First Gene Therapy for Alzheimer’s

L A   J O L L A, Calif., April 11 -- — California doctors have performed the first gene therapy procedure on an Alzheimer’s patient, in an attempt to use the controversial treatment to try to prevent the neurodegenerative disease.

University of California in San Diego physicians implanted genetically modified tissue on April 5 into a 60-year-old Caucasian woman with the early stages of the disease who asked to remain anonymous.

The therapy is not expected to cure her Alzheimer’s disease but to protect and possibly even restore some of her brain cells. It may also alleviate some symptoms, such as short-term memory loss.

Nerve Growth Factor Gene Transplanted

Scientists added the gene for the brain cell-stimulating molecule, called nerve growth factor, to millions of her cells before putting them into her brain. The hope is that the nerve growth factor will be able to prevent the inevitable cell brain death that occurs with Alzheimer’s.

Doctors performed the 11-hour procedure at UCSD’s John M. and Sally B. Thornton Hospital in La Jolla.

The patient, a former teacher from Oregon, will be monitored to see how the therapy works.

“This is the first attempt to translate this approach from animals to humans and when one makes that step there are always some unknown factors that one encounters,’ says Dr. Mark H. Tuszynski, UCSD neurologist and study leader.

Monkey Studies Support Human Surgery

“The rationale for doing the study is certainly supported by the fact that there is a wealth of information from primate studies that shows that this is an effective approach in preventing aging in monkeys and cell death in monkey brains."

Tuszynski says young monkey brains have dense nervous tissue in the brain. With aging, the density of the connections is reduced by 25 percent.

“If we introduce genetically engineered cells, there is a complete restoration to the level of a young monkey," Tuszynski says. “But monkeys don’t get Alzheimer’s disease and so we don’t know with certainty that this will prevent it …