This article is more than 2 years old
The big questions in CF research: highlights from the UK CF Clinical Trials Conference
What’s changed in 60 years?
With Cystic Fibrosis Trust marking its 60th anniversary this year, the conference was an opportunity to reflect on the past 60 years in clinical trials. Dr Tim Lee opened the day by taking us back to the early days of CF research in the UK to reflect on what’s changed and what's been achieved.
There was only one CF clinical trial in the first three years. We’ve come a long way since then, with treatments being produced at a remarkable pace over the last couple of decades.
One of the major developments in CF research has been the formation of clinical trial networks such as the Trust’s Clinical Trials Accelerator Platform (CTAP). This has made it far easier to run large trials across multiple CF centres, allowing more people to take part and speeding up the trial process.
How can CFTR modulators improve?
A big theme of the day was how we build on the initial successes of CFTR modulators and make these treatments better for everyone. Clinical trials have shown that CFTR modulators have impressive overall benefits; however, the amount of benefit can vary greatly between individuals. Some people also have to stop taking them, for example, due to side effects.
We need to better understand what’s causing these differences and why some people are less able to tolerate CFTR modulators. We also need more choices when it comes to CFTR modulators so people can access treatments they tolerate well and that aren’t overly burdensome in their daily lives.
Who else could benefit from CFTR modulators?
We also need a better understanding of the potential benefit of CFTR modulators for people with rare gene mutations. Some studies have found that people with certain rare mutations might see benefits. However, the evidence is limited, and more research is needed to understand which gene mutations could benefit from CFTR modulators.
Other researchers are looking at whether we could develop lab-based tests to predict who might benefit from different CFTR modulators. Professor Margarida Amaral spoke about her ongoing work in this area, which involves taking rectal biopsies to test how a person’s cells respond when treated with CFTR modulators. This could be used to predict whether a person would benefit from particular CFTR modulators.
“When we meet next year, let’s hope all of us have moved forward in this field and brought more answers to the CF community.”
Dr Tim Lee
What are the effects of CFTR modulators outside the lungs?
A top research priority for people with CF is understanding how CFTR modulators might affect other areas of the body, such as the gut, bone health, and mental well-being. CFTR modulators could have benefits outside the lung but could potentially exacerbate other problems, so we need to understand this better.
Understanding the effects of CFTR modulators outside of the lungs could help doctors and researchers answer questions about growing older with CF, which we know is another research priority for people with CF. The Trust is collaborating with people working within CF and other areas of medical research to try to answer these questions. You can read about some of this work in our Growing older with CF report.
What research is going on to improve gut symptoms?
Improving gut symptoms continues to be a priority for people with CF, even in the new era of CFTR modulators. GRAMPUS-CF is a Strategic Research Centre funded by the Trust that looks into different aspects of CF gut symptoms. As part of this, they are currently looking for participants for their GRAMPUS-CF study, which will explore what causes different gut symptoms.
What’s coming up in clinical trials this year?
We’re excited to see an extremely broad range of clinical trials coming down the pipeline, and we hope that they will start to answer some of these questions.
This includes clinical trials to continue developing CFTR modulators and other areas such as anti-infective and anti-inflammatory treatments. The first genetic therapy trials are already opening in the UK, as well as in other countries like the US and Australia.
CTAP continues to support upcoming trials by ensuring we have the infrastructure and resources to run them and working with trial sponsors to set up trials in the UK. You can find all trials currently recruiting in the UK on the Trials Tracker.
Cystic fibrosis (CF) is a genetic condition which causes sticky mucus to build up in the lungs and digestive system. It affects almost 11,000 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.