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BrainChild-01

Development stage
Phase 2
Lead developer
Seattle Children's
Modality
CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies
Administration
Intracranial, intra-Ommaya, Intra-ventricular, intra-VP Shunt
01

Overview

**BrainChild-01** is an investigational autologous CAR T-cell therapy developed by Seattle Children's for relapsed or refractory HER2-positive pediatric central nervous system (CNS) tumors, such as medulloblastoma, ependymoma, and glioma (excluding DIPG). It involves reprogramming a patient's own CD4+ and CD8+ T cells via lentiviral transduction to express a HER2-specific chimeric antigen receptor (CAR), which targets and destroys HER2-expressing tumor cells while sparing healthy brain tissue. The therapy is administered locoregionally through an indwelling catheter directly into the CNS resection cavity or ventricular system to bypass the blood-brain barrier, enabling repeated doses with potentially reduced systemic toxicity like cytokine release syndrome.[1][2][3][4][13]

Other names
HER2-specific chimeric antigen receptor T cellHER-2-specific chimeric antigen receptor T cellHER 2-specific chimeric antigen receptor T cellHER2-CAR T cellsHER-2-CAR T cellsHER 2-CAR T cellsHER2-specific CAR T cellsHER-2-specific CAR T cellsHER 2-specific CAR T cells
02

Targets

ERBB2 (Erb-b2 receptor tyrosine kinase 2)

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