Friday, August 19, 2016

The end of VX 661 study

As most of you know, for the past 7 months, I have been participating in a Vertex clinical trial testing a drug called VX-661 in combination with Ivacaftor. On Monday, Vertex released a statement saying they are stopping the trial for people with one copy of deltaF508 mutation and one copy of another minimal CFTR function mutation (what they refer to as "het-min" mutation combination). Meaning my part in this study is finished. The results thus far showed that the drug was not providing meaningful benefit and therefore was not worth the time, effort, and money to continue studying. This drug combination is still being studied in other mutation combinations and is thought to be more promising.

When I heard the news on Monday, I was filled with mixed emotions. For starters, I so desperately want there to be a cure for CF, and we're so close I can almost taste it! Yet we're not there yet, and my patience is growing thin waiting. Also, I've seen so many great success stories for people on Orkambi and Kalydeco, both drugs that will not work to treat my mutations, and I feel the clock ticking. I want to be seeing those kinds of improvements. I want my lung function to go up, not down. I want to be able to breathe fully and deeply. I can only do so much for myself when it comes to beating CF, and I want a chance to experience more life like those on Kalydeco and Orkambi. Don't get me wrong, those two drugs are not cures and are not the solution to every CF patient's problems, but they have helped some people tremendously. I had hoped VX 661 would help me, even if it was just for a little bit before a new drug came out that was even better.

However, I'm not surprised that the VX 661 study was discontinued. I really didn't see any noticeable improvement while in the study. My PFTs did get a nice boost in May, but I think that was more due to the fact that I was training for a 5k than the medicine. Overall, nothing about my health  changed, and I never felt any different. In a way, it's nice to be released from a study that's not working so I can possibly participate in more promising studies in the future.

Am I disappointed in the results of the VX 661 study? Absolutely. Do I regret my decision to participate in this study? Not at all. I can take pride in the fact that I helped researchers discover what doesn't work so they can try harder to find what does. Failure often comes many times before success, and I am happy to help science take one step further to finding that success. With my specific mutation combination, the researchers believe it will take a triple drug combination to correct the CFTR dysfunction, and Vertex is working fervently to get this combination to the public, with studies hopefully starting by the end of this year. I hope to be part of more promising studies in the future--there are a lot of exciting opportunities on the horizon!

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