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ACT Receives Approval for First Human Embryonic Stem Cell Trial in Europe

ACT Receives Approval for First Human Embryonic Stem Cell Trial in Europe

22 September 2011

Moorfields Eye Hospital in London is the site for Phase 1/2 trials to treat Stargardt's Disease using RPE derived from stem cells.

US company Advanced Cell Technology Inc (ACT) announced today that it has received clearance from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to begin treating patients as part of a Phase 1/2 clinical trial for Stargardt's Macular Dystrophy (SMD) using retinal pigment epithelium (RPE) derived from human embryonic stem cells.
ACT has received similar approval from the Gene Therapy Advisory Committee, which ethical oversight responsibility for clinical trials involving gene or stem cell therapies in the U.K.

Statement from the Company

"This is another important milestone for ACT and for the field of regenerative medicine," said Gary Rabin, CEO of ACT. "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. We recently announced the dosing of the first patients in our Phase 1/2 clinical trials for Stargardt's macular dystrophy and dry age-related macular degeneration (dry AMD) with hESC-derived RPE cells in the U.S., and both patients successfully underwent the outpatient transplantation surgeries. 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.

"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."

Stargardt's Macular Dystrophy affects an estimated 80,000 to 100,000 patients in the U.S. and Europe, and causes progressive vision loss, usually starting in people between the ages of 10 to 20. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, the retinal pigment epithelium. The first patient to be treated in the U.S. with stem cell-derived RPE cells was a young woman who was already legally blind as a consequence of this disease. This newly-approved clinical trial in Europe will be a prospective, open-label study designed to determine the safety and tolerability of RPE cells derived from hESCs following sub-retinal transplantation to patients with advanced SMD, and it is similar in design to the FDA-cleared U.S. trial initiated in July.

"This is the first time an embryonic stem cell trial has ever been approved anywhere else in the world," said Robert Lanza, M.D., ACT's chief scientific officer. "Stargardt's disease is currently untreatable, and is one of the leading causes of juvenile blindness in the world. Collectively, degenerative eye diseases afflict over 25 million people in the U.S. and Europe alone. These diseases have a devastating impact on patients and their families, which has been a strong motivating factor for developing this new treatment. In Stargardt's disease, the loss of RPE cells in the patient's macula causes a loss of photoreceptors -- the cones and rods with which we see -- leading to blindness. We believe that transplanting new, healthy RPE cells may provide an effective treatment for SMD and perhaps other macular degenerative diseases such as dry AMD. We are excited to start these trials in Europe, and look forward to analyzing the data we continue to collect in our ongoing trials to determine the engraftment and function of the transplanted RPE cells."

The trial will be led by Professor James Bainbridge, consultant surgeon at Moorfields Eye Hospital, and Chair of Retinal Studies at University College London.

"Stargardt's disease is a form of macular degeneration that causes disabling loss of sight in young people and is currently untreatable," said Professor Bainbridge. "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. The ability to generate retinal cells from stem cells in the laboratory has been a significant advance and the opportunity to help translate such technology into new treatments for patients is hugely exciting. Testing the safety of retinal cell transplantation in this clinical trial will be an important step towards achieving this aim."

RP Fighting Blindness Response

News that the trials are to be run in the UK was warmly welcomed by Prof Paul Bishop, Professor of Ophthalmology and Matrix Biology at Manchester Eye Hospital and Chair of the RP Fighting Blindness Medical Advisory Board. David Head, Chief Executive at RP Fighting Blindness, added "This is great news; advances in this type of technology offer the best prospects in the longer term for people facing sight loss as a result of RP.

"It's important to bear in mind that these are trials, not generally available treatments, and we will have to remain patient, but it is a fantastic step forward and yet more cause for optimism in the face of what has, untill now, been seen as an incurable condition."

Professor Bainbridge is well known to RP Fighting Blindness, having been involved in the clinical trials of gene therapy at the Institute of Ophthalmology over the last few years.

Patients with enquiries about the trial can contact the team at mol.therapy@ucl.ac.uk.