Department of Biosystems, Laboratory of Gene Technology, KU Leuven, Leuven, Belgium.
Microb Biotechnol. 2024 Jul;17(7):e14510. doi: 10.1111/1751-7915.14510.
The Global Burden of Disease report of 2019 estimated 14 million infection-related deaths, making it the second leading cause of death after ischaemic heart disease. Bacterial pathogens accounted for 7.7 million deaths and deaths attributable to bacterial antibiotic resistance amounted to 1.3 million, describing a clear demand for novel antibiotics. Antibiotic development had its golden age in 1930-1960. Following failures in the screening of chemical libraries for novel antibiotics at the beginning of this century, the high cost of launching new antibiotics (estimated at US$ 1.4 billion per registered drug) and difficulties in achieving a return of investment for novel antibiotics, pharmaceutical industry has mostly left the field. The current Lilliput review analyses the question whether scientific or economic hurdles prevented the registration of new antibiotics. Scientifically, substantial progress has been achieved over recent years to define the chemical properties needed to overcome the permeation barrier in Gram-negative pathogens; in extending the chemical space of antibiotic candidates by full modular synthesis of suitable molecules; by extending bioprospecting to previously 'unculturable' bacteria or unusual bacteria; by attacking bacterial targets on the outer bacterial membrane; and by looking for support from structural biology, genomics, molecular genetics, phylogenetic analyses and deep machine learning approaches. However, these research activities were mostly conducted by academic researchers and biotech companies with limited financial resources. It thus seems that the development of new antibiotics, frequently described as the drying of the pipeline, is less limited by lack of scientific insight than by lack of the mobilization of the monetary resources needed to bring these discoveries to the market despite recent financial push and pull efforts of the public sector.
2019 年全球疾病负担报告估计,有 1400 万人因感染相关疾病而死亡,这使其成为仅次于缺血性心脏病的第二大死亡原因。细菌病原体导致了 770 万人死亡,而细菌对抗生素耐药导致的死亡人数达到了 130 万,这表明人们对新型抗生素有着明确的需求。抗生素的发展在 1930 年至 1960 年达到了黄金时期。本世纪初,在筛选新抗生素的化学库方面遭遇失败后,开发新抗生素的成本很高(每注册一种新药估计为 14 亿美元),而且新型抗生素难以收回投资,制药行业大多已经退出了这一领域。目前的《小人国评论》分析了科学或经济障碍是否阻止了新型抗生素的注册。从科学角度来看,近年来在定义克服革兰氏阴性病原体渗透障碍所需的化学特性方面取得了实质性进展;通过全模块化合成合适的分子,扩展了抗生素候选物的化学空间;通过将生物勘探扩展到以前“不可培养”或不寻常的细菌,扩大了生物勘探的范围;通过攻击细菌外膜上的细菌靶标,以及通过从结构生物学、基因组学、分子遗传学、系统发育分析和深度学习方法中寻找支持,来寻找支持。然而,这些研究活动主要是由学术研究人员和生物技术公司进行的,他们的资金有限。因此,新型抗生素的开发,经常被描述为“管道干涸”,与其说是由于缺乏科学洞察力,不如说是由于缺乏将这些发现推向市场所需的货币资源的调动,尽管公共部门最近在财力方面进行了推动和拉动。