The FDA in the past year or so has issued draft guidances on regulation of stem cells that would, if finalized, make it crystal clear that what many American stem cell clinics are selling are unapproved drugs.
That would be a good thing for patients who are often being put at risk by many of the clinics. This is the reality no matter what some of the patient advocates who promote the clinics might say. Such finalized FDA regs that unambiguously state that, for example, fat stem cells are drugs, would be bad news for the clinics that are after patients’ money. Update: It is important to note that not all patients or patient advocacy groups are pro-clinic and anti-FDA. Every patient has a different story and I’ve met many that know the clinics aren’t some kind of Robin Hoods of stem cells.
It is perhaps not surprising then that at the planned upcoming public FDA meeting on stem cell regs in April, there’s going to be a crowd of anti-FDA, anti-regulation forces converging to speak. The agenda of this meeting, with a few notable exceptions, reads like a who’s who of the for-profit stem cell clinic world. I’ve pasted the agenda at the bottom of this post.
An interesting additional factor going on here is that there are some highly respected organizations also coming to the meeting that, reading the tea leaves, are also almost certainly going to advocate that the FDA be less comprehensive in its regulation of stem cells to accelerate translation. For instance, CIRM recently has made some statements about the FDA needing to speed up and maybe not be as strict.
CIRM probably realizes that there will be many folks at this FDA meeting who have no particular interest in participating in the standard FDA approval process at all. These stem cell clinic folks may cheer on statements from CIRM, if such are made, that come off as wanting a weaker FDA regulatory sphere for stem cells. I cannot imagine that CIRM would want that and I know that CIRM does not support these clinics.
As a result, there’s a vital need for CIRM to choose its approach to this meeting very carefully and cautiously. It may be a fine tightrope to walk. Accelerating evidence-based treatments based on stem cells is one thing, but weakening much-needed regulatory oversight that leads in the near future to clinics taking more money from more patients that they put at risk is quite another.
In short, CIRM’s agenda is very different than that of the clinics. Yet the stem cell clinic crowd could even be hoping to use CIRM’s words at this meeting for their own future publicity. They might even try to get their picture taken with CIRM staff to use on their websites. Historically the stem cell clinics have been very anti-CIRM, but that doesn’t mean that they won’t pounce on a PR opportunity if it arises.
The Alliance for Regenerative Medicine (ARM), which has done and continues to do amazing things, is also likely to push for a relatively more permissive stem cell regulatory construct at this meeting too. I hope that they are cautious not to align themselves with the dubious clinics. The membership of ARM consists of good citizens of the stem cell and regenerative medicine biotech community who work with the FDA and follow regs. This is a night and day difference from the stem cell clinics.
Regardless of how this meeting plays out, it will be fascinating to see and hear the views at this unprecedented gathering.
The agenda as of Feb. 10.
