HUMAN NEURONAL CELL THERAPY FOR PARKINSONS DISEASE

描述：HNT是有丝分裂后、终末分化的人类 可以移植到中枢神经系统的神经细胞 作为一种治疗神经疾病的方法。多巴胺能特性的研究 包括多巴胺合成和代谢的证据 体外培养，使其成为比其他细胞来源更好的替代品 为临床使用而开发。SBIR第一阶段结果显示 HNT神经元的多巴胺能活动的生理水平和 在神经元产生过程中优化和稳定活动的能力。 在第二阶段，一系列对照良好的大鼠6-OHDA和 帕金森病猴子MPTP模型将测量功能 HNT神经元移植带来的改善。具体来说， 这些实验将确定：(1)剂量-反应效果；(2) 纹状体和黑质同时移植的益处；(3)稳定性 长期疗效和(4)大鼠和非人类的安全性和有效性 灵长类动物。仔细评估是有必要探索的动物模型 临床功能。我们的最终目标是发展创新 HNT神经元移植技术在临床上的可行性 为100多万人提供的选择 帕金森氏症。 建议的商业应用： 商业应用-HNT神经元将在cGMPs下生产，以 通过以下途径提供治疗帕金森氏病的人类神经细胞 将这些细胞移植到患者的大脑中以取代细胞 已经被疾病破坏，从而恢复了正常的功能。 HNT-神经元将被出售给各大医院并由 神经外科医生。

DESCRIPTION: hNT are post-mitotic, terminally differentiated human neuronal cells that can be transplanted into the central nervous system as a treatment for neurological disorders. The dopaminergic properties of hNT-neurons include evidence of dopamine synthesis and metabolism in vitro, make them a superior alternative to other cell sources in development for clinical use. The SBIR Phase I results demonstrated physiological levels of dopaminergic activity in hNT neurons and the ability to optimize and stabilize the activity during neuron production. In Phase II, a well controlled series of studies in the rat 6-OHDA and monkey MPTP models of Parkinson's disease will measure functional improvements resulting from transplants of hNT neurons. Specifically, these experiments will determine: (1) dose-response efficacy; (2) benefits of simultaneous striatal and nigral transplants; (3) stability of long-term outcomes and (4) safety and efficacy in rats and non-human primates. Careful evaluation is animal models in necessary to explore clinical function. The ultimate goal is to develop the innovative transplantation technology using hNT neurons into a viable clinical option for the more than 1 million individuals afflicted with Parkinson's disease. PROPOSED COMMERCIAL APPLICATION: Commercial application - hNT-Neurons will be produced under cGMPs to provide human neuronal cells for treating Parkinson's disease by implanting these cells into the brains of patients to replace cells which have been destroyed by the disease thus restoring normal function. hNT-Neurons will be sold to major hospitals and implanted by neurosurgeons.