This text is part of the special Research section
Muscular dystrophy consists of a collection of 160 serious genetic neuromuscular diseases that lead to progressive deterioration of muscle strength over the years, thus limiting the motor and respiratory abilities of patients who suffer from it. At present, there is no curative treatment to counter his diagnosis.
Although certain interventions and prescriptions can relieve the discomforts and symptoms, the degenerative disease offers a reduced life expectancy; about 30 years, sometimes less. Since 2014, Professor Florian Bentzinger’s team from the University of Sherbrooke has been collaborating with the Nestlé Institute of Health Sciences on an avenue of cell therapy that could change the lives of those affected.
Holder of the CRMUS Chair in the regenerative medicine of skeletal muscle, Professor Bentzinger has been working for almost ten years on an innovative idea, that of grafting healthy stem cells into diseased muscle to allow them to add their healthy genome. “This is a particularity of skeletal muscle stem cells: in the context of a transplant, they can fuse with the fibers of a diseased cell,” explains the researcher. Because the stem cell that is transplanted is able to fuse with this muscle fiber, it can add its nucleus to it with all the normal and healthy genetic information, which makes it possible to stabilize the diseased fibers in the dystrophic muscles”, continues -he.
To do this, the research team uses induced pluripotent stem cells (iPSCs), human cells, often from skin or blood, which are reprogrammed in the laboratory. In 2012, Japanese researcher Shinya Yamanaka won the Nobel Prize for Medicine for this discovery, which is now considered a major advance in the world of science.
“iPSC cells are used all over the research world to produce other types of cells because they are pluripotent, that is, with the right protocol, they can produce any cell “, explains Professor Bentzigner. Thanks to iPSC cells, purchased from banks, it is therefore possible to produce an almost unlimited number of muscle stem cells necessary for the protocol established by the team from the University of Sherbrooke. She is also working on forms of muscular dystrophy that affect children, including Duchenne dystrophy, one of the most frequent diagnoses, as well as two rarer forms.
Towards lifelong treatment?
The researcher and his team published their protocol last June in The Embo Journal. This shows how to treat cultured iPSC cells to develop into a healthy muscle stem cell. The great advances of the protocol, says Professor Bentzinger, lie in the speed of its execution, which is carried out in two weeks instead of the three to five usually necessary for its purity of 99% as well as its production in suspension which, at the instead of being carried out on cell plates, can be carried out in solution.
For the moment, the tests carried out on mice are “very favorable”, specifies the professor. One of the reasons for his enthusiasm is the observation that once the transplant is carried out, a portion of the muscle stem cells remain active in the muscle and continue to divide, which generates a continuous production of new stem cells. healthy muscle that can, over time, fuse with diseased cells. “If, one day, we are able to develop a treatment, that would mean that we wouldn’t need to constantly inject new stem cells to have the fibers corrected. We could put in just once: it’s like a transplant for life! » he marks.
However, for the moment, the protocol is far from being applied in clinical testing on human patients. Before getting there, you have to think about transplantation, that is, the injection of cells into the muscle, but also about possible side effects and safety protocols. All of this is without mentioning the various levels of government approval to contend with. ” It is still early. When patients affected by muscular dystrophy contact me, I always explain that what we are doing is basic research. We are heading in the direction of a real treatment, but we are not there yet”, underlines the researcher.
Despite everything, Professor Bentzinger remains optimistic. “All the discoveries that led to clinical treatment started like this. With this publication of the protocol, we hope that clinicians and researchers will want to use it and develop it to go further,” he summarizes.
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