Minimal Change Disease (MCD), Focal and Segmental Glomerulosclerosis (FSGS), and Membranous Nephropathy (MN) are glomerular diseases that despite their rarity, account for a large fraction of prevalent ESRD. There is growing consensus that the presently employed histopathology-based classification of FSGS and MN is inadequate because it is not based on an understanding of the molecular basis of these diseases, and because it does not well predict the heterogeneous natural history or response to therapy of individuals within a given glomerular histopathological category. Given these inadequacies, it is not surprising that our therapeutic approach to these diseases is imperfect. We propose that several major barriers must be overcome before more effective interventional studies of primary non-inflammatory glomerular disease can be conducted. Among these barriers is the absence of specific biomarkers of glomerular disease that would allow refined, biologically relevant sub-classification of glomerular disease histopathology useful for defining subject inclusion and exclusion criteria in clinical studies. Such disease sub-classification might overcome the effects of study population heterogeneity that likely have complicated interpretation of past studies of these glomerular diseases. New glomerular disease biomarkers might also predict disease natural history, allow proper selection of and prediction of response to specific therapeutic intervention, allow early detection of disease, or provide indicators of disease activity. Importantly, a robust investigative infrastructure is presently lacking that would facilitate collection, cultivation, and access to human biological material and associated clinical data necessary for biomarker identification, for the identification of clinically relevant study endpoints, and for conducting pilot clinical studies that would advance the care of MCD, FSGS, and MN patients. For these reasons, we propose the establishment of a Nephrotic Syndrome RDCRN, a multidisciplinary research and education platform that brings together clinical and translational scientists and two lay research and patient education foundations, aimed at beginning to better study and educate patients with FSGS, MN, and MCD.

Public Health Relevance

MCD, FSGS, and MN are rare diseases that cause serious morbidity and high mortality, generating enormous individual and societal economic burden. Creation of the Nephrotic Syndrome Rare Disease Consortium will provide a readily accessible research and education resource that will significantly advance our ability to study, classify, characterize, diagnose, and treat non-inflammatory glomerular diseases.

Agency
National Institute of Health (NIH)
Institute
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Type
Specialized Center--Cooperative Agreements (U54)
Project #
5U54DK083912-04
Application #
8328121
Study Section
Special Emphasis Panel (ZRG1-HOP-Y (50))
Program Officer
Moxey-Mims, Marva M
Project Start
2009-09-08
Project End
2014-06-30
Budget Start
2012-07-01
Budget End
2013-06-30
Support Year
4
Fiscal Year
2012
Total Cost
$1,245,786
Indirect Cost
$420,523
Name
University of Michigan Ann Arbor
Department
Internal Medicine/Medicine
Type
Schools of Medicine
DUNS #
073133571
City
Ann Arbor
State
MI
Country
United States
Zip Code
48109
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