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For those of you who aren't so keen on the molecular biology dont fear. The new drug the team are trialing is called Ivacaftor and its mechanism is to restore some functioning to the chanels which fail to work in cystic fibrosis.
This was a Randomised placebo V. drug trial with 83 and 84 subjects in eah group respectively conducted over a 48 week period.
Outcome measures included lung function, weight gain and sweat chloride levels among other markers of the disease process. The trial reports a significant improvement in these markers and a reduction in pulmonary exacerbations during the trial period as well as a lower proportion of side effects than in the placebo group.
Unfortunatley the drug has currently only been shown to be effective in patients with the G551D mutation, and the majority of the UK Cystic fibrosis population are deltaF508 homozygous but this new medication appears promising. Ongoing longer term trials are needed to assess longer term outcomes and potenial side effects but it would appear that this is the first of hopefully a new generation of treatements for CF.
Should more money be invested into molecular technologies such as chanel potentiators of should we be putting our efforts into genetic therapies aiming to repair or replace the faulty DNA? Where does the future of CF treatments lie?