Drug Everolimus Does Not Improve Overall Survival in Patients with Advanced Liver Cancer

“Despite strong preclinical data, the drug everolimus failed to improve overall survival in patients with advanced liver cancer, compared to placebo, according to a study in the July 2 issue of JAMA.

“Patients with advanced hepatocellular carcinoma (HCC; a type of liver cancer) have a median overall survival of less than l year, largely because of the absence of effective therapies. The drug sorafenib is the only systemic therapy shown to significantly improve overall survival in advanced HCC; however its benefits are mostly transient and modest, and disease eventually progresses. In preclinical models, everolimus prevented tumor progression and improved survival, according to background information in the article.

“Andrew X. Zhu, M.D., Ph.D., of the Massachusetts General Hospital Cancer Center, Harvard Medical School, Boston, and colleagues randomly assigned 546 adults with advanced HCC whose disease progressed during or after sorafenib or who were intolerant of sorafenib to receive everolimus (n = 362) or placebo (n = 184), both given in combination with best supportive care and continued until disease progression or intolerable toxicity. In this phase 3 study, patients were enrolled from 17 countries between May 2010 and March 2012.

“The researchers found no significant difference in overall survival between the two groups…”

Editor’s note: This article describes a clinical trial with volunteer patients to test a potential new liver cancer drug called everolimus. Patients in the trial all had advanced hepatocellular carcinoma (HCC) and had been unsuccessfully treated with sorafenib. Half were given everolimus, and for comparison, half were given a “fake” placebo drug. Unfortunately, there was no significant difference in survival between the two groups.