A targeted therapy already used to treat advanced skin cancer is also
effective against the most common subtype of the brain tumor
medulloblastoma in adults and should be considered for treatment of
newly diagnosed patients, according to research led by St. Jude
Children's Research Hospital.
The drug, called Vismodegib, is designed to block a key protein in
the sonic hedgehog (SHH) signaling pathway. The pathway is normally
active during fetal development and is inappropriately switched on in
about 30 percent of medulloblastoma tumors, including about 60 percent
of tumors in adults and 25 percent of tumors in children. Only patients with the SHH subtype responded to vismodegib; however,
researchers also reported that the drug was not universally effective
against all tumors in the subtype."While it was disappointing that not all medulloblastoma patients with
the SHH subtype will benefit, for the right patients these results mark
the beginning of a new era of targeted therapy for treatment of this
tumor,"
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