Hope For Sufferers Of Parkinson's Disease

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

(CBS/AP) 
Kaplitt, who has a financial interest in Neurologix Inc., which paid for the research, noted that the 12 patients in the study still have Parkinson's symptoms. The amount of medication they were already taking for their symptoms didn't change significantly in the year after the surgery.

Of the 12 patients in the study, all showed at least a 25 percent improvement. Five patients, including Klein, had up to a 65 percent reduction in symptoms, reports Miller.

Current medicines can control symptoms but can't stop the disease from getting worse over time. They also can produce troublesome side effects, such as uncontrollable movement.

"I think it's a very important first step in gene therapy for Parkinson's. It's treating the symptoms of Parkinson's, not the underlying defect in Parkinson's,” Guy McKhann M.D. of the Columbia University Medical Center tells CBS.

Some patients gain relief from a surgical treatment called deep brain stimulation, in which electrodes are placed in the brain and connected to a programmable stimulator.

Kaplitt's procedure was aimed at achieving the same goal as that surgery, calming overactive circuitry in the brain. It gets overactive because it loses the normal supply of a chemical called GABA. The gene therapy was designed to make the brain produce more GABA.

For the gene therapy surgery, a tube about the width of a hair was threaded through a hole about the size of a quarter at the top of the skull. The tube delivered a dose of a virus engineered to ferry copies of a gene into cells of a brain region called the subthalamic nucleus. The gene copies enable the cells to pump out more GABA.

The Lancet paper reports that over a year, patients showed no side effects from the procedure. What's more, they showed improvements in an overall assessment of symptoms like tremors, stiffness and walking problems.

The improvements were evident at a checkup three months after the procedure and persisted to the end of the study, one year after the surgery, researchers reported. By that time, the overall amount of improvement from before surgery was about 24 percent when measured at times that patients were off their normal medication, and 27 percent at times when they were on medication.

Most of the effect appeared on just one side of the body. Because of concerns about safety with the untested procedure, the researchers treated only the brain circuitry controlling one side of the body.

Dr. Karl Kieburtz of the University of Rochester Medical Center, who didn't participate in Kaplitt's work, said the lack of any apparent side effects is itself significant.

But he urged caution in interpreting the evidence of benefits in symptoms. Other experimental therapies that looked good at such a preliminary stage have failed to pan out in more rigorous studies, he said, so more research is needed.

[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.