REGROW Act is Attack on Science-Based Stem Cell Trial Oversight

REGROW ActSenators Mark Kirk, Joe Manchin, and Susan Collins have proposed new legislation in the form of the REGROW Act that would substantially interfere with the FDA’s ability to properly regulate the development of new stem cell and regenerative medicine therapies based on hard science.

The REGROW Act would force the FDA to allow rushed introduction of experimental stem cell interventions into patients even if the science did not support that these were safe or effective at that time.

From the Kirk press release:

“Unfortunately the Food and Drug Administration (FDA) has identified the current lack of regulatory standards in this area of medicine as an impediment to treatment development. Countries like Japan and England are outpacing the U.S. in regenerative medicine therapy development due to new regulatory policies that the U.S. has yet to mirror.

S. 2689 will allow the United States to regain prominence in the field of regenerative medicine science and bring therapies quickly to the patients that need it most.”

This libertarian agenda fits with the recent BPC report that overreaches on FDA deregulation. See my take on the BPC report here.

You can also see a nationalistic agenda evident in the language that is not scientific. Just because some authorities in Japan have chosen to dramatically weaken regulation of stem cell therapies there and made other questionable decisions such as more widely allowing charging of patients to be in stem cell clinical trials, doesn’t mean the U.S. should follow the same somewhat reckless path.

These practices are highly questionable from a bioethics perspective and also from a simple common sense view that rushing ahead too fast can actually slow things down by leading to catastrophic patient outcomes. The Jesse Gelsinger case with gene therapy comes to mind and its interesting that some advocate a race car mentality for use of CRISPR in humans too.

My understanding is that the dubious stem cell clinic lobbying force is happy with the REGROW bill as it would shift the regulatory framework toward their philosophy even if they don’t get everything they want. REGROW proponents want to rocket forward with stem cells into patients by eliminating Phase III trials and establishing a risky conditional approval paradigm. I haven’t seen any evidence that Phase III clinical trials for stem cells are unnecessary nor that the conditional approval paradigm would be safe.

The REGROW Act summary page is here and the full text of the bill is here. Fortunately analysts predict only a 4% chance of passage of this dangerous bill.

20 thoughts on “REGROW Act is Attack on Science-Based Stem Cell Trial Oversight


  1. Paul,

    REGROW proponents don’t want to “rocket forward” to treatment, they want to rocket BACKWARD, to the 8-10 years “hard science” has already been established and applied in: Germany, France, Sweden, South Korea, Thailand, China, Mexico and others. Can you cite ANY international research (not n=1 single-case studies) that, since 2014 or so, declares publicly-available treatments in other countries as being direly unsafe? Serious, significant adverse effects/outcomes that that are not now established as easily avoidable or to have a clinical workaround?

    I don’t think you can.

    In addition, international research now has longevity studies on its side. iPSC’s (unless they can also “rocket forward”) will not be available for 10-15 years, and then, without longevity studies.

    The U.S. is behind. We need all oars in the water, Paul, and, the existing, (largely un-altered adult stem cell) treatment proponents know that. If the NIH and FDA will help to fuel innovation through recognition of global consensus of safety and efficacy studies, the benefits to iPSC research you’ll know more. And you’ll have to research less to perfect the holy grail of off-the-shelf iPSC’s).

    Not only will there be these obvious benefits, but imagine patients having the option of “compassionate care” decisions (no effective treatment, no hope prognosis, no established cure). It’s well established here in the U.S. that a person has a right to determine what happens to the tissue from his/her own body.

    I’d be glad to provide you journal references if you like, on the condition that you provide them to this forum without comment.

    Best,

    Doug Oliver (who went from legally blind to legal-to-drive in 8 weeks after autologous BMC treatments, isolated without reducers or culturing).

    Nashville, TN


    • @Doug,
      I’m not aware of “hard science” proving that MSCs are universally safe regardless of how different entities prepare and use them with their hodgepodge of methods.
      As to your demand of not making comments on references, this is an open forum for dialogue and exchange of information as well as opinions so that’s a no-go. If you have something to share, just post it without condition.
      While MSCs have a strong safety profile overall, there are many articles that raise different concerns about MSC safety such as this new one on chromosomal instability:
      http://www.celltherapyjournal.org/article/S1465-3249(15)01147-0/abstract?cc=y=
      Other risks include lack of expertise in areas being treated in patients, infection, autoimmune reaction, abnormal tissue growth. There are not many years of solid data to go on in most cases. For instance, SVF was not widely used in the past.
      There are safety issues with any kind of stem cell and pluripotent stem cells have an inherently higher risk for sure, but I don’t see labs working with pluripotent stem cell-based products claiming that they are not drugs, don’t need to go through Phase III, etc.
      Paul


  2. On the one hand, it’s good that stem cell therapeutics are on the active political agenda and I common sense will surely prevail. Nevertheless, the draft is badly written, is self-contradictory, and sadly leverages national interests claim to shoehorn in all those unregulated stem cell clinic practices, such as non-homologous use.

