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.

Tuesday, February 3, 2015

Sometimes A Message Just Misses The Boat


That was the case for me when I read a joint statement regarding the recent grass-roots interest in Genervon GM604.

Here's the statement:
http://www.alsa.org/news/archive/letter-to-the-als-community.html

And parts of it I read dozens of times, not believing what I was reading.

"Recent excitement about lithium in ALS and followed by no effect (or worsening) in subsequent studies underscores this point."
Lithium?  Are you sure you want to bring that one up?  Let's take a trip down memory lane. 
First, why would you choose the adjective "recent" for something that happened eight years ago?   
In 2007 there was a formal Italian study that made lithium sound like a miracle drug for ALS.  Lithium is readily available.  Every person with ALS I knew at the time had a chat with a physician and got a prescription for its off-label use.  Here's the really important part of all this.  A daughter of a  person with ALS put up a big shared spreadsheet online so that all trying lithium could track some basic data.  They did so.  Within weeks it was clear that this wasn't a wonder drug for ALS.  The excitement that a small, formal study had caused was put to rest very quickly by a broad base of grass-roots people who tried it and tracked their data.  
PatientsLikeMe stepped up to assist so that a better data repository than the spreadsheet could be used.  They published the patient-reported results far more quickly than the much more time-consuming and expensive lithium clinical trials that followed.  Here is an  article on the subject. 
Yesterday's statement mentioning lithium actually gives us a poster example for broad patient access to potential ALS therapies and patient reporting that can be much more definitive than small, slow, expensive trials.  I rest my case.
"...it is difficult to determine whether there would be any benefit to people with ALS if we were to repeat this in a larger study or the long term impact on quality of life."
Exactly!
"We do hear the voices that say, “This is exciting, let’s make sure this drug is available to all patients with ALS!”"
The voices don't stop with that sentence, though.  There have been numerous, thoughtful statements from those with ALS who want to advance the science through broader access to GM604.  It's not a selfish act of trying to survive.  It's a much more benevolent act of willingness to take risks with eyes wide open to advance the science more quickly.

Obviously you signers of yesterday's statement felt the heat of well over 100,000 signers on the change.org petition.  You clearly felt the heat of hundreds of postings and emails from people with ALS and their caregivers.  But were you even listening?

6 comments:

  1. Remember who funded a Phase II clinical trial for lithium, putting up around $2 million? ALS Association. Irony at hits best

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  2. And that was after the patient-supplied data had already told the story on lithium... and PALS asked (and rightly so), "Why are we wasting more money on this?"

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  3. What an awful statement from ALSA. If there is any PALS or CALS who feels that this association is helping us or feel identify in they way they think, i am willing to give a 1MM donation towards ALSA (nah, just kidding).

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  4. What an awful statements from ALSA. If there is any PALS who feels identify with ALSA or think they are helping us in any way i am willing to donate 1 MM to ALSA (nah, just kidding)

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  5. I was part of the group who put together the patient-led trial of lithium. First we did it because ALSA wasn't interested. Then we told ALSA to *not* waste money because our project clearly and scientifically showed no benefit.

    ALSA doesn't listen to anything but their own voices.

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    1. Wow, guess they forgot to include that part in their statement. I feel ashamed that the ALS Association who gather most donations worldwide after the IBC are those guys. I am from Argentina, and i know they are helping the local association to build an ALS Clinic, which is great, but they can just throw some money and not LISTEN to what the PALS community wants or desire; which in this case is to commit with the situation and to THINK HOW THEY CAN HELP US. Do they honestly believe this is the way? Sending those awful and inaccurate statements and then offering some money for a LONG Phase IIb trial? HMMM

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