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
>
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