Intrathecal Allogeneic B7-H3-targeted CAR γδ T Cells for Leptomeningeal Metastasis from Solid Tumors: Safety, Efficacy, and Immunological Dynamics in a Phase 1 Trial.
Early testing of off-the-shelf CAR T cells delivered directly to the brain shows promise for treating cancer spread to the spine and brain lining.
This Phase 1 trial evaluated intrathecal allogeneic B7-H3-targeted CAR γδ T cells (QH104) in 3 patients with leptomeningeal metastasis from solid tumors, demonstrating safety and preliminary anti-tumor activity. All patients had stable disease at day 30 with IFN-γ-associated immune remodeling in CSF, providing proof-of-concept for this novel off-the-shelf intrathecal CAR T strategy.
What the study was
- Study design
- Phase 1 clinical trial
- Population
- Patients with leptomeningeal metastasis from B7-H3-positive solid tumors (lung adenocarcinoma n=2, TNBC n=1)
- Sample size
- 3
- Category
- Treatment Innovation
- Maturity
- Exploratory
- Journal
- Clin Cancer Res
Why it surfaced
First published Phase 1 data for intrathecal allogeneic off-the-shelf CAR γδ T cells targeting B7-H3 in leptomeningeal metastasis — a disease with near-zero effective therapies. Novelty is very high (novel cell type, novel route, novel target in LM). Score capped by very small n=3; preliminary but mechanistically compelling with IFN-γ remodeling data.
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