Amanda Boston

Type of Porphyria: 
Variegate Porphyria (VP)

I first saw the opportunity to participate in research studies on the American Porphyria Foundation (APF) Facebook Page.   As soon I as I saw the post I knew I would love to volunteer!  As a young woman in my twenties, I have a vested interest in the race for a cure, and the development of new treatments.  Research studies even help document treatment options that are tried and true in some areas of the country but not others, which is a important in getting insurance companies to consider the treatments normal and standard and therefore a covered expense.  This too is crucial in getting patients treatment.  

I participated in three studies in 2014, the Longitudinal study, the Panhematin Study and the Anylam Study.  The Longitudinal Study required no travel, just some lab work coordinated through my personal physician.  The Panhematin Study involved traveling to the University of Texas Medical Branch (UTMB) in Galveston, TX.   I stayed there five days participated in a double blind study to prove the effectiveness of the IV drug Panhematin.  I already have this treatment available in my area and take it on a routine basis.  However others are not so fortunate and the results of this study will help more people get the treatment prescribed and then covered by insurance.   Then just last week, I went back to UTMB to begin participating in a study for a new drug through Alnylam.   The study involved a physical exam and some lab work, which will be repeated twice more at six months intervals. 

All my experiences at UTMB have been positive.  Last week was no exception.  I traveled that morning and arrived at 11am to meet Dr. Anderson, Dr. Ede, and Dr. Hallberg.  Dr. Anderson is very knowledgeable and friendly and I was at ease instantly.  I met him for the first time during the Panhematin study, and he knew what I needed from the moment I walked in.  He had obviously read my medical records and understood Porphyria and understood the symptoms I was experiencing.   He was quick to give me a Panhematin treatment at the onset of an attack and never put my health in jeopardy.  This time I had a simple exam where they took my blood pressure, pulse, temperature, weight and height and then I gave a blood and urine sample.  I flew home the next day, so it was a very small time commitment.

After the research was completed, I met with Desiree Lyon.  She is one of the nicest people I have ever met.  When she speaks about the foundation and research projects, you can truly feel the dedication and commitment.  I admire her strength to cope with Acute Intermittent Porphyria herself while working so hard to help others.    I met some other staff members as well who were friendly and took me out for dinner and made me feel at home and welcome.  The next morning I had asked if I could see more of the work they do at the foundation, and I had the privilege of observing all morning until my flight that afternoon.  I was impressed by the dedication and compassion of the staff!

I know that many people may hesitate to participate in research studies.  I had reservations at first myself, and my fears were quickly put to ease.  The time commitments have been small and the process has been all positive.  I hope this article encourages you to become more involved and participate in finding a cure for Porphyria.