First Surgery in Gene Therapy Trial for Alzheimer's

In a groundbreaking 11-hour procedure, doctors at the University of California, San Diego, School of Medicine (UCSD, USA) have surgically implanted a naturally occurring protein called nerve growth factor (NGF) into the brain of a patient with Alzheimer's disease. NGF promotes nerve cell growth and survival.

Several months earlier, a sample of the patient's own skin cells was collected in a biopsy and NGF genes isolated from the nervous system were inserted. Over a three-month period, the genetically engineered cells grown in culture divided and increased in number, producing large quantities of NGF. The patient received five implants of these modified cells, targeting a region at the base of the frontal lobe containing cholinergic cells, which undergoes profound degeneration in Alzheimer's patients.

Precise coordinates of the targets in the brain were determined by using a stereotaxic head frame, and special surgical instruments were used to insure that cells would be injected in the right location. Magnetic resonance scanning further verified the targeted area. After making a small hole on the right side of the patient's skull, the genetically modified cells were inserted through a fine needle. The patient will be closely monitored for any adverse events over the next few weeks and doctors will continue measurements of cognitive function.

The current trial is based on research conducted over the past 12 years in rats and monkeys with NGF. In February 2001, the research culminated in a finding that axons were restored in primate brains using genetically modified tissue implants. Previously, the axons had shriveled up and disappeared in aged monkeys. However, in monkeys that received the NGF cells, the axons were restored to normal levels and sometimes exceeded those levels.

"NFG gene therapy is not expected to cure Alzheimer's disease, 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,” said UCSD neurologist Mark H. Tuszynski, who lead the study and who conducted much of the earlier research.




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