KIAA1549

Paclitaxel-carboplatin chemotherapy was commenced with dose modifications for hepatotoxicity, complicated by infusion reactions, mild neuropathy, and mucositis. This case underlines the extreme morphological and molecular heterogeneity of hepatic carcinosarcomas, the rapid progression despite surgery, and the limited systemic treatment options available for such rare tumours.

Although no adjuvant therapy was required following complete resection, documentation of this fusion provides a rational framework for future molecularly guided treatment should disease recurrence occur. This case expands the spectrum of oncogenic BRAF fusion partners in LGG and underscores the importance of integrated RNA-based diagnostics and computational validation in precision neuro-oncology.

Demonstrating the mutation carried by the tumor is very critical in this regard. The purpose of this article is to focus on the treatment of thalamic glioma in pediatric patients in light of molecular information.

P1/2, N=50, Recruiting, St. Justine's Hospital | Not yet recruiting --> Recruiting

In this review, we synthesize current knowledge on MAPK pathway alterations in CNS tumors and evaluate the therapeutic landscape of MAPK inhibition, with emphasis on approved agents, emerging compounds, combination strategies, and novel drug-delivery technologies. We also discuss mechanisms that undermine treatment efficacy and highlight future directions aimed at integrating MAPK-targeted therapy into precision-based management of brain tumors.

High [18F] Fluciclovine uptake in pilocytic astrocytoma may reflect amino acid transporter expression, which may lead to overestimation of the WHO grade. Awareness of this pitfall is essential in pediatric neuro-oncology.

Dabrafenib plus trametinib has shown superiority over chemotherapy in pediatric low-grade gliomas and activity against high-grade diseases. Larotrectinib and entrectinib provide tumor-agnostic options for NTRK-fusion-positive tumors with central nervous system penetration. Selumetinib offers clinical benefits in NF1-associated plexiform neurofibromas and shows promise for treating NF1-related low-grade gliomas. Tovorafenib, a type II RAF inhibitor active in BRAF-altered tumors (including BRAFKIAA1549 fusion), achieved robust responses, thereby leading to FDA approval. ONC201 (dordaviprone) has received accelerated approval for the treatment of H3 K27M-mutant diffuse midline gliomas, with Japanese trials and patient-initiated programs expanding access. Abemaciclib, a CDK4/6 inhibitor, is under phase II evaluation for pediatric high-grade glioma and diffuse midline glioma, including sites in Japan. Neurosurgeons play a pivotal role in securing high-quality biopsies, thus enabling comprehensive molecular diagnostics and facilitating enrollment in international trials. This review summarizes current targeted therapies and ongoing studies and outlines practical considerations for integrating precision oncology into pediatric neuro-oncology in Japan.

This dual alteration necessitates multimodal therapeutic approaches targeting both pathways. This case emphasizes the importance of integrated diagnostics in glioma classification and personalized treatment, with further research needed to explore the implications of such rare co-occurrences.

The use of molecular diagnostics is emerging to be promising in the setting of pLGBG, which historically has been under investigated due to its anatomical location. Preliminary studies indicate that knowledge of molecular status can influence both diagnosis and treatment. Targeted treatment is the most exciting avenue for this, with exciting anecdotal data in the literature, and multiple clinical trials underway at this time.

ACTOnco+® identified a total of 329 heterozygous deletions, which were subsequently validated for reliability using FISH analysis. Gene profiling with ACTOnco+® exhibited comparable results to FoundationOne® CDx, thereby contributing to future personalized medicine endeavors.

DLGNT remains challenging to treat, with poor outcomes across modalities. Further investigation of treatment, including targeted therapies addressing activated pathways, is needed to improve patient survival.