Funding transnational collaborative research through joint transnational calls is one of the major objectives of E-Rare. This is the most important and effective joint activity to enhance the cooperation between European scientists working on rare diseases and thus reducing the fragmentation of research in this field. E-Rare launches calls on a yearly basis. The topic and eligibility criteria are specified every year and therefore may vary from one call to the other.

Repurposing doxycycline in the treatment of AL amyloidosis

Project Coordinator


Cristopher Venner Cross Cancer Institute, University of Alberta Edmonton, Alberta, Canada
Arnaud Jaccard CHU Limoges Limoges, France
Stefan Schönland Ruprecht-Karls-University Heidelberg Heidelberg, Germany
Carlos Fernández de Larrea Fundació Clinic per la Recerca Biomèdica (FCRB) Hospital Clinic de Barcelona (HCB) Barcelona, Spain
Ayse Salihoglu Istanbul University Cerrahpasa Faculty of Medicine Istanbul, Turkey

Light-chain (AL) amyloidosis is caused by a population of plasma cells (antibody-producing cells) producing antibody parts, light chains (LC), that deposit in tissue in an ordered form called amyloid. Heart involvement is responsible for almost all deaths. Most patients are treated with a powerful anti-plasma cell drug, bortezomib. However, this improves heart involvement only in a few cases. Doxycycline, a widely used antibiotic, was shown to disrupt amyloid deposits and counteract LC toxicity in laboratory studies and animal models. In a preliminary clinical study, patients receiving doxycycline during anti-plasma cell treatment survived longer compared to similar patients who had received only anti-plasma cell therapy in the past. In the present study, patients with AL amyloidosis with similar degree of heart involvement, will randomly receive doxycycline or standard antibiotics in addition to bortezomib-based therapy, in order to assess the safety and efficacy of doxycycline.

E-Rare 2012 - Created by Toussaint Biger