Yes, it is hard to accept Amgen's position. That's why a group of 
trial participants and PD advocates have been tryign to convince 
Amgen to change its decision for the last 2 years.

My guess is amgen didn't think they would make enough money on GDNF. 
The bottom line is profits over people.
Further complicated by the unexpected death of the person who was in 
charge of gDNF research and its main supporter at the company -- 
there was no one else with his knowledge of the treatment  to take 
over the project. And the execs at Amgen don't seem willing to change 
their minds, no matter what evidence is presented, or how many 
patients plead with them. 

Amgen has been withholding monkey toxicology data for two years that 
could eiether confirm or disprove their claims that GDNF caused brain 
lesions in some of the test monkeys. Some of the trial doctors 
believe the lesions were  caused by the sudden withdrawl from very 
high doses of GDNF in those monkeys - not from the substance itself. 
The tox data could clear this up.

There will surely be future advances in other treatments such as gene 
therapy and stem cells, but none are as close to clinical use as the 
infusion delivery of GDNF could be. We are all loosing a potential, 
powerful near term treatment. The saddest part of the story though is 
the plight of the trial participants in the UK and the US who were 
given their lives back for a short time and then had them ripped away 
from them again. They deserved to receive compassionate use of GDNF. 
The FDA said OK.... Amgen said NO!

Linda
full story at
www.pdpipeline.org
and www.grassrootsconnection.com

-- rayilynlee <[log in to unmask]> wrote:
It is so hard to understand Amgen's position.
----- Original Message -----
From: "[log in to unmask]" <[log in to unmask]>
To: <[log in to unmask]>
Sent: Tuesday, October 31, 2006 7:45 PM
Subject: Why won't they let Parkinson's sufferers take a life-
changing drug?


More on GDNF from the United Kingdom:
From the Daily Mirror
31/10/06 - Health section

Why won't they let Parkinson's sufferers take a life-changing drug?
By NIKKI MURFITT

When Tom Isaacs was 27 he was diagnosed with Parkinson's disease. The
condition gradually destroys the brain's ability to control the
muscles - there is no cure.

Determined to give himself the best possible prognosis, Tom embarked
on a very personal journey to meet leading scientists in the hope of
finding new treatments that would help him.

Three years ago it seemed he'd found the answer: a new drug GDNF
(glial derived neurotrophic growth factor). A group of Parkinson's
patients had been treated with GDNF at Bristol's Frenchay Hospital
and their transformation had been remarkable: sufferers who'd been
trapped in a living hell were suddenly able to walk, talk and smile
again.

Parkinson's is caused by a shortage of dopamine, a chemical messenger
involved in movement, mood and behaviour. Why this happens is still
not known.

GDNF seems to work by stimulating dopamine production and preventing
degeneration of the brain cells. The drug is delivered via a catheter
permanently implanted in the brain. The catheter is connected to a
Jaffa cake-sized pump sewn into the abdomen.

When GDNF was given to the Bristol patients the results were
astonishing. 'Men who had been unable to get up out of a chair
unaided were walking normally across a room. Their hand co-ordination
was unbelievable, in exercises they could move their hand easily from
left to right, something that had previously been impossible under
the onslaught of Parkinson's,' says Tom.

So impressive were the results that the study was rolled out to North
America and by September 2004, 50 patients were receiving the drug.
The Parkinson's Disease Society in the UK planned to hand over £1.2
million to fund further trials in Bristol.

But suddenly, at the end of 2004, and without warning, this lifeline
was snatched away. Amgen, the American drug company which holds the
patent for manufacturing GDNF, claimed the drug was dangerous as it
could cause brain damage and refused to continue prescribing it.

Amgen's decision has caused huge divisions within the Parkinson's
community, pitting scientists against each other and leaving patients
angry and frustrated.

As Tom explains: 'We were left with a situation where half the
doctors involved in the studies sided with Amgen and the other half
sided with the patients who wanted to keep taking the drug.

'The doctors didn't believe there was anything wrong with the drug
and yet it was being taken away. They couldn't offer their patients
an explanation because they did not agree there was one to give.'

Tom, now 38, had hoped to be able to have GDNF treatment himself, but
is now leading the fight to get the drug back in use, and with his
charity Movers and Shakers has raised more than £1 million to fund
further research.

One of those desperate to be given the treatment is Stephen Waites.
He was also 27 when he was told he had Parkinson's, and as his
condition deteriorated, he had to give up his flourishing
architecture business and relied instead on benefits.

