New Treatment Shows Promise Against Childhood Liver Cancer (Hepatoblastoma)

Researchers in France have identified a promising new therapeutic approach for hepatoblastoma, the most common malignant liver tumor in children, combining an EZH2 inhibitor with statins. The findings, published March 19 in Molecular Cancer, demonstrate successful elimination of cancer cells in mouse models and suggest potential efficacy against other cancers.

Hepatoblastoma affects approximately one in one million children under the age of 15, resulting in 10 to 15 new cases annually in France, according to data from Université Paris Cité. Current treatment typically involves a combination of chemotherapy and surgical removal of the tumor, achieving successful outcomes in around 80 percent of cases. However, aggressive forms of the disease, characterized by rapid cell proliferation, present a significant challenge.

The research team, from the Inserm and the University of Bordeaux, focused on the protein EZH2, which was found to be overexpressed in the most aggressive hepatoblastoma cases. They administered a chemical compound designed to neutralize EZH2 action to mouse models of the disease. “Coupled with statins, our EZH2 inhibitor effectively destroyed cancer cells and blocked tumor growth in mice,” explained Christophe Grosset, a research director at Inserm’s Institute of Oncology in Bordeaux.

Notably, the combination therapy also yielded positive results when tested on osteosarcoma (a bone cancer affecting children and young adults) and lung cancer cells cultivated in a laboratory setting, indicating a potentially broader application.

The strategy builds on the 2020 approval in the United States of the first anti-EZH2 treatment for epithelioid sarcoma, a rare and aggressive soft tissue tumor. Numerous studies are currently underway globally to assess the effectiveness of EZH2 inhibition against other cancers, including prostate cancer and certain lymphomas.

“Our results in mice suggest that the combination of this EZH2 inhibitor with statins could be promising for the most aggressive hepatoblastomas,” Grosset stated. “However, further preliminary work will be necessary to confirm its efficacy and safety in humans.”