Article
Mutated FGFR1 is an oncogenic driver and therapeutic target in high-risk neuroblastoma.
The Journal of clinical investigation - 1 Apr 2026
Werr Lisa, Boland Jana, Petersen Josephine, Iglesias Fiorella, Höppner Stefanie, Bartenhagen Christoph, Rosswog Carolina, Hellmann Anna-Maria, Kahlert Yvonne, Hemstedt Nadine, Ibruli Nadliv, Dammert Marcel A, Decarolis Boris, Werner Jan-Michael, Malchers Florian, Schramm Kathrin, Witt Olaf, Beiske Klaus H, Rognlien Anne Gro Wesenberg, Gunnes Maria Winther, Langenberg Karin P, Molenaar Jan, Bernkopf Marie, Taschner-Mandl Sabine, Hughes Debbie, George Sally L, Chesler Louis, Schulte Johannes H, Barone Giuseppe, Capasso Mario, Surrey Lea F, Bagatell Rochelle, Masliah-Planchon Julien, Schleiermacher Gudrun, Grüll Holger, Westermann Frank, Schultheis Anne M, Büttner Reinhard, Henssen Anton G, Eggert Angelika, Peifer Martin, Shukla Neerav N, Simon Thorsten, Hero Barbara, Reinhardt H Christian, Thomas Roman K, Fischer Matthias
Abstract excerpt
Fibroblast growth factor receptor 1 (FGFR1) is recurrently mutated at p.N546 in neuroblastoma. We examined whether mutant FGFR1 is an oncogenic driver, a predictive biomarker, and an actionable vulnerability in this malignancy. FGFR1 mutations at p.N546 were associated with high-risk disease and rapid tumor progression, resulting in dismal outcome for these patients. Ectopic expression of FGFR1N546K induced...
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