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Gene Therapy May Offer Hope For Alzheimer's Patients

April 25, 2005
Experimental gene therapy on a small number of people with Alzheimer's disease suggests it may be effective in slowing the disease's effects, according to researchers. The study is detailed in the journal Nature Medicine.

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In the clinical trial, eight patients in the early stages of Alzheimer's received gene therapy. After 22 months, six of the eight had a slower rate of cognitive loss, as measured by standard medical testing.

For the trial, the researchers used a gene that controls production of nerve growth factor, a naturally occurring protein that protects brain cells.

Lead author of the study, Mark H. Tuszynski of the University of California, San Diego, said the substance had been used in treatment of other diseases, with mostly negative effects. He said this was the first use of the protein - or of any gene - to treat Alzheimer's. Tuszynski is also a principal of Ceregene, the company holding the rights to this particular gene therapy technique.

Gene therapy has yet to yield any real results in the decade and a half it's been tried, but Tuszynski and his colleagues say there is reason to be hopeful in this case.

They say by using the gene to trigger natural growth factor production in the brain, precisely at the points where it's needed, the brain may be able to withstand the disease that is relentlessly degenerative, and inevitably fatal.

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