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mackenzie,

I would put my $10 million into research on the work being done by Avigen. They are using a gene therapy viral vector delivery system to repopulate the brain with the enzymes that convert levodopa to dopamine. These enzymes are lost along with the natural dopamine produced in our brains as the dopamine neurons die off. One of the problems with using Sinemet as a long-term medication is the onset of on -- off fluctuations and the appearance of dyskinesia after a few years of using this medication. The theory of the scientists at Avigen is that repopulation of the brain with the conversion enzymes will allow the brain to convert levodopa into dopamine as effectively as it did when a patient first started taking the drug. In other words, the loss of these enzymes causes the fluctuations and dyskinesia because conversion becomes haphazard as these enzymes continue to die off. In an extraordinary animal study using 35 primates, an unusually large number, the effectiveness of this!
  therapy
 was remarkable, with all of the primates showing a substantial increase in their ability to convert levodopa to dopamine smoothly and without fluctuations or dyskinesia. According to the Avigen researcher in charge of this project, Dr. Alvin Luk, they believe that the enzymes will continue to successfully convert levodopa into dopamine for as long as 15 years.

In other words, middle and late stage Parkinson's patients would be able to use this therapy to extend the effective life of their Sinemet for a decade or more, and the Sinemet would respond as effectively as it did when the patient first began carbidopa levodopa therapy. This would be a godsend to mid-and late stage patients and enable them to hold out for another decade or more with something approaching the quality of life they experienced when they first began carbidopa levodopa therapy. It would allow people who would otherwise be lost to this disease to hang on until a real therapy, one which effectively regresses the disease rather than simply masking and symptoms, can be found and reach the marketplace.

Avigen has recently reported success with the first phase 1 human clinical trial being conducted using this therapy.

So that's what I would do with my 10 million. It's a little selfish, because I've had PD for 10 years, but I think you could save a couple of generations of Parkinson's patients until the cavalry comes.

Best,

Greg Wasson


mackenzie <[log in to unmask]> wrote:
hi all,

i am curious about something. let's say you had $10m
(number chosen at random) to donate to PD research,
and just for this little inquiry, let's say there are
only two areas of research that you can fund. those
areas are:

1) research into alleviating some of the non-motor
problems of PD - for example, sleep disorders,
depression, fatigue, pain, and i am sure there are
more

or

2) research into finding treatments that achieve
something close (though not necessarily 100%) to the
degree of symptom relief that l-dopa provides but will
never cause dyskinesias (or any comparably disabling
side effect)

you can allocate your $10m any way you want - all in
#1, all in #2, or divided between them in any ratio
you like.

how would you allocate your $10M?

thanks for playing,
mackenzie

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