How an ‘Impossible’ Idea Led to a Pancreatic Cancer Breakthrough

And now that the protein-targeting strategy shows promise, multiple companies have jumped into the fray. Dozens of similar drugs are now being tested for cancers of the pancreas, lung and colon.
The drug that opened the floodgates, daraxonrasib, has been fast-tracked for review by the Food and Drug Administration and could win approval later this year. Until then, the agency has signed off on a plan by Revolution Medicines, the small Silicon Valley company developing the drug, to offer early access to some patients.
The pills, three taken daily, are not a cure — eventually, daraxonrasib stops working. Many patients do not respond. And it has side effects that can be harsh, including rash, diarrhea, fatigue, nausea and raw, split fingertips.
Until now, however, patients with pancreatic cancer have typically been offered grueling chemotherapy that does little to extend their lives.
A gland deep in the abdomen, the pancreas helps regulate blood sugar and digestion. Only 3 percent of these patients with cancer that has spread to distant parts of their body are still alive after five years. The disease kills more than 50,000 Americans a year.
Revolution tested daraxonrasib in a late-stage clinical trial in patients who had metastatic cancer and had already tried chemotherapy. For these patients, further treatment was viewed as a Hail Mary.
Patients who received the drug lived for a median of over 13 months, compared with less than seven months for patients who had chemotherapy, the company said in a news release.
Researchers will present the findings at a major cancer conference in Chicago later this month. The study has not yet been published in a peer-reviewed medical journal.
Scientists say the drug could turn out to be cancer’s equivalent of breaking the four-minute mile. “It’s the beginning, not the end,” said Dr. Elizabeth Jaffee, a pancreatic cancer researcher at Johns Hopkins University.




