Nav: Home

Discovery points to innovative new way to treat Duchenne muscular dystrophy

January 31, 2019

Researchers at The Ottawa Hospital and the University of Ottawa have discovered a new way to treat the loss of muscle function caused by Duchenne muscular dystrophy in animal models of the disease. As reported in Cell Stem Cell, the team restored muscle stem cell function that is impaired in Duchenne muscular dystophy, resulting in efficient regeneration of the muscle and preventing the progressive loss of muscle strength characteristic of the disease.

"This is a huge step forward in developing a new approach for treating Duchenne muscular dystrophy," said Dr. Michael Rudnicki, senior scientist and Director of the Regenerative Medicine Program at The Ottawa Hospital and a professor at the University of Ottawa. "We were able to preserve the muscle strength and function that are usually lost over time in this disease."

Duchenne muscular dystrophy is an inherited disease that weakens the muscles and causes death by the second or third decade of life. About one in 3,600 boys live with this condition, and there is no cure.

Dr. Rudnicki's team previously made the landmark discovery that Duchenne muscular dystrophy affects muscle stem cells, not just the muscle fibers. Muscle stem cells are responsible for repairing muscle fibers after injury and exercise. In people with Duchenne muscular dystrophy, their stem cells do not produce enough of the cells that will become muscle cells. This is because they are missing a protein called dystrophin, which properly orients the cell division needed to form new muscle cells.

The team found that activating a protein called epidermal growth factor receptor (EGFR) could restore this pathway and create new muscle cells, without the need for dystrophin.

The researchers inserted a gene that produced a steady supply of EGF into the muscle of a mouse model of Duchenne muscular dystrophy before symptoms of the disease began. After one month, the treated mice had 18 percent more muscle mass and less muscle scarring than untreated controls. The treated mice also had 30 percent more muscle fibers, and the muscles generated 32 percent more force compared to untreated mice. Muscle function was restored to almost normal levels.

This proof-of-concept study shows that the muscle repair pathway in Duchenne muscular dystrophy is correctable. However, EGF would not be a practical treatment because the protein cannot travel from the bloodstream into the muscles. The researchers' next step is to find a small-molecule drug that triggers this same pathway and can be easily delivered through the bloodstream. They will also look at the long-term effects of triggering this pathway, as the therapy would likely be taken throughout life.

"Duchenne muscular dystrophy is complex, and we will probably need a combination of treatments to address all aspects of the disease," said Dr. Rudnicki.
-end-
Acknowledgements: This research was possible because of generous donations to The Ottawa Hospital for regenerative medicine research. This research was also funded by the US National Institutes for Health, the Canadian Institutes for Health Research, Muscular Dystrophy Canada, the Muscular Dystrophy Association, the Ontario Institute for Regenerative Medicine, and the Stem Cell Network. It was also supported by StemCore Laboratories. Dr. Rudnicki is a founder of Satellos Bioscience Inc., a company that is advancing this research.

Full Reference: EGFR-Aurka Signaling Rescues Polarity and Regeneration Defects in Dystrophin-Deficient Muscle Stem Cells by Increasing Asymmetric Divisions. Yu Xin Wang, Peter Feige, Caroline E. Brun, Bahareh Hekmatnejad, Nicolas A.Dumont, Jean-Marc Renaud, Sharlene Faulkes, Dan Guindon, and Michael A. Rudnicki. Cell Stem Cell. January 31, 2019.

About The Ottawa Hospital: Inspired by research. Driven by compassion. The Ottawa Hospital is one of Canada's largest learning and research hospitals with over 1,100 beds, approximately 12,000 staff and an annual budget of over $1.2 billion. Our focus on research and learning helps us develop new and innovative ways to treat patients and improve care. As a multi-campus hospital, affiliated with the University of Ottawa, we deliver specialized care to the Eastern Ontario region, but our techniques and research discoveries are adopted around the world. We engage the community at all levels to support our vision for better patient care. See http://www.ohri.ca for more information about research at The Ottawa Hospital.

