Thaindian News

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

Sciences. (ANI)