干细胞科学的未来会是什么样子?
乔-安娜·瑞姆斯博士.D., 犹他大学血液学和恶性血液学部门的研究副教授, was an important part of a discussion related to that question recently at the Regenerative Medicine Partnering Forum, 在加利福尼亚举行的年度活动. 该论坛是名为“高原干细胞会议”的更广泛倡议的一部分.
由再生医学联盟组织, 加州再生医学研究所和桑福德再生医学联盟, the recent Stem Cell Meeting on the Mesa brought together the regenerative medicine industry with the scientific research community to advance discussion about moving stem cell science into cures.
除了作为研究副教授的工作, Reems —who presented at the conference — also serves as scientific director of the University of Utah Health Care's Cell Therapy and Regenerative Medicine Facility (CTRM).
她在访谈中分享了自己对新兴再生医学领域的看法&一个与 健康饲料.
问：您从事再生医学领域的工作. 对于那些不熟悉这个主题的人, how would you describe what you do on a day-to-day basis as well as what the field of regenerative medicine entails?
开始这个讨论, 大发娱乐需要定义什么是“再生医学”, 修复和/或替换受损或有缺陷的细胞和组织的一个相对较新的领域. The first successful regenerative medicine therapy occurred in 1956 when stem cells from the bone marrow of an identical twin were used to restore their sibling's bone marrow after they had undergone irradiation. 移植的干细胞, 能生长和分化成许多不同细胞类型的, succeeded in achieving a complete remission of the sibling's leukemia by replenishing the cellular elements of their blood stream.
起初，干细胞是从正常供体的骨髓中获得的, 后来，他们从动员到供体外周血中的干细胞中获得. 和, 1990年左右, a third source of stem cells from umbilical cord blood was reported that facilitated the restoration of hematopoiesis. 所有这些干细胞来源通常用于恢复造血细胞, 但也被用于非造血疾病. 最近, 具有恢复非造血细胞能力的干细胞的其他来源已被确定. 这些干细胞来源包括胚胎干细胞, 诱导多能干细胞（iPS）和多能间充质基质细胞（MSCs）. The potential of these stem cells as treatment modalities to repair and replace defective cells in a variety of disease states are currently under study. 特别是, there are numerous clinical trials exploring the potential of the MSC to treat disease states that involve inflammatory bowel disease, 肌肉骨骼损伤, 肝损伤, 烧伤和皮肤损伤, 脊髓损伤.
The University of Utah's CTRM was first established in 1990 to support the blood and marrow transplant (BMT) programs at the Huntsman Cancer Institute and Primary Children's Hospital. The day-to-day efforts of staff in supporting the BMT program include the collection of stem cells from donors/patients, transporting products to the CTRM facility where the quality of products is assessed and the stem cell product is prepared for infusion into patients. 在某些情况下, stem cell products are frozen and stored at <-150 °C for later use for transplantation.
To include applications of novel cellular therapy products for the repair of diseased or injured non-hematopoietic tissues, the scope of the CTRM was expanded in 2006 by leveraging the infrastructure already in place for the blood and marrow transplant program. 这个扩展包括, 但并不局限于为治疗肌萎缩性侧索硬化症等疾病准备细胞, 肝损伤，背部疼痛等. 大发娱乐提供新的细胞疗法是一个漫长的过程, arduous and labor intensive endeavor that ultimately leads to filing an investigational new drug (IND) application with the FDA to perform clinical trials. Steps along this process include transfer of technology from an academic or industrial partner to the CTRM and then optimization of procedures by the CTRM that outline the procurement, 隔离, 文化战略, 细胞的冷冻、储存方法及运输. 所有这些都在提交IND申请之前完成. Upon IND activation and under cGMP conditions products are then prepared for use in a clinical trial by the CTRM.
问：你为什么对再生医学充满热情? 是什么把你吸引到这个领域来的? 你喜欢你工作的哪一点?
The healing abilities of the body are amazing and the possibility that it may one day be possible to harness this capability is not only fascinating, but also carries a reward of having the potential to profoundly influence the quality of life and possibly the life expectancy of individuals. What I love the most about my job is that it is a privilege to be part of this cutting edge science and to have the opportunity to work with highly dedicated and skilled individuals at the CTRM.
问：目前再生医学领域面临的挑战和紧迫问题是什么?
A major challenge for the field of regenerative medicine is overcoming the difficulty in getting novel cell therapies to patients. 由于美国严格的监管.S.在美国，一种新的细胞疗法正在对患者进行治疗.S. 是否落后于其他国家. 因此，在国外寻求干细胞治疗的患者有所增加.
A major challenge for the CTRM is obtaining financial support that will accelerate innovations focused on the development of regenerative medicine in Utah as well as establishing working teams at the University of Utah that are focused on translating novel cell therapies specifically for treating patients under various medical disciplines. 例如, core working teams dedicated to delivering a cell therapy for 肝损伤 under gastroenterology or for macular degeneration under ophthalmology.
问：你认为干细胞的下一个重大突破会是什么? 地平线上有什么?
哇，有这么多的可能性. 鉴于目前正在进行的MSCs临床试验的数量, I expect that the next big breakthrough will be that these cells have successfully and reproducibly alleviated the symptoms of several specific diseases.
On the horizon is the development of new culturing technologies that support in vitro self-renewal of stem cell populations. The development of such new methods will permit growing stem cells in the laboratory in large quantities that will be sufficient for clinical use.
问：犹他大学的再生医学项目有什么重要意义? 这个项目取得了哪些突破, 你认为未来的发展方向是什么?
再生医学领域的增长主要是在公共和私营部门. The CTRM at the University of Utah is one of only a few academic centers with a Cell Therapy and Regenerative Medicine facility. Future goals of the CTRM include developing and delivering novel cell products via university collaborations and business partnerships as well as to build an internal core group of scientist, 临床医生和生物工程师专注于发展, engineering and providing patients with the highest quality clinical grade cellular and tissue engineered products.