Cystic fibrosis

Gene study paves the way for other trials: Expert

The findings of a recent gene therapy trial in CF may have been modest but it represents years of work that will lay the path for other trials, an expert says.

The randomised double blind placebo controlled phase 2 study of pGM169/GL67A reported by the limbic last month (here) found a stabilisation of FEV1 in the treatment group rather than an improvement.

But in a comment in the Lancet Respiratory Medicine Scott Bell from the QIMR Berghofer Medical Research Institute in Brisbane said it was important to acknowledge the study for its “crucial preparatory work and its potential to provide important direction for future clinical trials”.

He encourages clinicians and researchers to delve into the online supplementary file of the study (here) because it provides “logistical triumph” in successful completion of the study, and into the potential challenges of moving such therapies into the clinic.

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