Dual epitope anti-LILRB4 synthetic T-cell receptor and antigen receptor (STAR)-T-cell therapy for relapsed/refractory acute myeloid leukemia
Novel T-cell therapy targeting an immunosuppressive receptor shows 50% response in treatment-resistant AML, with clues to improve outcomes through rational combination strategies.
This first-in-human trial of a novel nanobody-based STAR-T cell therapy targeting LILRB4 (an immunosuppressive receptor highly expressed on monocytic AML blasts) achieved an overall response rate of 50% in 6 evaluable R/R AML patients with an acceptable safety profile — no ICANS and no grade ≥3 cytokine release syndrome. Single-cell RNA sequencing revealed that monocyte-mediated suppression of autologous T-cell function is a primary mechanism of non-response, pointing to rational combination strategies for future optimization.
What the study was
- Study design
- First-in-human Phase 1 trial (NCT05548088)
- Population
- Adults with LILRB4-positive relapsed/refractory AML
- Sample size
- 9
- Category
- Treatment Innovation
- Maturity
- Exploratory
- Journal
- Signal Transduction and Targeted Therapy
Why it surfaced
First-in-human STAR-T cell therapy targeting LILRB4 in AML achieves 50% ORR with excellent safety in a disease with extreme unmet need; novel antigen target and cell engineering approach differentiate from CAR-T. Mechanistic scRNA-seq data immediately actionable for next-generation design.
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