Chs Pharma, Inc.

Florida Atlantic University

Jang-Yen Wu

Howard Malcolm Prentice

Dipnarine Maharaj

Gene therapy using expression vectors containing a granulocyte colony-stimulating factor (GCSF) gene are able to protect cells in a cell-based as well as animal-based models for ischemic stroke.

Granulocyte colony-stimulating factor (GCSF) gene therapy for treating neurological diseases

Carbamathione agent protects brain tissue exposed to a cerebral ischemia/reperfusion injury, and minimizes the size of infarcts that develop as a result of the injury.

Treatment for ischemic stroke

Granulocyte colony-stimulating factor (G-CSF; a stem cell enhancer and facilitator), DETC-MeSO (a glutamate receptor partial antagonist and anti-excitotoxicity agent), and sulindac (a potent anti-oxidant and anti-inflammatory agent) each can protect brain tissue exposed to a cerebral ischemia/reperfusion injury, and minimize the size of infarcts that develop as a result of the injury. When administered in combination, these agents are effective at protecting brain tissue and minimizing the size of an infarct resulting from the injury at much lower concentrations compared to using a single agent.

The invention relates to the discovery that in an animal model of Parkinson's disease (PD), administration of granulocyte colony-stimulating factor (G-CSF) to rodents having 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine (MPTP)-induced PD restored the function of dopamine neurons. In these animals, G-CSF treatment increased the number of dopamine neurons in the substantia nigra (SN), G-CSF treatment partially restored the nigrostriatal pathway, and G-CSF restored the function of dopamine to the level before MPTP treatment. The invention also relates to the discovery that treatment of a human patient with corticobasilar ganglionic degeneration, a rare progressive neurological disorder characterized by Parkinsonism and coritcal dysfunction, with G-CSF resulted in a significant improvement in the patient's Unified Parkinson's Disease Rating Scale evaluations as well as measures of activity of daily living. The invention further relates to the discovery that G-CSF treatment of a patient who had suffered an acute stroke resulted in a significant improvement in neurological function, the patient having minimal observable disability seven years later. The methods described herein can be used to treat PD in a mammalian subject (e.g., rodent, human) as well as other neurodegenerative diseases such as Alzheimer's disease, spinal cord injury, and stroke.

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Boca Raton, FL, United States of America

Jang-Yen Wu