In the advanced form of dry AMD - so-called geographic atrophy - degeneration of retinal pigment epithelial cells occurs. These cells are known to nourish the light-sensitive photoreceptors of the retina. The degeneration of the RPE cells is accompanied by damage and loss of the photoreceptors located in the macula, which are essential for reading. Geographic atrophy is a late form of age-related macular degeneration. It was preceded by a progressive disease process. The slowly expanding atrophy areas correspond to absolute scotomas. Affected patients lose central vision as a result. Reading and recognising faces is no longer possible.
At the National Eye Institute, the research team has achieved something impressive. The researchers have managed to convert the patient's blood cells into induced pluripotent stem cells (iPS). The cells were programmed to develop into retinal pigment epithelial cells. In ophthalmology, we have been waiting a long time for such a successful cell therapy. Blood cells can be transformed into almost any kind of cell in the body. This offers us unimagined possibilities in the treatment of diseases that until now have led to blindness. In the future, glaucomatous damage to the optic nerve head could possibly also be remedied by cell therapy. The sound of the future no longer seems too far away. The research success of the National Eye Institute after a good 10 years of research and development of this innovative cell therapy is also a success for mankind.1
The patch was made from induced pluripotent stem (iPS) cells derived from the patient. The iPS cell-derived therapy was developed by a research team from the Ocular and Stem Cell Translational Research Section in collaboration with FUJIFILM Cellular Dynamics Inc. and Opsis Therapeutics. Led by Kapil Bharti, Ph.D., the National Eye Institute researchers succeeded in bringing hope to the world for a previously incurable disease. The patch, made from autologous stem cells, is the basis for regaining vision in the central visual field.1
The safety profile and efficacy of the patch were first tested preclinically by a research team from the National Eye Institute. The cell patch was previously manufactured at the Center for Cellular Engineering, Department of Transfusion Medicine, Clinical Center, NIH. This is where the assessment of the clinical quality of this new cell therapy took place. The next step was to test the effectiveness and safety of this innovative method, which was still experimental at that time, within the framework of a clinical phase I/IIa study. This task was undertaken by Prof. Dr. Ph.D. Amir H. (Wilmer Eye Institute, Johns Hopkins School of Medicine) together with clinical associate Dr. Shilpa Kodati (National Eye Institute). The surgery was performed at the NIH Clinical Center in Bethesda, Maryland.1