The Need

Treatment failure is one of the leading causes of death in patients with human acute leukemia, yet the cause of treatment failure is still largely unknown. Overexpression of the RNA gene miR-3151 and its host gene, Brain and Acute Leukemia, Cytplasmic (BAALC), is a sign of an ineffective treatment outcome. Targeting miR-3151 as the causal agent of chemo-resistance could improve the clinical outcome in leukemia cases that overexpress BAALC, allowing for improved rates of remission with resilient cases of acute leukemia. Treatment that regulates the expression of miR-3151 in patients with human acute leukemia would improve their chance of survival.

The Technology

The BAALC gene is a prognostic marker for human acute leukemias: AML and ALL. Leukemia patients overexpressing BAALC show poor outcome and chemo-resistance to treatment, but the causal mechanisms are unknown. The Ohio State University researchers, led by Dr. Albert de la Chapelle, discovered that miR-3151 is part of the primary transcript of BAALC. MiR-3151 is responsible for the failure to achieve clinical remission in human acute leukemia and potentially in melanoma and brain tumors. Targeting miR-3151 as the causal agent of chemo-resistance might improve clinical outcome in leukemia cases that overexpress BAALC.

Commercial Applications

• Oncology therapeutics

Benefits/Advantages

• Novel target for improved therapeutic treatment
• miR-3151 and BAALC inhibit apoptosis of AML cells and primary AML patient blasts
• Overexpression of miR-3151, alone and in combination with BAALC, causes increased leukemogenesis in vivo