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NephCure in Action
On the Science Front
Supported by a Scientific Advisory Board ( SAB) comprised of some of the most knowledgeable experts in Nephrotic Syndrome and FSGS, the NephCure Foundation and its founders have provided ongoing support to numerous individuals and groups working to find the cause and cure of those conditions. Below are direct activities of the foundation or of its founders.
Meetings and Conferences
The Bethesda Symposium -- In January 2005, NephCure joined the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) in sponsoring a major conference for more than 100 scientists studying glomerular diseases such as NS and FSGS. The experts discussed developments in the area of genetic research, emerging treatment trials, and new information about podocyte, a cell that seems to have a key role in the diseases.
The Podocyte Conference – Researchers from around the globe meet annually to share findings about the podocyte, whose structure and shape are linked to NS and FSGS. These meetings are held in the U.S. and abroad and NephCure has provided financial support for several of them.
American Society of Pediatric Nephrologists – Because so many NS and FSGS patients are youngsters, NephCure has provided support for the annual meeting of the ASPN, whose members treat our patients. ASPN officials have conducted programs dealing with our diseases and an ASPN past president, Frederick Kaskel, M.D., who is a principal investigator in the national FSGS Clinical Trial, serves on the NephCure Scientific Advisory Board.
Individual Grants -- Dr. Friedhelm Hildebrandt of the University of Michigan is one of the leading geneticists in the FSGS field. He has discovered genetic mutations which account for some portions of the FSGS pool. He currently is in the third year of a five-year funding program.
Dr. Marilyn Farquahr of the University of California, San Diego -- and one of the early pioneers in FSGS research -- has been granted a two-year fellowship to fund a post-doctoral researcher in her lab.
Funding for Ancillary Studies -- NephCure is participating in a matching grant program with NIDDK, for research ancillary to the national FSGS Clinical Trial. The Clinical Trial is studying the effect of several treatments on FSGS patients throughout the nation. NephCure and NIDDK have set aside these funds to be used for basic research using the biological materials collected during the trial.
Endowed University Chair -- The Robert C. Kelsch Collegiate Chair in Pediatric Nephrology at the University of Michigan was endowed with major support from a NephCure Founder. The Head of the Section of Pediatric Nephrology at Michigan traditionally holds the Chair. All proceeds from the Chair’s endowment are earmarked for basic research into the molecular mechanisms of FSGS and Minimal Change. Last year, funds were allocated to Richard Ransom, Ph.D. and William Smoyer, M.D. for work on the proteonomics of the glomerulus.
NephCure Advocacy in Action -- After the Bethesda symposium mentioned above, NephCure officials conducted numerous meetings with federal officials to encourage creation of an NIDDK “Funding Opportunity Announcement,” a first step in funding new research into glomerular disease. That announcement came in March of this year. Researchers are now being invited to apply for support ($275,000 for two years) for new research programs. The goal of this new source of funding is to identify the brightest new research programs that will then attract substantial funding in subsequent years.
A “Biobank” for NS and FSGS
There are 1 million filters (nephrons) in each human kidney and until recently scientists were forced to guess exactly how these amazingly small filters were constructed and worked. Equipped with modern technology, researchers are striving to understand the molecular structure of both health and diseased kidneys.
NephCure is planning to support a gifted researcher at the University of Michigan who will begin creating a “Registry” for Nephrotic Syndrome and FSGS. Essentially a collection of biopsy tissues and specimens from patients with the disease, along with their histories, this registry will be used by researchers to develop a system of markers which can subdivide different forms of FSGS into categories. Such classification may provide finer details as to prognosis, responsiveness to various drugs, and why some patients respond to treatments (such as steroids) and others do not. NephCure will provide funding and promote the collection effort.
An Epidemiological Study and Database for NS and FSGS
There never has been a nationwide effort to collect data from all idiopathic NS and FSGS patients to allow for a study of possible risk factors related to the disease. NephCure has been meeting with well-regarded researchers to discuss a project that will both collect this data and organize it into a web-based database available to researchers and the general public. Because of its credibility with the patient population and medical community, NephCure is perfectly positioned to fund and promote such a project. Major diseases have benefited greatly from large-scale epidemiological studies, but none have been conducted for NS and FSGS. Such a project will provide the tools that could lead to scientific breakthroughs. This project will complement the Biobank
Web-Based Updates on Important NS/FSGS Research
Both the scientific community and the lay public are clamoring for more information on these diseases and the NephCure staff fields frequent calls from families wanting to know “what’s the latest in science?” NephCure has contracted with a respected nephrologist and a postdoctoral Fellow at Harvard University, who will scout the scientific literature and provide us with quarterly reports that will be found on the nephcure.org web site.
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