EMBARGOED FOR RELEASE UNTIL 4 PM ET, January 07, 2015
Synthetic Oil Drug May Bring Promise for Huntington鈥檚 Disease
MINNEAPOLIS -
An early study suggests that a synthetic triglyceride oil called triheptanoin may provide hope for people with Huntington鈥檚 disease. The study is published in the January 7, 2015, online issue of the medical journal of the . Huntington鈥檚 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鈥檚 disease has a 50 percent chance of developing the disease. Symptoms usually appear between the ages of 30 to 50. 鈥淥ur 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,鈥 said study author Fanny Mochel, MD, PhD, with Piti茅-Salp锚tri猫re University hospital in Paris, France. 鈥淎lthough 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鈥檚 after one month of therapy.鈥 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鈥檚 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鈥檚 metabolism increased during the stimulation, then returned to the normal level. In people with Huntington鈥檚 disease, there was no change in metabolism. For the second part of the study, only people with Huntington鈥檚 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. 鈥淚f confirmed in other studies, the findings may be hopeful for people who have the family gene for Huntington鈥檚 and will eventually develop the disease,鈥 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. To learn more about Huntington鈥檚 disease, please visit