Draft Guidances Relating to the Regulation of Human Cells, Tissues or Cellular or Tissue-Based Products Part 15 Public Hearing Draft Agenda April 13, 2016 FDA White Oak Campus 10903 New Hampshire Ave, Building 31, Room 1503, Great Room Silver Spring, Maryland 20903
8:00-8:15 AM: Presiding Officer Opening Remarks and Introduction of Panel Members 8:15-9:46: Session 1 • 8:15-8:18: Allosource • 8:19-8:22: Atlanta Medical Center • 8:23-8:26: AZ Pain Centers • 8:27-8:30: Birth Tissue Recovery, LLC • 8:31-8:34: California Stem Cell Treatment Center and Cell Surgical Network • 8:35-8:38: CAREStream America • 8:39-8:42: Celebration Stem Cell Center • 8:43-8:46: Centeno-Schultz Clinic • 8:47-8:50: Harvard Apparatus Regenerative Technology • 8:51-8:54: INCELL Corporation LLC • 8:55-8:58: Intellicell BioSciences • 8:59-9:02: Johnson & Johnson • 9:03-9:06: LifeLink Tissue Bank • 9:07-9:10: LifeNet Health • 9:11-9:14: Long Island Plastic Surgical Group • 9:15-9:18: MedCentris • 9:19-9:22: Info Health Global • 9:23-9:26: Millennium Medical Technologies • 9:27-9:30: MiMedx Group, Inc. • 9:31-9:34: Minnesota Medical & Rehabilitative Services • 9:35-9:38: Musculoskeletal Transplant Foundation • 9:39-9:42: National Spine & Pain Centers • 9:43-9:46: Oregon Regenerative Medicine 9:47-9:55: Questions from panel 9:55-10:25 Break 10:25-11:56: Session 2 • 10:25-10:28: Organogenesis Inc. • 10:29-10:32: RTI Surgical • 10:33-10:36: StemGenex, Inc. • 10:37-10:40: U.S. Stem Cell Inc. (FKA BIOHEART Inc.) • 10:41-10:44: Robert W. Alexander, M.D. • 10:45-10:48: Scott R. Graham, M.D • 10:49-10:52: Shay Bess, M.D. • 10:53-10:56: Julie Cerrone • 10:57-11:00: Ann A. Connolly • 11:01-11:04: Shawn Connolly • 11:05-11:08: Elio Coradin • 11:09-11:12: Terri Coutee • 11:13-11:16: Charles S. Cox, Jr., M.D. • 11:17-11:20: Pamela Fricke • 11:21-11:24: Brian Gates • 11:25-11:28: Jarvis P. Green • 11:29-11:32: Candace Hart • 11:33-11:36: David Jacobs, M.D. • 11:37-11:40: Carolyn M. Salafia, M.D. • 11:41-11:44: George U. Sauter • 11:45-11:48: John Klimkiewicz, M.D. • 11:49-11:52: Samantha Wilkinson • 11:53-11:56: Rebecca Baergen, M.D. 11:56-12:00 Questions from panel 12:00-1:00: Lunch: Ala Carte items will be available for purchase on site 1:00-1:35: Session 3 • 1:00-1:03: MaryAnn Chirba, J.D. • 1:04-1:07: Case Western Reserve University • 1:08-1:11: Indiana University School of Medicine • 1:12-1:15: Wake Forest University School of Medicine • 1:16-1:19: Navigant Consulting • 1:20-1:23: OrthoKinetic Technologies, LLC • 1:24-1:27: Parenteau BioConsultants • 1:28-1:31: Alston & Bird LLP • 1:32-1:35: Topher Stephenson, M.D. 1:36-2:52: Session 4 • 1:36-1:42: Alliance for Regenerative Medicine • 1:43-1:49: Alliance of Wound Care Stakeholders • 1:50-1:56: American Association of Tissue Banks • 1:57-2:03: Bipartisan Policy Center • 2:04-2:10: California Institute for Regenerative Medicine • 2:11-2:17: Coalition of Wound Care Manufacturers • 2:18-2:24: Curemonos • 2:25-2:31: Foundation for the Accreditation of Cellular Therapy • 2:32-2:38: International Society for Cellular Therapy • 2:39-2:45: National Center for Health Research • 2:46-2:52: Patients for Stem Cells 2:52-3:00: Questions from panel 3:00-3:30: Break 3:30-4:53: Session 5 • 3:30-3:36: The Amputee Coalition • 3:37-3:43: The Cord Blood Association • 3:44-3:50: The Cure Alliance • 3:51-3:57: Academy of Regenerative Practices • 3:58-4:04: Alliance for the Advancement of Cellular Therapies • 4:05-4:11: American Association of Neurological Surgeons and Congress of Neurological Surgeons • 4:12-4:18: American College of Surgeons • 4:19-4:25: American Society of Plastic Surgeons • 4:26-4:32: Biologic Orthopedic Society • 4:33-4:39: International Cellular Medicine Society • 4:40-4:46: International Federation for Adipose Therapeutics and Science • 4:47-4:53: The Plastic Surgery Foundation 4:53-5:00: Questions from panel 5:00-Close: Closing statement from Presiding Officer
Wondering Paul if you are familiar with the report of the Bipartisan Policy Center that provides some thoughtful proposals on moving the regulatory needle safely?
