Risk factors in the development of stem cell therapy.
Risk factors in the development of stem cell therapy.
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DOI：
10.1186/1479-5876-9-29
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发表时间：
2011-03-22
影响因子：
7.4
通讯作者：
Hermsen HP
中科院分区：
文献类型：
--
作者：
Herberts CA;Kwa MS;Hermsen HP
干细胞疗法有望治疗退行性疾病、癌症和修复目前没有或有限的治疗选择的受损组织。干细胞疗法的潜力早已被认识到，诱导多能干细胞(IPSC)的产生促进了干细胞领域的发展，带来了越来越多的科学知识。尽管基于干细胞的医疗产品具有临床潜力，但也存在潜在的和意想不到的风险。这些风险值得在当前科学知识和经验的视角下进行彻底的讨论。在干细胞类药物临床应用之前，对潜在风险的评估应该是一个先决条件。干细胞医疗产品的风险状况取决于许多风险因素，包括干细胞的类型、分化状态和增殖能力、给药途径、预期的位置、体外培养和/或其他操作步骤、治疗的不可逆性、在发生不可逆组织丢失的情况下同时进行组织再生的必要性/可能性，以及移植细胞的长期存活。这些因素共同决定了与以干细胞为基础的医药产品相关的风险概况。已确定的风险(即在临床经验中确定的风险)或潜在/理论风险(即在动物研究中观察到的风险)包括肿瘤形成、不想要的免疫反应和外来病原体的传播。目前，还没有多能干细胞(即胚胎干细胞和IPSC)的临床经验。基于它们无限自我更新和高增殖率的特点，含有这些细胞的产品相关的风险(例如形成肿瘤的风险)被认为是高的，如果不被认为是不可接受的话。相比之下，在再生医学应用中使用间充质干细胞(MSC)进行的绝大多数小型临床试验没有报告重大的健康问题，这表明MSC疗法可能是相对安全的。然而，在一些临床试验中已经报告了严重的不良事件，这强调了需要更多的知识，特别是关于生物机制和长期安全性的知识。
Stem cell therapy holds the promise to treat degenerative diseases, cancer and repair of damaged tissues for which there are currently no or limited therapeutic options. The potential of stem cell therapies has long been recognised and the creation of induced pluripotent stem cells (iPSC) has boosted the stem cell field leading to increasing development and scientific knowledge. Despite the clinical potential of stem cell based medicinal products there are also potential and unanticipated risks. These risks deserve a thorough discussion within the perspective of current scientific knowledge and experience. Evaluation of potential risks should be a prerequisite step before clinical use of stem cell based medicinal products. The risk profile of stem cell based medicinal products depends on many risk factors, which include the type of stem cells, their differentiation status and proliferation capacity, the route of administration, the intended location, in vitro culture and/or other manipulation steps, irreversibility of treatment, need/possibility for concurrent tissue regeneration in case of irreversible tissue loss, and long-term survival of engrafted cells. Together these factors determine the risk profile associated with a stem cell based medicinal product. The identified risks (i.e. risks identified in clinical experience) or potential/theoretical risks (i.e. risks observed in animal studies) include tumour formation, unwanted immune responses and the transmission of adventitious agents. Currently, there is no clinical experience with pluripotent stem cells (i.e. embryonal stem cells and iPSC). Based on their characteristics of unlimited self-renewal and high proliferation rate the risks associated with a product containing these cells (e.g. risk on tumour formation) are considered high, if not perceived to be unacceptable. In contrast, the vast majority of small-sized clinical trials conducted with mesenchymal stem/stromal cells (MSC) in regenerative medicine applications has not reported major health concerns, suggesting that MSC therapies could be relatively safe. However, in some clinical trials serious adverse events have been reported, which emphasizes the need for additional knowledge, particularly with regard to biological mechanisms and long term safety.
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