'Funding shortfall' puts cystic fibrosis research on hold

 
Cystic fibrosis x-ray Mucus in the lungs can lead to infection

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UK scientists say pioneering research into cystic fibrosis has been delayed by funding problems caused by the economic downturn.

The researchers were hoping to begin a trial later this summer, using gene therapy to try to protect patients' lungs from potentially fatal damage.

They have been shortlisted for a prestigious award medical innovation.

The Cystic Fibrosis Trust says public donations for this type of work have fallen in the tough economic climate.

The Medical Futures Innovation Awards celebrate some of the best ideas for new treatments and help them attract funding.

But that is proving especially challenging at the moment.

The winners will be announced on Monday, but a team in the running, which is looking at pioneering gene therapy for cystic fibrosis (CF), says plans were in place to begin the world's biggest trial of its kind later this summer.

Funding Shortfall

But this has been put back while efforts continue to meet a shortfall of six million pounds.

The UK CF Gene Therapy Consortium has spent 10 years working on a novel way of treating the disease, which is caused by a faulty gene that controls the movement of salt and water through cells.

When this gene fails internal organs become clogged with sticky mucus which attracts infection. Most people with the disease die of chronic lung damage.

The scientists have developed a way of delivering droplets containing healthy genes into the lungs. They have completed initial safety trials, and say the early findings are "hugely encouraging".

Start Quote

You need a large critical mass of people doing the research, getting scientists and doctors working as a team, recruiting patients and making the material for the trial. It's been a decade of effort. No one else is capable of doing it. It is completely unique. ”

End Quote Professor Eric Alton UK CF Gene Therapy Consortium

However they say problems with funding mean the next trial to test clinical effectiveness will not get under way until March next year at the earliest.

Delay for patients

Professor Eric Alton, who is heading the research, says without further fresh backing the project could disintegrate.

"This project cannot be parked. If we do not get the funding patients will become more unwell. Some will die. A huge amount of hope and effort and science has been invested in this."

The consortium is talking to potential partners from the pharmaceutical industry and has applied for a grant from the National Institute for Health Research.

So far nearly all the £30m funding has come from the Cystic Fibrosis Trust. But the charity has seen a decline in annual public donations from about £10m in 2008, to about £8m now.

It is launching a fresh appeal targeting corporate and wealthy donors to try to meet the shortfall.

The charity's chief executive, Matthew Reed, says it would like to find a commercial partner.

"Cystic fibrosis undermines the quality and length of life This is a really significant moment in the history of the disease. We are very close to a clinical trial to test for efficacy. A lot of people are doing all they can to make it happen," he said.

 

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  • rate this
    +1

    Comment number 13.

    A good friend of mine died 10 years ago from complications caused by CF. Gene therapy was being talked about then and I find it disheartening that we are still no closer to a cure considering all the other wonderful technological advances we have. This may be simplistic but if only such research had the backing that say Apple have...

  • rate this
    0

    Comment number 12.

    Everyone has to do their bit for those fortunate. What about people who are very wealthy and most importantly healthy, to give quite a bit more to fund raising for the research into ALL of these gene trials which would benefit so many sufferers of all kinds of gene disorders?

  • rate this
    +1

    Comment number 7.

    I am a university scientist involved in clinical trials and would like to add that this problem is not limited to just cystic fibrosis, scientists in pretty much every disease area are facing the same problems

  • rate this
    -3

    Comment number 1.

    Perhaps there would be more money for research of this type if Universities did not charge an admin fee of 120% of scientist's salaries?
    Terms and conditions in Universities are not sustainable and there are limits on what research funders will pay.
    Doesn't take a Chair to see the decline of UK research since the implementation of full economic costings.

 
 

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