Unstoppable in our search for answers: 5 highlights from our Research Impact Report

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Funding and supporting research that will benefit everybody with cystic fibrosis (CF) is at the heart of everything we do at the Trust. We won’t stop until everyone can live without the limits imposed by cystic fibrosis. To celebrate the launch of our 2022 Research Impact Report, we share some of our research successes over the past year – all made possible because of your incredible support.

1. New insights into how M. abscessus infections are passed on

Non-tuberculosis mycobacteria (NTM), including M. abscessus, can cause an aggressive lung infection in people with CF that is extremely difficult to treat. Gaining a better understanding of this infection and the development of exciting new treatments for it are some of the aims of the UK CF Innovation Hub, a research partnership between the Trust and the University of Cambridge. Professor Floto, who heads up the Hub, and colleagues, recently made an exciting research breakthrough. They found evidence that the infection can be passed to people with CF by people who don’t have CF. This is the first report making this link. Following further research studies to understand more about it, this discovery could help reduce the number of people with CF infected with M. abscessus.

When I first got diagnosed with NTM, I was told that there was no ‘go-to’ treatment to tackle this bug. It was all a game of ‘trial and error’ and you may be lucky if the treatment worked and it got rid of the infection. But in most cases you were stuck with it. Research in this area is so important as it could lead to new medicines that are better at getting rid of this infection for good, and giving those who have it a better quality of life.

Sara, who has CF

Did you know? Since 2013, 287 research papers have been published directly as a result of our funding. These reports of new scientific knowledge can stimulate more research, lead to changes in policy or the development of new CF treatments.

2. A research ‘toolkit’ to accelerate the development of better treatments for CF infections

To live long and fulfilled lives everyone with CF needs access to effective medicines to treat CF lung infections, both for those able and unable to benefit from CFTR modulators like Kaftrio. New medicines to treat these infections are urgently needed due to antimicrobial resistance (AMR). This is where medicines such as antibiotics stop working as infection-causing bugs adapt themselves to avoid the effects of the treatment.

New medicines for CF infections need to be developed in a different way to medicines for other infections because of the environment within the lungs of people with CF. The CF AMR Syndicate, a partnership between Cystic Fibrosis Trust, Medicines Discovery Catapult (MDC) and LifeArc, have developed a ‘toolkit’ of resources to overcome some of these difficulties and accelerate the development of new medicines.

The idea is to make treating new CF infections, an exciting, attractive and easier area of research to work in – whether you are working in the ‘discovery’ end of the new medicines pipeline, or whether you are a biotech company seeking advice on the best ways to test a new medicine that would meet regulatory approval.

Dr Paula Sommer, Head of Research at Cystic Fibrosis Trust.
Dr Zoe Saynor

3. Involving people with CF every step of the way

The voices of those affected by CF are at the heart of everything we do. From defining our research priorities, to making vital funding decisions - your lived experience matters every step of the way. The Trust’s involvement group of over 100 volunteers contribute to a wide range of projects. They play a vital role in our Clinical Trials Accelerator Platform, they share their lived experience and views in focus groups, review draft documents written by researchers, and support the CF research community to develop new research studies. Recently this meant members of our involvement group helping to shape an exciting study on airway clearance and exercise, known as ExACT-CF.

“We believe that the support of the CF community for this project not only helped us design a project for people with CF, but was also essential in helping us get funding to do the study," said Dr Zoe Saynor, CF and exercise researcher at the University of Portsmouth.

Did you know? Since the start of the involvement programme, people with CF and their families have participated in around 30 different opportunities to get involved in research each year.

4. Developing the research talent of tomorrow

Since 2013 we’ve funded 113 early career researchers, ensuring the brightest and best scientists have the opportunity to work in CF research. Through each SRC award, we provide funding to train and support up to five early career researchers to work in the programme. Lucia Nicosia is studying for her PhD in Dr Patrick Harrison’s lab at University College Cork in Ireland. They’re part of the Trust’s ‘Therapeutic gene editing’ SRC led by Professor Stephen Hart.

“I’m going to visit two different labs within the SRC within a month. I’m going to France to test if my experiments work in cells that are a closer match for the lung cells in people with CF. A few weeks later I’ll visit a lab in London, where I can test if my genetic editing makes a working copy of the CF protein. Our research is giving people with CF hope, and I think that’s just incredible,” explained Lucia.

Dr Harrison added: “Working in an SRC network gives early career researchers such as Lucia lots of opportunities for exchange visits. She can bring back expertise in new techniques to our lab and build strong relationships between labs. It all helps move our CF research along faster.”

Did you know? The Trust has supported 22 Strategic Research Centres (SRCs) with 142 scientists and specialists from over 50 cities and 15 countries around the world.

5. Supporting CF clinical research trials across the UK

From developing CFTR modulators and antibiotics to new physio techniques, clinical trials play a vital role in helping ensure people with cystic fibrosis can access the newest treatments being developed. In 2017, we launched the Clinical Trials Accelerator Platform (CTAP) including the CF Trials Tracker database, with the aim to create more opportunities for the CF community to take part in clinical trials. Through the CTAP programme, we have brought together a network of 27 NHS CF centres who we have funded with specialist trial coordinators to oversee the running of CF clinical trials across the country. Collectively, our network of CTAP centres have supported over 40 CF research studies and helped over 1,000 babies, children and adults take part in a clinical trial since CTAP launched. Around 6 out of every 10 trials were to test medicines that restored the CF protein. Other studies investigated treatments for different aspects of CF including infections, and mucociliary clearance.

To me, taking part in clinical trials means playing my part in the "research relay race" against CF. The baton was passed to me by previous generations who trialled medicines and therapies I have benefitted from since I was a small child. I hope we can keep inspiring future generations to carry on picking up that baton, until the race is won.

Rob, who has CF

We won’t stop

It's thanks to our incredible supporters that we can continue to be at the cutting edge of CF research. Making breakthroughs and discoveries that change lives for the better. Now and in the future. But we know this is just the start. Incredible progress has been made, but there is still a long way to go until everyone with CF can truly live a life unlimited. We won’t stop until we can all lead the lives we want. Until cystic fibrosis stops challenging, damaging or shortening lives. And you can be part of it.

 This Christmas we’re joining together to make change happen. There are lots of ways you can support us this festive season and help ensure a life unlimited for everyone with CF. Find out more.

Read more about how our research is making a difference in our Research Impact Report.

Read our latest Research Impact Report

Cystic fibrosis (CF) is a genetic condition which causes sticky mucus to build up in the lungs and digestive system. It affects more than 10,800 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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