Hi Bernie,
Thanks for bringing this important document back into the discussion.
I have only read it once and have been wanting to read it again more recently and write a response.
I agree with the authors that the FDA regulatory approach to cellular therapies needs some reforms, but we diverge on a number of the specifics. Also the report seem overly optimistic to me, lacking a discussion of some sizable risks of the proposed reduced FDA regulation. For instance, what would be the reaction of the mushrooming for-profit stem cell clinic industry in the US to reduced regulation with their hodgepodge of methods, devices, and unapproved products?
Still I’m hopeful that collectively we all can chart a constructive path forward. Stay tuned for a post with more details on my reaction to the report.
Paul
@ Jenninger Ziegler
“We’ve NEVER promoted “clinics.” I’m surprised after interacting with you over the past 4 years, that’s what you think we do!
“What PFSC does promote is the right to share information so a patient can make an educated decision with family/doctors on where to seek adult stem cell therapy…”
You say tomato…
SOme for-profit stem cell clinic world make money…. And you John are defending multi billon industry to play with our health and own stem cell. There are lot of Doctors doing ASC treatment with VERY GOOD RESULTS… my friend after spine cord lesion, now is walking again… one day procedure using adipose derived stem cells everything done in one day….
Anyone interested in attending the April 13 hearing must obtain the FDA’s permission to do so, and the request period closed in early January. See below for info on submitting written comments to the FDA – the deadline is April 29, 2016.
Also, the speakers listed (thank you Paul!) for the April 13 hearing will address a host of concerns, many of which have little if anything to do with the potential risks and benefits of accelerating approval of emerging and unproven HCTP therapies. Many speakers are not anti-FDA. To the contrary, they simply want the FDA to do what it is supposed to do and are concerned that these drafts contravene that purpose. The 4 draft guidances (re: minimal manipulation, homologous use, adipose tissue and same surgical procedure exception to § 351) individually and collectively employ key terms that, as a matter of basic biology, are inaccurate or at least incomplete. These foundational problems risk casting even well-established treatments onto a regulatory pathway that does not fit the product or treatment being regulated. If lack-of-fit renders a pathway incapable of performing the basic risk benefit-calculus necessary to protect patients, then patients can lose access to existing safe and effective treatments. So, more than acceleration is on the table; regulatory regression is now a problem, too.
This lack of regulatory fit is akin to insisting that all plumbing problems be referred to the fire department. There is definitely a problem with the plumbing and no one denies that. There is definitely a need for an appropriate response, too, and at least most people would agree. The fire department is excellent at responding appropriately to certain problems, but it cannot – or at least should not – handle plumbing problems of any magnitude. Conversely, however excellent a plumber might be, he or she should not be the one evaluating whether my smoke and carbon monoxide detectors are safe and effective. There is an obvious lack of fit between the problem and the response. Who would ever accept this? But that is what these draft guidances risk doing at least for certain situations. Thus, the FDA’s April 13 hearing is about much more than reasons to accelerate or de-regulate; it is about making sure that the regulations do what they are supposed to do – and therefore do what all patients need and deserve them to do.
One example is the impact of the adipose and homologous use draft guidance documents on a woman’s longstanding options for breast reconstruction following mastectomy or radiation for breast cancer. Until these draft guidances, standard flap procedures were not on anyone’s radar when debating the pros and cons of how to regulate emerging HCTP therapies. Yet, they face serious jeopardy should the draft guidances be finalized in their current form. Why? According to the FDA, “the basic function” of the breast is lactation. Various flap procedures involve some degree of fat transfer because adipose is an integral part of the overall tissue being transferred. The FDA insists that to qualify as homologous use, the defect being repaired must be a subcutaneous ADIPOSE tissue defect, as opposed to replacing or supplementing a loss of breast tissue. Because fat does not restore lactation, using it to reconstruct the breast is non-homologous and thus not permitted. Put simply, a woman’s own fat can be used to fill the hollows of her cheeks, but it cannot be used to reconstruct her own breast because fat does not restore lactation. Rebuilding a secondary sex organ and restoring overall bodily integrity apparently do not matter even though federal law requires insurance coverage for breast reconstruction post-mastectomy.
