Kate Amato Foundation Awarded Projects
Novel Targeted Therapy to Eliminate Leukemic Stem Cells in Pediatric Acute Myeloid Leukemia
Project Goal: To develop a pioneering approach to target pediatric leukemic stem cells that could very likely lead to a new class of drugs for precision medicine in pediatric AML therapy.

Institution: The David Geffen School of Medicine at UCLA
Researchers: Dr. Martina Roos, Dr. Vivian Chang and Dr. John Chute
Year Awarded: 2018
Type of Childhood Cancer: Pediatric Acute Myeloid Leukemia
Project Description:
Dr. Martina Roos, Dr. Vivian Chang and Dr. John Chute at The David Geffen School of Medicine at UCLA, whose project is entitled “Novel Targeted Therapy to Eliminate Leukemic Stem Cells in Pediatric Acute Myeloid Leukemia,” and seeks to develop a pioneering approach to target pediatric leukemic stem cells that could very likely lead to a new class of drugs for precision medicine in pediatric AML therapy. Relapse following initial treatment is the biggest problem in pediatric acute myeloid leukemia (AML), a devastating blood cancer where blood producing stem cells are damaged and become abnormally functioning, chemotherapy-resistant leukemic stem cells. We identified novel compounds that inhibit important growth mechanisms of these leukemic stem cells and show remarkable anti-leukemia activity in animal models of pediatric AML. We believe that our project is a pioneering, innovative approach to target pediatric leukemic stem cells and has high potential to develop a new class of drugs for precision medicine in pediatric AML therapy.