A simple new lab test predicts acute kidney failure: About 4 million people die of acute kidney failure each year. They die primarily because the current diagnosis of acute kidney failure is woefully delayed, crippling our ability to institute potentially effective therapies in a timely manner. A research team led by Dr. Prasad Devarajan, the Louise M. Williams Endowed Chair, Professor and Director of Nephrology at CCHMC, has identified a new biomarker called neutrophil gelatinase-associated lipocalin (NGAL), which appears in the urine and blood of patients up to three days before the current tests for acute kidney failure become positive. Dr. Devarajan’s team has validated the biomarker in a wide variety of pediatric and adult patient populations, including those undergoing heart surgery, kidney transplantation, radio-contrast administration, sepsis, nephrotoxic medications, subjects admitted to intensive care units, and even patients randomly presenting to the emergency room. Their results have recently been published in prestigious medical journals such as Lancet and Annals of Internal Medicine. In addition, Dr. Devarajan has partnered with industry collaborators to design standardized clinical laboratory-based point-of-care kits that can measure NGAL in a drop of urine or blood and provide quantitative results in 30 minutes or less. It is anticipated that these simple new tests will become widely accessible to the medical community within the next year. The availability of an early biomarker like NGAL could revolutionize medical care and save lives, by providing clinicians with a desperately needed tool for predicting acute kidney failure in hospitalized and ambulatory subjects, allowing for accurate risk assessment, optimizing resource utilization, providing timely therapies, monitoring the response to therapies, and providing a kidney safety marker for future drug development.
A transplant drug proves effective in treating kidney tumors: One goal of medical research is to understand disease mechanisms in order to develop new and more effective treatments. This was true for the kidney and lung manifestations of tuberous sclerosis complex and a sporadic disease called lymphangioleiomyomatosis. The cell-signaling pathway that is disrupted in both these diseases is the same pathway that is suppressed by the transplant drug called sirolimus. A research team led by Dr. John Bissler, the Clark D. West Endowed Chair in Nephrology at CCHMC, conducted a study to determine whether sirolimus had any effect on the kidney tumors called angiomyolipomas and the lung manifestation called lymphangioleiomyomatosis found in both diseases. After one year of treatment with sirolimus, the average size of angiomyolipomas was reduced by nearly 50 percent in patients. Sirolimus also improved lung function in the lymphangioleiomyomatosis patients. These results have recently been published in New England Journal of Medicine. Dr. Bissler is now conducting a study to identify an optimum dosing schedule for this strategy, to understand the mechanisms of this response, and to identify biomarkers that predict the best response. Research in Dr. Bissler’s laboratory is also looking at drug combinations to further optimize the treatment of these patients.
