Gene therapy treatment successful in stabilizing cystic fibrosis and could be a path for gene therapy to treat flu and arthritis

One year of gene therapy treatment for cystic fibrosis resulted in 3.7% improvement and stabilization of the disease compared to a placebo group. Cystic fibrosis affects about 70,000 people worldwide. Mutations in a single gene – CFTR – cause problems around the body, but particularly in the lungs. People with cystic fibrosis produce thick, sticky mucus, which clogs up the organ and makes it a breeding ground for harmful bacteria.

The Lancet Respiratory Medicine had the published study. Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial

Over the course of the year, the lung function of people in the treatment group seemed to stabilise, while the control group showed a steady decline in lung power.

The lungs of people with cystic fibrosis typically get worse at a rate of about 2 to 3 per cent a year, and Rayner says that a small change to this rate of decline could mean the difference between needing a lung transplant at the age of 60 instead of 40. “That is a big deal – it could mean a very different kind of life for them and those around them,” he says.

Now we’ve worked out how to get genes into the lungs, similar techniques could be used to tackle other genetic lung diseases, says Gill, or even to start using the lungs to produce therapeutic proteins for other health problems.

“One application would be to express antibodies in the lung against respiratory viruses such as flu, or we could secrete anti-inflammatory proteins from the lung into the blood to treat conditions such as rheumatoid arthritis,” Gill says.

She says her team has some preliminary data showing that techniques like this could work, potentially allowing us to take advantage of the lungs’ position at the centre of our circulatory system for their delivery. “We are just beginning to think about how to take these ideas forward,” says Gill.

Cystic fibrosis lungs on the left and healthy lungs on the right

SOURCES – New Scientist, The Lancet Respiratory Medicine

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