Oct. 9, 2009
Contact: Julie O’Connor, julie.oconnor@wayne.edu
Joe Serwach, jserwach@umich.edu

URC garners $12.5 million in NIH research consortiums

The National Institutes of Health announced this week a second phase of the Rare Diseases Clinical Research Network totaling $117 million, including support for 19 research consortia including Michigan’s University Research Corridor.

Two URC)partners, the University of Michigan and Wayne State University, received $12.5 million to lead two of these international studies.

University of Michigan researchers received $6.25 million from the National Institute of Diabetes, Digestive and Kidney Diseases (NIDDKD) to study a rare kidney disease, nephrotic syndrome, that leads to kidney failure and the need for dialysis. The syndrome, which affects kidney filter cells, contributes to nearly 12 percent of cases of kidney failure, and an annual cost of more than $3 billion.

The Nephrotic Syndrome Rare Disease Consortium, led by Matthias Kretzler, M.D., professor of internal medicine at the U-M Medical School and nephrologist at the U-M Health System, will bring together a network of investigators from the United States and Canada. The team also includes two highly active patient interest groups, the Nephcure Foundation and the Halpin Foundation, to create a network for collecting patient information, facilitating clinical and observational studies and training future researchers.

In addition to the NIH award, NephCure committed $2 million and U-M contributed a total of $2 million.

Kretzler, along with U-M medical school researchers Akinlolu Ojo, M.D., professor in internal medicine, and Lawrence B. Holzman, M.D., professor of internal medicine, will provide the leadership for the multi-center consortium.

Wayne State University received $6.25 million from the National Institute of Neurological Disorders and Stroke to develop the Inherited Neuropathies Consortium, an international project aimed at developing a better understanding of and new treatments for the various forms of Charcot-Marie-Tooth disease.

Charcot-Marie-Tooth disease (CMT) is one of the most common genetic nerve diseases, affecting 1 in 2,500 people. CMT causes progressive muscle weakness, painful foot deformities and walking difficulty. As the disease progresses, weakness and muscle atrophy occur in the hands, resulting in difficulty with fine motor skills. To date, no effective therapies are available for any form of CMT. Wayne State University has one of the largest and most comprehensive CMT programs in the world.

Michael Shy, M.D., professor of neurology in the School of Medicine, is leading the study to provide insights into disease mechanisms, develop therapies and educate future research on inherited neuropathies such as CMT.

He is joined by Gyula Acsadi, associate professor of neurology at WSU and director of the Pediatric MDA Clinic and Pediatric Neurology Clinical Division Chief at Children’s Hospital of Michigan; the University of Pennsylvania; the National Hospital for neurology and neurosurgery and Newcastle University in London, England; the University College of London; University of Miami; and University of Rochester.

“These studies will give great hope to patients with these debilitating diseases,” said Hilary Ratner, vice president for research at Wayne State University. “This critical support from the NIH and others will put us closer to the development of new targeted treatment options.”

“As a premier research institution, we’re well-positioned to take advantage of a rapidly expanding knowledge of genetics and the power of bioinformatics to transform our understanding and treatment of rare diseases,” said James A. Shayman, M.D., associate vice president for research at the University of Michigan.