Advanced Cell Technology seeks to test embryonic stem cells to treat blindness

AFP (11/19, Oberman) reported that “embryonic stem cell therapy got a step closer to the clinic Thursday after” researchers from Advanced Cell Technology “said they filed a request for” Food and Drug Administration “approval of human trials” involving “a single injection of retinal cells derived from embryonic stem cells” to treat Stargardt’s disease, a common type of “juvenile blindness.” The technique “works by replacing lost” retinal pigment epithelium cells “which maintain the photoreceptors needed for vision,” explained Robert Lanza, MD, the company’s chief scientific officer.

Reuters (11/20, Fox) reports that if the agency approves the application, a human phase I/II trial would take place in 12 patients to prove safety and efficacy.

The UK’s Independent (11/20) reports that Advanced Cell Technology plans “to follow this with an application to treat age-related macular degeneration, which…is the most common cause of blindness.” In animal tests, “transplants of the human cells into rats with macular degeneration resulted in a ‘100 percent improvement’ in vision with no side-effects, Dr. Lanza said.”

The Los Angeles Times (11/19, Kaplan) “Booster Shots” blog reported that Stargardt’s disease “is a childhood version of macular degeneration and affects about one in 10,000 kids. Patients typically begin to lose their central vision between the ages of six and 20,” with “about half of victims” becoming “legally blind by age 50.” In most cases of the disease, which has no cure, “children inherent a faulty version of the ABCA4 gene or the CNGB3 gene from both parents,” which causes “photoreceptor cells in the retina” not to “get enough fuel,” so “they atrophy.” The UK’s Telegraph (11/20, Moore) also covers the story.


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