ArticleCancer biology & therapy2016
Complete and sustained response of adult medulloblastoma to first-line sonic hedgehog inhibition with vismodegib.
Article in Cancer biology & therapy, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
19 citing papers in PubMed.
- A 10-year-long ongoing complete response to vismodegib as first-line treatment in adult metastatic medulloblastoma.Acta oncologica (Stockholm, Sweden) · 2026Article
- Real-world management and long-term outcomes in adolescent, young adult, and adult medulloblastoma: Experience from a monocentric series with multimodal and targeted approaches.Neuro-oncology practice · 2026Article
- Outcomes of craniospinal radiotherapy in adult medulloblastoma: survival, long-term toxicities, and quality of life assessment with SF-12 and t-Cog.Strahlentherapie und Onkologie : Organ der Deutschen Rontgengesellschaft ... [et al] · 2026Article
- Cardiotoxicity prevention in thoracic radiotherapy: The effect of different melatonin doses on the level of oxidation markers -in vivo animal study.Toxicology reports · 2025Article
- Pediatric-Like Brain Tumors in Adults.Advances and technical standards in neurosurgery · 2024Review
- Review
- Review
- Review
- Molecular testing for adolescent and young adult central nervous system tumors: A Canadian guideline.Frontiers in oncology · 2022Review
- The multifaceted NF-kB: are there still prospects of its inhibition for clinical intervention in pediatric central nervous system tumors?Cellular and molecular life sciences : CMLS · 2021Review
- Identification of a potent antagonist of smoothened in hedgehog signaling.Cell & bioscience · 2021Article
- Review
- Circulating tumour DNA from the cerebrospinal fluid allows the characterisation and monitoring of medulloblastoma.Nature communications · 2020Article
- miR-196B-5P and miR-200B-3P Are Differentially Expressed in Medulloblastomas of Adults and Children.Diagnostics (Basel, Switzerland) · 2020Article
- Vismodegib as First-Line Treatment of Mutated Sonic Hedgehog Pathway in Adult Medulloblastoma.JCO precision oncology · 2020Article
- Updates on Management of Adult Medulloblastoma.Current treatment options in oncology · 2019Review
- Transforming Biomarker Development with Exceptional Responders.Trends in cancer · 2018Review
- Long-term outcomes of adult medulloblastoma patients treated with radiotherapy.Journal of neuro-oncology · 2018Article
- Differential response of SHH-expressing adult medulloblastomas to the sonic hedgehog inhibitor vismodegib: whole-genome analysis.Cancer biology & therapyArticle
Corrections and comments
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Authors and funding
6 authors.
Funding
Abstract
Medulloblastoma is an aggressive primitive neuroectodermal tumor of the cerebellum that is rare in adults. Medulloblastomas fall into 4 prognostically significant molecular subgroups that are best defined by experimental gene expression profiles: the WNT pathway, sonic hedgehog (SHH) pathway, and subgroups 3 and 4 (non-SHH/WNT). Medulloblastoma of adults belong primarily to the SHH category. Vismodegib, an SHH-pathway inhibitor FDA-approved in 2012 for treatment of basal cell carcinoma, has been used successfully in the setting of chemorefractory medulloblastoma, but not as a first-line therapy. In this report, we describe a sustained response of an unresectable multifocal form of adult medulloblastoma to vismodegib. Molecular analysis in this case revealed mutations in TP53 and a cytogenetic abnormality, i17q, that is prevalent and most often associated with subgroup 4 rather than the SHH-activated form of medulloblastoma. Our findings indicate that vismodegib may also block alternate, non-canonical forms of downstream SHH pathway activation. These findings provide strong impetus for further investigation of vismodegib in clinical trials in the first-line setting for pediatric and adult forms of medulloblastoma.
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