This week I am sharing an updated version of a previous blog post because I couldn’t let Clinical Trials Awareness Week go by without acknowledging the many, many patients who put their health and welfare on the line each and every day as human guinea pigs, in order to broaden the spectrum of treatment options for patients suffering from a myriad of conditions. I did it once and I’d do it again, except that now I seem to have aged out of most trials, I’ve been diagnosed too long, or I’ve been on too many treatments, all of which are to be expected when you’ve been diagnosed almost 17 years and living with symptoms for over 25 years easily. If you know anyone who has served in the capacity of human guinea pig, celebrate and honor them for doing for the greater good. This week, I proudly present my own experience as a clinical trial participant.
In honor of Clinical Trials Awareness Week (May 18-22, 2020), I decided to share my experience as a participant in the clinical trial for what was once FTY720, then Fingolimod, and ultimately became Gilenya, the first oral medication for relapsing forms of Multiple Sclerosis.
The months leading up to my search for alternate treatment options were some of the darkest of my life. I had exhausted all of the available approved medications (at that time only four different injectable options). After I had failed on a once a month infusion therapy, which to me was nothing more than hanging out with my friends once a month, except we all had IVs in our arms, I began my last remaining option: a daily injection, which ruled my world. I had horrible injection site reactions, regardless of how diligently I rotated my injection sites. I tried a heating pad, I tried ice, I tried morning, I tried night, I tried just after a shower. I tried everything to make things better, but to no avail. The worst part about the MS treatments is that they don’t offer any “relief”, and taking them is really all about the future. They are the best chance we have of slowing the progression of an inevitably progressive illness, but there is no instant gratification, and the results will never truly be known. In any case, I knew I wanted to be on treatment, but I did not feel that I could withstand the torture of the burning lumps, bruises, and excruciating after-effects of my current medication. 
With no other treatment options available to me, I dove into the world of clinical trials. Coincidentally, I had seen a segment on the news regarding the Novartis Freedoms II trial testing the efficacy and safety of an oral medication, and I learned that the trial was recruiting participants. I found an online screener, completed it, and was directed to a study center that was participating. After the online screener, I received a generic “thank you for your interest” email, advising me that if my preliminary responses indicated I was eligible, a study nurse would contact me. I felt deflated, again yearning for instant gratification yet not getting it. But several days later, I received a phone call from the study nurse, and spent about an hour discussing the course of my MS in great detail. At the end of our conversation, I was one step closer as we scheduled a face-to-face appointment to discuss the informed consent, which is a huge document outlining every single side effect that had been reported, as well as what my responsibilities would include. It was a pretty scary wad of papers that my husband and I scoured through (also enlisting the help of my dad, my sister, and my mother-in-law), and came back with a list of questions for my next appointment.
This picture was taken when I was still working, and still a human guinea pig.
In November of 2008, I had my first of many appointments at The Gimbel MS Center at Holy Name Hospital in Teaneck, NJ. It was one of the very first ever comprehensive care centers for MS and it has a great reputation, particularly relating to research studies. I met with the study nurse who went over the informed consent, point by point with me. She patiently answered all of my questions, understanding of how scary a proposition it is to enroll in a research study where you may (or may not) be taking a medication that is brand new, with side effects and efficacy yet to be discovered. Preliminary results from early trials can be encouraging but still do not tell the entire story. But that November day in ’08, I made a decision that undoubtedly changed my life for the better. I consented to a two year, double blind, placebo controlled clinical trial. I knew that the possibility existed that I would be on no medication at all for two years, but they were testing two different drug dosages versus placebo, so I felt that my odds were pretty good. Instead of a fifty/fifty chance of getting the product, I had a two out of three chance. In my mind, I just knew that the universe would take care of me.
Being in a clinical trial requires commitment. Even before I took my first dose, I had two full days of baseline testing which included visits with a dermatologist, an ophthalmologist, pulmonary exams, MRIs, EKGs, and I even had to wear a 24 hour Holter Monitor (which I told my students was recording everything going on in the classroom…and they believed it!). Additionally, I had to meet with the researching neurologist, the examining neurologist, and I underwent a battery of neurological tests (which I’m convinced are all meant as torture devices) with the study nurse. And this process was repeated every three months. As much as scheduling was often difficult because I was still working, being monitored so closely made me feel safe even in previously uncharted territory in the world of MS treatments.
It didn’t take long before I was convinced that I was on the product and not a placebo. I am very sensitive to medications, and I am known to experience side effects, even those deemed “rare” for most patients. This was no exception! In fact, there were times that I was forced to take a “study drug holiday” until things normalized in my system. I was doubtful that my elevated liver enzymes, or the variation of my absolute lymphocyte count could be attributed to the “placebo effect”! But I still stuck it out. I committed for a two year trial, and I knew in advance what the risks were. I could have opted out at any time, but I felt it was important to follow through. They might not have liked how my body reacted at times, but it was data that needed to be collected for the good of the MS Community.
This was a 3 month supply of my study drug. Every 3 months I had to bring what was remaining and switch for a new “kit”, even if there were pills left. They always gave a few extra just to allow for scheduling issues.
Nothing about the clinical trial surprised me, including the fact that they did not want to use me in an extension trial. In fact, they did not even want me to complete the trial with them. At month 21, the very product I had been taking, became commercially available as Gilenya, the first ever oral medication for relapsing forms of Multiple Sclerosis. I maintain that if my numbers were ideal throughout my time in the trial, I would have been asked to complete the “exit” portion, which in essence, would have sealed my numbers into the results of the trial (and also given me “free” study meds for 3 more months!). I transitioned to the commercial product, which was fine by me, because I still got what I wanted in the end: another treatment option.
This is what the Gilenya packaging looks like. It is the worst concept ever, particularly for patients who lack manual dexterity. I hope they re-visit this concept at some point! Also, it is only a 28 day supply, and runs about $5,000.
I almost jumped for joy when the packaging changed to a regular pill bottle and an actual 30 day supply!
Years later, we were “unblinded”, and as it turns out, I was on Gilenya the whole time. I learned a lot through the process, and I know it taught me more about being a “professional patient” than I ever thought possible. It’s true that my motives for enrolling in the clinical trial were self-serving, but ultimately I knew that I was also doing for the greater good. And although I am no longer on the product, I endorse it whole-heartedly because it served me well for over eight years, which is an amazing run where it comes to treatments for MS. I was patient number 002516, and I’m damn proud of it.
