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Dear List members, Just wish to pass along some interesting information that I've gathered over the past few days regarding two biotech companies engaged in clinical studies focused on gene therapies for neurodegenerative disorders. The companies: Ceregene and Neurologix both have studies underway involving in vivo (inside the body) gene therapy utilizing a non-replicating viral vector to deliver therapeutic genes to the nervous system. Ceregene has initiated a Phase 1 study of CERE-120 to treat Parkinson's disease. The study is being conducted at the U. of California, San Francisco Medical center and Rush University Medical Center in Chicago. CERE-120 is a novel gene therapy that delivers the neurturin (NTN) gene via an adeno-associated virus (AAV) type 2 vector delivery system. Neurturin is a member of the same protein family as GDNF and they have similar pharmacological properties. Many of us are aware of, have been disturbed by and have attempted to intervene in Amgen's decision to halt their trial of GDNF in spite of the positive results that the patients reported while receiving GDNF therapy. These patients have been struggling to persuade Amgen to continue to administer GDNF to them so that they can maintain the improvements they've realized with this treatment but Amgen has refused claiming that the drug wasn't effective and had safety concerns. Since Neurturin and GDNF are in the same protein family and have similar characteristics in maintaining survival of dopamine-producing nerve cells, I am heartened to know that Ceregene is actively researching this closely related protein. I recently spoke with Don Lee, a research assistant at Ceregene and I understand that there are 12 people involved in their study 6 at U.C. and 6 at Rush. Neurologix has recently announced positive interim results of their Phase 1 trial of their core technology referred to as "NLX". Twelve patients in total have undergone gene transfer in this trial, four in each of three dose cohorts. On speaking with Dr. Martin Kaplitt, I gained a better understanding of the goals they hope to attain with this treatment. It seems that the effects of this treatment would be similar to those achieved with STN Deep Brain Stimulation but with less invasive surgery and hardware involved. The procedure involves the infusion of AAV-GAD via a hair-thin catheter into the subthalamic nucleus. After the infusion period, the delivery catheter is withdrawn and the incision closed. No hardware is left behind following this procedure. GAD (glutamic acid decarboxylase) is an enzyme which synthesizes the major inhibitory neurotransmitter in the brain, (gamma)-aminobutyric acid (GABA). GABA has been shown to have a calming effect on the subthalamic nucleus thus providing improvement of motor function in Parkinson's patients similar to that experienced with DBS. Although there have been many preceding years of education and experience in this field the studies being conducted by these companies are in relatively early stages. Still, I am always encouraged by and grateful for the work that is going on in gene therapy for neurodegenerative disorders and wished to share it with you today. Dee ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn