Department of Medical Services and Clinical Research, NeuroGen Brain and Spine Institute, Surana Sethia Hospital and Research Centre, Mumbai, India.
Cell Transplant. 2012;21 Suppl 1:S79-90. doi: 10.3727/096368912X633798.
Neurological disorders such as muscular dystrophy, cerebral palsy, and injury to the brain and spine currently have no known definitive treatments or cures. A study was carried out on 71 children suffering from such incurable neurological disorders and injury. They were intrathecally and intramuscularly administered autologous bone marrow-derived mononuclear cells. Assessment after transplantation showed neurological improvements in muscle power and a shift on assessment scales such as FIM and Brooke and Vignos scale. Further, imaging and electrophysiological studies also showed significant changes in selective cases. On an average follow-up of 15 ± 1 months, overall 97% muscular dystrophy cases showed subjective and functional improvement, with 2 of them also showing changes on MRI and 3 on EMG. One hundred percent of the spinal cord injury cases showed improvement with respect to muscle strength, urine control, spasticity, etc. Eighty-five percent of cases of cerebral palsy cases showed improvements, out of which 75% reported improvement in muscle tone and 50% in speech among other symptoms. Eighty-eight percent of cases of other incurable neurological disorders such as autism, Retts Syndrome, giant axonal neuropathy, etc., also showed improvement. No significant adverse events were noted. The results show that this treatment is safe, efficacious, and also improves the quality of life of children with incurable neurological disorders and injury.
目前，神经紊乱疾病（如肌肉萎缩症、脑瘫以及脑和脊柱损伤）尚无明确的治疗方法或治愈方法。本研究对 71 名患有此类不治之症和损伤的儿童进行了研究。对他们进行了鞘内和肌肉内注射自体骨髓来源的单核细胞治疗。移植后的评估显示，肌肉力量的神经功能得到改善，FIM 和 Brooke 和 Vignos 量表等评估量表也发生了转变。此外，影像学和电生理学研究也显示在某些选择性病例中有明显的变化。平均随访 15±1 个月后，97%的肌肉萎缩症病例表现出主观和功能改善，其中 2 例 MRI 发生变化，3 例 EMG 发生变化。100%的脊髓损伤病例在肌肉力量、尿控制、痉挛等方面均有改善。85%的脑瘫病例有所改善，其中 75%的病例报告肌肉张力改善，50%的病例报告言语改善等症状。88%的其他不治之症的神经紊乱病例（如自闭症、雷特综合征、巨大轴突神经病等）也有改善。未观察到明显的不良事件。结果表明，这种治疗方法安全有效，还能提高患有不治之症的神经紊乱和损伤的儿童的生活质量。
