Brain Tumors (Other NF-Related Tumors)

    Actionable Target Abnormality Prevalence (%) Clinical Experience with Targeted Agent
    NF1/NF2 Loss 100 Everolimus (mTOR inhibitor):
    • Phase 2: RR 0% (20)
    EGFR Overexpression  40 Lapatinib (EGFR/HER2 inhibitor):
    • Phase 2, NF2: ORR 24% (time to response: 4.5 mo), PFS at 12 mo 64% (21)
    Erlotinib (EGFR inhibitor):
    • Phase 2, NF1: SD 5% (22)
    VEGFR Expression 100 Bevacizumab (VEGF inhibitor):
    • Retrospective review, NF2: ORR 55% (23)
    • Case study: 2 pts treated with single-agent SD of 9+ and 10+ mo, 2 pts treated with combination with temsirolimus (mTOR inhibitor) 9+ mo (with 33% volumetric reduction) (24)
    Other topics in Targets by Organ Site