Email Newsletter, Volume 1, Issue 1

Investigational drug improves survival for colorectal cancer patients

Axel Grothey, M.D.

An investigational drug called regorafenib slowed the progression of tumors and increased survival of patients with metastatic colorectal cancer, an international phase III trial found. The findings were presented earlier this year at the Gastrointestinal Cancers Symposium in San Francisco by Mayo Clinic Cancer Center oncologist Axel Grothey, M.D., principal investigator of the trial in the United States.

"For years, patients with metastatic colorectal cancer have faced a devastating impasse when standard chemotherapies have failed to halt the growth of tumors and physicians have run out of effective drugs to offer them," Dr. Grothey said. "This is the first novel agent in eight years to show improvement in overall survival of colon cancer patients who have run out of treatment options."

Researchers tested regorafenib in a phase III, randomized, placebo-controlled trial, conducted simultaneously in the United States, Europe, Japan, Australia and China. They looked at survival outcomes in 760 patients whose cancer had progressed despite standard chemotherapy regimens. Regorafenib, a multikinase inhibitor, slows cell proliferation and blood vessel growth and tempers a variety of biological pathways that are activated in tumors.

Researchers found that patients with metastatic colon cancer who were treated with the drug showed a 29 percent increase in overall survival when compared with those treated with placebo. The median length of survival for patients treated with the drug increased from five months to 6.5 months, a statistically significant jump. Overall, regorafenib reduced patients' risk of dying of cancer during the trial by 23 percent.

The trial, whose largest group of study participants in the United States was at Mayo Clinic, finished more than a year ahead of schedule.

"Patients with metastatic colorectal cancer who have failed all approved standard therapies have a poor prognosis," Dr. Grothey said. "This is the first and only agent in this setting that has demonstrated statistically significant overall survival benefit."

Mayo Clinic Cancer Center researcher Daniel J. Sargent, Ph.D., the Ralph S. and Beverley E. Caulkins Professor of Cancer Research, also participated in this study. This research was sponsored by Bayer Healthcare.

Watch a video of Dr. Grothey discussing this study.