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Human embryonic stem cells may help older people battle blindness

January 03, 2011|By Karen Kaplan, Los Angeles Times
  • A physician highlights the part of the retina that is destroyed in a patient with age-related macular degeneration.
A physician highlights the part of the retina that is destroyed in a patient… (Chiaki Kawajiri/Baltimore…)

A therapy derived from human embryonic stem cells may help millions of Americans battle a common form of blindness.

Advanced Cell Technology Inc. said Monday that it has received a green light from the U.S. Food and Drug Administration to begin a clinical trial to test its therapeutic cells as a treatment for dry age-related macular degeneration. The company plans to enroll a dozen patients in a Phase I/II trial, which would primarily test the safety of the cells and whether they are well-tolerated by patients.

Age-related macular degeneration is a disease in which the macula – in the middle of the retina in the back of the eye – is gradually destroyed. In the dry version of AMD, objects in the center of a patient’s visual field become blurry and they have trouble recognizing faces, according to this explainer from the National Eye Institute. Though there are treatments that may stall the progression of dry AMD, there is no cure. An estimated 10 million to 15 million Americans suffer from dry AMD, and that number is expected to rise as the population ages.

The scientists at Advanced Cell hope that these patients can be helped by the company’s retinal pigment epithelial (RPE) cells, which would theoretically replace the ones that have been destroyed by the disease. The RPE cells have restored vision in rats and mice with an animal version of the disease. In November, the FDA gave the company the go-ahead to test the RPE cells in children with a rare disease called Stargardt’s macular dystrophy.

The macular degeneration study will enroll patients at various sites across the country. So far, the Jules Stein Eye Institute at UCLA and the Ophthalmology Department at Stanford University School of Medicine are being considered, according to a statement from the company. More information about the trial will be posted at ClinicalTrials.gov soon, the company said.

As explained in previous Booster Shots posts, these RPE cells were derived from an unusual line of human embryonic stem cells. The stem cell line was created from a single blastomere cell that was extracted from an eight-cell embryo using a technique that does not require the destruction of an embryo. In fact, the blastomere extraction technique is commonly used in pre-implantation genetic diagnosis.

RELATED: FDA clears second clinical trial involving human embryonic stem cells

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