{
  "abstract": "Glioblastoma is a fatal disease with a median prognosis of 12-18 months. Recent studies have shown encouraging results using neoantigen-based vaccines to stimulate glioblastoma-directed immune responses, but overall immunogenicity has been low. Here, we report the results of an open-label, single-arm, phase 1 clinical trial (GT-20) to evaluate the safety and feasibility (primary endpoints) as well as immunogenicity and preliminary clinical activity (secondary endpoints) of GNOS-PV01 monotherapy, a DNA-based personalized therapeutic cancer vaccine administered following surgical resection and radiation for patients with MGMT unmethylated glioblastoma. The GT-20 study vaccinated nine patients, using up to 40 neoantigens per patient (range, 17-40) without causing any serious adverse events, unexpected toxicities or dose-limiting toxicities. The vaccine induced activation and expansion of circulating peripheral T cells in all evaluated patients, except one who was being treated with dexamethasone. The secondary endpoint was to evaluate 6 month progression-free survival and 12 month overall survival; each observed in 66.7% of patients. Median progression-free survival was 8.5 months, median overall survival was 16.3 months and survival at 24 months was 33%, including one long-term survivor still alive 4 years from the time of initial surgery. This study met the pre-specified endpoints and supports the use of GNOS-PV01 as a potentially impactful component of glioblastoma immunotherapy. ClinicalTrials.gov: NCT04015700 .",
  "abstract_shown": true,
  "affiliations": [
    "The Steve and Cindy Rasmussen Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, OH, USA",
    "Geneos Therapeutics, Philadelphia, PA, USA",
    "Department of Medicine, Washington University School of Medicine, St. Louis, MO, USA",
    "Division of Medical Oncology, Washington University School of Medicine, St. Louis, MO, USA",
    "Department of Pathology and Immunology, Washington University School of Medicine, St. Louis, MO, USA",
    "The Brain Tumor Center at Siteman Cancer Center, Washington University in St. Louis, St. Louis, MO, USA",
    "Department of Surgery, Washington University in St. Louis, St. Louis, MO, USA",
    "McDonnell Genome Institute, Washington University School of Medicine, St. Louis, MO, USA",
    "Biomedical Sciences Graduate Program, The Ohio State University College of Medicine, Columbus, OH, USA",
    "The Ohio State University, Columbus, OH, USA",
    "Department of Radiation Oncology, Washington University School of Medicine, St. Louis, MO, USA",
    "BioProcess Advantage LLC, Westfield, NJ, USA",
    "Department of Neurology, Mayo Clinic, Rochester, MN, USA",
    "Department of Neurosurgery, University of Missouri, Columbia, MO, USA",
    "Department of Neurosurgery, Washington University School of Medicine, St. Louis, MO, USA",
    "Department of Genetics, Washington University School of Medicine, St. Louis, MO, USA",
    "Department of Surgery, Washington University School of Medicine, St. Louis, MO, USA",
    "Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA",
    "Department of Neurosurgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA",
    "Andrew M. and Jane M. Bursky Center for Human Immunology and Immunotherapy Program, Washington University School of Medicine, St. Louis, MO, USA. tannerjohanns@wustl.edu",
    "Division of Medical Oncology, Washington University School of Medicine, St. Louis, MO, USA. tannerjohanns@wustl.edu",
    "The Brain Tumor Center at Siteman Cancer Center, Washington University in St. Louis, St. Louis, MO, USA. tannerjohanns@wustl.edu"
  ],
  "article_type": null,
  "arxiv": null,
  "author_total": 36,
  "authors": [
    {
      "affs": [
        0
      ],
      "name": "Elizabeth A.R. Garfinkle",
      "orcid": "0000-0002-7292-4347"
    },
    {
      "affs": [
        1
      ],
      "name": "Renzo F. Perales-Linares",
      "orcid": "0000-0001-5309-8249"
    },
    {
      "affs": [
        2
      ],
      "name": "Ryan C. Gimple",
      "orcid": "0000-0002-7189-3243"
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      "affs": [
        3
      ],
      "name": "Alexandra J. Livingstone",
      "orcid": "0000-0001-5471-4576"
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        4
      ],
      "name": "Kaleigh F. Roberts",
      "orcid": "0000-0001-6483-9153"
    },
    {
      "affs": [
        3,
        5
      ],
      "name": "Omar Hameed Butt",
      "orcid": "0000-0003-2252-3673"
    },
    {
      "affs": [
        6,
        5
      ],
      "name": "S. Peter Goedegebuure"
    },
    {
      "affs": [
        7
      ],
      "name": "Michael D. McLellan",
      "orcid": "0000-0002-2703-8784"
    },
    {
      "affs": [
        7
      ],
      "name": "Gue Su Chang",
      "orcid": "0009-0007-2885-7400"
    },
    {
      "affs": [
        7
      ],
      "name": "Jasreet Hundal",
      "orcid": "0000-0002-2584-5320"
    },
    {
      "affs": [
        1
      ],
      "name": "Jian Yan"
    },
    {
      "affs": [
        0
      ],
      "name": "Jaye B. Navarro"
    },
    {
      "affs": [
        8,
        0
      ],
