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Funds dry up as scientists pinpoint effective treatment for cystic fibrosis A last-ditch effort to save a £36m UK project that is developing a revolutionary treatment for cystic fibrosis is to be launched. Researchers and campaigners say they need to raise £6m in the next six weeks. If they fail, the project, which has involved more than 80 scientists working in Edinburgh, Oxford and London, will be abandoned, dashing the hopes of thousands of young people with the incurable wasting illness. The treatment perfected by the consortium’s scientists involves putting genes into the lungs of patients and has passed early clinical trials. But the recession has badly damaged the income of the Cystic Fibrosis Trust , the charity that has funded the 10-year programme. As a result, the funds that are needed to complete the work have dried up – just as scientists have closed in on their goal of an effective treatment. “Unless we raise the last £6m by the end of October, we will have to lay off staff,” said Professor Eric Alton, the consortium’s co-ordinator. “In addition, the medicines we have developed to treat patients have a limited storage life and will have to be thrown away. We will have to disband – with our target in sight.” Cystic fibrosis affects around 9,000 people in the UK and is caused by a mutant gene that prevents cells from producing healthy digestive juices, sweat and mucus. Individuals who carry a single copy of this gene are unaffected but those who inherit two copies – one from their father, one from their mother – are affected. Their bodily fluids become thick and sticky, clogging 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. In the past, people with cystic fibrosis would die in childhood. 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, which has centres in Edinburgh, London and Oxford, decided to tackle the disease at a genetic level. Backed by funding from the trust, they isolated the healthy version of the cystic fibrosis gene and coated it in a special fatty chemical known as a liposome. Patients could then inhale droplets of these liposome-coated genes which would be taken up by cells in their lungs to replace faulty genes. It sounds straightforward. In fact, it took dozens of researchers working for more than a decade to pinpoint the best section of DNA to isolate and to create the best liposome coating. “I was involved in the first trials of this treatment,” said cystic fibrosis patient Alix Stredwick, 33, who lives in Hackney, London, with her partner, Richard. “That was a safety trial. It proved the treatment caused no harm. In addition, doctors found that when they studied what was happening in my lungs – at a cellular level – they could see the effect they were hoping for. The gene was being incorporated into cells in my respiratory system and appeared to be making healthy proteins. It was a fantastic feeling being involved in that.” Now scientists are ready to carry out a second phase of clinical trials which will compare the effects of the gene therapy treatment with a placebo. A group of 130 patients has been enrolled and the toxicology tests completed – just as the consortium’s money has run out. As a result, the trust is launching a special CF Sprint campaign to raise money from the public. At the same time, the consortium scientists have applied to pharmaceutical companies for aid as well as to a number of government grant bodies. “I cannot believe that all this work we have done, after the herculean efforts that have been made, will be allowed to collapse,” said Matthew Reed, chief executive of the Cystic Fibrosis Trust. “In the scheme of things, £6m is not a vast sum though it is a very tough goal for a charity to raise in such a short space of time. We have to succeed, however.” This point was endorsed by Toby Smith, a cystic fibrosis patient. “This is a chance to turn a disease that was once a death sentence into a manageable condition like diabetes. It is not just this generation of patients who will benefit but all future generations.” Medical research Genetics Charities Health Robin McKie guardian.co.uk