Schoales Zoe, Ghosh Pratyusha, Vasylaki Anastasiia, Jaimes Edgar A, Williams Ryan
Department of Biomedical Engineering, City College of New York, New York, NY, USA.
Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Clin Kidney J. 2025 Sep 18;18(9):sfaf192. doi: 10.1093/ckj/sfaf192. eCollection 2025 Sep.
Kidney diseases are a substantial worldwide health burden, with high mortality and increasing incidence. Despite their prevalence, substantial gaps remain in the clinic in both diagnostics and therapeutics. Many novel treatments have failed in clinical trials or fallen out of use in the clinic due to side effects and poor efficacy, in large part due to poor therapeutic profiles in the kidney. Nanomedicines have begun to emerge as a potentially promising diagnostic or therapeutic delivery system. Based on their physicochemical properties, such as size, shape, surface chemistry, and so on, some nanotechnologies can target the kidneys. However, as of yet, no kidney-specific nanomedicines have reached clinical translation. While the field of renal nanomedicine is in its early stages and growing, some potential obstacles to translation include poor preclinical models, challenges in manufacturing scale-up, clinical trial design and the cost of translation. Here, we overview the current state of the kidney-targeting nanomedicine field and outline a potential framework for clinical translation. We focus on the paths of US Food and Drug Administration- approved nanomedicines and suggestions from other nanomedicine fields to inform our key considerations for translational success. We also highlight the importance of academic and clinical collaboration with industry and federal regulators. Several investigational technologies are just now at the cusp of scaling towards the clinic and we therefore aim to support this momentum for improving the lives of patients with kidney diseases.
肾脏疾病是全球范围内一项重大的健康负担，死亡率高且发病率不断上升。尽管其普遍存在，但在临床诊断和治疗方面仍存在巨大差距。许多新型治疗方法在临床试验中失败，或因副作用和疗效不佳而在临床上停用，这在很大程度上是由于在肾脏中的治疗效果不佳。纳米药物已开始作为一种潜在的有前景的诊断或治疗递送系统出现。基于其物理化学性质，如尺寸、形状、表面化学等，一些纳米技术可以靶向肾脏。然而，截至目前，尚无肾脏特异性纳米药物进入临床转化阶段。虽然肾脏纳米医学领域尚处于早期阶段且在不断发展，但转化过程中的一些潜在障碍包括临床前模型不完善、扩大生产规模的挑战、临床试验设计以及转化成本。在此，我们概述肾脏靶向纳米医学领域的现状，并勾勒出一个潜在的临床转化框架。我们重点关注美国食品药品监督管理局批准的纳米药物的路径以及其他纳米医学领域的建议，以指导我们实现转化成功的关键考虑因素。我们还强调学术、临床与行业及联邦监管机构合作的重要性。一些研究性技术刚刚处于迈向临床应用的边缘，因此我们旨在支持这一势头，以改善肾病患者的生活。
