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SHH inhibitors for the treatment of medulloblastoma
Journal article   Peer reviewed

SHH inhibitors for the treatment of medulloblastoma

Ayman Samkari, Jason White and Roger Packer
Expert review of neurotherapeutics, v 15(7), pp 763-770
01 Jul 2015
PMID: 26027634

Abstract

Clinical Neurology Life Sciences & Biomedicine Neurosciences & Neurology Pharmacology & Pharmacy Science & Technology
Medulloblastoma is the most common malignant brain tumor of childhood. It is currently stratified into four molecular variants through the advances in transcriptional profiling. They include: wingless, sonic hedgehog (SHH), Group III, and Group IV. The SHH group is characterized by constitutive activation of the SHH signaling pathway, and genetically characterized by mutations in patched homolog 1 (PTCH1) or other downstream pathway mutations. SHH inhibitors have become of great clinical interest in treating SHH-driven medulloblastoma. Many inhibitors are currently in different stages of development, some already approved for other SHH-driven cancers, such as basal cell carcinoma. In vitro and in vivo medulloblastoma studies have shown efficacy and these findings have been translated into Phase I and II clinical trials. In this review, we present an overview of SHH medulloblastoma, as well as a discussion of currently available SHH inhibitors, and the challenges associated with their use.

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Collaboration types
Domestic collaboration
Web of Science research areas
Clinical Neurology
Pharmacology & Pharmacy
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