Here's an obvious question. I assume there are people with PD who have come down with this type leukemia and have been treated with this drug.
Hopefully, their leukemia was put into remission but what happened with their PD? Did they see any improvements? Is anyone asking?

ashleyk, I think that's a great question. I would guess that given the population of PWP that have also taken nilotinib is not that high. CML in general is not that common (1 in 625 lifetime chance of occurence). Also, the drug has only been on the market for about 5 years and is only used for a subset of CML patients. Nevertheless, there must be some PWP that can be found that can at least provide some anecdotal information about the drug.

I think the biggest problem is that the information may not be that useful. First, the researchers are suggesting testing nilotinib on early stage patients. Therefore, you would have to narrow down the subset population even more to just those PWP who were recently diagnosed. More important, though, I believe is the dosing issue. When used for CML, the drug is given in very high dosages in order to cause cell autophagy. Essentially, kill the cancer cells. The opposite was done in this neurological research. Their theory was to use the drug in extemely low dosage in order to keep cells alive by ridding them of an excess of garbage a-syn.

What we really need right now is for the manufacturer, Novartis, to open up their pocketbook and fully support the funding of this new research. Given their ability to put this right into Phase 2, they should be able to move it along quicker than two university doctors at Georgetown U by themselves.

I keep thinking research of already approved drugs in MUCH LOWER DOSES might be carried out in small open label trials.

Patient funded ?? Kickstart funded ? Part patient funding with matching funds from MJFF ??

I, for one, am willing to make a significant donation.

i'd contact the researcher, nothing to lose. find out how serious he is about a phase2 study and if he would consider crowd funding. we can't keep waiting years to find out if a cere-120 might help imho, something has to change in their experimental designs imho. i hope that cere-120 failure would have stimulated more public discussion, it doesn't seemed to have. hardly a peep.

That's an interesting suggestion. I think I'll try to do it.

gulp, good luck. contacting pd researchers who have publicized great results with mice can be a full time job.

I would lke very much to participate in your funded clinical trial. Athough w/o placebos!

Rick Anderson