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, the maintenance of 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 to the affected muscle tissue on their own 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 aim of Generate Your Muscle is to test the effectiveness of the therapy after safety in patients with these and other hereditary muscle disorders, whereby 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 is first carried out 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.”
By: National Care Guide