Experimental pill offers hope for deadly pancreatic cancer

A novel pill has extended survival for patients with advanced pancreatic cancer. The drug, daraxonrasib, targets a mutated protein in over 90% of cases, offering a potential shift in treatment for a disease with a dismal five-year survival rate of 13%. Researchers called the findings a “very large step forward” despite the drug not curing the condition.

Study Results

The trial involved 500 patients with metastatic cancer that had stopped responding to prior treatments. Those taking daraxonrasib lived a median of 13.2 months, compared to 6.7 months for chemotherapy. Dr. Zev Wainberg of UCLA, who led the study, noted this is the first drug to show a significant survival advantage over chemotherapy in pancreatic cancer.

Patients on the pill reported fewer severe side effects and better quality of life, with many continuing treatment longer than those on chemotherapy. Dr. Rachna Shroff of the University of Arizona, who attended the American Society for Clinical Oncology meeting, described the results as “durable and meaningful,” noting patients stayed on the drug due to its benefits.

Mechanism and Development

The drug uses a molecular glue to bind multiple KRAS subtypes, a protein family central to pancreatic cancer growth. KRAS mutations, long deemed “undruggable,” are a key driver of the disease. Revolution Medicines developed the pill, and the FDA plans to expedite its review. Expanded access is already available for eligible patients.

Dr. Brian Wolpin of Dana-Farber Cancer Institute said the drug could become a new standard of care. Researchers also plan to test it earlier in the disease, exploring whether tumor shrinkage might make surgery possible for more patients. Common side effects include rash and mouth sores, though they are manageable.

Context and Future

Pancreatic cancer is among the deadliest cancers due to late detection. The American Cancer Society estimates 67,000 new cases in the U.S. this year, with over 52,000 deaths expected. Unlike other cancers with multiple treatment options, pancreatic cancer has lacked effective therapies until now.

Experts not involved in the study expressed cautious optimism. Dr. Andrew Coveler of Fred Hutchinson Cancer Center called the drug’s mechanism “drastically different” from existing options. Other experimental approaches, including vaccines targeting mutated proteins, are also in development.

The FDA’s expedited review and public interest—sparked by figures like former Sen. Ben Sasse sharing his experience—highlight the urgency. Oncologists report a surge in requests for the drug through the expanded access program. Researchers now aim to determine if the pill works better in specific KRAS subtypes, potentially refining its use in the future.