“The approval of a new medication this past October gives me hope that easier years are on the horizon. From the first day I received it, until now, I can’t help but smile with hope that things are looking up.”
My future was delivered to the Harrison Package Room at 11:02 AM. As soon as I got the notification, I threw on clothes and sprinted to the elevator with wet shower-dripping hair. Once I hit the ground floor, I ran to the package room as fast as I could. When the mailman looked at me, I beamed and handed over my school card. I had never been so excited about a single package in my entire life. He scanned my card, rustled around, and eventually handed me a thin white box. Although he had no idea how special the moment was, I like to think that if he knew what he was holding, he would feel the magic too. I grabbed the box with both hands, spoke a breathless “THANK YOU!” and was gone in a flash.
That morning I would take my first dose of Trikafta, a new medication that was approved for people with cystic fibrosis. It is a triple-combination modulator therapy made by Vertex Pharmaceuticals which targets the root cause of the disease, instead of just the symptoms. The reason that Trikafta is such a big deal, and warranted my race to the mail room, is that in the drug trials it was proven highly effective, with patients gaining over 10% of their lung function back within weeks. However, Trikafta isn’t the first modulator therapy to come out of Vertex. In the past Vertex has released two other similar therapies (Orkambi and Symdeko), but Trikafta is the one with the greatest reach within the CF community. This is because Trikafta is effective in people who have at least one copy of the most common mutation of cystic fibrosis (deltaF508). This essentially means that over 90% of the CF population can take the drug.
But if Vertex had already made modulator therapies before, why is Trikafta so much more effective? It all lies in the third piece of this triplecombination drug. Orkambi and Symdeko both only consisted of two medications. However, Trikafta uses the two medicines from Symdeko, while adding a third called Elexacaftor. It is this third medication that makes all the difference. Because I have two copies of the deltaF508 mutation, I was able to take both Orkambi and Symdeko. On Orkambi, I gained over 10 pounds within the first five months on the drug, but I didn’t feel a huge change within my lungs. Additionally, Orkambi was a powerful drug and the side effects, such as hair loss and chest tightness, eventually outweighed the benefits. Then, I began Symdeko which wasn’t as hard on the body, but I didn’t feel a huge difference in my lungs. While I am sure that it made me stronger, it was nothing like how Trikafta has made me feel.
After I took my first dose of Trikafta, I felt it working within hours. Over that first day, I coughed up a lot of mucus and cleared out parts of my lungs. After about three days, I stopped coughing as frequently and felt myself breathe easier. Just before I received the drug, I had gotten a cold and was struggling to get my health and sinuses under control. Once I swallowed that morning dose, I felt some of that infection calm down. My nose cleared up and my mucus became a lighter color. It was as if someone had turned back time a little to when it wasn’t as hard to breathe. The most notable change to me is that when I wake up in the morning my chest is no longer heavy from mucus. Before Trikafta, every morning I had to start my breathing treatments quick before I could do anything else, because of all the mucus that had built up overnight. I would spend the entire day coughing it up and then once I went to sleep it would build up again. Now, I wake up and no longer have as big of a burden in front of me. I still have to do all of my treatments, but I wake up and feel good even before I start them.
The response that I have had to the drug, isn’t limited to my case. I know that people across the nation, and even others in Tyler, are feeling the relief that I have felt. This drug isn’t a cure-all, it can’t undo the years of damage that CF has done, but it is a step in the right direction. With this drug, the door has been opened to so many more possibilities. Within the drug pipeline there are currently eighteen drugs that are being tested for correcting the root problem of CF. They all have the potential to be as great, or even better, than Trikafta. It is for this reason that I am certain that we will find a cure soon.
While it is, of course, because of all the hard work that the scientists at Vertex put in that have made this drug possible, it is also thanks to the people of Tyler, Texas, that it was developed. For twenty-two years, this city has fought with me and many others against this disease. Together, we have raised over $6 million. Ninety cents of every one of those dollars has gone directly to research, and it is from that research that Trikafta was born. So, although this drug is not a cure and the fight isn’t over, just know that your time and money have truly made history and that when I am taking in deeper breaths, I am always thinking of you, the people of Tyler, Texas, who have helped me get there.”