Xie Yunbo, Zhang Ziying, Pan Yuefei, Wang Fu-Sheng
Department of Infectious Diseases, The Fifth Medical Center of Chinese PLA General Hospital, 100 Western 4 Ring Road, Beijing, 100039, China.
Chinese PLA Medical School, Chinese PLA General Hospital, Beijing, 100085, China.
Hepatol Int. 2025 Jun 24. doi: 10.1007/s12072-025-10835-1.
Stem cell therapy has emerged as a promising approach for treating severe chronic liver diseases, including decompensated liver cirrhosis (DLC) and liver failure(LF), with mesenchymal stem cells (MSCs) being the most extensively investigated cell type in both preclinical and clinical trials.
To summarize the research progress in clinical studies of stem cells therapy for chronic liver diseases, providing a comprehensive overview of current knowledge.
Research on stem cell therapy for liver diseases was searched from databases such as PubMed, Web of Science, Google Scholar, and ClinicalTrials.gov.
As of March 31, 2025, over 110 clinical trials investigating stem cell therapies for chronic liver diseases have been registered, predominantly in Phase I and Phase II stages. MSCs derived from umbilical cord, bone marrow, or adipose tissue representing the primary cellular sources. Stem cell therapy has demonstrated favorable safety profiles in the treatment of liver diseases. Concurrently, emerging evidence highlights its preliminary efficacy in enhancing patient survival rates and promoting hepatic functional recovery. However, challenges remain, such as inconsistent efficacy across trials, the absence of standardized protocols, and unresolved safety concerns. Emerging technologies, including stem cell-derived exosomes, gene-edited stem cells, and 3D liver organoids, show promising preclinical potential but require further validation.
Stem cell therapy holds great potential for liver disease treatment. Although not yet a mainstream clinical option, continuous research may lead to effective, widely applicable treatments in the future.
干细胞疗法已成为治疗严重慢性肝病的一种有前景的方法，包括失代偿期肝硬化（DLC）和肝衰竭（LF），间充质干细胞（MSCs）是临床前和临床试验中研究最广泛的细胞类型。
总结干细胞治疗慢性肝病临床研究的进展，全面概述当前的知识。
从PubMed、Web of Science、Google Scholar和ClinicalTrials.gov等数据库搜索干细胞治疗肝病的研究。
截至2025年3月31日，已登记了110多项研究慢性肝病干细胞疗法的临床试验，主要处于I期和II期阶段。来自脐带、骨髓或脂肪组织的间充质干细胞是主要的细胞来源。干细胞疗法在肝病治疗中已显示出良好的安全性。同时，新出现的证据突出了其在提高患者生存率和促进肝功能恢复方面的初步疗效。然而，挑战依然存在，如各试验疗效不一致、缺乏标准化方案以及未解决的安全问题。包括干细胞衍生外泌体、基因编辑干细胞和3D肝脏类器官在内的新兴技术显示出有前景的临床前潜力，但需要进一步验证。
干细胞疗法在肝病治疗方面具有巨大潜力。虽然尚未成为主流临床选择，但持续的研究可能会在未来带来有效、广泛适用的治疗方法。
