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Ref Type
PMID (34250399)
Authors Farouk Sait S, Gilheeney SW, Bale TA, Haque S, Dinkin MJ, Vitolano S, Rosenblum MK, Ibanez K, Prince DE, Spatz KH, Dunkel IJ, Karajannis MA
Title Debio1347, an Oral FGFR Inhibitor: Results From a Single-Center Study in Pediatric Patients With Recurrent or Refractory FGFR-Altered Gliomas.
Journal JCO precision oncology
Vol 5
Issue
Date 2021
URL
Abstract Text

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
FGFR1 E765G missense unknown FGFR1 E765G lies within the protein kinase domain of the Fgfr1 protein (UniProt.org). E765G has been identified in the scientific literature (PMID: 34250399), but has not been biochemically characterized and therefore, its effect on Fgfr1 protein function is unknown (PubMed, Jan 2022).
FGFR1 V592M missense unknown FGFR1 V592M (corresponds to V561M in the canonical isoform) lies within the protein kinase domain of the Fgfr1 protein (UniProt.org). V592M has been identified in the scientific literature (PMID: 34250399), but has not been biochemically characterized and therefore, its effect on Fgfr1 protein function is unknown (PubMed, Jan 2022).
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
FGFR1 - TACC1 pilomyxoid astrocytoma predicted - sensitive Debio 1347 Case Reports/Case Series Actionable In a clinical case study, Debio 1347 treatment resulted in a partial response with a 74.5% maximal tumor reduction that was sustained for 9 months in a pediatric patient with optic pathway pilomyxoid astrocytoma harboring FGFR1-TACC1, who also harbored FGFR1 E765G (PMID: 34250399). 34250399
FGFR1 V592M FGFR1 K687E pilomyxoid astrocytoma predicted - sensitive Debio 1347 Case Reports/Case Series Actionable In a clinical case study, Debio 1347 treatment resulted in a significant clinical improvement and sustained stable disease at 14 months with a 12.2% maximal tumor reduction in a pediatric patient with optic pathway pilomyxoid astrocytoma harboring FGFR1 V592M and FGFR1 K687E (PMID: 34250399). 34250399
FGFR1 V592M FGFR1 K687E glioblastoma predicted - sensitive Debio 1347 Case Reports/Case Series Actionable In a clinical case study, Debio 1347 treatment resulted in a near complete response with a 96.3% maximal tumor reduction after 2 months of therapy in a pediatric patient with spinal cord glioblastoma harboring FGFR1 V592M and FGFR1 K687E, and the response was maintained for 11 months (PMID: 34250399). 34250399
FGFR3 - TACC3 central nervous system cancer predicted - sensitive Debio 1347 Case Reports/Case Series Actionable In a clinical case study, Debio 1347 treatment resulted in a prolonged stable disease and stable clinical symptoms at 26 months in a pediatric patient with a right cerebellar hemispheric rosette-forming glioneuronal tumor harboring an FGFR3-TACC3 fusion (PMID: 34250399). 34250399