But 30 years later in 2001, when he was 58, he became part of the
Frenchay Hospital trial. He says he was given back his life. 'I was
able to restart my business. I had more than 40 projects on the go
and I really had taken on a new lease of life. I could drive again
and even bought myself a new Jaguar.

'GDNF made me independent, and after so many years living with
Parkinson's, I began to get my confidence back. It was a cure because
it improved my condition more than 60 per cent - I could not ask for
more than that and I was delighted.

'When I was told at the beginning of last year that the drug was
being taken away I felt that they'd taken my life away along with it.
I had committed myself to a huge workload and without the drug I
couldn't fulfil my commitments.

'I've deteriorated since the GDNF was stopped - I can't drive any
more so the Jaguar has had to go and we also had to downsize the
house because I'd been able to start earning good money again but
that went too.

'I was willing to sign anything to absolve Amgen of responsibility if
anything went wrong, if only they'd let me keep taking GDNF, but they
were having none of it.

'I've been very low mentally for the past 12 months and I just can't
seem to rise above it.'

Tom adds: 'I think what hurts most is the total lack of communication
by Amgen, who have put up a brick wall and won't discuss the issue.
Two patients in America sued the company in an effort to
gain 'compassionate access' to the drug but lost.

'It has been a crushing blow, especially as it has not been seen to
be doing any harm to anyone, quite the reverse.' In the human trials
in America some of the catheters became dislodged and as a result
GDNF was floating around the brain instead of getting to where it was
supposed to go.

'There were fears that this would give rise to antibodies in the
brain which could then attack a patient's own reserves of GDNF and
lead to brain damage,' says Tom.

'One or two patients on the American trial had shown up in scans as
having antibodies, but their specialists said they'd had them there
for three years and it hadn't done them any harm.'

Amgen also said that several monkeys that had received high does of
the drug suffered irreversible brain damage, and subsequently died.

A spokesman for Amgen, David Polk, explained that the company had
stopped providing GDNF because of 'serious safety concerns' and the
absence of any demonstrated medical benefit.'

'Stopping the GDNF trial was an incredibly difficult decision, but
given what we learned about GDNF we could not ethically continue to
administer it to patients.

'We are actively pursuing further understanding of the toxicity and
safety issues, as well as the possibility for a safe and effective
alternate method of delivery.'

The patients understand that in a climate of litigation Amgen had to
be cautious. But, says Tom, 'They believe that to take the drug from
those on the trial who were willing to sign anything to stay normal
and seemingly Parkinson's-free just seems cruel.'

Dr Stephen Gill, a neurosurgoen who headed the Bristol trial,
says: 'GDNF is the best possible drug for Parkinson's and as close to
a cure as you are going to get and our results in Bristol showed
this. Unfortunately it wasn't demonstrated as well in the Amgen
trials in America.

'It was when we learned that our patients would be stopped from
having GDNF. The department was in tears and it was very stressful
for everyone involved.

'I believe that Amgen's tests, which led to them withdrawing the
drug, were inappropriate and were not a realistic test of the
toxicology of a drug. I do not feel that there is a safety issue at
all.'

Although the decision to withdraw GDNF may seem unfair, Dr Piers
Benn, a lecturer in Medical Ethics and Law at London's Imperial
College, says: 'It comes down to a risk-benefit analysis. If it's
believed that the drug may produce side effects then the drug company
has to operate cautiously and within the law.

'You can talk about informed consent from patients, but when someone
is ill and anxious for a chance to improve their condition, they
don't look at the situation objectively and don't always take into
account the risk involved.'

Funded by the Movers and Shakers charity, Dr Gill and his team are
investigating a safer form of delivery for the drug. Tom has also
asked Mary Baker, chair of the World Health Organisation's steering
committee on Parkinson's, to preside over a meeting with Amgen.

'If we can prove to Amgen that this new system is safe and effective
then they would have to reconsider their decision,' he says.

Meanwhile, Tom, whose condition is managed with a cocktail of 15
tablets a day, has given up his job as a chartered surveyor and now
raises funds to find a cure for Parkinson's.

He says: 'I know that neither GDNF or anything else is going to make
it go away, but it can improve the quality of my life.

Tom Issacs can be heard in Chasing A Cure, BBC Radio 4, November 8.

http://www.dailymail.co.uk/pages/live/articles/health/healthmain.html?
in_article_id=413634&in_page_id=1774

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