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.

FDA warp speed RMAT approval nukes stem cell clinic excuses

Many stem cell clinics are in a bind in 2017 in the new RMAT approval reality.

warp speed

Flickr image marked for re-use

Stem cell clinics say that they only want to help people by providing stem cell therapies to patients in a speedier way that they claim getting FDA approval for a trial just doesn’t allow, but that doesn’t ring true. It never has and now even less so.

The new RMAT designation out there based on the 21st Century Cures Act whereby the FDA can rapidly accelerate truly promising stem cell therapies in clinical trials makes this stem cell clinic excuse of the need for speed seem even less based on common sense. For more on RMATs see this background post. With RMAT, one eligibility criterion for that designation is that the illness in question must be serious or life threatening. The clinics have generally indicated in the past that what they treat are serious conditions. Will the FDA see it that way? It’ll depend on the conditions in each case.

The FDA has already approved 4 RMATs in just a few months so they are not holding back. If you really have good, solid clinical data for a serious or life-threatening condition, then you should apply to the FDA for an RMAT and in theory you can have both speed and FDA approval for a trial to move quickly. This new situation makes that general stem cell clinic marketing pitch of needing to get supposedly truly promising stem cells to patients more quickly than the FDA system allows ring very hollow.

Clinical speed with no real data is kind of like driving a race car with no brakes.

And if you are a patient, before you consider getting any stem cell treatment you have a new additional “must-ask” question for the clinics out there: “Have you applied for an RMAT from the FDA?” or “Do you have RMAT approvals?” Also, see my stem cell treatment guide for patients where I list 10 more questions to ask and things to think about. Asking your primary care doctor who is not involved in the stem cell treatment for their professional take in advance on your consideration of a stem cell treatment is a good idea.