Mesenchymal stem cell treatment sees success in new muscular dystrophy study

Scientists at the Klara Medical Center (KMC; Czestochowa, Poland) have shown that mesenchymal stem cells (MSCs) can be used for the treatment of muscular dystrophies – a group of progressive muscle diseases caused by a genetic mutation, resulting in muscle weakness.

The MSCs, derived from a substance found in the umbilical cord called Wharton’s jelly (WJ), were able to significantly improve muscle strength with no serious side effects in the latest compassionate-use study.

The results were recently published in the journal STEM CELLS Translational Medicine.

“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,” commented Beata Świątkowska-Flis, unit head at KMC’s Polish Center for Cell Therapies and Immunotherapy, and study leader.

Over 30 unique genes are involved in the pathogenesis of muscular dystrophies, making it difficult to target. Current treatment options are limited to supportive care and drugs such as steroids, which are associated with harmful side effects.

“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,” Świątkowska-Flis explained.

In the study, 22 people with varying types of muscular dystrophies received one to five intravenous and/or intrathecal injections per treatment course, which occurred up to twice a month. Following this, muscle strength was evaluated by using a set of CQ Dynamometer computerized force meters.

“In the group as a whole, we saw significant improvement in several body muscles, including limb, hip, elbow and shoulder,” Świątkowska-Flis reported. “In the most successful case, the patient began moving without a crutch, stopped rehabilitation and rejoined a full-time job.”

With these impressive results, the team now aim to conduct further studies to investigate the use of MSCs in treating 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,” Świątkowska-Flis concluded.

Ref:https://www.regmednet.com/mesenchymal-stem-cell-treatment-sees-success-in-new-muscular-dystrophy-study/کپی لینک کوتاه