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Updated: May 25, 2026
10:24
Regenerative Therapy by Suprachoroidal Cell Autograft in Dry Age-related Macular Degeneration: Preliminary In Vivo Report
Published on: February 12, 2018
Embryonic stem cell trials for macular degeneration: a preliminary report
Steven D Schwartz1, Jean-Pierre Hubschman, Gad Heilwell
1Jules Stein Eye Institute Retina Division, Department of Ophthalmology, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA.
Lancet (London, England)
|January 28, 2012
Summary
Human embryonic stem cell-derived retinal pigment epithelium (hESC-RPE) cells were transplanted into patients with macular dystrophies. The hESC-RPE cells demonstrated safety and potential vision improvement in early clinical trials.
Area of Science:
- Ophthalmology
- Regenerative Medicine
- Stem Cell Biology
Background:
- Human embryonic stem cells (hESCs) were discovered 13 years ago.
- This report details the first transplantation of hESC-derived cells into human patients.
- The study focuses on hESC-derived retinal pigment epithelium (RPE) for treating blinding conditions.
Purpose of the Study:
- To assess the safety and tolerability of subretinal transplantation of hESC-derived RPE.
- To evaluate the potential efficacy of hESC-RPE in patients with Stargardt's macular dystrophy and dry age-related macular degeneration.
- To establish clinical protocols for hESC-RPE transplantation.
Main Methods:
- Two prospective clinical studies were initiated (ClinicalTrials.gov: NCT01345006, NCT01344993).
- Subretinal transplantation of >99% pure, controlled hESC-differentiated RPE cells.
- Preoperative and postoperative ophthalmic examinations including visual acuity, fluorescein angiography, OCT, and visual field testing.
Main Results:
- Transplanted hESC-RPE cells integrated into the host RPE layer and persisted.
- No signs of hyperproliferation, tumorigenicity, or immune rejection were observed in patients within 4 months.
- Visual acuity improved in both patients; Stargardt's patient improved from hand motions to 20/800, and AMD patient improved from 21 to 28 ETDRS letters.
Conclusions:
- hESC-derived RPE cells are safe and well-tolerated in patients with macular dystrophies.
- Early evidence suggests potential for vision improvement and rescue of photoreceptors.
- Future therapeutic strategies aim for earlier intervention in disease progression.
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