Cell therapies for neurological disorders are entering the clinic and present unique challenges and opportunities compared with conventional medicines. They have the potential to replace damaged nervous tissue and integrate into the brain or spinal cord to produce functional effects for the lifetime of the patient, which could revolutionize the way clinicians treat debilitating neurological disorders. The major challenge has been cell sourcing, which historically relied mainly on fetal brain tissue. This has largely been overcome with the advent of pluripotent stem cell technology and the ability to make almost any cell of the nervous system at scale. Furthermore, advances in gene editing now allow the generation of genetically modified cells that could perform better and evade the immune system. With all the remarkable new approaches to treat neurological disorders, we take a critical look at the state of current clinical trials and how challenges may be overcome with the evolving technology and innovation occurring in the stem cell field.

神经系统疾病的细胞疗法正在进入临床，与传统药物相比，它带来了独特的挑战和机遇。它们有可能替换受损的神经组织并整合到大脑或脊髓中，从而在患者的一生中产生功能影响，这可能会彻底改变临床医生治疗使人衰弱的神经系统疾病的方式。主要挑战是细胞来源，它过去主要依赖于胎儿脑组织。随着多能干细胞技术的出现以及大规模制造神经系统中几乎任何细胞的能力，这在很大程度上已经被克服了。此外，基因编辑的进步现在允许产生转基因细胞，这些细胞可以表现得更好并逃避免疫系统。通过所有治疗神经系统疾病的非凡新方法，我们批判性地审视了当前临床试验的状态，以及如何通过干细胞领域不断发展的技术和创新来克服挑战。