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Molecular mechanisms and therapeutic targets in neuroblastoma

Journal article
Authors J. I. Johnsen
C. Dyberg
Susanne Fransson
M. Wickstrom
American Journal Of Pathology V. P. Stle Vp
Published in Pharmacological Research
Volume 131
Pages 164-176
ISSN 1043-6618
Publication year 2018
Published at Institute of Biomedicine, Department of Medical and Clinical Genetics
Pages 164-176
Language en
Links dx.doi.org/10.1016/j.phrs.2018.02.0...
Keywords Neuroblastoma, Targeted therapy, Investigational drugs, MYCN, ALK, Ras/MAPK, Clinical trial, childrens oncology group, high-risk neuroblastoma, bet-bromodomain, inhibition, phase-i consortium, mdm2 antagonist nutlin-3, refractory, solid tumors, high-dose chemotherapy, growth-factor receptor, wild-type, p53, n-myc, Pharmacology & Pharmacy
Subject categories Cancer and Oncology

Abstract

Neuroblastoma is the most common extracranical tumor of childhood and the most deadly tumor of infancy. It is characterized by early age onset and high frequencies of metastatic disease but also the capacity to spontaneously regress. Despite intensive therapy, the survival for patients with high-risk neuroblastoma and those with recurrent or relapsed disease is low. Hence, there is an urgent need to develop new therapies for these patient groups. The molecular pathogenesis based on high-throughput omics technologies of neuroblastoma is beginning to be resolved which have given the opportunity to develop personalized therapies for high-risk patients. Here we discuss the potential of developing targeted therapies against aberrantly expressed molecules detected in sub-populations of neuroblastoma patients and how these selected targets can be drugged in order to overcome treatment resistance, improve survival and quality of life for these patients and also the possibilities to transfer preclinical research into clinical testing. (C) 2018 The Authors

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