SAN FRANCISCO, Dec. 10 (Xinhua) -- U.S. scientists have found a new compound that offers a potential treatment for progressive kidney disease and may save the lives of millions of people, a new study showed.
The research, conducted by a team of scientists from the Broad Institute of MIT and Harvard University, Brigham and Women's Hospital, and Harvard Medical School, describes a new approach to prevent cell death in essential kidney cells.
The researchers, whose findings were published Friday in the journal Science, studied multiple animal models of kidney disease and discovered a compound that can avert cell demise and restore kidney function.
They started their investigation with a rare genetic type of kidney disease and used an animal model to understand the genes, proteins and pathways involved in the organ's deterioration.
The kidney damage often features the loss of the filtration cells, or podocytes, which normally keep essential proteins in the blood and filter out toxins.
If the cells are destroyed, proteins from the blood start spilling out into the urine, a symptom called proteinuria.
The researchers' experiment shows that a protein called Rac1 is activated in kidney disease characterized with genetic mutilation, which activates a protein called TRPC5 in a damaging feedback loop.
TRPC5 then destroys the podocytes after pumping calcuium ions into the filtration cells.
However, the U.S. scientists designed and tested a new compound that could block this process and identified a small molecule they called AC1903.
The AC1903 molecule proved to be able to protect the kidney's filtration cells in a genetic rat model of progressive kidney disease.
It can prevent further podocyte loss and suppress proteinuria, thus restoring the kidney function.
The results of animal test suggest that the new compound may form the basis for much-needed, mechanistically based therapies for progressive kidney diseases.
Progressive kidney diseases, which could be caused by obesity, hypertension, diabetes or rare genetic mutations, often lead to the destruction of blood-filtering cells
Treatment options for the illness were few and often costly in most countries in the past several decades.
