Clinical responses to trastuzumab deruxtecan in molecularly defined ependymoma - PubMed
5 hours ago
- #ependymoma
- #HER2-targeted therapy
- #antibody drug conjugate
- Two adults with molecularly defined ependymoma showed radiographic response or clinical stability with trastuzumab deruxtecan.
- HER2 expression was identified in both tumors via immunohistochemistry, aligning with prior ADC target profiling in CNS tumors.
- Multiplexed imaging revealed heterogeneous HER2 expression: one tumor had EGFR/MAPK-enriched proliferative niches, the other showed diffuse organization without strong coupling.
- The findings support HER2-directed antibody drug conjugates in ependymoma and emphasize integrated molecular/spatial profiling for rare CNS tumors.