Phase IIa clinical trial started at MUMC: administration of patient’s own muscle stem cells in m.3243A>G mutation carriers
More than half of the m.3243A>G mutation carriers become quickly fatigued and have muscle weakness, and at present no treatment is available for this. In the Maastricht UMC+ (the Netherlands) we have already been investigating for some years whether the administration of muscle stem cells can lead to an improvement in muscle function. To prevent rejection reactions, we want to use the patient’s own muscle stem cells for this. In our laboratory we have shown that around half of the m.3243A>G mutation carriers have mutation-free stem cells. Last year we carried out a safety study in which the patient’s own muscle stem cells were administered 1x in the lower leg of m.3243A>G-mutation carriers in the MUMC. However, 3 administrations are necessary to achieve an effective dose. The purpose of this phase IIa clinical trial is to investigate whether 3x administration of patient’s own muscle stem cells in the arm leads to an improvement in muscle strength and a reduction in fatigue. This therapeutic administration is at present only carried out in a study context.
If you are an m.3243A>G mutation carrier and would like more information on this study, then contact us via email@example.com.
For more information on the completed phase I study: