3 WilsonMed: Multimolecular targeting of copper overload in Wilson disease

Joint Transnational Call 2020 (JTC20202)

3 WilsonMed: Multimolecular targeting of copper overload in Wilson disease

Wilson disease (WD) results from a defect of copper (Cu) transporter ATP7B leading to high oxidative stress due to Cu accumulation, mostly in the liver. Currently, therapy is based on anti-Cu compounds identified decades ago. However, a portion of WD patients do not respond, show non-compliance, and miss adequate therapy in case of high emergency, including liver transplantation. WilsonMed is a unique partnership that unites leading experts of basic/translational research, clinicians, and PAOs to (i) provide novel treatment options, (ii) launch proof-of principle studies in preclinical models, (iii) assess predictive biomarkers for monitoring of therapy, and (iv) disseminate novel concepts first-hand to PAOs. The partners of WilsonMed are stemming from distinctive fields having long-lasting expertise of state-of-the-art research, including (i) novel chemically-tailored Cu chelators having improved efficacy, (ii) screening of anti-Cu moieties, including repurposed drugs (iii) compound-induced enhancement of tissue-specific autophagy, (iv) gene-editing/silencing methodology, and (v) establishment of novel WD models. Having achieved important milestones in their individual fields, the partners combine activities by sharing advanced cellular platforms, animal models, and multi-molecular concepts for the short-term implementation of innovative treatment strategies. The expertise of the WilsonMed partners and PAOs will be synergistically combined to contribute to a significant enhancement in the management and therapy of patients having WD.

  • Schmidt, Hartmut (Coordinator)
    Westfälische Wilhelms Universität [GERMANY]
  • Kroemer, Guido
    Institut National de la Santé de la Recherche Médicale [FRANCE]
  • Polishchuk, Roman
    Telethon Institute of Genetics and Medicine [ITALY]
  • Socha, Piotr
    Instytut „Pomnik – Centrum Zdrowia Dziecka” [POLAND]
  • Zischka, Hans
    Helmholtz Zentrum München & Technische Universität München [GERMANY]
  • Association Bernard Pépin pour la maladie de Wilson [FRANCE]
  • Associazione Nazionale Malattia di Wilson O.N.L.U.S. [ITALY]
  • Morbus Wilson e.V. [GERMANY]
  • Polish Association of Wilson Disease Patients [POLAND]

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