中国神经再生研究(英文版) ›› 2026, Vol. 21 ›› Issue (4): 1428-1446.doi: 10.4103/NRR.NRR-D-24-01337

• 综述:神经损伤修复保护与再生 • 上一篇    下一篇

治疗癫痫的干细胞修复策略

  

  • 出版日期:2026-04-15 发布日期:2025-07-26
  • 基金资助:
    国家自然科学基金项目(82471471、82471485)、陕西省科技创新领军人才特殊项目(tzjhjw); 陕西省重点研发计划项目(2023-YBSF-353);西京医院创新研究院联合基金项目(LHJJ24JH13)。

Stem cell repair strategies for epilepsy

Xiao Ma# , Zitong Wang# , Yinuo Niu, Jie Zhao, Xiaorui Wang, Xuan Wang, Fang Yang, Dong Wei, Zhongqing Sun* , Wen Jiang*   

  1. Department of Neurology, Xijing Hospital, Fourth Military Medical University, Xi’an, Shaanxi Province, China
  • Online:2026-04-15 Published:2025-07-26
  • Contact: Wen Jiang, MD, PhD, jiangwen@fmmu.edu.cn; Zhongqing Sun, PhD, sunzqtxj@fmmu.edu.cn.
  • Supported by:
    This work was supported by the National Natural Science Foundation of China, Nos. 82471471 (to WJ), 82471485 (to FY); Shaanxi Province Special Support Program for Leading Talents in Scientific and Technological Innovation, No. tzjhjw (to WJ); Shaanxi Key Research and Development Plan Project, No. 2023-YBSF-353 (to XW); the Joint Fund Project of Innovation Research Institute of Xijing Hospital, No. LHJJ24JH13 (to ZS).

摘要:

癫痫是一种严重的脑部疾病,目前药物治疗效果并不理想。最近,干细胞技术在治疗各种神经系统疾病方面显示出显著疗效,激发了人们对其治疗癫痫的潜力的兴趣。此综述的目的是基于已有文献探讨治疗癫痫的干细胞修复策略。目前已经有一些的临床前实验使用间充质干细胞,包括转基因间充质干细胞,研究其潜在的治疗效果。在临床试验中,来自患者自身的间充质干细胞降低了癫痫发作频率,改善了神经、认知和运动功能。另一种很有前景的治疗策略是神经干细胞治疗,这些细胞可以在体外生长,然后定向分化成特定的细胞类型,移植神经干细胞可以替代失去的抑制性中间神经元,为癫痫提供一种潜在的治疗方法。胚胎干细胞的特点是自我更新能力强,能够分化成任何类型的体细胞,在癫痫治疗中,它们可发挥3大功能:神经元再生、维持细胞平衡和恢复疗法,其中一个值得注意的治疗策略是将胚胎干细胞分化为γ-氨基丁酸能神经元,然后移植到病变部位,这种方法目前正在进行临床试验,可能成为治疗难治性癫痫的一个突破。诱导多能干细具有与供体相同的遗传背景,降低了免疫排斥风险和伦理问题,但有关诱导多能干细细胞疗法的研究仍处于临床前阶段。然而,干细胞疗法的费用较高而且存在安全问题,如肿瘤的形成和移植细胞的低存活率仍是挑战。总之,未来不断发展的干细胞疗法有望为癫痫的治疗带来新希望。

https://orcid.org/0000-0003-0612-2917 (Wen Jiang); https://orcid.org/0009-0000-5235-5880 (Zhongqing Sun)

关键词: 细胞疗法, 临床试验, 癫痫, γ-氨基丁酸能神经元, 间充质干细胞, 神经干细胞, 胚胎多能干细胞, 诱导多能干细胞, 星形胶质细胞转分化, 神经再生, 类器官, 细胞移植

Abstract: Epilepsy is a serious neurological disorder; however, the effectiveness of current medications is often suboptimal. Recently, stem cell technology has demonstrated remarkable therapeutic potential in addressing various neurological diseases, igniting interest in its applicability for epilepsy treatment. This comprehensive review summarizes different therapeutic approaches utilizing various types of stem cells. Preclinical experiments have explored the use and potential therapeutic effects of mesenchymal stem cells, including genetically modified variants. Clinical trials involving patientderived mesenchymal stem cells have shown promising results, with reductions in the frequency of epileptic seizures and improvements in neurological, cognitive, and motor functions reported. Another promising therapeutic strategy involves neural stem cells. These cells can be cultured outside the body and directed to differentiate into specific cell types. The transplant of neural stem cells has the potential to replace lost inhibitory interneurons, providing a novel treatment avenue for epilepsy. Embryonic stem cells are characterized by their significant capacity for self-renewal and their ability to differentiate into any type of somatic cell. In epilepsy treatment, embryonic stem cells can serve three primary functions: neuron regeneration, the maintenance of cellular homeostasis, and restorative activity. One notable strategy involves differentiating embryonic stem cells into γ-aminobutyric acidergic neurons for transplantation into lesion sites. This approach is currently undergoing clinical trials and could be a breakthrough in the treatment of refractory epilepsy. Induced pluripotent stem cells share the same genetic background as the donor, thereby reducing the risk of immune rejection and addressing ethical concerns. However, research on induced pluripotent stem cell therapy remains in the preclinical stage. Despite the promise of stem cell therapies for epilepsy, several limitations must be addressed. Safety concerns persist, including issues such as tumor formation, and the low survival rate of transplanted cells remains a significant challenge. Additionally, the high cost of these treatments may be prohibitive for some patients. In summary, stem cell therapy shows considerable promise in managing epilepsy, but further research is needed to overcome its existing limitations and enhance its clinical applicability.

Key words: astrocyte transdifferentiation, cell therapy, cell transplantation, clinical trials, embryonic pluripotent stem cells, epilepsy, gamma-aminobutyric acidergic neuron, induced pluripotent stem cells, mesenchymal stem cells, nerve regeneration, neural stem cells, organoid