An international team of researchers including scientists from the Mayo Clinic and the University of Zurich have shown that tolvaptan, a drug already used to treat low blood sodium levels in patients with heart failure and liver problems, may be a promising new treatment for PKD. The findings are the culmination of 15 years of research.

In a recent three-year study of 1400 patients worldwide, tolvaptan reduced cyst growth and kidney growth, reduced pain, and slowed the decline in kidney function in patients with autosomal dominant polycystic kidney disease (ADPKD). The adverse effects of increased urination and thirst as well as increased liver enzymes and blood sodium levels did lead to a higher rate of discontinuation of the drug compared to patients taking a placebo.

Sources:

Cystic Kidney Growth Curbed, ScienceDaily, November 5, 2012

Tolvaptan in Patients with Autosomal Dominant Polycystic Kidney Disease, New England Journal of Medicine, November 3, 2012

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UC Santa Barbara researchers, in collaboration with the biotech firm, Endocyte, have put a new twist on a common drug, which could successfully treat PKD in the future. In earlier research, UCSB scientist Thomas Weimbs had identified the immunosuppressant drug rapamycin as a good candidate for PKD treatment. While rapamycin effectively stopped PKD progression in mice, clinical trials showed that safe levels of the drug weren’t enough to stop kidney cysts in humans.

In this latest research, the scientists decided to find out if they could target PKD cysts in the same way they target cancer cells. Endocyte has successfully added folate to cancer drugs, thus delivering the treatment straight to cancer cells with folate receptors. The team then found that both mouse and human PKD cysts do, indeed, have folate receptors. Endocyte created the new drug, FC-rapa, which links folate with rapamycin and directly targets the kidneys. Treatment in mice was promising, preventing PKD cyst growth with fewer side effects.

Source:

New Drug Shows Promise for Kidney Disease, ScienceDaily, August 2, 2012

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Our spokesperson, Valen Keefer, continues to inspire the kidney disease and transplant community with her new blog for the PKD Foundation, PKD Will Not Beat Me. She’ll be sharing her story and fostering a positive space where people with PKD can come together to talk with each other, get information and lift each other’s spirits.

We are so proud of Valen and the wonderful work she does! Check out her first blog post and you’ll find out what PKD really stands for in Valen’s world.

A screen shot of Valen's blog

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Yale School of Medicine researchers have discovered that the PKD1 gene not only causes cysts to grow on the kidneys, but on the liver as well.  Their studies of families with liver-only polycystic disease led them to explore the relationship between that liver condition and the most common form of Polycystic Kidney Disease, ADPKD. They have determined that low expression of PKD1 leads to cysts and theorize that increased expression could slow cyst formation. They believe that targeting the activity of PKD1 could benefit those with liver-only polycystic disease, as well as children with recessive PKD and some adults with ADPKD.

Source:

Single Gene Controls Development of Many Forms of Polycystic Disease, Yale University, July 19, 2011

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Researchers from Fox Chase Cancer Center in Philadelphia have found that Aurora A kinase, an enzyme linked to tumor growth in cancer, may contribute to polycystic kidney disease. They discovered that Aurora A is over-expressed in the cells lining PKD cysts. It likely contributes to the disease by disrupting the activity of polycystin-2, a protein produced by the PKD-2 gene, which is often mutated in patients. Polycystin-2 acts as a calcium channel, allowing the flow of calcium ions into kidney cells and triggering functions important to kidney development. The scientists theorize that inhibiting Aurora A may restore polycystin-2 activity in some PKD patients. They hope continued research will explain why Aurora A is over-expressed in PKD and plan to conduct experiments to determine if inhibiting Aurora A slows the growth of cysts.

Sources:

Aurora A May Contribute to Kidney Disease, Rockefeller University Press, June 13, 2011

Research Abstract: Aurora A Kinase Activity Influences Calcium Signaling in Kidney Cells

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Australian researchers have discovered how to turn specialized human kidney cells back into generalized cells known as induced pluripotent stem cells or iPS cells. This type of cell reprogramming is a useful tool for studying genetic kidney diseases.  The scientists hope to generate iPS cells from patients with Polycystic Kidney Disease and Alport syndrome; the transformed cells would retain the genetic information that causes these diseases, providing scientists with greater insight into how these disorders work and how to develop stem cell therapies to treat them.

Source:

Stem Cell Tool in Kidney Disease, The Australian, May 18, 2011

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Researchers at UC Santa Barbara have discovered that the same molecular mechanism that causes cancer cells to multiply also causes cysts to grow in patients with ADPKD, the most common form of Polycystic Kidney Disease. They found that the transcription factor STAT3 is activated by a mutated protein present in ADPKD patients. UCSB researchers are now investigating whether experimental cancer drugs to inhibit STAT3 could one day prove an effective therapy for PKD.

Source:

UCSB Scientists Discover New Drug Target for Kidney Disease, UC Santa Barbara, April 26, 2011

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