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Molecular Profile FGFR1 N546K
Therapy Alpelisib
Indication/Tumor Type diffuse midline glioma, H3 K27M-mutant
Response Type sensitive

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
FGFR1 N546K diffuse midline glioma, H3 K27M-mutant sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, Piqray (alpelisib) inhibited viability of mouse glioma cells expressing H3.3 K28M (reported as H3.3 K27M) and FGFR1 N546K (reported as N457K) in culture (PMID: 37011011). 37011011
PubMed Id Reference Title Details
(37011011) A Compendium of Syngeneic, Transplantable Pediatric High-Grade Glioma Models Reveals Subtype-Specific Therapeutic Vulnerabilities. Full reference...