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Thanks, Dee and very interesting. Wish I understood it better. I do not look forward to having my batteries replaced. Also learned they can leak and cause chemical burns. Wish I wasn't so damn old now Ray ----- Original Message ----- From: "Dolores Buente" <[log in to unmask]> To: <[log in to unmask]> Sent: Sunday, October 02, 2005 7:03 AM Subject: PD and Biotechnology > 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 > ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn