[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"detail-sidebar-cat-0-en-105":3,"doc-seo-356817-105":59,"doc-detail-356817-en":130},{"code":4,"msg":5,"data":6},0,"success",[7,13,18,23,28,33,38,43,48,51,55],{"id":8,"doc_module":4,"doc_module_name":9,"category_name":10,"show_sort_weight":11,"slug":12},1,"Document","Story & Novel",90,"story-novel",{"id":14,"doc_module":4,"doc_module_name":9,"category_name":15,"show_sort_weight":16,"slug":17},2,"Literature",80,"literature",{"id":19,"doc_module":4,"doc_module_name":9,"category_name":20,"show_sort_weight":21,"slug":22},4,"Exam",70,"exam",{"id":24,"doc_module":4,"doc_module_name":9,"category_name":25,"show_sort_weight":26,"slug":27},5,"Comic",60,"comic",{"id":29,"doc_module":4,"doc_module_name":9,"category_name":30,"show_sort_weight":31,"slug":32},6,"Technology",50,"technology",{"id":34,"doc_module":4,"doc_module_name":9,"category_name":35,"show_sort_weight":36,"slug":37},7,"Healthcare",40,"healthcare",{"id":39,"doc_module":4,"doc_module_name":9,"category_name":40,"show_sort_weight":41,"slug":42},8,"Research & Report",30,"research-report",{"id":44,"doc_module":4,"doc_module_name":9,"category_name":45,"show_sort_weight":46,"slug":47},9,"Religion & Spirituality",20,"religion-spirituality",{"id":46,"doc_module":4,"doc_module_name":9,"category_name":49,"show_sort_weight":46,"slug":50},"World Cup","world-cup",{"id":52,"doc_module":4,"doc_module_name":9,"category_name":53,"show_sort_weight":52,"slug":54},10,"Lifestyle","lifestyle",{"id":56,"doc_module":4,"doc_module_name":9,"category_name":57,"show_sort_weight":24,"slug":58},19,"General","general",{"code":4,"msg":60,"data":61},"ok",{"site_id":62,"language":63,"slug":64,"title":65,"keywords":66,"description":67,"schema_data":68,"social_meta":123,"head_meta":125,"extra_data":127,"updated_unix":129},105,"en","paediatric-therapeutic-development-workshop-on-rhabdoid-tumours","Paediatric Therapeutic Development Workshop on rhabdoid tumours","","Rhabdoid tumours (RT) are aggressive pediatric malignancies affecting the central nervous system, kidneys, liver, and soft tissues, with survival rates below 30% in high-risk groups. Standard care relies on surgery plus intensive chemotherapy and radiotherapy, yet results remain poor due to substantial short- and long-term toxicities. A targeted development workshop aimed to enable therapeutic discovery by aligning strategies across intra-cranial and extra-cranial RT biology. Priorities include DDB1–CUL4-associated factor 5 small-molecule binders/degraders, possible superior EZH2 degrader approaches, subgroup value for FGFR/PDGFR inhibition, and MDM2-centered novel development and combination studies to drive early clinical trials.",{"@graph":69,"@context":122},[70,84,105],{"@type":71,"itemListElement":72},"BreadcrumbList",[73,77,79,82],{"item":74,"name":75,"@type":76,"position":8},"https://docshare.wps.com","Home","ListItem",{"item":78,"name":9,"@type":76,"position":14},"https://docshare.wps.com/document/",{"item":80,"name":35,"@type":76,"position":81},"https://docshare.wps.com/document/healthcare/",3,{"item":83,"name":65,"@type":76,"position":19},"https://docshare.wps.com/document/paediatric-therapeutic-development-workshop-on-rhabdoid-tumours/356817/",{"url":83,"name":65,"@type":85,"image":86,"author":91,"headline":65,"publisher":94,"fileFormat":97,"inLanguage":63,"description":67,"dateModified":98,"datePublished":99,"encodingFormat":97,"isAccessibleForFree":100,"interactionStatistic":101},"DigitalDocument",{"url":87,"@type":88,"width":89,"height":90},"https://docshare.wps.com/thumbnails/paediatric-therapeutic-development-workshop-on-rhabdoid-tumours/356817.png","ImageObject",300,407,{"name":92,"@type":93},"Đào","Person",{"url":74,"name":95,"@type":96},"DocShare","Organization","application/pdf","2026-10-28","2026-09-23",true,{"@type":102,"interactionType":103,"userInteractionCount":24},"InteractionCounter",{"@type":104},"ViewAction",{"@type":106,"mainEntity":107},"FAQPage",[108,114,118],{"name":109,"@type":110,"acceptedAnswer":111},"What