Research for Rare - Research for rare diseases

Speed Translation-Oriented Progress to Treat FSGS (STOP-FSGS)
Idiopathic (primary) FSGS is a rare disease affecting an estimated 2,000 people in Germany. Its secondary form represents a major cause for irreversible loss of renal function and life-long need for dialysis or transplantation. Building on novel methods and insights into patho-mechanisms of FSGS in the previous funding period, the present consortium brings together some of Germany’s most renowned FSGS experts. The consortium addresses key questions focussing on the initiation, diagnosis and novel therapies of FSGS. Regarding the initiation of FSGS, circulating permeability factors (Schiffer) and genetic causes (Huber) will be explored using innovative parabiotic animal models and high-end ‘omics platforms. Furthermore, the role of individual glomerular cells for regeneration (Endlich) and the loss of renal function (Moeller) will be investigated. Novel therapeutic concepts will be investigated in preclinical studies with a focus on candidate signalling pathways identified in the previous funding period (Boor, Moeller). In addition, available drugs will be screened using a novel in vivo assay (Endlich). Rapid translation to clinic continues to be a major focus of this consortium. Endlich will validate a new diagnostic test to differentiateprimary from secondary FSGS using super-resolution microscopy. Our specialized clinics for FSGS patients will provide access to novel therapies and participate in guidelines (Huber, Moeller, Schiffer). Finally, a national FOrMe FSGS patient registry will be created from existing regional biobanks to facilitate future clinical trials.Projects

Contact
Coordinating investigator:

Professor Dr. med. Marcus J. Moeller
RWTH University of Aachen,
Nephrology and Clinical Immunology
Pauwelsstrasse 30, D-52074 Aachen
Telephone 0241-8035204
Fax 0241-8082446
E-mail: mmoeller@ukaachen.de

Publications