Neural Regeneration Research ›› 2026, Vol. 21 ›› Issue (4): 1428-1446.doi: 10.4103/NRR.NRR-D-24-01337

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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).

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