The results of the CHOICES clinical trial show promise for people with rare CF variants in two exciting ways. The study showed that a new triple combination CFTR modulator therapy, developed by FAIR Therapeutics, was found to be safe and beneficial in many people with CF with rare CF variants. Participants were selected for the trial by testing their responsiveness to the trial medicines in lab tests following a biopsy.
CF diabetes is a difficult illness to manage alongside other CF symptoms, increasing the burden of treatment and risk of long term complications. In this Development Award, Dr Ildem Akerman and Prof Victoria Salem are exploring a new way to treat it.
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The aim of this Strategic Research Centre (SRC) is to design new and innovative medicines to act on the CFTR protein that will be bigger difference for more people with cystic fibrosis (CF).
Professor Nick Simmonds at the Royal Brompton Hospital is leading a research project on difficult to diagnose cystic fibrosis (CF). The title of his programme of research is Advanced Diagnostic Validation and Novel Clinical Evaluation across the CFTR spectrum, or ADVANCE-CFTR for short.
Led by Dr Mike Gray at Newcastle University, researchers from across Europe are designing chemicals in the lab that could be used as CF medicines in the future, for those with a specific type of ‘Class 1’ mutation, known as a premature termination codon or PTCs.
This Trust-funded Strategic Research Centre (SRC) programme will be run by Dr Paola Vergani at University College London, and aims to better understand the flow of bicarbonate in different organs affected by CF. Researchers involved hope their studies could inform future treatment choices and help us understand more about CF disease outside of the lungs.
The most effective way of treating cystic fibrosis (CF) is to treat the underlying cause of the condition and we want to do this for everyone. This includes developing future CFTR modulators with improved benefits and reduced side effects, and finding treatments for those who are unable to benefit from them.
