CLINICAL TRANSLATION CORE

临床翻译核心

• 批准号: 10427280
• 负责人: Sandesh Chakravarthy Sreenath Nagamani
• 金额: $ 14.11万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-07-22 至 2025-05-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10427280
• 关键词: Academia; Animal Model; Award; Basic Science; Bioinformatics; Biological; Biological Markers; Biological Specimen Banks; Clinic; Clinical; Clinical Investigator; Clinical Research; Clinical Trials; Collaborations; Collection; Coupled; Data; Databases; Development; Diagnosis; Diagnostic; Disease; Drug Industry; E-learning; Environment; Fostering; Funding; Future; Genetic; Genomics; Good Clinical Practice; Grant; Human; Human Subject Research; Infrastructure; Intellectual and Developmental Disabilities Research Centers; Intellectual functioning disability; International; Internet; Laboratories; Medicine; Mentorship; Modality; Molecular; Morbidity - disease rate; Natural History; Nervous system structure; Neurodevelopmental Disorder; Outcome; Outcome Measure; Patient Care; Patients; Phenotype; Positioning Attribute; Process; Proteomics; Publications; Publishing; Rare Diseases; Research; Research Activity; Research Design; Research Infrastructure; Research Personnel; Resources; Sampling; Scholarship; Scientist; Services; Structure; System; Techniques; Therapeutic Intervention; Training; Translating; Translational Research; Translations; United States; United States National Institutes of Health; Validation; Vision; Work; base; bench to bedside; clinical outcome assessment; clinical translation; clinical trial readiness; college; comparative efficacy; design; driving force; efficacy study; first-in-human; insight; lectures; metabolomics; next generation; novel; novel diagnostics; novel therapeutics; patient biomarkers; patient registry; pre-clinical; success; therapy development; transcriptomics; translational scientist; translational study; trial design; webinar

In its current form, the Baylor College of Medicine (BCM) IDDRC’s Clinical Translational Core (CTC) has provided an outstanding platform to integrate basic science researchers, clinical investigators, and clinicians with infrastructure and resources to enable the translation of many mechanistic discoveries from our center into the clinical realm. In this proposal, we are excited to build upon the success of our CTC and expand on the services using a new administrative and novel core structure that will allow us to leverage our strengths for developing therapies for IDD. The new structure and services were born from our “evolving Core theme” in order to accommodate and translate the new and exciting discoveries from our center investigators. The CTC will be composed of three units: Study Design and Implementation Unit, Biospecimen and Data Unit, and Center Navigation Unit that will collaborate closely with other cores of the BCM IDDRC, national and international networks, patient registries, patients, and stakeholders to facilitate all aspects of transitional research in IDD. A vision that we have for our CTC is that it should have an impact beyond our local center and that our processes and activities should be of use to other centers and IDD researchers across the United States. In line with that vision, the CTC will establish a Clinical Outcome Assessment Panel that will leverage the significant preclinical and clinical expertise at our center to develop and validate endpoints for clinical trials which would be critical for clinical trial readiness. Additionally, the CTC will work towards having a biospecimen repository from which precious samples can be shared with other centers and researchers. Finally, as the future of IDD research is dependent on young clinicians and scientists, the CTC will focus on training the next- generation of clinical and translational researchers in IDD. We strongly believe that the CTC will be the nidus by which: 1) basic researchers will have access to clinical information and human samples and thus be integrated into the clinical enterprise, 2) basic research questions will be validated in a clinical arena, and 3) newer diagnostic modalities and therapies will be fostered. The structure and services of the CTC will not only facilitate the translation of findings from the “bench-to-the- bedside”, but also drive research in the laboratory based on phenotypic observations made in the clinics. Such bidirectional translation will not only advance the basic science research but also make a significant impact on the care of the patients with IDD.

目前，贝勒医学院 (BCM) IDDRC 的临床转化核心 (CTC) 已 提供了一个将基础科学研究人员、临床研究人员和临床医生整合在一起的杰出平台 基础设施和资源，使我们中心的许多机械发现能够转化为现实 临床领域。在本提案中，我们很高兴能够在 CTC 的成功基础上再接再厉并扩展服务 使用新的管理和新颖的核心结构，使我们能够利用我们的优势来发展 IDD 的治疗方法。新的结构和服务源于我们的“不断发展的核心主题”，以便 容纳并翻译我们中心研究人员的新的、令人兴奋的发现。 CTC 将由三个单位组成：研究设计和实施单位、生物样本和数据单位 单元和中心导航单元将与 BCM IDDRC、国家和地区的其他核心密切合作 国际网络、患者登记处、患者和利益相关者，以促进过渡的各个方面 IDD 领域的研究。我们对 CTC 的愿景是它应该产生超出我们当地中心的影响， 我们的流程和活动应该对美国各地的其他中心和 IDD 研究人员有用。 根据这一愿景，CTC 将建立一个临床结果评估小组，该小组将利用 我们中心拥有重要的临床前和临床专业知识，可开发和验证临床试验的终点， 对于临床试验准备工作至关重要。此外，CTC 将致力于拥有生物样本 可以与其他中心和研究人员共享珍贵样本的存储库。最后，作为未来 IDD 研究的一部分依赖于年轻的临床医生和科学家，CTC 将重点培训下一代—— IDD 领域的临床和转化研究人员的一代。 我们坚信，CTC 将成为一个核心：1）基础研究人员将能够获得 临床信息和人体样本从而整合到临床事业中，2）基础研究 问题将在临床领域得到验证，并且3）将培育更新的诊断方式和疗法。 CTC 的结构和服务不仅将促进“从基准到实际”的研究结果的转化 床边”，而且还根据诊所的表型观察推动实验室研究。 双向翻译不仅会推进基础科学研究，而且会对 IDD 患者的护理。