Clinical Trials of Cell Therapy and Regenerative Medicine in Muscular Dystrophies

Cell therapy could form part of the future treatment of degenerative disorders of genetic origin that affect skeletal muscles, such as muscular dystrophies. This strategy could produce clinical benefits by fulfilling three fundamental properties: (a) genetic complementation, which makes it possible to restore in the patient’s muscle fibers the proteins whose deficiency of genetic origin is the cause of the disease, (b) formation of new muscle fibers, which open the possibility of reconstituting the skeletal muscle lost by the degenerative process of muscular dystrophy, and (c) generation of new satellite cells, which would allow restoring the pool of muscle-specific stem cells. Extensive research in rodents and other animal models has studied these properties. However, it has been the translational studies in macaques that have most helped define the transplantation protocols that have proven to be reproducible in clinical trials. Although muscle fiber regeneration Muscle fiber regenerationhas been shown to depend on satellite cells through their activation and conversion into myoblasts, several cell types other than satellite cells and myoblasts have been reported in animal studies to possess some of the myogenic properties necessary for cell therapy of muscular dystrophies. However, only the administration of satellite cell-derived myoblasts has so far proven to be able to fulfill these properties in clinical trials.