Cystic fibrosis treatment saved by last-minute £4m bailout
A last-minute bailout has stopped the closure of a revolutionary British project developing a treatment for cystic fibrosis. Professor Eric Alton, the leader of the project, has thanked the Observer for the vital role it played in helping to save the consortium of 80 scientists working on the initiative.
"Rescue has come at the very last minute and has saved 10 years' of pioneering work that could help thousands of young people with cystic fibrosis," he said. "The Observer has played a magnificent role in describing our work and highlighting the terrible position we found ourselves in when funding ran out. We are very grateful indeed."
The treatment being developed by the scientists based in Edinburgh, Oxford and London, which involves putting genes into the lungs of patients suffering the fatal, inherited wasting disease, passed early clinical trials in 2010. But the recession badly affected the Cystic Fibrosis Trust, the charity that had funded the programe, and work had to stop last year just as the consortium was closing in on its goal. The plight of the scientists and their patients was highlighted in a series of articles published in the Observer, and last week the Medical Research Council and the National Institute for Health Research announced they had said that they would provide more than £4m to allow the project to continue. "This funding will demonstrate how science in the lab can be translated into gene therapy for patients," said the health secretary Andrew Lansley.
Cystic fibrosis affects 9,000 people in Britain and is caused by a mutant gene that prevents cells from producing healthy digestive juices, sweat and mucus. Bodily fluids become thick and sticky and clog up lungs and digestive tracts, which then become infected. Around 150 babies a year are born with the disease in Britain. James Fraser Brown, the four-year-old son of Gordon Brown, is one of them.
The development of antibiotics has helped to keep them alive, but even today few live beyond their late 30s. Patients survive only by going through long daily physiotherapy sessions, the consumption of dozens of vitamin and digestive enzyme tablets, and the constant use of antibiotics and asthma inhalers.
Scientists at the Cystic Fibrosis Gene Therapy Consortium have isolated the healthy version of the gene and coated it in a fatty chemical known as a liposome. Patients inhale droplets of these liposome-coated genes, which should be taken up by cells in their lungs to replace their faulty genes.
"Rescue has come at the very last minute and has saved 10 years' of pioneering work that could help thousands of young people with cystic fibrosis," he said. "The Observer has played a magnificent role in describing our work and highlighting the terrible position we found ourselves in when funding ran out. We are very grateful indeed."
The treatment being developed by the scientists based in Edinburgh, Oxford and London, which involves putting genes into the lungs of patients suffering the fatal, inherited wasting disease, passed early clinical trials in 2010. But the recession badly affected the Cystic Fibrosis Trust, the charity that had funded the programe, and work had to stop last year just as the consortium was closing in on its goal. The plight of the scientists and their patients was highlighted in a series of articles published in the Observer, and last week the Medical Research Council and the National Institute for Health Research announced they had said that they would provide more than £4m to allow the project to continue. "This funding will demonstrate how science in the lab can be translated into gene therapy for patients," said the health secretary Andrew Lansley.
Cystic fibrosis affects 9,000 people in Britain and is caused by a mutant gene that prevents cells from producing healthy digestive juices, sweat and mucus. Bodily fluids become thick and sticky and clog up lungs and digestive tracts, which then become infected. Around 150 babies a year are born with the disease in Britain. James Fraser Brown, the four-year-old son of Gordon Brown, is one of them.
The development of antibiotics has helped to keep them alive, but even today few live beyond their late 30s. Patients survive only by going through long daily physiotherapy sessions, the consumption of dozens of vitamin and digestive enzyme tablets, and the constant use of antibiotics and asthma inhalers.
Scientists at the Cystic Fibrosis Gene Therapy Consortium have isolated the healthy version of the gene and coated it in a fatty chemical known as a liposome. Patients inhale droplets of these liposome-coated genes, which should be taken up by cells in their lungs to replace their faulty genes.
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