Scientists discover potential new drug therapy for kidney diseasesApril 27th, 2011 - 5:35 pm ICT by ANI
Washington, April 27 (ANI): Scientists at the Department of Molecular, Cellular and
Developmental Biology and the Neuroscience Research Institute at UCSB have
discovered a potential new drug therapy for patients with kidney diseases.
Statistics show that over 600,000 people in the U.S and 12 million worldwide are
affected by the inherited kidney disease known as autosomal-dominant polycystic
kidney disease (ADPKD), which is characterized by proliferation of cysts that
damage the kidneys, causing kidney failure in half of the patients by the age of 50.
Currently, no treatment exists to prevent or slow the cyst formation and most
ADPKD patients require kidney transplants or lifelong dialysis for survival, says
Thomas Weimbs, Associate professor in the Department of Molecular, Cellular
and Developmental Biology and the Neuroscience Research Institute at UCSB.
First, Weimbs and his research team discovered a molecular mechanism that
sheds light on the disease. The mechanism concerns polycystin-1, a protein that is
mutated in ADPKD patients. They found how this protein regulates a well-known
transcription factor called STAT3. Transcription factors transcribe information from
DNA to RNA, from specific genes. Second, the team discovered that STAT3 is
strongly and aberrantly activated in polycystic kidneys.
“The clinical significance of these discoveries lies in the fact that STAT3 is also
known to be aberrantly activated in many forms of cancer and is considered an
important drug target for cancer therapy,” said Weimbs.
“Numerous STAT3 inhibitors are currently being developed and tested, and several
experimental drugs are already available. Our results suggest that STAT3
activation is a driving force for the cyst growth that leads to polycystic kidneys in
ADPKD. Therefore, STAT3 may be a highly promising drug target for the treatment
of ADPKD,” he added.
Weimbs explained further that STAT3 is a signaling molecule that is activated in
response to many different growth factors binding to specific receptors on the
surface of kidney cells. In response to these growth factors hitting the cell, STAT3 is
activated. That causes STAT3 to turn on the expression of certain genes. This
activity causes the cells to proliferate, as they do in cancer.
The research is published in the Proceedings of the National Academy of
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