Adjuvant personalized multivalent neoantigen DNA vaccination for MGMT unmethylated glioblastoma: a phase 1 trial - Report - MDSpire

Adjuvant personalized multivalent neoantigen DNA vaccination for MGMT unmethylated glioblastoma: a phase 1 trial

  • By

  • Elizabeth A. R. Garfinkle

  • Renzo Perales-Linares

  • Ryan C. Gimple

  • Alexandra J. Livingstone

  • Kaleigh F. Roberts

  • Omar H. Butt

  • S. Peter Goedegebuure

  • Michael D. McLellan

  • Gue Su Chang

  • Jasreet Hundal

  • Jian Yan

  • Jaye B. Navarro

  • Sophia A. Paxton

  • Srestha Chattopadhyay

  • Neil Cooch

  • Alfredo Perales-Puchalt

  • Konstantina Stavroulaki

  • Sarah Rochestie

  • Joann Peters

  • Beth Junker

  • Jian L. Campian

  • Milan G. Chheda

  • Michael R. Chicoine

  • Albert H. Kim

  • Jon T. Willie

  • Gregory J. Zipfel

  • Joshua L. Dowling

  • Christopher A. Miller

  • Obi L. Griffith

  • Malachi Griffith

  • William E. Gillanders

  • Katherine E. Miller

  • Elaine R. Mardis

  • Niranjan Y. Sardesai

  • Gavin P. Dunn

  • Tanner M. Johanns

  • May 12, 2026

  • 0 min

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Clinical Report: Personalized Multivalent Neoantigen DNA Vaccination in GBM

Overview

This phase 1 study evaluated a personalized neoantigen DNA vaccine for patients with unmethylated MGMT glioblastoma. The trial demonstrated the feasibility and immunogenicity of the vaccine, targeting a range of neoantigens derived from multiple tumor regions.

Background

Glioblastoma (GBM) is a highly aggressive brain tumor with limited treatment options, particularly for patients with unmethylated MGMT promoters who do not benefit from standard chemotherapy. The need for innovative therapies is critical, as current immunotherapies have shown limited efficacy in this patient population. Personalized neoantigen vaccines represent a promising approach to enhance anti-tumor immune responses in GBM.

Data Highlights

ParameterValue
Patients screened34
Patients eligible13
Patients enrolled9
Median age59 years
Male patients67%
Neoantigens targeted17–40

Key Findings

  • The trial enrolled nine patients with newly diagnosed MGMT unmethylated GBM.
  • Personalized DNA vaccines were designed to target up to 40 neoantigens from multiple tumor regions.
  • All vaccinations were administered intramuscularly with electroporation and IL-12 as an adjuvant.
  • Immune responses were assessed using TCR sequencing and functional assays.
  • The study aimed to inform future combinatorial therapeutic approaches for GBM.

Clinical Implications

The findings suggest that personalized neoantigen DNA vaccines may provide a novel therapeutic option for patients with unmethylated MGMT glioblastoma. Further studies are warranted to evaluate the efficacy of this approach in combination with other treatments to enhance patient outcomes.

Conclusion

This phase 1 trial highlights the potential of personalized neoantigen DNA vaccines in eliciting immune responses against glioblastoma. Continued research is essential to optimize this strategy and improve survival in affected patients.

Related Resources & Content

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  8. WHO Grade 4 Adult-Type Diffuse Glioma Guideline - American Society for Radiation Oncology (ASTRO)
  9. Radiotherapy combined with nivolumab or temozolomide for newly diagnosed glioblastoma with unmethylated MGMT promoter: An international randomized phase III trial | Neuro-Oncology | Oxford Academic
  10. Neoantigen vaccine generates intratumoral T cell responses in phase Ib glioblastoma trial | Nature

Original Source(s)

Adjuvant personalized multivalent neoantigen DNA vaccination for MGMT unmethylated glioblastoma: a phase 1 trial

  • by Elizabeth A. R. Garfinkle, Renzo Perales-Linares, Ryan C. Gimple, Alexandra J. Livingstone, Kaleigh F. Roberts, Omar H. Butt, S. Peter Goedegebuure, Michael D. McLellan, Gue Su Chang, Jasreet Hundal, Jian Yan, Jaye B. Navarro, Sophia A. Paxton, Srestha Chattopadhyay, Neil Cooch, Alfredo Perales-Puchalt, Konstantina Stavroulaki, Sarah Rochestie, Joann Peters, Beth Junker, Jian L. Campian, Milan G. Chheda, Michael R. Chicoine, Albert H. Kim, Jon T. Willie, Gregory J. Zipfel, Joshua L. Dowling, Christopher A. Miller, Obi L. Griffith, Malachi Griffith, William E. Gillanders, Katherine E. Miller, Elaine R. Mardis, Niranjan Y. Sardesai, Gavin P. Dunn, Tanner M. Johanns

  • May 12, 2026

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