Department of Medicine-Neurosciences, Ottawa Hospital Research Institute, University of Ottawa, Ottawa, Ontario K1H 8L6, Canada.
Department of Medicine-Hematology, Ottawa Hospital Research Institute, University of Ottawa, Ottawa, Ontario K1H 8L6, Canada.
Cold Spring Harb Perspect Med. 2019 Mar 1;9(3):a029082. doi: 10.1101/cshperspect.a029082.
Multiple sclerosis (MS) is an autoimmune disorder that typically affects young people during their most productive years, causing irreversible damage and accumulation of disability. Treatments over time have had modest effects at completely controlling or suppressing disease activity, but are generally aimed at controlling early dominating inflammation that, over time, accumulates damage and leads to progressive disability. Some unfortunate patients are destined to deteriorate despite even newer and more effective agents because of the inability of these drugs to fully curb the inflammatory component of the disease. These patients require something more that might be capable of halting the disease process. Using high-intensity myeloablative chemotherapeutic agents, it is now possible to completely remove the peripheral immune system and replace it anew from autologous bone marrow-derived hematopoietic stem cells, purged of disease-causing MS cells. This procedure, referred to as hematopoietic stem cell transplantation (HSCT), produces a new immune system that appears tolerant and no longer attacks the central nervous system (CNS).
多发性硬化症（MS）是一种自身免疫性疾病，通常在年轻人生产力最高的时期影响他们，导致不可逆转的损伤和残疾积累。随着时间的推移，治疗方法在完全控制或抑制疾病活动方面效果有限，但通常旨在控制早期主导的炎症，随着时间的推移，炎症会积累损伤并导致进行性残疾。一些不幸的患者尽管有更新、更有效的药物，也注定会恶化，因为这些药物无法完全抑制疾病的炎症成分。这些患者需要更有效的治疗方法，可能有能力阻止疾病进程。使用高强度的骨髓清除性化疗药物，现在可以彻底清除外周免疫系统，并从自身骨髓来源的造血干细胞中重新建立新的免疫系统，这些干细胞已清除了导致 MS 的细胞。该程序称为造血干细胞移植（HSCT），可产生新的免疫系统，该免疫系统似乎具有耐受性，不再攻击中枢神经系统（CNS）。
