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11.12.2013

Study Finds New Treatment Option for Recurrent Kidney Disease

In a multi-institutional study led at the Miller School by George W. Burke III, M.D., professor of surgery, and Alessia Fornoni, M.D., Ph.D., associate professor of medicine and the Peggy and Harold Katz Family Chair in Kidney & Vascular Disease Research, physician-scientists have uncovered a new treatment option for recurrent focal segmental glomerulosclerosis (FSGS), an idiopathic disease that causes kidney failure.

The study, “Abatacept in B7-1-Positive Proteinuric Kidney Disease,” published November 8 in the New England Journal of Medicine, identifies abatacept, an inhibitor that targets B7-1, a co-stimulatory molecule that, when under stress, is expressed by podocytes, the kidney cells responsible for development of FSGS.

“To date, therapy for FSGS and associated kidney disorders has been nonspecific, often ineffective, and fraught with side effects,” said Burke, Director of the Division of Kidney and Pancreas Transplantation. “Our clinical and in vitro data indicate that B7-1 induction in primary and recurrent FSGS offers a rationale for using abatacept to treat a subgroup of patients with proteinuric kidney diseases.”

The best treatment option for FSGS, which primarily affects children in minority populations, is kidney transplantation. However, the disease frequently returns within hours to weeks after a kidney transplant.

In preclinical studies, the off-label use of abatacept, an FDA-approved drug for treating rheumatoid arthritis, was associated with the appearance of B7-1 in kidney biopsies performed before and after surgery. The presence of B7-1 was demonstrated through immunofluorescence staining conducted by a team of scientists led by Fornoni.

“Such an important study could have not been accomplished if not through this strong inter-institutional and interdisciplinary effort, which successfully brings together once again the clinical and translational expertise of faculty at the Miami Transplant Institute with the basic science expertise of those at the Peggy and Harold Katz Family Drug Discovery Center,” said Fornoni, who directs the Drug Discovery Center.

In addition to the Miller School, the multicenter study included Harvard Medical School, Kaohsiung Medical University in Taiwan, the Icahn School of Medicine at Mount Sinai (formerly the Mount Sinai School of Medicine), and University Medical Center Hamburg-Eppendorf in Germany.

Other Miller School co-authors of the study include Junichiro Sageshima, M.D., associate professor of surgery, Linda Chen, M.D., assistant professor of surgery, Gaetano Ciancio, M.D., professor of surgery, and Christian Faul, Ph.D., assistant professor of medicine.

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