Human Neuronal Cell Therapy for Parkinsons Disease

Period of Performance: 09/15/1998 - 08/31/1999


Phase 2 SBIR

Recipient Firm

Layton Bioscience, Inc.
Sunnyvale, CA 94086
Principal Investigator


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.