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Editorial
. 2011 Dec 7;2(6):47.
doi: 10.1186/scrt88.

Eyes open to stem cells: safety trial may pave the way for cell therapy to treat retinal disease in patients

Reinhold J Medina, Desmond B Archer, Alan W Stitt
Editorial

Eyes open to stem cells: safety trial may pave the way for cell therapy to treat retinal disease in patients

Reinhold J Medina et al. Stem Cell Res Ther. .

Abstract

A clinical trial using human embryonic stem cell (hESC) therapy for an inherited retinal degenerative disease is about to commence. The Advanced Cell Technology (ACT) trial will treat patients with Stargardt's macular dystrophy using transplanted retinal pigment epithelium derived from hESCs. Currently, no effective treatment is available for Stargardt's disease so a stem cell-based therapy that can slow progression of this blinding condition could represent a significant breakthrough. While there are some hurdles to clear, the ACT trial is a fine example of translational research that could eventually pave the way for a range of stem cell therapies for the retina and other tissues.

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Figures

Figure 1
Figure 1
Stargardt's macular dystrophy. (a) Fundus of a patient with advanced Stargardts disease. There are widespread white/yellow deposits at the posterior pole, indicating lipofuscin, RPE deposits and degeneration of the photoreceptors. Of note is the advanced foveal pigmentation at the centre of the macula (arrow). (b) A red free image showing extensive degeneration in the fovea and hyperfluorescent lipofuscin in RPE (arrows). (c,d) Ocular coherence tomography of a 30-year-old Stargardt's patient with vision of 6/36. There is extensive loss of cone photoreceptors evident in the fovea (arrow). Images courtesy of Miss Giuliana Silvestri.
See this image and copyright information in PMC

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