October 17, 2019

Old Drug, New Tricks, Provides Hope For Patients With Polycystic Kidney Disease

(Toronto, Canada – October 3, 2019) Dr. York Pei, an award-winning scientist at the University Health Network, has discovered a potential new use for an old drug, providing hope for patients suffering from polycystic kidney disease.

In a pre-clinical trial, Dr. Pei found Salsalate, a drug in the aspirin family, and an approved drug therapy for rheumatism, was successfully used to improve kidney survival and reduced cystic kidney disease severity. 

Polycystic Kidney Disease is a major cause of genetic kidney failure and affects thousands of Canadians and millions of people worldwide. One of the main molecules that modify disease progression in PKD is called AMPK-activated protein kinase. AMPK exists in low levels in those suffering from PKD so Dr. Pei decided to study known AMPK activators to see if the combination would have a positive synergistic effect. What he found was surprising.

"Salsalate as well as Metformin and Canagliflozin, two diabetes drugs, were tested because all three are AMPK activators," says Dr. Pei. "We found only the Salsalate was effective on its own and most importantly at levels that are achievable in humans."

Salsalate is a prodrug that gives rise to salicylic molecules, it is like aspirin but without certain adverse side effects. Traditionally Salsalate is used to treat diseases like rheumatoid arthritis and osteoarthritis.

"We used a moderately aggressive model that we believe provides the foundation for an eventual clinical trial," says Dr. Pei. "We'll test this in additional models but the good news is, it's safe and relatively inexpensive."

The study was partly funded by the PKD Foundation of Canada and through a grant provided by the Canadian Institutes of Health Research, Canada's federal funding agency for health research.

The study was published in EBioMedicine, an online journal published by The Lancet.

Dr. Pei is a Professor of Medicine at the University of Toronto and a Staff Nephrologist at the Toronto General Hospital, University Health Network, where he currently directs the Hereditary Kidney Disease Clinic.

About Toronto General Hospital and University Health Network
University Health Network consists of Toronto General and Toronto Western Hospitals, Princess Margaret Cancer Centre, Toronto Rehabilitation Institute, and The Michener Institute of Education at UHN. The scope of research and complexity of cases at University Health Network has made it a national and international source for discovery, education and patient care. University Health Network has the largest hospital-based research program in Canada, with major research in cardiology, transplantation, neurosciences, oncology, surgical innovation, infectious diseases, genomic medicine and rehabilitation medicine. University Health Network is a research hospital affiliated with the University of Toronto.