Vertex Scientists Win Prestigious Lasker Award for Revolutionary Cystic Fibrosis Treatment
核心洞察
Three scientists, including Vertex's Paul Negulescu, received the 2025 Lasker-DeBakey Clinical Medical Research Award for developing TRIKAFTA, a life-saving triple-drug combination therapy for cystic fibrosis (搜索).
The breakthrough treatment extends lifespans of CF (搜索) patients by decades, with those starting treatment in childhood expected to live near-average lifespans compared to a median survival age of 40 before 2019.
TRIKAFTA addresses the underlying molecular cause of CF (搜索) by combining three drugs that help restore proper ion transport in cells, fundamentally changing how this genetic disease is treated.
Vertex Pharmaceuticals Senior Vice President Paul Negulescu has been awarded the prestigious 2025 Lasker-DeBakey Clinical Medical Research Award alongside two colleagues for their groundbreaking work developing TRIKAFTA, a revolutionary treatment that has transformed the outlook for patients with cystic fibrosis (搜索) (CF (搜索)). The award recognizes their role "in the development of a novel, life-saving treatment for cystic fibrosis — namely, a triple-drug combination therapy, TRIKAFTA, that has helped countless people with this genetic disease."
Negulescu shares the $250,000 prize with Jesús (Tito) González, a former Vertex scientist now at Integro Theranostics (搜索), and Michael Welsh, Professor of Internal Medicine-Pulmonary, Critical Care and Occupational Medicine at the University of Iowa. The Lasker Awards, established in 1945 and often called the "American Nobels," are widely regarded as America's preeminent biomedical research prize.
Dramatic Impact on Patient Survival
The treatment has achieved remarkable clinical outcomes, extending the lifespans of people with CF (搜索) by decades. Patients who start treatment in childhood or adolescence are now expected to live near-average lifespans, a dramatic improvement from historical outcomes. When CF was first discovered in the 1930s, most patients died in early childhood. Even in the 2010s, before TRIKAFTA's approval in 2019, about half of CF patients died before age 40.
"Today, the estimated median age of survival for persons with cystic fibrosis (搜索) who were born between 2020 and 2024 and have access to treatment is 65 years," according to Dr. Eric Sorscher of Emory University. "Available projections suggest that health and longevity may increase further as modulators begin to be administered at younger ages."
Scientific Foundation and Development
The award recognizes several major scientific inflection points in CF (搜索) research, from understanding the cellular mechanisms that cause the disease to developing the first FDA-approved medicines that treat its underlying cause. Welsh's laboratory research in the 1980s and 1990s laid crucial groundwork by unraveling the physiological consequences of the most common genetic error in CF patients.
Working with cells from the airways of people with CF (搜索), Welsh and colleagues demonstrated that the delta-f508 mutation in the CFTR (搜索) gene prevents charged particles from passing through cellular membrane tubes. Their experiments showed that these tubes never reach the cell surface, causing ions to become trapped.
González co-invented a revolutionary screening system that could accurately track ion flow across cell membranes in real time. "This allowed us to screen tens of thousands of compounds a day," González explained, compared to traditional electrophysiology methods that could only test "a handful a day."
Negulescu led the molecular screening project at Aurora Biosciences (搜索) (later acquired by Vertex), searching for "potentiators" that boost ion flow and "correctors" that help position the cellular tubes correctly. This systematic approach led to the approval of several CF (搜索) drug iterations in 2012, 2015, and 2018, culminating in TRIKAFTA's approval in 2019.
Global Treatment Impact
TRIKAFTA combines three drugs to effectively treat most people with CF (搜索), addressing the underlying molecular cause of the disease rather than just managing symptoms. The treatment has reduced the number of lung transplants and hospitalizations for infection among CF patients while significantly improving their quality of life.
Vertex's CF (搜索) medicines now treat over 75,000 people with CF in more than 60 countries across six continents, representing approximately two-thirds of diagnosed CF patients eligible for CFTR (搜索) modulator therapy. The company has brought five transformational medicines to CF patients, fundamentally changing how this disease is treated.
Understanding Cystic Fibrosis
CF (搜索) is a rare, life-shortening genetic disease affecting more than 109,000 people globally, including 94,000 in North America, Europe, and Australia. The progressive, multi-organ disease affects the lungs, liver, pancreas, gastrointestinal tract, sinuses, sweat glands, and reproductive tract.
The disease is caused by defective or missing CFTR protein (搜索) resulting from mutations in the CFTR (搜索) gene. Children must inherit two defective CFTR genes — one from each parent — to develop CF (搜索). The vast majority of CF patients have at least one F508del (搜索) mutation, which leads to poor salt and water flow in and out of cells across multiple organs.
In the lungs, this dysfunction causes abnormally thick, sticky mucus buildup, leading to chronic infections and progressive lung damage. The median age of death historically was in the 30s, though projected survival continues to improve with treatment advances.
Recognition of Collaborative Achievement
"I'm honored to represent my co-leaders on the CF (搜索) program, Fred Van Goor and Sabine Hadida, and the more than a thousand people at Vertex who have worked over two decades to make the impossible possible," said Negulescu. "Together, we've brought five medicines including TRIKAFTA and now ALYFTREK to tens of thousands of people with CF around the world — medicines that have fundamentally changed the course of this disease."
The Lasker Foundation has awarded more than 400 prizes since 1945, recognizing leaders who have made major advances in understanding, diagnosing, treating, curing, and preventing human disease. Notably, 101 Lasker Laureates have also received the Nobel Prize, including 13 since 2018, underscoring the significance of this recognition for the CF (搜索) research team's transformative work.