Abstract:

BACKGROUND: Many animal and clinical studies have reported that the safe and effective usage of umbilical cord blood-derived mesenchymal stem cells (UCB-MSCs) transplantation for treatment of neurological genetic diseases.
OBJECTIVE: To investigate the feasibility and effect of UCB-MSCs transplantation in the treatment of spinal muscular atrophy (SMA).
METHODS: A child admitted at January 2010 had been confirmed as having SMA, and drug and rehabilitation therapies were invalid. Then, the child received UCB-MSCs transplantation via the first intravenous infusion and three times of subarachnoid injection, once a week, (4-6)×10⁷ cells once and four times as a course. Neurological physical examination, biochemical test, muscle enzymes detection, FIM scoring and electromyography (EMG) examination were conducted.
RESULTS AND CONCLUSION: Compared with prior to transplantation, the level of muscle enzymes decreased, FIM scores were increased from 68 to 93 points, EMG results showed that the motor units with re-contraction in each 10.0 ms were increased that the motor function was improved, the lower extremity muscle strength elevated, and the self-care ability was improved in the SMA child at 6 months after transplantation. During the 10-month follow-up, the child had no adverse effects. It is indicated that UCB-MSCs transplantation is effective to treat SMA, and the neurological function has a remarkable restoration.