Medical researchers have announced encouraging results from a clinical trial testing a new medication for advanced pancreatic cancer, one of the most lethal forms of the disease.
The experimental treatment, known as daraxonrasib, demonstrated significant benefits for patients whose cancer had spread and stopped responding to previous therapies. In a study of 500 participants, those receiving the daily medication survived for a median of 13.2 months, compared to 6.7 months for patients given standard chemotherapy treatments.
“While not curing the cancer, it is a very large step forward,” said Dr. Zev Wainberg, of the University of California, Los Angeles, who helped lead the study.
The medication works by targeting a mutated protein that drives tumor development in more than 90% of pancreatic cancer patients – a biological target that scientists have struggled to address for decades. The research findings were published in the New England Journal of Medicine and presented Sunday at the American Society for Clinical Oncology meeting in Chicago.
Dr. Rachna Shroff of the University of Arizona Cancer Center, who was not part of the research team, expressed her emotional reaction to the results. “Having treated pancreatic cancer for 16 years, I actually started crying” when first seeing the study results, she said from the ASCO meeting. She noted how “patients stayed on this treatment because it was providing durable and meaningful benefit to them.”
Patients taking the experimental pills experienced fewer severe side effects and reported better quality of life as their tumors decreased in size. Many participants continued using the medication longer than the comparison group remained on chemotherapy, and some were still taking it when data analysis concluded.
Dr. Brian Wolpin, of the Dana-Farber Cancer Institute, presented the findings Sunday. He indicated the drug should become “a new standard of care” for previously treated metastatic pancreatic cancer. Researchers plan to investigate its potential use earlier in treatment progression and explore whether tumor reduction might make more patients eligible for surgical intervention.
The most common side effects that could impact treatment continuation included potentially severe skin rashes and mouth sores, according to Wolpin.
Revolution Medicines, the company that developed the medication, funded the research. The Food and Drug Administration has committed to fast-track review of the drug while simultaneously allowing “expanded access” to qualifying patients. The treatment gained public visibility when former U.S. Sen. Ben Sasse discussed on “60 Minutes” how he experienced reduced pain while using it. Cancer specialists report being overwhelmed with patient requests as the special access program launches.
Pancreatic cancer ranks among the most fatal cancer types primarily because early detection proves difficult before it spreads to other organs. The American Cancer Society projects approximately 67,000 new diagnoses in the U.S. this year, with more than 52,000 deaths expected. The five-year survival rate stands at just 13%.
While other cancer types have benefited from diverse treatment alternatives beyond chemotherapy, pancreatic cancer has remained particularly challenging to treat effectively.
Cancer experts not participating in this research expressed hope that these results may represent a breakthrough moment in developing new treatment options, noting dozens of experimental medications currently under development.
The medication targets mutations in the RAS gene family, which typically controls cell growth. KRAS mutations play a particularly crucial role in pancreatic cancer development. However, the protein structure made it difficult for drugs to attach to the mutated proteins, leading scientists to consider this cancer driver “undruggable” for many years.
Revolution Medicines’ treatment uses what researchers describe as molecular adhesive to connect with multiple KRAS variations. Wainberg indicated that future research will examine whether the medication showed greater effectiveness against specific subtypes.
Dr. Andrew Coveler of the Fred Hutchinson Cancer Center, who was not involved in the study, predicted the drug will transform pancreatic cancer treatment approaches. “This thing works drastically differently,” he stated.
Wainberg noted that other medications in development focus on specific KRAS variations. Additional experimental approaches include vaccines designed to prevent cancer recurrence following pancreatic surgery by training the immune system to identify the mutated protein.
