Wouldn’t you like to be a RAT too? Stem cell clinics apply for new FDA accelerated approval path

RAT RMAT designationAre stem cell clinics going to somehow get the FDA to approve their stem cell interventions? To get recognized as legit? It seems they are trying now more than ever.

Remember the REGROW Act that would have greatly reduced regulations on investigational stem cells and then how the 21st Century Cures Act passed with some REGROW-related provisions, but more moderate ones?

The status quo of how the FDA regulated stem cell-based regenerative medicine therapies wasn’t working so great in past years so some reforms were (and still are) needed, but the key was hitting the “Goldilocks” sweet spot of just the right amount of regulatory change.

I was worried about REGROW in particular because I viewed it as having overshot by a mile by going for too much reduction in oversight (particularly in its original language, although it moderated somewhat over time) and I had mixed feelings on Cures, but it was at least relatively more balanced. Cures passed, was signed by Obama, and is now law. One of its main provisions on stem cells is so-called Regenerative Advanced Therapy (RAT, an unfortunate acronym choice that it seems the FDA wants to change to RMAT) designation that could speed up vetting of stem cell therapies.

My concerns about the legislative efforts were mainly stemming from the possibility that stem cell clinics would try to take advantage of the less stringent regulations. Could they get their interventions to be designated as RATs? Could some of the clinics get FDA approval?

Now some of the clinics are trying to make that a reality.

I’m hearing from multiple sources that a number of stem cell businesses running clinics have already applied to the FDA for RAT/RMAT designation that puts therapies automatically on the accelerated pathway. Most of these businesses have not publicly announced their applications, but US Stem Cell, Inc., a publicly-traded stem cell clinic company disclosed that it has applied for RAT status. You might recall this businesses as it was associated with the blinding of three of its patients. I’m hearing that other businesses are already applying for RAT too. US Stem Cell wrote:

“Following the passing of the 21st Century Cures Act, U.S. Stem Cell, Inc. has applied to the FDA for RAT Designation. We have recently heard from the FDA, who has requested additional information regarding the MARVEL Phase II/III trial. We have provided all requested information to the FDA and are hopeful that the FDA will continue their expeditious review of our MyoCell product. Thanks to the REGROW component of the Cures Act, the FDA will grant RAT designation for a regenerative medicine therapy that is intended to treat, modify, reverse, or cure a serious or life-threatening disease and demonstrates preliminary clinical evidence that the product has the potential to address unmet medical needs for a disease. We believe that our MyoCell product meets these requirements, as we have demonstrated clinical efficacy in both preclinical and clinical studies…”

Presumably, some of those working on investigational stem cell therapies from non-clinics are going for RAT designation too and some of those are probably not going to get approved. On the other hand, some stem cell clinic and non-clinic applications to the FDA may get the RAT stamp of approval.

Overall, how will the FDA decide what to approve as a RAT and what to reject as not a RAT? How effective will the FDA be able to be on vetting the host of RAT applications it is getting?

Showing how fast this could all happen, the FDA has already granted the now-re-named RMAT designation to some companies including Humacyte, which is in part funded by CIRM and this RMAT designation seems like a positive move.

Hold on to your hat (or chair, or pet rat, or whatever) as we will now all witness a high stakes regulatory experiment unfold in front of us in the coming months and years due to Cures. The outcome is likely to be mixed overall, but we can hope it’ll be on the whole a positive for the field and for patients. There’s no crystal ball on this though.

Splashy SacBee Ad for Stem Cell Clinic Nervana Raises More Questions

This morning my local paper The Sacramento Bee ran a full-page, super splashy ad for the local stem cell clinic, Nervana.

Remarkably the ad says, “As Seen in The Sacramento Bee”, referring to an article by reporter Claudia Buck that ran in the past in the SacBee Insight section that raised many concerns and questions about Nervana. I may have missed it, but it seems like it has been months since the last Nervana ad in the Bee.

What is going on here? It seems like there are few answers still and more questions now.

Nervana SacBee ad

I’ve written before about how Nervana has had a number of big full-page ads in the Bee. Nervana markets non-FDA approved stem cell offerings for neuropathy and other conditions for which in my opinion as a stem cell scientist there is no solid scientific basis for safety or efficacy.

This new ad takes the cake in this series of ads from the company as it has “Stem Cells” in huge blue font across the top and a very large “Neuropathy” below that in red font. In addition to that dash of splash and the odd mention of the clinic having been discussed in the Sac Bee before, importantly the ad makes medical claims that I see as unproven.

