Cabozantinib in the treatment of hepatocellular carcinoma

Mary Linton B Peters1, Rebecca A Miksad1

  • 1Division of Hematology/Oncology, Beth Israel Deaconess Medical Center, Boston, MA 02215, USA.

Insights

Cabozantinib, a tyrosine kinase inhibitor, shows promise for advanced hepatocellular carcinoma (HCC) patients previously treated with sorafenib. Further Phase III trials are evaluating its efficacy in improving outcomes for this challenging cancer.

Area of Science:

  • Hepatobiliary cancers
  • Medical oncology
  • Drug development

Background:

  • Limited systemic therapy advancements for hepatocellular carcinoma (HCC) between 2007 and 2017.
  • Need for improved treatment options to enhance patient outcomes and quality of life in advanced HCC.

Purpose of the Study:

  • To review the development of cabozantinib for advanced hepatocellular carcinoma (HCC).
  • To summarize the clinical evaluation of cabozantinib in HCC patients.

Main Methods:

  • Cabozantinib is an oral small-molecule tyrosine kinase inhibitor targeting MET, VEGFR2, AXL, and RET.
  • Positive Phase II study results led to a Phase III randomized controlled trial for advanced HCC patients previously treated with sorafenib.
  • Cabozantinib has orphan drug status in the USA for HCC indication.

Main Results:

  • Cabozantinib is approved for metastatic medullary thyroid cancer and previously treated renal cell carcinoma.
  • Ongoing Phase III trial evaluates cabozantinib in advanced HCC patients post-sorafenib therapy.
  • The drug targets key pathways implicated in cancer progression.

Conclusions:

  • Cabozantinib represents a potential new systemic therapy for advanced HCC.
  • Further investigation in Phase III trials is crucial to establish its role in HCC treatment.
  • The development of cabozantinib offers a new avenue for patients with previously treated advanced HCC.

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