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Current Oncology
Special Issues
Clinical Outcomes and New Treatments in Pediatric Brain Tumors
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Clinical Outcomes and New Treatments in Pediatric Brain Tumors

A special issue of Current Oncology (ISSN 1718-7729). This special issue belongs to the section "Neuro-Oncology".

Deadline for manuscript submissions: 15 August 2025 | Viewed by 5360

Special Issue Editors

Dr. Sébastien Perreault

E-Mail Website
Guest Editor
Division of Child Neurology, CHU Sainte-Justine, Montréal, QC H3T 1C5, Canada
Interests: pediatric neuro-oncologie; low-grade gliomas; targeted therapy; clinical trials
Dr. Craig Erker

E-Mail Website
Guest Editor
Division of Pediatric Hematology-Oncology, IWK Health Centre, Halifax, NS B3K 6R8, Canada
Interests: pediatric neuro-oncology; response assessment; pediatric high-grade glioma; infant medulloblastoma

Special Issue Information

Dear Colleagues,

Brain tumors are currently the leading cause of cancer deaths in pediatrics. The heterogeneity and aggressiveness of pediatric brain tumors have historically made them difficult to treat. The purpose of this Special Issue is to discuss the latest clinical outcomes and therapeutic treatments for pediatric brain tumors. Given the fact that new treatment approaches now overlap multiple tumor types, we will cover topics by reviewing various fields, from targeted therapies to new surgical techniques.

In this Special Issue, original research articles and reviews are welcome. Research areas may include (but are not limited to) the following:

  1. Medical and patient perspectives;
  2. Observational approaches;
  3. Targeted therapies;
  4. Other small molecules;
  5. Chemotherapy;
  6. Immunotherapy;
  7. Radiation therapy approaches;
  8. Surgery;
  9. New devices;
  10. Approaches to reduce toxicity.

We look forward to receiving your contributions.

Dr. Sébastien Perreault
Dr. Craig Erker
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Current Oncology is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2200 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • pediatric neuro-oncology
  • central nervous system tumors
  • treatment approaches

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Further information on MDPI's Special Issue policies can be found here.

Published Papers (6 papers)