I raise this as just one example of what is on the line here. This is not just a discussion about fringe groups doing fringe therapies. In no way do I minimize the importance of those concerns. Indeed, the FDA may have been targeting that category of dilemmas but in the process, created a much larger compendium of worries. This is what will be on my mind on April 13. Like every other speaker, I have 3 minutes to present. Thus, for speakers and non-speakers alike, submitting written comments is important. Here are instructions:
To see where various groups stand on various issues, review their comments on the adipose, homologous, minimal manipulation and same surgical procedure HCT/P Draft Guidance documents. Go to:
http://www.regulations.gov/#!searchResults;rpp=25;po=0;s=HCT%255C0P%252Badipose;fp=true;ns=true
Clicking on “Open Docket Folder” will direct you to comments from clinicians, researchers, professional associations, patient groups, etc. Comments may be submitted by clicking on “Comment Now!” The FDA’s public hearing is on April 13, but the written comment period extends to April 29, 2016.
Thank you again, Paul.
Hi Mary Ann,
Thanks for the lengthy, thoughtful comment. I also encourage everyone to submit their written comments to the FDA so they get diverse, public and expert comments.
I don’t really buy into the plumbing/fire department analogy. The FDA is not an emergency response unit if I understood that correctly. Much of what they do is day-to-day basic evaluation of data.
Certainly many (but as you point out not all) of the participants on the agenda are familiar for-profit clinics selling stromal vascular fraction (SVF) and some for very diverse entirely non-adipose related conditions. For instance, what do you think of the use of SVF for neurological conditions? I ask this in part to point out that there are legitimate questions about non-homologous use. Perhaps the case of breast reconstruction after cancer is a more complicated area as to decide about homologous use, but for others such as cardiac, neurologic, pulmonary, etc. it is very difficult to see how by any stretch of the imagination the uses being done on a daily basis with SVF out there are homologous use.
As a stem cell biologist often conducting basic science, I would also question the claim that the FDA draft guidances are inaccurate scientifically. Can you be more specific as to what you mean?
Thanks again,
Paul
Paul, if the ARM and the CIRM also want looser regulation that might tell you something. This is a medical procedure and should not be regulated for “clinics” but for Physicians under their licenses and regulatory bodies. Physicians are being trained in these procedures left and right, but most don’t do the procedures or don’t say it publicly, due to fears like the ones your blog creates. So, many patients miss the opportunity of getting the benefits (that you know exist). So, instead of making a career out of creating fear about FDA regulation you should make a career out of how to get these procedures safely to the people who need them the most (If you think you are doing the latter, you are not). Use your reach to facilitate that all these physicians perform the procedures and you will see the “clinics” with their abusive prices disappear.
How much faith can we really put in the FDA? Margaret Hamburg is being sued, Robert Califf’s nomination has been put on hold. What next? If our own stem cells administered by a physician were profitable to the tune of a mass produced pharmaceutical drug, would we even be having this discussion? Since Big Pharma, FDA and others aren’t making money off these therapies, it’s imperative that they be stopped.
Who really gives a hoot about the patients anyway? When we post here we always seem to get a lot of thumb’s down for our comments if they are even posted at all. And I have to agree with Tracy that anyone with such grave concerns as so many profess to have about our safety should be attending the meeting and speaking.
