Yongkui Jing, Ph.D. Ruibao Ren, M.D., Ph.D.
Mount Sinai School of Medicine Brandeis University

Development of Novel α, β-unsaturated Carbonyl Compounds as Therapeutic Agents in Myeloid Leukemia 

More than half of acute myeloid leukemia is caused by gene translocation and/or gene mutation. Gene translocation forms fusion products, which either block myeloid cell differentiation or protect cell death and have been found to cause leukemia in transgenic mice. Usually mutant genes are transcriptional factors that regulate gene expression network to keep a balance of cell proliferation and death. Mutation of these genes will lead to activation of a group of proteins that protect cells against death and lead to or contribute to the leukemogenesis. The gene fusion products and downstream targets of mutant genes have been used as therapeutic targets for leukemia.

Dr. Jing and Ren's research project has generated a novel group of small molecular compounds that target some fusion proteins and proteins that protect cell from death. These compounds might have potential to be developed as novel agents for leukemia treatment.