Targeting oncogenic transcriptional programs in Juvenile Myelomonocytic Leukemia

Dr. Julia Maxson, Ph.D. – Oregon Health & Science University – OHSU, Portland, OR

Juvenile Myelomonocytic Leukemia (JMML) is a deadly form of childhood cancer. Most children with JMML are diagnosed before the age of three. Children with JMML receive intensive chemotherapy and often receive a bone marrow transplant—where the child’s own bone marrow is destroyed and replaced with that from a donor. Even with these harsh therapies, many children with JMML will have their leukemias return. Indeed, only half of children with JMML will survive for the first five years after their diagnosis. We need better treatments for this aggressive blood cancer. One key to developing new treatments may lie in a gene called SETBP1. Mutations in this gene make the leukemia much more likely to return. If we could target this bad-acting gene, we might be able to stop this leukemia from coming back. We have recently discovered that drugs developed for a different type of childhood leukemia might also work against SETBP1. The goal of our studies is to understand how mutations in SETBP1 cause hard-to-treat disease and to determine whether we can use these drugs to kill SETBP1-mutant leukemia cells.