British scientists are embarking on research which could deliver the world's first stem cell treatment for blindness within five years.
British scientists are embarking on research which could deliver the world's first stem cell treatment for blindness within five years.
A team of leading researchers and clinicians is being assembled for the project, which will use embryonic stem cells to repair damage caused by age-related macular degeneration (AMD).
The leading cause of blindness among the elderly in the developed world, it affects around a quarter of people over the age of 60 in the UK to some degree.
An anonymous American philanthropist has defied his country's ban on embryonic stem cell research and put up £4m to launch The London Project to Cure AMD yesterday.
The scientists, led by teams from University College London, Moorfields Eye Hospital in London and the University of Sheffield, plan to transplant cells created in the laboratory straight into the eyes of patients.
They are confident of success, and predict that within 10 years the hour-long operation will be a routine day-surgery procedure.
AMD is associated with defects in retinal pigment epithelial (RPE) cells which support the light-sensitive photoreceptors of the retina. Without the underlying carpet of RPE cells, the rod and cone cells that respond to light cannot survive. In AMD, cells in the middle of the retina begin to die, causing loss of vision that spreads out from the centre of the visual field. The new project aims to generate RPE cells from embryonic stem cells in the laboratory and transplant them into the eyes of patients.
Stem cells are immature, dormant cells with the ability to turn into different cell types. Embryonic stem cells, among the most potent, are obtained from early stage embryos the size of a pinhead.
There are two types of AMD, "wet" and "dry". While much progress has been made in tackling wet AMD, which is characterised by leaking blood vessels, no treatment is available for dry AMD.
The embryonic stem cells (ESCs) will be grown at the University of Sheffield, where scientists already have eight experimental cell lines. A new facility at the university will develop cells that meet clinical standards.
The £4m donation will only pay for half of the entire project but is enough to get it started, said the scientists.
It is hoped that government funding and charities will provide the balance once the research is under way.
The US donor is said to have been frustrated by restrictions on stem cell research in his home country. President George W Bush has banned the state funding of new research involving embryonic stem cells, and patent rules stand in the way of private work.
The donor insisted as a condition for his support that the London Project work should not be commercialised. All the fruits of the research will be freely available to other scientists and doctors.
Enthusiasm in Scotland for the new possibilities for treatment was tempered by the fact that for many people already suffering from macular degeneration it will come too late.
John Legg, director of the Royal National Institute of Blind People, Scotland said: "This is particularly good news for the estimated 15,000 people in Scotland with dry AMD which currently has no treatment. However, even if all goes well with this project, the possibility of treatment being made available on the NHS is still five to seven years away."
For George Kay, 74, from Edinburgh, the news is welcome because his children and grandchildren will have hope of effective treatment if the disorder is passed on to them.
"My mother and her aunt both had it, so I am worried that it runs in my family, and I have three children and seven grandchildren. Our best hope is that future generations will have hope of treatment."
As a sufferer from wet AMD Mr Kay has benefited from laser surgery in 1989, but his vision has deteriorated, and he will undergo further laser surgery next month.
"My left eye is as bad as it was in 1989 so the treatment then has staved off decline for 18 years," he said.
Parents' appeal after life-saving transplant for girl of steel'
Three-year-old Eva Winston-Hart is injected with stem cells to fight a rare form of leukaemia as she lies surrounded by her favourite cuddly toys.
Her parents, Dave and Amy, of Market Harborough, Leicestershire, who were by her side during the operation, have appealed for better facilities to store cord blood, a rich source of stem cells, after a search for a donor in the UK failed. In March, a matching stem-cell donor was found in the US, following a scan of more than 11 million samples across the world.
Eva remained awake throughout the operation in the isolation ward at Birmingham Children's Hospital on May 23.
It could be weeks before they know if the transplant has been successful but there are already good signs. Yesterday, Mr Winston-Hart said: "On day 10, they took bloods and certain things are starting which the doctors were not expecting yet, so it's looking positive. It appears the stem cells are doing their job and producing new cells for her."
Eva was diagnosed with the condition seven months ago and her parents have been at her bedside around the clock. She has already overcome an E-coli infection and a bout of gastroenteritis.
Mr Winston-Hart said: "She's always been a fighter. Some days, I think she's made of steel."
Eva's procedure involved the injection of stem cells, found in a stored umbilical cord, into her bloodstream.
Mrs Winston-Hart travelled to Westminster in April to raise awareness about cord blood storage with members of the House of Lords. She said: "We went through many months of heartache. Our wait was made longer as the vast majority of cord blood in the UK is thrown in the bin.
"Every cord is potentially lifesaving and we feel the government really must take action to make sure there are proper facilities in place for saving cord blood."
