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Two new studies provide profound insights into the biology and development of childhood brain tumors

Two recent publications in pediatric neuro-oncology, produced in close collaboration with the Comprehensive Cancer Center Vienna at MedUni Vienna and Vienna General Hospital (AKH), expand our understanding of key mechanisms underlying the development and organization of rare, aggressive brain tumors in children and adolescents. The focus is on fundamental developmental processes—with potential implications for future therapeutic approaches.

The first study, co-first authored by MedUni Vienna, investigates pineoblastoma, a rare tumor of the pineal gland. Researchers investigated the development of pineoblastoma and discovered a shared molecular dependency that also occurs in other types of brain tumors, such as medulloblastoma and retinoblastoma. In the process, a high-resolution single-cell atlas of normal pineal gland development was created, and tumor samples from 38 patients were analyzed. It was found that pineoblastomas arise from early precursor cells and are highly dependent on light-sensitive genes. These genes proved to be essential for survival in several tumor types, making them a potential common target for future therapies. This study was recently published in the journal Cancer Cell and announced as a “Preview,” as well as highlighted as a “Research Highlight” in the journal Nature Reviews Cancer.

The second paper, co-authored by Meduni Vienna and published in the journal Nature, focuses on supratentorial ependymoma, another highly aggressive group of tumors in children and adolescents, and demonstrates that these tumors consist of distinct developmental-like cell states. Depending on the molecular subgroup, they differ significantly in their composition, maturation, and spatial organization. Using single-cell and spatial transcriptomics as well as live-cell imaging, the researchers identified two key precursor-like cell states reminiscent of early human brain development. One is particularly migratory. The other exhibits particularly vigorous and uncontrolled cell division. At the same time, influences from the brain environment can further shift the tumor cells into neuron-like states. Overall, the study provides a comprehensive picture of tumor heterogeneity and demonstrates how developmental programs, the microenvironment, and cell behavior collectively contribute to the aggressiveness of these brain tumors. 

Co-(first) authors Bernhard Englinger and Johannes Gojo from CCC Vienna summarize: “The two studies show that pediatric brain tumors in many cases represent misdirected developmental processes. A deeper understanding of these processes opens new ways for identifying common vulnerabilities across different tumor types and exploiting them therapeutically.”

Publication: Cancer Cell 
A tumor-associated photoreceptor signature unifies distinct central nervous system malignancies
Autoren: Gudenas BL*, Ahmad ST*, Englinger B*, Liu APY*, Zhao M*, Paul L, Hadley J, Li Y, Batts M, Mittal P, Wu SC, Lewis SA, Han K, Soliman T, Lin H, Janke L, Meredith D, Pfaff E, Gojo J, Cotter J, Klimo P Jr, Boop FA, Gajjar A, Robinson GW, Rosén G, Alexandrescu S, Jones DTW, Orr BA, Swartling FJ, Filbin MG, Northcott PA. https://www.sciencedirect.com/science/article/pii/S153561082600108X 
DOI https://doi.org/10.1016/j.ccell.2026.02.010

Publication: Nature 

Multidimensional profiling of heterogeneity in supratentorial ependymomas.

Authors: Jeong D, Danielli SG, Maaß KK, Ghasemi DR, Tetzlaff SK, Reyhan E, Jiang L, Katiyar S, Sundheimer JK, Lo Cascio C, Neyazi S, de Biagi-Junior CAO, Couvillon E, Castellani S, Pazyra-Murphy M, Mullally M, Dehler MP, Englinger B, Nascimento A, Cruzeiro GAV, Marques JG, Haase RD, Nguyen CM, Baumgartner AC, Rozowsky JS, Hack OA, Shaw ML, Lotsch-Gojo D, Bruckner K, Korshunov A, Pfister SM, Kool M, Nowakowski TJ, Gojo J, Baird L, Alexandrescu S, Pajtler KW, Venkataramani V, Filbin MG.https://www.nature.com/articles/s41586-026-10214-2
DOI https://doi.org/10.1038/s41586-026-10214-2