You can ask my entire family — they’ll tell you I was fully willing to participate in clinical trials from day one. I’m assuming it was a combination of the previous minimal research I had done before my diagnosis, being an adult (or being older because, let’s face it, 22 is not an adult. Ha!), accepting that I have lived a pretty full life, and the sheer and utter fear of not having a single answer for something that was going to drastically change and probably take away my life.
I always say there is a reason for everything. Everyone’s favorite cliché, I know, I apologize. But honestly, there is a reason I didn’t become noticeably symptomatic until about age 19. In my heart and soul, I know that my family would not have been able to handle having a daughter with FA as well as they are now.
Don’t get me wrong, my mom is probably one of the strongest people I know, as well as my grandma, but I honestly don’t think they would have been able to handle those trying situations as gracefully and suddenly that we have faced as a family. I also know that I probably wouldn’t have the mindset I do now if Friedreich’s ataxia had been part of my life at age 5, 10, or even 15.
That is why I give so much credit and honor to those parents of young children with FA, those who have had it for a long period, or just kids with FA in general. I am 25 years old and still in the elementary stages of progression, and I’m having a hard time dealing with FA.
I haven’t even touched on some of the difficulties my friends and other patients face, and I already feel like I’m drowning in loss and frustration some days. The harder pill to swallow is that it’s only going to get worse and there’s nothing I can do to stop it.
The only way I have even come close to feeling like I can help find an answer is by participating in clinical trials. Let me get this out of the way: By no means am I saying that you need to participate and sign up tomorrow. You have every single right as a patient or parent to question the process, the doctors, and the medication. You should be doing as much research as possible, having doubts, raising concerns about certain things.
Truth is, it’s scary and there is nothing I can say to take away that initial fear. I understand people might have certain views about the U.S. Food and Drug Administration and big pharma, and that’s totally fine. That is completely up to you, and it is a big decision for you and your family to make. If anything makes you or your child uncomfortable, don’t do it. I wanted to share my personal experiences with you in case you wanted a patient’s perspective.
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Since being diagnosed with FA in June 2015, I have completed the Acctimune/Interferon gamma trial and the most recent Reata/Moxie 408 study, now going into Phase 2 (NCT02255435), as well as being a part of the natural history study for FA patients. Acctimune required two shots, every other day, for about eight months; Moxie required taking a pill daily for about three months; and the natural history study is just a checkup once a year that tracks the progression of Friedreich’s with questionnaires, the dreaded peg test, and all the other basic FARS tests that patients regularly go through when seeing a neurologist.
I will go into depth about each trial in future articles, but I just wanted to share my own voice as a patient on the importance of participating. Only 15,000 people worldwide have FA, with about 5,000 to 6,000 of them living in the United States. That’s really not a lot of people if you think about it.
A good number of trials have requirements for physical capabilities, age, stage of progression, and other similar factors. Because FA is progressive and typically diagnosed before age 18, the search for potential candidates becomes scarce and even more difficult. Some studies require 30 to 100 or more patients, and it can be difficult to find someone who checks everything off the list. That’s why I think it’s important to participate and have more potential candidates to choose from.
Personally, I had to make a list when it came to weighing the pros and cons of joining the clinical trial realm, and I found there were more pros. How many times have you gone to a doctor and you have to be the one to teach them about FA? Annoying and disheartening, right?
When joining a trial, you now have access to doctors who not only specialize in Friedreich’s, but also have dedicated their lives to helping find a cure. Dr. Susan Perlman is my doctor at UCLA, where she is at the top of the neurology department and helps run an entire ataxia clinic on campus.
And don’t forget, the drug company will compensate you for travel expenses as far as gas or plane tickets or a hotel. I know the money part was a huge issue with my family and me, but once we actually learned how helpful the companies were and we didn’t really have to pay for anything, including the meds, it helped in my decision-making.
You always have the ability to withdraw at any time, which also made me feel a little more comfortable. But the biggest reason was the access to a specialist who knows everything about FA. To know that your doctor can answer, or find out, any question you have about your body and the disorder is a huge relief.
With these doctors, it’s their life’s mission to find a cure for something they aren’t even dealing with themselves. To experience and hear about other doctors in Philadelphia and Tampa, and how awesome they are with leading research on FA, justifies that in my mind. The comfort I now have at the UCLA neurology department is all because of Dr. Perlman and her amazing staff. They are responsible for my journey with clinical trials and helped me a lot during the past two years in dealing with my progression.
What I’d like you to take from this, really, is to give it some time. Do some in-depth research on clinical trials. Ask yourself what your genuine concerns are and express them. These doctors will answer your questions.
More people are needed to figure out if something may or may not work for the FA community. Results, negative or positive, are crucial to finding the right medications — and you could help contribute to finding a cure. Give it some genuine effort and draw your own conclusions.
I’m honestly not trying to draft you into taking part in clinical trials. I just had great experiences, and I wanted to help address concerns that I also once had.