The CF community received more great news today. Vertex has been working on several drugs that target the defective proteins that cause cystic fibrosis. An oral medicine called VX-770, geared towards the G551D mutation (which is one of Ellis' mutations!), has been in the trial phase, and Vertex released some of the amazing results. The VX-770 opens the gateway of the defective protein to allow the chloride to pass through the cell and prevent the build-up of mucus in the lungs, among other things.
In the study, 52 children (ages 6-11), with at least one copy of the G551D CFTR mutation, received VX-770 or the placebo as a single 150 mg tablet every 12 hours.
Preliminary Results
Lung Function: Results of the study showed that the difference in mean absolute improvement from baseline in lung function through 24 weeks in children treated with VX-770 was 12.5 percent. The difference in mean relative improvement from baseline was 17.4 percent compared to placebo.
Weight: Many people with CF have a hard time gaining and maintaining weight due to factors such as reduced pulmonary function, nutrition, chronic infection and inflammation. Those who received VX-770 experienced an average weight gain of approximately 8.1 lbs through 24 weeks compared to baseline while those in the placebo group gained approximately 3.9 lbs compared to baseline.
Sweat Chloride: People with CF typically have sweat chloride levels in excess of 60 mmol/L, while normal values are less than 40 mmol/L. Reduction in sweat chloride is considered to be a marker of improved CFTR function. In the study, the baseline sweat chloride level for both treatment groups was approximately 104 mmol/L (Ellis' sweat chloride levels are at 100). Through week 24, the mean sweat chloride level for individuals treated with VX-770 was below 60 mmol/L. Significant decreases in sweat chloride were not observed among those in the placebo group.
We expect to see more results in mid-July. I believe this drug will made available when Ellis is turning two years old. Keep up the good work Vertex!!!
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ReplyDeleteThis was a very well done study, BTW!! its was a double blind study meaning that neither the researchers nor the study participants knew if they were being administered the drug or placebo. Not to mention it was a randomized control study, little room for bias, BRAVO researchers, BRAVO!!!
ReplyDeleteThis makes there results even MORE promising :)