Lasker prize for medication that arrests cystic fibrosis
Who are the scientists who determined biomedical cause and engineered a therapy?
The Lasker Foundation announced that three scientists are recipients of the 2025 Lasker~DeBakey Clinical Medical Research Award for their work on TRIKAFTA, a drug that changed the course of medical care for individuals with cystic fibrosis and the lives of thousands of children and their families. The three men are Michael J. Welsh, MD., of the University of Iowa; and Jesús González Ph.D., and Paul A. Negulescu, Ph.D., of Vertex Pharmaceuticals.
People who have cystic fibrosis have excessive mucus in their lungs and disordeed enzymes that block the function of the liver or pancreas (or both). Historically, treatment has included “percussion” of the persons upper body.1 The disease develops in children and usually become so severe that most people who have it die before reaching adulthood.
Sportswriter Frank Defor wrote a moving revelation about his daughter, Alexandra Deford. In Alex: The Life Of A Child2 he chronicled his daughter’s case, treatment (including percussion), brief life (she lived to be eight year old). The insights into families coping with a child’s debilitating medical situation can help people to understand the demands that disability puts on a family. The book was turned into a TV movie.
Special education
Children with cystic fibrous may or may not require special education. Cystic fibrosis does not interfere directly with the learning. However, the medical difficulties may lead to other problems that require accommodations. For example, children with cystic fibrosis may be hospitalized for extended periods of time, requiring a hospital education program. One can find recommendations in some academic sources. Gathercole (2019) described how parents, nurses, and teachers in England sometimes found educational activities and treatment regimens incompatible. Strawhacker and Wellendorf (2004) provided general guidance aimed at an audience of school and clinic nurses.
The Cystic Fibrosis Foundation’s Web site is worth reviewing to learn more about the condition. It includes, for example “A Teacher’s Guide to Cystic Fibrosis” (including a subsection on IEPs).
Although the story about the development of TRIKAFTA may not be immediately relevant for special educators and the families of children with disabilities, I think there is an important connection to this award and our business. To me, the award for a momentous accomplish scientific accomplishment is a cause for celebration. If these researchers can study a problem and develop a powerful solution, that augurs well for many other problems that may seem intractable. Cystic fibrosis is a genetic disorder, and that may make it seem like it’s not going to be overcome, but here we have evidence that scientistic can find ways to counter the effects of those genes. This is a marker of hope for me.
How the therapy was developed
Unlike some other genetic disorders, cystic fibrosis has only been known for a relatively brief time. Over many years, Drs. Welsh, González, and Negulescu studied the cell mechanisms that result from defects or missing proteins caused by mutations in the CFTR gene. They found that the proteins were disrupting cells’ functioning. What is the CFTR protein and what does it do?
Michael Welsh explained in an article published 11 September 2025 in JAMA Network:
The overriding question was “What does CFTR do?” CFTR is a member of the adenosine triphosphate–binding cassette transporter family, which were all pumps. Speculation abounded. Is CFTR a pump? What does it pump? Does it transport a regulatory factor controlling chloride channels?
We posed the simplest hypothesis: CFTR is a chloride channel. If correct, amino acids lining the channel pore determine its ion selectivity. Guessing at CFTR’s structure, we altered charged amino acids lining the predicted pore.3 CFTR’s anion selectivity sequence is bromide > chloride > iodide > fluoride. Switching 2 positively charged lysines to negative amino acids converted the sequence to iodide > bromide > chloride > fluoride. I remember the thrill of discovery when Matt Anderson came running out of the patch-clamp room yelling “I can’t believe it worked.” The results indicated that CFTR is an anion channel. The next year, Christine Bear, John Riordan, and colleagues reached the same conclusion using biochemical reconstitution. This discovery linked the genetics to CF physiology and provided opportunities to better understand the disease. It also identified ion channel function as a target when hunting for therapeutics.
More about the award recipients:
Michael Welsh is a professor of internal medicine at the University of Iowa, where he holds an endowed chair and directs a biomedical institute. He obtained a bachelors degree in science and a medical degree (both from the University of Iwoa). He completed post-doctoral work at the Universitiy of San Fransisco and a felllowship at the University of Houston. He has been on the faculty at Iowa since 1981.
Jesús González is now the chief technology officer for Integrao Theranostics. https://integrotheranostics.com/. He completed undergraduate work in chemistry at Rice University and graduate work in chemistry at Princeton University. He did post-doctoral work at the Howard Hughes Medical Institute before joining Vertex.
Paul Negulescu, who is vice president of Vertex Pharmaceuticals, is a cell biologist. He graduated from the University of California, Berkeley, with degree in history and physiology. He completed a Ph.D. at Berkeley and then joined a company (Aurora Biosciences) that was subsequently acquired by Vetex.
There are Lasker Foundation awards are made in four areas: (a) Albert Lasker Basic Medical Research, (b) Lasker~DeBakey Clinical Medical Research Award; (c) Lasker~Koshland Special Achievement Award in Medical Science, and (d) Lasker~Bloomberg Public Service Award. Learn more about them from the Lasker Foundation https://laskerfoundation.org/awards/about-the-awards/
See other coverage of this award from
Carl Zimmer and Gina Kolata of the New York Times on 11 September 2025: “” Major Medical Prizes Given to Cell Biology and Cystic Fibrosis Pioneers: Many winners of the annual Lasker Awards have gone on to win a Nobel Prize in medicine or other fields.” https://www.nytimes.com/2025/09/11/health/lasker-awards-medicine.html
Andrea Corona of Drug Discovery News on 11 September 2025: “Lasker-DeBakey 2025 honors breakthroughs that transformed cystic fibrosis
A disease once synonymous with a shortened lifespan is now a model for how drug discovery can directly alter the natural history of genetic disorders.” https://www.drugdiscoverynews.com/lasker-debakey-2025-honors-breakthroughs-that-transformed-cystic-fibrosis-16627
US Proceedings of the National Academy of Sciences 11 September 2025: “PNAS Celebrates the 2025 Lasker Awards: PNAS has partnered with the Lasker Foundation to shine a spotlight on this year’s Lasker Award recipients. https://www.pnas.org/post/update/2025-lasker-awards”
Vertex: Vertex Scientists Awarded the 2025 Lasker~DeBakey Clinical Medical Research Award for Pioneering Discoveries in Cystic Fibrosis https://news.vrtx.com/news-releases/news-release-details/vertex-scientists-awarded-2025-laskerdebakey-clinical-medical
References
Gathercole, K. (2019). Managing cystic fibrosis alongside children’s schooling: Family, nurse and teacher perspectives. Journal of Child Health Care, 23(3), 425-436.
Strawhacker, M. T., & Wellendorf, J. (2004). Caring for children with cystic fibrosis: A collaborative clinical and school approach. The Journal of School Nursing, 20(1), 5-15.
Footnotes
In “percussion,” caregivers (often parens) repeatedly hit the child’s chest and back with a cupped hand. The idea is that the action loosens mucus in the lungs, making it easier for the person with cystic fibrosis to expel it and, thereby, breathe more easily.
Viking, the publisher, does not seem to be selling the title. I found copies on the used books site, Abe Books. (I make $0.00 from this referral.)