Since little has been mentioned about the lawsuit – here it is. You may want to plug your nose while you read it. The smell is pretty bad. http://www.qmed.com/news/former-fda-commissioner-jj-targeted-federal-lawsuit?cid=sm.qmed.site.qmedsocial&hootPostID=274e0ae283f928abd7add1129a5a5ca9
In the U.S. there are two FDA regulatory segments for heterogeneous herbal products
Dietary Supplements: http://www.fda.gov/Food/DietarySupplements/
and
Botanical Drugs: http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ucm090983.htm
At the end of the day they result in pretty much the SAME output
One has become a $100 billion industry with limited oversight
One has languished to produce any products despite having big pharma investment and involvement
Reason: people don’t care about the “FDA premium”
The stem cell segment is going to face this same existential dilemma but in 2016 its regulatory domain become the whole world
The notice says, “FDA will consider information it obtains from the public hearing in the finalization of these guidances.” I understand the need to let everyone have their say, but surely the the assessment of whether or not HCT/Ps in their current form are a risk to the public will require careful assessment of clinical evidence?
Patient advocacy has been instrumental in getting this on the table, but neither those groups nor any clinics will be presenting controlled clinical trial data in 6 minutes. So is there another meeting where we see the data on which the later regulatory rules are based, or will the hearing only result in a modification of the HCT/Ps guidances – which don’t appear to have been effective for any party.
It is so interesting to me to see the “thumbs down” on @Jennifer.Ziegler’s comments about no option patients seeing life improvements from adult stem cell therapy. We are not talking about cosmetic procedures here! Who would think so negatively about a person fighting for their life and getting results from an alternative treatment? PFSC is definitely not anti-FDA. In fact we look forward to working with the FDA in bringing accelerated access for adult stem cell therapy to their current regulatory framework. This is a new age in medicine, and we are excited to be a part of it! We are in agreement with CIRM but believe it or not, were not interested in a photo op! That’s a little berating if you ask me.
It’s a shame that you or any of your contemporaries didn’t take time out of you schedule to attend this hearing as this is obviously of grave concern to you. Most of language in your blog piece is pretty heavy handed and critical. It’s obvious you have strong feelings about this subject.
Hi Paul, just a quick Q from a lay person:
If a sponsor completes trials for (say) cell therapy in knee OA, would they have to complete the process again for the body’s many other joints?
Great blog and best wishes,
Matthew
“That would be a good thing for patients who are often being put at risk by many of the clinics. This is the reality no matter what some of the patient advocates who promote the clinics might say. Such finalized FDA regs that unambiguously state that, for example, fat stem cells are drugs, would be bad news for the clinics that are after patients’ money.”
Paul, as a founding member of Patients for Stem Cells, I would like to clarify on the above statement to show you this is NOT the agenda of Patients for Stem Cells. We’ve NEVER promoted “clinics.” I’m surprised after interacting with you over the past 4 years, that’s what you think we do! It’s really shows how little is understood about the reality of a “no option patient,” situation. As an Multiple Sclerosis patient myself (dxd 2004) I’ve had major quality of life improvements being treated with my own cells.
What PFSC does promote is the right to share information so a patient can make an educated decision with family/doctors on where to seek adult stem cell therapy, when all other FDA approved medications have harmed/failed. The reality is patients have the right to have their voices heard! An injustice is being done, not only to patients, but to physicians, who’s right to innovate under the practice of medicine is being infringed upon. Regenerative cell therapy, especially adult stem cell therapy, has proven to be safe and effective for thousands of patients. Patients know it, other countries know it, and I hasten to say iPC cell scientists know it.
Luckily Patiens for Stem Cells has been given a 6 minute time slot to share our stories. Will you be speaking/attending the hearing?
Best,
Jennifer Ziegler
Founding Member of Patients For Stem Cells
Patient Advisory Board Member, Alliance For The Advancement of Cellular Therapies
“They might even try to get their picture taken with CIRM staff to use on their websites.” You have got to be kidding, right?
Are you planning to go to this meeting? I’d like to be there.
Jeanne
Nope, can’t make it. Are you thinking of going, Jeanne? You could ask if they could squeeze you into the agenda. I hope they would. It’s a long way for US coast people to go for a 3-minute speaking slot, but the meeting overall would be quite interesting. It will be webcast…trying to find that link. Paul