Associated Press, November 25, 2010 at 9:06 PM: "Test OK'd for stem-cell treatment of rare disease":
NEW YORK — For only the second time, the U.S. government has approved a test in people of a treatment using embryonic stem cells — this time for a rare disease that causes serious vision loss. Advanced Cell Technology (ACT), a biotechnology company based in Santa Monica., Calif., said the research should begin early next year, following the green light from the U.S. Food and Drug Administration.
...ACT's experiment will focus on Stargardt disease, which affects only about 30,000 Americans. But the company hopes the same approach will work for similar and more common eye disorders like age-related macular degeneration, which affects millions. Stargardt is an inherited disorder that attacks central vision used for tasks like reading and recognizing faces. Some patients go totally blind, even losing peripheral vision, while others are severely impaired and can only perceive light or see their hands moving in front of their faces.
...The disease typically starts in adolescence. The key problem is that impaired scavenger cells fail to remove toxic byproducts from the eye, allowing them to build up and kill other cells. There is no proven treatment. In the new study, 12 patients will be treated with healthy scavenger cells, created in a laboratory from human embryonic stem cells. This early phase of the research is primarily to test the safety of various doses, injecting only one eye of each patient.
Los Angeles Times, November 22, 2010, 2:02 p.m.: "FDA clears second clinical trial involving human embryonic stem cells":
Another therapy derived from human embryonic stem cells is headed for clinical trials. Advanced Cell Technology Inc. said Monday that the 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.
...Advanced Cell Technology hopes to treat these patients by giving them new retinal pigment epithelium cells to replace the ones that are lost to the disease. The therapy has restored vision in rats and mice, according to published studies. The Phase I/II trial is designed to test the safety and tolerability of the RPE cells in humans.
Reuters, November 22, 2010 5:20pm EST: "Second U.S. company gets stem cell go-ahead":
The U.S. Food and Drug Administration has approved the second human trial of human embryonic stem cells -- this one testing cells in people with a progressive form of blindness, the company said on Monday. Massachusetts-based Advanced Cell Technology said it would start testing its stem cell-based treatment on 12 patients with Stargardt's macular dystrophy. It is the second trial of human embryonic stem cells to be approved by the FDA this year. Last month Geron Corp enrolled the first patient in its study using the cells in people whose spinal cords have been crushed.
Time, November 22, 2010: "FDA Approves Second Trial of Stem-Cell Therapy":
For only the second time, the Food and Drug Administration approved a company's request to test an embryonic stem cell-based therapy on human patients. Advanced Cell Technology (ACT), based in Marlborough, Mass., will begin testing its retinal cell treatment this year in a dozen patients with Stargardt's macular dystrophy, an inherited degenerative eye disease that leads to blindness in children. ...ACT's trial will involve injecting retinal pigment epithelium (RPE) cells, which nurture the retina, into volunteers with the most advanced forms of Stargardt's, in an attempt to replace dying and no longer functioning photoreceptor cells. In animals, the infusion of healthy cells improved vision and rescued the function of some diseased cells.
New Scientist, 22 November 2010, 16:36 GMT: "Stem cell trial for blindness gets green light":
Twelve people left almost blind by a hereditary condition that strikes in childhood are to receive the world's first eye therapy derived from human embryonic stem cells (hESCs). The treatment is for Stargardt's macular dystrophy, which affects 1 in 8000 people in the US. Their sight deteriorates from around age 6 when retinal pigment epithelial cells (RPEs) start to die off rapidly, possibly due to a defective gene. Without RPEs to support and nourish them, adjacent photoreceptor cells which capture light signals, die too and blindness is the result. People in the trial will be those whose vision has deteriorated to the point where they can see the movement of their own hand, but little else. They will receive injections into their eyes of between 50,000 and 200,000 RPEs.
"The goal is to halt the rate of photoreceptor loss," says Robert Lanza, chief scientist at Advanced Cell Technology (ACT) of Worcester, Massachusetts, the company that has been developing the treatment since first turning hESCs into RPEs in 2004. By implanting new RPEs, which do not contain the defective gene, the team hopes to prevent further deterioration or perhaps even reverse it.
Boston Globe, November 22, 2010: "ACT wins FDA’s approval to test stem cell therapy":
Advanced Cell Technology, a stem cell company with a research laboratory in Marlborough, has received federal approval to test a human embryonic stem cell treatment on patients with a rare disease that causes blindness. ...ACT filed an investigational new drug application with the Food and Drug Administration a year ago to use retinal cells derived from human embryonic stem cells to treat patients with Stargardt’s, a rare genetic disease that affects about 30,000 people in the United States. The disease causes progressive vision loss, starting in patients between 10 and 20 years old, and results in blindness.
