Department of Neurology, Antwerp University Hospital, 2650 Edegem, Belgium.
Laboratory of Experimental Hematology, Vaccine & Infectious Disease Institute (VAXINFECTIO), Faculty of Medicine and Health Sciences, University of Antwerp, 2610 Wilrijk, Belgium.
Int J Mol Sci. 2021 Jul 25;22(15):7925. doi: 10.3390/ijms22157925.
Cell-based therapies are gaining momentum as promising treatments for rare neurological autoimmune diseases, including neuromyelitis optica spectrum disorders and myelin oligodendrocyte glycoprotein antibody-associated disease. The development of targeted cell therapies is hampered by the lack of adequate animal models that mirror the human disease. Most cell-based treatments, including HSCT, CAR-T cell, tolerogenic dendritic cell and mesenchymal stem cell treatment have entered early stage clinical trials or have been used as rescue treatment in treatment-refractory cases. The development of antigen-specific cell-based immunotherapies for autoimmune diseases is slowed down by the rarity of the diseases, the lack of surrogate outcomes and biomarkers that are able to predict long-term outcomes and/or therapy effectiveness as well as challenges in the manufacturing of cellular products. These challenges are likely to be overcome by future research.
细胞疗法作为治疗罕见神经自身免疫性疾病（包括视神经脊髓炎谱系疾病和髓鞘少突胶质细胞糖蛋白抗体相关疾病）的有前途的治疗方法正在兴起。由于缺乏能够反映人类疾病的适当动物模型，靶向细胞疗法的发展受到阻碍。大多数细胞治疗方法，包括 HSCT、CAR-T 细胞、免疫耐受树突状细胞和间充质干细胞治疗，已经进入早期临床试验阶段，或已被用作治疗难治性病例的抢救治疗。由于疾病的罕见性、缺乏能够预测长期结果和/或治疗效果的替代结果和生物标志物，以及细胞产品制造方面的挑战，自身免疫性疾病的抗原特异性细胞免疫疗法的发展受到了阻碍。这些挑战很可能会被未来的研究克服。
