Abstract
Purpose :
Stargardt disease (STGD) is typically caused by defects in the ABCA4gene that lead to dysfunction and death of RPE cells. Transplantation of RPE cells has the potential to promote and protect retinal function. Our aim was to investigate the safety of subretinal transplantation of human embryonic stem cell-derived hESC-derived RPE cells in STGD.
Methods :
Twelve participants with STGD were administered subretinally a suspension of hESC-derived RPE cells at doses of 50 K, 100 K, 150 K and 200 K cells. Systemic immunosuppression was administered in all participants. We assessed safety, cell survival and retinal function.
Results :
Subretinal hyperpigmentation suggesting cell engraftment at the site of transplantation was observed in all participants. The extent of hyperpigmentation correlateddirectly with the dose of cells administered. A reduction in pigment density was evident during the follow-up period. We found no evidence of tumorigenicity or graft rejection. We measured no significant change in visual acuity or retinal sensitivity, except in one participant in whom a reduction of subretinal pigmentation was associated with reduced retinal function.
Conclusions :
Subretinal transplantation of hESC-derived RPE cells in STGD appears safe and well tolerated at 5 years.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.