Researchers Test Gene Therapy for Alzheimer's Disease

Desperate diseases call for innovative measures. That's why a group of specialists around the country are teaming up on what Alan Lerner, MD, calls, "a radically different approach to the treatment of Alzheimer's disease."

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Lerner, a neurologist at Case Western Reserve University and University Hospitals Case Medical Center, says Alzheimer’s patients have abnormally low levels of the brain chemical acetylcholine, a neurotransmitter essential for processing memory and learning. This is because people afflicted with Alzheimer’s experience nerve cell death in a region of the brain where many acetylcholine-producing cells, called cholinergic neurons, are located.

To increase production of acetylcholine, the researchers will deliver the gene responsible for nerve growth factor to the brain. More nerve growth factor, Lerner says, will lead to more cholinergic neurons, which will lead to more acetylcholine—effectively improving memory. The gene therapy won’t cure Alzheimer’s, but researchers believe it could slow the progression of the disease.

An earlier clinical trial suggests this new treatment is safe. Its effectiveness is now being tested in a larger group in a study, funded by the National Institute on Aging, at Case Western Reserve and 11 other sites nationwide.