U.K. regulators have given the green light to a small but groundbreaking embryonic stem cell treatment advanced by the Marlborough, MA-based biotech Advanced Cell Technology for Stargardt's macular dystrophy, an incurable disease. This is the first such ESC trial in Europe, which hopes to offer a glimpse of whether an injection of healthy retinal cells can arrest or even reverse the disease. A similar study was launched at UCLA in July while Geron has its own ESC study underway in the U.S. for a different target.
"This is a safety and tolerability study, so we are dealing with patients with advanced stage disease. Where we expect to get the most significant results is in earlier patients, before they have lost their photoreceptors. We're hoping to prevent the onset of blindness altogether in those patients," ACT CSO Robert Lanza told The Guardian.
James Bainbridge at the Moorfields Eye Hospital, who will conduct the trial in London, said it was "hugely exciting" to be given a chance to start testing these retinal cells made from ESCs.
It's also a big step for ACT ($ACTC), which has skirted financial disaster more than once over the years. This morning its shares were trading at 16 cents. "Building international relationships around our clinical programs, such as with Professor James Bainbridge at Moorfields Eye Hospital is very important to our strategy of developing new regenerative medicine therapies," says ACT CEO Gary Rabin.
In an online Q&A at The New York Times, Stephen Rose, the research chief for the Foundation Fighting Blindness, cautioned that the ACT ESC study is very early stage, with a big focus on safety at this point. But he also noted hopes that the ESC approach could provide a long-term solution, or even a cure.
"Advanced Cell Technology's cell treatment is also potentially a one-time procedure," says Rose. "Once the cells are in the eye, it is hoped that they will work for several years, perhaps a lifetime."
In a related story, the first patient in Geron's ESC study for spinal cord damage has been treated. Stanford investigators injected two million stem cells into a damaged spinal cord to replace damaged neurons.