ESC-Derived Retinal Pigmented Epithelial Cell Transplants in Patients: So Far, So Good

摘要

Many untreatable blinding diseases involve degeneration of retinal pigmented epithelial (RPE) cells, which has prompted exploration of the therapeutic potential of human-piuripotent-stem-cell-derived RPE. The first safety trials reported in The Lancet of embryonic-stem-cell-derived RPE cell transplants indicate no serious adverse outcomes and encourage further investigation. In a recent issue of The Lancet, Schwartz et al. (2014) reported on progress in phase l/ll clinical trials transplanting embryonic stem cell (ESC)-derived retinal pigment epithelial (RPE) cells in late-stage retinal degenerative disease. As the first report on the mid- to long-term safety of ESCs in cell therapy for any disease, this study is highly significant for the future development of ESC-based therapies. In the retina, RPE cells serve multiple roles essential for the function and survival of the light-sensitive photoreceptor cells; they form a polarized monolayer (on Bruch's basement membrane) between the blood supply of the choroid and the overlying photoreceptor cells. Degeneration of RPE cells is involved in many untreatable blinding eye diseases, including atrophic age-related macular degeneration (AMD), which is the most common cause of sight impairment in industrialized countries, and Stargardt disease, which is the most common cause of inherited sight loss.