US Stem Cell bid for FDA RMAT rejected?

Can a stem cell clinic business get FDA RMAT designation? At least one announced it was trying, but now seems to have given up.

Stem cell clinic business US Stem Cell, Inc. has reportedly announced that it is at least temporarily abandoning its efforts at getting Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. The company’s penny stock $USRM has been gyrating for months and I had earlier wondered if could be some fake news about it. The USRM news now seems real and not good on the RMAT front. Shares plummeted earlier this week (see earlier stock graph above).

US Stem Cell Inc.As to plans, here’s something from an apparent company PR:

“Until then, U.S. Stem Cell will focus on opening new clinics around the country to better serve patients in need. In addition to the original Sunrise clinic (that has successfully treated hundreds of patients and generated over $2m in revenues in the past 12 months alone), recent clinic openings include Miami and Palm Beach, Florida. Upcoming openings include Dallas, Texas (thanks in part to the early adoption of patient rights by the State of Texas), Chicago, Atlanta, and Denver – as well as other clinics in the northeast and the west coast.”

Opening more clinics…selling non-FDA approved offerings?

This may be what the company sees as a good business move, but in my opinion it puts more patients at potential risk and takes their money for “stem cell treatments” that are not conclusively proven to be safe or effective. I have not seen RCT data from USRM to support their commercial stem cell efforts.

What happened with the US Stem Cell, Inc. RMAT application? We may never know for sure, but rejection by the FDA is one possibility. Over at the RAPS site, a new piece on CBER has this to say (“Marks” refers to CBER Director Peter Marks):

“Thanks to the 21st Century Cures Act, FDA now has a new designation for regenerative medicines, known as the regenerative medicine advanced therapy (RMAT) designation. As of last week, Marks said there have been 19 requests for RMAT designations, 18 of which CBER has acted on, and four of which have been granted…’

Unless US Stem Cell, Inc. is the 1 out of the 19 applications on which the FDA has not acted, then it’s not looking promising for their RMAT just based on the odds. Another possibility is the FDA did not reject it, but asked US Stem Cell for a lot more data and that constitutes “acted on”.

It’s worth a reminder that this business was linked to the blinding of three patients in a presentation at a 2016 FDA meeting and in a NEJM publication. The company has had patient lawsuits too, which seem to have been settled out of court.

More broadly in the oversight arena, to my knowledge the FDA and CBER specifically have not issued any warning letters to or taken other actions (at least in the public domain) on stem cell clinics in ages despite hundreds of such businesses marketing unproven stem cell offerings without FDA approval. And whatever happened to those four key FDA draft guidances? Does CBER have enough funding and staff to tackle the burgeoning stem cell business arena? It remains unclear how the FDA and CBER will handle key challenges under the new Trump administration and with new FDA Commissioner Scott Gottlieb.

Good stem cell news on trials, FDA, cool new papers & more

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Guimarães-Camboa, et al, Cell Stem Cell figure

In the whirlwind that is the stem cell and regenerative medicine world, there are many concerning things that need attention, but also good stuff happens too and this post focuses on the positive.

The Asterias spinal cord injury clinical trial, a phase 1/2a trial called SCiStar, continues to make encouraging news with a clean safety profile and additional hints at possible positive indicators of efficacy. With the usual, important caveats such as that this is early and it is not an RCT, the SCiStar momentum is positive. I’m excited to see what the future holds for this one including from an RCT. You can read my interview from last month with Asterias leadership here.

I remain very enthusiastic about ViaCyte’s trial as well using a stem cell capsule product for treatment of Diabetes. Their joining forces with BetaLogics a year ago just made their position even stronger.

I’m going to do a post soon on an analysis on the total number of stem cell and regenerative medicine trials compared to historical data I collected. Stay tuned on that. I’m guessing it’ll be good news.

