Gene therapy could help Parkinson's symptoms

September 25, 2011 10:00:00 PM PDT
Parkinson's disease is one of the most common neurological disorders with as many as 60, 000 Americans diagnosed every year. Patients may have tremors, stiffness, and loss of motor control as the disease progresses.

Now, a new study is showing experimental gene therapy may hold real promise for some patients.

60 year old Walter Liskiewicz spends most of his time "tickling" the electronic ivories. The accomplished new-age jazz musician and singer has a string of hit songs under his stage name "Waldino."

"He was very prolific. People couldn't understand how he could write so many songs," said Connie Smith, Walter's wife. "It's because he was stuck in a chair."

For more than 18 years, Walter has struggled with Parkinson's disease. Increasing disabilities forced him to retire at age 44 from his first career as an oral surgeon. Medication helped control the disease early on, but eventually Liskiewicz started losing his ability to speak and sing.

"My life was going down the tubes," Walter Liskiewicz said.

Dr. Peter Lewitt heads the movement disorders program at Henry Ford Health System in Michigan. Lewitt is studying gene transfer therapy to treat Parkinson's patients.

"The foot is now in the door, opening perhaps a better way to treat people than just medications," Peter Lewitt, M.D., a director of the Parkinson's Disease and Movement Disorders Program at Henry Ford Health System, said.

During the transfer procedure, doctors attach a specialized gene onto a harmless virus and infuse it directly into the brain. Researchers believe that gene, known as GAD, regulates a chemical in the brain that can improve Parkinson's disease symptoms.

Soon after the surgery, Walter and Connie began to notice small, but meaningful changes.

"The eyes sparkling, the smile, the facial expressions," Connie said. "It was really exciting."

It's a cutting-edge procedure that may help this dentist-turned-performer not miss a beat.

Researchers say despite concerns that the gene therapy could have unforeseen risks, those enrolled in the study had no significant side effects.

They say the therapy could potentially be repeated and larger trials would need to be conducted before the FDA would approve the treatment as safe and effective.


Load Comments