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Molecularly targeted therapy
Source: Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 11, 2025 · Captured 2026-09-09.
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Context: Treatment of Recurrent Childhood Medulloblastoma and Other CNS Embryonal Tumors / Treatment Options
With increased knowledge of the molecular and genetic changes associated with different subtypes of medulloblastoma, molecularly targeted therapy, also called precision therapy, is being actively explored in children with recurrent disease.
In patients with recurrent SHH-activated medulloblastomas, the SHH PTCH1 inhibitor vismodegib demonstrated radiographic responses in 3 of 12 pediatric patients. Two of the responses were sustained for less than 2 months, and one response was sustained for more than 6 months. Response was only seen in patients with upstream variants of the SHH pathway, at the level of PTCH1 or SMO.[42] However, because of the development of irreversible growth plate fusions, the use of vismodegib is limited to skeletally mature children.[43]
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