    @Doug – the claim that Europe is somehow ahead and “hard science has already been established and applied” is incorrect – don’t believe everything politicians tell you. Governments and regulators in Germany, France and Japan are waiting for the US on this issue. Unregulated stem cell clinics and their practices are being slammed here too.

    Japan has only expanded a rule for pre-phase III approval in certain cases – it has not given carte blanche for non-homologous use and other changes listed in the REGROW draft. That is simply a political machination.

    In Germany, health insurers (guided by the G-BA and IQWiG) have stated that they will not cover therapies that have not demonstrated clinical efficacy and benefit over existing therapeutics – in the target patients, even if these are approved by EMA. Key here is “in the target patients” as an ethnically diverse population cannot be represented in a phase II trial, because there are insufficient patient numbers – this is even more of an issue in the US, but less so in Japan.

    So I’m not worried by this draft and its knock-on consequences for Europe or Japan – it will never be approved and 4% for effort is what I would give it too.


  3. @Jeff,
    but it is true there are some therapies only available for example in Germany. And there are some American Sport Stars who travel to Germany for treatment. Just look at google for example for Kobe Bryant, (I think Demi Moore, too ) … Kobe Bryant seems to be very fit, so his application seems to work…
    Can you answer the question, why Kobe Bryant needs to go to Germany for treatment?

    http://grantland.com/features/kobe-bryant-dr-chris-renna-regenokine-knee-treatment/


  4. @Bob – the treatment is still available in Germany, not because German regulators have approved it (as US politicians will have you believe), far from it – but because old German laws are still used to enable a person to carry our his learned trade – a law that unregulated practitioners use to prevent closure.

    Generally, these unregulated stem cell practices are de facto illegal under German law, but the older law prevails. For this reason you can still get homogenized goat brain injected into your muscles under the name “fresh cell therapy” – good luck with that!

    As I said, Germany is waiting to see how the FDA rules on these clinics before drafting their own regulations. For now they wait until someone is harmed or dies and then close the clinic. Since the XCell Center (www.telegraph.co.uk/news/worldnews/europe/germany/8500233/Europes-largest-stem-cell-clinic-shut-down-after-death-of-baby.html), the authorities have closed about 30 of them, but it’s easy money for these clinics to lure people desperate for hope, and they just pop up again in another state.


  5. @Jeff, but why should Germany be wrong?, because Kobe Bryant and others seem to be treated very successful.
    Why do you think Germany is waiting for the FDA? Sorry, I can not believe this. I don’t think Germany will shut down successful therapies because of the FDA.

    Besides it does not make sense to talk about of all stem cell therapies – you can not put them all under one umbrella. Of couse there are good ones and bad ones and the therapies Kobe Bryant received in Germany seem to be very successful.


  6. @Doug,
    could you please give us some more information about your successful treatment?
    “Doug Oliver (who went from legally blind to legal-to-drive in 8 weeks after autologous BMC treatments, isolated without reducers or culturing).”
    It sound amazing. Where have you been treated? What kind of cells have been used? How many cells?
    Thanks


  7. @Bob, “why should Germany be wrong?” – not sure I understand the question, but Germany has ruled that cells that are more than minimally manipulated or are used in a non-homologous fashion are drugs, and must be regulated by EMA (Europe’s FDA), so the situation is identical to the US.

    I agree with you that not all stem cell therapies are the same, but all “successful therapies” (when defined as drugs) must go through controlled clinical trials and demonstrate statistically significant safety and efficacy, compared to a standard of care (if there is one). This is how both US and Germany operate and a current bone of contention is when to call a cell a drug.

    Why are German regulators waiting for the FDA? Because FDA is taking the lead – they have issued guidelines, called a public hearing, made key rulings on some clinics, based on medical practices – whereas Germany has only shut down clinics based on criminal law – the FDA is ahead of Europe here.

    Btw, I don’t know why celebrities go to Germany for the same unregulated, unproven therapy they can get in Sacramento or Mexico. Maybe those clinics are now getting some bad press and Germany still sounds “trustworthy”.


  8. But the crucial point is that these therapies (in Germany) seem to work, because Kobe Bryant seems to be treated successfully. I think many people have been treated by these therapies in the last years (not only celebreties), so I don’t think that these therapies are not proofed at all. Maybe no double blind trials until today, but why should patients wait?, because therapies seem to work.


