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An important step in research into the treatment of muscle diseases: Specialists from a consortium led by Maastricht University have started a trial of treating patients with their own healthy stem cells. These experiments on their own stem cells are unique in the world.
This method offers hope to countless people who suffer from a loss of muscle mass, for example due to cancer or aging, but not all muscle diseases are curable. Because it should be possible to find healthy or repairable stem cells, explains Professor of Genome Analysis Bert Smits at Radio NOS 1 News.
Yesterday, the first two patients with a genetic muscle disorder were given healthy muscle stem cells. Millions of stem cells have been grown outside the body and released into the bloodstream during clinical trials to boost muscle production. “Boost with healthy stem cells that make healthy muscle tissue,” Smits explains. “But stem cells also fuse with existing muscle fibers. So we can also repair existing muscle.”
In this video, the researchers explain how they want to help patients with the new treatment:
The professor stresses that the huge advantage of using your own stem cells is that they are not rejected by the body. This happens with donor cells from other people. “If you want to suppress the rejection of donor cells, you have to use suppressants. These substances also prevent the transplantation of stem cells into the muscle. So they also have a negative effect on the treatment itself.”
The test started yesterday with the lower part of a sick leg. “We compare it to the other leg to see if it has an effect.”
To be able to tell if the inserted stem cells are actually doing what they’re supposed to do, they need to be visible in a muscle biopsy, which is a piece of muscle tissue taken from a muscle for research. “Because they are the body’s own cells, they are completely indistinguishable from the cells they already have, so we give them a color.” For example, under the microscope it can be seen how many new stem cells have reached the muscle.
The Sarah Foundation is one of the co-funders of the research. It was founded in 2016 by the parents of 7-year-old Sarah to raise funds for research into their daughter’s muscle disease, who has the rare muscle disease MDC1A.
However, Sarah herself cannot yet be cured with the new stem cells. She has a genetic defect in her stem cells “so we can’t do it the way we do now.”
The defect must first be corrected. “We’re working on it too. We’re making progress but we’re not at the point where we can do that yet.”
For some other muscle diseases, such as ALS, it’s more complicated. Because it is not always clear where or how the genetic defect occurs. Smeets: “With diseases where it’s a bit unclear where the effect is and how to fix it, it gets a little more difficult, because you have to know that the cells you’re giving back are healthy. So with ALS it’s a little bit more difficult. Because then you don’t know exactly how to make it. healthy”.
In the meantime, the researchers hope that the current study will provide important information about the effect of the treatment. “The doses, how many cells go into the muscle, how you can grow the cells in a safe way, it’s all really a win-win.”
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