Project 467033

Acute myeloid leukemia (AML) is an aggressive blood cancer with a 5-year survival rate of 21%. AML occurs when the bone marrow produces a large number of unhealthy cells that prevent the growth and functions of normal cells. The only cure for AML patients is to undergo a stem cell transplant where normal blood stem cells from a healthy donor are transplanted into the patients. These donor cells have the ability to prevent AML from re-emerging, but AML still frequently returns after transplant, leading to disease relapse. To reduce relapse and improve the long-term effectiveness of transplants, new treatment options are needed. Lysine-specific demethylase 1 (LSD1) is a protein that has been a promising therapeutic target for treating AML. Blocking the function of LSD1 has also been shown to increase immune cells ability to recognize and kill cancer cells. Therefore, this project aims to study the ability for LSD1 blockers to activate and enhance immune responses against AML in the context of stem cell transplant. We will characterize the underlying molecular mechanisms that contribute to a potentially heightened anti-leukemia immune response. Mice models that mimic the relevant biological environment will also be used to evaluate the safety and efficacy of LSD1 blockers. Eventually, our goal is to gather sufficient experimental evidence to support a clinical trial that further examines the LSD1 blockers ability to prevent relapse after stem cell transplant.