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WASHINGTON (AP) — A novel pill helped people with advanced pancreatic cancer live longer, researchers reported Sunday, raising hopes of long-needed better
treatments for one of the deadliest types of cancer. “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 drug, daraxonrasib, blocks a mutated protein that fuels tumor growth in more than 90% of pancreatic cancer cases — a target that had eluded treatment for decades.The daily pills nearly doubled survival time, with fewer severe side effects, in a study that randomly assigned the experimental drug or more chemotherapy to 500 patients whose metastatic cancer had stopped responding to prior treatment. The findings were published in the New England Journal of Medicine and presented Sunday at the American Society for Clinical Oncology meeting in Chicago.
Patients taking daraxonrasib lived for a median of 13.2 months compared with 6.7 months for those receiving chemotherapy. While this may seem like a small improvement, Wainberg noted it marked the first drug to demonstrate a substantial advantage over chemotherapy.
“Having treated pancreatic cancer for 16 years, I actually started crying when first seeing the study results,” said Dr. Rachna Shroff of the University of Arizona Cancer Center, who wasn't involved with the research. She was struck by how “patients stayed on this treatment because it was providing durable and meaningful benefit to them.”
Although the pills’ effects eventually wane, recipients used them for significantly longer than the comparison group remained on chemotherapy, reporting less pain and a better quality of life as their tumors shrank. Many continued using the drug after the data was analyzed, which Wainberg indicated means the survival gap may widen as researchers continue tracking them.
Dr. Brian Wolpin, of the Dana-Farber Cancer Institute, presented the findings Sunday. He asserted the drug should become “a new standard of care” for previously treated metastatic pancreatic cancer, adding that researchers will also explore its use earlier in the disease to determine if tumor shrinkage might allow more patients to qualify for surgery.
Side effects most likely to affect pill usage included a severe rash and mouth sores, he stated.
Revolution Medicines funded the study, and the Food and Drug Administration plans to expedite the review of the drug. Meanwhile, the agency is allowing what’s known as “expanded access” to the experimental drug for patients who meet certain criteria. The drug garnered public attention when former U.S. Sen. Ben Sasse described on “60 Minutes” how he experienced less pain while taking it. Oncologists are now receiving a surge of requests as the special access program begins.
Pancreatic cancer ranks among the most lethal forms primarily due to its difficulty in detection before it spreads to other organs. The American Cancer Society estimates that about 67,000 new cases will be diagnosed in the U.S. this year, with more than 52,000 fatalities resulting from the disease. The five-year overall survival rate stands at 13%.
Unlike other cancers that have benefitted from various chemotherapy alternatives, pancreatic cancer has posed a more significant challenge.
Cancer specialists not involved in the new research expressed optimism that this could mark a turning point in the search for new treatment options, with numerous experimental drugs currently in development.
The new drug targets mutations in the RAS gene family that typically regulate cell growth. KRAS mutations are particularly critical in driving pancreatic cancer. However, a structure that made it difficult for drugs to adhere to the mutated proteins had long rendered this cancer driver “undruggable.”
Revolution Medicines’ drug employs what can be described as a molecular glue to bind with multiple KRAS subtypes. Wainberg indicated that researchers will next investigate whether the drug works more effectively in specific subtypes.
The drug is set to transform pancreatic cancer treatment, according to Dr. Andrew Coveler of the Fred Hutchinson Cancer Center, who wasn’t involved in the research.
“This thing works drastically differently,” he remarked.
Wainberg noted that other drugs in development target specific KRAS subtypes. Other approaches in earlier testing phases include vaccines aimed at preventing recurrence after pancreatic cancer surgery by training the immune system to recognize the mutated protein.
