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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 |
Molecular Profile | Treatment Approach |
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Gene Name | Source | Synonyms | Protein Domains | Gene Description | Gene Role |
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Therapy Name | Drugs | Efficacy Evidence | Clinical Trials |
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Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
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Gene | Variant | Impact | Protein Effect | Variant Description | Associated with drug Resistance |
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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 |
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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 |