The combination of drug treatment doubled the survival time for patients with aggressive forms of colorectal cancer, according to late stage trial data published in the New England Journal of Medicine and presented at the American Society of Clinical Oncology Annual Meeting in Chicago.
The three treatment combinations included a standard chemotherapy drug, an antibody drug called cetuximab, and a Pfizer tablet called Brffovi, which targets a cancer mutation called BRAF V600E.
Dr. Lionel Cankyu Fonkoua, a gastrointestinal oncologist at Mayo Clinic Comprehensive Cancer Center in Rochester, Minnesota, said mutations appear in about 10% of colon cancer patients. Patients with the mutation tend to survive less than a year after diagnosis and do not respond well to standard chemotherapy treatments, said Vonkoua, who is not involved in the new trial.
According to Pfizer, these patients have a risk of death more than twice as high as those without mutations.
Braffovi was originally approved in 2020 for use with cetuximab in this group of patients after other treatments have failed. The new trial considered drug combinations to be what is called first-line therapy.
The Food and Drug Administration granted rapid approval of the treatment as its first-line approach in December, provided that Pfizer provides additional data to confirm its effectiveness. Agents often grant prompt approval for treatments that address serious or life-threatening conditions, particularly when there are unmet medical needs.
Dr. Christopher Liu, a professor of medicine at the University of Colorado School of Medicine, called the results “very shocking.”
“The patients are clearly long and represent a new standard of care for this particular subset of patients with this particular mutation,” Lieu said.
The study included more than 600 patients with mutations suffering from metastatic colorectal cancer.
Patients were randomized and only received three-agent combination treatment or standard chemotherapy. Some patients in the latter group also received bevacizumab, the first-line treatment for metastatic colorectal cancer.
The trial found that patients who received combination treatment live on average about 30 months, with or without bevacizumab, compared to about 15 months in patients receiving standard chemotherapy.
Furthermore, 47% of patients receiving combination treatment had no progression of the disease after 2 years.
Treatment was well tolerated and there were no unexpected safety concerns that investigators would have stopped trials.
“It’s a truly amazing discovery,” said Dr. Scott Kopetz, a professor of gastrointestinal medical oncology at the MD Anderson Cancer Center in Texas and a co-investigator of the trial. “To put this together with standard care chemotherapy, it will survive virtually long term for these patients who are truly unprecedented about this type of disease.”
According to the Centers for Disease Control and Prevention, more than 141,000 new cases are diagnosed in the United States each year, making it the fourth most common cancer. According to the American Cancer Society, approximately 52,900 people in the United States are expected to die from colorectal cancer this year.
Laurie Richie, 61, of St. Louis, received combination treatment in October 2023. Her results were not included in the clinical trial analysis.
Richie had been diagnosed with metastatic colorectal cancer with BRAF mutations. This is the diagnosis she describes as a “giant shock.”
Previous colonoscopy did not detect cancer. By the time she was diagnosed, the cancer had already reached stage 4. It eventually spread to the lungs and ovaries. She said her blood tests have consistently shown no signs of cancer since she received combination treatment.
She is still worried about cancer coming back, but she says she is now focused on living completely, including water skiing and downhill skiing.
“I learned to think of it as something in the trunk, not as a front seat,” she said. “It still feels a bit like a time bomb inside me, but I think the work I did in my mental health really helped me live with it.”
