Robertson Clinical and Translational Cell Therapy Program, Duke University, Durham, North Carolina.
Pediatr Res. 2018 Jan;83(1-2):364-371. doi: 10.1038/pr.2017.254. Epub 2017 Nov 8.
The concept of utilizing human cells for the treatment of medical conditions is not new. In its simplest form, blood product transfusion as treatment of severe hemorrhage has been practiced since the 1800s. The advent of hematopoietic stem cell transplantation (HSCT) began with the development of bone marrow transplantation for hematological malignancies in the mid-1900s and is now the standard of care for many hematological disorders. In the past few decades, HSCT has expanded to additional sources of donor cells, a wider range of indications, and the development of novel cell products. This trajectory has sparked a rapidly growing interest in the pursuit of innovative cell therapies to treat presently incurable diseases, including neurological conditions. HSCT is currently an established therapy for certain neurologically devastating inherited metabolic diseases, in which engrafting donor cells provide lifelong enzyme replacement that prevents neurological deterioration and significantly extends the lives of affected children. Knowledge gained from the treatment of these rare conditions has led to refinement of the indications and timing of HSCT, the study of additional cellular products and techniques to address its limitations, and the investigation of cellular therapies without transplantation to treat more common neurological conditions, such as autism spectrum disorder.
利用人类细胞治疗医疗状况的概念并不新鲜。最简单的形式是，自 19 世纪以来，血液制品输注已被用于治疗严重出血。造血干细胞移植（HSCT）的出现始于 20 世纪中叶开发骨髓移植治疗血液系统恶性肿瘤，现在已成为许多血液系统疾病的标准治疗方法。在过去的几十年中，HSCT 已扩展到其他供体细胞来源、更广泛的适应证以及新型细胞产品的开发。这种发展趋势引发了人们对寻求创新细胞疗法治疗目前无法治愈的疾病（包括神经疾病）的浓厚兴趣。HSCT 目前是某些神经破坏性遗传性代谢疾病的既定治疗方法，其中植入供体细胞提供终身酶替代治疗，可防止神经恶化并显著延长受影响儿童的寿命。从这些罕见疾病的治疗中获得的知识导致 HSCT 的适应证和时机得到了改进，对其他细胞产品和技术的研究也得到了改进，以解决其局限性，并研究了无需移植即可治疗更常见的神经疾病（如自闭症谱系障碍）的细胞疗法。
