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.