Regenerative medicine company Advanced Cell Technology (OTCBB:ACTC) reached another important milestone Thursday as health authorities in the UK gave the go-ahead for trials to treat patients with the degenerative eye disease, Stargardt's Macular Dystrophy, using stem cell technology, the first time such studies have been approved in Europe.
The company is currently carrying out a similar trial in the United States at the Jules Stein Eye Institute in Los Angeles.
The UK trials will begin in December at the prolific Moorfields Eye Hospital in London, the oldest and largest eye hospital in the world, under Professor James Bainbridge, consultant surgeon and Chair of Retinal Studies at University College London.
Bainbridge, who will lead the study, commented: "There is real potential that people with blinding disorders of the retina including Stargardt's disease and age-related macular degeneration might benefit in the future from transplantation of retinal cells."
Stargardt's causes progressive vision loss, usually starting in people between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in a layer of the retina, called the retinal pigment epithelium (RPE).
Advanced Cell's key RPE product is derived from human embryonic stem cells (hESC) and can potentially be used to supply an unlimited amount of healthy RPE cells to treat degenerative retinal diseases such as Stargardt's, which is currently untreatable, and is one of the leading causes of juvenile blindness in the world.
The disease affects an estimated 80,000 to 100,000 patients in the US and Europe, which represents a $25-30 billion worldwide market that has yet to be effectively addressed.
RPE already carries Orphan Drug designation in Europe, a status which typically conveys advantages to companies developing treatments for rare medical conditions and diseases such as exclusivity periods, where the drug can be marketed without competition for a period of time, and financial incentives for clinical trials.
Chairman and CEO of Advanced Cell Technology, Gary Rabin, said: "We are pleased that the Moorfields Eye Hospital in London has agreed to participate as a site for this study as we continue to assess the capabilities of hESC-derived RPE cells to repair the retina and reduce the impact of these devastating eye diseases."
Advanced Cell Technology's portfolio also includes the Hemangioblast (HG) platform for the treatment of blood and cardiovascular diseases, as well as the development of a phase two-approved Myoblast autologous adult stem cell therapy for the treatment of chronic heart conditions.