Phase II Trial Evaluating the Safety and Efficacy of an Allogeneic Stem Cell for

描述(由申请人提供)：心血管疾病(CVD)是一种重要的未得到满足的医疗需求，美国每年有100多万患者患有急性心肌梗死(AMI)。心脏病发作的流行也是进一步心血管疾病的风险因素，美国每年有超过590万人患有慢性心力衰竭(CHF)，并有67万新患者被诊断为CHF。充血性心力衰竭与高发病率和高死亡率以及高昂的护理费用有关。事实上，根据美国心脏学会的数据，总的来说，心血管疾病是美国主要的死亡原因，每年造成约2870亿美元的直接和间接成本。随着人口老龄化以及糖尿病和肥胖症发病率的增加，心血管疾病的发病率和相关护理费用预计也将上升。到2030年，美国65岁及以上人口将增加80%。尽管治疗费用和患病率都很高，但同时治疗急性心肌梗死和充血性心力衰竭的医疗选择有限。根据目前的临床数据，在接下来的几年里，细胞疗法很有可能在预防和治疗心功能不全方面发挥作用。在这项研究中，我们建议在该提案的第一阶段开发一种成人骨髓源细胞治疗产品MultiStem，该产品可以在冷冻瓶中配制，用于快速解冻和输送，以治疗急性缺血性损伤。对于第二阶段，我们计划研究细胞疗法在特定和新的心血管疾病患者中的效果，这些患者有心脏病发作，表现为非ST段抬高性心肌梗死(NSTEMI)，以确定我们是否可以降低发病率和死亡率，并为心脏提供功能益处。重要的是，NSTEMI人群的患病率正在增长，不同于首次ST段抬高急性心肌梗死的临床人群，后者在过去十年中一直在稳步下降。这些研究的成功完成将为第三阶段登记试验提供所需的数据，这将是将MultiStem用于治疗急性心血管损伤的上市所必需的。

DESCRIPTION (provided by applicant): Cardiovascular disease (CVD) represents a significant unmet medical need with over 1 million patients in the United States suffering from acute myocardial infarction (AMI) each year. This epidemic of heart attacks also represents a risk factor for further cardiovascular disease, with over 5.9 million individuals suffering from chronic heart failure (CHF) and > 670,000 new patients diagnosed with CHF annually in the United States. CHF is associated with high morbidity and mortality as well as high cost of care. In fact, taken together, CVD is the leading cause of death in the United States and is responsible for an estimated $287 billion dollars annually in direct and indirect costs, according the American Heart As the population ages and the incidence of diabetes and obesity increase, the incidence and associated cost of care for CVD Is expected to rise as well. By 2030, the number of people in US age 65 and over will increase by 80%. Despite the high cost of care and prevalence, the medical options to treat both AMI and CHF are limited. Based on the current clinical data, it would appear highly likely that cell therapy will play a role in the prevention ad treatment of cardiac dysfunction in the ensuing years. In this study, we proposed to develop in the Phase I portion of this proposal, an adult bone-marrow derived cell therapy product, MultiStem, that can formulated in a cryovial for rapid thaw and delivery to treat acute ischemic injury. For the Phase II portion, we plan to investigate the effects of cell therapy in a specific nd novel population of patients with cardiovascular disease, those patients with heart attacks that present as non-ST elevated myocardial infarcts (NSTEMI) to determine if we can reduce the morbidity and mortality and provide functional benefit to the heart. Importantly, the NSTEMI population is growing in prevalence unlike the clinical population of first-time ST elevation AMI which has been steadily declining over the past decade. Successful completion of these studies will provide the data required for a Phase III registration trial that will be required to market MultiStem for the treatment of acute cardiovascular injury.