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Case study: UK at the forefront of cystic fibrosis research

Dr Alexander Horsley, leading expert in cystic fibrosis, NIHR Clinician Scientist and deputy director of the NIHR Manchester Clinical Research Facility at Wythenshawe Hospital, tells us why there has been a huge increase in clinical trials activity in CF in the last five years, what this means for patients and what the future looks like for patients with cystic fibrosis.

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The last few years have been an exciting time for CF patients

Cystic Fibrosis (CF) is a genetic condition affecting almost 11,000 people in the UK. You are born with CF and cannot catch it later in life, but one in 25 people carry the faulty gene that causes it, usually without knowing. The gene affected by CF controls the movement of salt and water in and out of cells. People with CF experience a build-up of thick sticky mucus in the lungs, digestive system and other organs, causing a wide range of challenging symptoms affecting the entire body.

Currently there isn’t a cure for people with CF but there are a growing range of drug therapies available to manage the condition. Dr Alexander Horsley, leading expert in CF, NIHR Clinician Scientist, and deputy director of the NIHR Manchester Clinical Research Facility at Wythenshawe Hospital, tells us why there has been a huge increase in clinical trials activity in CF in the last five years, what this means for patients and what the future looks like for patients with CF.

Why has there been a rise in the number of studies into CF and how has this influenced the NIHR Clinical Research Network Portfolio of CF studies?

“The NIHR Clinical Research Network (CRN) Respiratory Disorders Specialty has seen an increase of more than 100% in CF trials entered onto the NIHR CRN Portfolio of studies in the last 5 years. During 2017 there were on average two new CF studies added to the Portfolio each month, making a total of 37 active studies into this condition. Around 70 per cent of these are interventional studies meaning they are looking at new medications or treatment options.

“The increase in clinical trials activity into CF has been driven in part by the new and exciting developments in Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator therapies. These are drugs that work directly on the faulty protein in CF, partially restoring its activity.

“The result of this is that CF now makes up 15 per cent of the CRN-supported studies in respiratory medicine, despite there being a smaller proportion of patients with this condition when compared to the overall number of patients with respiratory disease in the UK.”

Who is sponsoring these new studies in CF?

“The year-on-year increase in CF studies has been driven primarily by commercial clinical trials (those designed and sponsored by pharmaceutical companies) and this reflects the development of several new therapeutic areas in CF. These include agents that act to reduce inflammation in the lung, new inhaled antibiotic and mucolytic therapies, and in particular the new CFTR modulator agents that have already been transformative for some patients.”

How are patients benefiting from the increase in trials in CF?

“The last few years have been an exciting time for CF patients, and CF has now become one of the most active areas of research in respiratory medicine. Real advances and clinically significant improvements in symptoms and lung function have been shown with combinations of agents that will be relevant for most CF patients.”

What are some of the challenges around delivering CF trials and studies and can you describe some of the solutions being developed?

“CF trials tend to differ from trials in other disease areas. The nature of CF, with often complex studies delivered to patients with specific genetics and clinical characteristics, mean that eligible patients with clinical stability and the time to take part are often few in number. “The Cystic Fibrosis Trust has recognised this challenge, as well as the importance of developing new therapies and opening up research opportunities to as many patients as possible. To support recruitment activity and streamlined trial delivery, the Trust has recently launched the Clinical Trials Accelerator Platform (CTAP), putting CF-specific research support into CF centres across the UK to enable rapid adoption and recruitment to studies. “Through CTAP, the Cystic Fibrosis Trust is encouraging people with CF to get involved in the shape and design of trials as well as in the lay review process, as it’s proven that involving the public and patients and their ‘lived experience’ promotes better recruitment and retention into trials.”

How can patients find out more about what studies are available in CF?

"The Cystic Fibrosis Trust’s online Trials Tracker lists all CF trials in the UK and assists potential participants in identifying trials they could participate in. CTAP clinical trials coordinators are now based in 20 adult and paediatric centres throughout the UK and are currently supporting 25 trials, from phase 1 to phase 3, with a number of additional trials currently under negotiation."

What does the future look like for patients with CF?

"The UK is now at the forefront of trialling new CF therapies and has become a lead destination for phase 1 CF studies in particular. These are early trials and typically the first time the medication is used in patients. Several agents and combination therapies have now progressed from phase 1 to phase 2 and 3 trials with the support of the NIHR CRN. I’m delighted to also report that in the last year, all the CF trials open in the UK recruited to time and target, a significant achievement for this complex disease area.

This is a particularly important marker for commercially funded research to show that the UK can compare well with the rest of the world in clinical trial delivery. “None of the huge advances in CF care over the last few decades would have been possible without the efforts of patients and research teams. The next few years are likely to see considerable and exciting progress in CF treatments. The CF Trust, the NIHR CRN and the individual CF centres themselves are committed to maintaining the UK’s position as a leading destination for CF clinical trials. This opens up new opportunities for participants to access new treatment and care options as well as for the clinical teams and contributes to improved care for all patients with CF.”