Advanced Cell Technology Inc. (OTC:ACTC) is set to begin Europe’s first study involving human embryonic stem cells, as it begins a trial to consider an experimental treatment for a form of juvenile blindness.
"This is the first time an embryonic stem cell trial has ever been approved anywhere else in the world [outside the U.S.]," chief scientific officer Dr. Robert Lanza said.
The Marlborough, Mass.-based regenerative medicine company will inject stem cells derived from human embryos into the eyes of 12 enrolled patients, hoping to prove it an effective and safe treatment for Stargardt’s muscular dystrophy, an incurable disease that’s one of the main causes of blindness in young people.
The news is another boon for ACT, which earlier this year won a positive decision on its human embryonic stem cell-derived retinal pigment from the European Medicines Agency’s Committee for Orphan Medicinal Products, giving its stock a 7.6 percent boost just a day after it inexplicably sank 20 percent.
"This is another important milestone for ACT and for the field of regenerative medicine," said chairman & CEO Gary Rabin in prepared remarks. "Clearance from the MHRA to begin an SMD trial in the U.K. is the first step in our European clinical trial program. Europe not only represents the world’s second-largest pharmaceutical market, but it is also home to some of the best eye hospitals and surgeons in the world."
In July ACT became the second company in the U.S. to win FDA permission to study a therapy derived from human embryonic stem cells when it successfully dosed the first patients in U.S. clinical trials of stem cell implantation for blinding eye diseases that are generally considered untreatable.
Regulatory nods and successful trials not only help the company, but advance the field of human embryonic stem cell research in general, ACT said.
"It’s vindication," Lanza told Xconomy when the U.S. trials began. "In the early days, we were called murderers. We almost went under a few times. This was not easy."
The European trial, set to begin in December, will take place at Moorfields Eye Hospital in London.