COLUMBUS, Ohio – Treatment with the immunotherapeutic nivolumab (Opdivo) improved survival for patients with recurrent or metastatic head and neck squamous cell carcinoma that progressed after platinum-based chemotherapy compared with single-agent chemotherapy of the investigator’s choice, according to results from the CheckMate-141 phase III clinical trial presented at the American Association for Cancer Research (AACR) annual meeting Tuesday April 19, 2016.

“Recurrent or metastatic head and neck squamous cell carcinoma that is not responsive to platinum-based chemotherapy progresses very rapidly, and patients have a very poor prognosis,” said Maura L. Gillison, MD, PhD, a professor in the Department of Internal Medicine at The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute. “Treatment usually involves single-agent chemotherapy. However, no therapy has been shown to improve survival for this patient population. New treatment options are desperately needed.

“This study is the first randomized clinical trial to clearly demonstrate improved overall survival for patients with platinum-refractory recurrent or metastatic head and neck squamous cell carcinoma,” continued Gillison. “We hope that the results will establish nivolumab as a new standard of care option for this patient population and thereby fulfill a huge unmet need.”

CheckMate-141 was a randomized, phase III clinical trial designed to determine whether the PD-1 inhibitor nivolumab could extend overall survival for patients with platinum-refractory recurrent or metastatic head and neck squamous cell carcinoma compared with treatment of the investigator’s choice, which was any of the commonly used therapeutics docetaxel, methotrexate, or cetuximab.

Of the 361 patients enrolled in the clinical trial, 240 were randomly assigned to nivolumab and 121 to single-agent chemotherapy of investigator’s choice.

At the interim analysis, which was conducted after 218 events, patients assigned to nivolumab were found to have a 30 percent reduction in risk of death compared with those assigned therapy of investigator’s choice. Median overall survival was 7.5 months for those assigned nivolumab versus 5.1 months for those assigned therapy of investigator’s choice. At 12 months, 36 percent of the patients treated with nivolumab were alive compared with 17 percent of those assigned therapy of investigator’s choice.

For patients assigned nivolumab, the overall response rate (ORR, which is the proportion of patients who had a partial or complete response, as defined by RECIST 1.1 criteria) was 11.7 percent, with six complete responses and 22 partial responses, and for those assigned therapy of investigator’s choice, the ORR was 7.4 percent, with one complete response and eight partial responses.

Because certain types of head and neck squamous cell carcinoma, particularly those arising in the oropharynx (back of the throat, including the base of the tongue and tonsils), have been linked with human papillomavirus (HPV) infection, the investigators also evaluated the data based on the HPV status of the patients’ tumors.

The effect of nivolumab on overall survival was seen for both patients with HPV-positive disease and those with HPV-negative disease. Among patients with HPV-positive disease, median overall survival was 9.1 months for those assigned nivolumab versus 4.4 months for those assigned therapy of investigator’s choice, and among patients with HPV-negative disease, median overall survival was 7.5 months for those assigned nivolumab versus 5.8 months for those assigned therapy of investigator’s choice.

A survival benefit in patients treated with nivolumab was observed for the overall study population. Exploratory analysis suggested that the benefit was greater for patients treated with nivolumab whose tumors had PD-L1 expression (of 1 percent or greater) or were HPV-positive.

“Overall, our data are extremely exciting. This clinical trial has established head and neck squamous cell carcinoma as responsive to immunotherapy. We are hopeful that this represents the tip of the iceberg with regard to future benefit of immunotherapy for patients with head and neck squamous cell carcinoma,” added Gillison.

Please note that some numbers in this news release differ from those in the submitted abstract. Those in the news release are the most recent data available.

This study was funded by Bristol-Myers Squibb. Gillison’s role in the study was funded in part by the Oral Cancer Foundation. Gillison has consulted for Bristol-Myers Squibb, Eli Lilly and Company, and Merck in the past year.

# # #

About the OSUCCC – James

The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute strives to create a cancer-free world by integrating scientific research with excellence in education and patient-centered care, a strategy that leads to better methods of prevention, detection and treatment. Ohio State is one of only 45 National Cancer Institute-designated Comprehensive Cancer Centers and one of only four centers funded by the NCI to conduct both phase I and phase II clinical trials on novel anticancer drugs. As the cancer program’s 306-bed adult patient-care component, The James is one of the top cancer hospitals in the nation as ranked by U.S. News & World Report and has achieved Magnet designation, the highest honor an organization can receive for quality patient care and professional nursing practice. At 21 floors with more than 1.1 million square feet, The James is a transformational facility that fosters collaboration and integration of cancer research and clinical cancer care.

About the American Association for Cancer Research

Founded in 1907, the American Association for Cancer Research (AACR) is the world’s first and largest professional organization dedicated to advancing cancer research and its mission to prevent and cure cancer. AACR membership includes more than 35,000 laboratory, translational, and clinical researchers; population scientists; other health care professionals; and patient advocates residing in 104 countries. The AACR marshals the full spectrum of expertise of the cancer community to accelerate progress in the prevention, biology, diagnosis, and treatment of cancer by annually convening more than 30 conferences and educational workshops, the largest of which is the AACR Annual Meeting with nearly 19,300 attendees. In addition, the AACR publishes eight prestigious, peer-reviewed scientific journals and a magazine for cancer survivors, patients, and their caregivers. The AACR funds meritorious research directly as well as in cooperation with numerous cancer organizations. As the Scientific Partner of Stand Up To Cancer, the AACR provides expert peer review, grants administration, and scientific oversight of team science and individual investigator grants in cancer research that have the potential for near-term patient benefit. The AACR actively communicates with legislators and other policymakers about the value of cancer research and related biomedical science in saving lives from cancer. For more information about the AACR, visit www.AACR.org

MEDIA CONTACT:

Amanda J. Harper
Director, Media Relations, OSUCCC – James
614-685-5420 (direct)
614-293-3737 (media main)

*News release modified, as originally written by Karen Honey, AACR Senior Communications Manager.