Study Completed
Findings:
This study was negative. Although the drug proved safe and was well tolerated, due to low recruitment rates, there were no significant findings
Background:
Principal Investigators:
Noah Lechtzin, MD MHS
- Department of Medicine, Division of Pulmonary and Critical Care Medicine, Johns Hopkins University
Jeffrey Rothstein, MD PhD, Department of Neurology, Johns Hopkins University
Merit Cudkowicz, MD, Department of Neurology, Harvard University
Coordinating Center:
Johns Hopkins University, Multidisciplinary ALS Center
Participating Sites:
Johns Hopkins Hospital
Massachusetts General Hospital
Background:
Recent evidence suggests that neurotrophic factors may be beneficial in ALS. Buspirone is a commonly used anxiolytic agent. It is generally well tolerated with no severe side effects. Its mechanism of action for treating anxiety is unknown but it has high affinity for serotonin (5-HT1A) receptors. Buspirone may also mimic or stimulate the activity of endogenous neurotrophins such a nerve growth factor (NGF) and brain derived growth factor (BDNF). Because of these actions, preliminary studies have begun in ALS mice. Initial results from Evan Snyder at Harvard University are encouraging, with treated mice demonstrating improvements in pulmonary function compared to untreated littermates. We hypothesize that buspirone treatment will result in short-term benefits in pulmonary function in patients with ALS.
Study Design:
Double-masked, randomized, placebo-controlled clinical trial in which patients are assigned to receive buspirone or placebo. Pulmonary function and respiratory symptoms are followed over the course of this study.
Eligibility:
ALS patients 18 years and older with minimal to moderate pulmonary involvement.
