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

Poll: How will Trump’s FDA handle stem cells?

TGIF: Week’s Stem Cell Headlines, Papers, & Resources

TGIF science dart board

Modified Wikipedia image

What are the latest developments in stem cells and regenerative medicine in this first week of 2017 and right before the end of the past year? Here are some of the headlines and papers that have popped up that seem notable or surprising. Be sure to also check out our stem cell & regenerative medicine meeting hub for 2017.

Alexey’s review of Cell Therapy (including stem cells/regen med) in 2016. Must-read. This links to his part 2 and there is a link to part 1 there. This is on his Cell Trials blog.

A Rapid Pipeline to Model Rare Neurodevelopmental Disorders with Simultaneous CRISPR/Cas9 Gene Editing. Cool system described in Stem Cells article.

Scientists find link between maternal environment and neurodevelopment disorders. More on neurodevelopment. Environment and genetics do a tango in development.

NYSCF and PGP announce availability of unique new stem cell resource for scientific research. More stem cell resources the better.

What happens to rejected papers? Neuroskeptic takes a look. A little off topic, but very interesting.

Stem Cell Research: How Scientists Grew Stomach Tissue In A Lab. Does it get hungry?

How Trump Could Slow Medical Progress. Worries about Trump & biomedical sciences.

Will President Trump restrict embryonic stem cell research funding. More worries on Trump. 

Genetically engineered immune cells melt away deadly brain tumors (from CIRM blog) New approaches to brain tumors are urgently needed. Minor thing — not sure about the use of “melt away” as a metaphor here.

Top 20 Stem Cell Predictions for 2017

stem cell crystal ball

Stem cell crystal ball

Each year I make a list of predictions for the stem cell and regenerative medicine field for the coming new year. Later in this post I list my top 20 stem cell predictions for 2017. In looking at my past predictions I realized this will now be my 7th year doing stem cell/regenerative medicine yearly predictions.

You can see below links to these predictions for past years, which sometimes seems rather far removed from today and in other cases strike me as strangely apropos of our times.

What will 2017 bring? Below are my top 20 predictions in no particular order except starting with a few hopeful visions for the coming year.

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