Researching for the future
Past achievements
Commitments
Correcting the cause
Improving symptom control
The lungs
Infections
Weight
Quality Control


Researching for the future
The aim of our research is to understand, treat and cure Cystic Fibrosis. With many research projects underway at any one time, the CF Trust is the major funder of medical and scientific CF research in the UK.

Past achievements
The Cystic Fibrosis Trust has been instrumental in many of the world's key CF research discoveries. Over the last decade or so, CF Trust funded research has led to the development of CF mice; an important tool for testing new therapies and for understanding how the CF protein works. Our researchers identified Burkholderia cepacia and the ways in which it is acquired. This led to the preventative procedures to stop transmission from one person to another. Leading UK CF scientists were the first to develop and successfully test gene therapy using liposomes as the delivery system.

Commitments
We spend around £4.8 million on research each year. Because research projects last a number of years, the amounts of money committed for the duration of our current grants is huge.

Of the total research spend, around £4 million is for gene therapy and around £800,000 is for research into other aspects of CF such as infection, inflammation, drug therapy and the pancreas.

Correcting the cause
In the past, improving symptom control was our best hope of controlling CF, but since the discovery of the gene causing CF in 1989, and its product the cystic fibrosis transmembrane conductance regulator (CFTR) - researchers have been able to design novel ways of correcting the cause of CF, either by mending the faulty gene or adding in a healthy copy (gene therapy), or by activating or repairing the CFTR protein (protein therapy).

The Gene Therapy Consortium is now working to overcome the barriers to enable gene therapy to become a reality for people with CF (see CF Today for more information about gene therapy). Another approach currently being explored is to correct the faulty transport systems in airway cells.

Cystic fibrosis transmembrane conductance regulator controls the passage of salt and water in and out of cells. In many genetic mutations, including the most common DF508, CFTR is made within the cell but because it does not fold into the correct shape, the cells quality control system removes most of it before it can get to the cell membrane where it can operate. A number of research groups are developing new drugs to move CFTR to the membrane, or to activate it at the membrane surface. Other drugs are being developed to replace the function of CFTR activating alternative ion channels.

Cystic Fibrosis Trust funded scientists in Bristol, Cambridge, Dundee and Wales have investigated a range of potential drugs to understand how CFTR is controlled and ways of improving its function.

Improving symptom control
Around a quarter of l the CF Trust's research funds are directed at improving current clinical care by controlling symptoms better. Preventing and controlling lung infections, reducing inflammation and improving nutrition will all help keep people fit and healthy for longer.

The lungs
In Cystic Fibrosis it is important to understand the causes of lung infections and how to prevent and eradicate them. Lung infections along with the associated inflammation cause permanent damage to CF lungs. Inflammation is caused by white blood calls called neutrophils. How these cells are attracted to the lung, and ways to prevent this from happening, thereby reducing lung tissue damage is the focus of research by Dr Janis Shute in Portsmouth. She has been concentrating on the role of interleukin 8 (IL-8), which attracts neutrophils into the lungs, and the anti-inflammatory properties of heparin.

Infections
Why are some strains of bacteria more dangerous than others? Why do some respond to antibiotic treatment and others do not and can new antibiotics be found to control the infections? These are just a few of the questions CF Trust funded scientists are tackling at the moment.

Professor John Govan in Edinburgh runs the CF Microbiology Laboratory where hospitals around the country can get sputum samples analysed and obtain expert advice. He is also researching Burkolderia cepacia. His team is attempting to find out why B. cepacia is such a problem in CF, and is investigating new drugs including plant extracts and bacterial viruses to see if they can be used as effective treatments in Cystic Fibrosis.

Through a grant from the Big Lottery Fund, we have also founded the UK CF Microbiology Consortium based in centres across the UK to investigate aspects of infection in Cystic Fibrosis.

Weight
Studying and evaluating the use of hormones, vitamins and food supplements are a few of the research projects that have been undertaken to improve weight gain and overall nutrition for those living with Cystic Fibrosis.

Quality Control
Before funds are committed to research projects, a rigorous procedure is followed to ensure research funded is of the highest standard. Experts from all around the world are asked to comment upon the research proposal and a committee of medical and scientific experts (the Research Advisory Committee) then takes these reports into consideration and judges the overall quality and importance to Cystic Fibrosis.

The Research Advisory Committee makes recommendations to the CF Trust's board of Trustees who have final approval of funding projects that are of important and/or immediate clinical benefit to those with Cystic Fibrosis

Science has a system for assessing the quality of research before it is published. This system is called peer review. All of the medical research funded by the Cystic Fibrosis Trust is peer reviewed.

Sense About Science has produced a guide to help people to query the status of science and research reported in the media. Get the guide by clicking the e-button. It explains more about how scientists present and judge research and how you can ask questions of the scientific information presented to you. It is useful for those who follow debates about science and medicine in the news.