In vivo generation of anti-BCMA CAR-T cells in relapsed or refractory multiple myeloma: a phase 1 study - PubMed
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- #multiple myeloma
- #CAR-T therapy
- #clinical trial
- Study evaluates in vivo generation of anti-BCMA CAR-T cells for relapsed/refractory multiple myeloma.
- Phase 1 trial tested ESO-T01, a nanobody-directed lentiviral vector, without ex vivo manufacturing or lymphodepletion.
- Five heavily pretreated patients received a single infusion; median follow-up was 6.0 months.
- Primary endpoint was safety; no dose-limiting toxicities occurred, but all had grade 3+ adverse events.
- Cytokine release syndrome (CRS) occurred in four patients (three grade 3, one grade 2).
- Preliminary efficacy showed 4/5 patients achieved objective responses, including three stringent complete remissions.
- Minimal residual disease negativity (10^-5) was observed in all evaluable responders by day 60.
- Trial stopped early in 2025; findings support feasibility of in vivo CAR-T generation with immune-shielded vector.