Abstract:
The U.S. Food and Drug Administration (FDA) provides guidance for expanded access to experimental therapies, which in turn plays an important role in the Twenty-first Century Cures Act mandate to advance cell-based therapy. In cases of incurable diseases where there is a lack of alternative treatment options, many patients seek access to cell-based therapies for the possibility of treatment responses demonstrated in clinical trials. Here, we describe the use of the FDA\'s expanded access to investigational new drug (IND) to address rare and emergency conditions that include stiff-person syndrome, spinal cord injury, traumatic brain stem injury, complex congenital heart disease, ischemic stroke, and peripheral nerve injury. We have administered both allogeneic bone marrow-derived mesenchymal stem cell (MSC) and autologous Schwann cell (SC) therapy to patients upon emergency request using Single Patient Expanded Access (SPEA) INDs approved by the FDA. In this report, we present our experience with 10 completed SPEA protocols.
摘要:
美国食品和药物管理局(FDA)为扩大获得实验疗法提供了指导,这反过来又在二十一世纪治愈法案中发挥了重要作用,以推进基于细胞的治疗。在缺乏替代治疗选择的不治之症的情况下,许多患者寻求基于细胞的疗法,以获得临床试验中证明的治疗反应的可能性.这里,我们描述了使用FDA扩大获得研究新药(IND)来解决罕见和紧急情况,包括僵硬人综合征,脊髓损伤,创伤性脑干损伤,复杂的先天性心脏病,缺血性卒中,和周围神经损伤。我们已使用FDA批准的单例患者扩展访问(SPEA)IND在紧急要求下向患者进行了同种异体骨髓间充质干细胞(MSC)和自体雪旺细胞(SC)治疗。在这份报告中,我们介绍了10个完整的SPEA协议的经验。