In this first trial of gene therapy for cystic fibrosis (n=136), patients showed improvements in tests of lung function compared to placebo without safety concerns. Patients aged ≥12 years with cystic fibrosis were randomized to either gene therapy (nebulized pGM169/GL67A) or placebo (saline) at monthly intervals for one year. Patients’ lung function was assessed by volume of air forcibly exhaled in one second (FEV1).
Of the 62 patients who were given gene therapy, FEV1 was 3.7% higher than the placebo group after one year of treatment. The effects were inconsistent, however, where some patients showed better response than others. Significant FEV1 changes of 6.4% were seen in half of patients with the worst lung function at study initiation.
Gene therapy was well tolerated in general and both study groups experienced similar rates of adverse events, study authors noted.
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