Recently, we also saw evidence of fast action from FDA in response to the 21st Century Cures Act in terms of providing a clear document on Regenerative Advanced Therapy designations and applications. It’s still unclear how the Cures stem cell provisions will play out, but I consider quick, clear action from the FDA to be a positive. I wish they were this fast on other stuff like dealing with stem cell clinics marketing unapproved drug products.

There have been a number of cool papers recently that I recommend reading:

Perspectives on Cures Act stem cell provisions possible benefits & risks

The 21st Century Cures Act has some important provisions that would impact the stem cell and regenerative medicine field.  What is driving these efforts is a widespread sense that the status quo of regenerative medicine oversight isn’t working very well.

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I’m talking with key stakeholders and still trying to sort out my thoughts on the stem cell provisions in Cures. What would it mean? Cures’ language on stem cells is nuanced and its provisions also are not so black and white as REGROW was in my view.

You can see my interview with ARM’s Executive Director Michael Werner on Cures here. ARM supports Cures. ISSCR issued a statement this morning on Cures, which is upbeat but stresses the importance of data. The statement is not a clear thumbs up or down. Earlier this year, both ARM and ISSCR opposed REGROW.

Overall, there are both potential risks and benefits to Cures’ stem cell provisions, which I discuss below. Note that this post is focused primarily on the stem cell-related provisions of Cures. There is ambiguity in terms of how Cures will affect NIH overall or have broader outcomes for the life sciences.

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Chat with Michael Werner of ARM on Cures Act regenerative medicine provisions

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Michael Werner, Executive Director of ARM

One of the hottest topics in the world of stem cells this year has been the REGROW ACT and at a larger level the movement for changes in FDA oversight of investigational regenerative medicine therapies (see my past posts on it here), so the related provisions in the 21st Century Cures Act are stirring much discussion today. The bill is called Cures for short and you can read the latest draft here.

Cures may get a vote in Congress as early as tomorrow so this is a big week for the stem cell field and the life science more generally in the U.S. See sections 3033-3036 of the bill as those are on regenerative medicine.

I’m reaching out to a number of stakeholders for their perspectives. Earlier today I interviewed Michael Werner (MW), the Executive Director of the Alliance for Regenerative Medicine (ARM) on Cures’ potential impact on the stem cell field and I have posted that interview below.

Note that ARM opposed REGROW, but supports Cures. I will post my own perspectives on the potential risks and benefits of Cures for the stem cell field and patients in a separate piece most likely later today.

What are the key reasons that ARM supports the Cures Act regenerative medicine provisions? 

MW: Four things come to mind: first, there has been a lot of talk about Japan and other countries that have taken steps to support this field and now with Cures, the United States does, too. It sends a clear signal to researchers and commercial developers that regenerative medicine is a critical technology that will be supported. Second, it optimizes the pathway without reducing FDA standards or authority. Third, it also provides for resources and flexibility in this area. Fourth, the inclusion of the standards section directing the FDA to work with stakeholders is very important. The lack of standards has been identified as an obstacle by all key stakeholders.

Will there be effects on IND/BLA requirements from Cures?

MW: I don’t think this legislation would change those specifically. The issue is to make sure that all stakeholders work together on standards development and that the FDA is part of the process. Cures does that.

What about Cures’ pre-approval (meaning the definition of an “advanced regenerative therapy”) versus post-approval requirements?

MW: I think that FDA will be comfortable with the scientific basis of whether something meets the definition of an advanced regenerative therapy. Also, the patient’s condition has to be serious or life-threatening. It was written pretty carefully. 

What does “real-world evidence” mean and is the FDA on board with that?

MW: It means using sources like patient registries and other data collected outside the formal clinical trial process. ARM had a series of conversations with FDA about the use of real world evidence and when it makes sense to include it as part of the review process. If Cures passes, the agency still has to approve the use of real world evidence in specific circumstances and it can say “yes” or “no.”

Do you think it’ll pass? Obama sign it?

MW: I think it’ll pass, and I believe if it does, that President Obama will sign it.