• ”Promising is the very air o' th' time; it opens the eyes of expectation. Performance is ever duller…”

Posted February 23, 2012

”Promising is the very air o' th' time; it opens the eyes of expectation. Performance is ever duller…”
- William Shakespeare,The Life of Timon of Athens

Amid the clamor of this political season, the FDA’s budget renewal is being debated on the Hill. As part of this process, Senator Kay Hagan (D-NC) recently introduced legislation entitled the TREAT Act (Transforming the Regulatory Environment to Accelerate Access to Treatments). Its stated goal is to accelerate the FDA process for innovative drugs, particularly for currently incurable diseases. A quote from BIO, the country’s leading advocate for the biotech industry reads (in part) as follows:

“The TREAT Act will help break the institutional barriers to a faster idea-to-market pathway and speed new cures to patients most desperately in need. Through this bill, Senator Hagan has offered renewed hope to patients desperately in need of cures and breakthrough new medicines as well as a boost to our nation’s economy. The legislation will help the U.S. Food and Drug Administration (FDA) retain its leadership position as the global ‘gold standard’ for regulatory science and consumer protection.”

Wow… “promising” IS indeed the very air o th’ time. We will no doubt hear a great deal about this legislation and, as a company concentrating exclusively on fatal and/or incurable diseases and conditions, it is of great interest to Neuralstem. There is no question that major change is needed at the FDA to accelerate the development of innovative therapies. This legislation is an attempt to formally add this function to its mission, an attempt to force the agency to find a way to make both innovation and being the global “gold” standard compatible. The public does truly benefit from how “tough” the FDA is, but the public also benefits from access to potential breakthrough medicines. The two can no longer be seen as mutually exclusive. As BIO also stated:

“It is simply unacceptable that in the United States, as patients suffer and die, the time to take a new drug from discovery to patient is 10 to 15 years. More, regulatory uncertainty is having a major negative impact on the private funding of biomedical innovation, deteriorating the ability of companies to deliver cures and new medicines.”

So will this bill (if enacted) deliver on this promise?

First, Senator Hagan should be praised for trying. There are incredibly powerful entrenched interests involved here, and trying to change the entire culture of the FDA is no easy task. Add to that the hyper-adversarial nature of the current Congress and the very real budgetary restraints all agencies have to live within these days, and one can see how difficult this undertaking will be.

The bill creates all sorts of new boards and positions (including Chief Innovation Officer) inside the FDA to help facilitate the kind of change we all know is needed -- but no new money to accomplish anything, and no quantifiable goals or objectives. Creating this new accelerated pathway will be left to these new players; realistically, their first efforts are several years away (at best) from being enacted inside the agency.

Perhaps 20 years from now we will look back on this as a truly enabling piece of legislation that really did change the culture at the FDA and ushered in a new generation of innovative therapies. Only time will tell. And I think, in fact, that the grinding business of an agency, whose work is at the intersection of so much law and science and medicine and business, requires that basic change be enabled by this slow, unwieldy process. You really can’t turn an aircraft carrier on a dime. So what this bill is not is a quick fix.

To be fair, there are potentially important, specific changes championed by the legislation. Most notable among them is a new “standard” that the FDA may adopt to help speed along approval of new therapies for diseases such as ALS. The FDA is encouraged to consider surrogate markers of efficacy, which could “reasonably be expected” to provide an important benefit to patients, and to allow approval of such therapies in conjunction with further “post approval” testing and data collection by sponsors. For instance, if Neuralstem was to show that our cell therapy treatment can keep our patients’ breathing function strong enough to stay off of artificial breathing machines for a significant time, we would then be able to offer the therapy to many, rather than (theoretically) having to wait until the clinical trial patients die, to prove that they lived longer. If this were the case, it certainly would be progress; but it also certainly is NOT the only kind of accelerated and broader access to experimental medicines showing great promise that today’s generation of patients is looking for and needs.

It is too soon of course, to know how this legislation will fare as it moves through the inevitable maelstrom of the politics of budget battles in a Presidential election year. …Too soon to know if this is more “promise” than “performance, but it is a move in the right direction. I urge everyone reading this to do what they can to support this bill. In later blogs I will supply links that I think are helpful in this respect.

Still, it is not enough, not for the 30,000 ALS patients in the U.S., almost all of whom will most likely die before the benefits of this type of “big picture” approach can be realized. It is tremendously important for the future, but more needs to be done for today’s patients.

Neuralstem is a leader in developing innovative therapies to treat diseases like ALS; part of that leadership role must now go beyond the scientific and medical development of neural stem cell therapies. The company will start to work with groups inside and outside of the industry to help find a way to create broader and earlier access to promising therapies for ALS patients worldwide. In the coming weeks and months, I hope to outline these efforts and encourage your support for them.