Clinical trial results found that patients with previously treated metastatic pancreatic cancer survived a median of 13.2 months on new drug daraxonrasib, twice as long as 6.7 months on chemotherapy.Danielle Villasana/Reuters
When André Bourbeau learned last September that he had pancreatic cancer, he thought he would be dead by Christmas.
The 63-year-old retired civil servant from Cantley, Que., had good reason for a bleak outlook. Pancreatic cancer is among the grimmest diagnoses in oncology. Only one in 10 Canadians with the disease will be alive five years after being diagnosed, compared with nine in 10 with breast cancer. Mortality rates have barely budged in 40 years.
That’s why a new drug, daraxonrasib, that doubles survival time for people with pancreatic cancer, has been greeted rapturously by doctors and patients.
Mr. Bourbeau fears the chemotherapy that has kept him alive for the past 10 months will stop working soon. Daraxonrasib would be the next logical treatment for him.
Trouble is, he can’t get it. Neither can most Canadians with metastatic pancreatic cancer, some of whom have no time to spare.
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“There’s a drug at our doorstep that will double survival, and we can’t get it to the patient,” said George Zogopoulos, a surgeon who operates on pancreatic cancer patients at the McGill University Health Centre, where Mr. Bourbeau is being treated. “The greatness of humanity is to have made these advances. The failure is not to be able to deliver it.”
It is not unusual for there to be a lag of months or years between when researchers learn an experimental cancer drug works and when it becomes available to Canadians outside of clinical trials. Daraxonrasib is so new that the pill’s maker, California-based Revolution Medicines Inc., is still preparing its application to the United States Food and Drug Administration (FDA), the usual first stop for selling prescription medicines.
What’s different in the case of daraxonrasib is the desperation of the patients involved – most of whom had little cause for hope until now – and the degree of public excitement surrounding the new treatment.
When clinical trial results for daraxonrasib, a once-daily pill, were presented at an American oncology conference in Chicago at the end of May, the crowd responded with a standing ovation. The findings, published the same day in The New England Journal of Medicine, were that patients with previously treated metastatic pancreatic cancer survived a median of 13.2 months on daraxonrasib, twice as long as 6.7 months on chemotherapy.
“It started slow. There was applause, and then some people stood up, and it just grew like this surge of noise,” said Jennifer Knox, a pancreatic cancer specialist at the Princess Margaret Cancer Centre in Toronto, who was on stage in Chicago that day to put the trial results in context. Many people were crying, she recalled, even the handler giving her stage cues.
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“Pancreas cancer is just so abysmal,” Dr. Knox said later in an interview. “All the advances that have happened in other cancers just haven’t paid off in pancreatic cancer. Then, to see something work so well, it’s emotional.”
Pancreatic cancer is a formidable opponent for a few reasons. The organ, which secretes enzymes for digestion and insulin to regulate blood sugar, is located deep in the upper abdomen behind the stomach, making it hard to see on typical scans. By the time symptoms such as abdominal pain, weight loss and late-onset diabetes begin, the cancer, which tends to be aggressive, has often spread.
Making matters worse, more than 90 per cent of pancreatic cancer cases are driven by mutations in a gene called RAS, whose protein was thought to be “undruggable.” When working properly, RAS protein controls cellular growth by toggling on and off as needed. Mutations push RAS – short for rat sarcoma, a nod to how it was discovered – into a perpetual on state, fuelling tumour growth.
Dr. Knox described RAS proteins, which also drive some cases of lung and colorectal cancer, as a “slimy ball.” Drug designers struggled to make a compound that could hook into the mutated version and shut it off.
While Revolution Medicines prepares its formal submission to the FDA, the regulator is allowing the company to give some patients daraxonrasib through an Expanded Access Program, which it is doing for free.
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For now, that stopgap measure is available only to patients in the care of U.S. physicians.
Mark Johnson, a spokesperson for Health Canada, said some Canadian physicians have asked the department about bringing daraxonrasib to Canada for individual patients through its Special Access Program, which facilitates the importation of unlicensed drugs on a case-by-case basis. But he said in a written statement that the American pharmaceutical company has refused, and Health Canada can’t compel its co-operation.
Revolution Medicines wouldn’t comment on the timing of opening expanded access programs outside the U.S.
Dana Johnson, a spokesperson for the company, said in an e-mail that patients don’t have to live in the U.S. to qualify for the FDA program, but the request would have to come from a U.S. doctor. That means any Canadians pursuing that option would have to pay for some amount of American health care.
Mr. Bourbeau is looking at rallying other patients, oncologists and patient groups to write a letter to Revolution urging them to open an access program in Canada as soon as possible. “Not just for me,” he said, “but for other Canadians that could benefit from it.”
Ms. Johnson wrote that Revolution Medicines is planning to open clinical trial sites for daraxonrasib and a related drug in Canada soon.
Dr. Knox, Dr. Zogopoulos and Daniel Renouf, a medical oncologist and researcher who treats pancreatic cancer at the BC Cancer Agency, said they expect trial locations to open in Canada in the next couple of months – a timeline they would like to see sped up.
In the meantime, there are dozens of other RAS inhibitors in early stages of testing. Some of the companies developing them have Phase 1 trials, which are small and test for safety and appropriate dosing, open in Canada.
Connor Page with his wife Jen Lovrics and their two children Carter, left, and Finnigan. Mr. Page learned he had pancreatic cancer in 2022 at the age of 39.Supplied
Jen Lovrics saw the benefits of a Phase 1 trial firsthand when her husband, Connor Page, managed to nab a spot in one based in Cincinnati, Ohio. He learned he had pancreatic cancer in 2022 at the age of 39, when the Toronto couple’s boys were 4 and not quite 2.
The investigational drug was the RAS inhibitor that would go on to be named daraxonrasib. It gave Mr. Page about six extra months in the best health since his diagnosis. He used the time to make memories with his young family – skiing, swimming, playing board games, travelling to Florida and attending his children’s activities.
“The kids have a lot of memories from that time that can never be taken away,” Ms. Lovrics said. “When you’ve got young kids, the more time you have and the more time that feels happy and normal is priceless.”
Mr. Page and his family on Father's Day in 2023. Daraxonrasib gave him about six extra months in the best health since his diagnosis.Supplied
Mr. Page died in October, 2024, at the age of 41.
Stories such as Mr. Page’s are a reminder that more needs to be done to reduce the toll of pancreatic cancer, said Michelle Capobianco, chief executive officer of the patient group Pancreatic Cancer North America.
“The reality, still, is that early detection is the only solution for pancreatic cancer,” she said. “We’re looking for six years, not just six months.”