Pilot and Exploratory Science Core

试点和探索性研究核心-摘要 在OAIC的总体使命背景下，试点和探索性研究核心（PESC）旨在 为创新的试验研究项目提供催化支持-种子资金，核心支持和指导 产生FPT作用机制的数据，以促进更明确的机制研究， 指导疗效试验、假设生成或概念验证的探索性研究和回顾性研究的数据 分析为FPT干预提供信息的现有流行病学数据。 该方案的目标是： 目标1.为早期创新和跨学科研究建立翻译管道， 资金和基础设施： ·生成概念验证临床前数据，以加快FPT的开发。 ·目标识别的机制研究或FPT发挥作用的假设建立 骨骼肌和身体功能（例如，PES-1）。 ·在人类受试者或动物模型中进行转化干预或研究方案的试点测试， 安全性、可行性或最佳时程或剂量的确定（例如，PES-2、PES-3）。 目标2：建立PESC调查人员与OAIC核心资源之间的联系。 目标3：每半年监测一次，并指导PESC研究人员的研究项目取得成功。 目标4。促进试点和探索性项目成为高质量的同行评审出版物， 知识产权/专利，只要有可能。 我们从11项申请中选择了3项创新研究作为PESC的支持，因为这些研究 这些研究都与海外投资管理局的使命一致，即促进FPT的发展： PES-1将表征运动中的microRNA，并作为老年人合成代谢反应的生物标志物。 PES-2将评估家庭锻炼计划是否可以改善患者的功能结局 接受经导管主动脉瓣置换术 PES-3将确定经颅脑刺激是否可以改善老年人的身体功能。 在最初的5年资助期间，波士顿OAIC支持了11个PES项目，从1年到2.5年。75万美元的 OAIC的投资产生了1795万美元的校外资金，两项专利和75篇同行评审的出版物 包括《衰老细胞》（Guo 2013，Jasuja 2014）中的论文，一本关于医学转化生物学的书（Montano 2014，Elsevier Publishers）、PNAS（Hettmer 2011）、Circulation（Jefferson 2010）和NEJM（Basaria 2012）。 该PESC将由两名具有互补专业知识和指导技能的科学家领导;蒙蒂·蒙塔诺博士， 一位在肌肉和衰老方面具有专业知识的翻译科学家;和，道格拉斯基尔博士，一位临床研究者， 在肌肉骨骼衰老和功能促进疗法的干预试验方面的专业知识。

PILOT AND EXPLORATORY STUDIES CORE - SUMMARY Within the context of the OAIC's overall mission, the Pilot and Exploratory Studies Core (PESC) aims to provide catalytic support – seed funding, core support, and mentorship - for innovative pilot research projects that generate data on the mechanisms of FPT action to facilitate more definitive mechanistic studies, feasibility data to guide efficacy trials, hypothesis generating or proof-of-concept exploratory studies and retrospective analysis of existing epidemiologic data that inform FPT interventions. The aims of this PESC are: Aim 1. To build a translational pipeline for early stage innovative and interdisciplinary research by providing funding and infrastructure for: • Generation of proof-of-concept preclinical data to expedite the development of FPTs. • Mechanistic studies for target identification or hypotheses building by which FPTs exert their effects on skeletal muscle and physical function (e.g., PES-1). • Pilot testing of translational interventions or research protocols in human subjects or animal models for safety, feasibility, or determination of optimal time course or dosage (e.g., PES-2, PES-3). Aim 2: To build connectivity between PESC investigators and OAIC core resources. Aim 3: To monitor biannually and guide success of research projects by PESC investigators. Aim 4. To catalyze pilot and exploratory projects into high quality peer-reviewed publications, grant applications, and intellectual property/ patents, whenever possible. We selected 3 innovative studies for PESC support, from among a pool of 11 applications, because these studies are well aligned with the OAIC's mission of promoting the development of FPTs: PES-1 will characterize microRNAs in exercise and as biomarkers for anabolic response in aged adults. PES-2 will evaluate whether a home-based exercise program can improve functional outcomes in patients undergoing trans-catheter aortic valve replacement. PES-3 willl determine if transcranial brain stimulation can improve physical function in older adults. During its initial 5 years of funding, Boston OAIC supported 11 PES projects from 1 to 2.5 years. A $750,000 OAIC investment yielded $17.95 million in extramural funding, two patents, and 75 peer reviewed publications including papers in Aging Cell (Guo 2013, Jasuja 2014), a book on translational biology in medicine (Montano 2014, Elsevier Publishers), PNAS (Hettmer 2011), Circulation (Jefferson 2010) and NEJM (Basaria 2012). This PESC will be led by two scientists with complementary expertise and mentoring skills; Dr. Monty Montano, a translational scientist with expertise in muscle and aging; and, Dr. Douglas Kiel, a clinical investigator with expertise in musculoskeletal decline with aging and intervention trials of function promoting therapies.