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Two emerging biopharmaceutical companies, Advanced Cell Technology (ACT) and Stem Cells, Inc., are making significant progress in moving vision-saving stem cell treatments for retinal diseases into clinical trials. In 2010, ACT is planning to launch a Phase I clinical trial of their treatment, which involves transplantation of retinal-pigment-epithelial-like (RPE-like) cells into the eyes of people affected by Stargardt disease. Using a method of obtaining human embryonic stem cells that doesn’t harm the embryo, the RPE-like cells show strong potential for preserving vision by nourishing and supporting photoreceptors, the cells that enable people to see. ACT recently announced that they have submitted an investigational new drug application, also known as an IND, with the FDA to obtain authorization to begin their clinical study. Stem Cells, Inc. recently announced that their treatment, derived from human neural stem cells (nerve tissue), preserved vision in a rodent model of retinal degenerative disease. Foundation-funded researcher Raymond Lund, Ph.D., of the Oregon Health and Science University, conducted the study, noting that the transplanted cells were safe and demonstrated potential for “significant therapeutic value for clinical cases of retinal degeneration.” Dr. Lund’s lab and Stem Cells, Inc. are collaborating on additional preclinical studies before moving the treatment into a clinical trial. Stem cells hold promise for saving and restoring vision in people affected by a wide range of retinal degenerative diseases including: age-related macular degeneration, Stargardt disease, and retinitis pigmentosa. In less advanced stages of these diseases, they show potential for protecting and preserving a recipient’s existing retinal tissue from further degeneration. For more advanced disease where substantial vision has been lost, Foundation-funded investigators have shown that stem cells show potential for replacing degenerated tissue and thereby restoring vision. The Foundation Fighting Blindness supported several preclinical stem cell projects that have made these emerging therapies possible. The Foundation currently funds stem cell research at a number of research centers including: University of Wisconsin-Madison, Oregon Health and Science University, and the University of Washington. |
