A promising JDRF-funded trial looks at allopurinol—a 50-year-old gout treatment drug—to slow or stop loss of kidney function in people living with Type 1 Diabetes (T1D)

The Preventing Early Renal Function Loss in Diabetes (PERL) Consortium is conducting a multi-site, international clinical trial of allopurinol as an intervention for T1D-related kidney disease. Allopurinol is an inhibitor of the enzyme that produces uric acid, which has been used for decades to treat gout – a disease of the joint that occurs when uric acid levels are very high. It is inexpensive and safe medication. JDRF’s previously supported work that led to the discovery that high levels of uric acid in the blood are associated with rapid progression of kidney disease in people with T1D. You can click on this video to learn more about this trial and a trial participant’s experience.

Currently, “there are basically two ways to prevent kidney disease; one is good glycemic control,” said Alessandro Doria, M.D., Ph.D., Joslin Diabetes Center’s genetics core director and co-principle investigator of the PERL study. “In theory, if somebody can bring blood glucose down to normal levels and keep it to those levels most of the time, the risk of kidney disease is minimal.” But achieving such good control is challenging, added Dr. Doria.  More than 50 percent of the individuals with diabetes do not have satisfactory glycemic control. The second current option is to try to prevent kidney disease by using Renin-angiotensin system blockers or (RASB), but these are only partially effective. “Clearly there is the need for other medications to prevent this problem or to slow done the loss of kidney function if this has started, such as Allopurinol,” said Dr. Doria.

The PERL trial is also receiving funding from the National Institutes of Health through the Special Diabetes Program. The trial began recruiting participants in June 2014 and is expected to complete recruitment in the Fall of 2015. The 10-site study aims to recruit a total of 480 adult volunteers with T1D who are at increased risk for developing kidney disease to help test the drug. Participants will be randomly assigned to take either allopurinol or a placebo for three years, and their glomerular filtration rate—a test that measures how much blood passes through waste filters in the kidneys—will be measured at the beginning and end of the treatment period to determine if the patient taking allopurinol experiences less kidney function loss over time than those taking the placebo pills.  For more information or to support JDRF’s Complications research program, please click here.

Why it matters:

The PERL trial is designed to find an early intervention that can effectively prevent kidney damage. If allopurinol can reduce the rate of kidney function loss to half, this might provide a 10-to 15-year delay in reaching end-stage renal disease. Results from the study are expected in 2019, and should the findings demonstrate allopurinol’s effectiveness in averting the loss of kidney function in people with T1D, it could lead to a low-cost, safe and effective treatment for this life-threatening complication.