Let Your Voice Be Heard -
Stories from participants in the magnesium clinical trial
by Chris Rumer, PXEer
This article first appeared in the June 2014 eNewsletter.
Terry Mac Dermaid, PXE International: Chris, thanks for taking the time to answer my questions. Why did you volunteer to be a participant in Mt. Sinai's clinical trial testing magnesium supplementation as a treatment for PXE?
Chris Rumer, PXEer and Participant: I volunteered for the clinical trial because PXE is something I’ve lived with since I was 7 years old. Because it has received such little attention for most of my life, I feel this research opportunity is a duty of mine, to volunteer myself for the knowledge and betterment of others, which this research can bring. Also, as a scientist in my professional career, I understand how important research and clinical trials like these are. If each individual viewed this opportunity as something not worth pursuing, progress for a treatment for PXE would never be made. Lastly, this presented an opportunity for me to really learn more about PXE and to be able to ask specialists that are very familiar with PXE many questions I’ve had and to build a network for future reference that also allows me to communicate with others through PXE.org the knowledge that I’ve learned through this study. I couldn’t be happier with my decision.
Terry: What were your expectations regarding PXE before beginning the trial?
Chris: My expectations of this study began with the highest of hopes of taking a relatively simple medication to mitigate and maintain PXE conditions and possibly reverse them. At worst, my expectations were that I would learn a lot about PXE and should anything evolve negatively from the medication, that I have provided insight and knowledge to others with PXE.
Terry: Were there any surprises?
Chris: The biggest surprise for me was the annual appointment in January 2014 where I was expecting the same dosage and received instructions to take 2500 milligrams (mg) per day. This dosage made me feel fairly sick in my first several days and I had it reduced to 2000 mg per day. Knowing that there have been no negative impacts for anybody on a high concentration means that the envelope has been pushed a little farther and that can provide an even bigger impact than was originally planned.
Terry: What has been the hardest or easiest part of participating in the trial?
Chris: The easiest part of the study has been the great planning and communication with the medical staff at Mt. Sinai. The process has been seamless and I couldn’t be happier with the staff. The hardest part has been, in Pittsburgh, coordinating with my primary care physician to sample blood and urine for an outpatient purpose as well as shipping those samples via FedEx to Mt. Sinai. FedEx wouldn’t give me clear instructions on what to do and occasionally treated me poorly because I needed to ship a biological sample. The actions taken to resolve this were collecting several ClinicalPak packages and air bills from FedEx and scheduling a pick up at my house.
Terry: What was unusual, fun, or strange?
Chris: Though it’s not the most pleasant experience, I’ve found that there is a part of me that really enjoys visiting the ophthalmologist. From having PXE, I’ve learned so much about the eyes and how amazing they really are. I find myself captivated and so open to have a conversation about how eyes work. Dr. Fuchs has been much obliged to answer my questions and discuss with me what pictures would look like should I experience eye bleeds, etc. I found myself asking more curious questions about how PXE works and how the body works and responds to PXE as opposed to being a patient and having only the concern of, “what’s going to happen to me?” Though that is still the central question, I was surprised that I became so interested in the science behind PXE as well as the research taking place.
Terry: is it hard to be in a double blind, randomized trial, meaning that you don't know if you have taken the drug or the placebo in the first year?
Chris: I didn’t find it difficult and still don’t know if I was on a placebo during the first year. My biggest concern was monitoring how I felt. Knowing that 50% of the participants were on the magnesium oxide and knowing that every participant would be on it during year two eased the anxiety for me behind what was being taken. My neck was always the most impacted area as far as aesthetic symptoms of PXE. Since January of 2013, I’ve been monitoring my neck to see if there are changes. I’ve taken pictures and tried to analyze them myself.
Terry: Overall, how would you describe your experience participating in PXE research?
Chris: Participating in the PXE study is a rewarding experience. In addition to learning so much about PXE, it’s been great to get to know people participating in the study as well as the medical research team. Lastly, being a participant is a rewarding experience because I get to do my part in progressing the knowledge of PXE in working toward an effective treatment. Knowing that so many people can benefit from this is the most rewarding part of this experience.
Terry: Would participate in another clinical trial in the future, and would you recommend participating to someone else?
Chris: I’d likely participate in another trial. I have my limits regarding the medication I would try. Magnesium oxide is a relatively low-risk supplement that isn’t complex in chemical nature. I’m not sure how I’d feel about a complex drug that could have some serious side effects. Yes, I would recommend to others to participate in a clinical trial. I would provide them with information regarding my personal experience and give them some expectations and advice to ensure they feel comfortable in participation.
Terry: Chris, thanks for taking the time to share your experience with the PXE community!
Chris: My pleasure!