CIRM’s Randy Mills: New FDA rules for stem cells won’t fix the problem

For the last two days the Food and Drug Administration (FDA) has been holding a hearing in Bethesda, Maryland on new regulations that would tighten control over stem cell treatments. The FDA invited public testimony during the hearing on the regulations that would impact many of the clinics that currently offer unproven therapies

The testimony has been impassioned to say the least. Supporters of the clinics say they offer a valuable service and that patients should be allowed to decide for themselves how they want their own cells to be used. Opponents say the clinics are little more than snake oil sales people, offering bogus, unproven treatments.

One of those presenting was Randy Mills, CIRM’s President and CEO. Randy has been very vocal in the past about the need for the FDA to change the way it regulates stem cell therapies.

In California Healthline Randy explained why he thinks the rules the FDA is proposing will not fix the problem, and may even make it worse:

FDA Must Find A Middle Ground For Sake Of Patients


Randy Mills

We aren’t happy, as a lot of people aren’t happy, with the proliferation of these stem cell clinics — some of which are probably doing good work. But some are clearly making rather outlandish claims for which there’s no real data. 

There are a couple of conditions coming together to create this storm.

One is that the need is very real. These patients are really struggling. They don’t have alternatives. They’re desperate and they need help. It’s not in the realm of possibility to talk to somebody who is suffering as badly as these patients are and to say, ‘You have to wait a few more decades for the science to catch up.’

On the other hand, we have a regulatory paradigm that only provides two pathways to put a cell therapy onto the market. One pathway is the most intense regulatory requirement anywhere in the world for any product — the biologics license application through the FDA, which takes 10 to 20 years and costs over $1 billion.

The other is through the exemptions the FDA has made, which require absolutely no pre-market approval whatsoever. You can be on the market in days, with no data.

The regulatory burden associated with one is massive and the other is almost nonexistent.

So it’s not at all surprising that we’re seeing a proliferation of these stem cell clinics popping up that are operating under the assumption that they fall under the exemption.

What the FDA is doing now is saying, ‘We’re not happy with this. We’re going to define some terms more narrowly than in the past … and make it more difficult to legally be on the market under the less burdensome regulatory pathway.’

That’s what this meeting is about.

The problem with their strategy is twofold. It doesn’t address the patients, or the need side of the equation. And I don’t think it has a chance of actually working because the FDA will acknowledge that they do not have the resources to enforce these types of regulations at the clinic level.

They would have to be essentially regulating the practice of physicians, which is well beyond their capabilities. Even if they were able to enforce it, it would just drive these patients somewhere else.

We’re advocating for the creation of some middle pathway that would bring essentially unregulated therapies into the regulatory fold, but in a manner which could be complied with.

I would rather know these clinics are being regulated and collecting data than have them operating under the radar screen of the FDA. I would like there to be a formal pre-market review of these therapies before they’re put on the market. I would like there to be safety and efficacy data.

I’m going to try hard to get the FDA to see that just plugging this hole won’t make the problem go away.

Thinking that they’re going to strengthen the regulation and that patients are going to be satisfied that there’s absolutely no chance for help is naive.

There isn’t a lot of evidence to suggest these types of procedures are overly risky. It’s not that they don’t have risk, but everything in medicine does. If you’re a patient who has absolutely no alternative, you’re probably willing to take the chance.

6 thoughts on “CIRM’s Randy Mills: New FDA rules for stem cells won’t fix the problem

  1. Why don’t the ethical researchers listed here pick one and only one medical condition and as a large group provide a stem cell solution for that condition, producing a therapy useable by appropriate medical providers? It seems to me that this, just like cancer research is a bottomless pit of time and money wasted effort. No wonder it will take centuries and cost billions of dollars.

  2. Like you say, people should be allowed to make up their own minds about their own stem cells. Some of the ‘so called’ cure drugs on the market, cleared by the FDA can kill you, or cause cancer or more, how can they want to regulate our own decision to have our own stem cells re introduced to us as a possible cure for disease that there is no cure.

  3. I think that we, the research academic centers and stem cell industry, need to work more deeply in ways to communicate to lay public about the state of the art on treatment and expectations (including the risks of potential adverse effects), of stem cells. The main concern at this point is the patient well being,. But we need to keep in mind, that those who seek tratments come for very vulnerable population. There is no question that we need to protect the patient’s autonomy, allowing the benefits of this new therapeutics reach them, with a fair level of certainity has been identified. However, the challenge for us to hel define the level of certainity, and this still need to define the time to do so. Perhaps we need to define a specific plan for stem cell therapies and timelines to evaluate progress, as once was done to reach the Moon or now, to find a cure for Cancer.

  4. What is most troubling to me as a California Tax payer is that CIRM has taken the 3 billion dollars of our money and given those dollars to the academics to study embryonic stem cells and have not made any of those dollars available to California physicians trying to do clinical studies using autologous stem cells for treatment of California citizens. The academics who have been receiving these tax dollars are quick to criticize physicians for using “unproven stem cells”. Comments like “Stem cells need at least 10 more years of study before they are ready for the clinic” refer to the embryonic stem cells they are doing research on and not the human stem cells that are harvested from the patient and just returned to the same person. CIRM’s President and CEO Randy Mills should be looking into how CIRM can help practicing physicians do better clinical research so that the public and the academics can finally get the real truth about how safe and effective autologous stem cells are for a variety of health problems. Embryonic and IPS cells do have a long way to go but autologous stem cells are here now so lets use them in the most effective and safe way that we can. So far CIRM has failed to help the people of California because of this money grab by the Academics. Shame on CIRM and the Academics!

    • Dear David, thanks for the comment. We are always happy to work with anyone, academic, company or physician, who has a therapy that they want to get into a clinical trial. We don’t just fund academic researchers working with embryonic stem cells, we fund a wide variety of researchers using a wide variety of cell types. But I understand your frustration at the current regulatory system and how slow it is in moving the most promising therapies out of the lab and into clinical trials in patients. That’s why we started our Stem Cell Champions campaign, to get people like you to join us in trying to create a new regulatory approval process, one that is more responsive to the needs of the patients.

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