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Niclosamide for the Treatment of Relapsed/Refractory Pediatric Acute Myeloid Leukemia

Kathleen Sakamoto

Kathleen Sakamoto


Stanford University

Project Term: June 30, 2022 - June 30, 2025

Niclosamide is an FDA approved anti-parasitic drug that is well tolerated and acts synergistically with chemotherapy to kill AML cells. We will conduct a Phase I clinical trial with niclosamide in combination with cytarabine for children with relapsed/refractory pediatric AML. ShRNA/CRISPR screens demonstrated that Bcl-2 is upregulated in niclosamide resistant cells. We will study the effects of the Bcl-2 inhibitor venetoclax in combination with niclosamide in pediatric 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 conduct a Phase I clinical trial with niclosamide and cytarabine. In addition, our preliminary work showed that niclosamide synergizes with the FDA-approved drug venetoclax. We propose to study the effects of niclosamide and venetoclax in pre-clinical models of pediatric AML. These studies will allow us to design the next clinical trial following the Phase I trial. 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.

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