Wang Lily Li-Wen, Janes Morgan E, Kumbhojkar Ninad, Kapate Neha, Clegg John R, Prakash Supriya, Heavey Mairead K, Zhao Zongmin, Anselmo Aaron C, Mitragotri Samir
John A. Paulson School of Engineering & Applied Sciences Harvard University Cambridge Massachusetts USA.
Wyss Institute for Biologically Inspired Engineering Boston Massachusetts USA.
Bioeng Transl Med. 2021 Feb 26;6(2):e10214. doi: 10.1002/btm2.10214. eCollection 2021 May.
Cell therapies have emerged as a promising therapeutic modality with the potential to treat and even cure a diverse array of diseases. Cell therapies offer unique clinical and therapeutic advantages over conventional small molecules and the growing number of biologics. Particularly, living cells can simultaneously and dynamically perform complex biological functions in ways that conventional drugs cannot; cell therapies have expanded the spectrum of available therapeutic options to include key cellular functions and processes. As such, cell therapies are currently one of the most investigated therapeutic modalities in both preclinical and clinical settings, with many products having been approved and many more under active clinical investigation. Here, we highlight the diversity and key advantages of cell therapies and discuss their current clinical advances. In particular, we review 28 globally approved cell therapy products and their clinical use. We also analyze >1700 current active clinical trials of cell therapies, with an emphasis on discussing their therapeutic applications. Finally, we critically discuss the major biological, manufacturing, and regulatory challenges associated with the clinical translation of cell therapies.
细胞疗法已成为一种有前景的治疗方式,具有治疗甚至治愈多种疾病的潜力。与传统小分子药物和越来越多的生物制品相比,细胞疗法具有独特的临床和治疗优势。特别是,活细胞能够以传统药物无法做到的方式同时动态地执行复杂的生物学功能;细胞疗法拓宽了可用治疗选择的范围,将关键的细胞功能和过程纳入其中。因此,细胞疗法目前是临床前和临床环境中研究最多的治疗方式之一,许多产品已获批准,还有更多产品正在进行积极的临床研究。在此,我们强调细胞疗法的多样性和关键优势,并讨论其当前的临床进展。特别是,我们回顾了28种全球批准的细胞治疗产品及其临床应用。我们还分析了1700多项当前正在进行的细胞疗法活性临床试验,重点讨论其治疗应用。最后,我们批判性地讨论了与细胞疗法临床转化相关的主要生物学、生产和监管挑战。