Mukherjee · Cell reports. Medicine 2026 · preclinical animal and in vitro study · n=?

Ketogenic diet as a metabolic vehicle enhancing the therapeutic efficacy of mebendazole and devimistat in juvenile syngeneic high-grade glioma.

Level 5 - mechanism / opinion, no new human data

Preclinical in vitro and juvenile syngeneic mouse models with no human clinical data.

PubMed 42302750 · doi:10.1016/j.xcrm.2026.102845 · record verified 2026-08-26

What was done

Researchers evaluated the combination of nutritional ketosis via a ketogenic diet (KD) with mebendazole (MBZ) and devimistat (CPI-613) in juvenile syngeneic mice bearing invasive (VM-M3) and non-invasive (CT-2A, VM-NM1) glioblastoma models. The drugs were also tested in the human pediatric glioma cell line SF-188.

What was found

The abstract reports no numerical values, survival statistics, or effect sizes. Qualitatively, combining drug therapy with a ketogenic diet produced the greatest reductions in tumor invasion and progression and prolonged survival compared to individual therapies. In vitro, MBZ inhibited glycolysis and glutaminolysis in VM-M3 cells and decreased proliferation and viability in SF-188 cells. KD-enabled combination therapy also allowed lower drug dosing with reduced toxicity.

Why it matters

This study provides preclinical evidence that diet-induced ketosis can enhance the efficacy and reduce the required dosing and toxicity of repurposed metabolic drugs in high-grade glioma.

Limits

The findings are limited strictly to preclinical mouse and cell-line models, which may not translate to clinical efficacy or tolerability in human pediatric patients. The abstract provides no exact sample sizes, dosage details, or quantitative survival metrics.

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