Table 3

Potentially targetable genetic alterations identified

Genetic AlterationTargeted AgentTumor Type# of Patients
PIK3CA submTOR inhibitorInfiltrative astrocytoma3
hypermutationPD-1 inhibitorGlioblastoma3
PDGFRA amp or subdasatinibGlioblastoma3
KIAA1549-BRAF fusionMEK inhibitorPilocytic astrocytoma2
PTCH1 inactivationSMO inhibitorMedulloblastoma, nodular/desmoplastic2
FGFR1 sub or kinase domain dupkinase inhibitorLow-grade glioma2
H3F3A p.K27MpanobinostatDiffuse midline glioma2
AKT3 ampmTOR inhibitorGlioblastoma1
MAP2K1 exon 2 small in-frame delMEK inhibitorLow-grade astrocytic neoplasm1
ATG7-RAF1 fusionMEK inhibitorPleomorphic xanthoastrocytoma1
CDK4 amppalbociclibGlioblastoma1
SMARCB1 delEZH2 inhibitorChordoma1
PALB2 inactivationPARP inhibitorMedulloblastoma1
Genetic AlterationTargeted AgentTumor Type# of Patients
PIK3CA submTOR inhibitorInfiltrative astrocytoma3
hypermutationPD-1 inhibitorGlioblastoma3
PDGFRA amp or subdasatinibGlioblastoma3
KIAA1549-BRAF fusionMEK inhibitorPilocytic astrocytoma2
PTCH1 inactivationSMO inhibitorMedulloblastoma, nodular/desmoplastic2
FGFR1 sub or kinase domain dupkinase inhibitorLow-grade glioma2
H3F3A p.K27MpanobinostatDiffuse midline glioma2
AKT3 ampmTOR inhibitorGlioblastoma1
MAP2K1 exon 2 small in-frame delMEK inhibitorLow-grade astrocytic neoplasm1
ATG7-RAF1 fusionMEK inhibitorPleomorphic xanthoastrocytoma1
CDK4 amppalbociclibGlioblastoma1
SMARCB1 delEZH2 inhibitorChordoma1
PALB2 inactivationPARP inhibitorMedulloblastoma1

Abbreviations: sub, missense mutation; amp, amplification; dup, duplication; del, deletion; EZH2, enhancer of zeste homolog 2.

Table 3

Potentially targetable genetic alterations identified

Genetic AlterationTargeted AgentTumor Type# of Patients
PIK3CA submTOR inhibitorInfiltrative astrocytoma3
hypermutationPD-1 inhibitorGlioblastoma3
PDGFRA amp or subdasatinibGlioblastoma3
KIAA1549-BRAF fusionMEK inhibitorPilocytic astrocytoma2
PTCH1 inactivationSMO inhibitorMedulloblastoma, nodular/desmoplastic2
FGFR1 sub or kinase domain dupkinase inhibitorLow-grade glioma2
H3F3A p.K27MpanobinostatDiffuse midline glioma2
AKT3 ampmTOR inhibitorGlioblastoma1
MAP2K1 exon 2 small in-frame delMEK inhibitorLow-grade astrocytic neoplasm1
ATG7-RAF1 fusionMEK inhibitorPleomorphic xanthoastrocytoma1
CDK4 amppalbociclibGlioblastoma1
SMARCB1 delEZH2 inhibitorChordoma1
PALB2 inactivationPARP inhibitorMedulloblastoma1
Genetic AlterationTargeted AgentTumor Type# of Patients
PIK3CA submTOR inhibitorInfiltrative astrocytoma3
hypermutationPD-1 inhibitorGlioblastoma3
PDGFRA amp or subdasatinibGlioblastoma3
KIAA1549-BRAF fusionMEK inhibitorPilocytic astrocytoma2
PTCH1 inactivationSMO inhibitorMedulloblastoma, nodular/desmoplastic2
FGFR1 sub or kinase domain dupkinase inhibitorLow-grade glioma2
H3F3A p.K27MpanobinostatDiffuse midline glioma2
AKT3 ampmTOR inhibitorGlioblastoma1
MAP2K1 exon 2 small in-frame delMEK inhibitorLow-grade astrocytic neoplasm1
ATG7-RAF1 fusionMEK inhibitorPleomorphic xanthoastrocytoma1
CDK4 amppalbociclibGlioblastoma1
SMARCB1 delEZH2 inhibitorChordoma1
PALB2 inactivationPARP inhibitorMedulloblastoma1

Abbreviations: sub, missense mutation; amp, amplification; dup, duplication; del, deletion; EZH2, enhancer of zeste homolog 2.

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