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Gene Therapy Research May Relieve Parkinson's DiseaseAired October 26, 2000 - 2:38 p.m. ET
THIS IS A RUSH TRANSCRIPT. THIS COPY MAY NOT BE IN ITS FINAL FORM AND MAY BE UPDATED.
NATALIE ALLEN, CNN ANCHOR: And with that one we'll move on from the election for a moment to medical news. New gene therapy research may result in a breakthrough treatment for those suffering from Parkinson's disease. The experiment relieves severe symptoms of the disease in monkeys and experts say it might also work for humans.
Our medical correspondent Rhonda Rowland joins me now with more about this -- Rhonda.
RHONDA ROWLAND, CNN MEDICAL CORRESPONDENT: Well, Natalie, first of all, just for a background, most people know that Parkinson's disease is characterized by erratic body movements, by rigidity, by tremor and they believe that it is caused by a loss of dopamine in the brain, which is a chemical messenger-type thing. And so this study today was published in the journal, "Science."
And to help explain the experiment, we have some graphics to kind of walk through this. This particular study included eight old monkeys with brain changes associated with Parkinson's disease as well as 20 young monkeys that were given a chemically induced Parkinson's- like condition. So, it's not exactly Parkinson's disease but it is similar.
And these monkeys were given injections of gene therapy called GDNF. And this was injected directly into the brains of the monkeys. And they found several things. GDNF spurs production of the brain chemical dopamine. And they also found that it stabilized or prevented progression of Parkinson's disease. And it improved the symptoms. And, in fact, in three of the monkeys, they actually reverted back to normal, which was very exciting.
Now as for the future of this particular research, those scientists at Rush Presbyterian Medical Center in Chicago say that they still have to do another study in monkeys because they have to make sure that this is safe. That is a very big concern with gene therapy.
If it is successful, this next particular study, then they will go to the FDA and ask for permission to begin studies in humans. And this could happen in, perhaps, about a year. But even, Natalie, once they get that permission, it's still going to be, perhaps another three to five years before they actually start the study. So, this is still kind of long term.
ALLEN: Well, considering that this experiment was done in monkeys, how many of a breakthrough is this considered?
ROWLAND: Well, when we talk to Parkinson's disease researchers, they're very excited about this. They think that the next real breakthrough for Parkinson's disease will be in this type of a therapy. And the reason is because right now there are a number of drugs that are offered to patients but they don't work long term. They start to lose their effectiveness and then a lot of patients also have side effects that are very troubling.
The other option for these patients right now is brain surgery, where they actually go in and they destroy the part of the brain that's causing these erratic body movements. Or they can also get a pacemaker-like type of device that stops some of the tremors. But, as you imagine, Natalie, that is quite an undertaking to go for that surgery. It's very risky. So, of course to have something like this that could actually prevent the progression of this disease, this is extremely exciting.
ALLEN: So, if people have Parkinson's, should they be very hopeful, I would think, for this continuing on in human trials?
ROWLAND: Well, they should be hopeful. But again, it's going to take a little while, so you can't get your hopes up too much and hope that there is something new the next year or two. Also, we have to keep in mind, with gene therapy, even though the first human studies took place 10 years ago, and since that time there has been more than 300 studies in humans with gene therapy, there have only been four successful experiments where they have seen that it actually has worked and that's been in hemophilia, cancer, in immune deficiency disorder and heart disease. So, it has tremendous promise, but still, there's a lot of work to do.
ALLEN: Rhonda Rowland, thanks. It sounds encouraging.
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