Research conducted at MFT plays key role in transforming the lives of people with cystic fibrosis
Patients at Manchester Adult Cystic Fibrosis Centre (MACFC), based in Wythenshawe hospital – part of Manchester University NHS Foundation Trust (MFT) – are the first in Europe to have received the life-changing drug, Kaftrio, as part of standard NHS care.
The drug, which was made available on the NHS following approval by European regulators last year, had previously been tested within clinical trials across the world, including at MFT. At our Trust, the research involved adult participants at the NIHR Manchester Clinical Research Facility (CRF) at Wythenshawe Hospital, as well as paediatric participants at Manchester CRF at Royal Manchester Children’s Hospital (RMCH).
Cystic fibrosis (CF) is a rare genetic disease that affects around 10,000 people in the UK and is caused by mutations in the CFTR gene.
Kaftrio is a breakthrough therapy, known as a CFTR modulator, because it targets the core defect rather than the effects of the disease, and it could be suitable for 90 per cent of patients with CF.
In the last few years MFT has successfully delivered many clinical trials of this class of medications, both at Wythenshawe Hospital and at RMCH. Researchers from MACFC have served as national and global leads on a number of pivotal studies, while RMCH was the first UK centre to recruit patients in the Kaftrio trial.
Dr Anirban Maitra
Dr Anirban Maitra, Consultant Respiratory Paediatrician; Director of Cystic Fibrosis and Lead for Bronchiectasis and Non-CF Bronchiectasis at RMCH, said: “Kaftrio, the modern new generation CFTR modulator, has already made a considerable impact on people with cystic fibrosis.
“I am immensely proud to be associated with the first UK-based research trials of Kaftrio that took place at MFT, in the world class research facilities at RMCH, under the guidance of a very able research team.
I am truly honoured that this work has benefitted our children with cystic fibrosis.
Dr Peter Barry
Dr Peter Barry, Consultant Respiratory Physician, MACFC and medical lead for the roll out of these medications at Wythenshawe Hospital, said: “MFT’s Manchester Adult Cystic Fibrosis Centre has taken a leading role in trials which have led to the development of novel medications focused on the underlying defect in cystic fibrosis.
“Our investigators conducted this pivotal research at Manchester CRF at Wythenshawe Hospital. As a result, we were keenly aware of the positive influence these medications could have for the majority of people with CF attending our centre. We were delighted that these therapies rapidly moved from clinical trials to licensing in 2020.
“We have rapidly adapted our service during the COVID-19 pandemic to continue to provide safe and accessible clinical care to all our patients including the rapid implementation into clinical care of these novel therapies.
It is truly a tribute to the determination, drive and application of the entire CF MDT that our patients were actually the first in Europe to receive these transformative medications.
The MACFC team
*Photo taken before social distancing
