Friday, February 25, 2011


To some of you, those numbers and letters mean absolutely nothing. For a few of you, those random numbers and letters may change your entire future.

VX-770 is a medication for people with cystic fibrosis that has produced incredible results in Phase 3 clinical trials. It is the first medication of it's kind, targeting the actual gene defect rather than simply treating the symptoms. Revolutionary, life-changing and miraculous are all words I've heard people using to describe this drug.

Only about 4% of the CF population has the specific gene the VX-770 targets (G551D). I am not part of that 4%, but this is still HUGE news. It is an enormous leap forward; a stepping stone to a drug that will someday help everyone with CF, and it is one step closer to a cure. I'm not one to get all giddy when I hear about most drug trials, and hearing "a cure will be found in the next few years" gets old after hearing it for 15+ years. But you guys, there is proof backing this up. IT'S WORKING, and that is something to be excited about!

A couple articles about VX-770:


  1. I start the study monday. i hope i don't get a placebo.

  2. That is so cool Jenny. Wait, do you and Teresa have a different gene???

  3. No, Holly, we have the same genes. The study she's going to do is for the same drug, but it's a different version that specifically targets our gene mutation... if I understand that correctly. It's a little confusing even for me!

  4. we would need two drugs, the vx-770 but another drug (vx-809)also. I will be involved in testing the two drugs in combo with each other. (for people with the other mutation, the necessary protein is at the surface of the cells already, the 770 just helps it get out of the cells and working in the body. in our mutation, the protein is not yet at the surface of the cells. The 809 would get it to the surface,and then the 770 would fulfill the same purpose of getting it out into the body.)


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