Promise for Parkinson's - updates - FDA approves human use of gene therapy to treat the disease

The US Food and Drug Administration (FDA) has approved the first-ever human use of gene therapy to treat the devastating brain degeneration disorder known as Parkinson's disease.

In a clinical trial slated to begin this month, genes that manufacture a neurochemical called gamma-amino-butyric acid (GABA) will be injected into the brains of 12 willing, late-stage patients who have exhausted standard treatment methods. Researchers hope the treatment will calm tremors and slow progression of the disease. The gene transfer technique is a promising therapy, but extensive human clinical trials will be needed before it's ready for general use on all Parkinson's patients. However, that doesn't leave patients without other immediate options.

A second Parkinson's study, appearing in the October 2002 issue of the Archives of Neurology, concluded that a familiar dietary supplement retards progression of the disease-unlike conventional treatments, which ease symptoms but are not believed to affect the underlying disease process.

The naturally occurring compound known as co-enzyme Q10--or CoQ10--may help stop the nerve cell death that characterizes Parkinson's. CoQ10, also known as ubiquinone, has long been sold over the counter as an antioxidant shown to improve heart function. But Clifford Shults, PhD, and colleagues at the University of California at San Diego gave early-stage patients CoQ10 in doses of 300 mg, 600 mg or 1,200 mg daily--10 to 40 times the usual dosage. By the eighth month, the 23 patients on the highest dose showed significantly less impairment than did all others.

The results indicate that follow-up research at even higher doses should proceed aggressively, says Bernard Ravina, PhD, of the National Institute of Neurological Disorders and Stroke. The institute, based in Bethesda, Maryland, funded the CoQ10 study.

While Parkinsons affects about 1 percent of people over the age of 60, the disease can also strike at younger ages. About 25 percent of the 1.4 million Parkinson's patients in the United States were diagnosed before the age of 40.

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