Alzheimer”s research targets gene therapy

WASHINGTON – The first attempt at gene therapy for Alzheimer’s patients appeared to significantly delay worsening of the disease in a few people who have tested it, scientists reported Sunday.

Far more research is needed to see if the experimental treatment, requiring a form of brain surgery, really helps.

But if the approach pans out, researchers say, delivering protective substances, called growth factors, into a diseased brain holds the potential to rescue some dying brain cells.

In one patient, brain tissue showed new growth, which was a first, according to the study published in Sunday’s edition of the journal Nature Medicine.

“It won’t cure the disease,” said the lead researcher, Dr. Mark Tuszynski of the University of California at San Diego.

That is because Alzheimer’s destroys different types of cells in different parts of the brain, but the new gene therapy targets just one of those.

The preliminary success indicates similar approaches might help patients with other neurodegenerative diseases such as Parkinson’s, Tuszynski said.

Doctors at Chicago’s Rush University Medical Center have begun a second small study of gene therapy in Alzheimer’s patients. Tuszynski, who co-founded a biotechnology company that is funding the Chicago work, said he hopes larger studies will begin within another year.

“It’s cautious optimism with a big ‘C,’ ” Dr. David Bennett, of Rush University Medical Center, stressed. “It can’t be a cure, obviously, … but maybe it’ll do something.”