Project: Multimolecular targeting of copper overload in Wilson disease
| Acronym | WilsonMed (Reference Number: EJPRD20-006) |
| Project Topic | 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. |
| Network | EJP RD |
| Call | 2nd EJP RD Joint Transnational Call for Rare Diseases Research Project (JTC 2020) |
Project partner
| Number | Name | Role | Country |
|---|---|---|---|
| 1 | Universitätsklinikum Münster | Coordinator | Germany |
| 2 | Centre de Recherche des Cordeliers | Partner | France |
| 3 | Telethon Institute of Genetics and Medicine | Partner | Italy |
| 4 | Children’s Memorial Health Institute | Partner | Poland |
| 5 | Helmholtz Center Munich, German Research Center for Environmental Health | Partner | Germany |
| 6 | Morbus Wilson e.V. | Partner | Germany |
| 7 | Association Bernard Pépin pour la maladie de Wilson | Partner | France |
| 8 | Associazione Nazionale Malattia di Wilson O.N.L.U.S. | Partner | France |
| 8 | Polish Association of Wilson Disease Patients | Observer | Poland |
| 9 | University Clinical Center, Medical University of Warsaw | Observer | Poland |
| 10 | Poland | Observer | Poland |