Gene therapy for Alzheimer's disease

Doctors at the University of California, San Diego, have surgically implanted genetically modified cells into the brain of a patient suffering from Alzheimer's disease. A 60-year old woman was the first person to have gene therapy to combat the effects of the disease, in a procedure that involved drilling through her skull and injecting cells into her brain during an 11-hour operation.

The therapy is designed to prevent cell loss in the brain. The injection delivered skin cells that had taken from the patient some months before the brain operation, and which had been modified with a natural 'brain-survival molecule' called nerve growth factor (NGF). The procedure targets the area of the brain that supports memory and cognitive function. It has been shown in studies in primates that this part of the brain responds to NGF.

Critics of the trial say that it is not designed to get to the root of the disease itself and that there are already drugs which achieve a similar result as that intended by the operation. Neurologist Mark Tuszynski, who led the study into the procedure, said 'NGF gene therapy is not expected to cure Alzheimer's, but we hope that it might protect and even restore certain brain cells and alleviate some symptoms, such as short-term memory loss, for a period that could last a few years'.

The patient was diagnosed with Alzheimer's three years ago. Her husband said 'we have four children and one grandchild. If there is a genetic trail associated with this disease, we are concerned about their future. Our main motivation is to see if we can contribute to patient care in the future. If there are benefits for my wife, that will be a plus.'