First Month on Trikafta

The FDA approved Trikafta, the CFTR modulator, on Monday, October 21, 2019. Coincidentally I found myself on The Today Show that day discussing what a drug like this could do once it’s approved. A few hours later – boom! I’m officially on the clock waiting for the “magic drug” that could save my life. 

This drug will affect 90% of the cystic fibrosis population who have two things in common: they are at least 12 years old and have at least one copy of the Delta F508 gene. This is the fourth breakthrough CFTR modulator FDA has approved and the first one I can take. To say I was excited would be an understatement.

Before starting Trikafta, I had several fears. For one, what if my lung function doesn’t improve? I was skeptical to begin with as I’d never been on a CFTR modulator and therefore I wasn’t sure my 46-year-old body would take to it. This was a big deal. If it didn’t work, would anything really? Would I be letting everyone down? Would I be having to deal with my own depression? 

Another concern was what if the side effects are too much and I have to come off the drug. I’ve dealt with CF issues before but I never “knew” they were coming. It’s a little scary knowing you are going to have the flu (one of the side effects) before you even get it. 

And lastly, what if my improvement is great but my liver testing goes badly and therefore I have to go off the drug? Imagine feeling amazing for the first time in your life but a few weeks later a doctor tells you, “I’m glad you liked feeling great but the drug is damaging your body so you’ll have to come off and go back to being, well, you.” It’s like Cinderella at midnight turning back into a normal girl. I really need this glass slipper to fit.

I began taking Trikafta on Sunday, November 24, 34 long days after the drug was finally FDA approved. Dealing with insurance and my mail-order pharmacy was a nightmare but thanks to the Compass program at the CF Foundation and Vertex GPS, eventually the drug was delivered. My anticipation was great as I had fellow CF warriors tell me they felt a difference pretty quickly, including one person who noticed as early as half an hour on the drug that he could breathe deeper. Needless to say, when I was feeling very little difference the first couple of days, I became concerned that the drug wasn’t working and honestly became skeptical about everyone’s “improvement.” It didn’t help that I deal with clinical anxiety either. Then the results began coming back. My friends were having 5, 10, 15 and even 20% improvement on their pulmonary function tests after only a week or two. It was amazing.

As concerned as I was with the side effects, I wanted to have some so I knew the drug was effective. Peers had talked about the purge the first week where you develop flu-like symptoms and start coughing to the point of throwing up for a day or two. I didn’t feel anything like that until …

On the night of day three I began coughing. Those of us with CF know what our coughs feel and sound like but this one was different. This one had lots more mucus that slowly became watered down. The cough was like throwing up as each time I coughed, I felt like I needed to cough again. For years I tried to stop my cough because it made me feel sick and look different but I could tell that the watered-down cough was slowly helping so I spent night four in my basement, coughing until my normal green and yellow mucus began turning white and soon clear. Eventually, it went from gobs of sputum to what looked like rain. It appears THIS was my version of the purge. I coughed like this at night for the next two or three nights. 

Other side effects began soon after. I’d had a stomachache the first day, but not one I was concerned with until the issues with the cough subsided. That’s when one day I decided to go running while waiting on my car, which was in the shop. All of a sudden it felt like someone pointed a blow dryer on my rear end and things became very warm and began flowing out of control. I ran into the grocery store and fortunately, there was a handicapped stall where I was able to do my business. It was scary though. It was like a volcano and lasted for 15 minutes, and the cleanup was longer. It was embarrassing but I knew, just like the excessive nocturnal coughing, this was a side effect and meant the drug was kicking my body’s ass (literally this time) like it was supposed to.

A few days later, after being asked by everyone and their mother if I noticed a difference since starting the drug, I did. The changes were subtle but they were there. I was able to take a deep breath. For the first time in my life, actually, I was able to breathe all the way in and not feel congestion. I used to be afraid to laugh (though I love to tell jokes) because I feared another coughing jag. Not anymore. When I do my daily nasal sprays, I can actually feel the spray hitting my sinuses instead of sticking in my nose. Foods taste different. Fortunately, I still liked sushi. I gained about seven pounds, which is common on the drug. I have started eating like a normal person instead of eating everything within reach, as I’d been told to do since I was a youngster to avoid losing too much weight.

My energy is different. I still run two to three miles a day like normal but instead of clearing my throat maybe 30 to 40 times and coughing 10 to 12 times, I’m doing the former two to three times and the latter one to two times max. Instead of having a coughing spasm after my workouts, at most I cough maybe once and it doesn’t sound deep at all. And my mucus went from green and yellow to mostly clear with a little tint of yellow. Instead of sweating so much that I look like I emerged from a tidal wave, I look more like a guy who just walked out of his house after a nice nap. All of these subtle changes have led to amazing improvements but I had to know the big one: Was my lung function better?

Two weeks after taking the drug, I convinced myself to do a portable PFT at home. I told myself originally I would wait 30 days but the improvements that I was feeling were too great. I had to know. Two months earlier I had blown an FEV1 of 84% which was great for me and around my baseline. FEV1 is the forced air that you breathe out in the first second and the number one stat that doctors look at to determine lung function improvement or decline. That day I didn’t blow an 84, nor did I blow an 85, an 86, an 87 or an 88. I blew 89%. The best I’d done in maybe 10 years. I couldn’t believe it! 

I don’t feel like the same person anymore. Yes, I can tell I still have CF and I still do all my treatments and take all my meds, but I just feel that for the first time in my life I’m not terrified of waking up the next morning feeling worse. I’m not as scared to do a PFT and trust me I’m usually petrified. I work out with my trainer now and he says he can tell I have extra energy. I’m not coughing as much when I go out and when I do, hardly anything comes out. Things smell differently and though I’m usually very even keeled, I am now so excited about the way I’m feeling. 

I’m so excited to be on Trikafta and I’m so grateful for those who have worked so hard to make it a reality. Still there’s a part of me that feels a bit of guilt for talking about it. For one, I’ve lost so many friends to CF who cannot be here to feel these gains. And secondly, there is still ten percent of the population that can’t use this drug because it’s not a match for their genotype, or there is limited access to it in their country, or they have had transplants already, or they are too young to qualify (under 12). Everyone needs a drug like this and the only way I can avoid feeling guilty is that I make sure to advocate for the other ten percent. I’ve been there. When Kalydeco came out seven years ago, I was not a candidate. Soon after came Orkambi and Symdeko and again I was not a candidate. We need breakthrough CFTR modulators for everyone and now that I’ve seen what they can do, I’m going to sound greedy—we need a cure for cystic fibrosis.

I won’t stop fighting until that happens because although I can breathe easier now, I will breathe even easier once we all have our “magic drug.”

Live your dreams and love your life.

Best Wishes,

Andy