Centre for Molecular Therapeutics, Australian Institute of Tropical Health and Medicine, James Cook University, Cairns, Queensland, Australia.
PLoS Pathog. 2020 May 14;16(5):e1008508. doi: 10.1371/journal.ppat.1008508. eCollection 2020 May.
Parasitic helminths have coevolved with humans over millennia, intricately refining and developing an array of mechanisms to suppress or skew the host's immune system, thereby promoting their long-term survival. Some helminths, such as hookworms, cause little to no overt pathology when present in modest numbers and may even confer benefits to their human host. To exploit this evolutionary phenomenon, clinical trials of human helminth infection have been established and assessed for safety and efficacy for a range of immune dysfunction diseases and have yielded mixed outcomes. Studies of live helminth therapy in mice and larger animals have convincingly shown that helminths and their excretory/secretory products possess anti-inflammatory drug-like properties and represent an untapped pharmacopeia. These anti-inflammatory moieties include extracellular vesicles, proteins, glycans, post-translational modifications, and various metabolites. Although the concept of helminth-inspired therapies holds promise, it also presents a challenge to the drug development community, which is generally unfamiliar with foreign biologics that do not behave like antibodies. Identification and characterization of helminth molecules and vesicles and the molecular pathways they target in the host present a unique opportunity to develop tailored drugs inspired by nature that are efficacious, safe, and have minimal immunogenicity. Even so, much work remains to mine and assess this out-of-the-box therapeutic modality. Industry-based organizations need to consider long-haul investments aimed at unraveling and exploiting unique and differentiated mechanisms of action as opposed to toe-dipping entries with an eye on rapid and profitable turnarounds.
寄生虫性蠕虫与人类共同进化了数千年,它们精巧地调整和发展了一系列机制来抑制或改变宿主的免疫系统,从而促进自身的长期生存。一些蠕虫,如钩虫,在数量适中时几乎不会引起明显的病理变化,甚至可能对其人类宿主有益。为了利用这一进化现象,已经进行了人体蠕虫感染的临床试验,以评估其在一系列免疫功能障碍疾病中的安全性和疗效,结果喜忧参半。对活蠕虫治疗的小鼠和大型动物的研究令人信服地表明,蠕虫及其排泄/分泌产物具有抗炎药物样特性,代表了一个未开发的药物库。这些抗炎成分包括细胞外囊泡、蛋白质、聚糖、翻译后修饰和各种代谢物。虽然寄生虫启发疗法的概念具有很大的前景,但它也给药物开发界带来了挑战,因为后者通常不熟悉与抗体不同的外来生物制剂。鉴定和表征蠕虫分子和囊泡以及它们在宿主中靶向的分子途径,为开发受自然启发的、有效、安全且免疫原性低的定制药物提供了独特的机会。即便如此,仍有大量工作需要挖掘和评估这种非传统的治疗方式。基于行业的组织需要考虑长期投资,旨在揭示和利用独特和差异化的作用机制,而不是浅尝辄止,着眼于快速和有利可图的转变。