Medulloblastoma

P2, N=20, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Dec 2025 --> Dec 2026

We provide an overview of the current checkpoint inhibitor landscape for pediatric brain tumors, highlight barriers and summarize possible approaches that can be efficaciously explored in future clinical trials.

Although the lesion lacks histopathologic confirmation and a sporadic occurrence cannot be excluded, this observation raises the possibility that germline DNMT3A alterations may predispose to a broader range of tumors than previously recognized. This report underscores the need for continued documentation of unusual tumor presentations in TBRS to further elucidate the biology of DNMT3A-related tumorigenesis.

This case demonstrates that the extremely rare leptomeningeal spread of medulloblastoma can clinically and radiologically mimic TBM, posing a significant diagnostic challenge in pediatrics. In the absence of systemic signs of tuberculosis, lack of response to treatment, and the presence of progressive neuroimaging abnormalities, the diagnosis should be reconsidered. Early histopathological confirmation is essential to ensure the timely initiation of oncological treatment.

P2, N=23, Recruiting, H. Lee Moffitt Cancer Center and Research Institute | Trial primary completion date: Feb 2026 --> Feb 2027

UL88 emerged as the most strongly expressed HCMV gene across MB samples. These findings suggest a potential prognostic and therapeutic role for HCMV in MB, warranting validation in larger, prospective cohorts.

The limited antitumor activity of EphA2-CAR T cells could be overcome with second genetic modifications that increase T cell functionality including deletion of DNMT3A or the expression of a constitutively active IL-18 chimeric cytokine receptor. Thus, our study nominates EphA2-CAR T cells as a promising alternative to B7-H3-CAR T cells, which are actively being explored in clinical studies for medulloblastoma.

Executive deficits are observed in individuals with brain tumors or survivors, significantly affecting their academic, social, and emotional lives. Early identification, along with educational and neuropsychological support, is essential to preventing these deficits from interfering with academic, personal, and professional functioning.

The results demonstrated the potential of using cilia-promoting drugs, such as Alvocidib and Alisertib, to suppress cancer cell replication. Additionally, it shows the massive benefits of integrating accessible large language models to conduct sweeping, rapid, and accurate literature searches.

Integration of Wnt-centric gene signatures and machine-learning risk models now enables patient stratification and outcome prediction. Targeting context-dependent Wnt signaling, combined with rational combinatorial immunotherapy and TME reprogramming, offers a promising precisiononcology strategy to overcome therapeutic barriers in Wnt-driven malignancies.

A sonic hedgehog (SHH)-MB model, including a Gorlin syndrome patient neuroepithelial stem cell line (NES) and its tumor derivative (tNES), was used to evaluate single and combined treatments of PI3K, AKT, FGFR, and CDK4/6 inhibitors (BYL719, AZD5363, JNJ-42756493, and PD-0332991, respectively). This study illustrates that single and combined administrations of PI3K, FGFR, CDK4/6, and AKT inhibitors in a NES/tNES model have dose-dependent and additive/synergistic anti-MB activity impacting tumor growth. Their effects on tNES cells were generally more pronounced than on NES; however, the difference in proliferative capacity between the cells should be considered.

P2, N=232, Recruiting, Medical University of Vienna | N=100 --> 232