The CFTR modulator program began in 2000. This was the biggest breakthrough for CF since the CF gene was discovered in 1989. There was excitement and real hope that researchers were finally able to understand the genetic disease and the underlying cause of it.

I was about six years old when all this was going on and of course had no idea what any of that meant. I do remember my mom telling my older brother Ray and I that maybe one day we wouldn’t have cystic fibrosis. I remember Ray and I sat up in our bunk beds so excited as we talked about what our lives without CF would look like. We were most excited to someday not take pills and to not do vest therapies. This meant sleeping over at grandma’s house without packing anything and that was considered a big win for us.

Our mom said she would take us anywhere in the world when that day came. Ray and I stayed up late that night trying to decide where we should go. Since I was only 6 and he was 11, we didn’t have a huge understanding on just how big the world was. I offered up the idea of going to Indiana because our family had just went on a RV trip to Indiana and apparently I very much enjoyed that. Ray of course said we should go to Graceland because that’s where Elvis lived. We eventually ended up agreeing on Disney World.

Twenty years later and news broke in the fall of 2019 that the FDA has approved a medication that is “life changing” for 90% of people with CF. This new medication is a triple combination therapy, meaning they’ve been adding on to it over the years as improvements were made possible.

It’s been over 30 years of research in the making. Cystic fibrosis is known as an “orphan disease”, meaning it’s a rare disease that less than 200,000 people have. The Cystic Fibrosis Foundation is not funded by the government which means all the millions of dollars that have been raised and gone into research and awareness is because of the dedicated and passionate CF families and friends.

This disease has taken lives of children and young adults. It’s caused so much pain and worry for everyone affected. It’s been an ugly and relentless disease but in 2019, doctors are calling this new medication for cystic fibrosis, the greatest story in medicine. They are literally transforming a genetic disease! I can’t even get my head around that. I can’t believe I am lucky enough to be here and am able to benefit from this medicine.

I have been on Trikafta for over two months. I immediately gained six pounds within the first week. My cough went away after three days of starting it and my energy is at a level I haven’t experienced since high school. This is truly the miracle pill My family always hoped for.

Don’t get me wrong, I am THRILLED about this medicine! I don’t even know how to truly explain what this means but this feeling has to be the definition of bittersweet because I can’t help but be sad that my big brother Ray isn’t here to celebrate with me. He was the only person I knew growing up that also had CF. We sat with each other for hours each day during our vest treatments and waited for the other one to finish since we only had one machine, we chugged weight gain shakes together like it was a game, we went to every doctor appointment together which always ended up being an all day affair but with Ray it was fun because we usually were goofing off and laughing. 

Ray was my CF role model. He always confirmed that this was all okay. That boy preached that we were given CF because we are tough enough. He got a picc line first, was monitoring his blood sugar before me, stayed in the hospital before me. He did everything without fear. With Ray, this disease didn’t seem so scary because I wasn’t facing it alone.

Eventually I had to continue fighting our disease by myself. I have always had the most incredible support from my parents and medical team throughout the years but nothing has or ever will compare to an older sibling who knew exactly what I was going through all while leading the way.

I’ve been waiting for something like this new drug to come along since I was 5. This isn’t exactly how I envisioned it would be but out of respect for Ray and all those with CF who left this world too soon, I refuse to waste this gift when I have been given the opportunity to live a longer and healthier life. I promise to live life the way Ray would have and to continue to advocate for those with CF who still need Trikafta and other life saving medicines. The fight isn’t over until CF stands for "Cure Found." There is still work to be done.

 

Help Others Live STRONGER and LONGER-

 

 

Martha Garvey is a 26-year-old with cystic fibrosis. She was diagnosed at birth. She lives in Minnesota and works as a college instructor for young adults with autism. In Martha’s free time she is busy snowboarding, playing hockey, kayaking, or playing with her dog Charlie.

 

 

 

***Views expressed in the CFLF Blog are those of the bloggers themselves and not necessarily of the Cystic Fibrosis Lifestyle Foundation***