Note: I ran out of time and energy this week to write anything specifically for writer's, so I found a couple little write-ups I did for my Biotech course a week or so ago. They're basically discussion posts, so not formal essays or anything. But, I still got a 10/10 on both. 

Clinical Trials

Growing up- and also as an adult- I’ve participated as the guinea pig in numerous clinical trials in pursuit of effective medications and treatments for Cystic Fibrosis. Clinical trials exist to do just that: explore what medications do and do not work for every known illness out there, from symptoms associated with allergies, to something as severe as a rare, genetic disease. 

While there are numerous clinical trials going on all over the world at any given point, they’re usually very selective about who can and cannot participate. For instance, I may have Cystic Fibrosis caused by two delta-f508 mutations, but I didn’t qualify to participate in stage three clinical trials for Trikafta, because my lung function was too high. While that’s a “good” problem to have, it also wasn’t, because CF is more than just a lung condition. However, clinical trials have to operate within very specific parameters. In the case of Trikafta, scientists were using lung function as the dependent variable in the trial, because that’s the most dangerous part of Cystic Fibrosis. 

To conduct the Trikafta trials, doctors gave roughly half of the patients a placebo in place of the real drug, and the other half the actual drug. That way, they had a control group to compare the actual results of Trikafta. The Trikafta study was also a randomized, double-blind study, meaning that the patients who got Trikafta vs the placebo were randomly selected, such that not even the doctors knew whether or not they were giving the patient Trikafta. 

Like all medications, Trikafta went through all four stages of a typical clinical trial.

Stage one included only the sickest patients with CF that had at least one delta-f508 mutation, with the hope that their lung functions would improve just marginally from Trikafta. Stage two expanded slightly to more patients, but I still wasn’t qualified due to how healthy I was compared to most other people with CF. Then, Trikafta reached stage three of clinical trials, and I tried to participate, but again, my lung function was too high. 

Then, in October of 2019, the FDA unexpectedly approved Trikafta while it was in the middle of stage three clinical trials, because it was ridiculously effective. This meant that I could finally participate in stage four of the Trikafta trials. Except, I still had to wait my turn while manufacturing ramped up. For Christmas of that year, I finally got my first box of Trikafta, which changed my life (for the best). 

Today, Trikafta is still technically in stage four of the clinical trial. My doctors continue to closely monitor and track me. While I did notice a substantial increase in anxiety as a result of Trikafta, I was put on medication which quickly reduced my anxiety back to its normal levels (normal for me, that is). I also still have to get my blood drawn every 6 months to make sure that my liver doesn’t suffer from Trikafta too much, because Trikafta (and other medications like it) can cause liver damage. That also means that I don’t drink, because I don’t want to play stupid games and win stupid prizes. 

Like all medications, prior to going through the trial in the first place, it had to be approved by the hospital’s Institutional Review Board for ethical reasons. From there, patients participating in the trial (including myself, even after the FDA approved Trikafta) had to sign some paperwork to legally inform the institution that they understood, fully, what they were getting into, and were willing to give Trikafta a shot anyway, despite the potential risks involved. 

To protect both patients and institutions, numerous third-party regulatory agencies monitor clinical trails to make sure all ethical and clinical standards are upheld [1]. That’s really the only thing I don’t know much about, because I’ve never been involved in the regulatory side of clinical trials. 

Reference:

1. National Institutes of Health. (2024, June 10). Learn About Studies. Clinicaltrials.gov. https://clinicaltrials.gov/study-basics/learn-about-studies.