I found out on November the 20th that I was approved through my insurance to get the new CF drug, Trikafta. I’m just waiting to hear when I will receive it from the pharmacy.
Let me try to explain how CF works and what this new drug does. CF is caused by mutations in the CF gene. These mutations lead to defects in a specific protein called the cystic fibrosis transmembrane conductance regulator (CFTR) protein. As a result of these defects, the CFTR proteins don’t work the way they should. The most common mutation is the F508del mutation, which I have 2 copies of. Often called double delta 508. The F508del mutation causes 2 defects. Defect 1: fewer CFTR proteins get to the cell surface, where they are normally located. Defect 2: CFTR proteins don’t open correctly if they do reach the cell surface. Because of these defects, chloride ions cannot move into or out of the cells like they should. This causes thick, sticky mucus to build up in organs, such as the lungs.
Trikafta has 3 components that work together to target the underlying cause. Trikafta adds elexacaftor to tezacaftor and ivacaftor to target CFTR protein defects caused by the F508del mutation. By binding to different places on F508del-CFTR proteins, elexacaftor and tezacaftor work together to help more proteins reach the cell surface. Ivacaftor helps CFTR proteins stay open longer at the cell surface. Together, the 3 components help F508del-CFTR proteins function better. You can learn more about how Trikafta works Here.
Now for the non scientific stuff. Fellow CFers that have started Trikafta have noted more mucus production at the beginning along with more coughing to get it out. Then the cough dies down a bit once the lungs are more clear. Lung functions have gone up. Shortness of breath has decreased. Just to name a few things. I very much am one that says “I’ll believe it when I see it.” I’m not a skeptic but I’m a realist. So we shall see what it can do for me. The goal is that it will push lung transplant off longer. The damage/scar tissue in my lungs that is already there will not be reversed so I’m unsure how much lung function I might gain. It’s a lot of unknowns right now.
We will be keeping a close eye on my liver function since Trikafta is processed through the liver. I will also be keeping a close eye on my depression as we know the last modulator Symdeko messed with my antidepressants. They both were processed through the liver so the Symdeko basically ate up the antidepressants before it could processed. So I will be keeping an eye on all of that.
I will update once I actually have Trikafta in my hands and have been on it for a little bit so I can give a real review of how it is going. Thank you so much for all the love and support. It means the world to me.
Until next time…