Six Months Out of CF “Prison”

For years, I had heard a major breakthrough was seven to ten years away and every seven to ten years I would grow more frustrated that an innovative drug did not exist. The term seven to ten became like a jail sentence which led me to comparing having cystic fibrosis to serving a prison term.

In 2012, the first modulator was finally approved. It was called Kalydeco but only helped four percent of people with cystic fibrosis (CF) with specific gene mutations. In 2015, Orkambi was approved which would help 50 percent of the CF population who had two copies of the Delta F508 gene mutation. In 2018, Symdeko came out as an improvement over Orkambi as it caused less side effects and it could either work with two copies of the Delta F508 or one copy of the Delta F508 and a few other genes. Finally in October 2019, Trikafta was FDA approved for people with one copy of the Delta F508 and any other mutation with regards to the other gene. I was ecstatic as I have one copy of the Delta F508 and one copy of the W1282X which is a nonsense mutation. My doctor immediately prescribed Trikafta for me and a month later I had the drug in my hands.

It’s been six months since I began taking Trikafta. It is referred to as a triple-combination therapy, combining 3 drugs that target the defective CFTR protein. I refer to it as the miracle that has made my life much more livable.
For a person with cystic fibrosis, the chloride is not moving in and out of cells which causes thick mucus and difficulty absorbing food and lots of salt in sweat. The way these modulators work is that they help the protein function in a more normal capacity to channel chloride.

I began taking the drug in late November 2019 and after four to five days I began seeing a difference while only developing minimal side effects the first week with regards to stomach pain and an increase in coughing. One really cool thing about Trikafta which I think is less spoken about with regards to reduced symptoms is that it has helped my anxiety tremendously. I thought about it this morning while doing my therapy. I have to cough and spit out mucous every ten to fifteen minutes during my treatments and when I used to cough and spit I was worried that I would see blood or that my mucous would be dark yellow or green. Often times I would just close my eyes when I spit into the toilet because I knew the result in my head but I didn’t want to see it with my own eyes. Every now and then I would look and feel defeated when the colors were anything but white or light yellow.
Anytime my mucous changed colors meant that I needed to call my doctor and either get on an oral antibiotic or go in and get a culture and some PFT’s done. Since starting Trikafta, my mucous has almost always been clear or white. I have had less coughing spasms, too. For the first time in a long time, I also feel like I can finally take a deep breath.

Another thing I’ve noticed is that when I used to laugh that I would often cough. And when I would laugh really hard that I would often have a major coughing spasm. I would either run to the restroom or try and hold it in which never seemed to work. I used to be afraid to laugh because I knew that I wouldn’t be able to stop coughing and people would stare at me. Now I can laugh as freely as I would like. How wonderful because I love to laugh and even took a comedy class and performed on stage a few years ago. Now I can do it without the fear of coughing all over the place. It’s a good thing because in the age of COVID-19, people won’t just stare at me because I cough; they’ll be frightened of me too.

I have seen the biggest difference with Trikafta with my day to day life. I run every day and I used to cough three or four times per mile. Now I’m hardly ever coughing when I sweat. It’s amazing. My lung function (FEV1 or Forced Expiratory Value in the first second) has gone from 81 percentile (which compares my numbers against someone my age, height and weight) to 95 percentile. My doctor and I discussed it and we were even able to lower my number of vest treatments per day from three to two saving me an hour every day to spend with my wife and kids. That change has not only helped my day to day physical health but it has also played a role in my fight against depression and anxiety. Of course, I am in quarantine with a teenage girl and a preteen boy so the lessening of anxiety is not tremendous. Ha Ha!
I had my first liver test nearly three months ago and while my numbers were up a little, my doctor was not concerned. The drug does put those of us with cystic fibrosis at risk for liver failure unfortunately. I’ll continue getting tested every three to four months.

The last six months have definitely changed my life. I am grateful to Vertex Pharmaceuticals who developed the drug and all of the researchers who played a role. I also want to thank the Cystic Fibrosis Foundation for investing in companies like Vertex and for continuing their mission to treat and someday cure cystic fibrosis. The CF Foundation also realizes that Trikafta only helps those who have one copy of the Delta F508 gene and therefore only 90% of the CF population in the United States. They realize that we can’t consider this a complete success until we can treat 100% of people with cystic fibrosis and eventually that we can cure all of us with this disease.

While my “prison sentence” waiting for a breakthrough CF drug is over, I don’t see myself as a free man just yet. I don’t think that will happen until this disease is not only cured for me but for the other 70,000 people around the world with this disease. I can’t wait for that day and hopefully it will be in less than seven to ten years.

Live your dreams and love your life.

Best Wishes,
Andy