Experimental pill shows promise for deadly pancreatic cancer
New treatment extends survival and improves quality of life in advanced cases
Washington: A novel pill has helped people with advanced pancreatic cancer live longer, researchers reported Sunday, raising hopes for improved 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, called daraxonrasib, blocks a mutated protein that fuels tumour growth in more than 90% of pancreatic cancer cases — a target that had eluded treatment for decades.
In a study involving 500 patients whose metastatic (spreading) cancer had stopped responding to prior treatment, the daily pill nearly doubled survival time and resulted in fewer severe side effects compared to further chemotherapy. The findings were published in the New England Journal of Medicine and presented at the American Society for Clinical Oncology meeting in Chicago.
Patients taking daraxonrasib lived a median of 13.2 months, compared with 6.7 months for those receiving chemotherapy. While the improvement may seem modest, Wainberg said it marked the first drug to show a significant advantage over chemotherapy.
Dr Rachna Shroff of the University of Arizona Cancer Center, who was not involved in the research, said she was deeply moved by the results. She noted that patients remained on the treatment longer because it provided sustained and meaningful benefits.
Although the drug’s effects can diminish over time, patients used it for significantly longer than those on chemotherapy, reporting reduced pain and improved quality of life as tumours shrank. Many participants were still taking the drug when the data was analysed, suggesting the survival gap may widen as monitoring continues.
Dr Brian Wolpin of the Dana-Farber Cancer Institute, who presented the findings, said the drug could become “a new standard of care” for previously treated metastatic pancreatic cancer. Researchers also plan to study its use earlier in the disease, including whether tumour shrinkage could make more patients eligible for surgery.
The most common side effects affecting use of the drug include severe rash and mouth sores.
The study was funded by Revolution Medicines, and the US Food and Drug Administration plans to expedite its review. The agency is also allowing expanded access to eligible patients while the review is ongoing.
Pancreatic cancer remains one of the most deadly cancers, largely because it is difficult to detect before it spreads. The American Cancer Society estimates that about 67,000 new cases will be diagnosed in the United States this year, with more than 52,000 deaths. The five-year survival rate is around 13%.
Unlike other cancers, pancreatic cancer has been difficult to treat with alternatives to chemotherapy. However, experts not involved in the study said the findings could mark a turning point, with multiple experimental drugs currently in development.
Daraxonrasib targets mutations in the RAS gene family, which regulate cell growth. KRAS mutations are particularly important in driving pancreatic cancer but were long considered difficult to treat.
The drug works by acting as a form of molecular “glue”, binding to several KRAS subtypes. Researchers will now investigate whether it is more effective against specific subtypes.
Other treatments under development include drugs targeting specific KRAS mutations and vaccines designed to prevent recurrence after surgery by training the immune system to recognise the mutated protein.