CHICAGO (FRONTLINE MEDICAL NEWS) – Immune checkpoint inhibition with nivolumab every 2 weeks provides a better response than does standard treatment for advanced liver cancer, phase I/II results suggest.

The overall response rate was 19%, with 8 of the 42 evaluable patients experiencing at least 30% tumor shrinkage.

Moreover, responses to nivolumab (Opdivo) have been durable, with 62% of patients still alive at 12 months, Dr. Anthony El-Khoueiry reported in a press briefing in advance of the formal presentation of the study at the annual meeting of the American Society of Clinical Oncology.

In contrast, just 2% of patients have at least 30% tumor shrinkage with sorafenib (Nexavar) and the average overall survival is about 10-11 months. Sorafenib , a multitargeted tyrosine kinase inhibitor, is currently the only Food and Drug Administration–approved systemic treatment for advanced liver disease, he noted.

Nivolumab, a programmed death-1 (PD-1) inhibitor, is approved for previously treated melanoma and gained a second indication in March for use in previously treated squamous non–small cell lung cancer.

“This is the first study to show antitumor activity of a PD-1 immune checkpoint inhibitor in patients with liver cancer,” said Dr. El-Khoueiry of the University of Southern California Norris Comprehensive Cancer Center, Los Angeles.The durability of those responses was particularly impressive, Dr. Peter Paul Yu, ASCO president, said in an interview.

“This is a small study, but the signal is unusually robust in comparison to what the standard of care would be, which is why this is so promising,” he added.

Press briefing moderator Dr. Lynn Schuchter , chief of hematology-oncology at University of Pennsylvania in Philadelphia, said in a statement, “The fact that this drug might stop advanced liver cancer in its tracks for months, even a year, is great news for patients. To understand the full impact of this approach, however, larger trials are needed.”

Hear more about the promising results from the late-breaking abstract in our interview with Dr. El-Khoueiry .

The study was funded by Bristol-Myers Squibb. Dr. El-Khoueiry reported financial relationships with Bristol-Myers Squibb, GlaxoSmithKline, Genentech/Roche, Amgen, Exelixis, AstraZeneca, and Astex Pharmaceuticals. Several coauthors also had ties to BMS including employment and/or stock ownership. Dr. Schuchter reported institutional research funding from BMS, Genentech, GSK, Merck, and Roche.

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