Ray asked: 1. Who is doing further trials? when and where? THE FOLLOWING TEXT IS EXCERPTED FROM A FORBES MAGAZINE ARTICLE BY EMILY MULLIN. I URGE EVERYONE TO READ THIS EXCERPT. IF YOU JUST WANT THE SHORT ANSWER TO RAY'S QUESTION #1, SCROLL TO THE END OF THE EXCERPT. THE ENTIRE ARTICLE (VERY WORTHWHILE) CAN BE FOUND AT: http://www.forbes.com/sites/emilymullin/2015/10/21/could-a-cancer-drug-reverse-parkinsons-disease/ NOW HERE'S THE EXCERPT: ...The study [of nilotibid, at Georgetown] represents the first time a therapy appears to reverse cognitive and motor decline in patients with Parkinson’s disease, according to the researchers. “But it is critical to conduct larger and more comprehensive studies before determining the drug’s true impact,” said study author Dr. Fernando Pagan, an associate professor of neurology at Georgetown University, in a statement. The trial builds on animal studies conducted by Georgetown University assistant professor of neurology Dr. Charbel Moussa. In a study previously published in *Human Molecular Genetics* <http://hmg.oxfordjournals.org/content/early/2013/05/09/hmg.ddt192.abstract>, Moussa found that nilotinib prevented the buildup of toxic proteins associated with Parkinson’s in the brains of mice. The leukemia drug Tasigna (nilotinib) improved cognition and motor functions in patients with Parkinson’s disease in a small clinical trial. Nilotinib is approved to treat leukemia, and it works by forcing cancer cells into autophagy – a biological process that leads to the death of tumor cells in cancer. But in Parkinson’s patients, low levels may be enough to turn on just enough autophagy, or cell degradation, in neurons that the cells would clear malfunctioning proteins and nothing else. In other words, the drug seems to work by switching on the garbage disposal machinery inside neurons to clear toxic proteins from the cell, Moussa speculates. The clinical trial included 12 volunteers with Parkinson’s disease or a similar condition called dementia with Lewy bodies, the second most common type of progressive dementia after Alzheimer’s disease. Participants received daily doses of nilotinib in 150 mg and 300 mg doses – much smaller than what’s used to treat cancer, which is up to 800 mg daily. While the study’s primary objective was to test safety, 10 patients demonstrated what drug developers call “meaningful clinical improvements.” Researchers also reported that the drug was well tolerated with no serious side effects. It appears that in smaller doses once a day, nilotinib turns on autophagy for about four to eight hours – long enough to clean out the cells without causing cell death. Then proteins that build up again will be cleared when the drug is given again the next day,' Moussa said in a statement. While the results seem wildly promising, they should be interpreted with caution; the researchers did not study a control group for comparison. Also, nilotinib was not compared with other medications used to treat Parkinson’s in the study. Even if the drug is found effective in further studies, the cost could be out of reach for many patients. For leukemia patients taking nilotinib, the drug costs about $10,360 a month. "The Georgetown researchers are now planning larger clinical trials with nilotinib for patients with Parkinson’s and other neurodegenerative diseases, including Alzheimer’s disease, which are slated to begin in 2016.... Ray asked: 2. What is opinion of neuros? My neuro referred to the statement by the National Parkinson Foundation that most of us probably have seen (see http://bit.ly/NPF-nilotinib), which he thinks is a reasonable position to take right now. He thinks that at this point, to prescribe nilotinib off label for PD would be "unwise." He speaks of the smallness of the study, the fact that it was designed to study safety only, and the lack of a placebo group. Core message: Wait and see. Anyone else have a response from their neuro? Ray asked: 3. How willing is PD community to fight for progress? and outrageous drug costs/note that drug developer Dr. Druker’s research paid for by Leukemia and Lymphoma Society, not Novartis, drug producer Obviously, each of us has to assess our priorities and gauge our limits as to how willing and able we are to take an active role. And getting organized is a challenge. But given the stakes and the promising nature of this treatment, it seems to me that if there ever were a time to push ourselves, it's now. If anyone has thoughts or suggestions on what this might look like and how we might proceed, please speak up. Let's see if, together, we can generate some traction. Based on my almost 20 years with this disease, the time to start fighting for this research and for affordable treatments is now...or, more likely, yesterday. Kathleen ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn