Gene Therapy Studied for Alzheimer's

Delivers nerve growth factor to damaged neurons

FRIDAY, Sept. 24, 2004 (HealthDayNews) -- The use of gene therapy to deliver nerve growth factor into areas of the brain where neurons are degenerating, to reverse the ravages of Alzheimer's disease, is being investigated by Rush University Medical Center scientists.

If this technique proves successful, it could be a major step forward in finding a way to slow the course of Alzheimer's disease.

This approach uses the gene therapy agent CERE-110 to carry the nerve growth factor (NGF) gene. It codes for the NGF protein, which has a positive effect on brain cells, the researchers said.

One of the Rush scientists' key objectives is to deliver CERE-110 and its NGF cargo directly to the basal forebrain, the brain region that's almost always affected by Alzheimer's disease. The scientists don't want CERE-110 to travel to other parts of the brain, where it may cause side effects, they said.

The Chicago-based Rush team has started a two-year Phase I study to evaluate the safety and tolerability of two different doses of CERE-110. The study will include six to 12 people with mild to moderate Alzheimer's disease.

The study is being funded by Ceregene Inc., of San Diego, the developer and manufacturer of CERE-110.

More information

The U.S. National Institute on Aging has more about Alzheimer's disease.

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