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 its embryonic stem cell-based therapy is safe enough to resume testing in humans, hopefully before the end of next summer.
Embryonic stem cells have the ability to develop into multiple types of cells, leading to great excitement about their potential to replace diseased or damaged adult cells, as well as concerns about unintended side effects.
ACT has taken great care to select Stargardt’s as an initial target for its embryonic stem cell-derived therapies, Caldwell said. The firm would use stem cells to grow retinal pigment epithelial cells, which would be injected into patients’ eyes. One bonus of this strategy, says Caldwell, is that the retinal pigment cells have a different color than most cells, making them easy to track in the body.
There’s also a serious need for therapies, given that there are no FDA-approved treatments for the 25,000 Americans with Stargardt’s disease, which causes the degeneration of light-detecting cells, according to the American Macular Degeneration Foundation. Advanced Cell’s therapy would replace the cells that support these photoreceptor cells, with the intent of stymieing the progress of the disease. About the best way patients now have to slow the disease is to wear sunglasses to help protect the healthy photoreceptor cells that they still have.
When tested in mice with Stargardt’s disease, ACT’s cellular therapy showed that it could help fight the degenerative effects of the disease. The company is asking the FDA for permission to test the safety of the treatment in a dozen patients at three U.S. medical centers, including the University of Massachusetts Memorial Medical Center in Worcester and the Casey Eye Institute in Portland, OR.
Ed Mickunas, the company’s vice president of regulatory affairs, said that the firm may proceed with the development of its retinal cell therapy for other eye diseases such as age-related macular degeneration if the early study in Stargardt’s is successful.
Still, it’s going to cost a lot more money than ACT has on hand to bring its Stargardt’s treatment through clinical trials. The company told investors that it ended September with $590,000 in the bank and total cash or cash equivalents of $1.1 million. However, the company has agreements with lenders that will enable it to borrow funds to continue operating for at least two more years, Caldwell said. (In fact, the firm disclosed to the SEC earlier this month that it raised $2.8 million in a debt and options financing.)
The company’s stock has been hovering at around 10 cents per share, giving the company a market value just shy of $50 million.
ACT has reduced its operations to stay in business, closing labs in Alameda, CA, and Charlestown, MA, last year, Caldwell said. The firm is now operating with 14 full-time workers, with additional support from research partners and consultants. It also decided to save money by delaying its mid-stage clinical trial for a therapy in which adult stem cells—and not stem cells from embryos—are used to treat heart disease.
While the firm’s near-term success hinges on the success of its embryonic stem cell therapies, the firm is also participating in the hot field of using adult cells to make stem cells, Caldwell said. (This is not to be confused with treating people with adult stem cells, which has been done for years to treat cancer patients.) Technology that induces adult cells into a stem cell-like, or pluripotent, state has captured a lot of attention because it doesn’t require the use of embryos. Investors are swooning over companies like San Diego-based Fate Therapeutics, which is developing therapies based on such advances in stem cell research, while ACT and other earlier developers of stem cell therapies have struggled to fund their R&D efforts.
Caldwell said it’s important to proceed with the development of embryonic stem cell-based therapies because they are closer to being able to provide benefits to patients, while it will take many more years for approaches based on induced pluripotent stem (IPS) cells to catch up. “The IPS technology is still as science project,” Caldwell said.
Caldwell said that ACT is able to harvest human embryonic stem cells from without damaging embryos, using the same methods that are employed to test embryos for genetic disorders before babies are born.
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