Molecular Mechanisms and Treatment of Brain Cancer: Recent Advances and Future Trends

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Pharmacology".

Deadline for manuscript submissions: 25 June 2025 | Viewed by 1976

Special Issue Editor

Special Issue Information

Dear Colleagues,

Brain cancer is a serious issue in the global burden of diseases. It is estimated that 3.24 million new cancer cases and 1.66 million cancer deaths will be registered across Europe in 2024 and 40,800 of these deaths are from brain and central nervous system (CNS) cancers. Despite extensive efforts in molecular biology research, advances in biomedical engineering, artificial intelligence (AI) and big data science, brain tumors remain among the deadliest forms of cancer, resisting almost all conventional and novel treatments.

The aim of this Special Issue is to focus on recent advances and trends in the diagnosis, treatment (standard and newly developed, as well as natural substances) and the development of new in vitro, in vivo or ex vivo models in the field of brain cancer. Original articles and reviews are welcome for publication on the topic.

Dr. Terezia Kiskova
Guest Editor

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Keywords

  • brain cancer
  • molecular mechanism
  • treatment
  • diagnosis
  • in vitro
  • in vivo
  • models

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Published Papers (2 papers)

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Research

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29 pages, 6589 KiB  
Article
Identification of Deregulated miRNAs and mRNAs Involved in Tumorigenesis and Detection of Glioblastoma Patients Applying Next-Generation RNA Sequencing
by Dóra Géczi, Álmos Klekner, István Balogh, András Penyige, Melinda Szilágyi, József Virga, Andrea Bakó, Bálint Nagy, Bernadett Torner and Zsuzsanna Birkó
Pharmaceuticals 2025, 18(3), 431; https://doi.org/10.3390/ph18030431 - 19 Mar 2025
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Abstract
(1) Background: Glioblastoma (GBM) is one of the most aggressive brain tumors with a poor prognosis. Therefore, new insights into GBM diagnosis and treatment are required. In addition to differentially expressed mRNAs, miRNAs may have the potential to be applied as diagnostic biomarkers. [...] Read more.
(1) Background: Glioblastoma (GBM) is one of the most aggressive brain tumors with a poor prognosis. Therefore, new insights into GBM diagnosis and treatment are required. In addition to differentially expressed mRNAs, miRNAs may have the potential to be applied as diagnostic biomarkers. (2) Methods: In this study, profiling of human miRNAs in combination with mRNAs was performed on total RNA isolated from tissue samples of five control and five GBM patients, using a high-throughput RNA sequencing (RNA-Seq) approach. (3) Results: A total of 35 miRNAs and 365 mRNAs were upregulated, while 82 miRNAs and 1225 mRNAs showed significant downregulation between tissue samples of GBM patients compared to the control samples using the iDEP tool to analyze RNA-Seq data. To validate our results, the expression of five miRNAs (hsa-miR-196a-5p, hsa-miR-21-3p, hsa-miR-10b-3p, hsa-miR-383-5p, and hsa-miR-490-3p) and fourteen mRNAs (E2F2, HOXD13, VEGFA, CDC45, AURKB, HOXC10, MYBL2, FABP6, PRLHR, NEUROD6, CBLN1, HRH3, HCN1, and RELN) was determined by RT-qPCR assay. The miRNet tool was used to build miRNA–target interaction. Furthermore, a protein–protein interaction (PPI) network was created from the miRNA targets by applying the NetworkAnalyst 3.0 tool. Based on the PPI network, a functional enrichment analysis of the target proteins was also carried out. (4) Conclusions: We identified an miRNA panel and several deregulated mRNAs that could play an important role in tumor development and distinguish GBM patients from healthy controls with high sensitivity and specificity using total RNA isolated from tissue samples. Full article
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Review

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15 pages, 255 KiB  
Review
A 5-Year Update on the Clinical Development of Cancer Cell-Based Vaccines for Glioblastoma Multiforme
by Almohanad A. Alkayyal and Ahmad Bakur Mahmoud
Pharmaceuticals 2025, 18(3), 376; https://doi.org/10.3390/ph18030376 - 6 Mar 2025
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Abstract
Glioblastoma multiforme (GBM) is considered one of the most aggressive forms of brain cancer with a 15-month median survival, despite advancements in surgery, radiotherapy, and chemotherapy. The immune-suppressed tumor microenvironment and the blood–brain barrier are major contributors to its poor prognosis and treatment [...] Read more.
Glioblastoma multiforme (GBM) is considered one of the most aggressive forms of brain cancer with a 15-month median survival, despite advancements in surgery, radiotherapy, and chemotherapy. The immune-suppressed tumor microenvironment and the blood–brain barrier are major contributors to its poor prognosis and treatment resistance. In the last decade, significant progress has been made in developing cell-based vaccines to boost immune responses against GBM. This review provides an extensive update on recent clinical trials involving various cancer cell vaccines, including ICT-107, the α-type-1 DC vaccine, and others. Although these trials have demonstrated potential improvements in progression-free survival (PFS) and overall survival (OS), the diverse and immune-suppressed nature of GBM poses challenges for consistent therapeutic success. We discuss the details of these trials along with the potential mechanism of vaccine efficacy and immune activations. The findings of these trials highlight the significance of a personalized immunotherapy approach and suggest that patient stratification could significantly advance the clinical management of GBM. Full article
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