Granjeiro José Mauro, Borchio Priscila Grion de Miranda, Ribeiro Icaro Paschoal Brito, Paiva Katiucia Batista Silva
Division of Biological Metrology, The National Institute of Metrology, Quality, and Technology, Duque de Caxias 25250020, Rio de Janeiro, Brazil.
Laboratory of Regenerative Medicine, Arthur Sá Earp Neto University, Petropolis 25680120, Rio de Janeiro, Brazil.
World J Stem Cells. 2024 Oct 26;16(10):860-872. doi: 10.4252/wjsc.v16.i10.860.
The burgeoning field of bioengineering has witnessed significant strides due to the advent of stem cell models, particularly in their application in advanced therapy medicinal products (ATMPs). In this review, we examine the multifaceted impact of these developments, emphasizing the potential of stem cell models to enhance the sophistication of ATMPs and to offer alternatives to animal testing. Stem cell-derived tissues are particularly promising because they can reshape the preclinical landscape by providing more physiologically relevant and ethically sound platforms for drug screening and disease modelling. We also discuss the critical challenges of reproducibility and accuracy in measurements to ensure the integrity and utility of stem cell models in research and application. Moreover, this review highlights the imperative of stem cell models to align with regulatory standards, ensuring using stem cells in ATMPs translates into safe and effective clinical therapies. With regulatory approval serving as a gateway to clinical adoption, the collaborative efforts between scientists and regulators are vital for the progression of stem cell applications from bench to bedside. We advocate for a balanced approach that nurtures innovation within the framework of rigorous validation and regulatory compliance, ensuring that stem cell-base solutions are maximized to promote public trust and patient health in ATMPs.
由于干细胞模型的出现，生物工程这一新兴领域取得了显著进展，尤其是在其应用于先进治疗药品（ATMPs）方面。在本综述中，我们考察了这些进展的多方面影响，强调了干细胞模型增强ATMPs复杂性以及为动物实验提供替代方案的潜力。源自干细胞的组织特别有前景，因为它们可以通过为药物筛选和疾病建模提供更具生理相关性和伦理合理性的平台来重塑临床前研究格局。我们还讨论了测量中可重复性和准确性的关键挑战，以确保干细胞模型在研究和应用中的完整性和实用性。此外，本综述强调了干细胞模型符合监管标准的必要性，确保在ATMPs中使用干细胞转化为安全有效的临床治疗方法。随着监管批准成为临床应用的途径，科学家和监管机构之间的合作努力对于干细胞应用从实验室到床边的进展至关重要。我们倡导一种平衡的方法，即在严格验证和符合监管要求的框架内培育创新，确保基于干细胞的解决方案得到最大限度利用，以促进公众对ATMPs的信任和患者健康。
