Hope For Sufferers Of Parkinson's Disease

Experimental Gene Therapy Shows Promise For Easing The Symptoms Of Degenerative Disorder

(CBS/AP)  An experimental treatment for Parkinson's disease seemed to improve symptoms — dramatically so for one 59-year-old man — without causing side effects in an early study of a dozen patients.

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."

[bellebatinga]

I believe there is no PD,CANCER, etc. if we believe and serve GOD with TRUST AND FAITH,but obviously the human being forgoten who created EARTH and UNIVERSE,MOST THE PEOPLE BELIEVE THE DOCTORS AND MEDICINES ONLY, WITH OUT BELIEVE IN JESUS,BUT IGNORE THEY ARE JUST INSTRUMENT FROM GOD,WHY WE DONT CHANGE OUR WAY OF LEAVING.We are not better than the poorest people that they leave without food to eat and shelter to leave,but they dont have serious diseases,because they leave with GOD GRACE AND CARE.No matter what religion we have the important we served him with FAITH,So it is not to late those are leaving with PD and CANCER or loss of hope,just repent and call his NAME his door will open to us, WE CAN LEAVE WIHTOUT MEDICINES BUT WE CANNOT LEAVE WITHOUT GOD.GOD IS LOVE.

[railey6]

To address "cuttingras"'s ignorant comments. Not everyone who grew up in the 1960's did drugs. I for one never even experienced any kind of drugs. In fact it is a theory that those who smoked regular tobacco and drank a lot of caffine are less apt to develope PD. I also never smoked tobacco or anything else. I drink tea & sodas but not coffee. I only drank alcohol in moderation. Although, not considered heredity it appears in families. My grandfather had it. My mother is fine, now 81. But one of her four siblings had PD. I am one of six siblings and the only one who has PD. I was 53 when diagnosed with PD six years ago. If they knew what causes PD, they could find a cure so millions of people like me would not have to suffer from this awful disease.

[mgpm-2009]

My Mother in law has PD, and has been a health nut for years, long predating her diagnosis of PD. She avoids anything unnatural in her foods and takes all kinds of supplements, etc., has for many many years. Yet she still got PD. And her brother has it too.

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.

[last121868]

tim8559 GO AWAY!

[rational_1]

Hate to tell you but nothing you do or don't do with regard to diet or lifestyle will affect whether you get Huntington's disease. Some diseases (like Huntington's) are purely genetically determined. If you have greater than a certain number of trinucleotide repeats in your huntingtin gene then you are getting the disease no matter what you do; unfortunately this is a case of genetic destiny. The greater the number of these trinucleotide repeats, the younger you are when symptoms start. Now, you are right that for other diseases, environment clearly does play a role. For example if your identical twin is a schizophrenic, your chances of also being schizophrenic are about 50% (indicating a genetic component), but not 100% and this indicates a role for environment.

[getrealdiet]

Avoid synthetic food additive (linked to Parkinson's, Huntington's, and Alzheimer's), avoid bad fats and eat foods high in good fats and fat soluble vitamins and you won't be susceptible to brain diseases.

[rational_1]

Addressing cuttingras' questions...

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).

[scarletphd]

Billy is not just mislead, naive and uninformed...people like him are frequently dangerous because they refuse to take the correct medical action or intervention for disease or injury, and WORSE than that, they impose their ignorance on others, the most vulnerable of those: children.

[rational_1]

"It won't likely be any rat poison from USA drug companies. It will be from wholeistic living with herbs, vitamins, proper posture, and excercise. You don't need no stinkin drugs, alchohol, cigarettes, milk, or cornsyrup in all your foods. Wake up people, its your life!"
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.