View attachment 446A quick (easy to read) blurb on the recent drug combination therapies involving Kalydeco: The Kalydeco drug that effectively restores the flow of water and chloride through the CFTR protein channel has been successfully used to treat people with CF who carry the "celtic gene" (G551D), which is found in about 4% of CFers worldwide and 10-15% in Ireland (hence called the "celtic gene").
Recent combination trials in 1100 patients worldwide have used a second drug in combination with Kalydeco to restore CFTR protein folding defects. The very important breakthrough is that the combination therapies have been shown to improve lung function of patients with the common F508del mutation, which has about 60-70% prevalence worldwide within the CF population.
This means that we are on our way to devising a therapy that could treat over 50% of the CF population. Although the results are not as convincing as the Kalydeco treatments, they do clearly demonstrate that combination therapies have the potential to treat a much larger portion of the CF population.
Prof Stuart Elborn said: “While we had previously found an effective treatment for those with the ‘celtic gene’ this new combination treatment has the potential to help roughly half of those with cystic fibrosis, those who have two copies the F508DEL mutation."
These studies and their positive results offers a lot of hope for all with CF. A drug that treats the underlying causes of CF will alleviate symptoms and allow for better and longer lung function, significantly increasing life expectancy of people living with CF.
Comment below if you liked this explanation! I am trying to bring the science over to the CF community in an easy to understand manner.
Read external article
- Chris
Recent combination trials in 1100 patients worldwide have used a second drug in combination with Kalydeco to restore CFTR protein folding defects. The very important breakthrough is that the combination therapies have been shown to improve lung function of patients with the common F508del mutation, which has about 60-70% prevalence worldwide within the CF population.
This means that we are on our way to devising a therapy that could treat over 50% of the CF population. Although the results are not as convincing as the Kalydeco treatments, they do clearly demonstrate that combination therapies have the potential to treat a much larger portion of the CF population.
Prof Stuart Elborn said: “While we had previously found an effective treatment for those with the ‘celtic gene’ this new combination treatment has the potential to help roughly half of those with cystic fibrosis, those who have two copies the F508DEL mutation."
These studies and their positive results offers a lot of hope for all with CF. A drug that treats the underlying causes of CF will alleviate symptoms and allow for better and longer lung function, significantly increasing life expectancy of people living with CF.
Comment below if you liked this explanation! I am trying to bring the science over to the CF community in an easy to understand manner.
Read external article
- Chris