Research could help clinicians identify patients who may benefit from targeted therapy for liver cancer

New research from Roswell Park Cancer Institute offers clinicians treating patients with advanced liver cancer a way of determining which patients may benefit most from the targeted therapy sorafenib.

"Our study is the first to demonstrate the potent immunotherapeutic benefits of sorafenib and to suggest that this targeted treatment may extend survival among a subset of patients with liver cancer," says the paper's senior author, Yasmin Thanavala, PhD, Professor in the Department of Immunology and Member of the Tumor Immunology and Immunotherapy program at Roswell Park. "The drug has significant antiangiogenic properties, and also appears to beneficially impact the suppressed immune system of patients with advanced liver cancer."

Looking at blood samples from patients with advanced liver cancer, researchers evaluated the frequency and expression levels of several immunosuppressive cells and molecules before and after treatment with sorafenib. The team found a statistically significant reduction of the checkpoint molecule PD-1 and levels of its expression on important immune cells known as "helper," "killer" and regulatory T cells, and that this response was strongly linked to the patients' overall survival. They also observed a beneficial increase in the ratio of T effector cells to T regulatory cells following treatment with sorafenib. The findings suggest that sorafenib reduces the number of immune-suppressing cells in patients who respond to therapy, and that the combination of this targeted agent with immune-based therapies may improve treatment outcomes in patients with liver cancer.

"These results indicate that patients with an increased number of cells expressing the checkpoint molecule PD-1 before treatment are more responsive to sorafenib therapy. These increased numbers in pretreatment blood samples may be a biomarker indicating which patients will respond better to therapy and may help to predict overall patient survival," adds Dr. Thanavala.