Paediatric therapeutic development workshop on desmoplastic small round cell tumours
The sixth Paediatric Therapeutic Development Workshop focused on desmoplastic small round cell tumours (DSRCT). Less than 20% of patients with DSRCT survive beyond 5 years and there is no targeted therapy for this disease, which represents an urgent unmet need for new therapeutics. High priority targets include the EWSR1::WT1 fusion protein, the oncogenic driver, and targeting a junction-derived neopeptide presented by HLA-A*03 and HLA-A*11 using a T-cell receptor (TCR) is a novel approach. Other targets (LRRC15, SSTR3 and SSTR5, NECTIN4, STEAP1 and mesothelin) require further validation. Targeting ERBB2/HER2 or claudin-6 by antibody-drug conjugates (ADCs) and NTRK3 by a small molecule inhibitor are promising approaches and are currently being evaluated. Existing clinical assets targeting (in order of importance) B7-H3, the DNA damage response, androgen signalling, FGFR4 and STEAP1 are also high priorities for evaluation in DSRCT, but obtaining therapeutics for clinical trials is a barrier. In view of the rarity of DSRCT, a global initiative in preclinical and clinical research to establish international trials and collect data from patients treated in a harmonised form would benefit patients. Progress can be made by a combined approach of developing new agents whilst evaluating existing therapeutics in well-designed trials.
Authors
- JANET M. SHIPLEY (ORCID: https://orcid.org/0000-0001-6748-8678)
- Emily Kanaya Slotkin (ORCID: https://orcid.org/0000-0002-4463-5759)
- Konstantinos Klaourakis (ORCID: https://orcid.org/0000-0002-2881-6744)
- Patricia Blanc (ORCID: https://orcid.org/0000-0002-7318-8986)
- Joshua J. Waterfall (ORCID: https://orcid.org/0000-0002-3762-5050)
- Sophie C. Postel-Vinay (ORCID: https://orcid.org/0000-0001-5562-1857)
- David S. Moura (ORCID: https://orcid.org/0000-0002-6514-2419)
- Reineke A. Schoot (ORCID: https://orcid.org/0000-0002-8838-981X)
- Marc van de Wetering (ORCID: https://orcid.org/0000-0002-8195-9939)
- Geffen Lass
- Joseph S. Baxter (ORCID: https://orcid.org/0000-0002-2336-614X)
- Danh D. Truong (ORCID: https://orcid.org/0000-0003-3200-1297)
- Rob S. Sellar (ORCID: https://orcid.org/0000-0002-0472-7034)
- Paul H. Huang (ORCID: https://orcid.org/0000-0003-3972-5087)
- Arushi Agrawal (ORCID: https://orcid.org/0000-0002-1642-695X)
- William Tap
- Laura S. Danielson
- Michael T. Meister (ORCID: https://orcid.org/0000-0001-8311-721X)
- Andrew D.J. Pearson (ORCID: https://orcid.org/0000-0002-8738-5913)
- Corinne Mary Linardic (ORCID: https://orcid.org/0000-0002-3257-2885)
- Pamela R. Kearns (ORCID: https://orcid.org/0000-0003-2756-5813)
- Giovana Tardin Torrezan (ORCID: https://orcid.org/0000-0002-8659-5329)
- Daniel H. Orbach (ORCID: https://orcid.org/0000-0002-2520-139X)
- Bernadette Brennan (ORCID: https://orcid.org/0009-0003-2805-8079)
- Véronique Minard‐Colin (ORCID: https://orcid.org/0000-0002-0296-5207)
- Monika Sparber‐Sauer (ORCID: https://orcid.org/0000-0001-9551-2399)
- Joseph Aloysius Ludwig (ORCID: https://orcid.org/0000-0001-6631-8554)
- Leo Mascarenhas (ORCID: https://orcid.org/0000-0001-7790-0777)
- Lucy Métayer (ORCID: https://orcid.org/0000-0001-6682-5847)
- Christopher Austin Klebanoff (ORCID: https://orcid.org/0000-0001-9645-3896)
- Sandro Pasquali (ORCID: https://orcid.org/0000-0003-4815-6293)
- Sam Daems (ORCID: https://orcid.org/0009-0009-5540-5412)
- J Jaap Molenaar
- Siwan Oldham
- David Jenkinson
- Innovative Therapies for Children with Cancer (ITCC)
- On behalf of LifeArc
- Sheena Patel
- Madina Wane
- the Cancer Grand Challenge Protect team
- Silvia Stacchiotti
- Michela Casanova
- Stella Man
- Emma Halliwell
- Nikki Bowdidge
- Marc Ladanyi
- Patrick Grohar
- Janet Vasquez-Campbell
- Richard Bowdidge
- Katerina Lawlor
- Solomon English
- Andrea Ferrari
- Ana Teresa Amaral
- Aaron Weiss
Institutions
- LifeArc (GB)
- Cedars-Sinai Medical Center (US)
- Université Libre de Bruxelles (BE)
- Memorial Sloan Kettering Cancer Center (US)
- The University of Texas MD Anderson Cancer Center (US)
- Institute of Cancer Research (GB)
- Inserm (FR)
- Duke University (US)
- University of Milan (IT)
- Université Paris Sciences et Lettres (FR)
- Université Paris-Saclay (FR)
- Cancer Research UK (GB)
- Institut Gustave Roussy (FR)
- AC Camargo Hospital (BR)
- Smile Train (US)
- MaineHealth (US)
- Michigan Medicine (US)
- Michigan Center for Translational Pathology (US)
- Cancer Research Horizons (GB)
- University Children's Hospital Tübingen (DE)
- Duke Medical Center (US)
- Princess Máxima Center (NL)
- Instituto de Biomedicina de Sevilla (ES)
- Fondazione IRCCS Istituto Nazionale dei Tumori (IT)
- Royal Manchester Children's Hospital (GB)
- Klinikum Stuttgart (DE)
- Fondation Gustave Roussy (FR)
- University College London (GB)
- University of Coimbra (PT)
- University of Birmingham (GB)
- Institut Curie (FR)
- University of Tübingen (DE)
Publication Details
- Journal
- British Journal of Cancer
- Published
- 2026-10-14
- DOI
- https://doi.org/10.1038/s41416-026-03631-7
- Primary Topic
- Sarcoma Diagnosis and Treatment
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- LifeArc
- Instituto de Salud Carlos III