Synthetic Oil May Help Patients With Huntington's Disease

Preliminary research shows improvement in brain response

Thursday, January 8, 2015

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Huntington's Disease

THURSDAY, Jan. 8, 2015 (HealthDay News) -- Consuming a synthetic oil may help normalize brain metabolism of people with the incurable, inherited brain disorder known as Huntington's disease, a small new study suggests.

Daily doses of a triglyceride oil called triheptanoin -- which 10 Huntington's patients took with meals -- appeared to boost the brain's ability to use energy, researchers said. The scientists also noted improvements in movement and motor skills after one month of therapy.

Huntington's is a fatal disease causing the progressive breakdown of nerve cells in the brain. Both the study's author and an outside expert cautioned that the new findings are preliminary and need to be validated in larger studies.

Triheptanoin oil "can cross the blood-brain barrier and improve the brain energy deficit" common in Huntington's patients, said study author Dr. Fanny Mochel, an associate professor of genetics at Pitie-Salpetriere University Hospital in Paris.

"We know the gene mutation for Huntington's is present at birth and a key question is why symptoms don't start until age 30 or 40," Mochel said. "It means the body compensates for many years until aging starts. So if we can help the body compensate ... it may be easier to see the delay of disease onset rather than slow the disease's progression."

The study was published online Jan. 7 in the journal Neurology.

About 30,000 Americans exhibit symptoms of Huntington's, with more than 200,000 at risk of inheriting the disorder, according to the Huntington's Disease Society of America.

Each child of a parent with Huntington's stands a 50 percent chance of carrying the faulty gene. The disorder causes uncontrolled movements as well as emotional, behavioral and thinking problems. Death usually occurs 15 to 20 years after symptoms begin.

Mochel and her team broke the study into two parts. In the first part, they used MRI brain scans to analyze brain energy metabolism of nine people with early Huntington's symptoms and 13 healthy people before, during and after they viewed images that stimulated the brain.

The test was repeated one month later. In those without the disease, brain metabolism increased during visual stimulation, then returned to normal. In those with Huntington's, there was no change in their below-normal brain metabolism with visual stimulation.

In the second part, 10 people with Huntington's, including five participants from the first part, received triheptanoin oil three or four times a day. The odorless, flavorless oil contains special fatty acids believed to provide an alternative energy source for the brain, since Huntington's patients do not metabolize glucose properly.

Participants who had consumed the oil for a month underwent the visual stimulation test again, with researchers finding their brain metabolism normal.

But the study was not "blinded," meaning that participants and researchers knew who was receiving the oil. This can lead to the so-called placebo effect, where patients report improvements based on their expectations.

"In one month we saw some improvement [in motor skills]," Mochel said, "but it could be placebo-related because there was no control group."

George Yohrling, director of scientific and medical affairs for the Huntington's society, said the new research was "interesting" and noted that the use of triheptanoin oil appears to be safe, causing no significant side effects.

However, Yohrling said, "It's a really small study and a non-controlled, non-blinded, non-randomized study, which begs to be repeated in a larger, more conclusive manner."

Mochel's upcoming research, scheduled for launch this spring, seeks to accomplish that. It will include 100 Huntington's patients in a randomized study comparing triheptanoin oil to a placebo for six months before allowing all patients to receive the oil.

Yohrling said he feels the most promising research on Huntington's today focuses on drugs created specifically to target the disorder and its gene mutation. This year marks a milestone in Huntington's research, he said, because the first drug ever created specifically for Huntington's disease will be tested in humans.

"I'm more interested and hopeful that the pipeline of Huntington's disease drugs will be slowly filled with drugs specifically created with Huntington's in mind, and not Huntington's as an afterthought," he said.

SOURCES: Fanny Mochel, M.D., Ph.D., associate professor, genetics, Pitie-Salpetriere University Hospital, Paris, France; George Yohrling, Ph.D., director, scientific and medical affairs, Huntington's Disease Society of America, New York City; Jan. 7, 2015, online, Neurology

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