Our real case of treatment Primary torsion dystonia using stem cells therapy
Introduction: Primary torsion dystonia (PTD) is a neurological disorder characterized by prolonged or intermittent muscle contractions causing abnormal movements and/or postures with severe disability in young patients. Traditional treatments for PDD include oral antiparkinsonian drugs, botulinum neurotoxin, pallidotomy, and electrical stimulation of deep brain structures. Stem cell therapy is another promising area of treatment due to its neuroregenerative properties.
Objectives: The purpose of the study was to study the effect of combined (endolumbar, i.e. injection into the spinal canal of the spine and intravenous) administration of stem/stromal cells obtained from the umbilical cord (umbilical cord MSC) on reducing the symptoms of primary torsion dystonia.
Methods: Patient M., born on June 25, 2004, the disease developed gradually, 8 years ago signs of muscle dystonia appeared, especially in the right hand. Genetic tests were performed, which revealed mutations in the DYT-1 gene. A diagnosis of primary torsion dystonia was established. Conservative treatment was not effective. The patient was offered treatment as part of the research program “Investigation of the effectiveness of regenerative cell technologies in the neurosurgical treatment of patients with demyelinating diseases of the CNS and cerebral palsy.
As part of this study, mesenchymal stem cells were cultured from a healthy human umbilical cord and prepared according to the minimum criteria established by the International Association of Gene and Cell Therapy.3 injections of umbilical cord MSCs were administered with an interval of 6 months (intravenous and endolumbar at a dose of 6 × 10^7 per injection).
Endolumbar insertion was performed in accordance with the standard method of performing a lumbar puncture with compliance with the rules of asepsis and antiseptics in the operating room using local anesthesia.
Results: Before stem cell administration, the patient’s motor dystonia score on the Unified Dystonia Rating Scale (UDRS) was 15 points, which gradually decreased after the procedure to 13, 10, 7, and totaly 4 at 6, 12, 18, and 24 months, respectively. A significant improvement in the patient’s quality of life was observed.
Discussion: MSCs of the umbilical cord significantly reduce the clinical manifestations of dystonia. This result was achieved due to the neuroregenerative and neurotrophic effects of umbilical cord MSCs. The results of the study indicate the need for larger multicenter randomized studies using more advanced research methods.