Center for Neuroscience, SRI International, Menlo Park, CA, USA.
Pharmacol Biochem Behav. 2011 Aug;99(2):130-45. doi: 10.1016/j.pbb.2011.03.022. Epub 2011 Apr 1.
The investigation of novel drug targets for treating cognitive impairments associated with neurological and psychiatric disorders remains a primary focus of study in central nervous system (CNS) research. Many promising new therapies are progressing through preclinical and clinical development, and offer the potential of improved treatment options for neurodegenerative diseases such as Alzheimer's disease (AD) as well as other disorders that have not been particularly well treated to date like the cognitive impairments associated with schizophrenia (CIAS). Among targets under investigation, cholinergic receptors have received much attention with several nicotinic agonists (α7 and α4β2) actively in clinical trials for the treatment of AD, CIAS and attention deficit hyperactivity disorder (ADHD). Both glutamatergic and serotonergic (5-HT) agonists and antagonists have profound effects on neurotransmission and improve cognitive function in preclinical experiments with animals; some of these compounds are now in proof-of-concept studies in humans. Several histamine H3 receptor antagonists are in clinical development not only for cognitive enhancement, but also for the treatment of narcolepsy and cognitive deficits due to sleep deprivation because of their expression in brain sleep centers. Compounds that dampen inhibitory tone (e.g., GABA(A) α5 inverse agonists) or elevate excitatory tone (e.g., glycine transporter inhibitors) offer novel approaches for treating diseases such as schizophrenia, AD and Down syndrome. In addition to cell surface receptors, intracellular drug targets such as the phosphodiesterases (PDEs) are known to impact signaling pathways that affect long-term memory formation and working memory. Overall, there is a genuine need to treat cognitive deficits associated with many neuropsychiatric conditions as well as an increasingly aging population.
治疗与神经和精神障碍相关的认知障碍的新型药物靶标研究仍然是中枢神经系统 (CNS) 研究的主要焦点。许多有前途的新疗法正在进行临床前和临床开发,为阿尔茨海默病 (AD) 等神经退行性疾病以及其他迄今为止治疗效果不佳的疾病(如与精神分裂症相关的认知障碍,CIAS)提供了改善治疗选择的潜力。在研究中的目标中,胆碱能受体受到了广泛关注,几种烟碱激动剂(α7 和 α4β2)正在积极进行临床试验,用于治疗 AD、CIAS 和注意力缺陷多动障碍 (ADHD)。谷氨酸能和 5-羟色胺能 (5-HT) 激动剂和拮抗剂对动物的神经传递有深远影响,并改善了认知功能的临床前实验;其中一些化合物目前正在进行人体概念验证研究。几种组胺 H3 受体拮抗剂不仅在认知增强方面,而且在治疗因睡眠剥夺引起的嗜睡症和认知缺陷方面也处于临床开发阶段,因为它们在大脑睡眠中心表达。抑制性调谐(例如 GABA(A)α5 反向激动剂)或兴奋性调谐(例如甘氨酸转运蛋白抑制剂)的化合物为治疗精神分裂症、AD 和唐氏综合征等疾病提供了新的方法。除了细胞表面受体外,已知细胞内药物靶标,如磷酸二酯酶 (PDEs),会影响影响长期记忆形成和工作记忆的信号通路。总体而言,确实需要治疗许多神经精神疾病以及人口老龄化带来的认知障碍。