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Updated: Jan 31, 2026

Author Spotlight: Investigating Liver Cancer Pathogenesis Using Patient-Derived Organoids
Published on: August 18, 2023
Targeted therapy of hepatocellular carcinoma: present and future
1State Key Laboratory in Oncology in South China, Sir YK Pao Center for Cancer, Department of Clinical Oncology, Hong Kong Cancer Institute and Prince of Wales Hospital, The Chinese University of Hong Kong, Hong Kong. chanlam_stephen@cuhk.edu.hk
Abstract:
Following the encouraging results of sorafenib in advanced hepatocellular carcinoma (HCC), targeted therapy has become a new direction of research in the treatment of HCC. Emerging data provide evidence that the pathogenesis and progression of HCC are mediated by a number of molecular defects and dysregulated pathways. Novel targeted therapies are designed to inhibit the aberrant pathways at a molecular level with an aim to improve the clinical outcome. For the past few years, an increasing number of targeted agents have been tested in HCC in the clinical setting. This review aims to summarize the current status of clinical development of targeted therapy in HCC, with focus on novel agents targeting angiogenesis, signal transduction and epigenetic dysregulation of tumors. The review also discusses the lessons learned from outcomes of completed clinical trials and provides perspectives on future clinical trials in HCC.
Insights
Targeted therapy shows promise for advanced hepatocellular carcinoma (HCC) by inhibiting molecular pathways. This review summarizes current clinical trials and future directions for novel HCC treatments.
Area of Science:
- Hepatobiliary cancers
- Molecular oncology
- Clinical pharmacology
Background:
- Sorafenib demonstrated efficacy in advanced hepatocellular carcinoma (HCC), shifting treatment paradigms.
- HCC pathogenesis involves molecular defects and dysregulated pathways.
- Targeted therapies aim to inhibit these aberrant pathways for improved clinical outcomes.
Purpose of the Study:
- To review the current status of targeted therapy clinical development in HCC.
- To focus on novel agents targeting angiogenesis, signal transduction, and epigenetic dysregulation.
- To discuss lessons learned from completed trials and future perspectives.
Main Methods:
- Literature review of clinical trials involving targeted agents in HCC.
- Focus on agents targeting angiogenesis, signal transduction, and epigenetic mechanisms.
- Analysis of outcomes from completed clinical trials.
Main Results:
- An increasing number of targeted agents have been evaluated in HCC clinical settings.
- Novel agents target key molecular pathways implicated in HCC progression.
- Completed trials provide valuable insights into efficacy and safety.
Conclusions:
- Targeted therapy represents a significant advancement in HCC treatment.
- Understanding molecular pathways is crucial for developing effective therapies.
- Future clinical trials should build upon current findings and explore novel strategies.
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