^
    7d
    Azeliragon in MGMT Unmethylated Glioblastoma (clinicaltrials.gov)
    P2, N=30, Active, not recruiting, Cantex Pharmaceuticals | Trial completion date: Jun 2025 --> Dec 2026 | Trial primary completion date: Sep 2024 --> Sep 2026
    Trial completion date • Trial primary completion date
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    MGMT (6-O-methylguanine-DNA methyltransferase)
    |
    MGMT promoter methylation • IDH wild-type
    |
    azeliragon (TTP488)
    9d
    ARETHUSA: Pembrolizumab in MMR-Proficient Metastatic Colorectal Cancer Pharmacologically Primed to Trigger Hypermutation Status (clinicaltrials.gov)
    P2, N=107, Completed, IFOM ETS - The AIRC Institute of Molecular Oncology | Not yet recruiting --> Completed
    Trial completion • Tumor mutational burden
    |
    TMB (Tumor Mutational Burden) • MGMT (6-O-methylguanine-DNA methyltransferase)
    |
    MSI-H/dMMR • RAS mutation • MGMT promoter methylation
    |
    Keytruda (pembrolizumab) • temozolomide
    14d
    NKCC1: A key regulator of glioblastoma progression. (PubMed, Mol Oncol)
    These findings identify NKCC1 as a regulator of GBM progression and recurrence, linking chloride transporter imbalance to GABAergic signaling. Targeting NKCC1 and restoring chloride homeostasis may provide promising new treatment strategies.
    Journal
    |
    MGMT (6-O-methylguanine-DNA methyltransferase) • SLC12A2 (Solute Carrier Family 12 Member 2)
    |
    MGMT promoter methylation
    22d
    Identification of new prognostic molecular markers in glioblastoma: a single-center retrospective study. (PubMed, Oncologist)
    We identified new prognostic markers in GBM, showing for the first time that EGFR substitutions improve OS. Validation in external cohorts and preclinical studies is needed.
    Retrospective data • Journal
    |
    EGFR (Epidermal growth factor receptor) • TP53 (Tumor protein P53) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • NOTCH1 (Notch 1) • MGMT (6-O-methylguanine-DNA methyltransferase) • CDKN2B (Cyclin Dependent Kinase Inhibitor 2B)
    |
    MGMT promoter methylation
    25d
    MGMT Promoter and Enhancer Methylation in Melanoma Brain Metastases and Glioblastoma: Shared and Distinct Features. (PubMed, Cells)
    We focused on MGMT because MGMT promoter methylation is used as a predictive marker for temozolomide response in GB, but its role in MBM has been discussed controversially...GB exhibited higher methylation, particularly in promoter and intergenic enhancers, and stronger associations between methylation and overall survival than MBM. These results highlight both conserved and tumor-specific MGMT regulation, reflecting the complexity of epigenetic control in brain malignancies and emphasizing divergent evolution between MBM and GB.
    Journal
    |
    MGMT (6-O-methylguanine-DNA methyltransferase)
    |
    MGMT promoter methylation
    |
    temozolomide
    26d
    Personalized NeoAntigen Cancer Vaccine w RT Plus Pembrolizumab for Patients With Newly Diagnosed GBM (clinicaltrials.gov)
    P1, N=56, Recruiting, Dana-Farber Cancer Institute | Trial completion date: Feb 2027 --> Feb 2028 | Trial primary completion date: Feb 2026 --> Feb 2027
    Trial completion date • Trial primary completion date
    |
    MGMT (6-O-methylguanine-DNA methyltransferase)
    |
    MGMT promoter methylation
    |
    Keytruda (pembrolizumab) • temozolomide • Hiltonol (poly-ICLC) • Neo Vax (NEO-PV-01)
    28d
    Neoantigen-based Personalized DNA Vaccine With Retifanlimab PD-1 Blockade Therapy in Patients With Newly Diagnosed, Unmethylated Glioblastoma (clinicaltrials.gov)
    P1, N=27, Active, not recruiting, Washington University School of Medicine | Recruiting --> Active, not recruiting | Trial completion date: Jan 2030 --> Mar 2029 | Trial primary completion date: Apr 2027 --> Apr 2026
    Enrollment closed • Trial completion date • Trial primary completion date
    |
    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • MGMT (6-O-methylguanine-DNA methyltransferase)
    |
    IDH2 mutation • MGMT promoter methylation • IDH wild-type
    |
    Zynyz (retifanlimab-dlwr) • neoantigen DNA vaccine
    29d
    Glioblastoma survival in rural America: a 10-year experience from a quaternary care center. (PubMed, J Neurooncol)
    In a predominantly rural state served by a primary academic neuro-oncology program, median overall survival for GBM was 12.7 months indicating centralized care eliminates rural-urban survival gaps in GBM.
    Journal
    |
    MGMT (6-O-methylguanine-DNA methyltransferase)
    |
    MGMT promoter methylation • IDH wild-type
    |
    temozolomide
    30d
    Pediatric high-grade gliomas, H3-wildtype, IDH-wildtype: Refining diagnostic criteria and exploring clinical associations. (PubMed, Neurooncol Pract)
    The findings challenge current WHO diagnostic criteria regarding H3K27me3 retention and demonstrate the critical role of molecular diagnostics, particularly methylation profiling, in refining classification and guiding clinical management. These results advocate for re-evaluation of existing diagnostic frameworks to better accommodate the observed variability and associations in this challenging tumor subtype.
    Journal
    |
    MGMT (6-O-methylguanine-DNA methyltransferase) • MYCN (MYCN Proto-Oncogene BHLH Transcription Factor)
    |
    MGMT promoter methylation • IDH wild-type
    1m
    Pediatric H3 G34-mutant diffuse hemispheric glioma: clinical, imaging and molecular prognostic factors, MGMT expression, and temozolomide response. (PubMed, Acta Neuropathol)
    Our findings reaffirm the dismal outcomes of pediatric H3 G34-mutant DHG, which exhibits radiation resistance, frequent widespread disease, and a novel mechanism of MGMT regulation. Our data support the use of frontline TMZ in pediatric patients and underscore the importance of GTR when feasible.
    Retrospective data • Journal
    |
    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • MGMT (6-O-methylguanine-DNA methyltransferase)
    |
    MGMT promoter methylation
    |
    temozolomide
    1m
    Temporally-Modulated Pulsed Radiation Therapy Versus Standard Radiation Therapy for the Treatment of Newly Diagnosed, IDH Wildtype, MGMT-Unmethylated Glioblastoma (clinicaltrials.gov)
    P3, N=398, Not yet recruiting, NRG Oncology
    New P3 trial
    |
    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • MGMT (6-O-methylguanine-DNA methyltransferase)
    |
    MGMT promoter methylation • IDH wild-type • IDH1 R132
    |
    temozolomide
    1m
    NRG-BN007: Testing the Use of the Immunotherapy Drugs Ipilimumab and Nivolumab Plus Radiation Therapy Compared to the Usual Treatment (Temozolomide and Radiation Therapy) for Newly Diagnosed MGMT Unmethylated Glioblastoma (clinicaltrials.gov)
    P2/3, N=159, Completed, National Cancer Institute (NCI) | Trial completion date: Sep 2026 --> Dec 2025 | Active, not recruiting --> Completed
    Trial completion • Trial completion date • Tumor mutational burden
    |
    PD-L1 (Programmed death ligand 1) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • MGMT (6-O-methylguanine-DNA methyltransferase)
    |
    MGMT promoter methylation • IDH1 R132
    |
    Opdivo (nivolumab) • Yervoy (ipilimumab) • temozolomide • ABP 206 (nivolumab biosimilar)