Medulloblastoma

Importantly, pharmacological inhibition of Sirt1 demonstrated promising therapeutic efficacy in both subcutaneous transplantation and primary MB models. Our findings identify Sirt1 as a potential therapeutic target for SHH-driven MB and other cancers.

These findings support a role for seasonal triggers in ALL and warrant further investigation in larger, genetically stratified cohorts.

Loss of Pten in normal or SmoM2-expressing GCPs increased proliferation and enhanced progenitor state initially, but by 12 days Pten mutant SmoM2 tumors were highly differentiated due to increased survival of non-proliferating GCPs. Furthermore, macrophage infiltration and cytotoxicity appeared reduced in differentiated regions of tumors lacking Pten, indicating cell nonautonomous changes could also contribute to accelerated tumor growth.

ResiPOx is a brain-penetrant immunomodulatory therapeutic that reshapes the immune-privileged brain tumor microenvironment. Systemic administration activates myeloid-driven anti-tumoral immunity mediated by microglial and macrophage TAMs, and improves survival in preclinical models of DMG and MB.

P=N/A, N=74, Recruiting, University of California, San Francisco | Suspended --> Recruiting

Moreover, combining AMBRA1 knockdown with FDA-approved SHH inhibitors enhances antitumor efficacy. These findings identify the AMBRA1/βTrCP/REN axis as a key regulatory mechanism in SHH signaling and discover an unrecognized function of AMBRA1 in MBSHH, providing actionable insights into brain tumor biology.

P1/2, N=25, Recruiting, Fundación de investigación HM | Phase classification: P2 --> P1/2

P1, N=90, Recruiting, Seattle Children's Hospital | Trial completion date: May 2041 --> May 2042 | Trial primary completion date: May 2026 --> May 2027

The review also addresses challenges such as intra-tumour heterogeneity, limited tissue availability, technical variability, and ethical considerations in paediatric oncology. Finally, we explore future directions, highlighting integrative, longitudinal, and ethically grounded biomarker strategies that have the potential to optimise therapy, minimise long-term toxicity, and improve both survival and quality of life for children with medulloblastoma.

P1/2, N=65, Recruiting, Nationwide Children's Hospital | Trial completion date: Nov 2027 --> Nov 2033 | Trial primary completion date: Nov 2027 --> Nov 2028

P=N/A, N=74, Suspended, University of California, San Francisco | Recruiting --> Suspended

SRS presents as a reasonable primary option or should be considered for early intervention after surgery. Patients and families should be counseled that multiple SRS sessions might be needed to achieve eventual tumor control.