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There seems to be alot of interest in gene therapy and the research has started to move from the lab into clinical trials. The genes are transferred into brain cells by the use of a virus. Here are 4 gene therapy trials for PD in the Parkinson Pipeline Project database. All are reporting encouraging early results. CERE-120 (Neurturin) - Phase II trial (Ceregene is sponsor) This approach aims to deliver growth factors into the nigrostriatal system in an attempt to prevent the degeneration of substantia nigra neurons and the accompanying loss of dopamine, Genes for Neurturin (a nerve growth factor related to GDNF) are delivered to the brain through a virus an AAV (adeno-associated viral vector) delivery system AAV-hAADC-2 - phase I trial sponsored by Genzyme The gene coding for the enzyme that converts L-dopa to dopamine (AADC) is inserted into an common, non-pathogenic virus - also an AAV . The AAV will help to transport the gene into the brain cells. It is injected into the striatum during a surgical procedure. Patients who undergo the procedure would continue to take L-dopa; AAV-hAADC-2 is intended to provide, directly to the brain, the missing enzyme needed to convert L-dopa to dopamine ProSavin - phase I/II sponsored by Oxford Biomedica "ProSavin delivers to the patient's stratium three genes required for synthesis of dopamine. ProSavin is based on a LentiVector system (virus) carrying the genes AADC, TH and CH1. The virus can be delivered precisely into the brain by injection. The genes convert cells that normally do not produce dopamine into cells that do. Glutamic Acid Decarboxylase (GAD) Gene Therapy - phase II trial - Neurologix --GAD treatment involves injecting a small amount of a gene-carrying virus (AAV) into the STN region of the brain to calm it down by turning the excitatory chemical glutamate into the inhibitory brain chemical GABA." I'm guessing that they might exclude PWP who have dyskinesia from some of these studies because they want to compare the amount of time to onset of dyskinesia in those who are using traditional levodopa therapy and those who received gene therapy. For more information on these and other treatments in development- see the Parkinson Pipeline Project Treatment Database at:: www.pdpipeline.org Click on Database acccess. Linda -- kbachn <[log in to unmask]> wrote: this is very interesting. anyone knows the difference btw this gene therapy and the Ceregene one. upon reading the website, an article mentioned that they only recruite late stages of parkinson (undersandable) and those WITHOUT dyskinisea ( curious as to why this is so, does it mean they suspect or already know that this treatment wont work with those with dyskinesea ). any insight? Thank You and Regards, Ken ----- Original Message ----- From: "M.Schild" <[log in to unmask]> To: <[log in to unmask]> Sent: Saturday, December 15, 2007 2:47 AM Subject: Hopes rise over Parkinson's > Hopes rise over Parkinson's > > Scientists at an Oxford biotech firm are hoping they have made a > breakthrough > in the search for an effective Parkinson's Disease treatment. > Trials on the ProSavin gene therapy product, which has been developed by > Oxford Biomedica based at the Oxford Science Park, have started in France, > with experts confident of success. > Chief executive Prof Alan Kingsman said: "The product could represent a > fundamentally new approach for the treatment of Parkinson's Disease and > could > significantly expand the worldwide market for existing therapies." > > > He said the clinical trial followed more than 10 years of research. > Parkinson's disease is caused by the degeneration of dopamine-producing > nerve > cells, leading to impaired movement. > ProSavin uses gene therapy to restore dopamine production in the brain. > Initial tests have shown almost complete recovery of movement and other > behaviour with a single treatment effective for at least two years. > Current surgical approaches to treatment require the destruction of brain > tissue or the use of electrodes to stimulate the brain, both of which have > side effects. > 4:51pm Friday 14th December 2007 > > ---------------------------------------------------------------------- > 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