About the University of Ottawa --A crossroads of cultures and ideas: The University of Ottawa is home to over 50,000 students, faculty and staff, who live, work and study in both French and English. Our campus is a crossroads of cultures and ideas, where bold minds come together to inspire game-changing ideas. We are one of Canada's top 10 research universities--our professors and researchers explore new approaches to today's challenges. One of a handful of Canadian universities ranked among the top 200 in the world, we attract exceptional thinkers and welcome diverse perspectives from across the globe. http://www.uottawa.ca

Media Contact: Amelia Buchanan, Senior Communication Specialist, Ottawa Hospital Research Institute; ambuchanan@ohri.ca; Office: 613-798-5555 x 73687; Cell: 613-297-8315

The Ottawa Hospital

Related Stem Cells Articles:

More selective elimination of leukemia stem cells and blood stem cells
Hematopoietic stem cells from a healthy donor can help patients suffering from acute leukemia.
Computer simulations visualize how DNA is recognized to convert cells into stem cells
Researchers of the Hubrecht Institute (KNAW - The Netherlands) and the Max Planck Institute in Münster (Germany) have revealed how an essential protein helps to activate genomic DNA during the conversion of regular adult human cells into stem cells.
First events in stem cells becoming specialized cells needed for organ development
Cell biologists at the University of Toronto shed light on the very first step stem cells go through to turn into the specialized cells that make up organs.
Surprising research result: All immature cells can develop into stem cells
New sensational study conducted at the University of Copenhagen disproves traditional knowledge of stem cell development.
The development of brain stem cells into new nerve cells and why this can lead to cancer
Stem cells are true Jacks-of-all-trades of our bodies, as they can turn into the many different cell types of all organs.
Healthy blood stem cells have as many DNA mutations as leukemic cells
Researchers from the Princess Máxima Center for Pediatric Oncology have shown that the number of mutations in healthy and leukemic blood stem cells does not differ.
New method grows brain cells from stem cells quickly and efficiently
Researchers at Lund University in Sweden have developed a faster method to generate functional brain cells, called astrocytes, from embryonic stem cells.
NUS researchers confine mature cells to turn them into stem cells
Recent research led by Professor G.V. Shivashankar of the Mechanobiology Institute at the National University of Singapore and the FIRC Institute of Molecular Oncology in Italy, has revealed that mature cells can be reprogrammed into re-deployable stem cells without direct genetic modification -- by confining them to a defined geometric space for an extended period of time.
Researchers develop a new method for turning skin cells into pluripotent stem cells
Researchers at the University of Helsinki, Finland, and Karolinska Institutet, Sweden, have for the first time succeeded in converting human skin cells into pluripotent stem cells by activating the cell's own genes.
In mice, stem cells seem to work in fighting obesity! What about stem cells in humans?
This release aims to summarize the available literature in regard to the effect of Mesenchymal Stem Cells transplantation on obesity and related comorbidities from the animal model.
More Stem Cells News and Stem Cells Current Events

Trending Science News

Current Coronavirus (COVID-19) News

Top Science Podcasts

We have hand picked the top science podcasts of 2020.
Now Playing: TED Radio Hour

Processing The Pandemic
Between the pandemic and America's reckoning with racism and police brutality, many of us are anxious, angry, and depressed. This hour, TED Fellow and writer Laurel Braitman helps us process it all.
Now Playing: Science for the People

#568 Poker Face Psychology
Anyone who's seen pop culture depictions of poker might think statistics and math is the only way to get ahead. But no, there's psychology too. Author Maria Konnikova took her Ph.D. in psychology to the poker table, and turned out to be good. So good, she went pro in poker, and learned all about her own biases on the way. We're talking about her new book "The Biggest Bluff: How I Learned to Pay Attention, Master Myself, and Win".
Now Playing: Radiolab

Invisible Allies
As scientists have been scrambling to find new and better ways to treat covid-19, they've come across some unexpected allies. Invisible and primordial, these protectors have been with us all along. And they just might help us to better weather this viral storm. To kick things off, we travel through time from a homeless shelter to a military hospital, pondering the pandemic-fighting power of the sun. And then, we dive deep into the periodic table to look at how a simple element might actually be a microbe's biggest foe. This episode was reported by Simon Adler and Molly Webster, and produced by Annie McEwen and Pat Walters. Support Radiolab today at Radiolab.org/donate.