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Monday, November 1 2021
Press release

New hope for Duchenne muscular dystrophy patients

A research team at CHU Sainte-Justine discovers a new therapeutic agent

MONTREAL, November 1, 2021 – A research team led by Nicolas Dumont, researcher at CHU Sainte-Justine and professor at the Université de Montréal, discovered a new therapeutic molecule, Resolvin-D2. It has the potential to significantly improve the quality of life of patients with Duchenne muscular dystrophy (DMD) by stimulating muscle stem cell activity.

DMD is a rare genetic disease characterized by progressive muscle degeneration, affecting 1 in 4000 boys. The first symptoms appear between ages 3 and 5, and as the child grows, there is an irreversible loss of muscle function. Currently, the administration of glucocorticoids from early childhood is the standard patient treatment.

The results of this study were reported in Nature Communications.

DMD is characterized by muscle fragility that, at the slightest contraction, causes muscle fibres to tear. A vicious cycle of injury and healing begins. Over time, chronic inflammation develops, attacking healthy tissue and further destroying the muscles.

“Glucocorticoids have powerful anti-inflammatory effects that are widely used in clinical practice. To date, they are the only treatment that can effectively slow the progression of the disease,” explains Professor Nicolas Dumont, researcher at CHU Sainte-Justine and associate professor in the School of Rehabilitation at the Université de Montréal. However, when this molecule is taken chronically, its relative toxicity can cause significant side effects, such as osteoporosis, growth retardation in children or acute anxiety. In addition, glucocorticoids can directly attack the muscles and decrease their ability to heal. Thus, the beneficial effect on patients’ muscle function is undermined by harmful side effects.

“Our initial goal was to target a molecule that would be more effective than the current treatment and less toxic,” explains Junio Dort, first author of the study and a postdoctoral fellow at CHU Sainte-Justine. We reviewed molecules with anti-inflammatory properties similar to glucocorticoids and selected about ten. By testing these molecules in preclinical laboratory models, Resolvin-D2 proved to be the best candidate.” 

The Resolvin-D2 molecule is not only a very effective anti-inflammatory agent, but unlike glucocorticoids, it also stimulates the activity of muscle stem cells that are responsible for muscle healing.

“This property is extremely promising because the function of muscle stem cells is diminished in DMD. By restoring their ability to heal, it would therefore be possible to extend the life expectancy of patients, which is currently between 20 and 30 years,” emphasizes Professor Dumont.

“We’re continuing our research to create a more stable Resolvin-D2 molecule, one that degrades less rapidly and can be administered orally. Nonetheless, this molecule represents a strong therapeutic potential, which could translate into an improved quality of life for patients and a reduced economic burden for society,” concludes Professor Dumont.

About this study

The article "Resolvin-D2 targets myogenic cells and improves muscle regeneration in Duchenne Muscular Dystrophy" written by Junio Dort was published in  November 2021 in Nature Communications. The study was funded by the Canadian Institutes of Health Research, the Natural Sciences and Engineering Research Council of Canada, the Canada Foundation for Innovation, the Stem Cell Network, the CHU Sainte-Justine Foundation, the Fondation du Grand défi Pierre Lavoie and the Réseau de thérapie cellulaire, tissulaire et génique – ThéCell.

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About the CHU Sainte-Justine Research Centre

The CHU Sainte-Justine Research Centre is a leading mother-child research institution affiliated with the Université de Montréal. It brings together more than 210 research investigators, including over 110 clinician-scientists, as well as 450 graduate and postgraduate students focused on finding innovative prevention means, faster and less invasive treatments, as well as personalized approaches to medicine. The centre is part of CHU Sainte-Justine, which is the largest mother-child centre in Canada.

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CHU Sainte-Justine
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Maude Hoffmann
Communications, CHU Sainte-Justine Research Centre
maude.hoffmann.hsj@ssss.gouv.qc.ca

Media resource person:

Florence Meney
Assistant to the Director of Communications – External Media Liaison
CHU Sainte-Justine
Tel: 514-755-2516
florence.meney.hsj@ssss.gouv.qc.ca 

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Updated on 11/1/2021
Created on 10/29/2021
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