There has been a lot of press and interest lately about the recently-concluded Georgetown Univ. Dept. of Neurology clinical trial testing the cancer drug nilotinib (Novartis) on patients with Parkinson's Disease and Diffuse Lewy Body Disease. Simply Google "Georgetown nilotinib clinical trial" and you will see lots of articles.

While obviously very early in the clinical process, the results are extremely encouraging for people with these and related diseases, their families and caretakers. I called Georgetown a few times for answers about a Phase 2 trial, but they are likely inundated with expressions of interest, and as a result, have not responded to me.

For patients like my mother (age 78, who has both PD and LBD) who sadly don't have the luxury of time to wait for successive round(s) of trials to see their course, I wonder this: IS THERE ANY HOPE TO SOMEHOW TAKE ADVANTAGE OF THIS PROMISING DRUG NOW? I would love to know if there are any forums out there (online or elsewhere) where people are at least talking about the "compassionate use" of an approved drug like nilotinib for something that's it was not specifically intended for? The good news to me is that nilotinib is already an FDA-approved drug, and if there are doctors out there willing to prescribe it to PD and LBD patients with the appropriate care and monitoring, why not try it for people who basically have little hope of improvement otherwise. I see my mother declining daily, and it pains me to know that there may be something approved for use by the FDA that very well could help her NOW before it's too late. The very high cost of the drug, of course, is an issue that I simply don't have an answer for, but I would at least like to be part of a conversation about the possibilities.

If you have any ideas or thoughts on the subject, I would really love to hear them.

Thank you for listening,
Tim (first time NeoroTalk user)

Tim, I can certainly sympathize with your sentiments. It is very difficult in the U.S. to get compassionate use for a drug, particularly when the it would be an off-label use based on a very small, open-label study. I am not aware of any organized use of Nilotinib, or other similar drugs in the same class, for PD patients. Putting the cost aside for the moment, you would really need to get a combination of a neurologist and oncologist to help you out. This drug, even in lower dosage, can have serious side effects, particularly on red and white blood counts. Regular blood tests are required. If it works, you would also need the help of a neurologist to properly titrate down from current PD meds. On top of everything else, you have to realize that, in theory, the drug needs to be taken for life. So, it's not like you can just get someone to come up with a months worth of the drug to try it out. In essence, it would be difficult to do, but of course, not impossible. Of course, there is also that minor issue we put aside, the cost. At today's pricing, it would be approximately $3,000/month.

All that being said, I am aware of some docs who have tried it for Alzheimer's patients. None were successful to my knowledge, although it was used on late termers.

Gary

By the way, although I am not suggesting you actually try it, if you do, please make sure you have reputable doctors involved throughput the process. I'm sure for the right amount of money, you can always find someone, with a diploma hanging on their wall, willing to sell you the drug.