A small biotech firm is the second company to start human tests of embryonic stem-cell therapy.
JULY 14, 2011
BY ANTONIO REGALADO
In a bid to harness the potential of embryonic stem cells, surgeons in California have implanted lab-grown retinal cells into the eyes of two patients going blind from macular degeneration.
The procedures were carried out on Tuesday by Steven Schwartz, chief of the retina division at the Jules Stein Eye Institute at the University of California, Los Angeles. They were financed by Advanced Cell Technology, a biotech company with laboratories in Marlborough, Mass. that recently won approval from the U.S. Food and Drug Administration to test the treatment in 24 patients suffering from either dry advanced macular degeneration, or a juvenile form of the disease known as Stargardt's.
The two patients, whose names weren't released, are among the first volunteers ever to receive a treatment created using embryonic stem cells.
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Researchers see the start of a second set of tests, in blindness, as an important landmark for the stem-cell field.
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The start of the clinical trials is likely to stir hopes of a big payday among investors. Advanced Cell's stock price has risen 170 percent during the last year, valuing the firm at $285 million based on Tuesday's stock price. Investors hope it could be worth several billion should the therapy succeed. Nearly 10 million Americans suffer from some form of macular degeneration.
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"We are excited about this treatment, because we think this has the potential to slow the disease progression," said Steve Rose, chief research officer of the Foundation Fighting Blindness, a nonprofit that invested about $2 million in early animal testing of the treatment, but is no longer directly involved. "This company has had their ups and downs, and I am really happy to see they got into the clinic. We've had our fingers crossed."
During a recent visit to Advanced Cell's laboratories, a research technician adjusted a microscope to show off the company's lead product: cube-shaped retinal pigment epithelial cells growing in a petri dish. Some were translucent, while others already had the brownish coloring of a mature cell. (The pigment absorbs stray light in the eye, acting as a kind of glare shield.)
These retinal cells are the type that are killed off in macular degeneration, eventually leading to the death of photoreceptors, and the gradual loss of central vision. Advanced Cell believes that injecting new, lab-grown cells into the eye may cure the condition.
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