A research team at Wythenshawe Hospital have recruited the first UK patient to a study evaluating a new treatment for cystic fibrosis (CF).
Cystic fibrosis is a genetic condition affecting more than 10, 00 people in the UK. It is caused by reduced quantity and/or function of the CF transmembrane conductance regulatory (CFTR) protein due to mutations in the CFTR gene. People with cystic fibrosis 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.
The study will compare a new treatment called VX-659 in combination with two other drugs in patients who are homozygous for the Phe508del mutation in the CFTR gene. This is the most common gene mutation in patients with cystic fibrosis worldwide and almost half of the patients in the UK have two copies of this gene mutation. These new treatments look to target the faulty protein made as a result of the gene mutation and aim to improve protein function which targets the root cause of cystic fibrosis. This trial, which is part of a series of trials testing these new medications, is hoping to improve on current treatment options for these patients.
The research is led locally by Dr Peter Barry, who said:
Recruiting the first UK patient to this global study is an excellent achievement for the cystic fibrosis research team at Wythenshawe Hospital.
The Manchester Adult Cystic Fibrosis Centre is one of the largest and longest established adult cystic fibrosis centres in the UK, and we are committed to conducting translational research that has an immediate positive impact for our patients.