Pilot Demonstration

试点/示范项目 CREATE RDCRC试点/示范研究计划的主要目的是通过一个小型研究资助计划促进ALS和相关疾病的生物标记物的发现和验证，该计划在开发的早期阶段资助潜在生物标记物的研究。优先考虑具有最有前途的临床研究潜力的项目。Create Consortium的活动--对家族性和散发性疾病患者的纵向评估、深层表型数据的收集以及建立已在临床上详细注解的生物样本(血液、尿液、脑脊液)的存储库--都有望通过试点/示范研究计划加强和促进生物标志物的发现。第一个试点/示范项目将侧重于ALS脑脊液生物标记物的早期开发。在接下来的几年中，潜在的项目包括开发一种反映疾病进展的尿液生物标志物，以及一种反映皮质抑制和兴奋性之间失衡的神经成像标志物。CREATE RDCRC将利用每年一次的“申请申请”，征集申请，并选择最有希望获得资金的项目。

PILOT/DEMONSTRATION PROJECTS The primary purpose of the CREATE RDCRC pilot/demonstration research program is to promote biomarker discovery and validation for ALS and related disorders through a small research grant program that funds studies of potential biomarkers during their early stages of development. Priority is given to projects with the most promising clinical research potential. The activities of the CREATE Consortium - longitudinal evaluation of patients with familial and sporadic disease, collection of deep phenotypic data, and the establishment of a repository of biological samples (blood, urine, cerebrospinal fluid) that has been annotated clinically in a detailed manner - are all expected to enhance and facilitate biomarker discovery through the pilot/ demonstration research program. The first pilot/demonstration project will focus on early development of a cerebrospinal fluid biomarker for ALS. Potential projects in subsequent years include development of a urinary biomarker of disease progression and a neuroimaging marker of the imbalance between cortical inhibition and excitability. The CREATE RDCRC will utilize a "request for applications" on an annual basis, to solicit applications and to select the most promising project for funding.