JAMA Medical News How Did Cystic Fibrosis Go From Fatal to Treatable?
Sep 11, 2025
In this discussion, Michael J. Welsh, a renowned pulmonologist and award-winning researcher at the University of Iowa, shares insights into cystic fibrosis. He recounts his early experiences with patients and the journey that led to the breakthrough drug Trikafta. Listeners will learn how collaborative research transformed cystic fibrosis from a fatal condition to a manageable one. Welsh also highlights future directions for treatment, including gene editing, offering hope for improved patient outcomes.
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Early Patient Encounter That Sparked A Career
- Michael J. Welsh recalls a seven- or eight-year-old girl with CF who couldn't breathe well and smelled of Pseudomonas.
- Her prognosis — not surviving past her teens — deeply motivated his work on CF.
Modulator Therapy Transformed Outcomes
- Trikafta and other modulators have changed CF from progressive decline to actual improvement in many patients.
- University of Iowa saw lung transplants drop by about 75% after modulators became available.
Apical Membrane Defect Unifies CF
- The CFTR defect was pinpointed to the apical membrane of airway epithelial cells, unifying multiple organ manifestations.
- This explained why chloride couldn't exit cells and tied together sweat, lung, and pancreatic disease.