Development of a cellular therapy for dry Age Related Macular Degeneration (AMD) using retinal pigment epithelium (RPE) derived from human embryonic stem cells (hESC)

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Dry age related macular degeneration (AMD) is a degenerative disease of the retina, which leads to a progressive decrease in central visual acuity. In countries like the Federal Republic of Germany or the United States of America it is the main reason of blindness for the over 65 aged with approximately 12 million patients. It is caused by the cell death of the retinal pigment epithelium (RPE), which is integral for maintenance of healthy photoreceptors (PR) and together with the PR provides the transducing interface for visual perception. We hypothesize that lost RPE can be restored in an animal disease model through transplantation with human embryonic stem cells (hESC). Stem cells are premature and primitve cells, that have a big advantage in the underlying research project. They bear a high replication rate through cell division, which is supported by the fact that there is the relative paucity of cell numbers needed for each patient (1.5x105 RPE) which means that it is easily achievable with working and master cell banks. Additionally each cell has the potential to develop to a mature RPE cell. Preliminary results demostrate that hESC have disease-modyfing activity of of hESC-RPE in animal models of retinal dystrophy. In cooperation with the CalTech university it is planned to monitor blood flow dynamics in the choriocapillaris that supports the RPE and to measure changes in the photoreceptor pigment. Both are essential physiologic processes to evaluate the feasibility of hESC transplanted RPE.