Grant: Proof of Principle Program Phase I - Drug Development

Huntington's disease (HD) is the most common inherited neurodegenerative disorder and it is characterized by progressive motor dysfunction, cognitive decline and psychiatric symptoms. The disease is caused by a mutation in a protein, huntingtin, that affect the function and survival of brain cells. Currently, there is no cure for this disease. We have recently discovered that the production of gangliosides, lipid molecules that are important for normal functioning of brain cells, is impaired in HD. Our studies will explore the therapeutic application of various gangliosides in HD. We will determine their ability to decrease the toxicity of mutant huntingtin and HD symptoms in animal models. We will also determine the best route of administration of these potential drugs and their distribution in the brain of HD animal models. Our studies will be instrumental to the development and the commercialization of new treatments for HD.