Background Although human embryonic stem cells (hESCs) have been considered a potential therapeutic option for regenerative medicine, there are some concerns regarding tumorigenicity, immunogenicity and ethical considerations. Stargardt macular dystrophy (SMD) is the most common form of juvenile macular degeneration that causes early onset blindness. Therapeutic options for SMD remain limited, although several treatment strategies are currently under investigation. Here, we report a 3-year assessment of a phase I clinical trial involving subretinal transplantation of hESC-retinal pigment epithelium (RPE) cells in patients with SMD.
Methods This prospective, non-randomised clinical trial included three patients with SMD. All transplant recipients had central visual acuity no better than 20/400. Trans-pars plana vitrectomy was performed in the eye with poorer vision. RPE cells were reconstituted in balanced salt solution plus, then injected into the subretinal space using a semi-automated subretinal injection method.
Results No serious adverse events occurred throughout the 3-year period following the injection of hESC-RPE cells. The functional and anatomical results were favourable, compared with the natural course of SMD reported in the ProgStar study. One patient showed best-corrected visual acuity improvement, while the other patients had stable best-corrected visual acuity during the 3-year follow-up period.
Conclusion These results suggest the long-term safety, tolerability, and feasibility of subretinal hESC-derived RPE cell transplantation in regenerative medicine.
Trial registration number NCT01625559.
- Stem cells
- Clinical trial
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Contributors YS: Collection and assembly of data, data analysis and interpretation, manuscript writing; MJL: Other (cell works, collection and assembly of data); JC, SYJ, SYC, JHS: Other (screening of the patients and/or management of the adverse events); SHS: design and interpretation of genetic analysis; WKS: conception and design, provision of study material or patients, data analysis and interpretation, manuscript writing, final approval of manuscript.
Funding This research was supported by the CHA Biotech Co., Ltd., by their grant of the Korea Health Technology R&D Project through the Korea Health Industry Development Institute (KHIDI), funded by the Ministry of Health & Welfare, Republic of Korea: grant number HI12C0447 (A120506).
Competing interests None declared.
Provenance and peer review Not commissioned; externally peer reviewed.
Data availability statement No data are available.
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