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**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]
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