The use of umbilical cord‐derived mesenchymal stem cells in patients with muscular dystrophies: Results from compassionate use in real‐life settings

Muscular dystrophies are genetically determined progressive diseases with no cause‐related 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) to 22 patients with muscular dystrophies. Patients received one to five intravenous and/or intrathecal injections per treatment course in up to two courses every 2 months. Four standard doses of 10, 20, 30, or 40 × 10(6) UC‐MSCs per injection were used; the approximate dose per kilogram was 1 × 10(6) UC‐MSCs. Muscle strength was measured with a set of CQ Dynamometer computerized force meters (CQ Elektronik System, Czernica, Poland). Statistical analysis of muscle strength in the whole group showed significant improvement in the right upper limb (+4.0 N); left hip straightening (+4.5 N) and adduction (+0.5 N); right hip straightening (+1.0 N), bending (+7.5 N), and adduction (+2.5 N); right knee straightening (+8.5 N); left shoulder revocation (+13.0 N), straightening (+5.5 N), and bending (+6.5 N); right shoulder adduction (+3.0 N), revocation (+10.5 N), and bending (+5 N); and right elbow straightening (+9.5 N); all these differences were statistically significant. In six patients (27.3%) these changes led to improvement in gait analysis or movement scale result. Only one patient experienced transient headache and lower back pain after the last administration. In conclusion, UC‐MSC therapy may be considered as a therapeutic option for these patients.