Successfully Treated AML Patients May Hold the Key to New CAR T Cell Therapy
In animal models, the new MSK-developed CAR T cells targeting the U5 snRNP200 protein demonstrated remarkable effectiveness, sparing healthy blood‑forming cells while eliminating leukemia and providing long‑lasting protection.
Memorial Sloan Kettering Cancer Center researchers engineered a novel CAR T‑cell therapy that recognizes the U5 snRNP200 protein—a surface marker found mainly on acute myeloid leukemia (AML) cells but absent on normal hematopoietic cells. Preclinical studies in mouse models showed that these CAR T cells eradicate AML and B‑ALL cells, including CD19‑negative B‑ALL, while preserving healthy blood‑forming cells and eliciting durable immune memory. The therapy, derived from antibodies isolated from patients who achieved long‑term remission after bone marrow transplant, may offer a safer, more precise treatment for AML and potentially overcome current resistance mechanisms.
Consistent targeting of a leukemia‑specific surface protein could reduce toxicity to normal blood cells, improve survival for AML patients—especially those who cannot undergo transplant—and expand CAR T‑cell applicability to other leukemias such as CD19‑negative B‑ALL.
Evidence level: Korai humán adat. Kis vagy feltáró emberi adat.