Trio honored for work on Trikafta triple-drug combo medication for CF
Scientists to share medical research award, $250K prize
A trio of scientists is being honored for research contributions that helped lead to the development of Trikafta, a triple-drug combination medication for people with cystic fibrosis (CF).
The three winners of this year’s Lasker~DeBakey Clinical Medical Research Award are Paul Negulescu, PhD, senior vice president at Vertex Pharmaceuticals, which markets Trikafta; Jesús González, PhD, a former Vertex researcher; and Michael J. Welsh, MD, a scientist at the University of Iowa. The three will share a $250,000 prize.
The award, administered by the Lasker Foundation, was established in the 1940s to recognize scientists who help make major advances in understanding, diagnosing, treating, and preventing medical conditions. In announcing the winners, the foundation said these scientists’ contributions “culminated in a triple-drug combination that has transformed CF into a manageable condition.”
“Welsh, González, and Negulescu’s achievements are affording people with CF the chance to thrive now and to plan vibrant futures,” the announcement states.
Trikafta, a CFTR modulator therapy, has been approved in the U.S. since 2019 to target the underlying genetic defect responsible for CF. It’s now approved in more than 30 countries, including Canada and Australia, and is widely available in Europe.
CF is caused by mutations in the gene that provides instructions to make the CFTR protein, which is important for regulating the production of mucus. In people with CF, the lack of functional CFTR protein leads to thick, sticky mucus that builds up in organs and drives most disease symptoms.
Trikafta a combo of 3 CF medications: elexacaftor, tezacaftor, and ivacaftor
CFTR modulators are medications that can increase the functionality of the defective CFTR protein in people with CF caused by certain genetic mutations. Trikafta is a combination therapy containing three modulators: elexacaftor, tezacaftor, and ivacaftor. It’s indicated for CF patients with responsive mutations — including the most common disease-causing mutation, F508del.
Other CFTR modulator therapies approved for use in the U.S. include Kalydeco (ivacaftor), the dual-combination therapies Orkambi (lumacaftor/ivacaftor) and Symdeko (tezacaftor/ivacaftor), and a triple-combo therapy developed as a next-generation successor of Trkafta, which is called Alyftrek (vanzacaftor/tezacaftor/deutivacaftor). All of these medications are sold by Vertex.
Welsh — who this month was also awarded the Yergin-New International Prize for his contributions to CF research — helped lead early work to figure out how the CFTR protein works. His research laid the groundwork for the concept of developing modulator therapies to increase the protein’s function.
For their part, González and Negulescu worked on projects to screen thousands of compounds to potentially increase protein function. This ultimately led to the identification of the molecules used in Trikafta and other modulator therapies.
Negulescu said in a press release from Vertex that he was “honored to represent my coleaders on the CF program, Fred Van Goor and Sabine Hadida.” The scientist also gave a shout-out to “the more than a thousand people at Vertex who have worked over two decades to make the impossible possible.”
The Vertex release says the company’s CF therapies are now used in more than 60 countries.
“Together, we’ve brought five medicines including Trikafta … to tens of thousands of people with CF around the world — medicines that have fundamentally changed the course of this disease,” Negulescu said. “This award recognizes that collective effort and serves as a reminder of the work ahead in reaching all people with CF.”
About the Author
