Clinical Research Support Core

本提案的主题是加强项目中的目标发现/确认，并将 这些关于潜在靶点的发现包括BH 3蛋白/凋亡机制，泛素连接酶， 关键转录因子这些靶点中的一些或全部将作为潜在有用的治疗药物进行测试 通过早期临床试验。临床研究支持需要核心资源， 对在诊断/复发时获得的人白血病细胞进行这些临床试验和测定， 在治疗研究的过程中。在这个核心的人员将密切互动， 核心C中的生物统计学家负责试验设计、数据质量保证和结果分析。这一核心将是 负责获得所需的诊断和治疗后样本，并将其储存在组织中 在获得适当同意后，使用生物样本核心B中描述的银行贷款。因此，总体具体 临床研究支持核心的目标是： 1.在诊断和复发时从AML和高风险MDS患者收集细胞用于储存， 生物样本核心B中的注释，将执行关键生物学定义测定并提供细胞， 目标开发和验证项目 2.使用源自以下物质的药物进行临床试验（包括获取辅助研究的材料）： 项目中的临床前科学 为了实现这些目标，我们将： * 招募和入组合格患者（包括年龄、性别、 种族和种族群体）用于骨髓性白血病的临床试验 * 协调本申请中所述临床试验的操作。确保研究 遵循参数;确认患者登记的资格，治疗计划的依从性， HIPPA依从性、适当的剂量调整、辅助数据和标本的采集， 不良事件报告和毒性/反应评估 * 通过生物样本核心C样本采集研究样本并分发辅助样本 采购、储存和分发机制，以供联合国相关实验室的调查人员使用。 项目（在某些情况下，在爆破后不久，Core D可能会进行简单的分析 隔离（如需要） * 作为与制药公司，外部医生和医院的联络人， 收集研究样本和后续数据 * 提供中央数据管理，以收集和记录个体患者信息 相关性： 在这个项目中进行的研究将能够开发毒性更小和更具体的治疗方法 AML和高危MDS患者。我们计划将实验室的发现迅速转化为有用的 治疗

The theme of this proposal is to both enhance target discovery/confirmation in the projects and to translate such findings regarding potential targets including BH3 proteins/apoptosis machinery, ubiquitin ligases, and key transcription factors. Some or all of these targets will be tested as potentially useful therapeutic strategies by means of early phase clinical trials. Clinical Research Support Core resources are required to carry out these clinical trials and assays on human leukemia cells obtained both at diagnosis/relapse and during the course of therapeutic studies. The personnel in this core will closely interact with the biostatisticians in Core C for trial design, data quality assurance and analysis of results. This Core will be responsible for obtaining required diagnostic and post-treatment samples which will be stored in the tissue bank facility described in Biospecimen Core B after obtaining appropriate consent. Thus the overall Specific Aims of the clinical research support core are to: 1. Collect cells at diagnosis and relapse from patients with AML and higher-risk MDS for storage and annotation in Biospecimen Core B who will perform key biology-defining assays and provide cells to the projects for target development and validation 2. To conduct clinical trials (including obtaining material for ancillary studies) with agents derived from preclinical science in the projects To accomplish these aims we will: * Recruit and enroll eligible patients (including appropriate numbers of individuals across age, gender, ethnicity and racial groups) f o r t h e clinical trials in myeloid leukemias * Coordinate the operation of the clinical trials described in this application. Ensure that study parameters are followed; confirm eligibility for patient registration, treatment program adherence, HIPPA compliance, appropriate dose modification, collection of ancillary data and specimens, adverse event reporting, and toxicity/response assessment * Collect research specimens and distribute ancillary samples via the Biospecimen Core C sample acquisition, storage and distribution mechanism to investigators at the relevant laboratories in the Projects (in some cases straightforward assays may be carried out by Core D shortly after blast isolation if required) * Act as liaison with pharmaceutical companies, outside physicians and hospitals to coordinate the collection of research specimens and follow-up data * Provide central data management for collection and documentation of individual patient information RELEVANCE: The research carried out in this project will enable the development of less toxic and more specific therapies for patients with AML and high risk MDS. We plan to rapidly translate laboratory discoveries into useful therapies.