Thanks to everyone who have expressed their thoughts so articulately on the
subject of corporate motivation with respect to approval of new treatments.
Also thanks to Sid Levin for his advertisement of the Parkinson-FDA-Industry
initiative.

My own view of Pharmaceutical companies is more like Paul's.  Significant
advances in treatment are a goal, not only for profitability motivations but
for humanitarian, professional and scientific motivations as well.  Drug
company executives after all are people with families, they are often
scientists as well as businessmen and women.  I have been in contact with
Amgen executives regarding the NIL-A trials over the past 2 years, and my
impression is that they, as well as the FDA regulators, are motivated
primarily by science.  They are seeking scientific evidence to prove the
efficacy of the medication.

Of course this science is sponsored by a public corporation that also
considers the payoff when making huge investments necessary to prove
effectiveness.  Here is where we are at a crossroads.  The press release that
described the results was issued by Guilford Pharm., not Amgen, according to
Guilford Investor Relations to halt the steep slide in their stock price.
The stock price of Amgen which is a larger company with a number of
successful products, was in the meantime rising on a better than expected
earnings report.  Still unclear to me is why Guilford would characterize the
results as "does not produce a substantial reversal of the motor symptoms of
PD" without more qualification that the sample size was small and the trends
were in the predicted direction, i.e., the results were not conclusive but
positive.

Something is missing from this picture.  Is the increase of nerve endings of
1.2% and 2.5% for treatment groups vs -.0.15% placebo clinically
insignificant?  To me this is going in the right direction and given how
little is known about halting or reversing the direction of PD, it should be
a motivation for further study.   (the FDA does not now accept Spect/PET
scans as surrogate markers to measure progression anyhow, because it hasn't
been established that improvements in these measures are related to
functional improvements.  Until now there were no safe treatments that could
reverse deterioration in these Spect/PET measures. Amgen to its credit has
included these experimental measures in the protocol to collect the data
necessary to validate these measures).

Also 17% and 21% of patients that showed significant improvements in H&Y
scores. Who are these people? Remember all participants were < 5 years
diagnosed so they were presumably not severely impaired (i.e., had little
room for improvement), and adjustment for age and duration the results were
statistically significant.  The press release did not specify whether it was
the older longer diagnosed, younger-shorter-diagnosed,
older-shorter-diagnosed or younger-longer-diagnosed segment that was
significantly better, but this is an obvious avenue for further study.

It is especially important that this medication continues to be tested
because of anecdotal reports from participants in the trial that not only
motor symptoms were affected but more problematic mood, cognition, and
autonomic effects of PD (digestion, smell, etc.) not treatable by sinemet
were improved.  If only 20% of patients are affected by this medication, it
may qualify for "orphan drug" status which provides incentives (certain
patent rights, etc.) to develop drugs for small target groups.

Amgen has not made a corporate decision to continue development NIL-A.  I
believe and hope that the Guilford Press Release was a hasty response to
disappointing news, not a more calculated signal.  Given the facts, Phase III
is the next logical step.  I intend to communicate these views to my contacts
at Amgen.  Comments and feedback are appreciated. I would also appreciate
private responses from anyone with contacts and information at Amgen or
Guilford.

Perry Cohen
Washington DC
www.parkinsonscare.org

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