Robin McKie, science editor 

Charities step in to help cystic fibrosis sufferers

More than £20m is being raised at jumble sales and shops by charity workers to fund trials of a revolutionary cystic fibrosis treatment developed by British scientists. By contrast, the government has pledged only £2m to back the project.
  
  


More than £20m is being raised at jumble sales and shops by charity workers to fund trials of a revolutionary cystic fibrosis treatment developed by British scientists. By contrast, the government has pledged only £2m to back the project.

Cystic fibrosis is the most common severe inherited disease in the West. In Britain, more than 8,000 people - half of them under 16 - are affected. Last week, it was revealed that Fraser, the four-month-old son of Chancellor Gordon Brown and his wife, Sarah, had been diagnosed with the disease, which causes lung, pancreatic and liver damage.

'In 10 years, our gene therapy project could be alleviating the disease in young people like Mr Brown's son,' said Professor Eric Alton, of the Royal Brompton Hospital, London. This view was backed by Rosie Barnes of the Cystic Fibrosis Trust. 'This is the best hope we have of tackling this disease,' she said.

Scientists at the UK Cystic Fibrosis Therapy Consortium have found a treatment. 'We have taken the cystic gene and coated it in liposomes,' said Alton. 'Basically, we have put the gene in a drop of Fairy Liquid. We administer these droplets as a spray. In the lungs, when a globule comes in contact with cells on the surface of the lungs, the fatty acid fuses with its membrane. The gene slips into the cell.' The cystic fibrosis gene is then taken up by the cell's own DNA, rectifying the patient's abnormality. The technique has worked well on tests on mice. The team is preparing to launch clinical trials on 100 patients and results are expected by 2010. 'The market for cystic fibrosis treatments is too small to interest big companies,' added Kingsman. 'Our best chance is for a mid-sized company to become involved.'

Such an outcome would be a major victory for families affected by cystic fibrosis who are the Cystic Fibrosis Trust's key fund raisers. Initially, scientists had no common strategy, so researchers like Alton got together and agreed to set up the consortium to develop a single approach, with the trust providing funds.

'The government offered little help,' added Barnes. 'So we asked our members what they thought. They said, "Let's go ahead on our own." It has meant stopping grants for our other projects and raising £20m ourselves. So far we have raised £15m. That is a lot of jumble sales.'

 

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