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Cancers
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Study on Clinical Treatment of Brain Tumors
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Study on Clinical Treatment of Brain Tumors

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

Deadline for manuscript submissions: 13 March 2026 | Viewed by 2657

Special Issue Editor

Dr. Shinya Watanabe

E-Mail Website
Guest Editor
Department of Neurosurgery, Institute of Medicine, University of Tsukuba, Tennodai 1-1-1, Tsukuba 305-8575, Ibaraki, Japan
Interests: brain cancer

Special Issue Information

Dear Colleagues,

In recent years, the development of antineoplastic agents has undergone significant changes, especially in the development of molecularly targeted agents. Drugs targeting driver mutations have shown remarkable efficacy, and in the clinical development of anti-tumor drugs for rare subtypes. The number of cases in which regulatory approval has been granted based on the primary efficacy endpoint of response rate in phase II trials is increasing. In addition, many pharmaceuticals are being introduced into clinical practice as genomic medicine, and it is expected that more drugs will be developed for tumors based on genetic abnormalities that are less common in the future.

However, brain tumors, especially malignant brain tumors such as glioblastoma, are still an area with a poor prognosis and high unmet medical needs, and the development of new treatment methods, including not only pharmaceuticals but also biotechnology products and medical devices, is sought after, and various clinical trials are being conducted. In this area, it is necessary not only to develop treatments, but also to create a consensus on efficacy endpoints for optimal response.

This Special Issue presents recent studies on the clinical treatment of brain tumors, working toward turning various brain tumors into prolonged overall survival.

Dr. Shinya Watanabe
Guest Editor

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 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. Cancers is an international peer-reviewed open access semimonthly 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 2900 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

  • brain tumors
  • clinical treatment
  • anti-tumor drugs
  • clinical trials

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Published Papers (3 papers)

