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New Gene Target Discovered for Predicting the Risk of Extramedullary Infiltration in Leukemia for its Prevention and Treatment

New biomarker discovered for predicting the risk of extramedullary infiltration, which reportedly indicates poor outcome of leukemia

Background

Extramedullary infiltration (EMI) refers to leukemic cells found in organs or tissue outside the primary site in the bone marrow to other tissues and organs, significantly worsening the prognosis. Among the various forms of EMI, myeloid sarcoma, which involves tumor formation in subcutaneous tissues or organs, has been challenging to replicate pathogenesis in animal models. Underlying mechanisms of myeloid sarcoma onset remain poorly understood, hindering the development of effective biomarkers and therapeutic strategies.

Description and Advantages

Kyoto University researchers have successfully developed a novel mousemodel with a high incidence of myeloid sarcoma using immunodeficient mice implanted with specific acute myeloid leukemia (AML) cells.Additionally, they identified a gene associated with EMI among highly expressed genes in the tumor tissues formed in this animal model and in leukemia cells of EMI patients.Furthermore, the knockout of this gene (Fig. 1) or administration of inhibitors in AML cells (Fig. 2) ledto reduced infiltration ability, decreased tumor formation rates in mice, and prolonged overall survival.

Identification of the gene involved in human EMI
New strategy for the companion diagnostics and therapeutics for EMI

Development
Status
・Identified a novel gene as a marker for EMI and myeloid sarcoma development.
• Confirmed the tumorigenesis suppression through gene knockout or inhibitor administration in the model mice.
• Ongoing studies on myelodysplastic syndromes and chronic myeloid leukemia
Offer • Patent License
• Option for Patent License
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