Pilot and Demonstration Project Program

试点示范项目计划

基本信息

批准号：
8920715
负责人：
Bruce C Trapnell
金额：
$ 0.95万
依托单位：
CINCINNATI CHILDRENS HOSP MED CTR
依托单位国家：
美国
项目类别：
财政年份：
资助国家：
美国
起止时间：
至
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/8920715
关键词：
Academic Medical Centers Affect Alveolar Area Basic Science Birt-Hogg-Dube Syndrome Breathing Bronchoalveolar Lavage Clinical Clinical Data Clinical Research Cross-Sectional Studies Crystal Formation Data Densitometry Development Diagnostic Diagnostic Procedure Diagnostic tests Disease Foundations Funding Funding Opportunities Future Goals Grant Granulocyte-Macrophage Colony-Stimulating Factor Hermanski-Pudlak Syndrome Individual Interstitial Lung Diseases Interstitial Pneumonia Lead Lung Lung diseases Lymphangioleiomyomatosis Medical center Medicine Molecular Natural History Outcome Measure Pathogenesis Pathway interactions Patients Pediatric Hospitals Physicians Pilot Projects Process Pulmonary Alveolar Proteinosis Rare Diseases Research Research Personnel Research Project Grants Research Training Sampling Scientist Severity of illness Site Techniques Testing Therapeutic Training Translations Treatment Efficacy Trypsin alpha 1-Antitrypsin Deficiency bisphosphonate calcium phosphate cohort design improved interest method development mutation carrier novel novel diagnostics novel therapeutics patient population programs success therapeutic effectiveness therapeutic target therapy outcome tool treatment trial

项目摘要

The translation of hypothesis-driven basic research into clinically useful tests and therapies for patients is challenging, particularly for individuals with rare diseases, because of limited numbers of patients who are geographically widely distributed throughout the US and the world, physicians who rarely see more than one or two patients with rare diseases and have limited training in basic science research and clinical research, and basic science researchers have limited access to patient samples and clinical data. Additionally, the early development of methods and techniques to understand the disease pathogenesis, evaluate potential therapeutic targets, and advance clinical techniques to improve outcome measures for patients are not well-funded by traditional grant mechanisms available to academic medicine. Pilot/demonstration programs are the ideal opportunity to integrate patients and patient foundations, physicians, and scientists to make scientific advancements for individuals with rare diseases. Research in the rare lung diseases, Lymphangioleiomyomatosis (LAM) and Pulmonary Alveolar Proteinosis (PAP), has significantly benefitted from pilot program opportunities at Cincinnati Children's Hospital Medical Center (CCHMC), University of Cincinnati Medical Center (UCMC) and previously via the creation of the Rare Lung Disease Consortium (RLDC), part of the Rare Disease Clinical Research Network from 2003 through 2008. The new RLDC Pilot Program plans to build on these successes with LAM and PAP to continue to fund pilot/demonstration projects in LAM and PAP, as well as fund studies in Familial Interstitial Pneumonia (FIP), Pulmonary Alveolar Microlithiasis (PAM), Hermansky-Pudlak Syndrome (HPS), Alpha-1 anti-trypsin deficiency (A1AT) and additional rare lung diseases. We have solicited pilot proposals in the areas of FIP, PAM, PAP, that propose to perform a cross-sectional study of TERT mutation carriers in a cohort of patients with PIP, proof of concept treatment study with Etidronate for PAM, and a quantitative CT densitometry study to evaluate therapeutic efficacy for PAP. Additional preliminary concept are also included as potential projects for future years.

将假说驱动的基础研究转化为临床有用的测试和患者的疗法是具有挑战性的，特别是对于罕见疾病的人，由于地理位置上广泛分布在美国和世界上的有限的患者，他们很少看到一个或两种罕见的患者患有稀有疾病，并且在基础科学研究和临床科学研究的培训中很少有罕见的疾病培训，并且对基础科学研究的培训有限。此外，方法和技术的早期开发以理解疾病发病机理，评估潜在的治疗靶标以及进步的临床技术以改善患者的结果指标，这并不是由可用于学术医学的传统赠款机制充分资金。试点/示范计划是整合患者和患者基金会，医生和科学家的理想机会，以对罕见疾病的个体进行科学进步。罕见肺部疾病，淋巴管肌瘤病（LAM）和肺肺泡蛋白质病（PAP）的研究显着受益于辛辛那提儿童医院医疗中心（CCHMC）的试点计划机会，辛辛那提大学医学中心（UCMC）以及以前通过稀有的Lung Sytrim Consort（rld Cansort）（rldc）（RLDC）（RLDC）（rldc）（rld）（rld）（rld）（rld）（rld）（rld）（rlde）（Rld Cance） 2008年。新的RLDC飞行员计划计划以Lam和pap的成功为基础，以继续为LAM和PAP中的飞行员/示范项目提供资金，以及在家族性间质性肺炎（FIP）的基金研究中，肺肺泡微石质症（PAM），Hermansky-Pudlak-pudlak syndrome（HPS），Alpha-1 Antpha-andprysise（hermansky-pudlak andprysion）（hermansky-pudlak syprys）（PAM）。我们已经在FIP，PAM，PAP领域征求了试点建议，该提案建议在一组PIP患者中对TERT突变携带者进行横断面研究，PAM的概念治疗研究证明PAM的概念治疗研究和定量的CT CT密度仪研究以评估PAP的治疗效率。其他初步概念也被包括在未来几年的潜在项目中。