Clinical Trial of a Cancer Vaccine Targeting VEGF and KIF20A in Advanced Biliary Tract Cancer

Anticancer Res. 2021 Mar;41(3):1485-1496. doi: 10.21873/anticanres.14907.


Background: As the prognosis of biliary tract cancer (BTC) is extremely poor and treatment options are limited, new treatment modalities are urgently needed. We designed a phase II clinical trial to investigate the immune responses and clinical benefits of OCV-C01, an HLA-A*24:02-restricted three-peptide cancer vaccine targeting VEGFR1, VEGFR2, and KIF20A.

Patients and methods: Participants were patients with advanced BTC who had unresectable tumours and were refractory to standard chemotherapy. OCV-C01 was injected weekly until the discontinuance criteria were met.

Results: Six participants, including four patients positive for HLA-A*24:02, were enrolled in this study for assessment of efficacy. Four out of six patients exhibited vaccine-specific T-cell responses to one or more of three antigens. Log-rank tests revealed that vaccine-specific T cell responses contributed significantly to overall survival.

Conclusion: The cancer vaccine had positive effects on survival, indicating that this approach warrants further clinical studies.

Keywords: KIF20A; Translational research; VEGF; biliary tract cancer; cancer vaccine.

Publication types

  • Clinical Trial, Phase II

MeSH terms

  • Aged
  • Aged, 80 and over
  • Biliary Tract Neoplasms / drug therapy*
  • Biliary Tract Neoplasms / immunology
  • Biliary Tract Neoplasms / metabolism
  • Cancer Vaccines / administration & dosage*
  • Cancer Vaccines / adverse effects
  • Cancer Vaccines / immunology
  • Disease-Free Survival
  • Female
  • Fever / chemically induced
  • Headache / chemically induced
  • Humans
  • Kinesins / antagonists & inhibitors*
  • Kinesins / immunology
  • Male
  • Middle Aged
  • Molecular Targeted Therapy / methods
  • Prognosis
  • T-Lymphocytes, Cytotoxic / drug effects
  • T-Lymphocytes, Cytotoxic / immunology
  • T-Lymphocytes, Cytotoxic / metabolism
  • Vaccines, Subunit / administration & dosage*
  • Vaccines, Subunit / adverse effects
  • Vaccines, Subunit / immunology
  • Vascular Endothelial Growth Factor A / antagonists & inhibitors
  • Vascular Endothelial Growth Factor A / immunology
  • Vascular Endothelial Growth Factor Receptor-2 / antagonists & inhibitors*
  • Vascular Endothelial Growth Factor Receptor-2 / immunology


  • Cancer Vaccines
  • KIF20A protein, human
  • OCV-C01
  • Vaccines, Subunit
  • Vascular Endothelial Growth Factor A
  • KDR protein, human
  • Vascular Endothelial Growth Factor Receptor-2
  • Kinesins