Article (Scientific journals)
Molecular profiling and access to treatment for rare cancers in Europe.
Morfouace, M; Hoogstoel, F; Oliveira, J et al.
2026 • In Nature Communications, 17 (1)
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Keywords :
Humans; Europe/epidemiology; Female; Precision Medicine/methods; Mutation; Male; Molecular Targeted Therapy; Middle Aged; Adult; Immunotherapy; Aged; Neoplasms/genetics; Neoplasms/therapy; Rare Diseases/genetics; Rare Diseases/therapy; Europe; Neoplasms; Precision Medicine; Rare Diseases; Chemistry (all); Biochemistry, Genetics and Molecular Biology (all); Multidisciplinary; Physics and Astronomy (all)
Abstract :
[en] Arcagen is a clinical study proposing a comprehensive analysis of molecular and clinical characteristics of rare adult solid cancers (11 domains: sarcomas, gynecology, uro-genital, GI, NET, head and neck, thoracic, skin and eyes, CNS and CUP) across Europe, utilizing the EORTC-SPECTA platform with the EURACAN ERN. This study enrolled 1235 advanced stage patients. 893 were evaluable for molecular profiling toward a potential access to personalized therapy.In the 11 different domains, 69.4% (620/893) of patients had actionable mutations, among whom 93 (15% of 620 patients with currently actionable alterations) received targeted- or immune (IO)-therapies. The median overall survival of the patient population without treatment adaptation, with switch to targeted therapy, or immunotherapy was 1.1, 3.3 years, and not reached, respectively.Across Europe, high prevalence and diversity of molecular alterations for rare solid tumors were observed as well as significant disparities regarding access to personalized medicine approaches.
Disciplines :
Oncology
Author, co-author :
Morfouace, M ;  Institut Gustave Roussy, Direction de la Recherche Clinique, Villejuif, France. Marie.MORFOUACE@gustaveroussy.fr ; European Organization for Research and Treatment of Cancer Headquarters, Brussels, Belgium. Marie.MORFOUACE@gustaveroussy.fr
Hoogstoel, F ;  European Organization for Research and Treatment of Cancer Headquarters, Brussels, Belgium
Oliveira, J;  Instituto Portugues de Oncologia do Porto Francisco Gentil, Porto, Portugal
Penel, N ;  Department of Medical Oncology, Centre Oscar-Lambret, University of Lille, Lille, France ; ULR 2694 - Metrics: Evaluation des technologies de santé et des pratiques médicales, CHU of Lille, University of Lille, Lille, France
Tejpar, S ;  UZ Leuven, Leuven, Belgium
Ray-Coquard, I ;  Centre Leon Berard, University Claude Bernard Lyon 1, Lyon, France
Peron, J;  Department of Medical Oncology, Hospices Civils de Lyon, RESHAPE laboratory, Lyon, France
Pracht, M;  Centre Eugene Marquis, Department of Clinical Oncology, Rennes, France
Brasiuniene, B;  National Cancer Center of Vilnius University hospital Santaros clinic, Vilnius, Lithuania ; Faculty of Medicine of Vilnius University, Vilnius, Lithuania
Capela, A ;  Unidade Local de Saúde de Gaia e Espinho, Vila Nova de Gaia, Portugal
Gennigens, Christine  ;  Centre Hospitalier Universitaire de Liège - CHU > > Service d'oncologie médicale
Greillier, L;  Aix-Marseille Univ, APHM, INSERM, CNRS, CRCM, Hôpital Nord, Marseille, France
Even, C;  Head and Neck Department, Gustave Roussy, Villejuif, France
Huang, R;  Foundation Medicine Inc., Boston, Massachusetts, USA
Gorlia, T;  European Organization for Research and Treatment of Cancer Headquarters, Brussels, Belgium
Lombardi, G ;  Department of Oncology, Oncology 1, Veneto Institute of Oncology IOV-IRCCS, Padova, Italy
Gamulin, M ;  University of Zagreb, School of Medicine Department of Clinical Oncology, University Hospital Centre Zagreb, Department of Oncology, Zagreb, Croatia
Licitra, L;  Istituto Tumori, Milan, Italy
Girard, N;  Institut Curie, Paris, France
Klümpen, H J;  University Medical Center Groningen, Department of Medical Oncology, Groningen, The Netherlands
Idbaih, A;  Sorbonne Université, AP-HP, Institut du Cerveau - Paris Brain Institute - ICM, Inserm, CNRS, Hôpitaux Universitaires La Pitié Salpêtrière - Charles Foix, Paris, France
Van der Graaf, Wta;  Department of Medical Oncology, Netherlands Cancer Institute, Amsterdam, The Netherlands ; Department of Medical Oncology, Erasmus MC Cancer Institute, Erasmus Medical Center, Rotterdam, The Netherlands
Blay, J Y ;  Centre Leon Berard, University Claude Bernard Lyon 1, Lyon, France. jean-yves.blay@lyon.unicancer.fr
More authors (13 more) Less
Language :
English
Title :
Molecular profiling and access to treatment for rare cancers in Europe.
Publication date :
23 July 2026
Journal title :
Nature Communications
eISSN :
2041-1723
Publisher :
Nature Research, England
Volume :
17
Issue :
1
Peer reviewed :
Peer Reviewed verified by ORBi
Funding text :
Arcagen is funded by F. Hoffman-La Roche. The EORTC SPECTA platform is supported by Alliance Healthcare. Alliance Healthcare will become Cencora. Fanny Hoogstoel\u2019s work as Fellow at EORTC Headquarters was supported by the EORTC Cancer Research Fund.
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