Treatment of multiple sclerosis by transplantation of neural stem cells derived from induced pluripotent stem cells

• Published in: Science China. Life sciences 2016-09, Vol.59 (9), p.950-957
• Main Authors: Zhang, Chao, Cao, Jiani, Li, Xiaoyan, Xu, Haoyu, Wang, Weixu, Wang, Libin, Zhao, Xiaoyang, Li, Wei, Jiao, Jianwei, Hu, Baoyang, Zhou, Qi, Zhao, Tongbiao
• Format: Article
• Language: English
• Subjects: Animals; Biomedical and Life Sciences; Cell Differentiation; Cells, Cultured; Disease Models, Animal; Encephalomyelitis, Autoimmune, Experimental - immunology; Encephalomyelitis, Autoimmune, Experimental - physiopathology; Encephalomyelitis, Autoimmune, Experimental - therapy; Feasibility Studies; Humans; Induced Pluripotent Stem Cells - cytology; Life Sciences; Male; Mice, Inbred C57BL; Motor Activity; Multiple Sclerosis - immunology; Multiple Sclerosis - physiopathology; Multiple Sclerosis - therapy; Neural Stem Cells - cytology; Neural Stem Cells - transplantation; NSCs; Research Paper; Stem Cell Transplantation - methods; T-Lymphocytes - immunology; T-Lymphocytes - metabolism; Treatment Outcome; 中枢神经系统; 多发性硬化; 神经干细胞; 神经退行性疾病; 移植治疗; 细胞浸润; 自身免疫性疾病
• ISSN: 1674-7305; 1869-1889
• DOI: 10.1007/s11427-016-0114-9

Multiple sclerosis（MS） is an autoimmune disease of the central nervous system（CNS）, with focal T lymphocytic infiltration and damage of myelin and axons. The underlying mechanism of pathogenesis remains unclear and there are currently no effective treatments. The development of neural stem cell（NSC） transplantation provides a promising strategy to treat neurodegenerative disease. However, the limited availability of NSCs prevents their application in neural disease therapy. In this study, we generated NSCs from induced pluripotent stem cells（iPSCs） and transplanted these cells into mice with experimental autoimmune encephalomyelitis（EAE）, a model of MS. The results showed that transplantation of iPSC-derived NSCs dramatically reduced T cell infiltration and ameliorated white matter damage in the treated EAE mice. Correspondingly, the disease symptom score was greatly decreased, and motor ability was dramatically rescued in the iPSC-NSC-treated EAE mice, indicating the effectiveness of using iPSC-NSCs to treat MS. Our study provides pre-clinical evidence to support the feasibility of treating MS by transplantation of iPSC-derived NSCs.