How Scott Gottlieb may transform the FDA’s approach to stem cells

The Trump FDA commissioner nominee Dr. Scott Gottlieb Dr. Scott Gottliebcould dramatically alter how the agency regulates investigational stem cell therapies. How might such changes unfold? There are potential upsides and downsides to  the seismic shift that could be in the offing.

Gottlieb has written in the past about his perception of FDA over-regulation of stem cells such as in this piece in the WSJ. There he made a number of assertions that signal his view at least back then that when it comes to regulation of stem cells by the FDA, less is more.

More recently, a speech he gave at an ISSCR meeting in Berkeley last year is much more balanced in tone than the 2012 WSJ piece. This quote from the Berkeley talk, for example, is balanced and emphasizes standards:

“Expediting the development of these novel and transformative technologies like gene- and cell-based therapies doesn’t necessarily mean lowering the standard for approval, as I believe other countries have done. But it does mean having a framework that’s crafted to deal with the unique hypothetical risks that these products pose.”

Still that WSJ article is concerning.

For instance, he and his co-author Coleen Klasmeier, both former employees of the FDA, strongly criticized a federal court ruling in 2012 that the FDA could regulate laboratory-proliferated stem cells as a biological drug. Oddly, much of the basis for their criticism was far broader than the reality of the court ruling. They suggested, for instance, that the ruling opened a veritable Pandora’s box of FDA over-reach potential that extended to all autologous uses of stem cells (lab-grown or not) and beyond, but that was not the case as the ruling was focused on lab-grown stem cells. Even so they made largely unsupported generalizations such as the following:

“If the FDA’s victory is upheld on appeal, then conceivably nothing done as part of clinical practice is beyond the agency’s reach.”

In the intervening years since the 2012 court ruling and the WSJ article, we have seen that the sky hasn’t fallen from the FDA having obtained the defined authority to regulate lab-expanded stem cells as drugs. In fact, oddly enough if anything the more time that has passed, the less inclined FDA’s CBER biologics branch that oversees stem cells has been to take action on stem cell biologics. It’s hard to view the FDA issuing less than one warning letter per year to stem cell clinics even as there are upwards of 600 of these clinics as a form of overreach.

From a scientific and medical perspective, there are good reasons to regulate lab-grown stem cells as drugs. The cells are known to change their differentiation properties, accumulate mutations and epigenetic changes, and undergo other significant alterations in the dish in the lab. There is also, perhaps more directly related to the FDA’s mandate on biologics, substantial potential for contamination of cells in a lab during proliferation.

Since 2012, another big change has been the explosion of adipose stem cell clinics onto the scene, which use more than minimally manipulated liposuctioned material to make a biological drug product via enzymatic and other steps. However, the clinics argue their products aren’t drugs and they generally do not have any FDA approval to market stem cells. Nonetheless such offerings are being sold at hundreds of clinics around the country. How would a newly minted FDA commish Gottlieb view these clinics? Through the same lens as the autologous, homologous bone marrow-based approaches? What about the clinics using bone marrow and amniotic cells in non-homologous manners that make such products drugs? Continue reading

Open letter to FDA: take action now on stem cell clinics before Trump

Dear FDA,

The stem cell clinic clock is ticking on you.

Before the Trump administration rolls in to possibly tie your hands on many important areas of oversight including stem cell clinics, you should take bold action now.

Your CBER branch has been preternaturally quiet on taking actual regulatory actions on stem cell clinics for several years now even though there about 600 such clinics in the U.S. operating without any FDA approvals putting thousands of patients and the stem cell field at risk. Everyone knows that you are now aware of these clinics. One warning letter in a period of years is a drop in the bucket.

For these same past few years you have issued draft guidances that if implemented would substantially change how you regulate stem cells in ways that would quite helpfully put a stop to the mushrooming stem cell clinic industry.

You also held two public meetings on stem cells in 2016, which was historic, and you received both verbal and written comments from stakeholders. The REGROW Act is history and the Cures Act is now law, with important language reinforcing your role in stem cell oversight. The clinics do not by any stretch of the imagination meet the hurdles specified in Cures.

In short, much uncertainty is over. And you have done your due diligence. The time is right to tackle the dangerous stem cell clinic problem. You have sent clear signals that you feel strongly about proper stem cell oversight including via a late November opinion piece in the NEJM only a few weeks ago. But words are not enough.

Issue a large coordinated series of warning letters in the next couple weeks to the scores of clinics grossly violating your regulations by experimenting on thousands of patients for profit with unapproved drugs. Now is the time.

After Trump’s inauguration all bets are off as to whether the new administration’s FDA could still do anything about this serious problem. Strike now before his inauguration and make a profound positive difference.

Best regards,

Paul Knoepfler

Trump Has FDA in Crosshairs: Impact on Stem Cell Oversight?

fda-trump-healthcareWill Trump trump the FDA?

Our President-elect recently released his health care policy agenda and it looks like the FDA is a target in terms of reducing oversight. Over at the Washington Post, Carolyn Johnson has a nice article on this development.

One point in the agenda in particular has caught people’s attention as potentially the key indicator of what is to come for regulatory oversight of biomedical products:

  • “Reform the Food and Drug Administration, to put greater focus on the need of patients for new and innovative medical products”

This one sentence says a great deal in terms of signaling that Trump wants dramatically less oversight or at least that’s how “reform” is being interpreted.

For those of us in the stem cell field this sentence from Trump sounds very reminiscent of language from proponents of reduced regulation and even stem cell clinics, who say they are just in it for the patients but keep keeping racking up profits.

This signal from Trump does not bode well for stem cell and regenerative medicine oversight in the coming years, but we’ll have to stay tuned to see how this develops.