Trial Type: Blood Cancer
Trial Status: Recently Completed

Weili Sun, MD
Children’s Hospital of Los Angeles

Long term survival of children with acute myeloid leukemia (AML) has improved to nearly 60% over the last 50 years. The children who experience a relapse are more resistant to additional treatments because they develop a multi-agent drug resistance. As leukemia progresses, certain genes are “turned off” and this also contributes to their poor prognoses.

Epigeneticists study how turning on and off parts of the DNA sequence (instead of changing the actual sequence) affects how a cell functions. It has been show in laboratory tests that epigenetic modifiers may reverse AML chemo-resistance.

This multicenter clinical trial is the first pediatric study to test the concept of epigenetic intervention in concert with chemotherapy in children with AML. Dr. Sun and her team will evaluate whether drug resistance is reduced and if there is an improved response rate to this new combination of therapies. [Awarded 2012]


Clinical Summary
AML (acute myeloid leukemia) is the second most common leukemia in children; long term survival has improved to nearly approximately 60% over the last 50 years. However, treatment failure causes relapse in nearly half of all patients; once relapsed leukemia’s are more resistant to treatment and patient outcomes are very poor. Multi-agent drug resistance is the main challenge and the need for new strategies is urgent. As leukemia progresses, genes are “turned off” by a process called DNA methylation. DNA methylation may be reversed by agents called epigenetic modifiers, leading to novel treatment strategies. Drug induced demethylation has been shown to reverse chemo‐resistance of AML cells in vitro. The aim of this study is to test the novel epigenetic “priming” approach to use hypomethylating agent, 5-azacytidine (AZA), in combination with chemotherapy and evaluate whether epigenetic “priming” can reverse DNA methylation, overcome drug resistance, and increase response in relapsed/ AML. This is the first pediatric study to test the concept of epigenetic “priming” using DNA demethylating agents in combination with chemo- therapy in childhood AML.

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