†Department of Chemistry, University of Oxford, Chemistry Research Laboratory, Mansfield Road, Oxford, OX1 3TA, U.K.
‡Department of Pharmacology, University of Oxford, Mansfield Road, Oxford, OX1 3QT, U.K.
J Med Chem. 2015 Apr 9;58(7):2863-94. doi: 10.1021/jm500838d. Epub 2015 Jan 15.
A new paradigm for drug research has emerged, namely the deliberate search for molecules able to selectively affect the proliferation, differentiation, and migration of adult stem cells within the tissues in which they exist. Recently, there has been significant interest in medicinal chemistry toward the discovery and design of low molecular weight molecules that affect stem cells and thus have novel therapeutic activity. We believe that a successful agent from such a discover program would have profound effects on the treatment of many long-term degenerative disorders. Among these conditions are examples such as cardiovascular decay, neurological disorders including Alzheimer's disease, and macular degeneration, all of which have significant unmet medical needs. This perspective will review evidence from the literature that indicates that discovery of such agents is achievable and represents a worthwhile pursuit for the skills of the medicinal chemist.
一种新的药物研究范式已经出现，即刻意寻找能够选择性地影响存在于其组织中的成年干细胞增殖、分化和迁移的分子。最近，人们对药物化学在发现和设计影响干细胞的低分子量分子方面产生了浓厚的兴趣，这些小分子具有新颖的治疗活性。我们相信，从这样的发现计划中成功开发出的药物将对许多长期退行性疾病的治疗产生深远影响。其中包括心血管衰退、阿尔茨海默病等神经退行性疾病和黄斑变性等疾病，所有这些疾病都存在巨大的未满足的医疗需求。本文将综述文献中的证据，表明发现此类药物是可行的，并且代表了药物化学家技能的一项有价值的追求。
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