Scolding Neil J, Pasquini Marcelo, Reingold Stephen C, Cohen Jeffrey A
Department of Neurology, University of Bristol Southmead Hospital, Bristol BS10 5NB, UK.
Center for International Blood and Marrow Transplant Research (CIBMTR), Medical College of Wisconsin, Milwaukee, WI 53226, USA.
Brain. 2017 Nov 1;140(11):2776-2796. doi: 10.1093/brain/awx154.
The availability of multiple disease-modifying medications with regulatory approval to treat multiple sclerosis illustrates the substantial progress made in therapy of the disease. However, all are only partially effective in preventing inflammatory tissue damage in the central nervous system and none directly promotes repair. Cell-based therapies, including immunoablation followed by autologous haematopoietic stem cell transplantation, mesenchymal and related stem cell transplantation, pharmacologic manipulation of endogenous stem cells to enhance their reparative capabilities, and transplantation of oligodendrocyte progenitor cells, have generated substantial interest as novel therapeutic strategies for immune modulation, neuroprotection, or repair of the damaged central nervous system in multiple sclerosis. Each approach has potential advantages but also safety concerns and unresolved questions. Moreover, clinical trials of cell-based therapies present several unique methodological and ethical issues. We summarize here the status of cell-based therapies to treat multiple sclerosis and make consensus recommendations for future research and clinical trials.
多种具有监管部门批准的疾病修饰药物可用于治疗多发性硬化症，这表明该疾病的治疗取得了重大进展。然而，所有这些药物在预防中枢神经系统的炎症性组织损伤方面都只是部分有效，而且没有一种药物能直接促进修复。基于细胞的疗法，包括免疫消融后自体造血干细胞移植、间充质及相关干细胞移植、对内源性干细胞进行药理学操作以增强其修复能力，以及少突胶质前体细胞移植，作为多发性硬化症免疫调节、神经保护或受损中枢神经系统修复的新型治疗策略，已引起了广泛关注。每种方法都有潜在的优势，但也存在安全问题和未解决的疑问。此外，基于细胞的疗法的临床试验还存在一些独特的方法学和伦理问题。我们在此总结基于细胞的疗法治疗多发性硬化症的现状，并对未来的研究和临床试验提出共识性建议。
