Vertex

Taken from the CFF.org website
"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.
VX-770, the most advanced of the two compounds, is known as a potentiator and is designed to allow CFTR located at the cell surface to function correctly.
Vertex is also developing VX-809, known as a corrector, which is designed to move defective CFTR to its proper place in the cell."

In January of this year my doctor's office called me and asked me if I wanted to participate in a new drug trial. It was for Vertex 770. They wanted a group of CF patients who were homozygous (carried two of the same genetic mutations) for DeltaF508. I was one of the few patients at my CF Center that was eiligble for this. I jumped at the chance! In order to qualify for the study you had to have a FEV1 of 40%. Luckily at enrollment date my FEV1 was 50%. I began the 16 week study. My first PFT which is the one that counts was only 38%. But since I had already enrolled in the study at 50%, I was still able to partake. It was a double blind study. That means that both myself and my doctors did not know whether I was receceving the actual drug or the placebo. The study entailed me keeping in touch with the doctors every two weeks. I either had to go into the office for a sweat test, multiple blood draws, pulmonary function tests and EKG's or follow up with a phone interview. The pill was a large blue pill that I had to take twice a day after consuming a high calorie meal. To this day I still am not sure whether I was on the placebo or actual drug but I am glad that I participated! At the end of the 16 weeks you were able to continue on to the second part of the trial if you had either a 10% increase in your FEV1 and/or a decrease in your sodium chloride level from the baseline sweat test that was drawn. I am not sure what my last sweat test result was but, I did have an increase in my PFTs. Day One (FEV1 - 38%). Week 16 (FEV1 - 43%) which calculated to be over a 10% increase.

In June I began the open label part of the Vertex 770 study. The study is going to go over a 96 week period. I am now officially getting the real drug. I still have to go to the doctors for visits but not as often as the first part. When I do go I still get blood draws, EKG's, PFTs, and sometimes a sweat test. I also have to go for liver function tests every two weeks. I got hospitalized at the end of June and my doctors made me stop taking Vertex for a little while because I was running fevers and they wanted to make sure that it didn't have anything to do with the drug. I began taking it again continuously at the end of August. It's been almost a month now of taking it on a regular basis and I really do feel like it is making my mucus thinner.

I am excited for the future of CF drugs. Vertex has two different compounds VX-770 and VX-809 and from what I have been reading - eventually they will be tested together. The two compounds together are supposed to have the best result. One is a corrector and one is a potentiator - so each compound has different effects based on your mutations and the effect it has on the CFTR protein. Research has actually shown that VX-809 has a better result in my genetic mutation (DDF508) - so I am really hoping that my CF Center will soon be testing this compound as well.