A groundbreaking therapy that has transformed the outlook for people with cystic fibrosis (CF) has earned three scientists the 2024 Lasker-DeBakey Clinical Medical Research Award, often called the “American Nobel.”
The $250,000 prize was awarded to Dr. Michael Welsh of the University of Iowa, Paul Negulescu of Vertex Pharmaceuticals, and Jesús (Tito) González of Integro Theranostics for their pivotal roles in developing Trikafta, a drug that dramatically extends the lives of people with CF.
From Fatal Childhood Disease to Near-Normal Lifespan
Cystic fibrosis is a genetic disorder caused by mutations in the CFTR gene, which normally helps regulate the movement of salt and water in and out of cells. Defects in the gene lead to thick, sticky mucus that clogs the lungs and digestive tract, causing severe infections and organ damage.
When CF was first identified in the 1930s, most children with the disease died before reaching school age. Even a decade ago, half of patients did not live past 40. But with the introduction of Trikafta in 2019, life expectancy has shifted dramatically.
“Today, the estimated median survival for children born with cystic fibrosis between 2020 and 2024 is 65 years — a figure expected to rise as more patients begin treatment earlier in life,” wrote Dr. Eric Sorscher of Emory University in The New England Journal of Medicine.
How Trikafta Works
Trikafta combines three drugs that target the underlying molecular defect in CF. By helping the faulty protein reach the cell surface and function properly, the treatment restores the flow of salt and water through tissues, easing mucus buildup.
This breakthrough approach builds on decades of work:
Michael Welsh studied the effects of the most common CF mutation (delta-F508) and showed how the defective protein misbehaves inside cells.
Jesús González, as a postdoctoral researcher, co-developed a system to track how ions move across cell membranes — a tool that later accelerated drug discovery.
Paul Negulescu led the massive screening effort at Vertex Pharmaceuticals to identify and refine compounds that could fix the malfunctioning protein.
Their work led to a series of earlier CF drugs before culminating in Trikafta.
A Lifesaving Impact
Since its approval, Trikafta has become widely used, reducing hospitalizations, cutting the need for lung transplants, and allowing many people with CF to plan for futures once thought impossible.
“It’s very rare in medical research to see something make it all the way to patients with such dramatic effects,” said González. “This award is incredibly gratifying.”
Honoring Scientific Achievement
The Lasker Awards, established in 1945, are among the most respected prizes in medicine and often precede a Nobel Prize. Two other 2024 Lasker Awards were also announced:
Basic Medical Research Award: To Dirk Görlich (Germany) and Steven McKnight (U.S.) for discovering how proteins organize cells and how disruptions contribute to disease.
Special Achievement Award: To Lucy Shapiro (Stanford University) for her five-decade career advancing microbiology and global health policy.
As for the CF award, the impact is clear: what was once a childhood killer has become a manageable condition for most patients.
“Welsh, González, and Negulescu’s achievements are affording people with CF the chance to thrive now and to plan vibrant futures,” the Lasker Foundation said in its announcement.
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