Defeat Duchenne Canada is proud to announce its gift of $300,000 towards a breakthrough clinical trial, which may empower local researchers to discover a therapeutic solution to the degenerative disease Duchenne muscular dystrophy.
The clinical trial is building upon promising Phase I research involving myoblast transplantation. This therapeutic approach applies to all Duchenne patients, independently of the mutation of the patient. The successful progression of this trial could mark an incredible breakthrough for boys who are indiscriminately targeted by Duchenne muscular dystrophy. It may also produce a treatment for other recessive dystrophies.
Two London area researchers are heading up this novel trial: Neurologist Dr. Craig Campbell and Dr. Guido Filler, an expert in immunosuppression, are working in collaboration with Dr. Jacques P. Tremblay, a molecular biologist at Laval University in Québec.
This game-changing research would not be possible without Defeat Duchenne Canada’s monetary support. “Without the financial support from Defeat Duchenne Canada, I would have been forced to terminate the clinical trial of myoblast transplantation,” explains Dr. Jacques P. Tremblay. “This would have been a terrible loss for those affected by Duchenne because the results of the trials have, thus far, been extremely positive. The Phase I clinical trial had shown that the missing protein, dystrophin, was restored in the muscles of patients who received the myoblast transplantation. The first patient who participated in the current Phase I/II clinical trial showed significant stabilization after the graft was placed.”
The trial is now moving forward with the recruitment of patients who are more than 16 years old, reaching out to families who are currently coping with this menacing disease, which takes away boys’ and young men’s ability to walk, and eventually their lives.
“Duchenne is a devastating disease. While our community has made great strides over the past two decades, we still have so much further to go to enhance the lives of the boys and families coping with its life-altering consequences,” shares the Executive Director of Defeat Duchenne Canada, Perry Esler. “Our organization relies on people’s willingness to support solutions we may not have even imagined yet. It’s a big ask, but as you can see with these trials, our donors’ willingness to have faith in our researchers can have transformational results.”
With your help, we can defeat Duchenne. To learn more about why give to Duchenne research, click here.