Hope for muscle strength recovery in rare genetic muscle disorders

Under the name Generate Your Muscle, researchers wish to boost the production of muscle mass and tissue by healthy and endogenous muscle stem cells.

A consortium led by scientists from Maastricht UMC+ has begun clinical trials of an innovative stem cell therapy against, among other things, hereditary muscle disorders and muscle breakdown in the elderly. On July 13 and 28, the first two patients with a genetic muscle disorder caused by energy metabolism will be given healthy body muscle stem cells. It is the first worldwide trial to use such stem cells to systematically stimulate muscle mass. This provides hope to countless patients in search of a cure for genetic diseases that are often very rare.

Under the name Generate Your Muscle (GYM), researchers want to boost the production of muscle mass and tissue by endogenous muscle stem cells. According to scientists, the use of muscle stem cells in the body has a significant advantage in avoiding the necessary risks associated with transplantation of donor cells. What’s also unique about the trial is that even though the patients had a genetic disorder of energy metabolism, their muscle stem cells hardly had a genetic defect. So these stem cells can be used as a direct treatment. Clinical trial should demonstrate the safety of the treatment and will provide a first impression of the effectiveness of the intervention.

After all, maintaining energy, muscle mass and strength is essential to a healthy life. Genetic muscle disorders, such as muscular dystrophy, lead to a loss of muscle function. In addition, cancer patients and the elderly often experience severe loss of muscle mass. So far, there is no effective way to treat these muscle disorders.

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Stem Cell Therapy
The scientists involved saw that a specific type of muscle stem cell (the so-called mesoblast) could promote the production of healthy muscle fibers after being administered into the bloodstream. They also showed that this group of patients had healthy stem cells that could be used directly for treatment. In the experiment, muscle stem cells are transplanted into larger numbers of muscle biopsies. The body’s own stem cells are then reintroduced into the bloodstream, after which they travel to the affected muscle tissue on their own and ensure immediate recovery. Part of Maastricht’s preliminary research has been published in the journal Stem Cell Research and Therapy.

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The goal of Generate Your Muscle is to test the effectiveness of post-safe treatment in patients with these and other hereditary muscle disorders, where the genetic defect must be repaired or compensated if necessary. “This new clinical research is a huge step forward for this group of patients,” says project leader Prof. Dr. Bert Smits. In addition to affecting genetic muscle diseases, scientists also want to investigate the additional potential of stem cells in other forms of muscle breakdown (such as cancer or aging). To this end, further research is being conducted first into the quality and quantity of mesoblastoma cells for these groups of patients. Smits: “Ultimately, we also want to increase muscle stem cell production and make treatment more widely available, at a lower cost, and more quickly.”

Building your muscles is made possible thanks to the support of the European Interreg programme. UMC+ researchers work with universities in Hasselt, Liege and Leuven, with the University Hospital Aachen and with Scannexus and Kenko International. In addition, many patient organizations are involved that fight for a healthier future for patients with rare diseases. The province of Limburg is a partner in the form of a co-financier, as well as several patient organizations. In total, there is a research budget of 2.8 million euros.

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Sarah Foundation
The Sarah Foundation is one of the pioneers in accelerating research into muscle stem cell therapy. It was specifically established to more rapidly approach the treatment of children with the rare muscle disease MDC1A. MDC1A is a form of progressive muscular dystrophy, as a result of which patients (often young adults) experience muscle weakness throughout the body. So the parents of seven-year-old Sarah are raising money to support Smits’ research. So, Bram Verbrough, Sarah’s father and the foundation’s president, is thrilled with this important step in the investigation: for five years, all of the Foundation’s volunteers have worked hard to get to this point. If these trials are successful, we are not there yet, but the way to treat our daughter and all other patients is open.”

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Megan Vasquez

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