Unapproved Drug Therapy to Cure Inherited Form of Kidney Disease

The researchers from Mayo clinic have come up with a novel drug therapy that helps in treating inherited form of kidney disease known as autosomal dominant polycystic kidney disease (ADPKD).

Autosomal dominant polycystic kidney disease (ADPKD) is a genetic condition that causes multiple cysts to develop on the kidneys. This disease triggers between the age of 30 and 60. The symptoms of ADPKD can range from mild to severe. It is uncommon as only 1 in every 1,000 people are born with the condition. 

They have come with a medication tolvaptan that slowed down the pace of kidney cyst growth over three years of the study.

tolvaptan demonstrated a nearly 50 percent reduction in the rate of increase in total kidney volume  in ADPKD patients over the study period, compared to placebo.

"ADPKD is the most common inherited and the fourth most common overall cause of kidney failure worldwide," says lead author Vicente Torres, M.D., Ph.D., Mayo Clinic nephrologist.

"In most patients with this disease, relentless cyst growth within the kidneys destroys the tissue, causes hypertension and painful complications, and negatively impacts the quality of life," Dr. Torres says. "The results of this study reveal a potential treatment that blunts kidney growth, lessens associated symptoms and slows kidney function decline when given over three years."

Dr. Torres notes that while the trail findings are encouraging, tovaptan has not yet been approved for this indication.

"The data from this study are compelling and provide a strong basis for global regulatory filings," said William H. Carson, M.D., President & CEO, Otsuka Pharmaceutical Development & Commercialization, Inc. "Tolvaptan was discovered by Otsuka in Japan, and if approved by regulatory agencies, could become the first pharmaceutical therapy for patients who suffer from ADPKD. Our research philosophy is to take untraveled paths to discoveries that only Otsuka can reach."

The phase three clinical trial results were being presented November 3 at the American Society of Nephrology annual meeting and published online in the New England Journal of Medicine.