Infusion of HER2-Specific CAR T Cells into Brain or CNS “Feasible” in Pediatric Patients


ASTCT recently published an article in their journal Nucleus detailing CAR T-cell in pediatric patients with brain cancer.

The following was recently published in ASTCT’s Nucleus Plus: Emerging CAR T publication.

An interim analysis of the ongoing BrainChild–01 trial (NCT03500991) suggests that repetitive locoregional infusion of HER2-specific CAR T cells into the brain or the cerebrospinal fluid of children and young adults with recurrent or refractory tumors of the brain or central nervous system (CNS) may be well tolerated and may activate a localized immune response. The analysis was published in Nature Medicinein July.1

Locoregional delivery of CAR T cells has produced objective responses in adults with glioblastoma, but these are the first published findings on the viability of this approach in pediatric patients. Infusions are administered on an outpatient basis by means of a CNS catheter into either the tumor cavity or the ventricular system.

The 3 patients in this preliminary dataset were aged 16, 19, and 26 years at the time they received the investigational CAR T-cell therapy. All 3 patients were diagnosed before the age of 19 and had previously received at least 3 tumor-directed surgeries, at least 1 radiation treatment, and at least 1 chemotherapy regimen.

“The initial 3 patients experienced no dose-limiting toxicity and exhibited clinical, as well as correlative laboratory, evidence of local CNS immune activation, including high concentrations of CXCL10 and CCL2 in the cerebrospinal fluid,” report principal investigator Nicholas A. Vitanza, MD, and colleagues at the Seattle Children’s Research Institute. “This interim report supports the feasibility of generating HER2-specific CAR T cells for repeated dosing regimens…”


1. Vitanza NA, Johnson AJ, Wilson AL, et al. Locoregional infusion of HER2-specific CAR T cells in children and young adults with recurrent or refractory CNS tumors: an interim analysis. Nat Med. July 12, 2021. doi:10.1038/s41591-021-01404-8

2. Helwick C. HER2-Specific CAR T-Cell Trial Addresses Pediatric Brain and Spinal Cord Tumors. ASCO Post. July 19, 2021.

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