For two days, the FDA held a hearing in Bethesda on proposed regulations to tighten oversight of stem cell treatments. The agency invited public testimony on rules that would affect many clinics offering unproven therapies.
The testimony was impassioned. Supporters argued that clinics provide a valuable service and that patients should decide how their own cells are used. Opponents said the clinics act like snake oil salespeople, offering unproven treatments.
Randy Mills, CIRM’s President and CEO, was among the speakers. He has been outspoken about the need for the FDA to change how 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
We aren’t happy—and many others aren’t—with the rapid spread of stem cell clinics. Some may do good work, but others make outlandish claims with no data.
Two factors are driving this problem. First, the need is real. These patients are suffering, have no alternatives, and are desperate for help. It’s unrealistic to tell someone in that situation to wait decades for science to catch up.
Second, the regulatory system offers only two paths. One is the FDA biologics license application—the most intense regulatory requirement in the world, taking 10–20 years and costing over $1 billion. The other is an exemption requiring no pre‑market approval, allowing clinics to open in days with no supporting data.
The burden on one path is massive; on the other, almost nonexistent. So it’s no surprise many clinics operate under the assumption they qualify for the exemption.
Now the FDA is saying, “We’re not happy with this.” They plan to define key terms more narrowly and make it harder to legally operate under the lighter regulatory pathway.
Their strategy
That’s what this meeting is about.
Their strategy has two problems. It doesn’t address patients or the need side of the equation. It’s also unlikely to work because the FDA lacks the resources to enforce these regulations at the clinic level.
They would essentially have to regulate physicians’ practice, which is beyond their capacity. Even if enforcement were possible, it would just push patients elsewhere.
We are advocating for a middle pathway that brings unregulated therapies into a framework clinics can follow.
I would rather see these clinics regulated and collecting data than operating under the FDA’s radar. I want formal pre‑market review. I want safety and efficacy data.
I will push the FDA to see that simply plugging this hole won’t solve the problem. Believing stricter rules will convince desperate patients to give up hope is naive.
There is little evidence these procedures are unusually risky. They do have risks, but so does everything in medicine. If you have no alternatives, you may be willing to take that chance.
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.
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.
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.
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.
Here is the work Patients For Stem Cells did on the FDA stem cells hearing, published in MD Monthly.
http://www.mdmonthly.com/stem-cell-therapy-md-monthly/