4.7 Editorial Material

Access to new drugs in paediatric oncology: can we learn from the ongoing ONC201 saga?

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Summary: The imipridone ONC201 and its derivative ONC206 significantly reduce cell viability in pediatric diffuse midline gliomas (DMGs) and prolong the survival of DMG PDX models. Both drugs impair tumor cell growth and survival by activating mitochondrial metabolism and the integrative stress response (ISR), and trigger cell lineage differentiation. These findings are of great importance for the treatment of pediatric DMGs.

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