Alex Abela

When Alex Abela was a postdoctoral fellow at the University of California San Diego in 2014, Vertex Pharmaceuticals invited postdocs to tour the company’s research facility down the street from the university’s campus. Abela’s visit set him on a course that would change the lives of people with the life-threatening disease cystic fibrosis.

Of course, Abela didn’t know that as he toured the facility. But he connected with Vertex employees’ enthusiasm for their work. Alongside the incredible science, part of what drew him to the company was that researchers there stayed involved with hands-on chemistry longer than is the norm in some places, he says. “You can spend a lot of time designing stuff, and you can sell people on ideas . . . but there’s no substitute for just going in there and making it yourself if you really believe in something,” he says.

Abela joined Vertex in a contract position at the tail end of 2015 and started as a full-time employee the following summer. In his first year at Vertex, Abela synthesized a small molecule now known as elexacaftor.

Elexacaftor, as part of the triple-combination therapy Trikafta, improves the function of the cystic fibrosis transmembrane conductance regulator protein. This protein is defective or present in reduced amounts at the cell surface in people who have cystic fibrosis, a genetic disease. The protein regulates the movement of chloride ions across the cell surface. When it doesn’t work properly or isn’t present in sufficient amounts, mucus coating the lungs can become thick and sticky, making it progressively harder to breathe.

The fact that elexacaftor sounds quite a bit like Alex may not be such a coincidence, though Abela insists he had nothing to do with it. “There was a brainstorming session, and that name came out,” he says. “At some point, someone changed the name tag on my desk to Elex Abela, though, instead of Alex, as a joke.”

Trikafta can treat up to 90% of people with cystic fibrosis, according to a Vertex spokesperson. But Vertex has gotten flak for not making the therapy available in lower-income countries. More than 160,000 people around the world have cystic fibrosis, according to a 2022 study, and only about 12% of people living with the disease take Trikafta, which was launched in 2019. Since then, Trikafta has become a blockbuster; it generated almost $7.7 billion in revenue in 2022. Vertex says it is working to expand access to the drug, which people in more than 50 countries have used.

Abela’s excellence predates Vertex, according to his former PhD adviser, Bruce Lipshutz. Abela first joined Lipshutz’s team at the University of California, Santa Barbara, in 2006. That same year, Lipshutz recalls, he found out the lab was the number 1 polluter in the county. Abela developed key Suzuki-​Miyaura couplings that could be done at room temperature and in water rather than in organic solvents. These changes dramatically cut down on the amount of organic waste coming from Lipshutz’s lab.

“He deserves a lot of credit for establishing that that chemistry could be done,” Lipshutz says. “A lot of things that we’ve since done were based on his work.”

Abela also won two awards for his outstanding teaching and had a good rapport with the undergraduate students, Lipshutz says. He isn’t the least bit surprised with Abela’s success in creating elexacaftor. “There’s a lot of medicinal chemists out there that never get a drug to market, let alone an $8 billion drug. So it’s quite an accomplishment and I’m actually very proud of him,” Lipshutz says.

Abela is now a principal research scientist at Vertex and continues to work on treatments for cystic fibrosis. On occasion, people with cystic fibrosis and their families will stop by the Vertex San Diego site, where they can take a tour and meet the research team, Abela says. “It’s nice to work on something that you feel is important, but it’s kind of an abstract idea that you’re going to try to develop a medicine to help to treat a disease,” Abela says. These visits make clear the true impact of his work. “I wasn’t fully prepared for how much you feel like, ‘Wow, what I’m doing could help this person with this incredibly challenging disease.’ ”