thanks Kathleen
Don Reed's book Stem Cell Battles - Prop 71 and Beyond is supposed to be
about how folks can best battle for progress in disease research, book comes
out in December and I'm hoping for pointers on strategies for organizing.
lets start with Don's book
I think it should be noted that while a cancer drug may help PD, a PD drug
might help another disease or condition and there is no reason why a
leukemia patient should have to pay over $10,000 a month to stay alive.
Medical research and affordable meds are good and worthy causes not just
for Parkinson's.
And yes, Nic, it is very difficult for me to "type" - have tocorrecct
every other word, but I can barely speakk at all,,sooo computer is a
help
Lets organize!!!!
-----Original Message-----
From: Kathleen Cochran
Sent: Saturday, November 07, 2015 10:20 AM
To: [log in to unmask]
Subject: Re: Ray's questions #1, #2, #3
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
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