In cystic fibrosis the gene encoding a protein called the cystic fibrosis transmembrane conductance regulator (CFTR) is faulty. People with cystic fibrosis suffer from progressive lung infection. This lung damage reduces life expectancy. Agents for CFTR gene transfer may be an effective treatment as they deliver a correct copy of the CFTR gene to lung cells.
We found three trials with 155 participants to include in this review. The trials compare gene therapy to placebo nebulised to the lungs. The trials were of different designs and used different agents. This meant we could not combine them all in a meta-analysis. We found significant changes toward normal ion transport values in the lower airways of people who received gene transfer agents. However, we found no evidence that outcome measures which are clinically relevant to people with cystic fibrosis had improved. In one study "influenza-like" symptoms were significantly more common in people who received CFTR gene transfer agents. We conclude that at the moment there is no evidence to support the use of CFTR gene transfer agents as a treatment for lung disease in people with cystic fibrosis. We recommend that future studies are designed and reported clearly so that their results can be incorporated into a systematic review.