JPND Biological Resource Analysis to Identify New MEchanisms and phenotypes in Neurodegenerative Diseases (BRAIN-MEND)

神经退行性疾病包括阿尔茨海默病、帕金森病、肌萎缩性侧索硬化症、额颞叶痴呆、皮质基底变性、多系统萎缩和进行性核上性麻痹。我们目前根据症状和临床表现对这些疾病进行分类和治疗。虽然这种方法似乎合乎逻辑，但它没有考虑到每种疾病的原因或它们之间的任何重叠，这阻碍了新治疗方法的发展。更好的方法是根据病因对不同的病症进行重新分类，因为这需要对每种病症有深入但不同的理解，然后新的治疗方法就可以直接针对病因。这种重新分类、新的理解和对可能治疗方法的探索，就是BRAIN-MEND背后的理念。我们将采用由BRAIN-MEND联盟首创和发展的三种创新方法。我们将使用遗传学和表观遗传学的最新方法来确定神经退行性疾病的原因，以及它们影响的细胞系统。我们将使用尖端技术（基于一种称为网络分析的方法）从遗传和表观遗传结果中确定新的药物靶点。同时，将使用机器学习来分析医学文献和患者记录，以发现新的或未被识别的症状集群和其他可能提示新疾病组的临床特征。这三种不同的方法：寻找病因，寻找药物靶点，寻找新的疾病群体，将相互补充，这样我们就可以确定任何新的发现。BRAIN-MEND的一个关键成果将是理清不同的神经退行性疾病，这样对于任何患者群体，我们都能理解不同的病因如何产生相同的临床症状，而在其他情况下，相同的病因如何产生不同的临床症状。有了这种神经退行性疾病的新思维方式将增强我们对它们的理解，并使其更容易开发新的治疗方法。患者和护理人员将通过参与研究设计，例如，开发以患者为导向的网站，密切参与整个项目。BRAIN-MEND的合作伙伴非常有资格执行所提议的工作，具有出色的科学记录，可直接获得待研究的数据，以及将使用的科学方法方面的专业知识。所有结果将通过医学文献、在国际会议上的演讲、通过联盟博客、通过社会媒体以及通过相关机构的新闻办公室公布。

Neurodegenerative diseases include Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, frontotemporal dementia, corticobasal degeneration, multiple system atrophy and progressive supranuclear palsy. We currently classify and treat these conditions based on symptoms and clinical findings. Although this approach seems logical it does not take into account the causes of each condition or any overlap between them, which hinders the development of new treatments. A better approach would be to reclassify the different conditions based on their causes, because this would require a deep but different understanding of each condition, and new treatments could then be directly aimed at the cause. This reclassification, new understanding, and exploration of possible treatments, is the idea behind BRAIN-MEND. We will use three innovative approaches, pioneered and developed by the BRAIN-MEND consortium. We will use the latest methods in genetics and epigenetics to identify the causes of neurodegenerative diseases, and which cell systems they affect. We will use a cutting edge technique (based on a method called network analysis) to identify new drug targets from the genetic and epigenetic results. At the same time, will use machine learning to analyse medical literature and patient records to find new or under-recognized clusters of symptoms and other clinical features which might suggest new disease groups. These three different approaches: finding causes, finding drug targets, finding new disease groups, will act to complement each other so we can be sure of any new findings. A key outcome of BRAIN-MEND will be to disentangle the different neurodegenerative diseases, so that for any patient group, we understand how different causes produce the same clinical picture, while in other cases, the same cause can produce different clinical pictures. Having this new way of thinking of neurodegenerative diseases will enhance our understanding of them and make it easier to develop new treatments. Patients and caregivers will be intimately involved throughout the project, through participation in study design, and for example, development of a patient-orientated website. BRAIN-MEND partners are highly qualified to perform the work proposed, with outstanding scientific track records, direct access to the data to be studied, and expertise in the scientific methods that will be used. All the results will be publicized through medical literature, presentation at international conferences, through a consortium blog, through social media, and through the press offices of the institutions involved.