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细胞治疗临床试验概述:进展、挑战与未来方向。

Overview of Cellular Therapeutics Clinical Trials: Advances, Challenges, and Future Directions.

作者信息

Guo Meizhai, Zheng Bingyi, Zeng Xiaoling, Wang Xueting, Tzeng Chi-Meng

机构信息

Translational Medicine Research Center, School of Pharmaceutical Sciences, Xiamen University, Xiamen 361102, China.

出版信息

Int J Mol Sci. 2025 Jun 16;26(12):5770. doi: 10.3390/ijms26125770.

DOI:10.3390/ijms26125770
PMID:40565232
Abstract

Cellular therapeutics, encompassing stem cell-based regeneration and engineered immune cell platforms, have demonstrated efficacy in treating degenerative diseases, immune-related diseases, and oncology. However, low engraftment rates and limited long-term efficacy remain critical translational barriers. This review compiled clinical projects on cell therapy in China over the past five years (over 1200 patients across 172 clinical trials) to highlight its rapid development in recent years and illustrate the directions of indications for application. This review also analyzes published clinical achievements all over the world, revealing significant therapeutic improvements in degenerative disorders (40-60% improvement in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) scores and oncology (78% ctDNA clearance, < 0.001)). We propose integrating traditional Chinese medicine (TCM) bioactive compounds to enhance cell viability via C-X-C motif chemokine receptor (CXCR4) upregulation and mitochondrial biogenesis. Despite mechanistic insights, translational barriers include limited TCM validation (72% lacking single-cell omics) and regulatory misalignment. Future efforts should prioritize randomized trials and standardized TCM-cell therapy protocols to bridge discovery and clinical translation.

摘要

细胞疗法,包括基于干细胞的再生和工程化免疫细胞平台,已在治疗退行性疾病、免疫相关疾病和肿瘤学方面显示出疗效。然而,低植入率和有限的长期疗效仍然是关键的转化障碍。本综述汇编了中国过去五年的细胞治疗临床项目(172项临床试验中的1200多名患者),以突出其近年来的快速发展,并阐明应用适应症的方向。本综述还分析了世界各地已发表的临床成果,揭示了在退行性疾病(西安大略和麦克马斯特大学骨关节炎指数(WOMAC)评分提高40 - 60%)和肿瘤学(78%的循环肿瘤DNA清除率,< 0.001)方面有显著的治疗改善。我们建议整合中药生物活性化合物,通过上调C-X-C基序趋化因子受体(CXCR4)和线粒体生物发生来提高细胞活力。尽管有机制方面的见解,但转化障碍包括中药验证有限(72%缺乏单细胞组学)和监管不一致。未来的努力应优先进行随机试验和标准化的中药-细胞治疗方案,以弥合发现与临床转化之间的差距。

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Chronic kidney disease prevalence and outcomes in patients with type 2 diabetes or prediabetes at high cardiovascular risk: results from the CINEMA program.心血管高危2型糖尿病或糖尿病前期患者的慢性肾脏病患病率及转归:CINEMA项目结果
Am J Prev Cardiol. 2025 Apr 25;22:101004. doi: 10.1016/j.ajpc.2025.101004. eCollection 2025 Jun.
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New discoveries in therapeutic targets and drug development pathways for type 2 diabetes mellitus under the guidance of precision medicine.
精准医学指导下2型糖尿病治疗靶点与药物研发途径的新发现
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Mesenchymal stem cell-based therapy for type 1 & 2 diabetes mellitus patients: a systematic review and meta-analysis of randomized controlled trials.基于间充质干细胞的1型和2型糖尿病患者治疗:随机对照试验的系统评价和荟萃分析
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CAR-T cell therapy in T-Cell lymphoblastic leukemia/lymphoma: where do we stand now?嵌合抗原受体T细胞疗法治疗T细胞淋巴母细胞白血病/淋巴瘤:我们目前的进展如何?
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Adapting for the future: what can we learn from REMAP-CAP and COVID-19 pandemic trials?为未来做好准备:我们能从REMAP-CAP和新冠疫情试验中学到什么?
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