Transplantation of Multipotent Progenitor Cells in Stroke

Veraillie博士和他的同事开发的多潜能祖细胞(MPC) 在明尼苏达大学，似乎有使他们非常适合的属性 作为一种干细胞疗法。Athersys，Inc.拥有这些电池的专有权利。我们最近做了 与开发制造业的Athersys，Inc.建立了合作关系 使这些细胞可用于临床的技术。Athersys，Inc.将向我们提供这些电池。 我们在啮齿动物身上的初步数据表明，这些细胞作为中风的治疗方法非常有希望。在……里面 为了开发这些细胞在卒中中的作用，我们提出了一个翻译方案。建议数 一系列实验将作为继续进行骨髓基质细胞移植的临床前基础 缺血性中风患者。卒中后MFC剂量和移植时机的可行性、安全性和最佳剂量 为了在啮齿动物模型中产生疗效，将对成年缺血性中风模型进行检验。这个 遵循特定的目标将成为里程碑，并有“去和不去”的标准。 具体目标1：确定静脉注射大鼠的“长期”(即移植后6个月)疗效 一过性大脑中动脉闭塞模型中的MPC。 具体目标2：评估Athersys，Inc.生产的IV人MPC的长期疗效。 根据GLP异种一过性大脑中脑GMP制作方案 动脉闭塞模型。 具体目标3：评估静脉注射人类MPC以实现长期行为的有效性 对雄性和雌性以及成年和老年大鼠暴露于短暂性中脑的益处 动脉闭塞模型。 具体目标4：展示静脉注射人类MPC实现长期行为的有效性 永久性大脑中动脉结扎模型的益处。 特异性目标5：鉴定同种异体大鼠骨髓基质干细胞的组织相容性 异种人MPC进一步提供大鼠缺血性卒中模型中静脉注射MPC的安全性。 ？具体目标6：召开IND FDA筹备会议并汇编文件以备存档 IND用于缺血性卒中的干细胞治疗。 这项建议扩展了我们确定干细胞疗法在中风中的益处的科学平台。

Multipotent progenitor cells (MPCs), developed by Dr. Verfaillie and co-workers at the University of Minnesota, appear to have the properties which make them highly suitable as a stem cell therapy. Athersys, Inc. has the proprietary rights to these cells. We have recently developed a collaborative relationship with Athersys, Inc. which has developed manufacturing techniques to make these cells available for clinical use. Athersys, Inc. will supply these cells to us. Our preliminary data in rodents indicate that these cells are quite promising as a therapy for stroke. In order to develop the utility of these cells in stroke, we propose a translational protocol. The proposed set of experiments will serve as the preclinical basis for proceeding with transplantation of MPCs in ischemic stroke patients. Feasibility, safety, and optimal MFC dose and transplantation timing poststroke for producing efficacy in rodent models of adult ischemic stroke will be examined. The following specific aims will serve as milestones, with "go and no-go" criteria. ¿ Specific Aim 1: Determine the "long-term" (i.e., 6 months post-transplantation) efficacy of IV rat MPCs in transient middle cerebral artery occlusion model. ¿ Specific Aim 2: Evaluate the long-term efficacy of IV human MPCs produced by Athersys, Inc. according to GMP manufacturing protocols in a GLP xenogeneic transient middle cerebral artery occlusion model. ¿ Specific Aim 3: Assess the efficacy of IV human MPCs to achieve long-term behavioral benefits in male and female, and adult and aged rats exposed to transient middle cerebral artery occlusion model. ¿ Specific Aim 4: Demonstrate the efficacy of IV human MPCs to achieve long-term behavioral benefits in permanent middle cerebral artery ligation model. ¿ Specific Aim 5: Characterize stem cell histocompatibility in allogeneic rat MPCs and xenogeneic human MPCs to further provide safety of IV MPCs in rat ischemic stroke model. ¿ Specific Aim 6: Conduct IND FDA preparation meetings and assemble documents for filing IND for stem cell therapy in ischemic stroke. This proposal extends our scientific platform for determining the benefit of stem cell therapy in stroke.