Every year, researchers and clinicians gather to share the latest information on cystic fibrosis research and care at the North American Cystic Fibrosis Conference (NACFC). This year’s conference was held in Boston from Sept. 26–28.
The three plenaries are the highlight of the conference, and this year they covered the latest research in genetic therapies, cystic fibrosis-related diabetes (CFRD), and reproductive health. We asked three adults with CF to offer their key takeaways from each plenary.
Plenary 1: Challenges and Progress in the Pursuit of Genetic Therapies for CF
“We have work to do, but the time is right to pave the clinical development path for genetic medicines.” This quote from Daniel Siegwart, PhD, who spoke during the first plenary session, perhaps best summarizes the current outlook on CF clinical research and the benefits this research has for the CF community.
The main point from this plenary is that challenges remain with genetic therapies, however with challenges come progress. Progress is achieved through the pursuit of various gene therapies that target those with CF who do not qualify for a modulator. Progress is being made through the Cystic Fibrosis Foundation’s relentless pursuit of removing critical barriers to effective gene therapies for cystic fibrosis. These gene therapies are now being tested in people with CF through innovations in clinical trial design.
As always, the highest priority for the CF community is that all people with CF have access to clinical care and to research studies. As someone who is not eligible to take a modulator therapy, I urge those in the CF community to continue to pursue clinical research trials that best apply to you by searching the Clinical Trial Finder. By participating in clinical research trials, you are helping the Foundation, research scientists, and other people affected by CF overcome challenges.
— Schyler Kline, adult with CF
Plenary 2: The Tipping Point: The Journey to Understand and Advance Research in CFRD
This plenary focused on the prevalence of CFRD, the impact CF has on the pancreas, and current studies on how to advance the diagnosis, management, and treatment of CFRD.
I was diagnosed with CFRD in 2006, when I was 23 years old. I had been experiencing symptoms of CFRD earlier than that, which is just one reason why I found plenary 2 so compelling. CFRD affects one quarter of adolescents and half of adults with CF. Rebecca Hull-Meichle, PhD, discussed the study of the CF pancreas under the microscope, describing what happens at the cellular level; while Melissa Putman, MD, dove into CFRD on the clinical side, which has its own unique challenges.
I found it particularly interesting to learn that in 2023, only 30% of the CF population took the Oral Glucose Tolerance Test, recommended to be completed annually for those with CF ages 10 years and older. This test, used to diagnose diabetes, can be burdensome for people with CF to take and it’s not always straightforward in diagnosing CFRD. However, early detection is crucial as changes in the function of the pancreas often occur early on in life.
Insulin is the only treatment available for CFRD. Continuous glucose monitors and insulin pumps can help with managing CFRD. People with CFRD who take CFTR modulator drugs, such as Trikafta®, can have a small but significant improvement in A1C — a measurement of the average amount of glucose (sugar) in their blood over a three-month period — but their CFRD does not go away. With the increase in use of modulators and longer lifespans, there are rising rates of obesity and pregnancies in CF, which present other obstacles in managing CFRD.
In this plenary, they discuss how vital it is to have treatment plans tailored to each individual with CFRD. Exciting, up-and-coming technologies are discussed, which I, for one, count on for ease of treatment of CFRD in the future.”
— Molly Baker, adult with CF
Plenary 3: Reproductive Health in CF
During the third plenary, Molly Pam, an adult with CF, gave us a glimpse into her life. She and her husband struggled with deciding whether they should have kids. Molly discussed that she was able voice these concerns during a CF focus group called CFReSHC (CF Reproductive and Sexual Health Collaborative). This collaborative has made it clear that CF impacts more than just the lungs; it affects reproductive health as well. I was very engaged in this plenary session because it took my husband and me a while to conceive our daughter, who also has CF.
Raksha Jain, MD, discussed studies involving fertility and pregnancy with people with CF. She also talked about the MAYFLOWERS study, which showcases maternal and fetal outcomes in the era of modulators. I related to Dr. Jain’s presentation the most because I loved seeing the research, studies, statistics, and stories she shared.
Traci Kazmerski, MD, emphasized that the CF community wants — and needs — to discuss reproductive health with their CF care teams. This is a great perspective because before I became pregnant with my daughter, my care team helped me with the decision-making about having a child after I told them that I didn’t know what having kids would look like for me. Routine sexual and reproductive health discussions with care teams — beyond just having children — would be beneficial for adults living with CF.
— Madison Patterson, adult with CF
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