Muscle Stem Cell Therapy Project
Innovative stem cell therapy for the recovery of muscle mass and strength
A consortium led by scientists from Maastricht UMC + and Maastricht University has received more than three million euros in European funding (Interreg V EMR) for the development of autologous stem cell therapy for treating genetic and non-genetic muscle diseases. Under the name Generate Your Muscle (GYM), the researchers will use modified stem cells to compensate the loss of muscle mass and muscle strength. In a spin-off company the production of the specific stem cells will be scaled up.
Maintaining muscle mass and strength is essential for a healthy life. However, genetic muscle disorders (such as muscular dystrophies) cause the muscles to lose their function. In addition, cancer patients often have to deal with a loss of muscle strength and people simply lose muscle mass as they age. For all these disorders, there are no treatments available to restore this muscle breakdown.
Muscle Stem Cell therapy
The consortium involved has previously discovered that a certain type of muscle stem cells (so-called mesoangioblasts) can promote the production of healthy muscle fibers. These stem cells are collected from the patient, corrected in the event of a genetic disorder, and grown to larger numbers. The body's own corrected muscle stem cells are then brought back into the bloodstream, after which they move to the affected muscle tissue and ensure recovery of the affected muscle. Ultimately, the production of muscle stem cells will be scaled-up in a new spin-off company, with the aim of making the therapy available as broadly, cheaply, and quickly as possible.
The GYM project (Generate Your Muscle) has 3 key objectives:
- Defining safety and effectiveness of muscle stem cell therapy by clinical trials.
A specific group of patients with a muscle disease due to an mtDNA mutation makes healthy muscle stem cells, called mesoangioblasts, without the genetic defect. Their autologous mesoangioblasts are being cultured and administered in clinical trials to test safety and efficacy. - Defining the potential for muscle stem cell therapy
- Patients with a broad variety of diseases, like cancer and sarcopenia, suffer from loss of muscle mass and power. Mesoangioblasts will be collected from these patients to determine their therapeutic potential.
- Patients with neuromuscular diseases due to a genetic defect in a nuclear gene make only mesoangioblasts containing this defect, so this will be repaired using CRISPR/Cas9 technology or compensated in an alternative way. This will be developed initially for LAMA2-MD and Myotonic Dystrophy Type 1. - Industrial production of mesoangioblasts
A feasibility study has shown that setting up a company producing mesoangioblasts for therapy is a realistic option. For that the effectiveness (energy-boost) and specificity of the muscle stem cell therapy product will be optimized, the engraftment of the administered mesoangioblasts improved and the production process shortened.
The 3 million euro project GYM is being realized within the framework of Interreg V-A Euregio Meuse-Rhine with the support of the European Regional Development Fund of the European Union, the local authorities and the universities and companies involved. Project leaders Maastricht UMC+ and Maastricht University work within GYM together with Hasselt University, Université de Liège, KULeuven and the Uniklinik RWTH Aachen and with the companies Scannexus and Kenko International. The GYM project builds on the synergy between academic researchers and the biotechnology industry in the Euregio Meuse-Rhine and on the profitable climate that has been created for regenerative medicine in this region.
More information about the GYM Project.
