Results of a compassionate-use research study launched in STEM CELLS Translational Medicine reveal appealing outcomes for dealing with muscular dystrophies with mesenchymal stem cells (MSCs) originated from Wharton’s jelly (WJ), a compound discovered in the umbilical cable. Led by physicians at Klara Medical Center (KMC), Czestochowa, Poland, these WJ-MSC treatments led to considerable enhancement in a number of body muscles in the majority of the clients, without any severe adverse effects.
“Administration of WJ-MSCs in neurological indications is controversial; still, this paper shows that cell therapy is a reasonable experimental treatment option, although the eligibility criteria for treatment needs to be optimized,” stated Beata Świątkowska-Flis, M.D., Ph.D., neurologist, system head at KMC’s Polish Center for Cell Therapies and Immunotherapy and research study leader.
Muscular dystrophies include a group of muscle illness triggered by anomalies in an individual’s genes that lead to progressive muscle losing and weak point. This can ultimately cause death from breathing failure or cardiomyopathy.
“There are many kinds of muscular dystrophy, each affecting specific muscle groups, with signs and symptoms appearing at different ages, and varying in severity. Although over 30 unique genes are involved in their pathogeneses, a similar mutation in the same gene may cause a wide range of phenotypes, and distinct genes may be responsible for one identical phenotype. Because of this heterogeneity, pharmacologic treatments are limited,” stated Dr. Świątkowska-Flis.
The existing alternatives consist of helpful care and drugs. While steroids are the gold requirement in pharmacotherapy, they can have considerable adverse effects, amongst them weight gain, adolescence hold-up, behavioral concerns, and bone fractures.
“Although stem cells cannot resolve the underlying genetic conditions, their wide-ranging therapeutic properties may ameliorate the consequences of the involved mutations. Our study describes the clinical outcomes of the compassionate use of WJ-MSCs in patients with muscular dystrophies treated in real-life settings,” Dr. Świątkowska-Flis stated.
The research study included 22 individuals with differing kinds of muscular dystrophies. The group was similarly divided in between male and female, and the mean age was 33. Each individual got 1-5 intravenous and/or intrathecal injections per treatment course in as much as 2 courses every 2 months. Muscle strength was then evaluated by utilizing a set of CQ Dynamometer electronic force meters.
“In the group as a whole, we saw significant improvement in several body muscles, including limb, hip, elbow and shoulder,” Dr. Świątkowska-Flis reported. “In the most successful case, the patient began moving without a crutch, stopped rehabilitation, and rejoined a full-time job.”
While these outcomes are outstanding, the physicians warn that it is prematurely to identify the position of MSCs in dealing with muscular dystrophies.
“For example, we don’t know how long the therapeutic effect will last; it might be that the therapy should be repeated cyclically. Further studies are needed to optimize stem cell therapy both in terms of treatment scheme in a long period and possible synergy with pharmacological drugs and/or rehabilitation. Still, we believe the results are cautiously encouraging, especially in light of no other efficient treatment,” Dr. Świątkowska-Flis stated.
“While use of mesenchymal stem cells in neurological indications is controversial, this study shows that cell therapy is a reasonable experimental treatment option for this rare group of muscle diseases,” stated Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. “No side effects were observed and the data stemming from this study is potentially encouraging and of interest. We look forward to the continuation of this research to further document clinical efficacy.”
Reference: “The use of umbilical cord-derived mesenchymal stem cells in patients with muscular dystrophies: results from compassionate use in real-life settings” by Beata Świątkowska-Flis, Izabela Zdolińska-Malinowska, Dominika Sługocka and Dariusz Boruczkowski, 27 July 2021, Stem Cells Translational Medicine.
DOI: 10.1002/sctm.21-0027
