THALAMOSS - THALAssaemia MOdular Stratification System for personalized therapy of beta-thalassemia

Abstract:

THALAMOSS is aimed at development of universal sets of markers and techniques for stratification of β-thalassaemia patients into treatment subgroups for (a) onset and frequency of blood transfusions, (b) choice of iron chelation, (c) induction of fetal hemoglobin, (d) prospective efficacy of gene-therapy. At present, no framework exists to guide therapeutic decisions and personalised treatment of β-thalassaemia.
THALAMOSS Workpackages: WP1. Recruitment, patient characterization and development of erythroid precursor cells cultures; WP2. Omics analyses; WP3. Novel therapeutic approaches; WP4. Data analysis; WP5. Dissemination and exploitation; WP6. Regulatory and ethical issues; WP7. Management.
The impact of THALAMOSS is the provision of novel biomarkers for distinct treatment subgroups in β-thalassaemia (500-1000 samples from four European medical centres), identified by combined genomics, proteomics, transcriptomics and tissue culture assays, and establishment of routine techniques for detection of these markers. Translation of these activities into the product portfolio and R&D methodology of participating SMEs will be a major issue. THALAMOSS tools and technologies will (a) facilitate identification of novel diagnostic tests, drugs and treatments specific to patient subgroups and (b) guide conventional and novel therapeutical approaches for β-thalassaemia, including personalised medical treatments.
Key researchers of THALAMOSS are R.Gambari (Ferrara University, Italy), M. Kleanthous (The Cyprus Foundation for Muscular Dystrophy Research, Cyprus), S.Philipsen (Erasmus Universitair Medisch Centrum Rotterdam, The Netherlands), E.Katsantoni (Biomedical Research Foundation, Academy of Athens, Greece), S.Rivella (Weill Cornell Medical College, NY, USA), P.Holub (Masaryk University, Czech Republic), R.Galanello (Cagliari University, Italy), SL.Thein (King’s College Hospital, UK), E.Voskaridou (Laiko General Hospital, Greece). Participating SMEs are Biocep (Israel), NovaMechanics Ltd. (Cyprus) and IRBM (Italy). Industrial activities are also provided by Harbour Antibodies (The Netherlands).