Experimental ‘game-changing’ drug doubles pancreatic cancer survival time, study shows

An experimental drug that doubles the survival time of pancreatic cancer is being hailed as a breakthrough in the fight against the world’s deadliest cancer.

A Phase 3, 500-person trial combined the once-daily pill with standard chemotherapy in patients who had already gone through one unsuccessful round of traditional cancer treatment. The study found that the pill, daraxonrasib, stopped or slowed tumor growth by about a third compared to 10 percent in those given chemotherapy – and the survival time doubled from less than seven months to about 13.

The results of a study from the US biotech company Revolution Medicines were published in the New England Journal of Medicine on Sunday and receive a standing ovation at the annual meeting of the American Society of Clinical Oncology (ASOC) on the same day.

Canadian medical expert Dr. Bishal Gyawali, an associate professor at Queen’s University in Kingston, Ont., was among those in attendance.

“My first reaction was: This is the right kind of stand-up,” he told CBC News by phone from Chicago, where the ASOC meeting was underway. “Because pancreatic cancer was a difficult cancer to treat.”

“It’s a huge breakthrough for pancreatic cancer,” echoed Dr. Jennifer Knox, a pancreatic cancer specialist at the Princess Margaret Cancer Center in Toronto, is also at the ASOC meeting.

“We haven’t seen advances like this in any other treatment. There have been a lot of exciting breakthroughs in all of oncology; none of them have worked in pancreatic cancer. So this is a game changer.”

Pancreatic cancer known as the ‘silent killer’

The “silent killer” shows few early symptoms and is often caught too late, resulting in just a 13 percent five-year survival rate.

It is also stubbornly resistant to treatment, in part because by the time it is detected, the cancer has spread beyond the pancreas – a small gland deep in the abdomen – to other organs, such as the liver, lungs or stomach wall.

The result of a study showing a doubling of survival time for pancreatic cancer is “unprecedented” so far, Gyawali said, although he stressed that the experimental pill is still considered a second-line treatment and not a cure.

“There are still miles to go, but it’s exciting in the sense that it’s the first time [for] such a dangerous disease, we see such good results,” he said.

A ‘historic moment’ in cancer care

Daraxonrasib works by targeting a faulty protein that leads to uncontrolled tumor growth, marking a radical new approach to cancer treatment.

More than nine out of 10 cases of pancreatic cancer are driven by a mutation in the KRAS gene, which acts as the cell’s on-off factor. The mutation produces an abnormal version of the KRAS protein, which is stuck in “walking” mode, sending endless signals for cells to grow and multiply.

“It’s exciting that we can identify this change for the first time and break it down,” Gyawali said, calling the results “great.”

On social media, some doctors also took it as “success” as well as-“historic moment.”

“These results will change the way scientists, doctors and patients think about the treatment of pancreatic cancer,” the main investigator of the case, Dr. Brian Wolpin of the Dana-Farber Cancer Institute at Harvard, told Reuters.

The pill appears to lead to few life-changing side effects, the research team found. Adverse events that led people to stop treatment occurred in 1.2 percent of people in the daraxonrasib group, compared to 11 percent of those in the chemotherapy group.

However, the most common and serious side effect of the drug is rash, which about 14 percent of patients experience.

Pancreatic cancer patient Menta “Steve” Wallace, of Houston, Texas, was one of those people. Still, the 74-year-old told Reuters he was “very happy” with the overall results of daraxonrasib while taking part in an additional, ongoing Revolution Medicine study that is testing the drug in early-stage disease and in combination with other treatments.

Wallace said her last scan showed her tumor had shrunk by about 50 percent.

It hopes to be widely used

Daraxonrasib is the first in a new class of drugs called RAS(ON) inhibitors, which target various members of the RAS family of genes and associated proteins. KRAS is one gene in the RAS family, which is found in a variety of fatal human cancers.

“It’s been studied for decades, like 50 to 60 years, and it was thought to be uncontrollable,” said Knox, a Toronto-based oncologist.

But now there is hope that this promising finding could fuel the development of other RAS(ON) inhibitor-based therapies, not just for pancreatic cancer but other potentially fatal cancers.

“Lung cancer, colon cancer, biliary cancer, kidney cancer, stomach cancer — a lot of cancers have RAS mutations,” Knox said.

“There is power to help all of them.”

Limited access to medicine so far

Daraxonrasib is not yet approved in Canada or the US, although American patients can get the treatment through an early access program approved by the US Food and Drug Administration.

So, when will the drug be available to Canadians?

Revolution Medicines told CBC News that the company “actively preparing for the transfer of laws around the world,” but did not confirm that that includes Canada. Health Canada also has not responded to a CBC News inquiry about whether the agency is reviewing the daraxonrasib shipment.

More than 7,000 new cases of pancreatic cancer are diagnosed each year in Canada, according to the Canadian Cancer Societyand the disease kills more than 6,000 people each year.