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Cystic Fibrosis: Scientist Members of The Physiological Society Speak Out

Major advances in Cystic Fibrosis research mean that the future looks brighter for Gordon Brown’s family and all sufferers of this disease than it would have done 20 years ago

What does Cystic Fibrosis (CF) Mean For People?

Cystic Fibrosis is the UK’s most common life-threatening inherited disease. It is caused by a defective gene that clogs the internal organs, especially the lungs and digestive system, with thick sticky mucus resulting in chromic infections and inflammation in the lungs and difficulty digesting food. Over 7500 people in the UK are living with the disease, 5 babies are born with it each week. One person in 25 carries the faulty gene, and if 2 carriers have a baby, the child has a 1 in 4 chance of having CF.

How is the Future Getting Brighter?

Decades ago, the majority of children born with the disease were not expected to live beyond infancy. Now the average life expectancy is 36 and rising steadily. For the past two years, most parts of the UK now have more adults than children with CF.

Physiological Society Member Alastair Innes from the Western General Hospital in Edinburgh says “I run an adult CF clinic and we have several patients over the age of 50. About 70% of our adults with CF are in employment or education. We’ve achieved this through helping our patients with better nutrition, physiotherapy, the aggressive use of antibiotics, the development of multi-disciplinary team care, new high tech treatments and lung transplantation.

However the battle isn’t over yet. This progress carries a heavy burden of life long treatments and hospital supervision, with some patients, despite the best care, still dying in childhood. Most other patients still don’t live beyond middle age in a society where most people can expect to live into their 70s or even longer. Further research is vital and now offers the prospect of greatly improved treatments coming on line in the next few years”.

Research progress has been staggering since the gene responsible was identified in 1989, and Physiological Society members have made important contributions to this work. For instance the first ever measurements of the action of the CF gene product, called CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) were made in Newcastle by Mike Gray and Barry Argent using electrical measurements on human epithelial cells.

Physiologists helped show that the main role of CFTR is to control salt and fluid transport in epithelial cells and this process is reduced in CF tissue, where CFTR is faulty. This is probably the basic defect in CF.

Mike Gray of the Institute for Cell and Molecular Biosciences at the University of Newcastle says “Having this knowledge has helped enormously our understanding of the link between the gene defect and the clinical disease. This information would not have been possible without the work of many physiologists employing multidisciplinary approaches to understand the role of CFTR in normal cells, and then using this information to try and decipher what goes wrong in CF. However, we still are learning new things about CFTR that adds to the complexity of the disease, and basic research about CFTR is critical to the development of new therapies for CF”.

The UK is now at the forefront of efforts to develop gene therapy for CF through the UK CF Gene Therapy Consortium, a collaboration between scientists and clinicians based in London, Oxford and Edinburgh, funded by the Cystic Fibrosis Trust. Alastair, who is a member of this consortium says “Clinical Studies are already underway to prepare for a major multicentre trial of inhaled gene therapy in 2008, the first CF gene therapy trial in the world to go beyond safety testing to give multiple doses by aerosol inhalation with the aim of leading to clinical improvement. Physiological measurements are fundamental to this research in patients, enabling us to measure the electrical activity of airway lining cells and subtle changes in the ventilatory efficiency of the lungs in early disease and following treatment.”

Gene therapy isn’t the only research avenue for novel treatments that are being explored. UK physiologists, such as David Sheppard in Bristol, are also involved in developing non-gene therapy based approaches for the treatment of CF, such as drug therapy and non-CFTR ion channel therapy. The role of other genes that may contribute to, or modify, the outcome of the disease is also being explored.

Mike Gray says “Through a combination of all of these approaches it is likely that significant advances in the management of CF will be realised in the not too distant future. However, further sustained research about all aspects of CFTR is vital if we are to reach our goals of an improved quality of life and long-term survival in CF”

Mike Gray has also been working with The Physiological Society to progress CF research with international partners. He and Margarida Amaral (Lisbon) recently organised a meeting “New Frontiers in the Basic Science of CF” in Portugal in April 2006. The meeting was co-sponsored by The Physiological Society and the European Cystic Fibrosis Society.

Vicky Cowell of Patients Voice for Medical Advance says “I am really thankful that scientists have never given up hope in trying to tackle this disease, their research advances have paid off for my family. My daughter Laura has celebrated her 21st birthday this year, when she was born I was told that she probably wouldn’t live beyond her 6th birthday. But treatments have improved so fast that this gloomy prediction was proved wrong. What made the difference for Laura was being diagnosed early so that we could help her as soon as possible. I think that Gordon Brown is very lucky in that his son was born in Scotland where screening is the norm. Many parents in other parts of the UK are not so lucky, as a lot of Health Authorities do not routinely screen for CF early in a child’s life. Come on Gordon, make sure that early screening is available for every family in the UK”.

Mike Gray agrees “Early diagnosis is vital to help significantly reduce subsequent disease development. In Scotland they test all babies for CF, but this doesn’t happen in all of the UK”.

Elizabeth Bell | alfa
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