Gene Therapy for Alzheimer’s

(Ivanhoe Newswire) -- The first study to use gene therapy to treat Alzheimer’s disease shows the therapy markedly reduces the rate of cognitive decline seen in these patients.

Researchers from the University of California, San Diego School of Medicine tested the procedure on eight patients with early Alzheimer’s disease. First, researchers collected skin cells from the patients and grew them in the laboratory to express nerve growth factor (NGF) -- a protein known to help keep cells healthy and prevent cell death.

Then, they implanted the cells into the patients’ brains. Patients were followed for an average of 22 months. Results showed the rate of mental decline, as measured by a standard test, was reduced up to 51 percent. Brain scans also showed increased metabolic activity in the patients’ brains, which researchers say indicates they were healthier than they were prior to the procedure.

The investigators did learn an important lesson during the experiment, however. Initially, patients were implanted with the cells while under only light sedation. Two of the patients moved during the procedure, which caused bleeding in the brain, and one died. Subsequently, the researchers redesigned their method to implant the cells only on patients who were under general anesthesia.

The authors write, “We find that NGF gene delivery in the human brain has no long-term adverse safety consequences. However, subjects must be anesthetized to allow safe completion of the initial stereotaxic delivery procedure.”

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SOURCE: Nature Medicine, published online April 24, 2005