Article
Effective targeting of PDGFRA-altered high-grade glioma with avapritinib.
Cancer cell - 14 Apr 2025
Mayr Lisa, Neyazi Sina, Schwark Kallen, Trissal Maria, Beck Alexander, Labelle Jenna, Eder Sebastian K, Weiler-Wichtl Liesa, Marques Joana G, de Biagi-Junior Carlos A O, Lo Cascio Costanza, Chapman Owen, Sridhar Sunita, Kenkre Rishaan, Dutta Aditi, Wang Shanqing, Wang Jessica, Hack Olivia, Nascimento Andrezza, Nguyen Cuong M, Castellani Sophia, Rozowsky Jacob S, Groves Andrew, Panditharatna Eshini, Cruzeiro Gustavo Alencastro Veiga, Haase Rebecca D, Tabatabai Kuscha, Madlener Sibylle, Wadden Jack, Adam Tiffany, Kong Seongbae, Miclea Madeline, Patel Tirth, Bruckner Katharina, Senfter Daniel, Lämmerer Anna, Supko Jeffrey, Guntner Armin S, Palova Hana, Neradil Jakub, Stepien Natalia, Lötsch-Gojo Daniela, Berger Walter, Leiss Ulrike, Rosenmayr Verena, Dorfer Christian, Dieckmann Karin, Peyrl Andreas, Azizi Amedeo A, Baumgartner Alicia, Slaby Ondrej, Pokorna Petra, Clark Louise M, Cameron Amy, Nguyen Quang-De, Wakimoto Hiroaki, Dubois Frank, Greenwald Noah F, Bandopadhayay Pratiti, Beroukhim Rameen, Ligon Keith, Kramm Christof, Bronsema Annika, Bailey Simon, Stucklin Ana Guerreiro, Mueller Sabine, Skrypek Mary, Martinez Nina, Bowers Daniel C, Jones David T W, Jones Chris, Jäger Natalie, Sterba Jaroslav, Müllauer Leonhard, Haberler Christine, Kumar-Sinha Chandan, Chinnaiyan Arul, Mody Rajen, Chavez Lukas, Furtner Julia, Koschmann Carl, Gojo Johannes, Filbin Mariella G
Abstract excerpt
PDGFRA is crucial to tumorigenesis and frequently genomically altered in high-grade glioma (HGG). In a comprehensive dataset of pediatric HGG (n = 261), we detect PDGFRA mutations and/or amplifications in 15% of cases, suggesting PDGFRA as a therapeutic target. We reveal that the PDGFRA/KIT inhibitor avapritinib shows (1) selectivity for PDGFRA inhibition, (2) distinct patterns of subcellular effects, (3)...
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