Pioneers in Cystic Fibrosis Treatment Awarded the Prestigious Lasker Prize for Innovative Breakthroughs

Scientists Honored for Cystic Fibrosis Breakthroughs

New York, NY – September 11, 2025 – A trio of researchers is being celebrated for their transformative work in battling cystic fibrosis, a genetic disease affecting the lungs and other organs. Paul Negulescu, Jesús (Tito) González, and michael Welsh have been awarded the prestigious Lasker DeBakey Clinical Medical Research Award for dramatically improving the lives of individuals living with this challenging condition.

Revolutionizing Cystic Fibrosis Treatment

The Lasker Award, frequently enough referred to as “America’s Nobel Prize,” recognizes exceptional contributions to medical science. This year’s recipients are acknowledged for fundamentally changing how cystic fibrosis is understood and treated. Their collaborative research has led to therapies that address the underlying cause of the disease in many patients,considerably extending lifespans and improving quality of life.

Paul Negulescu, a Senior Vice President at Vertex Pharmaceuticals, played a pivotal role in the development of these innovative therapies. jesús González, formerly with Vertex, contributed crucial insights during the research phase. Professor Michael Welsh of the University of Iowa provided foundational scientific understanding of the disease mechanisms.

The Impact on Patients

Prior to these advancements, treatment for cystic fibrosis focused largely on managing symptoms. Today,thanks to the work of these scientists,many patients can receive medications that target the defective protein responsible for the disease’s progression. According to the Cystic Fibrosis Foundation, the median predicted survival age for individuals with cystic fibrosis has risen from the late 30s in the 1990s to the early 50s and beyond, with continued improvements anticipated.

“It is not only gratifying and rewarding,” stated Negulescu, “but our hope is that our work can serve as lessons for other research endeavors.” This sentiment underscores the broader implications of their discoveries, potentially paving the way for similar breakthroughs in other genetic diseases.

Understanding Cystic Fibrosis: A Quick Overview

Cystic fibrosis (CF) is a chronic genetic disorder that affects the lungs, pancreas, liver, intestines, sinuses, and sex organs. It’s caused by a defective gene that makes the body produce thick and sticky mucus, leading to various health problems. Approximately 70,000 people worldwide are affected by CF, with over 30,000 in the United States alone.

Key Statistic	Data (2024)
Worldwide CF Cases	Approximately 70,000
US CF Cases	Over 30,000
Median Predicted Survival Age (1990s)	Late 30s
Median Predicted Survival Age (Current)	Early 50s+

Did You Know? While there is currently no cure for cystic fibrosis, ongoing research continues to explore new therapies, including gene editing techniques, which may offer a potential cure in the future.

Pro Tip: Early diagnosis and consistent medical care are crucial for managing cystic fibrosis and maximizing a patient’s quality of life.

The Future of Cystic Fibrosis Research

The advancements honored by the Lasker Award mark a meaningful turning point in the fight against cystic fibrosis. Though, research is far from over. Scientists are actively investigating new ways to address the disease in patients who do not respond to current therapies, as well as exploring potential cures. Gene editing technologies, such as CRISPR, hold immense promise for correcting the defective gene responsible for CF.

Beyond treatment, research focusing on preventative measures and improving the overall well-being of individuals with cystic fibrosis is also critical.This includes studies on nutrition, exercise, and mental health support.

Frequently Asked Questions about Cystic Fibrosis

• what is cystic fibrosis? Cystic fibrosis is a genetic disorder affecting the lungs, pancreas, and other organs, causing thick mucus buildup.
• What are the symptoms of cystic fibrosis? Common symptoms include persistent coughing, wheezing, frequent lung infections, and difficulty digesting food.
• Is cystic fibrosis curable? Currently, there is no cure for cystic fibrosis, but treatments have significantly improved quality of life and increased lifespan.
• How is cystic fibrosis diagnosed? Diagnosis typically involves a sweat test, which measures the amount of salt in sweat, and genetic testing.
• What is the role of gene therapy in cystic fibrosis? Gene therapy aims to correct the defective gene responsible for cystic fibrosis, offering a potential cure.
• What support is available for people living with cystic fibrosis? The Cystic Fibrosis Foundation and numerous other organizations offer resources, support groups, and advocacy for individuals and families affected by CF.

What are your thoughts on the rapid advancements in genetic disease treatment? Do you know anyone impacted by conditions like cystic fibrosis and how have these changed their lives?

