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Thank you for sharing this intesting information.
A.G.

On 10/2/05, Dolores Buente <[log in to unmask]> wrote:
>
> 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
>
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