According to a report published in Medscape Medical News, a small proof-of-concept study suggests safety and some potential benefit in terms of efficacy from administration of octanoic acid, a metabolite of the long-chain alcohol 1-octanol, in patients with essential tremor (ET).
A single dosage was well tolerated. The primary desired outcome, postural tremor power measured by accelerometry at 80 minutes, was not met; however, a greater benefit was seen for later periods out to 5 hours.
“We did have some suggestion that there is efficacy, that there is some superiority for octanoic acid in secondary outcome parameters, which mainly are at later time points,” says study author Dietrich Haubenberger, MD, from the National Institutes of Neurological Disorders and Strokes (NINDS).
Haubenberger explains that the octanoic acid begins to take noticeable effect later than expected, anywhere from two and a half hours to five hours. The strongest effect for the right hand was recorded even after five hours.
Alcohol has been long identified as an agent that provides temporary relief from tremor symptoms for patients with ET. Of course, regular use of alcohol to treat ET presents problems. Studies have shown that 1-Octanol, a type of alcohol that is rapidly converted to octanoic acid, is effective in providing tremor relief. Researchers suggest that octanoic acid may be the active substance.
The study, designed to test the safety, efficacy and action of drugs in the body, was conducted to objectively measure tremor rather than functional outcomes or clinical scores.
Dr. Rodger Elble, of Southern Illinois University School of Medicine, is an ET researcher and member of the IETF Medical Advisory Board. As co-moderator of the study session, Dr. Elble remarks that “this was only a single-dose study, and the efficacy of octanol/octanoic acid needs to be confirmed in a longer randomized, placebo-controlled trial.”
At this point, it remains unclear from the study whether all ET patients respond to this treatment or only those that respond to ethanol, a form of alcohol.
These findings were presented at the Movement Disorders Society 15th International Congress of Parkinson’s Disease and Movement Disorders.