Μake us preferred on Google For four decades, cancer researchers fought to disable a stubborn mutation that made pancreatic cancer a death sentence—to little avail.
Now, doctors have a medicine that targets the mutation. The Food and Drug Administration’s approval of the drug from Revolution Medicines on Wednesday marks a milestone in treatment of one of the most intractable kinds of tumors.
“This is life-altering,” said Carla Kurkjian , an oncologist at Mercy Hospital in Oklahoma City, who wasn’t involved in the studies. “Not just for patients, as far as helping them extend their lives beyond anything we’d ever seen, but for us as physicians, to be able to have a different conversation than we’ve had for so long.”
The drug, called Rasonque, helped subjects in a late-stage study live more than 13 months, nearly twice as long as those on chemotherapy alone. When presented at the world’s largest gathering of cancer researchers last spring, the results drew a nearly minute-long standing ovation.
Yet the drug does have side effects, including a harsh rash across the body and gastrointestinal issues. Former Nebraska Sen. Ben Sasse , who went public in the spring with what he described as a significant reduction in his tumors while in a clinical trial of the drug, appeared on television with his face covered in red blotches.
Wall Street expects Rasonque, which was approved for use in patients for whom other treatments failed, to be a big seller. The drug could generate more than $20 billion in annual sales, according to pharmaceutical commercial intelligence firm Evaluate, if the drug proves to work safely in earlier stages of the disease and with different types of cancers.
“This is a monumental opportunity for patients and their families, and we need to do everything we can to clear the decks and make that happen as quickly as possible,” RevMed Chief Executive Mark Goldsmith said.
Rasonque is RevMed’s first approved drug.
Given the opportunity, competitors—including large pharmaceutical companies such as Eli Lilly and Pfizer —have been racing to develop their own RAS-targeting pancreatic-cancer drugs.
Some 67,000 people in the U.S. are diagnosed with pancreatic cancer each year, according to the American Cancer Society. They haven’t had good options for treatment and typically resort to chemotherapy. About seven in eight patients die within five years of diagnosis—many within a year.
The breakthrough caps a decadeslong scientific chase to solve one of the biggest mysteries in cancer treatment.
In the early 1980s, scientists identified a small number of mutated genes that drove most human cancers. A family of three closely related genes called RAS stood out. Together, the RAS mutations drive as much as a third of all human cancers, including most pancreatic tumors.
Drugmakers rushed to find treatments, but RAS proved especially tough to crack. Millions of chemical compounds failed to work. The problem was the shape of the protein generated by the mutated gene. It was flat, without a pocket that a typical drug could lock onto.
Given the shape, some scientists labeled RAS “undruggable” and moved on. Greg Verdine , then a Harvard researcher, looked to nature for a workaround.
In nature, there are compounds that are able to hook up with flat proteins like RAS by first latching onto a “helper” protein inside of human cells. The compounds are called molecular glues.
In 2012, Verdine launched a biotech, Warp Drive Bio, to look for molecular glues out in the wild. It sequenced 135,000 bacteria hunting for one. The research found six candidates, but none worked on RAS, so Verdine proposed designing a synthetic glue instead.
The next year, in 2013, cancer researcher Kevan Shokat found a pocket on the RAS protein that a drug could latch on to. Shokat, a chemical biologist at the University of California, San Francisco, co-founded RevMed. It bought Warp Drive Bio in 2018.
RevMed began testing the drug in its first patients in 2022.
Jay Williams, 58 years old, started taking Rasonque in July after rounds of chemotherapy and radiation and a major surgery failed to halt his cancer’s spread. He got the drug through a pathway that lets patients receive a drug before formal FDA approval.
A former executive at Disney , Williams said he and his wife have made it back to Disneyland several times this summer and he walks a mile a day. His next scan, in late August, will show whether the cancer is responding. He hopes Rasonque can help him spend more time with his family, including a son who is getting married this September.
“What it does is buy you time, and that’s one thing you don’t always have with pancreatic cancer,” he said.
Write to Xavier Martinez at xavier.martinez@wsj.com
