The leading cause of progressive blindness may be able to be treated by using human embryonic stem cells, according to the online Medical News Today (www.medicalnewstoday.com.) On Jan. 3, 2011, the United States Food and Drug Administration (FDA.) announced that it has approved clinical trials of an application to treat dry age-related macular degeneration (AMD) by using retinal pigment epithelial cells derived from human embryonic stem cells. About 10 to 15 million Americans have AMD.
The company Advanced Cell Technology, or ACT, stated that they have received permission to begin a clinical trial to treat patients with dry AMD, which is the most common form of macular degeneration in the world. The other form is wet macular degeneration, which is far rarer. Dry AMD can cause great vision loss over time, and even blindness. There are no current treatments available for this disease.
During the clinical trial, 12 patients will be enrolled in the study at different clinical sites. The sites that are being considered are the Jules Stein Eye Institute at UCLA, and the Ophthalmology Department at Stanford University School of Medicine. More sites may be considered.
Dr. Robert Lanza, ACT’s Chief Scientific Officer is optimistic about this possible stem cell treatment of dry AMD. He said that only a small number of cells are needed to treat each patient. Lanza stated, “Based on our animal model studies, we are very excited about the opportunity to treat patients. In a rat model of macular degeneration, we have seen a remarkable improvement in visual performance over untreated animals, without any adverse effects. We also maintained near-normal function in a mouse model of Stargardt’s disease, a form of juvenile macular degeneration.”
The initial part of the clinical trial will focus on safety. In following trials the ACT hopes to show that the retinal pigment epithelial cells that are injected into the retinal space will be able to slow or halt the progression of the disease, and even restore some sight to the patient.