REGROW Act 2.0 still no-go with big risks to patients & field

Mark Kirk REGROW ActThe REGROW Act is a bill that seeks to lessen the regulatory burden to accelerate getting stem cells to patients more quickly, but it over-reaches so much that it would almost certainly do harm to patients and maybe to the stem cell field as a whole.

The REGROW Act, which is sponsored by Senator Mark Kirk of Illinois, has had several incarnations, but the latest new one reflects some big changes so I’m going to call it REGROW 2.0.

A source provided me with a copy of REGROW 2.0 and it is surprising in a number of ways. Unfortunately the bottom line is that it is still a no-go because it would be so risky.

The original REGROW would have forced a conditional approval system on the FDA whereby still experimental stem cells could be given to patients with little data supporting this use.

REGROW 2.0 has dropped the extreme conditional approval so that seems like a good thing. Instead 2.0 focuses on accelerating approval, which on the surface also seems like it could be a positive thing. However, the devil is in the details.

Too broad on cell product type? The new version of the bill drops all requirements on the types of cells that would qualify including the previous filters for genetically modified cells. So anything goes for stem cell types in REGROW 2.0.

Too weak on medical conditions? The new version of the bill also would weaken in a really big way the standard requirements for accelerated approval for experimental stem cell products by the FDA. No longer would the patient need to be facing a very serious condition with unmet need such as a fatal illness, but now instead the acceleration could be used for many things that fit into the broader category it stipulates:

“the treatment of a serious condition for which the product is approved under this subsection may include a chronic, persistent, or recurring condition that affects day-to-day functioning without taking into account the availability or lack of alternative treatments.”

Devices mess. This could mean almost anything so REGROW 2.0 would put a big old loop-hole in there. REGROW 2.0 also still seems problematic related to stem cell devices as it is weak on the rules required for determining what can or cannot be used.

Stem cell clinics covered too? One of the other problems with both the original REGROW and its 2.0 version is that some of the not-so-good citizens within the stem cell arena may take advantage of its mandating of weaker oversight provisions.

FDA has issues but it’s not only problem. The bill also represents a philosophical shift toward the idea that the FDA is the only problem here with translation of stem cells. Yes, the FDA has some issues including a major lack of clarity and slowness, but it’s too simple to say the FDA is the only problem with the stem cell clinical translation arena.

For instance, experience so far teaches us that safe and effective cellular therapies are most often going to be very challenging at scientific and medical levels to produce and prove with convincing data that they are superior to the standard of care. Shortcuts around this rigorous standard help no one in the long run.

Who is writing REGROW and is there an industry connection? Even though Senator Kirk is the prime sponsor of the REGROW Act, the language of the bill certainly continues to feel like it was written by stem cell companies who want less oversight by the FDA. I wonder who exactly is the person or persons writing and rewriting REGROW? How much of a role if any does industry play in that?

Bottom line. What this all mean is that overall I still have to say “no” to REGROW Act 2.0 from my perspective. It’s too risky for patients and the field.

10 thoughts on “REGROW Act 2.0 still no-go with big risks to patients & field”

  1. The 30-day mortality rate for a total knee replacement (TKR) is about 1 in 400, or 0.25 percent

    Should we not be reporting on them instead of the lower risk of autologous adult cell therapy.

    I find it interesting that the scientific title is “unregulated clinics” but some how it has morphed to “unproven clinics” in the media these are two completely different statements.

    The evidence is clear in the literature for treatment of knee and hip disease with autologous adult cells with there now 6 level II studies and 4 level III studies (and 40 publications).

    Even the most ardent critic must say there is a “possible” case for efficacy using these cells.

    1. @Alb,
      If I understand the data correctly, 0.25% of patients (mostly elderly) who get TKR die, but this doesn’t mean that this are procedure-related deaths, right? In other words, the data we need is surgery-related deaths. Is the mortality rate for untreated controls around 0.25% as well because they are elderly patients with knee problems? Or is it much lower than those who had TKR?
      Admittedly TKR is a serious procedure that does have at times serious complications. With properly controlled data for stem cells used for orthopedic procedures I hope that some day we’ll be able to make a meaningful comparison for both safety and efficacy.

      1. @admin. The 30-day threshold is the modern equivalent of “dying on the table”. Ask any surgeon. These days, almost no one literally dies on the table. If they are that bad off, most surgeons will simply not operate because the risk of death outweighs any potential benefit. And if we apply the literal definition to autologous stem cell injections, I’m pretty sure not a single orthopedic patient has ever died during an injection into his or her knee. There is certainly no evidence of it and even a “dubious” clinic couldn’t get away with killing a patient and not reporting it. In any case, the 30-day threshold is certainly a valid measure when discussing procedure related deaths. That’s why it’s used.

