Scientists who developed a treatment for cystic fibrosis and dramatically raised patients' survival rates have received the U.S. Lasker Award. The Lasker Award is a prestigious prize given by the Lasker Foundation to people who have contributed to research in medicine and public health. Since 1945, 95 Lasker laureates have also won the Nobel Prize, earning it the nickname "America's Nobel."
The Lasker Foundation said on the 11th (local time) that this year's Lasker DeBakey Clinical Medical Research Award went to Paul Negulescu, executive vice president at U.S. drugmaker Vertex Pharmaceuticals, former researcher Jesús (Tito) González, and Michael Welsh, a professor at the University of Iowa's medical school. The total prize money is $250,000 (about 340 million won).
Cystic fibrosis is a genetic disease caused by mutations in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. As a result, sticky mucus abnormally builds up in major organs such as the lungs and pancreas, causing patients to suffer from breathing difficulties and malnutrition. There are about 40,000 patients in the United States today and around 100,000 patients across 94 countries worldwide.
Existing treatments were limited to symptomatic care that thins mucus or eases symptoms. Because of this, most patients did not survive childhood, and even those who did had an average life expectancy of less than 40. The laureates created a major turning point in cystic fibrosis treatment with a new approach that corrects the protein as it forms in cells. Based on this, Vertex succeeded in commercializing the triple-combination therapy Trikafta.
Trikafta reached the market after winning U.S. Food and Drug Administration (FDA) approval in 2019. The drug has been credited with dramatically improving patients' lung function and raising life expectancy to as high as over 80. About two-thirds of cystic fibrosis patients worldwide are currently using the drug.
The Lasker Foundation said, "This research transformed cystic fibrosis from a death sentence into a manageable disease," adding, "It has given patients the opportunity to plan for a vibrant future."
Negulescu, a laureate and executive vice president, said, "The solution lay in understanding the root cause of the disease and targeting it directly," adding, "It took a long time and was difficult, but it ultimately proved to be the right path." Former Vertex researcher González also noted, "Developing new drugs always feels like climbing a mountain, but having a moment to pause and reflect on the results is deeply meaningful," adding, "It would have been impossible without the efforts of thousands of researchers."
Trikafta's annual treatment cost reaches $300,000 (about 400 million won), making it hard for patients to use easily. Aware of this, Vertex is running a free donation program in 14 countries. Negulescu said, "The goal is to ensure that all patients can receive effective treatment with Trikafta."
The Lasker Award is considered a gateway to the Nobel Prize because many winners go on to receive the Nobel. The Breakthrough Prize, known as the "Silicon Valley Nobel," and Israel's Wolf Prize are regarded in a similar light. Negulescu, this year's Lasker laureate, also won the 2023 Breakthrough Prize in Life Sciences and is considered the top contender for the Nobel Prize.