Science Daily (January 25, 2010): Gene Therapy Study Seeks to Improve Brain Function in Alzheimer's disease

Publication Date: 
Mon, 01/25/2010

Alzheimer's disease (AD) researchers are testing the effectiveness of gene therapy for the first time to treat patients with this common brain disease. Emory University is one of 12 institutions participating in a nationwide study to test the experimental medication, CERE-110.

The Phase 2 clinical trial seeks to enroll a total of 50 study participants with mild to moderate AD.

Previously studied in animals and in a small study to assess safety in humans, CERE-110 appears to induce long-term production of Nerve Growth Factor (NGF) by brain cells. NGF is a naturally occuring protein that helps nerve cells, or neurons, survive in the brain. These neurons produce a chemical, acetylcholine, which plays a vital role in memory and cognitive function.

Since NGF supports the survival and function of the neurons that deteriorate in people with AD, we hope to slow the worsening of their symptoms with this new therapy, says James Lah, associate professor of neurology, Emory University School of Medicine and lead investigator of the study at Emory.