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