Authors
L Nicolas Gonzalez Castro, Yi-Chien Wu, Jia-Ren Lin, Maria-Joao Santiago-Ribeiro, Alice Laurenge, Alexandre Carpentier, Shannon Coy, Nancy U Lin, Karima Mokhtari, Keith L Ligon, Mehdi Touat, Sandro Santagata
Published in
NPJ precision oncology. Jun 20, 2026. Epub Jun 20, 2026.
Abstract
Despite evidence that antibody drug conjugates (ADCs) are active in brain metastases, their role in primary central nervous system (CNS) tumors remains unclear. We report two adults with molecularly defined ependymoma - a MYCN-amplified spinal tumor with intracranial dissemination and a supratentorial ZFTA-RELA-fused tumor with multiple recurrences - who demonstrated radiographic response and/or durable clinical and metabolic stability with trastuzumab deruxtecan (T-DXd). HER2 expression was identified by immunohistochemistry in both tumors, consistent with prior systematic profiling of ADC targets in CNS tumors. Multiplexed imaging showed broad but heterogeneous HER2 expression across tumor states in both cases; in the MYCN-amplified tumor, this occurred alongside EGFR/MAPK-enriched proliferative niches, whereas the ZFTA-RELA tumor showed more diffuse organization without strong coupling of EGFR and proliferation. These findings provide early clinical evidence supporting HER2-directed ADCs in ependymoma and highlight the value of integrated molecular and spatial profiling in interpreting therapeutic response in rare CNS tumors.
PMID:
42321415
Bibliographic data and abstract were imported from PubMed on 20 Jun 2026.
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