Five things about research to give us hope for the future

1. Scientists haven’t taken their feet off the CF research pedal!

Like you, CF researchers around the world know that getting approval for and access to Kaftrio isn’t the end of CF research. They’re still working extremely hard to develop better medicines to treat everyone with CF, regardless of whether or not they are able to benefit from Kaftrio. Areas of research range from new types of therapy, such as genetic therapies to treat all forms of CF; novel ways to diagnose CF lung infections; and collaborative, multidisciplinary working to truly understand CF diabetes to improve how the condition is managed.

2. Exciting partnerships are underway to tackle CF lung infections

Research to prevent people with CF developing lung infections, work to improve how they are diagnosed, and applying new and innovative ways to develop new medicines that beat antimicrobial resistance (AMR) is underway, thanks to incredible partnerships with the University of Cambridge, Medicines Discovery Catapult and LifeArc.

The Innovation Hub at the University of Cambridge is a world-class partnership between the Trust and the University. Starting in 2018 the programme has identified important new data on why some strains of the bacteria Mycobacterium abscessus cause such aggressive infections in people with CF. They have also applied a wide range of cutting-edge techniques to develop new potential medicines to treat resistant infections – techniques that could benefit people with CF and others with severe and hard-to-treat infections.

Continuing the momentum of the Innovation Hub at the University of Cambridge, last month, we were delighted to open a £15m funding call to create a Translational Innovation Hub network in a co-funding partnership with the medical charity LifeArc. With the research hubs starting next Summer, this will build on advances made at the Innovation Hub at the University of Cambridge.

Last year, the CF AMR Syndicate led the development of patient focussed checklists, or Target Produce Profiles (TPPs), for new medicines to treat CF lung infections. This is just one piece of a much larger ‘toolkit’ of resources the Syndicate, a partnership between the Trust, Medicines Discovery Catapult and LifeArc, have developed to speed up the process of getting new medicines approved for the treatment of CF lung infections.

3. Our Clinical Trials Accelerator Programme is going from strength to strength

Across the UK, the Trust has established a clinical trials network of 27 adult and paediatric CF centres. The Trials Accelerator Programme provides invaluable support to sponsors with the delivery of CF clinical trials and a platform which enables the CF community to gain timely access to these trials. The network comprises a mix of research-focused clinicians and coordinators with a range of experience, knowledge and skills in CF care and clinical trial delivery.

Right now, as you read this, there are opportunities to take part in CF clinical trials and research studies. Why not head on over to our Trials Tracker to see if any are of interest to you? If the studies available are not for you, why not mention to your CF team next time you talk to them that you are interested in finding out more about research opportunities?

4. You. Us. Research - Our priorities are your priorities

It is important to us that the research we fund and help make happen, is research that could make a difference to you. We were delighted that so many of you had your say in the QuestionCF research priorities project that was completed last year. You identified a list of your top 10 CF research priorities. Since then, we’ve been hard at work behind the scenes sharing the priorities with researchers and sparking new ideas about research studies that could be conducted to address them.

Just last week, these new research priorities were a topic of discussion in front of over 2,000 CF researchers, clinicians and healthcare professionals at the European CF Society conference!

Identifying the research priorities is one example of people from the CF community helping to shape future CF research. Others include helping to approve access to UK CF Registry data for research and shaping how CF clinical trials should be conducted. If you’d like to know more about how you can get involved in research, please contact colleagues in our Involvement team.

5. The value of the UK CF Registry

At such a difficult time in a family’s life, many parents of newly diagnosed babies with CF agree for data about their children to be included in the UK CF Registry, a request that we confirm with people with CF when they are older. We can’t overstate how important this data is.

It helps provide some baseline information for doctors to share with families in CF clinics – something you may have experienced for yourself.

It’s also really important at all stages of research – for example, CF scientists use information and statistics about symptoms to help them work out how research studies should be designed and use Registry data to monitor the effects of new medicines after they’ve been approved for use. Increasingly researchers are also using UK CF Registry data to conduct clinical trials, for example, the CF STORM trial. The aim of this trial is to find out if people receiving Kaftrio can safely start to reduce the number of treatments they have to manage as part of their daily healthcare routine.

“With data from the UK CF Registry, we can design clinical trials to fit into routine CF care, minimising the extra time it can take to participate in a clinical trial, but still getting answers about the effects of treatment or the safety of reducing medications,” said Dr Gwyneth Davies, co-chief investigator of the CF STORM study.

Look out for lots of updates throughout the week leading up to Wear Yellow Day on Friday. Unite with us by wearing yellow and raising money to help create a brighter future for people living with cystic fibrosis.

Cystic fibrosis (CF) is a genetic condition which causes sticky mucus to build up in the lungs and digestive system. It affects more than 10,900 people in the UK. One in 25 of us carries the faulty gene that causes it, usually without knowing.

Since 1964, we've supported people with cystic fibrosis to live longer, healthier lives -and we won’t stop until everyone can live without limits imposed by CF.

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