 |
 |
Thank you, Dee, for this heartening news. I just wonder if it could be possible that I might not have to spend my life unsteady on my feet, shuffling like a marionette, and shaking like a leaf. It seems there might be a chance... And Ray, there are a lot of braindead rock stars in your age range that would do anything to have your mental acuity. My son, 18, also complains about being born too early. He'd rather live in a Star Wars kind of universe. Rise above. Hold out. Hold on. Rick ----- Original Message ----- From: "rayilynlee" <[log in to unmask]> To: <[log in to unmask]> Sent: Monday, October 03, 2005 10:13 PM Subject: Re: PD and Biotechnology > 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 ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn