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

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。多发性硬化症（MS）是一种人类中枢神经系统（CNS）的炎症和脱髓鞘退行性疾病。这是一种世界性的疾病，在美国大约有30万人受到影响；这是一种年轻人的疾病，70-80%的人在20-40岁之间发病。MS是一种基于临床病程、磁共振成像（MRI）扫描评估以及活检和尸检材料病理分析的异质性疾病。该疾病表现为大量可能的缺陷组合，包括脊髓、脑干、颅神经、小脑、大脑和认知综合征。进行性残疾是大多数多发性硬化症患者的命运，尤其是在25年的情况下。一半的多发性硬化症患者在发病15年内需要拐杖行走。多发性硬化症是导致中青年神经功能障碍的主要原因，直到过去十年，还没有已知的有效治疗方法。目前还没有批准的治疗原发性进行性多发性硬化症的方法，也没有有效预防长期残疾的治疗方法。存在大量的证据致病作用的抗体和b细胞淋巴细胞在女士的发病机制和识别b细胞免疫疗法作为小说的合适的目标女士利妥昔单抗治疗的具体切除b细胞和有良好的安全性,使其成为一个潜在的有吸引力的药理剂进一步阐明b细胞的作用及其在病理生理学女士和功能测试b细胞消耗的治疗潜力治疗。

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.