Four research projects aimed at defeating Duchenne muscular dystrophy are being awarded a combined $1.7 million this year from Jesse’s Journey, officials with the charity announced late Monday.
The funding is the largest annual amount the London-based charity has doled out since it was founded in 1995, the same year John Davidson, 49, set out to push Jesse, his 15-year-old son and the charity’s namesake, 3,300 kilometres across Ontario in his wheelchair to raise money and find a cure for the life-threatening disorder.
“Every year we just get an enormous amount of projects that are submitted to our granting cycle, which is so encouraging and so hopeful for our families, because it just shows the dedication and the commitment to research in Duchenne muscular dystrophy,” said Nicola Worsfold, director of research and advocacy at Jesse’s Journey.
“We really didn’t know this year if we were going to be in a position to be able to grant what we wanted to this year. But because of the support and the commitment of our donors, our families and all that hard work that’s going on behind the scenes, we’re just really, really excited to be able to grant this amount this year.”
Among the research projects receiving funding is one led by SickKids president and CEO Dr. Ronald Cohn using the revolutionary gene-editing technique CRISPR-Cas9.
Developed by Emmanuelle Charpentier and Jennifer A. Doudna, CRISPR-Cas9 allows for laser-sharp snips in the long strings of DNA, enabling scientists to precisely edit specific genes to remove errors that lead to diseases. The pair received a Nobel Prize in chemistry for their work in 2020.
“(Cohn’s) team has successfully implemented a genome-editing technique in mice that imitates the genetic mutation of a patient with Duchenne,” read a release from Jesses’ Journey. “Using CRISPR-Cas9 technology, they have demonstrated restoration of full functionality to the disease-causing gene for the first time in a living animal (in vivo).”
“While this research is charging ahead in some areas, it has been hampered for patients with duplication mutations. Our research seeks to open up treatment avenues that we previously weren’t able to explore for these patients,” Cohn said in a statement.
Cohn said CRISPR-Cas9 and other precision medicine solutions had “emerged as a beacon of hope” for people with disorders like Duchenne.
“We also, this year, have granted awards to Dr. (Natasha) Chang, who is out of McGill University, and she’s doing some incredible work in stem cell research,” Worsfold said.
“We also have Dr. (Sachiko) Sato who’s at Laval University in Quebec, who’s working on some really interesting science on how to make muscles stronger. We also have international colleagues, Dr. (Niclas) Bengtsson, who’s out of University of Washington School of Medicine, also looking at gene editing in muscle stem cells,” she continued.
“We’re just very fortunate to have brilliant minds who are working on this important science to really help our children … we’re very fortunate and very excited.”
Officials with Jesse’s Journey say the charity has invested more than $14.7 million in research projects around the world since its founding.
Duchenne muscular dystrophy impacts approximately one in every 5,000 boys in Canada. The progressive, intramuscular disorder causes muscle weakness, loss of movement and early death, usually when patients are in their 20s or 30s.
“Even over the last 10 years, there have been a lot of advancements. Our boys are starting to live longer, they’re starting to walk longer,” said Worsfold, herself the mother of a 14-year-old who was diagnosed with Duchenne when he was six.
“When he was diagnosed, it just completely turns your world upside down. You’re basically told that your child will lose his ability to walk in his teens and he may not live past his 30th birthday. So you can imagine how that would completely devastate any family who has that diagnosis,” she said.
“It’s incredible the work that a foundation like Jesse’s Journey … the impact that we can have to really push science.
“We’re looking to push the boundaries of science and push the time. We can’t wait tomorrow. Our children can’t wait 10 years. We can’t wait 20 years. We don’t have that amount of time. Our goal is to not only push innovation in science, but to find the fastest way that we can to find drugs that are going to help our boys and help them live a longer life.”
— With files from Sawyer Bogdan and The Associated Press