Development of CAR T Cells Targeting a Surface RNA-Binding Protein for the Treatment of Acute Leukemias.

CAR T cells targeting surface U5 snRNP200, an RNA-binding protein absent from normal hematopoietic precursors, effectively treat AML and B-cell ALL with improved safety.

• Why it matters: Developing targeted therapies for AML is difficult due to the scarcity of antigens that distinguish leukemia cells from healthy blood cells, risking damage to normal tissue.
• What they did: Researchers engineered CAR T cells based on alloantibodies from cured AML patients, targeting U5 snRNP200, and enhanced them with IL-18 to improve efficacy and durability in preclinical models.
• The result: The modified CAR T cells achieved durable remission, prevented AML rechallenge, and demonstrated a promising platform for safer, more selective leukemia immunotherapy.