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.

Friday, January 16, 2015

It's Time To Think Out Loud

When I testified to a very stoic FDA panel at the ALS hearing two years ago, I envisioned thought-clouds above a lot of heads.  When people with ALS, their caregivers, drug developers, and organizations asked for faster approvals, I imagined FDA scientists facing us thinking, "Fine. Bring us something."

There was a lot that was unspoken in that room.

It's time to speak.  It's time to get a proposal on the table and work out the good, the bad, the ugly, the impossible, and, yes, the possible.  There is a willing drug developer.  There are people with ALS who are more than willing to accept risk beyond a normal clinical trial in order to advance the science and have a chance at a possible therapy.  There is an FDA that understands the urgency of ALS within the context of its serious responsibilities.

Let's think out loud this time, candidly.

The Genervon GM6 conversation has its roots (literally grassroots) on the forum at www.als.net .  To view the forum requires registration, but it is well worth it if you are interested in the science and challenges.

In addition here are some pertinent background links --

This blog's first post on the subject including a letter from Genervon.
This blog's second post on the subject about choices.
The change.org petition started by a gentleman with ALS with 19K signatures as of this morning.
An important blog from another gentleman with ALS who has taken GM6 and is working with Genervon.

Silence is easy.  It's time for some difficult conversations.  Out loud.


3 comments:

  1. Some smart people I respect are saying "Not so fast. We have very little data. We have made the mistake before in jumping on the bandwagon too soon."
    Other smart people I respect say " Its safe and its the best hope we've got right now, go for it."
    I believe we need some option between "Conditional Approval" and typical Phase 3. I'm not smart enough to know what that looks like. I do wish there was a better test case than GM6. There were just too few trial participants.

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  2. I suppose the "conditions" in the "conditional" approval could be tailored according to the situation.

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  3. I'm willing to participate with a phase 3 trayel.
    Berto Bisschop, Enschede, Netherlands.
    Bisschoo60@home.nl

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