      "name": "Sophia A. Paxton",
      "orcid": "0009-0000-8169-6895"
    },
    {
      "affs": [
        9,
        0
      ],
      "name": "Srestha Chattopadhyay"
    },
    {
      "affs": [
        1
      ],
      "name": "Neil S. Cooch",
      "orcid": "0000-0002-8017-9957"
    },
    {
      "affs": [
        1
      ],
      "name": "Alfredo Perales‐Puchalt",
      "orcid": "0000-0003-4904-5050"
    },
    {
      "affs": [
        10
      ],
      "name": "Konstantina Stavroulaki"
    },
    {
      "affs": [
        1
      ],
      "name": "Sarah Rochestie"
    },
    {
      "affs": [
        1
      ],
      "name": "Joann Peters"
    },
    {
      "affs": [
        11
      ],
      "name": "Beth Junker"
    },
    {
      "affs": [
        12
      ],
      "name": "Jian Li Campian",
      "orcid": "0000-0002-5317-8481"
    },
    {
      "affs": [
        2,
        5
      ],
      "name": "Milan G. Chheda",
      "orcid": "0000-0001-8282-9098"
    },
    {
      "affs": [
        13
      ],
      "name": "Michael R. Chicoine",
      "orcid": "0000-0002-0689-8603"
    },
    {
      "affs": [
        14,
        5
      ],
      "name": "Albert H. Kim"
    },
    {
      "affs": [
        14
      ],
      "name": "Jon Timothy Willie",
      "orcid": "0000-0001-9565-4338"
    },
    {
      "affs": [
        14,
        5
      ],
      "name": "Gregory Joseph Zipfel",
      "orcid": "0000-0003-2737-9084"
    },
    {
      "affs": [
        14,
        5
      ],
      "name": "Joshua L. Dowling",
      "orcid": "0000-0002-0340-7493"
    },
    {
      "affs": [
        15,
        2,
        7
      ],
      "name": "Christopher A. Miller",
      "orcid": "0000-0003-4266-6700"
    },
    {
      "affs": [
        15,
        2,
        7
      ],
      "name": "Obi L. Griffith",
      "orcid": "0000-0002-0843-4271"
    },
    {
      "affs": [
        15,
        2,
        7
      ],
      "name": "Malachi Griffith",
      "orcid": "0000-0002-6388-446X"
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    {
      "affs": [
        16
      ],
      "name": "William E. Gillanders",
      "orcid": "0000-0002-0465-3071"
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    {
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        17,
        0
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      "name": "Katherine E. Miller",
      "orcid": "0000-0003-1054-0476"
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    {
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        17,
        0
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      "name": "Elaine R. Mardis",
      "orcid": "0000-0002-5892-1553"
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    {
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        1
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      "name": "Niranjan Y. Sardesai",
      "orcid": "0000-0001-6920-1167"
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        18
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      "name": "Gavin P. Dunn",
      "orcid": "0000-0001-9302-4834"
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    {
      "affs": [
        19,
        20,
        21
      ],
      "name": "Tanner Michael Johanns",
      "orcid": "0000-0003-1094-1528"
    }
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  "bibtex": "https://research.draconitic.com/paper/eqnr5q301cc1t.bib",
  "cited_by": 9,
  "doi": "10.1038/s43018-026-01163-w",
  "fulltext_url": null,
  "id": "eqnr5q301cc1t",
  "issns": [
    "2662-1347"
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  "landing_url": "https://doi.org/10.1038/s43018-026-01163-w",
  "language": "en",
  "licence": "CC-BY-NC-ND",
  "licence_label": "CC BY-NC-ND",
  "licence_source": "journal",
  "licence_url": "https://creativecommons.org/licenses/by-nc-nd/4.0/",
  "metadata_licence": null,
  "native_subjects": [
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      "2716|Glioma Diagnosis and Treatment"
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  "openalex": "W7160926096",
  "pdf_url": null,
  "pmcid": "PMC13400305",
  "pmid": "42120910",
  "published": "2026-05-12",
  "publisher": "Nature Portfolio",
  "ris": "https://research.draconitic.com/paper/eqnr5q301cc1t.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T13:26:56Z",
      "native_id": "W7160926096",
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      "url": "https://doi.org/10.1038/s43018-026-01163-w"
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  "subjects": [
    {
      "field": "medicine",
      "origin": "openalex:2716|Glioma Diagnosis and Treatment",
      "subfield": "human-genetics"
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  ],
  "title": "Adjuvant personalized multivalent neoantigen DNA vaccination for MGMT unmethylated glioblastoma: a phase 1 trial",
  "title_text": "Adjuvant personalized multivalent neoantigen DNA vaccination for MGMT unmethylated glioblastoma: a phase 1 trial",
  "type": "peer-reviewed",
  "updated_at": "2026-11-04T13:21:15Z",
  "url": "https://research.draconitic.com/paper/eqnr5q301cc1t",
  "venue": "Nature Cancer",
  "versions": []
}