About Cystic Fibrosis Trust
The Cystic Fibrosis Trust is the UK’s only national charity dealing with all aspects of Cystic Fibrosis. It funds research to treat the symptoms of CF and to find an effective treatment. It also aims to ensure appropriate healthcare for those with Cystic Fibrosis and provides information, advice and support to people with CF and their families across the UK.
Cystic Fibrosis is one of the most common, life-threatening inherited diseases in the UK. It affects over 8,000 people and 1 in 25 of us carries the faulty gene that causes Cystic Fibrosis. If two carriers start a family, each child has a 1 in 4 chance of having Cystic Fibrosis.
Cystic Fibrosis affects the internal organs, especially the lungs and digestive system, by clogging them with thick, sticky mucus making it hard to breathe and to digest food. People with Cystic Fibrosis have to endure a rigorous daily treatment regime including physiotherapy to clear the chest, inhaled and injected drugs to thin mucus and fight infections and dozens of enzyme pills to help digest food.
Each week, five babies are born with CF and sadly, each week three young lives are lost. Around half of those with CF can expect to live over 35 years, but a baby born today can expect to live longer.
Each year we invest around £4 million in gene therapy research and around £800,000 in symptom control, infection, transplant and other research. We also spend around £1 million improving the care of those with CF and around £1 million on information, advice and support. None of this would be possible without the support of the CF community and the generosity of the public.
Our history
Cystic Fibrosis was first clearly described in the 1930s, although there had been reports of babies previously who almost certainly had Cystic Fibrosis.
In 1964, the Cystic Fibrosis Trust was founded by a concerned group of doctors, parents and friends to “get the research going” into Cystic Fibrosis. At the time, a child with CF was lucky to reach the age of five.
Due to advances in the understanding and treatment of CF, often funded by the CF Trust, the outlook for people with CF is much brighter although there is still much work to be done.
In 1989, the faulty gene that causes CF was discovered. Since then, a lot of time and money has been spent to repair or add a healthy copy of the faulty gene to the lungs of those with Cystic Fibrosis – gene therapy. There were no easy solutions and many scientists moved on to the next ‘big thing’.
In 2002, the CF Trust brought together the leading UK CF gene therapy teams from Edinburgh, London and Oxford. For the first time, talent, effort and results would be pooled to find a way of turning gene therapy research into clinical reality.
There are now 80 dedicated scientists and clinicians in the UK CF Gene Therapy Consortium. A gene therapy product has been developed, which in 2009 is undergoing a single dose pilot study in adults with Cystic Fibrosis.
If the pilot study is successful, a major multi-dose clinical trial in up to 100 people with CF will be carried out. This is the first time anywhere in the world that CF gene therapy has come this far.