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What's Hot in PD? Nilotinib and Repurposing Other Drugs to Treat Parkinson’s Disease

What if I told you that it was possible that existing and already FDA approved drugs could possibly be breakthrough treatments for Parkinson’s disease?  It is an exciting potential reality.  Our Center of Excellence at Georgetown University has been exploring one such compound, which has been previously used as an anti-cancer drug.  The drug is actually already FDA approved to address chronic myelogenous leukemia.

As Parkinson’s disease progresses, protein deposits accumulate in the brain.  These protein deposits are called Lewy Bodies. There has been a great interest in developing drug therapies that may have the potential to clear out these abnormal protein deposits.  Dr. Fernando Pagan at the NPF Center of Excellence at Georgetown University has been testing just such a therapy. The drug, called Nilotinib, has been used in chemotherapy for leukemia, but recently Georgetown University researchers repurposed the drug for its potential to “alter the abnormal protein build up in Parkinson disease and Diffuse Lewy Body Disease patients.” Dr. Pagan’s trial, which is listed on, is primarily interested in determining:

  • If disease symptoms change
  • If there are changes in the protein in the spinal fluid
  • Whether inflammatory markers in the blood change after taking Nilotinib.  

Nilotinib blocks the non-receptor Abelson (Abl) tyrosine kinase, but its exact mechanism of action in Parkinson’s disease is still under investigation. The hope is that this approach could slow down or stop Parkinson’s disease progression. The results of the first safety study should be available soon. The dosages used for this study were far less than those used for chemotherapy. We suspect that there will be many more anti-cancer and other FDA approved drugs that may be candidates for repurposing to Parkinson’s disease populations.

You can find out more about NPF's National Medical Director, Dr. Michael S. Okun, by also visiting the NPF Center of Excellence, University of Florida Health Center for Movement Disorders and Neurorestoration. Dr. Okun is also the author of the Amazon #1 Parkinson's Best Seller 10 Secrets to a Happier Life and 10 Breakthrough Therapies for Parkinson's Disease.