From: Rayilyn Brown 
Sent: Sunday, July 17, 2016 11:18 PM
To: parkinsn 
Subject: Tasigna Info

To: 'Charbel Moussa' <[log in to unmask]>
Subject: Why doesn’t Novartis or the Michael J Fox Foundation Jump on this?????????
Sent: Tuesday, July 12, 2016 2:01 AM

Please explain why you are not drowning in cold hard cash!!!!!!!


From: Charbel Moussa [mailto:[log in to unmask]]
Sent: Monday, July 11, 2016 5:57 AM
Subject: Re: The struggle to get Tasigna (nilotinib) cleared for human triL

Thanks for your your kind words and I hope you will be able to combat this disease. Thank you for offering all the help you can, that is wonderful.

We are collecting data from whoever provides evidence and so far it is overwhelming positive suggesting a very encouraging effect of the drug. However, the drug is still experimental and we have to be very cautious so the planned clinical study is so critical. We look forward to a more positive response from funding bodies. The FDA have approved all our plan so far and the only thing remains some logistical obstacles and enough funding. But we will get it done and we hopefully will conquer these diseases.

Thanks for your support and we hope to be able to help.

Many thanks

Charbel Moussa MD, PhD
Assistant Professor of Neurology
Director- Laboratory for Dementia and Parkinsonism
Clinical Research Director- Translational Neurotherapeutics Program
Georgetown University Medical Center (GUMC)
4000 Reservoir RD. NW. Bldg D, room 203-C
Washington DC 20057
Office: 202-687-7328
Lab: 202-687- 5146

Email: [log in to unmask]

https://sites.google.com/a/georgetown.edu/moussa-lab/home


On Jul 10, 2016, at 8:50 AM, he wrote:

    Dr. Charbel Moussa,
    You truly are my hero. I know there is no magic bullet for the complexities of the human body but to possibly halt the disease progression and buy time for the next development is the next best thing. I am one of Dr. Pagan's patients, I am 51 years old and I have early onset Parkinson's. I had an in depth discussion with Dr. Pagan and he told me the difficulties with the FDA and astonishingly the Micheal J. Fox Foundation, some of the reactions from your peers around the world and even Novartis.  

    I have 27 years experience in clinical labs. If there is any way I can help your team bring hope and relief to millions please let me know. If you are in need of laboratory equipment I will talk to my company to see what they are willing to contribute. If you need a lab assistant to free up your team for more productive work,  or someone to do grunt work, be a gofer, a chauffeur, go get food, whatever, please let me know.

    I did have one more thought especially after my talk with Dr. Pagan, and all the absurd obstacles thrown in your team's way. Obviously you are bound by certain laws, regulations, and ethics which a more than willing Parkinson's or Alzheimer patient is not. Since you are not prescribing, encouraging or condoning the use of an unapproved drug is there anyway to capture the data you are looking for from thousands of suffers who are desperate enough to take matters into their own hands. Surly there are neurologists or researchers who would be willing to help collect this data within your and the FDA's strict specifications and guidelines or travel to Georgetown or a handful of satellite labs so everything is with in your control? I think filming our physical improvement, results from your cognitive testing along with measuring the decrease in certain proteins in the CSF or DAT scans of the brain would be irrefutable data to prove effectiveness quickly.

    Just thinking out loud. Thank you and your team again for outstanding work.  
    ------------------------------------------------------------------------------------------------------------------------------------------------
    A promising therapy that may slow or stop Parkinson’s progression is moving forward. Today The Michael J. Fox Foundation (MJFF), the Van Andel Research Institute (VARI) in Michigan and the Cure Parkinson’s Trust (CPT) in the United Kingdom announced plans to collaborate to assess the clinical use and development of cancer drug nilotinib. Among the partners’ goals: planning a double-blind, placebo-controlled clinical trial of nilotinib, which MJFF hopes can begin in 2017.

     
To:  Michael J Fox,
    Georgetown University is ready to start human trials now, so why is your foundation starting at square one? They are betting on the wrong horse. Your foundation and Georgetown need to kiss and make up. We all know it’s about money, but let’s put the afflicted first. I will also forward my communication with the scientist who made this discovery, Charbel Moussa, MD, PhD.