What specific impact have CFTR modulators had on the daily lives of cystic fibrosis patients, as evidenced by real-world examples?

The 2025 Lasker Award: Recognizing Decades of Cystic Fibrosis Research

On September 11, 2025, the Lasker Awards – often hailed as “America’s Nobel” – were presented, with a notable portion of the recognition going to researchers who have revolutionized the treatment of cystic fibrosis (CF). this year’s prize acknowledges the decades-long journey to understand and combat this life-limiting genetic disorder, specifically highlighting the advancement of CFTR modulators. These groundbreaking therapies represent a paradigm shift in how we approach cystic fibrosis management.

Understanding Cystic fibrosis: A Brief Overview

Cystic fibrosis is a progressive, genetic disease that affects the lungs, pancreas, liver, intestines, sinuses, and sex organs. It’s caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.This gene provides instructions for making a protein that controls the movement of salt and water in and out of cells. When the CFTR protein isn’t functioning correctly, it leads to the buildup of thick, sticky mucus, causing a range of debilitating symptoms.

Key characteristics of CF include:

* Persistent coughing,sometimes with phlegm

* Frequent lung infections

* Wheezing and shortness of breath

* Poor growth or weight gain despite a normal appetite

* Salty-tasting skin

* Difficulty with digestion and absorption of nutrients

The Journey to CFTR Modulators: A Timeline of Finding

The path to effective cystic fibrosis treatment wasn’t overnight. It involved years of dedicated research, starting with the identification of the CFTR gene in 1989. This discovery opened the door to understanding the underlying cause of the disease and paved the way for targeted therapies.

Here’s a simplified timeline:

1. 1989: The CFTR gene is identified.
2. Early 2000s: Research focuses on understanding the different types of CFTR mutations and their impact on protein function.
3. 2012: Ivacaftor (Kalydeco) – the first CFTR modulator – is approved by the FDA. This drug specifically targets the G551D mutation, restoring some CFTR protein function in a subset of patients.
4. 2015: Lumacaftor/Ivacaftor (Orkambi) is approved, addressing the most common CFTR mutation, F508del.
5. 2018: Tezacaftor/Ivacaftor (Symdeko) offers improved efficacy for patients with F508del and other mutations.
6. 2019: Elexacaftor/Tezacaftor/Ivacaftor (Trikafta) – a triple combination therapy – receives FDA approval, benefiting approximately 90% of people with cystic fibrosis.

The Lasker Prize Recipients and Their Contributions

The 2025 Lasker Prize recognizes the collective efforts of several key researchers.While specific names are subject to official announcements, the award broadly acknowledges the contributions of scientists involved in:

* Gene Discovery: Those who identified and characterized the CFTR gene.

* Mechanism of Action Studies: Researchers who elucidated how different CFTR mutations affect protein function.

* Drug Development: Scientists and pharmaceutical companies responsible for designing, synthesizing, and testing CFTR modulators.

* Clinical Trial Leadership: Physicians and researchers who conducted rigorous clinical trials to demonstrate the safety and efficacy of these new therapies.

How CFTR Modulators Work: Restoring Protein Function

CFTR modulators aren’t a cure for cystic fibrosis, but they address the underlying defect by helping the CFTR protein function more effectively. Different modulators work in different ways:

* Correctors: Help the misfolded CFTR protein reach the cell surface. (e.g., Lumacaftor, Tezacaftor)

* Potentiators: Help the CFTR protein open properly, allowing chloride ions to flow through. (e.g., Ivacaftor)

* Amplifiers: Increase the amount of CFTR protein produced. (e.g., Elexacaftor)

The combination of these mechanisms, as seen in Trikafta, provides the most significant clinical benefit for the largest number of patients.

Benefits of CFTR Modulators: Improved Quality of Life

The impact of CFTR modulators on the lives of people with cystic fibrosis has been profound. Benefits include:

* Improved lung function

* Reduced frequency of pulmonary exacerbations (lung infections)

* weight gain and improved nutritional status

* enhanced quality of life

* Increased life expectancy – studies show a significant increase in median predicted survival for those on these therapies.

Real-World Impact: Patient Stories and Case Studies

Anecdotal evidence and clinical data consistently demonstrate the transformative power of CFTR modulators. Patients previously reliant on frequent hospitalizations and intensive therapies are now able to lead more active and fulfilling lives.

Such as, individuals who once struggled with daily breathing exercises and constant infections are now able to participate in sports, attend school regularly, and pursue careers. While long