Despite their disadvantages, preclinical models in vitro are still crucial for every area of biomedical science. They remain a necessary basis for biological, biochemical, and mechanistic studies of pathophysiology of human disease, evaluation of diagnostic tests, assessment of vaccines, as well as screening of potential and repurposed drugs before they are adapted to clinical use. In contrast to animal models in vivo, preclinical in vitro models are cost and time effective. They are easier to use, and, in most cases, they are not associated with ethical concerns. Therefore, they are extensively used in cancer research. Conditional cell reprogramming (CCR) has been one of the novel technologies utilized as a preclinical model in vitro for various common cancers and other diseases. It may be even more important for the research related to rare cancers—elusive, difficult to study, and with insufficient number of relevant models available. Applications of this technology for the basic and translational studies of rare cancers are described in this article. Evaluation of the mechanisms of tumorigenicity and metastasis in neuroblastoma, neuroendocrine cervical carcinoma, ependymoma and astrocytoma, as well as screening of potential drugs and other therapeutic approaches for the laryngeal and hypopharyngeal carcinoma and adenoid cystic carcinoma, demonstrate that the CCR technology is a potential reliable model for various aspects of rare cancer research in the future.
尽管存在不足,体外临床前模型在生物医学的各个领域仍至关重要。它们仍是人类疾病病理生理学的生物学、生化学及机制研究、诊断测试评估、疫苗评价,以及潜在药物和再利用药物在临床应用前筛选的必要基础。与体内动物模型相比,体外临床前模型具有成本效益和时间效率。它们更易于使用,且在大多数情况下不涉及伦理问题。因此,它们在癌症研究中被广泛应用。条件性细胞重编程技术已成为多种常见癌症及其他疾病体外临床前模型的新型技术之一。对于罕见癌症的研究而言,这一技术可能更为重要——罕见癌症难以捉摸、研究困难,且相关模型数量不足。本文阐述了该技术在罕见癌症基础与转化研究中的应用。通过对神经母细胞瘤、神经内分泌宫颈癌、室管膜瘤和星形细胞瘤的肿瘤发生和转移机制评估,以及对喉癌、下咽癌和腺样囊性癌的潜在药物及其他治疗方法的筛选,证明了条件性细胞重编程技术有望成为未来罕见癌症多方面研究的可靠模型。
Conditionally Reprogrammed Cells as Preclinical Model for Rare Cancers