Evaluation of orally active anti-inflammatory C5a receptor antagonists in a transgenic rat motor neurone disease model

转基因大鼠运动神经元疾病模型中口服活性抗炎 C5a 受体拮抗剂的评价

• 批准号: nhmrc : 455856
• 负责人: A/Pr Peter Noakes
• 金额: $ 35.58万
• 依托单位: The University of Queensland
• 依托单位国家: 澳大利亚
• 项目类别: NHMRC Project Grants
• 财政年份: 2007
• 资助国家: 澳大利亚
• 起止时间: 2007-01-01 至 2009-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/evaluation-orally-active-disease-model/98168
• 关键词: Evaluation; orally; active; anti; inflammatory

Motor neurone disease is a rapidly progressive and incurable disease, usually ending in death within 3-5 years of diagnosis. The disease usually arrives without warning, and results in a progressive loss of muscle control. There is no effective treatment, and available drugs increase life span by a few weeks at best. There is evidence that the disease involves an inflammatory component, but available anti-inflammatory drugs are ineffective. We have developed a new class of anti-inflammatory drugs, known as C5a antagonists, and in preliminary experiments have shown they are therapeutically effective in a transgenic rat model of motor neurone disease. We propose to investigate in more detail how these drugs work in the rat model, and demonstrate that a specific inflammatory pathway, which we can now block, is responsible for some of the disease's progression. This work may lead to an entirely new class of drugs being used to treat patients with this drastic disease.

运动神经元疾病是一种进展迅速且无法治愈的疾病，通常在诊断后3-5年内导致死亡。这种疾病通常在没有警告的情况下到来，并导致肌肉控制的逐渐丧失。没有有效的治疗方法，可用的药物最多能延长几周的寿命。有证据表明，这种疾病涉及炎症成分，但现有的抗炎药物无效。我们已经开发了一类新的抗炎药物，称为C5 a拮抗剂，并在初步实验中表明，它们在运动神经元疾病的转基因大鼠模型中具有治疗效果。我们建议更详细地研究这些药物如何在大鼠模型中起作用，并证明我们现在可以阻断的特定炎症通路是导致疾病进展的原因。这项工作可能会导致一种全新的药物被用于治疗这种严重疾病的患者。