Two weeks ago, I watched a livestream of the North American Cystic Fibrosis Conference (NACFC) as I popped Trikafta (elexacaftor/tezacaftor/ivacaftor) tablets out of their blister pack. Francis Collins, one of the researchers who discovered the cystic fibrosis transmembrane conductance regulator (CFTR) gene, was singing to an audience of thousands. I teared up when he sang, “Dare to dream till the story of CF is history.”
It was a poignant moment. A researcher who dedicated his life to cystic fibrosis sang about persisting until we find a cure as I took a medication to slow the progression of my disease.
Francis didn’t just reinforce the solidarity of the researchers, nurse coordinators, social workers, and doctors who work each day to improve our lives. He ignited the room through empathy. His serenade began with a song from “Five Feet Apart” that emphasizes the perspective of people who face the unknown every day: “I’m not givin’ up, … even when I’m down to my last breath, so don’t give up on me.”
That is our community’s plea.
This was my first time at NACFC, thanks to the U.S. Adult Cystic Fibrosis Association. My biggest takeaway from the conference is that we are a team. We aren’t stopping just because we have treatments available to us.
You may be wondering why I was watching the livestream. I realized that in order to conserve energy and optimize the hours between my daily breathing treatments, I couldn’t attend every session. Instead, I spent a good amount of time in the exhibitor hall, chatting with people I had only interacted with through email. I was astounded by the incredible science on display and was especially fascinated by the living, breathing pig lungs that were inhaling air through a positive expiratory pressure device.
Despite the emphasis that has been placed on modulator therapies like Trikafta, patients still need anti-infectives. Bacteria won’t just disappear from our lungs after decades of colonization. We also need regenerative medicine because many of us have underlying damage that modulators cannot treat. I was concerned that modulators might overshadow the importance of other research areas, but I was pleasantly surprised.
I saw hundreds of research posters on airway clearance devices, novel molecules as anti-infectives, and quorum sensing to eliminate bacterial communication, to name a few. I also met a researcher who knew me as patient “EB.” James Gurney heard of my case earlier this year when I was treated with the bacteriophage he was presenting at NACFC. The researcher-patient connection came full circle, and we were both amazed.
I stay up to date with research, but the results of a stem cell therapy in Phase 1 patient clinical trials gave me even more hope! I heard trial data on RNA therapy. Organoids are being utilized in theratyping procedures to deliver personalized medicine. Companies I’ve never heard of are trying to “outdo the success of Trikafta” by researching new modulators. I also learned that in multiple labs around the world, gene editing has been successful in fixing the CFTR gene!
Future treatments are fueled by the CF Foundation’s “Path to a Cure.” The plan is to provide modulators for everyone, treat comorbidities like infections and cystic fibrosis-related diabetes, optimize medical systems, facilitate mutual learning between doctors and patients, and ultimately find a cure. People are excited to open doors for patients. Medical professionals are starting to recognize our value in facilitating the path toward care partnerships.
I encourage patients and caregivers to watch the NACFC sessions that are available online. More than ever, I encourage people with CF to be active and open to participating in new clinical trials. Be inquisitive, be bold in trying new treatments, and share your health journey with researchers and care teams who need your valuable input.
Without the opportunity to attend NACFC, I wouldn’t have known that stem cell trials are happening in the CF community! I hope to attend NACFC again and remain an active part of the research community.
Note: Cystic Fibrosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Cystic Fibrosis News Today, or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to cystic fibrosis.