Background <p>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.</p> Objective <p>To summarize the research progress in clinical studies of stem cells therapy for chronic liver diseases, providing a comprehensive overview of current knowledge.</p> Methods <p>Research on stem cell therapy for liver diseases was searched from databases such as PubMed, Web of Science, Google Scholar, and ClinicalTrials.gov.</p> Results <p>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.</p> Conclusion <p>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.</p>

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Stem cells therapies for liver diseases: for current practice and future goals

  • Yunbo Xie,
  • Ziying Zhang,
  • Yuefei Pan,
  • Fu-Sheng Wang

摘要

Background

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.

Objective

To summarize the research progress in clinical studies of stem cells therapy for chronic liver diseases, providing a comprehensive overview of current knowledge.

Methods

Research on stem cell therapy for liver diseases was searched from databases such as PubMed, Web of Science, Google Scholar, and ClinicalTrials.gov.

Results

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.

Conclusion

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.