Nat Med: Clinical study of local infusion of HER2-specific CAR-T cells in the treatment of children and young patients with relapsed/refractory central nervous system tumors

CAR-T cells (chimeric antigen receptor T cells) have been confirmed to have clinical effects on malignant tumors of the hematopoietic system including glioblastoma, but the current CAR-T cell therapy strategy is in the central nervous system of children ( CNS) Feasibility and tolerability in tumors are still to be studied

CAR-T cells (chimeric antigen receptor T cells) have been confirmed to have clinical effects on malignant tumors of the hematopoietic system including glioblastoma, but the current CAR-T cell therapy strategy is in the central nervous system of children ( CNS) Feasibility and tolerability in tumors are still to be studied

In recent years, HER2 has been developed as a target of CAR-T cells, which is an attractive target for the treatment of central nervous system tumors

stem cell

However, compared with breast cancer , the expression of HER2 in central nervous system tumors is lower, which also limits the application of related HER2 targeting antibodies

Breast cancer researchers demonstrated that a medium-length CAR spacer domain can enhance the therapeutic effect of HER2-specific CAR-T cells in orthotopic transplantation of medulloblastoma models

Schematic diagram of the BrainChild-01 trial design

Schematic diagram of the BrainChild-01 trial design

The researchers translated these findings into BrainChild-01 (NCT03500991), a phase 1 clinical trial conducted at Seattle Children’s Hospital to evaluate the effects of these HER2-specific CAR-T cells on relapsed/refractory CNS tumors (including Diffuse midline glioma) repeated topical doses for children and young adults

The researchers translated these findings into BrainChild-01 (NCT03500991), a phase 1 clinical trial conducted at Seattle Children’s Hospital to evaluate the effects of these HER2-specific CAR-T cells on relapsed/refractory CNS tumors (including Diffuse midline glioma) repeated topical doses for children and young adults

Neuroimaging after local CAR-T cell perfusion

Neuroimaging after local CAR-T cell perfusion

The results showed that the first three patients did not experience dose-limiting toxicity and showed activation of local CNS immunity , including high concentrations of CXCL10 and CCL2 detected in the cerebrospinal fluid

The interim report of

Locoregional infusion of HER2-specific CAR T cells in children and young adults with recurrent or refractory CNS tumors: an interim analysis.

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