Huntington disease (HD) is an inherited neurodegenerative disease that affects more than 30,000 people in both the United States and Canada. HD is most often considered a movement disorder, but it also causes emotional and behavioral changes and cognitive decline. The symptoms associated with HD usually become more severe over time. Currently, there is no cure for HD, but there are medications to manage symptoms. In a multicenter phase III clinical study, researchers, including a VCU professor, will conduct research on a novel drug for the treatment of chorea in HD.
The Huntington Study Group (HSG), under the leadership of Samuel Frank, M.D., principal investigator of the study and associate professor of neurology from Boston University School of Medicine, and Claudia Testa, M.D., Ph.D., director of theVirginia Commonwealth University HD Program, the Joan Massey Chair in Clinical Parkinson’s Disease, associate professor of neurology in the VCU School of Medicine and the study’s co-principal investigator, is researching the drug, SD-809 ER, in the treatment of HD chorea. The new trial will be conducted at sites across the United States and Canada.
The study, sponsored by Auspex Pharmaceuticals Inc., will examine the efficacy, safety and tolerability of SD-809 ER for the treatment of chorea associated with HD.
HD is characterized by brain cell death that usually begins between the ages of 30 and 50. This cell loss results in motor, cognitive and behavioral signs and symptoms. Chorea, from the Greek “to dance,” is a type of involuntary movements and is a hallmark of the disease.
SD-809 ER has the same mechanism of action as the FDA-approved drug tetrabenazine, which is used to lesson chorea in people with HD. Because SD-809 ER is more slowly metabolized than tetrabenazine, it provides more consistent and predictable drug levels and can be given less often. The study will examine, in part, if a lower overall dose of SD-809 ER can be administered, which may be more convenient for persons with HD.
“I am excited to work with my colleagues in the Huntington Study Group to examine this new drug for the treatment of Huntington disease chorea,” said Testa. “Currently, there is only one approved treatment option available for chorea. We hope this study leads to a new option for our patients.”
Source: Virginia Commonwealth University