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
