Loyola University Chicago Stritch School of Medicine Archives

2014

Scholar Grant

Dr. Charles Hemenway

Maywood, IL

Acute myelogenous leukemia (AML) in children remains a challenging disease to treat successfully. We have reached a treatment plateau, despite high dose chemotherapy followed by allogeneic bone marrow transplantation for high-risk AML. Relapse remains the most common cause for failure of this aggressive treatment, followed by treatment-related deaths. Yet, improved understanding of the unique biology of AML has revealed clues about potential weaknesses of AML cells that could guide new treatments. For example, a protein known as FLT-3 is found in abundance in high-risk AML. Drugs recently have been developed that inhibit FLT-3 functioning, and some of these drugs are now being tested in Pediatric clinical trials. Another protein that is found at high levels in more aggressive types of childhood AML is known as MLLT11. Unlike FLT-3, however, the function of MLLT11 is poorly understood. One approach to uncover the function of a given protein is to identify other proteins that associate with it; knowing these protein partners allows predictions to be made and tested to determine the function of MLLT11. Once determined, a focused approach can be employed to develop drugs to block that function. We will take advantage of two distinct experimental techniques to identify protin partners of MLLT11 in order to reveal its function. We also will use genetic tricks to block MLLT11 in AML cells to determine its importance to the survival of these cells. Taken together, the findings of these experiments may provide new avenues for the successful treatment of childhood AML.