  9. @Bob – these unproven, uncontrolled clinics are no better in Germany than anywhere else. And whether the therapies even work is impossible to judge as they report no data i.e. unproven and uncontrolled.

    The only true measure of whether a drug therapy is better or safer than others is clinical trial data, where you compare the drug / cells against other treatments or placebo in a cohort of patients. One Kobe Bryant (n=1) taking who knows what other therapeutics and self-reporting that he feels better is no measure of therapeutic success.

    Btw, Bryant is listed as questionable for Wednesday’s game against the Heat, due to shoulder problems – so apparently Germany wasn’t that good.


  10. @Jeff
    As far as I know was Kobe Bryant treated by Professor Wehling in Germany. Professor Wehling has got interesting publications and he is also a professor of Chapel Hill in South Carolina, so his views and treatments should be accepted by other experts in the USA, too.
    Kobe Bryant is not n=1, because Professor Wehling has already treated many patients with his methods. So I think it is not true that there is no data.

    Because of these reasons I can understand the comment of Doug, who wrote: “The U.S. is behind.” and I hope the USA will take the lead again.

    http://www.mensjournal.com/magazine/the-body-that-heals-itself-20140217


    • @Bob,
      In science, we do not consider this kind of thing to be data when pro athletes go to a clinic and then we judge them based on how they perform in their games. That’s not data. If folks like Wehling are really getting consistent, safe, good results, what they must do is collect all this data in a rigorous manner, submit it for peer review, and publish it. Only then can we really be sure what the heck is going on. Otherwise it is not worth much of anything. The treatment/use of athletes by stem cell clinics both here in the US and abroad, and then media reports and PRs of the outcomes are essentially just advertising and not science.
      Paul


  11. @Paul
    Professor Wehling applies these methods probably over 10 years, so that his experience is certainly based on data of at least maybe 10 000 patients or even more. I hope, therefore, that he knows exactly what he’s doing and what his therapy can. Personally, I can’t decide whether his therapy holds actually, what it promises, but I think the probability that it is working (in view of the fact that he has so many experiences) is relative high. Patients must decide, if they trust him or not and many patients seem to trust him …
    Therefore, the United States should pay attention not to fall back and stay the country of medical progress.
    The outcome of some celebreties (n=1) is of course no data, but we can assume that these guys may have very good/maybe the best advisers.

    For example, if a sport star has a big knee problem, they will often be send to the Steadman Clinic in Colorado. This clinic has fixed already so many knees of sport stars. If you read about sports in newspapers you will know that, but I am not sure, if this clinic has also proved these special abilities by double clinical trials?
    Mabye Professor Wehling is the best option for new therapies? I can decide that ultimately not sure, but I think that despite lacking double blind studies you should take a second look to these therapies. It could be a shame, that patients had to go to Germany to get a treatment – in former times it was vice versa.


  12. @Bob – thanks for the info. I checked out Wehling’s therapy (Regenokine) and discovered it’s not stem cells, but re-injection of your own warmed up blood plasma. The idea has been around for about a decade under the name Orthokine, but was abandoned due to more effective treatments.

    Interestingly, Chris Centeno of Regenxx posted on this too. His conclusion: “Regenokine is not regenerative, but an anti-inflammatory shot that lasts for about 6-9 months and has generally long since been abandoned by US vets for more effective platelet rich plasma (http://www.regenexx.com/what-is-regenokine/) He also shows data on the ineffectiveness of the therapy in actual repair of tissue damage, which is not surprising if it’s only action is anti-inflammatory.


  13. One thing to consider: that millions of patients with untreatable conditions such as spinal injury, stroke, Parkinson’s disease, and many more are waiting. And many of these patients will die waiting.

    I’m sure many have seen the recently published study showing corticospinal tract repair achieved in rats. How many years until human trials/therapies? Affected patients would be prepared to take many risks.


  14. @Jeff,
    interesting facts, thank you, but the question is: who is right? Centeno or Wehling? Has Chris Centeno enough evidence for his opinion? I can not really decide which therapy is better? If anybody knows more, please let us know.
    Both, Wehling and Centeno, should have enough data to give us the necessary background information.
    I think that’s the same Paul already mentioned, we are very interested in more data.


  15. @Bob – the data is already out there and Centeno and Wehling don’t disagree: Regenokine is a minimally effective, short-term, non-regenerative, anti-inflammatory treatment. Wehling markets it as a “proven effective therapy”, which doesn’t contradict the statement above and is still legal for marketing.

    Obviously, he leaves out the fact that it’s now obsolete for humans (at least if you want effective therapy or cure). Aspirin does a similar job, is cheaper and I don’t need to go to Germany for it.


  16. I’m all for it just because Paul is against it. Paul has never been on the right side of any issue in his life.

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