Cystic fibrosis clinical trials team
12 October 2017
Royal Brompton & Harefield NHS Foundation Trust has become the first organisation in Europe to recruit patients for an important new study which looks at the potential for a new drug to be used in the treatment of Cystic Fibrosis.
The drug under study, known simply as FDL169, has already been shown to improve the function of the protein which is defective in patients with CF.
CF is an inherited condition where the lungs and digestive system become clogged with thick, sticky mucus which can cause problems with breathing and digestion from a young age. Over many years, the lungs become increasingly damaged and may eventually stop working properly. The condition affects over 70,000 people worldwide with an estimated 1 in every 2,500 babies in the UK being born with the condition.
The aim of this study is to see how safe the drug is and how it interacts with the body, with one group of patients receiving differing doses of the experimental drug and another group of patients receiving a placebo (a dummy drug). The drug is planned as a long-term treatment in a specific subset of patients with CF who have the most common genetic mutation (F508del-CFTR mutation).
The study is currently being undertaken at 14 sites across the world including Australia, Germany, Czech Republic and the UK, and is sponsored by Flatley Discovery Laboratory, a not-for-profit laboratory for CF drug discovery, based in the United States.
Dr Nicholas Simmonds, who is leading the study for the Trust, said: “We’re delighted to have the first European patient randomised into this important study of a new CFTR ‘modulator’ – an exciting area of drug development in CF. The drug development pipeline in CF is diverse and expanding, but there is still a need to discover more effective treatments for this life-limiting condition. Although at the early stages of clinical development, drugs such as FDL169 have great potential to fill this space and ultimately improve the outcomes for thousands of individuals with CF across the UK and beyond”.
Dr Simmonds, an adult CF physician, was recently the recipient of a research award which recognises the outstanding research contributions of clinicians in the NHS. His research covers a range of studies, from trials of novel drugs to improvements in clinical care for CF.
Dr Simmonds is also the associate director of the adult CF centre at the Trust, which is one of the largest of its kind in Europe. The centre is a world leader in the development of new treatments and has become a model for other units in the UK and Europe.
Dr Jenny Rivers, associate director for research, said: “The range of innovative CF research at the Trust led by Dr Simmonds and his colleagues is truly inspirational. It’s fantastic to see our researchers’ desire to recruit patients into these studies in an attempt to improve disease outcomes. Research such as this will help shape our understanding of the condition and its management in the future.”
If you would like to find out more about this research or any of the research undertaken at the Trust please contact us.