Six month follow up on Nilotinib trial.
 

During my 6 month follow up neuro appointment at Georgetown Medstar Hospital I inquired how the Nilotinib trial was going. One of my neurologists is involved with the trial and gave me a brief review. Nothing has been published because the trial is for 9 months but there are some very positive results appearing at 6 months. This trial has 10 "end stage" PWP (this is the term she used) and the purpose of the trial is to test the safety of Nilotinib, a drug used to tx leukemia. So far abnormal proteins and tau have been reduced in the brain. Levels have been measured in spinal fluid and cell activity has been detected in blood work. By cellular activity this means dopamine production by the cells once damaged by abnormal proteins. In fact enough dopamine was produced that Parkinson medications had to be reduced in some patients. There have also been positive cognitive changes. At least one patient who hadn't talked in years started talking. All the patients received Nilotinib no placebo group.
This trial will finish up in the fall and another trial will begin around November. This was the estimated time frame given to me today. I will be included in the next trial if they are testing mild disability PWP. She isn't sure if this next trial will include a placebo group but all patients will be giving the drug at different times in the trial.
Funding for this current trial is being done by 2 of the current patients. MJFF hasn't given any $$ and according to the neurologist isn't planning to provide any funding. GTH is looking at grants etc for funding for the next trial.
This is the first time I've been excited or had any hope concerning this disease. I'll keep everyone informed about what happens in November when, I hope, I'm entered in the new trial.
All the best to everyone,
Betsy

Thank you

Here some links: http://www.foxnews.com/health/2013/0...ia-discovered/

http://www.georgetown.edu/news/cance...ons-study.html

http://www.nature.com/srep/2014/1405...srep04874.html

I've been following this closely for over a year and have discussed the science behind the hypothesis with both neurologists and oncologists. They all thought that this could be something that would work (although, of course, we've heard that before). I was somewhat disappointed when the FDA only approved the phase I trial for mid to late stage volunteers. Once again, this is a case where the scientists believe that the earlier the intervention with the drug, the more likely it would show efficacy. I was somewhat surprised that they would tell you interim trial information prior to a formal release of data.

I have heard of some off-label attempts with alzheimers patients that did not work. Nevertheless, I hope they are able to go to a phase 2 study so we can get more information. I'm surprised that they can't just get funding from the manufacturer, Novartis, as it would seem to be to their benefit if the drug is approved for other use outside of CML (and I think now AML also).

Nilotinib trial
 

I agree with you about your surprise of trial information being released. I simply asked how the trial was going and kept asking questions as the information was forth coming. I was most excited about what appears to be dopamine production in this advanced group. Also motor improvement was noted, walking in particular. I asked about tremor but she wasn't sure said not all the patients had tremor to begin with. Who knows what will happen the last three months of the trial.
I return in November when it's anticipated the second phase of this trial will begin with all stages of PWP. I've been hearing about this trial for about 16 months from the neurologists in anticipation that if I'm eligible I could enter the trial at the appropriate time. I'm hoping it's November. I'll keep everyone posted.

i personally would never repeat interim results from a clinical trial that weren't made public, imho this dr. should not have revealed them, things can change, the results haven't been peer reviewed, plus most pd phase 1 trials succeed and phase 2 don't once the placebo affect is removed and other clinicians besides the original investigator become involved with the larger phase2 trial. if the results were that fantastic i assume they would seek to get approval asap to expand the trial or start a new one. the fear i have is that pd'ers will start bugging their dr's for this already approved drug.

sorry if i got this wrong.

Assuming the trials for nilotinib go well and in a timely fashion, how long would it take to bring this drug to market. Are we looking at maybe 3 years or more like 5? How many more trials are necessary?

This trial, conceptually, is somewhat similar to the Isradipine Steady-PD trial. In that study the FDA required a 1 year phase 2 (which was successfully completed). The phase 3 commenced the end of 2014/ beginning of 2015 and is designed as a 3 year trial. If the FDA required a similar timeline, and taking into account time for patient recruitment, data analysis, NDA submittal and FDA review, you would have a minimum of 6 years. Of course, there is interim monitoring and if the results warranted it, the trial could be stopped earlier.

I wonder if the dose is similar to the one used for leukemia. Like the rest of the chemotherapy tribe it has some rough side effects at that dose.

It's not. The standard dose for chemo is either 300mg or 400mg 2X/day. The dosage for this trial was either 150mg or 300mg once per day. This is the reason there have been much less side effects than with chemo use.