Science Highlights
Published on July 28, 2026
Assessment of PRAME-Directed Immunotherapy in AML
by Clinical Cancer Research
Liu X, Li J, Li X, et al. A Novel Nanobody-Based TCR-Like CAR T Therapy Targeting PRAME for the Treatment of Acute Myeloid Leukemia. Clinical Cancer Research. 2026; (doi: 10.1158/1078-0432.CCR-25-2726).
Scientists are encouraged by early findings that point to preferentially expressed antigen in melanoma (PRAME) as a feasible alternative to usual chimeric antigen receptor (CAR) T-cell therapy for acute myeloid leukemia (AML). Conventional CAR T-cell therapies often underperform in this disease setting due to a dearth of cell-surface antigens that are specifically expressed on leukemic blasts but are limited in or completely absent from normal hematopoietic stem/progenitor cells (HSPCs) and healthy tissues. In response, researchers shifted their focus to nanobodies aimed at PRAME, which is highly expressed in AML cells but not in HSPCs and is associated with unfavorable AML outcomes. Nanobodies developed to target PRAME425-433/human leukocyte antigen A2 were used as the building blocks of a novel T-cell receptor–like CAR T-cell intervention. The cells exhibited specific and robust antileukemic cytotoxicity against PRAME+ AML cells in vitro and in vivo but did not impair or disrupt normal HSPCs.
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