There was a burst of feeling last night in the stem cell community as word began circulating that a federal court had ruled in favor of the FDA defining manipulated stem cell products as drugs.
However, not everyone was pleased with the court ruling.
The clinics most directly affected by the ruling were understandably upset, but interestingly some long-time stem cell advocates also were worried.
They said there are potentially serious downsides to the federal court ruling.
What might be the negative repercussions?
The argument I’ve heard most often is that the direct consequence of this ruling will be that it will, rather than being helpful, actually be harmful because it will drive desperate patients to engage in stem cell tourism by going to other countries for treatment at clinics where patients are at even greater risk.
Is this argument legit?
It is difficult to say if more patients will leave the U.S. as a result of this ruling.
But regardless at its heart this stem cell tourism-based line of reasoning is a false argument because the U.S. must make appropriate laws regardless of the laws of other countries. For example, the U.S. has tax laws that are essential to provide revenue to fund our country. We know the U.S. tax laws lead some folks to take their money to Switzerland or the Cayman Islands to try to dodge U.S. taxes, but nonetheless the U.S. should not give up its tax laws. Similarly the U.S. must require to the best of its ability that medical treatments are safe even if the medical system in the U.S. ends up being far more restrictive than that in other countries. Perhaps this will lead some folks to leave the U.S. seeking more relaxed medical regulatory environments, but at the same time it keeps millions of Americans safer. The tradeoff is worth it.
A second argument is that this court ruling will impede medical innovation related to stem cell technology. I’m not sure I buy this argument either. We need a balance of innovation and safety. In my opinion from my years of experience with stem cells and decades growing cells in culture, once you take cells out of the body and grow them in a dish, they are fundamentally no longer the same any more. Every day cells are grown in culture the less “normal” they become and the more like cancer cells they become. I believe it is appropriate to call stem cells grown in culture “a drug”.
I know a few smart people disagree with me, but I believe they are in the minority.
As a patient, I can say that more patients will go offshore or to clinics that operate under the radar. There are already plenty of those in case anyone was not aware. The FDA was not even able to prevent a cantaloupe outbreak last year that killed quite a few people, so how are they going to find all of the clinics and doctors administering stem cell treatments and shut them down? This ruling does not protect patients. Patients feel protected when they go to their own doctor because states license doctors and doctors carry malpractice insurance. Going offshore does not afford patients that protection. Patients do not appreciate the “protection” that is the excuse that many give in support of this type of regulatory action. Many claim to worry that patients are throwing their money away on untested treatments. Why is that anyone’s concern other than the patient himself? Does anyone care if a patient throws his money away in Las Vegas or on a loser stock or a car or a bad investment of any kind? NO! When a patient has exhausted all other means of conventional medicine, why deny them the use of their own stem cells if that is what they want? As for safety, I feel comfortable with the knowledge that a physician treating me is licensed and that I have a right to sue for malpractice. I don’t need anyone else’s “protection”. Let’s talk about who really is protected by this ruling.
Barbara, these arguments are very illogical.
Just because some people gamble away money at a casino or lose money through bad shopping decisions has no bearing on the American medical system and certainly is a poor argument for the supposed appropriateness of patients potentially gambling away their lives because of a lack of regulation.
As to the idea that state licensing of physicians and malpractice insurance are adequate, somehow global protection for patients getting experimental treatments, that is naive at best. How will that help you if you’re dead? However, it is likely true that physicians giving unlicensed stem cell treatments will be frequently sued.
Finally, please clue us in as to who will be protected, if it is not patients, by the FDA being able to regulate stem cells as drugs? I really don’t see the answer?
Quote “I know a few smart people disagree with me, but I believe they are in the minority.”
Ouch Paul!
I think there are a few comments to be made here,
1. I think many people who read your blog may not have the clinical or professional education to make an informed comment about minimal manipulation, that be the case, I agree with you.
2. Maybe people are commenting more about the intrusiveness of our Government as a whole and in this discussion the FDA is the scapegoat.
