Clinical responses to trastuzumab deruxtecan in molecularly defined ependymoma.
Two patients with a rare brain tumor responded to HER2-targeted cancer therapy based on molecular testing, opening a potential new treatment avenue.
Two patients with molecularly defined ependymoma (confirmed HER2 expression by IHC) showed radiographic response or durable stability on trastuzumab deruxtecan, providing the first clinical evidence for HER2-directed ADC therapy in this rare CNS tumor. Integrated molecular and spatial profiling identified heterogeneous HER2 expression with distinct proliferative niches in each tumor subtype.
What the study was
- Study design
- Case series (n=2)
- Population
- Adults with molecularly defined ependymoma (MYCN-amplified and ZFTA-RELA-fused), treated with T-DXd after recurrence
- Sample size
- 2
- Category
- Treatment Innovation
- Maturity
- Exploratory
- Journal
- NPJ Precision Oncology
Why it surfaced
First clinical evidence of T-DXd (HER2-directed ADC) in molecularly defined ependymoma — high novelty and signal for rare CNS tumor precision oncology. Conservative scoring (n=2 case series) despite high-impact journal (NPJ Precis Oncol) and multi-institution team (Dana-Farber, Pitié-Salpêtrière).
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