SYSTEMATIC REVIEW article
Front. Pharmacol.
Sec. Experimental Pharmacology and Drug Discovery
Volume 16 - 2025 | doi: 10.3389/fphar.2025.1587440
Efficacy and safety of stem cells in the treatment of glaucoma: systematic review and meta-analysis based on animal experiments
Provisionally accepted
- 1School of clinical medicine, Shandong Second Medical University, Weifang, China
- 2Key Laboratory of Ophthalmology and Visual Science of Zibo City, Zibo Central Hospital, Shandong, China
- 3Department of Geriatrics, Zibo Central Hospital, Shandong, China
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Purpose: Glaucoma, characterized by progressive retinal ganglion cell (RGC) degeneration and optic nerve atrophy, remains a leading global cause of irreversible blindness, despite advancements in clinical management. While current therapies predominantly focus on lowering intraocular pressure (IOP), neuroprotective strategies to preserve visual function remain an unmet clinical need. Stem cells exhibit high proliferative capacity and multilineage differentiation potential, demonstrating notable efficacy in glaucoma treatment. Emerging preclinical evidence further highlights dual neuroprotective mechanisms (i.e., paracrine neurotrophic support and cellular replacement) of stem cell-based interventions. Accordingly, this systematic review and meta-analysis evaluated the therapeutic efficacy and safety of stem cell transplantation in experimental glaucoma models, with a focus on functional outcomes (IOP modulation), structural preservation (RGC survival, and nerve fiber layer integrity), and neuroprotective efficacy.Methods: A systematic literature retrieval from multiple online databases (e.g., PubMed, Web of Science, Embase, Cochrane Library, Scopus, CNKI, WFSD, VIP, and CBM) was conducted through January 3, 2025, to identify relevant animal experimental studies. This study further computed effect sizes of stem cell transplantation on indices such as IOP, RGC count. In the meta-analysis involving 19 studies, the stem cell transplantation group had significantly lower IOP (MD =-1.55,95% CI=-2.62 to -0.47) at weeks 3 and 4, higher RGC count at weeks 2, 3, and 4 (MD =23.06,95%CI=18.22 to 27.89), and greater nerve fiber layer thickness (MD = 10.69, 95%CI =9.44 to 11.94) compared to the control group. Meanwhile, the stem cell transplantation group also had higher retinal BDNF expression at weeks 2 and 4 (MD =0.75,95%CI=0.67 to 0.83), GDNF expression at weeks 1, 2, 3, and 4, and IGF-1 expression (MD = 0.49,95%CI =0.39 to 0.58). None of these studies reported any adverse systemic events.Conclusions: This meta-analysis provides preclinical evidence supporting stem cell transplantation as a multimodal therapeutic strategy for glaucoma, demonstrating significant efficacy in IOP modulation, neurostructural preservation, and neurotrophic factor expression. Given the severity of glaucomainduced ocular structural damage, it underscores the significance of stem cell transplantation as a secure and promising therapeutic option with notable neuroprotective potential, despite existing translational challenges regarding optimal cell sources, delivery routes, and long-term safety profiles.
Keywords: Stem Cells, Glaucoma, Meta-analysis, randomized controlled trials, animal experiment
Received: 07 Mar 2025; Accepted: 18 Jun 2025.
Copyright: © 2025 Wu, Liu, Zhang, Zhang, Wang, Lu and Yue. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Hui Lu, Key Laboratory of Ophthalmology and Visual Science of Zibo City, Zibo Central Hospital, Shandong, 255000, China
Tao Yue, Department of Geriatrics, Zibo Central Hospital, Shandong, 255000, China
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.