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Retinal Cell Implantation Shows Promise And Improvement In Advanced
Parkinson's Disease Patients

Date: Posted 4/18/2002 - An innovative procedure, in which retinal cells
are implanted in the brains of patients with advanced Parkinson’s disease
(PD), is showing promise, according to a group of Emory University
researchers.

In a one-year follow-up report to be given at the American Academy of
Neurology 54th Annual Meeting in Denver, Colo., on April 17, Ray Watts,
M.D., professor of neurology, Emory University School of Medicine, says
patients involved in this study have improved nearly 50 percent in motor
function after 12 months.

Dr. Watts will lead the discussion on the safety and efficacy of this pilot
study at the meeting.

In this Phase I trial, Dr. Watts and colleagues implanted retinal pigment
epithelial (RPE) cells attached to gelatin microcarriers into the brains of
six patients with advanced PD.

RPE cells, normally found in the back of the eye, are cultured in the
laboratory to produce cells for this treatment. These cells provide a
source of increased dopamine production.

Dopamine is a neurotransmitter produced by neurons in the brain that is
found in steadily decreasing amounts as the disease progresses. The
implanted retinal cells serve as a new source of dopamine production in
these patients.

"This is the first human intracerebral retinal cell implantation study in
the world and we are encouraged by the results so far," Dr. Watts reports.

"We’ve been following these six participants for over a year, and we’ve
found they’ve improved, on average, nearly 50 percent in motor function."

Improvement is being seen in their tremor, stiffness, slow movement and
balance, the most common motor functions affected by Parkinson’s disease.

Half of the participants are also showing improvement of dyskinesia, which
are involuntary movements that are a result of medications.

The epithelial cells used in the trial were taken from a human donor.
Hundreds of millions of cells were grown in cell culture from the donor
cells, and they were attached to tiny gelatin beads (or microcarriers)
prior to implantation.

Surgeons then implanted approximately 325,000 cells in each participant
during MRI-guided stereotaxic surgery.

The cells were implanted in five different areas of the striatum, the part
of the brain that controls movement.

The cells then live on the gelatin beads in the striatum and continually
provide a source of increased dopamine production. The novel cell product
is called Spheramine, developed by Titan Pharmaceuticals, Inc. and uses
Titan’s cell-coated microcarrier (CCM‘) technology.

Participants in the Emory study only had cells implanted on one side of the
brain, focusing on the side that was most affected by the disease.

This implantation procedure is still investigational and has not been
approved by the Food and Drug Administration (FDA) for general use.

Researchers say because it is performed stereotaxically with a needle,
there is less risk of infection or bleeding.

Participants did not have to take immune suppressant drugs, because the
brain is relatively immune protected and these cells appear to have low
tendency to produce an immune response when implanted with this
microcarrier technique.

The research for this Phase I, open-label trial was supported by a grant
from Titan Pharmaceuticals, Inc. and the National Institute of Neurological
Disorders and Stroke (NINDS), a division of the National Institutes of
Health (NIH).

'This new cell implantation therapy appears to be safe and well tolerated
in participants 12 to 15 months following implantation," says Dr. Watts.

"We will continue to follow these six participants for months to come. We
hope to begin a Phase II controlled trial soon implanting Spheramine on
both sides of the brain, instead of just one side."

Parkinson’s disease is a progressive disorder of the central nervous system
affecting over one million people in the United States.

Source: Emory University Health Sciences Center
(http://www.emory.edu/)

Note: This story has been adapted from a news release
issued by Emory University
Health Sciences Center for journalists and other members of
the public. If you wish to
quote from any part of this story, please credit Emory
University Health Sciences
Center as the original source. You may also wish to include
the following link in any
citation:

http://www.sciencedaily.com/releases/2002/04/020418073536.htm

janet paterson: an akinetic rigid subtype, albeit perky, parky
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