Three scientists who revolutionized the treatment of cystic fibrosis and prolonged the lifespan of patients with the deadly disease have won the Lasker Award, one of the most prestigious awards in medicine.
Boston-based Vertex Pharmaceuticals researcher Paul Negulescu, former Vertex researcher Jesús (Tito) González, and University of Iowa professor Michael Welsh, will share the $250,000 prize. The Lasker DeBakey Clinical Medical Research Award, as it’s formally known, is given for a “major advance that improves the lives of thousands of people,” according to the Lasker Foundation.
“It’s not only gratifying and rewarding,” said Negulescu, a senior vice president at Vertex. “Our hope is that our work can serve as lessons for other research.”
Since 1945, Lasker has awarded prizes to more than 400 scientists, nearly a quarter of whom also won the Nobel Prize, according to the foundation.
The three winners hold key roles in the development of a triple-drug combination therapy known as Trikafta, which helps to improve the quality, and length, of life for people with cystic fibrosis. Historically, people with the disease died before the age of 40; most would not survive early childhood, according to the Lasker Foundation.
About 40,000 children and adults are living with CF in the United States, and an estimated 105,000 have been diagnosed with the disease across 94 countries, according to the Cystic Fibrosis Foundation. Vertex’s drug, Trikafta, can increase the life expectancy of people diagnosed with CF to about 83 years if the medicine is taken around the age of 12, according to a 2023 study published in the Journal of Cystic Fibrosis.
The winners’ research “transformed CF from a death sentence into a manageable condition for more than 90 percent of people with the disease,” the Lasker Foundation said in a statement. “Their scientific innovations are helping afford those with CF the chance to thrive today and to plan vibrant futures.”
For González, who left Vertex in 2008 after more than a decade of CF research with the company, the award presents an opportunity to look back at the monumental achievement — a rarity in the drug development field, he said.
“You’re always pushing a rock up the hill, so it’s nice to take a break and see how well it worked out,” he said. “These results would not have been possible without the effort of thousands of people who contributed to this.”
In recent years, González has shifted his work to research methods of visualizing breast and lung cancers in surgery for a more efficient treatment.
Cystic Fibrosis is caused by mutations in the gene that results in excessive buildup of mucus in the lungs, pancreas, and other organs, essentially drowning CF patients. For decades, the treatment was mainly palliative. Patients would take a heaping amount of drugs to improve their breathing and ability to process food. Some used devices to inhale medicated mist or create sound waves to loosen mucus in their lungs.
Trikafta, which was developed at Vertex’s San Diego laboratory and approved by the US Food and Drug Administration in 2019, focuses on repairing the mucus proteins as they form in the cell, as opposed to most prior research, which sought to treat the disease by changing the defective gene, the foundation said.
“The lesson here is to understand the cause of the disease and go right at it,” Negulescu said. “It might take longer and be more difficult, but this is proof that it works.”
Globally, two-thirds of people diagnosed with CF are being treated with the Vertex drug, according to the company. But many in low- and middle-income countries can’t afford the drug due to its hefty price tag of $300,000 a year, which is paid for primarily by private insurers and reimbursement agreements in rich countries.
The company said it recognized accessibility challenges and has established a pilot donation program to provide CF medication to eligible people. The program is now available in 14 countries.
“Our goal is that everyone with CF has effective medication,” Negulescu said.
While continuing research on CF, Negulescu and the Vertex team are also developing nonopioid pain therapeutics. In January, the FDA approved Vertex’s medication for moderate to acute pain, Journavx, which was released to the market in March. As of mid-July, more than 110,000 prescriptions were filled across the country, according to the company.
Yogev Toby can be reached at yogev.toby@globe.com.
