The FDA’s stem cell meeting wrapped up today on day two with a diverse group of individual speakers. A series of patient testimonials today in favor of clinics was one thing that stood out. You can read my take of day one and the account of Jeanne Loring who was at the meeting. I’ve noted that on average about 350 people were watching the webcast of the meeting both days.
The patients gave powerful, often emotional testimonials today on their experiences at various stem cell clinics including Celltex and most prominently Stemgenex. I think there were 7 of its patients who spoke. I have no doubt of the patients’ sincerity in their belief that they’ve been helped.
Various stem cell clinic doctors also testified.
As with yesterday, a common statement was that there are “no side effects” of stem cell treatments, which is concerning as all medical therapies have some risk of side effects.
A number of health care practitioners and researchers voiced support for the FDA and its draft guidances. For instance, nurse practitioner Sheila Sabon DeCastro did an especially outstanding job at articulating the urgent need for the draft guidances to be finalized.
Jeanne Loring gave a talk regarding both stem cell clinics as well as her own clinical research to develop IPS cell-based products for Parkinson’s Disease. She did a great job.
Bioethicist Leigh Turner talked about the stem cell clinic marketplace in the US today including discussing our work together that we recently published in Cell Stem Cell on the astounding number of stem cell clinics in the US at this time. He was supportive of the FDA draft guidances and commended the agency for them.
However, Leigh was also critical (rightly so I believe) of the lack of appropriate FDA oversight of stem cell clinics recently and noted this inaction’s likely contribution to the growth of the stem cell clinic industry. For instance, Leigh mentioned that he contacted the FDA three years ago about specific clinics that concerned him, but that those clinics still operate and the FDA apparently has not done anything.
He ended with a very assertive concluding paragraph:
“The out-of-control marketplace for stem cell interventions needs effective regulatory oversight. I therefore hope the draft guidances are more than stage props and this hearing is more than public theater. When patient safety and public health are at stake, the FDA must do more than function as a paper tiger. It is time for action.”
It is time for action or who knows where things will be a year or two from now. A thousand clinics in the US administering unproven stem cell products to hundreds of thousands of patients?
Opponemts of stem cell treatments are left out of the profits. Hit corporate companies in the wallet, and they will want in on the action. Just a matter of time before stem cell txs are approved. Big pharma will continue to fight it. Humira doesn’t pay for itself.
Homologous use of stemcells is a contradiction by itself.I find the sentence that ‘adipose msc has no place in airway,brain or bloodstream’ ignoring some basic facts.No 1 is MSC is not a single cell but a mixed population of cells in varying differentiation pathways waiting for appropriate clues to take a particular direction towards tissue differentiation.There are no ‘adipose mscs’ but mscs in adipose tissue which are constantly replenished from bone marrow.Arnold Caplan’s statement about Stromal cell being mistakenly named as MSC but essentially a cell secreting paracrine factors underscores our evolving knowledge.As clinicians we have used intestine to supplement a defective/partially removed bladder wall;omental tissue with just fascia and fat for closing rents in intestine, uterus etc ; fascia in the place of tendons etc.Years after these surgeries on reopening we find the transformation of substitutes into the original tissue (change in collagen type).My request is not to have fixed ideas particularly about homologous use but be open to understand basic physiology with a understanding of embryology to see how all these Non-homogenous tissues evolved from single cell and what role do signals play on these cells.
That’s a very interesting perspective and I’ve no doubt that the FDA will need to review the evidence on tissue transplantation when reformulating their “non-homologous use” definition.
But even clinicians must have a scientific rationale for transplantation of tissues to novel sites. And surely the clinician would rule out, e.g. transplantation of omental tissue to say, the brain?
Where can one find the rules laid out for such pro/con arguments and could these be incorporated in FDA guidelines?
After 3 years, Dr LookGood finally shows up again. Are you going to provide your response to the FDA Form 483 concerns, as you said you would in August 2013? Do you still claim that your process is viable and is FDA compliant, as you told all your shareholders when you launched your public company and put out that launch video from the Hamptons that is still on youtube?
Looks like Dr LookGood got nothing to say to your comment Cathe. Shareholders are screwed.
After listening to 2 days of the FDA meeting I would like to ask Paul or anyone what is the scientific importance of Homologous in the regulations? If the tissue or cells are not homologous is there any risk for the patient? After thinking and thinking about this I can not see why homologous use is an issue at all and what role it plays in the science or regulatory positions. I would love to have someone explain this to me. If a tissue or cell is used for a nonhomologous use and it works then why is it bad? Looking forward to some education.
Good question but the truth is that “homologous use” is nothing more than a concept created by FDA to make these treatments illegal.
@Steven,
The idea of homologous use seems to be that using a biological product like stem cells that is homologous to the target organ to be treated would have lower risk overall inherently.
For instance, using lung cells to treat lungs, makes good sense.
So does using fat cells to treat fat-related issues, bone marrow for issues related to bone, cartilage, blood, etc.
Scientifically to me this seems like a valid concept.
Common sense also seems to dictate that you can’t just squirt any old type of cell into any random organ or into the blood stream and hope that things go well. Nature doesn’t work that way. To my knowledge, in each of us our fat stem cells don’t travel around our bodies naturally to other entirely different tissues in the thousands or millions and do helpful stuff. Blood cells may be do this via capillaries, but not fat cells.
Adipose MSCs have no business for instance naturally being in an airway, in the brain, in the bloodstream, etc.
Could they in theory still be helpful in such places?
Yes, in theory but there’s a higher safety hurdle to meet because of the lack of homology, which again seems logical to me. Another example, if one transplants SVF (fat stem & other cells) into a very different tissue like airways or heart, etc. and they engraft (which is unlikely given recent data, but possible) you could end up growing fat tissue inside an organ where it is harmful to have fat randomly sprouting up.
Paul
@Steven, Just focus on overseas in China and get the toxic debt paid off. That is the only way to get out from under this wet blanket!! Hoping the best to you!!!
Paul, thanks for giving an even handed assessment of the FDA Part 15 hearing. I truly hope this will lead to constructive solutions, like the “middle pathway” as proposed by the Bipartisan Policy Council and Arnold Caplan
http://www.cellr4.org/article/2109
I know you’ve posted some reports of adverse events happening to patients, but hear are the results of a FOIA request, against the 570 clinics you reported on. Only 4 adverse events were reported.
http://www.patientsforstemcells.org/foia/
@SammyJo,
I have strong opinions as you know and I know that you do too, but I tried to be balanced overall.
Unfortunately there have been more than 4 adverse events from stem cell clinics.
What exactly did you ask for in your FOIA? Can you please post your FOIA request.
Was the FDA able to comprehensively look for data from all 570 clinics that quickly?
I also expect that not all adverse events make it to the FDA. Maybe most do not. There have also been some at the state medical board level (e.g. just one: https://www.ipscell.com/2014/01/perspectives-on-emergency-suspension-of-stem-cell-clinic-doc/ ).
Then there were the deaths after stem cell treatments by Grekos.
Also, we’ve heard just in recent weeks that 3 or more patients were reportedly (e.g. Dr. Albini’s presentation) blinded by just one clinic in recent months so that alone is some serious adverse events just recently beyond what the FDA FOIA indicated and all from one business out of 570.
I know of other, in some cases very serious adverse events that have not yet entered the public domain too.
It’s in everyone’s best interest to get comprehensive adverse events data. Your doing the FOIA is one positive step, but I think there’s more to be done before we can be sure we’ve got the full story on adverse events.
Paul