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The hedgehog pathway inhibitor vismodegib produced responses in recurrent SHH-subgroup medulloblastoma but not in other subgroups, and only in tumours whose mutation lay upstream of the drug's target, showing that molecular subgrouping must guide its use.
Two phase 2 studies of vismodegib in 43 patients (adults and children) with recurrent medulloblastoma, analysed by molecular subgroup and mutation.
Responses occurred in 3 of 12 adults and 1 of 8 children with SHH-subgroup tumours and in none of the 20 non-SHH patients; responders had PTCH1 or SMO alterations, while tumours with downstream SUFU or GLI2 alterations did not respond, and growth plate fusion occurred in children.
Vismodegib or sonidegib is reserved for skeletally mature patients with relapsed SHH medulloblastoma and upstream pathway mutations, a niche defined by this trial.
Shares SHH-activated medulloblastoma, Journal of Clinical Oncology.
Shares Vismodegib, SHH-activated medulloblastoma.
Shares Vismodegib, SHH-activated medulloblastoma.
Shares Vismodegib, SHH-activated medulloblastoma.
Shares Vismodegib, SHH-activated medulloblastoma.