Hepatoblastoma: a combined strategy to target resistant forms

Promising results in preclinical models




The researchers first showed that the EZH2 protein is overexpressed in the most aggressive hepatoblastomas, confirming its potential role in tumor progression. They then tested their approach in murine models of the disease.  

“Combined with statins, our EZH2 inhibitor effectively destroyed cancer cells and blocked tumor growth in mice,” reports Christophe Grosset. This efficacy was also observed in vitro in other cancer types, including osteosarcoma and lung cancer cells.  




A promising avenue to be confirmed in humans  





These preclinical data open new therapeutic perspectives for the most aggressive forms of hepatoblastoma. They are part of a broader research effort on EZH2 inhibitors, already being explored in several cancers.  

As Christophe Grosset points out: “since the approval in 2020 in the United States of the first anti-EZH2 treatment for epithelioid sarcoma, a rare and aggressive soft tissue tumor, numerous studies are underway worldwide to evaluate the effectiveness of this therapeutic strategy against other cancers.”  

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



  Toward more targeted medicine in pediatric oncology  





Beyond hepatoblastoma, this study illustrates the growing interest in combinatorial approaches targeting both the epigenetic and metabolic mechanisms of tumor cells. It also highlights the potential for drug repurposing, such as statins, in oncology.  

If these results are confirmed in clinical settings, this strategy could expand the therapeutic arsenal against resistant pediatric cancers. This is particularly important given the limited options currently available for these patients and the major public health challenge posed by innovation in pediatric oncology.       



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About the Author – Elodie Vaz
Health journalist, CFPJ graduate (2023).
Élodie explores the marks diseases leave on bodies and, more broadly, on human life. A registered nurse since 2010, she spent twelve years at patients’ bedsides before exchanging her stethoscope for a notebook. She now investigates the links between environment and health, convinced that the vitality of life cannot be reduced to that of humans alone.