PROFILE · Innovative training network: Immunoprofile-directed stratification of patients with the autoimmune disorder thrombotic thrombocytopenic purpura

Innovative training network: Immunoprofile-directed stratification of patients with the autoimmune disorder thrombotic thrombocytopenic purpura

Problem The increasing prevalence of autoimmune disease represents a major challenge for the healthcare system in Europe. Current treatments are far from being optimal as virtually all patients receive the same, non-specific treatment. In the current PROFILE project, we study the autoimmune disorder acquired thrombotic thrombocytopenic purpura (aTTP). The unmet clinical needs are fine-tuned diagnostic tools and truly personalised treatment options. We want to identify how patients with aTTP match or differ from each other. Specific markers in each patient will be identified which will lead to a barcode profile. Patient stratification by barcode should allow better prediction of relapse. On top of that, treatment options will be identified and developed for each patient group. Impact to society The PROFILE project will result in improved diagnosis and treatment of patients suffering from aTTP. This will lead to an increased quality of life and a significant reduction in healthcare costs. Importantly, the strategy applied in this project to aTTP patients can easily be translated to many other autoimmune disease settings. Overall objectives Objective 1: To understand the role of specific markers (immunoprofiling markers: antibodies and T-cells) in the autoimmune disease aTTP Objective 2: To group patients with matching markers Objective 3: To link the markers with disease prognosis Objective 4: To develop novel treatment strategies To reach these objectives, the six early stage researchers (ESRs) working in the PROFILE project are receiving specialized training. They learn how to identify a clinical need, perform translational research, transfer technology and knowledge to the industry, manage intellectual property rights and act as competent managers. The scientific objectives and the high-level training and supervision of the ESRs is realised by the combination of highly skilled and specialized immunoprofiling experts from small and medium enterprises (Icosagen and Protobios, Estonia; Immudex, Denmark; Biokit, Spain; PharmaTarget, The Netherlands) as well as world-leading experts on acquired TTP from academia (KU Leuven, Belgium; Sanquin, The Netherlands; Assistance Publique-Hopitaux de Paris, France). Translational aspects are well covered by the inclusion of expert clinicians (Assistance Publique-Hospitaux de Paris, France) as well as representatives from patient groups (Stichting Zeldzame Bloedziekten, The Netherlands). Specific aspects of the training program are brought to the ESR during network wide events that are organised every 6 months. Importantly, these training sessions are open to all interested and are announced well in advance via our website (www.itn-profile.eu). Dissemination of results emanating from the PROFILE consortium to patients, clinicians and other groups of interest is ensured by the inclusion of the European Hematology Association (EHA) as a partner organisation.

Data: CORDIS, © European Union