Abstract

Nanomedicine, as a relatively new offshoot of nanotechnology, has presented vast opportunities in biomedical research for developing novel strategies to treat diseases. In the past decade, there has been a significant increase in in vitro and preclinical studies addressing the benefits of nanomedicines. In this commentary, we focus specifically on the efficacy- and toxicity-related translational challenges of nanocarrier-mediated systems, and briefly discuss possible strategies for addressing such issues at in vitro and preclinical stages. We address questions related specifically to the balance between toxicity and efficacy, a balance that is expected to be substantially different for nanomedicines compared to that for a free drug. Using case studies, we propose a ratiometric assessment tool to quantify the overall benefit of nanomedicine as compared to free drugs in terms of efficacy and toxicity. The overall goal of this commentary is to emphasize the strategies that promote the translation of nanomedicines, especially by learning lessons from previous translational failures of other drugs and devices, and to apply these lessons to critically assess data at the basic stages of nanomedicinal research.

Highlights

  • The last two decades have witnessed the advent of nanomedicines - a powerful new component of nanotechnology that presents new opportunities and challenges in almost all specialties of medicine

  • We will address questions related to the balance between toxicity and efficacy, a balance that is expected to be substantially different for nanomedicines compared to that for a free drug

  • How do we determine whether a specific nanomedicine is better? Should the efficacy of the nanoformulation be better than the free drug? Or should the adverse effect of the former be lower than that of the latter? Or both? we argue that the benefits of nanomedicines should be considered taking both the efficacy and adverse effects of the nanomedicine and the free drug together

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Summary

Introduction

The last two decades have witnessed the advent of nanomedicines - a powerful new component of nanotechnology that presents new opportunities and challenges in almost all specialties of medicine. While the objective of increasing efficacy and decreasing toxicity seems rather straightforward, the process, is fraught with challenges that are technical, biological and financial in nature In this commentary, we will focus on the efficacy- and toxicity-related translational challenges of nanocarrier-mediated drug delivery systems and briefly discuss possible strategies for addressing these issues at the in vitro and preclinical stages before attempting human translation. Platinum is lately being re-examined as a nanoparticle carrier for various biological applications, including cancer [18,19] It is well-known clinically that platinum is highly nephrotoxic [20,21], and one can pre-emptively predict that attempts to translate a platinum-based nanomedicinal formulations from the bench-side to the bed would pose nephro-toxicity challenges. We wish to take up one such basic evaluation at the in vivo and in vitro levels that could possibly add to the overall assessment of a nanomedicine

The Efficacy and Toxicity Dilemma
Case Study One
Case Study Two
Conclusion

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