FROM: LA Times, Feb 17, 2005

Amgen Drug Is Said to Work
A study says the trial Parkinson's treatment improved motor function,
contradicting the company's research.

By Denise Gellene, Times Staff Writer

Just days after Amgen Inc. said it would stop supplying patients an
experimental drug for Parkinson's disease, a research team from the
University of Kentucky reported in a medical journal that the medicine
worked in a clinical trial.

Don M. Gash, an author of the study, said he hoped Amgen would reconsider
its decision and provide the trial drug to 48 patients who participated
in company-supported studies. Many of the patients have been lobbying
Amgen for the drug, which they see as their only hope.

"Controversy can be good if it can get people not to take a rigid
position and not to create barriers," Gash said.

Amgen spokeswoman Marie Kennedy said she was not aware of the study.

Last week, Amgen said it would no longer provide the drug to patients
because its studies showed that it did not work and might cause permanent
harm. Kennedy said Wednesday that the company hadn't abandoned its
research, and would continue to support laboratory studies of the drug by
academics.

The study, published in the Journal of Neurology, reported that 10
patients who had received the drug, known as GDNF, had an average 30%
improvement in such areas as balance, gait and speed of hand movements.
The study followed the patients for six months.

However, seven of the 10 continued to receive GDNF for a full year before
Amgen stopped providing the medication. Those patients further improved
and saw a 45% increase in their motor function, Gash said. "It is the
difference between being wheelchair-bound and being able to walk for
several miles," he said.

Researchers saw no signs of brain damage that Amgen observed when the
company tested high doses of GDNF in monkeys, Gash said. The monkeys were
given six times more drug than patients in the University of Kentucky
study.

"The difference between poison and medicine is the dose," Gash said. "The
patients did very well and the side effect profile is excellent."

The report said researchers could not rule out the so-called placebo
effect in which the process of being treated, rather than the drug
itself, causes patients to improve.

An earlier Amgen-sponsored trial of 34 patients ended in disappointment.
The company divided the patients into two groups — half received GDNF and
the rest were in a control group given saline solution. At the end of six
months, Amgen concluded that GDNF was no better than a placebo.

But Gash said there were differences between the two studies that might
explain the results.

All patients received GDNF directly into their brains through tubes
connected to pumps implanted in their abdomens. In the University of
Kentucky study, the drug entered the brain in bursts through a catheter
with 40 holes that Gash likened to a "soaker hose." Amgen used a catheter
more like a garden hose that delivered the drug in a continuous drip.

Gash said he has discussed his findings with Amgen scientists. "We
certainly wish Amgen would reconsider," he said.

http://www.latimes.com/business/la-fi-amgen17feb17,1,865660.story?coll=la
-headlines-business&ctrack=3&cset=true

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Additionally a two year follow up of the Phase I study in Bristol, United
Kingdom has been published in Annals of Neurology, February 2005 issue
--  " Intraputamenal infusion of glial cell line-derived  neurotrophic
factor in PD: A two-year outcome study",   Nikunj K. Patel, et.al and
reports continued improvement  in all 5 patients over the 2 years and no
serious side effects. The authors concluded, that treatment with GDNF
resulted in improvement in symptoms AND slowed the disease progression!

"“Our results indicate GDNF’s potential as a therapeutic agent in PD,
from its ability not only to provide symptomatic relief, but also to
possibly modify the disease state, distinct from other current
therapeutic strategies; however, continuing dopamine replacement therapy
was required. The early onset of symptomatic improvement, accompanied by
an increase in 18F-dopa uptake immediately surrounding the cannula tip,
seems compatible with a functional upregulation in residual dopaminergic
neurons. The progressive and sustained improvement in symptomology, and
increased 18F-dopa uptake throughout the putamen at 24
months,15 is suggestive of reduced disease progression.”

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