OK, everyone, this is about much more than GM604.
Genervon and the FDA have been engaging in an interesting p.r. war, lobbing statements overhead while people with ALS and their caregivers are stuck in the trenches.
Genervon has been obvious (and, therefore, not very effective) in trying to make a very public appeal for FDA Accelerated Approval of GM604 for ALS.
The FDA has been very nuanced in making statements to cast aspersions on Genervon. I'm pretty sure this recent public statement cost the American taxpayer a small fortune http://www.fda.gov/Drugs/DrugSafety/ucm443242.htm
And the media know an interesting story when they see one.
So who is using whom here? I don't know. I don't really care.
We need to take a time-out from this p.r. circus and listen to the adults in the room -- the people with ALS and their caregivers.
This is about much more than Genervon. This is about a failed process for testing and approving therapies for ALS.
Fact: A number of years ago an FDA representative explained to some ALS advocates that the FDA can't just do things. They need to react to things that are brought to them, and, frankly, the FDA doesn't get much for ALS beyond a few traditional Phase 1,2,3 trials.
Fact: Genervon took them something different -- a request to use the Accelerated Approval path that was established for the FDA some years ago.
Fact: People with ALS and their caregivers have asked repeatedly for the chance to assume more risk in order to try investigational therapies and to advance the science more quickly. The FDA and our ALS organizations have nodded but delivered nothing to let that happen.
Fact: We don't know if GM604 will work, but its safety profile to-date does not put the fear-of-God in most people with ALS.
Fact: Traditional Phase 2 trials are not big enough to be definitive. They are not designed to look for efficacy, yet for a disease like ALS, every trial is being scrutinized for some sign of efficacy.
Fact: Traditional Phase 3 trials are dreadfully expensive; therefore, "promising" drug candidates sit on shelves while people with ALS die.
We have a failed process. It takes too damned long.
We have people with ALS and caregivers who are demanding that the word urgency finally be given some teeth in the fight against ALS. They are the ones who need to be heard in this conversation. They are smart. They are thoughtful. They are facing a fast and difficult path to death. They understand good science. They have eyes wide open about risk. They know p.r. volleys when they see them, too.
Genervon has pushed the boundaries of the same old same old with their request for Accelerated Approval. They have upset those who seem to believe that the existing Phase 1,2,3 trial model is the only way to do good science.
Listen to the adults in the room -- those with ALS and their caregivers. They are smarter than any of the p.r. wonks realize.
You will have a chance to listen to some of them in person http://www.meetup.com/ALS-Faster-Drug-Approval/events/221529700/ .
They are the ones acting like adults here. They need to be heard. And this is about much more than GM604.