Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

Emerging and Future Targeted Therapies for Pediatric Acute Myeloid Leukemia: Targeting the Leukemia Stem Cells

Version 1 : Received: 14 November 2023 / Approved: 15 November 2023 / Online: 16 November 2023 (02:22:24 CET)

A peer-reviewed article of this Preprint also exists.

Murphy, L.A.; Winters, A.C. Emerging and Future Targeted Therapies for Pediatric Acute Myeloid Leukemia: Targeting the Leukemia Stem Cells. Biomedicines 2023, 11, 3248. Murphy, L.A.; Winters, A.C. Emerging and Future Targeted Therapies for Pediatric Acute Myeloid Leukemia: Targeting the Leukemia Stem Cells. Biomedicines 2023, 11, 3248.

Abstract

Acute myeloid leukemia (AML) is a rare subtype of acute leukemia in the pediatric and adolescent population but causes disproportionate morbidity and mortality in this age group. Standard chemotherapeutic regimens for AML have changed very little in the past 3-4 decades, but addition of targeted agents in recent years have led to improved survival in select subsets of patients as well as a better biologic understanding of the disease. One key paradigm of bench-to-bedside practice in the context of adult AML currently is the focus on leukemia stem cell (LSC)-targeted therapies. Here we review current and emerging immunotherapies and other targeted agents that are in clinical use for pediatric AML, through the lens of what is known (and not known) about their LSC-targeting capability. Based on a growing understanding of pediatric LSC biology, we also briefly discuss potential future agents on the horizon.

Keywords

pediatric AML; immunotherapy; targeted therapy; leukemia stem cell (LSC)

Subject

Medicine and Pharmacology, Oncology and Oncogenics

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