ALS Advocacy

ALS Advocacy
Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Sunday, February 1, 2015

Please Take The Shot Before Time Runs Out!

I just submitted the following to the FDA (click here for link):

In October, 2012, I participated in your Patient-Focused Drug Development meeting.  A few months later I also attended your ALS hearing.  In both meetings a message we all heard over and over from those dealing with ALS was the meaning of "risk." It's clearly different for them.

My impression has been that the FDA isn't the stumbling block in our past failures to deliver an effective therapy for ALS.  We realize that you need for people to bring you proposals for ALS, and that just hasn't happened often enough.  We understand that Genervon is about to bring you a proposal.  I hope that you can work with them to find a path that will let people with ALS assume more risk so that the science and understanding of GM604 advance more quickly.  Please let those with ALS be a part of the solution with their eyes wide open that there are risks.  If you were to ask people with ALS to put all of their remaining chips on the table and pick between safety and attacking ALS, the chips would land on the latter every time.

I realize that you have much to consider.  I sincerely hope that you can work with a willing drug developer to come up with a plan that will give people living with ALS today a chance -- a chance to try and be part of a solution.  As you well know, people with ALS are faced with lousy choices every day.  We can't let the perfect get in the way of something that might be good.  We've done that for decades and it hasn't worked.   
Two years ago I was given six minutes to talk to the FDA about my thoughts on ALS.  I was driven to finish my words before Dr. Katz's light flashed on.  I made it.  Someone, somewhere died from ALS while I spoke.  People with ALS face a cruel, lethal clock every day.  We must find responsible ways to help them.  I hope that you can work with Genervon to do just that.  

Thank you.

Please submit your comments.  Time is quickly running out as Genervon is meeting with the FDA in early February.

It's like you've been passed the ball for a buzzer-beater three point shot for the win.  Organizations are saying, "We don't think you should shoot. Give us the ball."  I say, let it fly.



5 comments:

  1. Thank God we have you as our articulate, rational and personally dedicated advocate to seek solutions for us. We're forever in your debt.

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  2. Don't know about that, Grumpy... I just think that it would be a terrible waste not to take a cogent case to the FDA with what data and proposal Genervon has. We had an ALS FDA hearing and nothing has changed. Perhaps this will finally be a fulcrum of change.

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  3. My mother has ALS.



    She is on bed,she can not move with feet and hands,she can not speak and she is on ventilator because the breathing problems.
    Every day I have heartbreaking and dreadful feelings when I see her health condition. And her health condition get worser and worser.

    SHE HAS ALS 4 YEARS NOW AND I DONT KNOW HOW LONG SHE WILL BE ALIVE !!!!!!!
    SHE NEED TREATMENT AS FAST AS POSSIBLE-RIGHT NOW !!!!!!!!!!

    GENERVON´S GM604 IS HER ONLY HOPE FOR STAY ALIVE,AS WELL AS FOR HUNDREDS OF THOUSANDS PEOPLES LIVING WITH ALS IN THE WORLD.
    MY DEAR MOTHER AND HUNDREDS OF THOUSANDS PEOPLES LIVING WITH ALS IN THE WORLD CAN NOT WAIT YEARS OR MONTHS FOR EFFECTIVE TREATMENT BECAUSE THEY WILL BE DEAD.
    THEY NEED EFFECTIVE TREATMENT RIGHT NOW !!!!!!!!
    THE RISK USING TESTED,SAFE AND EFFECTIVE GM604 FOR PEOPLES WITH ALS IS NOTHING BECAUSE WHEN THEY DONT USE GM604 THEY WILL 100% DIE.

    I PLEASE FDA RESPONSIBLE PEOPLE FOR ACCELERATED
    APPROVAL OF GENERVON´S GM604 FOR USE IN ALS.PLEASE,PLEASE,PLEASE DONT KILL MY MOTHER AND HUNDREDS OF THOUSANDS PEOPLES WITH ALS IN THE WORLD AND GIVE THEM CHANCE TO LIVE WITH ACCELERATED
    APPROVAL OF GENERVON´S GM604 FOR USE IN ALS.

    ALL THE BEST.

    Matus Majernik-Loving Son on of Dear Mother living 4 years with ALS

    ReplyDelete
  4. My mother has ALS.



    She is on bed,she can not move with feet and hands,she can not speak and she is on ventilator because the breathing problems.
    Every day I have heartbreaking and dreadful feelings when I see her health condition. And her health condition get worser and worser.

    SHE HAS ALS 4 YEARS NOW AND I DONT KNOW HOW LONG SHE WILL BE ALIVE !!!!!!!
    SHE NEED TREATMENT AS FAST AS POSSIBLE-RIGHT NOW !!!!!!!!!!

    GENERVON´S GM604 IS HER ONLY HOPE FOR STAY ALIVE,AS WELL AS FOR HUNDREDS OF THOUSANDS PEOPLES LIVING WITH ALS IN THE WORLD.
    MY DEAR MOTHER AND HUNDREDS OF THOUSANDS PEOPLES LIVING WITH ALS IN THE WORLD CAN NOT WAIT YEARS OR MONTHS FOR EFFECTIVE TREATMENT BECAUSE THEY WILL BE DEAD.
    THEY NEED EFFECTIVE TREATMENT RIGHT NOW !!!!!!!!
    THE RISK USING TESTED,SAFE AND EFFECTIVE GM604 FOR PEOPLES WITH ALS IS NOTHING BECAUSE WHEN THEY DONT USE GM604 THEY WILL 100% DIE.

    I PLEASE FDA RESPONSIBLE PEOPLE FOR ACCELERATED
    APPROVAL OF GENERVON´S GM604 FOR USE IN ALS.PLEASE,PLEASE,PLEASE DONT KILL MY MOTHER AND HUNDREDS OF THOUSANDS PEOPLES WITH ALS IN THE WORLD AND GIVE THEM CHANCE TO LIVE WITH ACCELERATED
    APPROVAL OF GENERVON´S GM604 FOR USE IN ALS.

    ALL THE BEST.

    Matus Majernik-Loving Son on of Dear Mother living 4 years with ALS

    ReplyDelete
  5. We just need hope. The brutal reality of ALS is something PALS face whether they want to or not. Knowing that there is nothing to be done heightens the despair. Just give us a little hope that there are people working hard to help and that we can be contributors in the process of finding a cure. A life without hope is a desperate thing.

    ReplyDelete