Mab immunotherapies for myeloid leukemia patients with germline or somatic RUNX1 mutations.

本研究提供了初步证据，并提出证实2种细胞表面分子CD11a （ITGAL）和IL3RA （CD123）是hsc中RUNX1的直接靶点（可能是抑制靶点），并且在RUNX1突变的AML中失调。针对这两种表面分子的单克隆抗体疗法已经针对不同疾病通过了不同的临床试验阶段。我们计划在临床前模型和临床试验中证明这些抗体对RUNX1阳性AML有效。

This proposal presents preliminary evidence and proposes to confirm that 2 cell surface molecules, CD11a (ITGAL) and IL3RA (CD123) are direct (probably repression) targets of RUNX1 in HSCs, and are dysregulated in RUNX1 mutated AML. Monoclonal antibody therapies that target these two surface molecules have already passed different clinical trial phases for different diseases. We plan to show these antibodies are effective in RUNX1 positive AML in preclinical models and then clinical trials.