Cystic Fibrosis Gene Therapy Improves Lung Function in New Study

First Posted: Jul 06, 2015 08:14 AM EDT

A new gene therapy for cystic fibrosis could be huge in terms of improving lung function. Scientists have replaced the defective gene responsible for cystic fibrosis by using inhaled molecules of DNA to deliver a normal working copy of the gene to lung cells.

"Patients who received the gene therapy showed a significant, if modest, benefit in tests of lung function compared with the placebo group and there were no safety concerns," said Eric Alton, senior author of the new study, in a news release. "Whilst the effect was inconsistent, with some patients responding better than others, the results are encouraging."

Cystic fibrosis is a rare, inherited disease that's caused by mutations in a single gene called cystic fibrosis transmembrane conductance regulator (CFTR). It affects one in every 2,500 newborns in the UK and over 90,000 people worldwide. In this case, scientists have discovered around 2,000 CFTR mutations so far. These mutations cause the lining of the lungs to secrete unusually thick mucus, which can lead to recurrent life-threatening infections.

In this latest study, the researchers examined 136 CF patients at the ages of 12 and over from across the UK. Participants were randomly assigned to either 5ml of inhaled pGM169/GL67A or saline at monthly intervals over one year. Lung function was then evaluated using a common clinical measure of the volume of air forcibly exhaled in one second.

So what did they find? After a year of treatment, 62 patients who received the gene therapy had a better lung capacity than those who received the placebo. This was a result of stabilization of respiratory function rather than an improvement. However, the effects were inconsistent; some patients responded better than others.

"Publication of this trial is a landmark for cystic fibrosis patients and we are particularly grateful to the many patients across the UK who gave their time and effort to participate and make this collaborative venture a success," said Alastair Innes, one of the researcher, in a news release.

The findings are published in the journal The Lancet Respiratory Medicine.

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TagsCystic Fibrosis, Lung Cancer, Lung, Lungs, Gene Therapy