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Hi Ken, GDNF and neuturin are members of the Glial-cell-line-derived neurotrophic factor (GDNF) family ligands (GFLs). They help regulate the development and maintenance of the nervous system. According to the sponsor, Ceregene "The two molecules have similar pharmacological properties, and both have been shown to benefit the midbrain dopamine neurons that degenerate in Parkinson's disease and are responsible for the major motor impairments" I've been tracking CERE-120 for the Parkinson Pipeline Project. The results of the CERE-120 phase I trial seem very promising and close to the results of the GDNF phase I. When Amgen halted the GDNF trial, they refused to let any other company or researchers utilize their product. By using neuturin instead, Ceregene did not have to deal with Amgen. Let's hope the 2 molecules are indeed very similar, and the Phase II results will be very good. Ceregene seems like a very ethical biotech company that is committed to this research and wants to make sure the trials are done correctly, so there will be no lingering questions, as there were in the GDNF trials. The fact that the Michael J. Fox Foundation is providing funding is a very good sign. To see more about the currently recruiting phase II trial see Pdtrials.org: http://www.pdtrials.org/front/trial_detail.php?trial_id=128 To learn more about CERE-120 research see the Parkinson Pipeline database www.pdpipeline.org Linda p.s. When Jonas Salk was asked why he didn't patent his polio vaccine he replied, "Would you patent the sun? " Guess Amgen's answer would be YES!" www.pdpipeline.org -- kbachn <[log in to unmask]> wrote: 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] ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn In the body of the message put: signoff parkinsn