Trials of an experimental new treatment that could offer hope to young people suffering from a currently untreatable inherited eye condition will get underway soon at Moorfields Eye Hospital following approval today, Thursday 22 September, by the Medicines and Healthcare Products Regulatory Agency (MHRA).

The trials, due to start in the next few months, will investigate the safety of using retinal cells derived from stem cells to treat people with advanced Stargardt disease, a form of macular degeneration that causes disabling loss of sight in young people. The cells will be injected into the retina during an operation lasting up to an hour.

“There is real potential that people with blinding disorders of the retina, including Stargardt disease and age-related macular degeneration, might benefit in the future from transplantation of retinal cells,” says retinal surgeon Professor James Bainbridge, who will be conducting the trials at the National Institute for Health Research (NIHR) Biomedical Research Centre based at Moorfields and the UCL Institute of Ophthalmology.

“The ability to regenerate 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.”

“We are delighted to be the site for these very exciting new clinical trials in stem cell therapy, which have the potential to give hope and make such a difference to the lives of people with currently untreatable blinding retinal conditions,”adds Professor Peng Khaw, director of the biomedical research centre at Moorfields. 

“It is crucial that we continue to attract external investment for world-leading research in the UK, through the NIHR's transformational investment in research infrastructure through national biomedical research centres such as ours here at Moorfields and the UCL Institute of Ophthalmology. " 

The technology has been developed by US company Advanced Cell Technology (ACT), who got the go ahead to run similar trials in the United States in November 2010.

Further information for patients is available here.

Adapted from Moorfields Eye Hospital

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