University of Michigan Health System researchers will lead a $10.25 million effort to study rare kidney diseases that cause kidney failure and the need for dialysis.
The five-year project supports research of nephrotic syndrome, which describes a group of diseases that, though rare, generates an enormous individual and societal burden. 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 will bring together a network of investigators from the United States and Canada, and 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.
The National Institutes of Health Office of Rare Diseases, the National Institutes of Diabetes, Digestive and Kidney Disease awarded a $6.25 million grant to the U-M, with NephCure committing $2 million and U-M contributing a total of $2 million.
NephCure, a Pennsylvania-based patient advocacy and research funding organization, will continue to have a role in recruiting and educating patients. NephCure has contributed more than $6 million to research over the past several years.
"The kidney community has had a strong interest in this work for a long time, but, until now, we have not had adequate funding for a coordinated effort," says project director Matthias Kretzler, M.D., professor of internal medicine at the U-M Medical School and a nephrologist at the U-M Health system.
"This has the potential to change the way we understand and treat patients with nephrotic syndrome," he says. "NephCure's support was critically important in winning this award and will be vital to our long term success."
Kretzler, along with U-M medical school researchers Akinlolu Ojo, M.D., Ph.D., professor in internal medicine, and Lawrence B. Holzman, M.D., professor of internal medicine, will provide the leadership for the multi-center consortium.
"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," says James Shayman, M.D., associate vice president for research at the University of Michigan.
"We recognize that combining our own institutional resources with those of the NIH and NephCure Foundation creates a unique opportunity for promoting rare disease research in nephrotic syndrome," he says.
A seed grant from the university's Office of the Vice President for Research Rare Disease Initiative was instrumental in securing funding for the new consortium.
While the U-M will function as the central operational hub, clinical investigators from 11 academic medical centers will make up the research team.
One of the joint initiatives will be to establish and maintain a Web site with educational and networking resources for patients, families, physicians and scientists.
Researchers will investigate minimal change disease, focal and segmental glomerulosclerosis, and membranous nephropathy, diseases known to damage kidney filter cells and can lead to kidney failure and death.
Some types of the syndrome are hereditary, but it can also manifest later in life for reasons largely unknown. The network will use molecular medicine tools to unravel the mechanism responsible for the diseases and potentially develop new targeted treatment therapies.
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