Vertex Pharmaceuticals is developing two oral compounds designed to treat the basic defect in cystic fibrosis—a faulty gene and its protein product, called CFTR. In March 2009, Vertex Pharmaceuticals initiated a Phase 2a clinical trial of VX-809 in patients homozygous for the Deltaf508 mutation. Homozygous means that a person inherited the same gene from both parents. Ryan and I are both carriers of the Df508 mutation so Ben qualifies as a patient for this new drug. VX-809, a CFTR corrector, is designed to increase the concentration of F508del CFTR proteins at the cell surface. Cells that regulate Ben's salt/water ratio make everything they are supposed to make, but what he needs is for those cells to actually function...at this point they don't (causing mucus to be extra thick and sticky) and this medication allows his cells to open up a (chloride) channel allowing his cells to function. The VX-809 drug "pulls" everything to the cell wall, where all it needs is a channel to open up allowing their cells to function too.
I know this is all very confusing and I hope I am getting my facts completely straight. I'm still trying to wrap my mind around all the medical terms! Thank goodness for my RN mom. Basically this new drug could drastically improve lung function. The best part is it's taken orally. The drug is only in phase two (available to only adult CFer's) but once it gets to phase four it will be open for Ben to try. In phase three children (6-11) will be able to try it and in phase five it will be FDA approved! This could take many years but this is a HUGE deal in the battle against CF. Please continue to pray that this drug does what it needs to do. To read more, here is the article link: