MONDAY, April 9 (HealthScout) -- Patients with a rare, crippling nerve disorder could get relief from a natural supplement whose backers have a long history of promising benefits with little proof.
　　Researchers say some people with hereditary ataxia have made revival-style recoveries after taking the off-the-shelf compound, called coenzyme Q10. One patient, a young boy limited to a wheelchair was able to walk after taking the supplement, says a report on the treatment in the April 10 issue of Neurology.

Ataxia is the catchall phrase for a variety of nerve conditions, some with genetic causes. The disorders affect an estimated 150,000 Americans, says the National Ataxia Foundation. The wide range of symptoms include muscle atrophy, loss of coordination and other motor trouble. Ataxia also can cause shrinking of the cerebellum (the brain center that controls fine motor movement), speech difficulties, seizures and retardation.

In the latest study, researchers led by Dr. Salvatore DiMauro, a Columbia University neurologist, identified six patients with ataxia whose coenzyme Q10 (CoQ10) levels averaged about 70 percent lower than normal.

CoQ10, also known as ubiquinone, is a naturally occurring vitamin-like compound that helps cells pry energy from oxygen, a vital part of muscle health.

The molecule is also a powerful antioxidant. Supplements of the substance have been touted for everything from reducing heart attack risk and dental problems to preventing breast cancer. The American Heart Association says it cannot recommend CoQ10 until large studies have shown it to be safe and effective against heart disease. When a toothpaste with CoQ10 hit the market three years ago, saying it could reverse gum disease, the American Dental Association reacted skeptically, saying it had no clinical evidence that it worked. And the National Cancer Institute says a few studies have found that breast-cancer patients seemed to improve after taking CoQ10. However, the NCI says all the studies were flawed: None had a control group -- a standard practice for comparison purposes, in which one set of patients doesn't get the remedy under investigation; the number of patients was small, and everyone was taking several other drugs in addition to the CoQ10, so it was hard to tell exactly what was helping.

While the claims for other ailments have yet to be proven, CoQ10's effects on ataxia appear more solidly based.

The patients received 300 milligrams daily of CoQ10 supplements, building up to 3,000 milligrams a day as the treatment began to show promise. After a year, all had improved scores for coordination, balance and speech, the researchers say.

The average gain was 25 percent, but some patients saw far more radical improvement. The researchers say five patients who were unable to walk before the study could do so after a year on the supplement, albeit with the help of a person or a walker; one previously home-bound young woman is now able to go to work.

"These are the only patients [with CoQ10-deficiency ataxia] we found, and they all responded" to the therapy, DiMauro says. What's more, "there was no question that they had improved. It was quite impressive," he says.

Since submitting their study for publication, DiMauro's group has located a New York family with children suffering from CoQ10-related ataxia. They also are doing well on supplement therapy, DiMauro says.

Not everyone with ataxia would get help from the supplement, the researchers say. Dr. Jerry Mendell, a neurologist at Ohio State University in Columbus and a co-author of the study, says only a "very small fraction" of ataxia patients have CoQ10 deficiency. "It's not a common condition."

Even so, DiMauro says the findings may spur doctors to look more closely for the nutrient problem. He says early treatment is important because patients seem to respond less well to treatment as the condition progresses.

DiMauro says the variable severity of ataxia might reflect problems at one or more of the dozen steps in the complex natural synthesis of CoQ10 in the body. He and his colleagues are planning to look more closely at the life cycle of the molecule, and how changes in its structure affect the health of cells.
　　

From HealthScout