New Cystic Fibrosis project open and recruiting participants

The NIHR BioResource is expanding its Rare Disease programme with the Cystic Fibrosis (CF) project.

Cystic Fibrosis logo - small

The BioResource will provide a unique and ground-breaking opportunity to join up existing clinical data registries, providing information on the characteristics of CF, with genetic data. Ultimately, this will drive the development of more targeted CF therapies and large-scale clinical trials.

This new CF BioResource project is in complete collaboration with the Cystic Fibrosis Trust that also hosts the UK Cystic Fibrosis Registry

Speaking about the project, Dr Lucy Allen, director of research at Cystic Fibrosis Trust, said: “This will create a bank of people with CF who have agreed to be contacted about research, based on their genetic make-up. This will make research happen more quickly, speeding up advances in our understanding of CF.” 

What exactly is Cystic Fibrosis?

Cystic Fibrosis is one of the UK’s most common life-threatening genetic diseases that causes sticky mucus to build up in the lungs and digestive system. This causes lung infections and problems with digesting food. Around 10,600 people in the UK have CF; that's 1 in every 2,500 babies born.

Symptoms usually start in early childhood and vary from child to child, but the condition gets slowly worse over time, with the lungs and digestive system becoming increasingly damaged.

CF is caused by various genetic variants in the CF gene called Cystic Fibrosis Transmembrane conductance Regulator (CFTR). Currently, CFTR modulators can help reduce the symptoms caused by the condition and make it easier to live with, however about 10% of CF patients in the country are not responding to the CFTR modulator. More research could lead to the discovery of new CF drugs and help patients with their symptoms.

Image of mother with child suffering from Cystic Fibrosis

How many participants do we want to recruit and up until what period?

The aim is to recruit up to 1,500-2,000 CF patients to the NIHR BioResource in the first 2 years, and potentially up to as many as 6,000-8,000 participants over the course of the project.  

The first Cystic Fibrosis patients were recruited to the project in October, the same month the project opened; and within 3 weeks, a total of 6 patients had been recruited from two NHS Trust sites. Over the coming months, and into 2022, further clinical teams at NHS Trusts around the country will be prepared to recruit to the Cystic Fibrosis BioResource project.  

One of the first participants, 11 year old Billy said: “I took part in this research because I want other people like me to have better medicine as we go from generation to generation. Even though I have a fear of needles since I was little, I still decided to take part for other people’s wellbeing. I encourage other people with CF to take part. A couple seconds of pain from a needle could provide research that make CF people’s life better.” 

 The project is led clinically by Principal Investigators, Professor Andres Floto and Professor Alex Horsley, recognised experts in Cystic Fibrosis care and research.

Speaking about the project, Hannah Stark, Operations Lead for the NIHR BioResource said: “It has been brilliant to work with the Cystic Fibrosis Trust to get this important project up and running. We hope the BioResource can aid the research into the development of treatments for Cystic Fibrosis.”  

How to find out more 

Recruitment into the NIHR BioResource – Rare Diseases (RD) is via patient referral. The NIHR Bioresource is currently recruiting across more than 70 RD conditions; the full list of RD projects is available here.

Clinical teams wishing to express an interest can contact and/or their Clinical Trials Accelerator Platform (CTAP) centre coordinator. 

You can learn more about the Cystic Fibrosis BioResource project on the CF website.

For individuals interested in joining the NIHR BioResource – Rare Diseases, please discuss with your clinical care team at a future appointment.