Department of Biochemical Sciences "Alessandro Rossi Fanelli", Sapienza University of Rome, Rome, Italy.
Wiley Interdiscip Rev Nanomed Nanobiotechnol. 2024 Nov-Dec;16(6):e2016. doi: 10.1002/wnan.2016.
Over the past two decades, ferritin has emerged as a promising nanoparticle for drug delivery, catalyzing the development of numerous prototypes capable of encapsulating a wide array of therapeutic agents. These ferritin-based nanoparticles exhibit selectivity for various molecular targets and are distinguished by their potential biocompatibility, unique symmetrical structure, and highly controlled size. The hollow interior of ferritin nanoparticles allows for efficient encapsulation of diverse therapeutic agents, enhancing their delivery and effectiveness. Despite these promising features, the anticipated clinical advancements have yet to be fully realized. As a physiological protein with a central role in both health and disease, ferritin can exert unexpected effects on physiology when employed as a drug delivery system. Many studies have not thoroughly evaluated the pharmacokinetic properties of the ferritin protein shell when administered in vivo, overlooking crucial aspects such as biodistribution, clearance, cellular trafficking, and immune response. Addressing these challenges is crucial for achieving the desired transition from bench to bedside. Biodistribution studies need to account for ferritin's natural accumulation in specific organs (liver, spleen, and kidneys), which may lead to off-target effects. Moreover, the mechanisms of clearance and cellular trafficking must be elucidated to optimize the delivery and reduce potential toxicity of ferritin nanoparticles. Additionally, understanding the immune response elicited by exogenous ferritin is essential to mitigate adverse reactions and enhance therapeutic efficacy. A comprehensive understanding of these physiological constraints, along with innovative solutions, is essential to fully realize the therapeutic potential of ferritin nanoparticles paving the way for their successful clinical translation.
在过去的二十年中,铁蛋白已成为一种很有前途的药物输送纳米颗粒,推动了许多能够封装各种治疗剂的原型的开发。这些基于铁蛋白的纳米颗粒对各种分子靶标具有选择性,并具有潜在的生物相容性、独特的对称结构和高度可控的尺寸等特点。铁蛋白纳米颗粒的中空内部允许高效封装各种治疗剂,从而提高其递送和效果。尽管具有这些有前途的特性,但预期的临床进展尚未完全实现。作为一种在健康和疾病中都具有核心作用的生理蛋白,铁蛋白在用作药物递送系统时可能会对生理产生意想不到的影响。许多研究在体内给药时并未彻底评估铁蛋白蛋白壳的药代动力学特性,忽略了生物分布、清除、细胞转运和免疫反应等关键方面。解决这些挑战对于实现从实验室到临床的理想转变至关重要。生物分布研究需要考虑铁蛋白在特定器官(肝脏、脾脏和肾脏)中的自然积累,这可能导致脱靶效应。此外,必须阐明清除和细胞转运的机制,以优化铁蛋白纳米颗粒的递送并降低潜在毒性。此外,了解外源性铁蛋白引起的免疫反应对于减轻不良反应和提高治疗效果至关重要。全面了解这些生理限制以及创新的解决方案对于充分实现铁蛋白纳米颗粒的治疗潜力至关重要,为其成功的临床转化铺平了道路。