CORD BLOOD COLLECTION DEVICE

描述（由申请人提供）：虽然每年有30，000例骨髓移植试图治愈白血病，地中海贫血和镰状细胞病等疾病，但每年约有60，000名符合移植条件的患者因无法找到成功移植所需的几乎相同的供体匹配而死亡。人们对高质量脐带血作为移植用造血祖细胞来源的需求日益增长。然而，没有专门的设备可以安全，快速和方便地收集脐带血。NHLBI先前授予SBIR第一阶段和第二阶段赠款给法尔布鲁克工程公司。和CHORI（奥克兰研究所儿童医院）开发了一个独立的单元，该单元围绕脐带闭合，以安全的方式切割脐带，并将脐带血收集在含有抗凝剂的容器中。该申请寻求额外的资金，以继续评估和改进脐带血采集设备的过程，并进行FDA要求的临床前研究，作为销售该设备的510（k）上市许可申请的一部分。我们的假设是，我们的新设备将收集与传统系统相同或更多的脐带血，而不会使医护人员受到针刺伤害。我们的建议包括四个具体目标：1）。完成脐带血采集装置原型的设计; 2）在三个研究中心进行临床前试验; 3）申请FDA对我们的装置（UCB 1000）的监管批准;以及4）成立Cord Cell Technologies，Inc，并获得UCB装置生产和营销的融资。临床前试验和产品评价将在三个试验中心进行，这些试验中心将遵循相同的程序和方案：Reinas脐带血库，杜克大学，达勒姆，NC; MD安德森癌症中心，休斯顿，TX;和阿尔塔贝茨峰会医疗中心，伯克利，CA。我们将在每个研究中心采集50份脐带血样本，并运送至CHORI实验室。除了测量采集的总血量和评价每个单元的无菌性外，我们还将在血液采集、处理、冷冻和解冻后进行细胞计数、NRBC差异、活力（台盼蓝）、CD 3和CD 34流式细胞术以及CFU测定。将收集定性和主观数据，以评价医疗保健专业人员使用UCB 1000的易用性。我们在这些试验中成功的五个标准是：1）与传统方法相比，脐带血收集量相等或更好; 2）细胞活力水平相等或更好; 3）无菌水平相等或更好; 4）操作安全，不会使人员暴露于穿透皮肤的锋利边缘; 5）快速轻松地收集，不会对产科实践产生重大干扰。UCB 1000脐带血采集器将大大降低采集脐带血所需的专业知识水平，消除血液飞溅和针刺的风险，同时增加采集的无污染和体积充足的脐带血单位的百分比。我们相信这款设备将成为行业标准。由于没有足够的脐带血干细胞库存来确保及时为所有移植患者提供高质量的匹配，今天许多患者在等待匹配时死亡。需要改进安全有效收集脐带血的方法，以确保有足够的干细胞库存可供儿童和成人移植。本授权申请中描述的产品易于使用，将增加采集的单位数量，并最大限度地提高每次采集的脐带血体积，同时保护医护人员在采集过程中免受针刺和任何污染血液的影响。

DESCRIPTION (provided by applicant): Although 30,000 bone marrow transplants occur each year in attempts to cure diseases such as leukemia, thalassemia and sickle cell disease, approximately 60,000 patients eligible for transplant die every year without finding the nearly identical donor match required for a successful transplant. There is growing demand for high quality umbilical cord blood as a source of hematopoietic progenitor cells for transplantation. No dedicated device, however, exists to safely, quickly, and conveniently collect cord blood. NHLBI previously awarded SBIR phase I and II grants to Fallbrook Engineering, Inc. and CHORI (Children's Hospital, Oakland Research Institute) to develop a self-contained unit that closes around the umbilical cord, cuts the cord in a safe manner, and collects the cord blood in an anticoagulant-containing vessel. This application seeks additional funding to continue the process of assessing and improving the cord blood collection device and to conduct preclinical studies required by the FDA as part of a 510(k) application for marketing clearance to sell the device. Our hypothesis is that our new device will collect the same or greater volume of cord blood than the conventional systems without exposing the healthcare workers to needle-stick injuries. Our proposal consists of four specific aims: 1). Finalize Design of the Prototype Cord Blood Collection Device; 2) Conduct Pre- Clinical testing at three sites; 3) Request FDA Regulatory Approval for our device (the UCB 1000); and 4) Form Cord Cell Technologies, Inc, and obtain financing for production and marketing of the UCB devices. Pre-clinical testing and product evaluations will be conducted at three test sites that will follow the same procedures and protocols: Carolinas Cord Blood Bank, Duke University, Durham, NC; MD Anderson Cancer Center, Houston, TX; and Alta Bates Summit Medical Center, Berkeley, CA. We will collect 50 samples of cord blood at each site and ship to the CHORI laboratory. In addition to measuring total collected blood volume and evaluating sterility for each unit, we will perform cell counts, diff, with NRBC, Viability (Trypan blue), Flow cytometry for CD3 and CD34 and CFU assays following the collection, processing, freezing and thawing of the blood. Qualitative and subjective data will be collected to evaluate the ease of use of UCB 1000 by health care professionals. Our five criteria of success in these trials are 1) equivalent or better collection of cord blood volume compared to conventional methods; 2) equivalent or better level of cell viability; 3) equivalent or better level of sterility; 4) safe operation without exposure of personnel to sharp edges that penetrate the skin; and 5) rapid and easy collection without significant interference with obstetric practice. The UCB 1000 Cord Blood Collection Device will substantially reduce the level of expertise required to collect cord blood, eliminate the risk of blood splashing and needle sticks while increasing the percentage of cord blood units collected that are free of contamination and sufficient in volume. We believe this device will become the industry standard. Because there are not sufficient inventories of cord blood stem cells to assure a quality match for all transplant patients in a timely manner, many patients today die while waiting for a match. Improved methods for the safe and effective collection of cord blood are needed to ensure that adequate inventories of stem cells are available for transplantation in children and adults. The product described in this grant application is easy to use and will both increase the number of units collected and maximize the cord blood volume per collection while protecting healthcare workers from exposure to needle sticks and any contaminated blood during the collection process.