When researchers announced that a four-drug chemotherapy regimen can add years to the lives of some patients with earlier-stage pancreatic cancer, doctors didn't wait.
Trial results were released last spring, and were immediately "practice-changing," said Dr. Hedy Kindler, an oncologist at the University of Chicago who was not involved in the trial.
Dr. James Biagi, one of the researchers on the trial, made the same point. "The standard of care changed overnight," said Biagi, an associate professor of oncology at Queen's University in Kingston, Canada.
Experts say the findings represent major progress against a highly lethal disease.
In the study of nearly 500 patients with earlier-stage pancreatic tumors, researchers found that post-surgery chemotherapy with four drugs substantially extended patients' typical survival.
By the end of the study, the median survival rate among those patients was 54 months -- or 4.5 years. "Median" refers to the midpoint on the survival spectrum, so half the patients survived longer than that, while half died sooner.
In the world of pancreatic cancer treatment, the results are "remarkable," said Kindler.
"We've never seen a median survival of 54 months in this disease," she said. "That's far beyond what we expected."
Kindler wrote an editorial published with the study in the Dec. 20 issue of the New England Journal of Medicine.
The trial results were first released at the annual meeting of the American Society of Clinical Oncology in June.
"We all went home and changed how we treat this group of patients," Kindler said. "We didn't wait."
Still, Biagi and Kindler tempered their enthusiasm by citing some stark figures. Only about 20 percent of people diagnosed with pancreatic cancer are even able to have surgery. For most patients, the disease has already spread too extensively for surgery to be an option.
So the majority of pancreatic cancer patients do not stand to benefit from this approach.
In the United States, it's estimated that roughly 55,400 people will be diagnosed with pancreatic cancer this year, according to the American Cancer Society. More than 44,000 will die of the disease.
Pancreatic cancer carries a grim prognosis, in part, because it is often diagnosed late. There is no screening test for it, and people often have no symptoms until the cancer has spread, the cancer society says.
The only chance for a cure, Biagi said, is if the disease is caught early enough for the tumor to be surgically removed. The standard of care has been to follow surgery with six months of chemotherapy -- with a drug called gemcitabine.
But even then, 69 percent to 75 percent of patients have a recurrence within two years, according to Biagi's team.
In their trial, the researchers tested the effects of a four-drug chemo regimen versus standard chemo with gemcitabine. The regimen is a modified version of one already used for advanced pancreatic cancer. At that point, there is no hope for a cure -- but research shows that the regimen (called FOLFIRINOX) can help those patients live longer.
Biagi's team randomly assigned 493 patients to six months of either standard chemo or FOLFIRINOX following their surgery.
After three years, 63 percent of patients given the chemo cocktail were still alive, and 40 percent were free of a recurrence. Of patients given standard chemo, just under 49 percent were alive, and 22 percent were recurrence-free.
The overall median survival in the FOLFIRINOX group was 54 months -- compared with 35 months in the standard-care group.
The four-drug chemo came at a cost of more side effects, however. Three-quarters of patients had more serious "grade 3 or 4" side effects, versus 53 percent of those on standard chemo.
Those side effects included diarrhea, nausea and vomiting, fatigue and nerve damage in the limbs.
Not every patient who has surgery for pancreatic cancer would be a candidate for FOLFIRINOX, according to Biagi. The chemo needs to begin within 12 weeks of surgery, so patients have to recover well enough to tolerate that.
Ultimately, Biagi and Kindler said, researchers need to find ways to catch pancreatic cancer earlier -- and keep digging into the underlying biology of the disease, to develop more precise treatments.
"This new [chemotherapy] standard is an important step forward," Biagi said. "But it's not a complete answer."