Hope For Sufferers Of Parkinson's Disease
Experimental Gene Therapy Shows Promise For Easing The Symptoms Of Degenerative Disorder
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Play CBS Video Video Hope For Parkinson Patients Scientists have found a gene therapy treatment for the incurable brain disorder that affects millions of Americans. So far, so good. Michelle Miller reports.
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Nathan Klein, 59, was the first patient to be treated with Kaplitt's gene therapy procedure in 2003. (CBS)
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The gene therapy treatment involved slipping billions of copies of a gene into the brain to calm overactive brain circuitry.
The small study focused on testing the safety of the procedure rather than its effectiveness, and experts caution that it's too soon to draw conclusions about how well it works. But they called the results promising and said the approach merits further studies.
"We still have quite a bit more testing to do," said Dr. Michael Kaplitt of Weill Cornell Medical College in New York, an author of the study. Still, "the initial results are extremely encouraging."
"Our hope is that this will, in the future, be considered the first major milestone in the creation of a whole new field of medicine for brain diseases," Kaplitt told CBS News Correspondent Michelle Miller.
Kaplitt and collaborators report their results in this week's issue of the British medical journal, The Lancet.
They're not alone in trying gene therapy for Parkinson's. In April, another team told a medical meeting that its experiments, which delivered a different kind of gene to a different part of the brain, also appeared safe and gave a preliminary hint of benefit.
More than half a million Americans have Parkinson's. They endure symptoms that include tremors, rigidity in their limbs, slowness of movement and impaired balance and coordination. Eventually they can become severely disabled.
Nathan Klein, a 59-year-old freelance television producer in Port Washington, N.Y., said the disease left him "pretty messed up." It weakened his voice, impaired his walking and made his hand tremble so badly he couldn't hold a glass of wine without spilling it all.
Klein was the first patient to be treated with Kaplitt's gene therapy procedure in 2003, and he said his symptoms gradually subsided afterward. Nowadays, he said, apart from freezing now and then when he wants to walk, the symptoms are basically gone.
"I feel great. I swim, ride my bike, I do everything anybody who's normal can do," Klein told Miller.
"I'm elated," added Klein, who continues to take his regular pills for the disease. "It's unbelievable."
© MMVII, CBS Interactive Inc. All Rights Reserved. This material may not be published, broadcast, rewritten, or redistributed. The Associated Press contributed to this report.
Best-selling author Mitch Albom on his first nonfiction work since "Tuesdays with Morrie."





While I have no doubt that eating garbage and having chemicals in our food has an effect, it isn't the entire story. You cannot protect yourself entirely from disease by eating right. If you want to think that, if it makes you feel better to think that, well, go ahead.
My MIL's PD is progressing even though she takes supplements. She believes they are helping her slow the disease, but I don't know if that's true or not. I love her dearly, she's my best friend...I am hoping and praying that this new treatment proves very helpful over a large group of people, and that she can get it eventually. She was so vibrant, and to see this disease gradually sap that away is heartwrenching.
I don't think Parkinsons disease necessarily has a higher incidence now than in the past, but patients do tend to live longer and the treatments, such as L-Dopa, do allow them to at least temporarily (a few years) get somewhat of a respite from the disease. Plus, there are a lot more people now than in the past and people tend to live longer. Maybe that accounts for why you think there are more PD patients around.
By the way, you mentioned drug use - there was a designer drug that came out about 30 years ago, called MPTP, that some people thought could be a way to circumvent laws against illegal drug use. Unfortunately it turned out to be very neurotoxic to the exact same neurons that die out in PD. See this Wikipedia link if you're interested.
http://en.wikipedia.org/wiki/MPTP
Incidentally, this is in no way related to PD in the general population (just an interesting story).
Posted by billysmith6 at 07:01 PM : Jun 21, 2007
Ya, improving your posture will do wonders for your schizophrenia or Parkinson's disease! Probably cure Huntington's as well. Give me a break! Sure a proper diet and exercise are useful for maintaining health, but they won't do anything for you if the dopaminergic cells in your substantia nigra die, leaving you with Parkinson's disease. There's a whole lot to be said for modern medicine, but if you're banking on some eye of newt from your herbalist to treat your high blood pressure or congestive heart failure, then for your relatives' sakes, I suggest you up your life insurance.