It's time to start bringing the Summer Knowledge Project back towards Cystic Fibrosis. We aren't completely done exploring other diseases within the spectrum, but I'd like to do a post on a CF-related issue.
Many people have heard about VX-770. It is the biggest chance right now, at least with those who have the G551D mutation and maybe others, to correct the problem that the mutated gene causes. I put a period there at the end because it was indeed a statement, but oh, what a statement it is!
The G551D mutation was chosen, according to the CFF website because the CFTR gene of the G551D resides on the cell surface, and can be best manipulated. With the Delta F508 CFTR residing inside the cell, we just don't know yet if VX-770 will work alone. It may require a second drug to correct this underlying issue, called VX-809, which is still in development as well.
And let me tell you personally why I'm so excited: My daughter is a double-delta. Guess what mutation is being tested in part of the 3rd Trial? You guessed it! Double-delta! Oh, you have no idea the hope I have!
Yet you've undoubtedly come to know through some of my posts that I don't put my hope in medicines, treatments, or the hope of a cure. My hope remains and will remain in Christ, who will always love Samantha more than I or any one in the CFF community can. His will be done, not mine. But if I have a say, I pray that this works. Even if it doesn't work for the double-delta community, at least 3-5% of the CF population, those with the G551D mutation, can receive that hope, and the research community can take the lessons learned and move forward with the VX-809 drug research.
And that is what making strides to CURE FOUND is all about!
11 months ago