CQDM and the Government of Quebec invest in collaborative research to advance a promising new treatment for mitochondrial DNA depletion disorders

Source: The Institute and CQDM
November 18, 2025

Kenneth Myers, MD, PhD, Scientist at the Research Institute of the McGill University Health Centre (The Institute), has received new funding to advance research into an innovative therapy that could transform the lives of patients living with mitochondrial DNA depletion disorders—rare and often fatal diseases for which no effective treatment currently exists.

The project has earned a grant from the Government of Quebec’s Ministère de l’Économie, de l’Innovation et de l’Énergie (MEIE), awarded by the Consortium de recherche biopharmaceutique du Québec (CQDM) through its SynergiQc program. With total funding of $347,973 over two years, this initiative is part of a broader $2.25-million investment by CQDM to accelerate the development of cutting-edge therapies for complex diseases.

Addressing an urgent unmet need

Mitochondrial DNA depletion disorders (MDDDs) are severe genetic conditions that impair the body’s ability to produce energy at the cellular level. Caused by defects in the enzymes that maintain mitochondrial DNA, these disorders can lead to a wide range of serious symptoms—from seizures and liver failure to muscle weakness, hearing loss and cardiac problems. For many affected children, the disease progresses rapidly, often resulting in early mortality.

In collaboration with Liam Pharma for Mitochondrial Disease Inc., a Quebec-based biotechnology company, Dr. Myers, Scientist in the Child Health and Human Development Program at The Institute, and his research team will assess whether supplementation with deoxycytidine and deoxythymidine—two natural building blocks of DNA—can enhance mitochondrial function and energy production.

Building on promising results

Earlier pilot studies conducted by Dr. Myers’ team have already shown encouraging results: the treatment has been well tolerated over a 24-month period, with early signs of clinical improvement in children and young adults affected by MDDDs. The current project aims to expand this work by enrolling additional patients and gathering the data needed to confirm both safety and efficacy.

If successful, this therapy could become one of the first disease-modifying treatments for mitochondrial DNA depletion disorders—potentially transforming care for patients worldwide.

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