By Elodie Vaz | Published on March 27, 2026 | 4 min read
Hepatoblastoma
is the most common liver cancer in children. Although standard treatment
combining chemotherapy and surgery now cures about 80% of patients, some forms
remain particularly aggressive. These tumors are characterized by rapid
proliferation and resistance to conventional therapies, leaving clinicians with
limited options for poor-prognosis cases.
In this
context, a team from Inserm and the University of Bordeaux, led by Christophe
Grosset at the Bordeaux Institute of Oncology, has explored a new therapeutic
strategy published in Springer Nature on February 26. The objective:
eliminate resistant tumor cells by combining an EZH2 inhibitor (a protein
involved in epigenetic regulation) with statins, drugs commonly prescribed for
hypercholesterolemia.
A
combinatorial approach grounded in tumor biology
The
rationale behind this strategy relies on two complementary mechanisms. On the
one hand, EZH2 acts on chromatin by modifying DNA organization. As Christophe
Grosset, lead author of the study, explains: “it adds a tiny ‘chemical tag,’
the methyl group, onto a protein called histone H3, around which DNA is
wrapped. This modification changes chromatin organization by compacting it and
prevents the expression of certain genes, notably tumor suppressor genes, which
normally act as brakes on cancer development.”
On the
other hand, statins target cellular metabolism. “They block the production of
mevalonate, a molecule that cells use to grow, survive, and move. By reducing
mevalonate levels, statins slow cancer cell growth and migration, increase
their sensitivity to chemotherapy, and may help the immune system better
eliminate the tumor,” he adds.
Promising
results in preclinical model
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.
Read next: Liver, sugar, and pills: who's in
control?
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.
