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Articles by Sam Daems
Paediatric therapeutic development workshop on desmoplastic small round cell tumours
Abstract 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.
Paediatric Therapeutic Development Workshop on rhabdomyosarcoma
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Abstract The third in a series of Paediatric Therapeutic Development Workshops focused on rhabdomyosarcoma. Rhabdomyosarcoma is the most common soft tissue sarcoma in children, with 90% survival for those with the lowest risk disease, but just 20–30% in children with metastatic disease. An urgent unmet need exists to develop targeted therapeutics for high-risk disease and to reduce the toxicity of treatment.
By Joseph Baxter, Claudia Montiel Equihua, Jan J. Molenaar, Jamie M. Aye, Gianni Bisogno, Patricia Blanc, Willemijn B. Breunis, Julia C. Chisholm, Jacquelyn Crane, Sam Daems, Laura Danielson, Bram de Wilde, Adam D. Durbin Verified, Felipe Galvez-Cancino, Patrizia Gasparini, Birgit Geoerger, Simone Graham, Mark E. Hatley, Delphine Heenen, Christine M. Heske, Simone Hettmer, Peter Houghton, Pamela R Kearns, Charles Keller, Javed S. Khan, David M. Langenau, Geffen Lass, Leo Mascarenhas, Michael Meister, Jonathan Metts, Veronique Minard-Colin, Sapna Oberoi, Daniela Palacios, Abbe Pannucci, Seema Patel, Sheena M Patel, Silvia Pomella, Terry Rabbitts, Rossella Rota, Erin R Rudzinski, Beat W. Schäfer, Karin Straathof, Rajkumar Venkatramani, Marco Wachtel, Sara Wakeling, Zoe Walters, Sam Warburton, Brenda J. Weigel, Andrew Pearson, David Jenkinson Verified, Janet Shipley, Michela Casanova, Innovative Therapies, Bass Hassan, Elizabeth Hookham, Fiona Kinnis, Corinne Mary Linardic, John Jack Shern
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Nature
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Paediatric Therapeutic Development Workshop on rhabdoid tumours - British Journal of Cancer
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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.
By Jan J. Molenaar, Jaclyn A. Biegel, Patricia Blanc, Susan N. Chi, Sam Daems, Laura Danielson, Jarno Drost, Michael C. Frühwald, Amar Gajjar, James Geller, annie huang, Pascal D. Johann, Pamela R Kearns, Karsten Nysom, Suzanne O’Connor, Michael Ortiz, Jenny Parker, Seema Patel, Sheena M Patel, Charles Roberts, Daniel Williamson, Joanna S. Yi, Andrew Pearson, David Jenkinson Verified, Marcel Kool, Franck Bourdeaut, Innovative Therapies, Claudia Montiel Equihua, Itziar Areso, Niels E. Franke
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Nature
Verified
New models for the development of and access to CAR T-cell therapies for children and adolescents with cancer: an ACCELERATE multistakeholder analysis
Get full text access Log in, subscribe or purchase for full access. References 1. Labanieh, L ∙ Mackall, CL CAR immune cells: design principles, resistance and the next generation Nature. 2023; 614:635-648 2. European Medicines Agency Kymriah 3. US Food and Drug Administration FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine release syndrome 4. Del Bufalo, F ∙ De Angelis, B ∙ Caruana, I ∙ et al. GD2-CART01 for relapsed or refractory high-risk neuroblastoma N Engl J Med.
New models for the development of and access to CAR T-cell therapies for children and adolescents with cancer: an ACCELERATE multistakeholder analysis
Introduction Chimeric antigen receptor (CAR) T-cell therapy is a rapidly developing field that is often driven by academia.1 Accordingly, there has been a major increase in the number of CAR T-cell products in preclinical and clinical testing,1 with several authorised products available that are primarily oriented to adult diseases, with a single paediatric exception.2,3 In addition to established efficacy in paediatric B-cell precursor acute lymphocytic leukaemia, CAR T cells have shown...
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