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The embryonic stem cell derived retinal pigment epithelial cell trial for Stargardt macular dystrophy: preliminary phase 1 results in Asian
Author(s) -
KWON HJ,
SONG W,
CHOI J,
CHUNG SY,
KIM HJ,
LEE JH
Publication year - 2014
Publication title -
acta ophthalmologica
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.534
H-Index - 87
eISSN - 1755-3768
pISSN - 1755-375X
DOI - 10.1111/j.1755-3768.2014.f017.x
Subject(s) - medicine , ophthalmology , visual acuity , pars plana , retinal pigment epithelium , fluorescein angiography , transplantation , fundus (uterus) , posterior pole , retinal , retinitis pigmentosa , macular degeneration , vitrectomy , surgery
Purpose To report interim results of Stargardt macular dystrophy(SMD) patients who underwent human embryonic stem cell derived retinal pigment epithelial cells(hES‐RPE) transplantation. Methods hESC‐RPE cells were manufactured in a fully validated good‐manufacturing‐practice facility. Total pars plana vitrectomy with induction of posterior vitreous detachment was performed in the eyes with the worst vision. A volume of 150µL of RPE reconstituted in BSS Plus with the target dose of 50000 RPE cells was injected into the subretinal space. Immunosuppression was started at lower dosages of tacrolimus and mycophenolate mofetil. Ophthalmologic examinations; ETDRS visual acuity(VA), visual field examination(VF), fluorescein angiography, spectral‐domain OCT, and electroretinography were performed. Results A 45‐year‐old male SMD patient with initial VA of 1 letter(Counting fingers) had improved to 13 letters(20/640) and smaller central scotoma observed on goldman VF at 52 weeks. A 40‐year‐old male SMD patient with initial VA of 13 letters(20/640) improved to 30 letters(20/200) at 26 weeks. Subretinal pigmentations with multiple increased autofluorescence spots were noted. A 40‐year‐old male SMD patient with initial VA of 41 letters(20/160) has stable VA of 41 letters(20/160) at 4 weeks. Subretinal pigmentations around injection site were noted at 4 weeks. Conclusion Two of three SMD patients who received the target dose and relevant immunosuppression showed gradual improvements of visual acuity only in the study eye. No serious systemic or ocular adverse events occurred. Longer follow up and further randomized studies with a control group are needed to determine the safety, tolerability, efficacy.