Update on nilotinib, including MJFF webinar on Tuesday, Aug 2

This post may be of most interest to those dealing with Lewy Body Dementia (Dementia with Lewy Bodies and Parkinson’s Disease Dementia).

I first started hearing about the leukemia drug nilotinib back in 2013.  You can find an email sent to the group back in 2013 here:


In 2013, Georgetown published some basic research that showed that nilotinib getting rid of alpha-synuclein in the brains of transgenic mice with Parkinson’s.  A Georgetown press release mentioned that they wanted to do phase II studies in PD, LBD, MSA, and PSP — a surprising combination since PSP is not an alpha-synuclein disorder.

In October 2015, Georgetown announced results of a phase I clinical trial of nilotinib in 12 patients for a 6-month period.  Unfortunately there was a mix of diagnoses — 5 had a DLB (dementia with Lewy bodies), 3 had PD-MCI (PD-mild cognitive impairment), 2 had PDD (Parkinson’s disease with dementia), one had PD plus MCI, and one had PD.  Phase I studies are safety studies so I found it confusing that the researchers claimed the “drug improved cognition, motor skills, and non-motor function” in the 11 patients who completed the study.  (One had to drop out due to a serious side effect.)  The study size was very small and there was no placebo-control.  Also, the cancer drug costs several thousand dollars per month.

In November 2015, I asked several movement disorder specialists about use of nilotinib for PD or any of the atypical parkinsonism disorders.  All of the MDs scoffed at the idea.  One said that the nurse in the movement disorders clinic where he works worked for many years in oncology.  That nurse was frightened by the idea that anyone without leukemia would take nilotinib.

In December 2015, I learned that a member of our local support group (whose husband may have LBD) was able to obtain a prescription from a local physician for nilotinib.  It was costing them a bundle.

People are still talking about nilotinib, especially in the PD and LBD communities.  The Georgetown team’s phase I results were finally published in July 2016.  You can read the results here:

Nilotinib Effects in Parkinson’s disease and Dementia with Lewy bodies

Along with the Georgetown results, the journal published an accompanying editorial titled “Nilotinib – Differentiating the Hope from the Hype.”  The authors of the editorial said:  “We debate the safety of Nilotinib and the reported efficacy signals.  We emphasize that due to the small sample size, and lack of a control group, it is impossible to rule out a placebo effect.”  You can read that editorial here:

Nilotinib – Differentiating the Hope from the Hype

If nilotinib is of interest to you, I’d suggest checking out this Michael J. Fox Foundation overview of the scientific rationale behind nilotinib:


Or, if you still want more, the Michael J. Fox Foundation will be having a webinar on Tuesday, August 2nd, from 9 to 10am CA time about nilotinib as a possible treatment for PD.

You can register here:


The panelists “will discuss the scientific rationale behind nilotinib for Parkinson’s, findings from recent research and next steps in the study of this drug.  Q & A with the audience will follow the discussion.”