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Niclosamide for the treatment of relapsed pediatric acute myeloid leukemia

Kathleen Sakamoto

Kathleen Sakamoto


Stanford University

Project Term: July 1, 2019 - June 30, 2022

Niclosamide is an FDA approved anti-parasitic drug that is well tolerated in adults and children. AML cells are sensitive to niclosamide, act synergistically with chemotherapy in vitro, and inhibit the proliferation of primary AML stem cells in vivo. We propose to examine the effects of niclosamide in combination with chemotherapy in animal models of AML and study the mechanism of action of niclosamide in AML cells in anticipation of a clinical trial in children with relapsed AML.

Lay Abstract

The prognosis for relapsed AML in children is poor. We identified a drug known as niclosamide that was used to treat parasites and is well tolerated in children. Our preliminary data demonstrated that AML cells from children are sensitive to niclosamide alone and enhances the effects of chemotherapy. Our goal is to study the effects of niclosamide in animal models of AML and understand how niclosamide kills AML cells. The long-term goal of this project is to design a clinical trial with niclosamide and chemotherapy for children with relapsed AML to improve their survival and quality of life in the future.

Translational Research Program
Grant Subprogram
TRP Basic
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