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Novel drugs in clinical development for hepatocellular carcinoma

Oliver Waidmann1, Jörg Trojan

  • 1Universitätsklinikum Frankfurt, Medizinische Klinik 1 , Theodor-Stern-Kai 7, Frankfurt am Main , Germany +49 0 69 6301 7860 ; +49 0 69 6301 6448 ; trojan@em.uni-frankfurt.de.

Expert Opinion on Investigational Drugs
|June 26, 2015
PubMed
Abstract

Insights

Sorafenib is the only approved drug for advanced hepatocellular carcinoma (HCC). New targeted therapies show promise in specific patient subgroups, including MET-overexpressing tumors and those with high AFP levels, offering hope for improved survival in HCC treatment.

Area of Science:

  • Hepatocellular Carcinoma (HCC) Research
  • Oncology
  • Clinical Trials

Background:

  • Sorafenib remains the sole approved systemic therapy for advanced hepatocellular carcinoma (HCC).
  • Recent investigational agents targeting angiogenesis have largely failed in late-stage clinical trials due to toxicity or lack of efficacy.
  • There is a critical need for novel systemic treatments for advanced HCC.

Purpose of the Study:

  • To review recent clinical data on systemic agents for advanced HCC.
  • To summarize ongoing clinical trials in patients with advanced HCC.
  • To identify promising therapeutic strategies beyond current standards of care.

Main Methods:

  • Review of recent clinical trial data for systemic agents in advanced HCC.
  • Analysis of ongoing clinical trial landscape in advanced HCC.
  • Evaluation of efficacy and safety data for investigational therapies.

Main Results:

  • Several large trials in unselected advanced HCC patients yielded disappointing results.
  • Tivantinib (MET inhibitor) demonstrated survival benefit in MET-overexpressing tumors (Phase II trial).
  • Ramucirumab (VEGFR2 antibody) improved overall survival in patients with baseline AFP ≥ 400 ng/ml (Phase III trial).

Conclusions:

  • Targeted therapies show promise in specific advanced HCC subgroups.
  • Tivantinib and ramucirumab represent potential new treatment options for selected HCC patients.
  • Immunotherapeutics, including checkpoint inhibitors and oncolytic viruses, are under investigation for advanced HCC.
Keywords:
anti-CTLA-4anti-PD1cabozantinibeverolimushepatocellular carcinomaimmunotherapylenvatiniboncolytic virusramucirumabreceptor tyrosine kinase inhibitorrefametinibregorafenibsorafenibtivantinibtrebananib

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