Funded by the Dick Vitale Pediatric Cancer Research Fund in memory of Austin Schroeder
Over the past decades, the cure rate of pediatric leukemia has significantly increased because of improved understanding of diagnosis, chemotherapy combinations, and supportive patient care. However, patients with MLL-rearranged subtype leukemia still face an unfavorable outcome and few therapeutic options. To date, the five-year survival of patients in this subtype is less than 70%, much lower than most other patients. To better improve the treatment outcome and increase the survival, many novel therapeutic drugs have been identified. Among these drugs, the inhibitors against BET proteins hold a great promise. So far, the mechanisms controlling drug response and resistance are not well understood. To our knowledge, our proposed research fits the goal of the V-foundation. We have conducted a successful genome-wide screen. We are going to study the function of novel candidate genes in drug resistance models in vitro and in vivo, as well as the working mechanism. Completing our proposed work is expected to significantly prevent and treat drug resistance and relapse, saving more patients with this deadly disease.