Drug approved for CF

Yesterday was a very exciting day for the CF community! A drug by the name of Kalydeco was approved by the FDA for treatment of a small population of patients with CF. This is the first drug to target the underlying cause of the illness and is a great advancement in finding a cure. It is targeted for people who have the G551D mutation of Cystic Fibrosis. This is not the type that Kelsie has but a drug to target her type (delta F508) is the in the next one to come down the pipeline! So we are very hopeful that we will see that come to fruition before her lungs sustain too much damage.

Here is the information about the drug VX-809 that is being studied to treat Kelsie's type:

VX-809 is the second investigational oral drug candidate for the treatment of cystic fibrosis (CF) to be advanced into development as part of a successful collaboration with the Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT).

Cystic fibrosis is caused by defective or missing CFTR proteins, which result in poor ion flow across cell membranes, including in the lung, and the accumulation of abnormally thick, sticky mucus that leads to chronic lung infections and progressive lung damage. In people with the F508del mutation, CFTR proteins do not reach the cell surface in normal amounts. VX-809, known as a CFTR corrector, aims to increase CFTR function by increasing the trafficking, or movement, of CFTR to the cell surface. In people with the G551D mutation, CFTR proteins do not function normally at the cell surface. VX-770, known as a CFTR potentiator, aims to increase the function of defective CFTR proteins by increasing the gating activity, or ability to transport ions across the cell membrane, of CFTR at the cell surface. 

And here is the information about the drug that was just approved!

http://www.cff.org/aboutCFFoundation/NewsEvents/2012NewsArchive/1-31-FDA-Approves-Kalydeco.cfm