Lancet Study Shows Gene Therapy for Parkinson's Disease is Safe and Benefits
Some Patients
A novel gene therapy technique is safe and may be effective at staving off
worsening symptoms of Parkinson's disease, according to the first scientific
review of a dozen patients who have received the treatment over the last
three years. The results were published in the latest issue of Lancet.
 Manhasset, NY (Vocus/PRWEB ) June 20, 2007 -- A novel gene therapy technique
is safe and may be effective at staving off worsening symptoms of Parkinson's
disease, according to the first scientific review of a dozen patients who
have received the treatment over the last three years. The results were
published in the latest issue of Lancet.
This important study shows that gene therapy can be performed safely and may
benefits patients.
   The patients, half of whom live on Long Island, are in advanced stages of
the illness and were no longer responding to medicines when they signed on
for the experimental therapy. The study was conducted by Andrew Feigin, MD,
director of Neuroscience Experimental Therapeutics at The Feinstein Institute
for Medical Research and his colleagues in collaboration with Parkinson's
scientists at New York Presbyterian Hospital-Weill Cornell Medical Center in
Manhattan.
 One woman and 11 men received a surgical infusion of fluid containing a viral
vector and genes for a protein called GAD, glutamic acid decarboxylase. This
enzyme is critical in controlling a neurotransmitter called GABA. In
Parkinson's, GABA is reduced in an area of the brain called the subthalamic
nucleus. This region is working on overdrive in the disease process and GABA
is an inhibitory transmitter and is important in trying to calm this
hyper-reactive circuit.
 The gene therapy would be used to reduce symptoms and not alter the
underlying disease process. Finding novel therapies are key as many
Parkinson's patients stop develop complications after prolonged use of
traditional medicines.
 The Feinstein's David Eidelberg, MD, took brain scans before, during and
after the treatment and the scans show that the brain is re-working these
abnormal circuits. Dr. Feigin said that patients had about a 27 percent
improvement in symptoms, although the study was an open label design. The
scientists are now designing a double-blind placebo controlled trial that
would enroll far more patients in an attempt to see whether the gene therapy
is effective in reducing symptoms.
 The patients' scans showed a quieting of these areas, on the side of the
brain where the genes were infused. The study was designed to inject the
genes into one side of the brain. Normally, Parkinson's patients have
worsening symptoms on one side of the body.
 The novel strategy included packing genes that make an inhibitory chemical
called GABA into pieces of viruses that have been rendered non-infectious.
They began studying the experimental treatment in Parkinson's patients in
2003. Some patients continue to show improvement.
 Parkinson's patients have been willing to step up to the operating table for
relief from the tremors, stiffness and rigidity that characterize the
disease. Decades ago, surgeons began to make lesions in parts of the brain
involved in the disease, which lessened symptoms. Fetal stem cell surgery was
pioneered in Parkinson's patients. And in the past decade, the deep brain
stimulation has worked in as many as 70 percent of patients who have opted
for the surgical procedure. If it doesn't work, the electrodes can be
removed. (By comparison, it would be impossible to reverse gene therapy.)
 "Gene therapy could be a more natural way to treat the disease," said Dr.
Feigin. "This important study shows that gene therapy can be performed safely
and may benefits patients."
 The gene therapy technique was developed by Neurologix, a New Jersey-based
company.
 Parkinson's is a movement disorder caused by a progressive depletion of the
brain chemical dopamine in the substantia nigra. These dopamine-containing
cells control movement. When 70 to 80 percent of these cells are destroyed, a
person develops the first symptoms of disease: tremors, slowed movement,
muscle rigidity and problems with balance. The main medication used in
Parkinson's is L-dopa, which replaces dopamine in cells that are still
working normally. Over time, the cell death is so massive that the effects of
the medication disappear.
 About The Feinstein Institute for Medical Research Headquartered in
Manhasset, NY, and part of the North Shore-LIJ Health System, The Feinstein
Institute for Medical Research is home to international scientific leaders in
Parkinson's disease, Alzheimer's disease, psychiatric disorders, rheumatoid
arthritis, lupus, sepsis, inflammatory bowel disease, diabetes, human
genetics, leukemia, lymphoma, neuroimmunology, and medicinal chemistry.
Feinstein investigators rank in the top 6th percentile of all grant awards
funded by the National Institutes of Health. Feinstein Researchers are
developing new drugs and drug targets, and producing results where science
meets the patient. For more information, please visit
www.FeinsteinInstitute.org.
 Contact: Jamie Talan
 Science Writer
 516-562-1232

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