A robust Pilot and Feasibility Program will be an integral portion of the CONSORTIUM FOR HEREDITARY CAUSES OF NEPHROLITHIASIS AND KIDNEY FAILURE. The Rare Diseases Consortium will serve as an active incubator for novel ideas in nephrolithiasis research and patient care. Plans are to fund 2 projects at all times for the duration of this award. For this purpose, $100,000 has been budgeted each year, allowing approximately $50,000 per pilot project. One of the two pilot projects will be funded by money pledged from the Mayo Clinic Rochester research committee. The primary focus will be to support new protocols with high potential for advancing patient care amongst one or more of the patient cohorts in our consortium. Proposals from junior investigators will also be given special consideration for funding, although the program will also be open to established investigators as a means of encouraging them to develop new projects or avenues of research within this field. Projects with a high potential to produce preliminary data making them competitive for additional extramural funding will also be given higher priority. In years 1-2, we will fund a pilot project on developing technology for harvesting viable hepatocytes from livers removed at transplant. This project synchronizes well with aims of Project 1, and will open the door towards novel model systems and potential therapies for PH. The second pilot project will be funded by the Mayo Clinic Research Committee ($50,000 per year for 5 years pledged). These funds expand the overall resources and scope of the Rare Diseases Consortium. All proposals will be selected by the full Rare Diseases Consortium Pilot Project Committee, using a rigorous selection processed. Several possible candidates for future Pilot Projects have been identified. However, we have intentionally elected to not select this additional proposal at the time of submission. Instead, we will issue a new RFA and select the most promising proposal at the time of potential grant funding. In this manner, we will maintain flexibility and not lock the consortium into specific areas, but instead allow the possibility that novel areas can be supported that emerge over the next 12 to 18 months.

Public Health Relevance

The Pilot Program will fund 2 projects at all times for a period of 1-2 years each. Maximal budget is $50,000/year. Goals are to fund promising nephrolithiasis investigators and provide preliminary data in support of promising new ideas.

National Institute of Health (NIH)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Specialized Center--Cooperative Agreements (U54)
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Special Emphasis Panel (ZRG1-HOP-Y)
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Mayo Clinic, Rochester
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Whittamore, Jonathan M; Hatch, Marguerite (2017) The role of intestinal oxalate transport in hyperoxaluria and the formation of kidney stones in animals and man. Urolithiasis 45:89-108
Perinpam, Majuran; Enders, Felicity T; Mara, Kristin C et al. (2017) Plasma oxalate in relation to eGFR in patients with primary hyperoxaluria, enteric hyperoxaluria and urinary stone disease. Clin Biochem 50:1014-1019
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