Molecular profiling and access to treatment for rare cancers in Europe

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. Molecular characterization of rare cancer types can be challenging, and this can limit application of personalized therapy. Here, the authors compile a large clinical cohort of rare adult solid cancers to find 69% harbour actionable mutations.

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Publication Details

Journal
Nature Communications
Published
2026-07-23
DOI
https://doi.org/10.1038/s41467-026-75776-1
Primary Topic
Sarcoma Diagnosis and Treatment
Type
article
Field-Weighted Citation Impact
0.00

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article

Molecular profiling and access to treatment for rare cancers in Europe

Marija Gamulin, Sabine Tejpar, Marie Morfouace, J Oliveira et al.
Nature Communications
Sarcoma Diagnosis and Treatment
article

Molecular profiling and access to treatment for rare cancers in Europe

Marija Gamulin, Sabine Tejpar, Marie Morfouace, J Oliveira, Jean‐Yves Blay, Nicolas Penel, Giuseppe Lombardi, Andreia Capela, M. Pracht, Isabelle Laure Ray-Coquard, WTA van der Graaf, F. Hoogstoel, C. Gennigens, B. Brasiuniene, T. Gorlia, J. Peron, L. Licitra, H. J. Klümpen, R. Huang, C. Even, A. Idbaih, N. Girard, L. Greillier
article en

Abstract

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. Molecular characterization of rare cancer types can be challenging, and this can limit application of personalized therapy. Here, the authors compile a large clinical cohort of rare adult solid cancers to find 69% harbour actionable mutations.

Nature Communications
Université Claude Bernard Lyon 1 (FR), Centre National de la Recherche Scientifique (FR), University Medical Center Groningen (NL), University Hospital Centre Zagreb (HR), Inserm (FR), University of Liège (BE), Vilnius University (LT), Aix-Marseille Université (FR), Université de Lille (FR), Universitair Ziekenhuis Leuven (BE), Institut Gustave Roussy (FR), Boston Foundation (US), The Netherlands Cancer Institute (NL), Sorbonne Université (FR), Assistance Publique – Hôpitaux de Paris (FR), Hospices Civils de Lyon (FR), Centre Léon Bérard (FR), Istituto Oncologico Veneto (IT), Administração Regional de Saúde de Lisboa e Vale do Tejo (PT), Centre Eugène Marquis (FR), IPO Porto (PT), Hôpital Nord (FR), European Organisation for Research and Treatment of Cancer (BE), Centre Oscar Lambret (FR), Centre Hospitalier Universitaire de Liège (BE), Centro Hospitalar de Vila Nova de Gaia (PT), Erasmus MC Cancer Institute (NL), Institut du Cerveau (FR), National Cancer Institute (LT), Fondazione IRCCS Istituto Nazionale dei Tumori (IT), Institut Curie (FR)
F. Hoffmann-La Roche, Alliance Healthcare Foundation, European Network for Rare Adult Solid Cancers
Good health and well-being
Openalex Percentile: Top 9%
Sarcoma Diagnosis and Treatment
DOIOpen Access PDFOpenAlex
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