Hope for recovery of muscle strength in rare, hereditary muscle disorders

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

A consortium led by scientists from Maastricht UMC+ is starting clinical trials for an innovative stem cell therapy against, among other things, genetic muscle disorders and muscle breakdown in the elderly. On July 13 and 28, the first two patients with a hereditary muscle disorder of energy metabolism will be given healthy body’s own muscle stem cells. It is the very first trial worldwide in which such stem cells are used to induce muscle mass systemically. This offers hope for countless patients in the search for a treatment for hereditary and often very rare diseases.

Under the name Generate Your Muscle (GYM), the researchers want to promote the production of muscle mass and tissue by means of healthy, endogenous muscle stem cells. According to the scientists, the use of the body’s own muscle stem cells has the great advantage that the necessary risks associated with transplantation of donor cells are avoided. What is also unique about the trial is that although the patients have a hereditary muscle disorder of the energy metabolism, their muscle stem cells hardly have the hereditary defect. These stem cells can therefore be used directly as therapy. The 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 for a healthy life. Genetic muscle disorders such as muscular dystrophies cause the muscles to lose their function. In addition, cancer patients and the elderly often suffer from severe muscle mass loss. To date, there is no method to effectively treat these muscle disorders.

Stamceltherapie
The scientists involved saw that a certain type of muscle stem cells (so-called mesoangioblasts) can promote the production of healthy muscle fibers after administration to the bloodstream. They had also shown that this patient group had healthy stem cells that could be used directly for therapy. For the trial, the muscle stem cells are grown into larger numbers from a muscle biopsy. The body’s own stem cells are then reintroduced into the bloodstream, after which they move on their own to the affected muscle tissue and ensure recovery on the spot. Part of the Maastricht preliminary research has already been published in the journal “Stem Cell Research and Therapy”.

scale up
The goal of Generate Your Muscle is to test the effectiveness of the therapy after safety in patients with these and other hereditary muscle disorders, where the hereditary defect must be repaired or compensated if necessary. “This new clinical study is therefore a major step forward for this group of patients,” says project leader Prof. Dr. Bert Smeets. In addition to the effect in genetic muscle diseases, the scientists also want to investigate the further potential of the stem cells in other forms of muscle breakdown (such as cancer or aging). To this end, further research will first be conducted into the quality and quantity of the mesoangioblasts of these groups of patients. Smeets: “Ultimately, we also want to scale up the production of muscle stem cells and make the therapy available as broadly, cheaply and quickly as possible.”

Collaboration
Generate Your Muscle is made possible by a subsidy from the European Interreg programme. Researchers from Maastricht UMC+ are working together with universities in Hasselt, Liège and Leuven, with the university hospital in Aachen and with the companies Scannexus and Kenko International. In addition, several patient organizations are involved that fight for a healthier future for patients of rare diseases. The province of Limburg is a partner in the form of a co-financier, as are various patient organisations. In total, there is a research budget of 2.8 million euros.

Foundation for Sara
One of the champions for an acceleration in research into muscle stem cell therapy is the Sara Foundation. Specially founded to bring a treatment for children with the rare muscle disease MDC1A closer faster. MDC1A is a form of progressive muscular dystrophy, as a result of which (often young) patients suffer from muscle weakness throughout the body. The parents of seven-year-old Sara are therefore raising money to support Smeets’ research. Bram Verbrugge, father of Sara and chairman of the foundation, is therefore pleased with this important step in the research: ,,For five years all volunteers of the foundation have worked very hard to get to this point. If these trials are successful, we are not there yet, but the way to a treatment for our daughter and all other patients is open.”

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