BIRMINGHAM, Ala. — A small number of Parkinson's patients are participating in a study that injects an experimental drug into the brain with the hopes of rejuvenating damaged cells and slowing the progression of the disease.
Nine medical centers from Alabama and San Francisco to Chicago and New York enrolled patients to participate in the double-blind study and scientists will check the patients at years' end to see if their symptoms have improved.
Researchers say the drug, CERE-120, could possibly be used for a host of other common ailments of aging, including Alzheimer's disease.
CERE-120 contains genetic instructions for making the natural protein neurturin, which promotes growth.
Those instructions have been placed within a virus and the gene therapy is precisely injected into areas where brains have been damaged by Parkinson's disease.
What results is "a little factory pumping out neurturin," Jeffrey M. Ostrove, president and chief executive officer of Ceregene Inc., told the Birmingham News in a Sunday story.
The neurturin bathes, protects and rejuvenates damaged cells.
An early human trial indicates that the approach might work, and if all goes well the treatment could be available in a few years.
The early trial was conducted with a dozen Parkinson's patients primarily to see if CERE-120 was safe.
It was, and it also turned out that patients in the trial got better, on average improving scores on function tests by 36 percent, the company announced last April.
The trial is called double-blind because patients and most doctors in the study don't know who got the drug. Patients who didn't get the drug underwent a simulated surgery, which has been used in studies before, but it is unusual.
"We are really pushing the boundaries," said Dr. Ray L. Watts, chair of neurology at University of Alabama, Birmingham, one of the universities participating.
Watts is the principal investigator for CERE-120 at UAB, and he said about six Parkinson's patients from the area signed up for the study.
Some were injected with the drug, but others only had a small nick safely burred into their skulls that made it look and feel like they had a drug injection.
The surgeries and imitations were conducted by Dr. Bart Guthrie, a co-investigator. Guthrie and his team know who got what, but they won't evaluate patients or "break the blind."
Watts said the study will run until the end of the year. Then patients will be tested, and results will be evaluated.
Researchers are cautiously optimistic about the outcome and are already planning a third and final study that would complete testing.
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