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Neuroblastoma: Molecular Insights and Clinical Implications

A special issue of Cancers (ISSN 2072-6694). This special issue belongs to the section "Molecular Cancer Biology".

Deadline for manuscript submissions: 30 April 2026 | Viewed by 6106

Special Issue Editor


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Guest Editor
Division of Pediatric Surgery, Department of Surgery, Rush University Medical Center, Chicago, IL 60612, USA
Interests: neuroblastoma

Special Issue Information

Dear Colleagues,

Neuroblastoma has long frustrated clinicians as it is a very heterogeneous, oftentimes aggressive cancer of childhood. It is the most common non-brain solid tumor in children, and the survival remains under 70% for most patients. Therefore, physician scientists as well as bench scientists have long sought to understand this disease in order to find better targeted therapies. The articles in this issue will focus on some of the newer treatment modalities that go beyond traditional chemotherapies and include immunotherapies and small molecule therapies. This issue is dedicated to showcasing some of the most up-to-date developments in the field. The hope is that by understanding the molecular mechanisms of neuroblastoma, we will find new targets for therapy and improve survival in children with this tumor.

Dr. Mary Beth Madonna
Guest Editor

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Keywords

  • neuroblastoma
  • molecular mechanisms
  • targeted therapies
  • immunotherapies
  • small molecule therapies

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Published Papers (2 papers)

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Review

19 pages, 693 KB  
Review
MYCN-Driven Metabolic Networks Are a Critical Dependency of High-Risk Neuroblastomas
by Michelle G. Pitts, Lindsay T. Bryant, Michael D. Buoncristiani and Eric J. Rellinger
Cancers 2025, 17(19), 3256; https://doi.org/10.3390/cancers17193256 - 8 Oct 2025
Viewed by 524
Abstract
Neuroblastoma is a devastating pediatric solid tumor that, despite significant recent advances, still accounts for nearly 15% of all childhood cancer deaths. Patients are risk stratified based on a number of features, including amplification of the MYCN oncogene, yet targeting MYCN itself has [...] Read more.
Neuroblastoma is a devastating pediatric solid tumor that, despite significant recent advances, still accounts for nearly 15% of all childhood cancer deaths. Patients are risk stratified based on a number of features, including amplification of the MYCN oncogene, yet targeting MYCN itself has been unsuccessful to date. The complex interplay between this oncogene and its many metabolic targets has proven challenging and is only beginning to be understood in the context of pediatric tumors. It is increasingly recognized, however, that MYCN-driven metabolic rewiring and concomitant increases in biosynthetic precursors has the potential to drive many aspects of tumor development. Furthermore, emerging research suggests that improving overall therapeutic outcomes for neuroblastoma patients may well require individual metabolic profiling, allowing personalized simultaneous targeting of multiple metabolic nodes. In this review, we outline clinically relevant research involving MYCN-driven metabolic derangements, including increased glucose uptake, polyamine synthesis, glycosylation, and others, and attempt to summarize the influence of MYCN on important metabolic genes and druggable protein targets. We spotlight emerging research in glycosylation and its modulation as an often overlooked but increasingly promising therapeutic area. It is our hope that this document will provide utility for both clinicians and scientists seeking to understand how the MYCN oncogene and metabolism are critically intertwined. Full article
(This article belongs to the Special Issue Neuroblastoma: Molecular Insights and Clinical Implications)
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25 pages, 1258 KB  
Review
The Neuroblastoma Microenvironment, Heterogeneity and Immunotherapeutic Approaches
by Panagiotis Alkinoos Polychronopoulos, Oscar C. Bedoya-Reina and John Inge Johnsen
Cancers 2024, 16(10), 1863; https://doi.org/10.3390/cancers16101863 - 13 May 2024
Cited by 7 | Viewed by 4867
Abstract
Neuroblastoma is a peripheral nervous system tumor that almost exclusively occurs in young children. Although intensified treatment modalities have led to increased patient survival, the prognosis for patients with high-risk disease is still around 50%, signifying neuroblastoma as a leading cause of cancer-related [...] Read more.
Neuroblastoma is a peripheral nervous system tumor that almost exclusively occurs in young children. Although intensified treatment modalities have led to increased patient survival, the prognosis for patients with high-risk disease is still around 50%, signifying neuroblastoma as a leading cause of cancer-related deaths in children. Neuroblastoma is an embryonal tumor and is shaped by its origin from cells within the neural crest. Hence, neuroblastoma usually presents with a low mutational burden and is, in the majority of cases, driven by epigenetically deregulated transcription networks. The recent development of Omic techniques has given us detailed knowledge of neuroblastoma evolution, heterogeneity, and plasticity, as well as intra- and intercellular molecular communication networks within the neuroblastoma microenvironment. Here, we discuss the potential of these recent discoveries with emphasis on new treatment modalities, including immunotherapies which hold promise for better future treatment regimens. Full article
(This article belongs to the Special Issue Neuroblastoma: Molecular Insights and Clinical Implications)
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