Biology Cellular and Integrative Biology Anatomy and Cell Biology
Investigation of new treatments for polycystic kidney disease; Investiagtion of new treatments for hydrocephalus
We have recently conducted a series of experiments using animal models to study whether a class of FDA-approved drugs could be used for the treatment of polycystic kidney disease (PKD). PKD is the most common genetic disease in the population and in middle age the progression of this disease causes renal failure in over half of the affected individuals. Our studies are based on a series of unrelated experiments that serendipitously showed that some of the disease pathology in PKD may be amenable to treatment with insulin sensitizing agents that are used to treat type 2 diabetes in humans. These drugs have relatively few side effects and, therefore, they would be ideal treatment options for long-term therapy in PKD. The animal studies confirmed the tissue culture studies and we have now submitted a grant to the NIH to request funds to start a small human clinical trial.