The Use of Umbilical Cord-Derived Mesenchymal Stem Cells in Patients with Muscular Dystrophies: Results from Compassionate Use in Real-Life Settings

Abstract

Muscular dystrophies are genetically determined progressive diseases with no causerelated treatment and limited supportive treatment. Although stem cells cannot resolve the underlying genetic conditions, their wide-ranging therapeutic properties may ameliorate the consequences of the involved mutations (oxidative stress, inflammation, mitochondrial dysfunction, necrosis). In this study, we administered advanced therapy medicinal product containing umbilical cord-derived mesenchymal stem cells (UC-MSCs)

Fresh Umbilical Cord Blood—A Source of Multipotent Stem Cells, Collection, Banking, Cryopreservation, and Ethical Concerns

“…However, the presence of CD4+ and CD25+ T cells, and Foxp3 in umbilical cord blood and peripheral blood need more clarity, with various authors presenting differing claims [ 101 , 102 , 103 ]. Similarly, individuals with muscular dystrophies improved significantly in limb strengthening, stretching, bending, and straightening, with 27.3% improving their gait as well [ 70 ]. Three alopecia patients responded well by sprouting their hair from the 53rd to the 226th day of treatment.…”

253rd ENMC international workshop: Striated muscle laminopathies - natural history and clinical trial readiness. 24–26 June 2022, Hoofddorp, the Netherlands

“…From a therapeutic point of view, improvement (or inhibition) of MuSC function is an attractive therapeutic strategy for the treatment of SMLs' MuSC population by transient p38 inhibition, increasing the regeneration potential of aged mice [17]. Transplantation of wild type MuSC or mesenchymal stem cells has shown benefits in different muscular dystrophies [18][19][20]. Unfortunately, no major information on using stem cell-based strategies as potential therapies for SMLs has been reported.…”

Unproven cell interventions in Poland and the exploitation of European Union law on advanced therapy medicinal products

“…Analyses of PubMed revealed several publications from private institutions that offer commercial therapies ( Barczewska et al., 2019 , 2020 ; Siwek et al., 2020 ; Boruczkowski and Zdolińska-Malinowska, 2019a , 2019b ; Świątkowska-Flis et al., 2021 ). However, the studies were not randomized, the patient groups analyzed were heterogeneous in terms of disease and age, the patients received various numbers of injections, the “control” groups did not exist or were created from publicly available sources from numerous unrelated studies, and the methods for monitoring patients were poor.…”