First in border-crossing collaborative project for rare muscle disorders

Source photo: Maastricht UMC+

A successful collaboration and the pooling of knowledge with neighboring countries Germany and Belgium, has led to the border-crossing project GYM (Generate Your Muscle) being able to proclaim a world first. On Wednesday, July 13 and Thursday, July 28, the first two patients with a hereditary muscle disorder of the energy metabolism will be administered healthy body-own muscle stem cells. These are the very first trials worldwide using such stem cells for systemic induction of muscle mass. It offers hope for countless patients in the search for a treatment for inherited and often very rare diseases.

GYM

Generate Your Muscle (GYM) is a project that came about within the Interreg Euregio Maas-Rhine program. It concerns a cooperation between the universities of Aachen - Liège - Hasselt - Leuven - Maastricht and Scannexus in the field of stem cell therapy for rare (muscle) diseases. The project is supported by patient organizations - and/or foundations representing patients, such as the Foundation for Sara.

Unieke trial

Within the GYM project, researchers want to promote the creation of muscle mass and tissue by using healthy, body-own muscle stem cells. The uniqueness of this trial is that although the patients have a hereditary muscle disorder of energy metabolism, their muscle stem cells have virtually no hereditary defect. These stem cells can therefore be used directly as therapy. This clinical trial should demonstrate the safety of the treatment and will provide an initial picture of the effectiveness of the procedure. The aim of Generate Your Muscle is to test the therapy after safety especially for effectiveness in patients with this and other inherited muscle disorders, whereby if necessary the hereditary defect must be repaired or compensated.

A collaboration across the border

Generate Your Muscle is made possible by a grant from the European Interreg program. Researchers from Maastricht UMC+ are working together with universities in Hasselt, Liege and Leuven, with the University Hospital of 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 co-financing, as are several patient organizations. In total, there is a research budget of 2.8 million euros.

Interreg Euregio Maas-Rijn

Euregio Maas-Rhine is one of the 60 Interreg areas in Europe. In these border areas, subsidies are made available to stimulate cross-border cooperation. The goal is to stimulate innovation and sustainability in border regions and reduce barriers caused by borders. We invest in projects around innovation, economy and social inclusion in the border region between the Netherlands, Belgium and Germany. The Province of Limburg is one of the partner regions of Interreg Euregion Meuse-Rhine.

Source: Province of Limburg