Neurotrophic Agents for Treating Parkinson's Disaese

DESCRIPTION provided by applicant Parkinsonandapos s disease PD is a neurodegenerative disorder characterized by motor function and eventually cognitive decline GM depletion is validated as a therapeutic target for Parkinsonandapos s disease PD in clinical studies in which GM replacement stabilized motor function loss and may be disease modifying However GM has poor pharmacologic properties that include poor potency mg SC bid man and aqueous solubility mg in mL no oral bioavailability and limited blood brain barrier penetration crosses the BBB that makes continuous dosing by injection painful patient compliance difficult and chronic treatment undesirable for this devastating disease Our goal in this program is to assess GM analogs for their pharmacodynamic properties in a PD mouse model validate their potential efficacy for PD using a genetic PD mouse model and ultimately proceed to nonclinical ADMET studies and an IND PUBLIC HEALTH RELEVANCE GM depletion is validated as a therapeutic target for Parkinsonandapos s disease PD in preclinical and clinical studies in which GM replacement was capable of modifying the motor losses resulting from the disease state However GM has poor pharmacologic properties that make continuous dosing by injection painful patient compliance difficult and chronic treatment undesirable for this devastating disease We have developed novel potent orally bioavailable analogs of GM which can solve this road block and become the first disease modifying treatment for PD