Gene therapy performed on Alzheimer's patient

NEW YORK, Apr 10 (Reuters Health) - Doctors have performed the first-ever surgical procedure using gene therapy in the brain of an Alzheimer's patient, in an effort to stave off the effects of the debilitating disease, they announced at a press conference Tuesday in La Jolla, California.

The groundbreaking 11-hour procedure that took place on April 5 involved implanting genetically engineered cells into the brain of a 60-year-old woman from Oregon. The cells, skin cells taken from the patient herself, were engineered to produce a substance called nerve growth factor (NGF).

NGF is known to help restore connections between brain cells that deteriorate with ageing. The cells were grown in the lab to increase their numbers and genetically modified so that they would produce NGF.

The patient was diagnosed with Alzheimer's disease 3 years ago and is in the early stages of the disease.

The quantity of skin cells implanted amounted to about one hundredth of a teaspoon--or roughly two drops--and were implanted in a region of the brain approximately 1.5 centimetres in area, according to the project leader Dr. Mark H. Tuszynski of the University of California-San Diego and the Veterans Administration. The surgical procedure was led by Dr. Hoi Sang U.

The targeted region in the brain, which is called the nucleus basalis of Meynert, "helps regulate the function of all cells throughout the brain, which in turn affects a number of higher intellectual functions," such as memory and the ability to balance a chequebook, for example, Tuszynski told Reuters Health in a telephone interview.

While the technique is not considered to be a cure for the debilitating illness, Tuszynski is optimistic that the newly inserted NGF-producing genes will take over the role of failing cells and delay the rapid decline of brain function that is associated with Alzheimer's disease.

"The hope is to prevent ongoing cell loss in the brain of Alzheimer's disease patients and possibly improve the functioning of remaining cells," he said.

Tuesday, 5 days after the surgery, Tuszynski said that "the patient came through the surgery extremely well and she was released 48-hours after the procedure."

This was the first of eight scheduled surgeries in what is a preliminary clinical trial primarily designed to evaluate the safety of the procedure.

"The next surgery is scheduled to take place 3 months from now, and we have a patient already selected. We are still waiting to select the other six patients," Tuszynski said.

The patient will have her mental abilities evaluated every 3 months and compared to measurements taken just prior to the surgery. Researchers also expect to compare her abilities to known rates of mental decline that have been established for Alzheimer's patients.

Tuszynski also noted that the team plans to use magnetic resonance imaging scans to monitor the appearance and size of the newly implanted cells.

"This approach is based on 15 years of research," which has until now involved various animal studies using rats and monkeys, Tuszynski noted.