Journal Article (Review Article)

DKFZ-2026-00357

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Paediatric Therapeutic Development Workshop on rhabdoid tumours.

Montiel Equihua, C. ; Molenaar, J. J. ; Areso, I. ; Biegel, J. A. ; Blanc, P. ; Chi, S. N. ; Daems, S. ; Danielson, L. ; Drost, J. ; Franke, N. E. ; Frühwald, M. C. ; Gajjar, A. ; Geller, J. I. ; Huang, A. ; Johann, P. D. ; Kearns, P. ; Nysom, K. ; O'Connor, S. ; Ortiz, M. V. ; Parker, J. ; Patel, S. ; Patel, S. ; Roberts, C. W. ; Williamson, D. ; Yi, J. S. ; Pearson, A. D. ; Jenkinson, D. ; Kool, M.DKFZ* ; Bourdeaut, F. ; LifeArc (Collaboration Author) ; Cancer, I. T. f. C. w. (Collaboration Author) ; UK, C. R. (Collaboration Author) ; team, C. G. C. P. (Collaboration Author)

2026
Nature Publ. Group Edinburgh

Abstract: 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 drive early clinical studies.

Note: #DKTKZFB26# / epub

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Contributing Institute(s):

1. B062 Pädiatrische Neuroonkologie (B062)
2. DKTK HD zentral (HD01)

Research Program(s):

1. 312 - Funktionelle und strukturelle Genomforschung (POF4-312) (POF4-312)

Appears in the scientific report 2026

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Database coverage:
; BIOSIS Previews ; Biological Abstracts ; Clarivate Analytics Master Journal List ; Current Contents - Clinical Medicine ; Current Contents - Life Sciences ; DEAL Springer ; Ebsco Academic Search ; Essential Science Indicators ; IF >= 5 ; JCR ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

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