Designing a programme of support to encourage better use of nebulisers
Dr Martin Wildman, University of Sheffield, UK
Our contribution: £105,000
Partner: National Institute for Health Research (NIHR), £2 million
Effective use of nebulised treatments can help reduce lung exacerbations in people with cystic fibrosis. However, it is known that people with CF don’t always stick to their treatment plans, and this can affect their overall lung health. Working with the CF community, researchers in Sheffield have designed a support programme to encourage people with CF to use their nebulisers as they were prescribed. They are currently putting their programme into practice and will be looking at the results at the end of the project in 2020.
More information is available on the CFHealth Hub website, and you can follow updates on their Twitter account.
Which is the most accurate formula to calculate lung function?
Professor Janet Stocks, Institute of Child Health, UCL, UK
Our contribution: £30,150
Partner: Cystic Fibrosis Foundation, USA, £48,800
Forced vital capacity in one second (FEV1) is a frequently used measure of lung function in cystic fibrosis. It is used to monitor the benefits of drugs in clinical trials, by people living with CF to monitor their own lung function and by registries to track improvements in care over time. As FEV1 varies with body size, age, gender and ethnicity, equations are used to create a standardised measure of FEV1.
Previously there were several different equations used to standardise FEV1, including different equations for children and adults. Recently a new equation has been adopted internationally to be used for both children and adults, known as the ‘GLI’. Professor Stock’s research is using UK Cystic Fibrosis Registry data to compare the values for FEV1 calculated by the new equations compared to the original equations used. This re-analysis may give us important information about how lung function changes over time, and help predict which patients may have a more rapid decline in lung function.
Charity Partner, Registry study
Early studies investigating the effects of RPL554 in cystic fibrosis
Verona Pharmaceuticals, London, UK
Our contribution: £100,000
Partner: Verona Pharmaceuticals, £200,000
Verona Pharmaceuticals’ drug known as ‘RPL554’ was originally developed for the treatment of chronic obstructive pulmonary disease (COPD). They are now developing RPL554 as a potential treatment for CF as well as for COPD.
This research grant was awarded to Verona Pharmaceutical to help fund a phase 2a safety study of RPL554 in people with cystic fibrosis. The results of this study were announced in a press release in March 2018, where the drug was found to be safe. There was some evidence of a beneficial effect which needs to be followed up in larger studies. Quoted in the press release, Dr Janet Allen, Director of Strategy and Innovation at the Trust said: “We’re pleased to see these interesting results come through, and that our investment in this area continues to show promise. We look forward to seeing the next steps of developing RPL554 for the treatment of cystic fibrosis.”
Dietary manipulation to improve glycaemic control
Laura Birch, NIHR Biomedical Research Unit in Nutrition, Diet and Lifestyle, Bristol, UK
Our contribution: £25,000
Partner: NIHR, £269,498
Up to half of people with cystic fibrosis will develop cystic fibrosis-related diabetes (CFRD). For those that do, their diet must control blood sugar levels and incorporate the increased energy requirements that are needed for all people with cystic fibrosis. Currently there is little evidence to guide practice on how best to achieve this.
This is a pilot study to understand more about how the diet of people with CF affects their blood sugar levels, and if changing the diet is a useful and acceptable way for people with CF to manage their diabetes. The findings of this study will inform the design of future, large scale studies.
To carry out this study dietician Laura Birch is in receipt of a Health Education England/NIHR Clinical Doctoral Research Fellowship, a funding scheme allowing allied health professionals (physiotherapists, dieticians etc) to study for a PhD at the same time as continuing their clinical practice. The Trust has provided supplementary funding to assist with the clinical recruitment of the study and with its dissemination.
Drug screening for easier ways to clear mucus from the lungs
Enterprise Therapeutics, Sussex, UK
Our contribution: £97,500
Partner: Enterprise Therapeutics, £97,500
One of the roles of the CFTR protein is to keep the mucus that lines the lungs watery (thin) and moving freely. It does this by acting as a gate for chloride to move out of the cells lining the lungs into the mucus. When the CFTR protein is faulty in cystic fibrosis, the movement of chloride is blocked, so the mucus becomes thick and sticky.
