Science Highlights
Published on September 02, 2025
Targeting pediatric EPNs with B7-H3 CAR T-cells
by Clinical Cancer Research
Mehta S, Ippagunta S, Varadharajan S, et al. B7-H3 CAR T-Cells Are Effective Against Ependymomas, But Limited by Tumor Size and Immune Response. Clinical Cancer Research. 2025; (doi: 10.1158/1078-0432.CCR-24-3083).
Study results indicate that cell therapy using B7 homolog 3 (B7-H3/CD276) may be a promising pathway in the pursuit of targeted therapies to treat ependymomas (EPNs). Based on positive results in 29 of 44 pediatric EPN samples analyzed, the protein appears to be highly but heterogeneously expressed across patients and an appropriate candidate for chimeric antigen receptor (CAR) T-cell interventions. After engineering second-generation human B7-H3 CAR T cells, researchers tested them in multiple models. The cells exhibited robust anti-EPN cytolytic activity, expansion, and persistence in vitro, activity that was inversely associated with upregulation of the protein on CAR T cells. Potent and significant anti-tumor effects were also observed in human xenograft EPN models, but the response varied based on extent of tumor burden and was not durable. Although outcomes were limited with murine B7-H3 CAR T cells, the study authors believe the findings warrant additional clinical assessment of B7-H3 CAR T cells for EPNs.
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