Study: New drug treatment for Huntington's disease shows promise

University of Rochester Medical Center researchers have announced that a new drug treatment for Huntington's disease was well-tolerated and improved cognition in an early stage clinical trial.

The experimental drug dimebon (latrepirdine), being developed by Medivation, Inc. to treat Huntington's disease, appeared to be be effective in improving cognition safely, a recent clinical trial at the University of Rochester Medical Center indicated. Huntington's disease is a progressive, inherited neurodegenerative condition that impairs cognition, behavior and movement, according to the National Institute of Neurological Disorders and Stroke of the National Institutes of Health. Neurologist Karl Kieburtz, lead author of the study, said, "While more investigation needs to be done, these results are encouraging and show, for the first time, a statistically significant benefit in terms of improved cognitive function in patients with Huntington's disease." Kieburtz explained that Huntington's disease, for which no other treatments currently exist that alter the course of the illness or improve cognition, gradually destroys memory and the ability to think and learn, and results in a patient's eventual inability to work or perform the activities of daily life. According to background information provided by Kieburtz and the University of Rochester Medical Center: It is believed that functional impairment of the mitochondria, the part of cells that helps convert food to energy, contributes to the development of Huntington's disease. Latrepirdine is designed to enhance and stabilize mitochondrial function, an approach that has demonstrated improved outcomes in Alzheimer's disease. The team studied the affect of the drug on 91 patients with mild to moderate Huntington's disease during a 90 day period. Half of the participants received the drug and the other half a placebo. A cognitive evaluation test called the Mini-Mental State Examination, often utilized in clinical settings to evaluate the severity and stage of Alzheimer's disease and dementia due to other causes, was given to the patients to measure their orientation, memory and ability to follow commands. The researchers found improved scores for patients taking the drug, but no changes in the scores of those who took the placebo. This study was funded by Medivation, Inc., a San Francisco-based pharmaceutical company, and was published on Feb. 8, 2010, in the Archives of Neurology journal (Vol. 67, Number 2:154-160). Dimebon (latrepirdine) is also being tested in patients with Alzheimer's disease.