An early stage clinical trial of the experimental drug dimebon (latrepirdine) in people with Huntington's disease appears to be safe and may improve cognition. That is the conclusion of a study published today in the Archives of Neurology.
"This is the first clinical trial that has focused on what is perhaps the most disabling aspect of the disease," said University of Rochester Medical Center neurologist Karl Kieburtz, M.D., the lead author of the study. "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."
Huntington's disease is a progressive neurodegenerative disorder that impacts movement, behavior, cognition, and generally results in death within 20 years of the disease's onset. The disease steadily erodes a person's memory and their ability to think and learn. Over time, this cognitive impairment contributes to the loss of the ability to work and perform the activities of daily life. There are no treatments current available that effectively alter the course of the disease or improve cognition.
It is believed that mitochondria - the part of the cell that helps convert food to energy - plays a role in the development of Huntington's disease. Lampridine stabilizes and enhances mitochondrial function, a result that has been shown to improve behavioral, cognitive, and functional outcomes in Alzheimer's disease. Scientists speculate that this may have the same effect in patients with Huntington's disease.