Medulloblastoma

Unexpectedly, we demonstrated that MYC triggers lipid storage, creating a unique dependency on lipid droplet-mitochondria communications to sustain tumor maintenance in vivo. Together, this comprehensive analysis reveals a targetable vulnerability downstream of MYC that constitutes a promising therapeutic approach to treat currently untreatable medulloblastoma subtypes.

P2, N=1376, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: May 2026 --> Jan 2027

These findings provide new structure-function insights into a key signaling complex mutated in cancer and other diseases. Summary: Alphafold3 predicts a more extended interaction of the GLI1 transcription factor with the tumor suppressor SUFU than has been seen in published x-ray structures; these predictions are supported by structure-function experiments, including analysis of patient-derived missense variants of SUFU.

Cyclin D1 can be used as a complementary marker in WNT-AG detection. ROR2 negativity may indicate G3 tumors, though further studies are warranted to confirm its prognostic value.

Meta-analyses of clinical data reveal that neonatal hyperbilirubinemia (jaundice) caused by high levels of bilirubin, increases pediatric brain tumor risk. Collectively, these findings highlight the origins of sex differences in neurodevelopment and brain tumorigenesis, offering insights into early screening and prevention of pediatric brain tumors through targeted interventions addressing modifiable risk factors.

In flies, reduction of PARP activity ameliorated the tumor associated phenotypes of SA1-deficient tissue. Our in vivo and in vitro data suggest that impairment of PARP activity compensates for reduced cohesin activity, highlighting a vulnerability that might be pharmacologically exploited in brain tumors.

The results show that hsa_circ_PCNT promotes SHH-MB aggression through the hsa-miR-133b /TAGLN2 pathway. In summary, our research demonstrates that hsa_circ_PCNT occupies a crucial position in SHH-MB and emerges as a potential therapeutic target.

Innovations in targeting (e.g., GLUT1, RVG peptide, ApoE mimetic peptide) and non-invasive delivery (e.g., focused ultrasound) are critical to overcome the BBB limitations. This review highlights the different strategies that can be utilized to deliver RNA-based therapies to the brain and summarizes the recent clinical efforts to deliver the RNA.

One signature distinguished neuroblastoma samples with sensitivity to navitoclax, a BCL2 family inhibitor...The combination of multiomics analysis and drug sensitivity profiling identified two signatures related to drug sensitivity in pediatric solid tumors, contributing to the advancement of functional precision medicine and personalized treatment strategies. This article is part of a special series: Driving Cancer Discoveries with Computational Research, Data Science, and Machine Learning/AI .

Knockdown of JARID1B in human G3MB cell lines reduced growth, supporting potential as a therapeutic target. We conclude that a MYC-TGFβ-JARID1B axis represses target genes to drive G3MB and present new humanized models for G3MB to understand epigenetic dysregulation in G3MB.

P1, N=15, Recruiting, C17 Council | Not yet recruiting --> Recruiting | Trial completion date: Sep 2032 --> Dec 2032 | Trial primary completion date: Sep 2030 --> Dec 2030

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