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Joslin receives $24.3 million grant from the NIH for diabetic kidney disease study

Joslin Diabetes Center


IMAGE: Allessandro Doria, M.D., Ph.D., M.P.H., is Investigator in the Section on Genetics & Epidemiology at Joslin Diabetes Center and Associate Professor of Medicine at Harvard Medical School. view more

Credit: John Soares

BOSTON - (September 26, 2013) The National Institutes of Health awarded Joslin Diabetes Center $24.3 million to fund a clinical trial to study a potential treatment for kidney disease in people with type 1 diabetes. Alessandro Doria, M.D., Ph.D., M.P.H., an Investigator in the Section on Genetics and Epidemiology, Associate Professor of Medicine at Harvard Medical School and Associate Professor in the Department of Epidemiology at the Harvard School of Public Health, will lead the trial together with his co- Principal Investigator, Michael Mauer, M.D., Professor of Pediatrics and Medicine at the University of Minnesota Medical School.

The five-year trial will evaluate the possible benefits of allopurinol, an FDA approved uric acid lowering drug, in reducing kidney function loss among people with type 1 diabetes. The study stems from findings from Joslin and other investigators linking higher levels of serum uric acid to the risk of kidney complications in diabetes.

The research will be conducted at institutions in the Preventing Early Renal Function Loss in Diabetes (PERL) Consortium, composed of Joslin Diabetes Center, University of Minnesota, the Universities of Colorado, Toronto, and Michigan, Northwestern University, Albert Einstein College of Medicine and the Steno Diabetes Center in Denmark. The funding will be allocated to Joslin, who will then distribute funding to the additional seven clinical centers, plus a data center in Michigan and a central lab in Minnesota.

"We are doing this study to see if we can slow down the decline of kidney function by decreasing uric acid," commented Dr. Doria. "We have data indicating that moderately high serum uric acid levels predispose to diabetic kidney disease. However, we don't know whether this is due to uric acid itself or to something else that goes together with it. That's why this study is important - to determine if uric acid is the culprit or not."

Smaller studies have suggested benefit in slowing kidney function decline in patients with chronic kidney disease, but PERL is positioned to provide a much more definitive answer to this important question.

Kidney disease poses one of the greatest burdens for people with type 1 diabetes, with 10 to 15 percent of patients developing end stage renal disease (ESRD), meaning that they need hemodialysis or a renal transplant to survive. Diabetes is the leading cause of ESRD, and the number of people with diabetes, including type 1 and type 2, and kidney failure rose by 61 percent between 2000 and 2010. Currently, tight control of blood sugar and blood pressure are the only prevention tools. However, as many a 50 percent of people with diabetes cannot achieve optimal blood sugar control and blood pressure control is only partially effective.

Dr. Mauer commented that "since persons with diabetes represent more than 45 percent of the more than 115,000 new ESRD cases in the USA per year, and since the incidence of new cases of diabetes continues to grow, this study has large human suffering, public health and health cost implications."

"The aim of this study is to find a new way of preventing kidney complications in people with diabetes," Dr. Doria said.

This study will include approximately 500 patients with type 1 diabetes who are at increased risk of losing kidney function. Half the patients will take allopurinol and half will be randomly assigned to placebo for three years.

George King, M.D., Chief Scientific Officer, Director of Research at Joslin, Head of the Section on Vascular Cell Biology at Joslin and Professor of Medicine at Harvard Medical School, characterizes this study as the epitome of translational research in action.

"This shows how well positioned we are to translate what we are finding from the laboratory to the patients, and this exemplifies what we do best," said Dr. King. "This is a joint work of our basic, clinical research and the clinic, in bringing this research to bear, which I think is incredibly exciting."

Prior to being awarded this grant, Drs. Doria and Mauer together with Peter Rossing, M.D., head of research at the Steno Diabetes Center and Affiliated Professor at Aarhus University, Denmark conducted a small pilot of this study with the support of JDRF. From this, they have already established the infrastructure and procedures, which allows them to start recruiting patients for the study as early as in October.

"Diabetic nephropathy is a devastating complication of type 1 diabetes and new therapies are urgently needed to prevent progression to kidney failure," said Helen Nickerson, Ph.D., JDRF's Senior Scientific Program Manager, Complications. "This study, initially supported by JDRF, and now funded for a pivotal study using funds from the Special Diabetes Program, will test an existing therapy, allopurinol, to target those individuals most at risk for progression."

If Dr. Doria and the rest of the PERL consortium can demonstrate that allopurinol is capable of halting or slowing down loss of kidney function in people with type 1 diabetes, there will be a generic and safe medication to prevent or delay kidney failure by intervening at the early stages of kidney disease.

"If we see a robust effect [from this study], that will become a standard way of treating diabetic kidney complications," said Dr. Doria. "Also, given that allopurinol is relatively inexpensive and safe, perhaps many more people with diabetes could be treated with it, even if they are not at high risk for kidney complications. It will make a huge difference in the array of tools we have to prevent ESRD."

The findings from this study - both from perspective of public health and that of persons with diabetes - will be significant for better understanding and improving the health of people with diabetes and kidney complications.

"If this study is successful, we will have a new therapy for kidney complications," said Dr. Doria.


About Joslin Diabetes Center

Joslin Diabetes Center, based in Boston, Massachusetts, undertakes diabetes research, clinical care, education and health and wellness programs on a global scale. Joslin is dedicated to ensuring that people with diabetes live long, healthy lives and offers real progress in preventing and curing diabetes . Joslin is an independent, nonprofit institution affiliated with Harvard Medical School, and is recognized worldwide for driving innovative solutions in diabetes prevention, research, education, and care.

Our mission is to prevent, treat and cure diabetes. Our vision is a world free of diabetes and its complications. For more information, visit

About Joslin Research

Joslin Research comprises the most comprehensive and productive effort in diabetes research under one roof anywhere in the world. With 30 plus faculty level investigators, Joslin researchers focus on unraveling the biological, biochemical and genetic processes that underlie the development of type 1 and type 2 diabetes and related complications.

Joslin research is highly innovative and imaginative, employing the newest tools in genetics, genomics and proteomics to identify abnormalities that may play a role in the development of diabetes and its complications. Joslin Clinic patients, and others with diabetes, have the option of participating in clinical trials at Joslin to help translate basic research into treatment innovations.

Joslin has one of the largest diabetes training programs in the world, educating 150 M.D. and Ph.D. researchers each year, many of whom go on to head diabetes initiatives at leading institutions all over the globe. For more information, visit

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