Scientists Behind Life-Changing Cystic Fibrosis Treatment That Extends Life By Decades Win Medical ‘Nobel’ Prize

Three scientists whose groundbreaking work redefined the future of cystic fibrosis (CF) care have been awarded one of the world’s most prestigious honors in medicine: the Lasker-DeBakey Clinical Medical Research Award. Often referred to as the “American Nobel,” the award recognizes contributions that radically improve human health. This year, it went to Dr. Michael Welsh of the University of Iowa, Paul Negulescu of Vertex Pharmaceuticals, and Jesús (Tito) González, now of Integro Theranostics.Together, their decades of research led to the creation of Trikafta, a therapy that has extended the lifespan of cystic fibrosis patients by decades and fundamentally reshaped what it means to live with the disease.When cystic fibrosis was first described in the 1930s, it was considered a fatal childhood condition. Patients rarely survived past elementary school. Even as late as the 2010s, before Trikafta’s approval in 2019, half of patients with CF died before the age of 40.Also Read: Jason Collins Brain Tumor: NBA’s First Openly Gay Player, Diagnosed With Brain Tumor — Early Warning Signs You Might MissToday, the outlook is dramatically different. Children born with CF between 2020 and 2024 who have access to Trikafta now face a median survival age of 65 years — nearly indistinguishable from the general population.As Dr. Eric Sorscher of Emory University explained in The New England Journal of Medicine, “Available projections suggest that health and longevity may increase further as modulators begin to be administered at younger ages.”This shift marks one of the most profound turnarounds in modern medicine.The Role of the CFTR GeneCystic fibrosis is caused by mutations in a single gene: CFTR (cystic fibrosis transmembrane conductance regulator). The gene is critical for regulating the movement of ions across cell membranes, which in turn ensures proper water balance in tissues.In healthy cells, CFTR forms channels that allow ions to flow freely. But in CF, the mutated gene produces faulty channels. The result is thick, sticky mucus that clogs the lungs and digestive system, fuels recurrent infections, and damages organs over time.Dr. Michael Welsh, a pulmonologist and molecular biologist, helped illuminate the exact ways the most common CF mutation, delta-F508, disrupts cell function. He discovered two problems: the defective protein often gets “trapped” inside cells before reaching the surface, and even when it does reach the surface, it underperforms.In a pivotal experiment, Welsh showed that simply lowering the temperature of cells allowed the trapped protein to move correctly. “That meant it was not totally broken,” he later recalled — a crucial realization that opened the door to correcting the defect.Meanwhile, as a postdoctoral researcher in Nobel laureate Roger Tsien’s lab, Jesús (Tito) González developed a real-time system to track ion movement across membranes. Initially designed to study neurons, this tool proved invaluable for testing whether new drugs could restore CFTR function.Negulescu and the Push to Find a TherapyAt Vertex Pharmaceuticals, Paul Negulescu helped drive the systematic search for compounds that could repair CFTR defects. Guided by Welsh’s molecular insights and González’s imaging system, the team screened thousands of molecules. The result was Trikafta, a triple-drug therapy that addressed the underlying cause of CF for most patients.'Trikafta' Of Game-Changer in CF TreatmentApproved by the FDA in 2019, Trikafta combines three drugs — elexacaftor, tezacaftor, and ivacaftor — that work synergistically to help defective CFTR proteins fold correctly, reach the cell surface, and function effectively. The impact has been extraordinary. Since its introduction:Hospitalizations for lung infections have plummeted.The need for lung transplants has declined.Patients report stronger lung function, weight gain, and dramatically improved quality of life.For many families, the treatment has meant that milestones once unimaginable — graduating college, starting families, living into retirement — are now within reach.Recognition at the “American Nobels”The Lasker Awards, founded in 1945, celebrate biomedical achievements that shape the future of health. They are considered one of the highest honors in science, often predicting future Nobel Prizes.The recognition of Welsh, González, and Negulescu underscores the profound impact of their work. The $250,000 prize, while symbolic compared to the billions Trikafta has generated, highlights the ethical and humanitarian dimension of their achievement: turning a once uniformly fatal disease into a chronic, manageable condition.What CF Treatment Means for Medicine?The CF breakthrough is not just about one disease. It represents a paradigm shift in genetic medicine. By targeting the root molecular defect rather than simply managing symptoms, Trikafta has become a model for other genetic conditions, from sickle cell disease to rare metabolic disorders.It also illustrates the power of partnerships between academic researchers, biotech innovators, and patient foundations. The Cystic Fibrosis Foundation’s early investments in research were critical to advancing the work that ultimately led to Trikafta’s approval.While Trikafta has transformed care in wealthy countries, challenges remain. The therapy is expensive — with an annual price tag of over $300,000 in the U.S. — putting it out of reach for many patients globally.Furthermore, a subset of CF patients with rare genetic mutations still do not benefit from the drug, leaving an urgent need for alternative therapies. And as with all long-term treatments, researchers must continue monitoring for side effects and resistance.Roughly 100,000 people worldwide live with cystic fibrosis. For decades, their lives were defined by daily medical regimens, frequent hospitalizations, and shortened lifespans. Today, thanks to the pioneering work of Welsh, González, and Negulescu, those same patients are looking toward futures filled with possibility.

Scientists Behind Life-Changing Cystic Fibrosis Treatment That Extends Life By Decades Win Medical ‘Nobel’ Prize

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