CF BioResource project – understanding your cystic fibrosis

Everyone who has cystic fibrosis has one fault or another in both of their CF genes. But that’s where the similarity ends.

There are over 2,000 different types of fault (or mutation) in the CF gene. The current CFTR modulators can correct the most common of these faults, but we know there are around 1 in 10 people with CF in the UK who are unable to benefit from the current medicines.

Also, we know that CF affects everyone differently. Even two children with the same parents may have different symptoms, numbers of infections, complications or longevity despite similar treatment options. For example, some of those differences may be caused by natural, small differences in other (non-CF) genes. But we don’t know enough about which genes these are or how or when they exert their effects.

To develop treatments for the rare forms of CF and tailor personalised treatments for each individual living with CF, researchers need to use genetic information to help them approach the right volunteers for future research studies and clinical trials. Currently this genetic information is not easily available, adding time and expense to new research, which can slow down or even put doctors, scientists and pharmaceutical companies off from starting CF research studies and clinical trials.

The aim of a new resource is to help overcome this problem. The CF BioResource project launched in 2021 within the NIHR BioResource.

The NIHR BioResource will create a register of people with CF who have agreed to be contacted about research, based on their genetic make up. This would make it much quicker and easier for researchers and doctors to find the volunteers they need for their studies. People with CF who share their DNA sample are likely to be invited to volunteer for both research studies related to CF and about other disease areas (unrelated to CF).

It is an approach the NIHR BioResource is already using to help researchers better understand different diseases and conditions and develop new treatments for them.

People with CF can take part by sharing a blood (for adults) or saliva (for children) sample and information about themselves. Adults can join the project in person. Children and young people (aged 0-15) can join through the DNA, Children + Young People’s Health Resource (D-CYPHR), by signing up online or in-person.

After registering interest, people will be provided with a Participant Information Leaflet and Consent Form and decide whether they wish to join.

NIHR BioResource project explained in more detail

Martin, one of the Trust’s Trial Ambassadors who has CF spoke to the Principal Investigators of the CF BioResource project, Professor Alex Horsley and Professor Andres Floto to find out more about it. The conversation was facilitated by the Trust’s Involvement manager Lorna Allen.

What is the CF BioResource project all about?

The CF BioResource project will make more research possible, such as understanding why CF is varied within the same family. This information could be used to help manage people’s CF in the future, and new avenues for treatment. The Principal Investigators are excited about the difference the research could make for the CF community and attracting CF research studies into the UK.

How will it work?

Since 2021, more than 1,000 people with CF have joined the project. Their genetic information together with existing data from the UK CF Registry is what makes this project so exciting. Once someone has agreed to join the CF BioResource project they may be invited to take part in new research studies. The research may or may not be related to CF. It will be up to the individual on whether they choose to take part. We will benefit from the expertise and experience of the NIHR BioResource and D-CYPHR in facilitating these studies.

How can I get involved?

People can take part in the CF BioResource project through their CF centre. Once they have given consent to take part, a small blood sample will be taken and sent off to the NIHR BioResource. The sample can be taken at the same time as taking other blood samples, so it won’t require an extra needle. People might be asked to provide a further sample if there is a problem extracting DNA from the original sample they provide.

The NIHR BioResource is also offering an extra route to join for children and young people aged 0 to 15. Children and young people can join via D-CYPHR either in person or by consenting online and give a sample from home. Children and young people will be asked to provide a saliva sample instead of a blood sample.

Watch our video opposite to find out more about how adults can join the NIHR BioResource Rare Disease part of the project.

You can find lots of information and videos about how children and young people can join the D-CYPHR part of the study on the D-CYPHR website.

Why is Martin getting involved?

People will be approached about the CF BioResource project during their CF clinic visits to participating CF centres within our CTAP network.

You can find out which CF centres are participating by visiting our Trials Tracker - and we will keep this up to date as additional centres open. You can register your interest in taking part now by emailing us.