    I want to explain my situation so you understand my motivation. I just happen to be in the right place at the right time. My neurologist is Dr. Pagan and I have a medical background, and I love to do research on the internet so I am very well informed. After seeing Dr. Pagan, I was talking with one of his fellows, who are physicians that are specializing in movement disorders, and this physician told me that many patients don’t want to wait and they are getting the drug from Canada. Then this physician told me there are buying groups that band together to get the drug at an even cheaper cost. I am not waiting for the drug to be approved, I have ordered 112 pills at a cost of almost four thousand dollars. I don’t know why they sell it in this increment but I have three refills after this order. I have been informed I can get it at half that cost from Mexican pharmacies. I could also join a buying group but I wanted to get started on this drug asap so I went with the sure bet so I have a few months to figure out cheaper alternatives. So I am set, but I want to help anyone who is afflicted with Parkinson’s or Alzheimer’s who are willing to take the drug and can afford it. So far the cheapest I have found it is 120 pills, 150 mg, at $22 a pill from a brick and mortar pharmacy in Mexico. I was told the drug from this pharmacy is sealed in Novartis, the manufacturer of this drug, packaging, but I have to go there to get it. So I am going to take this drug, if it works I will get it for other people who also want it. For my own protection I can’t reveal how I do this.          

    I saw Dr. Pagan a couple of weeks ago and peppered him with questions about why it was taking so long to get the phase two study started. That is why I know this information. He explained the MJFF relationship and also explained the relationship with the FDA.

     Dr. Pagan said that members of the FDA that are assigned to Parkinson’s drugs are much more difficult to deal with than the members of the FDA assigned to Alzheimer’s drugs. They were getting nowhere with the FDA to start phase two so they gave their research data to the Alzheimer research group and got it approved for that disease with the FDA. Then they had a precedence to use in their fight to get it approved for human trials in Parkinson’s. Ironically the day that I saw Dr. Pagan he had a conference call with the FDA to find out if the FDA approved his study for human trials. The answer was yes; they are cleared to go.    

    This drug has been out for nine years treating CML (chronic myelogenous leukemia) at doses up to 800 mg daily. They noticed that Parkinson’s and Alzheimer patients with this leukemia and treated with nilotinib had significant improvement in their symptoms of their respective diseases.

    Georgetown conducted a small phase 1 trial with 12 patients, one dropped out early. They used two doses 150 mg and 300 mg for this safety study.   The study’s primary objective was to test safety. Researchers say that use of nilotinib, in doses much smaller than are used to treat cancer (which is up to 800 mg daily), was well tolerated with no serious side effects. I am just going to copy and paste some information:
    ------------------------------------------------------------------------------------------------------------------------------------------------------------------------------

TO:  Michael J. Fox  

Why is your foundation not giving money to Georgetown University Hospital Movement Disorders Research Program for funding the repurposing of a leukemia drug called Tasigna, generic: Nilotinib, that stops Parkinson's and Alzheimer's in its tracks and reverses the diseases by saving the "infected" cell by autophagy of the alpha-synuclein protein that kills the dopamine producing cells in the brain in Parkinson's and the Tau and Beta Amyloid proteins that cause Alzheimer's. Dr. Moussa has been working on this.

If that wasn't bad enough members of your research foundation went to Georgetown University Hospital and tried to strong-arm my neurologist Dr. Pagan and his research neurologist Dr. Charbel Moussa, Ph.D. (who should be presented the Nobel prize for Medicine) into giving them all the research data they had collected. When Dr. Pagan and Dr. Moussa refused, members of your foundation cut off all funding for the ground breaking discovery!!  Members of your foundation contacted Novartis, the manufacturer of this drug, and told them not to provide the drug to Georgetown University. Are you aware of this? 

I know that Dr. Moussa is listed as an inventor on a provisional patent application to use nilotinib and bosutinib as a therapeutic approach in neurodegenerative diseases, is that why your foundation is upset? Or is there an agreement to share all knowledge gained from research? Now Georgetown has to waste time raising money. Don’t we all deserve better? 

Did your foundation help with the long struggle to get it cleared for human trials by the FDA? Why didn’t your foundation help in the beginning, Georgetown has had to raise money from the public, and an NIH grant to get this far. They are on the precipice of changing Parkinson’s, Alzheimer’s, ALS (Lou Gehrig's Disease ) and Huntington’s treatment forever.

Now I see your foundation has partnered with Van Andel Research Institute and Cure Parkinson's Trust announced plans to collaborate on the clinical assessment of nilotinib as a potential treatment for PD. Georgetown had to raise money for first phase too. I implore you to consider funding Georgetown since they have FDA clearance for human trials, they are ready to start the trials now! How long will it take this new partnership to get up and running? I have no financial stake in this.  Could you also explain why Novartis is sitting on the sidelines??

I look forward to your response and acknowledgment of receipt of this letter. Thank for your time on this matter.














Ray
Rayilyn Brown
Past Director AZNPF
Arizona Chapter National Parkinson Foundation

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