Experimental pancreatic cancer drug offers new hope in major trial
An experimental drug for metastatic pancreatic cancer nearly doubled overall survival in a Phase 3 clinical trial, offering new hope for patients with one of the deadliest forms of cancer.
The findings, presented Sunday at the American Society of Clinical Oncology annual meeting and published simultaneously in the New England Journal of Medicine, showed that patients who received the investigational drug daraxonrasib lived, on average, about six months longer than those who received chemotherapy. The drug is not yet approved by the U.S. Food and Drug Administration.
"I think many of us would consider this a big win," Dr. Brian Wolpin, director of the Hale Family Center for Pancreatic Cancer Research and Gastrointestinal Cancer Center at Dana-Farber Cancer Institute, said in an interview with "Good Morning America" aired on Monday.
The global Phase 3 trial enrolled 500 patients with metastatic pancreatic cancer who had previously received one line of chemotherapy for metastatic disease.
Researchers found patients treated with daraxonrasib had a median overall survival of 13.2 months, compared with 6.7 months for those receiving chemotherapy. The treatment also reduced the risk of death by 60%.
Daraxonrasib is an oral targeted therapy designed to block RAS proteins, which play a key role in driving pancreatic cancer growth in most cases.
"The trial is trying to help people live as long as they can and actually live better with less side effects, less symptoms from the cancer, and that is a very big deal," Wolpin said.
Pancreatic cancer is among the deadliest cancers, in part because it is often diagnosed after it has already spread beyond the pancreas. Few patients with metastatic disease survive beyond a year, and treatment options after initial chemotherapy remain limited.
For patients like Jim, who participated in the clinical trial, the drug has helped provide renewed optimism.
"It's news that can set you back or it can push you forward," Jim told "GMA" of his diagnosis.
After beginning treatment with daraxonrasib, Jim said a tumor in his liver that had spread from his pancreas shrank significantly.
"The tumor that I had in my liver, which had spread from the pancreas, got reduced by 50%, which was incredible news," he said.
The improvement has allowed him and his family to start making plans again.
"We are now back in the mode of rescheduling trips," Jim said.
Carla Walker, another patient enrolled in the trial, told "GMA" the medication has reduced her tumors by 40% while keeping the mass in her pancreas stable.
She now focuses on spending time with her grandsons and looks forward to future milestones.
"I would love, love to make it to their graduation from high school, and that's like a long-term goal, and that's what I look forward to. I'm going to be there, no matter what," Walker said.
Researchers say the findings could represent a major shift in the treatment of metastatic pancreatic cancer if the drug receives FDA approval.
"This is the first RAS inhibitor evaluated in a large, randomized trial for patients with pancreatic cancer, and it demonstrates how important an impact these novel medicines are likely to have on the treatment of the disease," Wolpin said in a press release issued Monday to announce the result of the trial.
Earlier this month, the FDA granted permission for an expanded access program that allows some patients with previously treated metastatic pancreatic cancer to receive daraxonrasib while the drug continues through the regulatory review process.
"It is exciting to see that we may soon be able to help patients with metastatic pancreatic cancer in ways we haven't been able to before, improving both survival and quality of life," Wolpin added in the release.
