UCSD to launch gene treatment for Alzheimer’s disease

The Associated Press
Wednesday February 14, 2001

SAN DIEGO — University of California researchers hope to treat Alzheimer’s patients by using genetically altered cells to rebuild neurons in the brain. 

A study to begin next month will take skin cells from a patient, alter them genetically so that they produce a chemical called nerve growth factor, and then implant them in the same patient’s brain. 

A test of the procedure was found to reverse neuron deterioration in aging rhesus monkeys afflicted with a condition similar to Alzheimer’s, according to neurologists at the UC San Diego Medical Center. 

“This is the start of something that, if it is successful, could literally prevent or at least slow down the progression of the disease,” Dr. Mark Tuszynski, a neurologist heading the research, told The San Diego Union-Tribune. 

Alzheimer’s disease disrupts the way the brain works, affecting the parts of the brain that control thought, memory and language. Neither the cause nor the cure has been found. Currently, one in 10 people over the age of 65 and as many as half those over 85 have Alzheimer’s. 

The benefits of restoring nerve fibers remains to be seen since Alzheimer’s disease also involves other types of brain cell damage and deposits of amyloid plaques. 

“We don’t know enough to say how useful rescuing these neurons will be,” said Dr. Michael Selzer, a University of Pennsylvania neurologist studying nervous system regeneration. “It’s a worthwhile thing to try. It’s possible you would still develop dementia but maybe the memory problem would not be as severe.” 

In a report in Monday’s edition of Proceedings of the National Academy of Sciences, Tuszynski and neuroscientist James Conner found the procedure regenerated connection fibers, called axons, in the brains of five elderly monkeys. 

The improvement lasted at least a year after the altered cells were injected. Tuszynski said he couldn’t predict how long it might last in people. 

Human testing of the technique won federal approval a year ago. But the researchers have found only two appropriate patients after evaluating 500 applicants. They are looking for six more patients for the first phase of the testing. 

To be eligible, Alzheimer’s patients must be in early enough stages of the disease to be able to understand the risks of experimental treatment and must have no other medical problems. 

Tuszynski is a shareholder in a San Diego company, Ceregene, that has a financial interest in any gene therapy technology to emerge from the studies. 

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