A U.S.-based company has received permission to start Europe's first clinical trial involving human embryonic stem (hES) cells. Advanced Cell Technology (ACT), based in Marlborough, Massachusetts, received approval today from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to begin a trial that will treat 12 patients with Stargardt's macular dystrophy. The disease strikes people between the ages of 10 and 20, causing progressive vision loss. There is currently no treatment.
Eye surgeon James Bainbridge of Moorfields Eye Hospital and University College London will lead the U.K. trial. He and his colleagues will inject into patients' eyes retinal pigment epithelial cells that ACT derived from hES cells. In animal models, the cells have been able to stave off or even reverse disease progression. The phase I/II trial will primarily examine the treatment's safety, however.
Robert Lanza, ACT's chief scientific officer, says the trial has essentially the same design as the company's U.S. trial with Stargardt's patients, which received approval last year. The fact that the U.S. Food and Drug Administration had approved the trial, however, did not make it a shoo-in with U.K. authorities, Lanza says. "I thought it was going to sail right through. I was wrong; they were very rigorous."
The new trial is the first one using hES cells to receive approval outside the United States, Lanza says. The company plans to apply for approval for a second U.K. trial for age-related macular degeneration, he adds, which would complement its U.S.-based trial for the same condition. They then plan to wait until the first results are in before starting any additional trials, he says. The first two U.S. patients received the company's cells in July—one patient with Stargardt's disease and one with age-related macular degeneration. The first indications are positive, Lanza says. "We're very pleased with what we see so far."