Re: Altered cells deliver Parkinson's therapy to brain

["ljherman1@xxxxxxxx" <ljherman1@xxxxxxxx>]

-- Inge Clody <ingeclody@xxxxxxxx> wrote:
THIS IS WONDERFUL NEWS
HOWEVER...........
HOW LONG WILL IT TAKE BEFORE IT IS FDA APPROVED?

Yes, GDNF is very promising therapy, however this research using stem cell or 
gene therapy delivery methods is in pre-clinical stages, and  is still years 
away from clinical use.
An article in The Milwaukee Journal Sentinel, dec.15, 2005, stated that, 

"..The study's findings are fascinating and the approach clearly shows promise, 
said Bradley Hiner, an assistant professor of neurology at the Medical College 
of Wisconsin who treats Parkinson's patients at Froedtert Memorial Lutheran 
Hospital.

However, it is a disservice to Parkinson's patients to call it a potential new 
treatment. Doing so gets their hopes up when clinical trials likely are years 
away, he said.

"It's a little premature to think about this as a potential treatment for 
humans when only three primates were done and the results were equivocal at 
best," Hiner said. "There's obviously quite a bit of work before anyone is 
getting this transplanted into humans."

HOWEVER, GDNF, delivered by a pump infusion method has been used to 
successfully treat 15 advanced PD patients for a number of years in 2 Phase I 
clinical trials (at the Un. of Kentucky and at Frenchay Hospital in Bristol, 
UK). Their treatments and further human clinical trials were halted by Amgen,( 
the biotech company that produced the GDNF) for controversial reasons. An 
autopsy of one of the trial participants showed evidence of neuronal growth in 
his brain.

The trial participants and other PD advocates have been campaigning for over a 
year to convince Amgen to provide compassionate use of the treatment for the 
trial subjects, and restart the clinical trials.
This infusion form of delivery of GDNF could be available years before 
gene therapy or stem cell delivery is expected to be available, but Amgen holds 
the patent and so far is refusing to develop it further or lease it to another 
company.

For more about this issue, see:

http://pdpipeline.org/gdnf_overview.htm
http://www.grassrootsconnection.com/
and a video of before and after GDNF treatment
http://www.grassrootsconnection.com/grcissue_GDNF_research.htm#View_film

Linda Herman

On Wed, 28 Dec 2005 08:03:37 +0100 "M.Schild" <mmoo@xxxxxxxxxxxxxxxx>
writes:
> Altered cells deliver Parkinson's therapy to brain
> Tue Dec 27, 2005 7:17 PM GMT9
>
>    NEW YORK (Reuters Health) - Genetically modified nerve
> 'progenitor' cells
> can be used as mini-pumps to deliver nerve growth factor to the
> brain, a new
> study in animals shows.
>  The results suggest such an approach could be used to treat
> Parkinson's
> disease and other brain diseases in humans, Dr. Clive D. Svendsen of
> the
> University of Wisconsin-Madison and colleagues report.
>  A nerve growth factor called "glial cell line-derived neurotrophic
> factor" (abbreviated to GDNF) has been shown to protect
> dopamine-producing
> neurons, which are lost in Parkinson's disease, Svendsen and his
> team note in
> the research journal Gene Therapy.
>  In fact, it's safe to infuse GDNF into brain regions of patients
> with
> Parkinson's disease, according to some studies, and it seems
> effective.
> However, delivering the drug in this fashion is complex and only
> reaches a
> single point in the brain.
>  In the current study, using rats with symptoms akin to Parkinson's
> disease,
> the researchers investigated the effect of human neural progenitor
> cells
> engineered to produce GDNF.
>  The rats were transplanted with the modified cells, and after two
> weeks these
> were seen to have migrated to affected areas and to be secreting
> enough GDNF
> to extend the survival of dopamine neurons and promote outgrowth of
> nerve
> fibers.
>  By five weeks post-transplant, the animals showed a "strong trend
> toward
> functional improvement," and at eight weeks the cells were still
> releasing
> the growth factor.
>  Tests in elderly monkeys showed the cells survived and continued to
> release
> GDNF for three months after transplant. None of the animals in the
> studies
> developed brain tumors.
>  Svendsen and his colleagues conclude that their results "show that
> combining
> human progenitor cell therapy with ... gene therapy is a powerful
> approach to
> the future treatment of Parkinson's disease and other neurological
> conditions."
>  SOURCE: Gene Therapy, online December 15, 2005.
>
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Inge Clody
Minado, Reg.
www.flash.net/~minado1

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