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FDA Approves Groundbreaking Drug for Pancreatic Cancer, Offering New Hope for Older Adults
In a clinical trial, daraxonrasib extended survival for patients with advanced disease, a rare breakthrough for one of the deadliest cancers
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Key takeaways
- Pancreatic cancer is one of the leading causes of cancer death in the U.S., and the vast majority of cases affect adults 50 and older.
- A phase 3 trial found that the drug daraxonrasib doubled the median survival time of patients with advanced pancreatic cancer.
- Patients on the drug also reported less pain and a better quality of life.
The Food and Drug Administration has approved daraxonrasib, a new drug for patients with metastatic pancreatic cancer that experts are calling a major advance against one of the most aggressive and deadly forms of cancer.
In a clinical trial, the daily pill, which will be sold under the brand name Rasonque, doubled median survival time for patients with pancreatic cancer, compared with chemotherapy, the current standard of care when the disease has metastasized.
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“It’s a major breakthrough,” says Dr. Eileen M. O’Reilly, a gastrointestinal medical oncologist at New York’s Memorial Sloan Kettering Cancer Center and an author on that landmark study, published in May in The New England Journal of Medicine. “The field, I think, is very optimistic that this is going to lead to significant improvements in outcomes for people with this disease.”
The challenge of treating pancreatic cancer
Pancreatic cancer is the third leading cause of cancer death in the U.S. One of the reasons it’s so deadly is that there are very few treatment options that can effectively target and attack the cancer’s unique and aggressive cells, says Dr. Olatunji B. Alese, a medical oncologist at Emory University’s Winship Cancer Institute in Atlanta, who specializes in the treatment of gastrointestinal cancers.
Pancreatic cancer cells are often shielded under fibrous tissue that is hard for drugs, like chemotherapy, to penetrate. What’s more, they can be resistant to many treatments, which helps explain why a large proportion of clinical trials in the past 25 years have failed.
There’s also no routine screening for pancreatic cancer to catch it in its early, more treatable stages, as there is for other cancers, “which means about 80 percent of patients also [come] to the clinic for the first time with advanced cancer that cannot be removed,” Alese says.
According to the American Cancer Society, almost all patients are older than 45; about two-thirds are at least 65. An estimated 67,530 people will be diagnosed with pancreatic cancer this year, and about 52,740 people will die from the disease.
Targeting a common mutation
The abnormality on Helene Rubin’s pancreas showed up during a routine lung cancer screening. At the time, she wasn’t overly worried. It appeared to be a cyst, “and nobody thought it was cancerous,” says Rubin, 83, who lives in northern New Jersey.
But when doctors removed it four months later, she got unsettling news: It was pancreatic cancer.
Six months of chemotherapy kept the cancer at bay for about a year. Then Rubin learned the disease had spread to her lungs — a diagnosis that for many patients means they may have mere months to a year to live. Just 3 percent of patients with metastatic pancreatic cancer live five years after a diagnosis. Rubin’s doctor suggested she enroll in a clinical trial to improve her odds.
The daraxonrasib trial that Rubin began in February at Memorial Sloan Kettering Cancer Center rocked the world of cancer medicine. In the 500-person study, median overall survival was 13.2 months in the group randomly selected to get daraxonrasib and 6.7 months in the group receiving chemotherapy.
What’s more, 31.6 percent of the patients taking daraxonrasib had their tumors shrink or disappear, compared with 11.2 percent of those receiving chemotherapy.
Rubin says after about two months on the medication, her largest lung nodule was “dramatically” smaller. “We were all so happy about that,” she says.
Helene Rubin is taking the drug daraxonrasib for treatment of pancreatic cancer. In a recent clinical trial, the medication doubled the median survival time for patients.
Courtesy Helene Rubin
Daraxonrasib works by targeting KRAS, a gene that can have a mutation that's found in more than 90 percent of pancreatic cancers.
In healthy cells, KRAS acts as an on-off switch for cell growth. In mutated cells, however, KRAS is stuck in the “on” position, causing the cancer cells to grow unchecked. Daraxonrasib binds to the mutation like glue and switches KRAS from on to off.
“It works by blocking the mutation that is so vital to the survival of the pancreatic cancer cells,” Alese says. Until now, no therapies have effectively targeted KRAS.
Study participants taking daraxonrasib also reported less pain and fatigue, as well as a better quality of life. Still, the drug can cause side effects, including rash and gastrointestinal problems, though experts say it’s still more tolerable than chemotherapy.
Rubin was briefly hospitalized with gastrointestinal side effects when she first started daraxonrasib, but she says she hasn’t had any issues since her dose was adjusted and prefers it to chemotherapy.
“I didn’t want to go through [chemo] again,” she says. “I took it pretty hard.”
Potential beyond pancreatic cancer
Experts say daraxonrasib’s promise extends beyond pancreatic cancer. KRAS is a member of the larger RAS family, and RAS mutations also appear in several other cancers, including lung and colorectal cancer — the two leading causes of cancer death — as well as ovarian and endometrial cancers, among others.
“It’s one of the commonest abnormalities that’s seen in cancer in general,” O’Reilly says.
Alese says, “That’s a huge number of patients in the United States who [may] benefit from that approach.”
Clinical trials evaluating daraxonrasib’s effect on other cancers are already underway, Alese says, as are studies evaluating other RAS inhibitors for pancreatic cancer and other tumor types.
Researchers are also studying daraxonrasib’s effect on earlier stages of pancreatic cancer, and in combination with other treatments, such as chemotherapy or immunotherapy.
“It’s an exciting time in pancreatic cancer treatment, but it’s still sobering that despite the remarkable outcomes from this trial, the median overall survival for the patients who got daraxonrasib was just 13.2 months,” Alese says. “There’s still a lot we have to do.”
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