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Stem Cells Treat Vision Disorders in Animal Tests

Stem cell colony

Human embryonic stem cell colony (National Institute of General Medical Sciences, NIH)

4 May 2015. Tests with lab rats of a therapy for degenerating retinas in the eyes, show the treatments derived from human embryonic stem cells can restore visual functions. Results of the treatments, developed by Cell Cure Neurosciences Ltd., are scheduled to be reported today by a research team from Oregon Health and Science University at a meeting of Association for Research in Vision and Ophthalmology in Denver.

The study, led by OHSU’s Trevor McGill, tested Cell Cure’s lead product OpRegen, which is derived from human embryonic stem cells that are cultured in the company’s lab to transform into retinal pigmented epithelial cells that support and nourish human retinas, the layer of tissue inside the eye that changes light waves to nerve impulses. Cell Cure, a subsidiary of the biotechnology company BioTime Inc., is developing OpRegen as a potential treatment for age-related macular degeneration and retinitis pigmentosa.

Age-related macular degeneration is a common eye disorder, where damage to the macula, a small spot in the center of the retina, becomes damaged, resulting in progressive loss of vision. Retinitis pigmentosa is a family of inherited eye disorders that result in damage to the retina, specifically breakdown and failure of photoreceptor cells in the retina also leading to progressive vision loss.

Cell Cure Neurosciences is a spin-off company from Hadassah Medical Center in Jerusalem, Israel located on the its campus. The company says its technology cultures stem cells into highly purified retinal pigmented epithelial cells that are transplanted into the degenerating retinas of patients. Cure Cell adds that its process, called xeno-free, transforms the stem cells without exposure to animal products, thus bypassing many safety issues.

The study by McGill and colleagues induced lab rats of various ages with retinal degeneration, and tested 3 dosage levels of clinical-grade OpRegen, with a 4th group left untreated for comparison. The researchers used electroretinography that measures the electrical response of light-sensitive areas of the retina and optomotor responses — movements of the eye to stabilize the image on the retina — as indicators of visual acuity after the treatments.

The results show the rats receiving treatments with OpRegen outperformed the rats left untreated on the visual function tests, although there were no statistically reliable differences among the 3 dosage levels. In addition, the researchers found in the treated rats a regrown layer of retinal and photoreceptor cells that transform light waves in images to signals sent for the brain.

Cure Cell received clearance from Food and Drug Administration and Israel’s health ministry to conduct a clinical trial of OpRegen with individuals having geographic atrophy, a severe form of dry age-related macular degeneration, for which there’s no current therapy. In the dry form of macular degeneration, protein deposits accumulate on the retina distorting one’s vision. In geographic atrophy, the protein deposits accumulate into more of a mass, leading to damage and loss of photoreceptor cells. The trial is recruiting 15 patients in Israel to test OpRegen’s safety and efficacy.

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