For instance, to my knowledge there is no concrete evidence such as published peer-reviewed articles backing up the ad’s claim of essentially a cure by “getting rid” of neuropathy symptoms. It does qualify that I guess with “may be possible”, but I’m not sure that conditional will sink in for patients. It also makes other medical claims that the treatment is “safe and effective”. Is it objectively known for sure to be those things? Could they have rigorous unpublished data that they’ve run by the FDA that serves as a foundation for what they are doing medically?

Finally the ad has a coupon and says, “over 1,000 patients already served”. If indeed they’ve already done experimental stem cell transplants into that many patients at a cost of say roughly $5,000 each then that is $5 million in money taken from patients. Is that serving the community? Is the SacBee an enabler of guiding patients toward potentially risky, unproven, and expensive medical offerings? Is the FDA still basically sitting out regulating stem cell clinics? What about state regulators in California? Who knows?

I’m going to reach out to Nervana itself again too to try to get more clarity on this situation and their perspectives. The company website still lists Dr. Tushar Goradia as the leader so I’ll try to reach him.

Did blogger DrugMonkey drop the mic?

One of the blogs I’ve really valued over the years was written by a pseudonymous academic blogger called DrugMonkey, but for two months the Monkey’s blog has been silent.

Has he called it a day? Dropped the mic after successfully having big impact?

What’s the deal?

DrugMonkey

After many years could it be that the DrugMonkey decided to move on to focus on other things? He provided valuable, no-B.S. perspectives on science and in particular on NIH funding. There was also the occasional post on the science of drugs that gave that blog and blogger his name.

One of the commenter’s on DrugMonkey’s last post, many of whom have been lamenting the possibility of the end of that blog, noted that he is still very active on Twitter.

Maybe he’ll be back to the blog eventually?

DrugMonkey’s disappearance from his blog has made me think more about my own blog. I’ve been at this blog more than 7 years and on the web with various websites for about a decade. You can read more about my web history here. If DrugMonkey is done or even just taking a blog sabbatical, I’m curious what was the deciding factor.

I still find the educational outreach on this blog to be a meaningful, positive thing to do despite being crazy busy overall. While there are many potential or even concrete risks, especially for me blogging as myself by name about often controversial subjects including reporting on stem cell clinics that have at times even threatened me, I continue to feel strongly about keeping this effort going.

On Duke stem cells for autism trial, did new CNN article hit the mark?

Dawson KurtzbergWhat’s the scoop on stem cells for autism?

Could stem cells make a real difference for autism patients and their families?

CNN has a big, high-profile new article out today on the Duke stem cell trial for autism that has been ongoing for a while and I’m wondering how good a job CNN did on this one. Too often media over-hype stem cells and this includes for autism.

I’ve covered the Duke trial before here and here, which is run by Dr. Joanne Kurtzberg, a scholar in the translational stem cell field, and Geraldine Dawson, an autism expert (they are pictured above).

The CNN article has some balance and includes cautionary notes about not jumping to conclusions so those are positive factors in conveying the appropriate message, but on the other hand the authors, Wayne Drash and Dr. Sanjay Gupta, focus so heavily on a particular patient and family portrayed as a success story that that tips the balance to a bit of a net negative I think in terms of giving readers the wrong impression. But the included cautionary notes from Arnold Kriegstein, head of the UCSF Stem Cell Program, are helpful.

There are just so many unknowns about autism and about how stem cells might help it in terms of mechanisms. The study in question is not controlled and is open-label too, factors that complicate interpretations.

Overall, it’s a close call and this article is better than most media pieces on translation of stem cells to the clinic, but I don’t think the CNN authors were quite cautious enough about stem cells for autism in this piece.

What do you think?

SCOTS, Selling Stem Cells, & More Patient Claims of Blindness

Have more patients been blinded by stem cell clinics?

The recent NEJM paper reporting on the blinding of three patients in Florida may be just the beginning of information beginning to flow on negative outcomes for patients who are customers of stem cell clinics selling non-FDA approved offerings. The NEJM authors linked the loss of vision to interventions received by patients from the publicly-traded company US Stem Cell, Inc., but different patients also in Florida have been alleging that they were blinded by a different entity, the “SCOTS trial”.

Steven Levy Jeffrey Weiss

Drs. Steven Levy & Jeffrey Weiss, leaders of the SCOTS trial.

I’ve blogged about SCOTS several times before including the patient allegations of being blinded and various other concerns. Now there’s a new BBC investigation on these allegations reported in a striking radio broadcast.

Two physicians are central to the SCOTS trial, Drs. Steven Levy and Jeffrey Weiss. A number of patients have alleged negative experiences including patient George Gibson, who is one focus of the BBC report. But by contrast another patient named Doug Oliver has said that he had very good results from SCOTS. How do we in the broader stem cell community try to understand the SCOTS situation? It’s difficult right now, but can we learn anything from the BBC investigation? Continue reading