First patients in UK recruited to Cystic Fibrosis next generation therapy trial

Research undertaken by the Cystic Fibrosis team at Royal Manchester Children’s Hospital is underway with the first patients in the UK recruited to the Aurora F/MF Cystic Fibrosis research study.

The study aims to evaluate if oral medication with a new agent (VX-445) is effective and safe when taken in combination with two other medications; tezacaftor (TEZ) and ivacaftor (IVA), in patients with cystic fibrosis aged 12 years and over.

Cystic Fibrosis (CF) is the commonest inherited genetic condition affecting more than 10,400 people in the UK. A child will be born with CF when a faulty CFTR gene from both parents is genetically passed down. Most people with CF inherit two copies of the faulty F508del (F) gene from their parents, but there are more than 1,700 different ways the gene can be damaged. Depending on the type of mutation, the illness may be more or less severe.

In the UK, all newborn babies are screened with a simple heel prick test followed by specialist diagnostic tests so that CF can be identified and treated early. CF causes thick and sticky mucus to build up in the lungs and digestive system that cause lung infections and problems with digesting fat. Current treatment includes a range of medications to help children breathe better, prevent infections and get enough nourishment.

The aim is to help researchers understand if this triple combination therapy improves chloride transport (to make mucus less thick) specifically in children with one copy of the F508del (F) mutation genotype and one copy of a minimal function (MF) mutation genotype. 

The Aurora study is being led locally at Manchester University NHS Foundation Trust by Dr Anirban Maitra, CF Director, and Consultant Respiratory Paediatrician at Royal Manchester Children’s Hospital.

Dr Maitra said

We are proud and privileged as a team to have been at the forefront of cutting edge research in cystic fibrosis. We would like to thank the patients and their families who have volunteered to take part in this exciting study. We will continue to work hard to bring more research to the Children’s Hospital and open up new opportunities for new therapies and treatments to more people with CF.

The study is led nationally by Dr Ed Nash from Heart of England NHS Foundation Trust in Birmingham, and has been supported by the National Institute for Health Research (NIHR) Clinical Research Network: West Midlands. The project is sponsored and funded by Vertex Pharmaceuticals Inc.