Huntington's Could Be Treated With Synthetic Oil

A synthetic triglyceride oil could help treat those suffering from Huntington's disease.

Huntington's disease is an inherited condition that cause nerve cells to break down in the brain, affecting functions such as movement, memory and behavior, the American Academy of Neurology reported.

"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 Dr. Fanny Mochel, of Pitié-Salpêtrière University hospital in Paris. "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."

To make their findings the researchers looked at MRI scans of nine people in the early stages of Huntington's disease and 13 people without the disease. The brain images were taken while the participants were experiencing visual stimulation. The team found the brain's metabolism increased during visual stimulation in patients with healthy brains, but did not do so in those with Huntington's.

In the second part of the study, patients suffering from Huntington's received triheptanoin, a compound made of fatty acids that can act as an alternative to the brain's glucose. After receiving the treatment (which is administered as a flavorless oil included in meals), the patients showed increases in brain metabolism during visual stimulation.

"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 provided the investigational drug triheptanoin for the study. The research was published in a recent edition of the journal Neurology.

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American Academy of Neurology
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