Round up of research from UK CF Conference

Treatments and quality of life for people with CF have improved significantly over the last few years, but there is still much more that can and will be done to allow people with CF to live a long and full life. At our UK CF Conference (UKCFC) last week, 18 researchers from across the world explained how their Trust-funded research was adding to our scientific understanding and greater knowledge of how to treat cystic fibrosis. In each of the three sessions of the conference, we heard from people talking about living with different aspects of CF in pre-prepared videos. A massive thank you to Cicely, Grace and Angelo for recording the videos, and to Jade for her wonderful twitter take-over keeping us up to date throughout the day.

Understanding CF – from gene to protein function

The day began with an overview of how to correct or compensate for the faulty CFTR protein from Professor Margarida Amaral, co-Investigator on our ‘Restoring airway function using alternative channels’ Strategic Research Centre (SRC). Professor Amaral began by talking about the healthy CFTR protein, from how it is made from the CFTR gene to how it works. Much of the focus was on research to develop new treatments that could benefit everyone – including those who can’t benefit from CFTR modulators – adding “these are the people that are our priority right now”.

Later in the session, we heard from Raquel Martins-Centeio from the University of Regensburg, Germany and Laetitia Pinte from the University of Cambridge, who emphasized the importance of looking at the function of proteins (other than CFTR) to understand how we might develop effective treatments for everyone with CF in the future and also understand more about differences in CF symptoms in different individuals with cystic fibrosis.

“The talks considered the different aspects of what might be contributing to and causing CF and how they can be taken advantage of for all, including diet and the microbiome, modifier genes, non-CFTR channels,” commented Dr Lucy Allen, our Director of Research after the session.

Improving treatments for CF complications

The research topics highlighted in Dr Joanna Whitehouse’s opening talk of the session on research into the symptoms and complications of CF were all identified by the CF community as health priorities for research.

We heard from Grace on her experience of living with the digestive complication Distal Obstructive Intestinal Syndrome (DIOS), which was later followed by a talk from Hannover University-based Professor Ursula Seidler on SRC-funded research testing the effectiveness of possible new DIOS treatments in the lab.

We also heard from Dr Bibi Uhre-Nielsen about our Trust-funded SRC investigating markers for CF-related diabetes (CFRD). As part of her research, she conducted a more-extensive-than-usual Oral Glucose Tolerance Test (OGTT) in study participants where she is based in Copenhagen. Blood samples taken during the OGTT study are being analysed by SRC partners all around the world. The aim of their research is to detect early signs that CFRD may be developing from chemicals and other signals in blood samples. You can read more about the aims of their research on our website.

Why treating CF lung infections is still a research priority

Professor Jane Davies opened the final session of the conference on lung infections stressing that she very strongly believed that in the era of CFTR modulators, researchers still need to focus on lung infections. She explained that the early evidence we have (from those taking the modulator therapy Ivacaftor) is that there are still large numbers of people with CF taking these therapies living with long-term infections.

A key obstacle to overcome in treating lung infections is tackling antimicrobial resistance (AMR). We heard from early career researchers working within SRC programmes and our UK CF Innovation Hub investigating new ways to treat infections of Aspergillus fumigatus, Pseudomonas aeruginosa and Mycobacterium abscessus respectively. This included a talk from PhD student Tom Williams at Imperial College London who is studying whether immunotherapies might be an effective treatment for A. fumigatus-related CF complication allergic bronchio-pulmonary aspergilliosis (ABPA).

In her concluding comments Katherine Cowan who was facilitating the conference on the Trust’s behalf said, “I’ve been very struck by the intense commitment of the CF research community, it’s obvious that there’s a determination to work together to work out what causes CF, find better ways to treat it and ultimately to make a difference to the lives of people living with CF and their families.”

The recordings of the sessions of the conference are available to watch again from our website.

To help the Trust fund vital research like this and support people with CF during the COVID-19 pandemic and beyond, please consider making a donation today.