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Gene therapy trial marks a step forward for cystic fibrosis patients

Watch Al Jazeera's The Cure to learn more about this pioneering research

3 July 2015

A therapy that replaces the faulty gene responsible for cystic fibrosis (CF) in patients’ lungs has produced encouraging results in a trial at Royal Brompton Hospital.

During the clinical trial, 136 patients aged 12 and over received monthly doses of either the gene therapy or a placebo for one year. The results showed that patients who received the therapy had a modest, but significant, improvement in lung function compared with those receiving a placebo.

Patients in Scotland also took part in the trial and were treated at the Western General Hospital in Edinburgh.

CF is the most common incurable inherited disease in the UK, affecting around 10,000 people nationally and more than 90,000 worldwide. Patients’ lungs become filled with thick sticky mucus and they are vulnerable to frequent chest infections, which eventually destroy the lungs. 

The cause of CF – mutations in a gene located on chromosome 7 – was identified in 1989. This discovery opened the door to a gene therapy which introduces a normal copy of this gene. In the trial, patients inhaled molecules of DNA wrapped in fat globules (liposomes) that delivered the gene into the cells in the lung lining. 

The study was carried out by the UK Cystic Fibrosis Gene Therapy Consortium, a group of scientists and clinical teams from Royal Brompton & Harefield NHS Foundation Trust, Imperial College London, the Universities of Oxford and Edinburgh and NHS Lothian. The group came together in 2001 to develop a gene therapy and were supported by the Cystic Fibrosis Trust

The trial launched in 2012 and was funded by the National Institute for Health Research (NIHR) and the Medical Research Council (MRC). The findings are published today in The Lancet Respiratory Medicine.

It is the first to show that repeated doses of gene therapy can have a meaningful effect on CF and change the lung function of patients. However, further research is needed to make the therapy more effective before it is suitable for clinical use.

Professor Eric Alton, consultant physician at Royal Brompton Hospital and co-ordinator of the consortium from Imperial College London, said: 

“The results are encouraging and lay the groundwork for further trials that we hope can improve the effect. We are looking to carry out follow-up studies to assess higher and more frequent doses, combinations with other treatments and different methods of delivering the gene into the cells.

“Our aim is to achieve a step change in the treatment of CF that focuses on the basic defect rather than just addressing the symptoms. Eventually we hope gene therapy will push CF patients towards a normal life expectancy and improve their quality of life significantly.”

The CF centre at Royal Brompton Hospital is one of the largest in Europe, caring for around 700 adults and 350 children. In 1965 it established the UK’s first adult CF service in response to the increasing number of patients living with the condition into adulthood.


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