Clinic part of trial of very promising' Parkinson's treatment
Sunday, February 25, 2007
Zachary Lewis
Plain Dealer Reporter

Researchers at the Cleveland Clinic are collaborating with eight medical institutions nationwide to test what they call a promising new treatment for Parkinson's disease.

"This would be a whole new paradigm," said Nicholas Boulis, a Clinic neurosurgeon and the primary investigator in the local trial. "It's really opening up a whole new way of approaching Parkinson's."

Under examination is CERE-120, a manufactured gene transfer agent that prompts the brain to repair itself.

Specifically, CERE-120 is an adeno-associated virus carrying a gene that causes the brain to produce neurturin, a naturally occurring protein that repairs damaged dopamine neurons.

Dopamine loss leads to the involuntary movements symptomatic of Parkinson's. Thus, CERE-120 stands to halt or even reverse the effects of the disease.

"I think this is very promising," said Marilyn Brandt, president of the Parkinson's Education Program of Greater Cleveland, a local support group.

Nearly 50,000 Americans have Parkinson's disease diagnosed every year. The disease affects 4.3 million worldwide.

A unique feature of the CERE-120 trial is the method of delivery. Existing dopamine treatments such as patches distribute substances across the brain, but CERE-120 is administered through precise injection into the putamen, part of the brain that influences movement.

The surgery consists of drilling two holes in the skull and, navigating by magnetic resonance imaging, delivering eight CERE-120 injections.

"Basically, the putamen evaluates everything you're trying to do from a motor perspective and decides whether or not to let it happen," Boulis said. "To be effective, treatments need to be in the exact right part, and they need to be there a long time."

Results so far have been encouraging. Twelve patients who received the surgery in Phase I trials at the University of California-San Francisco and Rush University Medical Center in Chicago reported a 40 percent improvement in their motor skills.

Side effects included mild to moderate temporary dyskinesia, or involuntary movement. This was treated successfully by readjusting the dosage of traditional Parkinson's medications, Boulis said.

Another advantage of CERE-120: "You don't have a wire and battery in your body," Boulis said, referring to equipment left over from deep-brain stimulation, another Parkinson's treatment.

Fifty-one participants nationwide are being sought for the trial's second phase, and enrollment is still open. Seventeen patients will be part of a control group and not receive CERE-120 initially.

In addition to the work in Cleveland, San Francisco and Chicago, trials are taking place in Birmingham, Ala.; New York; Durham, N.C.; Portland, Ore.; Houston; and Philadelphia.

"We've got to try every avenue there is, and we've got to try them on people," Brandt said. "Everybody's hoping one of them will break through."

To be eligible for the trial, participants must be 35 to 75 years old, have had the disease for five years and have pursued dissimilar treatments without success. Exclusionary factors include atypical forms of the disease, chemotherapy, history of drug or alcohol abuse and the presence of medical conditions that could complicate surgery.

The Michael J. Fox Foundation says it has contributed more than $2.6 million in support of the trials.

****Thanks, Michael!