A new daily pill for pancreatic cancer is showing remarkable promise in early clinical trials.
The drug, daraxonrasib, is designed to block cancer signals linked to the RAS gene. In a phase 1/2 trial led by the Dana-Farber Cancer Institute and published in The New England Journal of Medicine, 168 patients with advanced pancreatic cancer and RAS mutations were tested. All had previously undergone at least one round of chemotherapy.
At the 300-milligram dose, about 30% of patients saw a positive response. Overall, roughly 90% experienced disease control, meaning their cancer shrank or stopped progressing.
Lead investigator Dr. Brian Wolpin of Dana-Farber stated that if future trials confirm these results, daraxonrasib could be a targeted therapy relevant to nearly all advanced pancreatic cancer patients. More than 90% of pancreatic cancers carry these harmful RAS mutations.
The most common side effects were rash, mouth inflammation, nausea, and diarrhea. Most patients tolerated the treatment with supportive care; few needed to stop therapy.
Because this was an early-stage study, it does not prove daraxonrasib is superior to standard chemotherapy. However, the results look substantially better than historical chemotherapy trials in this patient population.
As Wolpin noted, pancreatic cancer has had very few effective therapies. Daraxonrasib represents real momentum, though it is still investigational and not a cure.
Additional experts echoed cautious optimism. Dr. Brian Slomovitz of Mount Sinai Medical Center said that if the full dataset confirms these findings, it could be one of the most important breakthroughs in solid tumors, doubling survival time in pretreated patients.