NIH Pediatric Center of Excellence in Nephrology
Prasad Devarajan, MD, director of Nephrology and Hypertension, was awarded a prestigious five-year, $3.7 million Center of Excellence (P50) grant by the National Institute of Diabetes and Digestive and Kidney Diseases. Ours is one of only three such centers that were funded. The Center will fund groundbreaking translational research projects in three areas of unmet need, including acute kidney injury, nephrotic syndrome, and lupus kidney disease. It will bring together investigators performing cutting-edge bench-to-bedside research from several divisions within Cincinnati Children’s, including Nephrology, Cardiology, Critical Care, Rheumatology, Developmental Biology, and Bone Marrow Transplant. The Center will include funding for innovative Research Cores in Genomics, Proteomics, and Biomarker Development, to support the primary focus areas. The Center will also incorporate an Administrative Core, and an Enrichment Core for the training of future pediatric nephrology researchers.
Nephrology Clinical Laboratory launches new tests for atypical HUS
Hemolytic uremic syndrome, or HUS, is a serious disease that can lead to kidney failure and even death. Some forms of HUS (known as atypical HUS) are associated with defects in the regulation of the complement system, a portion of the immune system, and are even more lethal if not correctly identified and treated. After caring for several patients with atypical HUS for whom the testing required sending samples to other institutions, taking weeks and even months to complete, Bradley Dixon, MD, wanted to make identification of these atypical forms of HUS easier and faster. He developed several blood tests to rapidly evaluate such patients, which have now been launched by the Nephrology Clinical Laboratory. These tests can quickly assay for the most common causes of atypical HUS, identify conditions that can closely resemble atypical HUS, and help clinicians rapidly treat the disease to improve patient outcomes. The Clinical Laboratory has already become a regional resource for HUS testing in the Tri-state area, and remains a national leader in performing unique tests for the diagnosis of other complement disorders, acute kidney injury, and chronic kidney disease.
Nephrology Research leads to FDA Approval of New Drug for Tuberous Sclerosis
April 29, 2012 was a very good day for patients with a tumor predisposition syndrome called tuberous sclerosis complex. People affected with this disease develop renal tumors called angiomyolipomata. Until this last April, only surgery or endovascular procedures could help control individual tumors; however, many patients have bilateral, multifocal kidney involvement that was previously untreatable. Work pioneered here, including an eleven-country, placebo-controlled trial led by John Bissler, MD, has changed the outcome for these patients. The trial demonstrated that the drug Everolimus caused significant shrinkage of the renal tumors in patients suffering from tuberous sclerosis renal disease. The significance of these findings led the Food and Drug Administration to approve Everolimus for the treatment of patients with tuberous sclerosis complex. In the recent Tuberous Sclerosis Consensus Conference, Everolimus was recommended as the first line of therapy for these tumors. This represents the first approved drug that can alter the progressive nature of these tumors, and offers new hope for patients with Tuberous Sclerosis renal disease.