Cystic fibrosis research 'delay'

Mucus in the lungs can lead to infection Continue reading the main storyRelated StoriesCystic fibrosis gene cure closerDrug 'aids lung mucus clearance' 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".

Continue reading the main story“Start QuoteYou 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 QuoteProfessor Eric AltonUK 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.