A once-daily pill is said to have achieved what decades of chemotherapy research could not in pancreatic cancer
A once-daily pill has doubled survival for pancreatic cancer patients who failed chemotherapy, targeting a gene mutation researchers had long considered untreatable.
Full results from the 500-person Phase 3 RASolute 302 trial showed that daraxonrasib, developed by US biotech Revolution Medicines, cut the overall risk of death by 60 percent and doubled median survival to 13.2 months from 6.7 months for patients on standard chemotherapy, Reuters reported.
The findings were published Sunday in the New England Journal of Medicine and presented at the American Society of Clinical Oncology annual meeting in Chicago, where thousands of oncologists gave them a standing ovation.
According to Reuters, the drug also halted or reversed tumour progression in nearly a third of patients, compared with 10 percent in the chemotherapy group.
Only 1.2 percent of patients on daraxonrasib discontinued treatment due to side effects, compared with 11.2 percent in the chemotherapy group, CBC News reported.
Jennifer Knox, a medical oncologist at Princess Margaret Cancer Centre in Toronto, called the result "the biggest breakthrough for pancreatic cancer ever," telling the outlet that previous oncology advances had not worked in pancreatic cancer.
"This is the game changer," she said.
Pancreatic cancer kills upwards of 6,000 Canadians each year, according to the Canadian Cancer Society, and carries only a 13 percent five-year survival rate, CBC News reported.
About 80 percent of patients are diagnosed at the advanced or metastatic stage, when the cancer has already spread to other organs.
The Pancreatic Cancer Action Network called the results "the most significant advance we have ever seen" for the disease, noting that patients "not only live longer but also feel better."
Daraxonrasib is the first in a new class of drugs called RAS(ON) inhibitors, which target mutations in the RAS gene family that drive uncontrolled tumour growth.
More than nine in 10 pancreatic cancer cases involve a mutation in the KRAS gene, which produces an abnormal protein that stays permanently switched on, CBC News reported.
Bishal Gyawali, an associate professor at Queen's University in Kingston, Ont, who attended the meeting, told the same outlet the gene "was thought to be undruggable" until now.
The drug's potential extends beyond the pancreas.
Knox noted to CBC News that RAS mutations drive lung, colorectal, biliary tract, kidney, and gastric cancers as well.
"There's the potential to help all of those," she said.
Rash affected 86.3 percent of patients on daraxonrasib, with severe cases in 14 percent, though trial lead Brian Wolpin of Harvard's Dana-Farber Cancer Institute said it is largely manageable with antibiotics and topical steroids, per Reuters.
Severe or life-threatening side effects overall occurred in 43.6 percent of the daraxonrasib group, versus 57.5 percent of those on chemotherapy.
The drug is not yet approved in Canada or the US.
American patients can currently access it through an expanded access program authorised by the US Food and Drug Administration, which plans a speedy review.
Revolution Medicines told CBC News the company is "actively preparing regulatory submissions globally" but did not confirm whether Canada is included.
As of Monday, Health Canada had not received a submission.
Revolution Medicines chief executive Mark Goldsmith said the company is already testing daraxonrasib in earlier-stage disease and in combination with other treatments, according to Reuters, with the goal of extending its survival benefit further.
