Huntington's disease marker emerges in clinical trial

Huntington's disease is an inherited disorder with a flaw in a gene on chromosome 4 that leads to wasting of the tissue in the brain. This causes a range of symptoms, including mood changes, problems with balance and movement, and dementia, and it is generally fatal within 20 years. There are only a few treatments for the symptoms, and no cure. A study of Coenzyme Q10 (CoQ), sometimes used as an antioxidant food supplement, showed a clue to a potential biomarker as well as signs of activity for the treatment of the disease.

While it's not clear what causes Huntington's disease, one theory is that the genetic change behind the disorder causes abnormal proteins to build up in brain cells, which then leads to oxidative stress and cell death. CoQ is an antioxidant that could potentially reduce this cell damage. In the "Pilot Safety and Tolerability Study of Coenzyme Q10 in Huntington Disease and in Normal Subjects" (Pre-2CARE) study, the researchers looked at levels of 8-hydroxy-2'-deoxyguanosine (8OHdG) in 20 people with Huntington's disease, before and after treatment with CoQ. They found that some of the symptoms improved and the levels of the biomarker, which signals oxidative stress, fell about 20%. The data were published in the first issue of the new Journal of Huntington's Disease.

"This study supports the hypothesis that CoQ exerts antioxidant effects in patients with Huntington's disease and therefore is a treatment that warrants further study," says Dr. Kevin Biglan, a University of Rochester Medical Center neurologist and lead author of the study. "As importantly, it has provided us with a new method to evaluate the efficacy of potential new treatments."

As well as supporting the use of CoQ, a low-cost and widely used supplement in this fatal neurological disorder, this biomarker could also be used in screening for and monitoring other potential treatments for this and related diseases. A Phase III trial is under way.

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