Scientists Win Lasker Award for Cystic Fibrosis Breakthrough
- Three scientists - Paul Negulescu of Vertex Pharmaceuticals, Jesús (Tito) González (formerly of Vertex), and michael Welsh of the University of Iowa - have been awarded the 2025...
- Cystic fibrosis is caused by mutations in the CFTR gene, leading to the buildup of thick mucus in the lungs, pancreas, and other organs.
- Michael Welsh's research,beginning in the 1980s,was instrumental in identifying the chloride channel defect caused by the CFTR mutation.
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Lasker Award Honors Scientists for Cystic Fibrosis Breakthroughs
Table of Contents
Published September 11, 2025, at 17:41:58 PDT
The Breakthrough: Revolutionizing Cystic Fibrosis Treatment
Three scientists – Paul Negulescu of Vertex Pharmaceuticals, Jesús (Tito) González (formerly of Vertex), and michael Welsh of the University of Iowa – have been awarded the 2025 Lasker debakey Clinical Medical Research Award for their pivotal work in understanding and treating cystic fibrosis (CF). The $250,000 prize recognizes their contributions to significantly extending the lifespan and improving the quality of life for individuals living with this deadly genetic disease. Lasker Foundation Proclamation
Cystic fibrosis is caused by mutations in the CFTR gene, leading to the buildup of thick mucus in the lungs, pancreas, and other organs. For decades, treatment focused on managing symptoms. The work of Negulescu, González, and Welsh shifted the paradigm towards addressing the underlying genetic defect.
Understanding the CFTR Protein: The Foundation of Treatment
Michael Welsh’s research,beginning in the 1980s,was instrumental in identifying the chloride channel defect caused by the CFTR mutation. He demonstrated that the lack of functional CFTR protein disrupted salt and water balance in the airways, leading to the characteristic mucus buildup. Welsh’s early research on CFTR
Jesús González, while at Vertex, played a crucial role in developing high-throughput screening methods to identify compounds that could correct the defective CFTR protein. This work paved the way for the finding of ivacaftor, the frist CFTR modulator. Vertex Pharmaceuticals History
Paul negulescu led the clinical growth of ivacaftor and subsequent CFTR modulators, demonstrating their efficacy and safety in clinical trials. These modulators don’t cure CF, but they help the defective protein function more effectively, improving lung function and overall health. Ivacaftor clinical Trial Results
Impact and Current Treatments
Before CFTR modulators, the median predicted lifespan for individuals with CF was around 30 years. Today, with access to these therapies, many individuals with CF can live well into their 40s and beyond. The impact is particularly notable for those with specific CFTR mutations that respond well to the available modulators.
Currently,several CFTR modulators are available,including ivacaftor (Kalydeco),lumacaftor/ivacaftor (Orkambi),tezacaftor/ivacaftor (Symdeko),and elexacaftor/tezacaftor/ivacaftor (Trikafta). Trikafta, approved by the FDA in 2019, is effective for approximately 90% of people with CF. Cystic Fibrosis Foundation – Treatments
| CFTR Modulator | FDA Approval Year | Effective For |
|---|---|---|
| Ivacaftor (Kalydeco) | 2012 | Specific CFTR mutations (e.g., G551D) |
| Lumacaftor/Ivacaftor (Orkambi) | 2015 | F508del mutation (in combination with a responding mutation) |
| Tezacaftor/Ivacaftor (Symdeko) | 2018 | F508del mutation (in combination with a responding mutation) |
| Elexacaftor/Tezacaftor/Ivacaftor (Trikafta) | 2019 | Approximately 90
|