Molecular Biology and Phase 1 Study of GM-CSF and Intrathecal Trastuzumab in Children with Recurrent Posterior Fossa Ependymoma - PubMed
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- #Pediatric Oncology
- #Immunotherapy
- #Clinical Trial
- Study investigates safety and dosing of intrathecal trastuzumab combined with subcutaneous GM-CSF for pediatric recurrent posterior fossa ependymoma.
- ErbB2/Her2 identified as a top therapeutic antibody target for ependymoma via in silico screening.
- In vitro assays show trastuzumab induces antibody-dependent cell cytotoxicity in GM-CSF-stimulated co-cultures with ependymoma cell lines.
- Phase 1 clinical findings indicate the combination therapy is safe at tested dose levels, with 57% of patients completing all planned therapy.
- Median progression-free survival observed at 2.4 years; non-progressing patients had increased T-cell activity and reduced pro-tumor myeloid gene expression.
- Conclusion supports safety and suggests need for a larger phase 2 study to evaluate long-term efficacy in recurrent PFA and RELA-ST ependymoma.