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Clinical Trials of Cell Therapy and Regenerative Medicine in Muscular Dystrophies

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Handbook of Stem Cell Applications

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Abstract

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 has 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.

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Abbreviations

CD:

Cluster of differentiation

DAPI:

4′,6-diamidino-2-phenylindole

DMD:

Duchenne muscular dystrophy

DNA:

Deoxyribonucleic acid

FISH:

Fluorescent in situ hybridization

mdx:

X chromosome-linked muscular dystrophy

MTs/SMFs:

Myotubes and/or small muscle fibers

PCR:

Polymerase chain reaction

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Authors and Affiliations

  1. Axe Neurosciences, Research Center of the CHU de Quebec – CHUL and Department of Molecular Medicine, School of Medicine, Laval University, Quebec, QC, Canada

    Daniel Skuk

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  1. Daniel Skuk
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  1. Department of Basic Sciences, Sulaiman Alrajhi University, Al Bukayriyah, Saudi Arabia

    Khawaja H. Haider

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  1. Basic Sciences Department, Sulaiman AlRajhi University, AlQaseem, Saudi Arabia

    Khawaja Husnain Haider Ph.D

  2. Stem Cells and Tissue Engineering Research Group, Faculty of Dentistry, Cairo University, Cairo, Egypt

    Karim M. Fawzy El-Sayed

  3. Oral Medicine and Periodontology Department, Faculty of Dentistry, Cairo University, Cairo, Egypt

    Karim M. Fawzy El-Sayed

  4. Clinic for Conservative Dentistry and Periodontology, School of Dental Medicine, Christian Albrechts University, Kiel, Germany

    Karim M. Fawzy El-Sayed

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Skuk, D. (2024). Clinical Trials of Cell Therapy and Regenerative Medicine in Muscular Dystrophies. In: Haider, K.H. (eds) Handbook of Stem Cell Applications. Springer, Singapore. https://doi.org/10.1007/978-981-99-7119-0_17

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