TRANSLATIONAL RESEARCH
Wilson's disease (WD) is a rare inherited condition that causes copper levels to build up in several organs, especially the liver, brain and eyes. WilsonMed is a collaborative approach to research, management and treatment of patients with WD involving scientists, clinicians, industry and patients.
WilsonMed is a significant breakthrough in the management and treatment of patients with Wilson's disease through the synergistic combination of scientific expertise and patient experience.
Wilson disease (WD) is a genetic rare disease that results from a defect of copper (Cu) transporter ATP7B leading to progressive Cu accumulation. Currently, therapy is based on anti-Cu compounds with lifelong prescriptions.
Due to limitations of current therapy, improved therapy has to be developed to counteract devastating conditions of patients and their families.
WilsonMed aims to complete the preclinical development of selected anti-Cu compounds. The validated anti-Cu therapies will be developed for the market by all partners through the post-project steps of financing, formulation development, toxicology, clinical trials and regulatory approval.
The involvement of patient organisations throughout the project is crucial.
The new therapeutic approach includes advanced drug delivery into the brain and liver and developing animal models with further refinement
WilsonMed will thereby increase knowledge of the mode of action (MoA) of novel anti-Cu compounds as required by regulatory authorities and will protect th e knowledge generated to support the exploitation/commercialization of the results.
WilsonMed is a unique partnership that brings together leading experts in basic/translational research, clinicians, and Wilson disease patient organisations to develop new treatment options, initiate proof-of-principle studies in preclinical models, evaluate predictive biomarkers for monitoring therapy, and bring new concepts to patients first-hand. The WilsonMed project has received funding from the EJP RD initiative of the European Union’s Horizon 2020 research and innovation programme.
PRINCIPAL INVESTIGATOR AND CONSORTIUM COORDINATOR
Direktor der Klinik für Gastroenterologie, Hepatologie und Transplantationsmedizin
Universitätsklinikum Essen
Universität Duisburg-Essen
E-mail: gastroenterologie(at)uk-essen.de
Germany
PRINCIPAL INVESTIGATOR
Director of Research Inserm U1138 "Metabolism, cancer and immunity".
Institut National de la Santé et la Recherche Médicale (Inserm)
France
PRINCIPAL INVESTIGATOR
Associate Investigator Head of Advanced Microscopy and Imaging Core
Telethon Institute of
Genetics and Medicine (Tigem)
Italy
PRINCIPAL INVESTIGATOR AND CLINICAL EXPERT
Professor in the Department of Gastroenterology, Hepatology, Nutritional Disorders and Pediatrics.
Deputy Director for Research
The Children’s Memorial Health Insitute (CMHI)
Poland.
PRINCIPAL INVESTIGATOR
Group Leader “Oxidative Cell Death”
Institute of Molecular
Toxicology and
Pharmacology, Helmholtz
Center Munich, Germany.
Research Center for
Environmental Health,
Germany
PATIENT REPRESENTATIVE
Morbus Wilson e.V.
Germany
PATIENT REPRESENTATIVE
Associazione Nazionale Malattia di
Wilson
Italy
PATIENT REPRESENTATIVE
Polish patients group collaborating with Dr SOCHA
Poland
PATIENT REPRESENTATIVE AND PAO COORDINATOR
French patients group. Ms Roatta is also the project's patient coordinator.
France
2024-11-30
Founded on 24 July 2024, this organisation brings together patients and parents to raise awareness and promote research, access to treatment, and patient integration, under the leadership of Anna Anioł and Agnieszka Miodek.
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2024-08-22
The WilsonMed scientific team published two papers this summer. They are available in the ‘Resources’ section.
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2024-05-10
Per Humanus foundation presents the key points, needs and the actions implemented to support patients during the WilsonMed Project.
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