Tuesday, July 7, 2015

Medical Trials Without the Tribulations

     I have been fortunate enough to qualify for a variety of Cystic Fibrosis medical studies over the years.  All of them were done through the University of Michigan, and though not all of them went smoothly, they were a fascinating window into how new medicine is approved and the level of commitment that is needed to make strides in finding new and better ways to treat CF.

     To clarify, even though not every trial I was involved with went according to Hoyle, that was always an issue on my end, not the trial or the people running it.  Twice when I had started a study and officially qualified after the "screening" visit, I experienced a health exacerbation which prevented me from continuing.  I also had two that I was interested in but was unable to do.  The first one I was about to sign-on for, but before the screening visit I could tell I wasn't feeling great and ended up going on IV Tobrimyacin instead of doing the inhaled Levoquin trial.  The other was for one of the trials for Orkambi, which was just approved last week, but my FEV1 was at 27%, well below the 40% necessary to take part in the study.  

     There was another study I participated in which I had to end early, but it was unlike any other I'd ever been informed of.  This study wanted to take a close look at the mucus that CF people produce each day.  Since many people with CF cough up this mucus and dispose of it multiple times a day, some researchers decided they wanted to take advantage of all the information this "wasted" mucus could contain.  Specifically, they hoped to be able to identify patterns and perhaps even indicators of activity that precedes a person's pulmonary exacerbation.  What if there were signs that a CF person was on his or her way toward a downward slope, and that information was just being thrown away?  I agreed to take part, and unlike all of the other studies I'd taken part in, this time it didn't matter if I went on IVs or had a specific FEV1 percentage.  I just had to spit my mucus into a plastic flask twice a day.

     When I say "just," that's what it seemed like when I signed on.  In reality, I had to record the time, date, and other bits of info so that the two people in charge of the study would have as much data as possible to use as they looked at the "big picture" of what goes on inside the lungs of individuals with CF.  This was a unique study being done in only one or two hospitals in the nation, and I really wanted to contribute to it.  It had no financial compensation, but this was such a special study that it didn't mater to me that I wasn't being paid for the time I spent on helping with it.  I spit into plastic flasks, stored them in my refrigerator, and either dropped them off or prepared them for pick-up every 28 days continuously for nine months.  They wanted to get a full year's worth of samples, but there came a point where it was too much for me to juggle and I wasn't doing it reliably.  I felt bad about that, but the co-lead of the research team and I emailed back and forth and agreed that what I had provided was really useful, and if I'd reached the point where this was no longer and easy blip in my day, I should stop.

     So, since all I've discussed so far in a post that is allegedly about how great it is to be in medical trials are things that aren't so great, I will reiterate that they are, indeed, a wonderful thing to take part in.  The two trials I was able to do start to finish had several things in common.  The first is that being involved in a study means more trips to the hospital for tests and evaluations, which means a better picture of my overall health.  One of the trials I wasn't able to finish actually helped me realize my FEV numbers were down when I otherwise wouldn't have known that.  Not having to pay for an extra close eye on one's health is always a good thing.

     Speaking of payment, the trial visits were all paid for, and I was compensated for my time in taking part in them.  Depending on the study I was there for a few hours, but the checks I got were, in my opinion, more than enough to make it worth it from that perspective.  But the best part of the studies was knowing my contribution toward the end goal.  In the two I completed, it was to figure out the safety of, and then the efficacy of, inhaled Tobramycin powder.  While I didn't know if I'd gotten a placebo in the safety trial (and I guess I still don't for sure) I felt fabulous during it and was certain that I'd gotten the real deal.  I also don't know which level of dose I was randomly assigned during the efficacy study, but again, I felt really good the whole time.  The only difference between doing the powdered Tobi for the two studies and when it went to market (it was eventually approved and my doctor replaced my nebulizer Tobi with it) was that during the trial, I had to save all of the packaging garbage and even the capsules which had been filled with powder in order to prove I had done all of the medicine to those conducting the research.

     With Orkambi's recent approval, we should all be grateful not just for the scientists and doctors who made it possible, but for the individuals who volunteered to the a part of the studies that were crucial in getting the drug to market.  Like so many things in life, this was a team effort top to bottom, and everyone's hopeful that it Orkambi and the other medicines in the pipeline will repaint the canvass of those impacted by Cystic Fibrosis.


  1. As a human being, thank you for taking part in studies to try to help "people". As a cf Mom thank you, thank you, thank you Evin for participating to try to further research towards a better every day for my son!!!!!
    I am VERY curious how the mucous study ended up? Kodey is not a spitter! Ewwww I know! Never could get him to do it when he was little and he just still can't. BIG gag reflex??????
    Great post!!!!!

  2. I used to have a really hard time coughing stuff up too, but that changed, obviously! :)
    I never heard anything about the results of the study, but it was a very long term and in-depth thing, so perhaps they are still working on it.