Prospects of targeted therapy of acute leukemias in children

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Abstract

Acute leukemias (ALL and AML) amount to more than one third of all malignant diseases in children. Even modern intensive risk-adapted chemotherapy fails (non-responses and relapses) in 20% of children with ALL and in 40–50% of children with AML, and hematopoietic stem cell transplantation (HSCT) can salvage only 40-50% of resistant and relapsed patients. New data on aberrant activation of signaling pathways and aberrant epigenetic events in malignant cells lead to invent of new drugs that selectively affect different mechanisms of leukemic transformation. The presented review briefly describes the main types of targeted therapy of AL in children. It includes the assessment of the efficacy of enzymatic agents and their modifications, the possibility of inhibition of tyrosine kinases, proteasomes, epigenetic regulators of gene expression, histone deacetylase, disruptor of telomeric signaling-1, monoclonal antibodies and conjugated immunotoxins, bispecific antibodies activating T-cells and T-cells with modified chimeric antigen receptor.

About the authors

A. G. Rumyantsev

National Scientific and Practical Center of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev

Author for correspondence.
Email: info@fnkc.ru
ORCID iD: 0000-0002-1643-5960

MD, DSc, Professor, Academician of the Russian Academy of Science, General Director

Russia 117997, Moscow, Samory Mashela st., 1

Russian Federation

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Copyright (c) 2017 Rumyantsev A.G.

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