Chadman Kathryn K
New York State Institute for Basic Research in Developmental Disabilities , 1050 Forest Hill Road, Staten Island, NY 10302 , USA
Expert Opin Drug Discov. 2014 Dec;9(12):1389-91. doi: 10.1517/17460441.2014.962511. Epub 2014 Sep 24.
Currently, there are still just two medications approved by the U.S. FDA to treat the irritability that is often found in children with autism spectrum disorder (ASD). Furthermore, there are no drugs approved that treat the core symptoms of ASD. And while many drugs have had good outcomes in preclinical animal models, they have been met with very little success in human clinical trials. There are several factors likely contributing to this problem starting with the heterogeneity within the ASD phenotype. It is therefore important that researchers develop their knowledge about the neurobiological basis of ASD as well as possible subgroups within the disorder, based on either specific behavioral profiles or biological pathways. Furthermore, the development of valid biological markers will allow researchers to use more than parent or teacher reports to determine if a treatment is effective. This author believes that there a good chance of finding effective pharmacological treatments for the core symptoms of ASD with this increased knowledge of the biology and etiology.
目前，美国食品药品监督管理局（FDA）仅批准了两种药物用于治疗自闭症谱系障碍（ASD）患儿中常见的易激惹症状。此外，尚无获批用于治疗ASD核心症状的药物。虽然许多药物在临床前动物模型中取得了良好效果，但在人体临床试验中却收效甚微。有几个因素可能导致了这一问题，首先是ASD表型的异质性。因此，研究人员基于特定行为特征或生物学途径，深入了解ASD的神经生物学基础以及该疾病可能存在的亚组非常重要。此外，有效的生物标志物的开发将使研究人员在确定一种治疗方法是否有效时，不仅仅依赖家长或教师的报告。作者认为，随着对生物学和病因学的了解不断增加，很有可能找到针对ASD核心症状的有效药物治疗方法。
