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.

NEJM paper links 3 blinded patients to publicly-traded stem cell clinic

Do 3 blinded stem cell clinic patients with major or complete vision loss constitute a significant adverse outcome?

I would say so and a new paper details how this happened apparently at a particular publically-traded South Florida stem cell clinic business.

You can see the damaged retinas of one such patient below in an image from a new NEJM paper reporting the severe adverse outcomes. The red areas are hemorrhaging with other substantial damage to the retina as well.

How did this all happen?

stem cells eyes

Kuriyan, et al. 2017 NEJM Figure 2A

Last year the story began to break of multiple patients alleging they had been blinded by different businesses in South Florida. Dr. Thomas Albini presented on some information on this at the FDA meeting last fall, but things weren’t entirely clear. Back then there were also indications of lawsuits by patients related to alleged vision loss due to experimental stem cell offerings against various parties involved.

Now we have more details on some of the cases in this new NEJM article (Kuriyan, et al.) in which the authors attribute these patients’ experiences to a withdrawn “trial”, NCT02024269, which lists Bioheart (now known as US Stem Cell, Inc.) as the sponsor. I put “trial” in quotes because it was withdrawn and also because as best as I can tell this wasn’t a traditional FDA-approved trial of the kind normally based on pre-clinical data and an IND. US Stem Cell, Inc. is a publicly-traded company ($USRM) and its stock has been all over the place this year. I’m not aware of US Stem Cell having FDA approval for what it is doing.

The NEJM article oddly does not mention Bioheart or US Stem Cell, Inc. by name as the place where the patients were given the stem cells, but the authors do clearly link them together and other information further supports this connection. Continue reading

What’s the deal with US Stem Cell Inc stock?

The stem cell clinic business US Stem Cell Inc., formerly known as Bioheart, has seen its stock take a rollercoaster ride recently include a big run up and now today a big drop so what’s the scoop?

US Stem Cell, which includes a few subsidiaries such as US Stem Cell Clinic, focuses on the use of adipose stem cells to treat a variety of health conditions in people, as well as training and pet treatments.

US Stem Cell Inc Stock USRM

To my knowledge, the company does not have FDA approval such as an IND for the stem cell interventions that it sells. In addition, there is some debate over whether adipose stem cells/stromal vascular fraction is a biological drug. As a stem cell scientist and close follower of the field, I believe it is a drug. Note that I’m not aware of the FDA having taken any action on US Stem Cell or its competitors who use adipose stem cells. The company has also recently settled two patient lawsuits, which included allegations of harm to vision by a number of entities.

On the potential upside for investors, it seems there remains strong demand across the US and the world amongst patients for what stem cells clinics are offering even without FDA approval.

Why is the US Stem Cell Inc stock moving so much recently?

It looks like an investment research firm put out a report on the company and the report apparently details the company making deals outside the U.S. and in the Middle East so this may be part of what is driving the stock to move around.

There also seems to be something recent about a lawsuit settlement on social media, but seems pretty vague so hard to say if it is accurate or means something.

Any other thoughts?

Disclosure: I have no investment in stem cell/regenerative medicine stocks including US Stem Cell or its competitors.

Update on patients lawsuit against stem cell clinic, Stemgenex

StemGenexThe website Law360 has an interesting update on the proposed class action lawsuit against the San Diego stem cell clinic Stemgenex.  Note that it seems you can read the full Law360 article without a subscription if you open the site in Chrome as your web browser. See more background on Stemgenex and on this case here.

Not surprisingly, the plaintiffs and defense see this case in opposite ways as reflected in quotes in the Law360 article:

“Plaintiffs make non-specific and conclusory allegations with respect to all named defendants,” StemGenex said. “The second amended complaint is so devoid of any specific facts to support its contentions that it is impossible for defendants to reasonably prepare a defense.”

Brian Findley of Mulligan Banham & Findley, a lawyer for the plaintiffs, told Law360 Wednesday that the allegations are “quite specific” and cite false statistics, made-up online reviews and StemGenex employees. If customers told the company that the treatment hadn’t done anything, they were told it could take months to see an effect, or that they should buy another treatment, he said.”

A key issue in this case is the marketing of stem cell offerings from Stemgenex and the plaintiffs allege this marketing was problematic:

“The three StemGenex customers, Selena Moorer, Stephen Ginsberg and Alexandra Gardner, all say that they paid the company $14,900 for each stem cell treatments for lupus, diabetes and other ailments after being persuaded by the number of satisfied customers on the company’s website, but that the treatments had no effect.”

The Stemgenex website still lists an apparent 100% patient satisfaction marketing claim as of today, January 23, 2017 (see screenshot below).

stemgenex

Screenshot from Stemgenex website

According to the Law360 article, Stemgenex has made various arguments to support their motion for dismissal and they overall called the lawsuit a “fishing expedition.”

If you want to follow the case, here is some info:

“The case is Moorer v. StemGenex Medical Group Inc., et al., case number 3:16-cv-02816, in the U.S. District Court for the Southern District of California.”

It seems likely that more patient suits against stem cell clinics will emerge this year. Some, but not all of the other recent cases of this kind including against US Stem Cell, Inc. and its subsidiary US Stem Cell Clinic have been settled before any judgment was issued. I’m not sure of the status of a different proposed potential class action case against The Lung Institute. If you know of other such lawsuits please contact me or post a comment.

Dang: ad for stem cell clinic @Sciam article on our paper on stem cell clinics

What a strange Internet world we live in these days, huh?

Leigh Turner and I just published a piece in Cell Stem Cell on the state of the US stem cell clinic market finding 570 clinics and this paper has drawn a lot of media attention including a piece in Scientific American (Sciam).

Stem cell clinic advertisement

Screenshot from Scientific American article on paper on stem cell clinics that has a stem cell clinic ad at right. Note that that ad is not part of the article, but is an actual ad.

I’m glancing over that Sciam article just now and BOOM I see an ad for a stem cell clinic appear at the top and it is even one of the ones in the database we created for our article. The ad is focused on selling stem cells to treat autism in children, an area that raises a lot of questions.

How can it be a good thing to have an ad for a stem cell clinic right next to the article about the challenge of stem cell clinics in America? The way the web works these days, perhaps it isn’t so surprising that articles having anything to do with stem cells will often be accompanied by ads for stem cell clinics. This one just really stuck out to me today because of the context.