Biographic Design Consulting, Winston-Salem, NC, USA.
Regen Med. 2012 Nov;7(6 Suppl):89-92. doi: 10.2217/rme.12.69.
The regulatory requirement to demonstrate purity and potency presents a much bigger challenge to regenerative medicine compared with small-molecule drugs and protein biologics because of the desire to introduce living cells into the human body. Any cell population is inherently heterogeneous and bioresponsive - characteristics that make standardization by traditional methods extremely difficult. Standardization is on a 'critical path' to demonstrating purity and potency as I will discuss. Although difficult, I believe standardization is not impossible. In fact, I believe untapped resources of benefit to the regenerative medicine and cell therapy industries exist, particularly in the area of oncology molecular diagnostics. Leveraging the vast amounts of cellular biomarker data that are linked with clinical outcomes and the established reimbursement strategies generated by oncology product development efforts might accelerate the translation of regenerative medicine products from the bench to the clinics both scientifically and financially.
与小分子药物和蛋白质生物制剂相比，由于希望将活细胞引入人体，因此展示纯度和效力的监管要求对再生医学提出了更大的挑战。任何细胞群体本质上都是异质的和生物响应的 - 这些特征使得通过传统方法进行标准化变得极其困难。如我将讨论的那样，标准化是证明纯度和效力的“关键路径”。尽管困难，但我相信标准化并非不可能。事实上，我相信再生医学和细胞治疗行业存在未开发的有益资源，特别是在肿瘤分子诊断领域。利用与临床结果相关联的大量细胞生物标志物数据以及肿瘤产品开发工作所产生的既定报销策略，可能会在科学和财务上加速将再生医学产品从实验室转化为临床。