  2. 1. Phase III identifies risks of adverse effects in a much broader context (cohorts) of patients than Phase II.
    2. If Phase III is removed those risks will no longer be identified or dealt with.
    3. A drug approved after Phase II carries those unidentified adverse effects to the market, exposing patients, especially those not included in the Phase II cohorts, to increased risks.

    I asked the senators and their advisory teams about this in many letters and mails, but have received no answer. The only comment I heard was that, “a database will be created to follow up on any unexpected findings.”

    Some consolation for pregnant women and children, who were not included in Phase II trials.

    Advocates claiming it is worth it if only some patients gain benefits should be aware that the cost of that benefit may be the increased suffering of others.

    1. “Advocates claiming it is worth it if only some patients gain benefits should be aware that the cost of that benefit may be the increased suffering of others.”

      What is an acceptable amount of negative side effects for you? As I said in a previous post. Death is a very real side effect in a total knee replacement. TKR are getting performed everyday yet I haven’t seen an outcry from you about people dying.. Im sure you also know that the FDA has a “fast track” for medical devices to be approved without any of the regulation as long as its “similar” to something that has already been approved. I don’t see an outcry about that either. The truth is you have patients who have had tremendous results with stem cells and a VERY FEW case studies that point the other way.. Most of which involve the use of embryonic stem cells offshore. I just don’t see what you are basing your argument on?? Your intuition? Don’t you think that there would have been huge amounts of negative responses in just Dr. Centeno’s clinic alone?? He reports that Regenexx has treated 30k plus patients.. In all that data, it would seem like there would be bad rxn after bad rxn if you were right. Thats just Chris Centeno.. I imagine that there are probably 100k plus procedures so far if not way more.. The data would be there I would guess.. But again these are probably better questions for @admin to answer

      Im not saying that I am for the REGROW act in its current form but it seems like there needs to be some fast tracking of approvals. @admin already stated that from the data that he had seen, it didn’t look like bone marrow derived stem cells used to treat joints was all that dangerous. Im not saying that we should be fast tracking stem cell approval for ALS or Parkinsons, but I don’t think that we should be stifling innovation from these doctors because a 500M- 1Bil dollar trial wasn’t done to bring it to market with results that were consistent with what groups are already finding without forking out the money.. The only people that win would be big pharma in that situation.. Patients don’t win.. They lose

      1. @Dan, “What is an acceptable amount of negative side effects for you?”
        If I were a patient receiving a therapy for which there is no clinical data for my patient type (pregnant women, pediatric, over 70, etc), then any benefit I get or risk I am exposed to is unknown. That is unacceptable. If my patient type is one of those in the Phase II tests, then data is available and I can determine a risk / benefit ratio. But that’s a very narrow set of patients.

        It’s not about a level of side effects acceptable to a general patient population, it’s about the increased benefit over the standard of care versus risk for each patient. That’s how choice of therapy is weighed up by a physician.

        “The truth is you have patients who have had tremendous results with stem cells.” I read this often in comments but nobody has shown me the published data for any successful controlled, randomized clinical trials.

        Centeno’s work indicates there may be efficacy in some situations in some patients, but the studies are not statistically powered or controlled and cannot give definitive risk/benefit readouts. For this “very large controlled trials where side effects can be compared to placebo are needed in addition” (Centeno, April 11, 2016, The Niche).

  3. Paul,
    I think researchers in your position would welcome the opportunity to reverse engineer the proposed accelerated pathway for cellular therapies already showing safety and promise world wide… If even half of patients receiving these treatments during the trial approval period gained benefits, it would be life changing for so many. I see the danger of Wild West chaos, but with your knowledge and resources, you could Copt the data provided by the treatments, and produce the CURE. In what way is that disadvantageous to anybody?

    1. Hi Jennifer,
      Thanks for the comment. I’d be really interested in hearing what your specific concerns are. I’ve got some new ones of my own.

  4. Physicians who actually treat patients with stem cells in clinical trials are the ones in a position to judge weather REGROW is a reasonable approach.

    From the Statn article

    Dr. Charles Murry, the interim director of the University of Washington Institute for Stem Cell and Regenerative Medicine, said the institute supported the measure because it could lead to treatments “for today’s patients.” He noted that Phase 3 trials would be conducted during the conditional approval period, so any treatment could be pulled from the market if it’s shown not to work.

    “An attempt to accelerate therapies can increase the likelihood of complications from these therapies,” Murry said. “On the other hand, working slowly, while less risky, results in more people suffering from potentially treatable conditions. The goal will be to strike the right balance.”

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