RIKEN researchers report feasibility of iPSC-based autologous transplantation in AMD

Researchers at the RIKEN Center for Developmental Biology (Kobe, Japan) and colleagues reported data from a case study in an issue of the New England Journal of Medicine showing the feasibility of transplanting a sheet of retinal pigment epithelial (RPE) cells differentiated from autologous induced pluripotent stem cells (iPSCs) in a 77-year old Japanese woman with neovascular age-related macular degeneration (AMD). The patient underwent surgery in 2014 that included removal of the neovascular membrane and subsequent transplantation of the autologous iPSC-derived RPE cell sheet under the retina of the patient's right eye. The iPSCs were generated from the patient's skin fibroblasts.

At one year post-surgery, the transplanted sheet remained intact with no recurrence of the neovascular membrane and no serious complications or serious adverse events reported. Post-operative best corrected visual acuity (BCVA) did not improve, but was maintained at one year and throughout the follow-up period. Additionally, the patient’s National Eye Institute Visual Functioning Questionnaire (VFQ-25) score improved from 48.8 points before surgery to 58.3 points at one year post-surgery. However, the researchers said it is possible that the improvement in VFQ-25 score could have been obtained without surgery...