Unraveling the Origins and Therapeutic Vulnerabilities of RBM15-MKL1-Driven AMKL
Childhood leukemia is a devastating cancer and many current treatments are not effective for certain aggressive forms of the disease. This project aims to uncover how specific cancer-causing genes alter normal cell processes to drive leukemia. In particular, we are investigating how these changes cause cells in very young infants to become cancerous. By understanding these mechanisms, we can identify new waysto block the cancer’s growth. Our findings will pave the way for the development of more precise, targeted therapies, offering hope for children with high-risk leukemia who currently have limited treatment options.
Project Goals
Infantile acute megakaryoblastic leukemia (AMKL) is a devastating disease with a poor prognosis. Thisis because we do not yet understand how this leukemia develops or how to effectively treat it. A specific genetic change referred to as the t(1;22) translocation, creates a fusion protein called RBM15-MKL1 (RM), to cause this disease. Our research aims to discover how RM functions to promote cells to become cancerous, with the goal of identifying novel, more effective treatment approaches. Additionally, we will test targeted treatments both individually and in combination, with the goal of developing targeted therapies that are more effective and lesstoxic to improve outcomes for these young patients.
