By Elizabeth W, Los Angeles, California
There is a lot of fear when you live with a progressive disease like FSH muscular dystrophy. Your mind is constantly circling a sea of scary “what ifs.” What if I can no longer walk around the office? What if the next time I fall I hit my head? What if someday breathing becomes harder?
The idea of purposely adding another scary “what if” like, “What if I participate in a clinical study and the medicine makes me sicker?” was untenable to me for a long time. Clinical trials are experiments done on humans to see if a potential treatment is safe and effective. The phrase “experiments done on humans” instantly floods my brain with images of Frankenstein’s monster, or the devastating photos from the Tuskegee Syphilis Study. And it’s true that clinical trials do have a dark history that is fodder for depressing documentaries, but they also have a very bright and wonderful history of scientific breakthroughs that improve and save lives.
In my millennial lifetime, HIV/AIDS has gone from a death sentence to being treatable. In the 1980s, when research was just beginning, thousands of men and women volunteered for clinical trials. Knowing full well that the medicines probably would not help them in time, they gave their time, energy, and bodies with the hope that their sacrifice would someday help their community.
Something only an elite few can do
I wish I could say it was altruism alone that drove me to do a 180 about my decision to be a clinical trial participant, but it wasn’t. Those scary “what ifs” were not only getting to me mentally; some were coming true. Living with a chronic illness, I find emotions fluctuate between denial and depression. For a long time, it was easier to ignore what was happening to my body. But denial only works for so long with FSHD. As I lost muscle strength, what replaced it was pain – pain in the muscles that did still work, pain in my joints and ligaments because of the way I walk. Pain so deep that it feels like the muscle is literally being torn off the bone. That is hard to ignore.
I decided I could not live this way any longer. I decided I would put the same ambition, passion, and diligence I was giving to my work to my FSHD. This journey started with finding a physical therapist who specialized in neuromuscular diseases, followed by getting fitted for orthotics that would help me live with less pain and fewer falls. Because there are no treatments for FSHD, my journey of doing everything I could to help myself could have stopped there. But something inside me told me that I could do more.
As much as I hate having FSHD, having it also means I have the key to curing it. There are a lot of things in this world I can no longer do. And there are a lot of things I can do, but other people can do them, too. However, participating in FSHD research is something that I and only an elite few can do.
At the time I did my 180 about participating in research, Acceleron Pharma was recruiting for their FSHD clinical trial. They theorized that their myostatin inhibitor would help grow and strengthen affected muscles. Hoping to get into the study, I aggressively emailed with the drug company and my neurologist. I qualified and was enrolled in Phase 2b, which was testing dosage, so I knew I would be receiving the medicine rather than a placebo. Spoiler alert: If you did not already know, the Acceleron trial was halted.
Forced to face my fears
It would be easy, as a one-time clinical trial skeptic, to feel bitter after taking part in a trial for a drug that didn’t work. Participating in medical research as a test subject is a time suck that is both emotionally and physically taxing. In addition, the medicine was injected with an electrical current, which was pretty uncomfortable.
Yet, at the end of the trial, I was not at all bitter. All I was left with was a feeling of gratitude. Gratitude for my neurologist, who not only conducts these FSHD trials in addition to others like the spinal muscular atrophy trials that led to groundbreaking treatments like Spinraza®, but who also sees patients, teaches, and still manages to patiently listen to every one of my questions. I felt so grateful to the physical therapists, trial coordinators, phlebotomists, and all of the hospital staff who share the dream that one day soon there will be treatments for this disease, and have dedicated their careers to finding those treatments.
But most of all, I am grateful for the shift in perspective that participating in clinical trials has given me. Because the aim of trials is to show that a medicine works, part of participating is documenting all of the things that don’t work in the hope that, over the course of taking a medicine, it does start working. During the many muscle strength tests, stair climbs, walks on tippy toes, six-minute walks, surveys, and MRIs, I had to lay bare everything I could no longer do or struggled to do.
It’s like looking in the mirror, and instead of seeing your face, you see a monster. At first, the monster is hard to look at. Just talking about the monster would get me choked up with tears. But when I did the trials and had to do those tough physical things over and over again, I got well acquainted with all of the monster’s warts and scales. Doing the trial allowed me to stare straight into the eyes of the monster, see it for all it is, and put it on notice.
Because one day, thanks to people who do clinical trials, there will be no FSHD, and the monster will be gone. Based on the many breakthroughs and all the momentum happening in pharmaceutical research, I really believe that day is coming very soon.
The feeling that I’m making a difference
Even if I cannot be there for the day when there is no longer FSHD, I know the time I spent participating in research helped that day to come. That feeling of knowing I am making a difference for my patient community is what cemented my 180 from fearing clinical trials to being an advocate for participating. Now I am filled with excitement about all of the future studies and trials that I will aggressively try to get into, because I know that with each one, even if it is just a natural history study with no medicine, the study’s findings are helping researchers better understand the disease and making a huge difference.
This feeling has also inspired me to advocate and encourage others (starting with my own family!) to set their fears aside and enroll in research studies so that they, too, can feel what I feel – that together we are making a difference that will change the lives of FSHD patients for generations to come.
If you also want to make a difference, please visit the FSHD Society’s Clinical Trial page to learn more about the trials that are actively recruiting, or visit ClinicalTrials.gov for a full list of FSHD trials conducted in the United States.