*GDNF and other information: Part Two

[Guest guest]

Here is a link the University of Kentucky study. This is referenced

many other places as well.

http://www.parkinson.org/glialnerves.htm

FROM: The Associated Press State & Local Wire

March 18, 2002, Monday

University of Kentuck Announces Innovative Experimental Treatment for

PD

BYLINE: By Steve , Associated Press Writer

Researchers at the University of Kentucky are set to begin an

innovative

clinical trial of a device they believe can reverse the degenerative

effects of PD. The device is an implantable pump that delivers a

naturally occurring

protein that stimulates the growth of dopamine neurons in the brain,

by catheter directly

into the part of the brain that is damaged in PD.

" Current treatments of PD focus only on improving the symptoms of

the illness but do nothing to actually restore function to the parts

of the

brain ravaged by the disease, " Greg Gerhardt, director of the

school's

K. Udall PD Research Center of Excellence, said during a

news conference to announce the trial.

PD is a neurological condition most often seen in olderpeople that

progressively destroys brain cells and impairs control of movement

and speech.

Symptoms of PD include tremors, stiff limbs, slow or absent

movement, a lack of facial expressions a shuffling gait, stooped

posture,

depression and, in some cases, an impaired ability to think.

According to

the National Institute of Neurological Disorders and Stroke, PD

affects

as many as 1 million Americans.

The protein being used in the study is called Glia cell line-derived

neurotrophic factor (GDNF). " GDNF is a very important compound, "

Gerhardt said. " In animal models, we've shown that the direct infusion

of GDNF into the section of the brain affected by PD can actually

restore

function to brain cells that are damaged or dying. "

The study is a Phase I clinical research trial, meaning that

researchers

primarily will be investigating the safety of the device on

participants.

Researchers are looking for 10 subjects who will have the device

implanted and then studied for side effects or any other

irregularities.

" The patients will be studied for about 9 months after the pump and

catheter are implanted, " said principal investigator Dr. Slevin,

a

professor in the University of Kentucky's College of Medicine's

Department of Neurology. " Once we prove that it is safe, we will

expand the study to look more closely at its efficacy, how it

actually works. "

GDNF is found naturally in the human brain but tends to decrease as a

person

ages. It is believed that the destruction of dopamine neurons, which

are

aided in growth by the protein, causes the symptoms of PD. Laboratory

studies

have shown that GDNF both protects and promotes regeneration of

injured

dopamine neurons and may directly influence the degenerative disease

process.

" Basically, what we're trying to do is improve, or even restore,

normal

circuitry in the brain that has been cut off by Parkinson's, " said

Don Gash,

a professor in the college's Department of Anatomy and Neurobiology.

" We've seen very profound improvements in motor function of animals

who had

shown symptoms of late-stage Parkinson's. We have actually seen that

injured

neurons that have shrunk grow back to normal size range when treated

with

GDNF. "

The battery-powered pump, about the size of a small yo-yo and

refillable, is

implanted into the abdomen of the patient with a tiny tube connecting

it

with a small catheter in the brain. The pump's programmable computer

precisely

regulates the flow of a four-week supply of GDNF directly into the

brain via

the catheter. The pump currently is approved for delivery of drugs

directly to the fluid

around the spinal cord in patients with some conditions.

The Udall Parkinson's Research Center is one of only 11 such centers

in the

nation. The foundation for the new investigational treatment came

from basic

research done by Gerhardt and Gash at the center, and the trial is

being

funded by a $5 million grant from the National Institute of

Neurological

Disorders and Stroke, a division of the National Institutes of

Health. "

Comment from Dr. Abe Lieberman

Several years ago Amgen, a biotech company, infused GDNF through a

catheter

into the ventricles (the fluid reservoirs in the brain). The infusion

did NOT reverse the symptoms

of PD and the side effects including severe weight loss halted the

study. In the

University of Kentucky study the infusion is directly into the

substantia nigra,

the site of destruction in PD. It is hoped, but not known, that this

direct delivery

may succeed where the delivery into the ventricles did not help.

_________