Chantix® (varenicline)
Varenicline is a partial nicotine receptor agonist used as a smoking cessation aid. Dr. Theresa Zesiewicz of the University of South Florida noticed that the uncoordinated movements (ataxia) and balance problems in a patient with fragile X tremor /ataxia syndrome improved greatly after he started Chantix® 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, including Friedreich’s ataxia, and several of her case reports have been published in medical journals.
INACTIVE: Stages of Development for Chantix
The drug development process can be thought of as a series of stages, and drugs must successfully pass through each stage to become available to patients. This treatment has been evaluated, and the program has been discontinued. Thus, it is not in the pipeline.
June 2009: A Phase II pilot study, sponsored by FARA, began to investigate whether varenicline (Chantix®) improves neurological symptoms, such as balance, coordination, and sensory perception, all of which are significantly impaired in patients with FA.
This study also evaluated the safety of Chantix® in patients with FA. The double blind, randomized, placebo-controlled pilot study was led by principal investigator Dr. Zesiewicz, at the University of South Florida College of Medicine, and co-investigator Dr. David Lynch, at Children’s Hospital of Philadelphia.
Early 2010: The study was stopped as a result of concerns regarding safety and intolerability and insufficient evidence of efficacy. Twenty six subjects had been enrolled into the trial. The primary concern among those who were withdrawn was a worsening of gait and imbalance.