Medulloblastoma

P1, N=15, Recruiting, C17 Council | Not yet recruiting --> Recruiting

P3, N=96, Not yet recruiting, Nationwide Children's Hospital

P1, N=102, Recruiting, Jazz Pharmaceuticals | Trial primary completion date: Dec 2025 --> Jul 2026

Further, pharmacokinetic studies in mice revealed improved plasma disposition, higher AUC, and slower elimination for Lipo/GlaB compared to the free drug. These findings support the therapeutic value of Lipo/GlaB as a selective and potent strategy for targeting HH-dependent cancers, offering improved biopharmaceutical properties and in vivo efficacy compared to non-formulated GlaB.

P2, N=22, Active, not recruiting, St. Jude Children's Research Hospital | Trial completion date: Dec 2025 --> Dec 2035 | Trial primary completion date: Oct 2025 --> Oct 2035

This well-characterized model provides a physiologically and functionally relevant platform for further dissecting the reciprocal interactions present between various brain cancer cells and vascular endothelial cells, supporting the development of targeted therapeutic strategies and advancing our understanding of brain tumor biology.

Low-cost methods resolved 90% of cases; NanoString clarified the remainder, achieving 98.8% precision. This first molecular characterization of pediatric MB in Argentina demonstrates that a tiered, conventional-tools-based approach can deliver high diagnostic accuracy and prognostic value, supporting its integration into routine care in resource-limited settings.

P=N/A, N=500, Recruiting, Universitätsklinikum Hamburg-Eppendorf | Suspended --> Recruiting

This case represents the first report of a BCOR ITD in an IDH-mutant WHO grade 4 astrocytoma. The discovery expands the molecular spectrum of high-grade astrocytomas and highlights the need for further research into the biological, prognostic, and therapeutic significance of BCOR alterations in these tumors.

Consistently, GPC6 enhanced SHH pathway activity by upregulating GLI1 expression, supported ciliogenesis that is essential for signal transduction, and facilitated the secretion of SHH ligands via extracellular vesicles. These findings establish GPC6 as a critical regulator of SHH-MB progression and highlight its potential as a therapeutic target.

Immunofluorescence, NGS analysis, and DNA methylation profiling revealed that the PDTs faithfully mirrored the cellular nature, the genetic and epigenetic landscape of their matched primary tumors. Our study shows the feasibility of generating PDTs, even from rarer entities, that recapitulate the genetic and epigenetic features of primary tumors, highlighting their potential as models for tumor biology studies and precision medicine.

IDH1 inhibition reverses resistance-mediated chromatin changes and enables radiation re-sensitization. Ultimately, these findings demonstrate the efficacy of single-cell multiome analysis in elucidating resistance mechanisms and identifying targetable pathways for MYC-driven medulloblastoma.