Stem cells for the treatment of eye diseases?

Stem cells for the treatment of eye diseases? The US Food and Drug Administration (FDA) first approved the use of retinal epithelial cells derived from human embryonic stem cells in a study to treat juvenile macular degeneration.

11/23/2010

The US researchers want to treat the also known as Stargardt's disease eye disease using embryonic stem cells. To this end, twelve patients with juvenile macular degeneration will participate in a phase I / II study in which the participants implanted from the embryonic stem cells retinal epithelial cells under the retina (subretinal) implanted. The method has already been successfully tested in various animal studies on rats and mice, with the implanted cells protecting the photoreceptors of the retina from destruction and saving the eyesight of the animals. Robert Lanza of the biotech company Advanced Cell Technology (ACT), which manufactures the cells. „In rats, the stem cells brought a hundred percent improvement compared to untreated animals“, explained Lanza. The researchers hope to achieve similar therapeutic success in the use of the new method in people with macular degeneration.

The study is aimed specifically at patients with Stargardt's disease, a hereditary form of macular degeneration that results in the destruction of retinal pigment epithelium (RPE) cells on the retina. It is in most cases only as well „Yellow spot“ designated macula, the „Point of the sharpest vision“ affected by the disease. Stargardt's disease usually occurs between the ages of 10 and 20, and vision steadily declines. A therapy, with which the vision loss can be counteracted, does not exist so far and thus the patients suffer from a steadily progressive decrease of the visual acuity. The central visual field is the most affected. According to the researchers, around 25,000 people in the United States suffer from Stargardt's disease; in Germany, around 8,000 people are affected.

The US scientists around Dr. Steven Schwartz of the Jules Stein Eye Institute at the University of California, Los Angeles plans to implant 50,000 to 200,000 embryonic stem cell-derived RPE cells into the eyes of the twelve Stargardt patients under the retina. The researchers use the ACT-produced RPE cell preparation „MA09-hRPE“, Which in the US since the beginning of the year „Orphan drug status“ Has. Although the preparation is derived from human embryonic stem cells, it no longer contains any stem cells that could increase the risk of cancer, in this connection ACT emphasized. In addition to the study on juvenile macular degeneration, the company is also planning a study with patients suffering from age-related dry macular degeneration, Robert Lanza continued

Although the stem cell-derived RPE cells are now being used on humans, the manufacturer believes that it will take years before the procedure in the US becomes ever larger. Due to the highly controversial use of human embryonic stem cells, it is questionable whether the method would be allowed in Germany at all. The study also has to prove that there is an improvement in the eyesight of the twelve participants and that no serious side effects occur. (Fp)