SAN DIEGO — Neurosurgeons have injected genetically modified cells into the brain of an Alzheimer’s patient in a pioneering procedure that holds the hope of halting or reversing brain cell loss caused by the disease.
The 11-hour procedure at the University of California, San Diego marked the first use of human gene therapy in the treatment of brain disease, researchers said Tuesday.
Scientists took skin cells from a 60-year-old Oregon woman in the early stages of Alzheimer’s Disease, isolated genes that secrete a protein found in healthy brains called nerve growth factor and, on Thursday, injected two drops into her brain. She was discharged from the hospital two days later.
“Our hope is that this procedure will be a way of delaying the progress of the disease and improving the quality of life for several years,” said Dr. Mark Tuszynski, who led the study. “It’s unlikely to be a cure.”
Nerve growth factor received federal approval two years ago for human trials after a team of UCSD researchers showed the protein reversed deterioration in the brains of aging monkeys. Another patient will undergo the procedure in three months and researchers are seeking six more candidates for initial studies to determine whether the therapy is safe for humans. Future tests will gauge whether patients maintain their mental abilities.
If the procedure is a success, the implanted cells could begin to improve brain function over the next few weeks, but doctors cautioned that it would take years to determine whether it is a useful therapy for the general public.
Four million Americans have Alzheimer’s disease, which causes a decline in memory and the ability to care for oneself. One in 10 seniors over 65 and nearly half of those over 85 have Alzheimer’s disease, according to the Alzheimer’s Association.
Bill Thies, vice president of medical and scientific affairs for the Chicago-based Alzheimer’s Association, was cautiously optimistic about the new procedure.
“Anytime you start a clinical trial, you don’t know whether the benefits outweigh the risks,” Thies said. “You always want to be cautious at the beginning.”
He noted that Alzheimer’s only afflicts humans, and doctors may not experience the same successes as they had with monkeys. He also said the complexity of the procedure may also be a downside.
“We’re not going to do neurosurgery on 4 million people,” Thies said.
The therapy targeted an area in the brain of the former Oregon schoolteacher known as the cholinergic system, which is important for supporting memory and brain function and deteriorates severely under Alzheimer’s disease. Doctors were able to inject the genes into an area about the size of a Tic-Tac that lies deep within the brain.
The patient remained conscious during the entire procedure and was able to converse with her surgeon, Dr. Hoi Sang U, after the procedure. If the therapy proves to be successful, doctors said, the procedure could eventually be done on an outpatient basis.
Doctors also were hopeful that the procedure could be used for similar treatments for other degenerative brain diseases, such as Parkinson’s and Lou Gehrig’s disease or possibly even the injection of stem sells to restore brain damage, Sang U said.
“If this (therapy) doesn’t work, we have the technology to deliver something else,” he said.