Abstract 6 Combined Umbilical Cord Mesenchymal Stromal/Stem Cell Application For Primary Torsion Dystonia Treatment (Case Report)

Abstract

Abstract Introduction Primary torsion dystonia (PTD) is a neurological disorder characterized by sustained or intermittent muscle contractions causing abnormal movements and/or postures with severe disability of young patients. Traditional therapies for PTD include oral anti-parkinsonism drugs, botulinum neurotoxin, pallidotomy and deep brain stimulation. Stem cell therapy is another promising treatment option, due to its neuroregenerative properties. Objectives The objective of the study was to investigate the effect of combined (intrathecal and intravenous) administration of umbilical cord derive stromal/stem cells (UC-MCS) on PTD reduction. Methods Patient M, born on 25.06.2004, fell ill gradually, 8 years ago developed signs of muscle dystonia, especially in right hand. Genetic tests were carried out, which revealed mutations in the DYT-1 gene. The diagnosis of primary torsion dystonia was established. Conservative treatment was not effective. The patient was offered treatment in the frame of research scientific program "To investigate the effectiveness of regenerative cell technologies in the neurosurgical treatment of patients with demyelinating diseases of the CNS and cerebral palsy. № 0119U000112" UC-MSCs for this study were isolated from healthy human umbilical cord and prepared due to the minimal criteria established by the Mesenchymal and Tissue Stem Cell Committee of the International Society for Cellular Therapy. 3 injections of UC-MSC were performed with time interval of 6 month (intravenously and intrathecally in a dose of 6 × 107at each injection). Intrathecal administration was performed according to the typical method of performing lumbar puncture in compliance with the rules of asepsis and antiseptics in the operation room using local anesthesia. Results Prior to stem cells injection, the patient's Unified Dystonia Rating Scale (UDRS) dystonia movement score was 15, which progressively decreased after procedure to 13, 10, 7 and 4 after 6, 12, 18 and 24 months respectively. There was a significant improvement in the patient's quality of life. Discussion UC-MSC significantly reduce dystonia in patient. This result is probably achieved due to the neuroregenerative and neurotrophic effects of UC-MSCs. Study results suggest the need to provide larger multicenter randomized studies with more sophisticated methods of investigations.