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Anti-ROBO1 CAR-T cells are a second-generation chimeric antigen receptor (CAR) T-cell therapy designed to target the Roundabout guidance receptor 1 (ROBO1). ROBO1 is an axonal guidance protein that is aberrantly upregulated in malignant and treatment-refractory brain tumor initiating cells (BTICs), particularly in recurrent glioblastoma. Developed by researchers at McMaster University and the National Research Council Canada, these CAR-T cells utilize a single-domain antibody (nanobody) binding region to recognize ROBO1. In preclinical studies, the therapy has demonstrated potent anti-tumor activity, doubling median survival in patient-derived xenograft (PDX) models of recurrent glioblastoma and showing efficacy in models of lung-to-brain metastases and pediatric medulloblastoma.
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