Ransohoff Richard M
Third Rock Ventures, Boston, MA.
Neurol Neuroimmunol Neuroinflamm. 2025 Jan;12(1):e200352. doi: 10.1212/NXI.0000000000200352. Epub 2024 Dec 13.
Neurologic disease remains a cause of incalculable suffering, a formidable public health burden, and a wilderness of complex biology and medicine. At the same time, advances in basic science, technology, and the clinical development toolkit bring meaningful benefit for patients along with realistic hope for those whose conditions remain inadequately treated. This perspective focuses on cell-based therapies for neurologic disease, with particular emphasis on neuroimmunologic disorders and on the immunologic considerations of cell therapy for nonimmune conditions. I will consider the use of chimeric antigen receptor (CAR)-T effector cells and regulatory T-cell therapies for autoimmune conditions. I will briefly discuss the immune aspects of pluripotent stem cell (PSC)-derived neuronal therapies. With apologies for the omission, we do not discuss mesenchymal stem cells, glial progenitor cells, or CAR-NK cells, primarily for space limitations.
神经疾病仍然是造成难以估量痛苦的原因,是巨大的公共卫生负担,也是复杂生物学和医学的一片未知领域。与此同时,基础科学、技术以及临床开发工具包的进步为患者带来了切实的益处,也给那些病情仍未得到充分治疗的患者带来了切实的希望。这篇综述聚焦于针对神经疾病的细胞疗法,尤其着重于神经免疫性疾病以及细胞疗法在非免疫性疾病中的免疫学考量。我将探讨嵌合抗原受体(CAR)-T效应细胞和调节性T细胞疗法在自身免疫性疾病中的应用。我还将简要讨论多能干细胞(PSC)衍生的神经元疗法的免疫方面。由于篇幅限制,我们未讨论间充质干细胞、神经胶质祖细胞或CAR-NK细胞,在此表示歉意。