PRODUCING HUMAN NEURONAL CELLS TO TREAT STROKE

将神经组织移植到功能受损的中枢神经系统中，作为治疗中风和其他神经系统疾病的一种很有希望的方法。该技术的广泛临床应用受到目前适合移植的神经细胞供应有限的限制。为了克服这个问题，莱顿生物科学公司开发了LBS-神经元。这些细胞来源于NT2细胞系，通过形态学、生化和功能标准表现为真正的人类神经元。在临床前研究中，将这些细胞移植到中风动物模型中提供了相当大的治疗价值。目前正在进行一项试点临床试验，以评估lbs -神经元在慢性卒中缺陷患者的中枢神经系统移植的可行性。目前用于产生lbs -神经元的小规模生产过程对临床使用这些细胞的可用性施加了后勤限制。我们建议通过开发和评估几种可替代的细胞培养系统来克服这一限制，这些系统将允许大规模生产lb -神经元。通过这种方式，我们将开始产生质量控制的lbs -神经元，其数量足以进行更广泛的临床试验，并最终广泛应用于临床。提出的商业应用：神经移植是一种很有前途的治疗策略，但它需要合适的细胞来源。lbs -神经元是纯粹的人类神经元细胞，移植到中枢神经系统后具有存活、成熟和功能的能力。它们是根据gmp进行小规模生产的，但为了充分发挥其临床和商业潜力，大规模生产是必要的

Transplantation of neural tissue into the functionally compromised CNS shows great promise as a treatment for stroke and other neurological disorders. The widespread clinical use of this technique is constrained by the current limited supply of neural cells suitable for transplantation. To overcome this problem, Layton Bioscience, Inc. has developed LBS- Neurons. These cells, derived from the NT2 cell line, appear by morphological, biochemical and functional criteria to behave as authentic human neurons. In preclinical studies, transplantation of these cells into animal models of stroke provided considerable therapeutic value. A pilot clinical trial is currently underway to assess the feasibility of CNS transplants of LBS-Neurons in patients with chronic stroke deficits. The current small-scale production process for generating LBS-Neurons imposes logistical limits on the availability of these cells for clinical use. We propose to overcome this limitation by developing and evaluating several alternate cell culture systems that will allow for the large scale production of LBS-Neurons. In this way, we will begin to generate quality- controlled LBS-Neurons in quantities sufficient for more extensive clinical testing and, ultimately, widespread clinical applications. PROPOSED COMMERCIAL APPLICATIONS: Neural transplantation is a promising treatment strategy, but it requires an appropriate source of cells. LBS-Neurons are pure, human neuronal cells, with a demonstrated ability to survive, mature and function when transplanted into the CNS. They are produced on a small-scale according to GMPs, but 1arge-scale production is necessary in order to realize their full clinical and commercial potential