Gene therapy may slow progression of Alzheimer's, study shows

By Thomas H. Maugh II
Los Angeles Times

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The team, led by Dr. Mark Tuszynski, studied five women and three men, with an average age of about 70, who were in the early stages of Alzheimer's disease.

The study was focused only on determining the safety of the procedure, not its effectiveness. But in the process, the researchers found that the treatment delayed progression of the disease by 40 percent to 50 percent for at least two years, Tuszynski said.

By contrast, the Alzheimer's drugs now available delay progression by only about 5 percent and the effects persist for only about six months, he said.

"If the magnitude of these effects is borne out in larger, controlled trials, this could be a significant advance over existing therapies for Alzheimer's disease," said Tuszynski, who presented the results yesterday at a San Francisco meeting of the American Academy of Neurology.

The approach is based on preliminary studies in rats and monkeys that showed that implanting cells that secrete nerve growth factor into the brain restored atrophied brain cells to near normal size and quantity, and also restored axons connecting the brain cells.

The San Diego researchers collected skin cells from each patient, genetically engineered them to produce nerve growth factor, then implanted them through needles inserted into the center of the frontal lobe, which is important in memory and cognitive function.

One of those patients died five weeks later of a heart attack unconnected to the surgery.

The patients underwent standard cognitive testing before and after the surgery, and the rate of their decline was estimated by physicians. The patients also underwent positron emission tomography imaging, which showed a sharply increased metabolic rate in the region of the brain where the cells were implanted.

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