Pilot and Feasibility Program

一个强有力的试点和可行性计划将是联合会的一个组成部分， 肾结石和肾衰竭的遗传原因。罕见疾病联盟 将作为肾结石研究和病人护理新想法的活跃孵化器。 计划是在此奖项的持续时间内始终资助2个项目。为此目的，每年编列预算10万美元，使每个试点项目约有5万美元。两个试点项目中的一个将由马约诊所罗切斯特研究委员会承诺的资金资助。主要重点将是支持新的协议，这些协议具有在我们联盟中的一个或多个患者队列中推进患者护理的高潜力。初级研究人员的建议也将得到特别考虑，尽管该计划也将向已建立的研究人员开放，以鼓励他们在该领域开发新的项目或研究途径。具有产生初步数据的高潜力的项目，使其有竞争力获得额外的校外资金，也将得到更高的优先考虑。 在第1-2年，我们将资助一个试点项目，开发从移植时切除的肝脏中收获活肝细胞的技术。该项目与项目1的目标一致，并将为PH的新模型系统和潜在疗法打开大门。第二个试点项目将由马约诊所研究委员会资助（承诺5年每年50，000美元）。这些资金扩大了 罕见疾病联盟的整体资源和范围。所有提案都将由罕见疾病联盟试点项目委员会进行严格的筛选。已经确定了未来试点项目的几个可能候选项目。但是，我们有意选择在提交时不选择此附加提案。相反，我们将发布一份新的RFA，并在潜在的赠款资助时选择最有希望的提案。这样，我们将保持灵活性，不将财团锁定在特定领域，而是允许新领域的可能性， 支持在未来12到18个月内出现。

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.