GENE THERAPY FOR PARKINSON'S
More than one and a half million Americans suffer from Parkinson's disease. There is no cure. But now there is a new treatment that may improve patients' quality of life.
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At 58, Jean Erickson began noticing signs that something was very wrong.
Jean Erickson
Has Parkinson's
"I would freeze. I would start to walk and then suddenly I couldn't walk anymore."
Jean has Parkinson's disease -- a neurologic condition that causes tremors. Like many patients, her medications stopped working properly, so she volunteered for a clinical trial on gene therapy.
Chad Christine, MD
"The upper dots represented the tracks of the needles."
With the new therapy, doctors insert needles in the head to infuse a virus that releases a gene deep in the brain.
Chad Christine, MD
Neurologist
University of California, San Francisco
San Francisco, CA
"Our gene therapy is one that is directed at replacing an enzyme that is lost as Parkinson's disease progresses."
Jean is one of four people who's tried the therapy. So far, all the patients reported improvements and have not had to increase their meds.
Chad Christine, MD
"We are delighted to observe that patients seem to be getting improvement from this low dose and are hopeful that even higher doses will be more effective."
Jean hopes so, too.
Jean Erickson
"I can walk better. I can talk better. My speech, eating in restaurants, isn't as draining as it used to be."
It took courage to be one of the first to try a new treatment. Jean says she's glad she did.
As the clinical trial continues, patients will receive higher doses of the gene therapy. Doctors hope to see even more striking results. Right now, it looks like the therapy will be a one-time treatment.
BACKGROUND: Parkinson's disease is a movement disorder that is chronic and progressive, meaning symptoms continue and worsen over time. More than 1 million Americans suffer from Parkinson's disease. While about 15 percent of people with Parkinson's are diagnosed before the age of 40, incidence increases with age. Parkinson's disease occurs when a group of cells in an area of the brain called the substantia nigra begin to malfunction and die. These cells in the substantia nigra produce a chemical called dopamine. Dopamine is a neurotransmitter, or chemical messenger, that sends information to the parts of the brain that control movement and coordination. When a person has Parkinson's disease, their dopamine-producing cells begin to die, and the amount of dopamine produced in the brain decreases. Messages from the brain telling the body how and when to move are, therefore, delivered more slowly, leaving a person incapable of initiating and controlling movements in a normal way. Parkinson's disease can cause several different symptoms. Some of the most common symptoms include:
tremor of the hands, arms, legs, jaw and face
stiffness of the limbs and trunk
slowness of movement
impaired balance and coordination
CURRENT TREATMENTS: There are many therapies available used to treat the symptoms of Parkinson's. One of the most common is a medication called Levodopa. Levodopa is a substance that is converted into dopamine by an enzyme in the brain. It is then released by brain cells and activates dopamine receptors, allowing for normal function of the movement control centers of the brain. Between 70 percent and 80 percent of treated Parkinson's patients take levodopa therapy. It is considered the "gold standard" treatment. One problem with the current medication is that it can eventually "wear off" and become less effective at controlling symptoms.
A NEW APPROACH: Researchers are now studying gene therapy for patients with Parkinson's disease. There are a couple different clinical trials testing this method. One trial, conducted by researchers from the University of California, San Francisco, is testing whether replacing an enzyme that as lost as Parkinson's disease progresses can help patients. For the study, doctors insert needles in the head to infuse a harmless virus that releases a gene deep into the brain. Chad Christine, M.D., a neurologist at UCSF, says: "We use a virus that's able to carry the gene deep into brain cells that are living and are healthy in the region where dopamine is normally released." Researchers have tested the therapy on four patients to date. So far, the results have been encouraging. "Patients seem to notice that their medications are lasting a bit longer and that they're moving a little more easily. We've not had to increase the medications for any of the patients, and that's very encouraging in a disease that is normally progressing and requires more and more medication as time goes on," says Dr. Christine. "Our hope is to reverse the disease a little bit, not by slowing down the condition itself, but by improving the response to the medications."
Carol Hyman
University of California, San Francisco
3333 California St.
St. 103
San Francisco, CA 94143-0462
chyman@pubaff.ucsf.edu
http://pub.ucsf.edu/newsservices/
Copyright © 2006 Ivanhoe Broadcast News, Inc.
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