NACFC 2025| 6 Years of Highly Effective Modulator Therapy & Beyond: Reflecting Back, Looking Forward

Описание: While CFTR modulators were a significant breakthrough, gaps in understanding and challenges remain.

During plenary 1 of the 2025 North American Cystic Fibrosis Conference, Claire Keating, MD, from Columbia University Irving Medical Center, and Marcus Mall, MD, from Universitätsmedizin Berlin, examine the transformative impact of CFTR modulators as a form of precision medicine in the treatment of cystic fibrosis. The speakers reflect on key understandings from the development and clinical use of modulators, highlighting anticipated benefits, expansion of use, and unanticipated outcomes. Next-generation therapeutic approaches, including ion transport therapies that work beyond CFTR, are also discussed.

Livestreamed from Seattle Oct. 23, 2025.

0:00:00 | 6 Years of Highly Effective Modulator Therapy & Beyond: Reflecting Back, Looking Forward
00:01 | Michael Boyle, MD, welcomes the audience to Plenary 1
06:54 | KC White, shares a story of progress from the CF Foundation Therapeutics Lab
12:35 | Steven Rowe, MD, introduces Plenary 1
22:45 | Claire Keating, MD, discusses the impact of CFTR modulators on cystic fibrosis
23:59 | How have CFTR modulators affected overall lung function in people with CF?
25:38 | Use of highly effective modulators is increasing in the U.S.
26:48 | VIDEO: Sydney, an adult with CF, shares her experience with CFTR modulator therapy
30:44 | For most people with CF, modulators continue to be safe and effective
32:15 | How do modulators affect other measures of lung health, such as Lung Clearance Index (LCI), mucus plugging, and infection?
37:03 | How do modulators affect the pancreas, including CF-related diabetes (CFRD) and exocrine pancreatic function?
40:04 | How do modulators affect the digestive system, including gastrointestinal symptoms and liver disease?
43:15 | Can modulators affect mental health outcomes?
45:09 | What do we know about drug dosing and exposure?
47:14 | What do we know about modulators during pregnancy?
51:55 | Ongoing challenges and unmet needs
53:29 | Marcus A. Mall, MD, discusses new boundaries researchers are pushing to restore CFTR function
53:58 | Expansion of modulators to non-F508del mutations
58:34 | How do modulators restore CFTR function to different types of mutations?
1:00:23 | Next generation modulators may restore CFTR function to normal levels
1:06:54 | How could readthrough drugs restore CFTR function in nonsense (class I) mutations?
1:10:03 | What strategies are we pursuing to improve mucociliary clearance and reduce inflammation?
1:16:55 | Conclusion: What we know about modulator therapy and where future research is going