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Peer-reviewedCC BY-NC-NDNature Communications

Molecular profiling and access to treatment for rare cancers in Europe

11 more authors

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.

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BibTeX
@article{morfouace2026molecular,
  title = {{Molecular profiling and access to treatment for rare cancers in Europe}},
  author = {M. Morfouace and F. Hoogstoel and J. Oliveira and N. Penel and S. Tejpar and I. Ray-Coquard and J. Peron and M. Pracht and B. Brasiuniene and A. Capela and C. Gennigens and L. Greillier and C. Even and R. Huang and T. Gorlia and G. Lombardi and M. Gamulin and L. Licitra and N. Girard and H. Klümpen and A. Idbaih and Wta van der Graaf and J. Blay},
  journal = {Nature Communications},
  year = {2026},
  month = jul,
  doi = {10.1038/s41467-026-75776-1},
  url = {https://hal.science/hal-05749328v1},
  note = {CC BY-NC-ND},
}
RIS
TY  - JOUR
TI  - Molecular profiling and access to treatment for rare cancers in Europe
AU  - M. Morfouace
AU  - F. Hoogstoel
AU  - J. Oliveira
AU  - N. Penel
AU  - S. Tejpar
AU  - I. Ray-Coquard
AU  - J. Peron
AU  - M. Pracht
AU  - B. Brasiuniene
AU  - A. Capela
AU  - C. Gennigens
AU  - L. Greillier
AU  - C. Even
AU  - R. Huang
AU  - T. Gorlia
AU  - G. Lombardi
AU  - M. Gamulin
AU  - L. Licitra
AU  - N. Girard
AU  - H. Klümpen
AU  - A. Idbaih
AU  - Wta van der Graaf
AU  - J. Blay
PY  - 2026
DA  - 2026/07/23
JO  - Nature Communications
DO  - 10.1038/s41467-026-75776-1
UR  - https://hal.science/hal-05749328v1
AB  - 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.
SN  - 2041-1723
SN  - 2041-1723
ER  -