Grief is very real. It’s exhausting, it’s sneaky, and it’s unpredictable. And it spans to all areas of loss – actual, perceived, or even anticipated loss. Growing up with cystic fibrosis and an overactive mind, I’ve been in stages of grief throughout my life. I grieve the thought of my own mortality. I grieve the loss of my past reality as my physical limitations change. I grieve the friends I’ve lost to this disease (and others). I also grieve the strangers I’ve lost to this disease. But, most recently, this year I’ve been grieving the loss of the hope I had that change was on the horizon for me.
Last fall, I was fortunate to be selected for an exciting clinical trial. This was a first-of-its-kind medication for my genetic mutation, and preliminary data showed that it could and should be life-changing – a drug that would truly transform my world. Within weeks of starting, I was certain (and hoping) that I was on the placebo because I didn’t notice any change. For those 24 weeks, I felt the same as usual, but I held onto hope that my chance was coming. After the initial period, I had the option to transition into the open-label portion of the trial – AKA the actual drug with no more possibility of a placebo.
That day was March 21, 2019. I was giddy with excitement as I made my way to the study appointment, filled with disbelief that the day had finally come. For a little bit of toot-my-horn backstory, I’ve been raising money for the Cystic Fibrosis Foundation since I was tiny. My fundraising efforts started out small, but for the last decade, I’ve raised about $25,000 a year, sometimes more. As I’ve watched the Foundation make significant progress, I’d been patiently waiting for my drug – my miracle.
Within a few days of starting the drug, I noticed I wasn’t coughing much. Then, one morning, on a walk with the dogs, I was inspired to run ahead with one of them – a thought that would never have crossed my mind before. As I ran, I realized I had no shortness of breath. I stopped and tried it again. And again. And again. I couldn’t believe what was happening.
At my two-week follow-up appointment, the doctor asked how I was feeling. In that moment, I realized I hadn’t thought about breathing in days. I responded, “I don’t know. I think I might be feeling how normal people feel.” It was incredible that, after a lifetime of coughing, suddenly I had achieved something that was seemingly simply, yet unfamiliar to me – a freedom from the chains of shortness of breath and coughing.
My energy level was incredible. And, by incredible, I think I mean “typical.” I didn’t find myself getting back in bed throughout the day. I couldn’t remember the last time I’d said I was exhausted before noon. I was socializing and running errands; life was no longer about conserving energy.
At my four-week appointment in mid-April, I was raving about how great I felt. My lung function was climbing. I found myself putting the cart before the horse, thinking grandiose ideas – no more hospitalizations, no more IV antibiotics, no more sinus surgeries, etc. However, two days later, my phone started ringing and I saw that it was the study doctor. I knew this wasn’t likely to be a positive phone call. She explained that my liver enzyme levels had increased significantly and that I needed to be retested within two weeks.
I waited as long as possible, trying to give my liver time to resolve whatever hissy-fit it was throwing. I had labs repeated on the first of May, and I received the dreaded phone call the next night. My liver values had gotten so high that I was forced to discontinue the drug. To say I was devastated was an understatement. I had just gotten a taste of what life could be like – six weeks of normalcy, of effortless breathing, of freedom. Then, in the blink of an eye, it was gone.
The phone call came on a Thursday night and sent me spiraling into a very negative place. I spent the weekend sulking, and I gave myself full and complete permission to stay at the pity party until the lights came on and I was told to leave. I went from thinking the drug was too good to be true to realizing that it was, in fact, too good to be true. (A few days later came the leukemia diagnosis, but that's beside the point...)
In the time since, my liver has gone through a litany of evaluations. I got every blood test under the sun, an abdominal ultrasound, and an MRI with and without contrast. I saw a hematologist who deemed my liver to be perfectly healthy. With complete confidence, he told me that the dangerously high enzyme levels were caused by drug-induced liver injury. And, to twist the knife a little bit more, he told me that he wouldn’t recommend that I ever take the drug again, at any dose, due to risk of acute liver failure. Ouch.
The drug has since been submitted to the FDA for approval and will likely go to market in the near future. Of all the grief I have experienced, I don’t know that I’ve ever felt such grief over a medication. As I watch the community justifiably celebrate this groundbreaking announcement, it hurts. My liver is fine, but my heart hurts. After years of grieving my own health, I finally had a reason to rejoice – the prospect of a new beginning free from the grasp of my disease. Now, I simply try to squint my eyes while everyone around me revels in what’s to come.
I know I will continue to grieve as the news breaks and transforms so many lives around me. Yet, despite my grief, each day is still a small victory; it's a reminder that I've transcended the odds, and that’s a reason to celebrate. But still: dang. Grief is a hard pill to swallow.