A drug approved for smoking cessation may hold promise for people suffering from Friedreich’s Ataxia (FA), an inherited disease that causes progressive damage to the neuromuscular system. A new clinical study, sponsored by the Friedreich’s Ataxia Research Alliance (FARA), will investigate whether varenicline (Chantix®) improves neurological symptoms, such as balance, coordination, and sensory perception, all of which are significantly impaired in patients with FA. Chantix® is approved by the U.S. F ood and Drug Administration to help cigarette smokers stop smoking. Currently, there is no effective treatment for FA.
The double blind, randomized, placebo-controlled pilot study will be led by principal investigator Dr.Theresa Zesiewicz, professor of neurology at the University of South Florida College of Medicine, and co-investigator Dr . David Lynch, associate professor of neurology and pediatrics at Children’s Hospital of Philadelphia. Dr. Zesiewicz noticed that the uncoordinated movements (ataxia) and balance problems of a patient with fragile X tremor /ataxia syndrome imp roved greatly after he started varenicline in an attempt to quit smoking. The symptoms worsened when the medication was discontinued. Dr. Zesiewicz found similar results when treating patients with other types of ataxia, and several of her case reports were published last year in medical journals.