30. STEM CELL AND STEM CELL-DERIVED PRODUCTS IN OPHTHALMIC DISEASES: AN UPDATED REVIEW, 2020–2025

Pham Xuan Da1, Ngo Chi Thanh, Ngo Tuan Minh, Pham Tue Lien
1 University of Medicine and Pharmacy - Vietnam National University, Hanoi

Main Article Content

Abstract

Objective: To review the application of stem cell therapies and stem cell-derived products in ophthalmic diseases.


Methods: A narrative review was conducted using PubMed and Google Scholar. Clinical studies published between 2020–2025 on the efficacy, safety, and clinical outcomes of stem cell therapies and extracellular vesicle (exosome) products were analyzed.


Results: ESC/iPSC-derived RPE transplantation for late-stage dry AMD showed long-term safety and visual improvement. In LSCD, CALEC achieved 77% complete success at 18 months, while allogeneic SLET maintained epithelialization in 71% of cases at 12 months. Intrastromal ADSC injection improved vision and corneal curvature in keratoconus. MSC-derived exosomes and eye drops improved dry eye indices with good safety. In glaucoma, early studies suggested neuroprotective effects on retinal ganglion cells.


Conclusions: Current evidence supports the safety and potential efficacy of stem cell-based therapies in ophthalmology. However, larger trials with long-term follow-up are needed to confirm durable benefits.

Article Details

References

[1] Humayun MS, Clegg DO, Dayan MS, et al. Long‑term Follow‑up of a Phase 1/2a Clinical Trial of a Stem Cell‑Derived Bioengineered Retinal Pigment Epithelium Implant for Geographic Atrophy. Ophthalmology. 2024;131(6):682‑691. doi:10.1016/j.ophtha.2023.12.028.
[2] Kashani AH, Lebkowski JS, Rahhal FM, et al. One‑Year Follow‑Up in a Phase 1/2a Clinical Trial of an Allogeneic RPE Cell Bioengineered Implant for Advanced Dry AMD. Transl Vis Sci Technol. 2021;10(10):13.
[3] Kashani AH, Uang J, Mert M, et al. Surgical method for implantation of a biosynthetic RPE monolayer for geographic atrophy: experience from a phase 1/2a study. Ophthalmol Retina. 2020;4(3):264‑273.
[4] California Institute for Regenerative Medicine (CIRM). Phase 2/3 pivotal planning for jCell (famzeretcel) after FDA Type B meeting (January 2024) – progress update. 2024. (Grant progress page).
[5] Jurkunas UV, Qi X, Suri K, et al. Cultivated autologous limbal epithelial cell (CALEC) transplantation for limbal stem cell deficiency: a phase I/II clinical trial with xenobiotic‑free, serum‑free, antibiotic‑free manufacturing. Nat Commun. 2025; (ấn hành trực tuyến).
[6] Riedl J, Mais C, Langenbucher A, et al. Midterm results after allogeneic simple limbal epithelial transplantation from deceased‑donor eyes in LSCD. Acta Ophthalmol. 2025;103(2):e—e. (PMCID: PMC11810538).
[7] Ramin S, Ahadi M, Abbasi A, et al. Intrastromal injection of adipose‑derived stem cells in keratoconus: a pilot study. Int J Ophthalmol. 2023;16(6):949‑957.
[8] Habibi A, Khosravi A, Soleimani M, et al. Efficacy of topical mesenchymal stem cell exosome in Sjögren’s syndrome‑related dry eye: randomized triple‑blind clinical trial. BMC Ophthalmol. 2025;25:299.
[9] Zhang D, Chen T, Liang Q, et al. First‑in‑human prospective pilot trial of umbilical cord‑derived MSC eye drops for refractory dry eye disease. Stem Cell Res Ther. 2025;16:202.
[10] Hu BY, Zhou L, Li Y, et al. Mesenchymal stem cells for repairing glaucomatous optic nerve injury: advances and controversies. (Review). 2024.
[11] Ji S, Zhao H, Lyu P, et al. Human adipose tissue‑derived stem cell extracellular vesicles alleviate neuroinflammation in ocular hypertension models. Acta Neuropathol Commun. 2024;12.
[12] Pastor JC, Rusciano D, Delgado A, et al. Intravitreal allogeneic mesenchymal stem cells: safety considerations and clinical insights. 2023. (Review).
[13] Soundararajan P, et al. Allogeneic retinal pigment epithelial cell suspension manufactured at scale for AMD. npj Regen Med. 2025;10.
[14] Jiao YR, Chen KX, Tang X, et al. Exosomes derived from mesenchymal stem cells in diabetes and diabetic complications. Cell Death Dis. 2024;15:271.
[15] Durmaz E, et al. Utilizing extracellular vesicles as a drug delivery system in the eye. J Control Release. 2024.
[16] Rohowetz LJ, et al. Gene and cell therapies currently used and in development for AMD and IRDs. Front Ophthalmol. 2023;3:1098406.
[17] Liu H, Xu T, Wang J, et al. Advances in retinal pigment epithelial cell transplantation for retinal degenerative diseases. Stem Cell Res Ther. 2024.
[18] Klymenko V, et al. Recent progress in RPE cell‑based therapy for retinal degeneration. 2024.
[19] Wang LH, et al. Advances in neuroprotection in glaucoma: role of cell‑based and exosome strategies. Pharmaceuticals (Basel). 2024;17(10):1261.
[20] Lin F, et al. Tackling visual impairment: emerging avenues in retinal neuroprotection and cell therapy. 2025.