"Legally blind" patients have improved vision after four months of experimental stem-cell treatment, claim scienists


'Legally blind' patients have improved vision after four months of experimental stem-cell treatment, claim scientists


Marcus Hilton (pictured with his daughter Amelia), is the first person in Europe to take part in stem cell trial for a rare eye disease

Marcus Hilton (pictured with his daughter Amelia), is the first person in Europe to take part in stem cell trial for a rare eye disease

A British man has become the first in Europe to take part in a stem cell trial for a rare and devastating eye disease.

Marcus Hilton, 34, is taking part in the European arm of a trial at Moorfields Eye Hospital, London, to assess safety which has already shown promising results in the US.

He is the first of 12 patients in Europe with Stargardt's macular dystrophy (SMD) to receive retinal pigment epithelial (RPE) cells derived from human embryonic stem cells.

Mr Hilton was injected with the cells during a 90-minute operation on Friday. Although it will be years before the treatment is proven, early results from the US suggest the method is safe and could lead to a suitable therapy.

Mr Hilton, who has a four-year-old daughter Amelia, said he was 'thrilled' and 'excited' to be taking part in the trial. He has suffered from severe Stargardt's, a hereditary disease which causes loss of central vision, for as long as he can remember.

He was aged seven when a problem was picked up at school but the diagnosis was not confirmed until three years later.

'I was aged about 10 when it was diagnosed by specialists at Moorfields,' Mr Hilton said. 'Besides being unable to drive, having the disease has an impact and I have had to engineer my life around it.

'I would never read out of choice because it is too much like hard work. And I'm unable to read a newspaper. When I was at school there was also ongoing hassle with reading blackboards and text books.'

Mr Hilton, from Wakefield, West Yorkshire, who runs two bars, is hopeful he could eventually have some sight restored, which would enable him to read books with his daughter.

He said: 'I'm over the moon they have had early results in America showing this treatment could work. It could change many people's lives – to have some sight restored would be a dream come true.'

Mr Hilton took a course of immunosuppressant drugs before Friday's operation, which passed without any complications.

'I was put under anaesthetic and they made a small hole in the front of my right eye to inject the stem cells,' he said. 'At the moment, because it was only done a few days ago, everything is very blurred.'

Mr Hilton's trial was led by Professor James Bainbridge, consultant surgeon at Moorfields.

He said: '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.

'Testing the safety of retinal cell transplantation in this clinical trial is an important step towards achieving this aim. We are very pleased that the first transplant surgery has gone smoothly and look forward to seeing the results as the trial progresses over the next two years.

Mr Hilton was part of the trial at Moorfields Eye Hospital as part of a global study

Mr Hilton was part of the trial at Moorfields Eye Hospital as part of a global study

'While this is primarily a safety trial, we will have the opportunity to monitor engraftment of retinal cells and to assess any impact on sight.'

He added: 'We are very excited to be working with ACT on the first human embryonic stem cell trial in Europe. Stargardt's macular dystrophy is a serious and progressive disease that usually starts between the age of 10 and 20 years.

'It includes degeneration of RPE cells in the macula at the centre of the retina, the region specialised for high acuity vision. With the loss of RPE cells in the macula comes the eventual loss of light-sensitive photoreceptor cells, leading to blindness at the prime of life.

'We hope that transplantation of healthy RPE cells might also help in other significant degenerative eye diseases affecting the retina for which there are no effective treatment options – particularly dry age-related macular degeneration which is the leading cause of blindness in Europe.'

Gary Rabin, chairman and chief executive officer of Advanced Cell Technology (ACT), a US biotech company running the trial, said it the trial had 'taken another critical step'.

He said: 'The treatment of the first patient in Europe is tangible evidence that stem cell research and development of cell therapies is making progress.

'It is a milestone for scientists, stem cell advocates and patients hoping for cures as well, as much as it is one for ACT. We are honoured to be working with Prof Bainbridge at Moorfields Eye Hospital, and are very pleased with the smooth progress of the trial thus far.'

It is hoped further patients will be enrolled on the trial at Moorfields.