Novel therapy for treating Multiple Sclerosis

描述（由申请人提供）：已经尝试了多种疗法来治疗多发性硬化症。这种实验性疗法包括施用髓磷脂碱性蛋白、施用衍生自髓磷脂碱性蛋白的肽、注射髓磷脂碱性蛋白肽基序模拟物醋酸格拉替雷，以及最近用编码髓磷脂碱性蛋白的DNA治疗。使用这种疗法的早期临床试验显示没有或很少有临床益处。基于这些过去的临床试验，很明显，需要对多发性硬化症患者进行更有效的治疗。本文提出的SBIR I期研究的总体目标是开发用于治疗多发性硬化患者的新型制剂。为了开始完成这项任务，我们将在转基因大豆种子中表达一种新的蛋白质。这种新蛋白的成功表达将允许测试大豆制剂在治疗多发性硬化症中的功效。为了完成这一任务，提出了以下里程碑：里程碑#1：用于治疗多发性硬化症的新型融合蛋白将在转基因大豆中表达。在里程碑#1中，我们将建立大豆品系并批量生产大豆种子，这些种子可用于制备大豆制剂，用于表征（里程碑#2）和有效性研究（里程碑#3）。里程碑2：我们将表征新的大豆衍生的融合蛋白，并证明这种蛋白质保留其正常的性质和功能。里程碑3：证明由表达融合蛋白的种子制成的大豆制剂在治疗多发性硬化症模型中显示出功效。尽管旨在治疗多发性硬化症的人体临床试验令人失望且成功有限，但此类疗法的潜力太大，不容忽视。在这个I期SBIR中，我们建议在转基因大豆中表达一种新的融合蛋白，以便可以评估大豆制剂在治疗多发性硬化症模型中的功效。这项I期SBIR研究的成功将为未来的SBIR研究奠定基础，这些研究将证明这种治疗在多发性硬化症患者中的安全性和有效性。公共卫生相关性：根据过去的临床试验，很明显，多发性硬化症患者需要更有效的治疗方法。本文提出的SBIR I期研究的总体目标是开发用于治疗多发性硬化患者的新型制剂。这种新制剂的成功表达将允许测试治疗多发性硬化症的功效。

DESCRIPTION (provided by applicant): A variety of therapies have been attempted to treat Multiple Sclerosis. Such experimental therapies have included the administration of myelin basic protein, administration of peptides derived from myelin basic protein, injection of the myelin basic protein peptide motif mimic, glatiramer acetate, and most recently, treating with DNA encoding myelin basic protein. The early clinical trials using such therapy have showed no, or little, clinical benefit. Based on these past clinical trials, it is clear that more efficacious therapies for Multiple Sclerosis patients are needed. The overall goal for the SBIR Phase I studies proposed here is to develop a novel formulation for use in treating Multiple Sclerosis patients. To begin to accomplish this task, we will express a novel protein in transgenic soybean seeds. The successful expression of this novel protein will allow soy formulations to be tested for efficacy in treating Multiple Sclerosis. To accomplish this task, the following milestones are proposed: Milestone #1: A novel fusion protein for treating Multiple Sclerosis will be expressed in transgenic soybeans. In milestone #1, we will establish soybean lines and produce soybean seeds in bulk quantities that can be used to make soy formulations for characterization (Milestone #2) and for use in efficacy studies (Milestone #3). Milestone #2: We will characterize the novel soybean-derived fusion protein and demonstrate that this protein retains it normal properties and functionality. Milestone #3: Demonstrate that soy formulations made from seeds expressing the fusion protein shows efficacy in treating a model of Multiple Sclerosis. Despite the disappointments and limited success of human clinical trials aimed at treating Multiple Sclerosis, the potential for such therapies is too great to ignore. In this Phase I SBIR, we propose to express a novel fusion protein in transgenic soybeans so that soy formulations can be assessed for their efficacy in treating a model of Multiple Sclerosis. Success with this Phase I SBIR studies will set the stage for future SBIR studies which will demonstrate safety and efficacy of such therapy in Multiple Sclerosis patients. PUBLIC HEALTH RELEVANCE: Based on past clinical trials, it is clear that more efficacious therapies for Multiple Sclerosis patients are needed. The overall goal for the SBIR Phase I studies proposed here is to develop a novel formulation for use in treating Multiple Sclerosis patients. The successful expression of this novel formulation will allow testing for efficacy in treating Multiple Sclerosis.