Advanced Cell Technology is testing a stem-cell treatment for blindness that could preserve vision and potentially reverse vision loss.
A new treatment for macular degeneration is close to the next stage of human testing—a noteworthy event not just for the millions of patients it could help, but for its potential to become the first therapy based on embryonic stem cells.
This year, the Boston-area company Advanced Cell Technology plans to move its stem-cell treatment for two forms of vision loss into advanced human trials. The company has already reported that the treatment is safe (see “Eye Study Is a Small but Crucial Advance for Stem-Cell Therapy”), although a full report of the results from the early, safety-focused testing has yet to be published. The planned trials will test whether it is effective. The treatment will be tested both on patients with Stargardt’s disease (an inherited form of progressive vision loss that can affect children) and on those with age-related macular degeneration, the leading cause of vision loss among people 65 and older.
Although complete data from the trials of ACT’s treatments have yet to be published, the company has reported impressive results with one patient, who recovered vision after being deemed legally blind. Now the company plans to publish the data from two clinical trials taking place in the U.S. and the E.U. in a peer-reviewed academic journal. Each of these early-stage trials includes 12 patients affected by either macular degeneration or Stargardt’s disease.
The more advanced trials will have dozens of participants, says ACT’s head of clinical development, Eddy Anglade. If proved safe and effective, the cellular therapy could preserve the vision of millions affected by age-related macular degeneration. By 2020, as the population ages, nearly 200 million people worldwide will have the disease, estimate researchers. Currently, there are no treatments available for the most common form, dry age-related macular degeneration.