An embryonic field seems to be developing rapidly. Just two months after Advanced Cell Technology (ACT) launched the second and third trials involving human embryonic stem cell (ESC) derived products in an operating room at the University of California–Los Angeles, the Santa Monica-based biotech announced today that it had gained approval from the UK Medicines and Healthcare products Regulatory Agency to conduct the first ESC cell trial outside the US.
“This is extremely significant,” Robert Lanza, ACT’s chief scientific officer, told Nature Medicine. “This is the first time there’s been an embryonic stem cell therapy trial anywhere else in the world.”
The trial will essentially be a repeat performance of one of ACT’s ongoing trials in the US, but this time conducted at the Moorfields Eye Hospital in London. Led by Moorfields ophthalmologist James Bainbridge, the trial, which is scheduled to begin before the end of the year, will use retinal cells derived from ESCs to treat 12 people suffering from Stargardt’s macular dystrophy, a progressive juvenile vision loss disorder that affects about one in every 10,000 children.
According to Lanza, in the near future ACT also expects to gain UK approval to use the same cell therapy to treat people with age-related macular degeneration, a common cause of blindness in the elderly and the other disease currently under investigation by the company in US trials. Meanwhile, ACT is also in late-stage talks with regulators and clinicians in France, China and elsewhere to launch further global trials.
“We have things ready to move forward in other parts of the world,” says Lanza. “But we don’t want to put the cart before the horse. We want to see how the patients do first. Between the US and UK studies, we should have enough data before we storm ahead.”
In other clinical stem cell news, Geron Corp., the Silicon Valley biotech behind the world’s first ES cell trial, announced this week that it had treated a fourth patient with its experimental therapy involving ESC-derived neural precursor cells. The unidentified study subject, who recently suffered a serious spinal cord injury and is now paralyzed from the waist down, was treated over the weekend at Santa Clara Valley Medical Center near San Jose, California, the San Francisco Chronicle reported yesterday. Geron plans to treat at least ten people in its phase 1 safety study.