Carmichael Natasha, Day Philip J R
Faculty of Biology, Medicine and Health, The University of Manchester, Manchester, United Kingdom.
School of Biological Sciences and Manchester Institute of Biotechnology, The University of Manchester, Manchester, United Kingdom.
Front Pharmacol. 2022 Mar 8;13:852938. doi: 10.3389/fphar.2022.852938. eCollection 2022.
Despite the numerous scientific and technological advances made within the last decade the attrition rates for new drug discovery remain as high as 95% for anticancer drugs. Recent drug development has been in part guided by Lipinski's Rule of 5 (Ro5) even though many approved drugs do not comply to these rules. With Covid-19 vaccine development strategy dramatically accelerating drug development perhaps it is timely to question the generic drug development process itself to find a more efficient, cost effective, and successful approach. It is widely believed that drugs permeate cells via two methods: phospholipid bilayer diffusion and carrier mediated transporters. However, emerging evidence suggests that carrier mediated transport may be the primary mechanism of drug uptake and not diffusion as long believed. Computational biology increasingly assists drug design to achieve desirable absorption, distribution, metabolism, elimination and toxicity (ADMET) properties. Perfecting drug entry into target cells as a prerequisite to intracellular drug action is a logical and compelling route and is expected to reduce drug attrition rates, particularly gaining favour amongst chronic lifelong therapeutics. Novel drug development is rapidly expanding from the utilisation of beyond the rule of five (bRo5) to pulsatile drug delivery systems and fragment based drug design. Utilising transporters as drug targets and advocating bRo5 molecules may be the solution to increasing drug specificity, reducing dosage and toxicity and thus revolutionising drug development. This review explores the development of cell surface transporter exploitation in drug development and the relationship with improved therapeutic index.
尽管在过去十年里取得了众多科技进步,但抗癌药物新药研发的淘汰率仍高达95%。尽管许多已获批药物并不符合这些规则,但近期的药物研发在一定程度上受到了Lipinski的五规则(Ro5)的指导。随着新冠疫苗研发策略极大地加速了药物研发进程,或许现在是时候质疑通用的药物研发流程本身,以找到一种更高效、更具成本效益且更成功的方法了。人们普遍认为,药物通过两种方式渗透细胞:磷脂双分子层扩散和载体介导转运。然而,新出现的证据表明,载体介导转运可能是药物摄取的主要机制,而并非如长期以来所认为的那样是扩散。计算生物学越来越有助于药物设计,以实现理想的吸收、分布、代谢、排泄和毒性(ADMET)特性。完善药物进入靶细胞的过程作为细胞内药物作用的前提条件,是一条合乎逻辑且极具说服力的途径,有望降低药物淘汰率,尤其在慢性终身治疗药物中更受青睐。新型药物研发正从利用五规则之外(bRo5)迅速扩展到脉冲式给药系统和基于片段的药物设计。将转运体用作药物靶点并倡导bRo5分子可能是提高药物特异性、降低剂量和毒性从而彻底改变药物研发的解决方案。本综述探讨了细胞表面转运体在药物研发中的开发利用及其与改善治疗指数的关系。