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Review

16 pages, 298 KiB  
Review
Small-Molecule Drugs in Pediatric Neuro-Oncology
by Stephanie Vairy and George Michaiel
Curr. Oncol. 2025, 32(8), 417; https://doi.org/10.3390/curroncol32080417 - 25 Jul 2025
Viewed by 96
Abstract
Advances in molecular diagnostics have enabled precision medicine approaches in pediatric neuro-oncology, with small-molecule drugs emerging as promising therapeutic candidates targeting specific genetic and epigenetic alterations in central nervous system (CNS) tumors. This review provides a focused overview of several small-molecule agents under [...] Read more.
Advances in molecular diagnostics have enabled precision medicine approaches in pediatric neuro-oncology, with small-molecule drugs emerging as promising therapeutic candidates targeting specific genetic and epigenetic alterations in central nervous system (CNS) tumors. This review provides a focused overview of several small-molecule agents under investigation or in early clinical use, including ONC201, tazemetostat, vorasidenib, CDK inhibitors, selinexor, and aurora kinase A inhibitors, among others. Highlighted are their mechanisms of action, pharmacokinetic properties, early efficacy data, and tolerability in pediatric populations. Despite encouraging preclinical and early-phase results, most agents face limitations due to study heterogeneity, lack of large-scale pediatric randomized trials, and challenges in drug delivery to the CNS. The review underscores the critical need for robust prospective clinical trials for the integration of these therapies into pediatric neuro-oncology care. Full article
(This article belongs to the Special Issue Clinical Outcomes and New Treatments in Pediatric Brain Tumors)
26 pages, 359 KiB  
Review
Old Tools in a New Era: The Continued Relevance of Chemotherapy in Pediatric Neuro-Oncology
by Kathleen Felton, Lucie Lafay-Cousin and Sylvia Cheng
Curr. Oncol. 2025, 32(7), 410; https://doi.org/10.3390/curroncol32070410 - 20 Jul 2025
Viewed by 225
Abstract
Conventional chemotherapy continues to form the backbone of treatment for many pediatric central nervous system (CNS) tumors. Advances have been made especially in the molecular underpinning of certain pediatric CNS tumors, allowing for advancement and consideration in incorporating this molecular information in molecular [...] Read more.
Conventional chemotherapy continues to form the backbone of treatment for many pediatric central nervous system (CNS) tumors. Advances have been made especially in the molecular underpinning of certain pediatric CNS tumors, allowing for advancement and consideration in incorporating this molecular information in molecular targeted therapy or appropriate de-escalation or escalation of therapy. In very young children with embryonal CNS tumors, intensive high-dose chemotherapy approaches have been used with varied increased survival in medulloblastoma, atypical teratoid rhabdoid tumor (ATRT), and rare embryonal subtypes, but there are certain molecular risk groups that require new therapies, such as the ATRT MYC subtype. Some CNS tumors remain resistant or refractory to conventional chemotherapy, especially in relapsed disease. Strategies to explore combination therapies with chemotherapy, novel agents, and novel approaches are needed to improve survival in this population in the future. Full article
(This article belongs to the Special Issue Clinical Outcomes and New Treatments in Pediatric Brain Tumors)
14 pages, 606 KiB  
Review
Approaches to Reduce Toxicity in Pediatric Brain Tumors
by Hallie Coltin, Christina Coleman and Chantel Cacciotti
Curr. Oncol. 2025, 32(5), 281; https://doi.org/10.3390/curroncol32050281 - 15 May 2025
Viewed by 635
Abstract
Pediatric central nervous system (CNS) tumor survivors are highly susceptible to long-term toxicity due to tumor location and also the treatment received. Advancements in treatment techniques, risk-adapted approaches to therapy with adjustments to treatment regimens—including de-escalation when feasible—along with the addition of supportive [...] Read more.
Pediatric central nervous system (CNS) tumor survivors are highly susceptible to long-term toxicity due to tumor location and also the treatment received. Advancements in treatment techniques, risk-adapted approaches to therapy with adjustments to treatment regimens—including de-escalation when feasible—along with the addition of supportive therapy and surveillance in these survivors, serve to minimize and manage late effects of therapy. Full article
(This article belongs to the Special Issue Clinical Outcomes and New Treatments in Pediatric Brain Tumors)
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23 pages, 710 KiB  
Review
Precision Medicine for Pediatric Glioma and NF1-Associated Tumors: The Role of Small Molecule Inhibitors
by Samuele Renzi, Julie Bennett, Nirav Thacker and Chantel Cacciotti
Curr. Oncol. 2025, 32(5), 280; https://doi.org/10.3390/curroncol32050280 - 15 May 2025
Viewed by 1218
Abstract
Pediatric gliomas encompass the most common brain tumor in children and are subdivided into pediatric low-grade gliomas (pLGGs) and pediatric high-grade gliomas (pHGGs). The era of molecular diagnosis has shifted the treatment paradigms and management of these patients. RAS/MAPK pathway alterations serve as [...] Read more.
Pediatric gliomas encompass the most common brain tumor in children and are subdivided into pediatric low-grade gliomas (pLGGs) and pediatric high-grade gliomas (pHGGs). The era of molecular diagnosis has shifted the treatment paradigms and management of these patients. RAS/MAPK pathway alterations serve as the driver in the majority of pLGGs, a subset of pHGG and NF1-related plexiform neurofibromas (PNs). The role of small molecule inhibitors in the treatment of these tumors has evolved in the past decade, facilitated through multiple clinical trials and moving into earlier stages of treatment. Although these developments hold promise, questions remain regarding targeted therapy, the long-term toxicities, the duration of treatment and the potential effects on the natural history of the tumor behavior. Full article
(This article belongs to the Special Issue Clinical Outcomes and New Treatments in Pediatric Brain Tumors)
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Figure 1

