{"doi":"10.1080/15384047.2016.1220453","title":"Complete and sustained response of adult medulloblastoma to first-line sonic hedgehog inhibition with vismodegib","abstract":null,"journal":"Cancer Biology & Therapy","year":2016,"id":619924,"datarank":0.47032413238937254,"base_score":3.1354942159291497,"endowment":3.1354942159291497,"self_citation_contribution":0.47032413238937254,"citation_network_contribution":0.0,"self_endowment_contribution":0.47032413238937254,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":22,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":null,"is_data_producer":false,"deposit_databanks":null,"is_oa":false,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":null,"fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":1299714,"name":"Matthew Schomaker","orcid":"0009-0007-0789-0754","position":1,"is_corresponding":false},{"id":1427074,"name":"Jon D. Wilson","orcid":null,"position":2,"is_corresponding":false},{"id":1600027,"name":"Mary Ahrens","orcid":null,"position":3,"is_corresponding":false},{"id":293777,"name":"Michelle Dolan","orcid":null,"position":4,"is_corresponding":false},{"id":235000,"name":"Andrew C. Nelson","orcid":"0000-0002-4741-0010","position":5,"is_corresponding":false},{"id":259423,"name":"Emil Lou","orcid":"0000-0002-1607-1386","position":0,"is_corresponding":false}],"reference_count":0,"raw_metadata":{"has_enrichment":true,"resolved":true,"title":"Complete and sustained response of adult medulloblastoma to first-line sonic hedgehog inhibition with vismodegib","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.","is_dataset_classified":null,"base_score":0.0,"endowment":0.0,"datacite_reuse_total":0,"file_count":0,"downloads":0,"views":0,"has_version_chain":false,"is_dataset":false,"is_oa":false,"pmid":"27682250","pmcid":"PMC5079386","openalex_id":"https://openalex.org/W27682250","authors":[],"funders":[{"funder_name":"National Center for Advancing Transl","grant_id":"UL1TR000114","title":null},{"funder_name":"NCATS NIH HHS","grant_id":"KL2 TR000113","title":null},{"funder_name":"Minnesota Masonic Charities/Masonic Cancer Center","grant_id":"","title":null},{"funder_name":"Litman Family Fund for Cancer Research","grant_id":"","title":null},{"funder_name":"Randy Shaver Cancer Research and Community Fund","grant_id":"","title":null}],"total_grants":5,"fwci":0.0,"citation_percentile":0.00382924,"influential_citations":0,"citation_trend":[],"oa_status":"bronze","license":null,"oa_locations":[{"url":"https://www.tandfonline.com/doi/pdf/10.1080/15384047.2016.1220453?needAccess=true","host_type":"publisher"},{"url":"http://www.tandfonline.com/doi/pdf/10.1080/15384047.2016.1220453","host_type":"publisher"},{"url":"https://meetingorganizer.copernicus.org/EGU2010/EGU2010-10311.pdf","host_type":"conference"}],"fields_of_study":["Cryospheric studies and observations","Meteorological Phenomena and Simulations","Hydrology and Watershed Management Studies"],"mesh_terms":[],"keywords":["Snow","Arctic","Norwegian","Geography","Research center","Physical geography","Space research","Climatology","Meteorology","Engineering","Political science","Geology","Oceanography","Medulloblastoma","Sonic hedgehog","Targeted Therapy","Vismodegib"],"sdg_mappings":[{"sdg_number":0,"sdg_label":"Climate action"}],"linked_datasets":[],"clinical_trials":[],"software_tools":[],"database_accessions":[],"source":"live","citation_network_status":"fetched"},"created_at":"2026-08-03T09:13:28.475822Z","pmid":null,"pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}