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Scientific Publications and Presentations

Summary of research article published in a scientific journal about fibrolamellar carcinoma:

Article title:
Serum Procalcitonin: A Novel Tumor Biomarker for Diagnosis and Follow-Up in Fibrolamellar Hepatocellular Carcinoma
Date of publication:

November 20, 2025

Authors:
Jean-Charles Nault, Claudia Campani, Theo Z Hirsch, Ethan Neumann, Waqar Arif, Sandrine Imbeaud, Marina Baretti, Marianne Ziol, Sabrina Sidali, Pauline Roger, Manon Allaire, Mohamed Bouattour, Fabio Marra, Brice Fresneau, Neus Llarch, Jean-Marie Peron, René Gerolami, Eric Nguyen Khac, Pierre Nahon, Nathalie Ganne-Carrié, Julien Caldéraro, Aurélie Beaufrère, Valérie Paradis, Catherine Guettier, Angela Sutton, Mark Yarchoan, Jessica Zucman-Rossi
Publication description:
Pre-print of scientific article in MedRxiv

Doctors have long struggled to diagnose and monitor FLC partially because no blood tests (biomarkers) work reliably for it. Standard liver cancer markers like alpha-fetoprotein (AFP) are almost always normal in FLC.

This study reports a new and promising discovery: procalcitonin (PCT), a blood marker normally used to detect bacterial infections, turns out to be highly elevated in most patients with FLC and could serve as both a diagnostic and treatment‑monitoring biomarker.

Key findings of the study

  • Procalcitonin levels are unusually high in patients with FLC.
    • Based on the analysis of data from two independent patient groups (Europe and the U.S.), 83% of people with FLC had elevated PCT, far higher than infection-related levels.
    • In contrast, only about 3% of people with other liver cancers (classic HCC or cholangiocarcinoma) had high PCT.
  • FLC tumors produce procalcitonin.
    • RNA sequencing showed the gene that produces PCT (CALCA) is highly overexpressed in FLC, not in other liver tumors.
    • Spatial transcriptomics, a technique that reveals the specific cells in a tissue sample that express an active gene, confirmed that CALCA is active in tumor cells that carry the FLC fusion gene (DNAJB1‑PRKACA).
    • Immunohistochemistry (protein staining) found PCT protein inside tumor cells in 77% of FLC cases, and 0% of other liver cancers.
  • In four patients with repeated PCT measurements, changes in PCT levels correlated with tumor response. This suggests PCT could be a simple blood test to monitor treatment efficacy.
    • When tumors shrank in response to therapy, PCT dropped.
    • When tumors grew or progressed, PCT rose.

Implications

FLC has no reliable blood biomarker today. Most patients with metastatic FLC have dramatically elevated PCT levels, and these levels rise and fall with treatment response. Because FLC tumors produce PCT, the measurement of PCT levels could potentially serve as both a diagnostic tool and a monitoring tool for the disease.

The preprint of the full article can be accessed here.

This effort was partially funded by FCF.

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