3 WilsonMed: Multimolecular targeting of copper overload in Wilson disease

Abstract

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.

Partners

  • Schmidt, Hartmut (Coordinator)
  • Kroemer, Guido
  • Polishchuk, Roman
  • Socha, Piotr
  • Zischka, Hans

Patient Advocacy Organisations and countries

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)

Publication title

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