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(I tried to post this a couple of days ago, but I think I sent it into the ether - but if its a dupe, forgive me.)

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:
Thanks so much to everyone who has answered so far, and anyone else who has an opinion on the subject, please do send it on.



"Willy Loman never made a lot of money. His name was never in the paper. He's not the finest character that ever lived. But he's a human being, and a terrible thing is happening to him. So attention must be paid...Attention, attention must finally be paid to such a person." - Arthur Miller - Death of a Salesman
















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