Miguel Bronchud, Co-Founder and Director of Clinical Research at BO REAL BioTech, shared on LinkedIn:
“Molecular profiling and access to treatment for rare cancers in Europe – actionable mutations were found in over half the patients – but significant disparities regarding access to personalized medicine approaches.
In any case, finding a single Onco driver vulnerable to a curative approach remains difficult – but not impossible?
Gleevec, also marketed internationally as Glivec and sometimes referred to by its chemical name imatinib, entered the medical world with a bang because it almost ‘magically arrested the growth of malignant cells’, with a well tolerated oral drug.
This medication was initially approved for use by the U.S. Food and Drug Administration (FDA) in 2001 for the treatment of chronic myelogenous leukemia (CML), a rare form of cancer that affects certain types of white blood cells. Since its initial approval, Gleevec has also been approved for use in patients with several types of gastrointestinal Stromal tumors GISTs).
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 European 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. Access to personalized and appropriate drugs was limited by regulatory constraints or financial considerations in many cases.
Arcagen-EORTC Research Project 1843-is a collaborative project between EORTC and ERN EURACAN. From the EORTC headquarters, in Brussels, the authors would like to acknowledge Arnaud Poncin (data management), Carolina Fernandes (clinical operations), Marie-Sophie Robert (project management), and Cedric Mallien (EGA upload).
Arcagen is generously funded by F. Hoffman-La Roche. The EORTC SPECTA platform is supported by Alliance Healthcare. Alliance Healthcare will become Cencora. Fanny Hoogstoel’s work as Fellow at EORTC Headquarters was supported by the EORTC Cancer Research Fund.”
Title: Molecular profiling and access to treatment for rare cancers in Europe
Authors: M. Morfouace, F. Hoogstoel, J. Oliveira, N. Penel, S. Tejpar, I. Ray-Coquard, J. Peron, M. Pracht, B. Brasiuniene, A. Capela, C. Gennigens, L. Greillier, C. Even, R. Huang, T. Gorlia, G. Lombardi, M. Gamulin, L. Licitra, N. Girard, H. J. Klümpen, A. Idbaih, WTA Van der Graaf, J. Y. Blay
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