Rational combination platform trial design for children and young adults with diffuse midline glioma: A report from PNOC
Sabine Mueller,
Cassie Kline,
Andrea Franson
et al.
Abstract:Diffuse midline glioma (DMG) is a devastating pediatric brain tumor unresponsive to hundreds of clinical trials. Approximately 80% of DMGs harbor H3K27M oncohistones, which reprogram the epigenome to increase the metabolic profile of the tumor cells. We have previously shown preclinical efficacy of targeting both oxidative phosphorylation and glycolysis through treatment with ONC201, which activates the mitochondrial protease ClpP, and paxalisib, which inhibits PI3K/mTOR, respectively. This combination treatme… Show more
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