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FDA approval of kidney drug "a game changer" for PKD patients

05/30/2018

Claudia Iliescu’s youthful appearance belies her actual age of 47. Unfortunately, Claudia’s kidneys have not aged nearly as gracefully. Claudia suffers from autosomal dominant polycystic kidney disease (ADPKD), a chronic, hereditary condition in which cysts grow inside the kidneys, reducing kidney function, and ultimately resulting in kidney failure and a premature need for dialysis or transplant. 

About ADPKD and a new treatment

“It’s a devastating disease that impacts multiple family members for generations,” said Claudia. “My 70-year-old mother has been on dialysis for the past 10 years. My grandmother died at 56 from ADPKD complications. And I have many aunts, uncles and cousins on my mother’s side who have been affected by this disease.”

There is no cure for ADPKD, but now there is reason for optimism for Claudia and other ADPKD patients. On April 24, 2018, the U.S. Food and Drug Administration approved Tolvaptan, the first-ever disease-modifying drug for ADPKD. Tolvaptan slows the growth of kidney cysts and prolongs kidney function.

Seeking treatment 

Claudia was diagnosed with ADPKD at age 22, but didn’t start experiencing symptoms until her 30s. Starting in 2010, she began having occasional sharp pain and discomfort in her abdomen. Due to a combination of kidney and liver disease, it became difficult for her to eat or bend over.

In early 2012, Claudia entered a three-year clinical trial at Tufts Medical Center – on the recommendation of her nephrologist, Associate Chief of the Division of Nephrology Ronald Perrone, MD – to test the effectiveness of Tolvaptan for ADPKD. Dr. Perrone served as the Principal Investigator at the Tufts MC study site and helped design the study and monitor its progress. While a bit skeptical at first, Claudia was thrilled with the results. 

Clinical trial success

“Tolvaptan prevented my pain from getting worse and slowed the growth of the cysts in my kidneys,” she said. “And now the FDA approval confirms that Tolvaptan works. I am very hopeful that it will help me and other people diagnosed with ADPKD enjoy many more years without needing dialysis or a kidney transplant.” 

“This is a major breakthrough,” said Dr. Perrone. “PKD is the fourth-leading cause of kidney failure in the U.S.; it greatly impacts quality of life and can lead to other serious health complications. Now, a single pill taken twice per day can slow the disease’s progression, allowing patients to be able to enjoy additional years of adequate kidney function before requiring additional intervention. That’s a game-changer for these patients.” 

The Center for Polycystic Kidney Disease at Tufts Medical Center, the only PKD center in Boston, has extensive experience in diagnosing and managing chronic kidney disease. For more information, or to make an appointment, call 617-636-5866.

Learn more about the research and drug approval >