Five Reasons to Join Research in the Post-Trikafta Era
Last updated: August 2022
In the fall of 2019 something amazing happened to the CF community: the newest CFTR modulator, Trikafta, became available. Many lives were changed and the CF community celebrated (rightly so) and took a big breath of relief, both physically and metaphorically.
For many individuals after Trikafta, CF research was not on the forefront of their minds due to the new stability in health they were experiencing from the latest medication discovery. The sense of urgency towards researching bigger and better treatments was dulled a bit. Not to mention, the start of the pandemic limited many research opportunities to people in the CF community.
Despite all the changes and challenges in the last few years, CF research is more important than ever as we inch closer and closer to a cure. Here’s my five top reasons to join CF research in the post-Trikafta era.
1. Modulators are not for everyone
Trikafta has improved the quality of life and wellbeing of many people in the CF community. Unfortunately, the reality is CFTR modulators are not available or tolerated by everyone. In order to benefit from Trikafta, you must have certain mutations leaving 5-10% of the CF population without access. In addition, some individuals have experienced adverse side effects with Trikafta which makes it dangerous to take. That’s why it’s so very important to join a clinical trial especially if you are someone with a rare mutation to help increase access to high effective treatments.1
2. Help expand medication options
While the uptick in medication options to treat CF over the last decade has been fantastic, there’s still limited options when it comes to certain medications. For example, all current pancreatic enzymes used to help digest and absorb nutrients in food are derived from animal products, specifically pig pancreas. Currently, an exciting clinical trial is ongoing that is testing a pancreatic enzyme that is not derived from pigs.2 A great option for our vegan friends and others!
3. Observational studies are always an option
One myth about clinical trials is that your only purpose is as a guinea pig to test experimental drugs. Many people are concerned about the risk or side effects from study medications, but what they don’t realize is that there are observational studies in CF research as well. In observational studies, measurements are taken of different factors such as treatments and medication use, health outcomes, lifestyle variables, etc. without an introduction of an experimental medication. For example, an observational study on pregnancy in CF called MAYFLOWERS is currently enrolling and the data collected will be an important first for many women with CF.3
4. Receive top notch care
Undoubtedly, participating in clinical trials benefits the entire CF community, but it also can be a benefit to your own care. As a participant in a clinical trial, monitoring your health status and wellbeing is a very detailed and precise process. Lots of information is recorded to get a whole picture of your wellbeing. This type of top notch care can help catch changes like sickness early, keeping you healthy in the long run.
5. Different paths to a cure
Many people assume CFTR modulators are the only way to move forward towards a cure, when in fact there may be many paths to cure CF. The different ways the CFF is moving towards a cure include:4
- Repair CFTR function
- Restore CFTR function
- Fix CFTR gene
Each method has its own set of experimental treatments to achieve the goal of a cure along the way with many steps of research that require participation. This is an area of study that is so worth the people's time and attention because only through clinical trials will we inch our way to a cure.
If you're interested in learning more about clinical trials and research in the CF community, try this tool.
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