Glioblastoma multiforme (GBM) is the most common and deadly of the human brain 
cancers. The EGF receptor is often amplified in GBM and provides a potential 
therapeutic target. However, targeting the normal receptor is complicated by its 
nearly ubiquitous and high level of expression in certain tissues. A naturally 
occurring deletion mutant of the EGF receptor, EGFRvIII, is a constitutively 
active variant originally identified in a high percentage of brain cancer cases, 
and more importantly is rarely found in normal tissue. A peptide vaccine, 
rindopepimut (CDX-110, Celldex Therapeutics), is directed against the novel exon 
1-8 junction produced by the EGFRvIII deletion, and it has shown high efficacy 
in preclinical models. Recent Phase II clinical trials in patients with newly 
diagnosed GBM have shown EGFRvIII-specific immune responses and significantly 
increased time to progression and overall survival in those receiving vaccine 
therapy, as compared with published results for standard of care. Rindopepimut 
therefore represents a very promising therapy for patients with GBM.