Immunotherapy for amyloidosis

淀粉样变性的免疫治疗

• 批准号: G0901596/1
• 负责人: Mark Pepys
• 金额: $ 102.24万
• 依托单位: University College London
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2010
• 资助国家: 英国
• 起止时间: 2010 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=G0901596%2F1
• 关键词: Immunotherapy; amyloidosis

For 30 years the MRC has supported my work on a rare disease known as amyloidosis. We have discovered much of what is known about this condition and about a normal protein in the blood, known as serum amyloid P component (SAP), which contributes to the disease. Our MRC funded research on SAP led us to invent a new diagnostic test which has delivered significant improvements in treatment and survival of amyloidosis patients. We went on to establish the UK NHS National Amyloidosis Centre, the world?s leading centre for diagnosis and management of amyloidosis. The survival of our patients is not bettered anywhere else. However amyloidosis, which is responsible for the death of one per thousand of the population, is still an almost universally fatal disease. Alzheimer?s disease and maturity onset diabetes, both of which are associated with amyloid, inflict prolonged suffering on patients and carers, at great cost to society. We have now invented new medicines that specifically target SAP and amyloid, and produce clinical benefit in animal models which closely resemble human disease. Our most advanced drug, that removes SAP from the blood, helpfully slows disease progression in amyloidosis patients but this is not sufficient. We have now developed another novel treatment, using antibodies to SAP, which completely removes the amyloid deposits that cause disease. Our achievements have so impressed GlaxoSmithKline, the second largest pharmaceutical company in the world, that it has agreed to collaborate with us to develop these medications. They have insisted that our university research team shares the early risk in the long, difficult and extremely costly process of drug development. They are therefore deliberately not funding our component of the programme. The present proposal seeks the funding necessary for our contribution, while GSK conduct their own in house development work. This represents extraordinarily good value for the MRC and for the patients who will ultimately benefit from potentially life saving new treatments. Enabled by the laboratory research proposed here, the GSK collaboration will bring our key inventions into clinical trials as soon as possible and, within the 3 year period of the grant, the basic laboratory work, for which we now seek MRC support, will translate towards benefits for sick people.

30 年来，MRC 一直支持我对一种罕见疾病淀粉样变性的研究。我们已经发现了有关这种疾病以及血液中一种正常蛋白质的大部分信息，这种蛋白质被称为血清淀粉样蛋白 P 成分 (SAP)，它会导致这种疾病。 MRC 资助的 SAP 研究促使我们发明了一种新的诊断测试，该测试显着改善了淀粉样变性患者的治疗和生存率。我们随后建立了英国 NHS 国家淀粉样变性中心，这是世界领先的淀粉样变性诊断和管理中心。我们的患者的生存率在其他任何地方都没有得到改善。然而，导致千分之一人口死亡的淀粉样变性仍然是一种几乎普遍致命的疾病。阿尔茨海默病和成年型糖尿病都与淀粉样蛋白有关，给患者和护理人员带来长期痛苦，给社会造成巨大损失。我们现在已经发明了专门针对 SAP 和淀粉样蛋白的新药物，并在与人类疾病非常相似的动物模型中产生了临床益处。我们最先进的药物可以去除血液中的 SAP，有助于减缓淀粉样变性患者的疾病进展，但这还不够。我们现在开发了另一种新的治疗方法，使用 SAP 抗体，完全去除引起疾病的淀粉样蛋白沉积物。我们的成就给世界第二大制药公司葛兰素史克留下了深刻的印象，它同意与我们合作开发这些药物。他们坚持要求我们大学的研究团队在漫长、困难且成本极高的药物开发过程中分担早期风险。因此，他们故意不资助我们的该计划部分。目前的提案寻求我们的贡献所需的资金，而葛兰素史克则开展自己的内部开发工作。这对 MRC 和最终将从可能挽救生命的新疗法中受益的患者来说具有非常好的价值。通过这里提出的实验室研究，葛兰素史克合作将尽快将我们的关键发明投入临床试验，并且在资助的 3 年内，我们现在寻求 MRC 支持的基础实验室工作将转化为造福于病人。