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Surgeons perform first embryonic stem cell surgery aimed at reversing vision loss caused by AMD

29 September 2015

A cure for the most common form of blindness has been developed by British surgeons in a ground-breaking operation. Experts hailed the breakthrough as a “big step forward” in treatment of age-related macular degeneration (AMD).

It could now mean that surgery to reverse vision loss is routinely offered to the 600,000 patients who have the disease in the UK in the same way that cataract sufferers are treated.

Surgeons at Moorfields Eye in London carried out the first embryonic stem cell operation on a female patient. The patient suffers from the less common, but more aggressive wet AMD, characterised by leaking blood vessels, but scientists believe the same procedure can be applied to dry AMD.

The female patient, aged 60, who underwent surgery last month, is the first of 10 to take part in the trial. The procedure was successful, but it will be some months before the impact of it on her sight is known.

Surgeon Professor Lyndon Da Cruz from Moorfields Eye Hospital in London, said he was “very excited” by the promise held out by the new procedure. “The reason we are so excited is that we have been able to grow a perfect copy of the layer and transplant it. We are going right back to the roots of the disease.”

The cells were taken from donated embryos that were created during IVF treatment but never used. This is the first time experts have used a "patch" technique using embryonic cells with the aim of reversing vision loss in patients.
A US safety trial last year used a different technique, which restored vision in half of cases.

But the UK studies are further advanced, and having proved the technique is safe, researchers are now testing its effectiveness.

Prof Da Cruz has started the trial with wet AMD patients because there is potential to restore their sight faster following sudden vision loss, with results within about three months.

The study involves patients with wet AMD, because results can be seen more quickly, as vision loss is more sudden, but surgeons believe the technique with also work on patients with dry AMD.

Prof Chris Mason, Professor of Regenerative Medicine, UCL, described the trial as “potentially a big step forward towards curing a major cause of blindness.”

“If the AMD trials are successful, then by using embryonic stem cells as the starting material, the therapy can then be affordably manufactured at large scale. This will enable all patients to benefit and not just be an expensive bespoke therapy for a select few,” he said.

Prof Anthony Hollander, professor of stem Cell Biology, University of Liverpool, said the research would offer scientists new insights into the use of embryonic stem cells.

“The start of a trial using cells derived from embryonic stem cells for wet AMD is an important landmark.”

Current treatment for wet AMD involves regular injections of antibodies, which does not always work, and there is no treatment available for dry AMD, the more common type of the disease.

Experts said larger clinical trials would be needed before embryonic stem therapy became a mainstream treatment.

The operation is part of a programme, the London Project to Cure Blindness, which has been set up by Moorfields, the University College London Institute of Ophthalmology, the National Institute for Health Research and pharmaceutical giant Pfizer.

Clara Eaglen, from the Royal National Institute of Blind People, said: "We are hopeful that stem cell technology will significantly change the way in which people with sight loss are treated over the next decade."It is early days yet but this development does show that stem cells can be successfully transplanted into the eye, which is a great step forward."

Professor Michael Cheetham, member of the RP Fighting Blindness Medical Advisory Board commented, "This new clinical trial at the UCL Institute of Ophthalmology and Moorfields Eye hospital is another important step in the potential use of stem cells to treat blindness. The researchers have turned stem cells into retinal pigment epithelium (RPE cells) and put them on a patch then have transplanted this patch into the eye of one patient. They will treat 10 people in this trial to test if is safe and if it can help their vision. This therapy is designed for the wet and dry forms of age-related macular degeneration (AMD), but if it works it could also be used in the future for RP patients where the problem is mainly in their RPE cells and not their light sensitive photoreceptors. Examples of these types of RP are Sorbsy’s Fundus Dystrophy (TIMP3), or the visual cycle disorders (e.g. RPE65, LRAT), bestrophinopathies (BEST1) or phagocytosis problems (MerTK). Diseases like Stargardt’s (ABCA4), where the problem is that the photoreceptors ‘poison’ the RPE, or choroideremia (CHM), where both the RPE and photoreceptors are affected, might also be amenable to this type of therapy. However, stem cell therapy for the majority of RP patients will need stem cells to be made into photoreceptors and although this is still a long way off, if this trial is successful it will help bring that possibility closer."

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