Topic awaiting preservation: Gene Therapy for Parkinson's

Not my usual style, but still interesting.
Kind of wish I knew more about gene therapy.

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Doctors Try New Gene Therapy Treatment for Parkinson's Disease
Kerry Sheridan
New York
21 Aug 2003, 00:23 UTC

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For the first time ever, doctors are attempting to treat Parkinson's disease with an experimental new method, gene therapy. The first patient, a New York man, has just undergone the treatment.

Until now, gene therapy has never been used to treat the disease known as Parkinson's, which is characterized by tremors, shaking limbs, muscular stiffness and difficulty walking, and affects about 1.5 million Americans. It is caused by excess activity in a small region of the brain, the subthalamic nucleus.

A team of New York doctors has just performed a radical new treatment to try to change that. The method of gene therapy they are experimenting with does not attempt to cure Parkinson's disease, only treat it.

The procedure involves injecting a virus containing gene cells into the affected area of the brain. Since genes cannot penetrate cells directly but viruses can, doctors chose a virus called adeno-associated virus, or AAV, which is harmless to humans. AAV carries the gene into the affected cells.

Dr. Matthew During, a professor of molecular medicine at the University of Auckland in New Zealand, is part of the medical team. He explains that the gene treatment is designed to calm the overstimulated brain activity that causes Parkinson's.

"In Parkinson's disease, essentially, the accelerator is full on and there's no brake and this part of the brain is completely running out of control. What we're doing genetically here is delivering a gene that enables this part of the brain now to quiet its activity and reset the circuitry," he said.

On Tuesday, Nathan Klein became the first person to receive the experimental treatment. He is a 55-year-old New Yorker who suffers from Parkinson's disease and experiences tremors on the right side of his body.

Nathan Klein, standing with his wife and children at press conference

Doctors drilled a coin-sized hole into Mr. Klein's skull, then using a catheter the size of a strand of hair, injected 3.5 billion particles containing the gene into a part of the brain called the subthalamic nucleus, which is where Parkinson's is centered.

A day after his operation, Mr. Klein appears calm and rested. He says he feels fine, but feels no difference in his symptoms so far. Mr. Klein says being a pioneer in the field of gene therapy is a new experience. "It's overwhelming to be the first," he said. "Unfortunately, it will take away from the second and third and fourth."

Doctors at New York-Presbyterian Hospital were encouraged when Mr. Klein tested normal and showed no brain inflammation in an examination Wednesday.

Gene therapy procedures are not without controversy. Last year, a Pennsylvania teenager died during a gene therapy experiment. And in France, research was halted when children undergoing an experimental gene therapy to cure an immune disorder later developed a leukemia-like disease.

The Parkinson's gene treatment has been tested on hundreds of rats and seven primates. Some experts fear that there have not been enough primate trials to prove it works. They say there is a potential danger of viruses spreading through the brain, or of a reaction that could stop brain cells from sending impulses altogether. For example, the already overactive brain could begin to produce too much of the gene product, called GABA.

Dr. Michael Kaplitt is the director of neurosurgery at New York-Presbyterian Hospital. He says doctors have options to counter possible dangers. "We have the potential to back into the same area and put an electrical stimulator in and try to control the activity of this area and there's evidence for that in our animal studies that was published in the October science paper," he said. "Or in the extreme situation we could go back in and destroy this area as is done in some Parkinson's patients and actually eliminate the source of this GABA production."

The medical team emphasized that this is Phase one clinical trial, which means the primary objective is to find out if the treatment is safe for humans, not necessarily effective.

Twelve patients were chosen to participate. Mr. Klein received the lowest dose of the virus and gene, as will the next three patients. The following four patients will receive a higher amount, and the last four will receive the highest. Doctors say they will continue to monitor Mr. Klein's progress and wait one month before performing surgery on the next patient.

Wow. Sounds alot like a step into the future.
Gene therapy is an area that requires some caution, but it`s awesome thinking that we are coming to a stage where we can begin to make life easier for people whom (before) had no hope.

On the other side of the coin, we are also nullifying the evolutionary selective process. Who knows where that will lead ?
Could go either way.

<edit> Stupid Fingers</edit>

[This message has been edited by docilebob (edited 08-24-2003).]

This one hits a bit close to home since my Mom may have the beginnings of this disease. I don't think I have a very good understanding of gene therapy. It's not clear to me what it actually entails from this article. I don't see how getting genes into the problem areas of the brain results in the calming.

. . : slicePuzzle

I can`t explain the mechanics of it, but as I understand it, a faulty gene is similar to a program with an open loop. The injected genes are the patch.

I'm pretty sure it goes something like this:
A traditional virus will invade a cell.
The virus's(?) DNA "patches" the cell's DNA. Could be RNA.
The cell now follows the new code, which is to replicate the virus until the cell explodes and spills bunches and bunches of viri(?).
Bad virus! Bad! Go lie down!

With gene therapy, the virus's code is hi-jacked.
When the virus invades the cell, the hi-jacked code patches the cell's DNA with what "we" want.
The cell doesn't explode.

With Parkinson's, a part of the brain is over-active due to genes. The brain cells just plain fire away indiscriminately, much the same way that cancer cell just plain reproduce indiscriminately. Changine the code to a calmer genes leads to calmer cells. The end result being less shakes and what-not.

Also, in the case of Parkinson's, I really don't think you want the treatment to spill into other parts of the brain. I imagine this could lead to autistic-like side-effects.

If the genetic code for various diseases can be found, you could probably inject 1rst generation potential parents in the gonads to wipe them out before a child is born. You might even be able to take eugenics in a totally different direction than the one that Hitler dreamed of, which is a totally different can of mixed nuts.

Bugs, I'll keep a look-out for more Parkinson's stuff for ya. I'm sure I'll wander across something interesting eventually.

Bugs: My mother endured parkinson's so I have a bit of an idea of what you are about to experience. I say 'a bit' because a new generation of drugs seem to delay the on-set considerably and there is of course more known about the disease itself.

But you gotta watch the drugs. Closely. Not all the drugs will be parkinson's specific. Some will be presribed to reduce associated anxiety. Problem is not everyone responds the same to the various cocktails. When my mother started displaying symptons that just didn't seem parkinsons related I did a bit of digging. I found that certain 'cocktails' produced certain side effects. I took it upon myself to call my mother's doctor told him of the possible conflict. The prescription was changed....symptons reduced.

For anxiety & depression related drugs this is an excellent place to start.
http://www.drugawareness.org