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Updated: Jan 20, 2026

Author Spotlight: Investigating Liver Cancer Pathogenesis Using Patient-Derived Organoids
Published on: August 18, 2023
Early development of sunitinib in hepatocellular carcinoma
1Tucker Gosnell Center for Gastrointestinal Cancers, Massachusetts General Hospital Cancer Center, Harvard Medical School, Boston, MA, USA. azhu@partners.org
Abstract:
Sunitinib malate is an oral, multitargeted receptor tyrosine kinase inhibitor of VEGF receptors 1, 2 and 3; PDGF receptors alpha and beta, and other receptor tyrosine kinases implicated in tumor growth, angiogenesis and metastasis. Hepatocellular carcinoma (HCC) is a highly vascular tumor that overexpresses several angiogenic factors; VEGF and PDGF signaling pathways play a key role in HCC. Until recently, treatment options for advanced HCC were limited and conventional therapies have met with poor response rates. Sorafenib provided proof-of-concept for molecularly targeted therapy in advanced HCC and has recently been approved for treatment. However, not all patients can tolerate sorafenib and patients may experience tumor progression; therefore, additional treatment options are warranted. Sunitinib has shown early evidence of anti-tumor activity in Phase II trials in US, European and Asian patients with locally advanced, unresectable and metastatic HCC. A Phase III trial of sunitinib in HCC is ongoing.
Insights
Sunitinib malate, a receptor tyrosine kinase inhibitor, shows anti-tumor activity in advanced hepatocellular carcinoma (HCC) patients. This targeted therapy offers a potential new option for patients with this highly vascular liver cancer.
Area of Science:
- Oncology
- Pharmacology
Background:
- Hepatocellular carcinoma (HCC) is a highly vascular tumor driven by angiogenesis.
- Advanced HCC treatment options were limited, with conventional therapies showing poor response rates.
- Sorafenib demonstrated the potential of targeted therapy but has limitations in tolerability and efficacy.
Purpose of the Study:
- To evaluate the anti-tumor activity of sunitinib malate in patients with advanced, unresectable, and metastatic HCC.
- To explore sunitinib as an alternative targeted therapy for HCC.
Main Methods:
- Sunitinib malate, an oral multitargeted receptor tyrosine kinase inhibitor, was investigated.
- Early evidence of anti-tumor activity was assessed in Phase II clinical trials.
Main Results:
- Sunitinib demonstrated early evidence of anti-tumor activity in US, European, and Asian patients.
- The drug targets key signaling pathways like VEGF and PDGF involved in HCC growth.
Conclusions:
- Sunitinib malate shows promise as a targeted therapy for advanced HCC.
- Further investigation in a Phase III trial is ongoing to confirm efficacy and safety.
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