From Fox Foundation Database of awarded grants
Abstract
Effects of PYM50028 in Reversing Dopaminergic Neuronal Degeneration and Behavioral Impairments in Parkinson’s Disease

Therapeutics Development Initiative 2008
Objective/Rationale:

The objective of this project is to assess the effects of oral administration of Cogane™ in reversing the changes in the area of the brain involved in Parkinson’s disease and the associated movement disability using two pre-clinical models. In addition, this project will establish the therapeutic dose levels and duration of treatment that provide these benefits in these models to gain essential information on the appropriate dosing and design for a Phase II clinical study in Parkinson’s disease patients.

Project Description:

In the first study, Cogane™ will be administered orally every day for up to two months in an established pre-clinical model of Parkinson’s disease. Beneficial effects will be assessed by taking measurements of biochemical markers specific for the disease. The second study will use a more complex pre-clinical model of Parkinson’s disease. A range of doses of Cogane™ will be administered orally every day for up to 4 weeks and the levels of Cogane™ in the body will be measured. The third study using the same model will utilize the optimal dose of Cogane™, determined from the previous study, administered orally every day for up to 26 weeks. Beneficial effects will be assessed by taking measurements of biochemical markers and recording the movement disability characteristic of the disease.

Relevance to Diagnosis/Treatment of Parkinson’s Disease:

The body naturally produces proteins known as neurotrophic factors that stimulate the growth of nerves. In particular, one known as “GDNF” has been shown to re-grow damaged nerves in areas of the brain involved in Parkinson’s disease. Injection of GDNF in the brain showed beneficial effects in a small clinical study. Cogane™, which can be taken orally, stimulates the body to release GDNF and therefore has the potential to overcome many of the surgical difficulties associated with GDNF administration.

Anticipated Outcome:

This project will provide evidence on the ability of Cogane™ to reverse the changes in the area of the brain involved in Parkinson’s disease and the associated movement disability using pre-clinical models. In addition, this project will provide essential information on the appropriate dosing and design for a Phase II clinical study to assess the potential benefits of orally administered Cogane™ in Parkinson’s disease patients.