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Updated: Jun 12, 2026

A Three-Dimensional Spheroid Model to Investigate the Tumor-Stromal Interaction in Hepatocellular Carcinoma
Published on: September 30, 2021
Molecular approaches to treatment of hepatocellular carcinoma
Anja Lachenmayer1, Clara Alsinet, Charissa Y Chang
1Liver Cancer Program, Division of Liver Diseases, Mount Sinai School of Medicine, New York, USA.
Abstract:
Hepatocellular carcinoma (HCC) remains a highly complex disease resistant to commonly used chemotherapy and radiotherapy. As the sixth most common cancer worldwide with the third highest mortality rate and very poorly understood molecular pathways driving hepatocarcinogenesis, new treatment strategies are urgently needed for this devastating disease. The multikinase inhibitor sorafenib was the first molecular targeted drug in HCC that led to significant survival benefit in patients with advanced tumors. It is the first drug to be considered standard of care for advanced HCC and supports the importance of molecular therapies in the treatment of this cancer. Analyses of genetic and epigenetic alterations as well as different molecular pathways involved in the development of HCC help to identify potential new druggable targets. A variety of novel compounds are already under preclinical or clinical investigation, and accumulating evidence suggests that combination therapy targeting different pathways will potentiate anti-tumoral effects and will become the future therapeutic approach. In addition the establishment of a robust molecular classification will pave the way for a more personalized treatment scheme in HCC. In this article we review the current knowledge of the molecular pathogenesis of HCC and provide an overview of molecular targeted therapies in the management of HCC.
Insights
Hepatocellular carcinoma (HCC) is a complex cancer needing new treatments. Molecular targeted therapies, like sorafenib, show promise for advanced HCC, with combination therapies and molecular classification guiding future personalized medicine.
Area of Science:
- Hepatocellular carcinoma (HCC) research
- Molecular oncology
- Cancer therapeutics
Background:
- Hepatocellular carcinoma (HCC) is a leading cause of cancer mortality with poorly understood molecular drivers.
- Current treatments for HCC, including chemotherapy and radiotherapy, show limited efficacy.
- Sorafenib, a multikinase inhibitor, represents a significant advancement as the first molecular targeted therapy for advanced HCC.
Purpose of the Study:
- To review the current understanding of molecular pathogenesis in HCC.
- To provide an overview of molecular targeted therapies for HCC management.
- To highlight the potential of novel therapeutic strategies and personalized medicine in HCC.
Main Methods:
- Review of scientific literature on HCC molecular pathogenesis.
- Analysis of genetic and epigenetic alterations in hepatocarcinogenesis.
- Evaluation of preclinical and clinical data for molecular targeted therapies.
Main Results:
- Identification of key molecular pathways involved in HCC development.
- Demonstration of sorafenib's efficacy and its role as a standard of care for advanced HCC.
- Evidence supporting combination therapies and molecular classification for enhanced anti-tumoral effects.
Conclusions:
- Molecular targeted therapies are crucial for advancing HCC treatment.
- Combination therapies targeting diverse pathways hold significant therapeutic potential.
- A robust molecular classification system is essential for personalized HCC treatment strategies.
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