A phase II trial evaluating the clinical and immunologic response of HLA-A2(+) non-small cell lung cancer patients vaccinated with an hTERT cryptic peptide

Lung Cancer. 2014 Oct;86(1):59-66. doi: 10.1016/j.lungcan.2014.07.018. Epub 2014 Aug 5.


Objectives: The immunological and clinical responses of patients with NSCLC treated, in the context of an expanded action program, with the cryptic hTERT-targeting Vx-001 vaccine are presented.

Materials and methods: Forty-six HLA-A*0201-positive patients with advanced NSCLC and residual (n=27) or progressive (n=19) disease following front-line treatment received two subcutaneous injections of the optimized TERT572Y peptide followed by four injections of the native TERT572 peptide, every 3 weeks. Peptide-specific immune responses were monitored by enzyme-linked immunosorbent spot assay at baseline, and after the 2nd and the 6th vaccinations. Thirty-eight HLA-A*0201-positive matched patients were used as historical controls.

Results: Twenty-three patients (50%) completed the vaccination protocol and 87% received at least two administrations. Twelve patients (26%) without disease progression after the 6th vaccination received boost vaccinations. Three (7%) patients achieved a partial response and 13 (28%) disease stabilization. The disease control rate was significantly higher in patients with non-squamous histology compared to those with squamous-cell histology [n=14 (45%) versus n=2 (13%); p=0.03]. The median progression-free survival (PFS) and overall survival (OS) was 3.8 (range, 0.7-99.4) and 19.8 months (range, 0.7-99.4), respectively. Patients who developed immune response had a numerically higher PFS compared to those who failed to mount any (6.7 versus 2.7 months; p=0.090). However, the median OS for the immune-responders was significantly prolonged compared to non-responders (40.0 versus 9.2 months, respectively; p=0.02). Toxicity was <grade 2.

Conclusion: Vx-001 vaccine is well tolerated and induced a TERT-specific immunological response, which was significantly correlated with improved clinical outcome.

Keywords: Clinical efficacy; HLA-A2+; Immunologic response; NSCLC; Phase II; h-TERT.

Publication types

  • Clinical Trial, Phase II
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Cancer Vaccines / administration & dosage
  • Cancer Vaccines / adverse effects
  • Cancer Vaccines / immunology*
  • Carcinoma, Non-Small-Cell Lung / immunology*
  • Carcinoma, Non-Small-Cell Lung / mortality
  • Carcinoma, Non-Small-Cell Lung / pathology
  • Carcinoma, Non-Small-Cell Lung / therapy*
  • Chemotherapy, Adjuvant
  • Disease Progression
  • Female
  • HLA-A2 Antigen / immunology*
  • Humans
  • Immunotherapy
  • Lung Neoplasms / immunology*
  • Lung Neoplasms / mortality
  • Lung Neoplasms / pathology
  • Lung Neoplasms / therapy*
  • Male
  • Middle Aged
  • Peptide Fragments / immunology*
  • Radiotherapy, Adjuvant
  • Telomerase / chemistry
  • Telomerase / immunology*
  • Treatment Outcome


  • Cancer Vaccines
  • HLA-A2 Antigen
  • Peptide Fragments
  • TERT protein, human
  • Telomerase