Triheptanoin Oil Shows Promise for Huntington's Disease
Author: American Academy of Neurology
Published: 2015/01/10 - Updated: 2025/12/15
Publication Details: Peer-Reviewed, Research, Study, Analysis
Category Topic: Medical Research - Related Publications
Page Content: Synopsis - Introduction - Main - Insights, Updates
Synopsis: This research, published in Neurology, the medical journal of the American Academy of Neurology, presents preliminary findings from a small-scale study examining whether triheptanoin - a synthetic triglyceride oil composed of special fatty acids - could benefit people in the early stages of Huntington's disease. The study used MRI brain scans on nine early-stage Huntington's patients and 13 healthy controls to measure brain metabolism during visual stimulation, revealing that while healthy participants showed normal metabolic responses, those with Huntington's showed no metabolic change. After one month of taking triheptanoin with meals three to four times daily, the ten Huntington's patients who received the treatment demonstrated normalized brain metabolism and reported improvements in movement and motor skills.
While the researchers caution that the study was not blinded - meaning both researchers and participants knew who was receiving the drug - the findings offer valuable preliminary evidence that alternative energy sources to glucose might address the metabolic dysfunction underlying Huntington's disease, a devastating inherited disorder that causes progressive breakdown of nerve cells controlling movement, cognition, and behavior - Disabled World (DW).
- Definition: Synthetic Triheptanoin Oil
Synthetic triheptanoin oil is a specialized triglyceride composed of three seven-carbon (heptanoic) fatty acid chains attached to a glycerol backbone, manufactured through chemical synthesis rather than extracted from natural sources. This unique medium-chain fatty acid formulation was specifically engineered to treat long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare inherited metabolic conditions where the body struggles to convert certain fats into energy. Unlike typical dietary fats that contain even-numbered carbon chains, triheptanoin's odd-numbered structure allows it to generate both acetyl-CoA and propionyl-CoA during metabolism - the latter being particularly crucial because it can replenish key intermediates in the citric acid cycle, providing an alternative energy source for patients whose normal fat metabolism is impaired. Approved by the FDA in 2020 under the brand name Dojolvi, this medical food has become an essential therapeutic option for managing potentially life-threatening complications like hypoglycemia, muscle weakness, and cardiomyopathy in LC-FAOD patients, though it requires careful administration and monitoring due to gastrointestinal side effects and the need for precise caloric calculations in patient diets.
Introduction
Synthetic Triheptanoin Oil May Help Huntington's Disease
An early study suggests that a synthetic triglyceride oil called triheptanoin may provide hope for people with Huntington's disease. The study is published in the online issue of Neurology, the medical journal of the American Academy of Neurology.
Main Content
Huntington's disease is inherited and causes nerve cells to break down in the brain, especially areas involved in the control of movements, memory and thinking abilities, and emotions and behavior. A child of a parent with Huntington's disease has a 50 percent chance of developing the disease. Symptoms usually appear between the ages of 30 to 50.
"Our study suggests that this drug in the form of oil may be able to improve the brain metabolic profile in early stages of the disease," said study author Fanny Mochel, MD, PhD, with Pitie-Salpatriere University hospital in Paris, France.
"Although the results should be taken with great caution because researchers and participants in the study knew whether or not they were getting the drug, we saw improvement in movement and motor skills in people with Huntington's after one month of therapy."
For the study, researchers used MRI brain scans to analyze the energy profile before, during and after the brain was visually stimulated in nine people in the early stages of Huntington's disease and 13 people without the disease.
The average age of the participants was 46. The test was then repeated one month later. In the people without the disease, the brain's metabolism increased during the stimulation, then returned to the normal level. In people with Huntington's disease, there was no change in metabolism.
For the second part of the study, only people with Huntington's disease received triheptanoin, a compound made up of special fatty acids that can provide alternative energy to glucose in the brain. The 10 participants, which included five of the participants in the first part of the study, had the flavorless, odorless oil during meals three or four times a day for a month. Then they had the visual stimulation test again. The brain metabolism was now normal.
"If confirmed in other studies, the findings may be hopeful for people who have the family gene for Huntington's and will eventually develop the disease," Mochel said.
The study was supported by Ipsen and the French National Institute of Health and Research. Ultragenyx Pharmaceutical Inc. provided the investigational drug triheptanoin for the study.
Insights, Analysis, and Developments
Editorial Note: What makes this research particularly significant for the disability community is its potential to address a genetic condition that affects not just those currently living with symptoms, but also the many family members who carry the gene and face a future diagnosis. Huntington's disease typically strikes during prime working years, robbing people of independence and forcing families into difficult caregiving situations that can last decades. The fact that triheptanoin is a dietary oil rather than a traditional pharmaceutical could mean easier administration and potentially fewer side effects, though much larger controlled trials are needed before any real conclusions can be drawn. Perhaps most importantly, this work demonstrates how understanding the fundamental metabolic problems in neurodegenerative diseases can point toward practical interventions - a research approach that could eventually benefit people with various conditions where brain energy metabolism falters, from certain forms of epilepsy to other movement disorders - Disabled World (DW).Attribution/Source(s): This peer reviewed publication was selected for publishing by the editors of Disabled World (DW) due to its relevance to the disability community. Originally authored by American Academy of Neurology and published on 2015/01/10, this content may have been edited for style, clarity, or brevity.