'Degen-lock' - a novel way of protecting the nervous system in Motor Neurone Disease

“Degen-lock”——一种保护运动神经元疾病神经系统的新方法

• 批准号: 41643
• 金额: $ 41.39万
• 依托单位: TMN THERAPEUTICS LIMITED
• 依托单位国家: 英国
• 项目类别: Study
• 财政年份: 2020
• 资助国家: 英国
• 起止时间: 2020 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=41643
• 关键词: Degen; lock; novel; way; protecting

We are developing a treatment for a neurological disease called motor neuron disease (MND). There is no cure for MND and the current therapies on the market do little to delay disease progression.This condition is caused by the death of a particular subset of brain cells known as motor neurons. These cells normally instruct muscles when to move, and their death causes patients to experience a progressive and irreversible paralysis. As the disease advances many patients find it difficult to talk, swallow and breathe, putting them at risk of choking or respiratory failure. Over 80% of patients will not survive 5 years after diagnosis.One of the reasons brain cells die in MND is chronic inflammation in the brain. Some inflammation can be a good thing-- it kills viruses and bacteria and helps to remove debris and dead cells. However, when this inflammation gets out of control it can kill brain cells, accelerating the progression of MND.To treat MND an extremely strong, but specific, anti-inflammatory is required --protecting motor neurons but allowing the rest of the brain to benefit from positive effects of inflammation. We have therefore invented a new technology, 'degen-lock' which targets an anti-inflammatory effect to the brain cells which are dying in MND, protecting them from inflammation.This grant from innovate UK will allow us to test and develop the targeting system and create a prototype of our MND therapy.

我们正在开发一种治疗神经系统疾病的方法，称为运动神经元病（MND）。MND是一种无法治愈的疾病，目前市场上的治疗方法对延缓疾病进展几乎没有作用。这种疾病是由一种称为运动神经元的特定脑细胞亚群的死亡引起的。这些细胞通常指示肌肉何时运动，它们的死亡会导致患者经历进行性和不可逆的瘫痪。随着疾病的发展，许多患者发现难以说话，吞咽和呼吸，使他们面临窒息或呼吸衰竭的风险。超过80%的患者在确诊后5年内无法存活。MND脑细胞死亡的原因之一是大脑中的慢性炎症。一些炎症可能是一件好事-它杀死病毒和细菌，并有助于清除碎片和死细胞。然而，当这种炎症失控时，它会杀死脑细胞，加速MND的进展。为了治疗MND，需要一种非常强大但特异性的抗炎药物-保护运动神经元，但允许大脑的其他部分受益于炎症的积极影响。因此，我们发明了一种新技术，“degen-lock”，它针对MND中死亡的脑细胞产生抗炎作用，保护它们免受炎症的影响。英国创新基金会的这笔拨款将使我们能够测试和开发靶向系统，并创建我们的MND治疗原型。