It was great that Dr. Centeno weighed in on the case.
I guess it will be interesting to see if the FDA challenges CellTex to a duel, or if in fact everyone waits to see what the November election will bring.
This reader believes Washington does not want to “Mess with Texas” this close to the election.
Thanks for the comment, John. There certainly are many key questions and points.
I’m going to do a post soon in detail about why I believe these cells require more regulation.
I am also curious to see what the FDA does next with CellTex. No one can be sure, but insiders believe that more steps from the FDA are almost certainly already in the works. Some people think a warning letter is inevitable. Others think that is not necessarily the case, but that the FDA will soon conduct a 2nd visit to CellTex. Yet others have other ideas.
Paul,
Interesting ruling. We had a good 20 issues to be answered, many never got addressed, so we go up to the next court to get these items answered. We knew this wasn’t going to be decided at the circuit level, as if we won this first round they would appeal to the DC circuit. As to whether this will impact stem cell use in the U.S., you have a number of things that will still likely happen:
-Same day adipose SVF use. FDA has declared this a drug every way to Sunday, yet outside of closing down Intellicell, it seems like every month more clinics are popping up. I don’t agree with most of what’s going on here, but realize there are 1 million US physicians. If even 10% of them begin doing this, FDA would need to be at least100 times bigger to deal with this flood.
-Bone marrow concentrates-there are many of these machines 510K’ed-so I would expect this category to expand. These are being used without and IND or IDE under 21 CFR 1271.15.
-361 transplant tissues-We get approached every month by yet another vendor claiming that there are stem cells in some fetal tissue they are selling in a vial. Lee brought up that these are flying below the radar for now and FDA has cracked down on at least 2 companies. I’m dubious that much of these have live tissue, so I’ve asked our research guy to test some of them to see if they do contain lives MSCs. I would expect FDA to crack down on more of these companies.
On innovation-scientists need to remember that much of medical innovation doesn’t come through university labs or the FDA pathway, but through doctors who see clinical problems on the ground that need to be solved and solve them. They follow an inverse publication pathway compared to Pharma drugs-namely case reports, case series, comparison studies, and finally RCT’s. Reducing the access to cellular therapies will take out a part of this innovation pathway-there’s just no way to avoid it. On a recent Linkedin poll in an Orthobiologics group, 100% of the physicians responded that the biggest obstacle to innovation in this field was FDA (not my poll or group).
On the appeal-we knew this was a four quarter ball game. We won the first quarter with the Judge’s ruling, the second quarter they out scored us, but there are at least two more quarters to play. I’m not sure if you saw former FDA comissioner Andy Von Eschenbach’s comments in the WSJ a few montsh back on the RSI case: http://online.wsj.com/article/SB10001424052702303815404577331673917964962.html . He doesn’t think that the FDA is heading a good direction here.
We do need regulation, the big question is what type. Balancing regulatory error is critical-too much regulation and patients are harmed by a lack of innovation, too little and they are harmed by dangerous drugs. This is the critical question we hope this case will eventually answer.
Thanks for the comment, Chris. I wonder where will the field be in 5 years.
Paul, if we could just get everyone to pull this research wagon in the same direction, maybe we could be a farther piece down the road in five years than we could in ten years.
If you could grow stem cells in a perfect bioreactor that perfectly replicated the physiologic conditions of the human body, would that be minimal manipulation?
I think it is a very good thing for the FDA to regulate how cells are expanded even if they are autologous. I was involved in a case at a university hospital, where patients’ cells were being expanded for immunotherapy of cancer. The culture work was in a research laboratory, not a GMP facility and the QC was research grade and there was no proper QA. Several patients received microbial contamination from the cellular product that could have been prevented if such activity were properly regulated. This was an IRB approved clinical trial and both the MD and PhD involved in the trial were ethical people, so this was not done maliciously, or to make a quick buck, but because proper procedures to prevent mistakes were not in place. This is what the FDA is putting in place and what is necessary for patient protection, even with their own cells.