Novel Treatment for Polycystic Kidney Disease (PKD)
Inventors: Jordan Kreidberg, Shan Qin
Invention Types: Therapeutics
Research Areas: Urology
Keywords: Animal Model (mouse), New Indication/Use, Small Molecule/DrugFor More Information Contact: Caron, Connie
Polycystic Kidney Disease (PKD) is the most common life-threatening genetic disease, affecting 600,000 Americans and 12.5 million people worldwide. PKD causes fluid-filled cysts to grow on the kidneys. Progressive accumulation of renal cysts causes grossly enlarged kidneys, and leads to renal failure in the majority of patients. About 50% of patients will require dialysis or a kidney transplant by the time they reach their 60s. Many individuals with PKD experience end stage renal disease as early as their 20s. Drs. Kreidberg and Qin have shown that inhibiting a well known membrane receptor, c-Met, reduces the number and size of cysts in a murine embryonic kidney organ culture model of PKD.
The discovery of Drs. Kreidberg and Qin describes a new role for c-Met in the pathogenesis of PKD and indicates that it may be a therapeutic target for this disease. These findings further suggest that c-Met inhibitors may represent a new strategy to treat PKD.
There is currently no known treatment or cure for PKD. Dialysis and transplantation are the options for patients who develop kidney failure. Pain and hypertension medicines, and ultimately laparoscopic surgery to eliminate existing cysts, are often used to alleviate the symptoms associated with PKD. Several inhibitors of c-Met are currently in development as anti-cancer agents. The discovery herein reveals a potential new use for these inhibitors for treatment of PKD.
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Key Publications: Qin S, et al. Failure to ubiquitinate c-Met leads to hyperactivation of mTOR signaling in a mouse model of autosomal dominant polycystic kidney disease. J Clin Invest. 2010 Oct;120(10):3617-28.
Qin S, et al. c-Met and NF-kB-dependent overexpression of Wnt7a and -7b and Pax2 promotes cystogenesis in polycystic kidney disease. J Am Soc Nephrol. 2012 Aug;23(8):1309-18.
IPStatus: Pat. Pend.