Gene therapy hopes for AMD
Author: Mark Gould
A woman from Oxford has become the first person in the world to have gene therapy to try to halt age related macular degeneration (AMD).
Surgeons injected a synthetic gene into the back of Janet Osborne's eye in a bid to prevent more cells from dying. It is the first treatment to target the underlying genetic cause of AMD which affects about 600,000 people in the UK.
Mrs Osborne, 80, is the first of 10 patients, all with dry AMD, taking part in a trial of the gene therapy treatment, manufactured by Gyroscope Therapeutics, funded by Syncona, the venture capital arm of the Wellcome Trust.
An injection is made at the back of the eye, which delivers a harmless virus containing a synthetic gene. The virus infects the retinal cells and releases the gene. This enables the eye to make a protein designed to stop cells from dying and so keep the macula healthy.
The early stage trial, at Oxford Eye Hospital, is primarily designed to check the safety of the procedure and is being carried out in patients who have already lost some vision. If successful, the aim would be to treat patients before they have lost any sight, in a bid to halt AMD in its tracks.
The treatment was carried out under local anaesthetic last month at Oxford Eye Hospital by Robert MacLaren, professor of ophthalmology at the University of Oxford.
He told BBC news: "A genetic treatment administered early on to preserve vision in patients who would otherwise lose their sight would be a tremendous breakthrough in ophthalmology and certainly something I hope to see in the near future."
It is too early to know if Mrs Osborne's sight loss in her left eye has been halted but all those on the trial will have their vision monitored. Mrs Osborne told BBC News: "I find it difficult to recognise faces with my left eye because my central vision is blurred - and if this treatment could stop that getting worse, it would be amazing."
Last year, doctors at Moorfields Eye Hospital, in London, restored the sight of two patients with AMD by implanting a patch of stem cells over the damaged area at the back of the eye. It is hoped that stem cell therapy could help many people who have already lost their sight.
But the Oxford trial is different because it aims to tackle the underlying genetic cause of AMD and might be effective in stopping the disease before people go blind.