CF is caused by genetic mutations leading to mucus buildup in organs, and prior treatments mainly managed symptoms rather than addressing the root cause.

Despite its success, challenges such as high costs—around $300,000 annually—and limited effectiveness for some mutations remain, although access efforts are underway.

Their discovery revealed that the defective CFTR protein was not entirely broken but trapped or malfunctioning, leading to drugs that restore its mobility and function.

González co-invented a system to track ion flow in real-time, which accelerated drug screening and was vital in discovering effective CF modulators.

The new treatment has significantly extended life expectancy, with patients now living near-normal lifespans if treatment begins early, compared to early childhood deaths in the past.

Since its approval, Trikafta has increased CF patients' life expectancy to around 83 years if started at age 12, marking a major breakthrough in longevity and health outcomes.

This therapy has turned CF from a fatal disease into a manageable condition for over 90% of patients, with many children now living active, healthier lives.

Negulescu emphasizes that understanding disease causes at the molecular level is crucial, guiding their scientific approach to developing targeted therapies.

Three scientists—Dr. Michael Welsh, Paul Negulescu, and Jesú́s (Tito) González—won the 2025 Lasker-DeBakey Clinical Medical Research Award for their groundbreaking development of Trikafta, a triple-drug therapy that has transformed cystic fibrosis (CF) treatment.

Trikafta, approved in 2019, combines three drugs to target most CF cases, significantly reducing hospitalizations and lung transplants, and dramatically improving patients' quality of life.

Research from Welsh's lab in the 1980s and 1990s uncovered that the common delta-f508 mutation prevents CFTR proteins from reaching the cell surface, impairing ion flow essential for organ function.

Vertex has launched a pilot donation program across 14 countries to improve access to Trikafta for low- and middle-income populations.

The team used innovative techniques, including dye-based chemical screening and real-time ion flow tracking systems co-invented by González, to identify molecules capable of correcting the malfunctioning CFTR protein.