TRANSPLANTATION OF SUBRETINAL STEM CELL-DERIVED RETINAL PIGMENT EPITHELIUM FOR STARGARDT DISEASE: A Phase I Clinical Trial.

Abstract

PURPOSE:

To assess the safety of injecting human embryonic stem cell retinal pigment epithelial cell dose to treat Stargardt disease.

METHODS:

In this prospective, Phase I clinical trial, human embryonic stem cell retinal pigment epithelial cells in suspension were injected into the subretinal space in eyes with the worse best-corrected visual acuity (BCVA). After vitrectomy/posterior hyaloid removal, a partial retinal detachment was created and the human embryonic stem cell retinal pigment epithelial cells were administered. Phacoemulsification with intraocular lens implantation was performed in eyes with lens opacity. All procedures were optical coherence tomography-guided. The 12-month follow-up included retinal imaging, optical coherence tomography, visual field/electrophysiologic testing, and systemic evaluation. The main outcome was the absence of ocular/systemic inflammation or rejection, tumor formation, or toxicity during follow-up.

RESULTS:

The mean baseline BCVAs in the phacoemulsification and no phacoemulsification groups were similar (1.950 ± 0.446 and 1.575 ± 0.303, respectively). One year postoperatively, treated eyes showed a nonsignificant increase in BCVA. No adverse effects occurred during follow-up. Intraoperative optical coherence tomography was important for guiding all procedures.

CONCLUSION:

This surgical procedure was feasible and safe without cellular migration, rejection, inflammation, or development of ocular or systemic tumors during follow-up.

• Authors: Rodrigo A Brant Fernandes, Fernando H Lojudice, Lucas Zago Ribeiro, Natasha Ferreira Santos da Cruz, Murilo Ubukata Polizelli, Priscila C Cristovam, Francesco Innocenti, Lisangela Morimoto, Octaviano Magalhães Jr, Juliana Maria Ferraz Sallum, Fernando Marcondes Penha, Mari C Sogayar, Rubens Belfort Jr, Mauricio Maia
• Journal: Retina (Philadelphia, Pa.) (Retina) Vol. 43 Issue 2 Pg. 263-274 (02 01 2023) ISSN: 1539-2864 [Electronic] United States
• PMID: 36223778 (Publication Type: Clinical Trial, Phase I, Journal Article)

Chemical References

• Retinal Pigments

Topics

• Humans
• Retinal Pigment Epithelium (pathology)
• Stargardt Disease
• Prospective Studies
• Retinal Detachment (pathology)
• Stem Cells
• Inflammation
• Retinal Pigments
• Tomography, Optical Coherence