Department of Computer Science, University of Pisa, Pisa, Italy.
Department of Chemical & Systems Biology, Stanford University, Stanford, California, United States of America.
PLoS Comput Biol. 2022 May 4;18(5):e1009531. doi: 10.1371/journal.pcbi.1009531. eCollection 2022 May.
Schizophrenia is a debilitating psychiatric disorder, leading to both physical and social morbidity. Worldwide 1% of the population is struggling with the disease, with 100,000 new cases annually only in the United States. Despite its importance, the goal of finding effective treatments for schizophrenia remains a challenging task, and previous work conducted expensive large-scale phenotypic screens. This work investigates the benefits of Machine Learning for graphs to optimize drug phenotypic screens and predict compounds that mitigate abnormal brain reduction induced by excessive glial phagocytic activity in schizophrenia subjects. Given a compound and its concentration as input, we propose a method that predicts a score associated with three possible compound effects, i.e., reduce, increase, or not influence phagocytosis. We leverage a high-throughput screening to prove experimentally that our method achieves good generalization capabilities. The screening involves 2218 compounds at five different concentrations. Then, we analyze the usability of our approach in a practical setting, i.e., prioritizing the selection of compounds in the SWEETLEAD library. We provide a list of 64 compounds from the library that have the most potential clinical utility for glial phagocytosis mitigation. Lastly, we propose a novel approach to computationally validate their utility as possible therapies for schizophrenia.
精神分裂症是一种使人衰弱的精神疾病,会导致身体和社交方面的病态。全世界有 1%的人患有这种疾病,仅在美国每年就有 10 万例新病例。尽管精神分裂症很重要,但找到有效的治疗方法仍然是一项具有挑战性的任务,之前的工作进行了昂贵的大规模表型筛选。这项工作研究了机器学习在图上的优势,以优化药物表型筛选,并预测减轻精神分裂症患者过度神经胶质吞噬活动引起的大脑异常减少的化合物。给定一种化合物及其浓度作为输入,我们提出了一种方法,预测与三种可能的化合物效果相关的分数,即减少、增加或不影响吞噬作用。我们利用高通量筛选实验证明了我们的方法具有良好的泛化能力。该筛选涉及 2218 种化合物,浓度为 5 种不同浓度。然后,我们分析了我们的方法在实际环境中的可用性,即在 SWEETLEAD 库中优先选择化合物。我们提供了一份库中具有最具潜在临床用途的 64 种化合物的列表,用于减轻神经胶质吞噬作用。最后,我们提出了一种新的方法来计算验证它们作为精神分裂症治疗方法的效用。