The PEPvIII-KLH (CDX-110) vaccine in glioblastoma multiforme patients

Expert Opin Biol Ther. 2009 Aug;9(8):1087-98. doi: 10.1517/14712590903124346.


Conventional therapies for glioblastoma multiforme (GBM) fail to target tumor cells exclusively, resulting in non-specific toxicity. Immune targeting of tumor-specific mutations may allow for more precise eradication of neoplastic cells. EGFR variant III (EGFRvIII) is a tumor-specific mutation that is widely expressed in GBM and other neoplasms and its expression enhances tumorigenicity. This in-frame deletion mutation splits a codon, resulting in a novel glycine at the fusion junction producing a tumor-specific epitope target for cellular or humoral immunotherapy. We have previously shown that vaccination with a peptide that spans the EGFRvIII fusion junction (PEPvIII-KLH/CDX-110) is an efficacious immunotherapy in syngeneic murine models. In this review, we summarize our results in GBM patients targeting this mutation in multiple, multi-institutional Phase II immunotherapy trials. These trials demonstrated that a selected population of GBM patients who received vaccines targeting EGFRvIII had an unexpectedly long survival time. Further therapeutic strategies and potential pitfalls of using this approach are discussed.

Publication types

  • Review

MeSH terms

  • Animals
  • Brain Neoplasms / therapy*
  • Cancer Vaccines / immunology
  • Cancer Vaccines / therapeutic use*
  • Central Nervous System / immunology
  • Clinical Trials as Topic
  • ErbB Receptors / immunology
  • ErbB Receptors / metabolism
  • Glioblastoma / therapy*
  • Hemocyanins / chemistry
  • Hemocyanins / immunology*
  • Hemocyanins / metabolism
  • Humans
  • Immunotherapy / methods
  • Mice
  • Mutation
  • Peptides / chemistry
  • Peptides / immunology*
  • Prognosis


  • Cancer Vaccines
  • Pep-3 peptide
  • Peptides
  • Hemocyanins
  • ErbB Receptors
  • keyhole-limpet hemocyanin