Nivolumab Shows Promise as a Treatment Option for Advanced Liver Cancer

ASCO Annual Meeting
May 29, 2015

In an early-stage study, nivolumab (Opdivo) has shown encouraging results as a treatment for advanced liver cancer. Liver cancer is the leading cause of cancer deaths worldwide, accounting for more than 600,000 deaths each year. People diagnosed with advanced liver cancer especially need new treatment options, as there is currently only one drug approved by the U.S. Food and Drug Administration (FDA).

Nivolumab is a type of immunotherapy, which is a treatment designed to boost the body's natural defenses to fight the cancer. Nivolumab helps improve immune system function by blocking a protein called PD-1. PD-1 is found on the surface of T-cells, which are a type of white blood cell that directly helps the body’s immune system fight disease. Because PD-1 keeps the immune system from destroying cancer cells, stopping PD-1 from working allows the immune system to better eliminate the disease.

As part of this study, 42 people with advanced hepatocellular carcinoma (HCC), the most common type of liver cancer diagnosed in adults, received nivolumab. Overall, 19% of the participants had tumors that responded to the treatment. Eight people had tumors that shrank by more than 30%, and two had complete remissions. A remission is when cancer cannot be detected in the body and there are no symptoms. These benefits lasted for more than a year for four participants. In addition, nivolumab stopped tumors from growing for 48% of the participants.

The one-year survival rate was 62%. The one-year survival rate is the percentage of people who live at least one year after starting treatment.

What this means for patients

“We are encouraged to see that the response rate, as well as preliminary survival data, look quite promising,” said lead study author Anthony B. El-Khoueiry, MD, an associate professor of clinical medicine and the Phase I Program Director at the University of Southern California Norris Comprehensive Cancer Center in Los Angeles. “While we have to verify this early signal in larger studies, this is one of the first signs that immunotherapy with immune checkpoint inhibitors will have a role in the treatment of liver cancer.”

In this study, treatment with nivolumab caused mild to moderate side effects. The most common side effects were elevated liver enzyme levels, rash, and high levels of amylase and lipase. However, abnormal liver enzymes and elevated amylase and lipase levels did not cause any significant symptoms.

Nivolumab is currently only FDA-approved to treat advanced melanoma that has not responded to other standard treatments and advanced squamous non-small cell lung cancer. Talk with your doctor about all of your treatment options, including participating in a clinical trial.

Questions to ask your doctor

  • What stage of liver cancer do I have? What does this mean?
  • What are my treatment options?
  • What clinical trials are open to me? Where are they located, and how do I find out more about them?
  • What treatment do you recommend? Why?
  • What are the possible side effects of each treatment, and how can they be managed?

More Information

Guide to Liver Cancer

What is Immunotherapy?