Thank You  Rayilynlee,

Your postings have always been interesting and insightful.

w.r.t. the " UPDRS was 38 percent" improvement mentioned, does it mean if
the PWP was a 4 - then after 1 year of treatment, the PWP is a 2 (or 2.4
average) after the GDNF treatment ( is suspect that the patients results
were between 2 and 3).

This is really encouraging, how different was this study compared to the one
several years ago that was cancelled (you know : the controversy). is it new
and improved GDNF?

Regards, Ken


----- Original Message -----
From: "rayilynlee" <[log in to unmask]>
To: <[log in to unmask]>
Sent: Tuesday, April 17, 2007 1:45 PM
Subject: Gene Therapy for PD


> Newswise - It is estimated that 60,000 new cases of Parkinson's disease
> (PD)
> are diagnosed each year, adding to the estimated one to 1.5 million
> Americans who currently have the disease. The latest epidemiology studies
> indicate that worldwide numbers will increase from an estimated 4.1
> million
> in 2005 to 8.7 million people with PD by 2030. There were nearly 18,000
> PD-related deaths in the United States in 2004. While the condition
> usually
> develops after the age of 55, the disease may affect people in their 30s
> and
> 40s.
> Early in the disease, there is a loss of brain cells that produce the
> chemical dopamine. Normally, dopamine operates in a delicate balance with
> other neurotransmitters to help coordinate the millions of nerve and
> muscle
> cells involved in movement. Without enough dopamine, this balance is
> disrupted, resulting in tremor (trembling in the hands, arms, legs and
> jaw);
> rigidity (stiffness of the limbs); slowness of movement; and impaired
> balance and coordination - the hallmark symptoms of PD.
> In the last 10 years, protein substances called "growth factors" have been
> discovered that can slow or halt the death of dopamine-producing cells.
> One
> such factor, known as "GDNF" (Glia-Derived Neurotrophic Factor), has been
> used in clinical trials for PD. The results have been inconsistent,
> possibly
> related to the method of delivering the protein to the brain.
> Researchers at the University of California at San Francisco, and
> Rush-Presbyterian Medical Center in Chicago, in conjunction with Ceregene,
> Inc. of San Diego, Calif., recently undertook a Phase I trial using a
> novel
> strategy called "gene transfer" to deliver a growth factor to the brains
> of
> 12 patients with PD. All patients entered in the trial were judged to have
> inadequate control of their disease with standard levadopa therapy and
> would
> have otherwise been potential candidates for treatment interventions such
> as
> deep brain stimulation (DBS).
> The results of this study, Intrastriatal Gene Transfer with AAV-Neurturin
> for Parkinson's Disease: Results of a Phase I Trial, will be presented by
> Philip A. Starr, MD, PhD, 11:45 am to 12:00 p.m. on Monday, April 16,
> 2007,
> during the 75th Annual Meeting of the American Association of Neurological
> Surgeons in Washington, D.C. Co-authors are Leo Verhagen, MD, Paul S.
> Larson, MD, Roy Bakay, MD, Robin Taylor, RN, Deborah Cahn-Weiner, PhD,
> Raymond Bartus, MD, Jill L. Ostrem, MD, and William J. Marks Jr., MD.
> The growth factor gene was delivered as part of a modified virus, or
> "viral
> vector", called adeno-associated virus (AAV). This viral vector helps
> enable
> the gene to be delivered into the correct brain cells, but has been
> modified
> so that it cannot reproduce or damage brain cells. The growth factor gene
> neurturin was utilized, which is a protein closely related to GDNF.
> Neurturin has been shown in laboratory studies to help prolong survival of
> dopamine-making cells. AAV-neurturin was delivered directly to the brain
> via
> stereotactic injection through multiple (16) needle injections into the
> striatum, the part of the brain most deficient in dopamine. This was
> performed through small openings in the skull.
> The patients were studied using standard rating scales of movement in PD,
> the Unified Parkinson's Disease Rating Scale (UPDRS) prior to surgery and
> on
> a continual basis post surgery, at baseline, 1, 3, 6, 9 and 12 months, on
> and off medication. Two different doses of the viral vector were tested,
> the
> lower dose in the first six patients, and the higher dose in the remaining
> six patients. The following outcomes were noted:
> .There were no major adverse effects from this treatment at the low or
> high
> doses.
> .In nine of the 12 patients for which one-year outcome data was available,
> the improvement in the UPDRS was 38 percent.
> "Patients with PD urgently need therapeutic approaches that not only
> improve
> their symptoms and daily functions, but positively modify the underlying
> components of the disease, stated Dr. Starr.
> "Existing therapies for PD treat only the symptoms, and are effective for
> a
> limited period of time, so any trial that is safe and results in promising
> efficacy data is worth pursuing. The safety data and preliminary efficacy
> data that resulted from this Phase 1 study are encouraging, and clearly
> warrant the need for a larger, Phase II study," concluded Dr. Starr.
> Founded in 1931 as the Harvey Cushing Society, the American Association of
> Neurological Surgeons (AANS) is a scientific and educational association
> with more than 6,800 members worldwide. The AANS is dedicated to advancing
> the specialty of neurological surgery in order to provide the highest
> quality of neurosurgical care to the public. All active members of the
> AANS
> are certified by the American Board of Neurological Surgery, the Royal
> College of Physicians and Surgeons (Neurosurgery) of Canada or the Mexican
> Council of Neurological Surgery, AC. Neurological surgery is the medical
> specialty concerned with the prevention, diagnosis, treatment and
> rehabilitation of disorders that affect the entire nervous system,
> including
> the spinal column, spinal cord, brain and peripheral nerves.
>
>
>
> © 2007 Newswise.  All Rights Reserved.
>
> ----------------------------------------------------------------------
> To sign-off Parkinsn send a message to:
> mailto:[log in to unmask]
> In the body of the message put: signoff parkinsn
>

----------------------------------------------------------------------
To sign-off Parkinsn send a message to: mailto:[log in to unmask]
In the body of the message put: signoff parkinsn