• "You never want a serious crisis to go to waste"

    - Rahm Emanuel (attribution)

Posted: February 27th, 2013

As callous and cold hearted as this sounds, it is a political truism. There are some systems, some bureaucracies that are so gridlocked, or so moribund that only the urgency that comes from dealing with a crisis can create enough momentum to make progress. The FDA held a hearing on Monday to allow the ALS community to voice their opinions on how to proceed with ALS research. While it is unclear exactly what precipitated the calling of this hearing, the failure of the Phase III Biogen “dex” trial to treat ALS patients has perhaps, finally created a “crisis” mode at the FDA. The audience was able to assure the FDA that they are already in crisis mode.

In emotional and chilling testimonies, patients and their families spoke of living with and dying from ALS.  Parents wept for their children, dead and alive.  Speaker after speaker, from patients to care givers to clinicians to researchers, echoed the same themes.  The FDA needs to redefine “risk” for this patient population; using their existing model is not “protecting” these people from anything, it is killing them.  The FDA needs to allow broader access to drugs for this population if these drugs can improve the quality of life for patients, regardless of whether or not it can impact survival.  And finally, the FDA needs to dramatically speed up the entire process; no one has a moment to wait.  And,   in what I confess was a moment of immense pride personally, Ted Harada (patient 15 of in ALS trial) stood up and excoriated the FDA for slowing down a trial and a therapy that held enormous potential for ALS patients.  “Just look at me” he said, in more than words.  In his testimony, Ted proposed that it is time that the FDA did the right thing and treat this process with the same urgency that the ALS community feels.  He spoke with passion, elegance and conviction.

But to me, the most chilling and important testimony came from our site principal investigator at Emory, Dr. Jonathan Glass.  Dr. Glass is a clinician with one of the largest ALS practices in the U.S. who has seen over a thousand ALS patients in his career.  He is also a world class scientist and researcher who knows first-hand what our experience with the FDA has been like in the Neuralstem trial.  In short, Jon speaks here with more experience and authority than anyone else in the field, in the world, literally.  Add to that, the fact that outside of our trial, Jon will have to go before the FDA many more times throughout his career and you can understand that criticizing them over “one” experience could have lasting reverberations.  But such is his conviction and dedication to his patients, and to science, that he felt compelled to speak truth to power.  We should all be thankful.

What Jon said was basically this; Neuralstem has completed 18 surgeries over an artificially long, but agreed upon timeframe to demonstrate that both our cells and the intraspinal surgery route of administration are safe.  We did just that beyond any question.  Ten experts at NIH have reviewed the data from the first trial, and our proposed Phase II protocol, and found it worthy of millions of dollars of NIH funding.  Yet the FDA, without the expertise or experience in the field that the NIH can harness, continues to place arbitrary hurdles in our way, and demand an inappropriately slow time frame for the dosing of patients in the next trial.  

The problem is easy to fix, Dr. Glass added.  The FDA merely needs to admit when they don’t have the expertise to deal with a subject, and borrow it from another government agency that does; in this case the NIH.  They should rely on the NIH scientific and medical vetting at a level the FDA doesn’t have the resources to produce.  This is no time for “turf” wars or false egos, people are dying.  Furthermore, all the FDA needs to do to speed up the process is talk to us.  As Jon said, we are constantly “guessing” where they will be on any particular issue, and waiting the statutory 30 days for responses to every question.    

In a recent presentation at the New York Stem Cell meeting, I said that the FDA was not the enemy.  They are not.  They are under resourced, and in our particular case, without experience or expertise.  However, more to the point, they are trying to work from a model which simply isn’t applicable to this disease.  At the pace they are currently suggesting we proceed, even if we are right and our therapy works; almost every ALS patient alive today will be dead before it is approved.  The ALS community deserves better.  We all deserve better.

Had I said these things first, it would have sounded like just another CEO whining about the FDA being “tough.”  We all owe Dr. Glass a debt of gratitude for not just his service to the ALS community, but his willingness to speak up.  None of these problems are insurmountable.  Let us hope that the FDA takes the opportunity that this crisis presents to change the way they approach our trial, and any other novel therapy that has the potential to help this population.  I will conclude as Jon started, reminding everyone that we are all on the same team here.

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