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Massaro Laboratory: The biology of platelets and leukemia

Dr. Stephanie Massaro devotes her time to the study of acute megakaryoblastic leukemia (AMKL) is a rare form of leukemia that affects megakaryocytes, which are platelet-making blood cells. The disease most commonly strikes very young children. Approximately 30% of pediatric patients diagnosed with AMKL are infants who have a specific genetic abnormality that involves two genes, RBM15 and MKL1. These genes may play important roles in normal blood cell development. However, when they are incorrectly linked together, they may contribute to leukemia development by altering Notch signaling, a normal cell signaling pathway responsible for cellular growth and maturation. This aberrant Notch signal may cause a failure of the tumor suppressor protein Rb and result in uncontrolled growth of immature megakaryocytes. This abnormality is associated with an extremely poor outcome, with an average survival time of only eight months from diagnosis despite aggressive medical therapy.

This project combines clinical interest in malignant hematology and basic scientific approach to understanding the pathogenesis of AMKL. Dr. Massaro’s commitment to this project is strengthened by my interactions with the patients she follows clinically. This information may lead to superior childhood leukemia outcomes by improving diagnostic strategies and developing new immuno- and chemotherapeutic agents.