Two research projects investigating treatment options for Alport syndrome have been awarded funding by the Alport Syndrome Foundation, Pedersen Family, and the Kidney Foundation of Canada Research Funding Program. The projects include a study on drug repurposing to treat Alport syndrome, and a study on WISE antibody as a treatment for Alport syndrome.

Alport Syndrome is a rare hereditary kidney disease that causes kidney failure, hearing loss, and vision abnormalities. Most boys experience kidney failure by the time they reach their early 20s. Girls are affected too, with some impacted similarly to boys but most seeing later disease progression. Alport Syndrome often affects multiple family members across generations. There is currently no treatment proven to prevent the development of kidney failure in people with Alport Syndrome; however, early diagnosis is essential as there are medications to delay the progression of the disease.

More about the studies
Dr. James Scholey of the University of Toronto was awarded $100,000 for one-year study on “Drug Repurposing for the Treatment of Experimental Alport Syndrome. This study will target the patterns of gene expression in the kidney associated with the progression of kidney injury using an FDA-approved drug vorinostat. It will be determined if this drug prolongs the lifespan of mice and if combined treatment with ACE inhibitors (current first line of treatment for Alport Syndrome patients) provides additional benefit.

Dr. Jeffrey Miner of Washington University was awarded $76,500 for a one-year study on “WISE Antibody as a Treatment for Alport Syndrome.” The goal of this study is to test the effectiveness of two antibodies at slowing the progression of kidney failure both separately and with ACE inhibition in mice models.

“We are pleased with the response and quality of research which the program has attracted and the progress which is being made in finding new drugs to further delay the progression of this disease,” said Harold Pedersen on behalf of the Pedersen Family.