Cell Surgical Network, largest group of US clinics, using lab-expanded stem cells in patients?

Elliot Lander Mark BermanIs the largest affiliated group of stem cell clinics in America, Cell Surgical Network, now using laboratory-proliferated stem cells in patients?

Do they already have some kind of final FDA approval for this clinical approach given that lab-grown stem cells are generally viewed as drugs requiring premarket approval?

Over the years I’ve reached out to interview many members of our diverse community in the stem cell arena including those operating stem cell clinics. One past such past interview (here and here) was with the leaders of Cell Surgical Network, Drs. Mark Berman and Elliot Lander.

Even though more broadly those operating stem cell clinics across the U.S. and I don’t see eye to eye on many things, the interviews are valuable to the community, providing insights generally not otherwise found in the public domain.

Today’s post is a new, striking interview with Lander and Berman (pictured above). I invited them to do this short Q&A because there have been indications that their group of clinics may be gearing up to or already has been taking a different approach (at least compared to what I knew about in the past) to using stem cells in patients with the possible new approach involving laboratory-amplified stem cells.

For instance, on their website FAQ page they refer to using seemingly laboratory expanded cells (emphasis mine):

“Autologous lipo-aspirate can be frozen as SVF Stromal Vascular Fraction (contains mesenchymal and hematopoetic stem cells). SVF can be deployed for repeated treatments and also expanded under IRB approval as part of a safety trial providing vast quantities of autologous stem cells that could be used throughout that patient’s life.”

The question of lab expansion is such a crucial point because to my knowledge lab-expanded stem cells are considered a biological drug by the FDA requiring pre-market approval steps such as an IND, IDE, and/or BLA. Also, typically a safety trial of the type mentioned would be an FDA-approved, Phase I clinical trial based on an IND and I’m not aware of Cell Surgical Network having that.

To my knowledge, IRB approval alone is not a sufficient basis for doing a clinical trial on a biologic. Am I missing something here? Is Cell Surgical Network’s apparent IDE application with the FDA going to encompass data usually found in an IND as well? Why not do an IND and an IDE In this case?

The point of this interview was to try to clarify this situation. Thanks, to Berman and Lander  of CSN for doing it.

PK: I’m hearing that you are apparently growing adipose stem cells in the lab these days for clinical use (transplantation) in patients. Is that correct?

CSN: Yes – Our patients receive re-implantation (not transplantation) of their own cells under this protocol. 

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National Academies panel leaves door a crack open to future human genetic modification

Early human embryosA National Academies panel on human genome editing chaired by Alta Charo and Richard Hynes released its report today. While it covered both somatic and germline (heritable) human genetic modification, the latter topic is far more contentious. You can see the National Academies summary of their report here. You can read the full report here.

On germline genetic modification using technologies such as CRISPR, the authors of the report carefully discussed potential benefits and risks to the use of this technology. On my first brief reading of it, the report is mostly appropriately cautious in terms of outlining the potential risks as well as societal and bioethical issues in addition to potential benefits.

As to the more controversial area of potential human germline genetic modification, I appreciate the fact that the report unambiguously says that human germline editing is not ready to be tried and lists numerous hurdles to address first. However, although the report tackles most of the key considerations and risks (and for that the authors should be commended), there is still an interesting undertone to the report’s discussion that seems to strive to justify leaving the door open to potential future use of human germline editing.

For instance, the report makes a point of noting mostly hypothetical instances where existing technologies such as PGD that are far less controversial may hypothetically fall down and so possibly might need to be supplanted by germline editing using techniques like CRISPR, but such instances are now and in the future would continue to be exceedingly rare. Too much weight are placed on these.

The other point that needs to be made is that hypothetical future germline editing in human embryos (or even done earlier developmentally in, for example, germ cells) with reproductive intent would have to be preceded by a great deal of research that involves PGD-like testing of human embryos to monitor efficiency and specificity of editing, chimerism, and other issues so it is not as though germline editing could avoid PGD-related issues or destruction of embryos.

I’m also not convinced that the personal need by some individuals to have a genetically-related offspring should be a major consideration in decisions related to the permissibility of human germline gene editing. The related concept of parental autonomy is given too much weight by the report. While this argument has both aspects that intuitively make good sense, it also has some that could lead to promotion of germline modification.

For instance, what if parents want their kids to have certain traits that become potentially available via germline modification? In this regard, the ability of researchers and policy-makers both in the US and more globally to prevent parents and practitioners from going down the path to use germline human modification for traits rather than strictly health conditions may prove to be quite limited and it’s not clear had to address that risk. The report in some places does not use strong enough language to counter the idea of human germline modification.

On the whole, I have favored a moratorium on germline modification, a step that this report does not take. Of course there are possible problems with a moratorium too including enforceability (lack thereof) and that once you have a moratorium it might be hard to escape it (e.g. if the government follows suit with legal steps) even if down the road the science were to back up getting rid of it. Note that as the report mentions, for at least some months longer, in the US the FDA cannot consider applications for any human embryo genetic modification and then there’s the Dickey-Wicker Amendment that disallows federal funding of anything that involves ‘destruction’ of human embryos and such things as using CRISPR modified embryos to do PCR for research would qualify as destruction. But then there’s loads of private money out there for research as well.