Enterprise Therapeutics are looking for drugs that can boost the activity of another chloride gate called TRMEM16 which compensates for the blocked CFTR protein and waters down the mucus. Their drug development is being conducted in specially grown circles of cells known as ‘bronchospheres’, which are made up of the cells that line the lungs. These spheres of cells are grown in tiny holes in specially-adapted dishes in the lab. Each dish can grow 384 bronchospheres and two of these dishes would fit on an A5 piece of paper. So over 750 ways of testing new drugs are being fit into the space of an A5 piece of paper.
Understanding the research priorities of people affected by cystic fibrosis (Question CF)
Professor Alan Smyth, University of Nottingham, UK
Our contribution: £15,000
Partner: University of Nottingham, £15,000
In a traditional research funding model, researchers and clinicians determine which areas of cystic fibrosis research are carried out, where the views and priorities of people affected by CF are not always taken into account. Working with the James Lind Alliance, Professor Smyth asked people affected by CF what their priority topics were for CF research - the project is known as Question CF.
The top 10 list of research areas has been published, and work is ongoing to refine the questions and fund research to address them.
Studying the effects of pollution on cystic fibrosis
Dr Fred Piel, School of Public Health, Imperial College, London
Our contribution: £85,000
Partner: Imperial College London, £85,000
Lots of external factors might influence the health of our lungs, and this is particularly true for people with cystic fibrosis. Narrowing down which factors actually do have an effect is difficult. We are co-funding two PhD students at Imperial College London, who will be looking at the effects of general air pollution, and traffic pollution in particular, for people with cystic fibrosis.
The students will be working in the Small Area Health Statistics Unit (SAHSU) at Imperial College, supervised by Dr Piel. The researchers have access to lots of very detailed, routinely collected information, for example air quality for houses that are linked together by the same postcode. They will be able to link/overlay this information with data from the UK CF Registry.
You can read a more detailed overview of this research in an article on our website.
Academic Partner, Registry study
Computer analysis of lung CT scans to measure lung health
Dr Joseph Jacob, UCL
Our contribution: £50,000
Partner: University College London, London £50,000
Performing CT (computerised tomography) scans can help doctors assess lung damage. Currently, scans are inspected manually and it’s difficult to spot small changes within the lungs or monitor changes that happen over time.
In this research project we are co-funding a PhD student at the Centre for Medical Image Computing at UCL. The student will be developing a method to allow computer analysis of abnormal airway dilation on CT scans. This computer analysis method will be able to conduct a more detailed analysis than clinicians can do by hand and will be much faster. If successful, this method could help researchers assess the benefits of drugs in future clinical trials.
Increasing our knowledge of CF-related joint pain
Dr Andrew Jones, University of Manchester
Our contribution: £34,000
Partner: University of Manchester, £34,000
Some people with CF have problems with their joints. One cause is inflammation inside the joint which can lead to pain, stiffness and swelling. While there can be other causes, this is often a condition known as CF-related arthritis (CFA).
Currently, we don’t have a good idea of what CFA is or how we should diagnose and treat it. These issues are studied by Dr Elizabeth Clarke, a rheumatologist working with Prof Andrew Jones within the joint pain SRC over the last two years. Dr Clarke gathered information from people with CF and joint pain by asking them about their experience, examining their joints, using ultrasound equipment to look inside the joints and conducting blood tests.
Her research has led to a greater understanding and awareness of CFA, however, it has also highlighted additional important questions. One year of further funding will enable Dr Clarke to get a better picture of how flares of joint disease affect people over time, as well as build better understanding of how CF can impact the results of some of the blood tests we use to investigate inflammation in joints. She will also look at how we can better assess other causes of joint pain that people with CF may experience so that we can treat those causes faster and more directly.