Yong LIU Third Military Medical University, Chongqing, China "Team Report on Clinical Trial of Human Stem Cell Derived Retinal Pigment Epithelial Cell Transplantation for Macular Degeneration"
ABSTRACT
Macular degeneration, including age-related macular degeneration (AMD) and Stargardt macular degeneration (STGD1), seriously impairs the central vision in the elder and younger populations. Since the main degeneration and pathological changes of these diseases starts with the loss of retinal pigment epithelial (RPE) cells (retinal supporting cells), transplantation of human embryonic stem cells-derived retinal pigment epithelial (hESC-RPE) cells may become a promising new treatment option for macular degeneration. Here, we successfully generated RPE cells from clinical grade (CTS)-hESCs, which met the standard requirements for clinical applications. We implanted hESC-RPE cells into the subretinal space of 16 patients with wet-AMD due to neovascular disruption to RPE and 7 patients with STGD1. With optimized perioperative management and surgical protocols, patients who underwent the transplantation showed no adverse health events during two years follow-up. The visual changes and the fate of the transplanted cells had different manifestations. This talk will report our clinical results and discuss the challenges we face in this field.
BIO Yong Liu, associated professor, 49 years old, is director of Dept. of Ophthalmology, Southwest Hospital, Army Medical University of China. He received his M.D. degree from Third Military Medical University in 2008 and then performed postdoctoral research in Schepens Eye Research Institute, Harvard Medical School. He underwent rigorous training of vitreoretinal surgery and mainly engaged in the research of retinal stem cells transplantation for retinal diseases. From 2010, his team began clinical transplantation of fetal retinal progenitor cell for retinitis pigmentosa (WHO Trial Registration, ChiCTR-TNRC-08000193). Based on this work, a stem cell transplant team was formed and a GMP standard lab was established. From 2013 to 2019, He carried out a phase I clinical study focusing on the safety of the hESC-RPE cells for the treatment of wet age-related macular degeneration (AMD) and Stargardt macular degeneration (NCT02749734). Until now, he got four grants from National Natural Science Foundation of China and published 24 peer reviewed papers including journals of Stem Cell Reports and Retina.