RITUXIMAB (MABTHERA/RITUXAN) IN ADULTS WITH PRIMARY PROGRESSIVE MS

利妥昔单抗（MABTHERA/RITUXAN）治疗成人原发性进行性多发性硬化症

• 批准号: 7380544
• 负责人: Aaron M Miller
• 金额: $ 0.8万
• 依托单位: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-17 至 2007-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7380544
• 关键词: antineoplastics; monoclonal antibody; role

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Multiple Sclerosis (MS) is an inflammatory and demyelinating degenerative disease of the human central nervous system (CNS). It is a worldwide disease that affects approximately 300,000 persons in the United States; it is a disease of young adults, with 70-80% having onset between 20-40 years old. MS is a heterogeneous disorder based on clinical course, magnetic response imaging (MRI) scan assessment, and pathology analysis of biopsy and autopsy material. The disease manifests itself in a large number of possible combinations of deficits, including spinal chord, brainstem, cranial nerve, cerebellar, cerebral and cognitive syndromes. Progressive disability is the fate of most patients with MS, especially when a 25-year perspective is included. Half of MS patients require a cane to walk within 15 years of disease onset. MS is a major cause of neurological disability in young and middle-aged adults and, until the past decade, has had no known beneficial treatments. There are no approved therapies for primary progressive MS and no treatments effective for the prevention of long-term disability. Substantial evidence exists for a pathogenic role of antibodies and B-cell lymphocytes in the pathogenesis of MS and identifies B-cells as an appropriate target for novel immunotherapies for the treatment of MS. Rituximab specifically ablates B-cells and has a well-characterized safety profile that makes it a potentially attractive pharmacological agent to further elucidate the role of B-cells and their functions in MS pathophysiology and to test the therapeutic potential of a B-cell depleting therapy.

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子弹和调查员（PI）可能已经从其他NIH来源获得了主要资金，因此可以在其他清晰的条目中代表。列出的机构适用于该中心，这不一定是调查员的机构。多发性硬化症（MS）是人类中枢神经系统（CNS）的一种炎症性和脱髓鞘性疾病。这是一种影响美国约30万人的全球疾病。它是一种年轻人的疾病，有70-80％的发作在20-40岁之间。 MS是基于临床过程，磁反应成像（MRI）扫描评估以及活检和尸检材料的病理分析的异质疾病。该疾病以许多可能的缺陷组合体现出来，包括脊柱和弦，脑干，颅神经，小脑，脑和认知综合征。进行性残疾是大多数MS患者的命运，尤其是在包括25年观点时。一半的MS患者需要甘蔗在疾病发作后的15年内行走。 MS是年轻和中年成年人神经残疾的主要原因，直到过去十年才有已知的有益治疗方法。 没有批准的主要进行性MS的疗法，也没有对预防长期残疾有效的治疗方法。存在大量证据表明，抗体和B细胞淋巴细胞在MS发病机理中的致病作用，并将B细胞鉴定为用于治疗MS的新型免疫疗法的适当靶标。利妥昔单抗特异性消融B细胞，具有良好的安全性概况，使其成为一种有吸引力的药理学剂，以进一步阐明B细胞及其在MS病理生理学中的功能的作用，并测试B细胞枯竭治疗的治疗潜力。