March 2026 - Lisa Gabler-Pamer

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Dr. Lisa Gabler-Pamer

MedUni Wien RESEARCHER OF THE MONTH March 2026

Embryonal tumors with multilayered rosettes (ETMRs) are among the deadliest cancers, almost exclusively affecting young children and carrying a very poor prognosis. Until now, the cellular mechanisms underlying this extremely rare tumor type were poorly understood, and no targeted therapies were available.

In the present study (Beck, Gabler-Pamer et al.), researchers from Austria, the United States, and the Netherlands analyzed eleven tumor samples using cutting-edge single-cell technologies. They discovered that ETMRs are composed of different tumor cell types arranged in a hierarchical structure, similar to what is seen in a developing brain. These include stem cell–like tumor cells, more differentiated neuron-like cells, and intermediate tumor cells.

The researchers also found that these distinct cell types appear to cooperate: the stem cell–like cells divide rapidly and drive tumor growth, while other cells provide signals that support this growth. The fibroblast growth factor receptor (FGFR) signaling pathway plays a central role in this process. In laboratory experiments and in one patient case, specific inhibitors of the FGFR pathway showed initial effectiveness and slowed tumor growth.

These findings are significant because, for the first time at the single-cell level, they reveal how ETMRs are organized, and which molecular mechanisms drive their growth. This knowledge opens the door to new targeted therapies that could, in the future, improve survival outcomes for children affected by this devastating disease.

Selected Literature

1. A. Beck et al., “Cellular hierarchies of embryonal tumors with multilayered rosettes are shaped by oncogenic microRNAs and receptor–ligand interactions,” Nature Cancer 2025, pp. 1–21, May 2025, doi: 10.1038/s43018-025-00964-9.

2. A.-I. Schiefer et al., “Multicenter Evaluation of a Novel Automated Rapid Detection System of BRAF Status in Formalin-Fixed, Paraffin-Embedded Tissues,” Journal of Molecular Diagnostics, vol. 18, no. 3, 2016, doi: 10.1016/j.jmoldx.2015.12.005

3. L. Gabler et al., “TERT expression is susceptible to BRAF and ETS-factor inhibition in BRAF V600E /TERT promoter double-mutated glioma,” Acta Neuropathol Commun, vol. 7, no. 1, p. 128, 2019, doi: 10.1186/s40478-019-0775-6

4. L. Gabler et al., “Fibroblast growth factor receptor 4 promotes glioblastoma progression: a central role of integrin-mediated cell invasiveness,” Acta Neuropathol Commun, vol. 10, no. 1, pp. 1–16, Dec. 2022, doi: 10.1186/S40478-022-01363-2/FIGURES/6

5. D. Lötsch et al., “Targeting fibroblast growth factor receptors to combat aggressive ependymoma,” Acta Neuropathol, vol. 1, p. 3, May 2021, doi: 10.1007/s00401-021-02327-x