CNS Tumor

P1, N=70, Not yet recruiting, Robbie Majzner

We propose: (1) Refining age-specific GGT thresholds via repeated measurements and restricted cubic splines; (2) Integrating GGT with carbohydrate antigen 19-9, carcinoembryonic antigen, and KRAS/GNAS circulating tumor DNA in machine-learning radiomic models for personalized 5-year malignancy risks per Fukuoka guidelines; and (3) Validating cost-effectiveness in multi-ethnic populations before screening. This repositions GGT as a globally available redox biosignature for PCN early detection, potentially reducing pancreatic cancer burden.

Phakomatoses-associated CNS tumors show promising outcomes with multimodality management. Further research into targeted multimodality treatment is warranted.

They also represent the first PLAG1 fusions identified in pediatric supratentorial ependymal tumors. These cases highlight the value of integrating histology, methylation profiling, and fusion detection, and suggest a new candidate supratentorial ependymal subtype with PLAG1 fusions, pending validation in larger series.

Epstein-Barr virus (EBV)-associated smooth muscle rare tumors (EBV-SMTs) in HIV patients with low CD4 counts require clinical suspicion and Epstein-Barr virus (EBV) involvement in brain tumor diagnosis. Early HIV detection, CD4 monitoring, and antiretroviral therapy (ART) management can decrease the risk of these malignancies.

B7-H3 CAR T cells combined with ONC206 is a feasible and efficacious multi-agent approach against multiple DIPG models.

Our findings provide strong support for the combined targeting of TGF-β and PD-L1 as a promising immunotherapeutic strategy to overcome immunosuppressive barriers in glioblastoma and induce potent antitumor responses.

Around 5-6% of MBs are associated with inherited cancer predisposition syndromes, with common genetic variants including PTCH1, SUFU, TP53, and SMO. This report describes the first pediatric patient harboring a CHEK2 germline variant of uncertain significance and developing a EA- MB localized at the trigeminal nerve and subsequent CNS and EN metastases.

P=N/A, N=600, Not yet recruiting, Tata Memorial Centre | Initiation date: Dec 2025 --> Mar 2026

Given its rarity, diagnosis of cerebellar angiosarcoma requires a high index of suspicion, supported by advanced imaging and immunohistochemical profiling. Multidisciplinary management is essential, and further case documentation is necessary to guide therapeutic strategies and prognostication.

In addition, the in vivo efficacy was also assessed in xenograft mouse models of non-CNS tumors, where PSMA CAR T cell treatment resulted in significant tumor regression and robust accumulation of CAR T cells within the tumor microenvironment. Together, these findings establish PSMA as a promising surface antigen beyond prostate cancer and provide preclinical evidence supporting the development of PSMA-directed therapies for this highly lethal pediatric cancer.

Molecular diagnostics play a crucial role in guiding appropriate care and management for patients with TS. Early diagnosis, genetic testing, and preventive measures are essential for the effective management of this condition.