD.
Jérôme Frenette: «I’ve lost two children to a genetic disease and I know very well what parents are currently experiencing with Duchenne muscular dystrophy, the suffering, the stress caused and the hope that is linked to these diseases. »
About Jérôme Frenette
Professor Jérôme Frenette is a researcher with a background in physical therapy and post-graduate training in muscle physiology and immunology at the University of California in Los-Angeles. The focus of his research program is to understand the link between osteoporosis and muscle atrophy/disease through one common pathway, i.e. the RANK/RANKL/OPG pathway. His team is developing new drugs that would potentially alleviate, in tandem, osteoporosis and muscle degeneration in Duchenne muscular dystrophy patients or other forms of bone and muscle diseases.
Can you explain your osteoprotegerin (OPG) research project?
Jérôme Frenette: We are working on the role of a protein, a protein that protects the bone, and we are interested in this protein. The big question is: could this bone protein also preserve muscle tissue? We know that the phenomenon of atrophy, of muscular dysfunction, happens at the same time as osteoporosis, so we tried to group these two diseases, bone, muscle, with a single protein which is osteoprotegerin, which, as its name says, protects the bone, and our work demonstrates that it also protects muscle tissue.
What would be the benefits of this treatment for DMD?
Jérôme Frenette: The benefit that these children could have in the very short term is to gain strength. Our work shows that there are very significant strength gains. The degeneration process, which begins at birth and continues until 20-25 years, could be delayed for several years. So, with a bone protein, we could succeed in protecting the muscle and the bone with our treatment.
When are the clinical trials for patients?
Jérôme Frenette: We’re relatively lucky because the molecule is relatively well characterized. Work has been done with a well-known company, Amgen, who have developed this protein. They even brought it to clinical trials in postmenopausal women. So, I think we can go much faster because there have already been clinical trials on this protein. A window of approximately 2 to 5 years may be considered for clinical treatment in children.
More information about Professor Jérôme Frenette: Centre de recherche du CHU de Québec
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