are rhabdoid tumours (RT) and who do they mainly affect?","Question",{"text":112,"@type":113},"RT are malignancies of the central nervous system, kidneys, liver, and soft tissues, primarily affecting infants and young children.","Answer",{"name":115,"@type":110,"acceptedAnswer":116},"Why is there an unmet need for new therapies in RT?",{"text":117,"@type":113},"Current treatment outcomes are poor and are accompanied by substantial short- and long-term toxicities, highlighting the need for targeted therapies to improve outcomes and reduce harm.",{"name":119,"@type":110,"acceptedAnswer":120},"What therapeutic development priorities are highlighted in the workshop?",{"text":121,"@type":113},"The workshop emphasizes DDB1–CUL4-associated factor 5 as a promising target, prioritizing small-molecule binders/degraders; it also discusses EZH2 degraders, FGFR/PDGFR inhibitors for selected subgroups, and MDM2 as a key priority for novel development and combination trials.","https://schema.org",{"og:url":83,"og:type":124,"og:title":65,"og:site_name":95,"og:description":67},"article",{"robots":126,"canonical":83},"index,follow",{"doc_id":128,"site_id":62},356817,1790449274,{"code":4,"msg":5,"data":131},{"doc_id":128,"user_id":132,"nickname":92,"user_avatar":133,"doc_module":4,"category_id":34,"category_name":35,"doc_title":65,"doc_description":67,"doc_content":134,"file_id":135,"file_url":136,"file_type":137,"file_size":138,"view_count":24,"is_deleted":4,"is_public":8,"is_downloadable":8,"audit_status":8,"page_count":56,"language":139,"language_code":63,"site_id":62,"html_lang":63,"table_of_contents":140,"faqs":141,"seo_title":142,"seo_description":67,"update_tm":143,"read_time":144},1374402968488,"https://ap-avatar.wpscdn.com/davatar_29158cc5080c5b710cf443261637dec0","[www.nature.com/bjc](www.nature.com/bjc British)[ British](www.nature.com/bjc British) Journal of Cancer  \nREVIEW ARTICLE OPEN   \nClinical Studies  \nPaediatric Therapeutic Development Workshop on rhabdoid tumours  \nClaudia Montiel Equihua1,30, Jan J. Molenaar2,30, Itziar Areso1,30, Jaclyn A. Biegel3,4, Patricia Blanc5, Susan N. Chi6, Sam Daems7,8, Laura Danielson9, Jarno Drost2,10, Niels E. Franke2, Michael C. Frühwald 11,12, Amar Gajjar13, James I. Geller14, Annie Huang 15,16, Pascal D. Johann 11,12, Pamela Kearns 17,18, Karsten Nysom 19, Suzanne O’Connor20, Michael V. Ortiz21, Jenny Parker 1, Seema Patel1, Sheena Patel22, Charles WM Roberts23, Daniel Williamson24, Joanna S. Yi25, Andrew DJ Pearson 1,31 ✉, David Jenkinson1,31, Marcel Kool2,26,27,28,31, Franck Bourdeaut29,31 On behalf of LifeArc*, Innovative Therapies for Children with Cancer (ITCC)*, Cancer Research UK* and Cancer Grand Challenge PROTECT team*  \n© The Author(s) 2026  \n\n|  | Rhabdoid tumours (RT) are malignancies of the central nervous system, kidneys, liver and soft tissues that most commonly affect very young children with survival rates below 30% in high-risk cohorts. Treatment entails surgery, intensive chemotherapy and radiotherapy, associated with substantial short-and long-term toxicities. There is an unmet need to develop targeted therapies for RT to improve patient outcomes and mitigate the