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Pediatric Cancer: Leukemia

New Epigenetic Approach to Leukemia Therapy for Children

Weili Sun, MD
Children's Hospital of Los Angeles

Clinical Title: New Epigenetic Approach to Leukemia Therapy

Clinical Description:

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 chemotherapy in childhood AML.

Status: Active Trial

Accepting Patients? Yes

NIH Clinical Trial Link:

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