Functional improvement of patients with progressive muscular dystrophy by bone marrow and umbilical cord blood mesenchymal stem cell transplantations

Abstract

To investigate the feasibility of employing double transplantations of autologous bone marrow mesenchymal stem cells (BMSC) and umbilical cord mesenchymal stem cells (UMSC) in the treatment of progressive muscular dystrophy (PMD). A total of 82 cases were treated by the double transplantations of BMSC and CB-MSC. They were diagnosed by clinical manifestations, CK, LDH, genetic analysis, electromyography, MRI and pathologic examination of biopsied muscle specimens from July 2007 to July 2008. Control group was self-made at before and after treatment and cases were followed up for 3 - 12 months. treatment method: Eighty-two patients underwent the double transplantations of bone mesenchymal stem cell (BMSC) and human umbilical cord blood MSC (CB-MSC). (1) BMSC: 80 - 150 ml bone marrow sample was collected through a puncture at bilateral posterior superior iliac spine. Ficoll density gradient centrifuge was employed to separate individual monocyte for induced differentiation. (2) CB-MSC: 80 - 160 ml umbilical cord blood was harvested and processed likewise as above. (3) Stem cell transplantation: Both BMSC and CB-MSC were collected and prepared into 1 x 10(8)/ml and 1 x 10(7)/ml cell suspension respectively. They were transplanted in divided does into the extremity muscle and vein. The clinical and laboratory parameters were monitored at 3, 6, 9 and 12 months. It was found that 31 cases (37.8%) obtained a remarkable efficacy, 37 cases (45.1%) were effective and 14 cases (17.1%) had no change. Total effective rate was 82.9%. Seventy patients (85.4%) felt limbs warmly, appetite improved, gained weight, had better appetite and action were nimble. Activity of daily living scale (ADL) in 72 patients (87.8%) increased as compared with pre-treatment (P < 0.01). LDH decreased at post-treatment [(475 +/- 223) u/L vs (410 +/- 216) u/L, P < 0.05, t = 6.650]. Creatine kinase [(2952 +/- 2259) u/L vs (2841 +/- 2092) u/L, P = 0.223, t = 1.094] and creatine [(26 +/- 12) micromol/L vs (25 +/- 11) micromol/L, P = 0.306, t = 1.029] decreased slightly. Adherence to therapy among Children and no adverse reaction was reported during the course of treatment. The double transplantation of BMSC and CB-MSC is convenient, safe and effective in the treatment of progressive muscular dystrophy and can be considered as a new therapy of PMD. MSC represents a possible tool of cellular therapeutics for PMD.