toxicities of current therapy. Detailed research followed by a workshop had the objective of enabling the development of targeted therapeutics for RT. Given the inherent commonality of their biology (i.e. biallelic inactivation of SMARCB1 or more rarely SMARCA4) the therapeutic approach should be similar for intra-cranial and extra-cranial tumours. DDB1–CUL4-associated factor 5 is a promising target, and the development of small molecule binders/degraders is a priority. Enhancer of zeste 2 polycomb repressive complex 2 subunit (EZH2) degraders may have greater therapeutic potential than inhibitors. Fibroblast growth factor receptor and platelet-derived growth factor receptor inhibitors may have value in subgroups. Mouse double minute 2 homologue (MDM2) is a priority target for novel therapeutic development and combination trials. Combinations of EZH2, MDM2 inhibitors and selective inhibitors of nuclear export should be evaluated robustly preclinically and |  |\n| --- | --- | --- |\n|  |  |  |\n|  |  |  |\n|  |  |  |\n|  |  |  |\n|  |  |  |\n|  |  |  |\n|  |  |  |\n|  |  |  |\n|  |  |  |\n|  |  |  |\n|  |  |  |\n| drive early clinical studies. |  |  |\n|  | British Journal of Cancer (2026) 134:1510–1528; [https://doi.org/10.1038/s41416-026-03348-7](https://doi.org/10.1038/s41416-026-03348-7) |  |\n|  |  |  |\n\nINTRODUCTION  \nRhabdoid tumours (RT) are tumours of the central nervous system (CNS), kidneys, liver and soft tissues primarily affecting infants and young children. Around 200 patients are diagnosed across Europe and the USA each year [1] .  \nSurvival for children with RT remains very poor: the average 5-year overall survival (OS) is ~30–40% [2, 3] . Studies have revealed several molecular and clinical distinct RT subgroups and have enabled the development of risk stratification models [3] . Standard therapy typically comprises maximal safe surgery,  \n1LifeArc, London, UK. 2Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands. 3Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA. 4Department of Pathology and Laboratory Medicine, Children’s Hospital Los Angeles, Los Angeles, CA, USA. 5Imagine for Margo, Saint-Germainen-Laye, Paris, France. 6Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA, USA. 7Waterland Private Equity Investments, Antwerp, Belgium. 8I3h Institute, Université libre de Bruxelles, Brussels, Belgium. 9Cancer Research UK, London, UK. 10Oncode Institute, Utrecht, The Netherlands. 11Pediatrics and Adolescent Medicine, Swabian Children’s Cancer Center, Pediatric Neur","cbCain2ysqrx4783","https://ap.wps.com/l/cbCain2ysqrx4783","pdf",884780,"English","# INTRODUCTION","[{\"question\":\"What are rhabdoid tumours (RT) and who do they mainly affect?\",\"answer\":\"RT are malignancies of the central nervous system, kidneys, liver, and soft tissues, primarily affecting infants and young children.\"},{\"question\":\"Why is there an unmet need for new therapies in RT?\",\"answer\":\"Current treatment outcomes are poor and are accompanied by substantial short- and long-term toxicities, highlighting the need for targeted therapies to improve outcomes and reduce harm.\"},{\"question\":\"What therapeutic development priorities are highlighted in the workshop?\",\"answer\":\"The workshop emphasizes DDB1–CUL4-associated factor 5 as a promising target, prioritizing small-molecule binders/degraders; it also discusses EZH2 degraders, FGFR/PDGFR inhibitors for selected subgroups, and MDM2 as a key priority for novel development and combination trials.\"}]","Paediatric Therapeutic Development Workshop on rhabdoid tumours | PDF",1790127456,48]