CD34 SELECTED PERIPHERAL BLOOD PROGENITOR CELLS W/ IMMUNOBLATION

CD34 选择性外周血祖细胞（免疫）

• 批准号: 6265779
• 负责人: ALAN MARSHALL MILLER
• 金额: $ 1.12万
• 依托单位: TULANE UNIVERSITY OF LOUISIANA
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-07-19 至 1999-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6265779
• 关键词: CD34 molecule; autoimmune disorder; cell transplantation; clinical research; clinical trial phase I; human subject; human therapy evaluation; immunopathology chemotherapy; immunotherapy; radiation immunosuppression; stem cells

Severe autoimmune diseases can only partially be controlled by conventional immunosuppressive treatments. There are over 40 of these diseases including severe lupus, mixed connective tissue disease and multiple sclerosis. In general, these diseases have been considered chronic diseases, but a small percentage of them are aggressive and can lead rapidly to death. Immunosuppressive drugs, particularly corticosteroids, have been the mainstay of therapy, with cytotoxic agents being used in the more difficult to treat cases. Abnormal immune cells, which recognize self as something foreign to be rejected, are responsible for causing the problems related to these diseases. This study proposes to use a combination of high dose chemotherapy, with radiation therapy and the transplantation of blood stem cells depleted of immune cells in order to eliminate those immune cells that react to self, and replace them with a new immune system that does not recognize self as foreign. The risks of these procedures include predominantly those associated with high dose chemotherapy and radiation therapy, with lower risk of severe organ damage, and up to a 5 % chance of transplant of procedure related death. In order to assure that only patients with the appropriate stages of their diseases are referred for this procedure, all referrals must be reviewed by the appropriate academic faculty at Tulane Medical Center including rheumatology, neurology, pediatric immunology and other appropriate groups. In addition, where necessary, outside experts will be called upon to review potential candidates. All requirement of the Tulane Bone Marrow Transplant Program that apply to cancer patients, regarding patients physiologic and psychological functioning, will be applied to patients on this study. Alternative treatments to be offered to the patients will include further use of standard therapies. However, these will be patients who have been felt to have failed standard therapy or other investigational treatments as may become available for those specific disease entities.

严重的自身免疫性疾病只能通过传统的免疫抑制治疗来部分控制。这些疾病有40多种，包括严重的狼疮、混合性结缔组织病和多发性硬化症。一般说来，这些疾病被认为是慢性病，但其中一小部分具有侵袭性，可能会迅速导致死亡。免疫抑制药物，特别是皮质类固醇，一直是治疗的主要药物，细胞毒药物用于更难治疗的病例。异常免疫细胞是导致这些疾病相关问题的罪魁祸首，这种细胞认为自己是要被排斥的异物。这项研究建议将大剂量化疗、放射治疗和移植耗尽免疫细胞的血液干细胞相结合，以消除那些对自身产生反应的免疫细胞，并用一种新的免疫系统取而代之，这种免疫系统不会识别自己是异体。这些手术的风险主要包括与大剂量化疗和放射治疗相关的风险，严重器官损伤的风险较低，移植手术相关死亡的可能性高达5%。为了确保只有疾病处于适当阶段的患者才能接受这一程序，所有转诊必须由杜兰医学中心的适当学术人员进行审查，包括风湿学、神经病学、儿科免疫学和其他适当的群体。此外，如有需要，将请外部专家审查可能的候选人。杜兰大学骨髓移植计划适用于癌症患者的所有要求，关于患者的生理和心理功能，将适用于本研究的患者。为患者提供的替代治疗将包括进一步使用标准治疗。然而，这些患者将被认为没有通过标准治疗或其他可能适用于这些特定疾病实体的研究治疗。