Current status and perspectives of patient-derived rare cancer models
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REVIEW ARTICLE
Current status and perspectives of patient‑derived rare cancer models Tadashi Kondo1 Received: 26 April 2020 / Accepted: 7 June 2020 © Japan Human Cell Society 2020
Abstract Malignancies with extremely low incidences, such as less than 6 per 100,000 people annually, are defined as rare cancers. Approximately 200 malignancies are classified in this category, therefore the total number of patients with rare cancers is greater than that of patients with any single common cancer. However, because of the small numbers of patients, novel therapies have not been developed for individual rare cancers, and clinical outcomes remain dismal. Patient-derived cancer models are indispensable for both basic and pre-clinical studies, and their roles will increase in the era of post-genome medicine. Although patient-derived cancer models have long been used in oncology, they are not well developed for rare cancers. In the context of sarcoma, the presently available cell lines and xenograft models are limited and do not satisfy the needs of research. Indeed, the lack of effective therapies for rare cancers might be attributable to the paucity of adequate patient-derived cancer models for pre-clinical studies. To facilitate the establishment and availability of patient-derived rare cancer models, we need to create effective methods for model establishment, share the valuable clinical samples and established models, and implement guidelines to record the clinical data of donor patients and original tumors. Patient-derived rare cancer models are a public resource, and they should not be used exclusively but should rather be shared among the research community. Keywords Patient-derived cancer model · Rare cancer · Cell line · Organoid · Xenograft
Rare cancers and efforts towards better clinical outcome Rare cancers are defined as malignancies with an incidence of less than 6 per 100,000 people per year. Despite this nomenclature, they are never rare. Approximately 200 different malignancies are collectively categorized as rare cancers [1], and 24% and 15% of all malignancies are represented by rare cancers in Europe [2] and Japan [3], respectively. Therefore, the total number of patients with rare cancers is more than that of any single common cancer. Moreover, the overall incidence of rare cancers has increased annually [2]. Curiously, all childhood cancers are rare, and overall, patients with rare cancers are younger than those with common cancers [4]. Improved clinical outcomes for patients with rare cancers are thus an urgent issue.
* Tadashi Kondo [email protected] 1
Division of Rare Cancer Research, National Cancer Center Research Institute, 5‑1‑1 Tsukiji, Chuo‑ku, Tokyo 104‑0045, Japan
Because rare cancers are defined according to patient numbers, they demonstrate heterogeneous clinical features and molecular backgrounds. However, rare cancers share common problems that stem from their extremely low incidence and prevalence. For example, because of the paucity of information regarding diagnosis and treatm
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