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Compassionate Use of Umbilical Cord Stem Cells as Muscular Dystrophy Treatment

Review of "The use of umbilical cord-derived mesenchymal stem cells in patients with muscular dystrophies: results from compassionate use in real-life settings" from STEM CELLS Translational Medicine by Stuart P. Atkinson

Wharton's jelly mesenchymal stem cells (WJ-MSCs) derived from the human umbilical cord possess a similar therapeutic potential to MSCs isolated from the bone marrow [1], with recent research in a mouse model highlighting the relevance of WJ-MSCs to the treatment of muscular disorders [2]. In this study, the authors reported that the secretion of elevated levels of chemokine (C motif) ligand (XCL1) by WJ-MSCs inhibited the induced apoptosis of mouse skeletal myoblast cells, thereby supporting their application as a promising treatment strategy for myopathies and other skeletal muscle diseases.

In a recent STEM CELLS Translational Medicine article, researchers led by Beata Świątkowska-Flis and Dominika Sługocka (Polish Center of Cell Therapy and Immunotherapy in Częstochowa, Częstochowa, Poland) describe the results from their recent evaluation of the compassionate use of WJ-MSCs in patients with muscular dystrophies treated in real-life settings [3]. The results provide evidence that WJ-MSC therapy, and the presumed amelioration of disease-associated oxidative stress, inflammation, mitochondrial dysfunction, and necrosis, represents a reasonable experimental treatment option.

  • Twenty-two muscular dystrophy patients received one to five intravenous and/or intrathecal injections of WJ-MSCs per treatment course in up to two courses every two months
    • Patients comprised eleven men and eleven women of a median age of 33 years
    • Muscular dystrophies included limb-girdle muscular dystrophy (10), facioscapulohumeral muscular dystrophy (6), myotonic dystrophy (1), Becker dystrophy (1), and unspecified disease (4)
    • The four standard doses used were 10, 20, 30, or 40 × 106 WJ-MSCs per injection
  • Analysis of muscle strength using computerized force meters demonstrated heterogeneous responses but also statistically significant improvements in twelve patients
    • These improvements prompted improvements in gait (3) or movement (3) results in six patient
    • Of note, one patient experienced partial deterioration (in lower limbs) during therapy and follow-up
  • Patients tolerated WJ-MSC treatments well
    • Transient headache and lower back pain after the last administration in one patient noted as an adverse effect

While the authors have provided encouraging results, they also highlight several weaknesses in their study, which include the open and uncontrolled design of the analysis, a lack of measurement of secretion of biologically active compounds by WJ-MSCs, the lack of blood test biomarker evaluation to monitor disease progression and safety as well as common clinical tests, and the short and qualitative follow-up.

For more on umbilical cord stem cell treatments for muscular dystrophies, stay tuned to the Stem Cells Portal!


  1. Mattar P and Bieback K, Comparing the Immunomodulatory Properties of Bone Marrow, Adipose Tissue, and Birth-Associated Tissue Mesenchymal Stromal Cells. Frontiers in Immunology 2015;6:560.
  2. Kwon S, Ki SM, Park SE, et al., Anti-apoptotic Effects of Human Wharton's Jelly-derived Mesenchymal Stem Cells on Skeletal Muscle Cells Mediated via Secretion of XCL1. Molecular Therapy 2016;24:1550-1560.
  3. Świątkowska-Flis B, Zdolińska-Malinowska I, Sługocka D, et al., The use of umbilical cord-derived mesenchymal stem cells in patients with muscular dystrophies: Results from compassionate use in real-life settings. STEM CELLS Translational Medicine 2021;10:1372-1383.