Think Tank

FDA Wants To Tighten The Screws On Stem Cell Clinics

The U.S. Food and Drug Administration is proposing to get tougher on clinics that offer unapproved stem cell therapies, and is hosting a critical public hearing on the issue Monday and Tuesday in Bethesda, Md.

A study published recently in the journal Cell Stem Cell found that 570 clinics across the country are offering pricey stem cell treatments for both cosmetic and medical purposes. The clinics claim to treat a wide variety of problems, from spinal cord injuries to autism.

Authors Leigh Turner of the University of Minnesota and Paul Knoepfler of the University of California, Davis identified 113 such clinics in California alone, the most of any state.

“There are clear grounds for concern that some of the companies we found are not compliant with federal regulations,” they wrote.

Many of these clinics say they use stem cells derived from patients’ own fat. They argue their treatments are exempt from the agency’s rigorous approval process because the fat cells are “minimally manipulated” and shouldn’t be classified as drugs subject to FDA regulation.

But late last year, the FDA sent a warning letter to a doctor saying that the processing of fat cells in his clinics, including one in California, “would not meet the definition of minimal manipulation” and his product was therefore considered a drug that required agency approval.

Nearly 100 stem cell clinics, doctors, researchers and organizations plan to testify at the hearing about these issues.

California Healthline interviewed three participants, with divergent views, all California-based experts whose professional lives revolve around stem cell science.

Jeanne Loring is a professor and stem cell researcher at The Scripps Research Institute in La Jolla. She supports tighter FDA controls.

Elliot Lander is co-medical director and co-founder of Cell Surgical Network, a Rancho Mirage, California-based group comprising about 100 stem cell clinics, most of them in the U.S. He is wary of the FDA’s plan for more regulation.

C. Randal Mills is CEO of the California Institute for Regenerative Medicine, the state’s stem cell agency, based in Oakland. He calls for a middle ground between the FDA’s strict approval process and total exemption from that process.

The transcript below has been edited for length and clarity.

More Regulation Needed To Protect Patients

The “pro” argument supporting FDA regulation is that we don’t want to be hurting people. And we don’t want people to get taken for their money with therapies that don’t work.

We want to be able to tell people which therapies are likely to work and what the dangers and probabilities are.

The only real scientific argument against regulation is that the FDA needs to come up with a faster method for approving evidence-based therapies.

We met with the FDA and we plan to begin clinical trials with stem cells in 2018. We can take anybody’s cells and make them into the same cell type as embryonic stem cells, with the difference that they actually match the person they come from.

Our clinical trial will transplant those cells into a specific part of the brain that’s involved in movement disorders in people with Parkinson’s Disease.

We have agreed they are a drug and we expect the FDA to regulate us.

The FDA is arguing that the fat cells that these stem cell clinics are using should be classified as drugs. I agree with them.

There’s no scientific evidence that the fat cells these clinics are using are going to do the patient any good. And there’s no evidence that shows they are safe.

I’m a stem cell scientist. I need scientific evidence before I will believe anything. Regulation will help determine the efficacy and safety of those fat cells.

There are several lawsuits or potential lawsuits brewing over these stem cell treatments. People were promised they would get improvements and didn’t. And there are cases where people were actually harmed by stem cells.

Some people truly believe they have been helped by the stem cell injections. I’m not going to argue with them. It’s a very personal and emotional response. It’s not something that can be scientifically validated. People are really desperate, especially really sick people and their families.

That means patients are at risk. That’s what bothers me a lot. There’s nothing we can do to talk somebody out of going to a clinic if they feel that’s the only option they have. We just want to make sure nobody gets hurt. We also don’t want people to go broke. These treatments are not covered by insurance and they cost tens of thousands of dollars.

Over-Regulation By FDA Could Stifle Innovation

Right now, the FDA appears to be under a lot of political pressure to regulate stem cell clinics. But the stem cell clinics we work with are not using external sources of stem cells from other people. All we’re doing is surgical procedures that help people use their own stem cells from their own fat.

The FDA has never been involved in the regulation of surgery. The regulation of surgery and medical practice is governed by state medical boards and a number of other entities including hospital peer review committees, plaintiff’s attorneys and malpractice carriers.

The FDA’s intention was that if cells undergo significant changes to their biologic properties, they are considered a manufactured medication. But if we’re just taking fresh stem cells from one part of the body and moving them under sterile conditions to another part of the body, then the cells are not changed in any meaningful way.

However the FDA is proposing that any modification of fat, including the act of liposuction itself, would count as more than minimal manipulation. That would put hundreds of thousands of daily surgeries under the jurisdiction of the FDA. That can cause unintended consequences that can stymie innovation.

If it puts the practice of surgery under the direct jurisdiction of the FDA, then it could interfere with our surgical medical practices, which up until now has allowed us to use any FDA-approved drugs and devices in any way we see fit to help our patients, as long as we believe it is safe.

We’re proposing the FDA institute less sweeping regulation and maybe a model like our organization has used: Clinics who want to work with the cells should do it under investigative protocols and avoid irresponsible advertising and collect data.

If we happen to find millions of healing cells just under our skin, we want to use those to help us prevent and treat disease. It should not be criminalized. My concern is over wide-sweeping regulations. In an effort to rein in the fringe doctors, it may hurt the entire industry.

It’s safe. It works. It’s here to stay. Patients are going to demand it. Patients are entitled to it. We can’t pretend it’s something that is illegal.

To the extent there’s no risk of the transmission of disease, these procedures performed by clinics really should be left alone.

We have a revolutionary, disruptive technology, but it can get squelched if we’re not vigilant.

FDA Must Find A Middle Ground For Sake Of Patients

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.