Advanced Cell Technology Betting Future on Embryonic Stem Cell Therapy for Eye Disease

Advanced Cell Technology is banking on an experimental cellular therapy for a rare eye disease to show the world that controversial embryonic stem cells have a place in treating diseases. Amid serious financial constraints at the Worcester, MA-based biotech firm (OTCBBACTC), a plan is in place to pioneer the use of cellular therapies made with human embryonic stem cells as treatments for people.

The biotech last month asked the FDA for permission to conduct a Phase I/II clinical trial of its stem cell therapy for Stargardt’s Macular Dystrophy, a genetic disease that is a leading cause of blindness in children. Bill Caldwell, the company’s chairman and CEO, told me this week that the experimental Stargardt’s treatment is the firm’s top priority and its best chance to prove that embryonic stem cells can make effective treatments for diseases.

The company and the field of human embryonic stem cells both need a big win. Advanced Cell Technology (ACT) has operated for 15 years without a commercial success, and the firm has been forced to nearly halve its operations since last year to stay afloat. Research on human embryonic stem cells has been mired in controversy and political attacks over fears about destroying human embryos to harvest stem cells—even though ACT and other firms have found ways to produce stem cells without harming embryos. And companies like ACT have been slow to move embryonic stem cell therapies into human clinical trials, despite the promise of such cells to provide the building blocks for regenerating tissues to repair organs damaged by serious conditions like heart disease, macular degeneration, and spinal cord injuries.

“This industry is in need of successes,” said Caldwell. “The technology of embryonic stem cells is very powerful and it needs validation.”

ACT has the burden of trying to become one of the first companies to test human embryonic stem cells as a therapy in humans, meaning the FDA, which must okay the clinical trials, has lots of questions about their potential health effects. Caldwell said that his firm has been in discussions with the FDA about the its proposed clinical trial with Stargardt’s patients, and the he hopes to have addressed all the agency’s concerns in time to begin the pioneering study in the third quarter of 2010.

The FDA has only authorized one company to test the use of embryonic stem cell therapies in humans: Menlo Park, CA-based biotech Geron, which was forced delay the its first human study for treating victims of spinal chord injuries in August due to the FDA’s concens about data it received on the treatment after it granted permission to conduct the trial. Geron (NASDAQ:GERN) said in October that it’s conducting animal tests to show the FDA that … Next Page »

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