Cedars-Sinai Board of Governors Regenerative Medicine Institute, Los Angeles, CA, USA.
Nat Med. 2024 Oct;30(10):2756-2770. doi: 10.1038/s41591-024-03281-3. Epub 2024 Oct 15.
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.
神经疾病的细胞疗法正在进入临床应用，与传统药物相比，它们带来了独特的挑战和机遇。这些疗法有可能替代受损的神经组织，并与大脑或脊髓整合，为患者的整个生命周期产生功能效果，这可能彻底改变临床医生治疗神经退行性疾病的方式。主要的挑战一直是细胞来源，从历史上看，这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几乎任何神经细胞的能力，这一问题在很大程度上得到了解决。此外，基因编辑技术的进步现在允许生成经过基因修饰的细胞，这些细胞可能表现更好，并逃避免疫系统。随着所有这些治疗神经疾病的新方法的出现，我们对当前临床试验的状况进行了批判性的审视，并探讨了如何通过干细胞领域正在发生的技术进步和创新来克服挑战。
