The survival time of the combination drug treatment doubled, according to late trial data published in the New England Journal of Medicine on Friday, published at the American Society of Clinical Oncology’s annual meeting in Chicago.
The three-treatment combination includes a standard chemotherapy drug, an antibody drug called cetuximab and a pill of the Pfizer drug Braftovi, whose targeting is called BRAF V600E.
About 10% of patients with colorectal cancer have shown the mutation, said Dr. Lionel Kankeu Fonkoua, a gastrointestinal oncologist at the Mayo Clinic Comprehensive Cancer Center in Rochester, Minnesota. Patients with the mutation tend to survive less than a year after diagnosis and have poor response to standard chemotherapy treatment, Fonkoua, who is not involved in the new trial.
According to Pfizer, these patients have a risk of death more than twice as high as patients without mutations.
Braftovi was approved in 2020 with cetuximab in this group of patients after other treatment failures. The new trial treats drug combinations as so-called first-line therapy.
The Food and Drug Administration granted approval for rapid training in its first-line approach in December, provided that Pfizer provided additional data to confirm its effectiveness. The agency often grants rapid approval of treatments that address serious or life-threatening conditions, especially if medical needs are not met.
Dr. Christopher Lieu, a professor of medicine at the University of Colorado School of Medicine, was not involved in the study, saying the results were "very influential".
"Patients obviously live longer, which represents a new standard of care for this particular subset of patients with this specific mutation," Lieu said.
The trial included more than 600 patients with mutations in metastatic colorectal cancer.
Patients were randomly assigned to obtain three-drug combined treatment or standard chemotherapy alone. Some patients in the latter group also received bevacizumab, a first-line treatment for metastatic colorectal cancer.
The trial found that patients who received combination therapy survived on average about 30 months, while patients with standard chemotherapy had or did not have bevacizumab.
More importantly, 47% of patients who received combination therapy did not have disease progression after two years, meaning their cancer did not grow or spread.
This treatment is good, without unexpected safety issues, which can cause investigators to block the trial.
“It’s a very amazing discovery,” said Scott Kopetz, MD, PhD, professor of gastrointestinal medicine oncology at the Anderson Cancer Center, Texas, a co-primary investigator in the trial. “When we combine it with standard care chemotherapy, our survival rates are really long for truly unprecedented patients of these disease types.”
According to the Centers for Disease Control and Prevention, more than 141,000 new cases of colorectal cancer 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 are expected to die from colorectal cancer in the United States this year.
Laurie Ritchie, 61, of St. Louis, received joint treatment in October 2023. Her results were not included in the clinical trial analysis.
Ritchie was diagnosed with metastatic colorectal cancer with a BRAF mutation — a "huge shock."
No cancer was found in previous colonoscopy. By the time she was diagnosed, the cancer had reached its stage 4. It ended up spreading on her lungs and ovaries. Since she received combined treatment, she said her blood tests have never shown any signs of cancer.
Although she is still worried about cancer coming back, she says she is now focusing on total life, including water skiing and downhill skiing.
“I’ve learned to think of it as something in the trunk, not the front seat,” she said. “It’s still kind of like the ticking time bomb inside me, but I think my work on mental health has really helped me to endure it.”