At Dana-Farber Cancer Institute in Boston, researchers have published results from the first human trial of daraxonrasib, a daily pill that offers new hope to pancreatic cancer patients whose tumors carry RAS gene mutations. The findings, published in The New England Journal of Medicine, reveal that 90% of patients experienced disease control—meaning their cancer either shrank or stopped advancing—a result that has stunned researchers and could reshape how this devastating disease is treated.
Pancreatic cancer remains one of the most difficult cancers to treat effectively, and the promise of a targeted therapy is particularly significant. More than 90% of pancreatic cancers carry harmful RAS gene mutations, yet existing drugs targeting these mutations have largely failed to work against the types most common in pancreatic cancer. Daraxonrasib is designed differently: it blocks multiple active cancer signals that fuel tumor growth, making it potentially relevant to nearly all patients with advanced pancreatic cancer.
The phase 1/2 clinical trial, led by Dr. Brian Wolpin, tested daraxonrasib in 168 patients with advanced pancreatic cancer who had already received at least one round of chemotherapy. At the 300-milligram dose—the amount planned for larger phase 3 trials—approximately 30% of patients experienced a positive response, meaning their tumors actually shrank. But the more striking finding was the 90% disease control rate: the vast majority of patients saw meaningful clinical benefit rather than rapid tumor progression.
"If supported by data from future clinical trials, daraxonrasib would be a targeted therapy relevant to nearly all patients with advanced pancreatic cancer," Wolpin said in a statement. In an interview with Fox News Digital, he called it "one of the most promising therapy advances we've seen in pancreatic cancer," a field that has historically had "very few effective therapies." The lead investigator also noted that results from daraxonrasib "looked substantially better than what we have seen in prior clinical trials of chemotherapy in patients with previously treated metastatic pancreatic cancer."
The drug did produce side effects—most commonly rash, mouth inflammation, nausea, and diarrhea—but the trial showed these were manageable. Most patients tolerated treatment with supportive care measures, and very few stopped therapy because of adverse effects. This suggests that daraxonrasib could offer a viable option for patients who might otherwise face limited choices.
Yet researchers emphasize caution and humility. This was an early-stage trial without a randomized control arm directly comparing daraxonrasib to chemotherapy, so it does not definitively prove the drug's superiority. The trial also included only patients with advanced disease who had already received prior treatments, leaving open questions about how daraxonrasib might perform in earlier disease stages. Additional research is needed to determine how best to sequence or combine therapies for the most durable responses and potential cures.
For patients and families affected by pancreatic cancer, Wolpin offered measured optimism: daraxonrasib signals "real momentum" toward effective treatments. It is not a cure, and it remains investigational. But it represents a genuinely new direction in pancreatic cancer therapy—one where a single daily pill, tailored to the genetic mutations driving the disease, could soon offer many more patients a fighting chance.