scRNA-seq and multiplexed imaging of ETMR tumors. Credit score: Nature Most cancers (2025). DOI: 10.1038/s43018-025-00964-9
A research led by researchers at Dana-Farber/Boston Kids’s Most cancers and Blood Issues Middle and the Broad Institute of MIT and Harvard has uncovered crucial insights into the biology of embryonal tumor with multilayered rosettes (ETMR), a uncommon and aggressive mind tumor affecting younger youngsters.
The findings, revealed in Nature Most cancers, provide promising avenues for focused therapies that might enhance outcomes for sufferers with this devastating illness.
ETMRs are pushed by a novel genetic alteration generally known as the C19MC microRNA cluster, which retains tumor cells in an immature and aggressive state. By single-cell and spatial evaluation of affected person tumors, the analysis group found that ETMR cells mimic early mind improvement, forming a hierarchy of stem-like and neuron-like cells. This mobile cooperation is essential for tumor progress, with extra mature cells offering indicators that assist stem-like cells thrive.
Co-lead writer of the research, Dr. Volker Hovestadt, defined, “We discovered that FGFR and NOTCH receptors are expressed by proliferative NSC-like cells, while more differentiated malignant cells within the same tumor supply the corresponding ligands, indicating a coordinated role in sustaining tumor growth. Targeting these interactions may represent a critical vulnerability in ETMR.”
The research highlights the potential of repurposing present FDA-approved FGFR inhibitors, which have proven early indicators of success in a single affected person handled below compassionate use.
“This is the first time we are able to look at this highly lethal tumor through this new lens of single cell sequencing,” emphasised Dr. Mariella Filbin, co-director of the Mind Tumor Middle at Dana-Farber/Boston Kids’s Most cancers and Blood Issues Middle.
“It is fascinating how all tumor cells, even though different, help each other to survive and grow. Learning how to disrupt their cooperation, for example by blocking one of their communication lines, will be imperative to treat this cancer in the future.”
The research requires additional medical trials to check FGFR and NOTCH inhibitors in ETMR sufferers and explores antisense therapies focusing on C19MC miRNAs, which have proven promise in lab settings. These findings provide a robust rationale for more practical focused therapies, probably remodeling the therapy panorama for youngsters with ETMR.
Extra info:
Alexander Beck et al, Mobile hierarchies of embryonal tumors with multilayered rosettes are formed by oncogenic microRNAs and receptor–ligand interactions, Nature Most cancers (2025). DOI: 10.1038/s43018-025-00964-9
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FDA-approved FGFR inhibitors present promise towards uncommon and aggressive pediatric mind tumor (2025, Could 26)
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