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April 5, 2026Cancer Research

Abstract 3707: Durable remission of high-risk and refractory neuroblastoma by PHOX2B derived peptide-HLA directed CAR T cells: An IND enabling study

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Authors

MWMuzamil Y. WantRKRicha KapoorDGDavid Groff

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Overview

IND-enabling study demonstrates potent efficacy of PHOX2B CAR T cells in refractory neuroblastoma, suggesting a novel treatment approach.

Key Points

  • The aim is to explore the efficacy and safety of PHOX2B-targeted CAR T cells in treating high-risk neuroblastoma.
  • IND-enabling studies of PHOX2B PC-CAR T cells using lentiviral transduction of T cells
  • Characterization of PHOX2B expression across various solid tumors via RNA-seq and IHC
  • Assessment of safety using cross-reactivity algorithms and testing on normal primary cell lines
  • Evaluation of cytotoxic activity using IncuCyte, cytokine profiling, and flow cytometry analysis
  • PHOX2B PC-CAR T cells achieved complete regression in neuroblastoma xenografts for over 50 days
  • High specificity was demonstrated with no cross-reactivity in non-HLA-A24/23 cancers
  • Immunophenotyping showed CD8+ effector-memory predominance after T-cell infusion
  • Durable responses were seen for over 120 days, with relapse in some models leading to spontaneous regression or re-treatment.

Cite This Study

Want et al. (2026) studied this question.

synapsesocial.com/papers/69d1fe18a79560c99a0a4a72https://doi.org/10.1158/1538-7445.am2026-3707
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