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.
Connie Smith, Walter’s wife, says, "He was very prolific. People couldn't understand how he could write so many songs. It's because he was stuck in a chair."
For more than 18 years, Liskiewicz 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.
Walter says, "My life was going down the tubes."
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.
Dr. LeWitt says, "The foot is now in the door, opening, perhaps, a better way to treat people than just medications."
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.
Connie says, "Just to see him…the eyes sparkling, the smile, the facial expressions. It was really exciting.”
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 trails would need to be conducted before the FDA would approve the treatment as safe and effective.
BACKGROUND: Parkinson's disease is a progressive disorder of the nervous system that affects movement. It develops gradually, often starting with a barely noticeable tremor in just one hand. While tremor may be the most well-known sign of Parkinson's disease, the disorder also commonly causes a slowing or freezing of movement. There's no cure for Parkinson's disease, but medications can help control some of the symptoms of Parkinson's disease, and in some case, surgery may be helpful. (SOURCE: Mayo Clinic)
CAUSES: A small region deep within the brain is the source for the symptoms of Parkinson's disease. When brain neurons in this part of the brain begin to die, these cells can no longer manufacture the molecule dopamine -- a chemical critical for controlling movement. The exact cause of Parkinson's disease is unknown, but several factors appear to play a role, including genes. Researchers have found specific genetic mutations that likely play a role in Parkinson's disease. In addition, scientists suspect that many more changes in genes -- whether inherited or caused by an environmental exposure -- may be responsible for Parkinson's disease. Exposure to toxins or certain viruses may trigger Parkinson's signs and symptoms. (SOURCE: Mayo Clinic)
NEW GENE THERAPY: For the first time, gene therapy has proven successful in Parkinson's patients. The therapy uses a virus that is stripped of its infectious properties and delivered with a thin tube into the brain's subthalamic nucleus -- a structure "the size of a pine nut" that is involved with movement. Researchers followed 45 patients for six months after the procedure at seven U.S. medical centers. Half the patients showed improvements early on, which they still sustained six months later. Most current therapies and research approaches target dopamine to treat motor symptoms associated with Parkinson's disease. In contrast, the focus of the current gene therapy strategy is on increasing GABA -- a brain neurotransmitter that regulates movement. In Parkinson's disease, GABA is reduced in the area of the brain known as the subthalamic nucleus, causing it to be overactive. Investigators feel this might be a better way to help advanced Parkinson's disease. (SOURCE: Henry Ford Health System)
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