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Thanks, Ken. Credit must go to Diane Wyshak who daily sends me tens of emails. I try to select a variety of topics to plain text and post. I am probably going to be writing the newsletter for the AZ Chapter of NPF and would like to know what kind of posts List readers find most helpful: Research and new drug/treatment trials Inspirational stories and bios of PWP Politics of research: world, national, state Advocacy Action Alerts Miscellaneous Re this GDNF study - it sounds like a new one to me, but I would refer you to Linda Herman and Claire Salamon, who are experts on GDNF trials. Ray ----- Original Message ----- From: "kbachn" <[log in to unmask]> To: <[log in to unmask]> Sent: Wednesday, April 18, 2007 11:18 AM Subject: Re: Gene Therapy for PD 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 ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn