Brain Tumors (Medulloblastoma)

    Actionable Target Abnormality Prevalence (%) Clinical Experience with Targeted Agent
    PTCH1 Mutation (hedgehog pathway activation)   7 Vismodegib (SMO/hedgehog inhibitor):
    • Phase 1, pediatric pts: ORR 33% (activated hedgehog pathway), 0% (without activation) (26)
    • Phase 1, advanced disease: 1/1 PR
    Erismodegib (SMO/hedgehog inhibitor):
    • Phase 1, advanced disease: ORR 8% (CR) (27)
    • Phase 1: 1 PR and 1 metabolic PR (activated hedgehog pathway) (28)
    VEGFR Expression 100 Bevacizumab (VEGF inhibitor):
    • Phase 1, with irinotecan and temozolomide, pediatric pts: ORR 67% at 3 mo and 55% at 6 mo, PFS , OS 13 mo (29)
    Other topics in Targets by Organ Site