Childhood Cancer

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Single-Cell Profiling of Acute Myeloid Leukemia for High-Resolution Chemo-immunotherapy Target Discovery

Stanford University School of Medicine
Charles Gawad, MD/PhD
Grant Type: 
Single-cell Pediatric Cancer Atlas Grant
Year Awarded: 
Type of Childhood Cancer: 
Leukemia, Acute Myeloid Leukemia (AML)
Project Description: 

Lay Summary: Despite enormous efforts to find better treatments for children with acute myeloid leukemia (AML), it remains one of the most difficult to treat pediatric cancers. The children that are fortunate enough to survive the intense treatment regimens suffer both short-term and lifelong side effects of their treatments. A major cause of the challenges in treating AML is the differences between cells present within leukemia that have varied responses to a given treatment. This study will utilize recently developed technologies that are able to analyze AML samples one cell at a time, providing insights into the disease at the cellular level. We produce this higher-resolution understanding of the disease using these new technologies and will make our data immediately accessible to the research community, with the aim of accelerating our efforts to find new ways to cure all children with AML.

Lay Summary Project Goal: The goal of this project is to use new technologies, including methods recently invented in my lab, to characterize the genetic material present in thousands of AML cells isolated from each patient. We anticipate that studying the disease one cell at a time will enable us to better understand why some cells are resistant to specific treatments and to determine how all the cell types interact to cause both the formation of the disease and their persistence during treatment. The complementary technologies we will utilize for this study will provide different views of each cell, including how the DNA has changed and which genes are turned on and off. The data produced from these new tools will be integrated into an online tool where researchers worldwide can easily search the cells in each sample for new clues on the biology of the disease. The ultimate goal of this study is to use this deeper understanding of AML to devise better methods and strategies for diagnosing and treating children with this deadly pediatric cancer.