In Which I Talk About Trikafta!

I have now been on Trikafta for somewhere around six months. I have been on full doses, half doses with one in the morning and one at night, half doses with two in the morning, and a whole lot of trouble in between. Ultimately, Trikafta has been a real difference-maker. How? What does it do? How can you tell it helps? Let's talk about how it has affected me, specifically.

Trikafta is a CFTR modulator. The CFTR is a protein that reaches the surface of a cell, opens up, and allows chloride into and out of the cells. This keeps your body from developing thick, sticky mucus in your organs. For Cystic Fibrosis patients, our CFTR proteins don't function correctly. This causes our mucus to get quite thick and sticky in places like the lungs. This opens the body up to harmful bacteria to get into the lungs or other organs and cause infections and other problems. 

Trikafta works by targeting the CFTR defects that are caused by the F508del mutation that most CF patients have. The combination of medications in Trikafta (ivacaftor, tezacaftor, elexacaftor) help the CFTR proteins to reach the surface of the cell and to stay open longer once they get there. This has the effect of reducing the thickness and amount of mucus produced, which results in less coughing; my digestion has been better due to the smaller amount of mucus in my intestines, and I am having less joint pain and general soreness. It has really been incredible. 

I first started Trikafta late last year when my clinic here in NYC got me approved for the Vertex Pharmaceuticals compassionate release program due to my health status at the time. My average lung function at the time was around 30%. I was sick enough for hospitalization nearly monthly. I had also developed a near-constant hemoptysis (bleeding of the lungs). Additionally, I was in one of the worst headspaces in recent memory due to the frequency of my hospitalizations and how everything seemed to be tumbling downhill quickly, and with seemingly no end in sight. This was not a good time for myself or my family. I couldn't walk more than a few blocks at a time without becoming extremely winded and needing to rest. Anything more than a single flight of stairs was often too much for me. I was missing a ton of time from work because I just couldn't be effective. I was quite depressed and it was becoming a problem. Most days I felt barely able to be out of bed for an hour or so at a time. I was missing a lot of time with Aurora and just not able to be the parent Aurora deserved. Then came the news that I was approved for compassionate release for Trikafta.

After my first couple of doses, I felt awful. Initially, you get these flu-like symptoms that wreak havoc for about two to three days. Achy body, fever, lethargy, and more! However, once you get about four days in, these symptoms start to subside. Next, you start to notice the changes for the positive that come. The most immediate differences that I noticed were how thin my mucus secretions were and how much more energy I had. Secondary to these, I realized that I was far less achy than usual. For the first time in years I could sleep flat! This was one of the most notable changes for me. It may seem insignificant, but it was a gigantic deal to me.

Several years ago I had actually purchased a new mattress and an adjustable bed frame specifically because I was having such difficulty sleeping at night. Quite literally, as soon as I laid down I would begin large, severe coughing fits that would often last through the night. Practically overnight, these problems vanished within a few days of starting Trikafta. 

This honeymoon period was short-lived. A few weeks into starting the medication I began having some of the worst chest pain I've had, possibly ever. Eventually, after several trips to the emergency room that gave no indication of what was happening, I saw a specialist and got a special type of MRI. We had the information we were seeking. One of the pancreatic cysts I have had for my entire life had suddenly grown from around one or two centimeters to about fifteen! To compare this to something tangible, it was about the size of a grapefruit. It had grown so large that it was pushing my liver into a crescent shape.

When all of this got started, I had been moved to a half dose of the medication. Initially, this was due to the possibility of the medication affecting my pancreas or liver. As it turns out, it didn't. After surgery (about a month and a half ago), it was discovered that my pancreatic fluid was draining into the cyst instead of my stomach. Additionally, I had some stones in my common bile duct. The surgeon put a stent connecting my stomach to my pancreas and I was to come back about a month later to have it removed, and to remove the stones.

Fast forward to now, and I have had the stent and the stones removed and seem to be doing really well! My hope is that I will be able to get blood work done soon and get back to a full dose of the medication. A half dose is fine, but it is no substitute for a full dose. My secretions are thicker and I cough more on the half dose. I still have the energy and general feeling of being able to get out and do things. My lung function, at last check, was around 45-50%. That is a huge increase! Now that I am feeling so much better physically, I have felt more able to tackle my mental roadblocks. I've started using a daily planner to keep up with my days and important events, and this has helped significantly with my ability to focus on tasks and feel like I'm contributing to my family. 

I am grateful that I can take Trikafta and that it works for me. There are still some that cannot yet benefit. It is incredible to think that just a few years ago I would tell middle school students that I wasn't sure we would ever see a cure or medication that would help as much as Trikafta. Here we are. Certainly not a cure, and certainly not where we would like to be with therapies, but this is a magnificent start. I am looking forward to getting back to a full dose, and see how I feel after having been at half for so long. It is crazy to think that nearly fifteen years ago I was in clinical trials for some of the medication I am taking today. I am grateful that I could contribute in some small way through my prior donation drives and clinical trials. Here's to the next breakthrough!