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Updated: May 15, 2026

A Three-Dimensional Spheroid Model to Investigate the Tumor-Stromal Interaction in Hepatocellular Carcinoma
Published on: September 30, 2021
New agents on the horizon in hepatocellular carcinoma
1Massachusetts General Hospital Cancer Center, Harvard Medical School, 55 Fruit Street, LH/POB 232, Boston, MA 02114, USA.
Abstract:
Despite the successful approval and extensive application of sorafenib, the prognosis for patients with advanced hepatocellular carcinoma (HCC) remains poor. Fortunately, there have been renewed and continued interests and active research in developing other molecularly targeted agents in HCC during the past few years. While there is early evidence of antitumor activity of several agents in phase I/II studies, phase III efforts with a few targeted agents have failed, highlighting the challenges of new drug development in HCC. This review summarizes the current status of other molecularly targeted agents under development in advanced HCC.
Insights
Despite sorafenib use, advanced hepatocellular carcinoma (HCC) patients have a poor prognosis. This review examines new molecularly targeted agents for HCC, noting challenges in drug development despite early promising results.
Area of Science:
- Hepatocellular Carcinoma Research
- Molecularly Targeted Therapies
- Oncology Drug Development
Background:
- Sorafenib is approved for advanced hepatocellular carcinoma (HCC), but patient prognosis remains poor.
- Despite sorafenib's use, significant research is ongoing for novel molecularly targeted agents in HCC.
- Challenges exist in developing new drugs for HCC, as evidenced by mixed results in clinical trials.
Purpose of the Study:
- To review the current development status of molecularly targeted agents for advanced HCC.
- To summarize early-phase and late-phase clinical trial findings for new HCC therapies.
- To highlight the challenges and progress in targeted therapy research for HCC.
Main Methods:
- Literature review of ongoing research and clinical trials.
- Analysis of phase I, II, and III study outcomes for targeted agents in HCC.
- Synthesis of data on antitumor activity and development challenges.
Main Results:
- Several targeted agents show early evidence of antitumor activity in phase I/II studies.
- Phase III trials for some targeted agents have unfortunately failed to meet endpoints.
- The development landscape for advanced HCC targeted therapies is complex and challenging.
Conclusions:
- New molecularly targeted agents are under investigation for advanced HCC.
- Clinical trial outcomes underscore the difficulties in bringing new HCC drugs to market.
- Continued research is crucial for improving treatment options for advanced HCC patients.
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