Integrated training in development and clinical practice of cell-based therapies

 描述(申请人提供)：干细胞治疗市场价值27亿美元，目前主要由造血干细胞(HSC)移植市场组成。造血干细胞疗法正在以大约10.6%的复合增长率增长，干细胞疗法的进步正在从血液系统恶性肿瘤的治疗扩展到心血管、神经和其他疾病的治疗。血液产品为几种新型细胞疗法的制造提供了原材料，包括干细胞和免疫细胞疗法。随着自然杀伤(NK)细胞、嵌合抗原受体T细胞(CAR-T细胞)、抗原特异性T细胞、间充质干细胞/基质细胞等试验的初步结果令人振奋，该领域只会继续扩大。随着基于血液的研究的科学家人数减少，细胞治疗领域的成功关键是将重点放在培训最新的研究人员和新的研究人员上，也许是在考虑一个方向。新的研究人员以及可能接受过血液研究基础知识培训的科学家可能缺乏将技术从工作台扩大到床边的经验。具体地说，他们可能没有用于处理血液和干细胞疗法的先进技术，以及用于产品表征和治疗后患者评估的复杂分析经验。此外，大多数研究人员对基于细胞的治疗的监管要求缺乏经验，这在将潜在的治疗从试验台转移到床边是一个明确的挑战。在细胞疗法的开发和临床实践方面的综合教育计划将有助于遏制基于血液的研究人员的衰落，并促进血液病和其他疾病的治疗进展。明尼苏达大学(UM)是提供这样一个项目的理想选择，它在细胞疗法的开发方面有着良好的记录。UM的cGMP设施，分子和细胞治疗(MCT)，作为细胞治疗的生产援助(PACT)小组的成员非常成功，这是NHLBI赞助的合同。MCT一直是几个广受欢迎的PACT网络研讨会、设施研讨会和专业组织(如AABB和ISCT)[3]教育项目的组成部分，并培养了来自世界各地的众多医学生、住院医生、研究员和访问学者。密歇根大学的生物保存核心资源(BioCoR)是国家唯一的保存资源，设有细胞疗法保存培训课程，并研究细胞治疗保存的技术和方法。这些资源和东道主使密歇根大学非常适合提供这种短期的教育经验，以丰富血液学劳动力。(摘要结束)

 DESCRIPTION (provided by applicant): The stem cell therapy market is valued at $2.7 billion, currently consisting largely of the hematopoietic stem cell (HSC) transplant market. HSC therapies are growing at roughly 10.6 % compound growth rate, and the advancement in stem cell therapies is broadening beyond the treatment of hematological malignancies into the treatment of cardiovascular, neurological, and other disorders. Blood products provide raw material for the manufacturing of several novel cell therapies, including stem cells and immune cell therapies. With promising early results in trials with natural killer (NK) cells, chimeric antgen receptor T-cells (CAR-T cells), antigen-specific T cells, mesenchymal stem/stromal cells, and others, the field will only continue to expand. With a decline in scientists in blood-based research, it is critical to the success of the field of cell therapy to focus efforts on training crrent researchers, as well as new investigators, perhaps contemplating a direction. New investigators as well as scientists who may be trained in the fundamentals of blood-based research may lack experience scaling up the techniques from the bench to the bedside. Specifically, they may not have experience in the advanced techniques used to process blood and stem-cell based therapies, and the sophisticated assays used for product characterization and post-treatment patient assessment. Furthermore, most investigators do not have experience with the regulatory requirements of cell-based therapies, a definite challenge in moving potential therapies from the bench to the bedside. An integrated education program in development and clinical practice of cell-based therapies would be instrumental in countering the decline of blood-based investigators and fostering advances in the treatment of hematological and other disease. The University of Minnesota (UM) is ideally positioned to provide such a program, with a proven track record of development of cellular therapies. The cGMP facility of the UM, Molecular & Cellular Therapeutics (MCT), has been highly successful as a member of the Production Assistance for Cellular Therapies (PACT) group, an NHLBI-sponsored contract. MCT has been an integral part of several well- received PACT webinars and facility workshops and education programs with professional organizations (e.g., AABB and ISCT) [3] and has educated numerous medical students, residents, fellows, and visiting scholars from around the world. The Biopreservation Core Resource (BioCoR) at the UM is the nations only resource in preservation and has training courses in preservation of cellular therapies and research into technology and methods of preservation for cellular therapies. These resources and a host makes the UM well suited to offer this short term educational experiences to enrich the hematology workforce. (End of Abstract)