Defeat Duchenne Canada Grants $1M to DMD Treatment Research

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by Mary Chapman |

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Defeat Duchenne Canada is investing $1.14 million in three research projects aimed at providing better treatments for Duchenne muscular dystrophy, the most common type of muscular dystrophy and one that affects about 1 in 3,500 male births.

The trio of three-year projects joins six other research efforts that are in the second and third year of funding. This latest funding round brings to $16 million the total awarded in the organization’s 27-year history.

Research is the road to hope for Duchenne muscular dystrophy, but this future can only be achieved through collaboration with our scientific community,” Nicola Worsfold, national vice president of research at Defeat Duchenne Canada, said in a press release. “We have proudly funded 56 projects from world-renowned researchers focused on discovering investigational treatments to improve the health and quality of life of children and young adults living with Duchenne.”

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There is no cure for DMD, although advancements in care have made it possible for patients to live longer and more fulfilling lives. Current guidelines recommend that people with DMD be regularly treated with anti-inflammatory medications called corticosteroids. For patients with specific disease-causing mutations, there are also multiple approved exon-skipping treatments, which help to produce a working dystrophin protein.

Defeat Duchenne Canada grant recipients for this year include Michael Rudnicki, PhD, director of the Regenerative Medicine Program at the Ottawa Hospital Research Institute, Ottawa, Ontario. He’s discovered a protein called Wnt7a that could become a DMD treatment candidate due to its ability to repair muscle damage. Rudnicki will work to deliver the protein via circulation to all bodily muscles. In particular, his team is investigating exosomes, small vesicles found naturally in human bodies and which carry information between cells.

Through the project, titled “Systemic Delivery of Wnt7a for Treating Duchenne Muscular Dystrophy,” Rudnicki and his lab will engineer new versions of exosomes carrying Wnt7a for delivery to muscle cells. The hope is that the prospective therapy will stimulate the muscles’ own capacity for regeneration and growth, independent of the underlying mutation.

Another project, “Phenotypic Screening of Deimmunized DMD Gene Therapy Vectors,” will be led by Guy Odom, PhD, of the University of Washington School of Medicine. Gene therapy works by introducing a smaller iteration of dystrophin, the protein lacking in DMD patients. However, due to an immune response against the introduced dystrophin form, some clinical trial participants have had severe side effects.

Odom’s team hopes to skirt such a immune response by engineering certain variants of utrophin, a protein structurally and functionally similar to dystrophin, to allow improved function. The overarching aim is to develop a safer and more productive version of a therapeutic protein for DMD.

The other grant goes to Louis Kunkel, PhD, of Boston Children’s Hospital. Kunkel is widely recognized for his 1986 identification of DMD as the causative gene in Duchenne. This current project is titled “A Novel Therapeutic Approach for Duchenne Muscular Dystrophy Centered on the NOTCH Pathway Modulation of Muscle Satellite Cells.”

A DMD hallmark is a decline in motor function due to muscle breakdown as a result of a lack of dystrophin. Kunkel’s team wants to see whether muscle regeneration can be improved independently of the amount of dystrophin protein present.

The lab has discovered a biochemical pathway, Notch, a master regulator of muscle satellite cells responsible for muscle regeneration. The aim is to use these findings to develop a DMD therapy for all patients, regardless of their specific mutation.