Advanced Cell Technology, Inc. (ACT), a leader in the field of regenerative medicine, announced that it has received clearance from the UK Medicines and Healthcare products Regulatory Agency (MHRA) to begin treating patients as part of a phase I/II clinical trial for Stargardt’s Macular Dystrophy (SMD) using Retinal Pigment Epithelium (RPE) derived from human Embryonic Stem Cells (hESCs). ACT received similar approval from the the Gene Therapy Advisory Committee (GTAC), which has responsibility for the ethical oversight of proposals to conduct clinical trials involving gene or stem cell therapies in the UK. The European Medicines Agency (EMA) previously granted Orphan Drug designation for the company's RPE cell product for use in treating SMD.
“This is another important milestone for ACT and for the field of regenerative medicine,” said Gary Rabin, chairman and 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 I/II clinical trials for Stargardt’s macular dystrophy and dry Age-related Macular Degeneration (dry AMD) with hESC-derived RPE cells in the US, and both patients successfully underwent the outpatient transplantation surgeries. Clearance from the MHRA to begin an SMD trial in the UK is the first step in our European clinical trial programme. 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 programmes, 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 US 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 US 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 US 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, MD, 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 US 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 analysing 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.”
Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. As many as 30 million people in the US and Europe suffer from macular degeneration, which represents a $25-30 billion worldwide market that has yet to be effectively addressed.