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Research

14 pages, 633 KiB  
Article
Trends in Efficacy Endpoints in Phase II Glioblastoma Trials: A Regulatory Science Analysis (FY2020–FY2022)
by Shinya Watanabe, Makoto Maeda, Narushi Sugii, Masanobu Yamada, Yoshihiro Arakawa, Kimika Nakamura, Koichi Hashimoto and Eiichi Ishikawa
Cancers 2025, 17(5), 855; https://doi.org/10.3390/cancers17050855 - 1 Mar 2025
Viewed by 868
Abstract
Background/Objectives: In glioblastoma trials, efficacy evaluation often deviates from the standard Response Evaluation Criteria in Solid Tumors (RECIST), an objective response rate (ORR) method, because of the unique nature of brain tumors. In phase II trials from the fiscal years (FYs) 2017–2019, [...] Read more.
Background/Objectives: In glioblastoma trials, efficacy evaluation often deviates from the standard Response Evaluation Criteria in Solid Tumors (RECIST), an objective response rate (ORR) method, because of the unique nature of brain tumors. In phase II trials from the fiscal years (FYs) 2017–2019, primary endpoints (PEs) were overall survival (OS) at 29%, ORR at 20%, progression-free survival (PFS) at 17%, and OS rate at 10%. Clinical trial methodologies have likely evolved in recent years. This study analyzed trends in efficacy endpoint settings for phase II trials from FY2020 to FY2022 compared with FY2017–2019. Methods: Using Clarivate’s Cortellis™ Clinical Trial Intelligence database, 116 phase II glioblastoma trials initiated between April 2020 and March 2023 were identified. After exclusions, 88 trials were analyzed. Trial characteristics, PEs, secondary endpoints (SEs), and designs were summarized and compared to prior data. Results: Of 101 PEs in the 88 trials, approximately half targeted newly diagnosed patients, and most tested pharmaceutical products. The most common PEs were FS (22%), OS (20%), and PFS rate (17%), while among 299 SEs, OS (15%), PFS (15%), and quality of life (14%) were most frequent. Time-to-event outcomes were employed in 74 (73%) trials, whereas ORR was used as a PE in only 7 trials (8%). ORR as a PE was significantly lower than in FY2017–2019 (p = 0.022). Conclusions: Recent glioblastoma trials show increased diversity in efficacy endpoints with less reliance on ORR compared to earlier periods, reflecting evolving strategies to address the unique challenges of glioblastoma treatment and evaluation. Full article
(This article belongs to the Special Issue Study on Clinical Treatment of Brain Tumors)
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17 pages, 1272 KiB  
Article
Clinical Impact of a Next-Generation Sequencing Approach for Glioblastoma Patients
by Catalina Vivancos Sánchez, María Isabel Esteban Rodríguez, Alberto Peláez García, Mario Taravilla-Loma, Víctor Rodríguez-Domínguez, Carlos Rodríguez-Antolín, Rocío Rosas-Alonso, Itsaso Losantos-García, Alberto Isla Guerrero and María Luisa Gandía-González
Cancers 2025, 17(5), 744; https://doi.org/10.3390/cancers17050744 - 22 Feb 2025
Viewed by 771
Abstract
Objectives: The purpose of this study is to assess the clinical impact of next-generation sequencing (NGS), as an increasingly available and advantageous tool, for glioblastoma patients. Methods: Adult patients aged less than 65, and surgically treated for glioblastoma between 2010–2021, were included. Tumor [...] Read more.
Objectives: The purpose of this study is to assess the clinical impact of next-generation sequencing (NGS), as an increasingly available and advantageous tool, for glioblastoma patients. Methods: Adult patients aged less than 65, and surgically treated for glioblastoma between 2010–2021, were included. Tumor samples were analyzed with NGS using the Oncomine Comprehensive v3 (OCA) panel and Ion Reporter Genexus v5.9.1 (Thermo Fisher Scientific). Results: Thirty-two patients were included, with a median age of 47.7 years and a median overall survival of 25 months. Identification of mutations by NGS resulted in a change in diagnosis in two cases. In all patients but one, at least one genetic alteration was detected (median of three per patient), most commonly EGFR amplification. In 93.7% of patients, biomarkers that make them potentially eligible for a clinical trial were found. No survival differences were seen regarding genetic alterations, although a trend towards better survival for those patients without CDK4 mutation was observed (p = 0.088). Conclusions: The use of NGS provides useful information for diagnosis, especially in young patients, and it will probably become valuable for clinical decision-making as more therapeutic targets and treatments emerge. For the moment, it is crucial for scientific progress to happen. Full article
(This article belongs to the Special Issue Study on Clinical Treatment of Brain Tumors)
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17 pages, 1423 KiB  
Article
A Retrospective, Nationwide, Multicenter Study on Diagnosis and Treatment Outcome of Pediatric Optic Pathway/Hypothalamic Gliomas Including Analysis of Risk Factors for Progression After Systemic Anticancer Therapy
by Carlien A. M. Bennebroek, Judith van Zwol, Maartje C. Montauban van Swijndregt, Giorgio L. Porro, Rianne Oostenbrink, Anne T. M. Dittrich, Jan W. Pott, Lisethe Meijer, Etienne J. M. Janssen, Sylvia Klinkenberg, Noel J. Bauer, Irene C. Notting, Maria M. van Genderen, Michael W. Tanck, Pim de Graaf, Peerooz Saeed and Antoinette Y. N. Schouten-van Meeteren
Cancers 2025, 17(5), 716; https://doi.org/10.3390/cancers17050716 - 20 Feb 2025
Viewed by 604
Abstract
Background: The current standard therapy for pediatric optic pathway/hypothalamic glioma (OPHG) is systemic anticancer therapy (SAT) over surgery and radiotherapy. Nevertheless, recurrent radiological or clinical tumor progression after SAT forms a considerable challenge. Sporadic OPHGs are considered to have a higher tendency toward [...] Read more.
Background: The current standard therapy for pediatric optic pathway/hypothalamic glioma (OPHG) is systemic anticancer therapy (SAT) over surgery and radiotherapy. Nevertheless, recurrent radiological or clinical tumor progression after SAT forms a considerable challenge. Sporadic OPHGs are considered to have a higher tendency toward progression after first-line systemic anticancer therapy (SAT) compared to neurofibromatosis type-1-associated (NF1) OPHGs. Methods: The objective of this study was to conduct a national retrospective cohort analysis of children who received various treatments for a progressive OPHG, involving the hypothalamus and/or chiasm and/or optic radiations. The study aimed to examine the differences in clinical course and the range of treatment modalities applied to both sporadic and NF1-associated OPHGs between 1995 and 2020. Additionally, we sought to identify risk factors for 3- and 5-year progression following first- and second-order SAT. Results: In total, 136 children received treatment, of whom 49 of 136 (36.0%) had NF1. Within a median of 7.5 years (range: 0.1–23.8 years) of follow-up, sporadic OPHGs received more treatments compared to NF1-associated OPHGs (median of 2 (range: 1–8) vs. median of 1 (range: 1–7) (p < 0.01)). Nine children with sporadic OPHGs (6.6%) died. Of 112 children (82.4%) receiving SAT, 92% received combined first-line vincristine and carboplatin. These children had a 3- and 5-year progression-free survival of 61.8% (95% CI: 51.0–72.6%) and 48.4% (95% CI: 38.0–58.8%), respectively. Sporadic OPHGs had a higher rate of second progression (p < 0.01). Starting first-line vincristine and carboplatin at an age below one year was the only independent risk factor for progression. Conclusions: In this national historic cohort of pediatric OPHGs, four out of five children received SAT. Sporadic OPHGs received a higher number of various SATs compared to NF1-associated OPHGs, but the sporadic appearance of OPHGs was not an independent risk factor for progression after combined vincristine and carboplatin, as ‘age below one year at the start’ was the only factor. Full article
(This article belongs to the Special Issue Study on Clinical Treatment of Brain Tumors)
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