Skip to main content

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!


Popular posts from this blog

Exciting news!!

It's been awhile since I've written a blog post. This semester has been busy--not only because of school stuff, but also because of exciting life things. About a month ago, the most amazing man got down on one knee and asked me to marry him! And of course, I said yes!

Honestly, there were times that I questioned if I would ever marry someone. Living with cystic fibrosis is hard. Choosing to be with someone with cystic fibrosis is almost crazy. Think about it; I am not a normal 23 year old. I have to plan and plan and plan to make sure I fit breathing treatments, exercise, and eating into my schedule. When I travel, I have to take a crazy amount of stuff with me--my Vest, nebulizers, compressor, pills, inhaled medication, puffers, and snacks. I have to make sure I sleep 8-9 hours a night because my body uses more energy than most, and I need sleep to fight infection. I have to have a course of IVs at least twice a year. It's hard to be spontaneous and adventurous because CF…

End of semester update

The past few weeks have been rough.

I got a cold about 3 weeks ago. No big deal, right? I hadn't gotten a cold in 10 months, which is basically a record for me. Surely my body could fight a cold off. Boy, was I wrong. After a week of fighting the cold, I started running a high fever and felt like a truck had run me over. I finally called the doctor for some reinforcements and started on an oral antibiotic and a course of steroids. Meanwhile, my school semester had just picked up, and I was down to the last 2 weeks of school. I had to finish a research paper, take 2 finals, write a final progress report for my practicum, and give really good, productive therapy. After 4 days on the antibiotic and steroids (Monday with one week of school left), I still was feeling crummy. I checked my oxygen and noticed that it was low--running at about 90-94%. I'm pretty positive the cold had turned into pneumonia. Even walking from my bedroom to my living room made me winded. It took me two-th…

A note to incoming college freshman

Recently, I was talking to a young woman who will be starting her freshman year of college in August. She didn't ask for my advice, but I gave it anyway. I tend to do that sometimes--blame my desire to help other people succeed as best as I can, I guess. Anyway, that got me thinking, what would I write if I were completely honest and vulnerable with incoming college freshman? What would I tell them that no one else seems to tell them? My freshman year was only 5 years ago, so my memories are still fresh. I came up with a list of things I found out to be true my freshman year. They may not all be true for you, but I hope and pray you gain some insight out of the lessons I learned.

1. Join a church and get plugged in to a small group. This should be your first order of business the minute you step on campus. Besides, you know, finding your dorm room and where all your classes are. Getting in a small group was literally the best thing I did freshman year. Not only did I start forming…