Wang Zijie, Ma Yanan, Hu Xiqi, Xia Ying
Department of Neurosurgery, Haikou Hospital Affiliated with Xiangya Medical College, Central South University, Haikou, China.
Front Neurol. 2025 Aug 22;16:1596484. doi: 10.3389/fneur.2025.1596484. eCollection 2025.
As an emerging therapeutic strategy, stem cell transplantation has demonstrated promising potential in the management of refractory epilepsy. Epilepsy, a prevalent neurological disorder characterized by recurrent seizures, affects approximately one-third of patients worldwide who exhibit resistance to existing antiepileptic drugs (AEDs). Consequently, exploring novel treatment modalities is imperative. Recent studies have indicated that stem cell transplantation improves neurological function in epilepsy through multiple mechanisms. Research has revealed that transplanted stem cells mitigate seizure-induced neuronal damage by replacing dead or dysfunctional neurons, secreting beneficial neurotrophic factors (e.g., brain-derived neurotrophic factor, BDNF), and releasing anti-inflammatory cytokines. Preclinical studies and early-phase clinical trials have shown that stem cell transplantation significantly reduces seizure frequency and enhances patients' quality of life. However, current research is limited by small sample sizes and short-term follow-ups, necessitating further validation of long-term efficacy. Despite its therapeutic promise, stem cell transplantation faces critical challenges. First, technical details such as the cell source, processing, method of transplantation, and timing have yet to be standardized, leading to potential variability in efficacy and safety across different stem cell types. Second, complications like immune rejection and tumorigenesis pose significant safety risks. Future research should focus on optimizing stem cell selection and processing, designing robust clinical trials to evaluate long-term safety and efficacy, exploring combinatorial approaches with existing therapies, and developing advanced biomaterials to enhance transplantation success. Additionally, monitoring post-transplant cell survival and functionality, along with identifying epilepsy-specific biomarkers, will be crucial to refining the precision and safety of stem cell-based therapies.
作为一种新兴的治疗策略，干细胞移植在难治性癫痫的治疗中已展现出了有前景的潜力。癫痫是一种常见的神经系统疾病，其特征为反复发作的癫痫发作，全球约三分之一的患者对现有的抗癫痫药物（AEDs）耐药。因此，探索新的治疗方式势在必行。最近的研究表明，干细胞移植通过多种机制改善癫痫患者的神经功能。研究发现，移植的干细胞可通过替代死亡或功能失调的神经元、分泌有益的神经营养因子（如脑源性神经营养因子，BDNF）以及释放抗炎细胞因子来减轻癫痫发作引起的神经元损伤。临床前研究和早期临床试验表明，干细胞移植可显著降低癫痫发作频率并提高患者的生活质量。然而，目前的研究受样本量小和随访时间短的限制，需要进一步验证长期疗效。尽管干细胞移植具有治疗前景，但仍面临严峻挑战。首先，诸如细胞来源、处理、移植方法和时机等技术细节尚未标准化，导致不同类型干细胞的疗效和安全性存在潜在差异。其次，免疫排斥和肿瘤发生等并发症带来了重大安全风险。未来的研究应侧重于优化干细胞的选择和处理，设计强有力的临床试验以评估长期安全性和疗效，探索与现有疗法的联合应用方法，以及开发先进的生物材料以提高移植成功率。此外，监测移植后细胞的存活和功能，以及识别癫痫特异性生物标志物，对于提高基于干细胞疗法的精准性和安全性至关重要。
