The second clinical trial involving stem cells from human embryonic cells has received the go-ahead. The news came from Advanced Cell Technology Inc. this Monday. The company announced that the U.S. Food and Drug Administration has cleared the way for its Phase I/II trial of retinal cells for patients with Stargardt’s macular dystrophy, a childhood version of macular degeneration.
Up to 12 patients will be enrolled at several sites across the country, including the Casey Eye Institute in Portland, Ore., the University of Massachusetts Memorial Medical Center in Worcester, and UMDNJ – New Jersey Medical School in Newark.
Stargardt’s macular dystrophy is a childhood disease that affects about one in 10,000. Initially patients begin to lose their central vision between the ages of 6 and 20. As SMD progresses, things may look blurry and distorted and patients may have trouble adjusting to low light. About half of victims are legally blind by age 50. There is no cure.
This new stem cell technology would treat these patients by giving them new retinal pigment epithelium cells to replace the ones that are lost to the disease. The therapy has been successful in lab animals. The cells were grown from a line of human embryonic stem cell that was made by extracting a single blastomere cell from an eight-cell embryo. The technique is commonly used in pre-implantation genetic diagnosis and does not require the destruction of an embryo. Last month, a patient in Atlanta became the first patient to be treated in an FDA-approved clinical trial involving a therapy derived from human embryonic stem cells.
Robert Lanza, ACT’s chief scientific officer, said that these RPE cells have undergone strict tests to meet safety standards. The protocol involves injecting 50,000 to 100,000 RPE cells into the vitreous cavity of the eye. If successful, the treatment could be extended to address other degenerative eye diseases as well, including retinitis pigmentosa and macular degeneration. Lanza said, “We’re starting out with a safety study in those with advanced disease…But hopefully if we get in earlier in the progression of the disease we might see greater impact on visual improvement. What this approval shows is that the readiness of the FDA to work with researchers to move exciting new stem cell based therapies out of the lab and into the clinic.”