Cornelis M van Tilburg, Lindsay B Kilburn, Sébastien Perreault, Rene Schmidt, Amedeo A Azizi, Ofelia Cruz-Martínez, Michal Zápotocký, Katrin Scheinemann, Antoinette Y N Schouten-van Meeteren, Astrid Sehested, Enrico Opocher, Pablo Hernáiz Driever, Shivaram Avula, David S Ziegler, David Capper, Arend Koch, Felix Sahm, Jiaheng Qiu, Li-Pen Tsao, Samuel C Blackman, Peter Manley, Till Milde, Ruth Witt, David T W Jones, Darren Hargrave, Olaf Witt
BACKGROUND: Pediatric low-grade glioma (pLGG) is essentially a single pathway disease, with most tumors driven by genomic alterations affecting the mitogen-activated protein kinase/ERK (MAPK) pathway, predominantly KIAA1549::BRAF fusions and BRAF V600E mutations. This makes pLGG an ideal candidate for MAPK pathway-targeted treatments. The type I BRAF inhibitor, dabrafenib, in combination with the MEK inhibitor, trametinib, has been approved by the United States Food and Drug Administration for the systemic treatment of BRAF V600E-mutated pLGG...
January 30, 2024: BMC Cancer