Focal segmental glomerulosclerosis (FSGS) is a clinical-pathologic syndromes characterized by the accumulation of fibrotic proteins in glomeruli, initially involving only some glomeruli (focal) and involving portions (segments) of the affected glomeruli. FSGS can be classified as follows: idiopathic FSGS, genetic FSGS and post-adaptive FSGS (associated with glomerular hypertrophy and hyperfiltration, and due to reduced renal mass, renal toxins, obesity, and sickle cell disease). A related syndrome is collapsing glomerulopathy, associated with podocyte hyperplasia whereas FSGS is associated with podocyte depletion. Collapsing glomerulopathy can be classified as HIV-associated or idiopathic. Many patients with podocyte diseases, including minimal change nephropathy (MCN), FSGS, and collapsing glomerulopathy are refractory to all conventional remittive therapy. Current studies: * ritiuximab + cyclosporine: single center, open label * sparsentan: multicenter, placebo-controlled RCT, industry-sponsored (IRB submission pending) * MaNAc: phase 1B, single center, open label for pharmacokinetics and response (IRB approval pending, CRADA formerly with New Zealand Pharmaceuticals, now with Escala) We are carrying out studies of a dual-function molecule, cannabinoid receptor antagonist and AMPK agonist, in mouse models of FSGS, in collaboration with George Kunos, NIAAA

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U.S. National Inst Diabetes/Digst/Kidney
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Limou, Sophie; Dummer, Patrick D; Nelson, George W et al. (2015) APOL1 toxin, innate immunity, and kidney injury. Kidney Int 88:28-34
Cravedi, P; Kopp, J B; Remuzzi, G (2013) Recent progress in the pathophysiology and treatment of FSGS recurrence. Am J Transplant 13:266-74
Sharma, Kumar; Karl, Bethany; Mathew, Anna V et al. (2013) Metabolomics reveals signature of mitochondrial dysfunction in diabetic kidney disease. J Am Soc Nephrol 24:1901-12
Xu, Qihe; Kopp, Jeffrey B (2012) Retinoid and TGF-? families: crosstalk in development, neoplasia, immunity, and tissue repair. Semin Nephrol 32:287-94
Sharma, Kumar; Ix, Joachim H; Mathew, Anna V et al. (2011) Pirfenidone for diabetic nephropathy. J Am Soc Nephrol 22:1144-51
Gordon, Judit; Kopp, Jeffrey B (2011) Off the beaten renin-angiotensin-aldosterone system pathway: new perspectives on antiproteinuric therapy. Adv Chronic Kidney Dis 18:300-11
Wong, Yuen Fei; Kopp, Jeffrey B; Roberts, Catherine et al. (2011) Endogenous retinoic acid activity in principal cells and intercalated cells of mouse collecting duct system. PLoS One 6:e16770
Barisoni, Laura; Schnaper, H William; Kopp, Jeffrey B (2009) Advances in the biology and genetics of the podocytopathies: implications for diagnosis and therapy. Arch Pathol Lab Med 133:201-16
Joy, Melanie S; Gipson, Debbie S; Dike, Mary et al. (2009) Phase I trial of rosiglitazone in FSGS: I. Report of the FONT Study Group. Clin J Am Soc Nephrol 4:39-47