News | 20 January 2020

Innovative stem cell therapy for recovering muscle mass and strength

European funding to develop treatment for genetic muscle disorders

A consortium led by researchers at Maastricht UMC+ and Maastricht University has received three million euros in European funding to develop a stem cell therapy for genetic muscle disorders and muscle atrophy in the elderly, among other things. Under the name Generate Your Muscle (GYM), the researchers aim to use modified stem cells to stimulate the generation of muscle mass and tissue. Production of the specific stem cells will be scaled up in a future spin-off firm.

iStockiStockMaintaining muscle mass and strength is essential for a healthy lifestyle. Genetic muscle disorders (such as muscular dystrophies), however, cause the muscles to lose function. Cancer patients also often lose muscle strength, while even healthy people experience a loss of muscle mass as they get older. At the moment, there are no methods for treating muscle atrophy of this kind.

Stem cell therapy
Researchers previously found that a certain type of muscle stem cell (mesoangioblasts) can stimulate the generation of healthy muscle fibres. These stem cells are harvested from the patient, modified in the event of a genetic disorder, and cultured. The stem cells are then reintroduced into the patient's bloodstream and subsequently migrate on their own to the affected muscle tissue, where they stimulate recovery. The Maastricht team recently published a report on its preliminary research in the scientific journal Stem Cell Research and Therapy.

Scaling up
The goal of Generate Your Muscle is to continue testing the safety and effectiveness of the therapy in patients with hereditary muscle disorders. 'This new clinical trial represents a major step forward for this group of patients,' says principal investigator Prof. Bert Smeets. In addition to its impact on genetic muscle disorders, the researchers also aim to investigate the potential of stem cell therapy for other forms of muscle atrophy (for example caused by cancer and ageing). They will begin by conducting more research into the specific mesoangioblasts of these patient groups. 'In the end, we would like to scale up the production of muscle stem cells in a new spin-off firm,' says Smeets. 'Our aim in doing so is to make the therapy available as widely, inexpensively and quickly as possible.'

Foundation for Sara
One of the champions of accelerating research into muscle stem cell therapy is the 'Stichting voor Sara' (Foundation for Sara). The foundation supports research into therapies for children with MDC1A, a rare muscle disorder. MDC1A is a type of progressive muscular dystrophy in which patients (many of them children) suffer from muscle weakness throughout the body. The parents of four-year-old Sara are raising money to support the modification of MDC1A patients' muscle stem cells as part of Prof. Smeets' broader research so that they are suitable for this type of therapy. Bram Verbrugge, Sara's father and chairman of the foundation, is therefore delighted with this new step in the clinical trial: 'When we set up our foundation, there was no treatment available for our daughter and no one was investigating her muscle disorder. The hard work of many volunteers and numerous donors made it possible to start the trial in Maastricht. The funding gives the researchers much more capacity to carry out their promising research. This could lead to a treatment for the incurable and life-threatening muscular disorder MDC1A and it may also address the consequences of numerous other diseases and age-related deficiencies. We couldn't have wished for more.'

Generate Your Muscle is made possible by funding from the European Union's Interreg programme. Maastricht UMC+ and Maastricht University have joined forces in this programme with the universities in Hasselt, Liège and Leuven in Belgium, Aachen University Hospital in Germany, and Scannexus and Kenko International.