Cystic fibrosis

Another step along the path of translational gene therapy


A proof of concept trial has shown that non-viral gene therapy can stabilise FEV1 in cystic fibrosis patients.

The results provide “another step along the path” of translational cystic fibrosis gene therapy and should encourage the rapid introduction of more potent gene transfer vectors into early phase trials, wrote the authors in the paper published in  Lancet Respiratory Medicine.

The two-year study randomised 136 CF patients aged 12 years or older to either 5ml of nebulised pGM169/GL67A (gene therapy) or saline (placebo) at monthly intervals over one year.

In the 62 patients who received the gene therapy, FEV1 was 3.7% greater compared to placebo, one-year results showed.

Although the findings were encouraging, they saw a stabilisation of FEV1 in the pGM169/GL67A group rather than an improvement, said the study authors led by Professor Eric Alton from the National Heart and Lung Institute at Imperial College London.

“This stabilisation took place over a 1 year period and further work will be needed to see if this effect is maintained,” they wrote.

The mean difference was also modest, and only recorded in some individuals, they added.

“Further improvements in efficacy and consistency of response to the current formulation, or its combination with CFTR potentiators, are needed before gene therapy was suitable for clinical practice,” they concluded.

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