Those of us who attended last September's Parkinson's Unity Walk in New York
were privileged to hear a presentation by Dr. William Langston, president of
the Parkinson's Institute in California and author of the book, The Case of
the Frozen Addicts.
Ken Aidekman, chairman of the Unity Walk, transcribed the talk so it would be
available to a wider section of the Parkinson's community. Here is the text.
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I left Carmel by the Sea in sunny California about 10 o'clock last night so
that I could be here with you today and join you for this very, very special
occasion. I can't tell you how important I think things like this are.
Maybe I should start this talk by telling you next year we will be stronger
and bigger and we will pass that bill (The Morris K. Udall Bill).
I stand before you as a physician and researcher who has dedicated his entire
professional life to trying to find the cause and cure and ways to prevent
this disease. And with the help of people like you I think we can do that.
I'd like to just quickly review the week that we've had again - you've heard
different pieces of it, but let's just think about what we heard. My week
started one week ago in Sacramento where I met with a rally of over 300
patients working in the same way that you are today; a phenomenal gathering,
a lot of excitement and enthusiasm. On Tuesday Dr. Carly Kantor from our
institute and Muhammad Ali and his wife, Lonnie, had a press conference in
Washington and announced a $1.2 million drug trial for Pramipexole in
minorities. On Thursday . . . we passed the Udall Bill in the Senate. We
didn't quite make it in the House, but we'll be back. And today - this Unity
Walk. That's a phenomenal week for Parkinson's Disease. (Applause)
I'm going to take just a minute to talk a little bit about some of the
research that's going on. But first I want to try to share an insight with
you. Basic science isn't an easy thing to do in life. I once heard science
defined as "The process of going up alleys to see if they're blind." This
isn't easy work. And what I don't think you realize - and the insight I
would like to share with you - is how much it inspires those of us in the
clinic and the laboratory to see events like this. It means a tremendous
amount to those of us working in the field. And I can tell you that people
like myself - I'm going to go back and work twice as hard to solve this
disease after seeing an event like this. I want to thank you for that.
(Applause)
While I am at it I would like to thank the organizers of this event. I think
we should give them a big round of applause. Thank you all - all the people
that worked very hard on this.
Let's talk a little bit about the research. I have a little speech but I'm
terrible when I try to read things so I think I'm just going to talk from the
heart here. I want to give you a feeling of how vibrant the research area
is. I went to a meeting of a very large pharmaceutical company on Thursday
of this week. Their business office has pooled not a scientific review of
the literature, but a business listing of all of the companies and all of the
drugs and techniques that are being studied for use with Parkinson's disease.
There were over forty companies on that list and over a hundred different
strategies for treating Parkinson's disease. I hadn't even heard of half of
the companies listed. They were biotechs. There is a tremendous amount
going on now. That's the good news.
One of the areas I want to talk about is "Searches into the cause". There is
a huge amount of work in this area. Epidemiologic studies directed toward
trying to find agents in the environment that might trigger the disease. NIH
has just put out a request for proposals to look at the genetics of the
disease.
Some of you may know that there is a huge twin study going on which is
looking at all of the twins (from the United States) who served in World War
II. There were twenty thousand twin pairs. They've been followed for the
last fifty years now. We have now identified enough Parkinson's patients in
that cohort - they're (all) in their seventies - to probably make a
definitive statement on the genetics of this disease. That's probably less
than a year away.
New therapies. There are a lot of new drugs being tested. Some of you have
probably heard about Pramipexole. That looks like a promising new dopamine
agonist that may be on the market as soon as . . . early next year. There
are a number of other new agonists that are also being tested.
There are a whole series of other therapeutic strategies being tested with
this disease; drugs that block the breakdown of dopamine. These are new
strategies that have never been tried before. These are drugs that are
actually being tested in humans right now. So on the pharmacological front
there's a lot going on.
What about neurosurgery? Well, if you read the papers like I do, there's a
lot going on there, too. Everybody's heard about pallidotomy. Thalamotomy's
been around a long time. It's also an effective treatment for Parkinson's.
I still believe a lot of work needs to be done with pallidotomy before we're
going to know its place in history, but a lot of work is being done right
now.
There's a whole new technique called Deep Brain Stimulation where you can
reverse the process if you get side effects. This is being done not just for
tremors in Parkinson's, but also it's being attempted now in the Pallidum.
So we may have a whole new strategy in terms of that type of treatment of
the disease.
But, there's much more; the whole issue of growth factors. I think everybody
here has probably read about Amgen. A little known fact I find with my
patients is that while you have an eighty percent dopamine depletion - that
means eighty percent of the dopamine is gone, or more, by the time you get
Parkinson's - only about sixty percent of the cells are gone. What does that
mean? It means that there are a lot of cells still there that aren't doing
their job; they're not pulling their weight. Growth factors, conceivably,
could turn those cells back on. And that's where the excitement is. That
could be surgery without the surgery.
Amgen has actually started human trials with the most promising of these
(growth factors). It's called GDNF. They're taking what I call a 'down and
dirty' approach. Nothing fancy. They're just putting a small tube straight
into the brain and literally pouring this in to see if it will help reverse
the symptoms of Parkinson's. I think there's a chance it may work. We will
know soon. There are many other growth factors that are being worked on. I
think this is just the first stage in trying to treat this with, again, a
whole new strategy.
The whole world of molecular biology is having a tremendous impact on
research. All the way from looking at genetically determined risk factors -
we now have some data - we found some mutations in the human gene that
increase the risk for getting Parkinson's up to fourteen fold. This doesn't
mean that you are going to get the disease if you have it but your risk goes
up. We suspect the disease is the combination of both genetically inherited
risk and something in the environment. Neither by itself may be enough.
It's going to be a complicated pattern but we're really making some progress
in this area.
The final area I'm going to mention is the whole area of genetic engineering.
This is stuff that we're actually doing in the lab right now. In one set of
experiments, for example, we're taking skin cells and genetically engineering
them so that they can make dopamine. You can then take those very same skin
cells and put them back into the donor. So a person could come in, have a
skin biopsy, have those skin cells 'trained' to make dopamine genetically,
transplanted into the brain and conceivably replace the missing dopamine. No
supply problems, no ethical problems, no immunological problems.
But we're even going further than that. There are now techniques to actually
induce the genes straight into the brain without having to go through cells.
It's called in vivo gene therapy. This is being done in animals as we
speak. You can put genes in for growth factors, dopamine making enzymes
right into the area where it's missing. They can burst the DNA of the cells
so they can start making missing substances.
How close are we to the cause? the cure? I can't answer that. My sense is
that there are hundreds of things going on right now and I think that there's
a real hope that suddenly someplace they are going to come together and we're
going to have an answer to this disease. But I will tell you it's only going
to happen with the support and help of people such as you, Margot Zobel, Ken
Aidekman, Joan Samuelson and all of you here. So I thank you for all of the
scientists and physicians working with this disease.
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We hope that Dr. Langston's words have helped to lift your spirits and to
energize you.
And speaking of energy, it's not too early to start getting involved with the
next Unity Walk, which will take place on Saturday, September 27, 1997. Mark
your calendars! This time we will be walking in the middle of Central Park,
where thousands of New Yorkers will get to see us, hear our message and see
our logo, "Fund the Research, Find the Cure."
To pre-register for the Walk, or get information on organizing a "team" of
walkers, or sign up as a volunteer, or make a contribution, send an e-mail to
[log in to unmask], or call the Walk hotline: (212) 580-6505.
Let's keep the spotlight on Parkinson's this year -- and pass the Udall bill
in the process!
- Margot Zobel
