Monday morning
To all:
Let me say at the outset (as I stated some months ago in this forum) that I
believe the real future for the treatment of Parkinson's and other
neurodegenerative disorders is in "neurotrophic (growth) factors", theraputic
substances which protect neurons from deteriorating and even revitalize dying
or dead neurons or regenerate new neurons.
Most of the research in this area is being carried out not in government labs
but in biotech companies financed in the good old fashioned free-enterprise
way by public stock offerings and other forms of non-governmental financing.
I should stress that many of these companies are still in the research and
development phase, have never made a profit, and are considered to be highly
speculative and risky from an investment standpoint. Still, a lot of
exciting research is going on which will result in some amazing medical
breakthroughs in the next five to ten years.
There is important news this morning in this field which, while not directly
or immediately affecting Parkinson's, has promising potential for treatment
of Parkinson's disease and other neurodegenerative disorders.
Cephalon, Inc., a biotech company based in Pennsylvania, has for many months
been conducting clinical trials of a substance it developed known as
"Myotrophin", which is a recombinant form of human insulin-like growth factor
- 1 (IGF-1), a neurotrophic factor. Myotrophin is being used (in trials
only, so far) in the treatment of patients suffering from Amyotrophic Lateral
Sclerosis (ALS), commonly known as "Lou Gehrig's disease". ALS is a
devastating, fatal disease of the nervous system characterized by the
chronic, progressive degeneration of motor neurons. The loss of spinal motor
neurons leads to muscle weakness, muscle atrophy, and eventually, usually
within 3 to 5 years of diagnosis, to the patient's death.
This morning, Cephalon announced the results of its trials in Myotrophin, and
they were viewed by analysts as being very favorable, apparently showing a
slowing or even halting of the progression of the disease and improving
movement and coordination. I should stress that all we have so far is
information from trial results involving a relatively small number (260, I
believe) of ALS patients, and even assuming everything is favorable, it will
still take an agonizingly long time (1.5 years or more) to be approved by the
FDA and be commercially available.
The implications for Parkinson's disease patients (and I count myself among
that select and distinguished group) are exciting. If neurotrophic factors
can be shown to protect and enhance the growth or re-growth of neurons and
slow or halt the progression of one devastating form of neurodegenerative
disorders, it stands to reason that is can be done for others, such as
Parkinson's. Cephalon and other biotech companies are currently working to
develop "small molecule" growth factors which will pass through the
blood-brain barrier, as would be necessary for Parkinson's and Alzheimer's
diseases. Myotrophin is a "large molecule" factor.
As I'm typing this, my broker has called and I've just listened to Cephalon's
president, Frank Baldino, on a brokerage conference call, describe the
trials, the "placebo patients" who progressed (deteriorated) normally with
this swift moving disorder, meeting certain readily observable deterioration
"milestones", while the "medication patients" had yet to hit the milestones
by the end of the 9 month trial. He said it was the first time in history
that an engineered molecule has been shown to slow or halt the progression of
a neurodegenerative disorder. He described it as "exciting news" both for
ALS patients as well as others suffering from neurodegenerative disorders.
I agree. It is very, very exciting news. Nothing will change overnight for
us as a result of the disclosures today, but there IS hope on the horizon for
us; there IS good reason for us to hang on, fight the good fight, coordinate
our efforts and resources, publicize our cause, work for increased funding of
research, exercise, eat wisely, be sensitive to proper levels of medications,
and live our lives as fruitfully as we can. There IS a light at the end of
the tunnel that we're in, and the light just got a little brighter. I
continue to believe that Parkinson's disease will eventually be cured.
Best regards,
Larry Allen ("[log in to unmask]")
