Long-Term Clinical Efficacy of Human Umbilical Cord Blood Mononuclear Cell Transplantation by Lateral Atlanto-Occipital Space Puncture (Gong’s Puncture) for the Treatment of Multiple System Atrophy

• Published in: Cell transplantation Vol. 31; p. 9636897221136553
• Main Authors: Gong, Dianrong, Wang, Weifei, Yuan, Xiaoling, Yu, Haiyan, Zhao, Min
• Format: Journal Article
• Language: English
• Published: Los Angeles, CA SAGE Publications 01.01.2022; Sage Publications Ltd; SAGE Publishing
• Subjects: Adverse events; Atrophy; Autonomic nervous system; Basal ganglia; Brain diseases; Brain injury; Cell therapy; Central nervous system diseases; Cerebellar ataxia; Cerebellum; Cord blood; Fetal Blood; Hematopoietic Stem Cell Transplantation; Humans; Leukocytes (mononuclear); Mesenchyme; Movement disorders; Multiple System Atrophy - therapy; Neurodegenerative diseases; Original; Prospective Studies; Punctures; Stem cells; Transplantation; Treatment Outcome; Umbilical cord; neurodegenerative disease; multiple system atrophy; cord blood mononuclear cells; parkinsonism; Gong’s puncture; lateral atlanto-occipital space puncture
• ISSN: 0963-6897; 1555-3892
• DOI: 10.1177/09636897221136553

Multiple system atrophy (MSA) is a sporadic, progressive neurodegenerative disease characterized by autonomic nervous dysfunction with parkinsonism or cerebellar ataxia. Mesenchymal stem cell therapy or transplantation of human umbilical cord blood mononuclear cells (hUCB-MCs) may inhibit progression in MSA, but long-term studies are lacking. In addition, injection of stem cells via lateral atlanto-occipital space puncture (LASP, or Gong’s puncture) may efficiently target areas of brain injury and avoid the disadvantages of other methods. This prospective study investigated the long-term clinical efficacy of transplantation of hUCB-MCs via LASP for the treatment of MSA. Seven patients with MSA who received hUCB-MC transplantation via LASP were followed for 3 to 5 years. Neurological function was evaluated before (baseline), at 3, 6, and 12 months, and annually after the first transplantation using the Unified MSA Rating Scale (UMSARS); a lower score indicated improvement. Adverse events were recorded. The best therapeutic effect was observed 3 to 6 months after the first hUCB-MC transplantation. The total UMSARS score at the timepoint of best effect (25.71 ± 11.87) was significantly lower than the score before treatment (42.57 ± 7.96; P = 0.001), but also significantly lower than at the end of follow-up (35.14 ± 18.21; P = 0.038). The UMSARS II score (findings on neurological examination) at the timepoint of best effect was significantly lower than before treatment (P = 0.001). There were no serious adverse events. In conclusion, transplantation of hUCB-MCs via LASP is a safe and effective treatment for MSA.