After new drug’s ‘unprecedented’ results for pancreatic cancer, doctors look at other uses - NBC News

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Every single patient with advanced pancreatic cancer who walked into Dr. Zev Wainberg’s office told him they would rather take an experimental medication than endure another round of chemotherapy.

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Wainberg, co-director of UCLA Health’s GI Oncology Program, was leading a clinical trial of a new drug called daraxonrasib. All the study participants previously had chemotherapy that was starting to fail.

“Statistically, I knew only half of them get the pill, and we don’t get to choose,” Wainberg said. “I put a lot of patients on the chemo arm, and none of them are alive anymore.”

“It’s one of the most emotional studies I’ve ever been a part of,” he said.

Enthusiasm around daraxonrasib is reaching a fever pitch. In the Phase 3 trial of 500 patients, the drug was shown to double the survival time of patients with advanced pancreatic cancer, a notoriously deadly cancer: 13.2 months, on average, compared to 6.7 months for people who got chemo. On Sunday, Wainberg and his colleagues presented those results at the American Society of Clinical Oncology’s annual meeting in Chicago. The full study was simultaneously published in the New England Journal of Medicine.

When Revolution Medicines, which makes the drug, released the trial’s preliminary findings in April, Dr. Rachna Shroff, chief of the division of hematology and oncology at the University of Arizona Cancer Center in Tucson, said she “started crying tears of joy.”

“It’s that big of a game-changer for those of us who treat pancreatic cancer,” she said. “It’s unprecedented.”

Now, the excitement is spilling over to other types of cancer. Daxaronrasib, which is taken as three pills once a day, works by targeting a mutation in the KRAS gene found across many cancers, including lung, colorectal, ovarian, endometrial and a type of bile duct cancer called cholangiocarcinoma.

“Pancreas cancer may be the first for this drug, but there will be others,” said Dr. Brian Wolpin, who also led research on daraxonrasib and directs the Hale Family Center for Pancreatic Cancer Research at Dana-Farber Cancer Institute. “Now the floodgates open.”

The Food and Drug Administration has already put the drug on a fast track toward approval for pancreatic cancer, and earlier this month said it would permit Revolution Medicines to give it to patients outside of clinical trials in an expanded access program.

Dr. Mark Goldsmith, chief executive officer for Revolution Medicines, declined to provide a timeline for when the company will file for FDA approval. “Our professionals are working literally 24/7 to get this material prepared as quickly as possible,” he said.

Most pancreatic cancers are diagnosed in later stages of the disease, when surgery isn’t an option.

“Even with our best chemotherapies, the average benefit is around 6 months, sometimes as little as weeks or months,” said Dr. Sameek Roychowdhury, a gastrointestinal medical oncologist at the Ohio State University Comprehensive Cancer Center. “It’s barely enough time for families to grasp the situation.”

Just 3% of patients diagnosed with metastatic pancreatic cancer are alive five years later, according to the American Cancer Society.

Debby Orcutt, 71, was diagnosed with Stage 4 pancreatic cancer in April 2024. The cancer had already spread to her liver. Prior to her diagnosis, her only symptom had been a nagging pain in her lower left abdomen that got worse at night.

When chemotherapy started failing Orcutt, she enrolled in the clinical trial for daraxonrasib — and was one of the participants who actually got the drug.

Since she began taking the pill in January 2025, the spot on her liver has vanished. And the tumor on her pancreas has shrunk by 80%, according to her oncologist at Dana-Farber.

“I feel great every single day,” said Orcutt, of Spencer, Massachusetts. “I do not dwell on the fact that I have pancreatic cancer.”

Neither Roychowdhury nor Shroff was involved with clinical trials of daraxonrasib, but both have already begun curating lists of patients to get the drug once it’s available.

“There’s not a doubt in my mind that the second it becomes available, I will start using it,” Shroff said.

What is daraxonrasib and how does it work?