HUMAN NEURONAL CELL THERAPY FOR PARKINSONS DISEASE

描述：hNT是有丝分裂后，终末分化的人 可以移植到中枢神经系统的神经细胞 作为治疗神经系统疾病的药物多巴胺能特性 hNT-神经元包括多巴胺合成和代谢的证据， 使其成为其他细胞来源的上级替代品， 开发用于临床。SBIR第一阶段的结果表明， hNT神经元中多巴胺能活性的生理水平和 在神经元产生过程中优化和稳定活动的能力。 在II期，在大鼠6-OHDA和 帕金森病的猴MPTP模型将测量功能性 hNT神经元移植带来的改善。具体地说， 这些实验将确定：（1）剂量-反应功效;（2） 纹状体和黑质同时移植的好处;（3）稳定性 长期结局和（4）在大鼠和非人类中的安全性和有效性 灵长类动物仔细评价是动物模型中需要探索的 临床功能最终目标是发展创新 利用hNT神经元移植技术， 为超过100万患有这种疾病的人提供选择 帕金森氏症。 拟定商业应用： 商业应用-hNT-神经元将在cGMP下生产， 提供用于治疗帕金森病的人神经元细胞， 将这些细胞植入患者的大脑中， 被疾病破坏的细胞恢复了正常功能 hNT神经元将出售给各大医院，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.