Vismodegib as First-Line Treatment of Mutated Sonic Hedgehog Pathway in Adult Medulloblastoma

Kian, Waleed; Roisman, Laila C.; Goldstein, Iris M.; Abo-Quider, Abed; Samueli, Benzion; Wallach, Nadav; Alguayn, Farouq; Shalata, Walid; Levitas, Dina; Belochitski, Olga; Levin, Daniel; Fenig, Eyal; Lavrenkov, Konstantin; Tokar, Margarita; Peled, Nir; Yakobson, Alexander

doi:10.1200/po.19.00264

Cited by 6 publications

(2 citation statements)

References 13 publications

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“…Research efforts are looking to find targeted therapies and immunotherapeutic options for medulloblastoma. Vismodegib is a small molecule inhibitor of the SHH pathway which has shown some efficacy against SHH-activated medulloblastoma in adults [87]. An ongoing clinical trial at St. Jude is investing this in combination with adjuvant chemotherapy for SHH-activated medulloblastoma in children (NCT01878617).…”

Section: Treatmentmentioning

confidence: 99%

Pediatric Central Nervous System Tumor Overview and Emerging Treatment Considerations

Damodharan

Puccetti

2023

Brain Sciences

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Pediatric central nervous system (CNS) tumors are the most common solid tumor in children, with the majority being glial in origin. These tumors are classified by the World Health Organization (WHO) as either being low grade (WHO grade 1 and 2) or high grade (WHO grade 3 and 4). Our knowledge of the molecular landscape of pediatric brain tumors has advanced over the last decade, which has led to newer categorizations along with an expansion of therapeutic targets and options. In this review, we will give an overview of common CNS tumors seen in children along with a focus on treatment options and future considerations.

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Section: Treatmentmentioning

confidence: 99%

Pediatric Central Nervous System Tumor Overview and Emerging Treatment Considerations

Damodharan

Puccetti

2023

Brain Sciences

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“…Kian et al have reported the case of an adult 24-year-old woman affected by desmoplastic MB, SHH-mutated and TP53 wild-type, harboring a non-sense mutation in position R135 of the PTCH1 gene, which achieved a remarkable and prolonged radiological response to the SMO antagonist vismodegib [ 41 ]. At the time of diagnosis, the MRI showed a wide right cerebellar and vermis lesion with multiple intramedullary metastases causing spinal cord compression.…”

Section: Treatment Of Shh Subgroupmentioning

confidence: 99%

Molecular Targeted Therapies: Time for a Paradigm Shift in Medulloblastoma Treatment?

Gatto¹,

Franceschi

Tosoni

et al. 2022

Cancers

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Medulloblastoma is a rare malignancy of the posterior cranial fossa. Although until now considered a single disease, according to the current WHO classification, it is a heterogeneous tumor that comprises multiple molecularly defined subgroups, with distinct gene expression profiles, pathogenetic driver alterations, clinical behaviors and age at onset. Adult medulloblastoma, in particular, is considered a rarer “orphan” entity in neuro-oncology practice because while treatments have progressively evolved for the pediatric population, no practice-changing prospective, randomized clinical trials have been performed in adults. In this scenario, the toughest challenge is to transfer the advances in cancer genomics into new molecularly targeted therapeutics, to improve the prognosis of this neoplasm and the treatment-related toxicities. Herein, we focus on the recent advances in targeted therapy of medulloblastoma based on the new and deeper knowledge of disease biology.

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Pediatric-Like Brain Tumors in Adults

Dias,

Richards,

Elliot

et al. 2024

Advances and Technical Standards in Neurosurgery

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Vismodegib as First-Line Treatment of Mutated Sonic Hedgehog Pathway in Adult Medulloblastoma

Cited by 6 publications

References 13 publications

Pediatric Central Nervous System Tumor Overview and Emerging Treatment Considerations

Pediatric Central Nervous System Tumor Overview and Emerging Treatment Considerations

Molecular Targeted Therapies: Time for a Paradigm Shift in Medulloblastoma Treatment?

Pediatric-Like Brain Tumors in Adults

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