Polycystic Kidney Disease (PKD) is the most frequently inherited kidney disorder and affects nearly 600,000 Americans and as many as 12 million people worldwide. Currently, there are no FDA approved treatments for PKD.
Researchers at the University of Kansas have created a novel compound and method for treating PKD that leads to the inhibition of cyst growth.
HOW PKD AFFECTS THE KIDNEY
The mammalian Target of Rapamycin (mTOR) pathway supports cell survival and plays an important role in the regulation of cell growth and proliferation. KU inventors have created a novel compound and method for treating PKD utilizing a novel liver kinase B1 (LKB) activator to stimulate AMPK, an energy sensor that impedes mTOR signaling, cell growth, cell proliferation and chloride-dependent fluid secretion.
The unique, boron-based chemical compounds have shown:
- Reduced cyst formation and inhibiting cyst growth of human PKD cells
- Improved efficacy and potency without the potential problems associated with metformin, particularly lactic acidosis.
- Effective at nano-molar concentrations
This approach is superior to other methods because LKB1 is involved in the regulation of energy metabolism, nutrient sensing, glucose homeostasis, and cell growth. Therefore, agents that target LKB1 function will be substantially more effective in their mode of action to hinder cyst growth in PKD.
This treatment approach does not have some of the common side-effects (i.e. lactic acidosis) and has shown a high level of effectiveness in human PKD cells, making it a viable option for future commercialization potentially improving the lives of millions affected by this disease.
If you are interested in learning more, a summary of this new technology is available. To download a white paper about the technology, please fill out the form below.