Muscle stem cell therapy

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GYM (Generate Your Muscle) aims to develop and commercially produce a stem cell therapy for recovery of muscle mass and strength in patients with muscle disease and muscle breakdown. GYM  will make the EMR a leader in stem cell therapy for muscle disease. The challenges the GYM project addresses impact both the vulnerable population and the socio-economic status of the EMR.  1. GYM combats the major health care problem of loss of muscle mass and power, which impacts the EMR more than other regions. Retaining muscle mass and strength is important for a long, healthy life, but: - Genetic muscle diseases lead to serious loss of function in 1 in 1,000 people and often premature death.  - Muscle breakdown is an important negative predictor in cancer patients and determines both the chance of recovery after surgery and the quality of life. - Muscle breakdown affects 10-20% of the older population, leading to loss of function, reduced quality of life and sometimes even death. For each case, no effective treatment exists and the development of a generic therapy has an enormous impact in preventing severe suffering and improving quality of life. In addition, the economic significance is big. For genetic muscle disease, the costs are at least €500,000 per patient, while those of age-related muscle loss already amount to. 1.5% of health care costs. This is even a bigger issue in the EMR, having a higher degree of aging and lower life expectancy, especially in specifc, vulnerable areas. Insufficient physical exercise, overweight, smoking (cancer), age and poor socio-economic status are key determing factors. GYM has the potential to combat the accompanying loss of muscle mass and to improve quality of life and life expectancy and save health care costs. 2. GYM will improve the socioeconomic status of the EMR, compensating the loss of old industry. A higher socio-economic status will generate high-quality jobs, a better environment and improved quality of life. GYM will contribute to this by fostering cross-border collaborations and establishing a highly innovative company in the EMR for the production of muscle stem cells, which  can become an international player, making use of regional supply companies.  Cross-border cooperation is key to this project due to available synergistic expertise and complementary infrastructure on each site of the border in knowledge institutions as well as in companies.  The GYM treatment consists of administering large numbers of healthy, autologous muscle stem cells, called mesoangioblasts as autologous somatic Cell Therapy Medicinal Product (asCTMP), to the patient's blood. These mesoangioblasts migrate to  affected muscles, growing  into healthy muscle fibers and compensating loss of muscle mass and strength. Based on succesful preclinical studies and production acoording to Good Manufacturing Practice (GMP), a phase I/II clinical (Technological Readiness Level [TRL] 6/7) can already be executed.  The project has 3 objectives: 1. Defining safety and partial effectiveness of muscle stem cell therapy (Phase I / II Clinical Trial) A specific group of patients with a genetic muscle disease make healthy mesoangioblasts without the genetic defect. Therefore, their autologous mesoangioblasts can be administered directly after culture. GYM will perform a phase I / II clinical trial (TRL6/7) for this patient group, aimed at safety and (partially) effectiveness. 2. Defining the market for muscle stem cell therapy. - Large groups of patients with a broad variety of diseases, like cancer, sarcopenia and stroke, suffer from loss of muscle mass and power. Mesoangioblast will be collected from these patients to determine their therapeutic potential. The treatment is tested preclinically in a mouse model with colon cancer.  - Patients  with muscle diseases due to a genetic defect in a nuclear gene make only mesoangioblasts containing this defect and this first needs to be repaired. After correction and showing safety, and compliance with GMP, this treatment can be applicable to all genetic muscle diseases. 3. Industrial production of mesoangioblasts. A feasibility study has been carried out, which shows that setting up a company is a realistic option, provided that a good IP position is built up. The latter is achieved through method development, where on the one hand the effectiveness and specificity of the muscle stem cell therapy is increased and on the other hand the production process is shortened and optimized. The company will be established as spin off during the project. The momentum exists in the EMR to become a key player in muscle stem cell therapy, embedded in the excellent frontal area of ​​regenerative medicine, this region aims to be. II (Technological Readiness Level [TRL] 6.

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