14 pages, 488 KiB  
Review
Tumor-Treating Fields and Related Treatments in the Management of Pediatric Brain Tumors
by Julien Rousseau, Sarah Lapointe and David Roberge
Curr. Oncol. 2025, 32(4), 185; https://doi.org/10.3390/curroncol32040185 - 21 Mar 2025
Viewed by 1483
Abstract
Pediatric primary brain tumors pose significant therapeutic challenges due to their aggressive nature and the critical environment of the developing brain. Traditional modalities like surgery, chemotherapy, and radiotherapy often achieve limited success in high-grade gliomas and embryonal tumors. Tumor-treating fields (TTfields), a non-invasive [...] Read more.
Pediatric primary brain tumors pose significant therapeutic challenges due to their aggressive nature and the critical environment of the developing brain. Traditional modalities like surgery, chemotherapy, and radiotherapy often achieve limited success in high-grade gliomas and embryonal tumors. Tumor-treating fields (TTfields), a non-invasive therapy delivering alternating electric fields, has emerged as a promising approach to disrupt tumor cell division through mechanisms such as mitotic disruption, DNA damage, and tumor microenvironment modulation. TTfields are thought to selectively target dividing tumor cells while sparing healthy, non-dividing cells. While TTfields therapy is FDA-approved for the management of glioblastoma and other cancers, its application in pediatric brain tumors remains under investigation. Preclinical studies reveal its potential in medulloblastoma and ependymoma models, while observational data suggest its safety and feasibility in children. Current research focuses on optimizing TTfields’ efficacy through advanced technologies, including high-intensity arrays, skull remodeling, and integration with immunotherapies such as immune checkpoint inhibitors. Innovative device-based therapies like magnetic field-based technologies further expand the treatment possibilities. As clinical trials progress, TTfields and related modalities offer hope for addressing unmet needs in pediatric neuro-oncology, especially for tumors in challenging locations. Future directions include biomarker identification, tailored protocols, and novel therapeutic combinations to enhance outcomes in pediatric brain tumor management. Full article
(This article belongs to the Special Issue Clinical Outcomes and New Treatments in Pediatric Brain Tumors)
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28 pages, 1398 KiB  
Review
Pediatric CNS Radiation Oncology: Recent Developments and Novel Techniques
by Justin Oh, Samir Patel, Mary-Pat Schlosser, Andrew J. Arifin, Carol Oliveira, Anne-Marie Charpentier and Derek S. Tsang
Curr. Oncol. 2025, 32(3), 180; https://doi.org/10.3390/curroncol32030180 - 20 Mar 2025
Cited by 1 | Viewed by 1258
Abstract
Radiation therapy (RT) is a cornerstone in the management of pediatric central nervous system (CNS) tumors. Recent advancements in RT delivery and techniques aim to enhance therapeutic effectiveness while minimizing both acute and long-term complications associated with pediatric brain RT. This paper highlights [...] Read more.
Radiation therapy (RT) is a cornerstone in the management of pediatric central nervous system (CNS) tumors. Recent advancements in RT delivery and techniques aim to enhance therapeutic effectiveness while minimizing both acute and long-term complications associated with pediatric brain RT. This paper highlights innovative developments in the field, including the clinical indications, benefits, and challenges of proton therapy and stereotactic radiotherapy. The ongoing refinement of risk-adapted RT volumes is highlighted, with examples of newly proposed germinoma RT volumes and hippocampal-sparing RT. Additionally, emerging experimental approaches, including FLASH therapy and theranostics, are also discussed as promising future directions. Further prospective, multi-institutional collaborative studies are essential to validate and expand upon the benefits outlined in this review. Full article
(This article belongs to the Special Issue Clinical Outcomes and New Treatments in Pediatric Brain Tumors)
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