What are your impressions of the report?

For more of my thoughts and concerns on human genetic modification you can read my book GMO Sapiens and watch my TED talk (below).

Dr. Oz Explosive Exposé on Stem Cell Clinics Airs Tomorrow

American stem cell clinics put thousands of patients at risk each year through hawking expensive, unproven and unapproved medical interventions, and now Dr. Oz is reportedly taking them on in a new show set to air tomorrow. dr-oz

Those running the clinics have affixed the buzz phrase “stem cells” onto a whole range of stuff ranging from A (actual stem cells, but unproven) to Z (zombie cells; aka not really living cells of the stem cell variety.)

While at times in the past the Dr. Oz Show has been criticized for how it discussed unproven health interventions, from what I can tell on the stem cell front now, they are very serious about exposing how risky the stem cell clinic industry has become.

A clip of the Dr. Oz show I was able to see in last week was striking. Another clip above from the show of Montel Williams is quite intense.

I was able to get this quote from Dr. Oz himself about this situation and the show:

“These stem cell clinics are using the potential of legitimate research to take advantage of patients desperate for help. These physicians are violating not only the trust of their patients but also the law and hopefully our show will push the FDA to use its authority to shut them down.”

I’ve set my DVR to record it. If you have any interest at all in stem cells as a patient, scientist, physician, student, grant funder, science writer, FDA person, FTC person, etc., you should check this out.

Dr. Sally Temple, President of ISSCR, is also on the show. I’m told that the stem cell clinic segment will be the second half of the show.

Japan IPS cell trial for vision to start in new incarnation

Masayo Takahashi, IPS cell trialIt’s been a long road, but the first ever IPS cell clinical study in humans is starting up again in a new incarnation. You might say it has been regenerated in a novel form.

Masayo Takahashi (高橋 政代) first started the pioneering IPS cell study a few years back in 2014, but it ended up getting put on hold in the summer of 2015 in the midst of changing regs in Japan and the discovery of some mutations in the IPS cell derivatives.

About a year after the hold was put in place, we got news that the study would be restarted in a new incarnation, and now more definitively the study is on track to start up again with 5 patients in Japan with the wet type of macular degeneration.  You can read the RIKEN PR here.

The PR provides more info including a co-leader:

“The project will be led by Yasuo Kurimoto and Masayo Takahashi of Kobe City Medical Center General Hospital, and Osaka University’s Graduate School of Medicine/ Faculty of Medicine, and will be conducted in collaboration with the RIKEN Center for Developmental Biology (CDB) and Kyoto University’s Center for iPS Cell Research and Application (CiRA).”

This is great news.

Yasuo Kurimoto is the surgeon who did the initial surgery in the study before it stopped.

One of the most striking things about the new form of this IPSC study is that it will reportedly focus on allogeneic use of IPSC rather than autologous. The foundation for this switch is that allogeneic cells derived from the IPSC can be used in matched recipients and in a relatively genetically homogenous population such allogeneic cells can be used in a number of patients with a good possibility that there won’t be rejection of the transplant.

Embryonic stem cell-based therapies can be used in the same allogeneic way and those are being studied for macular degeneration as well. I still feel like in the long run that the most power and unique positive contribution from IPSC clinically speaking may come from autologous use, especially in diverse populations such as the U.S. Their use in disease modeling is quite impactful as well.

Fractals, fibonacci, & food: romanesco wows in garden

As some of you readers know, in addition to being a scientist, I’m also an avid gardener and photographer. Every now and then I do posts on gardening or photography. I feel like I bring a scientist’s eye to the garden and that was true a few days ago when I captured the above picture of Broccoli Romanesco.
romanesco-knoepflerBoth normal people and scientists have been wowed at the fractal patterns in nature for centuries. This winter I mainly planted regular broccoli and cauliflower but threw in 3 romanesco plants for the heck of it later on. It’s been a dark, cold, and wet winter so when I pop out to the garden often it has been dodging rain drops to just get broccoli or cauliflower to eat.

Meanwhile the romanescos buried in the garden have also been growing, mostly unobserved. With all the other cruciferous vegetables gone for stir fry  and other dishes, one day recently with a nice break in the weather I thought to check on the romanesco and was struck by its beauty both in a naturalistic sense and mathematically. I start thinking of Fibonacci numbers too.

mantis on sunflower

There’s both a randomness and organization to a plant like this. I also in a way am reminded of sunflowers as well when I look at romanesco. Above is one of the more striking photos of sunflowers from my garden in past years with two praying mantises (manti?) hunting for bees. Note the fractal-like spiral in the flower.

While the romanesco may not be a perfect fractal, it is remarkable even so. At some point of course we’ll have to eat this amazing romanesco flower, which also happens to be a nutritional powerhouse. By the way, latter in the year one mantis caught and ate a bee. It’s crazy what you can see out in the garden.

For more pics and videos including from my garden, check this page out.